Tag Archives: interferon

Interferon Improves Myelofibrosis-Free Survival in AYA Patients with ET and PV

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By Melissa Badamo – Last Updated: May 27, 2025

While cytoreductive drugs did not reduce thrombosis risk in adolescent and young adult (AYA) patients with essential thrombocythemia (ET) and polycythemia vera (PV), interferon “significantly improved” myelofibrosis-free survival (MFS) compared with other treatments, according to a study published in Leukemia.

The retrospective study explored the long-term complications and impact of cytoreductive drugs on patient outcomes, thrombotic risk, and progression to secondary myelofibrosis (sMF) in 348 patients diagnosed with ET (n=278) or PV (n=70) before the age of 25 years. The primary end points were MFS, thrombosis-free survival (TFS), and overall survival. Secondary end points included identification of risk factors associated with thrombotic events and progression to sMF.

In the ET cohort, 147 (53%) patients had JAK2 mutations, 43 (16%) had CALR mutations, 3 (1%) had MPL mutations, and 85 (30%) were triple negative (TN). All patients with PV had JAK2 mutations.

A total of 237 (68%) patients were treated with a cytoreductive drug, including 185 patients with ET (66.5%) and 52 patients with PV (74.3%). Patients received one line of therapy (n=97; 41%), two lines of therapy (n=82; 35%), or three or more lines of therapy (n=58; 24%). The most prescribed first-line treatments were hydroxycarbamide (n=126; 53%), interferon (n=55; 23%), anagrelide (n=52; 22%), and alternative drugs (n=4; 2%). The median follow-up was 8.5 years.

Thrombotic Risk

Forty-four patients presented 57 thrombotic events, with a risk of 1.9 per 100 patient-years. The 10- and 20-year probability of TFS was 86.8% and 78.8% for the entire cohort, 86.9% and 80.0% for patients with ET, and 84.4% and 76.3% for patients with PV.

In a multivariate analysis, elevated white blood cell count (>11 × 109/L; hazard ratio [HR], 2.7; P=0.012) and the absence of splenomegaly at diagnosis (HR, 5.7; P=0.026) were associated with increased risk for thrombosis.

Choice of first treatment did not correlate with differences in TFS. The 10- and 20-year TFS were 83.9% and 79.9% for interferon, 81.4% and 70.2% for hydroxycarbamide, and 91.6% and 76.3% for anagrelide (P=0.281)

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Interferon Lowers Myelofibrosis Risk in Young Patients With Polycythemia Vera, Essential Thrombocytopenia

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May 15, 2025

Author(s): Alexandra Gerlach, Associate Editor

Early treatment with interferon reduced secondary myelofibrosis (sMF) risk in adolescent and young adult (AYA) patients with polycythemia vera (PV) or essential thrombocytopenia (ET), according to study data published in Leukemia.1

Red blood cells | Image Credit: © fotogurmespb - stock.adobe.com

MF is an incurable, rare hematologic malignancy characterized by the overproduction of red blood cells. This causes bone marrow scarring, which leads to severe anemia—a key factor impacting overall survival (OS) for patients with MF. It can develop as a primary disease or secondary to ET and PV, as well as progress to acute myeloid leukemia in some cases. Although MF is primarily diagnosed in older adults, 20% of cases are in individuals under the age of 40.1,2

AYA patients with ET or PV face unique clinical challenges due to their longer life expectancy because of a lack of data on long-term outcomes and treatment strategies. Existing guidelines are specific to older populations, despite the unique clinical considerations in younger patients.1

“These patients are expected to live for several decades, and the accumulation of additional thrombotic risk factors (age, cardiovascular conditions, additional mutations) may progressively heighten this risk,” the authors wrote. “This highlights a substantial and emerging concern regarding thrombotic events in this younger demographic.”1

The study evaluates complication rates, including thrombosis and progression to sMF, and the impact of interferon (IFN) on outcomes in patients diagnosed before the age of 25. The primary outcomes of the study were thrombosis-free survival (TFS), myelofibrosis-free survival (MFS), and OS in the entire cohort and for ET and PV patients separately, and the impact of treatment on these outcomes. Secondary outcomes were identification of risk factors associated with thrombotic events and sMF.1

The study included a total of 348 patients, of whom 278 were diagnosed with ET and 70 with PV, with a median age at diagnosis of 20 years. The incidence of thrombotic events was about 1.9 per 100 patient-years, with elevated white blood cell count (≥ 11 × 109/L) and absence of splenomegaly at diagnosis identified as significant risk factors. However, cytoreductive therapy did not reduce thrombotic risk. The incidence of sMF was 0.7 per 100 patient-years, with CALR mutations and a history of thrombosis associated with higher risk.1

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Pegylated Interferons: Still a Major Player for the Treatment of Myeloproliferative Neoplasms

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Michael Daunov, DO1 and Rebecca B. Klisovic, MD1

Overview

Over the past 35 years, interferons have been explored in various formulations for the management of Philadelphia chromosome–negative myeloproliferative neoplasms (MPNs), such as essential thrombocythemia (ET), polycythemia vera (PV), and myelofibrosis, and remain a key tool in caring for patients with these diseases. These agents are excellent cytoreductive agents with high rates of hematologic response, are helpful in symptom management, and have a long track record of safety and manageable toxicities. More recently, they have shown promise in sustaining responses over many years, with associated reductions in driver mutations (JAK2, MPL, CALR) of these diseases, particularly in PV and ET. Since reductions in molecular mutant allele burden have been correlated with several response outcomes such as reductions in both thrombotic risk and disease progression, there is emerging proof that interferons may offer disease-modifying activity. These long-term benefits and their use as the preferred agent in young pregnant women who need cytoreduction make interferons often the first choice in young adult population who harbor a lifetime risk of progression. Looking forward, the prospect of sustained treatment-free responses, like chronic myeloid leukemia after deep molecular response, and normal life expectancy may also be on the frontier. Despite relative rookies such as JAK inhibitors in the MPN landscape, the veteran in the game, interferon, remains a key player.

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Advances in Interferon Therapy for Myeloproliferative Neoplasms

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Kumar Das, Dibash PhD

Oncology Times 46(6):p 1,14, June 2024. | DOI: 10.1097/01.COT.0001024068.38723.15

In the ever-evolving landscape of myeloproliferative neoplasms (MPNs), clinicians continue to explore and refine treatment strategies to improve patient outcomes. A recent review published in Therapeutic Advances in Hematology sheds light on the pivotal role of interferons, particularly pegylated formulations, in managing MPNs effectively (2024; doi: 10.1177/20406207241229588).

The advent of pegylated interferons, including peginterferon alfa-2a and ropeginterferon alfa-2b-njft, marks a significant turning point in MPN therapeutics. These agents, renowned for their potent immunomodulatory capabilities and profound impact on disease progression, have reshaped treatment paradigms outlined in the National Comprehensive Cancer Network (NCCN) Guidelines for polycythemia vera (PV), essential thrombocythemia, and primary myelofibrosis. This article delves deep into the multifaceted influence of pegylated interferons, shedding light on their efficacy, safety profiles, and future implications in MPN management.

Clinical trials, including landmark Phase II and III studies such as MPD-RC 111 and MPD-RC 112, have provided crucial insights into the efficacy of pegylated interferons. These trials meticulously assessed response rates, molecular remissions, and hematological improvements in MPN patients resistant to or intolerant of hydroxyurea. Noteworthy reductions in JAK2 V617F variant allele frequency (VAF) have underscored the molecular response achievements of pegylated interferons, highlighting their disease-modifying potential.

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Interferons in the treatment of myeloproliferative neoplasms

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Pankit Vachhani, John Mascarenhas , Prithviraj Bose , Gabriela Hobbs, Abdulraheem Yacoub, Jeanne M. Palmer , Aaron T. Gerds, Lucia Masarova, Andrew T. Kuykendall, Raajit K. Rampal, Ruben Mesa and Srdan Verstovsek

Abstract: Interferons are cytokines with immunomodulatory properties and disease-modifying effects that have been used to treat myeloproliferative neoplasms (MPNs) for more than 35 years. The initial use of interferons was limited due to difficulties with administration and a significant toxicity profile. Many of these shortcomings were addressed by covalently binding polyethylene glycol to the interferon structure, which increases the stability, prolongs activity, and reduces immunogenicity of the molecule. In the current therapeutic landscape, pegylated interferons are recommended for use in the treatment of polycythemia vera, essential thrombocythemia, and primary myelofibrosis. We review recent efficacy, molecular response, and safety data for the two available pegylated interferons, peginterferon alfa-2a (Pegasys) and ropeginterferon alfa-2b-njft (BESREMi). The practical management of interferonbased therapies is discussed, along with our opinions on whether to and how to switch from hydroxyurea to one of these therapies. Key topics and questions related to use of interferons, such as their safety and tolerability, the significance of variant allele frequency, advantages of early treatment, and what the future of interferon therapy may look like, will be examined. Pegylated interferons represent an important therapeutic option for patients with MPNs; however, more research is still required to further refine interferon therapy.

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A Mother’s Story: When Your Child is Diagnosed with an MPN

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No one can prepare you for a cancer diagnosis of a child. Our daughter was four years old and began to complain about headaches. I assumed it was her eyes and made an appointment with an optometrist. Her sight was perfectly normal. A visit to her pediatrician lasted an hour with little insight, only suggestions to watch her diet, limit TV time and give her lots of water. She rarely watched TV and carried water with her throughout the day. Our diet doesn’t include sugar except from fresh fruit and we don’t eat boxed or canned food. I wasn’t optimistic. Eventually, her headaches became more severe on occasion, similar to migraines. We were sent to a neurologist. He requested blood work after she underwent an MRI. Thankfully, the blood work identified the problem-ET or essential thrombocythemia. We had never heard of it and had no idea what this meant for the future. A hematologist became our savior. With the proper diagnosis and medication, our daughter began to feel better, albeit a few side effects from the meds. Yogurt is a staple to help with GI issues, and a nap and early bedtime help fatigue. Yes, our little girl had what I would call fatigue. Our lives have changed but we do not let her ET control us. We control her ET. We manage her diagnosis as part of our daily lives as we would manage any other chronic disease. That is not to say it’s been easy. We have our moments of fear and doubt, but that doesn’t last as long as it used to. We keep very good records of her doctor visits, her blood levels and her overall health. We ask how she is feeling and pay attention to any changes that could be due to her ET. She is now 12 and enjoying a normal childhood. We are looking at Interferfon as a possible “next protocol,” if we think it will be better for her.  We stay informed and are very pleased to see all of the clinical trials and new drugs on the horizon. It’s easy to say don’t panic if your child is diagnosed with an MPN. I would simply say, gather the facts, stay informed, be the voice they cannot be, and remember to take good care of yourself.

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