Details

Details

Title 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

IRB INCT1Z16

CC 17-119

Hospital Main Campus

Disease Essential Thrombocythemia (ET), Myelofibrosis

Description

Description

Primary Objectives
  • Describe clinical characteristics and evolution of disease burden in the ET and MF cohorts per Protocol definitions.
Secondary Objectives
  • Describe patterns of treatment, therapies, and clinical management.
  • Describe disease progression over time.
  • Describe the patterns of hematocrit, hemoglobin, WBC count, and platelet counts over time.
  • Describe the comorbidities associated with disease and progression.
  • Describe changes in patient-reported symptoms and QOL.
  • Describe the rate and time to leukemic transformation.
  • Describe rate of all-cause mortality and aggregate causes of mortality.
  • Additional objectives for MF cohort only:
    • Describe reasons for patient ineligibility based on DIPSS during screening.
    • Describe time to first disease-related intervention or first progression event during the period of observation.
Exploratory Objectives

At the time of study enrollment, patients from the MF and ET cohorts will be invited to participate in any of 3 optional substudies, which require additional peripheral blood, BM, or saliva sample collection for study participation. Patients who elect to participate in any of the substudies will be required to sign additional consent forms documenting their willingness to participate in each of the substudies.

  • Patients in the peripheral blood study will provide samples at approximately 6-month intervals.
  • Patients in the BM study will provide samples at approximately 12-month intervals.
  • Patients in the saliva study will only provide a sample at the baseline visit.
Inclusion Criteria

Inclusion Criteria

  1. Age ≥ 18 years.
  2. MF cohort: Diagnosis of MF (including PMF, PET-MF, or PPV-MF) and low-risk using DIPSS risk categorization OR intermediate-1 risk by DIPSS by reason of age alone.
  3. ET cohort: Diagnosis of ET and age ≥ 60 years OR history of thromboembolic events OR currently receiving ET-directed therapy (eg, HU, anagrelide, interferon, busulfan, ruxolitinib).
  4. Willing and able to provide written informed consent.
  5. Willing and able to complete patient assessment questionnaires either alone or with minimal assistance from a caregiver and/or trained site personnel.
  6. Under the supervision of a physician for the current care of MF or ET.
  7. Individuals participating in investigational drug studies sponsored by other companies may be enrolled, if the other study allows concurrent enrollment in an observational study.
  8. Individuals participating in interventional studies of other sponsors (non-Incyte) may participate in the MOST study, if the other study does not prohibit participation in an observational study.
Exclusion Criteria

Exclusion Criteria

  1. Individuals who are participating in blinded investigational drug studies.
  2. 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.
  3. Life expectancy ≤ 6 months.
  4. Diagnosis of secondary AML, myelodysplastic syndrome, chronic myelogenous leukemia, or secondary thrombocytosis.