Polycythemia Vera
Conditions
Keywords
ruxolitinib, polycythemia vera, PV, jakavi
Brief summary
Jakavi® therapy for polycythemia vera (PV) has so far been studied exclusively in clinical trials and at selected clinical trial centres. This observational study is intended to document the therapy of PV in daily practice with a broad patient population and a geographically representative selection of German centres (both hospitals and practices). The prospective mapping of daily practice reality is thus the main goal of this project.
Detailed description
To achieve meaningful results in accordance with the study objective and to obtain long-term data from daily clinical practice in a real-world setting, the observation period under Jakavi® therapy is specified as 36 months.
Interventions
OTHERJakavi
Prospective observational study. There is no treatment allocation. Patients administered Jakavi by prescription and administered according to the SmPC.
Sponsors
Novartis Pharmaceuticals
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to 120 Years
Healthy volunteers
No
Inclusion criteria
* Adult male and female patients with PV for whom Jakavi® therapy is indicated according to the European summary of product characteristics * Patients who have been informed about this NIS and gave written consent
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Thromboembolic events | Up to 36 months | Number of patients with thromboembolic events. |
| Overall survival | Up to 36 months | Overall survival for JAK inhibitor naive and pretreated patients |
| Hematology | Up to 36 months | Number of patients with changes in different blood count values over time (hematocrit, erythrocytes, thrombocytes, leukocytes) |
| Number of phlebotomies | Up to 36 months | Total number of phlebotomies |
| Change in spleen size (or volume) | Up to 36 months | Measured by palpation/sonography/CT/MRI |
| Quality of Life (QoL) - MPN-SAF TSS; MPN-10 | Up to 36 months | The Myeloproliferative Neoplasm (MPN) Symptom Assessment Form Total Symptom Score (MPN-SAF TSS; MPN-10) questionnaire contains the ten most clinically relevant symptoms reported by patients with MPNs. It includes disease related symptoms each scored from 0 (absent) to 10 (worst imaginable). Total Scores range from 0-100, with higher scores indicating a greater number of symptoms and severity. |
| Dosing | Up to 36 months | Number of patients by initial dosing and number of patients with dose modifications will be provided |
| Treatment interruptions | Up to 36 months | Number of patients with treatment interruptions |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in constitutional symptoms | Up to 36 months | Number of patients with change in constitutional symptoms |
| Quality of Life (QoL) - (SF-36) | Up to 36 months | The Short Form-36 questionnaire consists of questions measuring physical function, physical role limitation, pain, general health, vitality, social function, emotional role limitations, and mental health status. The scores that can be obtained from the scale vary between 0 and 100 and the increase in the scores indicates that the quality of life is high. |
| Bone marrow aspiration and biopsy | Up to 36 months | Number of Biopsies per visit |
| Molecular examination | Up to 36 months | Number of performed assessments of JAK2V617F mutation per visist |
| Eastern Cooperative Oncology Group (ECOG) performance status | Up to 36 months | The ECOG performance status is a scale used to assess how a patient's disease is progressing, assess how the disease affects the daily living abilities of the patient, and determine appropriate treatment and prognosis. The grade ranges from 0 (fully active, able to carry on all pre-disease performance without restriction) to 5 (dead) |
Countries
Germany
Outcome results
None listed