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

Completed

• Conditions: Post-essential Thrombocythemia Myelofibrosis; Primary Myelofibrosis; Post-polycythemia Vera Myelofibrosis
• Interventions: Other: Jakavi

• Registration Number: NCT05044026
• Lead Sponsor: Novartis Pharmaceuticals

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.

Basic Information
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Recruitment & Eligibility
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Study & Design
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Trial Locations

Study Timeline

• Study Start: 2012-09-20
• Study First Submitted: 2021-09-06
• Study First Submitted QC: 2021-09-06
• Study First Posted: 2021-09-14
• Primary Completion: 2022-09-19
• Study Completion: 2022-09-19
• Status Verified: 2023-09
• Last Update Submitted: 2023-09-15
• Last Update Posted: 2023-09-21

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 1012

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.

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Exclusion Criteria

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Arm B: Pretreated patients	Jakavi	Patients pretreated with a JAK-inhibitor for more than 14 days prior to the baseline visit
Arm A: JAK inhibitor naive	Jakavi	JAK-inhibitor-naive patients, treatment start with ruxolitinib less than 14 days prior to the baseline visit

Primary Outcome Measures

Name	Time	Method
Number of patients with co-morbidities	Up to 36 months	Number of patients with co-morbidities was collected
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
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).
Ruxolitinib start and end dose	Up to 36 months	Ruxolitinib start and end dose was collected
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.
Therapy discontinuation and dose adjustments	Up to 36 months	Number of participants with therapy discontinuation and dose adjustments was collected
Blood transfusion dependency	Up to 36 months	Number of patients with blood transfusion dependency 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
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

Trial Locations

Locations (1)

Novartis Investigative Site; 🇩🇪 Aachen, Germany