Hematology

Survey data reveal strong support for gene therapy in sickle cell disease, but inconsistent referral practices, limited knowledge, and insurance barriers hinder implementation.

Liquid biopsy significantly reduced the median turnaround time from 46.8 days to just 6.5 days, offering a substantial improvement in diagnostic efficiency.

Castellucci and colleagues identified that a primary-outcome event had occurred in 44 (3.3%) of the 1,345 patients taking apixaban, compared to 96 (7.1%) of the 1,355 in the rivaroxaban group.

A large retrospective study identified risk factors for CNS relapse after transplant in adults with acute lymphoblastic leukaemia, highlighting elevated risk in Philadelphia chromosome positive disease.

A study identifies the ribosome biogenesis factor MYBBP1A as essential for acute myeloid leukaemia cell growth, revealing a potential therapeutic vulnerability.

Analysis of patient liver biopsies and cell models suggests epigenetic silencing may limit durability of AAV gene therapy, offering insight into long term variability in haemophilia treatment.

A transcriptional classifier identifies tumour reactive T cells in multiple myeloma and predicts response to immunotherapy, offering a potential biomarker for treatment selection.

A new study identifies a paralog dependency regulating MEF2C stability in acute myeloid leukaemia and demonstrates a peptide approach that suppresses disease progression in preclinical models.