High-dose cyclophosphamide for severe aplastic anemia: long-term follow-up.
Robert A Brodsky, Allen R Chen, Donna Dorr...
https://pubmed.ncbi.nlm.nih.gov/20018919Actively Recruiting
Phase 2
Age: 1Year - 75Years
All Genders
ID02828592
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
Led by Northside Hospital, Inc. · Updated on 2026-04-20
20
Participants Needed
2
Research Sites
52 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Severe aplastic anemia is a rare and serious bone marrow failure caused by an immune-related problem, leading to very low blood cell counts and high risks of infections and bleeding. This research is studying a type of bone marrow transplant using partly matched donors for patients who do not have fully matched related donors. The study aims to evaluate the success of this transplant approach and reduce complications like graft failure and graft versus host disease. Patients will receive a preparative treatment combining Fludarabine, Cyclophosphamide, and low-dose radiation before undergoing a haploidentical bone marrow transplant. After transplant, Cyclophosphamide is given on Days 3 and 4 to help prevent rejection, followed by immunosuppressive medications Tacrolimus and Mycophenolate Mofetil starting Day 5. Mycophenolate Mofetil is stopped on Day 35, while Tacrolimus continues until Day 180. This treatment plan is designed to support successful engraftment and reduce complications. During the study, participants will have monthly tests to measure donor cell engraftment for up to two years after transplant. Researchers will also monitor for treatment-related side effects, acute and chronic graft versus host disease, and overall survival at 100 days and one year. The total participation time includes close follow-up and evaluations to assess how well the transplant works and to track safety outcomes.
CONDITIONS
Brief Title
Haploidentical Bone Marrow Transplant With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia
Who Can Participate
Age: 1Year - 75Years
All Genders
Eligibility Criteria
You may qualify if you...
- Availability of a 3/6 to 5/6 matched related donor with negative HLA cross-match in host vs. graft direction
- Age 65 years or younger if previously treated, and 75 years or younger if previously untreated
- Karnofsky Performance Status of 70% or higher
- Diagnosis of aplastic anemia with at least two of: neutrophils below 500/mm3, platelets below 20,000/mm3, or absolute reticulocyte count below 40,000/microL
- Bone marrow with marked hypocellularity (less than 25% normal) or moderate hypocellularity with 70% non-myeloid precursors plus peripheral blood criteria
You will not qualify if you...
- Poor heart function with left ventricular ejection fraction below 40%
- Poor lung function with FEV1 and FVC less than 50% predicted
- Poor liver function with bilirubin 2 mg/dL or higher
- Poor kidney function with creatinine 2.0 mg/dL or higher or creatinine clearance less than 40 mL/min
- Prior allogeneic transplant
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
1
2
3
Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Run-in Period
Duration - 6 days
Participants undergo a preparative regimen including Fludarabine, Cyclophosphamide, Rabbit ATG, and Total Body Irradiation before transplantation.
Daily visits from Day -6 to Day -1
Treatment
Duration - Approximately 180 days
Participants receive haploidentical bone marrow transplantation followed by post-transplant Cyclophosphamide and immunosuppression with Tacrolimus and MMF.
Hospitalization for transplant and treatment; visits on Days +3 and +4 for Cyclophosphamide dosing; immunosuppression monitoring visits until Day +180
Follow-up
Duration - Up to 2 years
Participants are monitored for engraftment, graft-versus-host disease, survival, and other post-transplant outcomes.
Monthly visits for chimerism tests and assessments up to 2 years
Trial Site Locations
Total: 2 locations
1
Blood and Marrow Transplant Group of Georgia
Atlanta, Georgia, United States, 30342
Actively Recruiting
2
Northside Hospital
Atlanta, Georgia, United States, 30342
Actively Recruiting
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Research Team
M
Melhem Solh, MD
S
Stacey Brown
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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Published Research Related To This Trial
Allogeneic BM transplantation for the treatment of aplastic anemia: current results and expanding donor possibilities.
Gérard Socié
https://pubmed.ncbi.nlm.nih.gov/24319167Current concepts in the pathophysiology and treatment of aplastic anemia.
Neal S Young
https://pubmed.ncbi.nlm.nih.gov/24319166How I treat acquired aplastic anemia.
Phillip Scheinberg, Neal S Young
https://pubmed.ncbi.nlm.nih.gov/22517900Comparison of Outcomes of Hematopoietic Cell Transplants from T-Replete Haploidentical Donors Using Post-Transplantation Cyclophosphamide with 10 of 10 HLA-A, -B, -C, -DRB1, and -DQB1 Allele-Matched Unrelated Donors and HLA-Identical Sibling Donors: A Multivariable Analysis Including Disease Risk Index.
Asad Bashey, Xu Zhang, Katelin Jackson...
https://pubmed.ncbi.nlm.nih.gov/26359881Total Body Irradiation-Based Myeloablative Haploidentical Stem Cell Transplantation Is a Safe and Effective Alternative to Unrelated Donor Transplantation in Patients Without Matched Sibling Donors.
Scott R Solomon, Connie A Sizemore, Melissa Sanacore...
https://pubmed.ncbi.nlm.nih.gov/25797174T-cell replete haploidentical donor transplantation using post-transplant CY: an emerging standard-of-care option for patients who lack an HLA-identical sibling donor.
A Bashey, S R Solomon
https://pubmed.ncbi.nlm.nih.gov/24842530Criteria for severe aplastic anaemia.
C Rozman, P Marín, B Nomdedeu...
https://pubmed.ncbi.nlm.nih.gov/2889870Different outcomes between cyclophosphamide plus horse or rabbit antithymocyte globulin for HLA-identical sibling bone marrow transplant in severe aplastic anemia.
Elias Hallack Atta, Adriana Martins de Sousa, Marcelo Ribeiro Schirmer...
https://pubmed.ncbi.nlm.nih.gov/22796534