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

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.