AI Article Synopsis
- Bone marrow transplantation (BMT) is used to treat congenital metabolic diseases, but there is a risk of developing leukemia from intensive treatment.
- A case study details an 8-month-old girl with hypophosphatasia who received haploidentical BMT and multiple mesenchymal stem cell (MSC) transplants from her father, resulting in therapy-related leukemia by age 32 months.
- Despite this complication, a second round of BMT and MSC transplants at 40 months led to the girl's complete recovery, highlighting the complexities of treatment and potential risks associated with cytotoxic agents.
Article Abstract
Bone marrow (BM) transplantation (BMT) is one of the treatment strategies for congenital metabolic disease, but leukemia secondary to intensive cytoreductive treatment is a major concern. Besides BM cells, mesenchymal stem cells (MSC) are also used for transplantation. An 8-month-old girl with hypophosphatasia underwent transplantation of haploidentical BM cells followed by two transplants of MSC obtained from her father to facilitate osteogenesis. Fludarabine(Flu)/cyclophosphamide (CPA)/anti-thymocyte globulin were used for myeloablative conditioning, but the patient developed therapy-related leukemia harboring t(9;22)(q34;q11.2); minor BCR-ABL (t-leukemia with Ph) at the age of 32 months. At the age of 40 months she underwent a second BM and third MSC transplant from the same donor. Thereafter, she achieved complete histological and molecular remission. The present case suggests that the combination of cytotoxic agents (Flu/CPA) and MSC led to t-leukemia with Ph as a consequence of chromosome instability and suppression of host anti-tumor immunity.
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| http://dx.doi.org/10.1111/ped.12012 | DOI Listing |
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