Haploidentical Hematopoietic Stem Cell Transplantation in Thalassemia
Issued Date
2022-01-01
Resource Type
ISSN
03630269
eISSN
1532432X
Scopus ID
2-s2.0-85135218437
Pubmed ID
35920292
Journal Title
Hemoglobin
Volume
46
Issue
1
Start Page
2
End Page
6
Rights Holder(s)
SCOPUS
Bibliographic Citation
Hemoglobin Vol.46 No.1 (2022) , 2-6
Suggested Citation
Anurathapan U., Pakakasama S., Songdej D., Pongphitcha P., Chuansumrit A., Andersson B.S., Hongeng S. Haploidentical Hematopoietic Stem Cell Transplantation in Thalassemia. Hemoglobin Vol.46 No.1 (2022) , 2-6. 6. doi:10.1080/03630269.2022.2059671 Retrieved from: https://repository.li.mahidol.ac.th/handle/20.500.14594/83891
Title
Haploidentical Hematopoietic Stem Cell Transplantation in Thalassemia
Other Contributor(s)
Abstract
Allogeneic hematopoietic stem cell transplantation (HSCT) is the only established treatment that is potentially curative, but it is limited by the availability of donors and the medical condition of the patient. To expand the donor pool to include haploidentical related donors, we introduced a program consisting of a pharmacologic pre transplant immune suppression phase (PTIS) and two courses of dexamethasone (DXM) and fludarabine (FLU) followed by pre transplant conditioning with intravenous FLU busulfan (BU) and post transplant graft-vs.-host disease (GvHD) prophylaxis with cyclophosphamide (CPM), tacrolimus, and mycophenolate mofetil. We transplanted 83 consecutive transfusion-dependent patients with thalassemia; the 3-year projected overall and event-free survival is over 96.0%, and there have been no secondary graft failures. Of the first 31 patients, we had two graft failures, both of them occurring in patients with extremely high titers of anti-donor-specific human leukocyte antigen (HLA) antibodies [anti-donor specific antibodies (DSAs)], but after adjusting the PTIS to include bortezomib (BORT) and rituximab (RIX) for patients with high titers of anti-DSAs and using pharmacologic dose guidance for BU, we had no graft failures in the last 52 patients. Six (7.0%) of 83 patients developed severe GvHD. We conclude that this is a safe and efficacious approach to allogeneic HSCT in thalassemia.
