Allogeneic hematopoietic stem cell transplantation for Chediak-Higashi syndrome.
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.
Department of Pediatrics, Graduate School of Medicine, Kyoto University, Kyoto, Japan.
Katsutsugu
K
Umeda
Human Health Sciences, Graduate School of Medicine, Kyoto University, Kyoto, Japan.
Souichi
S
Adachi
Division of Hematology and Oncology, Shizuoka Children's Hospital, Shizuoka, Japan.
Yasuo
Y
Horikoshi
Department of Community Pediatrics, Perinatal and Maternal Medicine, Tokyo Medical and Dental University, Tokyo, Japan.
Kohsuke
K
Imai
Department of Pediatrics, Hirosaki University Graduate School of Medicine, Hirosaki, Japan.
Kiminori
K
Terui
Department of Pediatrics, Iwate Medical University, Morioka, Japan.
Mikiya
M
Endo
Department of Pediatrics, Yamagata University School of Medicine, Yamagata, Japan.
Tetsuo
T
Mitsui
Division of Pediatric Hematology and Oncology, Ibaraki Children's Hospital, Mito, Japan.
Keisuke
K
Kato
Department of Hematology/Oncology, Saitama Children's Medical Center, Saitama, Japan.
Katsuyoshi
K
Koh
Department of Pediatrics, Yokohama City University School of Medicine, Yokohama, Japan.
Ryosuke
R
Kajiwara
Division of Hematology and Oncology, Shizuoka Children's Hospital, Shizuoka, Japan.
Rieko
R
Ito
Department of Pediatrics, Hyogo College of Medicine, Nishinomiya, Japan.
Yoshitoshi
Y
Otsuka
Department of Hematology/Oncology, Osaka Medical Center and Research Institute for Maternal and Child Health, Izumi, Japan.
Masami
M
Inoue
Department of Pediatrics, Ehime University Graduate School of Medicine, Toon, Japan.
Eiichi
E
Ishii
Department of Cell Transplantation and Regenerative Medicine, Tokai University School of Medicine, Isehara, Japan.
Hiromasa
H
Yabe
D000328:Adult; D002609:Chediak-Higashi Syndrome / Q000628:therapy*; D002648:Child; D002675:Child, Preschool; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D051359:Lymphohistiocytosis, Hemophagocytic / Q000628:therapy; D008279:Magnetic Resonance Imaging; D008297:Male; D012189:Retrospective Studies; D019172:Transplantation Conditioning; D014184:Transplantation, Homologous; D016896:Treatment Outcome; D055815:Young Adult
Chediak-Higashi syndrome; hematopoietic stem cell transplantation; reduced-intensity conditioning