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Biol Blood Marrow Transplant. 2014 Dec;20(12):2066-71. doi: 10.1016/j.bbmt.2014.07.016. Epub 2014 Jul 23.

Outcomes of thalassemia patients undergoing hematopoietic stem cell transplantation by using a standard myeloablative versus a novel reduced-toxicity conditioning regimen according to a new risk stratification.

Author information

1
Department of Pediatrics, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand.
2
Department of Pediatrics, Chiangmai University Hospital, Chiangmai, Thailand.
3
Department of Pediatrics, Khonkaen University, Khonkaen, Thailand.
4
Department of Pediatrics, Siriraj Hospital, Mahidol University, Bangkok, Thailand.
5
Department of Pediatrics, Phramongkutklao Hospital, Bangkok, Thailand.
6
Queen Sirikit National Institute of Child Health, Bangkok, Thailand.
7
Center of Pharmaceutical Outcomes Research, Department of Pharmacy Practice, Naresuan University, Phitsanulok, Thailand.
8
Department of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand.
9
Department of Medicine, Siriraj Hospital, Mahidol University, Bangkok, Thailand.
10
Department of Stem Cell Transplantation and Cellular Therapy, University of Texas MD Anderson Cancer Center, Houston, Texas.
11
Department of Pediatrics, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand. Electronic address: suradej.hon@mahidol.ac.th.

Abstract

Improving outcomes among class 3 thalassemia patients receiving allogeneic hematopoietic stem cell transplantations (HSCT) remains a challenge. Before HSCT, patients who were ≥ 7 years old and had a liver size ≥ 5 cm constitute what the Center for International Blood and Marrow Transplant Research defined as a very high-risk subset of a conventional high-risk class 3 group (here referred to as class 3 HR). We performed HSCT in 98 patients with related and unrelated donor stem cells. Seventy-six of the patients with age < 10 years received the more conventional myeloablative conditioning (MAC) regimen (cyclophosphamide, busulfan, ± fludarabine); the remaining 22 patients with age ≥ 10 years and hepatomegaly (class 3 HR), and in several instances additional comorbidity problems, underwent HSCT with a novel reduced-toxicity conditioning (RTC) regimen (fludarabine and busulfan). We then compared the outcomes between these 2 groups (MAC versus RTC). Event-free survival (86% versus 90%) and overall survival (95% versus 90%) were not significantly different between the respective groups; however, there was a higher incidence of serious treatment-related complications in the MAC group, and although we experienced 6 graft failures in the MAC group (8%), there were none in the RTC group. Based on these results, we suggest that (1) class 3 HR thalassemia patients can safely receive HSCT with our novel RTC regimen and achieve the same excellent outcome as low/standard-risk thalassemia patients who received the standard MAC regimen, and further, (2) that this novel RTC approach should be tested in the low/standard-risk patient population.

KEYWORDS:

Myeloablative; Reduced toxicity; Thalassemia

PMID:
25064743
PMCID:
PMC5538782
DOI:
10.1016/j.bbmt.2014.07.016
[Indexed for MEDLINE]
Free PMC Article

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