Abstract

Objective: Hematopoietic stem cell transplantation is recommended as a first-line treatment in a pediatric severe aplastic anemia, if the patient has an HLA-matched family donor. In this study, we aimed to evaluate the allogeneic hematopoietic stem cell transplantation outcomes of our patients who were followed with a diagnosis of aplastic anemia.

Material  and  Methods: From April 2010 to July 2017, 10 pediatric acquired aplastic anemia patients treated with allogeneic hematopoietic stem cell transplantation at our center were evaluated retrospectively from patient charts and computer records.

Results: A total of 12 allogeneic stem cell transplantations had been performed in 10 patients with severe and very severe acquired aplastic anemia. Mean age during transplantation was 10.9±3.76 (min=4.6-max=16.1) years and 80% of the patients were female. Mean interval from diagnosis to transplantation was 10.1±7.65 (min=3-max=31) months and all of the donors were siblings. The mean nucleated cell count was 4.69±2.17x108 (min=1.13x108-max=8.43x108) and CD34+ cell count was 3.25±1.39x106 (min=1.65x106-max=6.55x106). Mean neutrophil engraftment time was 16.2±0.8 (min=15-max=17) days and platelet engraftment time was 26±6.5 (min=16-max=37) days. Acute graft versus host disease was not observed in any patient and limited chronic pulmonary graft versus host disease was seen in only one patient. Primary graft failure developed in one patient and secondary graft failure in another patient. None of the patients developed myelodysplastic syndrome (MDS), leukemia, paroxysmal nocturnal hemoglobinuria (PNH) or secondary malignancy during follow-up. One patient died due to intracranial hemorrhage on day +16 after transplantation. Mixed chimerism was present in 66.7% and all patients are now followed up with complete hematological remission.

Conclusion: We achieved 90% overall survival in our patients over a 7-year follow-up period. Considering the high rates of relapse and clonal hematopoiesis after immünosuppressive treatment in studies reported in the literature, we believe that allogeneic hematopoietic stem cell transplantation can be used as a first-line treatment in children with aplastic anemia, especially if there is an HLA-compatible family donor.

Keywords: Child, Acquired aplastic anemia, Stem cell transplantation

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Copyright and license

Copyright © 2019 The Author(s). This is an open access article distributed under the Creative Commons Attribution License (CC BY), which permits unrestricted use, distribution, and reproduction in any medium or format, provided the original work is properly cited.

How to cite

1.
Işık P. Allogeneic Stem Cell Transplantation in Children with Acquired Aplastic Anemia. Turk J Pediatr Dis [Internet]. 2019 Mar. 26 [cited 2025 Aug. 23];13(2):57-62. Available from: https://turkjpediatrdis.org/article/view/620