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Medium- and long-term functional outcomes in a multicenter cohort of children with juvenile dermatomyositis
A M Huber1, B Lang, C M LeBlanc
1Hospital for Sick Children, Toronto, Ontario, Canada.
Insights
Most juvenile dermatomyositis patients achieve good physical function, but many experience chronic symptoms and require ongoing treatment years after diagnosis. Further research is needed to improve long-term outcomes.
Area of Science:
- Pediatric Rheumatology
- Immunology
- Clinical Outcomes Research
Background:
- Juvenile dermatomyositis (DM) is a rare autoimmune disease affecting children.
- Understanding long-term functional outcomes is crucial for managing this chronic condition.
Purpose of the Study:
- To evaluate the long-term functional outcomes in a cohort of patients diagnosed with juvenile dermatomyositis.
- To identify factors influencing disease course and patient well-being.
Main Methods:
- Retrospective inception cohort study of 80 patients diagnosed between 1984-1995 at Canadian pediatric centers.
- Follow-up included telephone or in-person interviews assessing physical function (CHAQ), educational/vocational achievement, growth, calcinosis, and patient satisfaction.
- Data on disease presentation, treatment, and course were obtained via chart review.
Main Results:
- Sixty-five patients (81%) were followed for a median of 7.2 years.
- Most patients (95%) received corticosteroid treatment; 63% received second-line agents.
- Median Childhood Health Assessment Questionnaire (CHAQ) score was 0, indicating excellent physical function, with only 8% experiencing moderate-to-severe disability.
- Females and patients with a chronic continuous disease course had higher CHAQ scores.
- Calcinosis developed in 34% of patients and persisted in 22%.
- At follow-up, 40% had persistent rash, 23% reported weakness, and 35% were still on medication over 3 years post-diagnosis.
Conclusions:
- Patients in this cohort generally experienced favorable functional outcomes with good physical function.
- However, a significant proportion had chronic disease manifestations, including persistent rash and ongoing medication use.
- Further research is warranted to enhance long-term management and improve outcomes for individuals with juvenile dermatomyositis.
Objective:
To evaluate functional outcomes in a cohort of patients with juvenile dermatomyositis (DM).
Methods:
A retrospective inception cohort of patients diagnosed as having juvenile DM between January 1, 1984 and January 1, 1995 was established at 4 Canadian tertiary care pediatric centers. Informed consent was obtained. Each subject was interviewed by telephone or in person. The primary outcome was physical function, as measured by the Childhood Health Assessment Questionnaire (CHAQ). Additional outcomes were educational and vocational achievement, growth, development of calcinosis, patient satisfaction with outcome, and development of other illnesses. Data regarding illness presentation, treatment, and disease course were obtained through chart review.
Results:
Sixty-five of 80 patients (81%; 46 females and 19 males) could be contacted. The median followup time was 7.2 years (range 3.2-13.9 years), with a median age at followup of 13 years (range 7-26 years). Twenty-four patients (37%) had a monocyclic course, while the remaining 41 (63%) had a chronic continuous or polycyclic course. Sixty-two patients (95%) were treated with corticosteroids, while 41 (63%) received a second-line agent. Physical function was excellent, with a median CHAQ score of 0 (range 0-2.50). Eighteen patients had scores >0, and only 5 had moderate-to-severe disability, as defined by a CHAQ score >1.0. Females had higher CHAQ scores, and all but 1 of the patients with scores >0 were female (range 0-2.50; P = 0.015). Patients with a chronic continuous course also had higher CHAQ scores. Sixteen patients in the chronic continuous group had CHAQ scores >0 (range 0-2.50; P = 0.0009). Calcinosis developed in 22 patients (34%) and persisted to followup in 14. Development of calcinosis was not related to initial therapy, sex, or disease course, but was significantly associated with higher CHAQ scores (range 0-1.0 versus 0-2.5; P = 0.01). At the time of followup, 26 patients (40%) still had rash, 15 (23%) still reported weakness, and 23 (35%) continued taking medications, despite the fact that all were at least 3 years postdiagnosis. There was 1 death.
Conclusion:
In general, patients in this cohort had favorable outcomes. Most had CHAQ scores of 0, and only 8% met our definition of moderate-to-severe disability. However, many patients continued to have chronic disease, persistent rash, and continued taking medications >3 years after diagnosis. Further research is needed to improve outcomes for patients with juvenile DM.