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UNIVERSIDADE ESTADUAL DE CAMPINAS FACULDADE DE ODONTOLOGIA DE PIRACICABA GLEYSON KLEBER DO AMARAL SILVA ANÁLISE DA EXPRESSÃO DE PROTEÍNAS RELACIONADAS AO REPARO DO DNA E PROLIFERAÇÃO CELULAR EM AMELOBLASTOMA E CARCINOMA AMELOBLÁSTICO ANALYSIS OF THE EXPRESSION OF PROTEINS RELATED TO DNA REPAIR AND CELLULAR PROLIFERATION IN AMELOBLASTOMA AND AMELOBLASTIC CARCINOMA PIRACICABA 2017

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Page 1: ANALYSIS OF THE EXPRESSION OF PROTEINS RELATED … · Fregnani, Dr. André Caroli, Dr. Fernando Soares, Dr. Hélder Pontes, Dra. Vivian Wagner, À Fabiana e ao Adriano por toda a

UNIVERSIDADE ESTADUAL DE CAMPINAS

FACULDADE DE ODONTOLOGIA DE PIRACICABA

GLEYSON KLEBER DO AMARAL SILVA

ANÁLISE DA EXPRESSÃO DE PROTEÍNAS RELACIONADAS AO REPARO DO DNA

E PROLIFERAÇÃO CELULAR EM AMELOBLASTOMA E CARCINOMA

AMELOBLÁSTICO

ANALYSIS OF THE EXPRESSION OF PROTEINS RELATED TO DNA

REPAIR AND CELLULAR PROLIFERATION IN AMELOBLASTOMA

AND AMELOBLASTIC CARCINOMA

PIRACICABA

2017

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GLEYSON KLEBER DO AMARAL SILVA

ANÁLISE DA EXPRESSÃO DE PROTEÍNAS RELACIONADAS AO REPARO DO DNA

E PROLIFERAÇÃO CELULAR EM AMELOBLASTOMA E CARCINOMA

AMELOBLÁSTICO

ANALYSIS OF THE EXPRESSION OF PROTEINS RELATED TO DNA REPAIR AND

CELLULAR PROLIFERATION IN AMELOBLASTOMA AND AMELOBLASTIC

CARCINOMA

Dissertação apresentada à Faculdade de

Odontologia de Piracicaba da Universidade

Estadual de Campinas como parte dos

requisitos exigidos para a obtenção do título

de Mestre em Estomatopatologia, na Área de

Patologia.

Orientador: Prof. Dr. Pablo Agustin Vargas

Coorientador: Prof. Dr. Felipe Paiva Fonseca

ESTE EXEMPLAR CORRESPONDE À

VERSÃO FINAL DA DISSERTAÇÃO

DEFENDIDA PELO ALUNO GLEYSON

KLEBER DO AMARAL SILVA, E

ORIENTADA PELO PROF. DR. PABLO

AGUSTIN VARGAS.

PIRACICABA

2017

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DEDICATÓRIA

Dedico este trabalho aos meus pais, Ednamay Amaral Silva e Walter Benedicto da

Silva, que sempre me estimularam e nunca mediram esforços para minha formação

educacional e moral.

À minha irmã Greyce Kellen do Amaral Silva, por todo apoio e companheirismo

durante nossas caminhadas de idas e vindas da escola e pelo apoio e conselhos ao logo da

minha vida. Estas e outras lembranças estarão presentes eternamente em minha mente.

À minha tia Maura Cassia Ferreira de Macedo, por estar presente em grande parte

da minha vida me permitindo experimentar de atividades e momentos felizes.

À minha amada Danielle Ferreira Sobral de Souza, pelo companheirismo, apoio e

estímulo na realização dos meus sonhos.

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AGRADECIMENTOS

À Faculdade de Odontologia de Piracicaba da Universidade Estadual de Campinas

(Unicamp), na pessoa de seu diretor, Prof. Dr. Guilherme Elias Pessanha.

À Profa. Dra. Cínthia Pereira Machado Tabchoury, coordenadora do Programa de

Pós-Graduação da Faculdade de Odontologia de Piracicaba da Universidade Estadual de

Campinas (Unicamp).

Ao Prof. Dr. Márcio Ajudarte Lopes, coordenador do Programa de Pós-Graduação

em Estomatopatologia, da Faculdade de Odontologia de Piracicaba da Universidade Estadual

de Campinas (Unicamp).

Ao meu orientador, Prof. Dr. Pablo Agustin Vargas, agradeço por todas as

oportunidades de aprendizagem, crescimento e por ser um exemplo de profissional sempre

tratando com os assuntos com seriedade, mas também permitindo momentos de descontração

em ocasiões oportunas. Obrigado por suas orientações e confiança no meu trabalho.

Ao meu coorientador, Prof. Dr. Felipe Paiva Fonseca, por todas as trocas de

experiências, ensinamentos e acompanhamento durante esta fase tão importante em minha

vida. Sou grato por sua grande contribuição no meu amadurecimento profissional.

Agradeço ao Prof. Dr. Oslei Almeida pela oportunidade e seus ensinamentos

durante nossas práticas na Rotina e por sempre estimular os seus alunos a estudar e buscar o

conhecimento, uma demonstração clássica de andragogia. Ao Professor Dr. Alan Roger, pelos

ensinamentos sempre que possíveis, especialmente quando nos encontramos no Orocentro.

Aos Professores Drs. Jacks Jorge, Ricardo Colleta e Edgar Graner, agradeço igualmente pelos

ensinamentos transmitidos durante a pós-graduação. À todos os demais colaboradores na

realização deste projeto e publicação dos artigos: Profa. Manoela Martins, Prof. Eduardo

Fregnani, Dr. André Caroli, Dr. Fernando Soares, Dr. Hélder Pontes, Dra. Vivian Wagner,

À Fabiana e ao Adriano por toda a ajuda e momentos de aprendizado e

descontração nos laboratórios.

Os meus mais sinceros agradecimentos a todos os colegas e amigos do curso pela

companhia, momentos de trocas de experiências pessoais e profissionais, especialmente à

Debora Lima, Leonardo Reis, Debora Campanela, Celeste Sánchez e Ciro Dantas pela

companhia e trocas de experiências e conhecimentos teóricos e práticos, muitas vezes

verdadeiras aulas, e por tornarem os dias em Piracicaba mais agradáveis.

À Fundação de Amparo à Pesquisa do Estado de São Paulo pela concessão de

bolsa de estudo (processo FAPESP: 2015/21520-8).

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AGRADECIMENTOS ESPECIAIS

Agradeço a Deus, nosso Pai Celestial, por todas as provas, expiações e desafios

como instrumentos para minha evolução moral e espiritual. Lhe agradeço pelo simples dom

da existência.

Aos meus pais, professores da vida e de profissão, agradeço por servirem de fonte

de inspiração e por todo o amor e carinho me ofertado. Sei que não foi fácil para mim chegar

até aqui, mas reconheço que vocês tiveram de abrir mão de muitas coisas e sonhos para que

eu pudesse conquistar os meus.

Agradeço a minha irmã, Greyce Amaral, e a minha tia, Maura Macêdo, por todo o

carinho o carinho, amor e contribuição para a minha formação moral. Muitas lembranças me

vêm à mente neste momento, como quando íamos e voltávamos da escola cantando músicas e

fazendo brincadeiras para nos distrair. São tantas coisas para agradecer que certamente as

palavras não conseguem descrever.

À Danielle Ferreira, pelo companheirismo, seu amor, compreensão e paciência

principalmente nestes últimos dias. Sabemos que nós temos uma grande e linda história e que

existem muitos amigos que fazem parte dela. Hoje quero agradecer especialmente por

acreditar em mim, no meu potencial, principalmente quando muitas vezes eu mesmo duvido.

Obrigado por me fazer acreditar nos meus sonhos e buscar lutar por ele.

Aos entes queridos que jamais saíram da lembrança, sinto na obrigação de

agradecer todo o amor e carinho por mim. A distância física ainda dói, mas nos momentos

mais difíceis sei que vocês intercederam por mim de alguma maneira. De todos, faço o

agradecimento especial para minha querida avó Socorro, a qual tenho a certeza que continua a

me abençoar. Obrigado minha amada avó.

Agradeço aos professores e amigos Danyel Elias da Cruz Perez e Flavia Maria da

Cruz Perez pelos ensinamentos ao longo da graduação e pela inspiração como exemplos de

profissionais, professores e orientadores. Ao professor Danyel faço um agradecimento

especial por toda a consideração e orientação enquanto fui seu aluno durante a graduação e no

programa de Iniciação Científica. Por esses e outros motivos sou eternamente grato.

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RESUMO

O ameloblastoma é um tumor odontogênico benigno, porém com comportamento local

agressivo devido seu potencial de infiltração e destruição óssea, afetando preferencialmente a

região posterior de mandíbula. A sua contraparte maligna, o carcinoma ameloblástico, pode

surgir de novo ou desenvolver-se de um ameloblastoma prévio, estando associado a um alto

índice de recidivas e metástases linfonodal e a distância. Embora sejam raros, ambos

representam os tumores odontogênico benigno e maligno mais frequentemente diagnosticados

nos principais serviços de Patologia Oral. Os mecanismos moleculares envolvidos com a

etiopatogenia destes tumores são pouco conhecidos, e apesar de alterações no sistema de

reparo de erros de pareamento de bases do DNA, conhecido como sistema mismatch (MMR)

favorecerem o desenvolvimento de diferentes neoplasias humanas, a importância destes no

desenvolvimento do ameloblastoma e do carcinoma ameloblástico ainda carece de estudos. O

MMR humano compreende o conjunto de proteínas divididas em dois grupos, hMutS e

hMutL. Sua principal função é de corrigir erros de pareamentos e de loops de inserção e

deleção das bases nitrogenadas no DNA. Uma variedade de neoplasias e condições

potencialmente malignas possuem alteração desse sistema. Os objetivos deste trabalho estão

divididos em capítulos e compreendem: (i) conceituar a função das proteínas do sistema

mismatch nas lesões neoplásicas humanas, com ênfase nas que acometem a região oral; (ii)

analisar quantitativamente a imunoexpressão das proteínas hMutS em germes dentais

humanos, ameloblastomas e carcinomas ameloblásticos, correlacionando seus resultados com

a expressão de BRAF-V600E e com a recorrência tumoral. Foi observado que expressão

imunohistoquímica das proteínas hMutS em 10 amostras de germes dentais humanos, 39

ameloblastomas e 2 carcinomas ameloblásticos apresentam um decréscimo sequencial nos

níveis de expressão nos grupos analisados, especialmente das proteínas hMSH2 e hMSH6 em

ameloblastomas (p=0.0059) em relação ao germe dental humano (p<0.0001). A expressão

imunohistoquímica das proteínas hMutS em 73 amostras de ameloblastomas contidas em um

bloco de 1.0 mm de tissue microarray permitiu associar seus resultados com a taxa de

recorrências e presença da mutação BRAF-V600E. Os casos de ameloblastomas com

sobrexpressão das proteínas hMSH2, hMSH3 apresentaram a presença de BRAF-V600E

(p<0.05) e foram associados com a recorrência da lesão (p=0.035). Estes dados sugerem que a

sobrexpressão de hMutS pode estar indiretamente relacionada mecanismos moleculares e

genéticos envolvidos na progressão e recorrência tumoral.

Palavras-chave: ameloblastoma; tumores odontogênicos; hMSH2; hMSH3; hMSH6; proteínas

mismatch.

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ABSTRACT

Ameloblastoma is a benign odontogenic tumor, but with aggressive local behavior due to its

potential for infiltration and bone destruction, preferentially affecting the posterior region of

the mandible. Its malignant counterpart, ameloblastic carcinoma, may arise de novo or

develop from a previous ameloblastoma, being associated with a high rate of relapses and

lymph node metastases and distance. Although rare, they both represent the benign and

malignant odontogenic tumors most frequently diagnosed in the main Oral Pathology

services. The molecular mechanisms involved in the etiopathogenesis of these tumors are

poorly understood, and although alterations in the DNA bases pairings repair system, known

as the mismatch system (MMR), favor the development of different human neoplasms, the

importance of these in the development of ameloblastoma and ameloblastic carcinoma still

lacks studies. Human MMR comprises the set of proteins divided into two groups, hMutS and

hMutL. Its main function is to correct errors of pairings and loops of insertion and deletion of

the bases in the DNA. Several neoplasms and potentially malignant conditions have alteration

of this system. The objectives of this work are divided into chapters and include: (i)

conceptualizing the function of the proteins of the mismatch system in human neoplastic

lesions, with emphasis on those that affect the oral region; (ii) quantitatively analyze the

immunoexpression of hMutS proteins in human dental germs, ameloblastomas and

ameloblastic carcinomas, correlating their results with the expression of BRAF-V600E and

with tumor recurrence. Immunohistochemical expression of hMutS proteins in 10 human

dental germ samples, 39 ameloblastomas and 2 ameloblastic carcinomas showed a sequential

decrease in expression levels in the analyzed groups, especially the hMSH2 and hMSH6

proteins in ameloblastomas (p = 0.0059) in relation to human dental germ (p <0.0001). The

immunohistochemical expression of hMutS proteins in 73 samples of ameloblastomas

contained in a 1.0 mm block of tissue microarray allowed to associate their results with the

rate of recurrences and the presence of the BRAF-V600E mutation. The cases of

ameloblastomas with overexpression of the hMSH2, hMSH3 proteins showed the presence of

BRAF-V600E (p <0.05) and were associated with lesion recurrence (p = 0.035). These data

suggest that the overexpression of hMutS may be indirectly related molecular and genetic

mechanisms involved in tumor progression and recurrence.

Keywords: ameloblastoma; odontogenic tumors; hMSH2; hMSH3; hMSH6; mismatch

proteins.

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SUMÁRIO

1 INTRODUÇÃO 11

1.1 Ameloblastoma 11

1.2 Carcinoma ameloblástico 11

1.3 Sistema de reparo mismatch 12

2 ARTIGOS 14

2.1 Artigo: Mismatch repair system proteins in oral benign and malignant lesions 14

2.2 Artigo: Prognostic significance of hMSH2, hMSH3 and hMSH6 expression in

ameloblastoma 27

3 DISCUSSÃO 50

4 CONCLUSÃO 52

REFERÊNCIAS 53

ANEXOS 56

Anexo 1: Certificado de Direitos Autorais da Journal of Oral Pathology and

Medicine 56

Anexo 2: Carta de aceite para publicação do Journal of Oral Surgery, Oral

Medicine, Oral Pathology, Oral Radiology 57

Anexo 3: Certificado do Comitê de Ética em Pesquisa FOP/Unicamp 58

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1 INTRODUÇÃO

1.1 Ameloblastoma

O ameloblastoma é um tumor odontogênico epitelial sem a formação de tecido

mineralizado que se origina de remanescentes de tecido odontogênico como a lâmina dentária,

órgão do esmalte, restos de Malassez e do revestimento epitelial de cistos odontogênicos,

especialmente os cistos dentígeros. (Dhanuthai et al., 2012; Masthan et al., 2015; McClary et

al., 2016). Primeiramente descrito por Cusack (1827), esta rara neoplasia acomete os ossos

gnáticos e representa cerca de 1% de todos os tumores orais, variando entre 9% – 11% dentre

os tumores odontogênicos (Masthan et al., 2015). Apresenta pico de ocorrência entre os 30-60

anos de idade, sem predileção pelo gênero e com incidência global de 0,5 casos/milhão/ano

(Fregnani et al., 2002; McClary et al., 2016). As manifestações clínicas apresentam

sintomatologia discreta, normalmente associada a um crescimento de volume indolor e lento,

porém localmente agressivo e infiltrativo, resultando em alta taxa de recorrência. Possui forte

predileção pela mandíbula em relação à maxila (13:1), preferencialmente a região posterior e

em terceiros molares não erupcionado (Dhanuthai et al., 2012). É radiograficamente

observado como imagens radiolúcidas uniloculares ou multiloculares, podendo apresentar

deslocamentos e reabsorções dentárias (Dhanuthai et al., 2012; McClary et al., 2016). Quando

associado às características microscópicas, pode ser classificado como ameloblastoma

unicístico, sólido ou multicistico (a maioria dos casos) ou periférico, apresentando seus

padrões histológicos variáveis (Dhanuthai et al., 2012; Masthan et al., 2015; McClary et al.,

2016).

A abordagem cirúrgica representa o tratamento padrão para o ameloblastoma,

porém os índices de recidiva da lesão estão fortemente relacionados com a modalidade

terapêutica empregada. No ameloblastoma unicístico, a taxa de recorrência corresponde a 4%

para o tratamento radical, como a ressecção em bloco, contra 17% do tratamento conservador,

como a curetagem da lesão, enquanto que no multicístico temos um índice de recorrências de

8% quando utilizado tratamento radical contra 38% quando tratamentos conservadores são

realizados (Antonoglou and Sándor, 2015).

1.2 Carcinoma ameloblástico

Os tumores odontogênicos malignos são neoplasias raras que correspondem a

menos de 6% de todos os tumores odontogênicos nas principais séries epidemiológicas

publicadas. Dentre estas lesões raras, o carcinoma ameloblástico corresponde ao tumor

odontogênico maligno mais comumente diagnosticado, com uma incidência estimada de

apenas 1,79 casos para cada 10 milhões de pessoas/ano. Esta neoplasia combina as

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características histológicas do ameloblastoma com diferentes graus de atipia celular. Pode

surgir de novo, sem uma neoplasia benigna prévia, ou desenvolver-se de um cisto ou tumor

odontogênico benigno pré-existente, como um ameloblastoma, recebendo a denominação de

carcinoma ameloblástico secundário (Hall et al., 2007; Pirklbauer et al., 2012).

Embora sua apresentação clínica seja variável, uma tumefação local de

crescimento rápido, associada a dor e parestesia, são as manifestações mais relatadas. Possui

ligeira predileção pelo sexo masculino, com idade média de 40 anos, tendo a região posterior

da mandíbula como o sítio mais afetado. Radiograficamente apresenta imagens radiolúcidas

mal definidas, com eventuais focos radiopacos sugestivos de calcificações distróficas,

podendo causar reabsorções e deslocamentos dentários (Benlyazid et al., 2007). A análise

histológica do carcinoma ameloblástico demonstra regiões de diferenciação ameloblástica

com graus variáveis de pleomorfismo e atipia celular, além de figuras de mitose atípicas,

necrose tecidual e eventuais focos de invasão perineural (Fitzpatrick et al., 2015; Lucca et al.,

2010).

Pouco se conhece sobre suas alterações genéticas ou moleculares, o que limita o

aprimoramento da abordagem terapêutica desta entidade. Portanto, os pacientes são tratados

por meio de abordagem cirúrgicas radicais, como a remoção em bloco do tumor ou a ampla

ressecção com margens de segurança, causando um elevado grau de morbidade terapêutica.

Além disso, altos índices de recorrência que variam de 28,3% a 90% são relatados na

literatura dependendo em especial da abordagem cirúrgica empregada. A frequência de

metástases a distância chega a 22% e os locais mais acometidos são o pulmão, cérebro e

múltiplos ossos. O prognóstico é considerado sombrio com taxas de sobrevida após cinco

anos próximas de 70%, diminuindo para cerca de 20% em pacientes com metástases

(Benlyazid et al., 2007; Hall et al., 2007).

1.3 Sistema de reparo mismatch

Os mecanismos genéticos envolvidos nessas manifestações neoplásicas ainda são

pouco compreendidos. Uma das linhas de estudo pouco explorada está relacionada com o

sistema de reparo de bases nitrogenadas mal pareadas do DNA, conhecido como sistema

mismatch (MMR). O mecanismo deste sistema é mediado pela associação de proteínas que

mantêm a fidelidade do material genético replicado, corrigindo erros no pareamento de bases

e impedindo a instabilidade de microssatélites. O sistema humano é homólogo ao encontrado

em na bactéria Escherichia coli (E. coli), sendo denominados homólogos MutS (hMutS) e

MutL (hMutL) (Hsieh and Yamane 2008). Em tumores odontogênicos, o estudo deste

mecanismo foi apenas abordado por Castrilli et al. (2001) de forma descritiva através de

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estudo imunohistoquímico em 25 casos de ameloblastomas. Neste estudo, os autores

identificaram a expressão positiva das proteínas hMSH2 e hMLH1 (componentes dos grupos

hMutS e hMutL, respectivamente), mas não realizaram estudos quantitativos ou comparativos

com germes dentais humanos, por exemplo.

Neste contexto, propusemos um estudo mais aprofundado da expressão

imunohistoquímica das proteínas relacionadas ao sistema mismatch em ameloblastomas e

carcinomas ameloblásticos, tendo como controle o epitélio ameloblástico normal de germes

dentais humanos, comparando seus resultados e correlacionando-os com as características

clínicas e potencial prognóstico.

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2 ARTIGOS

2.1 Artigo: Mismatch repair system proteins in oral benign and malignant lesions

Artigo publicado no Journal of Oral Pathology and Medicine, 2017 Apr;46(4):241-245. doi:

10.1111/jop.12484. Epub 2016 Aug 10. (Anexo 1)

Gleyson Kleber do Amaral Silva1

Manoela Domingues Martins2

Hélder Antônio Rebelo Pontes3

Eduardo Rodrigues Fregnani4

Márcio Ajudarte Lopes1

Felipe Paiva Fonseca1

Pablo Agustin Vargas1

1. Department of Oral Diagnosis - Piracicaba Dental School – University of Campinas

(Piracicaba/Brazil)

2. Department of Pathology - School of Dentistry - Federal University of Rio Grande do

Sul (Porto Alegre/Brazil).

3. Service of Buccal Pathology – João de Barros Barreto University Hospital – Federal

University of Pará (Belém/Brazil).

4. Department of Oral Medicine – Sírio-Libanês Hospital (São Paulo/Brazil).

Corresponding Author:

Prof. Dr. Pablo Agustin Vargas

Piracicaba Dental School – University of Campinas (UNICAMP)

Department of Oral Diagnosis – Pathology

Av. Limeira, 901 ZipCode: 13414-903 Piracicaba – São Paulo – Brazil

Tel.: +55 19 21065319 E-mail: [email protected]

Keywords: Mismatch repair system, MutS, MutL, MSH, MLH, oral cancer, leukoplakia.

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Abstract

Different environmental agents may cause DNA mutations by disrupting its double-

strand structure; however, even normal DNA polymerase function may synthesize

mismatch nucleotide bases, occasionally demonstrating failure in its proofreading

activity. To overcome this issue, mismatch repair (MMR) system, a group of proteins

specialized in finding mispairing bases and small loops of insertion or deletion,

works to avoid the occurrence of mutations that could ultimately lead to innumerous

human diseases. In the last decades, the role of MMR proteins in oral carcinogenesis

and in the development of other oral cavity neoplasms has grown, but their importance

in the pathogenesis and their prognostic potential for patients affected by oral

malignancies, especially oral squamous cell carcinoma (OSCC), remain unclear.

Therefore, in this manuscript we aimed to review and critically discuss the currently

available data on MMR proteins expression in oral potentially malignant lesions, in

OSCC, and in other oral neoplasms to better understand their relevance in these lesions.

Keywords: leukoplakia; mismatch repair system; MLH; MSH; MutL; MutS; oral cancer

Introduction

It is known that different endogenous and exogenous agents may cause DNA

mutations by disrupting its double-strand structure (1); however, DNA damage can also be

originated because of natural replication errors, including failures of DNA polymerase

activity (2) that may incorporate mismatched nucleotide bases, predisposing individuals to

innumerous human diseases (3).

Twelve different single-base incorporated errors can be found (4). Because these

genetic errors may pass unnoticed by the proofreading activity of DNA polymerase, the post-

replicated DNA strand is additionally checked by a mismatch repair (MMR) system that

consists of a group of proteins specialized in finding not only mispairing bases, but also small

loops of insertion or deletion, and ultimately providing their corrections (5).

Important new data were provided on the structure and function of MMR in the

human genome stability, but its importance on the development of different malignant and

potentially malignant human diseases has only recently been investigated. Therefore, in this

manuscript we aimed to review the current data available on MMR expression pattern and its

importance for the progression of oral squamous cell carcinoma (OSCC) and oral potentially

malignant lesions, as well as to provide an overview on their importance for the pathogenesis

of other oral cavity neoplasms.

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MMR system and your proteins

Mismatch repair system was initially investigated in Escherichia coli, being

classified in four different groups as MutS, MutL, MutH, and MutU (6), whereas following

studies in eukaryotic cells defined only two homologous groups, hMutS and hMutL (7),

which include the somatic human proteins hMSH2 (gen locus on 2p21), hMSH3 (5q14.1),

hMSH4 (1p31), hMSH5 (6p21.3), hMSH6 (2p16), hMLH1 (3p21), hPMS1 (2q31.1), hPMS2

(7q22.2), and hMLH3 (14q24.3) (8, 9).

Although bacterial MutS and MutL groups are formed by homodimers, in

eukaryotic cells these groups comprise heterodimers composed by two related, but distinct

protein subunits (10). hMutS contains the heterodimers hMSH2- hMSH6 (called hMutSα) and

hMSH2-hMSH3 (called hMutSβ), whereas hMutL contains the heterodimers hMLH1-hPMS2

(hMutLa), hMLH1-hPMS1 (hMutLb), and hMLH1-hMLH3 (hMutLγ) (5).

As a post-replication event, hMutS scans the new DNA strand looking for

mismatches and loops of insertion or deletion. However, hMutSα more efficiently recognizes

base–base mismatches and insertion/deletion mispairs in which one strand contains one or

two unpaired nucleotides, whereas hMutSβ preferentially identifies loops of insertion/deletion

of two to over 10 nucleotides (10). Following the recognition of a mispaired, hMutS

undergoes an ATP-induced conformational change that activates it and promotes its

interaction with hMutL proteins. Subsequently, Proliferating Cell Nuclear Antigen (PCNA)

activates hMutL to incise the daughter strand hundreds of base distant from the mismatch.

Once hMutL nicks the DNA, hMutS activates EXO1 (an exonuclease) to progressively excise

the DNA containing the mismatch or the insertion/deletion loop, while DNA polymerases d/e

initiate the new strand synthesis from the nicked point (11) (Fig. 1).

However, MMR genes can also be disrupted by mutations or hypermethylations,

predisposing cells to acquire an increased amount of DNA mismatches and potentially

developing a neoplastic phenotype. This is better exemplified by Lynch syndrome and its

increased incidence of hereditary non-polyposis colorectal cancer (HNPCC) (12).

In addition, studies demonstrating abnormal expression of MMR components in

breast (13), prostate (14), urothelial (15), and lung cancers (16) have also been described,

usually showing a lower expression of MMR proteins.

Similarly, alterations in the MMR complex have also been correlated with the

onset and progression of oral potentially malignant lesions and OSCC, with some new

evidences that these proteins could play a role as prognostic determinants (Table 1). All these

studies support additional investigations to better understand the biological implication of

MMR mutation and epigenetic modifications in the neoplastic context.

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MMR expression in oral and lip potentially malignant lesions

Leukoplakia is the most common potentially malignant lesion of the oral cavity,

and several studies looked forward to assessing the MMR protein expression pattern in this

lesion. Caldeira et al. (17) evaluated hMLH1 immunoexpression in different grades of

dysplasia, demonstrating a lower expression in more dysplastic lesions. These results were

confirmed by the same group that showed a lower expression of hMLH1 in more dysplastic

OL, also revealing an inverse correlation with p53 and AgNOR expression (18). These

authors suggested that hMLH1 alterations would represent an early molecular event in oral

carcinogenesis, what was supported by Jessri et al. (19) who confirmed a reduced expression

of hMLH1, hPMS2, and hMSH2 in OL and in OSCC when compared to normal oral mucosa,

also attributing a diagnostic role for these proteins. Sengupta et al. (20) showed that the

promoter regions of hMLH1 and hMSH2 were hypermethylated in 63% of their OL samples,

and this epigenetic feature correlated with the presence of microsatellite instability in

dysplastic cells and with tobacco use by the patients. Fernandes et al. (21) also described

altered hMLH1 expression in oral epithelium associated with tobacco use, with a higher

expression in smokers than non-smokers.

Similar results were described for actinic cheilitis (AC), the most common

potentially malignant lesion of the lip. Sarmento et al. (22) immunohistochemically analyzed

forty cases of lower lip AC demonstrating that hMLH1 and hMSH2 were less expressed in

more dysplastic lesions. These results were also obtained by Oliveira et al. (23) who showed

higher expression of hMLH1 in AC with mild dysplasia, whereas Souza et al. (24) found a

lower expression of hMSH2 in lip cancer than in AC.

Taken together, currently available studies that investigated MMR proteins in oral

and lip potentially malignant lesions have predominantly evaluated hMLH1 and hMSH2

proteins, always using immunohistochemical technique.

Although this methodology precludes the functional state of the proteins, most of

the results suggest that they are downregulated in more dysplastic lesions, making patients

more susceptible to additional DNA mutations and consequent malignant transformation.

However, further analyses with different laboratorial techniques are necessary to confirm this

assumption and to better understand the role played by MMR system in the pathogenesis of

OL and AC.

MMR expression in oral squamous cell carcinoma

Oral cancer is the sixth most common malignancy in the world (25), and

squamous cell carcinoma accounts for more than 90% of all cases (26). Different authors have

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attempted to determine the importance of MMR proteins for OSCC pathogenesis, and Lo

Muzio et al. (27) results represent one of the earliest evidences that a reduction in the

expression of hMSH2 and hMLH1 could be associated with OSCC development.

This initial observation was subsequently confirmed by Jessri et al. (19) that

revealed a progressive reduction of hMLH1, hMSH2, and hPMS2 from mild, moderate, and

severe dysplasia to OSCC. In a following study, Jessri et al. (25) further confirmed the lower

expression of hMLH1, hMSH2, hPMS2, and hMSH6 in more dysplastic OL and even more in

OSCC, also highlighting the diagnostic utility of hMSH6 in the identification of carcinoma in

situ. Sarmento et al. (22) confirmed the lower expression of hMLH1 and hMSH2 in OSCC

and Helal et al. (28) additionally identified a significant correlation between the low hMSH2

and p53 expressions in OSCC, suggesting that hMSH2 would play a role in the late stages of

oral carcinogenesis.

In addition to mutational status of MMR proteins, their frequently described lower

expression in OSCC can also be explained by epigenetic events. Czerninski et al. (29)

demonstrated that 50% of their OSCC cases had hypermethylation of the promoter region of

hMSH2 and hMLH1, similar to the results of González-Ramírez et al. (30) who observed that

76% of their OSCC cases presented hyper- methylation of the promoter regions of hMLH1,

whereas Sengupta et al. (20) showed that tobacco use increased susceptibility to hMLH1 and

hMSH2 hypermethylation. Tawfik et al. (31) assessed the expression and the methylation

pattern of hMLH1 promoter, observing that 30.6% of their Head and Neck Squamous Cell

Carcinoma (HNSCC) cases (including oral cavity) presented a low hMLH1 expression, with

86.7% of the cases having hypermethylation of the promoter region. On the other hand, Wang

et al. (32) failed to correlate hMLH1 and hMSH2 methylation, exon mutation, and gene

expression with the presence of microsatellite instability in patients with HNSCC.

Genetic polymorphisms of MMR genes in patients affected by OSCC were also

investigated, and some studies showed that single nucleotide polymorphism (SNP) of hMLH1

and hMSH3 can be associated with OSCC predisposition (33, 34). Nogueira et al. (35)

showed that inherited hMLH1 c.-93G>A, hMSH2 c.21119C>G, hMSH3 c.3133G>A, and

EXO1 c.1765G>A polymorphisms are predictors of clinical outcomes of patients affected by

HNSCC.

Few authors attempted to demonstrate the clinical significance of MMR proteins

in patients affected by OSCC. Cell lineages with low expression of hMSH2 and hMLH1 were

shown to be more resistant to the chemotherapeutic effect of cisplatin (36, 37), whereas our

recent study evidenced that lower hMSH2 expression was associated with higher T stage and

that both hMSH2 and hMSH6 expression predicted a poor prognosis (38).

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In contrast to the observed for potentially malignant lesions, although

immunohistochemical studies still predominate in the evaluation of MMR system in OSCC,

there are several genetic analyses currently available. However, functional assays and

different in vitro and in vivo analyses are still necessary to fully understand the role of MMR

proteins in the development of OSCC. Moreover, only one study investigated the prognostic

potential of MMR proteins, demanding additional analyses. Finally, some reports used

squamous cell carcinomas from different head and neck regions in their studies, what could

preclude stronger conclusions given the biological heterogeneity of these lesions. On the other

hand, available evidences point toward an important role played by the loss of MMR system

to OSCC pathogenesis.

MMR expression in other oral neoplasms

Mismatch repair proteins have also been investigated in other oral neoplasms like

in oral melanoma where hMLH1, hMSH2, hPMS1, and hPMS2 expressions were shown to be

frequently decreased (39, 40). Regarding salivary gland tumors, Tobón-Arroyave et al. (41)

suggested that the lower expression of hMLH1 and hMSH2 in cases of pleomorphic adenoma

would indicate a higher risk for malignant transformation of this tumor. However, no

significant differences were observed between benign and malignant salivary gland tumors in

two other studies (42, 43), whereas salivary gland adenocarcinomas demonstrated higher

expression of hMSH2 and hMLH1 when compared with pleomorphic adenomas in Castrilli et

al. (43) investigation. These results demonstrate divergent conclusions regarding MMR

proteins in salivary gland tumorigenesis.

Regarding odontogenic tumors, MMR proteins were investigated only in

ameloblastomas. Castrilli et al. (44) evaluated the expression of hMSH2 and hMLH1 proteins

by immunohistochemistry in 25 cases, observing a nuclear positivity in all of them,

speculating that the development and progression of ameloblastomas would not depend on a

defect in the MMR system.

Conclusion

Mismatch repair system is an important group of proteins for DNA repair and

maintenance of the genomic stability. Disruption of this system has been suggested to be

important in the pathogenesis of different oral lesions, including potentially malignant

disorders and malignant neoplasms. However, most of the studies currently available are

limited to demonstrate the immunoexpression of these proteins (more frequently of hMSH2

and hMLH1), and the size of the samples investigated is usually small, rarely correlating their

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expression patterns with clinicopathological parameters. Therefore, although we have

evidences that the loss of MMR proteins is important for the development of several oral

lesions, further studies are warranted to better understand their importance as possible

therapeutic targets, especially in the OSCC context, where these proteins also remain to be

further validated as prognostic markers.

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Acknowledgement

This research has been supported by grants from the São Paulo State Research

Foundation (FAPESP 2015/21520-8 and FAPESP 2015/16056-0).

Conflict of interest statement

The authors state that they have no potential conflict of interest that could bias the

results obtained in this study.

Ethics committee approval

Not applied.

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Figure 1: Schematic illustration of MMR system, exemplifying hMutSα (hMSH2 and

hMSH6) and hMutLa (hMLH1 and hPMS2) function. (A) Mismatch base (red triangle) was

incorporated in the DNA strand during replication and not recognized by DNA polymerase.

(B) hMutSα recog- nized the error and recruited hMutLa to form a complex. (C) PCNA

activates hMutLa endonuclease function to incise the daughter strand both distally and

proximally to the mispairing position. (D) hMutSα activates EXO1 to remove the excised

DNA containing the mismatch. (E) DNA polymerase with PCNA initiates the new DNA

synthesis from the nicked region.

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Table 1. Results obtained by the currently available studies that investigated MMR proteins in oral lesions.

Authors Lesion investigated MMR proteins

investigated

Main results obtained

Fujieda et al.,

1998

OSCC hMSH2 Lower expression of hMSH2 showed more resistance to

cisplatin.

Lo Muzio et al.,

1999

OSCC hMSH2/hMLH1 Reduction of these proteins may be related with OSCC.

Korabiowska et

al., 1999

Oral melanoma hMSH2/hMLH1/hPMS1/hPMS2 Lower expression correlated with high aneuploidy ratio.

Lo Muzio et al.,

2000

Oral melanoma hMSH2/hMLH1 Low expression of the proteins.

Wang et al., 2001 OSCC hMLH1/hMSH2 There is no association between MMR proteins and MSI.

Ohki et al., 2001 SGT hMSH2 hMSH2 expression is not significantly different between benign

and malignant tumors.

Castrilli et al.,

2001

Ameloblastoma hMSH2/hMLH1 All cases were positive for the proteins.

Castrilli et al.,

2002

SGT hMSH2/hMLH1 hMSH2 and hMLH1 expressions are not significantly different

between benign and malignant tumors.

Sengupta et al.,

2007

Leukoplakia/HNSCC hMSH2/hMLH1 Hypermethylation of the promoter regions and positive

correlation with tobacco use.

Czerninski et al.,

2009

OSCC hMSH2/hMLH1 OSCC presented hypermethylation of hMSH2 and hMLH1

promoters.

Tobón-Arroyave

et al., 2009

PA hMSH2/hMLH1 Lower expression correlated with a higher risk for malignant

transformation.

Adachi et al.,

2010

HNSCC hMLH1 Low expression of hMLH1 showed more resistance to cisplatin.

Souza et al., 2011 AC hMSH2 Lower expression in severe dysplasia.

González-

Ramírez et al.,

2011

OSCC hMLH1 Presence of hypermethylation of hMLH1.

Tawfik et al., HNSCC hMLH1 86.7% of the sample showed hypermethylation.

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2011

Caldeira et al.,

2011a

Leukoplakia hMLH1 Lower expression in severe dysplasia.

Caldeira et al.,

2011b

Leukoplakia hMLH1 Lower expression in severe dysplasia.

Helal et al., 2012 OSCC hMSH2 Lower expression in OSCC.

Sarmento et al.,

2013

AC/OSCC hMLH1/hMSH2 Lower expression in OSCC.

Jha et al., 2013 OSCC hMLH1 Polymorphism in this gene may be related with OSCC

predisposition.

Mondal et al.,

2013

Leukoplakia/OSCC hMSH3 Polymorphism in hMSH3 may be related with OSCC

predisposition.

de Oliveira et al.,

2014

AC hMLH1 Lower expression in severe dysplasia.

Nogueira et al.,

2015

OSCC hMLH1/hMSH2/hMSH3/EXO1 Polymorphisms in the MMR system may be related with OSCC

predisposition.

Jessri et al.,

2015a

OSCC hMSH2/hMSH6/hMLH1/hPMS2 Focal lack of hMSH6 indicates carcinoma in situ.

Jessri et al.,

2015b

Leukoplakia/OSCC hMSH2/hMSH6/hMLH1/hPMS2 Lower expression in severe dysplasia (except hMSH6).

Wagner et al.,

2016

OSCC hMSH2/hMSH6 Independent prognostic markers

AC: Actinic cheilitis; PA: Pleomorphic adenoma; Oral squamous cell carcinoma; HNSCC: Head and neck squamous cell carcinoma; SGT: Salivary gland tumors.

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2.2 Artigo: Prognostic significance of hMSH2, hMSH3 and hMSH6 expression in

ameloblastoma

Artigo aceito para publicação no periódico Oral Surgery, Oral Medicine, Oral Pathology, Oral

Radiology. (Anexo 2)

Gleyson Kleber do Amaral-Silva1, Celeste Sánchez-Romero1, Vivian Petersen Wagner2,

Manoela Domingues Martins2, Hélder Antônio Rebelo Pontes3, Eduardo Rodrigues Fregnani4,

Fernando Augusto Soares5, Oslei Paes de Almeida1, André Caroli Rocha6, Alan Roger

Santos-Silva1, Felipe Paiva Fonseca7 and Pablo Agustin Vargas1

1. Department of Oral Diagnosis - Piracicaba Dental School – University of Campinas

(Piracicaba/Brazil)

2. Department of Pathology - School of Dentistry - Federal University of Rio Grande do Sul

(Porto Alegre/Brazil).

3. Service of Buccal Pathology – João de Barros Barreto University Hospital – Federal

University of Pará (Belém/Brazil).

4. Oral Medicine Department – Sírio-Libanês Hospital (São Paulo/Brazil).

5. Department of Pathology, A. C. Camargo Cancer Center (São Paulo/Brazil).

6. Medical School, Clinics Hospital, University of São Paulo (São Paulo/Brazil).

7. Department of Oral Surgery and Pathology – School of Dentistry – Universidade Federal

de Minas Gerais (Belo Horizonte/Brazil).

Corresponding Author:

Prof. Dr. Pablo Agustin Vargas, FRCPath

Piracicaba Dental School – University of Campinas (UNICAMP)

Department of Oral Diagnosis – Oral Pathology

Av. Limeira, 901 ZipCode: 13414-903 Piracicaba – São Paulo – Brazil

Tel.: +55 19 21065319 E-mail: [email protected]

Conflict of interest statement

The authors state that they have no potential conflict of interest that could bias the results

obtained in this study. This research has been supported by grants from the São Paulo State

Research Foundation (FAPESP 2015/21520-8).

Word count for the abstract: 177 Manuscript word count: 3162

Number of references: 44 Number of figures/tables: 6 figures and 1 table

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Abstract

Aims: To investigate hMutS proteins in human dental germ, ameloblastomas and

ameloblastic carcinoma, and to determine whether the expression of these proteins has any

prognostic potential.

Methods and results: Ten cases of human dental germs, 39 ameloblastomas and 2

ameloblastic carcinomas were used to determine the distribution of the proteins during the

carcinogenesis process. Simultaneously, another sample of 73 ameloblastomas was arranged

in tissue microarray and their clinical, microscopic and radiographic features, treatment

outcome, presence of BRAF-V600E mutation and follow-up data, were assessed to determine

the prognostic relevance of the proteins. Immunohistochemistry was performed using

antibodies against hMutS (hMSH2, hMSH3, hMSH6) and Ki67. hMSH2 and hMSH6 were

significantly down-expressed in ameloblastomas (p=0.0059) than in human dental germs

(p<0.0001). hMSH2, hMSH3 and the protein combinations were significantly associated with

BRAF-V600E mutation (p<0.05). Simultaneous over-expression of hMutS was associated

with recurrences (p=0.035); however, the proteins did not predict the disease-free survival of

patients (p>0.05).

Conclusions: hMutS proteins are down-regulated in ameloblastoma; moreover, simultaneous

over-expression of these proteins in ameloblastoma was associated with recurrence, but did

not predict disease-free survival.

Keywords: ameloblastoma; odontogenic tumors; hMSH2; hMSH3; hMSH6; mismatch

protein.

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Introduction

Ameloblastoma is one of the most important odontogenic tumors given its high

frequency, local aggressiveness and infiltrative potential1-3. Surgery remains the gold standard

of treatment, ranging from conservative to aggressive approaches that lead to a recurrence rate

varying from 6% to over 51%, and different degrees of morbidity.4 Although malignant

odontogenic tumors are rarely identified, representing less than 6% of all odontogenic tumors,

ameloblastic carcinoma is the most common subtype. It may arise de novo or secondarily to a

previous benign ameloblastoma.5,6 Adult males are more affected and mandible is the most

involved location.7 Wide surgical resection is the recommended management, but there is a

recurrences rate of up to 90% and distant metastases are present in approximately 22% of the

cases.5,7

Ameloblastoma and ameloblastic carcinoma pathogenesis is poorly understood,

but a growing number of studies have attempted to define the molecular events that may drive

their development and clinical behavior. Recently, much attention has been paid to the

Mitogen activated protein kinases (MAPK) pathway mutations, especially in the B-Raf

(BRAF) oncogene,8–10 which was shown to be altered in approximately 60% of

ameloblastomas, potentially determining an increased ameloblastoma aggressiveness.11

In an attempt to avoid genetic mutations or decrease their frequency, humans

contain a complex group of proteins organized in the so-called mismatch repair system

(MMR), which is responsible for identifying and correcting mismatch and small loops of

deletion and insertion in DNA bases.12,13 The MMR system consists of hMutS with its

heterodimers hHMSH2-hMSH6 (called hMutSα) and hHMSH2-hMSH3 (called hMutSβ), and

of hMutL with its heterodimers hMLH1-hPMS2 (hMutLα), hMLH1-hPMS1 (hMutLβ) and

hMLH1-hMLH3 (hMutLγ).12 The failure in this system, either by mutations or by epigenetic

events, may lead to the development of pathological conditions, including different human

cancers,14,15 however, its importance for the pathogenesis of odontogenic tumors

remainsunclear and demands to be better understood.16 Therefore, the aim of this study is to

investigate the expression pattern of hMSH2, hMSH3 and hMSH6 in human dental germ,

ameloblastomas and ameloblastic carcinomas, and to determine whether the expression of

these proteins has any prognostic potential for patients affected by ameloblastomas.

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Material and Methods

Samples

For investigating the different expression patterns of hMutS proteins in benign

and malignant tumors and in their normal counterpart, 10 formalin-fixed paraffin-embedded

human dental germs present in surgical specimens sent for microscopic analysis of other

lesions, 39 cases of ameloblastomas and 2 cases of ameloblastic carcinomas were retrieved

from the files of three Brazilian institutions (Federal University of Pará – Belém; Federal

University of Rio Grande do Sul – Porto Alegre; and University of Campinas – Piracicaba) in

a 12-year period from January 2003 to January 2015. New 5 μm histological sections stained

with haematoxylin and eosin (H&E) were used to confirm the original diagnoses by two oral

pathologists. Clinical data regarding sex, age, tumor location, predominant microscopic

pattern and radiographic features were also retrieved from the patients’ dental charts.

Simultaneously, another set of 93 cases of ameloblastomas with full

clinicopathological data available (sex, age, tumor location, tumor size, tumor duration,

vestibular/palatine cortical disruption, basal cortical disruption, predominant microscopic

pattern, radiographic pattern, recurrences, follow-up data and BRAF-V600E

immunohistochemical status) were retrieved from the files of the Department of Pathology of

the A. C. Camargo Cancer Center (São Paulo – Brazil) and organized in a tissue microarray

(TMA) block to investigate the prognostic potential of hMutS proteins. The disease-free

survival (DFS) was obtained by calculating the time between the treatment date and the date

of recurrence or the last follow-up data obtained.The study was approved by the Ethics

Committee of the Piracicaba Dental School, University of Campinas (protocol 54172016.8).

Tissue microarray

The construction of the TMA block was previously described.11 Briefly, after the

microscopic review of the 93 ameloblastoma cases, areas of interest in each case were

selected from their respective H&E stained sections, and a TMA block was constructed

(Beecher Instruments, Silver Springs, USA). The areas identified in the donor paraffin block

were punctured twice with a 1.0 mm needle, and the two cylinders obtained were transferred

to the receptor paraffin block. A case was considered appropriate when at least 25% of the

total area of the cylinder with neoplastic tissue was available for analysis. Sequential 4 µm

thick sections were cut and collected on adhesive slides. A map specifying the exact position

of each core was made and a cylinder of normal liver tissue was included in the lower left

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corner of the TMA block for orientation. TMA protocol used for this study was previously

validated by our group.1

Immunohistochemistry

Immunohistochemical reactions were carried out in 3µm histological sections of

all cases. Samples were dewaxed with xylene and then hydrated in an ethanol series. Antigen

retrieval was performed using citrate solution (pH 6.0) in a pressure cooker for 3 minutes, and

the endogenous peroxidase activity was blocked using 10% hydrogen peroxide. After washing

in PBS buffer (pH7.4), slides were incubated overnight with primary antibodies against

hMSH2 (Polyclonal, Santa Cruz Technology, California, USA, diluted 1:100), hMSH6

(Polyclonal, Cell Marque, California, USA, diluted 1:50), hMSH3 (Polyclonal rabbit

antihuman, Novus Biologicals, Newcastle, UK) and Ki-67 (Monoclonal, clone MIB1,

DakoCytomation, Glostrup, Denmark, diluted 1:100). All slides were subsequently exposed

toavidin-biotin complex, horseradish peroxidase reagents (LSAB Kit-DakoCytomation), and

diaminobenzidine tetrahydrochloride (DAB, Sigma, St. Louis, MO, USA), and subsequently

counter-stained with Carazzi haematoxylin. Positive control sections were used for each

antibody, including colonic samples for hMSH2, hMSH3 and hMSH6, and oral squamous cell

carcinoma (OSCC) samples for Ki-67. Negative controls were obtained by omitting the

primary antibodies.

Conventional quantitative analysis

In the human dental germ, the 39 cases of ameloblastomas and the 2 ameloblastic

carcinomas, the expression of hMSH2, hMSH3, hMSH6 and Ki-67 was obtained by one

observer by counting the percentage of positive cells in a total of 1,000 cells per case in

hotspot areas, using photomicrographs in 400x taken with a DFC345 Photomicroscope (Leica

DMR, Leica Microsystems, Wetzlar, Germany) and assessed with ImageJ software (version

1.51c, National Institutes of Health, Bethesda, MD, USA).

Digital analysis

The immunohistochemical reactions against hMSH2, hMSH3 and hMSH6 done in

the ameloblastoma cases of the TMA sections were then scanned into high-resolution images

using the Aperio Scanscope CS Slide Scanner (Aperio Technologies Inc, Vista, CA). The

digital images obtained in .svs format were visualized using the ImageScope software (Aperio

Technologies Inc., Vista, CA). hMSH2, hMSH3 and hMSH6 nuclear stainings were analyzed

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using the Nuclear V9 algorithm (Aperio Technologies Inc, Vista, CA) with the following

input parameters: averaging radius: 0.9; curvature threshold: 2.5; lower threshold: 0; upper

threshold: 230; minimum nuclear size: 22; maximum nuclear size: 165; minimum roundness:

0.3; minimum compactness: 0.1; minimum elongation: 0.2; clear area objective: 240; and an

intensity threshold ranging from 0 to 230, in which strong staining was considered from 0

to185 and weak staining was from 185 to 230. At least 1000 cells were quantified in each case

and the percentage of positive cells was determined.

Statistical analysis

Initially, D’Agostino–Pearson test was used to determine the normality

distribution of the obtained data. Subsequently, to compare the immunoexpression of hMSH2,

hMSH3, hMSH6 and Ki67 between human dental germ and ameloblastoma, the unpaired

Student t-test and Mann–Whitney test were used. To investigate the association of hMSH2,

hMSH3 and hMSH6 with clinicopathological parameters, results were categorized as above

and below the mean positivity and the two-tailed Fisher’s exact test and the chi-square test

were used. Pearson and Spearman tests were used to investigate the correlation of hMSH2,

hMSH3 and hMSH6 with the expression of Ki67. Survival analysis was calculated using the

Kaplan–Meier method and the Log-rank univariate test was used to compare the groups.

Because only two cases of ameloblastic carcinoma could be retrieved, the results obtained in

this lesion were only descriptively described. GraphPad Prism version 5.01 (La Jolla, CA,

USA) and IBM SPSS version 20 (Chicago, IL, USA) software were used for statistical

analyses and a pvalue ≤ 0.05 using a 95% confidence interval was considered statistically

significant.

Results

Expression of hMSH2, hMSH3, hMSH6 and Ki67 in human dental germ, ameloblastoma

and ameloblastic carcinoma

In the ameloblastoma cases, males and females were affected in 17 cases each,

and in 5 cases the gender was not available. The mean age of this sample was 26 years (range

20–30 years) and the posterior region of the mandible was the most affected (77% or 30

cases) followed by the posterior region of maxilla (23% or 9 cases). Tumors predominantly

presented as multilocular images (38% or 15 cases), whereas in 36% (14 cases) of the cases

the radiographic exam revealed a unilocular image and in 25.6% of the cases data were not

available (10 cases). Microscopically, 41% of the cases (16/39 cases) predominantly

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presented the follicular pattern, whereas 59% of the cases (23/39 cases) predominantly

presented the plexiform pattern. Due to its rarity, only two cases of ameloblastic carcinoma

could be retrieved, one of them affecting a 28-year-old female patient whose details have been

described previously17 and the other affecting the posterior mandible of a 23-year-old male

patient.

Evaluating hotspot areas of the epithelial component of the human dental germs a

Ki67 proliferative index of 6.67% was obtained and positive cells were present both in the

ameloblastic layer and in the central stellate reticulum area. In ameloblastomas the

proliferative index was 3.76% and positive cells were predominantly located in the basal layer

of the neoplastic epithelial nests. This variation was significantly different (p < 0.0005). The

mean proliferative index of the ameloblastic carcinoma cases achieved 63% (Figure 1).

The expression of hMSH2, hMSH3 and hMSH6 proteins was found in all

investigated cases with similar distribution patterns, but different rates. All proteins were

observed in both epithelial and mesenchymal compartments of the human dental germs. In the

epithelial component the proteins were predominantly presented in the ameloblastic layer,

although some cells in the stellate reticulum were also positively stained, achieving in the

hotspot areas of the epithelium an immunoreactivity of 60.2%, 60.2% and 60.6% to hMSH2,

hMSH3 and hMSH6, respectively (Figures 2A, 3A and 4A). In ameloblastoma cases, the

proteins were also present in both peripheral and central areas of the neoplastic epithelial

nests and strands. Positive nuclei of granular cells were rarely observed and the stromal cells

were negative. Positivity in ameloblastomas achieved 43.2%, 59.5% and 40.9% to hMSH2,

hMSH3 and hMSH6, respectively (Figures 2B, 3B and 4B). Although only two cases of

ameloblastic carcinoma were analyzed, we observed a mean positivity of 38.5%, 39.5% and

41.8% to hMSH2, hMSH3 and hMSH6, respectively, also lower than those observed in the

normaltissue, but not too different from the benign tumor (Figures 2C, 3C and 4C).

Statistically, we observed that the expression of all three proteins was decreased in

ameloblastomas if compared to their normal counterpart, and significance was obtained for

hMSH2 (p = 0.0059) and hMSH6 (p < 0.0001), whereas hMSH3 did not achieve statistical

significance (p = 0.8836) (Figures 2D, 3D and 4D). Statistical analyses were not performed

for ameloblastic carcinoma.

The expression of hMSH2, hMSH3 and hMSH6 proteins did not correlate with

Ki67 proliferative index in human dental germs (p = 0.56, p = 0.59 and p = 0.43, respectively)

and in ameloblastomas (p = 0.50, p = 0.51 and p = 0.42, respectively).

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Correlation of hHMSH2, hMSH3 and hMSH6 with clinicopathological and follow-up

features of ameloblastoma

To investigate whether the expression of hMSH2, hMSH3 and hMSH6 proteins

would have any prognostic significance for patients affected by ameloblastoma, we used

TMA sections initially containing 93 cases. However, because the TMA block had been used

in previous studies and because of the aggressiveness of the immunohistochemical reactions

to the neoplastic tissue, 73 cases remained in the TMA sections to be quantified. The

clinicopathological and follow-up data regarding this final sample have been described

previously.11

All cases arranged in the TMA sections showed nuclear positivity for the three

markers in both peripheral and central areas of the nest and islands, with no staining in the

stromal cells. The sections were then digitized and the reactions quantified using the Nuclear

V9 algorithm (Aperio Technologies Inc, Vista, CA), the results of which were categorized as

above and below the mean positivity to be correlated with the clinicopathological parameters

and follow-up status of the patients. Table 1 summarizes the results obtained. Briefly, we

observed that hMSH2 and hMSH3 were significantly associated with cases presenting BRAF-

V600E mutation (p = 0.007 and p = 0.021, respectively) [data on BRAF V600E expression in

this same sample have been previously described – Fregnani et al.11], whereas hMSH6 did not

show any association. Moreover, when we organized the cases expressing high levels of

hMSH2/hMSH3 (hMutSβ), hMSH2/hMSH6 (hMutSα) and hMSH2/hMSH3/hMSH6

simultaneously, we also observed a significant association with BRAF-V600E status (p =

0.009; p = 0.007 and p = 0.002, respectively).

Although the expression of each protein individually did not demonstrate a

significant association with the presence of recurrences, when all three proteins were

simultaneously overexpressed a statistical significance was obtained (p = 0.035). No other

significant association with clinicopathological parameters was found. Finally, using

univariate analysis we were unable to find a significant difference in the DFS rates of patients

with higher and lower expression of hMSH2, hMSH3, hMSH6 and their combinations (p >

0.05), although it could be seen that in all these situations those patients with a lower

expression presented a higher DFS rate (Figures 5 and 6).

Discussion

To avoid the accumulation of genetic mutations, humans count with a complex

group of proteins that form the mismatch repair system (MMR), which is responsible for

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identifying and correcting errors in the pairing process of nucleotide bases during the DNA

replication.12,16 Here, we investigated the importance of hMutS proteins for ameloblastoma

pathogenesis and behavior because the components of this system were shown to be

deregulated in different human diseases14,18-24. Our results demonstrated that hMSH2 and

hMSH3 expression is associated with BRAF-V600E mutation and that hMSH2, hMSH3 and

hMSH6 simultaneous over-expression is associated with more recurrences, although the

proteins did not predict the DFS rate of these patients.

Very little is known about MMR proteins in odontogenic tumors. The only

research in this context is from Castrilli et al.,25 which observed the expression of hMSH2 and

hMLH1 in 25 ameloblastoma cases. However, the authors did not use a control group to

determine whether the proteins were over- or down-expressed, and did not attempt to

determine their clinical significance. In the present study, using a sample of human dental

germs in different stages of development we observed that the proteins are down-regulated in

ameloblastomas; moreover, the two cases of ameloblastic carcinoma also showed a lower

expression of hMSH2, hMSH3 and hMSH6, suggesting that this system would be down-

regulated in the tumorigenesis process of ameloblastoma/ameloblastic carcinoma, possibly

allowing the tumors to undergo more genetic mutations.

Similar to our results, investigating oral carcinogenesis Jessri et al.19 observed a

lower expression of hMLH1, hMSH2 and hPMS2 in OSCC than in oral leukoplakia, which

has also been documented by Sarmento et al.26 during lip carcinogenesis. On the other hand,

Xuan et al.27 observed an opposite distribution of hMSH2 during gall bladder carcinogenesis,

describing a higher expression in the malignancy and a lower expression in its preceding

nonneoplastic conditions. Taken together, these results suggest that the expression pattern of

MMR molecules during human tumorigenesis may differ depending on the tissue analyzed.

In accordance with some previous reports where hMutS proteins have not

predicted the survival rates of patients affected by different neoplasms,28,29 we did not observe

a correlation of hMutS expression patterns with prognostic factors in ameloblastoma either;

nevertheless, Wagner et al.18 demonstrated a significant prognostic role for hMSH2 and

hMSH6 in OSCC, Stark et al.30 found a prognostic potential for hMSH6 in glioblastoma and

Perrin et al.31 also showed that hMSH2 is a prognostic indicator for colon cancer. Thus, our

results and these studies illustrate the variable prognostic implications of MMR proteins in

different human neoplasms.

In some malignancies altered expression of MMR proteins was shown to be

associated with clinicopathological parameters such as tumor size, lymph node metastasis,

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clinical stage and chemotherapy resistance.18,32,33 In line with these reports, in our research a

significant association was demonstrated between the simultaneous over-expression of

hMSH2/hMSH3/hMSH6 and the presence of recurrences. As previously hypothesized,34 we

may also speculate that cases presenting hMutS over-expression could carry other molecular

events that would lead to a tumor with a higher potential of recurrences, stimulating the

expression of hMutS proteins as an attempt to repair other genetic errors. Therefore, hMutS

over-expression may represent an indicator of other molecular events.

In the last few years new insights into the molecular characteristics of

ameloblastoma have been obtained and different groups, including ours, have demonstrated

the importance of BRAF-V600E mutation for ameloblastoma development and behavior.9,11,35

Recent reports showed that 46 to 82% of ameloblastomas carry the BRAF-V600E

mutation9,36,37 and using the same sample used in this study, we demonstrated that BRAF-

V600E immunoexpression seems to be an important determinant of aggressiveness.11 Here,

we observed that except for hMSH6, the other proteins and their respective combinations

were all significantly associated with the presence of BRAF-V600E mutation, but we cannot

determine whether the presence of the BRAF mutation led to a higher expression of the

proteins or whether the lower expression of hMutS proteins in ameloblastomas, if compared

to human dental germs, would favor the occurrence of BRAF-V600E mutation. Interestingly,

in colorectal cancer Miyakura et al.38 and Koinuma et al.39 had already demonstrated that

BRAF somatic mutations are strongly associated with hMLH1 promoter methylation, whereas

Waldmann et al.40 showed that BRAF-V600E mutation was present in 67% of their colorectal

sample deficient for hMLH1 and Aparicio et al.41 showed that BRAF-V600E mutation was

very frequent in elderly patients affected by colorectal cancer deficient for MMR proteins.

These reports confirmed the importance of MMR system disturbance for the occurrence of

BRAF mutationsin colorectal cancer, but more studies are necessary to determine whether this

direct association is also true in ameloblastomas.

We also investigated the possible correlation of hMutS proteins expression with a

higher proliferative index measured by Ki67. Similar to previous analyzes that could not find

a significant correlation between MMR proteins and the proliferative potential of other

tumors,42 we did not obtain such correlation in ameloblastomas either. On the other hand,

Helal et al.43 demonstrated a significant correlation of hMSH2 with p53 expression in OSCC

and Köster et al.44 in breast cancer, suggesting that hMutS proteins would be more important

for the control of the apoptosis of tumor cells than for their proliferative potential.

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Despite the new data described in this study, it has the limitation of not having

investigated the functional status of the proteins, not allowing us to determine whether this

protein system is properly acting for DNA maintenance. Moreover, due to the rarity of

ameloblastic carcinoma, only a very small sample could be used, precluding statistical

analyzes and the acquisition of stronger results.

In conclusion, hMutS proteins were down-regulated in ameloblastoma; moreover,

simultaneous over-expression of these proteins in ameloblastoma was associated with

recurrence, but did not predict disease-free survival.

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Figures legends

Figure 1. Ki67 expression. A) In the epithelial component of the human dental germs Ki67

was predominantly observed in the peripheral palisade cells (DAB; 400x). B) Only some cells

were positive in ameloblastoma, more commonly in the basal cells (DAB, 400x). C) In

ameloblastic carcinoma Ki67 immunostaining was high and observed in peripheral and

central areas of epithelial nests and strands (DAB, 400x). D) Ki67 immunoexpression was

significantly higher in human dental germs than in ameloblastomas (Mann-Whitney test. p =

0.0005). Because of the small sample size of ameloblastic carcinoma, statistical analysis could

not be done for this group.

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Figure 2. hMSH2 expression. A) Nuclear expression of hMSH2 protein in human dental

germs (DAB, 400x). B) In ameloblastoma the nuclear expression was seen both in the

peripheral and central areas of the neoplastic islands and strands (DAB, 400x). C) hMSH2

was also observed in ameloblastic carcinomas (DAB, 400x). D) hMHS2 was significantly

reduced in ameloblastomas than in human dental germs (Mann-Whitney test. p = 0.0059).

Because of the small sample size of ameloblastic carcinoma, statistical analysis could not be

done for this group.

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Figure 3. hMSH3 expression. A) Positive expression in human dental germs (DAB, 400x). B)

Nuclear positivity in both peripheral and central cells of neoplastic islands and strands of

ameloblastoma (DAB, 400x). C) Both cases of ameloblastic carcinomas were also positive for

the protein (DAB, 400x). D) There was no significant difference in the expression of hMSH3

between human dental germs and ameloblastoma (Unpaired Student t-Test. p = 0.8836).

Because of the small sample size of ameloblastic carcinoma, statistical analysis could not be

done for this group.

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Figure 4. hMSH6 expression. A) Peripheral and central cells of the epithelial component of

human dental cells were positive for hMSH6 (DAB, 400x). B) Ameloblastoma was diffusely

positive (DAB, 400x). C) In ameloblastic carcinoma positivity was seen in both cases

analyzed (DAB, 400x). D) hMSH6 expression was significantly reduced in ameloblastomas

than in human dental germs (Unpaired Student t-Test. p < 0.0001). Because of the small

sample size of ameloblastic carcinoma, statistical analysis could not be done for this group.

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Figure 5. Analysis of hMSH2, hMSH3 and hMSH6 expressions as prognostic determinants

for patients affected by ameloblastoma. Results showed that patients expressing lower levels

of the proteins presented higher survival rates, but not significantly different from those

expressing higher levels of the proteins. A) High and B) low expression of hMSH2 in

ameloblastomas. C) Kaplan-Meyer survival curves were not statistically different according

to Log-rank univariate analysis (p = 0.137). D) High and E) low expression of hMSH3 in

ameloblastomas. F) Survival curves were not significantly different (p = 0.627). G) High and

H) low expression of hMSH6 in ameloblastomas. I) No statistical difference was seen

between survival rates of patients expressing different levels of this protein (p = 0.543). Using

digital analysis, blue nuclei represent negative cells and orange nuclei represent positive cells.

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Figure 6. Analysis of the importance of hMSH2, hMSH3 and hMSH6 combination for

determining the survival rates of patients affected by ameloblastomas. Although patients

expressing higher levels of the proteins exhibited a shorter survival than those expressing

lower levels of the proteins combined, no statistical significance could be obtained using the

univariate analysis. A) Survival curves obtained when hMSH2 and hMSH3 were

simultaneously over-expressed (p = 0.495). B) Survival curves obtained when hMSH2 and

hMSH6 were simultaneously over-expressed (p = 0.180). C) Survival curves obtained when

hMSH2, hMSH3 and hMSH6 were simultaneously over-expressed (p = 0.148).

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Table 1. Analysis of the association between clinicopathological parameters obtained from the 73 cases of ameloblastoma with the expression of

hMSH2, hMSH3, hMSH6 and their combination (Chi-square and Fisher’s exact tests).

hMSH2 hMSH3 hMSH6 hMSH2/hMSH3 hMSH2/hMSH6 hMSH2/hMSH3/hMSH6

Sex p = 0.890

p = 0.402

p = 0.629

p = 0.569

p = 1.000

p = 0.673

Age p = 0.834

p = 0.687

p = 0.207

p = 0.456

p = 0.336

p = 0.533

Tumor site p = 0.784

p = 0.720

p = 0.664

p = 0.724

p = 0.203

p = 0.661

Tumor size p = 0.344

p = 0.161

p = 0.091

p = 0.863

p = 0.794

p = 0.726

Tumor duration p = 0.192

p = 0.538

p = 0.925

p = 0.105

p = 0.833

p = 0.582

Microscopic features p = 0.340

p = 0.517

p = 0.411

p = 0.706

p = 0.461

p = 0.249

Radiographic features p = 0.535

p = 0.873

p = 0.492

p = 0.628

p = 0.592

p = 0.503

Vestibular/palatine cortical

disruption

p = 0.819

p = 0.576

p = 0.325

p = 0.857

p = 0.358

p = 0.456

Basal cortical disruption

p = 0.594

p = 0.346

p = 0.191

p = 0.365

p = 0.218

p = 0.218

Recurrences p = 0.106

p = 0.417

p = 0.227

p = 0.368 p = 0.056

p = 0.035*

BRAF V600E status p = 0.007* p = 0.021* p = 0.077 p = 0.009*

p = 0.007*

p = 0.002*

* Statistically significant p value.

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3 DISCUSSÃO

O ameloblastomas é o principal tumor odontogênico benigno comumente

diagnosticado nos serviços de diagnóstico oral (Nalabolu et al., 2017). Apresenta

características de agressividade local, além de índice importante de recorrência baseado na

modalidade terapêutica empregada. A progressão do tumor para o carcinoma ameloblástico

secundário é possível, apesar de ser uma ocorrência rara (Dhanuthai et al., 2012; Milman et

al., 2016). Assim como outros estudos, nos identificamos uma predominância do padrão

histológico sólido nas amostras de ameloblastomas (Dhanuthai et al., 2012; Fregnani et al.,

2010).

Os mecanismos de desenvolvimento destes tumores ainda não estão

suficientemente definidos, embora recentes estudos tenham identificado a expressão da

mutação BRAF-V600E em ameloblastomas, associando sua presença com o maior potencial

de agressividade do tumor (Fregnani et al., 2016). Esta mutação corresponde à substituição do

aminoácido valina (V) por ácido glutâmico (E) em decorrência da inserção de um nucleotídeo

timina no lugar de adenina no códon 600, gerando um mismatch do tipo transversão de

purinas (Davies et al., 2002). De acordo com Rossetti et al. (2015), este erro de pareamento é

o mais crítico por criar pontes de hidrogênio promiscuas.

Como avaliado em nossa primeira publicação, os seres humanos possuem um

sistema de proteínas homólogo ao identificado nas bactérias E. coli, onde apresentam a função

de identificação de erros de pareamento correto das bases do DNA após a replicação, além de

inserção ou deleção de bases. Nosso estudo também demostrou uma variedade de lesões orais

que apresentam alterações importantes nos níveis de expressão destas proteínas (Amaral-Silva

et al., 2017).

No nosso segundo estudo nós identificamos a expressão imunohistoquímica das

proteínas hMutS em germes dentais humanos normais, bem como a redução progressiva desta

expressão em ameloblastomas, com exceção da proteína hMSH3 (p = 0.8836). Não foi

possível obter uma análise estatística desta relação com o carcinoma ameloblástico devido ao

número reduzido de amostras (n = 2). Os casos de ameloblastomas que apresentaram

sobrexpressões das proteínas hMSH2 e hMSH3 isoladamente mostraram significativa

associação com a presença da mutação BRAF-V600E (p = 0.007 e p = 0.021,

respectivamente). Quando analisada a expressão simultânea de hMSH2/hMSH3 (hMutSβ),

hMSH2/hMSH6 (hMutSα) e hMSH2/hMSH3/hMSH6, também encontramos a associação

com BRAF-V600E (p = 0.009; p = 0.007 e p = 0.002, respectivamente).

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Alguns autores relacionam a deficiência de proteínas MMR com BRAF-V600E

em câncer colorretal esporádico, correlacionando os achados com o prognóstico e tempo de

sobrevida (Aparicio et al., 2014; Luey et al., 2014; Sinicrope et al., 2016; Toon et al., 2014;

Waldmann et al., 2015; Wang et al., 2016). Nós encontramos que, apenas de reduzidas, as

sobrexpressões simultâneas em relação as médias das proteínas hMSH2/hMSH3/hMSH6

apresentaram, além da relação estatística com BRAF-V600E, uma importante correlação

clínica com a presença de tumores recorrentes.

Assim como Theocharis et al. (2011), nós acreditamos que a sobrexpressão de

hMutS poderiam estar estimulada diante de outros eventos moleculares associados ao

potencial de recorrência, na tentativa de reparar possíveis erros de pareamento de bases desses

gens também sobrexpressos.

Não houve correlação estatística relevante entre a expressão das proteínas hMutS

e Ki67. Apenas observamos que a nossa expressão média de Ki67 nos ameloblastomas

(3,76%) estava um pouco abaixo da faixa relatada por Meer et al. (2003) (entre 4,6% a 8,8%).

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4 CONCLUSÃO

Com base nos resultados obtidos em nosso estudo, podemos concluir que:

1) Alterações no sistema mismatch estão presentes em diversas formas de

condições potencialmente malignas e em neoplasias humanas. A ausência

da expressão das suas proteínas são fatores diagnósticos para a Síndrome

de Lynch. Muitas condições patológicas apresentam redução na expressão

das proteínas, sendo isso potencialmente relacionado com eventos

epigenéticos;

2) A expressão imunohistoquímica média das proteínas hMutS estão

sequencialmente reduzidas no ameloblastoma e no carcinoma

ameloblástico em relação ao epitélio odontogênico do germe dental

humano normal;

3) A sobrexpressão das proteínas hMutS no ameloblastoma, sejam em

conjunto ou isoladas (exceto hMSH6), apresentam relação estatisticamente

significante com a presença da mutação BRAF-V600E;

4) A sobrexpressão simultânea das proteínas hMSH2/hMSH3/hMSH6 em

ameloblastomas apresentam potencial indicador para a recorrência

tumoral.

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Characteristics of Individuals with Mismatch Repair Deficient Colorectal Cancer. Digestion

2015;91:286–93. doi:10.1159/000381284.

Wang J, Andrici J, Sioson L, Clarkson A, Sheen A, Farzin M, et al. Loss of INI1 expression

in colorectal carcinoma is associated with high tumor grade, poor survival, BRAFV600E

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doi:10.1016/j.humpath.2016.04.018.

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ANEXOS

Anexo 1: Certificado de Direitos Autorais da Journal of Oral Pathology and Medicine.

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Anexo 2: Carta de aceite para publicação do Journal of Oral Surgery, Oral Medicine, Oral

Pathology, Oral Radiology.

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Anexo 3: Certificado do Comitê de Ética em Pesquisa FOP/Unicamp.