Abstract
Backgroundβ-Thalassemia major (β-TM) has become a public health problem in mainland China. Hematopoietic stem cell transplantation (HSCT) has remained the only cure for β-TM in mainland China since 1998.MethodsThis multicenter retrospective study provides a comprehensive review of the outcomes of 50 pediatric patients with β-TM who received HSCT between 1998 and 2009 at five centers in mainland China. Both related (n = 35) and unrelated donors (n = 15) with complete human leukocyte antigen matches were included. The stem cell sources included bone marrow (BM), peripheral blood stem cells, umbilical cord blood (UCB) and a combination of BM and UCB or a combination of BM and peripheral blood stem cells from a single sibling donor.ResultsThe probabilities of 5-year overall survival (OS) and thalassemia-free survival (TFS) after the first HSCT were 83.1 and 67.3%, respectively. Graft failure (GF) occurred in 17 patients. Univariate analyses showed that umbilical cord blood transplantation (UCBT) was one of the potential risk factors for decreased OS (P = 0.051), and that UCBT (P = 0.002) was potentially related to TFS. GF incidence was distinct between the UCBT and non-UCBT groups (P = 0.004). Four cases of UCB-BM combined transplantation led to decreased risks of mortality and recurrence. In the UCBT group, related donor transplantation produced more favorable results than unrelated donor transplantation in OS (P = 0.009) but not in TFS (P = 0.217).ConclusionsGF was the primary cause of UCBT failure. Though UCBT from related donors was not favorable, the combined transplantation of UCB and BM could improve the prognosis of UCBT.
Highlights
Thalassemia is the most common single-gene disorder worldwide and is considered a major public health issue. β-Thalassemia major (β-TM) occurs in homozygous or1 3 Vol:.(1234567890)World Journal of Pediatrics (2018) 14:92–99In the 1980s, a large-scale survey of hemoglobinopathies was carried out [3]
At the beginning phase of Hematopoietic stem cell transplantation (HSCT) for the treatment of β-TM, doctors in mainland China were faced with numerous difficulties, including human leukocyte antigen (HLA) matching techniques, the toxicity of conditioning regimens, graft rejection, severe graft-versushost disease (GVHD), infections, patients’ high risk status, and a lack of appropriate donors
Global data have suggested that umbilical cord blood transplantation (UCBT) from related donors had similar engraftment levels compared to bone marrow transplantation (BMT) but lower incidences of GVHD, which further promoted the use of UCBT in treating β-TM [19,20,21,22]
Summary
Thalassemia is the most common single-gene disorder worldwide and is considered a major public health issue. β-Thalassemia major (β-TM) occurs in homozygous or1 3 Vol:.(1234567890)World Journal of Pediatrics (2018) 14:92–99In the 1980s, a large-scale survey of hemoglobinopathies was carried out [3]. Hematopoietic stem cell transplantation (HSCT) has remained the only cure for βTM [5] since the first bone marrow transplantation (BMT) for β-TM reported in December 1981 [6]. This expensive procedure was not successfully performed in mainland China until 1998 [7]. The majority of patients with β-TM are distributed throughout southern China and HSCT is performed by several qualified hospitals. To provide a comprehensive review of the outcomes of children receiving HSCT for β-TM in China, the present study retrospectively analyzed the data from children receiving HSCT for β-TM between 1998 and 2009 in a multicenter study group of the Pediatric Branch of the Chinese Medical Association
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