Background: Myeloproliferative neoplasms (MPNs) are rare malignancies that include myelofibrosis (MF), polycythemia vera (PV), and essential thrombocythemia. The objective of this study is to assess current clinical practices of hematologist/oncologists (hem/oncs) related to current and emerging therapies for MPNs, in order to identify knowledge, competency, and practice gaps.
Methods: A continuing medical education (CME)-certified clinical practice assessment consisting of 25 multiple-choice questions was developed to measure knowledge, skills, attitudes, and competence of hem/oncs regarding current and emerging MPN therapies. The self-assessment was available online to physicians without monetary compensation or charge. Respondent confidentiality was maintained, and responses were de-identified and aggregated for reporting. The activity launched on November 21, 2019 and responses collected through July 12, 2020 were analyzed and reported.
Results: A total of 2,035 learners participated in the activity and 218 hem/oncs answered all questions in the assessment. Hem/onc demographics and patient load distributions are reported in Table 1. A majority (77%) of hem/oncs lack confidence treating adverse events of MPN therapies (Table 2). Baseline knowledge and competence of hem/oncs were grouped into themes:
Knowledge/Competence with Risk Stratification, Prognostic Scoring, and Symptom Assessment: While 87% and 63%, demonstrated knowledge about variables included on the MPN Symptom Assessment Form Total Symptom Score and the Dynamic International Prognostic Scoring System (DIPSS), respectively, only 50% demonstrated knowledge about variables included in the mutation-enhanced International Prognostic Scoring System (MIPSS70). Additionally, only 27% demonstrated competence risk stratifying a patient with high-risk MF according to DIPSS.
Knowledge of Available Therapies: While 62% demonstrated knowledge that the 5-year data from the COMFORT-1 trial found ruxolitinib demonstrated an overall survival benefit in patients with intermediate-2 (INT-2) and high-risk MF, only 57% demonstrated knowledge that in addition to reduction in spleen volume, ruxolitinib improved symptoms of patients with MF in the COMFORT trials. Additionally, only 56% demonstrated knowledge that in the RESPONSE trial ruxolitinib increased hematocrit control in patients with PV. A majority (77%) did not demonstrate knowledge that both ruxolitinib and fedratinib are approved for the first-line treatment of patients with high-risk MF.
Knowledge of Emerging Therapies:Momelotinib: 72% did not demonstrate knowledge that in the SIMPLIFY-2 trial momelotinib increased symptom improvement compared to best available therapy (BAT) in patients with MF who were pretreated with ruxolitinib. Pacritinib: 47% did not demonstrate knowledge that in the PERSIST trial pacritinib did not demonstrate improved overall survival compared to BAT. Ropeginterferon: 80% did not demonstrate knowledge of the efficacy outcomes in patients with PV who received ropeginterferon in the PROUD-PV study.
Knowledge and Competence Managing Adverse Events (AE): A majority (75%) demonstrated competence treating ruxolitinib-associated thrombocytopenia in a patient with INT-2 MF, however only 35% and 44%, respectively, demonstrated knowledge of the need to address thiamine deficiency before starting a patient on fedratinib (encephalopathy risk) and knowledge that diarrhea was the most common nonhematologic AE seen with ruxolitinib in patients on the COMFORT-II trial.
Conclusions: This research identified several knowledge, competence, and confidence deficits for hem/oncs related to current and emerging MPN therapies in the areas of: (1) Risk stratification and use of prognostic scoring tools, (2) Clinical trial safety and efficacy data for currently available and emerging therapies, (3) Personalizing treatment selection for patients with MPNs, (4) Preventing and managing treatment-related AEs.
Additional education is needed to address these gaps for hem/oncs who care for patients with MPNs, which is expected to translate into improved clinical performance and better patient outcomes.
Acknowledgements: This CME activity was supported by an independent educational grant from Incyte Corporation.
No relevant conflicts of interest to declare.
Asterisk with author names denotes non-ASH members.