Thalassemia-free and graft-versus-host-free survival: outcomes of hematopoietic stem cell transplantation for thalassemia major, Turkish experience

We report the national data on the outcomes of hematopoietic stem cell transplantation (HSCT) for thalassemia major (TM) patients in Turkey on behalf of the Turkish Pediatric Stem Cell Transplantation Group. We retrospectively enrolled 1469 patients with TM who underwent their first HSCT between 198...

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Published in:	Bone marrow transplantation (Basingstoke) Vol. 57; no. 5; pp. 760 - 767
Main Authors:	Yesilipek, M. Akif, Uygun, Vedat, Kupesiz, Alphan, Karasu, Gulsun, Ozturk, Gulyuz, Ertem, Mehmet, Şaşmaz, İlgen, Daloğlu, Hayriye, Güler, Elif, Hazar, Volkan, Fisgin, Tunç, Sezgin, Gülay, Kansoy, Savaş, Kuşkonmaz, Barış, Akıncı, Burcu, Özbek, Namık, İnce, Elif Ünal, Öztürkmen, Seda, Küpesiz, Funda Tayfun, Yalçın, Koray, Anak, Sema, Bozkurt, Ceyhun, Karakükçü, Musa, Küpeli, Serhan, Albayrak, Davut, Öniz, Haldun, Aksoylar, Serap, Okur, Fatma Visal, Albayrak, Canan, Yenigürbüz, Fatma Demir, Bozkaya, İkbal Ok, İleri, Talia, Gürsel, Orhan, Karagün, Barbaros Şahin, Kintrup, Gülen Tüysüz, Çelen, Suna, Elli, Murat, Aksoy, Basak Adaklı, Yılmaz, Ebru, Tanyeli, Atila, Akyol, Şule Turan, Siviş, Zuhal Önder, Özek, Gülcihan, Uçkan, Duygu, Kartal, İbrahim, Atay, Didem, Akyay, Arzu, Bilir, Özlem Arman, Çakmaklı, Hasan Fatih, Kürekçi, Emin, Malbora, Barış, Akbayram, Sinan, Demir, Hacı Ahmet, Kılıç, Suar Çakı, Güneş, Adalet Meral, Zengin, Emine, Özmen, Salih, Antmen, Ali Bülent
Format:	Journal Article
Language:	English
Published:	London Nature Publishing Group UK 01-05-2022 Nature Publishing Group
Subjects:	692/499 692/699/1541/13 Blood diseases Cell Biology Chimerism Graft-versus-host reaction Hematology Hematopoietic stem cells Internal Medicine Medicine Medicine & Public Health Patients Pediatrics Public Health Stem cell transplantation Stem Cells Survival Survival analysis Thalassemia Transplantation Transplants Turkey
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Summary:	We report the national data on the outcomes of hematopoietic stem cell transplantation (HSCT) for thalassemia major (TM) patients in Turkey on behalf of the Turkish Pediatric Stem Cell Transplantation Group. We retrospectively enrolled 1469 patients with TM who underwent their first HSCT between 1988 and 2020 in 25 pediatric centers in Turkey. The median follow-up duration and transplant ages were 62 months and 7 years, respectively; 113 patients had chronic graft versus host disease (cGVHD) and the cGVHD rate was 8.3% in surviving patients. Upon the last visit, 30 patients still had cGvHD (2.2%). The 5-year overall survival (OS), thalassemia-free survival (TFS) and thalassemia-GVHD-free survival (TGFS) rates were 92.3%, 82.1%, and 80.8%, respectively. cGVHD incidence was significantly lower in the mixed chimerism (MC) group compared to the complete chimerism (CC) group ( p < 0.001). In survival analysis, OS, TFS, and TGFS rates were significantly higher for transplants after 2010. TFS and TGFS rates were better for patients under 7 years and at centers that had performed over 100 thalassemia transplants. Transplants from matched unrelated donors had significantly higher TFS rates. We recommend HSCT before 7 years old in thalassemia patients who have a matched donor for improved outcomes.
Bibliography:	ObjectType-Article-1 SourceType-Scholarly Journals-1 ObjectType-Feature-2 content type line 23
ISSN:	0268-3369 1476-5365
DOI:	10.1038/s41409-022-01613-w