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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:

Completed

Funding Sources:

Supported by the Juvenile Dermatomyositis Research Centre and grants from the Cathal Hayes Research Foundation.

Publications:

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 ]

Contact:

Clarissa Pilkington, M.B.B.S.
Juvenile Dermatomyositis Research Centre
Institute of Child Health
University College of London
London, UK
clarissa.pilkington@gosh.nhs.uk
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