Moderate-dose Cyclophosphamide for Childhood Acquired Aplastic Anemia

Phase 4

• Conditions: Aplastic Anemia
• Interventions: Drug: cyclophosphamide,cyclosporine A

• Registration Number: NCT01995331
• Lead Sponsor: Xiaofan Zhu

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 Description

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.

Study Timeline

• Study Start: 2012-03
• Status Verified: 2013-11
• Study First Submitted: 2013-11-21
• Study First Submitted QC: 2013-11-21
• Last Update Submitted: 2013-11-21
• Study First Posted: 2013-11-26
• Last Update Posted: 2013-11-26
• Primary Completion: 2015-03
• Study Completion: 2015-03

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 30

Inclusion Criteria

• Acquired Childhood Severe Aplastic Anemia (SAA)

Exclusion Criteria

• not Childhood and Acquired Severe Aplastic Anemia

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
moderate-dose cyclophosphomide	cyclophosphamide,cyclosporine A	-

Primary Outcome Measures

Name	Time	Method
The reponse of Moderate-dose Cyclophosphamide for Childhood Acquired Aplastic Anemia	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).

Secondary Outcome Measures

Name	Time	Method
The side effect of Moderate-dose Cyclophosphamide for Childhood Acquired Aplastic Anemia	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.

Trial Locations

Locations (1)

Department of Pediatrics,Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College; 🇨🇳 Tianjin, Tianjin, China