Real-World Treatment Trends Among Patients with Metastatic Castration-Sensitive Prostate Cancer: Results from an International Study.

BACKGROUND: Continuous androgen deprivation therapy ± first-generation non-steroidal antiandrogen was previously the standard-of-care for patients with metastatic castration-sensitive prostate cancer (mCSPC). Treatment intensification with novel hormonal therapy (NHT) or taxane chemotherapy is now approved and guideline-recommended for these patients. METHODS: Physician-reported data on adult patients with mCSPC from the Adelphi Prostate Cancer Disease Specific Programme were analyzed descriptively. We evaluated real-world treatment trends for patients with mCSPC in 5 European countries (United Kingdom, France, Germany, Spain, and Italy) and the United States (US), looking at differences between patients initiating treatment in 2016-2018 and in 2019-2020. We also investigated treatment trends by ethnicity and insurance status in the US. RESULTS: This study found that most patients with mCSPC do not receive treatment intensification. However, greater use of treatment intensification with NHT and taxane chemotherapy was observed in 2019-2020 than in 2016-2018 across 5 European countries. In the US, greater use of treatment intensification with NHT in 2019-2020 than in 2016-2018 was observed for all ethnicity groups and those with Medicare and commercial insurance status. CONCLUSIONS: As the number of patients with mCSPC who receive treatment intensification increases, more patients who progress to metastatic castration-resistant prostate cancer (mCRPC) will have been exposed to intensified treatments. Treatment options for patients with mCSPC and mCRPC overlap, suggesting that an unmet need will emerge for new therapies. Further studies are needed to understand optimal treatment sequencing in mCSPC and mCRPC.

Real-World Treatment Patterns Among Patients With Metastatic Castration-Resistant Prostate Cancer: Results From an International Study.

BACKGROUND: There is limited real-world evidence on how increasing use of treatment intensification in metastatic castration-sensitive prostate cancer (mCSPC) has influenced treatment decisions in metastatic castration-resistant prostate cancer (mCRPC). The study objective was to evaluate the impact of novel hormonal therapy (NHT) and docetaxel use in mCSPC on first-line treatment patterns among patients with mCRPC in 5 European countries and the United States (US). METHODS: Physician-reported data on patients with mCRPC from the Adelphi Prostate Cancer Disease Specific Program were descriptively analyzed. RESULTS: A total of 215 physicians provided data on 722 patients with mCRPC. Across 5 European countries and the US, 65% and 75% of patients, respectively, received NHT, and 28% and 9% of patients, respectively, received taxane chemotherapy as first-line mCRPC treatment. In Europe, patients who had received NHT in mCSPC (n = 76) mostly received taxane chemotherapy in mCRPC (55%). Patients who had received taxane chemotherapy, or who did not receive taxane chemotherapy or NHT in mCSPC (n = 98 and 434, respectively) mostly received NHT in mCRPC (62% and 73%, respectively). In the US, patients who had received NHT, taxane chemotherapy, or neither in mCSPC (n = 32, 12, and 72, respectively) mostly received NHT in mCRPC (53%, 83%, and 83%, respectively). Two patients in Europe were rechallenged with the same NHT. CONCLUSIONS: These findings suggest that physicians consider mCSPC treatment history when making first-line treatment decisions in mCRPC. Further studies are needed to better understand optimal treatment sequencing, especially as new treatments emerge.

Physician- and patient-reported symptom concordance and association with quality of life in hepatocellular carcinoma.

Aim: To investigate the association of discordance in patient- and physician-reported symptoms on health-related quality of life (HRQoL) in hepatocellular carcinoma (HCC). Patients & methods: Data were drawn from a point-in-time survey of physicians and patients conducted in Germany, Italy and Spain (October 2018 - January 2019). Physicians and their consulting patients independently reported baseline characteristics, symptoms, treatment history and satisfaction, and HRQoL derived using the Functional Assessment of Cancer Therapy-Hepatobiliary (FACT-Hep) questionnaire. Results: Of 486 patients analysed, tiredness (73.3%, 73.7%), pain/aches (67.7%, 66.9%) and weight/appetite loss (54.3%, 53.7%) were the most common and concordant patient and physician-reported symptom domains, respectively. The symptom domains showing the largest discordance were reflux/indigestion (14.6%, 5.1%), neurological (11.9%, 5.6%), dermatological (9.3%, 6.2%) symptoms and jaundice (4.7%, 10.3%). Reduced HRQoL was observed with increasing symptom-reporting discordance. Conclusion: Further studies should investigate how symptom-reporting discordance influences patient satisfaction and HRQoL.

Impact of next-generation hormonal agents on treatment patterns among patients with metastatic hormone-sensitive prostate cancer: a real-world study from the United States, five European countries and Japan.

BACKGROUND: Until five years ago, the metastatic hormone-sensitive prostate cancer (mHSPC) treatment landscape was dominated by the use of androgen deprivation therapy (ADT) alone. However, novel hormonal agents (NHAs) and chemotherapy are now approved for male patients with mHSPC. This study aimed to understand the impact NHA approvals had on mHSPC real-world treatment patterns and to identify the key factors associated with NHA or chemotherapy (± ADT) usage vs ADT alone. METHODS: Data were collected from the Adelphi Prostate Cancer Disease Specific Programme (DSP)™, a point-in-time survey of physicians and their consulting patients conducted in the United States (US), five European countries (France, Germany, Italy, Spain, and the United Kingdom), and Japan between January and August 2020. Data were analysed using descriptive statistics for individual countries, regions, and all countries combined. Pairwise analyses were used to further investigate differences between treatment groups at global level. RESULTS: 336 physicians provided data on 1195 mHSPC patients. Globally, at data collection, the most common mHSPC regimen initiated first was ADT alone (47%), followed by NHAs (± ADT) (31%, of which 21% was abiraterone, 8% was enzalutamide, and 2% was apalutamide) and chemotherapy (± ADT) (19%). The highest rates of ADT alone usage were observed in Japan (78%) and Italy (66%), and the lowest in Spain (34%) and in the US (36%). Our results showed that clinical decision making was driven by patient fitness, compliance, tolerance of adverse events, and balance of impact on quality of life vs overall survival. CONCLUSIONS: This real-world survey offered early insights into the evolving mHSPC treatment paradigm. It showed that in 2020, ADT alone remained the most common initial mHSPC therapy, suggesting that physicians may prefer using treatments which they are familiar and have experience with, despite clinical trial evidence of improved survival with NHAs or chemotherapy (± ADT) vs ADT alone. Results also indicated that physicians prescribed specific mHSPC treatments primarily based on the following criteria: patient preference, disease burden/severity, and the performance status and comorbidities of the patient. To fully appreciate the rapidly changing mHSPC treatment landscape and monitor NHA uptake, additional real-world studies are required.

Real-World Treatment Patterns in Metastatic Castration-Resistant Prostate Cancer Across Europe (France, Germany, Italy, Spain, and the United Kingdom) and Japan.

INTRODUCTION: Prostate cancer (PC) is the second most common cancer, and the fifth most common cause of cancer-related mortality among male patients, worldwide. In Europe and Japan, the incidence of PC in men in 2020 exceeded that of lung cancer. Although national and regional clinical guidelines for the treatment of metastatic castration-resistant prostate cancer (mCRPC) are available in Europe and Japan, a literature review did not identify a published comparison of differing guidelines, but identified a lack of studies reporting treatment patterns of approved mCRPC treatments in Europe and Japan in normal clinical practice. The objective of this real-world study was to compare national treatment guidelines and real-world treatment for mCRPC in Europe and Japan. METHODS: Physician-reported demographics, clinical characteristics, and treatment data of patients with mCRPC were drawn from the Adelphi Prostate Cancer Disease Specific Programme™, conducted in five European countries and Japan (2020) and analysed descriptively. RESULTS: All current treatment guidelines recommended the use of novel hormonal agents (NHA-abiraterone/enzalutamide) and chemotherapy (mainly docetaxel), with some intercountry differences, with NHA rechallenge accepted in Germany, Italy and Japan, but not in France, Spain or the United Kingdom. Overall, 271 physicians provided data for 1753 patients. At 1st-line (1L), the most common treatment was NHAs followed by (→) chemotherapy, in all countries. Chemotherapy was the most common 2nd-line (2L) treatment, except in Japan, where 2L NHA use was preferred, and Spain, where both were used equally. NHA â†’ chemotherapy and chemotherapy â†’ NHA were the first and second usual 1L â†’ 2L sequence in most countries, except for France, where the second most common sequence was NHA â†’ NHA, and Japan, with androgen deprivation therapy alone â†’ NHA. CONCLUSION: Real-world mCRPC treatment patterns largely reflected national guidelines. It is expected that guidelines and treatment patterns will change with the development of new treatment options.

Real-world homologous recombination repair mutation testing in metastatic castration-resistant prostate cancer in the USA, Europe and Japan.

Aim: To assess homologous recombination repair mutation (HRRm) testing patterns in metastatic castration-resistant prostate cancer. Methods: A point-in-time, international survey conducted January-August 2020. Results: Three-quarters of physicians (oncologists, urologists, specialist surgeons) globally reported access to genetic/genomic testing and just over half were HRRm testers. Surveyed physicians reported HRRm testing and positivity rates for 1913 patients, which were 18.1% and 33.7%, respectively. Of patients tested (n = 347), the most common HRR genes tested were BRCA (91.6%) and ATM (47.3%). Conclusion: Overall testing rates were low, with physicians mostly testing patients they considered higher risk. Increased awareness and education are needed to encourage broader testing, to understand familial risk and to identify patients with worse outcomes or those eligible for life-prolonging treatments.

Metastatic castration-resistant prostate cancer (mCRPC) is cancer that has spread beyond the prostate gland and that no longer responds to hormone therapy. Genetic testing is now recommended for patients with mCRPC to help doctors understand familial risk and identify patients who may benefit from new treatments. The authors asked doctors questions about their patients, such as their age, symptoms and genetic testing. The authors found that overall genetic testing rates were low, with doctors mostly testing patients they thought were at higher risk of developing cancer. Increased doctor awareness and education are needed to encourage more genetic testing in mCRPC patients. However, doctors claimed that the cost of these tests was a challenge to conducting genetic testing.

Validity of a single-item indicator of treatment side effect bother in a diverse sample of cancer patients.

PURPOSE: With higher efficacy of cancer therapies, the numbers and types of side effects experienced by patients have also increased, evidencing a need for brief assessments of side effect bother. The Functional Assessment of Cancer Therapy-General (FACT-G) includes the item "I am bothered by side effects of treatment" (GP5). This study aimed to confirm GP5's validity in a large, diverse, real-world patient sample. METHODS: Real-world data were drawn from 10 Adelphi Disease Specific Programmes (DSP™) conducted between 2015 and 2019 in France, Germany, Italy, Spain, the UK and the USA, covering 10 cancer sites. We examined correlations between GP5 responses and varied measures of patient-reported global health and the number of side effects experienced. We explored whether more advanced patients and those with worse Eastern Cooperative Oncology Group Performance Status Rating (ECOG PSR) reported greater side effect bother. Finally, we conducted differential item functioning (DIF) assessment using the Mantel-Haenszel approach. RESULTS: The sample included 6755 advanced cancer patients. GP5 responses were distributed similarly across most cancer sites. A moderate, negative correlation (rpolyserial = - 0.43) between GP5 responses and global health evidenced convergent validity. Known groups validity was evidenced by dichotomised distributions of GP5, showing expected results between cancer stage 2 vs. 3 and 4 and with ECOG PSR (p < 0.001). Little evidence of DIF was found. CONCLUSION: GP5 exhibited evidence of validity across cancer sites and countries and appeared to measure the same construct across these countries. GP5 has significant promise as a summary indicator of side effect bother.

Real-world symptoms, disease burden, resource use and quality of life in US patients with advanced renal cell cancer.

Aim: To assess symptoms, healthcare resource utilization and health-related quality of life in advanced renal cell carcinoma (aRCC) clinical practice. Materials & methods: The USA point-in-time survey of physicians and patients was conducted between February and September 2019. Results: Data were available for 227 patients. Mean (standard deviation) number of symptoms was 3.4 (3.2); differences were observed across International Metastatic RCC Database Consortium risk categories (p < 0.001), with fewer symptoms in favorable-risk patients. Disease burden, measured by greater healthcare resource utilization and worse health-related quality of life, was high, particularly in International Metastatic RCC Database Consortium intermediate- or poor- versus favorable-risk patients. In total, 45 patients (21.6%) were hospitalized due to aRCC within a 6-month period, 35 (16.8%) had one hospitalization and ten (4.8%) experienced ≥2 hospitalizations due to aRCC. Mean (standard deviation) 19-Item Functional Assessment of Cancer Therapy Kidney Symptom Index score was 53.6 (13.2) for this population, significantly lower than the reference value (59.8; p < 0.001). Conclusion: A clear need exists for improved disease management in patients with aRCC.

Lay abstract Late-stage/advanced renal cell carcinoma (aRCC) is kidney cancer that has spread to other body parts. aRCC is expensive to treat and affects patients in many ways. New treatments have become available, including tyrosine kinase inhibitors and immuno-oncology therapies. The type of treatment recommended depends on the patient's International Metastatic RCC Database Consortium risk score. This is a way of classifying patients as having a good, intermediate or poor survival risk. We asked physicians questions about their patients such as their age, how long they had aRCC, their treatment and symptoms, and asked patients how aRCC affected their lives, including how often they visited doctors and hospitals. aRCC had the greatest effect on patients with poor-risk scores. Those patients had more symptoms and worse quality of life than patients with intermediate or good risk scores. Treatment also affected patients' lives, although not as much as risk score. Patients with aRCC need better treatment options to help improve their quality of life.

Treatment patterns, outcomes and clinical characteristics in advanced renal cell carcinoma: a real-world US study.

Aim: Assessing treatment patterns, outcomes and clinical characteristics in advanced renal cell carcinoma clinical practice. Materials & methods: A US cross-sectional physician survey conducted February-September 2019. Results: Surveyed physicians reported first-line treatment of 445 patients involving tyrosine kinase inhibitor monotherapy (51.0%), immuno-oncology (IO/IO combination) therapy (25.8%) or other regimens (23.1%). A total of 60.9% had physician-assessed IMDC risk. Of these 61.9, 50.9 and 27.6% of patients with favorable, intermediate and poor risk, respectively, received tyrosine kinase inhibitor monotherapy. A total of 16.7, 26.9 and 34.5% of patients with favorable, intermediate or poor risk received IO/IO combination therapy. Complete/partial responses (∼35% patients) remained comparable across first-line treatments. Conclusion: Guideline-recommended therapies are not widely prescribed. Many patients experienced poor clinical outcomes highlighting a need for more effective treatments.

Patient Characteristics and Real-World Treatment Patterns Among Early Users of PCSK9 Inhibitors.

BACKGROUND: Proprotein convertase subtilisin/kexin type 9 inhibitors (PCSK9i) reduce low-density lipoprotein cholesterol (LDL-C) levels and are approved for patients with familial hypercholesterolemia or atherosclerotic cardiovascular disease who require additional LDL-C lowering. OBJECTIVE: Our objective was to characterize patients receiving PCSK9i medications in real-world practice and describe physician-reported treatment patterns among dyslipidemia patients using PCSK9i or other lipid-lowering therapy. METHODS: We analyzed data from a point-in-time Adelphi dyslipidemia disease-specific programme (DSP) survey conducted in the USA in 2016. Physicians provided treatment history, laboratory values, patient characteristics, and comorbidities for treated patients. To ensure sufficient numbers of PCSK9i-treated patients, we conducted systematic oversampling of patients being prescribed PCSK9i. Outcomes included patient characteristics and physician-reported treatment patterns. RESULTS: The DSP included 159 physicians, who provided information on 1522 patients (304 PCSK9i; 1218 non-PCSK9i). Mean ± standard deviation (SD) baseline LDL-C levels were 180.0 ± 39.7 mg/dl for PCSK9i patients and 159.2 ± 40.5 mg/dl for non-PCSK9i patients. Prior statin use was reported in 69.1% of PCSK9i patients and 19.5% of non-PCSK9i patients, and physician-reported statin intolerance was observed in 31.6% of PCSK9i and 5.3% of non-PCSK9i patients. Use of statins only was reported in 40.5% of PCSK9i and 88.8% of non-PCSK9i patients. The most common physician-reported reasons for change to PCSK9i were lack of efficacy (70.2%) and muscle-related symptoms (myalgia 28.6%; myopathy 11.1%). CONCLUSIONS: Physicians surveyed appeared to prescribe PCSK9i medications appropriately. PCSK9i-treated patients had higher rates of cardiovascular comorbidities and physician-determined statin intolerance, had higher LDL-C levels, and received more lines of therapy than non-PCSK9i patients.

Reasons for discontinuation of GLP1 receptor agonists: data from a real-world cross-sectional survey of physicians and their patients with type 2 diabetes.

AIM: Nonadherence to glucagon-like peptide-1 receptor agonists (GLP1 RAs) is relatively common among patients with type 2 diabetes mellitus (T2DM). This study sought to identify reasons why patients discontinue GLP1 RAs. MATERIALS AND METHODS: Retrospective data from the Adelphi Diabetes Disease Specific Programme were used. Physicians managing patients with T2DM were surveyed via face-to-face interviews, and patients treated for T2DM were surveyed via self-completed questionnaires. Patient data were stratified by current versus prior GLP1 RA use. RESULTS: Physicians (n=443) most frequently reported inadequate blood glucose control (45.6%), nausea/vomiting (43.8%), and gastrointestinal (GI) side effects (36.8%) as reasons for GLP1 RA discontinuation. Patients (n=194) reported the GI-related issues "Made me feel sick" (64.4%) and "Made me throw up" (45.4%) as their top reasons for discontinuation. The most common problems reported (excluding cost) for those currently using GLP1 RAs were "Prefer oral medication over injections" (patients 56%, physicians 32.6%), "Made me feel sick" (patients 38.1%, physicians 16.3%), and "Did not help lose weight" (patients 25.4%, physicians 18%). The most bothersome problems for patients globally (frequency reporting very/extremely bothersome) (excluding cost) were "Difficult to plan meals around" (55.6%), "Made me throw up" (51.6%), and "Caused weight gain" (50%). CONCLUSION: Both patients and physicians reported GI-related issues as a prominent factor, but disparities between patient experiences and physician perceptions were revealed, suggesting gaps in physician-patient communication. Understanding patients' expectations of GLP1 RAs and physicians' patient-management practices may help increase GLP1 RA adherence and thereby potentially enhance diabetes care.

Trends in medication use in patients with type 2 diabetes mellitus: a long-term view of real-world treatment between 2000 and 2015.

BACKGROUND: Despite the availability of a variety of treatments, many patients with type 2 diabetes mellitus (T2DM) are not achieving glucose control. We analyzed successive waves of the Adelphi Real World Diabetes Disease Specific Programmes (DSPs) to assess treatment patterns reported by primary care physicians (PCPs) and specialists and the effect of treatment on levels of glucose control. METHODS: Data were collected between 2000 and 2015 in the US and EU5 (France, Germany, Italy, Spain, and the UK). Physicians completed patient record forms for the next 10 patients consulting with T2DM. Key aspects captured were change over time in therapy usage, time to insulin introduction, and glycated hemoglobin (HbA1c) levels. RESULTS: Over 12 DSP waves, 3,555 specialists and 5,109 PCPs completed questionnaires for 70,657 patients. Treatment patterns changed considerably over time as new agents were introduced. The number of agents prescribed per patient increased over time, as did HbA1c levels at which physicians stated they would introduce insulin. The greatest improvements in HbA1c levels occurred during 2000-2008, with little improvement since 2008. CONCLUSION: In this real-world setting, the proportion of patients with T2DM achieving good glucose control has not increased greatly since 2008. A better understanding of how to individualize treatment pathways may be required to improve control in these patients.