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

Shunichi Kato, Hiromasa Yabe, Hiromitsu Takakura, Hideo Mugishima, Mika Ishige, Akemi Tanaka, Koji Kato, Nao Yoshida, Souichi Adachi, Norio Sakai, Yoshiko Hashii, Toya Ohashi, Yoji Sasahara, Yasuyuki Suzuki, Ken Tabuchi

Research output: Contribution to journalArticle

8 Citations (Scopus)

Abstract

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 patients conditioned by RIC (74.6%) was comparable or slightly better than in those who underwent MAC with irradiation (57.9%) or without irradiation (54.2%) in 2000-2010. A more pronounced trend was observed toward differential EFS for UCBT in 2000-2010: RIC (62.9%), MAC with irradiation (20.0%), and MAC without irradiation (42.1%). The difference between RIC and MAC with irradiation was significant (p < 0.03). In summary, we report a Japanese registry analysis of HSCT for IEM with improving survival in UCBT. The introduction of RIC after 2000 was considered to contribute to this improvement. UCBT could be recommended for those who lack an HLA-identical sibling donor.

Original languageEnglish
Pages (from-to)203-214
Number of pages12
JournalPediatric Transplantation
Volume20
Issue number2
DOIs
Publication statusPublished - 2016 Mar 1

Keywords

  • hematopoietic stem cell transplantation
  • inborn errors of metabolism
  • national survey

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Transplantation

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