Transforming growth factor beta receptor type I (TGF-βRI) kinase inhibitors IOA-359 and IOA-360 stimulate erythropoiesis in MDS.

Overactivation of the Transforming Growth Factor Beta (TGF-β) pathway is implicated in the pathogenesis of cytopenias in Myelodysplastic syndromes (MDS) and Acute Myeloid Leukemia (AML). IOA-359 and IOA-360 are potent small molecule inhibitors of the TGF-beta Receptor type I kinase (TGF-βRI, also referred to as ALK5, activin receptor-like kinase 5) that abrogate SMAD phosphorylation in hematopoietic cell lines. Both inhibitors were able to inhibit TGF-β mediated gene transcription at specific doses.

Investigating home-based opioid use among youth with sickle cell disease using ecological momentary assessment.

Background: Opioids are a common and essential treatment for acute sickle cell disease (SCD) pain. However, opioids carry well-known adverse side effects, including potential development of hyperalgesia and nociplastic pain. We characterized opioid use in youth with SCD using ecological momentary assessment (EMA) data, and investigated the relationships between home-based opioid use, pain, and a range of biopsychosocial factors.