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
Shunichi KatoKen Tabuchi

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.

References

Aug 1, 1980·The American Journal of Medicine·H M ShulmanE D Thomas
Sep 10, 1999·Human Genetics·B J PoorthuisO P van Diggelen
Jun 1, 2000·Bone Marrow Transplantation·C Peters, W Krivit
Feb 15, 2001·The New England Journal of Medicine·E D KakkisE F Neufeld
Mar 7, 2003·Bone Marrow Transplantation·C PetersUNKNOWN Working Party on Inborn Errors, European Bone Marrow Transplant Group
Jan 31, 2006·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·Paul L MartinJoanne Kurtzberg
Sep 18, 2008·Orphanet Journal of Rare Diseases·David MooreChristine Lavery
Jan 27, 2009·The Journal of Pediatrics·Nathalie GuffonRoseline Froissart
Apr 28, 2009·American Journal of Medical Genetics. Part a·Hsiang-Yu LinJu-Li Lin
Jun 27, 2009·Molecular Genetics and Metabolism·Ken ArayaKeiichi Ozono
Nov 10, 2009·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·Andrea BacigalupoMary Horowitz
Mar 24, 2010·Pediatric Clinics of North America·Jaap J BoelensCharles Peters
Mar 16, 2013·Blood·Jaap Jan BoelensUNKNOWN Centre for International Blood and Marrow Research
Jul 26, 2014·Molecular Genetics and Metabolism Reports·Pravin PatelShunji Tomatsu

❮ Previous
Next ❯

Citations

Feb 3, 2018·Journal of Inherited Metabolic Disease·Sandra SirrsEric M Yoshida
Nov 20, 2018·Journal of Human Genetics·Hiroshi Kobayashi
Jan 29, 2020·Geriatrics & Gerontology International·Yoh MurayamaYoshinori Fujiwara
Jun 10, 2019·Journal of Autism and Developmental Disorders·Yuma IshimotoYuki Matsumoto
Aug 4, 2019·Hypertension Research : Official Journal of the Japanese Society of Hypertension·Satoshi UmemuraNobuhito Hirawa
Jan 1, 2019·Bioscience, Biotechnology, and Biochemistry·Hiroshi HamajimaHiroshi Kitagaki
Feb 6, 2017·International Journal of Radiation Biology·Kotaro OzasaKazunori Kodama
Sep 24, 2016·Food Additives & Contaminants. Part A, Chemistry, Analysis, Control, Exposure & Risk Assessment·Naoki OchiHisashi Fujii
Nov 1, 2016·Therapeutic Innovation & Regulatory Science·Shoyo ShibataTakeshi Suzuki
Sep 12, 2019·Journal of Cross-cultural Gerontology·Rumiko Tsuchiya-ItoBjörn Slaug
Sep 18, 2016·Journal of Neuroscience Research·Norio Sakai, Takanobu Otomo
Feb 14, 2020·International Journal of Hematology·Katsutsugu UmedaUNKNOWN Inherited Disease Working Group of the Japan Society for Hematopoietic Cell Transplantation
Dec 12, 2018·Bone Marrow Transplantation·Katsutsugu UmedaUNKNOWN Inherited Disease Working Group of the Japan Society for Hematopoietic Cell Transplantation

❮ Previous
Next ❯

Related Concepts

Related Feeds

Blood And Marrow Transplantation

The use of hematopoietic stem cell transplantation or blood and marrow transplantation (bmt) is on the increase worldwide. BMT is used to replace damaged or destroyed bone marrow with healthy bone marrow stem cells. Here is the latest research on bone and marrow transplantation.

Alternative Complement Pathway

The Alternative Complement Pathway is part of the innate immune system, and activation generates membrane attack complexes that kill pathogenic cells. Discover the latest research on the Alternative Complement Pathway.

Allogenic & Autologous Therapies

Allogenic therapies are generated in large batches from unrelated donor tissues such as bone marrow. In contrast, autologous therapies are manufactures as a single lot from the patient being treated. Here is the latest research on allogenic and autologous therapies.