title: Hematopoietic SCT in children with Griscelli syndrome: a single-center experience.
abstract: In total, 11 consecutive pediatric patients with Griscelli syndrome (GS) type 2, who received allogeneic hematopoietic SCT (aHSCT) at our center between 1993 and 2007, were reviewed. The median age at transplantation was 8.2 months (range, 4-36.3 months) and the median time from diagnosis to transplantation was 3.7 months (range, 1.4-19.5 months). Seven patients developed an accelerated phase and were treated with chemotherapy before transplantation. At the time of transplantation, all patients were in clinical remission. The source of grafts was matched-related marrows in eight patients and partially mismatched unrelated cords in three patients. All patients were engrafted at a median time of 15 days (range, 12-36 days). Grade I-II acute GVHD and veno-occlusive disease occurred in three and one patient, respectively. A total of 10 patients are now alive and disease free at a median of 4.8 years post-HSCT. The post transplant course was complicated by CMV infection in four patients. One patient died in remission from septic shock, 6 months after transplantation. Chimerism studies at the last contact are available for nine patients: six patients have complete donor chimerism and three have stable mixed chimerism. Early aHSCT from matched-related donors or unrelated cord blood for children with GS is feasible.
authors.affiliation: Department of Pediatric Hematology/Oncology, King Faisal Specialist Hospital & Research Center, Riyadh, Saudi Arabia. aahmari@kfshrc.edu.sa
authors.forename: A
authors.initials: A
authors.lastname: Al-Ahmari
authors.forename: A
authors.initials: A
authors.lastname: Al-Ghonaium
authors.forename: M
authors.initials: M
authors.lastname: Al-Mansoori
authors.forename: A
authors.initials: A
authors.lastname: Hawwari
authors.forename: A
authors.initials: A
authors.lastname: Eldali
authors.forename: M
authors.initials: M
authors.lastname: Ayas
authors.forename: H
authors.initials: H
authors.lastname: Al-Mousa
authors.forename: A
authors.initials: A
authors.lastname: Al-Jefri
authors.forename: B
authors.initials: B
authors.lastname: Al-Saud
authors.forename: A
authors.initials: A
authors.lastname: Al-Seraihy
authors.forename: S
authors.initials: S
authors.lastname: Al-Muhsen
authors.forename: M
authors.initials: M
authors.lastname: Al-Mahr
authors.forename: H
authors.initials: H
authors.lastname: Al-Dhekri
authors.forename: H
authors.initials: H
authors.lastname: El-Solh
mesh_terms: D002675:Child, Preschool; D003586:Cytomegalovirus Infections; D006086:Graft vs Host Disease; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods* / Q000401:mortality; D006648:Histocompatibility; D006801:Humans; D007153:Immunologic Deficiency Syndromes / Q000150:complications / Q000628:therapy; D007223:Infant; D051359:Lymphohistiocytosis, Hemophagocytic; D016116:Piebaldism / Q000150:complications / Q000628:therapy; D000081207:Primary Immunodeficiency Diseases; D016019:Survival Analysis; D016896:Treatment Outcome