MPN (Myeloproliferative Neoplasms)
Conditions
Keywords
Polycythemia, Polycythemia Vera, Hematologic Diseases, Myeloproliferative Disorders, Bone Marrow Diseases, Hydroxyurea, Antineoplastic Agents, Therapeutic Uses, Pharmacologic Actions, Antisickling Agents, Hematologic Agents, Enzyme Inhibitors, Molecular Mechanisms of Pharmacological Action, Nucleic Acid Synthesis Inhibitors
Brief summary
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.
Interventions
None listed
Sponsors
Incyte Corporation
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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 | Every 3 months for approximately 36 months | 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). |
| 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
| Measure | Time frame | Description |
|---|---|---|
| Incidence (frequency) of adverse events (for those patients on active therapies, including PHL) | Baseline through end of study. Approximately 36 months | — |
| Burden of phlebotomy (PHL) | Every 3 months for approximately 36 months | A patients self-reported assessment of burden including pain at site, difficulty and inconvenience of scheduling and cost factors. |
| Patient-reported outcomes as assessed by Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS) for assessment of the severity of symptoms | Every 3 months for approximately 36 months | 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. |
| Work Productivity and Activity Impairment Questionnaire - Specific Health Problem (WPAI-SHP) score for assessment of work productivity and activity impairment | Every 3 months for approximately 36 months | 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. |
| 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 | Every 3 months for approximately 36 months | The amount of health care received by a paid healthcare worker or by an unpaid caregiver |
Countries
Puerto Rico, United States
Outcome results
None listed