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Deprecated: Implicit conversion from float 229.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 J+Pediatr+Hematol+Oncol 2016 ; 38 (8): 627-35 Nephropedia Template TP
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High-Dose Cyclophosphamide is Effective Therapy for Pediatric Severe Aplastic Anemia #MMPMID27467367
Gamper CJ; Takemoto CM; Chen AR; Symons HJ; Loeb DM; Casella JF; Dezern AE; King KE; McGonigle AM; Jones RJ; Brodsky RA
J Pediatr Hematol Oncol 2016[Nov]; 38 (8): 627-35 PMID27467367show ga
Objective: Use of high-dose cyclophosphamide without hematopoietic stem cell transplant (HSCT) to treat severe aplastic anemia (SAA) has been controversial due to concern for increased infectious toxicity as compared to anti thymocyte globulin (ATG) and cyclosporine (CSA). 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 less 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 HLA-matched sibling donor were treated with cyclophosphamide 50 mg/kg/day for 4 consecutive days then received daily G-CSF until neutrophil recovery, transfusion support, and antimicrobial prophylaxis. Results: Overall survival was 85%, with hematologic response of 79% and complete response of 66%. Cumulative incidences of bacterial infection (86%) and fungal infection (62%) were high but deaths due to infection were rare, as were clonal evolution (1/28), clinically relevant PNH (1/28), and relapse (2/28). Conclusion: Response rates and survival following high-dose cyclophosphamide in pediatric patients with SAA exceed those seen in adults and compare favorably to ATG/CSA with manageable infectious toxicity.