Myelofibrosis and Essential Thrombocythemia Observational Study (MOST)

Completed

• Conditions: MPN (Myeloproliferative Neoplasms)

• Registration Number: NCT02953704
• Lead Sponsor: Incyte Corporation

Brief Summary

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).

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2016-11-01
• Study First Submitted QC: 2016-11-01
• Study First Posted: 2016-11-03
• Study Start: 2016-12-31
• Primary Completion: 2022-03-29
• Study Completion: 2022-03-29
• Status Verified: 2022-05
• Last Update Submitted: 2022-05-11
• Last Update Posted: 2022-05-13

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 1469

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.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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

Name	Time	Method
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 rate and time to leukemic transformation	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 reasons for patient ineligibility based on Dynamic International Prognostic Scoring System (DIPSS) during screening (MF patients only)	At screening
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 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 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.
Description of patterns of treatment, therapies, and clinical management	Approximately every 3-6 months through end of study, up to at least 36 months