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Allogeneic hematopoietic stem cell transplantation for Chediak–Higashi syndrome
Authors:Katsutsugu Umeda  Souichi Adachi  Yasuo Horikoshi  Kohsuke Imai  Kiminori Terui  Mikiya Endo  Tetsuo Mitsui  Keisuke Kato  Katsuyoshi Koh  Ryosuke Kajiwara  Rieko Ito  Yoshitoshi Otsuka  Masami Inoue  Eiichi Ishii  Hiromasa Yabe
Affiliation:1. Department of Pediatrics, Graduate School of Medicine, Kyoto University, Kyoto, Japan;2. Human Health Sciences, Graduate School of Medicine, Kyoto University, Kyoto, Japan;3. Division of Hematology and Oncology, Shizuoka Children's Hospital, Shizuoka, Japan;4. Department of Community Pediatrics, Perinatal and Maternal Medicine, Tokyo Medical and Dental University, Tokyo, Japan;5. Department of Pediatrics, Hirosaki University Graduate School of Medicine, Hirosaki, Japan;6. Department of Pediatrics, Iwate Medical University, Morioka, Japan;7. Department of Pediatrics, Yamagata University School of Medicine, Yamagata, Japan;8. Division of Pediatric Hematology and Oncology, Ibaraki Children's Hospital, Mito, Japan;9. Department of Hematology/Oncology, Saitama Children's Medical Center, Saitama, Japan;10. Department of Pediatrics, Yokohama City University School of Medicine, Yokohama, Japan;11. Department of Pediatrics, Hyogo College of Medicine, Nishinomiya, Japan;12. Department of Hematology/Oncology, Osaka Medical Center and Research Institute for Maternal and Child Health, Izumi, Japan;13. Department of Pediatrics, Ehime University Graduate School of Medicine, Toon, Japan;14. Department of Cell Transplantation and Regenerative Medicine, Tokai University School of Medicine, Isehara, Japan
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.
Keywords:Chediak–  Higashi syndrome  hematopoietic stem cell transplantation  reduced‐intensity conditioning
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