A practical guide to understanding, using and including patient reported outcomes in clinical trials in ovarian cancer

다국어 초록 (Multilingual Abstract)

Health related quality of life (HRQOL) is a key priority for patients with ovarian cancer as there is significant morbidity associated with the disease and the treatment. It is therefore essential to include measures of HRQOL and patient reported outcomes (PROs) in all clinical trials and ideally report them in the initial manuscript. The results of these analyses help interpret the primary trial endpoints which are typically progression free survival and overall survival from the perspective of the patients, but can also assist with regulatory approval of new drugs and inform future patients regarding the potential benefits and downsides of the treatment as well as help support clinical recommendations. Including PROs in clinical trials allows patient-defined clinical benefits to be assessed in parallel to traditional survival outcomes to provide a more holistic overview and aid in the interpretation of the trial results. Given the importance of these instruments in clinical trials, greater effort is required to improve the appropriate inclusion, quality of analyses and reporting of PROs. It is also essential that all clinicians understand the intricacies of the selection, implementation and interpretation of these measures of HRQOL and PRO's and how important their contribution is to clinical trials as well as clinical practice. This review is a practical guide for clinicians to gain a better understanding of PROs and how they can be incorporated into ovarian cancer trials.

참고문헌 (Reference)

1 Mercieca-Bebber R, "The patient-reported outcome content of international ovarian cancer randomised controlled trial protocols" 25 : 2457-2465, 2016

2 Basch E, "The missing voice of patients in drug-safety reporting" 362 : 865-869, 2010

3 Mokkink LB, "The COSMIN study reached international consensus on taxonomy, terminology, and definitions of measurement properties for health-related patient-reported outcomes" 63 : 737-745, 2010

4 Calvert M, "The CONSORT patient-reported outcome (PRO) extension: implications for clinical trials and practice" 11 : 184-, 2013

5 Motzer RJ, "Sunitinib versus interferon alfa in metastatic renal-cell carcinoma" 356 : 115-124, 2007

6 Basen-Engquist K, "Reliability and validity of the functional assessment of cancer therapy-ovarian" 19 : 1809-1817, 2001

7 Donovan KA, "Recommended patient-reported core set of symptoms and quality-of-life domains to measure in ovarian cancer treatment trials" 106 : dju128-, 2014

8 Revicki DA, "Recommendations on health-related quality of life research to support labeling and promotional claims in the United States" 9 : 887-900, 2000

9 Basch E, "Recommendations for incorporating patient-reported outcomes into clinical comparative effectiveness research in adult oncology" 30 : 4249-4255, 2012

10 Greimel ER, "Randomized study of the Arbeitsgemeinschaft Gynaekologische Onkologie Ovarian Cancer Study Group comparing quality of life in patients with ovarian cancer treated with cisplatin/paclitaxel versus carboplatin/paclitaxel" 24 : 579-586, 2006

11 Harano K, "Quality-of-life outcomes from a randomized phase III trial of dose-dense weekly paclitaxel and carboplatin compared with conventional paclitaxel and carboplatin as a first-line treatment for stage II-IV ovarian cancer: Japanese Gynecologic Oncology Group Trial (JGOG3016)" 25 : 251-257, 2014

12 Zee B, "Quality-adjusted time without symptoms or toxicity analysis of interferon maintenance in multiple myeloma" 16 : 2834-2839, 1998

13 Ledermann JA, "Quality of life during olaparib maintenance therapy in platinum-sensitive relapsed serous ovarian cancer" 115 : 1313-1320, 2016

14 Joly F, "Quality of life and/or symptom control in randomized clinical trials for patients with advanced cancer" 18 : 1935-1942, 2007

15 Patil S, "Q-TWiST analysis to estimate overall benefit for patients with metastatic renal cell carcinoma treated in a phase III trial of sunitinib vs interferon-${\alpha}$" 106 : 1587-1590, 2012

16 Sherrill B, "Q-TWiST analysis comparing ipilimumab/dacarbazine vs placebo/dacarbazine for patients with stage III/IV melanoma" 109 : 8-13, 2013

17 Kaye SB, "Phase II, open-label, randomized, multicenter study comparing the efficacy and safety of olaparib, a poly (ADP-ribose) polymerase inhibitor, and pegylated liposomal doxorubicin in patients with BRCA1 or BRCA2 mutations and recurrent ovarian cancer" 30 : 372-379, 2012

18 Bellmunt J, "Pembrolizumab as second-line therapy for advanced urothelial carcinoma" 376 : 1015-1026, 2017

19 Bottomley A, "Patient-reported outcomes: assessment and current perspectives of the guidelines of the Food and Drug Administration and the reflection paper of the European Medicines Agency" 45 : 347-353, 2009

20 Friedlander ML, "Patient-reported outcomes in ovarian cancer clinical trials" 24 (24): x64-x68, 2013

21 Friedlander M, "Patient-reported outcomes (PRO) in ovarian cancer clinical trials-lost opportunities and lessons learned" 27 (27): i66-i71, 2016

22 Stockler MR, "Patient-reported outcome results from the open-label phase III AURELIA trial evaluating bevacizumab-containing therapy for platinum-resistant ovarian cancer" 32 : 1309-1316, 2014

23 Blumenthal GM, "Oncology drug approvals: evaluating endpoints and evidence in an era of breakthrough therapies" 22 : 762-767, 2017

24 Pujade-Lauraine E, "Olaparib tablets as maintenance therapy in patients with platinum-sensitive, relapsed ovarian cancer and a BRCA1/2 mutation (SOLO2/ENGOT-Ov21): a double-blind, randomised, placebo-controlled, phase 3 trial" 18 : 1274-1284, 2017

25 Ledermann J, "Olaparib maintenance therapy in platinum-sensitive relapsed ovarian cancer" 366 : 1382-1392, 2012

26 Ledermann J, "Olaparib maintenance therapy in patients with platinum-sensitive relapsed serous ovarian cancer: a preplanned retrospective analysis of outcomes by BRCA status in a randomised phase 2 trial" 15 : 852-861, 2014

27 King MT, "Measuring what matters MOST: validation of the Measure of Ovarian Symptoms and Treatment, a patient-reported outcome measure of symptom burden and impact of chemotherapy in recurrent ovarian cancer" 27 : 59-74, 2018

28 Miyaji T, "Japanese translation and linguistic validation of the US National Cancer Institute's patient-reported outcomes version of the common terminology criteria for adverse events (PRO-CTCAE)" 1 : 8-, 2017

29 Bennett BK, "Impact of oxaliplatin-induced neuropathy: a patient perspective" 20 : 2959-2967, 2012

30 Gourgou-Bourgade S, "Impact of FOLFIRINOX compared with gemcitabine on quality of life in patients with metastatic pancreatic cancer: results from the PRODIGE 4/ACCORD 11 randomized trial" 31 : 23-29, 2013

31 Vaughn DJ, "Health-related quality-of-life analysis from KEYNOTE-045: a phase III study of pembrolizumab versus chemotherapy for previously treated advanced urothelial cancer" 36 : 1579-1587, 2018

32 Friedlander M, "Health-related quality of life (HRQOL) and patient-centered outcomes with maintenance olaparib compared with placebo following chemotherapy in patients with germline (g) BRCA-mutated (m) platinum-sensitive relapsed serous ovarian cancer (PSR SOC): SOLO2 phase III trial" 35 : 2017

33 Calvert M, "Guidelines for inclusion of patient-reported outcomes in clinical trial protocols: the SPIRIT-PRO Extension" 319 : 483-494, 2018

34 Food and Drug Administration (US), "Guidance for industry: patient-reported outcome measures: use in medical product development to support labelling claims" Food and Drug Administration

35 Friedlander M, "Getting the most out of follow-up: a prospective study using the Measure of Ovarian Symptoms and Treatment concerns (MOST) symptom index to evaluate and track adverse effects (AEs) and detect symptoms of recurrence in patients with ovarian cancer (OC) following first line chemotherapy (1LT)" American Society of Clinical Oncology 2018

36 Kluetz PG, "Focusing on core patient-reported outcomes in cancer clinical trials-response" 22 : 5618-, 2016

37 Wilson MK, "Fifth Ovarian Cancer Consensus Conference of the Gynecologic Cancer InterGroup: recurrent disease" 28 : 727-732, 2017

38 Joly F, "Fifth Ovarian Cancer Consensus Conference of the Gynecologic Cancer InterGroup: recommendations on incorporating patient-reported outcomes in clinical trials in epithelial ovarian cancer" 78 : 133-138, 2017

39 Cocks K, "Evidence-based guidelines for determination of sample size and interpretation of the European Organisation for the Research and Treatment of Cancer Quality of Life Questionnaire Core 30" 29 : 89-96, 2011

40 Katsumata N, "Dose-dense paclitaxel once a week in combination with carboplatin every 3 weeks for advanced ovarian cancer: a phase 3, open-label, randomised controlled trial" 374 : 1331-1338, 2009

41 Basch E, "Development of the National Cancer Institute's patient-reported outcomes version of the common terminology criteria for adverse events (PRO-CTCAE)" 106 : dju244-, 2014

42 Mercieca-Bebber R, "Design, implementation and reporting strategies to reduce the instance and impact of missing patient-reported outcome (PRO) data: a systematic review" 6 : e010938-, 2016

43 Park SB, "Clinical and genetic predictors of paclitaxel neurotoxicity based on patient- versus clinician-reported incidence and severity of neurotoxicity in the ICON7 trial" 28 : 2733-2740, 2017

44 Luckett T, "Choosing between the EORTC QLQ-C30 and FACT-G for measuring health-related quality of life in cancer clinical research: issues, evidence and recommendations" 22 : 2179-2190, 2011

45 Mols F, "Chemotherapy-induced neuropathy and its association with quality of life among 2- to 11-year colorectal cancer survivors: results from the population-based PROFILES registry" 31 : 2699-2707, 2013

46 Pignata S, "Carboplatin plus paclitaxel once a week versus every 3 weeks in patients with advanced ovarian cancer (MITO-7): a randomised, multicentre, open-label, phase 3 trial" 15 : 396-405, 2014

47 Schulz KF, "CONSORT 2010 statement: updated guidelines for reporting parallel group randomised trials" 340 : c332-, 2010

48 Pujade-Lauraine E, "Bevacizumab combined with chemotherapy for platinum-resistant recurrent ovarian cancer: the AURELIA open-label randomized phase III trial" 32 : 1302-1308, 2014

49 Greimel E, "An international field study of the reliability and validity of a disease-specific questionnaire module (the QLQ-OV28) in assessing the quality of life of patients with ovarian cancer" 39 : 1402-1408, 2003

50 Hoskins P, "Advanced ovarian cancer: phase III randomized study of sequential cisplatin-topotecan and carboplatin-paclitaxel vs carboplatin-paclitaxel" 102 : 1547-1556, 2010

51 Wilson MK, "A systematic review of health-related quality of life reporting in ovarian cancer phase III clinical trials: room to improve" 23 : 203-213, 2018

52 Mercieca-Bebber R, "A systematic evaluation of compliance and reporting of patient-reported outcome endpoints in ovarian cancer randomised controlled trials: implications for generalisability and clinical practice" 1 : 5-, 2017