Allogeneic hematopoietic stem cell transplantation for Chediak-Higashi syndrome

作者：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

来源：Pediatric Transplantation, 2016, 20(2): 271-275.

DOI：10.1111/petr.12626

• Chediak-Higashi syndrome
• hematopoietic stem cell transplantation
• reduced-intensity conditioning

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.

• 出版日期2016-3