MPN PROGRESSion Registry: Observational Study Tracking Symptoms, Treatments, and Disease Progression in People With Myeloproliferative Neoplasms (MPNs)

NCT07362225 | Recruiting

• 1 other identifier: MPNRF-PROG-2025-001
• Study Type: observational
• Target: 5,000
• Locations: 1 country
• Sites: 2

Brief Summary

The MPN PROGRESSion Registry is a multi-year, observational research study designed to improve understanding of myeloproliferative neoplasms (MPNs)-a group of rare, chronic blood cancers that include polycythemia vera (PV), essential thrombocythemia (ET), primary myelofibrosis (MF), pre-fibrotic primary myelofibrosis (pre-PMF), secondary myelofibrosis, myeloproliferative neoplasm-unclassifiable (MPN-U), MPN in accelerated phase (MPN-AP), and MPN in blast phase (MPN-BP), post-MPN Acute Myeloid Leukemia (AML), and MDS/MPN overlap syndrome as defined above per WHO 2022 criteria, including patients originally diagnosed with one of these conditions but who have received one or more SCTs and/or BMTs . These conditions are characterized by abnormal blood cell production in the bone marrow and may lead to complications such as blood clots, bleeding, bone marrow fibrosis, and, in some cases, progression to acute leukemia. The central hypothesis of the registry is that collecting and analyzing real-world, longitudinal data-including electronic health records (EHRs), laboratory values, treatments, and patient-reported outcomes (PROs)-from a diverse population of people living with MPNs will help identify patterns and predictors of disease progression, treatment response, quality of life, and long-term outcomes. These insights are intended to guide future research, inform clinical guidelines, and support improvements in patient care. The registry is non-interventional and observational; participants do not receive investigational treatments, and all medical care continues under the supervision of their own physicians. Data collection includes EHRs, PRO surveys, patient-reported symptom and lab tracking, insurance claims, and, in the future, may include linkages with other relevant disease registries and datasets. Potential collaborations under consideration include those with the European LeukemiaNet (ELN) MPN Registry, the Mayo Clinic MPN Database, the Center for International Blood and Marrow Transplant Research (CIBMTR), the SEER Program, Harmony Alliance Foundation, and the National Cancer Database (NCDB). The registry emphasizes the patient voice, incorporating lived experiences related to hallmark MPN symptoms such as fatigue, pruritus (itching), bone pain, night sweats, and social and emotional impacts. Participants will be followed for at least five years, with many enrolled for ten years or longer, to capture the natural history of disease and long-term outcomes. PRO surveys will be completed approximately every six months, and EHR data will be regularly reviewed to track changes in clinical status, treatment, and disease evolution. Statistical analyses will use descriptive and inferential methods to examine clinical characteristics, symptom burden, disease trajectories, and patient-centered outcomes. Planned subgroup analyses may compare differences across diagnoses, treatment approaches, demographics, or genomic factors. Analytic plans will be finalized during the course of the study and may evolve in response to emerging scientific questions. The registry is open to adults (18 years or older) living in the United States who have been diagnosed with any of the included MPN subtypes and are willing to share health information and complete study surveys. Individuals currently enrolled in interventional clinical trials or unable to provide informed consent may be excluded. Participation is voluntary, and participants may withdraw from the study at any time without affecting their medical care. Privacy and data security are core priorities. Participant data will be securely stored and managed in accordance with all applicable privacy laws and research regulations. No identifiable information will be shared with external parties without appropriate authorization. Oversight is provided by a Steering Committee and a Patient Engagement Advisory Committee (PEAC), ensuring rigorous scientific, ethical, and patient-centered governance. The registry is sponsored by the MPN Research Foundation, a nonprofit organization advancing research and patient advocacy in myeloproliferative neoplasms (MPNs). Participants can contact the registry team at any time with questions and will receive periodic updates on study findings. This study aims to address critical gaps in understanding the real-world experiences of people with MPNs-such as symptom burden over time, risk factors for progression, and how different treatments impact patient outcomes. Findings may inform clinical trial design, support biomarker discovery, and contribute to the development of updated treatment recommendations. The registry is committed to including participants from diverse backgrounds and clinical settings to ensure findings are broadly applicable across the MPN community. Summary results will be shared through scientific publications, presentations, and other dissemination efforts to advance MPN research and care globally.

Conditions

Polycythemia Vera; MF; Accelerated Phase MPN; ET (Essential Thrombocythemia); Polycythemia Vera (PV); Essential Thrombocythemia (ET); Primary Myelofibrosis (MF); Primary Myelofibrosis (PMF); Myelofibrosis; Myelofibrosis (MF); Myelofibrosis, Primary; Myelofibrosis, Post ET; Myelofibrosis, Post PV; Myelofibrosis (PMF); Myelofibrosis，MF; Myelofibrosis; Primary Myelofibrosis; Post-polycythemia Vera Myelofibrosis; Post-essential Thrombocythemia Myelofibrosis; Myelofibrosis Due to and Following Polycythemia Vera; Myelofibrosis Transformation in Essential Thrombocythemia; Myelofibrosis With High Molecular Risk Mutations; Secondary Myelofibrosis; Secondary Myelofibrosis in Myeloproliferative Disease; Secondary Myelofibrosis (Post-Polycythemia Vera Myelofibrosis, Post-Essential Thrombocythemia Myelofibrosis); Post-Polycythemia Vera Myelofibrosis; Post-polycythemia Vera Myelofibrosis (PPV-MF); Post-polycythemia Vera Myelofibrosis (Post-PV MF); Post-polycythemia Vera Myelofibrosis(Post-PV MF); Post-PV MF; Post-Essential Thrombocythemia Myelofibrosis; Post-essential Thrombocythemia Myelofibrosis (PET-MF); Post-essential Thrombocythemia Myelofibrosis(Post-ET MF); Post-essential Thrombocythemia Myelofibrosis (Post-ET MF); Post-ET MF; Pre-fibrotic Myelofibrosis; Myeloproliferative Disorder; Myeloproliferative Disorders; Myeloproliferative Disorders (MPD); Myeloproliferative Neoplasms (MPNs); Myeloproliferative Neoplasm(MPN)-Associated Myelofibrosis; Myeloproliferative Neoplasm With 10% Blasts or Higher; Myeloproliferative Neoplasms; MPN; MPN (Myeloproliferative Neoplasms); MPN-associated Myelofibrosis; Myeloproliferative Neoplasm, Unclassifiable; Myeloproliferative Neoplasm, Not Otherwise Specified; Accelerated Phase Myeloproliferative Neoplasm; Blast Phase MPN; Blast Phase Myeloproliferative Neoplasm; Thrombocythemia Myelofibrosis (PET-MF); Thrombocythemia, Essential; Thrombocythemia, Hemorrhagic; Agnogenic Myeloid Metaplasia; Chronic Idiopathic Myelofibrosis; Idiopathic Myelofibrosis; MDS/MPN Crossover Syndromes

Keywords

Myeloproliferative Neoplasms; Polycythemia Vera; Essential Thrombocythemia; Secondary Myelofibrosis; Pre-fibrotic Primary Myelofibrosis; Myeloproliferative Disorder; Myeloproliferative Neoplasm, Unclassifiable; Myeloproliferative Neoplasm, Accelerated Phase; Myeloproliferative Neoplasm, Blast Phase; Chronic Myeloproliferative Disease; Hematologic Neoplasms; Bone Marrow Neoplasms; Blood Cancer; Disease Progression; Biomarker Discovery; Patient-Reported Outcomes; Electronic Health Records; Real-World Evidence; Longitudinal Study; Observational Study; Registry Study; Natural History Study; Quality of Life; Rare Diseases; Chronic Hematologic Diseases; Risk Stratification; Symptom Burden; Medical Claims Data; Longitudinal Data Collection; Patient-Centered Research; Health Outcomes Research; Clinical Outcomes; Clinical Guidelines Development; Regulatory Science; Drug Development; Treatment Response

Outcome Measures

Primary Outcomes (1)

• Time to Disease Progression

  Time from enrollment to the first documented disease progression event, defined as transformation between MPN subtypes (for example, essential thrombocythemia or polycythemia vera to myelofibrosis), development of accelerated-phase MPN (MPN-AP), progression to blast phase (MPN-BP), or meeting updated World Health Organization (WHO) or International Working Group for Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) criteria for disease progression. This outcome measures the rate and timing of disease evolution over time and evaluates associations with treatments, exposures, and clinical risk factors among adults diagnosed with MPNs.

  Assessed continuously from enrollment over a minimum of 5 years per participant, with extended follow-up beyond 10 years for participants who remain active in the registry.

Secondary Outcomes (7)

• Overall Survival

  Assessed continuously from enrollment over a minimum of 5 years per participant, with extended follow-up beyond 10 years for active participants.

• Change in MPN-SAF Total Symptom Score (TSS)

  Assessed every 3-6 months over a minimum of 5 years per participant, with extended follow-up beyond 10 years where applicable.

• Event-Free Survival

  Assessed continuously from enrollment over a minimum of 5 years per participant, with extended follow-up beyond 10 years where applicable.

• Patient Global Impression Scale - Severity (PGI-S)

  Time Frame: Assessed every 3-6 months over a minimum of 5 years per participant, with extended follow-up beyond 10 years where applicable.

• EORTC QLG Core Questionnaire (QLQ-C30

  Time Frame: Assessed every 3-6 months over a minimum of 5 years per participant, with extended follow-up beyond 10 years where applicable.

Study Arms (1)

MPN Patient Cohort

Adults (18 years and older) diagnosed with a myeloproliferative neoplasm (MPN), including polycythemia vera (PV), essential thrombocythemia (ET), primary myelofibrosis (MF), secondary myelofibrosis, pre-fibrotic primary myelofibrosis (pre-PMF), myeloproliferative neoplasm-unclassifiable (MPN-U), myeloproliferative neoplasm-accelerated phase (MPN-AP), myeloproliferative neoplasm-blast phase (MPN-BP), or post-MPN acute myeloid leukemia (AML), and myelodysplastic syndrome (MDS)/MPN overlap syndrome as defined above per WHO 2022 criteria, including patients originally diagnosed with one of these conditions but who have received one or more stem cell transplants (SCTs) or bone marrow transplants (BMTs) enrolled in a prospective observational registry study to track clinical outcomes, disease progression, treatment patterns, patient-reported outcomes, and long-term health trajectories over time.

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Adults (18 years and older) diagnosed with a myeloproliferative neoplasm (MPN), including polycythemia vera, essential thrombocythemia, primary myelofibrosis, secondary myelofibrosis, pre-fibrotic primary myelofibrosis, MPN-unclassifiable, MPN-accelerated phase, or MPN-blast phase, post-MPN AML, or MDS/MPN overlap syndrome (including patients originally diagnosed with one of these conditions but who have received one or more SCTs or BMTs) residing in the United States. Participants are enrolled exclusively through direct-to-patient outreach using online recruitment, social media, digital advertising, and patient advocacy networks. All participants must provide electronic informed consent, have accessible electronic health record (EHR) data, and be willing to complete digital patient-reported outcome surveys. The study aims to capture a diverse national cohort for real-world, longitudinal research on disease progression, treatment patterns, and outcomes .

You may qualify if:

• Adults aged 18 years or older at the time of enrollment.
• Confirmed diagnosis of a myeloproliferative neoplasm (MPN), including one or more of the following subtypes, according to WHO 2022 criteria, including patients originally diagnosed with one of these conditions but who have received one or more stem cell transplants (SCTs) or bone marrow transplants (BMTs):
• Polycythemia vera (PV)
• Essential thrombocythemia (ET)
• Primary myelofibrosis (PMF)
• Secondary myelofibrosis (post-ET or post-PV MF)
• Pre-fibrotic primary myelofibrosis (pre-PMF)
• Myeloproliferative neoplasm-unclassifiable (MPN-U)
• Myeloproliferative neoplasm, accelerated phase (MPN-AP)
• Myeloproliferative neoplasm, blast phase (MPN-BP)
• Post-MPN acute myeloid leukemia (AML)
• Myelodysplastic syndrome (MDS)/MPN overlap syndrome
• Myeloproliferative neoplasm, blast phase (MPN-BP)
• Ability to provide informed consent electronically or through a legally authorized representative.
• Willingness to share health information, including electronic health records (EHR), laboratory values, and survey responses.

You may not qualify if:

• Individuals under 18 years of age.
• Inability to provide informed consent, either directly or through a legally authorized representative.
• Currently enrolled in an interventional clinical trial where participation would interfere with the ability to participate in this observational registry (based on investigator or sponsor judgment).
• Any condition that, in the judgment of the study team, would make participation in the registry infeasible or unsafe.

Sponsors & Collaborators

• MPN Research Foundation: lead
• Sobi, Inc.: collaborator
• Karyopharm Therapeutics Inc: collaborator
• GlaxoSmithKline: collaborator
• Memorial Sloan Kettering Cancer Center: collaborator

Study Sites (2)

MPN Research Foundation

Chicago, Illinois, 60654, United States

RECRUITING

Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States

RECRUITING

Related Links

• MPN Research Foundation MPN PROGRESSion Registry Webpage

MeSH Terms

Conditions

Polycythemia Vera; Thrombocythemia, Essential; Primary Myelofibrosis; Myeloproliferative Disorders; Blast Crisis; Hematologic Neoplasms; Bone Marrow Neoplasms; Disease Progression; Rare Diseases

Condition Hierarchy (Ancestors)

Neoplasms by Site; Neoplasms; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Blood Coagulation Disorders; Thrombocytosis; Blood Platelet Disorders; Hemorrhagic Disorders; Leukemia, Myelogenous, Chronic, BCR-ABL Positive; Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Cell Transformation, Neoplastic; Carcinogenesis; Neoplastic Processes; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Raajit Rampal, MD, PhD

  Memorial Sloan Kettering Cancer Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Stephanie Scobey, MPN, BS, RN

CONTACT

847-449-5307; sscobey@mpnrf.org

Sara Douglas, MSN, RN, OCN

CONTACT

7734923766; sdouglas@mpnrf.org

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 6, 2026
• First Posted: January 23, 2026
• Study Start: September 26, 2025
• Primary Completion (Estimated): September 8, 2035
• Study Completion (Estimated): September 8, 2035
• Last Updated: January 23, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)