- https://doi.org/10.14740/jh2184
Post-Transplant Cyclophosphamide Allows Allogeneic Hematopoietic Stem-Cell Transplantation Across Donor Types for Nonmalignant Hematologic Diseases.
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The aim of the study was to compare post-transplant cyclophosphamide (PTCY)-based regimens with historical regimens using calcineurin inhibitor and methotrexate (CNI-MTX) for allogeneic hematopoietic stem-cell transplant (HCT) in nonmalignant hematologic disorders. We conducted a single-center, retrospective review of patients with acquired severe aplastic anemia (N = 18) or Diamond-Blackfan anemia (N = 1) who underwent allogeneic HCT from 2011 to 2024. Patients received graft-versus-host disease (GVHD) prophylaxis with either CNI-MTX or PTCY-mycophenolate mofetil-tacrolimus. Primary endpoints were overall survival (OS) and disease-free survival (DFS) without graft failure at 1 year after transplantation. In the CNI-MTX cohort (N = 14) with severe aplastic anemia, 11 patients received fludarabine-cyclophosphamide-thymoglobulin (ATG)-total body irradiation (TBI), while three received cyclophosphamide-ATG allogeneic HCT. Donors were matched-unrelated (N = 7), matched-related (N = 6), or mismatched-unrelated (N = 1). Graft sources included bone marrow (N = 12) or peripheral blood stem cells (N = 2). One patient developed grade 3 skin acute GVHD, and none had chronic GVHD. There was primary graft failure (N = 6), stable mixed T-cell chimerism (N = 4), and 100% donor chimerism (N = 4). Four patients with primary graft failure underwent salvage second transplants at a median of 103 days (35-322) after the first transplant. Five patients with primary graft failure died at a median of 6 months (0.89-9.3) from the first transplant. The PTCY cohort (N = 5) included four patients with severe aplastic anemia and one with Diamond-Blackfan anemia. All underwent fludarabine-cyclophosphamide-ATG-TBI allogeneic HCT. Donors were matched-related (N = 1), matched-unrelated (N = 2), syngeneic (N = 1), or haploidentical (N = 1). Graft source was peripheral blood stem cells (N = 3) for matched-related, matched-unrelated, and syngeneic transplants, and bone marrow (N = 2) for haploidentical and matched-unrelated donor transplants. Donor chimerism was 100% (N = 3) and mixed chimerism (N = 2). All patients became transfusion-independent, and none developed GVHD or graft failure. The 1-year OS rate was 64.29% vs. 100%, the 1-year DFS rate was 57.14% vs. 100%, and the 1-year GVHD-free, graft failure-free survival (GRFS) was 50% vs.100% for the CNI-MTX and PTCY cohorts, respectively. Despite a trend toward better OS, DFS, and GRFS for PTCY, the OS, DFS, and GRFS time distributions were not statistically significantly different (P = 0.1448, 0.0919, and 0.0627, respectively). Allogeneic HCT with uniform conditioning of fludarabine-cyclophosphamide-ATG-TBI with PTCY GVHD prophylaxis is effective for adults with severe aplastic anemia or Diamond-Blackfan anemia across donor types (matched-related, syngeneic, matched-unrelated, haploidentical) and should be prospectively compared with historical regimens using CNI-MTX GVHD prophylaxis.
- Abstract
5 - 10.1182/blood.v126.23.2031.2031
- Dec 3, 2015
- Blood
Using Haploidentical (haplo) Donors and High-Dose Post-Transplant Cyclophosphamide (PTCy) for Refractory Severe Aplastic Anemia (SAA)
- Discussion
- 5
- 10.1002/ajh.26296
- Jul 31, 2021
- American Journal of Hematology
Phase II clinical trial of one dose of post-transplant cyclophosphamide for graft versus host disease prevention following myeloablative, peripheral blood stem cell, matched-unrelated donor transplantation.
- Abstract
- 5
- 10.1182/blood-2023-189914
- Nov 28, 2023
- Blood
Excellent Outcomes of HLA Matched Related Donor Transplant for Adults with Severe Sickle Cell Disease Using a Non-Myeloablative Conditioning with Thiotepa and Post-Transplant Cyclophosphamide: Multi-Center International Experience
- Front Matter
- 17
- 10.1053/j.gastro.2005.01.015
- Mar 1, 2005
- Gastroenterology
Resetting the immune system in refractory Crohn’s disease: Is autologous hematopoietic stem cell transplantation the way forward?
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- 10.1182/blood-2023-187386
- Nov 2, 2023
- Blood
Haploidentical Hematopoietic Cell Transplant with Post-Transplant Cyclophosphamide Expedites Donor-Derived Immune Reconstitution in a Pediatric Severe Aplastic Anemia Cohort
- Research Article
- 31
- 10.1016/j.exphem.2012.06.003
- Jun 12, 2012
- Experimental Hematology
Impact of anti-HLA antibodies on allogeneic hematopoietic stem cell transplantation outcomes after reduced-intensity conditioning regimens
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- 8
- 10.1016/j.jtct.2023.04.015
- Apr 24, 2023
- Transplantation and Cellular Therapy
Prospective Study of a Modified Post-Transplantation Cyclophosphamide Regimen for Severe Aplastic Anemia Patients with HLA-Haploidentical Transplantation
- Abstract
- 1
- 10.1182/blood-2023-188719
- Nov 28, 2023
- Blood
Post Transplant Cyclophosphamide (PTCY) in Peripheral Blood Stem Cell Transplant Results in Successful Engraftment and Substantially Low Frequency of Chronic Graft Versus Host Disease (GvHD), without Impaired Cellular Immune Reconstitution
- Abstract
- 1
- 10.1182/blood-2019-121900
- Nov 13, 2019
- Blood
The Path to Cure: Using Haploidentical (haplo) Donors and High-Dose Post-Transplant Cyclophosphamide (PTCy) for Treatment-Naïve and Refractory Severe Aplastic Anemia (SAA)
- Abstract
- 10.1182/blood-2023-182333
- Nov 28, 2023
- Blood
Superior Outcome of Bone Marrow Transplantation Using Fludarabine and Cyclophosphamide with ATG over Fludarabine and Cyclophosphamide without ATG Conditioning Regimen in Severe Aplastic Anemia
- Research Article
- 211
- 10.1016/j.bbmt.2011.06.006
- Jun 25, 2011
- Biology of Blood and Marrow Transplantation
The Incidence of Veno-Occlusive Disease Following Allogeneic Hematopoietic Stem Cell Transplantation Has Diminished and the Outcome Improved over the Last Decade
- Abstract
- 2
- 10.1182/blood-2020-142753
- Nov 5, 2020
- Blood
A Novel TBI Free Conditioning Protocol for Haploidentical Transplant in Acquired Aplastic Anemia: (FluCAB-Prime)
- Research Article
- 10.1182/blood-2024-207952
- Nov 5, 2024
- Blood
Trial in Progress: Cureaa - a Clinical Trial of Upfront Haploidentical or Unrelated Donor BMT to Restore Normal Hematopoiesis in Aplastic Anemia, Blood and Marrow Transplant Clinical Trials Network (BMT CTN) #2207
- Research Article
- 4
- 10.1097/mph.0000000000001981
- Oct 28, 2020
- Journal of Pediatric Hematology/Oncology
Between 2014 and 2020, 31 patients with severe aplastic anemia (SAA) underwent full match allogeneic hematopoietic stem cell transplantation at our center. Of the 31 patients with SAA, 19 had acquired aplastic anemia, 2 had Diamond Blackfan anemia and 10 had Fanconi anemia. Donors were either matched sibling (n=29), related donors (n=2), or unrelated donors (n=3). Peripheral blood stem cells were the graft source in all the cases except 1. Fludarabine-based reduced intensity conditioning was used in all except for patients with a diagnosis of Diamond Blackfan anemia. All patients except 1 achieved hematologic recovery in the form of neutrophil engraftment at 13 days (range, 9 to 17), whereas platelet engraftment occurred at 14 days (range, 10 to 18). Graft versus host disease (GvHD) prophylaxis consisted of cyclosporine and methotrexate ±antithymocyte globulin (horse/rabbit). Acute GvHD developed in 12.9% patients, whereas no patients developed chronic GvHD till the time of last follow-up. The 2-year overall survival for the entire cohort was 93.21±4.6%. In patients with SAA, allogeneic stem cell transplant using fludarabine-based conditioning regimens are very well tolerated and have excellent outcomes in a full match setting.
- Abstract
- 10.1182/blood.v128.22.3417.3417
- Dec 2, 2016
- Blood
Chimerism Analysis after Haploidentical Stem Cell Transplantation: Is It Necessary for All Patients?
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