Clarissa Pilkington, M.B.B.S.
Severe clinical features, including severe muscle weakness, cutaneous or gastrointestinal ulcerations, interstitial lung disease and convulsions, would respond to intravenous pulse cyclophosphamide therapy.
Open label, retrospective
Types & Number of IIM Studied:
8 patients with juvenile dermatomyositis
Severe or treatment resistant juvenile dermatomyositis despite treatment with high dose oral corticosteroids and at least 1 DMARD therapy or IVIG. Severe manifestations included cutaneous or gastrointestinal ulceration, or interstitial lung disease.
IMACS Core Set Measures Included:
Physician Global Activity-VAS, Patient Global Activity-VAS, Muscle Strength Testing-MMT, Functional Assessment Tools-CMAS, CHAQ, Laboratory-Muscle Enzymes, Extramuscular Assessment-Extramuscular Global Activity, MD Assessment of Outcome
Examined improvement in core set measures at 6 month and last follow-up evaluation.
Adverse event assessment
Key Trial Dates:
Supported by the Juvenile Dermatomyositis Research Centre and grants from the Cathal Hayes Research Foundation.
Riley P, Maillard SM, Wedderburn LR, Woo P, Murray KJ, Pilkington CA. Intravenous cyclophosphamide pulse therapy in juvenile dermatomyositis. A review of efficacy and safety., Rheumatology(Oxford), 2004 Apr;43(4):491-6. [Abstract]
- Clarissa Pilkington, M.B.B.S.
Juvenile Dermatomyositis Research Centre
Institute of Child Health
University College of London