Pacritinib versus best available therapy for the treatment of myelofibrosis irrespective of baseline cytopenias (PERSIST-1): an international, randomised, phase 3 trial

Result code in IS VaVaI

<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F65269705%3A_____%2F17%3A00067542" target="_blank" >RIV/65269705:_____/17:00067542 - isvavai.cz</a>

Result on the web

<a href="http://dx.doi.org/10.1016/S2352-3026(17)30027-3" target="_blank" >http://dx.doi.org/10.1016/S2352-3026(17)30027-3</a>

DOI - Digital Object Identifier

<a href="http://dx.doi.org/10.1016/S2352-3026(17)30027-3" target="_blank" >10.1016/S2352-3026(17)30027-3</a>

Result language

angličtina

Original language name

Pacritinib versus best available therapy for the treatment of myelofibrosis irrespective of baseline cytopenias (PERSIST-1): an international, randomised, phase 3 trial

Original language description

Background Available therapies for myelofibrosis can exacerbate cytopenias and are not indicated for patients with severe thrombocytopenia. Pacritinib, which inhibits both JAK2 and FLT3, induced spleen responses with limited myelosuppression in phase 1/2 trials. We aimed to assess the efficacy and safety of pacritinib versus best available therapy in patients with myelofibrosis irrespective of baseline cytopenias. Methods This international, multicentre, randomised, phase 3 trial (PERSIST-1) was done at 67 sites in 12 countries. Patients with higher-risk myelofibrosis (with no exclusions for baseline anaemia or thrombocytopenia) were randomly assigned (2: 1) to receive oral pacritinib 400 mg once daily or best available therapy (BAT) excluding JAK2 inhibitors until disease progression or unacceptable toxicity. Randomisation was stratified by risk category, platelet count, and region. Treatment assignments were known to investigators, site personnel, patients, clinical monitors, and pharmacovigilance personnel. The primary endpoint was spleen volume reduction (SVR) of 35% or more from baseline to week 24 in the intention-to-treat population as assessed by blinded, centrally reviewed MRI or CT. We did safety analyses in all randomised patients who received either treatment. Here we present the final data. This trial is registered with ClinicalTrials. gov, number NCT01773187. Findings Between Jan 8, 2013, and Aug 1, 2014, 327 patients were randomly assigned to pacritinib (n = 220) or BAT (n = 107). Median follow-up was 23.2 months (IQR 14.8-28.7). At week 24, the primary endpoint of SVR of 35% or more was achieved by 42 (19%) patients in the pacritinib group versus five (5%) patients in the BAT group (p = 0.0003). 90 patients in the BAT group crossed over to receive pacritinib at a median of 6.3 months (IQR 5.8-6.7).

Czech name

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Czech description

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Type

J_imp - Article in a specialist periodical, which is included in the Web of Science database

CEP classification

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OECD FORD branch

30205 - Hematology

Project

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Continuities

I - Institucionalni podpora na dlouhodoby koncepcni rozvoj vyzkumne organizace

Publication year

2017

Confidentiality

S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů

Name of the periodical

Lancet Haematology

ISSN

2352-3026

e-ISSN

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Volume of the periodical

4

Issue of the periodical within the volume

5

Country of publishing house

GB - UNITED KINGDOM

Number of pages

12

Pages from-to

"E225"-"E236"

UT code for WoS article

000405451800011

EID of the result in the Scopus database

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