Journal of comparative effectiveness research最新文献

{"title":"Validity of HINT-8 in evaluating health-related quality of life among cervical cancer patients.","authors":"Ah-Yeon Lee, Soo Hyeon Lee, Min-Ju Kim, Ji-Eun An, Gyeong-U Hong, Woo-Jeong Sim, Hyunji Lim, Tae-Wook Kong, Byung Su Kwon, Sung Taek Park, Kyeong A So, Won Moo Lee, Jung-Yun Lee, Dae Hoon Jeong, Min Chul Choi, Youn Jin Choi, Jae-Kwan Lee, Tae-Hwe Heo, Kyung-Jin Min, Su-Yeon Yu","doi":"10.57264/cer-2025-0194","DOIUrl":"10.57264/cer-2025-0194","url":null,"abstract":"<p><p><b>Aim:</b> Cervical cancer, caused primarily by human papillomavirus infection, affects patients' health-related quality of life (HRQoL). Despite research on quality of life in cancer patients, studies using the HINT-8 instrument, especially for cervical cancer, are scarce. This study evaluates the validity of HINT-8 in measuring HRQoL among Korean cervical cancer patients by comparing it with EQ-5D-5L. <b>Materials & methods:</b> Demographic and clinical characteristics were analyzed. Mean, median and standard deviations for each domain of both instruments were calculated. Spearman and Pearson correlation coefficients assessed relationships between HINT-8 and EQ-5D-5L indices and domains. <b>Results:</b> Increased problem intensity in HINT-8 domains (e.g., stair climbing, pain and vitality) was correlated with decreased EQ-5D-5L index, indicating a higher response level in HINT-8 aligns with lower EQ-5D-5L scores. Significant variables were consistent across tools, showing no substantial functional difference. A strong correlation between HINT-8 and EQ-5D-5L indices (r = 0.771, p < 0.01) supports HINT-8's effectiveness in evaluating HRQoL for cervical cancer patients. <b>Conclusion:</b> The HINT-8 demonstrated a strong correlation with the EQ-5D-5L index and a substantially lower ceiling effect, suggesting that it can capture subtle differences in HRQoL among cervical cancer patients. Furthermore, the HINT-8 may provide complementary information beyond the EQ-5D-5L by capturing distinct dimensions such as memory, sleep and happiness, which are not explicitly represented in the EQ-5D-5L descriptive system. These findings support the validity of the HINT-8 as a complementary instrument for assessing HRQoL in Korean cervical cancer patients.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250194"},"PeriodicalIF":2.8,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13435966/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148578919","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Lung cancer screening in Lithuania: a cost-effectiveness analysis.","authors":"Hilde Ten Berge, Greta Piazza, Dianne Ramaker, Xuanqi Pan, Titas Bukšnys, Saulius Cicėnas, Skaidrius Miliauskas, Giedre Smailyte, Rolandas Zablockis, Marius Zemaitis, Edvardas Danila","doi":"10.57264/cer-2026-0034","DOIUrl":"10.57264/cer-2026-0034","url":null,"abstract":"<p><p><b>Aim:</b> This study aimed to assess the cost-effectiveness of the annual volumetric-enabled low-dose computed tomography (LDCT) lung cancer screening (LCS), based on the NELSON screening outcomes, versus no screening for the asymptomatic high-risk population in Lithuania. <b>Materials & methods:</b> The previously established model with a decision tree with an integrated state-transition Markov trace was adapted to assess the health benefits and the financial consequences of LCS from the Lithuanian healthcare system perspective. Individuals aged 50-74 years and with a smoking history underwent LCS with LDCT in the screening arm, and it was compared with the absence of screening across a lifetime horizon. The primary outcomes included the clinical benefits (lung cancer cases detected per stage and premature lung cancer deaths averted), direct costs (recruitment, diagnostic and treatment costs) of LCS implementation, quality-adjusted life years, life-years and the incremental cost-effectiveness ratio. One-way sensitivity analysis and probabilistic sensitivity analysis were conducted to ascertain the result's robustness. <b>Results:</b> Annual LCS with volumetric-enabled LDCT for 170,808 eligible individuals in Lithuania resulted in 6117 additional early-stage (I and II) lung cancers detected and 1874 averted late-stage (III and IV) lung cancers, leading to 2606 premature lung cancer deaths averted and 21,639 life-years gained at an uptake rate of 46.5%. The incremental cost-effectiveness ratio was €1372 per quality-adjusted life year with incremental costs of €21.1 million and 15,391 quality-adjusted life years gained. The results were robust based on sensitivity and scenario analyses. <b>Conclusion:</b> This study demonstrated that annual LCS with volumetric-enabled LDCT for a high-risk population could be cost-effective compared with no screening in Lithuania.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e260034"},"PeriodicalIF":2.8,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13435896/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148471025","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"R WE ready for reimbursement? A round-up of developments in real-world evidence relating to health technology assessment: part 27.","authors":"Paul Arora, Sreeram V Ramagopalan","doi":"10.57264/cer-2026-0144","DOIUrl":"10.57264/cer-2026-0144","url":null,"abstract":"<p><p>In this update, we review the US FDA's updated compendium of real-world evidence (RWE) use in medical device regulatory decisions, a comprehensive scan of RWE utilization in Canadian drug reimbursement submissions, and Institute for Clinical and Economic Review's use of RWE to inform US Medicare drug price negotiations under the Inflation Reduction Act.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e260144"},"PeriodicalIF":2.8,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13435971/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148436878","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A disease progression model comparing the long-term mobility and respiratory outcomes of adults with late-onset Pompe disease receiving cipaglucosidase alfa plus miglustat versus alglucosidase alfa.","authors":"Amy Dymond, Will Green, Matthew Crabtree, Alasdair MacCulloch, Neil Johnson, Simon Shohet, Vera Gielen, Sophie Clarke, Patrick Deegan, Jordi Diaz-Manera","doi":"10.57264/cer-2025-0202","DOIUrl":"10.57264/cer-2025-0202","url":null,"abstract":"<p><p><b>Aim:</b> Late-onset Pompe disease (LOPD) is a rare lysosomal disease primarily impacting muscle strength and respiratory function. LOPD has a substantial burden despite the availability of alglucosidase alfa (alg). Patients often require mobility and respiratory support over time. Cipaglucosidase alfa in combination with miglustat (cipa + mig) is one of two more recently approved treatments for adults with LOPD. Given limited data on the lifetime trajectory to mobility and respiratory support in LOPD, a patient-level simulation model was developed to compare the long-term impact of cipa + mig with alg on these outcomes. <b>Materials & methods:</b> The patient-level simulation predicts lifetime mobility and respiratory disease progression outcomes based on the 6-min walk distance and %predicted forced vital capacity for alg and cipa + mig for the overall LOPD population using available data and assumptions from experienced clinicians. PROPEL/PROPEL open-label extension (NCT03729362) and ATB200-02 (NCT02675465) studies informed outcomes for four years with cipa + mig and one year with alg. French Pompe disease registry data were used thereafter. <b>Results:</b> Based on the available data and clinical assumptions, the model predicts cipa + mig slows the overall progression of LOPD, allowing patients an additional 2.72 years without mobility or respiratory support compared with alg. People receiving alg may be wheelchair dependent and require invasive respiratory support for an additional 2.57 and 1.55 years, respectively. <b>Conclusion:</b> Cipa + mig may delay disease progression compared with alg over the lifetime of a patient with LOPD, which would increase the amount of time spent without mobility and respiratory support dependency.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250202"},"PeriodicalIF":2.8,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13435892/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148447447","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Transparency in positive airway pressure therapy trials: an evaluation of reporting quality.","authors":"Petra Stazić Kunčić, Diana Gujinović, Renata Pecotić, Zoran Đogaš, Ana Marušić, Marija Roguljić","doi":"10.57264/cer-2026-0058","DOIUrl":"10.57264/cer-2026-0058","url":null,"abstract":"<p><p><b>Aim:</b> Accurate and detailed reporting of nonpharmacological interventions such as positive airway pressure (PAP) therapy is essential for reproducibility, yet previous research shows frequent reporting deficiencies. Due to the complexity of PAP therapy, we evaluated the quality of PAP intervention reporting in ClinicalTrials.gov and publications, and assessed consistency between them. <b>Materials & methods:</b> ClinicalTrials.gov was searched on 16 May 2023, for adult obstructive sleep apnea trials involving PAP therapy. Trials were evaluated for registration completeness (21 WHO Trial Registration DataSet items) and intervention reporting (adapted 12-item Template for Intervention Description and Replication checklist). Publications identified on 3 September 2025, following a 2-year publication period, were compared with registered data for participant characteristics, outcomes and intervention reporting. <b>Results:</b> Among 299 included trials, 178 corresponding publications were identified. Reporting of PAP therapy was frequently incomplete in registry and publications, with key procedural details - such as mask fitting, device acclimatization and adherence strategies - missing in the majority of records. Substantial discrepancies were also found between the registered and published data. Sample size calculation was reported in 56.1% of publications but in none of the trial registrations, with participant numbers differing in 29.3% of articles. Inconsistencies were most common for secondary outcomes (43.3%), while primary outcomes differed from those registered in 24.2% of publications. <b>Conclusion:</b> Incomplete and inconsistent reporting of PAP therapy across registry and corresponding publications may be compatible with selective reporting. Rather than pointing to individual misconduct, these discrepancies indicate that key information on protocol changes, outcomes and adherence is fragmented across sources, limiting the ability of clinicians and researchers to appraise trial findings in full. Strengthening adherence to trial registration requirements and structured intervention-reporting frameworks is therefore essential to improve transparency and reinforce confidence in the evidence base for obstructive sleep apnea management.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e260058"},"PeriodicalIF":2.8,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13435968/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148562136","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Effect of dose tapering policy on the prescription of advanced therapy for rheumatoid arthritis: a retrospective analysis of clinical and cost outcomes in Taiwan.","authors":"Ko-Jen Li, Chao-Hsiun Tang, Chia-Li Chang, Han-Fang Liao, Pei-Jung Yen, Wen-Yi Shau","doi":"10.57264/cer-2026-0020","DOIUrl":"10.57264/cer-2026-0020","url":null,"abstract":"<p><p><b>Aim:</b> To evaluate the impact of the Taiwan National Health Insurance program policy for advanced rheumatoid arthritis (aRA) therapies on treatment prescription patterns and costs in patients with rheumatoid arthritis (RA). <b>Materials & methods:</b> We performed a retrospective analysis of patients with RA aged ≥18 years who had commenced aRA treatment in the National Health Insurance research database in Taiwan between 2011 and 2017. Follow-up data were aggregated every 3 months to determine the proportion of the covered RA prescription days and the medical cost. Associations of the tapering policy initiation in 2014, aRA dose, non-aRA prescription and cost were analyzed using a generalized linear model. <b>Results:</b> In total, 9099 patients with RA were included in this study. Before the policy implementation, the probability of aRA dose tapering increased significantly once treatment duration reached 24 months, with adjusted odds ratio = 3.27 (p < 0.001); this further increased to 4.00 (p < 0.001) after the policy was implemented. The difference between both adjusted odds ratios was statistically significant (p = 0.012). Dose tapering for aRA was associated with reduced medical costs and decreased use of other RA prescription drugs. However, the number of reductions decreased after the dose tapering policy was initiated, suggesting an unintended effect of the policy. <b>Conclusion:</b> The policy implementation increased the dose tapering of aRA prescriptions and was associated with decreased medication costs and the use of other RA treatments. However, a moderate partial offset of the reduction in outcome measures suggests unintended consequences of the policy.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e260020"},"PeriodicalIF":2.8,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13435894/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148620256","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Mortality risk estimation for autistic older adults: comparing novel machine-learning derived weights versus standard weights for the Charlson Comorbidity Index.","authors":"Madison Blake, Melica Nikahd, J Madison Hyer, Brian W Patterson, Bethany J Wolf, Lauren Bishop, Brittany N Hand","doi":"10.57264/cer-2026-0053","DOIUrl":"10.57264/cer-2026-0053","url":null,"abstract":"<p><p><b>Aim:</b> We aimed to compare Quan and colleagues (2011) established weights for the Charlson Comorbidity Index (CCI) conditions to autism-specific weights for predicting mortality risk in autistic older adults. <b>Materials & methods:</b> We used inpatient healthcare claims from autistic older adults (aged 65+; n = 2829) using the Medicare Standard Analytic Files from 2021 to 2023. We used a machine learning technique called stochastic hill climbing to assign weights to the 12 CCI conditions to maximize predictive ability for 30-day and 1-year mortality. We then compared the resulting area under the curve (AUC) against the established weights. <b>Results:</b> The established weights had poor predictive ability for 30-day (AUC: 0.68; 95% CI: 0.62-0.74) and 1-year mortality (AUC: 0.67; 95% CI: 0.63-0.72). The autism-specific weights also had poor predictive ability for 30-day (AUC: 0.67; 95% CI: 0.61-0.73) and 1-year mortality (AUC: 0.67; 95% CI: 0.62-0.71). <b>Conclusion:</b> The established and autism-specific CCI weights performed similarly in predicting mortality among autistic older adults. Findings may suggest adjusting CCI weights alone is insufficient to accurately predict mortality risk in autistic older adults, and additional health conditions not currently captured by the CCI may need to be added to better predict mortality in this population. Future studies on developing an autism-specific mortality risk index are warranted.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e260053"},"PeriodicalIF":2.8,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13436062/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148604070","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Real-world comparative effectiveness of analogue and human insulin on glycemic control in children and adolescents with Type 1 diabetes in Lebanon using continuous glucose monitoring data.","authors":"Sahar Masri, Manuel Albela, Iza Ciglenecki, Philippa Boulle, Tania Hachem, Sola Bahous, David Prieto-Merino, Pablo Perel","doi":"10.57264/cer-2026-0036","DOIUrl":"10.57264/cer-2026-0036","url":null,"abstract":"<p><p><b>Aim:</b> In high income settings, insulin analogues were associated with improved glycemic control among adults with diabetes, mainly measured through HbA1c and hypoglycemic episodes. In low- and middle-income countries, especially those affected by humanitarian crises, analogue insulins may be helpful as proper diabetes care for this population remains challenging; yet, supporting evidence is lacking. <b>Materials & methods:</b> Routinely collected continuous glucose sensor data from patients with Type 1 diabetes, aged 4 to 18 years old, between April 2019 and December 2022 was retrospectively extracted. Changes in glycemic metrics while using analogue insulins were compared with human insulin using multiple linear regression with random effects. <b>Results:</b> Forty-five patients included in the study used continuous glucose monitor devices for an average duration of 73 days (±53) while using human insulin and 170 days (±83) while using analogue insulins. Compared with human insulin, analogue insulins were associated with an additional 26 min (95% CI; 15, 37, p < 0.001) per day spent within the normal target range and a reduction of 11 min (95% CI; -16, -5) and 10 min (95% CI; -13, -7) in overall and nocturnal hypoglycemia, respectively. Analogue insulin was also associated with a reduction of 6 mg/dl in 24 h mean glucose. <b>Conclusion:</b> Insulin analogues were associated with slight improvements in time within and below range compared with human insulin, yet within patient glycemic variability remains high. Further research is needed to explore its acceptability among patients, impact on quality of life and its cost-effectiveness and sustainability in low resource contexts.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e260036"},"PeriodicalIF":2.8,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13436064/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148471005","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Comparing characteristics and outcomes between hospitalized adults on a pea protein or dairy/soy protein formulas: initial findings.","authors":"Vanessa Millovich, Irene Olsen Shingara, Guilherme S Lopes, Manu Tyagi, Zhun Cao, Christina J Valentine","doi":"10.57264/cer-2026-0035","DOIUrl":"10.57264/cer-2026-0035","url":null,"abstract":"<p><p>Enteral formulas are an essential part of nutrition support to prevent or treat malnutrition and minimize hospital length of stay (LOS). Yet not all formulas are tolerated and may leave nutritional needs unmet. Unique pea protein plant-based formulas (PPPBF) are nutritionally complete and have accumulated evidence of good tolerance, but impact on health economic outcomes (HEO) is largely unknown. <b>Aim:</b> To examine differences in patient and clinical characteristics and HEOs of hospitalized adults using PPPBF versus dairy and/or soy protein (DSP) formulas to inform further research. <b>Materials & methods:</b> Retrospective comparative cohort study examined real-world data (Premier Healthcare Database) from adults (≥18 year) admitted to US hospitals who were prescribed a formula between 1 January 2020 and 30 September 2023. Patient and clinical characteristics and HEOs were compared between the PPPBF and DSP groups by unadjusted descriptive analysis. <b>Results:</b> Preliminary analyses were conducted on inpatients (n = 65,338 DSP; n = 243 PPPBF) from 60 US hospitals. PPPBF (versus DSP) group was younger (mean [SD] 63.6 [17.0] vs 66.7 [17.3] years; p = 0.006) and had a higher diagnosis rate for malnutrition, weight loss, food allergies, irritable bowel syndrome and/or inflammatory bowel disease on admission. Overall, formula intake was primarily oral but higher in PPPBF versus DSP (100 vs 78.3%, p < 0.001). Charlson Comorbidity Index indicated PPPBF (versus DSP) was sicker (median 4.0 vs 3.0; p < 0.001). Yet PPPBF group had shorter LOS (by ∼2 days; p < 0.001) and lower mortality rate by discharge (5.8% vs 11.5%; p = 0.005) without significant difference in 90-day readmission/outpatient visit rates after discharge (unadjusted comparisons). <b>Conclusion:</b> Preliminary evidence in hospitalized adults observed a shorter LOS in PPPBF users, despite higher baseline acuity and without significant difference in readmission/outpatient visit rates compared (unadjusted) to DSP users. Adjusted analyses and further research are needed.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e260035"},"PeriodicalIF":2.8,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13329859/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148031365","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Acthar Gel treatment in patients with rheumatoid arthritis, systemic lupus erythematosus, or dermatomyositis/polymyositis: analysis of physician-reported charts.","authors":"Kyle Hayes, Amit Patel, Priyanka P Shanbhag, Destri R Evans, Mary Prince Panaccio, Johanna Purcell, George J Wan","doi":"10.57264/cer-2025-0200","DOIUrl":"10.57264/cer-2025-0200","url":null,"abstract":"<p><p><b>Aim:</b> To examine the characteristics, treatment patterns, and physicians' assessments of outcomes for patients with rheumatoid arthritis (RA), systemic lupus erythematosus (SLE), or dermatomyositis/polymyositis (DM/PM) who received Acthar Gel. <b>Materials & methods:</b> This survey-based medical chart review study asked rheumatologists who met specific inclusion criteria to abstract data from patient records covering the period from April 2022 to November 2024. Eligible patients were adults ≥18 years diagnosed with RA, SLE, or DM/PM, treated with Acthar Gel for ≤24 months. An online questionnaire screened and identified contributing physicians and collected anonymized patient data (baseline demographic, medical history, concomitant medications, Acthar Gel treatment history, and physicians' assessment of health status, symptom severity, treatment outcomes with Acthar Gel). <b>Results:</b> The study population comprised 73 patients with RA (average age 50 years; 49 [67%] female; 44 [60%] White/non-Hispanic), 56 with SLE (average age 42 years; 47 [84%] female; 28 [50%] African-American), and 104 with DM/PM (average age 52 years; 69 [66%] female; 62 [60%] White/non-Hispanic). Patients had received Acthar Gel for an average of 9 (RA) or 8 months (SLE, DM/PM), with most receiving treatment at the time of the study. Per physicians' assessment, health status improved in 68 (93%) patients with RA, 50 (89%) patients with SLE, and 100 (96%) patients with DM/PM after starting treatment with Acthar Gel. The most common treatment goals achieved in patients with improved overall health status were improved overall symptoms, pain, physical function, and corticosteroid use in the RA cohort; overall symptoms, pain, corticosteroid use, and fatigue in the SLE cohort; and overall symptoms, strength, physical function, and corticosteroid use in the DM/PM cohort. <b>Conclusion:</b> These findings support the use of Acthar Gel as a potential treatment option for appropriate patients with RA, SLE, or DM/PM.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250200"},"PeriodicalIF":2.8,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13329898/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148317329","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}