Article
Hematopoietic stem cell transplantation for inborn errors of metabolism: A report from the Research Committee on Transplantation for Inborn Errors of Metabolism of the Japanese Ministry of Health, Labour and Welfare and the Working Group of the Japan Society for Hematopoietic Cell Transplantation.
Pediatric transplantation - 1 Mar 2016
Kato Shunichi, Yabe Hiromasa, Takakura Hiromitsu, Mugishima Hideo, Ishige Mika, Tanaka Akemi, Kato Koji, Yoshida Nao, Adachi Souichi, Sakai Norio, Hashii Yoshiko, Ohashi Toya, Sasahara Yoji, Suzuki Yasuyuki, Tabuchi Ken
Abstract excerpt
A total of 216 patients with IEM were treated by allogeneic HSCT in Japan from 1985 until 2010. The results of UCBT have improved, and the OS rate of UCBT (81.9%) was not different from those of RBMT (87.2%) or UBMT (73.9%) in 2000-2010. However, EFS rates in RBMT (73.2%) and UBMT (62.2%) were better than that in UCBT (49.5%), and the difference between RBMT and UCBT was significant (p = 0.01). The EFS rate of...
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