Myelofibrosis

Navtemadlin has demonstrated meaningful efficacy and safety as a monotherapy or combination treatment in various preclinical and clinical trials.

Pelabresib is an investigational, oral, small molecule that inhibits bromodomain and extraterminal (BET) proteins.

The data support the use of supportive anemia care agents in combination with ruxolitinib (Jakafi; Incyte Corp).

Anemia is a very common adverse effect associated with the use of ruxolitinib.

Anemia is a common adverse effect of ruxolitinib, calling for new approaches to mitigate risks.

The addition of navitoclax was associated with durable response and potential disease modification.

Rusfertide may sustain hematocrit levels in patients with polycythemia vera.

According to the study, 20% of patients with myeloproliferative neoplasms progress to blast phase.

The nomogram model demonstrated good predictive capabilities to assess progression risk to overt primary myelofibrosis.

Approximately 15% of patients with polycythemia vera will progress to myelofibrosis.

The study was conducted on a small subgroup of Japanese patients with myelofibrosis.

Treosulfan showed superior benefits to busulfan as a conditioning regimen before allogenic hematopoietic stem cell transplantation (allo-HSCT).

Pelabresib, a BET inhibitor, demonstrated meaningful reductions in spleen inflammation and anemia.

Fedratinib is an orally available, small molecule inhibitor of JAK-2 approved for treatment of myelofibrosis.

The findings may offer patients treatment options beyond symptom palliation.

INCA033989 is a monoclonal antibody that selectively targets mutant calreticulin oncogenic function.

Researchers found that PF4/Cxcl4, a key driver of fibrosis, was up regulated on all proteomes.

Luspatercept reduced the need for transfusion in patients with myelofibrosis (MF).

Disease-related factors include several prognostic scoring systems, such as DIPSS, whereas patient-related factors involve age, comorbidities, spleen size, and anemia.

It can also help identify patients who would benefit from disease-specific therapies.

Experts discuss the ramifications of red blood cell transfusion shortages on myelofibrosis patient management and delve into the scarcity’s effects, patient prioritization, and the struggle for consistency in care amid supply challenges.

Zahra Mahmoudjafari, PharmD, MBA, BCOP, FHOPA, and a panel of pharmacists explore pharmacist roles in myelofibrosis care, treatment challenges, patient outcomes, and pharmacist responsibilities within interdisciplinary teams.

The model may help clinicians identify patients who would benefit from disease-specific therapies or enrollment into clinical trials.

Expert explains how the role of pharmacists in myelofibrosis treatment has evolved significantly over the past decade.

Ruben A. Mesa, MD, FACP, discusses the results of the SIMPLIFY-1 and MOMENTUM trials and how the data led to the approval of momelotinib to treat intermediate or high-risk myelofibrosis in adult patients with anemia.

Most patients with myelofibrosis will develop anemia over the course of the disease, with more than 30% discontinuing treatment as a result.

Pharmacists also have the resources to help advocate for better care for patients.

Despite a slew of barriers with patient management, pharmacists have the training and resources to advocate for better patient care and help patients with hematologic myeloproliferative neoplasms advocate for themselves.

The regulatory submission includes data from the MOMENTUM phase 3 clinical trial that met all primary and key second efficacy end points.