Associations between hematology/oncology fellows' training and mentorship experiences and hematology-only career plans
There are significant concerns relating to the training of hematologists to provide sufficient numbers of skilled practitioners to treat an aging population. Masselink and colleagues explored the impact of mentorship during the training of hematology and oncology fellows, providing interesting data on how these relationships influence career choices.
Rational design of a trimeric APRIL-based CAR-binding domain enables efficient targeting of multiple myeloma
Chimeric antigen receptor T cells directed against B-cell maturation antigens is a promising approach to the treatment of relapsed refractory multiple myeloma. However, escape mutants have been identified, similar to what has been observed in the treatment of patients with B-cell lymphomas. Maus and colleagues developed a novel approach to target multiple antigens that may overcome this limitation of achieving durable responses.
Analysis of the cost-effectiveness of treatment strategies for CML with incorporation of treatment discontinuation
Tyrosine kinase inhibitors have revolutionized the care of patients with chronic myeloid leukemia. However, because multiple choices are available, there remains an ongoing question of the most cost-effective strategy for initial treatment. The analysis by Kanda and colleagues reports interesting and important findings analyzing this question, including situations in which tyrosine kinase inhibitors could be safely discontinued.
Etranacogene dezaparvovec (AMT-061 Phase 2b): normal/near normal FIX activity and bleed cessation in hemophilia B
Gene therapy for patients with hemophilia is an area of intense interest and investigation. Pipe and colleagues explored the use of etranacogene dezaparvovec, a recombinant AAV5 vector encoding factor IX under the control of a liver-specific promoter. These highly encouraging results in 3 patients set the stage for larger studies, which are eagerly anticipated.
Induction of fetal hemoglobin production is an attractive alternative for the treatment of hemoglobinopathies such as sickle cell anemia and thalassemia. Weiss and colleagues explored CRISPR-Cas9–mediated disruption of DNA regulatory elements to enhance fetal hemoglobin production, providing preclinical evidence to support this approach.
It is well recognized that while patients with the factor V leiden variant are more prone to developing deep venous thrombosis, they do not have an increased proportional risk of pulmonary embolus. To explain this paradox, Gross and colleagues explored deep vein thrombosis and pulmonary embolism development in wild-type mice as compared to factor V leiden heterozygous and homozygous mice. They reach interesting findings that may help explain this clinical observation.