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Phase II Study of Bone Marrow Transplantation Using Related Donors in Patients With Aplastic Anemia

This study has been completed.
University of California, Los Angeles
Information provided by:
National Center for Research Resources (NCRR) Identifier:
First received: October 18, 1999
Last updated: June 23, 2005
Last verified: April 2002

OBJECTIVES: I. Evaluate the efficacy of related, HLA-identical bone marrow transplantation following cyclophosphamide (CTX) and antithymocyte globulin in patients with aplastic anemia.

II. Evaluate the efficacy of related, HLA-nonidentical bone marrow transplantation following CTX and total-body irradiation/total-lymphoid irradiation in patients with aplastic anemia.

Condition Intervention Phase
Aplastic Anemia
Drug: anti-thymocyte globulin
Drug: cyclophosphamide
Drug: cyclosporine
Drug: mesna
Drug: methotrexate
Drug: methylprednisolone
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Efficacy Study
Primary Purpose: Treatment

Resource links provided by NLM:

Further study details as provided by National Center for Research Resources (NCRR):

Estimated Enrollment: 10
Study Start Date: February 1995
Detailed Description:

PROTOCOL OUTLINE: Patients with HLA-identical donors receive cyclophosphamide/mesna, antithymocyte globulin (ATG), and methylprednisolone (MePRDL), followed by allogeneic marrow transplantation.

Patients with HLA-nonidentical (5/6 or 4/6) donors and those with a systemic reaction to ATG receive the same dosage of myeloablative chemotherapy and rescue. In addition, these patients are treated with total-body irradiation, total-lymphoid irradiation, and methotrexate.

All patients received MePRDL and cyclosporine for graft-versus-host prophylaxis.


Ages Eligible for Study:   up to 55 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No


--Disease Characteristics-- Severe aplastic anemia with 2 of the following criteria: Polymorphonuclear neutrophils no more than 0.5 x 10 to the ninth/L Platelets no more than 20 x 10 to the ninth/L Reticulocytes no more than 30 x 10 to the ninth/L Hypocellular bone marrow Related donor aged 6 weeks or more, matching at least 4 of 6 HLA A, B, and DRI loci, as follows: HLA-identical family member for patients aged less than 40 years HLA-nonidentical family member for patients aged less than 55 years failing antithymocyte globulin or other immunosuppressive therapy The following eligible for transplantation as initial therapy: Patients aged less than 40 years with HLA-matched donor Patients aged 40-45 years with HLA-identical donor No neoplastic or preneoplastic evolution --Patient Characteristics-- Age: Under 40 (55 if ATG failure) Hepatic: No severe liver dysfunction Renal: No severe renal dysfunction Cardiovascular: No severe cardiac dysfunction Other: No other organ dysfunction that would compromise survival after transplant No HIV infection

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Please refer to this study by its identifier: NCT00004323

United States, California
Center for Health Sciences
Los Angeles, California, United States, 90024-1714
Sponsors and Collaborators
University of California, Los Angeles
Study Chair: Mary Carol Territo University of California, Los Angeles
  More Information

No publications provided Identifier: NCT00004323     History of Changes
Other Study ID Numbers: 199/11863, UCLA-94020602
Study First Received: October 18, 1999
Last Updated: June 23, 2005
Health Authority: United States: Federal Government

Keywords provided by National Center for Research Resources (NCRR):
aplastic anemia
hematologic disorders
rare disease

Additional relevant MeSH terms:
Anemia, Aplastic
Bone Marrow Diseases
Hematologic Diseases
Antilymphocyte Serum
Methylprednisolone Hemisuccinate
Methylprednisolone acetate
Prednisolone acetate
Prednisolone hemisuccinate
Prednisolone phosphate
Abortifacient Agents
Abortifacient Agents, Nonsteroidal
Alkylating Agents
Anti-Inflammatory Agents
Antimetabolites, Antineoplastic
Antineoplastic Agents
Antineoplastic Agents, Alkylating
Antineoplastic Agents, Hormonal
Antirheumatic Agents
Autonomic Agents
Central Nervous System Agents
Dermatologic Agents
Enzyme Inhibitors
Folic Acid Antagonists processed this record on November 25, 2014