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Allogenic Hematopoietic Cell Transplantation in Thalassemia Major

A Single-center Retrospective Analysis From India

Choudhary, Dharma DM; Doval, Divya MD; Sharma, Sanjeev K. DM; Khandelwal, Vipin MD; Setia, Rasika MD; Handoo, Anil MD

Journal of Pediatric Hematology/Oncology: July 2019 - Volume 41 - Issue 5 - p e296–e301
doi: 10.1097/MPH.0000000000001475
Online Articles: Original Articles

Thalassemia is a major public health problem in developing countries. Sibling matched hematopoietic stem cell transplantation (HCT) is the recommended treatment for thalassemia major (TM). We retrospectively analyzed our data of thalassemia major patients who underwent HCT at a tertiary care center in Northern India from January 2008 to September 2017. The primary end points were overall survival (OS) and thalassemia-free survival (TFS), and secondary end points were complications post HCT (graft-versus-host-disease [GVHD], hemorrhagic cystitis [HC], and sinusoidal obstruction syndrome [SOS]). Data of 203 transplants for 200 patients (3 s transplants) were evaluated. Median follow-up period was 29.1 months (range, 0.3 to 116.7 mo). The overall survival (OS) was 88.5% and TFS was 82%. Class risk analysis showed a significantly higher OS and TFS in class I and class II compared to class III high risk group (OS: P=0.0017; TFS: P=0.0005) and (OS: P=0.0134; TFS: P=0.0027) respectively. Acute and chronic GVHD was seen in 59 (29.5%) and 18 (9%) patients, respectively, and SOS and HC were seen in 23 (11.5%) and 11 (5.5%) patients, respectively. This study reconfirms that allogenic HCT is feasible in developing world with the overall survival and TFS comparable to that reported in Western literature and should be considered early in all TM patients with available matched sibling donors.

Center for Bone Marrow Transplant, BLK Super Speciality Hospital, New Delhi, India

The authors declare no conflict of interest.

Reprints: Dharma Choudhary, DM, BLK Super Speciality Hospital, Building No-5, Pusa Road, Rajendra Nagar, New Delhi, Delhi 110005, India (e-mail:

Received July 20, 2018

Accepted February 1, 2019

Copyright © 2019 Wolters Kluwer Health, Inc. All rights reserved.