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Intravenous Pulse Cyclophosphamide Therapy in Treatment-Refractory Juvenile Dermatomyositis

Principal Investigator:

Clarissa Pilkington, M.B.B.S.

Study Hypothesis:

Severe clinical features, including severe muscle weakness, cutaneous or gastrointestinal ulcerations, interstitial lung disease and convulsions, would respond to intravenous pulse cyclophosphamide therapy.

Study Design:

Open label, retrospective

Types & Number of IIM Studied:

8 patients with juvenile dermatomyositis

Entry Criteria:

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

Primary Outcome:

Examined improvement in core set measures at 6 month and last follow-up evaluation.

Secondary Outcome:

Adverse event assessment

Key Trial Dates:


Funding Sources:

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
London, UK
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