Publication:
Hematopoietic Stem Cell Transplantation for Severe Thalassemia Patients from Haploidentical Donors Using a Novel Conditioning Regimen

1

Issued Date

2020-01-01

Resource Type

Article

ISSN

15236536
10838791

DOI

10.1016/j.bbmt.2020.01.002

Other identifier(s)

2-s2.0-85083185919

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Mahidol University

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SCOPUS

Bibliographic Citation

Biology of Blood and Marrow Transplantation. (2020)

Suggested Citation

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 Hematopoietic Stem Cell Transplantation for Severe Thalassemia Patients from Haploidentical Donors Using a Novel Conditioning Regimen. Biology of Blood and Marrow Transplantation. (2020). doi:10.1016/j.bbmt.2020.01.002 Retrieved from: https://repository.li.mahidol.ac.th/handle/123456789/54692

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Title

Hematopoietic Stem Cell Transplantation for Severe Thalassemia Patients from Haploidentical Donors Using a Novel Conditioning Regimen

Author(s)

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

Other Contributor(s)

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

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.

Keyword(s)

Medicine

Availability

URI

https://repository.li.mahidol.ac.th/handle/123456789/54692

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Scopus 2020