A Prospective, Two-arm, Non-interventional Study of JAKAVI® (Ruxolitinib) in Patients With Myelofibrosis

Novartis Pharmaceuticals1,012 enrolled

Overview

This was a prospective, two-arm, non-interventional study of JAKAVI® (Ruxolitinib) in patients with myelofibrosis

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.

Study Type

OBSERVATIONAL

Enrollment

1,012

Conditions

Primary Myelofibrosis Post-polycythemia Vera Myelofibrosis Post-essential Thrombocythemia Myelofibrosis

Interventions

JakaviOTHER

Prospective observational study. There is no treatment allocation. Patients administered Jakavi by prescription and administered according to the SmPC.

Eligibility

Sex: ALLMin age: 18 YearsMax age: 120 Years

Medical Language ↔ Plain English

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: \-

Locations (1)

Novartis Investigative Site

Aachen, Germany

Outcomes

Primary Outcomes

Safety and tolerability

Evaluation of all occurring adverse events, serious adverse events and serious and non-serious adverse drug reactions

Time frame: Up to 36 months

Spleen size (or volume) reduction

Spleen size (or volume) reduction was measured by palpation

Time frame: Up to 36 months

Eastern Cooperative Oncology Group (ECOG) performance status

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).

Time frame: Up to 36 months

Change in the number of patients with constitutional symptoms

Number of patients with change in constitutional symptoms was collected

Time frame: Up to 36 months

Assessment of the Quality of Life (QoL) - Myeloproliferative Neoplasm - Symptom Assessment Form (MPN-SAF)

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.

Time frame: Baseline, month 1, month 3, month 6, month 12, month 24 and month 36

Assessment of the Quality of Life (QoL) - Short Form-36 (SF-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.

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.