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Human Leukocyte Antigen (HLA)-Haploidentical Hematopoietic Stem Cell Transplantation for Patients With Aplastic Anemia

Information source: Asan Medical Center
ClinicalTrials.gov processed this data on August 20, 2015
Link to the current ClinicalTrials.gov record.

Condition(s) targeted: Aplastic Anemia

Intervention: anti-thymocyte globulin (Biological); filgrastim (Biological); Fludarabine (Drug); Cyclophosphamide (Drug); CD3┬▒CD19 depleted hematopoietic stem cell transplantation (Procedure)

Phase: Phase 1/Phase 2

Status: Completed

Sponsored by: Asan Medical Center

Official(s) and/or principal investigator(s):
Ho Joon Im, MD & PhD, Principal Investigator, Affiliation: Asan Medical Center


Rationale: Chemotherapy with fludarabine, cyclophosphamide and anti-thymocyte globulin may induce the engraftment cross the immunologic barrier in the setting of HLA-haploidentical allogeneic hematopoietic cell transplantation. In addition, depletion CD3▒CD19 cells may contribute to prevent developing severe acute graft versus host disease (GVHD) in haploidentical transplantation. Purpose: This phase I/II trial is to evaluate the safety and efficacy of fludarabine, cyclophosphamide and antithymocyte globulin with CD3▒CD19 depleted graft from haploidentical donors in treating patients with aplastic anemia.

Clinical Details

Official title: HLA-haploidentical Allogeneic Hematopoietic Cell Transplantation Using CD3▒CD19 Depletion for Patients With Aplastic Anemia After Conditioning of Fludarabine, Cyclophosphamide and Antithymocyte Globulin

Study design: Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Primary outcome: To assess the engraftment rate and survival of CD3┬▒CD19 depleted haploidentical peripheral blood stem cell transplantation after conditioning with fludarabine, cyclophosphamide and anti-thymocyte globulin.

Secondary outcome:

To assess engraftment and graft failure

To estimate the risk of acute GVHD

To assess treatment related mortality

To estimate overall survival


Minimum age: N/A. Maximum age: 21 Years. Gender(s): Both.


Inclusion Criteria:

- Diagnosis of life-threatening marrow failure (severe aplastic anemia) of nonmalignant

etiology meeting 2 of the following criteria:

- Granulocyte count < 500/mm3,

- Corrected reticulocyte count < 1%,

- Platelet count < 20,000/mm3

- No HLA-identical family member or closely matched (8 of 8 HLA-locus match) unrelated

marrow donor available

- HLA-haploidentical related donor available

Exclusion Criteria:

- Paroxysmal nocturnal hemoglobinuria or Fanconi anemia

- Clonal cytogenetic abnormalities or myelodysplastic syndromes

- Active fungal infections

- HIV positive

- Severe disease other than aplastic anemia that would severely limit the probability

of survival during the graft procedure

- Pregnant or nursing

Locations and Contacts

Asan Medical Center, Seoul 138-736, Korea, Republic of
Additional Information


Related publications:

Woodard P, Cunningham JM, Benaim E, Chen X, Hale G, Horwitz E, Houston J, Kasow K, Leung W, Wang W, Yusuf U, Handgretinger R. Effective donor lymphohematopoietic reconstitution after haploidentical CD34+-selected hematopoietic stem cell transplantation in children with refractory severe aplastic anemia. Bone Marrow Transplant. 2004 Feb;33(4):411-8.

Kremens B, Basu O, Grosse-Wilde H, Sauerwein W, Schaefer UW, Havers W. Transplantation of CD34-enriched peripheral stem cells from an HLA-haplotype mismatched donor to a patient with severe aplastic anemia. Bone Marrow Transplant. 2001 Jan;27(1):111-3.

Lacerda JF, Martins C, Carmo JA, Louren├žo F, Juncal C, Ismail S, Lacerda JM. Haploidentical stem cell transplantation with purified CD34+ cells after a chemotherapy-alone conditioning regimen in heavily transfused severe aplastic anemia. Biol Blood Marrow Transplant. 2005 May;11(5):399-400.

Klingebiel T, Cornish J, Labopin M, Locatelli F, Darbyshire P, Handgretinger R, Balduzzi A, Owoc-Lempach J, Fagioli F, Or R, Peters C, Aversa F, Polge E, Dini G, Rocha V; Pediatric Diseases and Acute Leukemia Working Parties of the European Group for Blood and Marrow Transplantation (EBMT). Results and factors influencing outcome after fully haploidentical hematopoietic stem cell transplantation in children with very high-risk acute lymphoblastic leukemia: impact of center size: an analysis on behalf of the Acute Leukemia and Pediatric Disease Working Parties of the European Blood and Marrow Transplant group. Blood. 2010 Apr 29;115(17):3437-46. doi: 10.1182/blood-2009-03-207001. Epub 2009 Dec 29.

Starting date: July 2009
Last updated: December 30, 2012

Page last updated: August 20, 2015

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