Statistical analysis of patient-reported outcome data in randomised controlled trials of locally advanced and metastatic breast cancer: a systematic review

Pe, Madeline; Dorme, Lien; Coens, Corneel; Basch, Ethan; Calvert, Melanie; Campbell, Alicyn; Cleeland, Charles S.; Cocks, Kim; Collette, Laurence; Dirven, Linda; Dueck, Amylou C.; Devlin, Nancy; Flechtner, Hans‐Henning; Gotay, Carolyn; Griebsch, Ingolf; Grøenvold, Mogens; King, Madeleine; Koller, Michael; Malone, Daniel C.; Martinelli, Francesca; Mitchell, Sandra A.; Musoro, Jammbe Z.; Oliver, Kathy; Piault-Louis, Elisabeth; Piccart, Martine; Pimentel, Francisco; Quinten, Chantal; Reijneveld, Jaap C.; Sloan, Jeff A.; Velikova, Galina; Bottomley, Andrew

doi:10.1016/s1470-2045(18)30418-2

Cited by 71 publications

(67 citation statements)

References 109 publications

(44 reference statements)

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“…A recent review on the use of PROs in a more general context resulted in similar conclusions [13] beyond the aforementioned problems of precise definition [10] of endpoints. Either we have to use a single summary score, other composite endpoints, or we have to address the problem of multiple testing with several or multiple endpoints.…”

Section: How Did Skepticism About Early Integration Studies Arise?mentioning

confidence: 77%

Early Integration of Palliative and Oncological Care: Con

Schuler¹

2018

Oncol Res Treat

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For almost a decade, ‘early integration' has become a buzzword in the palliative care community. Can this still be an issue of controversy? The goals of care in palliative medicine are beyond any criticism and in fact should be, at least in theory, goals of good oncological care. However, the reality presents a different picture. The care of cancer patients requires improvement, and the studies on the early integration of palliative care (EIPC) reveal deficits in the oncological practice. However, the limitations and methodological weaknesses of these EIPC studies are insufficiently analyzed and discussed. The main criticisms relate to the incomplete definition of primary endpoints, published analyses deviating from the study protocols and insufficient consideration for multiple testing. If this criticism is justified, a possible consequence would be to overrate the achievable effects of EIPC and to limit the use of these studies in guiding policies. Improving the care of cancer patients by fostering their primary care by oncologists could provide one of the alternative approaches, but needs to be evaluated in future studies. Unmet needs in physical, psychic, spiritual or social care need to be addressed. Whether this requires a multiprofessional team in all cases is another issue of discussion.

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Section: How Did Skepticism About Early Integration Studies Arise?mentioning

confidence: 77%

Early Integration of Palliative and Oncological Care: Con

Schuler¹

2018

Oncol Res Treat

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“…The majority of the publications on the use of PROs within breast cancer patients deal with the patient's evaluation of a treatment. 10,41 Using PRO data in clinical trials examining side effects of adjuvant endocrine therapy has revealed a higher frequency of both physical and psychosocial symptom burden than based on clinicians' assessment. [42][43][44][45][46] Thus, one of the potential benefits with the application of systemically obtained PRO data, used proactively in survivorship, may be to provide a more comprehensive evaluation that includes the patient's perception of problems experienced during follow-up care.…”

Section: Discussionmentioning

confidence: 99%

<p>Are patient-reported outcomes useful in post-treatment follow-up care for women with early breast cancer? A scoping review</p>

Riis¹,

Bechmann²,

Jensen³

et al. 2019

PROM

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Background: Patient-reported outcomes (PROs) are frequently used to evaluate treatment effects and quality of life in clinical trials. The application of PROs in breast cancer clinics is evolving but their use to generate real-time information for use in follow-up care is uncommon. This proactive use might help to shift healthcare delivery toward a more patient-centered approach by acting as a screening tool for unmet needs or a dialogue tool to discuss issues proposed by the patient. Aims: This review aims to determine the effects and feasibility of using PROs proactively during follow-up care in early breast cancer. Materials and methods: A systematic search was conducted in January 2019 in PubMed, Cochrane Library, Embase, and CINAHL. Studies that exclusively concerned women treated for early breast cancer where PROs were used as a proactive tool during follow-up were included. Results: The search revealed a total of 653 records and four eligible studies were identified; three of which concerned the use of PROs both as a screening tool and as a dialogue tool, and one study in which PROs were used solely as a screening tool. The studies explored the feasibility of collecting and integrating PROs in the clinic and their ability to detect otherwise unrecognized problems. All of the included studies were prone to bias, but they point to potential benefits in respect of better symptom management in follow-up care. Conclusion: Our search identified a small number of low to moderate quality studies of the proactive use of PROs during follow-up after treatment for early stage breast cancer. The limited evidence available suggests that PROs may be useful for providing a more complete picture of the patient's symptoms and problems, possibly leading to improvements in symptom management.

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“…4 Finally, health-related quality of life (HRQoL) scales are often multidimensional and include several subscales, which can lead to complex result patterns. 5 The European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30) is often used to collect data on HRQoL and symptom burden in patients with cancer. 6 However, these data can be analyzed using a variety of methods, as exemplified in different clinical studies of CRC (see Supplemental Table 1 in the online version).…”

Section: Introductionmentioning

confidence: 99%

Health-related Quality of Life in the Phase III LUME-Colon 1 Study: Comparison and Interpretation of Results From EORTC QLQ-C30 Analyses

Lenz

Argilés

Yoshino

et al. 2019

Clinical Colorectal Cancer

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Based on European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30) outputs from the LUME-Colon 1 study, we compared and discussed different statistical methods for evaluating health-related quality of life data in oncology clinical trials. The different analyses consistently showed that patients' overall global health status/quality of life status was not impaired by active treatment with nintedanib versus placebo, and that patients perceived some benefits with nintedanib compared with placebo. Introduction: We used European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30) data from the LUME-Colon 1 study to illustrate different methods of statistical analysis for healthrelated quality of life (HRQoL), and compared the results. Patients and Methods: Patients were randomized 1:1 to receive nintedanib 200 mg twice daily plus best supportive care (n ¼ 386) or matched placebo plus best supportive care (n ¼ 382). Five methods (mean treatment difference averaged over time, using a mixed-effects growth curve model; mixed-effects models for repeated measurements (MMRM); time-to-deterioration (TTD); status change; and responder analysis) were used to analyze EORTC QLQ-C30 global health status (GHS)/QoL and scores from functional scales. Results: Overall, GHS/QoL and physical functioning deteriorated over time. Mean treatment difference slightly favored nintedanib over placebo for physical functioning (adjusted mean, 2.66; 95% confidence interval [CI], 0.97-4.34) and social functioning (adjusted mean, 2.62; 95% CI, 0.66-4.47). GHS/QoL was numerically better with nintedanib versus placebo (adjusted mean, 1.61; 95% CI, À0.004 to 3.27). MMRM analysis had similar results, with better physical functioning in the nintedanib group at all timepoints. There was no significant delay in GHS/QoL deterioration (10%) and physical functioning (16%) with nintedanib versus placebo (TTD analysis). Status change analysis showed a higher proportion of patients with markedly improved GHS/QoL and physical functioning in the nintedanib versus placebo groups. Responder analysis showed a similar, less pronounced pattern. Conclusion: Analyses of EORTC QLQ-C30 data showed that HRQoL was not impaired by treatment with nintedanib versus placebo. Analysis and interpretation of HRQoL endpoints should consider symptom type and severity and course of disease.

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Statistical analysis of patient-reported outcome data in randomised controlled trials of locally advanced and metastatic breast cancer: a systematic review

Cited by 71 publications

References 109 publications

Early Integration of Palliative and Oncological Care: Con

Early Integration of Palliative and Oncological Care: Con

<p>Are patient-reported outcomes useful in post-treatment follow-up care for women with early breast cancer? A scoping review</p>

Health-related Quality of Life in the Phase III LUME-Colon 1 Study: Comparison and Interpretation of Results From EORTC QLQ-C30 Analyses

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