Objective: To investigate the safety and tolerability of high-dose cyclophosphamide without stem cell rescue in scleroderma.
Methods: An open-label, single-site, uncontrolled study design entered patients with active diffuse cutaneous scleroderma. Patients were treated with cyclophosphamide (50 mg/kg) intravenously daily for 4 consecutive days (total 200 mg/kg) followed by granulocyte colony-stimulating factor (5 µg/kg/day). The primary clinical efficacy end point was the modified Rodnan skin score (mRSS). Secondary end points included the Health Assessment Questionnaire-Disability Index (HAQ-DI), physician global assessment (PGA) and pulmonary function tests.
Results: Six patients (4 men, 2 women) aged 19–60 years were entered into the study. One patients died early in the protocol, thus five patients had follow-up data. The percentage reduction of the mRSS in these five evaluable patients within 1 month of treatment was 60%, 55%, 41%, 31% and 0%. The patient with no decline in skin score at 1 month showed a decrease in skin score from 41 to 26 by the 3-month visit, a 37% improvement. Three patients sustained the improvement after treatment for 24, 12 and 12 months. Two patients relapsed at 12 and 6 months after treatment. The PGA and HAQ-DI scores improved in five of the six patients by 72% and 79% respectively at 3 months. The only serious adverse event was a death that occurred owing to infection after neutrophil count recovery.
Conclusions: High-dose cyclophosphamide without stem cell rescue can lead to a clinically significant improvement in skin score and measures of disease severity in patients with diffuse cutaneous scleroderma.
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Competing interests: Under a licensing agreement between Revimmune, Inc and the Johns Hopkins University, RAB is entitled to a share of royalty received by the university on sales of products described in this (article). The terms of this arrangement are being managed by the Johns Hopkins University in accordance with its conflict of interest policies. The other authors have no conflict of interest.
Ethics approval: Approved by the Institution Review Board of Johns Hopkins University and Clinical Research Review Committee Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins.
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