Real-world treatment patterns, healthcare resource use and disease burden in patients with multiple myeloma in Europe.

Aim: To investigate treatment patterns, healthcare resource utilization and disease burden in patients with multiple myeloma (MM). Methods: Point-in-time survey of physicians and their patients presenting in a real-world clinical setting, collected across Europe between May and November 2021. Results: In total, 173 physicians provided data for 2179 patients with MM. Treatments received became more diverse as line of therapy increased, dictated by previous treatment choices. Overall, 25% of all patients were tri-exposed, and experienced a higher degree of healthcare resource utilization, disease burden and impairment than non-tri-exposed patients. Conclusion: The treatment landscape in MM is complex and evolving. There is an unmet need for more effective therapies to reduce disease burden, particularly in tri-exposed patients.

There are many new treatments available for patients with multiple myeloma. While outcomes such as survival, symptoms and health problems experienced have improved, patients still continue to relapse and fall ill again. This means their current treatment stops working and they have to change to a new treatment to prevent their disease from developing further. Patients who have received three different types of treatment are classed as being 'tri-exposed', and they experience greater problems with their health. To better understand this course of events, we used information from a survey of doctors and their patients with multiple myeloma across Europe in 2021. We looked at patient's symptoms, the treatments they received, how and when they accessed healthcare (including hospital visits and tests) and the overall difficulties experienced due to their illness. We found that patients were broadly treated according to the most recent European guidelines, although differences were seen between countries. When patients had to switch therapy, the type of treatment received next depended on what they had previously been prescribed, meaning that treatment choices became increasingly complicated. Overall, 25% of patients in our study were classed as tri-exposed, and had more hospitalisations, required more hospital tests, had greater health problems and experienced more difficulties at work than those who were not tri-exposed. Despite recent developments in the treatment of multiple myeloma, there is still a need for more effective therapies. This is especially true for patients who are tri-exposed, who have limited treatment options and experienced greater health problems.

Drivers of physician decision-making and patient perspectives across lines of therapy in multiple myeloma in the USA.

Aim: To explore treatment selection for relapsed/refractory multiple myeloma (RRMM), which remains complex due to heterogeneity of available treatments and lack of defined standard of care. Patients & methods: The Adelphi Real World MM Disease Specific Programme surveyed physicians in the USA and their patients with MM to collect real-world data on patterns and perceptions of MM treatment across lines of therapy (LOT). Results: Triplets were the most common regimens across each LOT. Physicians reported efficacy-related factors, health insurance coverage, and clinical guidelines as key determinants of treatment choice regardless of LOT. Patients identified better quality of life as the most important treatment benefit. Conclusion: The DSP RW data highlight drivers of RRMM treatment choice from physicians' and patients' perspectives and need for a more holistic approach to guidelines and clinical trials that encompasses patient perspectives.

Multiple myeloma (MM) is an incurable cancer of white blood cells. Treatments tend to become less effective when taken for long periods of time, meaning that patients often receive several different treatments over the course of their disease. Many different drugs and drug combinations are now available for patients with MM. As there is no standardized approach for the treatment of MM, it can be challenging for physicians to choose between the various complex treatment options for their patients, and the key factors that influence physicians' treatment choices remain unclear. In this study, we used information from a survey of physicians and their patients with MM in the USA to investigate which treatments were used most commonly and how physicians and patients made decisions about their treatment. We found that patients with MM were typically treated with a combination of three drugs which differed between patients. When deciding which treatment to prescribe to their patients, physicians primarily considered factors related to how well a treatment works and, in-turn, prolong a patient's life, but this was also considered by physicians to be an area for improvement. While patients also favored treatments associated with a longer life, they more commonly favored those associated with a better quality of life. These findings show that quality of life is important to patients receiving treatment for MM and should be taken into account by physicians when choosing a treatment for their patients.

Real-world patient-reported outcomes and concordance between patient and physician reporting of side effects across lines of therapy in multiple myeloma within the USA.

PURPOSE: We aimed to explore patient-reported outcomes (PROs) and patient and physician concordance of side effects perception across lines of therapy (LOT) in multiple myeloma (MM) within the United States of America (USA). METHODS: Data were drawn from the Adelphi Real World MM III Disease Specific Programme™, a point-in-time survey of hemato-oncologists/hematologists and their patients with MM conducted in the USA between August 2020 and July 2021. Physicians reported patient characteristics and side effects. Patients reported side-effect bother and health-related quality of life (HRQoL) using validated PRO tools (European Organisation for the Research and Treatment of Cancer Quality of Life Core Questionnaire/-MM Module [EORTC QLQ-C30/-MY20], EQ-5D-3L and Functional Assessment of Cancer Therapy-General Population physical item 5). Descriptive, linear regression and concordance analyses were performed. RESULTS: Records from 63 physicians and 132 patients with MM were analyzed. EORTC QLQ-C30/-MY20 and EQ-5D-3L scores were consistent across LOTs. Scores tended to be worse with higher side-effect bother; patients "very much" bothered by side effects had lower median (interquartile range) global health status scores (33.3 [25.0-50.0]) than those "not at all" bothered (79.2 [66.7-83.3]). Patient and physician concordance on side-effect reporting was poor to fair. Patients frequently reported fatigue and nausea as bothersome side effects. CONCLUSION: HRQoL of patients with MM was worse with greater side-effect bother. Discordant patient and physician reporting of side effects indicated a need for improved communication during management of MM.

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.

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 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.

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 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.

Qualitative and Quantitative Assessment of Patient and Carer Experience of Chemotherapy (Docetaxel) in Combination with Androgen Deprivation Therapy (ADT) for the Treatment of Metastatic Hormone-Sensitive Prostate Cancer (mHSPC).

INTRODUCTION: Recent studies suggest that docetaxel plus androgen deprivation therapy can prolong survival among men with metastatic hormone-sensitive prostate cancer (mHSPC). However, as a cytotoxic therapy, there is a need to understand the experiences of men with mHSPC receiving docetaxel and their carers in a real-world setting. METHODS: During phase 1, semi-structured qualitative interviews were conducted with men with mHSPC (n = 31) and their carers (n = 14) in Europe to elicit in-depth data concerning their experiences with docetaxel. Eighteen men were also asked to record their experiences in a diary for 7 days. During phase 2, men with mHSPC (n = 161) and carers of men with mHSPC (n = 135) completed an online survey comprising self-report questionnaires including the Cancer Therapy Satisfaction Questionnaire, Brief Fatigue Inventory, Functional Assessment of Cancer Therapy-Prostate, EuroQol-5-Dimensions and the Burden Scale for Family Caregivers (carers only). RESULTS: At the outset of therapy, men reported a willingness to take docetaxel to prolong their life, despite being fearful of the potential side effects and impacts on their daily lives. Patient and carer experiences were generally consistent with pre-treatment expectations. However, variations in individual experiences and their ability to tolerate side effects were evident. Fatigue emerged as a prominent symptom with the majority (n = 98, 60.9%) of men reporting experiencing moderate-severe fatigue in the past 24 h. Participant ratings of fatigue were strongly correlated with health-related quality of life (r = - 0.82). Nausea, diarrhoea and sore mouth were also among the most bothersome symptoms for participants. CONCLUSIONS: Findings from this study highlight that real-world experience of docetaxel may differ from that observed in clinical trials and that care must be taken to ensure that treatment options are tailored to the needs of individual patients to promote not only how long patients survive but also the quality of that survival. FUNDING: Janssen.