Myelofibrosis and Essential Thrombocythemia Observational Study (MOST)

Study Overview
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Brief Summary
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The purpose of this prospective, longitudinal, noninterventional study is to describe clinical characteristics, evolution of disease burden, and treatment patterns in patients with select subcategories of essential thrombocythemia (ET) or myelofibrosis (MF).

Official Title
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Prospective, Longitudinal, Non-Interventional Study of Disease Burden and Treatment of Patients With Low-Risk Myelofibrosis (MF) or High-Risk Essential Thrombocythemia (ET) or ET Patients Receiving ET-Directed Therapy

Conditions
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MPN (Myeloproliferative Neoplasms)

Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: MF cohort: Diagnosis of MF and low-risk using DIPSS risk categorization OR intermediate-1 risk by DIPSS by reason of age alone. ET cohort: Diagnosis of ET and age ≥ 60 years OR history of thromboembolic events OR currently receiving ET-directed therapy (eg, hydroxyurea, anagrelide, interferon, busulfan, ruxolitinib, etc). Willing and able to provide written informed consent. Willing and able to complete patient assessment questionnaires either alone or with minimal assistance from a caregiver and/or trained site personnel. Under the supervision of a physician for the current care of MF or ET. Exclusion Criteria: Individuals who are participating in blinded investigational drug studies. Individuals who are participating in Incyte investigational/interventional drug trials (company- or investigator-sponsored studies) until they have completed the 30-day end of study visit. Life expectancy ≤ 6 months. Diagnosis of secondary acute myeloid leukemia, myelodysplastic syndrome, chronic myelogenous leukemia, or secondary thrombocytosis.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Myelofibrosis Cohort<br>Patients will be categorized as low-risk using Dynamic International Prognostic Scoring System (DIPSS) risk OR intermediate-1 risk by DIPSS by reason of age alone. | |
| Essential Thrombocythemia Cohort<br>Patients will be age ≥ 60 years OR have history of thromboembolic events OR currently receiving ET-directed therapy. | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Description of the clinical characteristics and evolution of disease burden in essential thrombocythemia (ET) and myelofibrosis (MF) patients |  | Approximately every 6 months through end of study, up to approximately 36 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Description of patterns of treatment, therapies, and clinical management |  | Approximately every 3-6 months through end of study, up to at least 36 months | 
| Description of disease progression over time |  | Approximately every 3-6 months through end of study, up to at least 36 months | 
| Describe hematocrit, hemoglobin, white blood cell (WBC) count, and platelet counts over time |  | Approximately every 3-6 months through end of study, up to at least 36 months | 
| Description of the comorbidities associated with disease and progression |  | Approximately every 3-6 months through end of study, up to at least 36 months | 
| Description of changes in patient-reported symptoms and quality of life (QOL) |  | Approximately every 3-6 months through end of study, up to at least 36 months | 
| Description of the rate and time to leukemic transformation |  | Approximately every 3-6 months through end of study, up to at least 36 months | 
| Description of rate of all-cause mortality and aggregate causes of mortality |  | Approximately every 3-6 months through end of study, up to at least 36 months | 
| Description of reasons for patient ineligibility based on Dynamic International Prognostic Scoring System (DIPSS) during screening (MF patients only) |  | At screening | 
| Description of time to first disease-related intervention or first progression event during the period of observation (MF patients only) |  | Baseline to end of study, up to 36 months. | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Myelofibrosis, essential thrombocythemia, clinical characteristics, disease burden, patient-reported outcomes