Evidence map›Paper›PMID 24258498›Full record

Trial reportCancer2014

A phase 2 study of ruxolitinib, an oral JAK1 and JAK2 Inhibitor, in patients with advanced polycythemia vera who are refractory or intolerant to hydroxyurea.

Srdan Verstovsek, Francesco Passamonti, Alessandro Rambaldi, Giovanni Barosi, Peter J Rosen, Elisa Rumi, Elisabetta Gattoni, Lisa Pieri, Paola Guglielmelli, Chiara Elena and 8 more

Open access · hybridAbstract readClinical Trial, Phase II
In one paragraph

Trial report in Cancer, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 75 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
75citing papers in PubMed, 2 pooled it
15.4field-weighted citation impact, top 1% of its field
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

75 citing papers in PubMed, 2 syntheses or guidelines pooled it, 174 citations in OpenAlex.

  1. Guideline
  2. Guideline
  3. Trial
  4. Trial
  5. Trial
  6. Trial
  7. Trial
  8. Trial
  9. Review
  10. Article
  11. Observational
  12. Polycythaemia vera.Nature reviews. Disease primers · 2025
    Review
  13. Review
  14. Review
  15. Review
  16. Article
  17. Article
  18. Review
  19. [Systemic pruritus: what is new in diagnosis and treatment?]Dermatologie (Heidelberg, Germany) · 2022
    Review
  20. Review

15 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

18 authors at 10 institutions in 2 countries.

Srdan Verstovsek
Francesco Passamonti
Alessandro Rambaldi
Giovanni Barosi
Peter J Rosen
Elisa Rumi
Elisabetta Gattoni
Lisa Pieri
Paola Guglielmelli
Chiara Elena
Shui He
Nancy Contel
Bijoyesh Mookerjee
Victor Sandor
Mario Cazzola
Hagop M Kantarjian
Tiziano Barbui
Alessandro M Vannucchi
Incyte (United States) · USUniversity of Florence · ITIstituti di Ricovero e Cura a Carattere Scientifico · ITOspedale Papa Giovanni XXIII · ITThe University of Texas MD Anderson Cancer Center · USOspedale di Circolo e Fondazione Macchi · ITOspedale di Santo Spirito · ITPoliclinico San Matteo Fondazione · ITTower Cancer Research Foundation · USUniversity of Pavia · IT

Funding

Tumor Evolution and Metastasis ProgramP30CA016672 · NCI · UNIVERSITY OF TX MD ANDERSON CAN CTR · PI Scott Kopetz · 1985 to 2026
$290.8M
NCI NIH HHS P30 CA016672
6 · The paper itself

Abstract

backgroundPolycythemia vera (PV) is a myeloproliferative neoplasm associated with somatic gain-of-function mutations of Janus kinase-2 (JAK2). Therapeutic options are limited in patients with advanced disease. Ruxolitinib, an oral JAK1/JAK2 inhibitor, is active in preclinical models of PV. The long-term efficacy and safety of ruxolitinib in patients with advanced PV who are refractory or intolerant to hydroxyurea were studied in a phase 2 trial.

methodsResponse was assessed using modified European LeukemiaNet criteria, which included a reduction in hematocrit to <45% without phlebotomy, resolution of palpable splenomegaly, normalization of white blood cell and platelet counts, and reduction in PV-associated symptoms.

resultsThirty-four patients received ruxolitinib for a median of 152 weeks (range, 31 weeks-177 weeks) or 35.0 months (range, 7.1 months-40.7 months). Hematocrit <45% without phlebotomy was achieved in 97% of patients by week 24.Only 1 patient required a phlebotomy after week 4. Among patients with palpable splenomegaly at baseline, 44% and 63%, respectively, achieved nonpalpable spleen measurements at weeks 24 and 144. Clinically meaningful improvements in pruritus, night sweats, and bone pain were observed within 4 weeks of the initiation of therapy and maintained with continued treatment. Ruxolitinib treatment also reduced elevated levels of inflammatory cytokines and granulocyte activation. Thrombocytopenia and anemia were the most common adverse events.Thrombocytopenia of grade 3 or anemia of grade 3 (according to National Cancer Institute Common Terminology Criteria for Adverse Events,version 3.0) occurred in 3 patients each (9%) (1 patient had both) and were managed with dose modification.

conclusionsRuxolitinib was generally well tolerated and provided rapid and durable clinical benefits in patients with advanced PV who were refractory or intolerant to hydroxyurea.

Indexed as

AdultAgedAged, 80 and overAnemiaContraindicationsDrug-Related Side Effects and Adverse ReactionsFemaleGranulocytesHematocritHumansHydroxyureaJanus Kinase 1Janus Kinase 2MaleMiddle AgedNitrilesHydroxyureaJAK1 protein, humanJAK2 protein, humanJanus Kinase 1Janus Kinase 2NitrilesPyrazolesPyrimidinesruxolitinib

Identifiers

PMID24258498
PMCPMC4231215
OpenAlexW2114995805

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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.