Donor Stem Cell Transplant After Busulfan, Fludarabine, Methylprednisolone, and Antithymocyte Globulin in Treating Patients With Bone Marrow Failure Syndrome

NCT00731328 | Completed Phase 2

• 2 other identifiers: CDR0000600351AMC-UUCM-2008-0038
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief Summary

RATIONALE: Giving low doses of chemotherapy and antithymocyte globulin before a donor stem cell transplant helps stop the growth of abnormal cells. It may also stop the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune cells and help destroy any remaining abnormal cells (graft-versus-tumor effect). PURPOSE: This phase II trial is studying how well a donor stem cell transplant works after busulfan, fludarabine, methylprednisolone, and antithymocyte globulin in treating patients with bone marrow failure syndrome.

Conditions

Leukemia; Myelodysplastic Syndromes; Nonmalignant Neoplasm; Paroxysmal Nocturnal Hemoglobinuria

Keywords

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

Outcome Measures

Primary Outcomes (1)

• Donor cell engraftment

  neutrophil count over 500/ul

  10-35 days after transplantation

Secondary Outcomes (4)

• Regimen-related toxicities as assessed by NCI's Common Toxicity Criteria

  0-60 months after transplantation

• Acute and chronic GVHD

  15-100 days; 100 days to 4 years

• overall survival

  0-60 months

• event-free survival

  0-60 months after transplatation

Interventions

nonmyeloablative allogeneic hematopoietic stem cell transplantation PROCEDURE

infusion of mobilized donor hematopoietic progenitor cells

Eligibility Criteria

• Age: Up to 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

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

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• Asan Medical Center: lead

Study Sites (1)

Asan Medical Center - University of Ulsan College of Medicine

Seoul, 138-736, South Korea

Location

MeSH Terms

Conditions

Leukemia; Myelodysplastic Syndromes; Neoplasms; Hemoglobinuria, Paroxysmal; Anemia, Refractory; Anemia, Aplastic

Condition Hierarchy (Ancestors)

Neoplasms by Histologic Type; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Diseases; Anemia, Hemolytic; Anemia; Bone Marrow Failure Disorders

Study Officials

• Kyoo H. Lee, MD

  Asan Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor of Internal Medicine

Study Record Dates

• First Submitted: August 8, 2008
• First Posted: August 11, 2008
• Study Start: April 1, 2008
• Primary Completion: December 1, 2013
• Study Completion: December 1, 2013
• Last Updated: December 31, 2015

Record last verified: 2015-12

Locations

KR (1)