PERSIST-1: A PHASE III STUDY OF PACRITINIB (PAC) VS
BEST AVAILABLE THERAPY (BAT) IN PRIMARY
MYELOFIBROSIS (PMF), POST-POLYCYTHEMIA VERA MF
(PPV-MF) OR POST-ESSENTIAL THROMBOCYTHEMIA MF
(PET-MF)
Claire Harrison1, Anita Szoke2, Aleksandr Suvorov3, Miklos Egyed4,
Ritam Prasad5, Jiri Mayer6, Janos Jakucs7, Anna Elinder8, Christian
Recher9, Peter A te Boekhorst10, Steven Knapper11, Tim
Somervaille12, Fabio Ciceri13, Fahd Quddus14, Nicole Straetmans15,
Dietger Niederwieser16, James P Dean17, Tanya Granston17, JeanJacques Kiladjian18, Alessandro Vannucchi19, Jyoti Nangalia20,
Adam Mead21, Ruben Mesa22
1Guy’s and St Thomas’ NHS Foundation Trust, London, UK; 2Albert Szent-Györgyi
Clinical Center, University of Szeged, Szeged, Hungary; 3First Republican Clinical
Hospital of Ministry of Health of the Udmurt Republic, Izhevsk, Russia; 4Kaposi
Mor Teaching Hospital, Kaposvar, Hungary; 5Royal Hobart Hospital, Hobart,
Australia; 6University Hospital Brno, Brno, Czech Republic; 7Békés Megyei Pándy
Kálmán Kórház, Gyula, Hungary; 8North Shore Hospital, Takapuna, New Zealand;
9Institut Universitaire du Cancer Toulouse, Toulouse, France; 10Erasmus
University Medical Center, Rotterdam, Netherlands; 11Cardiff University, Cardiff,
UK; 12The Christie NHS Foundation Trust, Manchester, UK; 13San Raffaele
Scientific Institute, Milan, Italy; 14Upstate Oncology Associates, Greenville, SC,
USA; 15Centre Hospitalier Jolimont-Lobbes, Haine-Saint-Paul, Belgium;
16Universitatsklinikum Leipzig, Leipzig, Germany; 17CTI BioPharma Corp, Seattle,
WA, USA; 18Hôpital Saint Louis, Paris, France; 19University of Florence, Florence,
Italy; 20University of Cambridge, Cambridge, UK; 21Oxford University, Oxford, UK;
22Mayo Clinic, Scottsdale, AZ, USA
INTRODUCTION
MF is a rare hematologic malignancy, the symptoms of which have a
substantial negative impact on both patient quality of life and overall
survival (OS)1-3
As early as 1 year from the time of diagnosis, the incidence of
disease-related thrombocytopenia, anemia, and red blood cell (RBC)
transfusion requirements increase dramatically4
Thrombocytopenia is a prognostic factor for shorter OS and risk of
leukemic transformation5
Current treatments have not demonstrated the ability to
simultaneously improve splenomegaly, symptoms, and cytopenias in
myelofibrosis patients
Pacritinib (Figure 1) is a selective JAK2/FLT3 inhibitor (Table 1) not
associated with clinically significant treatment-emergent anemia or
thrombocytopenia in clinical studies6
CONCLUSIONS
Significantly greater proportion of patients with ≥35% reduction of
spleen volume, regardless of baseline platelet count (p=0.0003)
Significant treatment effect (p<0.05) in highest-risk subset (baseline
platelets <50,000/μL)
Significant reduction in TSS (p<0.0001)
Significantly higher proportion of patients became RBC transfusion
independent (p<0.05)
Patients with baseline platelets <50,000/μL had mean increase in
platelet counts of 35% by Week 24
Based on preliminary data, pacritinib may be disease modifying and
warrants combination studies with other potentially disease-modifying
agents in myeloproliferative neoplasms
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