Primary myelofibrosis
HTML-код
- Опубликовано: 17 май 2022
- The bone marrow is the place where all blood cells are manufactured and released to the blood stream.
Myelofibrosis means that the bone marrow becomes fibrotic and is no longer a suitable place for blood cells to be produced.
So, our body used other organs, such as spleen or liver to produce blood cells.
However, in these organs other than the bone marrow blood cell productions are not efficient enough to supply the blood cells to meet the body's demand.
New drug approval: Momelotinib
On September 15, 2023, the FDA approved momelotinib for the treatment of patients with intermediate- or high-risk myelofibrosis, including primary or secondary myelofibrosis, and disease-related anemia.Findings from the phase 3 MOMENTUM trial (NCT04173494), as well as data from a subpopulation of adult patients with anemia from the phase 3 SIMPLIFY-1 trial (NCT01969838), supported the regulatory decision.
Twenty-five percent of patients with symptomatic and anemic myelofibrosis who were previously exposed to a JAK inhibitor experienced at least a 50% reduction in tumor symptom score (TSS) with momelotinib vs 9% of patients treated with danazol, which translated to a treatment difference of 16% (95% CI, 6%-26%; P < .01). Additionally, 30% of patients in the momelotinib arm achieved transfusion independence (TI) vs 20% of those in the danazol arm, which translated to a noninferiority treatment difference of 14% (95% CI, 2%-25%; P = .023). Spleen volume reduction and a decrease in anemia-related symptoms were also observed with momelotinib.
At 22:40, I wrongly described that grape juice is a strong CYP3A4 inhibitor. It is grapefruit juice, not grape juice.
New 5th edition of WHO classification was published.
No major changes in PMF.
www.nature.com/articles/s41375-022-01613-1#Tab8