Prospective Observational Study Of Patients With Polycythemia Vera In US Clinical Practices (REVEAL)

Study Overview
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Brief Summary
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This is a Phase IV, multicenter, non-interventional, non-randomized, prospective, observational study in an adult population (patients >18 years old) of men and women who have been diagnosed with clinically overt PV and are being followed in either community or academic medical centers in the United States who will be enrolled over a 12-month period and observed for 36 months from the date the last patient is enrolled.

Official Title
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Prospective, Non-interventional Study of Disease Progression and Treatment of Patients With Polycythemia Vera in United States Academic or Community Clinical Practices

Conditions
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MPN (Myeloproliferative Neoplasms)

Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Age ≥18 years Diagnosis of Polycythemia Vera (PV) 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 PV including but not limited to watchful waiting, acetylsalicylic acid (ASA) 81mg or greater, antithrombotic therapy, Phlebotomy (PHL), Hydroxyurea (HU), interferon (recombinant or pegylated), busulfan, anagrelide Exclusion Criteria: Participation in an active clinical trial in which the study treatment is blinded Life expectancy <6 months Diagnosis of myelofibrosis (MF) [including primary MF, post-PV MF, or post-essential thrombocythemia MF (post-ET MF)] Diagnosis of secondary Acute Myeloid Leukemia (AML) Diagnosis of Myelodysplastic Syndrome (MDS) History of or active plan to proceed to allogeneic hematopoietic stem cell transplant in next 3 months Splenectomy

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 |
| --- | --- |
| Cohort A<br>Patients with clinically overt PV (and not exhibiting any of the characteristics listed for Cohort B), managed with: Watchful waiting (with or without aspirin)*, or Phlebotomy (PHL) alone (with or without aspirin)* - or HU alone (without concomitant PHL, with or without aspirin). (*Unless patient has a history of intolerance or clinical resistance/ refractoriness to hydroxyurea [HU] (as assessed by the treating physician) - in which case, s/he belongs to Cohort B) | |
| Cohort B<br>Patients with clinically overt PV, with one or more of the following disease characteristics: Treatment with HU and PHL in combination or Treatment with any agent other than HU or aspirin (e.g., recombinant interferon (IFN) or pegylated IFN preparations, busulfan, anagrelide) or A history of thrombosis (venous or arterial) or A history of intolerance or clinical resistance/ refractoriness to HU (as assessed by the treating physician) or Presence of documented splenomegaly (clinically assessed by palpation) or Presence of one or more of the following uncontrolled symptoms related to PV despite therapy (Symptoms deemed uncontrolled as per physician's judgment) Tiredness Difficulty sleeping Itching Muscle aches and/or bone pain Night sweats Sweats while awake Other | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Description of patterns of the disease Polycythemia Vera (PV), and associations of such patterns with patients' exposures or treatments |  | Every 3 months for approximately 36 months | 
| Incidence (frequency) of disease progression |  | Every 3 months for approximately 36 months | 
| Incidence (frequency) of healthcare resources utilization | Healthcare resources were defined as a. Medical visits (including Office visits, ER visits, and hospitalizations), b. phlebotomy (PHL) procedures and c. prescriptions (including PV-related prescriptions, PV-related over-the-counter (OTC) medications, and prescription medications for co-morbid conditions). | Every 3 months for approximately 36 months | 
| Incidence (frequency) of complications due to PV |  | Every 3 months for approximately 36 months | 
| Incidence (frequency) and description of PV-related symptoms |  | Every 3 months for approximately 36 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence (frequency) of adverse events (for those patients on active therapies, including PHL) |  | Baseline through end of study. Approximately 36 months | 
| Patient-reported outcomes as assessed by Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS) for assessment of the severity of symptoms | The MPN-SAF TSS is a validated 10-item instrument that efficiently assesses the prevalence and severity of PV symptoms in both clinical practice and trial settings. Higher scores indicate more severe symptoms and greater inactivity with a possible total score range of 0-100. | Every 3 months for approximately 36 months | 
| Work Productivity and Activity Impairment Questionnaire - Specific Health Problem (WPAI-SHP) score for assessment of work productivity and activity impairment | WPAI-SHP is a validated 6-item instrument that measures the effect of overall health and specific symptoms on productivity at work and outside of it. All outcomes are presented as a proportion, with 0% representing minimal/no impairment and 100% representing maximal impairment or productivity loss. | Every 3 months for approximately 36 months | 
| European Organization for Research and Treatment of Cancer Core Quality of Life Questionnaire (EORTC QLQ-C30) for Health Related Quality of Life (HRQoL) |  | Every 3 months for approximately 36 months | 
| Caregiver burden | The amount of health care received by a paid healthcare worker or by an unpaid caregiver | Every 3 months for approximately 36 months | 
| Burden of phlebotomy (PHL) | A patients self-reported assessment of burden including pain at site, difficulty and inconvenience of scheduling and cost factors. | Every 3 months for approximately 36 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Hematologic Agents, Enzyme Inhibitors, Molecular Mechanisms of Pharmacological Action, Nucleic Acid Synthesis Inhibitors, Polycythemia, Polycythemia Vera, Hematologic Diseases, Myeloproliferative Disorders, Bone Marrow Diseases, Hydroxyurea, Antineoplastic Agents, Therapeutic Uses, Pharmacologic Actions, Antisickling Agents