What to Know About Myeloproliferative Disorders Clinical Trials

Posted on March 10, 2025March 10, 2025 by mpn_admin

Medically reviewed by Julie Scott, DNP, ANP-BC, AOCNP — Written by Hope Gillette on March 6, 2025

Myeloproliferative disorders, now referred to as myeloproliferative neoplasms (MPNs), include a group of blood cancers that develop when bone marrow produces too many red blood cells, white blood cells, or platelets.

Some forms of MPNs, such as essential thrombocythemia (ET) respond well to current treatmentTrusted Source, but others, such as primary myelofibrosis, have fewer effective options.

Depending on the specific diagnosis you received, your healthcare team may recommend participating in an MPN clinical trial to expand your treatment possibilities.

Divesiran Is Tolerable and Shows Positive Early Signals in Polycythemia Vera

Posted on February 27, 2025February 27, 2025 by mpn_admin

February 26, 2025

Author(s): Kyle Doherty

Fact checked by: Megan Hollasch

Divesiran (SLN124), a novel small interfering RNA (siRNA), was safe and displayed signals of efficacy in the treatment of patients with polycythemia vera, according to findings from the phase 1/2 SANRECO trial (NCT05499013).¹

Initial results from SANRECO presented during the 2024 ASH Annual Meeting showed that divesiran reduced phlebotomy frequency in patients (n = 21). A total of 79 phlebotomies occurred across all patients prior to dosing; there were 5 phlebotomies during the treatment period and 2 during follow-up among all patients. Divesiran also induced hepcidin in all patients and decreased hematocrit in all cohorts of patients treated.

Additionally, patients did not experience any dose-limiting toxicities. Most treatment-emergent adverse effects (TEAEs) were grade 1 in severity (84%) and there were no TEAEs above grade 2 reported. There were also no treatment-related serious AEs or TEAEs leading to treatment discontinuation.

Divesiran is a first-in-class GalNAc-conjugated siRNA that targets TMPRSS6, a negative regulator of the HJV/BMP/SMAD signaling pathway that induces hepcidin expression. The agent is designed to have a long duration of action, and, notably, it’s target sequence is unique to TMPRSS6 and was selected to maximize TMPRSS6 knock down. Investigators hypothesized that inhibiting TMPRSS6 would raise hepcidin levels and lower iron delivery to the bone marrow, leading to reduced erythropoiesis.

Underreporting of Patient-Reported Outcomes Seen in Blood Cancer Trials

Posted on August 5, 2024August 5, 2024 by mpn_admin

Caleb Rans, PharmD

August 2, 2024

The incorporation of patient-reported outcomes (PROs) in clinical trials provides a means for researchers to measure health-related quality of life (HRQOL) and patient-specific outcomes. When evaluating subjective symptoms, for example, patients are usually the most reliable source of feedback. Consequently, PROs are seen as the gold standard for assessing subjective symptoms.¹

In a systematic review published in JAMA Network Open, Kishan Patel, MD, MHS, of the department of internal medicine at Brigham and Women’s Hospital in Boston, Massachusetts, and colleagues evaluated the prevalence of PROs as prespecified endpoints in randomized clinical trials (RCTs) of hematological malignancies. They also assessed reporting of PROs in associated primary trial publications.

According to the authors, PROs are not often collected or reported in solid tumor trials, but less is known about RCTs focusing on blood cancers.²

Combination Therapies and New Research Drive Progress in Myelofibrosis

Posted on March 11, 2024March 11, 2024 by mpn_admin

March 7, 2024

Jordyn Sava

2023 brought a wave of positive developments for patients with myeloproliferative neoplasms (MPNs), particularly myelofibrosis. According to Raajit K. Rampal, MD, PhD, one study of particular interest was the phase 3 MANIFEST-2 trial (NCT04603495) of ruxolitinib (Jakafi) with pelabresib (CPI-0610).¹

This study, in addition to the TRANSFORM-1 trial (NCT04472598), showed significant improvement in spleen size and potential benefits in symptom reduction with combination therapies compared with single-agent treatments, suggesting that these combinations could become valuable options for treating patients with myelofibrosis upfront.^1,2

Other studies, including early data of TP-3654 and selinexor (Xpovio), show potential for further advancements in myelofibrosis treatment.

“There is a lot to be excited about for the first time in a very long time. There are all of these other small molecule inhibitors in clinical trials [and] I think we will learn a lot from that,” said Rampal, hematologic oncologist at Memorial Sloan Kettering Cancer Center, in an interview with Targeted Oncology^TM.