I’m 38 today and was diagnosed with ET at age 21 while in college in 2007, following a routine blood test. I took the blood test so that I could donate bone marrow to earn money ($400 at the time) for an upcoming study abroad trip. Apart from the increased platelet count, I did not have other noticeable symptoms. The blood test results indicated an increased platelet count, and I was not permitted to donate bone marrow. I underwent a bone marrow biopsy many months later, which confirmed the diagnosis of ET. I am JAK2+.Between that time period in college and today, I’ve been on and off hydroxyurea, on and off baby aspirin, got married, had two children, learned more about bleeding and other symptoms related to high platelet counts and have met new members of the MPN community. I wish I knew then how much hormone levels and clotting issues can impact women’s reproductive health. Interestingly, I learned after my son was born that I also carry the gene for hemophilia A, an incurable bleeding disorder. Call me if you’d like to talk about both clotting or bleeding!These days, I do occasionally experience incredible fatigue and frequently find myself scratching itchy skin (particularly after the shower) and have noticed some vision changes but am not sure if that can be attributed to ET. Today, I’m so grateful for the knowledge I’ve gained and the support available to me in the MPN community, especially MPN Advocacy and Education International.
Dr Jennifer Vaughn: Patients With MPN, MDS Should Discuss Long-Term Priorities Upfront
Laura Joszt, MA
Justina Petrullo
Justina Petrullo
With most patients with myeloproliferative neoplasms (MPNs) and myelodysplastic syndromes (MDS) experiencing long-term, chronic disease, it’s important to discuss their priorities and set up the relationship with their providers upfront, explained Jennifer Vaughn, MD, assistant professor in the division of hematology at The Ohio State University Comprehensive Cancer Center – James Cancer Hospital and Solove Research Institute.
About 1 in 5 Hematologic Cancer Patients Had Severe COVID-19, Despite Vaccination
In a study of more than 6000 patients with hematologic cancers, 21% developed severe COVID-19, despite being vaccinated. Researchers reported these results in JAMA Network Open.
The study included 6122 patients from the national Veterans Health Administration who had hematologic cancers. All patients had been vaccinated against COVID-19 but had a confirmed case of COVID-19 between January 1, 2021, and September 30, 2022.
The patients had chronic lymphocytic leukemia (CLL; n=1206) or other non-Hodgkin lymphomas (NHLs; n=1731), plasmacytoid neoplasms (n=1014), myeloproliferative neoplasms (MPNs; n=1144), myelodysplastic syndromes (MDS; n=518), chronic myeloid leukemia (CML; n=180), acute myeloid leukemia (AML; n=172), or Hodgkin lymphoma (n=157).
A total of 1301 paints (21.3%) had severe COVID-19, which was defined as dying within 28 days of SARS-CoV-2 infection, requiring mechanical ventilation, or requiring hospitalization with the use of dexamethasone, evidence of hypoxemia, or the use of supplemental oxygen.
The proportion of patients with severe COVID-19 was similar among those with lymphoid malignancies (21.6%) and those with myeloid malignancies (20.5%). The rate of severe COVID-19 was highest in patient with MDS (28.2%) and lowest in patients with Hodgkin lymphoma (12.1%).
Non-Driver Gene Mutations Have Adverse Prognostic Impact in Primary Myelofibrosis
A study published in the American Journal of Hematology has confirmed the adverse prognostic impacts of numerous non-driver gene mutations in primary myelofibrosis (PMF).
Researchers analyzed the impact of non-driver somatic mutations in patients with PMF treated at 60 institutions in Spain. Targeted next-generation sequencing (NGS) sequencing evaluating up to 56 non-myeloproliferative neoplasm driver genes was conducted. Of those, the team focused on 20 genes consistently analyzed across NGS panels. Only pathogenic or likely-pathogenic genetic variants with a variant allele frequency (VAF) higher than 1% were considered mutations.
A total of 312 patients with PMF according to World Health Organization criteria at the time of diagnosis and with availability of NGS data were included in the analysis. The median age of the cohort was 68 years. At a median follow-up duration of 4 years, 9% of patients had progressed to acute myeloid leukemia, and 47% had died.
Overall, 72% of patients had non-driver mutations, with 47% having 2 or more mutations (range, 0-7). The most frequent mutations detected were in ASXL1 (34%), TET2 (22%), SRSF2 (17%), U2AF1 Q157 (9%), CBL (8%), EZH2 (7%), TP53 (6%), DNMT3A (6%), and SETBP1 (5%).
Years After Genetic Finding, Drugs Targeting CALR-Mutant Myeloproliferative Neoplasms Enter Trials
NEW YORK – More than a decade after mutations in the CALR gene were first linked to the development of myeloproliferative neoplasms, CALR-targeted drug candidates are advancing to Phase I clinical trials.
If these drugs reach the market, they could provide a treatment option for a group of patients with myelofibrosis and essential thrombocythemia who typically must wait until their condition turns serious to attempt a risky stem cell transplant.
About 300,000 patients in the US have myeloproliferative neoplasms. Kapila Vigas, CEO of the MPN Research Foundation, said patients can have very different presentations of the disease, and it can take “years or decades” to get a diagnosis. Although myeloproliferative neoplasms are classified as chronic cancers that patients can live with for many years with blood count monitoring, Vigas said some patients can abruptly progress, and their condition can become serious.
“That uncertainty is really concerning to patients,” Vigas said. “We think from a psychosocial perspective, it’s worse than an acute cancer because while cancer may be more serious, it’s predictable, and there’s a plan and a protocol, whereas when you’re diagnosed with [a myeloproliferative neoplasm] watch and wait is almost a first-line approach. It just adds to the anxiety.”
Incyte Launches The Unseen Journey to Elevate the Hidden Impact of Myeloproliferative Neoplasms (MPNs) on Patients’ Lives through Generative AI
February 29, 2024
– The Unseen Journey brings to life the often-misunderstood impact of common myeloproliferative neoplasm (MPN) symptoms through AI-generated images developed from the words and experiences of real patients
WILMINGTON, Del.–(BUSINESS WIRE)–Incyte (Nasdaq: INCY) today announced the launch of The Unseen Journey, a program that brings to life the hidden emotional and physical toll of myeloproliferative neoplasms (MPNs), a group of rare, chronic and progressive blood cancers. Through the use of generative artificial intelligence (AI), the stories and experiences of MPN patients were transformed into unique images to help them show their health care team and their loved ones the significant impact of their MPN symptoms.
The Unseen Journey highlights the stories of people living with MPNs who were asked to describe their symptoms and how they impact their lives in their own words. As each patient described their symptoms and experiences, generative AI tools transformed their words into images that visually depict the patient’s most burdensome symptoms. The resulting images provide a vivid look at the reality of living with an MPN.
“MPN symptoms can be difficult to recognize and describe and every patient’s experience is different, which can sometimes create a challenge for patients, their loved ones and their health care teams to understand the impact of the condition to daily life,” said Ann Brazeau, CEO and Founder, MPN Advocacy and Education International. “These AI-generated images paint a vivid picture of what it is like to live with an MPN, and I hope they will help create a new level of awareness and empathy for those with these conditions.”
Rusfertide More Than Triples Responses Vs Placebo in Phlebotomy-Dependent Polycythemia Vera
Caroline Seymour
Treatment with rusfertide led to a 60% response rate (n = 18/30) vs 17% (n = 5/29) with placebo in patients with phlebotomy-dependent polycythemia vera (P = .002), according to updated findings from part 2 of the phase 2 REVIVE trial (NCT04057040) which were published in the New England Journal of Medicine.1
The international trial was designed with 3 parts: a 28-week, open-label, dose-finding portion in which rusfertide was added to a patient’s ongoing therapy of phlebotomy alone or cytoreductive therapy with optional phlebotomy; a double-blind, randomized withdrawal portion wherein patients were randomly assigned to receive rusfertide or placebo for 12 weeks (weeks 29 to 41); and an open-label extension period following patients on rusfertide therapy for up to 3 years.
Findings from part 1 showed that the estimated mean number of annual phlebotomies was 8.7±2.9 during the 28 weeks before the first dose of rusfertide and 0.6±1.0 during part 1 (estimated difference, 8.1 phlebotomies per year). Moreover, the mean maximum hematocrit level was 44.5±2.2% during part 1 vs 50.0±5.8% during the 28 weeks before the first dose of rusfertide. Patient quality of life was also improved on rusfertide, with a lower severity of disease-related symptoms.
“Rusfertide appears to represent a significant step forward in treating [patients with] polycythemia vera through its unique approach of limiting the amount of iron available for blood cell production,” Marina Kremyanskaya, MD, PhD, an associate professor of medicine (hematology and medical oncology) at Icahn School of Medicine at Mount Sinai in New York, New York, and lead author of the study, stated in a news release.2 “Pending further clinical studies, this injectable agent could become a valuable therapeutic tool for a disease which many patients and their physicians struggle to bring under control.”
PharmaEssentia’s BESREMi (ropeginterferon alfa-2b-njft) Now Recommended as a Preferred First-line Cytoreductive Therapy for Polycythemia Vera in Updated NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines)
Update based on NCCN’s determination of superior efficacy, safety and evidence for BESREMi in high and low-risk patients, reflecting an ongoing shift in PV care toward earlier intervention
BURLINGTON, Mass.–(BUSINESS WIRE)–PharmaEssentia USA Corporation, a subsidiary of PharmaEssentia Corporation (TWSE: 6446), a global biopharmaceutical innovator based in Taiwan leveraging deep expertise and proven scientific principles to deliver new biologics in hematology and oncology, today announced that the National Comprehensive Cancer Network Clinical Practice Guidelines in Oncology (NCCN Guidelines®) have recently been updated to include ropeginterferon alfa-2b-njft, marketed as BESREMi®, as a preferred first-line cytoreductive therapy option for the treatment of adults with symptomatic, low-risk polycythemia vera (PV). Ropeginterferon alfa-2b-njft is the only preferred therapeutic option for both high-risk and low-risk (symptomatic) PV regardless of treatment history.1
“The recent update to the NCCN Guidelines® reflects the ongoing shift in PV care towards earlier intervention, focusing on long-term patient health,” said Rami Komrokji, M.D., Vice Chair of the Malignant Hematology Department and Head of the Leukemia and MDS Section at Moffitt Cancer Center.
Broad next generation integrated sequencing of myelofibrosis identifies disease-specific and age-related genomic alterations
Abstract
The NUTRIENT Trial (NUTRitional Intervention among myEloproliferative Neoplasms): Results from a Randomized Phase I Pilot Study for Feasibility and Adherence
Laura F. Mendez Luque1,2, Julio Avelar-Barragan3, Hellen Nguyen1, Jenny Nguyen1, Eli M. Soyfer1, Jiarui Liu1, Jane H. Chen1, Nitya Mehrotra1, Xin (Helen) Huang1, Heidi E. Kosiorek4, Amylou Dueck4, Alexander Himstead1, Elena Heide1, Melinda Lem1, Kenza El Alaoui1, Eduard Mas1, Robyn M. Scherber5, Ruben A. Mesa6, Katrine L. Whiteson3, Andrew Odegaard1, Angela G. Fleischman1
ABSTRACT
Purpose: Chronic inflammation is integral to Myeloproliferative Neoplasm (MPN) pathogenesis. JAK inhibitors reduce cytokine levels, but not without significant side effects. Nutrition is a low- risk approach to reduce inflammation and ameliorate symptoms in MPN. We performed a randomized, parallel-arm study to determine the feasibility of an education-focused Mediterranean diet intervention among MPN patients.