Efficacy Results | Myelofibrosis Clinical Trials

Jakafi is indicated for treatment of intermediate or high-risk myelofibrosis (MF), including primary MF, post–polycythemia vera MF and post–essential thrombocythemia MF in adults.

Image of texts that says COMFORT-I Primary Endpoint

4.4 YEARS MEDIAN DURATION OF SPLEEN RESPONSE AMONG PRIMARY RESPONDERS (n=65)^3†

Primary Endpoint Results

Image that shows COMFORT-I: Percent Change in Spleen Volume in Individual Patients from Baseline to Week 24 or Last Observation

Each bar represents an individual patient's response.
Derived from Verstovsek S et al. N Engl J Med. 2012;366(9):799-807.

of patients experienced some reduction in spleen volume on Jakafi^2,4

Most patients receiving placebo experienced an increase in
spleen volume.^1,2

Image that shows COMFORT-II Primary Endpoint‡: 29% of patients receiving Jakafi achieved a ≥35% reduction in spleen volume at week 48 vs 0% of patients receiving best available therapy§ (P <0.0001)1,5

Image that shows COMFORT-I Secondary Endpoint||: 46% of patients receiving Jakafi achieved a ≥50% improvement in Total Symptom Score (TSS) vs 5% of patients receiving placebo (P <0.0001)1,2

MEDIAN TIME TO SYMPTOM RESPONSE WAS <4 WEEKS FOR PATIENTS RECEIVING JAKAFI¹

Image that shows COMFORT-I

Worsening of TSS is truncated at 150%.¹
Derived from Verstovsek S et al. N Engl J Med. 2012;366(9):799-807.

of patients experienced some improvement in symptoms on Jakafi³

Most patients receiving placebo had worsening of symptoms.²

Debilitating Symptoms of MF^1,2

Abdominal discomfort
Early satiety
Pain under left ribs
Pruritus
Night sweats
Bone/muscle pain

Jakafi overall survival data

COMFORT-I 5-year overall survival probability data⁶: Jakafi and placebo

At 3 years, survival probability was 70% for patients originally randomized to Jakafi and 61% for those originally randomized to placebo¹
Overall survival was a prespecified secondary endpoint in COMFORT-I¹

Image that shows Overall Survival Kaplan-Meier Curves by Treatment Group in COMFORT-I trial

Adapted with permission from the Journal of Hematology & Oncology.⁶

^aThe 5-year overall survival analysis is not included in the Full Prescribing Information for Jakafi. Although the 3-year overall survival analysis is presented in the Full Prescribing Information, P values and hazard ratios are omitted from the overall survival Kaplan-Meier curves.³

^bCOMFORT-I was not designed to compare survival probabilities between Jakafi and placebo at 3 or 5 years.³

^cPatients randomized to placebo were eligible to cross over to receive Jakafi because of progression-driven events or at the physician’s discretion; however, these patients continued to be grouped within their original randomized assignment for analysis purposes.³

COMFORT-II 5-year overall survival probability data⁷: Jakafi and best available therapy

At 3 years, survival probability was 79% for patients originally randomized to Jakafi and 59% for those originally randomized to best available therapy¹
Overall survival was a prespecified secondary endpoint in COMFORT-II¹

Image that shows Overall Survival Kaplan-Meier Curves by Treatment Group in COMFORT-II

Adapted with permission from Leukemia.⁷
BAT, best available therapy.

^bCOMFORT-II was not designed to compare survival probabilities between Jakafi and best available therapy at 3 or 5 years.³

^cPatients randomized to best available therapy were eligible to cross over to receive Jakafi because of progression-driven events or at the physician’s discretion; however, these patients continued to be grouped within their original randomized assignment for analysis purposes.³

Review Jakafi safety data

Video

Video

Jakafi^® (ruxolitinib) in Intermediate-2 or High-Risk Myelofibrosis (MF): Spleen Volume, Symptom, and Overall Survival Data

Hematologist-oncologist Dr Gary Grad reviews data from the COMFORT^¶ trials in MF, which included patients with intermediate-2 or high-risk myelofibrosis. Dr Grad begins with a discussion of risk level and corresponding survival in MF. He then discusses results including reduction in spleen volume, symptom reduction (COMFORT-I only), and overall survival probability (which was a secondary endpoint in both trials).

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^¶COMFORT, COntrolled MyeloFibrosis study with ORal JAK inhibitor Treatment

*COMFORT-I (COntrolled MyeloFibrosis study with ORal JAK inhibitor Treatment-I) was a randomized, double-blind, placebo-controlled phase 3 study with 309 patients with intermediate-2–risk or high-risk MF. The primary endpoint was the proportion of patients achieving a ≥35% reduction in spleen volume from baseline to week 24 as measured by computed tomography (CT) or magnetic resonance imaging (MRI).^1,2

^†Duration of spleen response was defined as the interval between the first spleen response measurement that was a ≥35% reduction from baseline and the date of the first measurement that was no longer a ≥35% reduction from baseline that was also a >25% increase from nadir.³

^‡COMFORT-II (COntrolled MyeloFibrosis study with ORal JAK inhibitor Treatment-II) was a randomized, open-label phase 3 study with 219 patients with intermediate-2–risk or high-risk MF. The primary endpoint was the proportion of patients achieving a ≥35% reduction in spleen volume from baseline at week 48 as measured by CT or MRI.^1,5

^§Best available therapy in COMFORT-II included hydroxyurea (46.6%) and glucocorticoids (16.4%), as well as no medication, anagrelide, epoetin alfa, thalidomide, lenalidomide, mercaptopurine, thioguanine, danazol, peginterferon alfa-2a, interferon-α, melphalan, acetylsalicylic acid, cytarabine, and colchicine.³

^||A secondary endpoint was the proportion of patients with a ≥50% reduction in TSS from baseline to week 24 as measured by the daily patient diary, the modified Myelofibrosis Symptom Assessment Form. TSS encompasses core symptoms of MF: abdominal discomfort, early satiety, pain under left ribs, pruritus, night sweats, and bone/muscle pain. Symptom scores ranged from 0 to 10, with 0 representing symptoms “absent" and 10 representing symptoms “worst imaginable." These scores were added to create the daily total score, which has a maximum of 60. At baseline, mean TSS was 18.0 in the group receiving Jakafi and 16.5 in the group receiving placebo.^1,2