Moderate-dose Cyclophosphamide for Childhood Acquired Aplastic Anemia

Xiaofan Zhu

Status and phase: Unknown; Phase 4

Conditions: Aplastic Anemia

Treatments: Drug: cyclophosphamide,cyclosporine A

Study type: Interventional

Funder types: Other

Identifiers

NCT01995331

YL20102601

Details and patient eligibility

About

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.

Full 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.

Enrollment: 30 estimated patients

Sex: All

Ages: 1 to 18 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Acquired Childhood Severe Aplastic Anemia (SAA)

Exclusion criteria

• not Childhood and Acquired Severe Aplastic Anemia

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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30 participants in 1 patient group

moderate-dose cyclophosphomide

Other group

Treatment:

Drug: cyclophosphamide,cyclosporine A