The myMPN patient registry for patients with myeloproliferative neoplasms (MPNs) enables research and care for patients with MPNs and allows prospective interfacing in this disease state, explained researchers in a presentation at the American Association of Cancer Research (AACR) 2019 Annual Meeting in Atlanta, Georgia. The registry was created by the MPN Research Foundation for patients with MPNs, including essential thrombocythemia (ET), myelofibrosis (MF), and polycythemia vera (PV). Patient enrollment began in September 2017 and is the first patient-focused registry in this therapeutic area.
MPNs are rare hematologic malignancies that have severe symptoms and clinically complicated sequalae. Patient registries can allow longitudinal assessment and monitoring of clinical outcomes in rare diseases. Regional or institutional limitations and the absence of patient-reported outcomes around symptoms and quality of life have affected the usefulness of other existing patient registries for MPNs.
The myMPN registry currently encompasses 744 patients, 62% of whom are female. Mean age is 61 years (range, 18 to 94). Though 11.5% of patients were not aware of their mutation status, 38% have ET, 36% have PV, 23% have MF, and 3% have an alternative MPN diagnosis.
Since initiating enrollment, 2100 reported disease-related events included 825 blood draws, 298 phlebotomies, 207 changes in medication for MPN treatment, 144 bone marrow biopsies, 77 transfusions, 39 bleeding or thrombotic events, and 30 genetic testing events.
In 6 patients, ET transformed to MF, and in 4 patients, PV transformed to MF. More than half of patients (n=400) completed 675 independent symptom assessments. Symptom scores are generally similar to those in previously published cohorts. Additionally, here the scores provide an opportunity for longitudinal evaluation of symptom scores.
Future goals of the myMPN registry are to enable patients to connect with their clinicians about their registry information, assess factors affecting disease progression and transformation, expand beyond the United States to other English-speaking countries, and create a compendium medical record and specimen registry.
Scherber RM, Whyte L, Woehrle M, et al. A snapshot of myeloproliferative neoplasms in the United States: Analysis of the “myMPN” patient registry. Poster presented at: American Association for Cancer Research 2019 Annual Meeting; March 29-April 3, 2019; Atlanta, GA. Abstract 4912/5.