9th annual unite walk in the jungle on may...

The Official Newsletter of the Florida Hemophilia Association

Winter 2019

NHF Washington DaysWashington, D.C.March 27-29

HFA Annual ConferenceSan Diego, CAApril 3-6

BDCF State Day and TrainingTallahassee, FLApril 17-18

Lighten Up!Johnson & Wales (North Miami)Sunday, April 28

9th Annual Unite Walk in the JungleJungle IslandSunday, May 5

(B)LEAD: Action League ofBleedersJune 13-16

Dolphin Day CampJune 28 and July 12

36th Annual Florida BleedingDisorders ConferenceWest Palm Beach, FLJuly 26-28

UPCOMING EVENTS

AND PROGRAMS

INSIDE

Executively SpeakingPage 2

Events/Program NewsPage 3- 6, 8, 11 -12

NHF News3 - 14

FHA Resource CenterPage 15

This is the time to remember those we’ve lost and to honor those living with ableeding disorder. Help raise awareness by sharing your personal story.

Bleeding Disorders Awareness Month Month is a great time to invite your friends, family, and coworkers to join you at the

9th Annual Unite Walk in the Jungle on May 5th.Let’s rally together to continue to raise awareness and crucial funds for

our important programs and services

SIGN UP TODAY!www.uniteforbleedingdisorders.org/event/florida

Turn to page 3 for information on registering for the walk.

Hello everyone!

We are off to a great start for 2019, and we arelooking forward to providing you with some newand exciting programming throughout the year!The Bleeding Disorder Coalition of Florida, a jointeffort between FHA and HFGF, will be heading toTallahassee on April 17 -18 for our AdvocacyTraining and State Days. If you are interested in

attending, please reach out as we’d love to have you participate to helpeducate our State representatives and tell your personal story andstruggles of living with a bleeding disorder. I am excited to report that bothchapters have collectively received a bunch of proclamations for “MarchHemophilia Awareness Month” and for “World Hemophilia Day”!

In additional news, our 9th Annual Unite Walk in the Jungle is scheduledfor May 5 at Jungle Island! We currently have 25 teams signed up. We’veset our goal to have 50 fund-raising teams this year. Teams are the coreof the Unite Walk in the Jungle. As a team captain and a participant, youplay a critical role in recruiting, motivating and inspiring others toparticipate. The walk is the biggest fund-raiser throughout the countrythat UNITES the bleeding disorders community. Funds raised will be usedto assist our overall mission of finding better treatments and cures andprevent complications of bleeding disorders by providing education,emotional support, advocacy, research and services. With your support,we can surpass this year’s goal of $110,000. Please contact the FHA officeif we can assist you in setting up your teams or if you need letters ofrequests for sponsorships, in-kind donations, etc.

Finally, I want to express my deepest gratitude to everyone who continuesto support FHA! You make it possible for us to continue our mission ofserving the bleeding disorder community year after year!

¡Hola a todos!

Hemos comenzado el 2019 con buen pie y esperamos ofrecerles nuevos yemocionantes programas durante todo el año. La Bleeding DisorderCoalition of Florida (Coalición de Trastornos Hemorrágicos de Florida), unesfuerzo conjunto de la FHA y la HFGF, se dirigirá a Tallahassee el 17 y 18de abril para los días estatales y de capacitación para la defensa de lospacientes (Advocacy Training and State Days). Si le interesa asistir,comuníquese con nosotros, ya que nos encantaría que participara paraayudar a informar a nuestros representantes estatales, y para que cuentesu historia personal y los desafíos de vivir con un trastorno hemorrágico.Me complace notificar que ambas sedes han recibido en forma conjuntaun montón de proclamaciones para el “March Hemophilia AwarenessMonth” (mes de concientización sobre la hemofilia en marzo) y el “WorldHemophilia Day” (día mundial de la hemofilia).

En otras noticias, nuestra 9a Caminata anual en la selva (9th Annual UniteWalk in the Jungle) está programada para el 5 de mayo en Jungle Island.Actualmente tenemos 25 equipos inscritos. Nuestra meta para este año escontar con 50 equipos para recaudar fondos. Los equipos son parte vitalde la Unite Walk in the Jungle. Como capitán de un equipo y participante,usted cumple una función esencial para reclutar, motivar e inspirar a otrosa participar. La caminata es la actividad para recaudar fondos más grandeen todo el país que UNE a la comunidad de trastornos hemorrágicos. Losfondos recaudados se usarán para ayudar a nuestra misión global deencontrar mejores tratamientos y curas y prevenir las complicaciones delos trastornos hemorrágicos mediante la educación, el apoyo emocional,la defensa, las investigaciones y los servicios. Con su ayuda, podemossobrepasar la meta de $110,000 para este año. Comuníquese con la oficinade la FHA si es que podemos ayudarles a crear sus equipos o si necesitancartas de solicitud de patrocinio, de donaciones en bienes o servicios, etc.

Por último, quiero expresar mi más profunda gratitud a todos los quecontinúan apoyando a la FHA. Ustedes hacen posible que podamoscontinuar nuestra misión de servir a la comunidad de trastornoshemorrágicos año tras año.

2 Winter 2019 • LIFELINE

ExecutivelySpeaking MensajeEjecutivo

Debbi AdamkinFHA Executive Director

Debbi AdamkinDirectora Ejecutiva de FHA

3Winter 2019 • LIFELINE

for the 9th Annual Unite Walk in the JungleSunday, May 5, 2019 at Jungle Island

YOUR PARTICIPATION MATTERS! DON’T WAIT, FORM YOUR TEAM TODAY:

www.uniteforbleedingdisorders.org/event/florida2019

We’ve set our walk goal high for this year. Funds raised from the walk will go to theimportant services and programs we provide toour families.

Please contact us if you need help setting up and orpersonalizing your page at 305-235-0717 [email protected]

We can’t do it without you!

para la 9a Caminata anual Unite en la selvaDomingo, 5 de mayo de 2019 en Jungle Island

¡TU PARTICIPACIÓN ES IMPORTANTE! NO ESPERES, FORMA TU EQUIPO HOY MISMO:

www.uniteforbleedingdisorders.org/event/florida2019

Nuestro objetivo es alto para este año. Los fondos que se recauden con la caminata sedestinarán a los importantes servicios y programasque ofrecemos a nuestras familias.

Si necesitas ayuda para configurar tupágina o para personalizarla,comunícate con nosotros: 305-235-0717 o [email protected]

¡No podemos lograrlo sin ti! Cómo inscribirte:1. Visita www.uniteforbleedingdisorders.org/event/florida20192. Haz clic en “Register Now!”

Si ya has caminado antes: 1. Haz clic en “Login to your account” e ingresa tu nombre de

usuario y contraseña*Si olvidaste tu contraseña, haz clic en “Forgot Password?”

Si no has caminado antes:1. Ingresa tu nombre, dirección de correo electrónico, crea tu

contraseña y luego continúa al próximo paso2. Sigue las instrucciones3. ¡Llena tu información y comienza a recaudar fondos!

Página personal y página del equipoPersonaliza tus páginas para recaudación de fondos personal ydel equipo con historias personales, fotos y mensajes.

How to Register:1. Visit www.uniteforbleedingdisorders.org/event/florida20192. Click “Register Now!”

If you are a returning walker: 1. Click “Login to your account” and enter your Username

and Password*If you forgot your password, click “Forgot Password?”

If you are a new walker:1. Enter your name, email address, create your password,

and then continue to the next step2. Follow the instructions3. Fill in your information and start fundraising!

Personal Page and Team PageCustomize your Personal and Team Fundraising pages withpersonal stories, photos, and messages.


End of the Year E V E N T


THANK YOU SPONSORS

Special Thanks to Event Sponsors, Volunteers


Let’s All Lighten Up!Sunday, April 28, 2019, 10:00 a.m.

SPACE IS LIMITED, PLEASE RSVP BEFORE APRIL 8 to:[email protected]

Program is intended for Adults, Teens (13 & up)

Johnson and Wales • 1701 NE 127th Street • North Miami, FL 33181

EL ESPACIO ES LIMITADO, por favor confirme su asistencia antes del 8 de Abril a: [email protected] Programa está destinado a Adultos,Adolescentes (13 y hacia arriba)

Johnson and Wales1701 NE 127th Street • North Miami, FL 33181

The Lighten Up Program is back! On April 28, 2019, the Florida Hemophilia Association,together with the University of Miami Hemophilia Treatment Center and Johnson and Wales, will

bring you this fun, informative cooking and nutrition program.

Have a favorite recipe that could use some lightening up? Submit it to us before March 30th and itmay be one of the recipes that the Chefs at Johnson and Wales recreate into a healthier version.

Even if you’re not a Cook you will have an opportunity to be a “Chef for the Day” preparing food in astate of the art kitchen. Afterwards we’ll all enjoy our delicious creations.

¡Todos aligeran!Domingo, 28 de abril de 2019, 10:00

¡El Aligerar para Arriba programa está de vuelta! En 28 de Abril de 2019, la Asociación dehemofilia de la Florida, junto con el Centro de Tratamiento de hemofilia de la Universidad de Miami y

Johnson y Wales, llegará este divertido programa de cocina y nutrición informativa.

¿Tiene una receta favorita que podría usar algún rayo? Envíela a nosotros antes del 30 de marzo y puedeser una de las recetas que los Chefs de Johnson y Wales se recrean en una versión más saludable.

Incluso si no eres cocinero tienes la oportunidad de ser un "Chef para el Día" preparación de alimentosen una cocina de vanguardia. Luego todos disfrutaremos de nuestras deliciosas creaciones.


Florida Hemophilia Association is a non-profit, community-basedorganization, dedicated to the advocacy, education and support offamilies affected by bleeding disorders. We strive to continue to not onlyeducate our members by adding programs and events, many at therequest of our members, but also in bringing you all together to knowyou are not alone in this journey. We listen to you and do our very bestto add programs that fit your and your family’s needs, but these eventsare not free to host. They are made possible by supporters, donationsand fundraisers, to name a few. We arrange food, reserve space, reservehotel rooms, and more, depending on the event, all according to thenumber of people that RSVP. We want to be able to continue to offerthese programs throughout the year. Many times people will RSVP andnot attend, without calling, texting, or emailing to let us know that theywon’t be able to make it. We know things come up unexpectedly andsometimes last minute, especially if it’s due to a bleed, illness, orsomething else that prevents you from making an event and weunderstand. However, please contact us to let us know, even if it is lastminute! We simply need to know these things, so that we maypotentially recoup some of the costs when possible, which, in turn, willenable us to use those funds for another event. Again, we understandthat sometimes things come up at the last minute but please give us acall to let us know.

Thank you so very much…and see you soon!

La Florida Hemophilia Association es una institución comunitaria sinfines de lucro, dedicada a la defensa, educación y apoyo de familias afectadaspor los trastornos hemorrágicos. Pretendemos continuar no solo informandoa nuestros miembros al agregar más programas y actividades, muchos asolicitud de nuestros miembros, sino también uniéndolos a todos ustedes paraque sepan que no están solos en este trayecto. Los escuchamos y hacemos lomejor que podemos para agregar programas que se ajusten a las necesidadesde ustedes y de sus familias, pero estas actividades conllevan gastos. Se llevana cabo gracias a colaboradores, donaciones y recaudadores de fondos, porsolo mencionar algunos. Hacemos arreglos para las comidas, reservamoslocales, reservamos habitaciones de hotel y otras cosas más, según la actividad,en función de la cantidad de personas que confirman su asistencia. Queremospoder seguir ofreciendo esos programas durante todo el año. Muchas veces,las personas confirman su asistencia y luego no asisten, sin llamar, sin enviarun mensaje de texto o por correo electrónico para dejarnos saber que nopodrán asistir. Sabemos que las cosas se presentan inesperadamente y, a veces,al último minuto, sobre todo si se debe a un sangrado, enfermedad o algunaotra cosa que le impida acudir a una actividad, y lo entendemos. No obstante,le pedimos que se comunique con nosotros para notificarnos, así sea al últimominuto. En verdad necesitamos estar enterados de esas cosas, para tener laoportunidad de recuperar algunos de los costos cuando sea posible; lo que, asu vez, nos permitirá usar esos fondos para otra actividad. Le reiteramos queentendemos que, a veces, las cosas se presentan en el último minuto, pero lerogamos que nos llame para notificarnos.

¡Muchísimas gracias… y nos vemos pronto!

Please email [email protected] request forms or for more information.

JUNE 28 • JULY 12


Welcome to Our New Staff Member Cathy Lopez!

Let’s extend a warm welcome to Cathy, as the new part-time administrativeassistant. Cathy has over 20 years’ experience in administrative work and we’reso excited to have her join the FHA team!

For more information, please contact us by email [email protected] or call 305-235-0717.

If you are a Teen age 15-20 or a Young Adult age 21-26 with a Bleeding Disorder, you can qualify for thisawesome program! You’ll participate in challenging team-building exercises, gain leadership and life skills, and

have a lot of fun! Keep an eye on your email, because applications will go out in March!


NHF Newsletter Story • February 2019

esearchers from the University of California (UC),Davis have been investigating whether two genetically

engineered cell types could be customized for long termtherapeutic benefit in individuals with hemophilia A. To thisend, Aijun Wang, PhD co-director of the surgicalbioengineering laboratory at UC Davis and his team treatedhemophilia A mice subjects with this unique gene therapy.

Through an intramuscular injection, the mice subjectsreceived human-sourced endothelial colony-forming cells(ECFCs) and placenta-derived mesenchymal stromal cells(PMSCs). ECFCs can multiply and take on the characteristicsof endothelial cells, which typically line the walls of bloodvessels in organs such as the heart, liver and intestines and areknown to produce FVIII. PMSCs are, according to theauthors, “well established as a stem cell therapy product for awide variety of diseases and conditions.”

The ECFCs and PMSCs were specifically engineered with aversion of the FVIII gene, along with repurposed retrovirusescalled lentiviral vectors. These vectors act as vehicles, carryingthe customized genetic material to elicit the production ofFVIII. One advantage in using lentiviruses is that mostpatients do not generate antibodies to this type of vector,avoiding an immune response that would otherwise render thetreatment ineffective. Another benefit of using lentiviral

vectors is their large size, enabling them to deliver greaterconcentrations of the FVIII gene to produce a more optimaltherapeutic effect.

While a drawback to using transplanted cells can be a verybrief period of viability – in this case only short-term FVIIIproduction – the outcomes here were promising. The resultsshowed that the co-transplant of PMSCs and ECFCs resultedin the most optimal and enduring transplants, yielding afunctional FVIII inside the mice’s bodies over at least sixmonths. Investigators also observed that the more successfultransplants were performed a few days after birth, versus thoseperformed in adult mice. Following therapy, neonatalhemophilia A mouse models with less than 1% of FVIII sawa two-thirds reduction in blood loss in response to a cut.

“This work demonstrated that co-transplantation of ECFCswith PMSCs at the neonatal age is a potential strategy toachieve stable, long-term engraftment, and thus holds greatpromise for cell-based treatment of hemophilia A,” concludedthe authors.

The study, “Potential Long-Term Treatment of HemophiliaA by Neonatal Co-Transplantation of Cord Blood-DerivedEndothelial Colony-Forming Cells and PlacentalMesenchymal Stromal Cells,” was published in the journalStem Cell Research & Therapy.

R

Gene Therapy with Transplanted Cells shows promise in Hemophilia A miceHemophilia News Today, January 28, 2019

nvestigadores de la Universidad de California (UC) enDavis han estado investigando si dos tipos de células

modificadas genéticamente pudieran personalizarse paraofrecer un beneficio terapéutico a largo plazo en personas conhemofilia A. A estos efectos, Aijun Wang, PhD, co-director dellaboratorio de bioingeniería quirúrgica de UC Davis y suequipo trataron ratones con hemofilia A con estarevolucionaria genoterapia.

A través de una inyección intramuscular, las ratas recibieroncélulas endoteliales humanas formadoras de colonias

(endothelial colony-forming cells, ECFC) y células estromalesmesenquimatosas derivadas de placenta (placenta-derivedmesenchymal stromal cells, PMSC). Las ECFC se puedenmultiplicar y tomar las características de las célulasendoteliales, que habitualmente recubren las paredes de losvasos sanguíneos en órganos como el corazón, el hígado y losintestinos, y que se sabe que producen factor VIII. Según losautores, las PMSC están “bien consolidadas como unproducto de tratamiento con células madre para una ampliavariedad de enfermedades y afecciones”.

I

Genoterapia con células trasplantadases promisoria en ratones con hemofilia AHemophilia News Today, 28 de Enero de 2019

(Continue page 14)


NHF Newsletter Story • February 2019

Las ECFC y las PMSC se genomodificaron específicamentecon una versión del gen del factor VIII, junto con retrovirusmodificados llamados vectores lentivirales. Esos vectoresactúan como vehículos que llevan el material genéticopersonalizado para provocar la producción del factor VIII.Una de las ventajas de usar los lentivirus es que la mayoría delos pacientes no genera anticuerpos contra este tipo de vector,lo que evita una respuesta inmunitaria que, de otra forma,haría que el tratamiento fuera ineficaz. Otro beneficio de usarlos vectores lentivirales es su gran tamaño: eso les permitellevar mayores concentraciones del gen del factor VIII paraproducir un efecto terapéutico óptimo.

Aunque uno de los inconvenientes del uso de célulastrasplantadas puede ser un muy breve período de viabilidad (eneste caso, la producción de factor VIII solo a corto plazo), losresultados en este estudio fueron promisorios. Los resultadosmostraron que el trasplante concomitante de PMSC y ECFCdio lugar a los trasplantes más óptimos y duraderos al producirun factor VIII funcional dentro de los cuerpos de los ratones

durante por lo menos seis meses. Los investigadores tambiénobservaron que los trasplantes más exitosos se realizaron apocos días del nacimiento, en comparación con los que serealizaron en ratones adultos. Luego del tratamiento, losmodelos neonatales de ratas con hemofilia A que tenían menosde un 1% de factor VIII mostraron una reducción de dos terciosen la pérdida de sangre como respuesta a una cortadura.

“Este trabajo demostró que el trasplante concomitante deECFC con PMSC durante la etapa neonatal es una posibleestrategia para lograr que el prendimiento del injerto seaestable y a largo plazo y, por lo tanto, muy prometedor para eltratamiento basado en células de la hemofilia A”, concluyeronlos autores.

El estudio, “Potential Long-Term Treatment of HemophiliaA by Neonatal Co-Transplantation of Cord Blood-DerivedEndothelial Colony-Forming Cells and PlacentalMesenchymal Stromal Cells”, se publicó en la revista StemCell Research & Therapy.

Genoterapia con células trasplantadas es promisoria enratones con hemofilia A – Cintinued...


SCHOLARSHIP FUNDS

FHA will be awarding up to FOUR (4) $1,000 scholarships for the 2019-2020 academic year.

In order to qualify for this scholarship, the applicant must be diagnosedwith Hemophilia, Von Willebrand Disease or related inherited bleedingdisorder OR the parent of a child diagnosed with Hemophilia, VonWillebrand disease or other related bleeding disorder. Applicantsmust be currently enrolled in good academic standing at ORaccepted into an accredited college, university or trade schooland pursuing a degree, certification or license. Applicants mustbe Florida residents.

For more information or to obtain an application packet, please contact

[email protected] or call 305.235.0717

APPLICATIONS ARE NOW BEING ACCEPTED!

CENTERS FOR BLEEDING DISORDERS*Hemophilia Treatment Center (HTC)

ABOUT THIS PUBLICATION

Florida Hemophilia

Association Office

(888) 880-8330

Executive Director

Debbi Adamkin(305) [email protected]

National Hemophilia

Foundation

(800) 424-2634

Hemophilia Federation of America

(800) 230-9797

LA Kelley

Communications, Inc.

Free resource material onHemophilia(978) 352-7657

CONTACT NUMBERSMISSION STATEMENT

Do the 5

1 Get an annual comprehensive check-up at a hemophilia treatment center. 2 Get vaccinated – Hepatitis A and B are preventable.3 Treat bleeds early and adequately. 4 Exercise to protect your joints. 5 Get tested regularly for blood-borne infections

Johns Hopkins All Childen's

Outpatient Care Clinic

Pediatric Cancer andBlood Disorders Center601 5th Street South,Third FloorSt. Petersburg, FL 33701Phone: (727) 767-4931www.allkids.org*HTC

Arnold Palmer Hospital

for Children

The Haley Center for Children’s Cancer and Blood Disorders92 West Miller St., MP 318Orlando, FL 32806Phone: (321) 841-8588www.orlandohealth.com*HTC

Joe DiMaggio

Children’s Hospital

Pediatric Specialty Center1150 N. 35th Ave.Suite 520Hollywood, FL 33021Phone: (954) 986-2234

Lee Memorial Hospital

Department of PediatricHematology & Oncology9981 So. Healthpark Dr.Suite 156Ft. Myers, FL 33908Phone: (239) 332-1111

Nemours Children’s

Clinic, Jacksonville

Department of PediatricHematology/Oncology807 Children’s WayJacksonville, FL 32207Phone: (904) 697-3789(904) 697-3600www.nemours.com*HTC

Nicklaus Children’s

Hospital

Kidz Medical ServicesDivision ofHematology/Oncology3100 SW 62 AveSuite 121Miami, Florida 33155Phone: (305) 662-8360*HTC

University of Florida

Hemophilia Treatment CenterDivision of Pediatric Hematology/Oncology1600 SW Archer RoadGainesville, FL 32610Phone: (352) 273-9120www.peds.ufl.edu/divisions/hemonc/*HTC

Nemours Children’s

Clinic, Orlando

Department of PediatricHematology/Oncology13535 Nemours ParkwayOrlando, FL 32827Phone: (904) 697-3789,(904) 697-3600

Sacred Heart Pediatric

Hemophilia Program,PensacolaPhone: (850) 416-7712

St. Joseph’s Children’s

Hospital

Pediatric HematologyOncology Out-patientClinic3001 W. Dr. MartinLuther King Jr. Blvd.Tampa, FL 33607Phone: (813) 554-8294or (813) 321-6820*HTC

The University of Miami

Hemophilia Treatment

Center Pediatrics and Adults

University of Miami Miller

School of Medicine

Department of Pediatrics Div of Hematology/OncologyLocator Code D8201601 NW 12th Ave., Room 5019Miami, FL 33136Phone: (305) 243-0834www.htcextras.org*HTC

USF Adult Hemophilia

Center

Hematology 4th Floor13220 Laurel DriveTampa, FL 33612Phone: (813) 974-3725*HTC

LIFE LINE is the officialNewsletter of the FloridaHemophilia Association.It is produced quarterly anddistributed free of charge torequesting members of thebleeding disorder community.

Florida Hemophilia

Association Headquarters

915 Middle River Drive, Suite 421Ft. Lauderdale, FL 33304Toll Free: (888) 880-8330www.floridahemophilia.org

Translation:

Roxanna Delgado

Design and Production:

Group M, Advertising & Design305-235-2538

15WINTER 2019 • LIFELINE

FHA ResourceCenterThe Florida Hemophilia Association, Inc (FHA) is a Not-for-Profit organization that isdedicated to enhancing the quality of life in the bleeding disorders community by creatingprograms and services that provide education, emotional support and advocacy. We arecontributing toward research to ultimately find a cure.

Florida Hemophilia Association(Formerly known as Florida Chapter, NHF)915 Middle River Drive, Suite 421Ft. Lauderdale, FL 33304

9th annual unite walk in the jungle on may...

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