Comparison of Cy-Atg vs Flu-Atg for the Conditioning Therapy in Allo-HCT for Adult Aplastic Anemia

• Status: Unknown status
• Trial end date: 2016-02-01
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study is to reduce the regimen related toxicities and transplantation related mortality after allogeneic stem cell transplantation in adult acquired aplastic anemia (AA), the trials of reduced dose of Cy along with fludarabine and ATG were performed.11-21 The investigators preliminary data of randomized comparison of cyclophosphamide plus fludarabine versus cyclophosphamide alone in addition to anti-thymocyte globulin for the conditioning therapy in allogeneic hematopoietic cell transplantation for bone marrow failure syndrome supports reduced dose of Cy along with fludarabine and ATG.

Conditioning regimen without Cy may reduce RRT because Cy-containing conditioning remains several RRT such as hemorrhagic cystitis, SOS and graft versus host disease (GvHD). Recently there were small trials of fludarabine and ATG (Flu-ATG) for the conditioning regimen of alloHSCT.22-24 These data raised the feasibility of fludarabine and ATG without Cy for patients with AA.

This new conditioning regimen of Flu-ATG will be compared to standard regimen of Cy- ATG in a randomized controlled trial.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Cooperative Study Group A for Hematology
• Treatments: Antilymphocyte Serum; Cyclophosphamide; Fludarabine; Fludarabine phosphate; Thymoglobulin; Vidarabine

Criteria

Inclusion Criteria:

• Severe aplastic anemia

• Severe aplastic anemia (SAA) is defined as a hypocellular bone marrow (cellularity<25%) and two or more of the following: granulocyte count <500/ml, platelet count <20,000/ml, and corrected reticulocyte count <1.0%

• Very severe aplastic anemia (VSAA) is defined as the criteria for SAA plus a granulocyte count <200/ml

• Patients should be 15 years of age or older, but younger than 65 years.

• The performance status of the patients should be 70 or over by Karnofsky performance scale (see Appendix I).

• Patients must have adequate hepatic function (bilirubin less than 2 mg/dl, AST and ALT less than three times the upper normal limit)

• Patients must have adequate renal function (creatinine less than 2.0 mg/dl).

• Patients must have adequate cardiac function (ejection fraction > 45% on echocardiogram).

Exclusion criteria:

• Patients should not have major illness or organ failure.

• Patients must not have a psychiatric disorder or mental deficiency severe as to make compliance with the treatment unlikely, and making informed consent impossible.

• Patients must not be in pregnancy.

• Hypoplastic myelodysplastic syndrome

• Paroxysmal nocturnal hemoglobinuria