Flu+CPM+rATG Conditioning Regimes for Unrelated Bone Marrow Transplantation (UBMT)(or Mobilized Peripheral Blood)in Severe Aplastic Anemia (SAA)

Fludarabine, Cyclophosphamide Plus Thymoglobulin Conditioning Regimen for Unrelated Bone Marrow (or Mobilized Peripheral Blood) Transplantation in Severe Aplastic Anemia


Lead Sponsor: The Korean Society of Pediatric Hematology Oncology

Source The Korean Society of Pediatric Hematology Oncology
Brief Summary

Anti-thymocyte globulin (ATG) has been used in severe aplastic anemia as a part of the conditioning regimen. Among the many kinds of ATG preparations, thymoglobulin had been found to be more effective in preventing GVHD and rejection of organ transplants. As the fludarabine based conditioning regimens without total body irradiation have been reported to be promising for BMT/PBSCT from alternative donors in SAA, thymoglobulin was added to fludarabine and cyclophosphamide conditioning to reduce GVHD and to allow good engraftment in UBMT/UPBSCT.

Detailed Description

GVHD prophylaxis recommendation tacrolimus (0.03 mg/kg/day i.v. by continuous infusion from day -2 and taper with an oral form until 1 year after BMT/PBSCT) methotrexate (15 mg/m2 i.v. on days 1 and 10 mg/m2 i.v. on days 3, 6, 11)

Overall Status Unknown status
Start Date January 2006
Completion Date December 2012
Primary Completion Date August 2012
Phase Phase 2
Study Type Interventional
Primary Outcome
Measure Time Frame
To evaluate the engraftment potential, incidence and severity of acute graft versus host disease,toxicity of conditioning regimen for UBMT in SAA. From Jan. 1. 2006 to Dec. 31. 2008. For 3 years.
To evaluate overall and EFS follow-up of 1 year after UBMT/PBSCT. From Jan. 1. 2006 to Dec. 31. 2008. For 3 years
Secondary Outcome
Measure Time Frame
To evaluate chronic GVHD and immunologic recovery after UBMT/PBSCT. and the efficacy of UBMT/PBSCT before immuno-suppressive therapy with anti-thymocyte globulin in severe aplastic anemia and long term toxicity of non-TBI based conditioning From Jan. 1. 2006 to Dec. 31. 2008. For 3 years.
Enrollment 30

Intervention Type: Drug

Intervention Name: cyclophosphamide, fludarabine , thymoglobulin

Description: cyclophosphamide (50 mg/kg once daily i.v. on days -9, -8, -7 & -6) fludarabine (30 mg/m2 once daily i.v. on days -5, -4, -3 & -2) thymoglobulin (2.5 mg/kg once daily i.v. on days -3, -2 & -1)

Arm Group Label: Fludarabine



Inclusion Criteria:

- Diagnosis of severe aplastic anemia defined by any two or three peripheral blood criteria

- and either marrow criterion.

- Peripheral blood

1. Neutrophils < 0.5 x 109/l

2. Platelets < 20 x 109/l

3. Corrected reticulocytes < 1%

- Bone marrow

1. Severe hypocellularity (< 25%)

2. Moderate hypocellularity (25-30%) with hematopoietic cells representing < 30% of residual cells

- No prior hematopoietic stem cell transplantation.

- Age: no limits.

- Performance status: ECOG 0-2.

- Patients must be free of significant functional deficits in major organs, but the following eligibility criteria may be modified in individual cases.

1. Heart: a shortening fraction > 30%, ejection fraction > 45%.

2. Liver: total bilirubin < 2 × upper limit of normal; ALT < 3 × upper limit of normal.

3. Kidney: creatinine <2 × normal or a creatinine clearance (GFR) > 60 ml/min/1.73m2.

- Patients must lack any active viral infections or active fungal infection.

- Appropriate donor is available: Matched in 6/6 of A, B, DR loci.

- Patients (or one of parents if patients age < 19) should sign informed consent.

Exclusion Criteria:

- Pregnant or nursing women.

- Malignant or nonmalignant illness that is uncontrolled or whose control may be jeopardized by complications of study therapy.

- Psychiatric disorder that would preclude compliance.

- Congenital aplastic anemia including Fanconi anemia.

- Manipulated bone marrow.

Gender: All

Minimum Age: N/A

Maximum Age: 25 Years

Healthy Volunteers: No

Overall Official
Last Name Role Affiliation
Hyo Seop Ahn, M.D, Ph.D Principal Investigator The Korean Society of Pediatric Hematology Oncology
Facility: Seoul National University Hospital
Location Countries

Korea, Republic of

Verification Date

March 2012

Responsible Party

Name Title: Hyo Seop Ahn

Organization: Seoul Natioanl University Hospital

Has Expanded Access No
Condition Browse
Number Of Arms 1
Arm Group

Label: Fludarabine

Type: Experimental

Study Design Info

Allocation: Non-Randomized

Intervention Model: Single Group Assignment

Primary Purpose: Treatment

Masking: None (Open Label)

Source: ClinicalTrials.gov