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

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

Who can join

Ages 1 to 75 · All sexes

Inclusion 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:

Peripheral Blood (must fulfill 2 of 3):

* \<500 PMN/mm3
* \<20,000 platelets
* absolute reticulocyte count \<40,000/microL

Bone Marrow (must be either):

* markedly hypocellular (\<25% of normal cellularity)
* moderately hypocellular with 70% non-myeloid precursors and patient meets peripheral blood criteria above

Exclusion Criteria:

* 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

About the study

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.

What is being tested

• Fludarabine (drug)
• Cyclophosphamide (drug)
• Total Body Irradiation (radiation)
• Rabbit ATG (drug)
• Cyclophosphamide (drug)

Sponsor: Northside Hospital, Inc. · Participants: 20 · Started: Sep 9, 2016

Contact the study team

• Melhem Solh, MD · Phone: 404-255-1930
• Stacey Brown · Phone: 404-851-8238

Official record on ClinicalTrials.gov — NCT02828592

Locations in the U.S.

Conditions

• Aplastic Anemia

From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.