Post-essential Thrombocythemia Myelofibrosis, Post-polycythemia Vera Myelofibrosis, Primary Myelofibrosis
Conditions
Keywords
Primary Myelofibrosis, PMF, post-polycythemia vera myelofibrosis, PPV MF, post-essential thrombocythemia myelofibrosis, post ET MF, Ruxolitinib, Jakavi
Brief summary
This was a prospective, two-arm, non-interventional study of JAKAVI® (Ruxolitinib) in patients with myelofibrosis
Detailed description
The purpose of this NIS was to gather data from the daily clinical practice of the Jakavi®-treatment in a broad patient population. In order to evaluate the direct effect of Jakavi®, only JAK inhibitor naive patients were documented in the first study arm; patients pretreated with JAK inhibitors were documented in the second study arm to evaluate the long-term efficacy of Jakavi® in this subpopulation. The documentation of all patients was carried out prospectively and began after the baseline visit. The medical decision on which therapeutic and diagnostic measures to take was made solely by the responsible physician. The observational period per patient was 36 months. The visit schedule after the baseline visit was set by the responsible physician according to standard clinical care, the clinical condition of the respective patients and the SmPC.
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
* Male and female patients with Primary Myelofibrosis (PMF), post-Polycythemia Vera-Myelofibrosis (PPV-MF), or post-Essential Thrombocythemia-Myelofibrosis (post-ET-MF), for whom Jakavi® therapy is indicated. * Patients that were informed about all aspects of this NIS and provided written informed consent.
Exclusion criteria
\-
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of patients with co-morbidities | Up to 36 months | Number of patients with co-morbidities was collected |
| Blood transfusion dependency | Up to 36 months | Number of patients with blood transfusion dependency was collected |
| Number of patients with concomitant medications | Up to 36 months | Number of patients with concomitant medications prescribed for myelofibrosis therapy and for the management of side effects was collected |
| Safety and tolerability | Up to 36 months | Evaluation of all occurring adverse events, serious adverse events and serious and non-serious adverse drug reactions |
| Spleen size (or volume) reduction | Up to 36 months | Spleen size (or volume) reduction was measured by palpation |
| 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). |
| Change in the number of patients with constitutional symptoms | Up to 36 months | Number of patients with change in constitutional symptoms was collected |
| Assessment of the Quality of Life (QoL) - Myeloproliferative Neoplasm - Symptom Assessment Form (MPN-SAF) | Baseline, month 1, month 3, month 6, month 12, month 24 and month 36 | The MPN-SAF questionnaire contains important questions that cover MF-specific symptoms whose analysis is part of the standard of care. 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. |
| Assessment of the Quality of Life (QoL) - Short Form-36 (SF-36) | Baseline month 6, month 12, month 24 and month 36 | This 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. |
| Overall survival | Up to 36 months | Overall survival for JAK inhibitor naive and pretreated patients |
| Ruxolitinib start and end dose | Up to 36 months | Ruxolitinib start and end dose was collected |
| Therapy discontinuation and dose adjustments | Up to 36 months | Number of participants with therapy discontinuation and dose adjustments was collected |
Countries
Germany
Outcome results
None listed