Evidence map›Paper›PMID 34551507›Full record

ArticleHaematologica2022

Retrospective analysis of pacritinib in patients with myelofibrosis and severe thrombocytopenia.

Srdan Verstovsek, Ruben Mesa, Moshe Talpaz, Jean-Jacques Kiladjian, Claire N Harrison, Stephen T Oh, Alessandro M Vannucchi, Raajit Rampal, Bart L Scott, Sarah A Buckley and 3 more

Erratum issuedAbstract read
In one paragraph

Article in Haematologica, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 24 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
24citing papers in PubMed, 1 pooled it
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

24 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Review
  3. Review
  4. Article
  5. Review
  6. Article
  7. JAK Inhibitors for Myelofibrosis: Strengths and Limitations.Current hematologic malignancy reports · 2024
    Review
  8. Novel selective inhibitors of macropinocytosis-dependent growth in pancreatic ductal carcinoma.Biomedicine & pharmacotherapy = Biomedecine & pharmacotherapie · 2024
    Article
  9. Article
  10. Haematologica · 2024
    Article
  11. Review
  12. Article
  13. Review
  14. Review
  15. Article
  16. Article
  17. Review
  18. Myelofibrosis.Blood · 2023
    Review
  19. Review
  20. Emerging Treatment Options for Myelofibrosis: Focus on Anemia.Therapeutics and clinical risk management · 2023
    Review
4 · The record

Corrections and comments

  • Erratum issued
    2024
5 · Who and what money

Authors and funding

13 authors.

Srdan VerstovsekMD Anderson Cancer Center, Houston. sverstov@mdanderson.org.
Ruben MesaMayo Clinic, Scottsdale.
Moshe TalpazUniversity of Michigan, Comprehensive Cancer Center, Ann Arbor.
Jean-Jacques KiladjianHopital Saint-Louis, Universite Paris Diderot, Paris.
Claire N HarrisonGuy's and St Thomas' NHS Foundation Trust, London.
Stephen T OhWashington University School of Medicine, St. Louis.
Alessandro M VannucchiUniversity of Florence, Azienda Ospedaliera-Universitaria Careggi, Florence.
Raajit RampalMemorial Sloan Kettering Cancer Center, New York.
Bart L ScottFred Hutchinson Cancer Research Center, Seattle.
Sarah A BuckleyCTI BioPharma Inc, Seattle.
Adam R CraigCTI BioPharma Inc, Seattle.
Karisse Roman-TorresCTI BioPharma Inc, Seattle.
John O MascarenhasIcahn School of Medicine at Mount Sinai, New York.

Funding

X-RAY CRYSTALLOGRAPHYP30CA008748 · NCI · SLOAN-KETTERING INSTITUTE FOR CANCER RES · PI Nicholas D Socci · 1985 to 2026
$347.4M
NCI NIH HHS P30 CA008748
6 · The paper itself

Abstract

Thrombocytopenia is common in patients with myelofibrosis (MF) and is a well-established adverse prognostic factor. Both of the approved Janus kinase (JAK) inhibitors, ruxolitinib and fedratinib, can worsen thrombocytopenia and have not been evaluated in patients with severe thrombocytopenia (platelet counts <50×109/L). Pacritinib, a novel JAK2/interleukin-1 receptor-associated kinase 1 inhibitor, has been studied in two phase III trials (PERSIST-1 and PERSIST- 2), both of which enrolled patients with MF and severe thrombocytopenia. In order to better characterize treatment outcomes for this population with advanced disease, we present a retrospective analysis of efficacy and safety data in the 189 patients with severe thrombocytopenia treated in the PERSIST studies. The proportion of patients in the pacritinib group meeting efficacy endpoints was greater than in the BAT group for ≥35% spleen volume reduction (23% vs. 2%, P=0.0007), ≥50% modified Total Symptom Score reduction (25% vs. 8%, P=0.044), and self-reported symptom benefit ("much" or "very much" improved; 25% vs. 8%, P=0.016) at the primary analysis time point (week 24). The adverse event profile of pacritinib was manageable, and dose modification was rarely required. There was no excess in bleeding or death in pacritinib-treated patients. These results indicate that pacritinib is a promising treatment for patients with MF who lack safe and effective therapeutic options due to severe thrombocytopenia.

Indexed as

AnemiaPrimary MyelofibrosisThrombocytopeniaBridged-Ring CompoundsHumansJanus Kinase 2NitrilesProtein Kinase InhibitorsPyrimidinesRetrospective Studies11-(2-pyrrolidin-1-ylethoxy)-14,19-dioxa-5,7,26-triazatetracyclo(19.3.1.1(2,6).1(8,12))heptacosa-1(25),2(26),3,5,8,10,12(27),16,21,23-decaeneBridged-Ring CompoundsJanus Kinase 2NitrilesProtein Kinase InhibitorsPyrimidines

Identifiers

PMID34551507
PMCPMC9244834

What OpenQuestion holds

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Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.