Validation and reference values of the EORTC QLQ-CML24 questionnaire to assess health-related quality of life in patients with chronic myeloid leukemia

References

• Bjorkholm M, Ohm L, Eloranta S, et al. Success story of targeted therapy in chronic myeloid leukemia: a population-based study of patients diagnosed in Sweden from 1973 to 2008. J Clin Oncol. 2011;29(18):2514–2520.
   PubMed Web of Science ®Google Scholar
• Hochhaus A, Breccia M, Saglio G, et al. Expert opinion—management of chronic myeloid leukemia after resistance to second-generation tyrosine kinase inhibitors. Leukemia. 2020;34(6):1495–1502.
   PubMed Web of Science ®Google Scholar
• Bower H, Bjorkholm M, Dickman PW, et al. Life expectancy of patients with chronic myeloid leukemia approaches the life expectancy of the general population. J Clin Oncol. 2016;34(24):2851–2857.
   PubMed Web of Science ®Google Scholar
• Hochhaus A, Larson RA, Guilhot F, et al. Long-term outcomes of imatinib treatment for chronic myeloid leukemia. N Engl J Med. 2017;376(10):917–927.
   PubMed Web of Science ®Google Scholar
• Saglio G, Kim DW, Issaragrisil S, et al. Nilotinib versus imatinib for newly diagnosed chronic myeloid leukemia. N Engl J Med. 2010;362(24):2251–2259.
   PubMed Web of Science ®Google Scholar
• Kantarjian H, Shah NP, Hochhaus A, et al. Dasatinib versus imatinib in newly diagnosed chronic-phase chronic myeloid leukemia. N Engl J Med. 2010;362(24):2260–2270.
   PubMed Web of Science ®Google Scholar
• Cortes JE, Kim DW, Kantarjian HM, et al. Bosutinib versus imatinib in newly diagnosed chronic-phase chronic myeloid leukemia: results from the BELA trial. J Clin Oncol. 2012;30(28):3486–3492.
   PubMed Web of Science ®Google Scholar
• Rosti G, Castagnetti F, Gugliotta G, et al. Tyrosine kinase inhibitors in chronic myeloid leukaemia: which, when, for whom? Nat Rev Clin Oncol. 2017;14(3):141–154.
   PubMed Web of Science ®Google Scholar
• Radich JP, Deininger M, Abboud CN, et al. Chronic myeloid leukemia, version 1.2019, NCCN Clinical Practice Guidelines in Oncology. J Natl Compr Canc Netw. 2018;16(9):1108–1135.
   PubMed Web of Science ®Google Scholar
• Efficace F, Baccarani M, Breccia M, et al. Chronic fatigue is the most important factor limiting health-related quality of life of chronic myeloid leukemia patients treated with imatinib. Leukemia. 2013;27(7):1511–1519.
   PubMed Web of Science ®Google Scholar
• Pinilla-Ibarz J, Cortes J, Mauro MJ. Intolerance to tyrosine kinase inhibitors in chronic myeloid leukemia: definitions and clinical implications. Cancer. 2011;117(4):688–697.
   PubMed Web of Science ®Google Scholar
• Guerin A, Chen L, Ionescu-Ittu R, et al. Impact of low-grade adverse events on health-related quality of life in adult patients receiving imatinib or nilotinib for newly diagnosed Philadelphia chromosome positive chronic myelogenous leukemia in chronic phase. Curr Med Res Opin. 2014;30(11):2317–2328.
   PubMed Web of Science ®Google Scholar
• Efficace F, Cannella L. The value of quality of life assessment in chronic myeloid leukemia patients receiving tyrosine kinase inhibitors. Hematology Am Soc Hematol Educ Program. 2016;2016(1):170–179.
   PubMedGoogle Scholar
• Noens L, Hensen M, Kucmin-Bemelmans I, et al. Measurement of adherence to BCR-ABL inhibitor therapy in chronic myeloid leukemia: current situation and future challenges. Haematologica. 2014;99(3):437–447.
   PubMed Web of Science ®Google Scholar
• Unnikrishnan R, Veeraiah S, Mani S, et al. Comprehensive evaluation of adherence to therapy, its associations, and its implications in patients with chronic myeloid leukemia receiving imatinib. Clin Lymphoma Myeloma Leuk. 2016;16(6):366–371e3.
   PubMed Web of Science ®Google Scholar
• Noens L, van Lierde MA, De Bock R, et al. Prevalence, determinants, and outcomes of nonadherence to imatinib therapy in patients with chronic myeloid leukemia: the ADAGIO study. Blood. 2009;113(22):5401–5411.
   PubMed Web of Science ®Google Scholar
• Marin D, Bazeos A, Mahon FX, et al. Adherence is the critical factor for achieving molecular responses in patients with chronic myeloid leukemia who achieve complete cytogenetic responses on imatinib. J Clin Oncol. 2010;28(14):2381–2388.
   PubMed Web of Science ®Google Scholar
• Efficace F, Baccarani M, Breccia M, et al. International development of an EORTC questionnaire for assessing health-related quality of life in chronic myeloid leukemia patients: the EORTC QLQ-CML24. Qual Life Res. 2014;23(3):825–836.
   PubMed Web of Science ®Google Scholar
• Aaronson NK, Ahmedzai S, Bergman B, et al. The European Organization for Research and Treatment of Cancer QLQ-C30: a quality-of-life instrument for use in international clinical trials in oncology. J Natl Cancer Inst. 1993;85(5):365–376.
   PubMed Web of Science ®Google Scholar
• Bacik J, Mazumdar M, Murphy BA, et al. The Functional Assessment of Cancer Therapy-BRM (FACT-BRM): a new tool for the assessment of quality of life in patients treated with biologic response modifiers. Qual Life Res. 2004;13(1):137–154.
   PubMed Web of Science ®Google Scholar
• Cella D, Jensen SE, Webster K, et al. Measuring health-related quality of life in leukemia: the functional assessment of cancer therapy-leukemia (FACT-Leu) questionnaire. Value Health. 2012;15(8):1051–1058.
   PubMed Web of Science ®Google Scholar
• Hahn EA, Glendenning GA, Sorensen MV, et al. Quality of life in patients with newly diagnosed chronic phase chronic myeloid leukemia on imatinib versus interferon alfa plus low-dose cytarabine: results from the IRIS Study. J Clin Oncol. 2003;21(11):2138–2146.
   PubMed Web of Science ®Google Scholar
• Cortes JE, Gambacorti-Passerini C, Deininger MW, et al. Patient-reported outcomes in the phase 3 BFORE trial of bosutinib versus imatinib for newly diagnosed chronic phase chronic myeloid leukemia. J Cancer Res Clin Oncol. 2019;145(6):1589–1599.
   PubMed Web of Science ®Google Scholar
• Williams LA, Gonzalez AG, Ault P, et al. Measuring the symptom burden associated with the treatment of chronic myeloid leukemia. Blood. 2013;122(5):641–647.
   PubMed Web of Science ®Google Scholar
• Cortes JE, Lipton JH, Miller CB, et al. Evaluating the impact of a switch to nilotinib on imatinib-related chronic low-grade adverse events in patients with CML-CP: the ENRICH study. Clin Lymphoma Myeloma Leuk. 2016;16(5):286–296.
   PubMed Web of Science ®Google Scholar
• Efficace F, Cocks K, Breccia M, et al. 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. Crit Rev Oncol Hematol. 2012;81(2):123–135.
   PubMed Web of Science ®Google Scholar
• US Food and Drug Administration. Patient-focused drug development: collecting comprehensive and representative input. Guidance for Industry, Food and Drug Administration Staff, and Other Stakeholders; 2020; [cited 2020 Sep 15]. Available from: https://www.fda.gov/media/139088/download
   Google Scholar
• US Food and Drug Administration. Principles for selecting, developing, modifying, and adapting patient-reported outcome instruments for use in medical device evaluation. Draft Guidance for Industry and Food and Drug Administration Staff, and other Stakeholders; 2020; [cited 2020 Sep 15]. Available from: https://www.fda.gov/media/141565/download
   Google Scholar
• Nolte S, Liegl G, Petersen MA, et al. General population normative data for the EORTC QLQ-C30 health-related quality of life questionnaire based on 15,386 persons across 13 European countries, Canada and the Unites States. Eur J Cancer. 2019;107:153–163.
   PubMed Web of Science ®Google Scholar
• Dewolf L, Koller M, Velikova G, et al. EORTC quality of life group translation procedure. 3rd ed. EORTC Quality of Life Group Publication, Brussels; 2009. (ISBN 978-2-930064-38-3).
   Google Scholar
• Efficace F, Stagno F, Iurlo A, et al. Health-related quality of life of newly diagnosed chronic myeloid leukemia patients treated with first-line dasatinib versus imatinib therapy. Leukemia. 2020;34(2):488–498.
   PubMed Web of Science ®Google Scholar
• Cronbach LJ, Warrington WG. Time-limit tests: estimating their reliability and degree of speeding. Psychometrika. 1951;16(2):167–188.
   PubMedGoogle Scholar
• Nunnally JC. Psychometric theory. 3rd ed. New York: McGraw-Hill; 1994.
   Google Scholar
• Shrout PE, Fleiss JL. Intraclass correlations: uses in assessing rater reliability. Psychol Bull. 1979;86(2):420–428.
   PubMed Web of Science ®Google Scholar
• Koo TK, Li MY. A guideline of selecting and reporting intraclass correlation coefficients for reliability research. J Chiropr Med. 2016;15(2):155–163.
   PubMed Web of Science ®Google Scholar
• Hu L-t, Bentler PM. Cutoff criteria for fit indexes in covariance structure analysis: conventional criteria versus new alternatives. Struct Eq Model. 1999;6(1):1–55.
   Web of Science ®Google Scholar
• Schermelleh-Engel K, Moosbrugger H, Müller H. Evaluating the fit of structural equation models: tests of significance and descriptive goodness-of-fit measures. Methods Psychol Res. 2003;8(2):23–74.
   Google Scholar
• Cocks K, King MT, Velikova G, et al. Evidence-based guidelines for interpreting change scores for the European Organisation for the Research and Treatment of Cancer Quality of Life Questionnaire Core 30. Eur J Cancer. 2012;48(11):1713–1721.
   PubMed Web of Science ®Google Scholar
• Papadakos JK, Charow RC, Papadakos CJ, et al. Evaluating cancer patient-reported outcome measures: readability and implications for clinical use. Cancer. 2019;125(8):1350–1356.
   PubMed Web of Science ®Google Scholar
• Sacha T, Gora-Tybor J, Wasak-Szulkowska E, et al. Quality of life and adherence to therapy in patients with chronic myeloid leukemia treated with nilotinib as a second-line therapy: a multicenter prospective observational study. Clin Lymphoma Myeloma Leuk. 2017;17(5):283–295.
   PubMed Web of Science ®Google Scholar
• Tan BK, Chua SS, Chen LC, et al. Efficacy of a medication management service in improving adherence to tyrosine kinase inhibitors and clinical outcomes of patients with chronic myeloid leukaemia: a randomised controlled trial. Support Care Cancer. 2020;28(7):3249–3249.
   PubMed Web of Science ®Google Scholar
• Efficace F, Baccarani M, Breccia M, et al. Health-related quality of life in chronic myeloid leukemia patients receiving long-term therapy with imatinib compared with the general population. Blood. 2011;118(17):4554–4560.
   PubMed Web of Science ®Google Scholar
• Efficace F, Rosti G, Aaronson N, et al. Patient- versus physician-reporting of symptoms and health status in chronic myeloid leukemia. Haematologica. 2014;99(4):788–793.
   PubMed Web of Science ®Google Scholar
• Basch E, Barbera L, Kerrigan CL, et al. Implementation of patient-reported outcomes in routine medical care. Am Soc Clin Oncol Educ Book. 2018;38:122–134.
   PubMedGoogle Scholar
• Ector GI, Westerweel PE, Hermens RP, et al. The development of a web-based, patient-centered intervention for patients with chronic myeloid leukemia (CMyLife): design thinking development approach. J Med Internet Res. 2020;22(5):e15895.
   PubMed Web of Science ®Google Scholar
• Efficace F, Castagnetti F, Martino B, et al. Health-related quality of life in patients with chronic myeloid leukemia receiving first-line therapy with nilotinib. Cancer. 2018;124(10):2228–2237.
   PubMed Web of Science ®Google Scholar