HLA-HAPLOIDENTICAL FAMILIAL DONOR HEMATOPOIETIC CELL TRANSPLANTATION AFTER REDUCED INTENSITY CONDITIONING OF BUSULFAN, FLUDARABINE, AND ANTI-THYMOCYTE GLOBULIN FOR PATIENTS WITH BONE MARROW FAILURE SYNDROME - A PHASE 2 STUDY
N/A
75 Years
Both
Leukemia, Myelodysplastic Syndromes, Nonmalignant Neoplasm, Paroxysmal Nocturnal Hemoglobinuria
Trial Information
HLA-HAPLOIDENTICAL FAMILIAL DONOR HEMATOPOIETIC CELL TRANSPLANTATION AFTER REDUCED INTENSITY CONDITIONING OF BUSULFAN, FLUDARABINE, AND ANTI-THYMOCYTE GLOBULIN FOR PATIENTS WITH BONE MARROW FAILURE SYNDROME - A PHASE 2 STUDY
OBJECTIVES:
- To evaluate the efficacy of HLA-haploidentical familial donor hematopoietic stem cell
transplantation after reduced-intensity conditioning regimen comprising busulfan,
fludarabine phosphate, and anti-thymocyte globulin in patients with bone marrow failure
syndromes.
OUTLINE:
- Reduced-intensity conditioning regimen: Patients receive busulfan IV daily on days -7
and -6, fludarabine phosphate IV over 30 minutes on days -7 to -2, anti-thymocyte
globulin (ATG) IV over 4 hours on days -4 to -1, and methylprednisolone IV over 30
minutes starting 30 minutes before ATG on days -4 to -1.
- HLA-haploidentical donor hematopoietic stem cell transplantation: Patients receive
donor hematopoietic stem cells via Hickman catheter over 1 hour on days 0 or 1.
- Graft-versus-host-disease prophylaxis (GVHD): Patients receive cyclosporine IV over 2-4
hours every 12 hours starting on day -1 (cyclosporine can be given orally once oral
medication can be tolerated) and methotrexate IV on days 2, 4 , 7, and 12. In the
absence of GVHD, cyclosporine is tapered starting between days 30 to 60.
After completion of study treatment, patients are followed periodically for 1 year.
Inclusion Criteria
DISEASE CHARACTERISTICS:
- Diagnosis of any of the following bone marrow failure syndromes:
- Severe aplastic anemia, meeting 1 of the following criteria:
- Not responsive to immunosuppressive therapy
- With recurrent cytopenia after immunosuppressive therapy or allogeneic
hematopoietic cell transplantation
- Low-risk myelodysplastic syndrome, including any of the following:
- Refractory anemia
- Refractory anemia with ringed sideroblasts
- Refractory cytopenia with multi-lineage dysplasia
- Paroxysmal nocturnal hemoglobinuria, meeting 1 of the following criteria:
- With thrombotic episodes
- With severe cytopenia
- No willing, suitable HLA-compatible donor in family or in donor registries
- Related donor with HLA-haploidentical mismatch at three or less of 6 loci
- Patients with very severe neutropenia (< 200/μL) or febrile episodes, who feel
urgent need for allogeneic hematopoietic cell transplantation, are eligible
without a search for HLA-matched unrelated donors
PATIENT CHARACTERISTICS:
- Karnofsky performance status 70-100%
- Bilirubin < 2.0 mg/dL
- AST < 3 times upper limit of normal
- Creatinine < 2.0 mg/dL
- Ejection fraction > 40% by MUGA scan
PRIOR CONCURRENT THERAPY:
- See Disease Characteristics
Type of Study:
Interventional
Study Design:
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Outcome Measure:
Donor cell engraftment
Outcome Description:
neutrophil count over 500/ul
Outcome Time Frame:
10-35 days after transplantation
Safety Issue:
No
Principal Investigator
Kyoo H. Lee, MD
Investigator Role:
Principal Investigator
Investigator Affiliation:
Asan Medical Center
Authority:
Korea: Food and Drug Administration
Study ID:
CDR0000600351
NCT ID:
NCT00731328
Start Date:
April 2008
Completion Date:
March 2017
Related Keywords:
- Leukemia
- Myelodysplastic Syndromes
- Nonmalignant Neoplasm
- Paroxysmal Nocturnal Hemoglobinuria
- refractory anemia
- refractory anemia with ringed sideroblasts
- refractory cytopenia with multilineage dysplasia
- childhood myelodysplastic syndromes
- de novo myelodysplastic syndromes
- previously treated myelodysplastic syndromes
- secondary myelodysplastic syndromes
- aplastic anemia
- paroxysmal nocturnal hemoglobinuria
- Neoplasms
- Hemoglobinuria
- Leukemia
- Hemoglobinuria, Paroxysmal
- Myelodysplastic Syndromes
- Preleukemia
- Pancytopenia
Name | Location |
|---|
