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  • Letter to the Editor
  • Published:

Do current response criteria in classical Ph-negative myeloproliferative neoplasms capture benefit for patients?

  • G Barosi1,
  • A Tefferi2 &
  • T Barbui3
  • on behalf of the ad hoc committee ‘Definition of clinically relevant outcomes for contemporarily clinical trials in Ph-neg MPNs’

Leukemia volume 26, pages 1148–1149 (2012)Cite this article

A major goal of the clinical research on the efficacy of drugs in cancer is to produce well-structured instruments for the evaluation of response to therapy. Response criteria should prove reliable in the assessment of drug effect on the tumor and represent a measure of benefit for the patient. Guidelines to produce reliable measurements of response in solid tumors have been introduced in 2000(ref.1) and recently revised by the RECIST working group.2

In classical Philadelphia-negative (Ph-neg) myeloproliferative neoplasms (MPNs), that is, polycythemia vera (PV), essential thrombocythemia (ET) and primary myelofibrosis (PMF), the discovery of JAK2 V617F mutation and other molecular abnormalities has unveiled new targets for therapies and brought to the development of an array of new experimental drugs aimed at modifying the natural history of the diseases. As a consequence, the doctors engaged in clinical research and the industry claiming for drug approval have to face the issue to test new drugs against end points that measure clinically relevant benefits for the patients.

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Acknowledgements

This work was supported by European LeukemiaNet (ELN), an EU-funded consortium (LSHC-CT-2004-503216). The experts participating in the Delphi consensus process were: T Barbui (Bergamo, Italy), G Barosi (Pavia, Italy), F Cervantes (Barcelona, Spain), G Finazzi (Bergamo, Italy), C Harrison (London, UK), JJ Kiladjianian (Paris, France), E Lengfelder (Heidelberg, Germany), R Mesa (Scottsdale, AZ, USA), A Pardanani (Rochester, MN, USA), F Passamonti (Varese, Italy), J Samuelsson (Stockholm, Sweden), A Tefferi (Rochester, MN, USA), AM Vannucchi (Florence, Italy), S Verstovsek (Huston, TX, USA).

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Authors and Affiliations

  1. IRCCS Policlinico S. Matteo Foundation, Pavia, Italy

    G Barosi

  2. Mayo Clinic, Rochester, MN, USA

    A Tefferi

  3. Ospedali Riuniti di Bergamo, Bergamo, Italy

    T Barbui

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  1. G Barosi
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  2. A Tefferi
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on behalf of the ad hoc committee ‘Definition of clinically relevant outcomes for contemporarily clinical trials in Ph-neg MPNs’

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Correspondence to G Barosi.

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Barosi, G., Tefferi, A., Barbui, T. et al. Do current response criteria in classical Ph-negative myeloproliferative neoplasms capture benefit for patients?. Leukemia 26, 1148–1149 (2012). https://doi.org/10.1038/leu.2011.337

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