Real-world evidence to guide healthcare policies in oncology

Donia, Marco; Hansen, Steen Werner; Svane, Inge Marie

doi:10.18632/oncotarget.27077

Cited by 10 publications

(9 citation statements)

References 10 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…This prospective real-world study presents long-term outcomes (≥ 3 years of follow-up) of patients with advanced melanoma treated with ipilimumab and other therapies. Safety outcomes and OS in patients treated with ipilimumab 3 mg/kg were similar to those previously reported in ipilimumab randomised clinical trials, despite the inclusion of patients who are typically excluded from randomised controlled trials (e.g., those with ECOG PS ≥ 2 or active/untreated brain metastases) [ 12 , 13 ]. This study also showed that patients who switched therapy (from ipilimumab or non-ipilimumab therapies) had longer OS than those who did not switch therapy.…”

Section: Discussionsupporting

confidence: 70%

“…Although efficacy and safety results from randomised controlled trials of ipilimumab are available, data from long-term real-world studies are lacking. Real-world studies are being increasingly used to complement results from clinical trials as they represent patients who are diagnosed and treated routinely, including those who did not meet the selection criteria for registration into randomised controlled trials (e.g., patients with Eastern Cooperative Oncology Group performance status [ECOG PS] ≥ 2 or active/untreated brain metastases) [ 12 , 13 ]. Since the introduction of anti–PD-1 antibodies, which have shown superior first-line efficacy compared with ipilimumab [ 14 , 15 ], ipilimumab has been less commonly used as first-line monotherapy.…”

Section: Introductionmentioning

confidence: 99%

See 1 more Smart Citation

Long-term real-world experience with ipilimumab and non-ipilimumab therapies in advanced melanoma: the IMAGE study

et al. 2021

View full text Add to dashboard Cite

Background Ipilimumab has shown long-term overall survival (OS) in patients with advanced melanoma in clinical trials, but robust real-world evidence is lacking. We present long-term outcomes from the IMAGE study (NCT01511913) in patients receiving ipilimumab and/or non-ipilimumab (any approved treatment other than ipilimumab) systemic therapies. Methods IMAGE was a multinational, prospective, observational study assessing adult patients with advanced melanoma treated with ipilimumab or non-ipilimumab systemic therapies between June 2012 and March 2015 with ≥3 years of follow-up. Adjusted OS curves based on multivariate Cox regression models included covariate effects. Safety and patient-reported outcomes were assessed. Results Among 1356 patients, 1094 (81%) received ipilimumab and 262 (19%) received non-ipilimumab index therapy (systemic therapy [chemotherapy, anti–programmed death 1 antibodies, or BRAF ± MEK inhibitors], radiotherapy, and radiosurgery). In the overall population, median age was 64 years, 60% were male, 78% were from Europe, and 78% had received previous treatment for advanced melanoma. In the ipilimumab-treated cohort, 780 (71%) patients did not receive subsequent therapy (IPI-noOther) and 314 (29%) received subsequent non-ipilimumab therapy (IPI-Other) on study. In the non-ipilimumab–treated cohort, 205 (78%) patients remained on or received other subsequent non-ipilimumab therapy (Other-Other) and 57 (22%) received subsequent ipilimumab therapy (Other-IPI) on study. Among 1151 patients who received ipilimumab at any time during the study (IPI-noOther, IPI-Other, and Other-IPI), 296 (26%) reported CTCAE grade ≥ 3 treatment-related adverse events, most occurring in year 1. Ipilimumab-treated and non-ipilimumab–treated patients who switched therapy (IPI-Other and Other-IPI) had longer OS than those who did not switch (IPI-noOther and Other-Other). Patients with prior therapy who did not switch therapy (IPI-noOther and Other-Other) showed similar OS. In treatment-naive patients, those in the IPI-noOther group tended to have longer OS than those in the Other-Other group. Patient-reported outcomes were similar between treatment cohorts. Conclusions With long-term follow-up (≥ 3 years), safety and OS in this real-world population of patients treated with ipilimumab 3 mg/kg were consistent with those reported in clinical trials. Patient-reported quality of life was maintained over the study period. OS analysis across both pretreated and treatment-naive patients suggested a beneficial role of ipilimumab early in treatment. Trial registration ClinicalTrials.gov, NCT01511913. Registered January 19, 2012 – Retrospectively registered, https://clinicaltrials.gov/ct2/show/NCT01511913

show abstract

Section: Discussionsupporting

confidence: 70%

Section: Introductionmentioning

confidence: 99%

Long-term real-world experience with ipilimumab and non-ipilimumab therapies in advanced melanoma: the IMAGE study

et al. 2021

View full text Add to dashboard Cite

show abstract

“…A major strength of our population-based registry over the meta-analyses discussed in the introduction is that we also report data from patients with more advanced melanoma and a worse clinical performance score that do not meet the in- and exclusion criteria [ 25 , 26 ]. Another advantage of our registry is that we were able to adjust survival for patient baseline (tumor) characteristics and known risk factors.…”

Section: Discussionmentioning

confidence: 99%

“…The previously mentioned meta-analyses included large randomized controlled trials, however, a vast proportion of patients with advanced melanoma treated in daily practice do not meet the in- and exclusion criteria of these trials [ 25 , 26 ]. Another limitation of these analyses was that the authors lacked additional information on patient-specific data, including the distribution of known risk factors among men and women [ 27 ]; this is important as the comparison between men and women in the setting of a randomized controlled trial can still be confounded, as it is not sex that is randomized.…”

Section: Introductionmentioning

confidence: 99%

Sex-Based Differences in Treatment with Immune Checkpoint Inhibition and Targeted Therapy for Advanced Melanoma: A Nationwide Cohort Study

Kooij

Dekkers

Aarts

et al. 2021

Cancers

View full text Add to dashboard Cite

Recent meta-analyses show conflicting data on sex-dependent benefit following systemic treatment for advanced melanoma patients. We examined the nationwide Dutch Melanoma Treatment Registry (July 2013–July 2018), assessing sex-dependent differences in advanced melanoma patients (stage IIIC/IV) with respect to clinical characteristics, mutational profiles, treatments initiated, grade 3–4 adverse events (AEs), treatment responses, and mortality. We included 3985 patients, 2363 men (59%) and showed that although men and women with advanced melanoma differ in clinical and tumor characteristics, the safety profile of immune checkpoint inhibition (ICI) is comparable. The data suggest a 10% survival advantage for women, mainly seen in patients ≥60 years of age and patients with BRAF V600 mutant melanoma. Following ICI there was no survival difference.

show abstract

“…13 This national cohort includes more than 13,000 men undergoing C-RT or H-RT in England, collected at least 18 months after diagnosis, providing contemporary evidence on functional outcomes from large-scale 'real-world' clinical practice. 14…”

Section: Introductionmentioning

confidence: 99%

Patient-Reported Functional Outcomes After Hypofractionated or Conventionally Fractionated Radiation for Prostate Cancer: A National Cohort Study in England

Nossiter

Sujenthiran

Cowling

et al. 2020

JCO

View full text Add to dashboard Cite

PURPOSE The aim of the current study was to determine patient-reported functional outcomes in men with prostate cancer (PCa) undergoing moderately hypofractionated (H-RT) or conventionally fractionated radiation therapy (C-RT) in a national cohort study. PATIENDS AND METHODS All men diagnosed with PCa between April 2014 and September 2016 in the English National Health Service undergoing C-RT or H-RT were identified in the National Prostate Cancer Audit and mailed a questionnaire at least 18 months after diagnosis. We estimated differences in patient-reported urinary, bowel, sexual, and hormonal function—Expanded Prostate Cancer Index Composite short-form 26 domain scores on a 0 to 100 scale—and health-related quality of life—EQ-5D-5L on a 0 to 1 scale—using linear regression with adjustment for patient, tumor, and treatment-related factors in addition to GI and genitourinary baseline function, with higher scores representing better outcomes. RESULTS Of the 17,058 men in the cohort, 77% responded: 8,432 men received C-RT (64.2%) and 4,699 H-RT (35.8%). Men in the H-RT group were older (age ≥ 70 years: 67.5% v 60.9%), fewer men had locally advanced disease (56.5% v 71.3%), were less likely to receive androgen-deprivation therapy (79.5% v 87.8%), and slightly more men had pretreatment genitourinary procedures (24.2% v 21.2%). H-RT was associated with small increases in adjusted mean Expanded Prostate Cancer Index Composite short-form 26 sexual (3.3 points; 95% CI, 2.1 to 4.5; P < .001) and hormonal function scores (3.2 points; 95% CI, 1.8 to 4.6; P < .001). These differences failed to meet established thresholds for a clinically meaningful change. There were no statistically significant differences in urinary or bowel function and quality of life. CONCLUSION This is the first national cohort study comparing functional outcomes after H-RT and C-RT reported by patients. These real-world results further support the use of H-RT as the standard for radiation therapy in men with nonmetastatic PCa.

show abstract

Real-world evidence to guide healthcare policies in oncology

Cited by 10 publications

References 10 publications

Long-term real-world experience with ipilimumab and non-ipilimumab therapies in advanced melanoma: the IMAGE study

Long-term real-world experience with ipilimumab and non-ipilimumab therapies in advanced melanoma: the IMAGE study

Sex-Based Differences in Treatment with Immune Checkpoint Inhibition and Targeted Therapy for Advanced Melanoma: A Nationwide Cohort Study

Patient-Reported Functional Outcomes After Hypofractionated or Conventionally Fractionated Radiation for Prostate Cancer: A National Cohort Study in England

Contact Info

Product

Resources

About