Hematopoietic stem cell transplantation in children with Griscelli syndrome: A single-center experience
Corresponding Author
Baris Kuskonmaz
Division of Bone Marrow Transplantation, Department of Pediatrics, Hacettepe University Faculty of Medicine, Ankara, Turkey
Correspondence
Baris Kuskonmaz, Division of Bone Marrow Transplantation, Department of Pediatrics, Hacettepe University Faculty of Medicine, Ankara, Turkey.
Email: [email protected]
Search for more papers by this authorDeniz Ayvaz
Division of Immunology, Department of Pediatrics, Hacettepe University Faculty of Medicine, Ankara, Turkey
Search for more papers by this authorMuge Gokce
Division of Hematology, Department of Pediatrics, Hacettepe University Faculty of Medicine, Ankara, Turkey
Search for more papers by this authorTuba Turul Ozgur
Division of Immunology, Department of Pediatrics, Hacettepe University Faculty of Medicine, Ankara, Turkey
Search for more papers by this authorFatma V. Okur
Division of Bone Marrow Transplantation, Department of Pediatrics, Hacettepe University Faculty of Medicine, Ankara, Turkey
Search for more papers by this authorMualla Cetin
Division of Hematology, Department of Pediatrics, Hacettepe University Faculty of Medicine, Ankara, Turkey
Search for more papers by this authorIlhan Tezcan
Division of Immunology, Department of Pediatrics, Hacettepe University Faculty of Medicine, Ankara, Turkey
Search for more papers by this authorDuygu Uckan Cetinkaya
Division of Bone Marrow Transplantation, Department of Pediatrics, Hacettepe University Faculty of Medicine, Ankara, Turkey
Search for more papers by this authorCorresponding Author
Baris Kuskonmaz
Division of Bone Marrow Transplantation, Department of Pediatrics, Hacettepe University Faculty of Medicine, Ankara, Turkey
Correspondence
Baris Kuskonmaz, Division of Bone Marrow Transplantation, Department of Pediatrics, Hacettepe University Faculty of Medicine, Ankara, Turkey.
Email: [email protected]
Search for more papers by this authorDeniz Ayvaz
Division of Immunology, Department of Pediatrics, Hacettepe University Faculty of Medicine, Ankara, Turkey
Search for more papers by this authorMuge Gokce
Division of Hematology, Department of Pediatrics, Hacettepe University Faculty of Medicine, Ankara, Turkey
Search for more papers by this authorTuba Turul Ozgur
Division of Immunology, Department of Pediatrics, Hacettepe University Faculty of Medicine, Ankara, Turkey
Search for more papers by this authorFatma V. Okur
Division of Bone Marrow Transplantation, Department of Pediatrics, Hacettepe University Faculty of Medicine, Ankara, Turkey
Search for more papers by this authorMualla Cetin
Division of Hematology, Department of Pediatrics, Hacettepe University Faculty of Medicine, Ankara, Turkey
Search for more papers by this authorIlhan Tezcan
Division of Immunology, Department of Pediatrics, Hacettepe University Faculty of Medicine, Ankara, Turkey
Search for more papers by this authorDuygu Uckan Cetinkaya
Division of Bone Marrow Transplantation, Department of Pediatrics, Hacettepe University Faculty of Medicine, Ankara, Turkey
Search for more papers by this authorAbstract
GS2 is a rare autosomal recessive disease characterized by hypopigmentation, variable immunodeficiency with HLH. HSCT is the only curative treatment for GS2. We analyzed the outcome of 10 children with GS2 who underwent HSCT at our center between October 1997 and September 2013. The median age of the patients at transplant was 13.5 months (range, 6-58 months). All of the patients developed HLH before HSCT and received HLH 94 or HLH 2004 protocols. Donors were HLA-identical relatives in 8 patients, HLA-mismatched relatives in 2 patients. Engraftment was achieved in all except one patient. None of the patients developed acute GVHD. Chronic GVHD occurred in one and veno-occlusive disease occurred in four patients. Eight of the patients are under remission without any neurologic sequelae—median time of disease-free survival is 92.4 months. The present study shows successful transplant outcome without long-term neurologic sequelae in patients with GS2 who underwent HSCT from HLA-related donors.
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