Complete and sustained remission of juvenile dermatomyositis resulting from aggressive treatment

Abstract

Objective: To assess the time needed to achieve sustained, medication-free remission in a cohort of patients with juvenile dermatomyositis (DM) receiving a stepwise, aggressive treatment protocol.

Methods: Between 1994 and 2004, a cohort of 49 children with juvenile DM who were followed up at a single tertiary care children's hospital using disease activity measures according to a specific protocol received standardized therapy with steroids and methotrexate. If a patient's strength or muscle enzyme levels did not normalize with this initial therapy, additional medications were added in rapid succession to the treatment regimen. The primary outcome measure was time to complete remission. Additional outcome measures were onset of calcinosis, effect of treatment on height, and complications resulting from medications.

Results: Forty-nine patients were followed up for a mean+/-SD of 48+/-30 months. All but 1 patient received 2 or more medications simultaneously. Transient localized calcifications occurred in 4 patients (8%), and 2 additional patients (4%) had persistent calcinosis. Despite the aggressive therapy, complications associated with treatment were mild and were primarily attributable to steroids. No persistent effect on longitudinal growth was observed. A complete, medication-free remission was achieved in 28 patients; the median time to achievement of complete remission was 38 months (95% confidence interval 32-44 months). None of these patients experienced a disease flare that required resumption of medications during the subsequent period of observation (mean+/-SD 36+/-19.7 months).

Conclusion: Our findings suggest that aggressive treatment of juvenile DM aimed at achieving rapid, complete control of muscle weakness and inflammation improves outcomes and reduces disease-related complications. In more than one-half of the children whose disease was treated in this manner (28 of 49), a prolonged, medication-free remission was attained within a median of 38 months from the time of diagnosis.

Figure 1

Time to complete clinical response and clinical remission in patients with juvenile dermatomyositis. Patients were considered to have had a complete clinical response once strength and muscle enzyme levels had normalized, with no evidence of active myositis for ≥6 months while receiving therapy. The median time to complete clinical response was 21 months (95% confidence interval [95% CI] 18–24 months), and the median time to complete remission was 38 months (95% CI 32–44 months). There was a lag between complete clinical response and clinical remission, since medications were tapered slowly, while normal strength and muscle enzyme levels were maintained. Values are the percentage of patients with clinical response or clinical remission at each time point; bars show the 95% CI.

Figure 2

Height of the patients with juvenile dermatomyositis. Growth deceleration was demonstrated after the initiation of treatment, but height percentiles returned to pre-disease levels over time (mean of 4 years after diagnosis). Values are the mean and SD. * = P < 0.05 versus pre-disease level (baseline).