Efficacy data in splenomegaly

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 trials2,3

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)

COMFORT‑II3

Phase III, open-label RCT

JAKAVI® (n=146) vs BAT (n=73)

Assessment of the efficacy and safety profile of JAKAVI®
Primary endpoint: proportion of patients achieving SVR35 from baseline at Week 48

Patients with int-2 or high-risk MF, post-polycythaemia vera (PV) MF or post-essential thrombocythaemia (ET) MF

JAKAVI® may help achieve durable spleen volume reduction in your patients with MF3


Patients achieving SVR35 at Week 48 (primary endpoint):*3

 

Circle with the text 28% JAKAVI® (n=144) P<0.001.
Circle with the text 0% BAT (n=72) P<0.001.

Adapted from Harrison C, et al. 2012.3

In the COMFORT-I Phase III RCT, 41.9% and 0.7% of patients treated with JAKAVI® (n=155) vs placebo (n=154) achieved SVR35 at Week 24, respectively (primary endpoint, p<0.001).2

COMFORT-II: mean percentage change in palpable spleen length from baseline at Week 48 (prespecified exploratory endpoint)3

COMFORT-II graphic showing the mean percentage change in palpable spleen length from baseline at week 48 (prespecified exploratory endpoint).3

Adapted from Harrison C, et al. 2012.3

COMFORT-II: 5-year follow-up – duration of spleen response (secondary endpoint)†4

Graph showing COMFORT-II: 5-year follow up - duration of spleen response (secondary endpoint).4

Adapted from Harrison C, et al. 2016.4

JAKAVI® maintained spleen response with continued therapy – the probability of maintaining a spleen response† was 0.51 (95% CI: 0.38–0.62) at 3 years and 0.48 (95% CI: 0.35–0.60) at 5 years for patients initially randomised to JAKAVI®4

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 Summary of Product Characteristics for more information.

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

To learn more about the overall survival data with JAKAVI®, click here

*Patients treated with JAKAVI® achieved reduction in spleen length at Week 4 from baseline, confirmed by reduction in spleen volume at Week 24, which was maintained to Week 48. The median time to 1st CT/MRI observation of an SVR35 was 12.3 weeks.3
†Spleen response was defined as the interval from first spleen volume measurement of ≥35% reduction from baseline at any time on study and the first scan that is no longer a 35% reduction and that is a >25% increase over on-study nadir.4

BAT, best available therapy; CI, confidence interval; ET, essential thrombocythaemia; int-2, intermediate-2; MF, myelofibrosis; PV, polycythaemia vera; RCT, randomised controlled trial; SVR35, ≥35% spleen volume reduction.

References

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

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

  3. Harrison C, et al. N Engl J Med 2012;366:787–798 and supplementary appendix.

  4. Harrison C, et al. Leukemia 2016;30:1701–1707.

UK | September 2025 | FA-11448865-1

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.