Hematopoietic Stem Cell Transplant in Pediatric Sickle Cell Disease: The Cook Children’s Experience

Background Current treatments for sickle cell disease are mainly supportive and are aimed at symptom control rather than curing the disease. The only known cure for sickle cell disease is hematopoietic stem cell transplantation. Successful transplantation has been found to eliminate further end-organ damage related to sickle cell disease. A study of transplant experiences and assessment of outcomes could potentially improve the future management of sickle cell disease patients. Purpose The objective of this study was to evaluate a single institution experience of patients undergoing hematopoietic stem cell transplantation at Cook Children’s Medical Center for treatment of Sickle Cell Disease. The data was analyzed to assess indications and outcomes of the transplanted sickle cell patients in order to evaluate transplant as a viable treatment for sickle cell disease. Materials and Methods A comprehensive retrospective chart review was performed on all patients who received a hematopoietic stem cell transplant for the treatment of Sickle Cell Disease from March 25, 1999 to May 24, 2013 at Cook Children’s Medical Center. The clinical variables collected consisted of demographic data, disease and medication history, transplant indications and preparations, donor source, engraftment information, and post-transplant complications. All electronic data was stored on Cook Children’s server using REDCap database. Results Data was collected on 20 patients who met inclusion criteria. Thrombotic stroke was the main clinical indication for transplant. All 20 patients continue to survive post-transplant. Sixteen patients achieved successful long term engraftment. These patients had a median period of follow-up of 4.5 years, ranging from 11 months to 9 years 1 month. Two patients received a 2nd transplant with subsequent failure to engraft. Fourteen developed graft versus host disease, 11 had neurological symptoms, 17 acquired infection, and 1 was found to have intracranial hemorrhage post- transplant. Discussion Preliminary results support the use of hematopoietic stem cell transplant as a viable treatment for sickle cell disease. The survival rate for patients as well as the successful engraftment rate following first transplant were suggestive of the viability of stem cell transplant. Further analysis of post transplant complications, specifically delineating graft versus host disease in relation to organ system and grade, will aid in improving management of these patients.