title: 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.
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.
authors: Department of Pediatrics, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand.
authors: Usanarat
authors: U
authors: Anurathapan
authors: Department of Pediatrics, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand.
authors: Samart
authors: S
authors: Pakakasama
authors: Department of Pediatrics, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand.
authors: Pimsiri
authors: P
authors: Mekjaruskul
authors: Department of Pediatrics, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand.
authors: Nongnuch
authors: N
authors: Sirachainan
authors: Department of Pediatrics, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand.
authors: Duantida
authors: D
authors: Songdej
authors: Department of Pediatrics, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand.
authors: Ampaiwan
authors: A
authors: Chuansumrit
authors: Department of Pediatrics, Chiangmai University Hospital, Chiangmai, Thailand.
authors: Pimlak
authors: P
authors: Charoenkwan
authors: Department of Pediatrics, Khonkaen University, Khonkaen, Thailand.
authors: Arunee
authors: A
authors: Jetsrisuparb
authors: Department of Pediatrics, Siriraj Hospital, Mahidol University, Bangkok, Thailand.
authors: Kleebsabai
authors: K
authors: Sanpakit
authors: Department of Pediatrics, Siriraj Hospital, Mahidol University, Bangkok, Thailand.
authors: Bunchoo
authors: B
authors: Pongtanakul
authors: Department of Pediatrics, Phramongkutklao Hospital, Bangkok, Thailand.
authors: Piya
authors: P
authors: Rujkijyanont
authors: Queen Sirikit National Institute of Child Health, Bangkok, Thailand.
authors: Arunotai
authors: A
authors: Meekaewkunchorn
authors: Center of Pharmaceutical Outcomes Research, Department of Pharmacy Practice, Naresuan University, Phitsanulok, Thailand.
authors: Rosarin
authors: R
authors: Sruamsiri
authors: Department of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand.
authors: Artit
authors: A
authors: Ungkanont
authors: Department of Medicine, Siriraj Hospital, Mahidol University, Bangkok, Thailand.
authors: Surapol
authors: S
authors: Issaragrisil
authors: Department of Stem Cell Transplantation and Cellular Therapy, University of Texas MD Anderson Cancer Center, Houston, Texas.
authors: Borje S
authors: BS
authors: Andersson
authors: Department of Pediatrics, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand. Electronic address: suradej.hon@mahidol.ac.th.
authors: Suradej
authors: S
authors: Hongeng
mesh_terms: D000293:Adolescent; D000328:Adult; D064591:Allografts; D002066:Busulfan / Q000008:administration & dosage; D002648:Child; D002675:Child, Preschool; D003520:Cyclophosphamide / Q000008:administration & dosage; D018572:Disease-Free Survival; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D008297:Male; D019653:Myeloablative Agonists / Q000008:administration & dosage; D012307:Risk Factors; D015996:Survival Rate; D013789:Thalassemia / Q000401:mortality* / Q000628:therapy*; D019172:Transplantation Conditioning / Q000379:methods*; D014740:Vidarabine / Q000008:administration & dosage / Q000031:analogs & derivatives
chemical_list: D019653:Myeloablative Agonists; D003520:Cyclophosphamide; D014740:Vidarabine; D002066:Busulfan; C024352:fludarabine
keywords: Myeloablative; Reduced toxicity; Thalassemia