AI Article Synopsis
- The study aimed to assess the effectiveness and safety of haploidentical HSCT along with BM-MSC transfusion for treating children with severe aplastic anemia (SAA) based on data from 25 cases.
- All 25 children achieved neutrophil engraftment, while 92% experienced platelet engraftment, though 64% developed acute graft-versus-host disease (aGVHD) and some complications like CMV viremia and infections occurred.
- The findings support that the combined treatment is safe and feasible, as nearly all children survived with a median follow-up of 14 months.
Article Abstract
Objective: To investigate the efficacy and safety of haploidentical hematopoietic stem cell transplantation(hi-HSCT) combined with bone-marrow derived mesenchymal stem cell (BM-MSC) tranfusion for treatment of children with severe apastic anemia(SAA).
Methods: The clinical data of 25 children with SAA undergoing hi-HSCT and BM-MSC tranfusion were retrospectively analyzed from August 2014 to July 2016.
Results: neutrophil engraftment was achieved in all 25(100%) children, with the median time 12(11-22) days. The median time of platelet engraftment was 21(11-130) days in 23(92%) children. Acute graft-versus-host disease(aGVHD) was observed in 16(64%) cases, including 11 case of grade I and 5 cases of aGVHD grade II-IV, and one of them died of grade IV of skin, gut and liver at day 115; 5 cases of chronic GVHD were found, all of them were limited cGVHD. Cytomegalovirus (CMV) viremia was observed in 23(92%) cases, but no CMV disease was developed after therapy. 3 cases of post-transplant lymphoroliferative disease with 23 EBV viremia positive occurred, all of them were cured after rituximab. Hemorrhagic cystitis appeared in 9 cases with only one case of grade III, 22 children suffered from infection, involving 10 cases in lung and 4 cases in liver, 1 patient was diagnosed as Guillain-Barre syndrome. Autoimmune hemolytic anemia was recorded in 1 patient, 22 children survived during a median following-up time of 14(3-27) months.
Conclusion: The hi-HSCT combined with BM-MSC transfusion for treatment of children with SAA has been confirmed to be safe and feasible.
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| http://dx.doi.org/10.7534/j.issn.1009-2137.2017.04.035 | DOI Listing |
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