Abstract 4912: A snapshot of myeloproliferative neoplasms in the United States: Analysis of the “myMPN” patient registry
Authors: Scherber Robyn M. , Whyte Lindsey , Woehrle Michelle , Harrison Claire , Mascarenhas John , Verstovsek Srdan , Moliterno Alison , Mesa Ruben A. 
Published: August 2019

Abstract
Introduction: The myeloproliferative neoplasms (MPNs) are an uncommon type of hematologic malignancy which can be accompanied by a medically complex sequalae and severe symptom burden. Patient registries allow for the evaluation and monitoring of clinically meaningful outcomes in rare diseases over time. Although patient registries exist for MPNs, the utility of these registries has been limited by inclusion of only particular institutions and/or regions or the lack of patient reported outcomes, specifically symptoms and quality of life. In September 2017, the “myMPN” patient registry began enrollment as the first MPN patient-centered registry. The purpose of this analysis is to report patient-reported disease features, outcomes, and events uploaded to the registry to date.
Methods: The “myMPN” patient registry was created by the MPN Research Foundation’s steering committee and hosted on the Genetic Alliance registry platform. Utilizing previous questions created for MPN populations and validated assessment tools, the myMPN patient registry allows patients to input data disclosures, disease features, treatments, blood counts and symptoms. The registry has been granted independent IRB approval.
Results: Accrual: To date, the registry has 744 participants. Of these, 62% were female and mean age was 61 years (range 18-94). Disease-related information: The registry includes 38% essential thrombocythemia (ET) patients, 36% polycythemia vera (PV) patients, 23% myelofibrosis (MF) patients, and 3% patients who reported an alternative MPN diagnosis. 11.5% of patients were not aware of their mutation status. Disease events: Over the year since study initiation, there have been 2,100 reported disease-related events, which have included 825 blood draws, 298 phlebotomies, 207 MPN medication changes, 144 bone marrow biopsies, 77 transfusions, 39 thrombotic or bleeding events, and 30 genetic testing events. Since registry initiation, 6 patients reported a new ET to MF transformation, and 4 patients reported a PV to MF transformation. Disease Symptom Burden: To date, 400 patients have completed 675 independent symptom assessments. Many have completed two or more symptom assessments. In general, MPN-10 symptom scores are similar to previously published cohorts, but provide data on longitudinal symptom change.
Conclusions: The myMPN patient registry facilitates the research and care of MPN patients by clinicians, researchers, patients, patient-advocates, and caregivers a common platform to interface prospectively. Future goals of the registry are to 1) explore variables related to disease progression/transformation, 2) expand outside of the United states to other English-speaking countries, 3) allow patients to connect with their physicians regarding their registry information, and 4) to develop a compendium medical record and specimen registry.
Citation Format: Robyn M. Scherber, Lindsey Whyte, Michelle Woehrle, Claire Harrison, John Mascarenhas, Srdan Verstovsek, Alison Moliterno, Ruben A. Mesa. A snapshot of myeloproliferative neoplasms in the United States: Analysis of the “myMPN” patient registry [abstract]. In: Proceedings of the American Association for Cancer Research Annual Meeting 2019; 2019 Mar 29-Apr 3; Atlanta, GA. Philadelphia (PA): AACR; Cancer Res 2019;79(13 Suppl):Abstract nr 4912.