Jornal de Pediatria - Vol. 79, Nº1, 2003 63

0021-7557/03/79-01/63

Jornal de Pediatria Copyright © 2003 by Sociedade Brasileira de Pediatria

ORIGINAL ARTICLE

Health related quality of life survey about children and adolescents with juvenile idiopathic arthritis Tatiana B. Brasil,1 Virgínia P.L. Ferriani,2 Claudia S.M. Machado3

Abstract Objective: to study the impact of chronic arthritis on health related quality of life by means of two selfreported tools: the parents’ version of the Childhood Health Assessment Questionnaire (CHAQ) and the Childhood Health Questionnaire PF50® (CHQ). Methods: both tools were filled in after proper instructions by 36 parents, during 1-2 clinic visits. The Disability Index (CHAQ) and the Physical and Psychosocial scores (CHQ) were compared to the core set of outcome measures, namely 1) physician’s global assessment, 2) parents’ global assessment, both scored by 10 cm visual analogue scale, 3) number of joints with active arthritis, 4) number of joints with limited range of motion, 5) erythrocyte sedimentation rate. Results: there was significant difference for all measures of disease activity, being higher in the polyarticular as compared to oligoarticular except for erythrocyte sedimentation rate, parents’ global assessment, and psychosocial score. This leads to different parents’ perceptions of disease activity and outcome. The responsiveness of the outcome measures during two follow-up visits of patients receiving active treatment indicated better responsiveness of physicians’ global assessment among the subjective measures, and intermediate responsiveness of the self-reported measures in comparison to the number of active and limited joints, and erythrocyte sedimentation rate. Conclusions: the responsiveness of two health related quality of life tools indicates their relative sensitivity for assessing clinical improvement during active treatment in Juvenile Idiopathic Arthritis patients. J Pediatr (Rio J) 2003;79(1):63-8: juvenile idiopathic arthritis, health related quality of life, functional assessment, self-reported tools, outcome.

Introduction The assessment of inflammatory activity in chronic arthritis in adults and children includes clinical measures such as joint mobility, joint deformities, radiological

alterations, laboratory measures and functional capacity measurements as well. Nevertheless, these are actually disease descriptors that reflect the physician’s perception of the patient’s clinical condition, but do not reflect the patient’s or his parents’ values and perception about his health status. Since the last decade, a great emphasis has been placed on incorporating the patient’s values and perception into his health status, and several tools have been developed with this purpose.1-3 ‘Quality of life’ in children has been defined as a subjective and multidimensional concept that includes

1. Medicine graduate student, School of Medicine of Botucatu, Universidade Estadual Paulista. 2. PhD, Assistant Professor, Department of Child Care and Pediatrics, School of Medicine of Ribeirão Preto, Universidade de São Paulo. 3. PhD, Assistant Professor, Department of Pediatrics, School of Medicine of Botucatu, Universidade Estadual Paulista. Manuscript received Mar 07 2002. Accepted for publication Oct 09 2002.

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64 Jornal de Pediatria - Vol. 79, Nº1, 2003 functional capacity and psychosocial interaction of the child and his family, while ‘health-related quality of life’ incorporates the assessment of physical symptoms, functional capacity, and the psychosocial impact of the disease on the child and his family.4 In two recent editorial reviews5,6 the authors emphasize a great number of tools under development, and the need of cultural adaptation as well as the adequacy of these tools for their application to parents and patients. In an original article, Feldman et al.7 made use of a tool developed to measure quality of life and health-related quality of life - ‘quality of my life’ -, comparing their scores with the physical and functional capacity measures, and observed the variability of the measures regarding an ample age range, diagnosis, and physical capacity. It is important to point out that these indicators should be able to detect the important clinical changes, and demonstrate the effects of the treatment even in a small series of patients, because pediatric rheumatic diseases are considered rare. Two self-report tools were used in the present study: the Child Health Assessment Questionnaire - CHAQ (parents’ version) and the Child Health Questionnaire CHQ-PF50® (parents’ form) with the purpose of studying the impact of chronic arthritis on the physical, functional, and psychosocial aspects of health-related quality of life in patients with juvenile idiopathic arthritis. The usefulness of these tools was assessed by the criteria used to indicate the improvement or worsening of the patient’s clinical condition in daily practice, associating the physical capacity indices and level of satisfaction with the clinical parameters of disease activity.

Patients and methods Between February 2000 and October 2001, 43 patients, whose parents declared to be literate, were selected. After obtaining the Free and Informed Consent from the parents or legal guardians, the participants were requested to fill out the questionnaires, which contained information about the survey and instructions for filling out the questionnaires. Thirty-six patients with regular visits to the service in this period and whose parents had voluntarily filled out both questionnaires, were included in the study. This was a convenience sample selected from a cohort of 150 patients with chronic arthritis registered at the Pediatric Rheumatology Outpatient Clinic of Hospital das Clinicas School of Medicine of Botucatu, a service of secondary and tertiary reference in pediatrics. Thirty-six cases of juvenile idiopathic arthritis were assessed8 - 17 with oligoarticular evolution and 19 with polyarticular evolution - and they were compared considering the descriptors, activity measures, and functional and quality of life assessments. During the period of study, 30 patients received active drug treatment. The patients in the oligoarticular group received nonsteroidal anti-inflammatory drugs and intra-articular corticosteroid injections (n=14).

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In the cases with polyarticular evolution, besides the use of regular oral anti-inflammatory nonsteroidal and corticosteroid drugs (n=16), 13 patients began the use of oral or parental methotrexate as the only drug with a modifying action on the disease activity and were assessed in two visits with an interval of four to six months. The disease activity was assessed and classified into four categories:1- active (with increase in the number of joints with active synovitis despite the treatment), 2- stable (the number of joints with synovitis remained stable during the treatment), 3- inactive (no evidence of synovitis and no medication for a period shorter than two years), 4- in remission (no evidence of synovitis and no medication for a period longer than two years.9 The clinical and laboratory parameters and the internationally standardized activity measures were the following: 1- assessment of overall activity made by the doctor in a 10 cm visual analogical scale (VAS) based on two points (‘inactive’ and ‘very serious’), 2- assessment of general activity made by the parents in a 10 cm visual analogical scale (VAS) based on two points (‘very well’ and ‘very bad’), 3- the count of the number of joints with active synovitis or number of active joints, 4- the count of joints with limited range of motion, 5- the erythrocyte sedimentation rate as a laboratory parameter of inflammatory activity, and 6- the functional capacity by means of a specific tool.10 Functional and Quality of Life Assessments The functional assessment was carried out through the application of the CHAQ “Childhood Health Assessment Questionnaire” (Parents’ version), 11 (adapted and valid),12-14 which assesses the functional capacity and independence in eight daily-life activities, assessing the degree of difficulty or limitations attributed to the disease in a 0-3 scale; the higher the number, the lower the capacity. Health-related quality of life was assessed through the Child Health Questionnaire CHQ - PF50 (Landgraf et al. Healthact - Boston®)15 (Parents’ version),14 applicable to children who were older than five years by means of fifteen health concepts. Each concept has a 0-100 score; the higher the score, the better the health condition, and the level of satisfaction and well-being, resulting in physical and psychosocial scores (0-70). Only the valid questionnaires, i.e., the ones entirely filled out, were analyzed; 90% of the CHAQ and 83.7% of the CHQ questionnaires were used. Statistical analysis The comparison between the values of clinical measures and the scores for functional capacity and health-related quality of life between oligoarticular and polyarticular groups was assessed through Mann-Whitney’s test. The comparison of the measures applied to the same patient in consecutive visits was assessed through Wilcoxon test. The

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statistical responsiveness of the scores for functional, clinical, and quality of life measures was calculated through the Standardized Response Mean (SRM) and through Effect Size (ES).17 The SRM was calculated as the median variation between the initial visit and the final visit, dividing the difference of the interquartile range by 3/4. Therefore, the highest SRM and ES values indicate greater responsiveness. We used Statmate - Prism Graph Pad 3.0® for the statistical analysis.

Results Thirty-six patients were included in the study. Fourteen of them were male, aged between 3.3 and 15.4 years (median 10.3). The duration of the disease in the first assessment lasted from 0.2 to 8 years (median 2.6). Among these, four patients presented antinuclear antibodies and one presented chronic iridocyclitis. The summarized clinical description of our patient population is presented in Table 1. The comparison of the clinical measures of disease activity, presented in Table 2, indicates that the oligoarticular and polyarticular groups did not differ as to age, duration of the disease and erythrocyte sedimentation rate. The only statistically significant difference was noticed in the number of joints with active inflammation, number of joints with limited range of motion and functional capacity. Among the subjective measures the one of the disease activity assessed by the doctor using a visual analogical scale showed a significant difference between the oligoarticular and polyarticular groups. However, these groups did not differ as to the measure of the parents’ perception in the assessment of general well-being. On the other hand, the parents’

Table 1 -

Clinical characteristics and classification of patients with juvenile idiopathic arthritis

Variable

n

Subtype of presentation Oligoarticular Polyarticular Systemic

22 10 4

Subtype of evolution Oligoarticular Polyarticular

17 19

Activity of the disease Active Stable Inactive Healing

20 10 3 3

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subjective assessment of the pain scale indicated a significant difference between these groups. The polyarticular group showed higher pain scores, hence, showing different parents’ perception levels about the activity. There was statistical difference between the functional capacity scores (CHAQ) of the oligoarticular and polyarticular groups. The polyarticular group had greater limitation or incapacity. We verified that the scores in each of the eight CHAQ categories were higher in the polyarticular group than in the oligoarticular group. The accounts given by the parents about the need of adaptations and devices were registered in only three questionnaires and the pick up category (pencil adapter, can opener), but the help from another person for the accomplishment of tasks was registered in 15 questionnaires under the following categories: get dressed, get up, walk, clean oneself, reach, pick up, and perform daily activities and domestic chores. The item with the greatest indication of aid was the get dressed one. In the assessment of health-related quality of life by means of a generic tool, the physical score (CHQ) showed a significant difference between both groups, with the highest scores in the oligoarticular group, indicating a better health condition. However even though the psychosocial scores (CHQ) were higher in the oligoarticular form, they did not statistically differ in the polyarticular form. These results, as a whole, indicate differences in the perception about the health condition in relation to the subjective assessment of the doctor and the clinical measures of activity. We verified a significant difference in the functional capacity, physical, and psychosocial scores (CHQ) after effective changes in the course of the treatment, observed in the second visit in three patients from the polyarticular group. The analysis of responsiveness, the statistical measure to assess the changes in functional capacity and the health status are presented in Table 3. The objective measures, which include the number of joints with active arthritis, the joints with limited range of motion, and the erythrocyte sedimentation rate present greater responsiveness. In the measures assessed by means of self-report tools, the physical capacity scores (CHAQ) and physical and psychosocial scores (CHQ) presented intermediate responsiveness. Among the subjective measures the general assessment of the activity by the doctor was the most responsive one. The parents’ subjective scores of pain and assessment of general well-being presented the lowest responsiveness.

Discussion The pediatric version of the health assessment questionnaire11,18,19 - the Childhood Health Assessment Questionnaire (CHAQ) with translation into several languages20 allows the use of this tool for the assessment of chronic arthritis in pediatric and adult patients. In the present study we confirmed the applicability of this

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66 Jornal de Pediatria - Vol. 79, Nº1, 2003

Table 2 -

Comparison between disease activity in 36 patients with juvenile idiopathic arthritis

Variable

Oligoarticular Polyarticular median (minimum-maximum)

Significance

Age (months) Arthritis duration (months)

129 (39-168) 24 (6-72)

111 (60-185) 33 (2-96)

NS NS

Joint variables Number of active joints Number of limited joints

1 (0-4) 1 (0-3)

16 (0-38) 12 (0-40)

p = 0.002 p = 0.005

Laboratory variable Erythrocyte sedimentation rate (mm)

13 (4-32)

28 (3-70)

NS

Functional capacity - CHAQ Functional capacity index (0-3)

0 (0-0.87)

1.25 (0-2.5)

p = 0.002

Global evaluation of the activity (VAS 0-10 cm) Global evaluation performed by physician Global evaluation performed by parents Pain evaluation performed by parents

1 (0-4) 0 (0-7) 0 (0-5.8)

4.5 (0-8) 3 (0-7.4) 3 (0-9.5)

p = 0.0003 NS p = 0.03

39.1 (34.5-57.1) 50.7 (9.7-57.1)

18.6 (2.8-47.6) 36.3 (8.4-53.8)

p = 0.001 NS

Quality of life - CHQ Physical index Psychosocial

CHQ - Child Health Questionnaire PF50 ® NS - nonsignificant

CHAQ - Childhood Health Assessment Questionnaire VAS - Visual and analogical scale

questionnaire when assessing the functional capacity profiles according to the parents’ perception in routine consultations. The functional capacity score, as well as the scores in the

Table 3 -

eight CHAQ categories indicate greater incapacity and activity in the polyarticular group. The infrequent use of special devices for the accomplishment of the activities may

Statistical analysis of the responsiveness of activity in a group with polyarticular juvenile idiopathic arthritis evaluated on two consecutive visits

Variable

Difference Medians

SRM

ES

Global evaluation of the activity (VAS 0-10 cm) Global evaluation performed by physician 5 Global evaluation performed by parents 3.6 Pain evaluation performed by parents 3.5

2.5 1.7 1.75

5.5 3.8 2.3

2.4 1.7 3.9

Functional capacity (CHAQ) Functional capacity index(0-3)

1.4

0.9

3.1

1.0

Quality of life (CHQ) Physical index Psychosocial index

18.6 36.3

-14.8 -13.9

4.8 2.7

0.7 1.3

Articular variables Number of active articulations Number of limited articulations

22 17

14 11

3.7 4.9

2.7 2.4

Laboratory variables Erythrocyte sedimentation rate (mm)

35

11.5

7.7

0.7

SRM - Standard response mean VAS - Visual and analogical scale CHQ - Child Health Questionnaire PF50 ®

Median Baseline values

ES - Effect size CHAQ - Childhood Health Assessment Questionnaire

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reflect a sociocultural characteristic of independence in activities, because another person’s aid in several categories of activities was registered in a great proportion of cases in the polyarticular group. The oligoarticular group presented a lower functional repercussion; however the assessment of general well-being from the parents’ perception did not differ from that of the polyarticular group. This may reflect differences in subjective assessment and perception about the disease, as described by Ruperto et al. 1999.17 The complete effect of arthritis on the patient and its prognosis is not entirely perceived without the use of a generic tool for the assessment of quality of life. Just like the Medical Outcome Health Survey (SF36) for adults,21 the Child Health Questionnaire (CHQ) has been used in several pediatric chronic diseases15 and the versions translated into Portuguese have been published in full recently.13,14 The prognosis of chronic arthritis varies quite a lot in children22-26 and the perception of different dimensions and the prognosis may vary in different cultures and societies.5,25,26 Nonetheless, there are few published studies about health-related quality of life in nonindustrialized countries. This concept includes the impact of the physical incapacity caused by the disease on school performance, social life, and on several different activities within and without the family, increasing the dimension of the impact of the disease on the child and his family. In the study developed by Feldman et al.7 two generic tools were compared - quality of life in a broader sense and healthrelated quality of life - by using a specific tool, the CHAQ physical capacity score. Considering that the authors have found an agreement among the measures, they emphasize the importance of simultaneously applying the general and generic assessment of quality of life, the functional capacity and the clinical measures of disease activity, both in daily practice as in therapeutic essays. Responsiveness is the appropriate statistical measure to assess changes to the health status or to the perception about the health status by means of therapeutic intervention. In order to be qualified as responsive, a measure must have consistent changes over time, or be compared to another measure whose value is known.28 In agreement with other studies17,27 we verified a greater responsiveness of the doctor’s general assessment and a lower responsiveness in the parents’ perception measures, among the subjective measures. The measures assessed by means of a tool showed relative sensitivity to assess the clinical improvement in patients with arthritis who were receiving specific treatment. In spite of lower responsiveness, we concluded that the physical and psychosocial dimension assessed in a comprehensive way by means of tools is applicable and useful in the routine clinical care of patients. This may also facilitate its practical application in future therapeutic essays for juvenile idiopathic arthritis.

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References 1. Ba’Pham, Klassen R. Assessing clinical measures and clinical disagreement. In: Moyer, VA Elliott EJ, Davis RL, Gilbert R, Klassen T, Logan S, et al., editores. Evidence-based Pediatrics and Child Health. London: BMJ Books - BMJ Publishing Group; 2000.p.71-78. 2. Waters E. Assessing quality of life. In: Moyer VA, Elliott EJ, Davis RL, Gilbert R, Klassen T, Logan S, et al., editores. Evidence-based Pediatrics and Child Health. London: BMJ Books - BMJ Publishing Group; 2000.p.79-90. 3. Jenney MEM, Campbell S. Measuring quality of life. Arch Dis Child 1997;77:347-54. 4. Strand CV, Russel AS. WHO/ILAR task force on quality of life. J Rheumatol 1997;24:1630-3. 5. Burgos-Vargas R. Assessment of quality of life in children with rheumatic disease [editorial]. J Rheumatol 1999;26:1432-4. 6. Tucker LB. Whose life is it anyway? Understanding quality of life in children with rheumatic diseases [editorial]. J Rheumatol 2000;27:8-11. 7. Feldman BM, Grundland B, McCullough L, Wright V. Distinction of quality of life, health related quality of life and health status in children referred for rheumatologic care. J Rheumatol 2000;27:226-33. 8. Petty RE, Southwood TR, Baum J, Bhettay E, Glass DN, Manners P, et al. Revision of the proposed classification criteria for juvenile idiopathic arthritis. Durban 1997. J Rheumatol 1998;25:1991-4. 9. Arguedas OA. Juvenile chronic arthritis - A study on epidemiology and outcome in Costa Rican children [dissertation]. Göteborg University-Sweden; 2001. 10. Giannini EH, Ruperto N, Ravelli A, Lovell DJ, Martini A. For the Pediatric Rheumatology International Trials Organization. Preliminary definition of improvement in juvenile arthritis. Arthritis Rheum 1997;40:1202-9. 11. Singh G, Athreya BH, Fries JF, Goldsmith DP. Measurement of health status in children with juvenile rheumatoid arthritis. Arthritis Rheum 1994;37(12):1761-9. 12. Len C, Goldenberg J, Ferraz MB, Hilario MO, Oliveira LM, Sacchetti S. Cross cultural reliability of the Childhood Health Assessment Questionnaire. J Rheumatol 1994;21:2349-52. 13. Melo-Gomes JA, Ruperto N, Canhão H, Fonseca JA, Quintal A, Salgado M, et al. for the Pediatric Rheumatology International Trials Organisation (PRINTO) The Portuguese version of the Childhood Health Assessment Questionnaire (CHAQ) and the Child Health Questionnaire (CHQ). Clin Exp Rheumatol 2001;19 (Suppl 23):126-130. 14. Machado CSM, Ruperto N, Silva CHM, Ferriani VPL, Roscoe I, Campos LMA, et al. for the Pediatric Rheumatology International Trials Organisation (PRINTO) The Brazilian version of the Childhood Health Assessment Questionnaire (CHAQ) and the Child Health Questionnaire (CHQ). Clin Exp Rheumatol 2001;19 (Suppl 23):25-29. 15. Landgraf JM, Albertz L, Ware JE Jr. The Child Health Questionnaire: A user’s manual. 2nd ed. Boston, The Health Act; 1999. 16. Guillemin F, Bombardier C, Beaton D. Cross-cultural adaptation of health-related quality of life measures: literature review and proposed guidelines. J Clin Epidemiol 1993;46:1417-32. 17. Ruperto N, Ravelli A, Migliavacca D, Viola S, Pistorio A, Duarte C, et al. Responsiveness of clinical measures in children with oligoarticular juvenile chronic arthritis. J Rheumatol 1999;26:1827-30. 18. Fries JF, Spitz P, Kraines RG, Holman HR Measurement of patient outcome in arthritis. Arthritis Rheum 1980;23:137-45.

68 Jornal de Pediatria - Vol. 79, Nº1, 2003 19. Timko C, Stovel KW, Moos RH, Miller JJ III Adaptation to juvenile rheumatic diseases. A controlled evaluation of functional disability with a one-year follow up. Health Psychology 1992;11:67-76. 20. Ruperto N, Ravelli A, Pistorio A, Mallattia C, Cavuto S, GadoWest L, et al. For the Pediatric Rheumatology International Trials Organisation (PRINTO) Cross-cultural adaptation and psychometric evaluation of the Childhood Health Assessment Questionnaire (CHAQ) and the Child Health Questionnaire (CHQ) in 32 countries. Review of the general methodology. Clin Exp Rheumatol 2001;19 (Suppl 23):1-9. 21. Ware JE Jr, Sherbourne CD. The MOS 33-item short-form health survey (SF-36) Conceptual framework and item selection. Med Care 1992;30:473-83. 22. Wallace CA, Levinson JE. Juvenile rheumatoid arthritis: outcome and treatment for the 1990s. Rheum Dis Clin North Am 1991;17:891-905. 23. Andersson Gare B, Fasth A. The natural history of juvenile chronic arthritis: a population based cohort study I. Onset and disease process. J Rheumatol 1995;22:295-307. 24. Andersson-Gäre B. Juvenile Arthritis - Who gets it, where and when? A review of current data in incidence and prevalence. Clin Exp Rheumatol 1999;17:367-74. 25. Ruperto N, Levinson JE, Ravelli A, Shear ES, Tague L, Murray K, et al. Long term health outcomes and quality of life in American and Italian inception cohorts of patients with juvenile rheumatoid arthritis. I. Outcome Status. J Rheumatol 1997; 24:945-51.

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26. Ruperto N, Levinson JE, Ravelli A, Shear ES, Tague L, Murray K, et al. Long term Health Outcomes and Quality of Life in American and Italian Inception Cohorts of Patients with Juvenile Rheumatoid Arthritis. II. Early predictors of outcome. J Rheumatol 1997;24:952-8. 27. Ruperto N, Ravelli A, Falcini F, Lepore L, Buoncompagni A, Gerlone V, et al. Responsiveness of outcome measures in juvenile chronic arthritis. Rheumatology 1999;38:176-80. 28. Dempster H, Porepa M, Young N, Feldman BM. The clinical meaning of functional outcome scores in children with Juvenile Arthritis. Arthritis Rheum 2001;44:1766-74.

Corresponding author: Dr. Claudia S. M. Machado Departamento de Pediatria Faculdade de Medicina de Botucatu, UNESP CEP18618-970 - Botucatu, SP, Brazil Tel./Fax: +55 14 6802.6274 / 6802.6083 / 6822.0421 E-mail: [email protected]