Pacritinib is an oral next-generation multikinase inhibitor with specificity for JAK2 and FLT3 being evaluated to treat myelofibrosis in two Phase III trials.

At EHA 2015, new data from the Phase 3 PERSIST-1 study show that pacritinib is more effective than the best available therapy (BAT) at improving disease symptoms and quality of life (QoL) for patients with myelofibrosis was presented.

Myelofibrosis is a rare blood cancer associated with significantly reduced QoL and shortened survival.

Most patients with myelofibrosis present with enlarged spleens as well as many other potentially devastating symptoms such as: abdominal discomfort, bone pain, feeling full after eating little, itching, night sweats, and tiredness.

PERSIST-1 was designed to compare the efficacy and safety of pacritinib to the best available therapy (BAT), which included a range of off-label treatments, in 327 patients with myelofibrosis, regardless of their platelet counts.

The study also measured patient-reported outcomes (PROs), which have become important for approval of new therapies, to determine whether pacritinib reduced patients’ symptom burden and helped them feel better.

Results at EHA showed that patients who received pacritinib experienced a greater degree of relief (median) from symptoms compared to BAT, specifically: abdominal discomfort (46% improvement with pacritinib vs no improvement with BAT); bone pain (32% improvement with pacritinib vs 8% improvement with BAT); feeling of early fullness (45% improvement with pacritinib vs 1% worsening with BAT); itching (48.5% improvement with pacritinib vs 10% improvement with BAT); night sweats (69.5% of improvement with pacritinib vs no improvement with BAT); and fatigue (27.5% improvement with pacritinib vs 4% worsening with BAT).

Results were determined using the Myeloproliferative Neoplasm Symptom Assessment (MPN-SAF TSS) Forms 1.0 and 2.0, which are specific sets of questions patients answer daily (using an electronic diary) and which are based on a questionnaire originally developed by Ruben A. Mesa, MD, Deputy Director of the Mayo Clinic Cancer Center in Scottsdale, Arizona, USA.

Watch the press conference for more information.

Source: EHA