Background: Severe hemophilia A is managed with factor VIII replacement or hemostatic products that stop or prevent bleeding. Data on gene therapy with hematopoietic stem-cell (HSC)-based expression of factor VIII for the treatment of severe hemophilia A are lacking.
Methods: We conducted a single-center study involving five participants 22 to 41 years of age with severe hemophilia A without factor VIII inhibitors.
Granulocyte transfusions (GTx) combat infections in neutropenic patients. However, immune-mediated off-target effects in transplant settings are unknown. Between January 2020 and December 2021, all transplants that used GTx during the peri-transplant period were analysed.
View Article and Find Full Text PDFTotal marrow and lymphoid irradiation (TMLI) can deliver higher doses of irradiation without increased toxicity. This study evaluated TMLI and cyclophosphamide in patients undergoing stem cell transplantation for acute lymphoblastic leukemia (ALL). A total of 58 patients underwent matched related, unrelated, or haplo-identical donor transplantation using TMLI.
View Article and Find Full Text PDFBackground: The karyotype is a major determinant of prognosis in myelodysplastic syndrome (MDS). Details of the cytogenetic profile of MDS in South Asia are limited because cytogenetic services are not widely available.
Methods: We performed a retrospective analysis of the cytogenetic and clinicopathologic profile of adult primary MDS seen consecutively at a tertiary-care centre in South India between 2003 and 2017.