Drugs - Real World Outcomes最新文献

{"title":"Determinants of the Financial Impact of Orphan Drugs in Italy: Differences Between Expected and Observed Pharmaceutical Expenditure.","authors":"Francesco Saverio Mennini, Americo Cicchetti, Paolo Sciattella, Filippo Rumi, Matteo Zanuzzi, Angelica Carletto, Annalisa Sammarco, Federica Romano, Pierluigi Russo","doi":"10.1007/s40801-024-00463-w","DOIUrl":"10.1007/s40801-024-00463-w","url":null,"abstract":"<p><strong>Background: </strong>The increasing demand for orphan drugs and the financial challenges associated with their reimbursement highlights the need to understand the dynamics between expected and actual pharmaceutical expenditures, particularly in the context of pricing and reimbursement negotiations.</p><p><strong>Objective: </strong>The study aims to identify the potential determinants of the difference in expected pharmaceutical spending after negotiation and the actual expenditure incurred (ΔS), as well as the difference in expected pharmaceutical spending before and after the price and reimbursement negotiation process (ΔSn) for rare disease drugs in Italy.</p><p><strong>Methods: </strong>The analysis focused on orphan drugs authorised by European Medicines Agency, with reimbursement applications to the Italian Medicines Agency from January 2013 to January 2019. Expected post-negotiation spending was estimated by applying negotiated discounts and financial-based market entry agreements to the expected pharmaceutical expenditure. The actual expenditure was approximated through company turnovers. Beta regression models were applied to identify potential determinants of ΔS and ΔSn.</p><p><strong>Results: </strong>The study analysed 52 reimbursed orphan drugs, with 41 (78.8%) with a single indication, 25 (48.1%) antineoplastics and immunomodulators and 18 (34.6%) conditionally/fully innovative. The median expenditure in the first 3 years post-commercialisation was 7.6% lower than expected post-negotiation. The reduction was less pronounced for innovative drugs (p = 0.011), drugs with a prices and reimbursement procedure in the subsequent 3 years (p = 0.007) and those with multiple indications (p = 0.021). Payment-by-result was the only significant variable associated with the expected spending ratio before and after negotiation (p = 0.002).</p><p><strong>Conclusion: </strong>The actual expenditure for orphan drugs aligns with the expected post-negotiation. Yet, innovative orphan drugs exhibit a higher actual expenditure than estimated, suggesting the market values their added therapeutic value or the actual therapeutic need they meet, and configuring innovativeness status as the main determinant of the orphan drugs financial impact in the multiple regression analysis.</p>","PeriodicalId":11282,"journal":{"name":"Drugs - Real World Outcomes","volume":" ","pages":"25-33"},"PeriodicalIF":1.9,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11829855/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142791362","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Trends in Reports of Statin-Associated Immune-Mediated Necrotizing Myopathy Using the Japanese Adverse Drug Event Report Database.","authors":"Akina Takami, Gen Terashima, Takumi Tajima, Koki Yamashita, Ataru Igarashi","doi":"10.1007/s40801-025-00481-2","DOIUrl":"10.1007/s40801-025-00481-2","url":null,"abstract":"<p><strong>Background: </strong>In October 2016, precautions of the package inserts for ethical drugs containing statins were revised for immune-mediated necrotizing myopathy (IMNM).</p><p><strong>Objective: </strong>Our objective was to assess the trend in statin-associated IMNM reported before and after the release of the revised precautions in Japan.</p><p><strong>Methods: </strong>We investigated the number of case reports and estimated annual incidence rate of statin-associated IMNM using the Japanese Adverse Drug Event Report and Japan Medical Data Center (JMDC) databases, respectively. To identify IMNM case reports, we used the preferred term \"immune-mediated myositis\" (MedDRA version 27.1).</p><p><strong>Results: </strong>We identified 172 statin-associated IMNMs in 145 case reports of patients between 1 April 2004 and 31 March 2023. The most common suspected statin administered to the patients was rosuvastatin (34.3%), followed by pitavastatin (25.0%) and atorvastatin (22.1%). No statin-associated IMNM was reported in patients who were treated with combination agents containing statins. The number of reported statin-associated IMNMs increased from 3 in 2015 to a peak of 51 in 2019, after which it was 22 (2020), 17 (2021), and 21 (2022) in the following years. The estimated annual incidence rate did not differ with statins, it rarely exceeded 5 per 1,000,000 patients.</p><p><strong>Conclusions: </strong>There was an increasing trend in the number of statin-associated IMNM after the revised precautions of package inserts for statins were released.</p>","PeriodicalId":11282,"journal":{"name":"Drugs - Real World Outcomes","volume":" ","pages":"145-152"},"PeriodicalIF":1.9,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11829879/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143074332","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Medication Errors and Error Chains Involving High-Alert Medications in a Paediatric Hospital Setting: A Qualitative Analysis of Self-Reported Medication Safety Incidents.","authors":"Sini Kuitunen, Mari Saksa, Anna-Riia Holmström","doi":"10.1007/s40801-024-00469-4","DOIUrl":"10.1007/s40801-024-00469-4","url":null,"abstract":"<p><strong>Background: </strong>Paediatric patients are prone to medication errors, but an in-depth understanding of errors involving high-alert medications remains limited.</p><p><strong>Objective: </strong>We aimed to investigate incident reports involving high-alert medications to describe medication errors, error chains and stages of the medication management and use process where the errors occur in paediatric hospitals.</p><p><strong>Methods: </strong>A retrospective document analysis of self-reported medication safety incidents in a paediatric university hospital in 2018-20. The incident reports involving high-alert medications were investigated using an inductive qualitative content analysis and quantified (frequencies and percentages). A systems approach to medication risk management based on the Theory of Human Error was applied.</p><p><strong>Results: </strong>Altogether, 560 medication errors were identified within the study sample (n = 426 incident reports). Most medication errors were associated with administration (43.1 %, n = 241/560) and prescribing (25.2 %, n = 141/560). Error chains involving two to four medication errors in one or more stages of the medication management and use process were present in 26.1% (n = 111/426) of reports, most of which originated from prescribing (62.2%; n = 69/111). The medication errors (n = 560) were classified into 14 main categories, the most common of which were wrong dose (13.9%; n = 78/560), omission of a drug (12.9%; n = 72/560) and documentation errors (10.0%; n = 56).</p><p><strong>Conclusions: </strong>Paediatric medication error chains often start from prescribing and pass through the medication management and use process. Systemic defences are especially needed for manual tasks leading to wrong doses, drug omission and documentation errors. Intravenous medications and chemotherapeutic agents, optimising drug formularies and handling, and high-alert drug use at home require further actions in paediatric medication risk management.</p>","PeriodicalId":11282,"journal":{"name":"Drugs - Real World Outcomes","volume":" ","pages":"45-61"},"PeriodicalIF":1.9,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11829867/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142806011","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Mental Health-Related Disability Days and Costs Among Patients with Treatment-Resistant Depression Initiated on Esketamine Nasal Spray and Conventional Therapies in the USA.","authors":"Manish K Jha, Maryia Zhdanava, Aditi Shah, Arthur Voegel, Anabelle Tardif-Samson, Dominic Pilon, Kruti Joshi","doi":"10.1007/s40801-024-00478-3","DOIUrl":"10.1007/s40801-024-00478-3","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Introduction: &lt;/strong&gt;Treatment-resistant depression (TRD) is related to disproportionate unemployment and productivity burden in the USA. The current study describes real-world mental health (MH)-related disability days and costs of patients with TRD initiated on esketamine nasal spray or conventional therapies in the USA.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;Adults with TRD were selected from Merative™ MarketScan&lt;sup&gt;®&lt;/sup&gt; Commercial database (from January 2016 to January 2023) and classified into four cohorts (esketamine, ECT [electroconvulsive therapy], TMS [transcranial magnetic stimulation], and SGA [second-generation antipsychotics] augmentation) based on therapy initiated (index date) on/after 5 March 2019 (esketamine approval date for TRD). Patients had ≥ 12 months of health plan eligibility pre-index date and disability information available pre- and post-index in the Merative™ MarketScan&lt;sup&gt;®&lt;/sup&gt; Health and Productivity Management database (from January 2016 to December 2021). MH-related disability days (i.e., short- or long-term) and associated costs (US dollars [USD] 2022) were reported per-patient-per-month for the 6 months pre- and post-index.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;The study comprised four cohorts: esketamine (n = 107; mean age: 45.5 years, female: 54.2%), ECT (n = 55; mean age: 47.6 years, female: 41.8%), TMS (n = 443; mean age: 46.1 years, female: 57.3%), and SGA (n = 4374; mean age: 44.8 years, female: 59.1%). In month 6 pre-index, mean number of MH-related disability days was 1.7 in the esketamine cohort, 1.2 in the TMS cohort, 1.3 in the ECT cohort, and 0.8 in the SGA augmentation cohort; mean MH-related disability costs were US $443 in the esketamine cohort, US $339 in the TMS cohort, US $178 in the ECT cohort, and US $143 in the SGA augmentation cohort. In all cohorts, a peak in mean MH-related disability days and costs was observed 1 month after therapy initiation followed by a decreasing trend. In month 6 post-index versus month 6 pre-index, the mean number of MH-related disability days trended lower in the esketamine cohort (- 0.4 days), remained the same in the TMS cohort and largely the same in the SGA augmentation cohort (+ 0.1 days), and trended higher (+ 1.6 days) in the ECT cohort. In the same timeframe, MH-related disability costs trended lower in the esketamine and TMS cohorts, with observed reductions of US $312 and US $123, respectively. Costs remained largely the same in the SGA augmentation cohort (+ US $26), and trended higher (+ US $353) in the ECT cohort.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusions: &lt;/strong&gt;In this descriptive study, initiation of esketamine was associated with trends toward lower MH-related disability days and costs. Conventional therapies demonstrated varied patterns, with no consistent trend toward reductions in disability days across all therapies and no observed cost-savings trends for SGA augmentation and ECT. These trends suggest potential economic and societal gains of esketami","PeriodicalId":11282,"journal":{"name":"Drugs - Real World Outcomes","volume":" ","pages":"35-43"},"PeriodicalIF":1.9,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11829853/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143001928","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Group-Based Trajectory Models to Evaluate the Association of Lipid Testing and Statin Adherence.","authors":"Yun-Yi Pan, Sandeep Devabhakthuni, Catherine E Cooke, Julia F Slejko","doi":"10.1007/s40801-024-00472-9","DOIUrl":"10.1007/s40801-024-00472-9","url":null,"abstract":"<p><strong>Background and objective: </strong>Performing lipid testing after statin initiation is recommended to monitor response. Inadequate response may indicate non-adherence, which is associated with an increased risk of cardiovascular events and increased costs. Group-based trajectory modeling is an approach to establish probabilistic developmental trajectories of adherence, differentiating individuals by their distinct longitudinal medication-taking behaviors. We examined whether lipid testing is associated with distinct trajectories of statin adherence among individuals enrolled in a Medicare fee-for-service plan in the USA.</p><p><strong>Methods: </strong>A retrospective cohort study was conducted using the Centers for Medicare & Medicaid Chronic Condition Warehouse 5% sample of Medicare fee-for-service data between 2006 and 2015. Statin use and lipid testing were identified using claims data. The proportion of days covered was calculated for each 30 days after the index date, which was used to estimate the probability of belonging to each potential adherence trajectory.</p><p><strong>Results: </strong>In a cohort of 138,101 statin initiators, four statin adherence trajectory groups were identified. The four groups were differentiated as \"rapid decline\" (21.53%), \"gradual decline\" (10.25%), \"decline first then improve later\" (26.47%), and \"high adherence\" (41.75%). Compared with \"high adherence,\" initiators who had lipid tests within 360 days after statin initiation were less likely to fall into \"rapid decline\" (adjusted odds ratio: 0.661; 95% confidence interval 0.641-0.683), \"gradual decline\" (adjusted odds ratio: 0.834; 95% confidence interval 0.801-0.868), and \"decline first then improve later\" groups (adjusted odds ratio: 0.936; 95% confidence interval 0.910-0.962).</p><p><strong>Conclusions: </strong>Lipid testing is positively associated with greater use of statin medication across different adherence trajectories in the present study.</p>","PeriodicalId":11282,"journal":{"name":"Drugs - Real World Outcomes","volume":" ","pages":"75-81"},"PeriodicalIF":1.9,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11829858/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142871664","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Effects of the Japanese Kampo Medicines Rikkunshito, Shakuyakukanzoto and Goreisan on Lenvatinib Plasma Concentrations in Japanese Patients with Thyroid Cancer.","authors":"Kazuma Fujita, Akifumi Suzuki, Mitsuji Nagahama, Kiminori Sugino, Chie Masaki, Koichi Ito, Masatomo Miura","doi":"10.1007/s40801-024-00467-6","DOIUrl":"10.1007/s40801-024-00467-6","url":null,"abstract":"<p><strong>Background: </strong>Kampo medicines are often used in Japan as therapy for the side effects induced by oral kinase inhibitors. However, the pharmacokinetic interactions between Kampo medicines and oral kinase inhibitors such as lenvatinib have not been studied.</p><p><strong>Objective: </strong>We investigated the effects of Kampo medicines (rikkunshito, shakuyakukanzoto and goreisan) on the steady-state plasma trough concentration (C₀) of lenvatinib in patients with thyroid cancer.</p><p><strong>Methods: </strong>Thirty-nine patients receiving lenvatinib therapy at Ito Hospital between May 2015 and December 2019 were enrolled. The mean C₀ of lenvatinib with Kampo medicine, at the same dose as before initiating Kampo medicines, was used.</p><p><strong>Results: </strong>After the repeated administration of rikkunshito (n = 21), shakuyakukanzoto (n = 10) or goreisan (n = 8), the mean C₀ of lenvatinib and the laboratory test values of patients did not change significantly. In contrast to rikkunshito, which alleviates emesis by enhancing gastric emptying, the C₀ values of lenvatinib with a proton pump inhibitor (PPI) (n = 16) or histamine H₂ receptor antagonist (H2RA) (n = 4) were significantly lower than the C₀ values without a PPI or H2RA (P = 0.007). The mean (range) change rate of the C₀ of lenvatinib with a PPI or H2RA versus without a PPI or H2RA was 88.6% (69.9-115%), and was significantly greater than the change rate for rikkunshito (P = 0.029). There was no significant difference between the C₀ of lenvatinib with a prokinetic agent (n = 7) versus without a prokinetic agent (P = 0.365).</p><p><strong>Conclusions: </strong>Although these Kampo medicines are reported to inhibit drug-metabolizing enzymes and drug transporters, the risk of drug interactions for patients receiving lenvatinib therapy is low. Patients should feel confident that they can receive Kampo medicines as supportive care for lenvatinib therapy without a risk of drug interactions that could affect treatment efficacy.</p>","PeriodicalId":11282,"journal":{"name":"Drugs - Real World Outcomes","volume":" ","pages":"153-160"},"PeriodicalIF":1.9,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11829860/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142767309","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Lipid-Lowering Efficiency and Safety of Alirocumab 300 mg Using a 2-mL Autoinjector Device in Real-World Practice: The MARS Study.","authors":"Klaus G Parhofer, Peter Bramlage, Constanze Gries, Cornelia Harder, Christiane Look, W Dieter Paar, Ursula Rauch-Kröhnert","doi":"10.1007/s40801-024-00471-w","DOIUrl":"10.1007/s40801-024-00471-w","url":null,"abstract":"<p><strong>Background: </strong>Alirocumab is a fully human monoclonal antibody to proprotein convertase subtilisin kexin type 9 used for the reduction of low-density lipoprotein cholesterol (LDL-C) in high-risk patients not reaching their LDL-C target. Recently, a 2-mL prefilled autoinjector has been developed to support the monthly 300-mg dosing regimen with a single-injection administration.</p><p><strong>Methods and objectives: </strong>Monthly application of 300 mg AlirRocumab (Praluent^®) using the 2-mL SYDNEY Device (MARS) is a non-interventional, open, prospective, multi-center cohort study conducted in Germany between 2021 and 2023 with an observational period of 12 weeks. Patients included had primary hypercholesterolemia (heterozygous familial or non-familial) or mixed dyslipidemia and confirmed vascular disease and other risk factors or confirmed familial heterozygous hypercholesterolemia. Primary objectives were to assess the effectiveness of the 2-mL SYDNEY autoinjector measured by the lipid-lowering effect of alirocumab and to document therapy satisfaction, patient adherence, and persistence. Secondary objectives were to assess safety (adverse events) and tolerability.</p><p><strong>Results: </strong>A total of 146 patients were analyzed: 110 (75.3%) patients were proprotein convertase subtilisin kexin type 9 inhibitor naïve and 36 (24.7%) were pre-treated with a proprotein convertase subtilisin kexin type 9 inhibitor. Patient mean age was 65.6 years with a preponderance of male gender (59.6%). At 12 weeks, the LDL-C value had decreased by a median of 59.5 mg/dL (1.5 mmol/L) in naïve patients (median relative decrease: - 52.0%). In the pre-treated group, the LDL-C value remained mainly unchanged (median slight numerical relative increase: 1.6%). Treatment satisfaction was rated similarly in both groups with most patients being satisfied/very satisfied and rating the injection as effective, safe, and easy to handle. Twenty-three adverse events in 13 patients (8.0%) were documented. Three patients experienced one serious adverse event each; for five patients, an adverse drug reaction was observed, although none was serious. The occurrence of adverse events was similar in both groups.</p><p><strong>Conclusions: </strong>Alirocumab 300 mg administered with the 2-mL SYDNEY autoinjector was safe and effective in lowering LDL-C after 12 weeks in a routine clinical setting in Germany. The treatment schedule was perceived to be beneficial with excellent device acceptance and satisfaction, potentially increasing patient adherence.</p><p><strong>Clinical trial registration: </strong>Clinicaltrials.gov: NCT05129241.</p>","PeriodicalId":11282,"journal":{"name":"Drugs - Real World Outcomes","volume":" ","pages":"63-74"},"PeriodicalIF":1.9,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11829868/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143058386","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Medication Hazards and Outcome Patterns of Pediatric Drug-Associated Liver Injury in Taiwan: An Analysis of 1998-2017 Spontaneous Adverse Drug Reaction Reports.","authors":"Yu-Ting Huang, Yen-Ming Huang, Ni-Chung Lee, Ping-Ing Lee, Yunn-Fang Ho","doi":"10.1007/s40801-024-00475-6","DOIUrl":"10.1007/s40801-024-00475-6","url":null,"abstract":"<p><strong>Background and objectives: </strong>Accumulating pediatric efficacy and safety data on drug use is inherently challenging yet essential. This study aimed to analyze the frequency and compute the odds of pediatric drug-associated liver injury across age groups (early childhood, middle childhood, and adolescence) and therapeutic categories using adverse drug reactions (ADRs) reporting data spanning nearly two decades.</p><p><strong>Methods: </strong>We analyzed the reports of suspected ADRs occurring in children and adolescents in the Taiwan National Adverse Drug Reaction Reporting System during the period from May 1998 until July 2017. Standardized Medical Dictionary for Regulatory Activities Queries were utilized to identify suspected hepatic ADRs. Outcome patterns across age groups were compared using the chi-squared test, and disproportionality analysis was employed to calculate reporting odds ratios (RORs) of hepatic versus nonhepatic reports.</p><p><strong>Results: </strong>Among 16,673 reports, 484 (2.9%) were identified as suspected hepatic ADRs, involving 193 distinct drugs. The mean age of affected individuals was 8.2 years. Outcome types in adolescents were predominantly serious (91.8%). Antibacterials for systemic use (18.8%) and antiepileptics (8.7%) were the most frequently implicated therapeutic categories. Drugs with high ADR occurrence rates and significant RORs included oxacillin (5.2%; ROR: 12.07), methotrexate (4.1%; ROR: 9.07), and phenobarbital (2.7%; ROR: 5.04). Some medications exhibited higher ratios of used-versus-recommended doses, suggesting inappropriate dosing.</p><p><strong>Conclusions: </strong>Pediatric drug-associated liver injury was not uncommon and may result in serious outcomes. This study underscores the need for heightened vigilance in administering certain high-risk drugs and attentiveness in proper dosing for children, including adolescents.</p>","PeriodicalId":11282,"journal":{"name":"Drugs - Real World Outcomes","volume":" ","pages":"105-114"},"PeriodicalIF":1.9,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11829874/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142946408","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Comparative Efficacy of Lenvatinib Plus Immunotherapy and Regorafenib Plus Immunotherapy After Lenvatinib Failure for Advanced Hepatocellular Carcinoma: A Retrospective Study.","authors":"Zeyu Yu, Bin Leng, Ran You, Lingfeng Diao, Qingyu Xu, Guowen Yin","doi":"10.1007/s40801-024-00480-9","DOIUrl":"10.1007/s40801-024-00480-9","url":null,"abstract":"<p><strong>Background: </strong>The combination of regorafenib and immune checkpoint inhibitor (ICI) has been the most popular second-line systemic therapy for advanced hepatocellular carcinoma (HCC). However, considering the good anti-tumor performance of lenvatinib, combined immunotherapy on the basis of lenvatinib after first-line lenvatinib failure is also popular in clinical practice. This study aimed to compare the efficacy and safety of regorafenib plus ICI (TACE-R-I) versus lenvatinib plus ICI (TACE-L-I) in patients with advanced HCC after lenvatinib failure.</p><p><strong>Methods: </strong>In this single-center retrospective study, 164 patients with advanced HCC were enrolled from January 2019 to March 2024 in China. All patients were aged ≥ 18 years, clinically or pathologically diagnosed with HCC. All patients received trans-arterial chemoembolization (TACE) as local treatment. Overall survival (OS), progression-free survival (PFS), and treatment-related adverse events (TRAEs) were compared between groups. The Cox regression model was used to analyze the factors associated with OS and PFS.</p><p><strong>Results: </strong>We compared 77 patients from each group after propensity score matching (PSM). There was no significant difference in the OS (p = 0.255) or PFS (p = 0.387) between groups. However, in the subgroup (distant metastases, Barcelona Clinic Liver Cancer (BCLC) stage C or tumor thrombus), the TACE-R-I group showed better survival benefit than the TACE-L-I group. The multivariable Cox regression model suggested that BCLC stage and alpha-fetoprotein (AFP) were independently associated with OS. Distant metastases, tumor thrombus and Child-Pugh were independent associated factors for PFS (p < 0.05). The frequency of grade ≥ 3 TRAEs was not significantly different between groups (p ≥ 0.05).</p><p><strong>Conclusion: </strong>Our study demonstrated that in patients with greater tumor burden, the TACE-R-I group showed better OS and PFS benefits than the TACE-L-I group. However, in the overall population of HCC patients, there was no significant difference in efficacy and safety between the groups.</p>","PeriodicalId":11282,"journal":{"name":"Drugs - Real World Outcomes","volume":" ","pages":"135-143"},"PeriodicalIF":1.9,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11829866/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143001923","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Patients' Health-Related Quality of Life and Use of Medicinal Cannabis: A Cross-Sectional Survey Study.","authors":"Frederik Rosenbæk, Sonja Wehberg, Line Bjørnskov Pedersen, Jesper Bo Nielsen, Jens Søndergaard","doi":"10.1007/s40801-024-00479-2","DOIUrl":"10.1007/s40801-024-00479-2","url":null,"abstract":"<p><strong>Background: </strong>Studies on medicinal cannabis (MC) have primarily investigated effects on diseases and symptoms, while there is only sparse knowledge on patients' health-related quality of life. Our aim was, firstly, to compare the health-related quality of life of patients (MC users and non-users) within four specified diagnostic indications (multiple sclerosis, paraplegia, neuropathy, and nausea and vomiting after chemotherapy) with that of patients with other diagnostic indications (MC users only) and the adult population (non-users only). Secondly, we estimate the associations between use of MC and health-related quality of life for patients in the four specified diagnostic indications.</p><p><strong>Methods: </strong>We collected data on quality-adjusted life years (QALYs), using EQ-5D-3L, and patients' self-reported use of MC in a Danish nationwide online survey distributed to 23,846 patients in October 2020. We compared QALY scores of all groups using a two-tailed t-test, listed QALY scores of MC users versus non-users, and investigated associations between QALY score and MC use using unadjusted and adjusted linear regression analyses. Significance level was set to p-value < 0.05.</p><p><strong>Results: </strong>A total of 9265 patients took part in the survey. All diagnostic indications had a statistically significant lower QALY score than the adult population (0.87). Paraplegia patients had the lowest QALY score, being 0.36 lower, followed by other diagnostic indication (- 0.34), multiple sclerosis (- 0.20), neuropathy (- 0.13), and nausea and vomiting after chemotherapy (- 0.06). MC users had a statistically significant lower QALY score than non-users (0.44 vs 0.74). Users redeeming 1-6 and ≥ 7 MC prescriptions (except for paraplegia patients) had a statistically significant lower QALY score than non-users, ranging between 0.11-0.24 and 0.26-0.32 lower than non-users, accordingly. Although, it should be noted that the number of users was small when stratifying by number of prescriptions.</p><p><strong>Conclusion: </strong>Patients with either multiple sclerosis, paraplegia, neuropathy, or nausea and vomiting after chemotherapy had a significantly lower health-related quality of life than individuals from the adult population. Users of medicinal cannabis also had a significantly lower health-related quality of life compared with non-users, in all diagnostic indications.</p>","PeriodicalId":11282,"journal":{"name":"Drugs - Real World Outcomes","volume":" ","pages":"125-133"},"PeriodicalIF":1.9,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11829861/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142964262","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}