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Haploidentical family donor hematopoietic stem cell transplantation (Haplo-HSCT) has been increasingly used for patients who require transplant for optimum therapy, but lack a human leukocyte antigen (HLA)-matched donor. Important variables that require consideration in choosing one of potentially many haplo-donors include evaluation for presence of donor-specific anti-HLA antibodies, the age and gender of the donor, the parental relationship of the donor especially for pediatric patients, and ABO compatibility. Three major platforms have been reported as valid methods of undertaking haplo-HSCT. Ex vivo T cell depleted transplant may allow for low rates of graft-versus-host disease, even without pharmacological prophylaxis after transplant. Major impediments such as delayed immune recovery and subsequent infections may be overcome by depletion of specific T cell subsets, as done in αβ T cell/B cell depleted transplants. Alternatively, T cell replete haplo-HSCT may be undertaken with post-transplantation cyclophosphamide, or by administering intensive pre- and post-transplant immunosuppression including use of anti-thymocyte globulin. All three haplo-HSCT platforms have been successfully used to treat children with hematologic malignancies. As this method of HSCT is relatively new, long-term follow-up is necessary to improve outcomes and clarify the toxicities of this transplant modality.
Flow chart of the febrile neutropenic episodes enrolled in the present study. PIPC/TAZ, pi-peracillin/tazobactam; MEPM, mero-penem; IVIG, intravenous immuno-globulin.
(A) The computed tomo-graphy of the patient’s brain at admission shows a 6.5 cm acute intracranial hemorrhage in the left frontal lobe with perilesional edema and midline shifting to the right. (B) An initial peripheral blood smear reveals left-shifted myeloid hyper-plasia with dysplasia (myeloblasts 2%, myelocytes 20% and metamy-elocytes 3% of the leukocytes) (Weight-Giemsa stain, ×400). (C) A bone marrow smear reveals in-creased granulocytic series with 8.7% myeloblasts and dysgranulo-poiesis (Weight-Giemsa stain, ×400). (D) A follow-up bone marrow smear of the patient 3 months after the initial evaluation shows increased myeloblasts of 14% (Weight-Giemsa stain, ×400). (E) Magnetic reson-ance imaging of brain 11 months later shows en-cephalomalacic change with hemo-siderin deposit in left frontal lobe and corpus callosum, sequelae of previous intracranial hemorrhage.
Clin Pediatr Hematol Oncol 2021;28:67-102 |
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