Review
Hematopoietic stem cell transplantation for children with thalassemia major in china†
Jian-Pei Fang MD,
Lu-Hong Xu PhD,
Corresponding Author
Jian-Pei Fang MD
Department of Pediatrics, Second Affiliated Hospital of Sun Yat-sen University, Guangzhou 510120, PR China
Department of Pediatrics, Second Affiliated Hospital of Sun Yat-sen University, No. 107, West Yan Jiang Road, Guangzhou, PR China.===Search for more papers by this authorLu-Hong Xu PhD
Department of Pediatrics, Second Affiliated Hospital of Sun Yat-sen University, Guangzhou 510120, PR China
Search for more papers by this authorJian-Pei Fang MD,
Lu-Hong Xu PhD,
Corresponding Author
Jian-Pei Fang MD
Department of Pediatrics, Second Affiliated Hospital of Sun Yat-sen University, Guangzhou 510120, PR China
Department of Pediatrics, Second Affiliated Hospital of Sun Yat-sen University, No. 107, West Yan Jiang Road, Guangzhou, PR China.===Search for more papers by this authorLu-Hong Xu PhD
Department of Pediatrics, Second Affiliated Hospital of Sun Yat-sen University, Guangzhou 510120, PR China
Search for more papers by this author†
The authors have no conflicts of interest to disclose.
Abstract
Thalassemia is the most common single-gene disorder worldwide that is considered a major public health issue. Hematopoietic stem cell transplantation (HSCT) is the only curative treatment for thalassemia major, the form of the disease reflecting homozygosity for a mutant allele. In China, many patients with thalassemia major cannot financially afford life-long regular supportive care with blood transfusions and iron chelation. Although HSCT is expensive, it is a one-time treatment that is possible in some patients. Disease-free survival rates have been 52–82% after HSCT in China. Graft rejection is the main cause of failure following HSCT. Humoral immune activity may play an important role in engraftment of donor cells. This article reviews the current status of HSCT for children with thalassemia major in China. Pediatr Blood Cancer. 2010;55:1062–1065. © 2010 Wiley-Liss, Inc.
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