Clinical Trial: Moderate-dose Cyclophosphamide for Childhood Acquired Aplastic Anemia

Study Status: Withdrawn
Recruit Status: Unknown status
Study Type: Interventional

Official Title: Moderate-dose Cyclophosphamide for Childhood Acquired Aplastic Anemia

Brief Summary: Severe aplastic anemia (SAA)is characterized by the depletion of hematopoietic precursors associated with life-threatening complications. High-dose cyclophosphamide has been found to yield a complete response (CR) in adults and children with SAA.However, the optimal dosage of cyclophosphamide for patients in childhood remains unclear. So we explore the ideal dosage of cyclophosphamide for the treatment of children with SAA.

Detailed Summary: Tisdale et al. (2000,2002) attempted to compare immunosuppression using ATG/CSA with high-dose cyclophosphamide (50 mg/kg/d for 4 consecutive days) plus CSA in a randomized trial of newly diagnosed adults with SAA. Both groups received CSA as part of the treatment regimen. However, the trial was terminated prematurely due to excessive morbidity among the patients treated in the cyclophosphamide arm. They documented that invasive fungal infections were severe among the cyclophosphamide group. Between January 2008 through May 2009, in our department, nine pediatric patients with a diagnosis of SAA were enrolled a study with lower dose of cyclophosphamide with 30mg/kg/day for 4 consecutive days and combination with CSA, this study shows promise for children with severe aplastic anemia.Now we want explore the dosage of cyclophosphamide.
Sponsor: Xiaofan Zhu

Current Primary Outcome: The reponse of Moderate-dose Cyclophosphamide for Childhood Acquired Aplastic Anemia [ Time Frame: 36 months ]

Complete response (CR) was defined as achieving normal levels of hemoglobin adjusted for age, platelet count >100×109/L, and ANC>1.5×109/L. Partial response (PR) was defined as transfusion independence, reticulocyte count >30×109/L, platelet count >30×109/L, and ANC >0.5×109/L above the baseline. Persistence of transfusion requirement or death was evidence of no response (NR).


Original Primary Outcome: Same as current

Current Secondary Outcome: The side effect of Moderate-dose Cyclophosphamide for Childhood Acquired Aplastic Anemia [ Time Frame: 36 months ]

To report the death rate and the death cause , the infection rate and the pathogenic bacteria, especialy the fungal infection rate.

To report other toxicity.



Original Secondary Outcome: Same as current

Information By: Chinese Academy of Medical Sciences

Dates:
Date Received: November 21, 2013
Date Started: March 2012
Date Completion: March 2015
Last Updated: November 21, 2013
Last Verified: November 2013