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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

15 participants

Study Classification

INTERVENTIONAL

Study Start Date

2008-04-30

Study Completion Date

2013-12-31

Brief Summary

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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.

Detailed Description

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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.

Conditions

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Leukemia Myelodysplastic Syndromes Nonmalignant Neoplasm Paroxysmal Nocturnal Hemoglobinuria

Keywords

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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

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Interventions

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nonmyeloablative allogeneic hematopoietic stem cell transplantation

infusion of mobilized donor hematopoietic progenitor cells

Intervention Type PROCEDURE

Eligibility Criteria

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Inclusion Criteria

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

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Responsible Party

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Kyoo-Hyung Lee

Professor of Internal Medicine

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Kyoo H. Lee, MD

Role: PRINCIPAL_INVESTIGATOR

Asan Medical Center

Locations

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Asan Medical Center - University of Ulsan College of Medicine

Seoul, , South Korea

Site Status

Countries

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South Korea

Other Identifiers

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AMC-UUCM-2008-0038

Identifier Type: -

Identifier Source: secondary_id

CDR0000600351