Fedratinib Alleviates Symptoms, Reduces Spleen Volume in MDS, MPNs

Fedratinib can reduce symptoms and spleen volume in patients with myelodysplastic syndrome (MDS) or myeloproliferative neoplasms (MPNs), according to research presented at the ASH Annual Meeting 2024.

Researchers are evaluating fedratinib, a JAK2 inhibitor, in a phase 2 trial. The trial (NCT05177211) enrolled 25 patients with atypical chronic myeloid leukemia (n=6), chronic neutrophilic leukemia (n=5), MDS/MPN-unclassifiable (n=8), and MDS/MPN with ring sideroblasts and thrombocytosis (n=6).

At baseline, the median patient age was 68.8 (range, 39.9-84.7) years, and the median time from diagnosis to treatment was 7.1 months. Most patients had splenomegaly (83%), and the median MPN-Symptom Assessment Form Total Symptom Score was 21 (range, 1-73). Prior treatments included hydroxyurea (36%), ruxolitinib (20%), luspatercept (8%), and hypomethylating agents (12%). Patients had a median of 3 pathogenic mutations.

“Most of these patients had multiple mutations, and most had a signaling mutation, an epigenetic mutation, and a splicing mutation,” said study presenter Andrew Kuykendall, MD, of the Moffitt Cancer Center in Tampa, Florida.

The patients received fedratinib at a dose of 400 mg daily in 28-day cycles. They could continue on treatment as long as they had a clinical benefit. At last follow-up, 11 patients were still on study treatment.

The median duration of fedratinib treatment was 10.8 months, and 21 patients were evaluable for efficacy at 24 weeks. Three patients discontinued fedratinib prior to 24 weeks for reasons unrelated to toxicity or lack of efficacy (eg, cost) and were considered non-responders.

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Recognizing Symptoms of Myeloproliferative Neoplasms and Clinical Trial Challenges

October 24, 2024

Author(s): Mary Caffrey, Laura Joszt, MA

The symptoms of myeloproliferative neoplasms can be variable and common, which can make it difficult to diagnose if you aren’t looking for the right thing, said Ruben Mesa, MD, FACP, executive director of Atrium Health Wake Forest Baptist Comprehensive Cancer Center and president of Atrium Health Levine Cancer.

He also discusses the challenges with getting patients enrolled in clinical trials, such as the limited availability of them and patient factors that make it difficult to participate.

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Education on MPN Symptoms, Treatments Leads to Greater Involvement in Care

October 3, 2024

Author(s): Darlene Dobkowski, MA

Fact checked by: Alex Biese

Learning more about the different symptoms and treatment goals of myeloproliferative neoplasms (MPNs) can help patients be more involved in management decisions throughout the disease trajectory, an expert said.

“It’s not just the doctor and the nurses; it’s the person who has the disease [that] is the main person, so their involvement is very important,” said Dr. Swati Goel at the recent CURE® Educated Patient® Updates in MPNs at Montefiore Medical Center in the Bronx, New York.

Goel is the Leader of the Myeloproliferative Disorder Clinic, assistant director of the hematology-oncology fellowship program and associate professor in the department of oncology and medicine at Montefiore Einstein in New York, New York.

Throughout the event, Goel discussed that there are three types of MPNs: polycythemia vera (PV), essential thrombocythemia (ET) and myelofibrosis.

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Predictors of symptom scores in myeloproliferative neoplasms: A real-world retrospective cohort study

Muhammad Ali KhanSyed Arsalan Ahmed NaqviIrbaz Bin Riaz, and Jeanne M. Palmer

Abstract

Background: Although high symptom burden indicates poor survival and informs treatment decisions, little is known about the impact of demographic, clinical, and laboratory features on total symptom score (TSS) in patients with myeloproliferative neoplasms (MPN).
Methods: Patients with MPN (polycythemia vera (PV), essential thrombocythemia (ET), and myelofibrosis (MF)) were identified from the retrospective chart review. TSS, individual symptom scores (fatigue, early satiety, abdominal discomfort, inactivity, concentration problems, fever, night sweats, itching, bone pain, weight loss), demographic characteristics (race, ethnicity, age, gender), clinical features (time since diagnosis, depression status, obesity status, spleen size), laboratory results and season at the time of visit were recorded from the clinical encounter when index assessment of TSS was performed for each patient. Normality was assessed using visual inspection of data distribution, whereas multicollinearity was assessed using various inflation factors. A univariable regression followed by a multivariable regression analysis was conducted using a backward selection approach. A p-value <0.05 indicated a statistically significant association of a given feature with TSS.
Results: The chart review identified 252 patients (PV: 78; ET: 81; MF: 93). Mean age was 59 (SD: 17.7), 67 (SD: 13.0), and 68 (SD: 10.9) years for ET, PV, and MF respectively. Most patients were white (PV, MF: 92%; ET: 83%) and females (ET: 75%; PV: 60%; MF: 53%). The TSS of patients was highest with PV (mean: 18.5; SD: 16.9) followed by MF (mean: 18.1; SD: 15.4) and ET (mean: 14.3; SD: 15.9). Fatigue was the most reported symptom whereas the least reported symptoms were fever and weight loss. Univariable regression analyses showed depression (B: 17.7; p=0.02), female gender (B: 10.6; p=0.01), platelet count (B: 0.03; p=0.03), and hemoglobin (Hb) (B: -2.6; p=0.01) in PV patients, depression (B: 19.8, p=2×10-5) in ET patients and depression (B: 11.0, p=0.03), white blood cell (WBC) count (B: 0.2; p=0.01), neutrophil count (B: 0.3, p=0.01), and non-neutrophil WBC count (B: 0.6; p=0.02) in MF patients to have significant association with TSS. Multivariable regression analyses (Table) showed Hb (B: -2.5; p=0.01) and platelet count (B: 0.02; p=0.03) in PV patients, depression (B: 19.7; p=2×10-5) in ET patients and depression (B: 12.3, p=0.01) and WBC count (B: 0.3; p=0.002) in MF patients to have a significant association with TSS.
Conclusions: Depression in ET and MF and low Hb in PV were identified as significant drivers of symptom burden. Identifying and managing patients with these comorbidities could improve their quality of life with a potential survival benefit.

MPN Symptoms Mimic Other Conditions, Open Communication Is Key

Brielle Benyon

Myeloproliferative neoplasm (MPN) symptoms can often appear as another condition, making it essential that patients find a cancer care team that they trust and can have open communication with, according to Patrick Buxton.

Buxton, who is a clinical nurse manager at Fred Hutchinson Cancer Center in Seattle and a 2023 MPN Hero, explained that certain side effects like constant fatigue could mimic conditions such as depression. That is why it is important for patients to work with their oncology team to establish a baseline of lab results and symptoms and keep them up to date on how they are feeling.

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