Evidence map›Paper›PMID 38060001›Full record

Trial reportAnnals of hematology2024

Ropeginterferon phase 2 randomized study in low-risk polycythemia vera: 5-year drug survival and efficacy outcomes.

Tiziano Barbui, Alessandra Carobbio, Valerio De Stefano, Alberto Alvarez-Larran, Arianna Ghirardi, Greta Carioli, Francesca Fenili, Elena Rossi, Fabio Ciceri, Massimiliano Bonifacio and 31 more

Open access · greenAbstract readClinical Trial, Phase IIRandomized Controlled Trial
PubMed Publisher
In one paragraph

Trial report in Annals of hematology, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
15citing papers in PubMed, 1 pooled it
4.9field-weighted citation impact, top 5% 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

15 citing papers in PubMed, 1 synthesis or guideline pooled it, 17 citations in OpenAlex.

  1. Pooled it
  2. Trial
  3. Trial
  4. Article
  5. Article
  6. Review
  7. Review
  8. Ropeginterferon alfa-2bWorld journal of clinical oncology · 2025
    Article
  9. Review
  10. Review
  11. Article
  12. Polycythaemia vera.Nature reviews. Disease primers · 2025
    Review
  13. Review
  14. Article
  15. Article
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

41 authors at 20 institutions in 2 countries.

Tiziano BarbuiFROM, Fondazione Per La Ricerca Ospedale Di Bergamo ETS, Papa Giovanni XXIII Hospital, 24127, Bergamo, Italy. tbarbui@fondazionefrom.it.ORCID http://orcid.org/0000-0003-2747-6327
Alessandra CarobbioFROM, Fondazione Per La Ricerca Ospedale Di Bergamo ETS, Papa Giovanni XXIII Hospital, 24127, Bergamo, Italy.
Valerio De StefanoDipartimento Di Scienze Radiologiche Ed Ematologiche, Sezione Di Ematologia, Università Cattolica del Sacro Cuore-Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.
Alberto Alvarez-LarranHematology Department, Hospital Clínic, Barcelona, Spain.
Arianna GhirardiFROM, Fondazione Per La Ricerca Ospedale Di Bergamo ETS, Papa Giovanni XXIII Hospital, 24127, Bergamo, Italy.
Greta CarioliFROM, Fondazione Per La Ricerca Ospedale Di Bergamo ETS, Papa Giovanni XXIII Hospital, 24127, Bergamo, Italy.
Francesca FeniliFROM, Fondazione Per La Ricerca Ospedale Di Bergamo ETS, Papa Giovanni XXIII Hospital, 24127, Bergamo, Italy.
Elena RossiDipartimento Di Scienze Radiologiche Ed Ematologiche, Sezione Di Ematologia, Università Cattolica del Sacro Cuore-Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.
Fabio CiceriUnità Operativa Di Ematologia E Trapianto Midollo Osseo, IRCCS Ospedale San Raffaele, Milan, Italy.
Massimiliano BonifacioDipartimento Di Ingegneria Per La Medicina Di Innovazione, Sezione Di Biomedicina Di Innovazione, Università Di Verona, Verona, Italy.
Alessandra IurloUOC Ematologia, Fondazione IRCCS Cá Granda-Ospedale Maggiore Policlinico, Milan, Italy.
Francesca PalandriIRCCS Azienda Ospedaliero-Universitaria Di Bologna, Istituto Di Ematologia "Seragnoli", Bologna, Italy.
Giulia BenevoloSC Ematologia U, A.O.U. Città Della Salute E Della Scienza, Turin, Italy.
Fabrizio PaneUOC Di Ematologia E Trapianti Di Midollo, Azienda Ospedaliera Universitaria Federico II Di Napoli, Naples, Italy.
Alessandra RiccoU.O. Ematologia Con Trapianto Azienda Ospedaliera Universitaria "Consorziale Policlinico" Di Bari, Bari, Italy.
Giuseppe CarliUOC Ematologia, Azienda ULSS 8 Berica Ospedale San Bortolo Di Vicenza, Vicenza, Italy.
Marianna CaramellaDivisione Ematologia, ASST Grande Ospedale Metropolitano Niguarda, Milan, Italy.
Davide RapezziDivisione Di Ematologia, Azienda Ospedaliera S. Croce E Carle Di Cuneo, Cuneo, Italy.
Caterina MusolinoUOC Ematologia, Azienda Ospedaliera Universitaria Policlinico "G Martino", Messina, Italy.
Sergio SiragusaDivisione Ematologia, Azienda Ospedaliera Universitaria Policlinico "P. Giaccone", Palermo, Italy.
Elisa RumiDepartment of Molecular Medicine, University of Pavia, Pavia, Italy.
Andrea PatriarcaSCDU Ematologia, Azienda Ospedaliero Universitaria Maggiore Della Carità, Novara, Italy.
Nicola CascavillaUO Ematologia IRCCS "Casa Sollievo Della Sofferenza", San Giovanni Rotondo, Italy.
Barbara MoraHematology, Ospedale Di Circolo, ASST Sette Laghi, Varese, Italy.
Emma CacciolaUO Di Emostasi/Ematologia Centro Federato FCSA, Dipartimento Di Scienze Mediche, Chirurgiche E Tecnologie Avanzate "G. F. Ingrassia, Azienda Ospedaliera Universitaria Policlinico "G. Rodolico-San Marco", Catania, Italy.
Laura CalabresiCRIMM, Azienda Ospedaliera Universitaria Careggi, Dipartimento Di Medicina Sperimentale E Clinica, Università Di Firenze, Florence, Italy.
Giuseppe Gaetano LoscoccoCRIMM, Azienda Ospedaliera Universitaria Careggi, Dipartimento Di Medicina Sperimentale E Clinica, Università Di Firenze, Florence, Italy.
Paola GuglielmelliCRIMM, Azienda Ospedaliera Universitaria Careggi, Dipartimento Di Medicina Sperimentale E Clinica, Università Di Firenze, Florence, Italy.
Francesca GesulloCRIMM, Azienda Ospedaliera Universitaria Careggi, Dipartimento Di Medicina Sperimentale E Clinica, Università Di Firenze, Florence, Italy.
Silvia BettiDipartimento Di Scienze Radiologiche Ed Ematologiche, Sezione Di Ematologia, Università Cattolica del Sacro Cuore-Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.
Francesco RamundoDipartimento Di Scienze Radiologiche Ed Ematologiche, Sezione Di Ematologia, Università Cattolica del Sacro Cuore-Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.
Francesca LunghiUnità Operativa Di Ematologia E Trapianto Midollo Osseo, IRCCS Ospedale San Raffaele, Milan, Italy.
Luigi ScaffidiDipartimento Di Medicina, Azienda Ospedaliera Universitaria Integrata Di Verona, Verona, Italy.
Cristina BucelliUOC Ematologia, Fondazione IRCCS Cá Granda-Ospedale Maggiore Policlinico, Milan, Italy.
Daniele CattaneoUOC Ematologia, Fondazione IRCCS Cá Granda-Ospedale Maggiore Policlinico, Milan, Italy.
Nicola VianelliIRCCS Azienda Ospedaliero-Universitaria Di Bologna, Istituto Di Ematologia "Seragnoli", Bologna, Italy.
Marta BelliniUOC Ematologia, ASST Papa Giovanni XXIII, Bergamo, Italy.
Maria Chiara FinazziUOC Ematologia, ASST Papa Giovanni XXIII, Bergamo, Italy.
Gianni TognoniCentro Di Coordinamento Nazionale Dei Comitati Etici, Agenzia Italiana del Farmaco, Rome, Italy.
Alessandro RambaldiUOC Ematologia, ASST Papa Giovanni XXIII, Bergamo, Italy.
Alessandro Maria VannucchiCRIMM, Azienda Ospedaliera Universitaria Careggi, Dipartimento Di Medicina Sperimentale E Clinica, Università Di Firenze, Florence, Italy.
Ospedale Papa Giovanni XXIII · ITAzienda Ospedaliero-Universitaria Careggi · ITUniversità Cattolica del Sacro Cuore · ITFondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico · ITIRCCS Ospedale San Raffaele · ITIstituto di Ematologia di Bologna · ITUniversity of Milan · ITAzienda Ospedaliera Citta' della Salute e della Scienza di Torino · ITAzienda Ospedaliera Universitaria Integrata Verona · ITAzienda Ospedaliera Universitaria Policlinico "G. Martino" · ITAzienda Ospedaliera Universitaria Policlinico "Paolo Giaccone" di Palermo · ITAzienda Sanitaria Ospedaliera S.Croce e Carle Cuneo · ITAzienda Socio Sanitaria Territoriale Grande Ospedale Metropolitano Niguarda · ITAzienda Universitaria Ospedaliera Consorziale - Policlinico Bari · ITCasa Sollievo della Sofferenza · ITFederico II University Hospital · ITHospital Clínic de Barcelona · ESItalian Medicines Agency · ITOspedale di Circolo e Fondazione Macchi · ITOspedale San Bortolo · IT

Funding

Fondazione AIRC per la ricerca sul cancro ETS 1005Fondazione AIRC per la ricerca sul cancro ETS 21267
6 · The paper itself

Abstract

In patients with low-risk polycythemia vera, exposure to low-dose Ropeginterferon alfa-2b (Ropeg) 100 µg every 2 weeks for 2 years was more effective than the standard treatment of therapeutic phlebotomy in maintaining target hematocrit (HCT) (< 45%) with a reduction in the need for phlebotomy without disease progression. In the present paper, we analyzed drug survival, defined as a surrogate measure of the efficacy, safety, adherence, and tolerability of Ropeg in patients followed up to 5 years. During the first 2 years, Ropeg and phlebotomy-only (Phl-O) were discontinued in 33% and 70% of patients, respectively, for lack of response (12 in the Ropeg arm vs. 34 in the Phl-O arm) or adverse events (6 vs. 0) and withdrawal of consent in (3 vs. 10). Thirty-six Ropeg responders continued the drug for up to 3 years, and the probability of drug survival after a median of 3.15 years was 59%. Notably, the primary composite endpoint was maintained in 97%, 94%, and 94% of patients still on drug at 3, 4, and 5 years, respectively, and 60% of cases were phlebotomy-free. Twenty-three of 63 Phl-O patients (37%) failed the primary endpoint and were crossed over to Ropeg; among the risk factors for this failure, the need for more than three bloodletting procedures in the first 6 months emerged as the most important determinant. In conclusion, to improve the effectiveness of Ropeg, we suggest increasing the dose and using it earlier driven by high phlebotomy need in the first 6 months post-diagnosis.

Indexed as

Polycythemia VeraBloodlettingHematocritHumansPhlebotomyRisk FactorsLow-riskPhlebotomyPolycythemia veraRopeginterferon alfa-2b

Identifiers

PMID38060001
OpenAlexW4389449724

What OpenQuestion holds

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Registered trials

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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.