Hematopoietic SCT in children with Griscelli syndrome: a single-center experience

Bone Marrow Transplant. 2010 Aug;45(8):1294-9. doi: 10.1038/bmt.2009.358. Epub 2010 Jan 11.

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.

MeSH terms

  • Child, Preschool
  • Cytomegalovirus Infections
  • Graft vs Host Disease
  • Hematopoietic Stem Cell Transplantation / adverse effects
  • Hematopoietic Stem Cell Transplantation / methods*
  • Hematopoietic Stem Cell Transplantation / mortality
  • Histocompatibility
  • Humans
  • Immunologic Deficiency Syndromes / complications
  • Immunologic Deficiency Syndromes / therapy
  • Infant
  • Lymphohistiocytosis, Hemophagocytic
  • Piebaldism / complications
  • Piebaldism / therapy
  • Primary Immunodeficiency Diseases
  • Survival Analysis
  • Treatment Outcome

Supplementary concepts

  • Griscelli syndrome type 2