Relationship between patient-reported outcomes and clinical outcomes in metastatic castration-resistant prostate cancer: post hoc analysis of COU-AA-301 and COU-AA-302

Cella, David; Traina, Shana; Li, T.; Johnson, K; Ho, KL; Molina, Arturo; Shore, Neal D.

doi:10.1093/annonc/mdx759

Cited by 26 publications

(15 citation statements)

References 17 publications

Supporting

Mentioning

Contrasting

Unclassified

Order By: Relevance

“…However, when In patients with metastatic castration-resistant prostate cancer, pain is generally reported alongside poor HRQoL and has been shown to be prognostic of overall and progression-free survival. 14,17 Although pain is not a salient symptom in patients with non-metastatic castration-resistant prostate cancer, 25 pain progression may be associated with disease progression in this population.…”

Section: Discussionmentioning

confidence: 99%

“…12 Overall survival and disease progression outcomes are used to evaluate new treatment approaches, but to be truly valuable to patients, new treatments should not only delay disease progression but also maintain or, ideally, improve, health-related quality of life (HRQoL) without worsening symptoms. 13 Interestingly, HRQoL has been shown to be prognostic of survival in metastatic castration-resistant prostate cancer, 14,15 a finding that was confirmed by analysis of data from two phase 3 studies of enzalutamide in patients with metastatic castration-resistant prostate cancer. 16,17 In addition, HRQoL is central to cost-utility analysis in many countries and is increasingly influencing reimbursement decisions, especially for oncology treatments.…”

Section: Introductionmentioning

confidence: 92%

See 1 more Smart Citation

Patient-reported outcomes following enzalutamide or placebo in men with non-metastatic, castration-resistant prostate cancer (PROSPER): a multicentre, randomised, double-blind, phase 3 trial

Tombal

Saad

Penson

et al. 2019

The Lancet Oncology

View full text Add to dashboard Cite

Background In the phase 3, double-blind, placebo-controlled PROSPER trial of patients with non-metastatic castration-resistant prostate cancer, enzalutamide significantly improved metastasis-free survival (primary endpoint). Here, we report the results of patient-reported outcome (PRO) measures of this study. Methods Patients ≥18 years of age with non-metastatic castration-resistant prostate cancer and a prostate-specific antigen doubling time of ≤10 months were randomised (2:1) via interactive voice/web recognition system to receive enzalutamide (160 mg/day) or placebo. Randomisation was stratified by prostate-specific-antigen-doubling time (<6 months vs ≥6 months) and baseline use of a bone-targeting agent (yes or no). The primary endpoint was metastasis-free survival (MFS), reported elsewhere. Exploratory endpoints, reported here, included pain progression and health-related quality of life (HRQoL), assessed by the Brief Pain Inventory Short Form (BPI-SF), the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-PR25), the Functional Assessment of Cancer Therapy-Prostate (FACT-P), and the EuroQoL 5-Dimensions 5-Levels health questionnaire visual analogue scale (EQ-VAS). Patients completed questionnaires at baseline, Week 17, and every 16 weeks thereafter during treatment. Pre-defined questionnaire thresholds were used to identify clinically meaningful changes. Enrollment for PROSPER is complete and follow-up continues. This trial is registered with ClinicalTrials.gov (NCT02003924). Findings Patients were enrolled from 26 November 2013 to 28 June 2017. Median followup time for all patients was 18•5 months (interquartile range [IQR] 10•7-29•2 months) in the enzalutamide group and 15•1 months (IQR 7•4-25•9 months) in the placebo group. Patients randomised to receive enzalutamide or placebo had similar baseline PRO scores. Changes in 6 Further analyses are needed to determine potential associations between pain, HRQoL, and disease progression in non-metastatic castration-resistant prostate cancer. Implications of all the available evidence As patients with non-metastatic castration-resistant prostate cancer generally have low levels of pre-treatment pain and favourable HRQoL compared to patients with advanced disease, treatments should aim to improve clinical outcomes while maintaining pain control and HRQoL. The demonstrated efficacy, tolerability, and HRQoL profile suggest that enzalutamide represents a treatment option for patients with non-metastatic castrationresistant prostate cancer. PROSPER patient recruitment sites, principal investigators and patients recruited by site* Country Site # Study centre Principal investigator # patients randomised Denmark 421 Herlev Hospital Per Rathenborg

show abstract

Section: Discussionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 92%

Patient-reported outcomes following enzalutamide or placebo in men with non-metastatic, castration-resistant prostate cancer (PROSPER): a multicentre, randomised, double-blind, phase 3 trial

Tombal

Saad

Penson

et al. 2019

The Lancet Oncology

View full text Add to dashboard Cite

show abstract

“…Due to the psychological implications of being diagnosed with metastatic disease, it is important to use patient-reported outcomes to match the instrumental data of MFS with the actual benefit in terms of both the delay of time to symptom worsening and global QoL. It is worth noting that in the most recent trials on PCa, the advantage in MFS is well supported by evidence of clinical benefit in terms of improvement in PFS on next-line therapy (PFS2),49 56 symptom delay and pain progression46 49 and QoL 48 49 57–63…”

Section: Bone Health During Nhtmentioning

confidence: 99%

Bone health management in the continuum of prostate cancer disease: a review of the evidence with an expert panel opinion

et al. 2020

View full text Add to dashboard Cite

Bone health impairment is a frequent detrimental consequence of the high bone tropism of prostate cancer (PCa) cells. It is further worsened by administration of androgen-deprivation therapy (ADT), the current standard of care in the management of advanced PCa, through a rapid and dramatic increase in bone turnover and body mass changes. As a result, patients may experience substantial pain and poor quality of life (QoL) and have an increased risk of death. Notwithstanding the importance of this issue, however, bone health preservation is not yet a widespread clinical goal in daily practice. To address this urgent unmet need, following a thorough discussion of available data and sharing of their clinical practice experience, a panel of Italian experts in the field of bone health and metabolism formulated a number of practical advices for optimising the monitoring and treatment of bone health in men undergoing ADT during all phases of the disease. The rationale behind the venture was to raise awareness on the importance of bone preservation in this complex setting, while providing an instrument to support physicians and facilitate the management of bone health. Current evidence regarding the effects on bone health of ADT, of novel hormone therapies (which improve progression delay, pain control and QoL while consistently carrying the risk of non-pathological fractures in both non-metastatic and metastatic PCa) and of bone turnover inhibitors (whose use is frequently suboptimal) is reviewed. Finally, the expert opinion to optimise bone health preservation is given.

show abstract

“… 43‐46 A randomized trial showed that screening for these symptoms was associated with a 7‐month survival advantage over usual care, which in part may have been because patients in the intervention group had better performance status at progression and thus were more likely to receive optimal treatment 46,47 . Finally, several studies have shown that PROs enhance prediction of survival in myelodysplastic syndromes, 48 multiple myeloma, 49 early‐stage colorectal cancer, 50 advanced breast cancer, 51 metastatic castration‐resistant prostate cancer, 52 metastatic renal cell carcinoma, 53 advanced cancers, 54,55 and a variety of tumor types 56,57 . In fact, some data suggest that PROs predict survival better than provider‐rated performance status 58 …”

Section: The Need For Pghd In Cancer Carementioning

confidence: 99%

Innovations in research and clinical care using patient‐generated health data

Jim

Hoogland

Brownstein

et al. 2020

CA A Cancer J Clinicians

114

View full text Add to dashboard Cite

Patient-generated health data (PGHD), or health-related data gathered from patients to help address a health concern, are used increasingly in oncology to make regulatory decisions and evaluate quality of care. PGHD include self-reported health and treatment histories, patient-reported outcomes (PROs), and biometric sensor data. Advances in wireless technology, smartphones, and the Internet of Things have facilitated new ways to collect PGHD during clinic visits and in daily life. The goal of the current review was to provide an overview of the current clinical, regulatory, technological, and analytic landscape as it relates to PGHD in oncology research and care. The review begins with a rationale for PGHD as described by the US Food and Drug Administration, the Institute of Medicine, and other regulatory and scientific organizations. The evidence base for clinic-based and remote symptom monitoring using PGHD is described, with an emphasis on PROs. An overview is presented of current approaches to digital phenotyping or device-based, real-time assessment of biometric, behavioral, self-report, and performance data. Analytic opportunities regarding PGHD are envisioned in the context of big data and artificial intelligence in medicine. Finally, challenges and solutions for the integration of PGHD into clinical care are presented. The challenges include electronic medical record integration of PROs and biometric data, analysis of large and complex biometric data sets, and potential clinic workflow redesign. In addition, there is currently more limited evidence for the use of biometric data relative to PROs. Despite these challenges, the potential benefits of PGHD make them increasingly likely to be integrated into oncology research and clinical care.

show abstract

Relationship between patient-reported outcomes and clinical outcomes in metastatic castration-resistant prostate cancer: post hoc analysis of COU-AA-301 and COU-AA-302

Abstract: PROs are significantly associated with clinically relevant time-to-event efficacy outcomes in clinical trials and may complement and help predict traditional clinical practice methods for monitoring patients for disease progression.

Cited by 26 publications

References 17 publications

Patient-reported outcomes following enzalutamide or placebo in men with non-metastatic, castration-resistant prostate cancer (PROSPER): a multicentre, randomised, double-blind, phase 3 trial

Patient-reported outcomes following enzalutamide or placebo in men with non-metastatic, castration-resistant prostate cancer (PROSPER): a multicentre, randomised, double-blind, phase 3 trial

Bone health management in the continuum of prostate cancer disease: a review of the evidence with an expert panel opinion

Innovations in research and clinical care using patient‐generated health data

Contact Info

Product

Resources

About