Allogeneic hematopoietic stem cell transplantation for children with severe aplastic anemia

Division ofHematology-Oncology, Department ofPediatrics, Faculty ofMedicine, Srinakharinwirot University Nakhon Nayok, Thailand.
Journal of the Medical Association of Thailand = Chotmaihet thangphaet 06/2013; 96 Suppl 1:S18-24.
Source: PubMed


Allogeneic hematopoietic stem cell transplantation (allo-SCT) is a potentially curative treatment for severe aplastic anemia (SAA). This is a single institutional review to study the feasibility of using allo-SCT for Thai children with SAA.
Nine children with SAA (7 matched-sibling donor-SCT, 1 matched-unrelated donor-SCT and 1 haploidentical-SCT) underwent allo-SCT between October 2002 and September 2010. Cyclophosphamide and anti-thymocyte globulin (CY/ATG) were used as conditioning regimen for 4 patients with matched-sibling donor-SCT CY/ATG and fludarabine were used for 3 patients with matched-sibling donor-SCT and one patient with haplo-identical SCT. One matched-unrelated donor-SCT received CY/ATG and total body irradiation.
Eight of 9 patients (89%) achieved neutrophil engraftment within 13.5 days (range 6.0-22.0). One matched-sibling donor-SCT recipient who failed to achieve engraftment died from acute renal failure and gram-negative sepsis on day 21 post allo-SCT. One matched-sibling donor-SCT case developed late graft failure on day 72 and died from invasive fungal infection. For graft versus host disease (GVHD), a haplo-identical-SCT patient died from steroid refractory grade IV acute GVHD. At last follow-up, six patients (67%) alive at a median follow-up time of 76.4 months (range 2.3-88.8). Overall survival (OS) and event-free survival (EFS) at 5 year was 63% and 65%, respectively.
Allo-SCT is a feasible curative treatment for children with SAA in Thailand. Graft failure and severe GVHD in alternative donors SCT are responsible for major causes of death. OS and EFS probabilities are stable after the first year post transplant.

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    ABSTRACT: The treatment of pediatric severe aplastic anemia (SAA) with allogeneic hematopoietic stem cell transplantation (allo-HSCT), presents major challenges including the risks of graft failure, septic complications, and graft-versus-host disease (GVHD). Additive infusions of human umbilical cord derived mesenchymal stem cell (hUC-MSC) may be administered to improve patient survival. We retrospectively examined 37 pediatric patients with SAA who received allo-HSCT and subsequent infusions of hUC-MSC suspension at a dose of 1.0 × 10(6 )/kg. The times and doses of hUC-MSC infusions were increased in patients with severe GVHD. All patients received hUC-MSC infusions. The median time to post-transplantation neutrophil count of greater than 0.5 × 10(9 )/L was 14 days (range, 11-20 days) and time to post-transplantation platelet count of greater than 20 × 10(9 )/L was 19 days (14-29 days). The overall frequency of acute GVHD (aGVHD) was 45.9% (17/37). These aGVHD episodes occurred at a median time of post-transplantation 47 days (15-83 days). The frequency of chronic GVHD (cGVHD) was 18.9% (7/37); cGVHD developed from aGVHD in 10.8% (4/37) of patients. The GVHD-associated mortality rate was 18.9% (7/37) and aGVHD-specific mortality rate was 8.1% (3/37). The median overall survival time was 35 months (9-67 months) and the three-year overall survival rate was 74.2% (28/37). Seven patients died of GVHD, one patient died of a severe invasive fungal infection, and one patient died of renal failure. In conclusion, post-transplantation hUC-MSC infusions seemed to be safely infused in children with SAA who have previously received allo-HSCT.
    Pediatric Hematology and Oncology 01/2014; 31(1). DOI:10.3109/08880018.2013.867556 · 1.10 Impact Factor