Patient-reported outcomes of patients with myelofibrosis or essential thrombocythemia enrolled in the MOST study

Abstract

The Myelofibrosis and Essential Thrombocythemia Observational STudy (MOST; NCT02953704) is an ongoing, noninterventional study assessing clinical characteristics and patient-reported outcomes (PROs) of patients with myelofibrosis (MF) or essential thrombocythemia (ET). This analysis assessed PROs at enrollment; symptom burden and quality of life (QoL), work productivity, and activity were assessed using validated questionnaires in patients with low- or intermediate-1-risk (age-alone) MF, or high- or low-risk ET (receiving ET-directed therapy) at enrollment. In MF and ET cohorts, fatigue had highest mean symptom score. Women had higher mean total symptom scores (TSS), mean symptom scores, and reduced QoL versus men. In patients with MF, mean TSS and symptom scores were similar between risk groups. Patients with low-risk ET had higher mean TSS and symptom scores than patients with high-risk ET. In conclusion, patients with lower risk MF and low- or high-risk ET experience significant symptom burden affecting QoL and ability to work.

Keywords:

• Myelofibrosis
• essential thrombocythemia
• observational study
• patient-reported outcomes
• symptom burden
• quality of life

Acknowledgements

The authors wish to thank the patients and their families, the investigators, and the site personnel who participated in this study. Medical writing and editorial assistance were provided by Rachel Shparberg, PhD, of Envision Pharma Group (Philadelphia, PA, USA), and was funded by Incyte Corporation.

Author contributions

All authors were involved in conceptualization, methodology, data curation, writing review, and editing drafts. RS, PK, and HR were involved in compiling and analyzing the study data and interpreted data in collaboration with all authors. All authors had full access to all the data in the study and provided input on additional analyses before the writing of the report.

Disclosure statement

Ellen Ritchie has a consulting role at Celgene, Incyte Corporation, Novartis, and Pfizer; participated in the speakers’ bureau for Ariad, Celgene, Incyte Corporation, and Novartis; received research funding from Astellas, Bristol Myers Squibb, Novartis, NS Pharma, and Pfizer; received travel support from Celgene and Novartis. Anas Al-Janadi holds honoraria from GSK and Karyopharm Therapeutics. Craig Kessler sits on the advisory board for Incyte Corporation and received research support from Incyte Corporation. Patricia Kalafut and Haobo Ren hold employment and stock ownership from Incyte Corporation. Robyn Scherber was a previous employee and stock owner of Incyte Corporation. Ruben Mesa has a consulting role at Incyte Corporation, La Jolla Pharmaceutical Company, Novartis, and Sierra Oncology; received research funding from AbbVie, Celgene, CTI Biopharma, Genotech Pharma, Incyte Corporation, Promedior, and Samus.

Data availability statement

Incyte Corporation (Wilmington, DE) is committed to data sharing that advances science and medicine while protecting patient privacy. Qualified external scientific researchers may request anonymized datasets owned by Incyte for the purpose of conducting legitimate scientific research. Researchers may request anonymized datasets from any interventional study (except phase 1 studies) for which the product and indication have been approved on or after 1 January 2020 in at least one major market (e.g. United States, European Union, Japan). Data will be available for request after the primary publication or 2 years after the study has ended. Information on Incyte’s clinical trial data sharing policy and instructions for submitting clinical trial data requests are available at: https://www.incyte.com/Portals/0/Assets/Compliance%20and%20Transparency/clinical-trial-data-sharing.pdf?ver=2020-05-21-132838-960

Additional information

Funding

This work was supported by Incyte Corporation (Wilmington, DE, USA).