Data Shows How Ruxolitinib is the Cornerstone of Treatment in Myelofibrosis

Ruben Mesa, MD, director of the UT Health San Antonio MD Anderson Cancer Center, discusses the COMFORT studies that demonstrated how ruxolitinib became a standard of care for patients with myelofibrosis.

In the COMFORT-I trial (NCT00952289), patients experienced improvements in their individual myelofibrosis-related symptoms, and most patients treated with ruxolitinib experienced a total symptom score (TSS) of 50% or greater. Moreover, patient reported outcomes showed that these patients self-reported their condition as either “much improved” or “very much improved” based on the Patient Global Impression of Change scale. Spleen volume reduction was also associated with these responses.

In the COMFORT-II study (NCT00934544), long-term findings showed that 53.4% of patients in the ruxolitinib arm of the study achieved a 35% or greater reduction in spleen volume at any time with a 0.48 (95% CI, 0.35-0.60) probability of maintaining their response at 5 years. In comparison, no patients on the best available therapy arm had a decrease in spleen volume at week 48 in the study compared with 28% of patients on ruxolitinib (P < .001).

Mesa also discusses the results of a pooled analysis of the COMFORT trials that showed patients on ruxolitinib had a longer-term overall survival (OS) compared with other therapies at a median of 3 years of follow-up (HR, 0.65; 95% CI, 0.46-0.90; P = .01). An estimated OS at week 144 of treatment also showed stronger results for patients of ruxolitinib at 78%.

TRANSCRIPTION:

0:08 | The main studies that led to the use of ruxolitinib [for patients with] myelofibrosis goes back to the COMFORT studies, the COMFORT-I and the COMFORT-II studies. In the COMFORT-I study, ruxolitinib was compared against placebo, with primary end points for improvement of splenomegaly and symptoms. In COMFORT-II, a parallel study done in Europe, the same end points with a comparator arm of best alternative therapy [were assessed]. With each of these studies, we saw the ruxolitinib was vastly superior to both control groups for improvements in splenomegaly and symptoms.

0:40 | Over time, we've also seen that there's a strong signal that use of ruxolitinib leads to an improvement in survival, both with long-term analysis of a pooled analysis of both the COMFORT studies as well as real world evidence that has come out from the use of ruxolitinib. So, it really has become the cornerstone of therapy for myelofibrosis. [It has] now been approved a little over 10 years, whether alone or, as the field continues to evolve, with potential combinations.