Haploidentical Bone Marrow Transplant With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia

Description

Severe aplastic anemia is a rare and serious form of bone marrow failure related to an immune-mediated mechanism that results in severe pancytopenia and high risk for infections and bleeding. Patients with matched sibling donors for transplantation have a 80-90% chance of survival; however, a response rate with just immunosuppression for those patients lacking suitable HLA-matched related siblings is only 60%. With immunosuppression, only 1/3 of patients are cured, 1/3 are dependent on long term immunosuppression, and the other 1/3 relapse or develop a clonal disorder. Recent studies have shown that using a haploidentical donor for transplantation has good response rates and significantly lower rates of acute and chronic GVHD.

Conditions

Severe Aplastic Anemia

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Study Overview

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Study Details

Study overview

Severe aplastic anemia is a rare and serious form of bone marrow failure related to an immune-mediated mechanism that results in severe pancytopenia and high risk for infections and bleeding. Patients with matched sibling donors for transplantation have a 80-90% chance of survival; however, a response rate with just immunosuppression for those patients lacking suitable HLA-matched related siblings is only 60%. With immunosuppression, only 1/3 of patients are cured, 1/3 are dependent on long term immunosuppression, and the other 1/3 relapse or develop a clonal disorder. Recent studies have shown that using a haploidentical donor for transplantation has good response rates and significantly lower rates of acute and chronic GVHD.

A Study of T-Cell Replete, HLA-Mismatched Haploidentical Bone Marrow Transplantation With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia Lacking HLA-Matched Related Donor

Haploidentical Bone Marrow Transplant With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia

Condition
Severe Aplastic Anemia
Intervention / Treatment

-

Contacts and Locations

Atlanta

Blood and Marrow Transplant Group of Georgia, Atlanta, Georgia, United States, 30342

Atlanta

Northside Hospital, Atlanta, Georgia, United States, 30342

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • * Availability of 3/6 - 5/6 matched (HLA-A, B, DR) related donor who must have negative HLA cross-match in the host vs. graft direction
  • * Age \<= 65 years for previously treated and \<= 75 years for previously treated patients
  • * KPS \>= 70%
  • * Aplastic Anemia that meets the following criteria:
  • * \<500 PMN/mm3
  • * \<20,000 platelets
  • * absolute reticulocyte count \<40,000/microL
  • * markedly hypocellular (\<25% of normal cellularity)
  • * moderately hypocellular with 70% non-myeloid precursors and patient meets peripheral blood criteria above
  • * poor cardiac function (LVEF \<40%)
  • * poor pulmonary function (FEV1 \& FVC \<50% predicted)
  • * poor liver function (bili \>= 2mg/dL)
  • * poor renal function (creatinine \>= 2.0mg/dL or creatinine clearance \<40mL/min)
  • * prior allogeneic transplant

Ages Eligible for Study

1 Year to 75 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Northside Hospital, Inc.,

Melhem Solh, MD, PRINCIPAL_INVESTIGATOR, Blood and Marrow Transplant Group of Georgia

Study Record Dates

2026-08-31