Overall survival (OS) data

JAKAVI® (ruxolitinib) is indicated for the treatment of disease-related splenomegaly or symptoms in adult patients with primary myelofibrosis (also known as chronic idiopathic myelofibrosis), post polycythaemia vera myelofibrosis or post essential thrombocythaemia myelofibrosis. JAKAVI® is also indicated for adult patients with polycythaemia vera who are resistant to or intolerant of hydroxyurea.1

For full safety information, please refer to the Summary of Product Characteristics.

Study designs: Randomised, controlled trial2

Study

Type of study

Regimen

Study purpose and description

Study population

COMFORT‑I2

Phase III, double-blind, randomised controlled trial (RCT)

JAKAVI® (n=155) vs placebo (n=154)

Assessment of the efficacy and safety profile of JAKAVI®
Primary endpoint: proportion of patients achieving ≥35% spleen volume reduction (SVR35) from baseline at Week 24

Patients with intermediate-2 (int-2) or high-risk myelofibrosis (MF)

Real-world study3

Study

Type of study

Regimen

Study purpose and description

Study population

ERNEST3

Registry of patients with MF from 13 centres in 5 European countries

JAKAVI® (n=50) 
vs hydroxyurea (HU) (n=50)

To analyse the impact of JAKAVI® on overall survival (OS) by using prospectively collected real-world data

Patients with MF treated at international centres with expertise in the management 
 of MF

In a pooled analysis of a randomised controlled trial, the 5-year OS data (exploratory analysis of the ITT population) found:*4

Chart showing the 5-year OS data from a pooled analysis of a randomised controlled trial.

Adapted from Verstovsek et al., 2017.4

JAKAVI® was observed to extend OS – a primary goal of treatment in patients with primary 
MF ineligible for stem cell transplantation (HR=0.70; 95% CI: 0.54–0.91)4–6

JAKAVI® was observed to extend OS by 3 years vs those who crossed over from control 
(5.3 vs 2.3 years; HR=0.35; 95% CI: 0.23–0.59)‡4

In a 10-year follow-up prospective real-world registry with propensity score-matched groups, the OS with HU and JAKAVI® showed:3 

chart showing the OS with HU and JAKAVI® from a 10-year follow-up prospective real-world registry with propensity score-matched groups.3

Adapted from Guglielmelli P, et al. 2022.3

Propensity score (PS) matching analysis balanced patients who had been treated or not with JAKAVI®, by forming matched sets of 1 treated and 1 randomly sampled, non-treated patients (1:1 matching) who shared a similar PS. The PS was estimated by logistic regression of exposure to JAKAVI® on baseline covariates at the beginning of treatment.3

In the real world, longer mOS was observed with JAKAVI® vs HU3

mOS with JAKAVI® was not statistically significant regardless of first-line or post-HU use (6.4 years vs 7.8 years, respectively; p=0.99)3

The most frequently reported haematological adverse drug reactions included anaemia (83.8%), thrombocytopenia (80.5%) and neutropenia (20.8%). The three most frequent non-haematological adverse drug reactions were bruising (33.3%), other bleeding (including epistaxis, post-procedural haemorrhage and haematuria) (24.3%) and dizziness (21.9%).1 Please refer to the SmPC for more information.

To learn more about the efficacy data of JAKAVI® in splenomegaly, click here

To learn more about how JAKAVI® may help control MF symptoms, click here

*Exploratory analysis of OS in the Phase III COMFORT-I and COMFORT-II studies (a secondary endpoint in both studies) using pooled intent-to-treat data from patients randomised to JAKAVI® and the control groups.4
†The control groups in COMFORT-I and COMFORT-II received placebo or BAT, respectively.4
‡The crossover-corrected treatment effect was estimated using an RPSFT method and through censorship of survival time at the time of crossover.4

BAT, best available therapy; CI, confidence interval; DIPSS, dynamic international prognostic scoring system; ERNEST, European Registry for Myeloproliferative Neoplasms: Toward a Better Understanding of Epidemiology, Survival, and Treatment; HR, hazard ratio; HU, hydroxyurea; int-1, intermediate-1; int-2, intermediate-2; MF, myelofibrosis; mOS, median overall survival; OS, overall survival; PS, propensity score; RPSFT, rank-preserving structural failure time.

References

  1. JAKAVI® (ruxolitinib) Summary of Product Characteristics.

  2. Verstovsek S, et al. N Engl J Med 2012;366:799–807. 

  3. Guglielmelli P, et al. Blood Adv 2022;6:373–375.

  4. Verstovsek S, et al. J Hematol Oncol 2017;10:156.

  5. Barbui T, et al. J Clin Oncol 2011;29:761–770.

  6. Barbui T, et al. Leukemia 2018;32:1057–1069.

UK | September 2025 | FA-11448878-1

Adverse events should be reported. Reporting forms and information can be found at www.mhra.gov.uk/yellowcard. Adverse events should also be reported to Novartis online through the pharmacovigilance intake (PVI) tool at www.novartis.com/report, or alternatively email [email protected] or call 01276 698370.