rheumatology 1999 petty 739 42

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Rheumatology 1999;38:739742

Paediatric Rheumatology Workshop/Series Editor: P. Woo

Prognosis in children with rheumatic diseases:justification for consideration of new therapies

R. E. Petty

Division of Rheumatology, Department of Paediatrics, University of BritishColumbia, Vancouver, Canada and Division of Immunology, Department ofPaediatrics, University of Utrecht, Utrecht, The Netherlands

The introduction of any new therapy is based on dissatis- All studies of the outcome of childhood arthritisdescribe the results of treatment instituted a decade orfaction with the effectiveness or the side-effects of currentmore earlier. It is likely that current therapy is moretherapy, i.e. the prognosis of the disease as it is currentlyefficacious and, therefore, long-term studies of prognosistreated. In order to determine the appropriateness of amay have little relevance to prognosis in patients innew therapy such as autologous bone marrow trans-whom optimal treatment is initiated today. The outcomeplantation (ABMT), it is important to have an under-

of published studies is, thus, likely to be less optimisticstanding of the anticipated long-term prognosis of thethan is actually the case at present.diseases for which it is proposed. Although ABMT is

being considered for a number of rheumatic diseases ofWhat is meant by outcome?

childhood, the present discussion will focus on chronicA number of parameters must be considered whenarthritis. Three aspects will be considered:evaluating the outcome of childhood arthritis. Examples

1. What are the difficulties in evaluating the outcome of such measurements include mortality, morbidity,of a chronic disease such as juvenile rheumatoid erosions, drug toxicity, function and quality of life.arthritis (JRA)? Mortality is seldom an imminent issue in most patients

2. What is the outcome of the diseases as currently with chronic arthritis. However, the long-term effects oftreated? childhood arthritis on lifespan are not yet known. In

3. What are the disease or demographic characteristics disorders in which mortality may increase with diseasethat predict unfavourable outcome at disease onset? duration, we are often left with incomplete data, since

long-term studies of children with rheumatic diseases

are limited. In adults with rheumatoid arthritis, it isEvaluating outcomeestimated [4] that the lifespan is shortened by from 3 to

Studies of the outcome of systemic or polyarticular 18 yr. This increased mortality is contributed to byarthritis are few and fraught with difficulty in interpret- complications of therapy as well as the disease itself.ation because of a number of factors. The orderly Function, health status and assessment of the quality ofevaluation of studies of outcome requires that the life are somewhat overlapping aspects of outcome [ 5].patients studied meet recognized diagnostic or classi- Estimates of functional outcome in chronic arthritisfication criteria. The criteria of the American College have most frequently used the Steinbrocker scale [6 ].of Rheumatology (ACR) [1 ], the European League Measurement of health status in childhood arthritisAgainst Rheumatism ( EULAR) [2 ] or the International most often uses the Childhood Health AssessmentLeague of Associations for Rheumatology (ILAR) [3] Questionnaire (CHAQ) [ 7], which is useful in childrenare acceptable for the description of chronic childhood with chronic arthritis of all types, over a wide range ofarthritis. Unfortunately, not all studies of prognosis ages. The Juvenile Arthritis Functional Assessment Scalehave faithfully adhered to accepted diagnostic and classi-

(JAFAS) [8 ] and Juvenile Arthritis Functional Assessmentfication criteria, thereby making the interpretation of Report (JAFAR) [9 ] are somewhat more complicated anddata somewhat difficult. Furthermore, rheumatic dis- do not adequately evaluate children under the age of 8 yr.eases may be difficult to diagnose accurately at onset, Quality of life scales [1012] are being developed, andand changes in diagnosis over time must be taken into have also been used recently in limited studies.account.

Current outcome of chronic childhoodarthritisAccepted 15 March 1999.

Correspondence to: R. E. Petty, Division of Rheumatology,Mortality

Department of Paediatrics, University of British Columbia,Studies of mortality in children with chronic arthritisVancouver, Canada and Division of Immunology, Department of

Paediatrics, University of Utrecht, Utrecht, The Netherlands. are few. In a 1976 survey, Baum and Gutowska [13]

1999 British Society for Rheumatology739


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R. E. Petty740

calculated the mortality from JRA to be 4.2% in Europe outcome. Furthermore, most of the patients were not inremission at the time of last follow-up. There is noand 1.1% in the USA. This discrepancy was ascribed

almost totally to the much higher frequency of amyl- doubt that this outcome has dramatically improved inthe last three decades, but some children with polyarticu-oidosis in children with systemic or severe polyarticular

disease in Europe. More recent estimates have placed lar or systemic-onset disease still encounter significantimpairment of function. In a study published in 1984the mortality rate at 50% of patientsclinical experience, most paediatric rheumatologistswith polyarticular onset JRA had very limited independ-believe that, of all children with chronic arthritis, thoseence or were confined to bed or a wheelchair.with systemic arthritis who have polyarticular disease,Approximately 25% of those with systemic onset andand children with extensive symmetrical polyarthritis,10% of those with oligoarticular onset had such a seriousespecially with rheumatoid factor, are most likely to

T 1. Mortality in juvenile rheumatoid arthritis have a poor prognosis, i.e. they are most likely to havea prolonged course, least likely to go into remission,

Onset type n %most likely to have a poor functional outcome, mostlikely to have complications of disease or therapy, andSystemic 21 63.6

Polyarticular 6 18.2 most likely to experience a poor quality of life. TheOligoarticular 4 12.1 evidence to support the clinical impression is somewhatUnknown 2 6.0 limited, however. The results of some of the more recent

studies will be summarized below.Adapted from Wallace and Levinson [15].Flato et al. [18] have described outcome 10 yr after

onset of disease in a group of 54 children with JRA, 25T 2. Causes of death in juvenile rheumatoid arthritisof whom had persistently oligoarticular disease and

Disease related 12 28 of whom had a polyarticular disease course afterCardiac 9

oligoarticular, polyarticular or systemic onsets. The pur-Renal amyloid 1pose of their study was to identify factors assessed onEncephalitis 1

Macrophage activation syndrome 1 first admission, or within the first 5 yr of disease, thatTreatment related 12 predicted an unfavourable outcome. Evaluation of dis-

Infection 4 ease outcome, as measured by the presence of remission,Surgery/anaesthesia 3

the presence of erosions and the score on a disabilityAdrenal insufficiency 1index, was studied with respect to disease onset andAcute myocardial infarct (steroid) 1

Transfusion reaction 1 course. Remission, based on the ACR criteria, requiredDuodenal perforation 1 fulfilment of five or more of the following criteria for atReyes syndrome 1

least 6 months, irrespective of drug therapy: (a)


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Prognosis in children with rheumatic diseases 741

tendon sheaths; (d) erythrocyte sedimentation rate Schneider et al. [19] studied 38 consecutive patients(ESR)


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Murray K et al. Longterm health outcomes and qualityReferencesof life in American and Italian inception cohorts ofpatients with juvenile rheumatoid arthritis. I. Outcome1. Brewer EJ, Bass J, Baum Jet al.Current proposed revisionstatus. J Rheumatol 1997;24:94551.of JRA criteria. Arthritis Rheum 1997;20(suppl.):1959.

11. Ruperto N, Ravelli A, Levinson JE, Shear ES, Murray K,2. European League against Rheumatism: EULAR BulletinTague BLet al. Longterm health outcomes and quality ofNo. 4. Nomenclature and classification of arthritis inlife in American and Italian inception cohorts of patientschildren. Basel: National Zeitung, 1977.with juvenile rheumatoid arthritis. II. Early predictors of3. Petty RE, Southwood TR, Baum J et al. Revision of the

outcome. J Rheumatol 1997;24:9528.proposed classification criteria for juvenile idiopathic arth- 12. Duffy CM, Arsenault L, Watanabe Duffy KN, Paquinritis: Durban 1997. J Rheumatol.JD, Strawczynski H. The juvenile arthritis quality of life4. Felson DT. Epidemiology of the rheumatic diseases. In:questionnairedevelopment of a responsive index forKoopman WJ, ed. Arthritis and allied conditions, 13th

juvenile rheumatoid arthritis and juvenile spondyloarthrit-edn. Baltimore: Williams and Wilkens, 1996:9.ides. J Rheumatol 1997;24:73846.5. Duffy CM, Duffy KN. Health assessment in the rheumatic

13. Baum J, Gutowska G. Death in juvenile rheumatoiddiseases of childhood. Curr Opin Rheumatol 1997;

arthritis. Arthritis Rheum 1977;20(suppl.):2535.9:4407.

14. Rennebohm R, Corell JK. Comprehensive management6. Steinbrocker O, Traeger CH, Batterman RC. Therapeutic

of juvenile rheumatoid arthritis. Nurs Clin North Amcriteria in rheumatoid arthritis. J Am Med Assoc

1984;19:64762.1949;140:65962.

15. Wallace CA, Levinson JE. Juvenile rheumatoid arthritis:7. Singh G, Athreya BH, Fries JF, Goldsmith DP. outcome and treatment for the 1990s. Rheum Dis Clin

Measurement of health status in children with juvenile North Am 1991;17:891905.rheumatoid arthritis. Arthritis Rheum 1994;37:17619. 16. Stillman JS, Barry PE. Juvenile rheumatoid arthritis: series

8. Lovell DJ, Howe S, Shear ER, Hartner S, McGirr G,

2. Arthritis Rheum 1977;20(suppl.):1715.Schulte Met al. Development of a disability measurement 17. De Inocencio J, Lovell DJ. Clinical and functional moni-tool for juvenile rheumatoid arthritis: The Juvenile toring, outcome measures and prognosis of juvenileArthritis Functional Assessment Scale. Arthritis Rheum chronic arthritis. Baillieres Clin Paediatr 1993;1:769801.1989;32:13905. 18. Flato B, Aasland A, Vinje O, Forre O. Outcome and

9. Howe S, Levinson J, Shear E, Hartner S, McGirr G, predictive factors in juvenile rheumatoid arthritisSchulte Met al. Development of a disability measurement and juvenile spondyloarthropathy. J Rheumatol 1998;tool for juvenile rheumatoid arthritis: the Juvenile Arthritis 25:36675.Functional Assessment Report for Children and their 19. Schneider R, Lang BA, Reilly BJ, Laxer RM, SilvermanParents. Arthritis Rheum 1991;34:87380. ED, Ibanez Det al. Prognostic indicators of joint destruc-

tion in systemic-onset JRA. J Pediatr 1992;120:2005.10. Ruperto N, Levinson JE, Ravelli A, Shear ES, Tague BL,

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