High-dose Cyclophosphamide is Effective Therapy for Pediatric Severe Aplastic Anemia.

ABSTRACT:

Objective

Use of high-dose cyclophosphamide without hematopoietic stem cell transplant to treat severe aplastic anemia (SAA) has been controversial due to concern for increased infectious toxicity as compared with antithymocyte globulin and cyclosporine A. As children often tolerate dose-intensive therapy better than adults, we sought to perform a detailed retrospective analysis of both treatment response and toxicity in 28 patients younger than 22 years of age treated with 29 courses of high-dose cyclophosphamide as the sole form of immunosuppression.

Study design

Children and adolescents with SAA who lacked an human leukocyte antigen-matched sibling donor were treated with cyclophosphamide 50 mg/kg/d for 4 consecutive days then received daily granulocyte colony stimulating

SUBMITTER: Gamper CJ 

PROVIDER: S-EPMC5074865 | biostudies-literature | 2016 Nov

REPOSITORIES: biostudies-literature