Efficacy and Safety of Ruxolitinib in Patients With Myelofibrosis

Efficacy and Safety of Ruxolitinib in Patients With Myelofibrosis: A Retrospective Multicenter Study

Ruxolitinib is a Janus kinase (JAK) 1/2 inhibitor currently used in the treatment of Myelofibrosis (MF). Ruxolitinib confirmed improvements in splenomegaly, MF-related symptoms and survival benefit in COMFORT and JUMP studies. At present, the real-world data on the efficacy and safety of ruxolitinib in the treatment of MF in China is still insufficient. The aim of this study was to evaluate the efficacy and safety of ruxolitinib in patients with MF and to provide guidance for the usage of ruxolitinib in MF in China.This was a retrospective, multicenter study of MF patients who received ruxolitinib treatment in Shandong province from August 2012 to December 2021. Data were analyzed using SPSS. Overall survival (OS) and Event-free survival (EFS) were estimated using the Kaplan- Meier method.

Study Overview

• Status: Completed
• Conditions: Myelofibrosis
• Intervention / Treatment: Drug: Ruxolitinib

• Study Type: Observational
• Enrollment (Actual): 130

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Probability Sample

Study Population

MF patients including primary and secondary MF patients who received ruxolitinib treatment are going to be recruited.

Description

Inclusion Criteria:

1. Male or female patients aged ≥18 years with a diagnosis of primary or secondary MF by World Health Organization and International Working Group for Myeloproliferative Neoplasms Research and Treat- ment (IWG-MRT) criteria;
2. Received ruxolitinib treatment for ≥3 months.

Exclusion Criteria:

1. Malignant tumors with other progression or myelofibrosis secondary to other diseases;
2. Exclude myelofibrosis patients after splenectomy;
3. Patients with poor compliance with case follow-up or lost to follow-up.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
MF patients who received ruxolitinib treatment	Drug: Ruxolitinib

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The proportion of patients with a ≥35% reduction in palpable spleen volume from baseline.	Reduction in spleen volume is measured by magnetic resonance imaging/computerized tomography (MRI/CT).	From Week 0 through Week 24

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The proportion of patients with ≥50% reduction in Total Symptom Score (TSS) from baseline.	TSS is assessed by the MPN-10.	From Week 0 through Week 24

Collaborators and Investigators

• Sponsor: Qilu Hospital of Shandong University

Study record dates

Study Major Dates

• Study Start (Actual): August 1, 2012
• Primary Completion (Actual): December 1, 2021
• Study Completion (Actual): December 1, 2021

Study Registration Dates

• First Submitted: May 23, 2022
• First Submitted That Met QC Criteria: June 5, 2022
• First Posted (Actual): June 8, 2022

Study Record Updates

• Last Update Posted (Actual): June 8, 2022
• Last Update Submitted That Met QC Criteria: June 5, 2022
• Last Verified: May 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Bone Marrow Diseases; Hematologic Diseases; Myeloproliferative Disorders; Primary Myelofibrosis
• Other Study ID Numbers: QiluH MF

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No