Comprehensive and long-term outcomes of enzyme replacement therapy followed by stem cell transplantation in children with Gaucher disease type 1 and 3.

Autor: Anurathapan U; Division of Hematology and Oncology, Department of Pediatrics, Faculty of Medicine Ramathibodi Hospital, Mahidol University, Bangkok, Thailand., Tim-Aroon T; Division of Medical Genetics, Department of Pediatrics, Faculty of Medicine Ramathibodi Hospital, Mahidol University, Bangkok, Thailand., Zhang W; Department of Pathology and Laboratory Medicine, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA.; Department of Pediatrics, University of Cincinnati College of Medicine, Cinicnnati, Ohio, USA., Sanpote W; Department of Pediatrics, Lampang Hospital, Lampang, Thailand., Wongrungsri S; Department of Pediatrics, Lampang Hospital, Lampang, Thailand., Khunin N; Division of Medical Genetics, Department of Pediatrics, Faculty of Medicine Ramathibodi Hospital, Mahidol University, Bangkok, Thailand., Chutipongtanate S; Department of Environmental and Public Health Sciences, University of Cincinnati College of Medicine, Cincinnati, Ohio, USA., Chirdkiatgumchai V; Division of Developmental and Behavioral Pediatrics, Department of Pediatrics, Faculty of Medicine Ramathibodi Hospital, Mahidol University, Bangkok, Thailand., Ngiwsara L; Laboratory of Biochemistry, Chulabhorn Research Institute, Bangkok, Thailand., Jaovisidha S; Department of Diagnostic and Therapeutic Radiology, Faculty of Medicine Ramathibodi Hospital, Mahidol University, Bangkok, Thailand., Khongkraparn A; Division of Medical Genetics, Department of Pediatrics, Faculty of Medicine Ramathibodi Hospital, Mahidol University, Bangkok, Thailand., Pakakasama S; Division of Hematology and Oncology, Department of Pediatrics, Faculty of Medicine Ramathibodi Hospital, Mahidol University, Bangkok, Thailand., Svasti J; Laboratory of Biochemistry, Chulabhorn Research Institute, Bangkok, Thailand., Setchell KDR; Department of Pathology and Laboratory Medicine, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA.; Department of Pediatrics, University of Cincinnati College of Medicine, Cinicnnati, Ohio, USA., Wattanasirichaigoon D; Division of Medical Genetics, Department of Pediatrics, Faculty of Medicine Ramathibodi Hospital, Mahidol University, Bangkok, Thailand., Hongeng S; Division of Hematology and Oncology, Department of Pediatrics, Faculty of Medicine Ramathibodi Hospital, Mahidol University, Bangkok, Thailand.

Publikováno v: Pediatric blood & cancer [Pediatr Blood Cancer] 2023 Mar; Vol. 70 (3), pp. e30149. Date of Electronic Publication: 2022 Dec 23.