Evidence map›Paper›PMID 35007321›Full record

Trial reportBlood2022

A randomized phase 3 trial of interferon-α vs hydroxyurea in polycythemia vera and essential thrombocythemia.

John Mascarenhas, Heidi E Kosiorek, Josef T Prchal, Alessandro Rambaldi, Dmitriy Berenzon, Abdulraheem Yacoub, Claire N Harrison, Mary Frances McMullin, Alessandro M Vannucchi, Joanne Ewing and 33 more

2 registry-linked trialsOpen access · bronzeAbstract readClinical Trial, Phase IIIRandomized Controlled Trial
In one paragraph

Trial report in Blood, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 85 papers, 7 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
85citing papers in PubMed, 7 pooled it
17.9field-weighted citation impact, top 1% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT01259856 phase3completednot on this map

Randomized Trial of Pegylated Interferon Alfa-2a Versus Hydroxyurea Therapy in the Treatment of High Risk Polycythemia Vera (PV) and High Risk Essential Thrombocythemia (ET)

TypeinterventionalSponsorRonald HoffmanRan2011 to 2017Enrolled168ConditionsHigh Risk Polycythemia Vera, High Risk Essential ThrombocythemiaArmsPEGASYS, Hydroxyurea, Aspirin
NCT05850273 recruitingnot on this mapstarted 2023, after this paper: background citation

Mechanism of Action of Interferon in the Treatment of Myeloproliferative Neoplasms

TypeobservationalSponsorInstitut National de la Santé Et de la Recherche Médicale, FranceRan2023 to 2033Enrolled80ConditionsMyeloproliferative Neoplasm
3 · Its place in the literature

Who cites it

85 citing papers in PubMed, 7 syntheses or guidelines pooled it, 120 citations in OpenAlex.

  1. Pooled it
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  7. [Chinese guideline for the diagnosis and treatment of polycythemia vera (2022)].Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2022
    Guideline
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25 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

43 authors at 20 institutions in 5 countries.

John MascarenhasThe Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY.ORCID 0000-0002-8400-0483
Heidi E KosiorekDepartment of Quantitative Health Sciences, Mayo Clinic, Scottsdale, AZ.
Josef T PrchalDivision of Hematology and Hematologic Malignancies, Huntsman Cancer Institute, University of Utah, Salt Lake City, UT.ORCID 0000-0002-8019-2940
Alessandro RambaldiHematology and Bone Marrow Transplantation Unit, ASST Papa Giovanni XXIII, Bergamo, Italy.
Dmitriy BerenzonComprehensive Cancer Center, Wake Forest Baptist Health, Comprehensive Cancer Center, Winston-Salem, NC.
Abdulraheem YacoubKansas University Cancer Center, Leawood, KS.ORCID 0000-0001-5293-4620
Claire N HarrisonDepartment of Haematology, Guy's Hospital, London, United Kingdom.
Mary Frances McMullinQueen's University Belfast, Belfast, United Kingdom.ORCID 0000-0002-0773-0204
Alessandro M VannucchiUniversity of Florence, Azienda Ospedaliero Universitaria Careggi, Florence, Italy.ORCID 0000-0001-5755-0730
Joanne EwingHeart of England NHS Foundation Trust, UHB, Birmingham, United Kingdom.
Casey L O'ConnellKeck School of Medicine, University of Southern California, Los Angeles, CA.
Jean-Jacques KiladjianUniversité de Paris, Assistance Publique-Hôpitaux de Paris (AP-HP), Hôpital Saint-Louis, Centre d'Investigations Cliniques, INSERM, Paris, France.ORCID 0000-0002-8121-438X
Adam J MeadOxford University Hospitals NHS Foundation Trust, Oxford, United Kingdom.ORCID 0000-0001-8522-1002
Elliott F WintonWinship Cancer Institute, Emory University School of Medicine, Atlanta, GA.
David S LeibowitzOncology Department, Palo Alto Medical Foundation Sutter Health, Cupertino, CA.ORCID 0000-0001-5254-9829
Valerio De StefanoSection of Hematology, Department of Radiological and Hematological Sciences, Catholic University, Fondazione Policlinico Universitario A. Gemelli Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS), Rome, Italy.ORCID 0000-0002-5178-5827
Murat O ArcasoyDuke University School of Medicine, Durham, NC.
Craig M KesslerGeorgetown University Medical Center, Washington, DC.
Rosalind CatchatourianOncology Department, John H. Stroger Jr Hospital of Cook County, Chicago, IL.
Damiano RondelliDivision of Hematology/Oncology, University of Illinois at Chicago, Chicago, IL.
Richard T SilverRichard T. Silver Myeloproliferative Neoplasms Center, New York Presbyterian Weill Cornell Medical Center, New York, NY.
Andrea BacigalupoSection of Hematology, Department of Radiological and Hematological Sciences, Catholic University, Fondazione Policlinico Universitario A. Gemelli Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS), Rome, Italy.
Arnon NaglerHematology Department, Chaim Sheba Medical Center, Tel Hashomer, Israel.
Marina KremyanskayaThe Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY.
Max F LevineCenter for Hematologic Malignancies, Memorial Sloan-Kettering Cancer Center, New York, NY.ORCID 0000-0001-5156-9086
Juan E Arango OssaCenter for Hematologic Malignancies, Memorial Sloan-Kettering Cancer Center, New York, NY.ORCID 0000-0003-1109-6178
Erin McGovernCenter for Hematologic Malignancies, Memorial Sloan-Kettering Cancer Center, New York, NY.ORCID 0000-0002-7949-0609
Lonette SandyThe Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY.
Mohamad E SalamaMayo Clinic, Rochester, MN.
Vesna NajfeldThe Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY.
Joseph TripodiThe Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY.ORCID 0000-0003-3033-1081
Noushin FarnoudCenter for Hematologic Malignancies, Memorial Sloan-Kettering Cancer Center, New York, NY.ORCID 0000-0001-5355-7775
Alexander V PensonCenter for Hematologic Malignancies, Memorial Sloan-Kettering Cancer Center, New York, NY.ORCID 0000-0003-2429-2370
Rona Singer WeinbergNew York Blood Center, New York, NY.ORCID 0000-0002-4503-1295
Leah PriceDepartment of Population Health and.ORCID 0000-0002-5092-4768
Judith D GoldbergDepartment of Population Health and.ORCID 0000-0003-3758-6976
Tiziano BarbuiPapa Giovanni XXIII Hospital, Foundation for Clinical Research (FROM), Bergamo, Italy.ORCID 0000-0003-2747-6327
Roberto MarchioliCardiovascular, Renal and Metabolic Medical and Scientific Services, IQVIA, Milan, Italy.
Gianni TognoniDepartment of Anaesthesia and Emergency Urgency, IRCCS, Ospedale Maggiore Policlinico, Milan, Italy.
Raajit K RampalLeukemia Service, Department of Medicine, Center for Hematologic Malignancies, Human Oncology and Pathogenesis Program, Memorial Sloan-Kettering Cancer Center, Myeloproliferative Neoplasm Research Consortium (MPN-RC), New York, NY; and.
Ruben A MesaUT Health San Antonio Cancer Center, San Antonio, TX.
Amylou C DueckDepartment of Quantitative Health Sciences, Mayo Clinic, Scottsdale, AZ.ORCID 0000-0002-9912-1085
Ronald HoffmanThe Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY.
Icahn School of Medicine at Mount Sinai · USMemorial Sloan Kettering Cancer Center · USMayo Clinic in Arizona · USIstituti di Ricovero e Cura a Carattere Scientifico · ITOspedale Papa Giovanni XXIII · ITAtrium Health Wake Forest Baptist · USAzienda Ospedaliero-Universitaria Careggi · ITCornell University · USDuke University · USEmory University · USGeorgetown University · USGuy's Hospital · GBHeart of England NHS Foundation Trust · GBInserm · FRIQVIA (United Kingdom) · GBJohn H. Stroger, Jr. Hospital of Cook County · USNew York Blood Center · USNew York University · USOspedale Maggiore · ITOxford University Hospitals NHS Trust · GB

Funding

X-RAY CRYSTALLOGRAPHYP30CA008748 · NCI · SLOAN-KETTERING INSTITUTE FOR CANCER RES · PI Hongbiao Carl Lekaye · 1985 to 2026
$347.4M
Women's Cancer ProgramP30CA015083 · NCI · MAYO CLINIC ROCHESTER · PI AKHILESH PANDEY · 1985 to 2026
$151.3M
Tissue BankP01CA108671 · NCI · UNIVERSITY OF ILLINOIS AT CHICAGO · PI Ronald Hoffman · 2006 to 2026
$82.0M
Genomic profiling and development of murine models of transformation of MPNsK08CA188529 · NCI · SLOAN-KETTERING INST CAN RESEARCH · PI RAMPAL, RAAJIT · 2014 to 2018
$891k
Cancer Research UK 26988Cancer Research UK 27125Cancer Research UK 27723Medical Research Council G84/6443Medical Research Council MC_UU_00016/15Medical Research Council MC_UU_12009/16Medical Research Council MR/L006340/1Medical Research Council MR/M00919X/1NCI NIH HHS K08 CA188529NCI NIH HHS P01 CA108671NCI NIH HHS P30 CA008748NCI NIH HHS P30 CA015083
6 · The paper itself

Abstract

The goal of therapy for patients with essential thrombocythemia (ET) and polycythemia vera (PV) is to reduce thrombotic events by normalizing blood counts. Hydroxyurea (HU) and interferon-α (IFN-α) are the most frequently used cytoreductive options for patients with ET and PV at high risk for vascular complications. Myeloproliferative Disorders Research Consortium 112 was an investigator-initiated, phase 3 trial comparing HU to pegylated IFN-α (PEG) in treatment-naïve, high-risk patients with ET/PV. The primary endpoint was complete response (CR) rate at 12 months. A total of 168 patients were treated for a median of 81.0 weeks. CR for HU was 37% and 35% for PEG (P = .80) at 12 months. At 24 to 36 months, CR was 20% to 17% for HU and 29% to 33% for PEG. PEG led to a greater reduction in JAK2V617F at 24 months, but histopathologic responses were more frequent with HU. Thrombotic events and disease progression were infrequent in both arms, whereas grade 3/4 adverse events were more frequent with PEG (46% vs 28%). At 12 months of treatment, there was no significant difference in CR rates between HU and PEG. This study indicates that PEG and HU are both effective treatments for PV and ET. With longer treatment, PEG was more effective in normalizing blood counts and reducing driver mutation burden, whereas HU produced more histopathologic responses. Despite these differences, both agents did not differ in limiting thrombotic events and disease progression in high-risk patients with ET/PV. This trial was registered at www.clinicaltrials.gov as #NCT01259856.

Indexed as

Polycythemia VeraThrombocythemia, EssentialThrombosisDisease ProgressionHumansHydroxyureaInterferon-alphaHydroxyureaInterferon-alpha

Identifiers

PMID35007321
PMCPMC9101248
OpenAlexW4205527069

What Socratic holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.