Allogeneic hematopoietic stem cell transplantation for Chediak–Higashi syndrome. (29th October 2015)

• Record Type: Journal Article
• Authors: Umeda, Katsutsugu; Adachi, Souichi; Horikoshi, Yasuo; Imai, Kohsuke; Terui, Kiminori; Endo, Mikiya; Mitsui, Tetsuo; Kato, Keisuke; Koh, Katsuyoshi; Kajiwara, Ryosuke; Ito, Rieko; Otsuka, Yoshitoshi; Inoue, Masami; Ishii, Eiichi; Yabe, Hiromasa
• Is Part Of: Pediatric transplantation. Volume 20:Number 2(2016:Mar.)
• Page Start: 271
• Page End: 275
• Publication Date: 2015-10-29
• Subjects: Chediak–Higashi syndrome -- hematopoietic stem cell transplantation -- reduced‐intensity conditioning; Transplantation of organs, tissues, etc. in children -- Periodicals; 617.95408305
• DOI: 10.1111/petr.12626
• Languages: English
• ISSNs: 1397-3142

Abstract

Abstract: The clinical outcome of allogeneic HSCT was retrospectively analyzed in eight patients with CHS. In total, six of these eight patients are alive. Four of five patients transplanted with MAC achieved prompt engraftment, and three of the four patients, including two patients with AP before transplant, are alive without disease. In contrast, three patients transplanted with RIC without active AP disease achieved prompt engraftment and survive long term. RIC‐HSCT might be an alternative treatment for CHS similar to other types of HLH, at least for patients without active AP disease.