Pediatric Rheumatology Online Journal → July 2003 → Dermatomyositis → Treatment → Abstract #125

CYCLOPHOSPHAMIDE USE IN JDM - RETROSPECTIVE CASENOTE REVIEW OF EFFICACY AND SAFETY

P. Riley,¹ S. Maillard,¹ P. Woo,¹ C. Pilkington.¹

¹Juvenile Dermatomyositis Research Centre, Institute of Child Health, Great Ormond Street, London, United Kingdom

Background A proportion of Juvenile Dermatomyositis (JDM) patients have disease refractory to conventional treatments, or have a vasculopathic pattern which is considered high risk for disease related morbidity and mortality, including complications such as ulceration, calcinosis, GI vasculitis and respiratory compromise. This study examines the efficacy and safety of IV Cyclophosphamide (CYP) use in these cases.
Methods Retrospective casenote review of 12 JDM patients treated with CYP over 1998-2002.Clinical parameters used included the Childhood Myositis Assessment Scale (CMAS), muscle strength score (five muscle groups), vasculitic score (score from each system involved), skin score, muscle enzymes (CK, LDH), inflammatory marker (ESR), full blood count (FBC), and steroid dose.
Results The cohort consisted of equal sex ratio, 3 Afro-Caribbean, 9 Caucasian, mean age at diagnosis 6.4 years (range 2-10 years), mean time from diagnosis to CYP treatment 1.2 years (0.2-3.8 years). Previous drugs included steroids, methotrexate, cyclosporin, azathioprine and immunoglobulin.
Patients were given 6 boluses of IV CYP at monthly intervals. They were then reviewed on an individual basis as to further monthly to 3 monthly doses.
Two patients were given CYP whilst ventilated in PICU but subsequently died. At 6 months,10 patients showed a significant improvement in the following -CMAS(p=0.01), muscle strength (p=0.01), vasculitic score (p=0.01), skin (p=0.01), LDH (p=0.03).CK(p=0.09) and prednisolone dose (p=0.08) were reduced, but calcinosis was unchanged. ESR did not change significantly.
The clinical improvement was maintained post CYP.
The mean cumulative dose was 5.88mg/m(3-9 mg/m). Mean number of doses was 8.5 (6-14). Significant side effects included lymphopaenia which resolved after completion of CYP treatment. One patient developed febrile neutropaenia, 3 herpes zoster infections, 4 suffered alopecia and 3 low grade infections.
Conclusion CYP was effective in treating severe and resistant JDM. In our experience there were no serious side effects.