Myelofibrosis symptom control

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 trials^2,3

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

Symptom burden and treatment goals

Patients reported that MF symptoms have a substantial impact on their daily life^4–7

Patients with MF (n=174) regularly cancel plans, miss work and feel depressed or anxious^4–7

The International MPN Landmark Survey was developed and funded by Novartis.

Maintenance of QoL is one of the main goals of MF treatment in the non-transplant setting according to the BSH guidelines⁸

Which treatment goals are most important to your patients?

International MPN Landmark Survey: most important treatment goals in MF^‡4

Graph showing which treatment goals are most important to MF patients from the International MPN Landmark survey.

Adapted from Harrison CN, et al. 2017.⁴

Symptom control in the COMFORT-I and COMFORT-II trials

JAKAVI® may help patients reach their goal of improved symptom control from baseline^2,3,9

COMFORT-I: proportion of patients with ≥50% reduction in total symptom score (TSS) over time (secondary endpoint)²

Graph showing the proportion of patients with ≥ 50% reduction in total symptom score (Tss) over time (secondary endpoint) in the COMFORT-I and COMFORT-II trials.2

Adapted from Verstovsek S, et al. 2012.²

Assessed by the modified Myelofibrosis Symptom Assessment Form (MF-SAF) in a double-blind, Phase III RCT (intent-to-treat analysis)*^§2

At Week 24, significantly more patients achieved symptom improvement with JAKAVI® vs placebo from baseline (p<0.001)²

COMFORT-II: mean change from baseline in EORTC QLQ-C30 symptom scores to Week 48 in a double-blind, Phase III RCT (prespecified exploratory endpoint)³

Negative scores indicate a decrease in symptoms; p-values were not reported.

Decreases in MF-related symptoms were observed with JAKAVI® vs an increase of symptoms with BAT³

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 of JAKAVI® in splenomegaly, click here

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

*Data from patients with MF (n=174, including 45 UK patients) in the international MPN Landmark Survey (N=699, including 286 UK patients).⁴
^†MF patients in full- or part-time employment at the time of the survey.⁴
^‡Patients and physicians were asked to assign rankings for their top three treatment goals. Patients were asked: Other than a cure for your condition, what are your three most important treatment goals? Physicians were asked: Other than a cure, what is your most important treatment goal for therapy?⁴
^§Improvements in symptoms were observed in patients treated with JAKAVI® at Week 4, maintained to Week 24; the primary endpoint was the proportion of patients with an SVR35 at Week 24. Each value plotted represents the moving average for the previous 7 days. Shortened 7-item modified MF-SAF form used, covering night sweats, itching, abdominal discomfort, pain under the ribs on the left side, early satiety, muscle/bone pain and inactivity.²

BAT, best available therapy; BSH, British Society for Haemtaology; CI, confidence interval; EORTC QLQ-C30, European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30; int-2, intermediate-2; MF, myelofibrosis; MF-SAF, myelofibrosis symptom assessment form; MPN, myeloproliferative neoplasm; OR, odds ratio; PV, polycythaemia vera; QoL, quality of life; RCT, randomised controlled trial; SVR35, ≥35% spleen volume reduction; TSS, total symptom score.

References

JAKAVI® (ruxolitinib) Summary of Product Characteristics.
Verstovsek S, et al. N Engl J Med 2012;366:799–807 and supplementary appendix.
Harrison C, et al. N Engl J Med 2012;366:787–798.
Harrison CN, et al. Ann Hematol 2017;96:1653–1665.
Petruk C and Mathias J. Adv Ther 2020;37:2050–2070.
Brochmann N, et al. Clin Epidemiol 2019;11:23–33.
Yu J, et al. BMC Cancer 2018;18:420–424.
McLornan DP, et al. Br H Haematol 2024;204:136–150.
Mesa R, et al. J Clin Oncol 2017;35:3844–3850.

UK | September 2025 | FA-11448876

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.

Medicines

Myelofibrosis symptom control

Study designs: Randomised, controlled trials2,3

Symptom burden and treatment goals

Patients reported that MF symptoms have a substantial impact on their daily life4–7

Maintenance of QoL is one of the main goals of MF treatment in the non-transplant setting according to the BSH guidelines8