Allogeneic Hematopoietic Stem Cell Transplantation For Treatment Of Congenital Hereditary Diseases In Hematopoietic System

Objective: To observe the outcome and analyze the prognostic factors of hematopoietic stem cell transplantation(HSCT) in children with congenital hereditary diseases(CHD) in hematopoietic system.Methods: Fifteen patients with CHD accepted HSCT in our medical institute from February 2015 to March 2016 and were followed up until April 2016, which included 11 patients with Wiskott-Aldrich syndrome(WAS) disease, 3 with Fanconi Anemia(FA), and1 with severe congenital neutropenia(SCN). Nine patients with WAS and 1 patient with SCN accepted unrelated cord blood transplantation(UCBT), 2 patients with WAS accepted haploidentical stem cell transplantation, 2 patient with FA accepted matched unrelated donor transplantation and the other accepted matched sibling transplantation. The nutritional status before transplantation, age of transplantation, HLA matched degree,conditioning regimen, post-transplantation graft versus host disease(GVHD), infection status were analyzed.Results: Ten patients with WAS succeeded in transplantation. The median time of granulocyte engraftment was 12.5 days, ranging from 11 to 15 days. The median time of platelet engraftment was 35 days and the median time of STR>95% was 17 days. Five patients suffered from acute graft versus host disease(aGVHD), namely 3 with skin GVHD(I), and 1 with intestinal GVHD(I), 1 with both GVHD in skin and intestine. Four of them had severe pneumonia,2 had hemorrhagic cystitis(HC), 5 had Cytomegalovirus(CMV) viremia, and 1 had CMV retinopathy. None of them had veno-occlusive disease(VOD). Three patients with FA succeeded in engraftment as well. The time of granulocyte engraftment was 12 days, 9 days and 10 days, respectively. The time of platelet engraftment was 26 days, 7 days and 13 days, respectively. The time of STR>95% was 18 days, 20 days and 23 days, respectively. None of them suffered from GVHD. One patient suffered of several lethal complications like severe pneumonia, hemorrhagic cystitis,hypertensive encephalopathy, cerebral hernia, dropsy of serous cavity, hemorrhage of digestive tract, iatrogenic diabetes and CMV viremia. The patients with SCN succeeded in UCBT. The time of granulocyte engraftment was 14 days. The time of platelet engraftment was 38 days. The time of STR>95% was 12 days. He had skin GVHD(II) and CMV viremia. However, he didn’t have HC or VOD or other serious infection. The median time of follow-up was 110 days and 14 patients were survived with one patient with WAS died before engraftment.Conclusion: Allo-HSCT is the most effective method to cure congenital hereditary diseases of hematopoietic system.