Background:There is paucity of data regarding long-term outcome and cumulative damage in children with juvenile dermatomyositis (JDM) from the Indian subcontinent.Objectives:To assess the long-term outcome and cumulative damage in children with JDM receiving treatment at a tertiary hospital in southern India.Methods:Retrospective review of records and cross-sectional assessment of outcome and damage in 29 patients with JDM at a tertiary hospital in Kochi, India. The disease course was categorized as monocyclic, polycyclic and chronic progressive. Cumulative damage was assessed using the IMACS myositis damage index (MDI).Results:Twenty-nine patients (male-16) diagnosed with definite or probable juvenile dermatomyositis based on the Bohan and Peter criteria and having a minimum follow-up period of 3 years each were enrolled. Of these, 20 children were diagnosed and initiated on treatment at our institute (inceptional) and 9 were diagnosed elsewhere and referred to our centre for further management (non-inceptional). The mean age at disease onset was 7.01 ± 3.34 years (range 1.0 to 13.5 years). The median interval from onset to diagnosis was 3 months (range- 3 weeks to 8.75 years). Delayed diagnosis defined as interval from onset to diagnosis exceeding 6 months was noted in case of 8 children. Among patients in the non-inceptional group, six were considered to have not received standard of care treatment prior to referral to our centre. Standard of care treatment was defined as initiation of a treatment regimen comprising of a combination of glucocorticoids with an immunosuppressive agent within 4 weeks of diagnosis. A total of 11 children had a delayed diagnosis and/or had not received standard of care treatment prior to referral.At our centre, all patients received oral steroids and subcutaneous methotrexate as standard therapy. Pulse steroids were used to induce remission in 12 patients and as a rescue for relapses in 2 patients. Intravenous immunoglobulin was used in 10 children with severe myositis, oropharyngeal weakness, refractory cutaneous disease including calcinosis and concomitant infection, where affordable with good results. Hydroxychloroquine was added in 15 patients with dominant cutaneous manifestations. Mycophenolate mofetil, azathioprine and tacrolimus were used in patients refractory or intolerant to methotrexate. Cyclophosphamide and rituximab were used in 4 patients each with refractory disease and extra-muscular manifestations such as interstitial lung disease. Seven patients with refractory calcinosis received pamidronate infusions.The total follow-up duration was 121.06 patient-years. A monocyclic course was observed in 11/29 (37.9%), chronic progressive course in 16/29 (55.2%) and polycyclic course in 2/29(6.9 %). Nearly half of the patients (14/29) had damage in at least one organ using the MDI (MDI ≥1). 38% of the patients (11/29) had at least one sign of cutaneous damage, the most common being calcinosis (n=11) and lipodystrophy (n=4). This was followed by skeletal damage (n=6, joint contractures-5, osteoporosis with fracture-1), muscle and pulmonary (pulmonary fibrosis) damage in 3 patients each, endocrine damage in 2 patients (diabetes mellitus-1, growth failure and delayed development of secondary sexual characteristics-1) and chronic infection in one patient. All patients with damage in at least one organ had a chronic progressive course, indicating damage accrual. Delayed diagnosis and lack of standard of care treatment prior to referral (i.e., longer duration of untreated/ sub-optimally treated disease) were associated with an increased risk of cumulative damage (p< 0.05).Conclusion:Nearly half of the patients had damage in at least one organ using the MDI. Cutaneous damage was the most common, followed by skeletal, muscle, pulmonary and endocrine damage. Longer duration of untreated/ sub-optimally treated disease significantly increases the risk of cumulative damage, highlighting the need for an early diagnosis and referral to pediatric rheumatology services.Disclosure of Interests:None declared
B Cell Activating Factor, Renal Allograft Antibody-Mediated Rejection, and Long-Term Outcome
