Critical Reviews in Oncology / Hematology
Volume 81, Issue 2 , Pages 123-135, February 2012

Time for a new era in the evaluation of targeted therapies for patients with chronic myeloid leukemia: Inclusion of quality of life and other patient-reported outcomes☆

  • F. Efficace

      Affiliations

    • Italian Group for Adult Hematologic Diseases (GIMEMA), GIMEMA Data Center and Health Outcomes Research Unit, Via Benevento 6, 00161 Rome, Italy
    • Corresponding Author InformationCorresponding author. Tel.: +39 06 441 639831; fax: +39 06 4402516.
    • ,
  • K. Cocks

      Affiliations

    • University of Leeds, Clinical Trials Research Unit, Leeds, UK
    • ,
  • M. Breccia

      Affiliations

    • University of Rome “Sapienza”, Department of Hematology, Rome, Italy
    • ,
  • M. Sprangers

      Affiliations

    • University of Amsterdam, Academic Medical Center, Department of Medical Psychology, Amsterdam, The Netherlands
    • ,
  • C.A. Meyers

      Affiliations

    • The University of Texas, M.D. Anderson Cancer Center, Department of Neuro-Oncology, Houston, TX, USA
    • ,
  • M. Vignetti

      Affiliations

    • University of Rome “Sapienza”, Department of Hematology, Rome, Italy
    • ,
  • M. Baccarani

      Affiliations

    • University of Bologna, S. Orsola-Malpighi Hospital, Department of Hematology and Oncology, Bologna, Italy
    • ,
  • F. Mandelli

      Affiliations

    • Italian Group for Adult Hematologic Diseases (GIMEMA), GIMEMA Data Center and Health Outcomes Research Unit, Via Benevento 6, 00161 Rome, Italy
    • ,
  • GIMEMA and EORTC Quality of Life Group

Accepted 22 February 2011. published online 25 March 2011.

Abstract 

Health-related quality of life (HRQOL) and other patient-reported outcomes (PROs) might be crucial in comparing effectiveness of treatments as they could provide invaluable information to better inform clinical decision-making. This is particularly true in the era of targeted therapies (TT). A systematic review was undertaken on all studies with CML patients published from 1980 to 2010 and including a PRO evaluation. Out of 619 articles scrutinized, 15 met eligibility criteria and no study was published before 1995. Six dealt mainly with interferon-based therapies, 7 with bone marrow transplantation and only 2 evaluated PROs in the context of TT. No disease-specific, validated PRO instrument for these patients was found. The main evidence being that Imatinib provides clear advantage in terms of HRQOL over interferon-based treatments. There is lack of data concerning PROs in patients treated with current TT. Documenting HRQOL and side effects of CML treatments, from the patients’ perspective is needed to evaluate overall treatment effectiveness and net clinical benefit of newer therapeutic strategies.

Keywords: Chronic myeloid leukemia, Quality of life, Systematic review, Patient-reported outcomes

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☆ Presented at the 52nd Annual Meeting of the American Society of Hematology (ASH), December 4–7, 2010, Orlando, FL, USA.

PII: S1040-8428(11)00053-9

doi:10.1016/j.critrevonc.2011.02.007

Critical Reviews in Oncology / Hematology
Volume 81, Issue 2 , Pages 123-135, February 2012

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