Drugs - Real World Outcomes最新文献

Background: Schizophrenia spectrum disorders (SSD) are chronic psychiatric conditions with high rates of medication nonadherence, relapse, and hospitalization. Long-acting injectable antipsychotics (LAIs) aim to improve adherence; their real-world use in inpatient settings is not yet well understood.

Objective: To investigate prescription patterns of LAI antipsychotics in a real-life setting among psychiatric inpatients with SSD in Australia.

Methods: This retrospective cross-sectional study was conducted at a major Australian tertiary center. It investigated prescription trends, demographics, hospitalization outcomes, and substance use among inpatients with SSD who received oral, LAI, or combined oral-LAI treatment. Readmission rates were also analyzed in patients with a history of medication nonadherence.

Results: Among the total of 510 inpatients with SSD, 26.6% received LAIs, 40% were treated with combined oral-LAI therapy, and 33.3% were prescribed oral antipsychotics alone. Second-generation LAIs were most prevalent (87.5%), with paliperidone being the most frequently used (53.7%). The combined oral-LAI therapy group had the highest rates of nonadherence (83.8%) and substance use (82.8%). Among patients with a history of medication nonadherence, those receiving LAIs had lower 30-day readmission rates compared with the oral antipsychotic treatment group.

Conclusions: Findings align with global trends favoring second-generation LAIs and highlight the rising yet understudied use of combined oral-LAI therapy. High nonadherence and substance use in the combined oral-LAI therapy group call for targeted interventions. While LAIs may reduce readmissions in nonadherent patients, further research is needed to assess combined therapy's effectiveness and optimize prescribing. These insights reinforce the role of LAIs in relapse prevention and the need for tailored adherence strategies.

Background: The optimal duration for thromboprophylaxis after colorectal cancer surgery remains uncertain. We sought to compare the effectiveness and safety of long-term thromboprophylaxis to that of short-term thromboprophylaxis in preventing venous thromboembolism (VTE) after colorectal cancer surgery.

Methods: In our retrospective study, patients undergoing colorectal cancer surgery were divided into the short-term (< 7 days) and long-term (≥ 7 days) thromboprophylaxis groups based on the low molecular weight heparin prophylaxis regimen. Propensity score matching was performed for both groups, and comparative analysis of the incidence of asymptomatic or symptomatic VTE and bleeding complications was conducted. Multivariable logistic regression analysis was performed in the unmatched cohort to explore the association of potential risk factors with postoperative VTE.

Results: A total of 140 patients undergoing colorectal cancer surgery were included. After matching, there were 57 patients in each group. VTE occurred in 18 patients (15.8%) within 6 months after surgery, with 12 cases (21.1%) in the short-term thromboprophylaxis group and six cases (10.5%) in the long-term thromboprophylaxis group (P = 0.123). There were no significant differences in the incidence of bleeding complications between the two groups. Multivariable logistic regression analysis indicated that long-term thromboprophylaxis can reduce the risk of postoperative VTE (odds ratio 0.34, 95% confidence interval 0.12-0.95; P = 0.039).

Conclusions: Long-term thromboprophylaxis (≥ 7 days) demonstrated comparable effectiveness and safety to shorter regimens (< 7 days) in preventing postoperative VTE in patients with colorectal cancer, while suggesting potential sustained protective benefits during extended follow-up periods exceeding 6 months. Whether VTE prophylaxis should be extended to 28 days post-surgery requires further research.

Background and objective: Nontuberculous mycobacteria (NTM) are opportunistic pathogens that can cause lung disease (NTMLD) in susceptible individuals, but NTMLD management is challenging. This study aims to describe real-world NTMLD treatment patterns in Belgium.

Methods: This retrospective study used data from the IQVIA longitudinal pharmacy database. Patients with presumed NTMLD (i.e., who initiated prespecified NTM treatments from October 2015 through September 2019) were included. Variables of interest were initiated prescribed regimens, medication possession rate (MPR), and treatment persistence, switches, and restarts.

Results: Overall, 199 presumed NTMLD patients initiated 72 triple- and 130 dual-drug regimens. The average triple-drug therapy MPR was 88%, and median treatment duration was 225 days. Sixty percent and 30% of patients remained on initial therapy at 6 and 12 months, respectively. Therapy switches were common, with up to five switches per patient. Seventeen percent of initiated therapies were stopped for more than 60 days but restarted within 1 year.

Conclusion: Despite inherent methodological limitations, results indicate therapy switches, premature treatment interruption, and restarting multidrug oral NTM treatment are common. These findings underscore the need for improved management of NTMLD through enhanced monitoring as well as more tolerable and effective treatment options.

Background and objective: Sexual dimorphism in drug efficacy, beyond pharmacokinetics (PK), remains underexplored. Significant sex differences exist in drug metabolism and adverse events, highlighting the need for personalized medicine. The objective of our study was to assess whether there are sex differences in the pharmacodynamic (PD) response to valproic acid (VPA) in photosensitive epilepsy, focusing on electroencephalographic (EEG) biomarkers (e.g., photoparoxysmal response [PPR] raw data and transformed PPR data, the standardized photosensitivity range [SPR]) that cannot be attributed to pharmacokinetics alone. On the basis of some exploratory published evidence plus our own clinical observations of VPA treatment in patients with epilepsy plus photosensitivity over time, we hypothesized that an EEG pharmacodynamic difference might exist between females and males.

Methods: We conducted a retrospective, observational, single-center, within-patient EEG cohort study conducted on antiseizure medicine (ASM)-naïve photosensitive individuals before and after VPA treatment (nonrandomized). The data we reviewed had been collected from a referral hospital in the Netherlands from 1990 to 2000. Changes in EEG data, including raw PPR data (transformed into SPR), were analyzed before and after VPA therapy in 48 patients, including 27 females and 21 males, ranging in age from 8 to 50 years old for the entire cohort. Co-primary outcomes included a between-sex comparison in the distribution of within-patient SPR changes from pre-VPA to steady-state VPA therapy, and complete PPR elimination on EEG. Secondary outcomes included the comparison of percentage of males and females meaningfully responding to VPA across SPR change categories, VPA dose, potential impact of plasma [VPA] concentrations on SPR changes, and associaton of patient age with SPR values. Statistical analyses included univariate linear regression models, chi-squared tests, non-parametric Wilcoxon-Mann-Whitney tests, and Fisher's exact tests.

Results: Our first co-primary outcome revealed a statistically significant difference in the distribution of within-patient SPR changes from pre-VPA to steady-state VPA therapy. Males experienced a significantly greater reduction in SPR compared with females. The mean decrease in SPR was -7.0 ± 2.6 in males only versus -3.9 ± 3.3 in females only (p = 0.0018). The next co-primary outcome, the percent of patients with complete PPR elimination, or a SPR value = 0 on second EEG, was observed in ten (47.6%) males compared with four (14.8%) females, a 3.2-fold difference (p = 0.0237). One secondary outcome, the percentage of males with a VPA clinically meaningful to optimal response was 1.93-fold greater than females, at 100:51.8%, respectively (p < 0.0001). Between-sex VPA total daily milligram dose did not differ. Plasma [VPA] concentrations, although nearly twice as high in females, were not st

Background and objective: Hereditary angioedema presents as recurrent, unpredictable, and often debilitating attacks of cutaneous/submucosal swelling. This study assessed the characteristics and treatment patterns of patients receiving long-term prophylaxis with the plasma kallikrein inhibitor lanadelumab in US clinical practice.

Methods: This retrospective longitudinal study, based on a physician panel-based medical chart review, included patients with a diagnosis of hereditary angioedema due to C1 esterase inhibitor deficiency/dysfunction (HAE-C1INH-Type1/2), initiating lanadelumab in/after August 2018 (index date), and with ≥ 3 months' post-index follow-up (Part 1, N = 186) and, additionally, a dosing interval extension after initiating lanadelumab 300 mg every 2 weeks (Part 2, N = 75).

Results: Patients in Part 1 were predominantly aged ≥ 18 years (95.7%) with HAE-CINH-Type1 (90.3%); Part 2 included a higher proportion of patients with HAE-C1INH-Type2 (28.0% vs 9.7%). In Part 1, 115/165 (69.7%) patients with hereditary angioedema attack information experienced 371 attacks in the 3 months pre-index; these were mostly mild/moderate (60.4%) and most commonly affected the lips (38.0%) and hands (32.9%). In total, 19/155 (12.3%) patients had 39 attacks during the post-index period (mean ± standard deviation [interquartile range] attack rate: 0.1 ± 0.3 [0.0, 0.0] per month). In Part 2, a dosing interval extension was enabled by well-controlled disease (74/75, 98.7%); most patients (86.7%) transitioned from every 2 weeks to every 4 weeks dosing. Among patients with attack information, 7/72 (9.7%) experienced a hereditary angioedema attack while receiving an initial every 2 weeks dosing regimen and 4/75 (5.3%) after an extended-interval dosing regimen.

Conclusions: Lanadelumab dosing intervals can be individualized to maintain effective disease control. A dosing interval extension may be considered in well-controlled disease.

Background: In low- and middle-income countries, sulfonylureas are commonly prescribed due to cost-effectiveness. However, data comparing their real-world impact, especially when used alone versus in combination with metformin, remain limited.

Objective: This study aimed to assess the effectiveness of glipizide and glipizide plus metformin in individuals with type 2 diabetes (T2D) using real-world data.

Methods: Data was obtained from 11,949 individuals with T2D who were prescribed either glipizide or glipizide+metformin and had at least one follow-up within 1 year at a tertiary diabetes care centre in India. The primary outcome was the change in glycated hemoglobin (HbA1c) levels from baseline to follow-up. Secondary outcomes included changes in fasting plasma glucose (FPG), postprandial glucose (PPG), body mass index (BMI), and estimated glomerular filtration rate (eGFR).

Results: The mean age of participants was 56 ± 11 years, 59% (n = 7008) were male, and the mean diabetes duration was 10.2 ± 8 years. In the glipizide group (n = 6034), HbA1c decreased from 8.8% to 7.9% (p < 0.001), FPG decreased by 16 mg/dL (p < 0.001), and PPG decreased by 29 mg/dL (p < 0.001). In the glipizide + metformin group (n = 5915), HbA1c levels declined from 8.9% to 7.8% (p < 0.001), and FPG and PPG declined by 23 mg/dL and 44 mg/dL, respectively (p < 0.001). BMI remained stable in the glipizide group, while a reduction of 0.2 kg/m² was observed among overweight/obese individuals in the glipizide + metformin group. The use of glipizide and glipizide + metformin effectively improved glycemic control without adverse anthropometric changes. C-peptide levels were preserved across all treatment groups, demonstrating sustained β-cell function. HbA1c reductions were observed consistently across all eGFR categories. Furthermore, as glipizide plus metformin is one of the least expensive antidiabetic drugs in India (₹1460/year [$16.87]) it can help improve accessibility to treatment even among those in lower socio-economic statuses.

Conclusions: Glipizide as monotherapy or in combination with metformin, significantly improved glycemic control even in those with decreasing renal function, with no adverse effects on weight and with preservation of β-cell function. While long-term studies are needed to assess the sustainability of these benefits, glipizide can be considered a cost-effective therapeutic option for T2D in low- and middle-income countries.

Background: Previous research in Canada has examined opioids prescription dispensing at the population level but did not examine the potential relationship with area-level income and rates of opioid dispensing.

Objective: The aim was to estimate average and annual opioid dispensing rate ratios (RRs) between lowest and highest income quintile geographic areas in Canada.

Methods: We performed a population-based retrospective study using the National Prescription Drug Utilization Information System (NPDUIS) between 2010 and 2018 that contains prescription records for all public drug plan beneficiaries (65+) in all Canadian provinces, excluding Quebec, Nova Scotia, and New Brunswick. We used census median household income, calculated at the Forward Sortation Area (FSA-the first three letters of the postal code) to assign income quintiles. Morphine milligram equivalent (MME) was calculated for all opioid dispensing and was divided by population of the FSA quintile. Population census year 2016 was used for population and income estimations. We calculated the average and annual RR between lowest and highest quintiles and stratified them by patients' sex. The significance of the trend of annual RR was tested by linear regression.

Results: The average MME per capita for the 65+ population ranged from 2321.8 in quintile 1 to 5831.9 in quintile 5. The RR between highest and lowest quintile was 2.5 (95% confidence interval [CI] 1.3-3.7), and was more profound for males (3.2, 95% CI 1.4-4.9) than females (2.2, 95% CI 1.2-3.3). Over the study period, the RR reduced slightly from 2.7 to 2.3 (p < 0.01). However, this trend was only significant for females.

Conclusion: Inequity in opioid prescriptions dispensing was persistent over time. Patients in the lowest income quintiles received higher amounts of opioids per capita, with some sex variation. Dispensing policies must take these equity issues into account.

Background: Wilson's disease (WD) is an autosomal recessive disorder characterized by abnormal copper accumulation, leading to multi-organ damage. The economic impact of WD in Italy has not been comprehensively studied.

Aims: The objectives were to determine the economic burden of WD, describe the demographic and clinical characteristics, and estimate the treatment distribution over time, using real-world data from Italy.

Methods: A retrospective multicenter longitudinal chart review study was conducted across six Italian reference centers for WD management. Patients with at least one visit for WD in 2019-2020 were included. Demographic, clinical, and treatment data were collected from medical records, and healthcare resource utilization and related costs were estimated over a 12-month follow-up. Treatment patterns from diagnosis to 2021 were also described.

Results: A total of 243 patients with WD were included (183 adults, 60 minors). Median age at diagnosis was 11 years in adults and 7 years in minors. At enrollment, hepatic involvement was the most frequent clinical manifestation (84.7% of adults; 80% of minors), while 13.1% of adults and 16.7% of minors were asymptomatic. In adults, use of D-penicillamine and zinc decreased, while trientine tetrahydrochloride use increased over time. In minors, treatment remained stable. The average annual cost per patient was €10,394 for adults (mainly driven by pharmacological treatment) and €1351 for minors. Costs increased with the number of disease manifestations.

Conclusion: The economic burden of WD in Italy varies with disease severity and treatment strategy, highlighting the need for optimized management practices to mitigate costs while enhancing patient care.

Background: Partial response to standard-dose proton pump inhibitors (PPIs) in gastroesophageal reflux disease (GERD) is common, yet real-world data on its burden and management in Indian settings remain limited.

Objective: This study aimed to understand the burden, clinical profile, drug utilization patterns across specialties, the effectiveness of Pantoprazole 80 mg dual delayed-release (DDR) formulation, and management strategies used in the treatment of partial responders with clinically diagnosed GERD in Indian settings.

Methods: This was a multicentric, retrospective observational study. Data on adult patients with GERD with a follow-up duration of at least 4 weeks from baseline were extracted from electronic medical records (EMR) of outpatient settings from five centers, which included drug utilization patterns, clinical and treatment profiles, and effectiveness of PPIs.

Results: Among EMRs of 5205 patients with GERD, 38.0% were on rabeprazole and 36.6% on pantoprazole (mean age: 53.3 years; standard deviation: 14.3 years), and 55.0% were male. Heartburn was the primary complaint in 76.0% of cases. Cardiovascular co-morbidities with dyslipidemia were reported in 66.7% (1742/2610) patients. Pantoprazole and rabeprazole were preferred across specialties, where 31.1% (592/1906) and 17.7% (350/1979) adhered to treatment, respectively. Total burden of partial responders was 41.7%, including patients who switched PPIs, changed PPI dosage, or added other medications. Pantoprazole 40 mg twice daily (BD) showed 49.1% improvement in heartburn and 50.9% in abdominal pain. Pantoprazole 80 mg DDR once daily demonstrated significantly higher symptom relief, with a 60.2% reduction in heartburn (p < 0.001) and a 66.1% reduction in abdominal pain (p < 0.001). These findings suggest that higher-dose pantoprazole therapy may be a clinically effective strategy for managing partial responders to standard-dose PPIs.

Conclusions: A significant proportion of patients with GERD were partial responders to PPIs. Pantoprazole and rabeprazole had high patient adherence across disciplines. Both pantoprazole DDR 80 mg once daily (OD) and 40 mg BD demonstrated significant symptom reduction in partial responders, supporting their use in optimizing GERD management in Indian clinical settings.