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Title: Hematopoietic Stem Cell Transplantation for Severe Thalassemia Patients from Haploidentical Donors Using a Novel Conditioning Regimen
Authors: Usanarat Anurathapan
Suradej Hongeng
Samart Pakakasama
Duantida Songdej
Nongnuch Sirachainan
Pongpak Pongphitcha
Ampaiwan Chuansumrit
Pimlak Charoenkwan
Arunee Jetsrisuparb
Kleebsabai Sanpakit
Piya Rujkijyanont
Arunotai Meekaewkunchorn
Yujinda Lektrakul
Pornchanok Iamsirirak
Pacharapan Surapolchai
Somtawin Sirireung
Rosarin Sruamsiri
Pustika Amalia Wahidiyat
Borje S. Andersson
University of Indonesia, RSUPN Dr. Cipto Mangunkusumo
Khon Kaen University
Samitivej Hospital (Sukhumvit)
Faculty of Medicine, Ramathibodi Hospital, Mahidol University
University of Texas MD Anderson Cancer Center
Maharaj Nakorn Chiang Mai Hospital
Thammasat University
Faculty of Medicine, Siriraj Hospital, Mahidol University
Queen Sirikit National Institute of Child Health
Phramongkutklao College of Medicine
Sunpasitthiprasong Hospital
Keywords: Medicine
Issue Date: 1-Jan-2020
Citation: Biology of Blood and Marrow Transplantation. (2020)
Abstract: © 2020 Patients with severe thalassemia commonly have a survival that is significantly shorter than that of the general population. Allogeneic hematopoietic stem cell transplantation (allo-SCT) 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 pretransplant immune suppression phase (PTIS) and 2 courses of dexamethasone and fludarabine, followed by pretransplant conditioning with fludarabine-i.v. busulfan and post-transplant graft-versus-host disease (GVHD) prophylaxis with cyclophosphamide, tacrolimus, and mycophenolate mofetil. We transplanted 83 consecutive transfusion-dependent patients with thalassemia (median age, 12 years; range, 1 to 28 years) with a minimum follow-up of 6 months (median, 15 months; range, 7 to 53 months); the 3-year projected overall and event-free survival is over 96%, and there have been no secondary graft failures. Of the first 31 patients, we had 2 graft failures, both of them occurring in patients with extremely high titers of anti-donor-specific HLA antibodies (anti-DSAs), but after adjusting the PTIS to include bortezomib and rituximab for patients with high titers of anti-DSAs and using pharmacologic dose guidance for busulfan, we had no graft failures in the last 52 patients. Six (7%) of 83 patients developed severe GVHD. We conclude that this is a safe and efficacious approach to allogeneic SCT in thalassemia, yielding results comparable to those available for patients with fully matched donors.
ISSN: 15236536
Appears in Collections:Scopus 2020

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