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Study of Allogeneic Bone Marrow and T-Cell Depleted, CD34+ Peripheral Blood Stem Cell Transplantation in Patients With Aplastic Anemia

The recruitment status of this study is unknown. The completion date has passed and the status has not been verified in more than two years.
Verified October 2003 by Office of Rare Diseases (ORD).
Recruitment status was:  Active, not recruiting
Information provided by:
Office of Rare Diseases (ORD) Identifier:
First received: June 6, 2001
Last updated: June 23, 2005
Last verified: October 2003


I. Determine the effect of supplementation with donor T-cell depleted, CD34+ peripheral blood stem cells on durable engraftment and incidence of graft-versus-host disease in patients with aplastic anemia undergoing allogeneic bone marrow transplantation.

Condition Intervention
Graft Versus Host Disease
Aplastic Anemia
Drug: anti-thymocyte globulin
Drug: cyclophosphamide
Drug: cyclosporine
Drug: methylprednisolone
Procedure: Allogeneic Bone Marrow Transplantation

Study Type: Interventional
Study Design: Primary Purpose: Treatment

Resource links provided by NLM:

Further study details as provided by Office of Rare Diseases (ORD):

Estimated Enrollment: 3
Study Start Date: April 2001
Detailed Description:

PROTOCOL OUTLINE: Bone marrow and peripheral blood stem cells (PBSC) are harvested from a HLA identical or 1 antigen mismatched related donor. PBSC are selected for CD34+ cells and T cells are depleted.

Patients receive cyclophosphamide IV over 2 hours on days -6 to -3 and anti-thymocyte globulin IV with methylprednisolone IV over 10-12 hours on days -5 to -3. T-cell depleted PBSC and bone marrow are infused on day 0. Patients receive cyclosporine IV over 12-24 hours on days -1 to 120 followed by a taper and methylprednisolone IV on days 7-64 for graft-versus-host disease prophylaxis.

Patients are followed every 30 days for 1 year, every 60 days for 2 years, and then as needed for a minimum of 3 years.


Ages Eligible for Study:   15 Years to 55 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


--Disease Characteristics--

  • Diagnosis of life-threatening or transfusion-dependent marrow or immune-mediated failure involving one or more hematopoietic cell lines evidenced by one or more of the following: Granulocyte count less than 500/mm3 Platelet count less than 20,000/mm3 Absolute reticulocyte count less than 60,000/mm3
  • Immune-mediated anemia and/or thrombocytopenia must have failed corticosteroids and cyclosporine
  • Paroxysmal nocturnal hemoglobinuria with progressive marrow failure allowed
  • Either congenital or acquired severe granulocytopenia with a history of life threatening infections No diagnosis of Fanconi's anemia
  • HLA identical or 1 antigen mismatched related donor meeting the following criteria: No prior or concurrent malignancy except localized basal cell or squamous cell skin cancer (malignancies judged to be cured by local surgery, such as head and neck cancer or stage I breast cancer, considered on an individual basis) Negative pregnancy test

--Patient Characteristics--

  • Hepatic: Hepatitis B surface antigen negative Bilirubin no greater than 2.0 mg/dL Transaminases no greater than 2 times upper limit of normal (in the absence of Gilbert's disease)
  • Renal: Creatinine no greater than 2.0 mg/dL
  • Cardiovascular: No history of coronary artery disease LVEF at least 40% by MUGA
  • Pulmonary: FEV1 at least 50% predicted FVC at least 50% predicted DLCO at least 50% predicted
  • Other: No uncontrolled diabetes mellitus No other illness that would preclude aggressive chemotherapy No prior or concurrent malignancy except localized basal cell or squamous cell skin cancer (malignancies judged to be cured by local surgery, such as head and neck cancer or stage I breast cancer, considered on an individual basis) No psychiatric illness or mental deficiency that would preclude study Not pregnant or nursing Fertile patients must use effective contraception HIV negative
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Please refer to this study by its identifier: NCT00017654

United States, Illinois
Northwestern Memorial Hospital
Chicago, Illinois, United States, 60611
Sponsors and Collaborators
Northwestern Memorial Hospital
Study Chair: Richard K. Burt Northwestern Memorial Hospital
  More Information Identifier: NCT00017654     History of Changes
Other Study ID Numbers: 199/14983
Study First Received: June 6, 2001
Last Updated: June 23, 2005

Keywords provided by Office of Rare Diseases (ORD):
aplastic anemia
disease-related problem/condition
graft versus host disease
hematologic disorders
rare disease

Additional relevant MeSH terms:
Graft vs Host Disease
Anemia, Aplastic
Hematologic Diseases
Immune System Diseases
Bone Marrow Diseases
Antilymphocyte Serum
Methylprednisolone Hemisuccinate
Prednisolone acetate
Methylprednisolone acetate
Prednisolone hemisuccinate
Prednisolone phosphate
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists
Enzyme Inhibitors
Antifungal Agents
Anti-Infective Agents
Dermatologic Agents processed this record on April 27, 2017