Immunoablative high-dose cyclophosphamide without stem-cell rescue for refractory, severe autoimmune disease
R A Brodsky1, M Petri, B D Smith
1Johns Hopkins University School of Medicine, Baltimore, Maryland 21205, USA.
Annals of Internal Medicine
|December 29, 1998
Summary
High-dose cyclophosphamide without stem-cell rescue effectively treats severe autoimmune diseases. This immunoablative therapy induced complete remission in most patients, offering a safer alternative to transplantation.
Area of Science:
- Immunology
- Hematology
- Rheumatology
Background:
- High-dose cyclophosphamide without stem-cell rescue has shown efficacy in aplastic anemia.
- This study investigates its use in other severe autoimmune conditions.
Observation:
- A prospective phase II study was conducted at Johns Hopkins University and Hahnemann University.
- Eight patients with refractory, severe autoimmune diseases received treatment.
Findings:
- Seven out of eight patients showed significant improvement, with five achieving complete remission and two partial remission.
- Remission was durable, lasting up to 21 months in some cases.
- The therapy was well-tolerated, with rapid recovery of neutrophil and platelet counts.
Implications:
- Immunoablative high-dose cyclophosphamide is a viable treatment for refractory autoimmune diseases.
- It offers an alternative to stem-cell transplantation, avoiding risks associated with autograft reinfusion.
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