Highlights from

ASH 2020

62nd Annual Meeting & Exposition of the American Society of Hematology

Virtual 5 - 8 December 2020

Updates from the Myelofibrosis and Essential Thrombocythemia Observational (MOST) Study: Myelofibrosis

Patients receiving treatment for essential thrombocythemia and myelofibrosis report a greater symptom burden than patients who are not receiving treatment. The prospect of suffering treatment related symptoms may be contributing to a reluctance to receive treatment for these myeloproliferative disorders.

The Myelofibrosis and Essential Thrombocythemia Observational Study (MOST) (NCT02953704) aims to describe the clinical characteristics, treatments, and disease burden experienced by patients with essential thrombocythemia or myelofibrosis. The study began in 2016, and Dr Rami Komrokji (H. Lee Moffitt Cancer Center and Research Institute, Florida, USA) shared interim real-world data about 205 patients with myelofibrosis enrolled in MOST [1].

For inclusion in the myelofibrosis cohort of this prospective, longitudinal, non-interventional study, patients have to score as either low-risk or intermediate-1 risk using the Dynamic International Prognostic Scoring System. Of the 205 patients in this analysis, 85 (41.5%) were low-risk and 120 (58.5%) intermediate-1 risk. Participants in MOST report their symptoms using 2 measures: the myeloproliferative neoplasm (MPN) symptom assessment form, and the Total Symptom Score (TSS), which is the patient’s grading of symptom severity using a scale of 0 (absent) to 100 (worst imaginable). The combination of these scores constitutes the patient’s symptom burden. At the time of the presented analysis, objective findings of palpable splenomegaly, blood count, and comorbidities were generally comparable between the 2 cohorts. Mean TSS was lower in the low-risk cohort than in the intermediate-1 risk cohort.

Of the 85 patients in the low-risk cohort, 48 were receiving current myelofibrosis-directed treatment; 47 were not. Of the 120 in the intermediate-1 risk cohort, 71 were receiving current myelofibrosis-directed treatment; 49 were not. While differences in mean TSS were not significant, the proportion of patients with a mean TSS ≥20 was greater in treated versus untreated patients in both low- and intermediate-1-risk groups. Several patients from both cohorts who had received treatment prior to enrolment had discontinued therapy by the time of enrolment. Researchers speculate that the higher symptom burden experienced by patients receiving treatment contributed to the decision to discontinue treatment. These findings reveal that there is a need to develop effective and more well-tolerated second-line therapies for the treatment of myelofibrosis.

  1. Komrokji R, et al. To Treat or Not to Treat? Understanding Treatment Patterns in Patients with Lower-Risk Myelofibrosis at the Time of Enrollment in the MOST Study. 62nd ASH Annual Meeting, 5-8 December 2020. Abstract 152.

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