Moderate-dose Cyclophosphamide for Childhood Acquired Aplastic Anemia

NCT ID: NCT01995331

Last Updated: 2013-11-26

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE4
• Total Enrollment: 30 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2012-03-31
• Study Completion Date: 2015-03-31

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.

Conditions

Aplastic Anemia

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

moderate-dose cyclophosphomide

Group Type: OTHER

cyclophosphamide,cyclosporine A

Intervention Type: DRUG

Drug,cyclophosphamide,cyclophosphamide (30 mg/kg/day) administered intravenously (IV) over 1 hr for 4 consecutive days Drug,cyclosporine A,5mg-12mg/kg.d,CSA was administered orally 40 days after the fourth dose of cyclophosphamide and maintained for 3 years. The dose of CSA was adjusted to maintain trough drug concentration above 150 μg/L and peak drug concentration above 300 μg/L.

Drug, human granulocyte colony-stimulating factor (rhG-CSF), 5 μg/kg/day subcutaneously starting 24 hrs after the fourth dose of cyclophosphamide, and it was withdrawed when ANC was >1×109/L.

Interventions

cyclophosphamide,cyclosporine A

Drug,cyclophosphamide,cyclophosphamide (30 mg/kg/day) administered intravenously (IV) over 1 hr for 4 consecutive days Drug,cyclosporine A,5mg-12mg/kg.d,CSA was administered orally 40 days after the fourth dose of cyclophosphamide and maintained for 3 years. The dose of CSA was adjusted to maintain trough drug concentration above 150 μg/L and peak drug concentration above 300 μg/L.

Drug, human granulocyte colony-stimulating factor (rhG-CSF), 5 μg/kg/day subcutaneously starting 24 hrs after the fourth dose of cyclophosphamide, and it was withdrawed when ANC was >1×109/L.

Intervention Type: DRUG

Other Intervention Names

Drug,Mesnaum

Eligibility Criteria

Inclusion Criteria

• Acquired Childhood Severe Aplastic Anemia (SAA)

Exclusion Criteria

• not Childhood and Acquired Severe Aplastic Anemia

• Minimum Eligible Age: 1 Year
• Maximum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Xiaofan Zhu

UNKNOWN

Sponsor Role: lead

Responsible Party

Xiaofan Zhu

chief physician

Responsibility Role: SPONSOR_INVESTIGATOR

Principal Investigators

Xiaifan Zhu, MD

Role: PRINCIPAL_INVESTIGATOR

Department of Pediatrics, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences

Locations

Department of Pediatrics,Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College

Tianjin, Tianjin Municipality, China

Countries

China

Other Identifiers

YL20102601

Identifier Type: -

Identifier Source: org_study_id