The MPN PROGRESSion Registry® is a patient-centric research initiative, designed to help provide better understanding of myeloproliferative neoplasms (MPNs) — a group of rare blood cancers that include, essential thrombocythemia (ET), polycythemia vera (PV), and myelofibrosis (MF). MPNs can gradually change into more aggressive forms of disease over time — such as progressing into another MPN subtype or transforming into secondary Acute Myeloid Leukemia (sAML). As a result, progression and its causes are a major concern for MPN patients, and a priority area of focus for researchers.

The MPN PROGRESSion Registry will track patient health records, genetic tests, treatments, and patient experiences over the next 5 to 10 years — striving to become the most comprehensive, longest-term collection of MPN patient health data in the United States — to answer a critical question: Why do MPNs progress, and what can we do to slow, stop, or reverse that progression?

By bringing together patients, doctors, researchers, and industry experts, the MPN PROGRESSion Registry aims to help the MPN community:

• understand the risk factors and causes of MPN disease progression.

• determine new ways to define, measure, and track progression of MPNs.

• improve treatments to help MPN patients live healthier, longer lives.

• provide insights to help personalize care.

• ultimately, discover ways to predict or reverse disease change

Many patients living with MPNs often worry about if, when, and how their disease will progress. Unfortunately, we still have a lot to learn about MPN disease progression. The current information and data available can be hard for researchers to access, not widely applicable from patient to patient, and is often missing key information specific to MPNs.

MPNs are complex diseases. They can be unpredictable, with no two patients’ experiences being exactly alike. Many questions remain unanswered as to why some patients’ conditions progress rapidly, while others remain stable. The MPN PROGRESSion Registry® will collect extensive data to help researchers, clinicians, and the pharmaceutical industry:

• discover what indicates progression — before it happens.

• better understand the different paths MPN diseases can take.

• provide a comprehensive resource that can be studied to gain actionable insights — and be put into practice to improve overall patient care.

• determine and agree to a widely accepted consensus, on:

  • what the defined stages of progression are.

  • the biomarkers that define the start and end of each stage.

• apply the best available therapeutics on an individualized basis.

• eventually, help identify the specific risk factors for progression — and help find a cure.

The MPN PROGRESSion Registry® stands out due to its scale, wide-reaching goals, and patient-centered approach:

• Knowledge insights: Aims to improve disease understanding and address challenges in the field of MPN progression.

• Large scale with a years-long approach: Designed to be the largest MPN registry in the United States, aiming to enroll thousands of patients in a long-term initiative that will follow participants for 5 to 10 years.

• Useful & comprehensive data: Widespread collection of data from medical records, genetic tests, patient-reported outcomes, and potentially more to maximize study outcomes.

• Broad representation: Participants will come from various locations, demographics, and backgrounds, and will include patients from across the spectrum of MPN diseases. This will ensure that the data reflects diverse patient biology and experiences.

• Actionable: Designed to deliver early insights into the patient journey, as well as improve understanding over the long term.

• Ease of enrollment & use: Sign-up is straightforward, and ongoing participation is easy for patients wishing to enroll.

• Trusted: The registry was created by MPN Research Foundation, and built by the experts at IQVIA, a global leader in patient registry technologies. There is rigorous oversight, and all data is highly confidential and secure.

• Goodwill: Patients can feel good being part of this exciting project and community.

The MPN PROGRESSion Registry® has been meticulously developed by MPN Research Foundation over a number of years, in collaboration with MPN patients, and leading scientific, research, and registry development experts. By incorporating best practices in data collection and analysis, the Registry is designed to yield reliable and impactful results.

The need and idea for a registry centered around MPN disease progression originated in meetings of the MPN Research Foundation Progression Research Network. In response, the Foundation began working to bring an MPN patient registry to life.

The MPN PROGRESSion Registry has been thoughtfully designed
During development, a feasibility study was conducted to assess and confirm the project’s viability and identify any possible roadblocks to long-term success. The study’s findings are detailed in this collaborative whitepaper and comprehensive report published by the MPN Research Foundation and IQVIA.

There is a planned, phased design for expanding the types of data included in the MPN PROGRESSion Registry over time. Each year of the study, additional and more complex data sets will be incorporated. This approach will enable greater impact, by delivering early insights while still expanding over time to meet larger, longer-term objectives.

The MPN PROGRESSion Registry is also structured to allow for continual improvement. It will regularly draw on participant insights and expert guidance to ensure it remains in line with cutting-edge research needs and priorities in the future.

The MPN PROGRESSion Registry® has an advisory Registry Steering Committee that provides input and oversight. The committee includes leading MPN specialists and other renowned researchers. Their expertise and guidance ensure the Registry’s design and analysis meet the highest scientific standards.

The MPN PROGRESSion Registry Steering Committee is:

• Raajit Rampal, MD, PhD – Director, Myeloproliferative Neoplasms Program at Memorial Sloan Kettering Cancer Center (Lead-PI)

• Doug Tremblay, MD – Assistant Professor of Medicine at Ichan School of Medicine at Mount Sinai (Co-PI)

• Jan Bewersdorf, MD, FACP – Assistant Professor of Medicine at Yale University (Co-PI)

• Gaith Abu-Zeinah, MD – Assistant Professor of Medicine at Weill Cornell Medicine

• Sibel Blau, MD – Medical Director at Northwest Medical Specialties and President of Quality Cancer Care Alliance Network (QCCA)

• Andriy Derkach, PhD – Assistant Attending Biostatistician at MSKCC (Statistician)

• Mithat Gönen, PhD – Chief, Biostatistics Service at MSKCC (Statistician)

• Michael Grunwald, MD, FACP – Chief, Leukemia Division at Atrium Health

• Claire Harrison, MD, DM, FRCP, FRCPath – Professor of Hematology at Guy’s and St. Thomas’ NHS Foundation Trust, London, UK

• Andrew Kuykendall, MD – Clinical Researcher and Hematologist/Oncologist at Moffit Cancer Center

• Jeanne M. Palmer, MD – Vice Chair and Section Chief for Hematology at Mayo Clinic

• Tyler Parsons, PhD – Postdoctoral Research Fellow at Washington University School of Medicine in St. Louis

• Anand Patel, MD – Assistant Professor of Medicine at UChicago Medicine

• Robyn Shapiro, JD – Lawyer/Bioethicist at Health Sciences Law Group

• David Shoultz, PhD, MBA – Chief Scientific Officer at MPN Research Foundation

• Shireen Sirhan, MD – Assistant Professor, Oncology at Jewish General Hospital

• Kapila Viges – Chief Executive Officer at MPN Research Foundation

The MPN PROGRESSion Registry is entirely funded by the MPN Research Foundation, making participation completely free. Additional support is provided by individual donors, foundations, corporations, pharmaceutical companies, or other collaborative sponsors and partners.

The MPN PROGRESSion Registry® is a team effort, involving expert organizations dedicated to improving care and research for people living with myeloproliferative neoplasms (MPNs). The key development partners are:

IQVIA
IQVIA built and manages the Registry patient and data portal. IQVIA is a global leader in healthcare technology and data solutions, with years of experience creating tools like patient registries to help researchers uncover valuable insights and advance medical research.

Sobi
Sobi is a biopharmaceutical company that works to improve the lives of people with rare diseases, including MPNs. Their partnership provides primary funding and expert support to help the Registry succeed. Sobi brings valuable industry knowledge and resources to this project, and their focus on rare diseases makes them a great fit for this important work.

Karyopharm
Karyopharm Therapeutics Inc. is a commercial-stage pharmaceutical company dedicated to pioneering novel cancer therapies. Their primary focus is on developing novel drugs which they hope will help treat patients with certain blood cancers or solid tumors. Karyopharm’s core technology leverages the inhibition of nuclear export as a mechanism to treat patients. Their partnership contributes both financial support and specialized knowledge to the Registry efforts.

GSK
GSK is a focused biopharma company working to prevent and treat disease with specialty medicines, vaccines and general medicines. They focus on the science of the immune system and advanced technologies, investing in four core therapeutic areas — respiratory, immunology and inflammation; oncology; HIV; and infectious diseases — to impact health at scale. Their partnership contributes both financial support and specialized knowledge to the Registry efforts.