Centers for Disease Control and Prevention. Health-Related Quality of Life (HRQOL): hrqol Concepts. Available at https://www.cdc.gov/hrqol/concept.htm. (Accessed 5 November 2019)
European Medicine Agency. Reflection paper on the regulatory guidance for the use of heath related quality of life (HRQL) measures in the evaluation of medicinal product. 2005 Available at https://www.ema.europa.eu/documents/scientific-guideline/reflection-paper-regulatory-guidance-use-healthrelated-quality-life-hrql-measures-evaluation_en.pdf. (Accessed 21 January 2019)
Gnanasakthy, A., Mordin, M., Evans, E., Doward, L., DeMuro, C., A review of patient-reported outcome labeling in the United States (2011-2015). Value Health 20 (2017), 420–429.
Johnson, J.R., Temple, R., Food and Drug Administration requirements for approval of new anticancer drugs. Cancer Treat Rep 69 (1985), 1155–1159.
Kluetz, P.G., O'Connor, D.J., Soltys, K., Incorporating the patient experience into regulatory decision making in the USA, Europe, and Canada. Lancet Oncol 19 (2018), e267–e274.
Basch, E., Abernethy, A.P., Mullins, C.D., Reeve, B.B., Smith, M.L., Coons, S.J., et al. Recommendations for incorporating patient-reported outcomes into clinical comparative effectiveness research in adult oncology. J Clin Oncol 30 (2012), 4249–4255.
Mesa, R.A., Gotlib, J., Gupta, V., Catalano, J.V., Deininger, M.W., Shields, A.L., et al. Effect of ruxolitinib therapy on myelofibrosis-related symptoms and other patient-reported outcomes in COMFORT-I: a randomized, double-blind, placebo-controlled trial. J Clin Oncol 31 (2013), 1285–1292.
Deisseroth, A., Kaminskas, E., Grillo, J., Chen, W., Saber, H., Lu, H.L., et al. U.S. Food and Drug Administration approval: ruxolitinib for the treatment of patients with intermediate and high-risk myelofibrosis. Clin Cancer Res 18 (2012), 3212–3217.
Topp, M.S., Zimmerman, Z., Cannell, P., Dombret, H., Maertens, J., Stein, A., et al. Health-related quality of life in adults with relapsed/refractory acute lymphoblastic leukemia treated with blinatumomab. Blood 131 (2018), 2906–2914.
Bonnetain, F., Dahan, L., Maillard, E., Ychou, M., Mitry, E., Hammel, P., et al. Time until definitive quality of life score deterioration as a means of longitudinal analysis for treatment trials in patients with metastatic pancreatic adenocarcinoma. Eur J Cancer 46 (2010), 2753–2762.
Kabbinavar, F.F., Wallace, J.F., Holmgren, E., Yi, J., Cella, D., Yost, K.J., et al. Health-related quality of life impact of bevacizumab when combined with irinotecan, 5-fluorouracil, and leucovorin or 5-fluorouracil and leucovorin for metastatic colorectal cancer. Oncologist 13 (2008), 1021–1029.
Charton, E., Bachet, J.B., Hammel, P., Desramé, J., Chibaudel, B., Cohen, R., et al. Impact on health-related quality of life deterioration-free survival of a first-line therapy combining nab-paclitaxel plus either gemcitabine or simplified leucovorin and fluorouracil for patients with metastatic pancreatic cancer: results of the randomized phase II AFUGEM GERCOR clinical trial. Cancer Med 8 (2019), 5079–5088.
Taphoorn, M.J.B., Dirven, L., Kanner, A.A., Lavy-Shahaf, G., Weinberg, U., Taillibert, S., et al. Influence of treatment with tumor-treating fields on health-related quality of life of patients with newly diagnosed glioblastoma: a secondary analysis of a randomized clinical trial. JAMA Oncol 4 (2018), 495–504.
Lee, C.K., Novello, S., Ryden, A., Mann, H., Mok, T., Patient-reported symptoms and impact of treatment with Osimertinib versus chemotherapy in advanced non-small-cell lung cancer: the AURA3 trial. J Clin Oncol 36 (2018), 1853–1860.
Chi, K.N., Protheroe, A., Rodriguez-Antolin, A., Facchini, G., Suttman, H., Matsubara, N., et al. Patient-reported outcomes following abiraterone acetate plus prednisone added to androgen deprivation therapy in patients with newly diagnosed metastatic castration-naive prostate cancer (LATITUDE): an international, randomised phase 3 trial. Lancet Oncol 19 (2018), 194–206.
Charton, E., Cuer, B., Cottone, F., Efficace, F., Touraine, C., Hamidou, Z., et al. Time to deterioration in cancer randomized clinical trials for patient-reported outcomes data: a systematic review. Qual Life Res 29 (2020), 867–878.
Harland, S., Staffurth, J., Molina, A., Hao, Y., Gagnon, D.D., Sternberg, C.N., et al. Effect of abiraterone acetate treatment on the quality of life of patients with metastatic castration-resistant prostate cancer after failure of docetaxel chemotherapy. Eur J Cancer 49 (2013), 3648–3657.
Cella, D., Grunwald, V., Nathan, P., Doan, J., Dastani, H., Taylor, F., et al. Quality of life in patients with advanced renal cell carcinoma given nivolumab versus everolimus in CheckMate 025: a randomised, open-label, phase 3 trial. Lancet Oncol 17 (2016), 994–1003.
Cella, D., McKendrick, J., Kudlac, A., Palumbo, A., Oukessou, A., Vij, R., et al. Impact of elotuzumab treatment on pain and health-related quality of life in patients with relapsed or refractory multiple myeloma: results from the ELOQUENT-2 study. Ann Hematol 97 (2018), 2455–2463.
Ludwig, H., Ponisch, W., Knop, S., Egle, A., Hinke, A., Schreder, M., et al. Quality of life in patients with relapsed/refractory multiple myeloma during ixazomib-thalidomide-dexamethasone induction and ixazomib maintenance therapy and comparison to the general population. Leuk Lymphoma 61 (2020), 377–386.
Ludwig, H., Moreau, P., Dimopoulos, M.A., Mateos, M.V., Kaiser, M., Hajek, R., et al. Health-related quality of life in the ENDEAVOR study: carfilzomib-dexamethasone vs bortezomib-dexamethasone in relapsed/refractory multiple myeloma. Blood Cancer J, 9, 2019, 23.
Reck, M., Taylor, F., Penrod, J.R., DeRosa, M., Morrissey, L., Dastani, H., et al. Impact of nivolumab versus docetaxel on health-related quality of life and symptoms in patients with advanced squamous non-small cell lung cancer: results from the CheckMate 017 study. J Thorac Oncol 13 (2018), 194–204.
Reck, M., Brahmer, J., Bennett, B., Taylor, F., Penrod, J.R., DeRosa, M., et al. Evaluation of health-related quality of life and symptoms in patients with advanced non-squamous non-small cell lung cancer treated with nivolumab or docetaxel in CheckMate 057. Eur J Cancer 102 (2018), 23–30.
Efficace, F., Castagnetti, F., Martino, B., Breccia, M., D'Adda, M., Angelucci, E., et al. Health-related quality of life in patients with chronic myeloid leukemia receiving first-line therapy with nilotinib. Cancer 124 (2018), 2228–2237.
Laetsch, T.W., Myers, G.D., Baruchel, A., Dietz, A.C., Pulsipher, M.A., Bittencourt, H., et al. Patient-reported quality of life after tisagenlecleucel infusion in children and young adults with relapsed or refractory B-cell acute lymphoblastic leukaemia: a global, single-arm, phase 2 trial. Lancet Oncol 20 (2019), 1710–1718.
Cottone, F., Anota, A., Bonnetain, F., Efficace, F., Time to quality of life improvement as a method for longitudinal analysis of treatment efficacy. Qual Life Res, 26, 2017, 37.
Anota, A., Hamidou, Z., Paget-Bailly, S., Chibaudel, B., Bascoul-Mollevi, C., Auquier, P., et al. Time to health-related quality of life score deterioration as a modality of longitudinal analysis for health-related quality of life studies in oncology: do we need RECIST for quality of life to achieve standardization?. Qual Life Res 24 (2015), 5–18.
Schunemann, H.J., Guyatt, G.H., Commentary–goodbye M(C)ID! Hello MID, where do you come from?. Health Serv Res 40 (2005), 593–597.
Aaronson, N.K., Ahmedzai, S., Bergman, B., Bullinger, M., Cull, A., Duez, N.J., 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 85 (1993), 365–376.
Osoba, D., Rodrigues, G., Myles, J., Zee, B., Pater, J., Interpreting the significance of changes in health-related quality-of-life scores. J Clin Oncol 16 (1998), 139–144.
Fine, J.P., Gray, R.J., A proportional hazards model for the subdistribution of a competing risk. J Am Stat Assoc 94 (1999), 496–509.
Gray, R.J., A Class of K-sample tests for comparing the cumulative incidence of a competing risk. The Ann Stat 16 (1988), 1141–1154.
Putter, H., Fiocco, M., Geskus, R.B., Tutorial in biostatistics: competing risks and multi-state models. Stat Med 26 (2007), 2389–2430.
Austin, P.C., Lee, D.S., Fine, J.P., Introduction to the analysis of survival data in the presence of competing risks. Circulation 133 (2016), 601–609.
Castagnetti, F., Breccia, M., Gugliotta, G., Martino, B., D'Adda, M., Stagno, F., et al. Nilotinib 300 mg twice daily: an academic single-arm study of newly diagnosed chronic phase chronic myeloid leukemia patients. Haematologica 101 (2016), 1200–1207.
European Medicine Agency. ICH E9 (R1) addendum on estimands and sensitivity analysis in clinical trials to the guideline on statistical principles for clinical trials. 2017 Available at https://www.ema.europa.eu/en/documents/scientific-guideline/draft-ich-e9-r1-addendum-estimands-sensitivity-analysis-clinical-trials-guideline-statistical_en.pdf. (Accessed 15 April 2020)
Lesko, C.R., Lau, B., Bias due to confounders for the exposure-competing risk relationship. Epidemiology 28 (2017), 20–27.
Matsuyama, Y., Yamaguchi, T., Estimation of the marginal survival time in the presence of dependent competing risks using inverse probability of censoring weighted (IPCW) methods. Pharm Stat 7 (2008), 202–214.
Anota, A., Barbieri, A., Savina, M., Pam, A., Gourgou-Bourgade, S., Bonnetain, F., et al. Comparison of three longitudinal analysis models for the health-related quality of life in oncology: a simulation study. Health Qual Life Outcomes, 12, 2014, 192.
Musoro, J.Z., Bottomley, A., Coens, C., Eggermont, A.M., King, M.T., Cocks, K., et al. Interpreting European Organisation for research and treatment for cancer quality of life questionnaire core 30 scores as minimally importantly different for patients with malignant melanoma. Eur J Cancer 104 (2018), 169–181.
Maringwa, J.T., Quinten, C., King, M., Ringash, J., Osoba, D., Coens, C., et al. Minimal important differences for interpreting health-related quality of life scores from the EORTC QLQ-C30 in lung cancer patients participating in randomized controlled trials. Support Care Cancer 19 (2011), 1753–1760.
Kvam, A.K., Fayers, P., Wisloff, F., What changes in health-related quality of life matter to multiple myeloma patients? A prospective study. Eur J Haematol 84 (2010), 345–353.
Musoro, J.Z., Coens, C., Fiteni, F., Katarzyna, P., Cardoso, F., Russell, N.S., et al. Minimally important differences for interpreting EORTC QLQ-C30 scores in patients with advanced Breast cancer. JNCI Cancer Spectr, 3, 2019, pkz037.
Fiteni, F., Anota, A., Bonnetain, F., Oster, J.P., Pichon, E., Wislez, M., et al. Health-related quality of life in elderly patients with advanced non-small cell lung cancer comparing carboplatin and weekly paclitaxel doublet chemotherapy with monotherapy. Eur Respir J 48 (2016), 861–872.
