Early initiation of low-dose gilteritinib maintenance improves posttransplant outcomes in patients with R/R FLT3mut AML
This article by Terao et al provides insights into the efficacy of gilteritinib as a posttransplant maintenance therapy in patients with acute myeloid leukemia with relapsed or refractory fms-like tyrosine kinase 3 mutations (R/R FLT3mut AML).
CD14+/CD31+ monocytes expanded by UM171 correct hemophilia A in zebrafish upon lentiviral gene transfer of factor VIII
This article by Elnaggar and colleagues extends the analysis of native and transgenic factor VIII to a zebrafish model and supports the premise of restricted expression to the monocyte lineage, currently being pursued in US and India trials.
Downregulated KLF2 in polycythemia vera and essential thrombocythemia induces prothrombotic gene expression
Thrombosis in polycythemia vera (PV) and essential thrombocythemia (ET) are regulated by Kruppel-like factor 2 (KLF2), a transcription factor for inflammation and thrombosis. Even though JAK-STAT signaling in PV and ET is downregulated by KLF2, it is only the downregulation of KLF2 in platelets but not leukocytes that correlates with thrombotic risk. These findings by Song et al suggest that other factors (ie, inflammatory factors) are involved in PV/ET thrombotic risk which are the focus of future studies.
Second malignancies among older patients with classical myeloproliferative neoplasms treated with hydroxyurea
Controversy exists as to whether hydroxyurea use is associated with a higher risk of secondary myeloid malignancies. The conclusions by Wang and colleagues, based on data from a large SEER database (that is independently analyzed), indicates that hydroxyurea therapy does not lead to the development of secondary malignancies including acute myeloid leukemia and myelodysplastic syndrome.
HLA-DR expression on monocytes and outcome of anti-CD19 CAR T-cell therapy for large B-cell lymphoma
Bourbon et al have evaluated the use of HLA-DR expression on monocytes as a predictive biomarker for patients receiving anti-CD19 chimeric antigen receptor (CAR) T-cell therapy. Due to the related efforts and costs of producing patient-specific CAR T-cell therapies, biomarkers are certainly needed to help optimize treatment decisions. This is the largest study of HLA-DR expression on monocytes in patients receiving CD19 CAR T cells, and this is of interest since biomarkers are needed to optimize treatment decisions regarding whether or not patients should receive CAR T cells.
The safety and efficacy of N8-GP (turoctocog alfa pegol) in previously untreated pediatric patients with hemophilia A
Königs et al present the first report of a glycoPEGylated extended half-life (EHL) factor VIII replacement product (N8-GP) used for the treatment of previously untreated patients (PUPs) with severe hemophilia A. This report is interesting as the prospect of using EHL products in PUPs is still new, and further data is needed to guide clinicians and researchers in best clinical practices and new areas for research in hemophilia A. Although the authors identified a previously undescribed decrease in incremental recovery, it returned to normal with continued N8-GP dosing. They conclude that N8-GP prophylaxis is efficacious for the prevention and treatment of bleeding episodes in PUPs with severe hemophilia.