Journal of comparative effectiveness research最新文献

{"title":"Eculizumab or ravulizumab treatment effect in people with neuromyelitis optica spectrum disorder: a plain language summary of three studies.","authors":"Alfredo Damasceno, Mariano Marrodan","doi":"10.57264/cer-2024-0177","DOIUrl":"10.57264/cer-2024-0177","url":null,"abstract":"<p><strong>What is this summary about?: </strong>Neuromyelitis optica spectrum disorder (NMOSD for short) is a rare autoimmune health condition, meaning that the body's natural defense system (the immune system) attacks the body's own tissues. This summary describes NMOSD and the results of three studies of the effects of treatment with two medicines called eculizumab and ravulizumab. Eculizumab and ravulizumab are approved to treat people with a type of NMOSD called AQP4-Ab+ NMOSD. The three studies included in this summary are the PREVENT and CHAMPION-NMOSD clinical studies and a study done in everyday clinical practice in Japan.</p><p><strong>What are the key conclusions from these studies?: </strong>The PREVENT study was a phase 3 study (a large study testing safety and effectiveness of a treatment before it is approved) with three parts. The PREVENT main study compared eculizumab to a placebo (a treatment with no active ingredients, used to test how well a new treatment works). A long-term follow-up study assessed the safety and effectiveness of eculizumab over time, without a placebo group. Another long-term follow-up focused on people taking eculizumab alone, without other immunosuppressive treatments. The CHAMPION-NMOSD study was another phase 3 study testing ravulizumab, a treatment based on eculizumab but given less often, using the placebo group from the PREVENT study for comparison. A daily clinical practice study with eculizumab in Japan looked at how eculizumab works in everyday medical practice (outside of controlled clinical trials) after the medication was already approved. The studies found eculizumab and ravulizumab to be safe and effective for preventing relapses in AQP4-Ab+ NMOSD. More than 95% of people treated with these medicines stayed relapse-free during the months or years of the studies follow-up periods. Most people reported side effects that were mild or moderate. The most common side effects were headache, runny nose or sore throat (nasopharyngitis), and infections in the upper respiratory system. The daily practice study confirmed that eculizumab works well in everyday medical practice.</p><p><strong>What do the findings of the study mean?: </strong>These studies suggest that eculizumab and ravulizumab are safe and effective treatments for people with AQP4-Ab+ NMOSD. Eculizumab may help reduce or stop the need for other treatments that weaken the immune system (immunosuppressive therapies). These treatments help people maintain their ability to carry out daily activities and their quality of life.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240177"},"PeriodicalIF":1.9,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11963372/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143399377","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 evidence: state-of-the-art and future perspectives.","authors":"Heather Fitzke, Tamanah Fayzan, Jonathan Watkins, Evgeny Galimov, Benjamin F Pierce","doi":"10.57264/cer-2024-0130","DOIUrl":"10.57264/cer-2024-0130","url":null,"abstract":"<p><p>Recent developments in digital infrastructure, advanced analytical approaches, and regulatory settings have facilitated the broadened use of real-world evidence (RWE) in population health management and evaluation of novel health technologies. RWE has uniquely contributed to improving human health by addressing unmet clinical needs, from assessing the external validity of clinical trial data to discovery of new disease phenotypes. In this perspective, we present exemplars across various health areas that have been impacted by real-world data and RWE, and we provide insights into further opportunities afforded by RWE. By deploying robust methodologies and transparently reporting caveats and limitations, real-world data accessed via secure data environments can support proactive healthcare management and accelerate access to novel interventions in England.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240130"},"PeriodicalIF":1.9,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11963347/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143573175","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":"Trends in adoption of knotless tissue control devices in robotic surgery.","authors":"Barbara H Johnson, Sinchana T, Stephen S Johnston, Najmuddin Gunja","doi":"10.57264/cer-2024-0229","DOIUrl":"10.57264/cer-2024-0229","url":null,"abstract":"<p><p><b>Aim:</b> Trends in the use and comparative outcomes for barbed and conventional sutures have not been well-reported for robotic surgery. <b>Materials & methods:</b> This retrospective study used hospital discharge data in the US to assess suture use during robotic colorectal surgery (CR), total hysterectomy (TH) and ventral hernia repair (VHR) performed between 1 October 2015 and 30 June 2022. We first examined quarterly trends in use of barbed sutures, (specifically STRATAFIX™ knotless tissue control devices [KTCD]) and then compared clinical and economic outcomes between KTCD and conventional sutures. Outcomes included wound-related complications (infection/wound dehiscence) through 90 days post discharge, operating room (OR) time, hospital costs and 30/60/90 days readmissions. Propensity score weighting was used to address potential confounding in the comparisons of outcomes, performed separately for each procedure. Generalized linear models, accounting for hospital-level clustering, with link functions and error distributions tailored to the empirical distribution of outcomes were used to test for statistically significant differences in outcomes between the KTCD and conventional suture cohorts. <b>Results:</b> We identified 15,875 patients with CR (668 KTCD), 175,963 patients with TH (15,075 KTCD) and 32,469 patients with VHR (6776 KTCD). Over the study period, the proportion of robotic surgeries using KTCD tripled for CR (2.0-6.4%) and TH (2.9-10.4%) and more than doubled for VHR (12.2-25.3%). Compared with conventional sutures, KTCD was associated with significantly shorter OR time for TH (-19.1 min, 95% CI: [-30.2, -8.0]) and VHR (-17.3 min, 95% CI: [-31.4, -3.2]), and was numerically shorter, but did not reach statistical significance for CR (-23.2 min 95% CI: [-48.1 to 1.7]). All other outcomes were similar between the two suture cohorts for all procedures, apart from CR 90-day readmissions, which were lower for the KTCD cohort (-2.8%, 95% CI: [-5.2 to -0.4%]). <b>Conclusion:</b> Adoption of KTCD has grown substantially over the past 6 years. While most clinical and economic outcomes were similar between the two groups, KTCD was associated with lower OR time versus conventional sutures for TH and VHR and lower 90-day readmissions for CR.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240229"},"PeriodicalIF":1.9,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11963344/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143647928","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":"Evaluation of reporting in time-driven activity-based costing studies on cardiovascular diseases: a scoping review.","authors":"Nayê Balzan Schneider, Erica Caetano Roos, Miriam Allein Zago Marcolino, Fabio Caldana, Filipe Rodrigues Vargas do Nascimento, Sérgio Renato da Rosa Decker, Ana Paula Beck da Silva Etges, Carisi Anne Polanczyk","doi":"10.57264/cer-2024-0013","DOIUrl":"10.57264/cer-2024-0013","url":null,"abstract":"<p><p><b>Aim:</b> This scoping review evaluates the application of the time-driven activity-based costing (TDABC) methodology in cardiovascular disease (CVD) studies. <b>Materials & methods:</b> The evaluation was conducted using the 32-item TDABC Healthcare Consortium Consensus Statement Checklist. A systematic search was performed in Medline, Embase and Scopus in September 2023, including only full-text, peer-reviewed studies reporting the application of TDABC in CVD research. <b>Results:</b> Twenty studies were included in the review. The positive response rate for individual studies ranged from 31 to 81%. The most frequently addressed checklist item was the clear definition of study objectives, while presenting costs per patient included in the analysis was the least reported item. Although 70% of the studies achieved a positive response rate above 50%, adherence to the TDABC checklist remains inconsistent. <b>Conclusion:</b> There is significant room for improvement in the reporting of TDABC methodology in CVD studies. Providing a more comprehensive and standardized description of the methodology would enhance the utility, reproducibility and accuracy of the information generated, supporting the development of evidence-based health policies and improving accountability in healthcare cost assessments.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240013"},"PeriodicalIF":1.9,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11963363/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143501842","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":"Burden of illness for patients with primary biliary cholangitis: an observational study of clinical characteristics and healthcare resource utilization.","authors":"Robert G Gish, Joanna P MacEwan, Alina Levine, Dannielle Lebovitch, Leona Bessonova, Darren Wheeler, Radhika Nair, Alan Bonder","doi":"10.57264/cer-2024-0174","DOIUrl":"10.57264/cer-2024-0174","url":null,"abstract":"<p><p><b>Aim:</b> To evaluate the clinical characteristics and healthcare resource utilization for acute care and its costs for patients with primary biliary cholangitis (PBC) with or without cirrhosis. <b>Materials & methods:</b> This retrospective observational cohort study was conducted using two datasets (Komodo's Healthcare Map™ [Komodo Health] and Optum Clinformatics^® Data Mart [CDM] database) between 2015 and 2023. Patients (≥18 years) with PBC were identified based on ≥1 inpatient or ≥2 outpatient claims. Healthcare resource utilization for acute care (hospitalizations and emergency department [ED] visits [not leading to hospitalization]) were assessed in both datasets, and associated medical costs were evaluated in Optum CDM. <b>Results:</b> In Komodo Health, of the 29,758 patients with PBC (mean age: 59.2 years), 21.6% had cirrhosis and 50.4% of patients with cirrhosis had Medicaid or Medicare coverage. Of the total 8143 patients in Optum CDM (mean age: 67.0 years), 20.7% had cirrhosis, and most were enrolled in Medicare (69.7%). There was a larger proportion of men in the cirrhosis group compared with the no-cirrhosis group in Komodo Health (31.7 vs 16.3%) and Optum CDM (29.7 vs 16.5%). Annually, among patients with cirrhosis who had a hospitalization, 69.3% had additional hospitalizations, and among patients who had an ED visit, 52.9% had additional ED visits in Komodo Health; similar results were observed in Optum CDM. Among patients with at least one acute-care event, the mean annual acute-care costs with and without cirrhosis were $113,568 and $47,436, respectively. <b>Conclusion:</b> Data from two large healthcare claims databases showed that the majority of patients who had at least one acute-care event experienced additional acute-care events, particularly among those with cirrhosis. Timely treatment to avoid hospitalization and disease progression may help mitigate the clinical and economic burden for patients with PBC.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240174"},"PeriodicalIF":1.9,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11963345/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143567244","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 18.","authors":"Paul Arora, Sreeram V Ramagopalan","doi":"10.57264/cer-2025-0014","DOIUrl":"10.57264/cer-2025-0014","url":null,"abstract":"<p><p>In this update, we discuss recent publications examining the use of real-world data as a measure of treatment effectiveness in submissions to the National Institute of Health and Care Excellence, the results of a pilot study from the Coalition to Advance Real-World Evidence initiative and a validation study of synthetic data.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250014"},"PeriodicalIF":1.9,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11963373/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143441107","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":"The role of health economic evidence in clinical practice guidelines for colorectal cancer: a comparative analysis across countries.","authors":"Xiaoyu Yan, Yue Wang, Aixia Ma, Hongchao Li","doi":"10.57264/cer-2024-0226","DOIUrl":"10.57264/cer-2024-0226","url":null,"abstract":"<p><p><b>Aim:</b> Colorectal cancer (CRC) is among the most prevalent malignancies globally and causes massive resource consumption and economic burden. Health economic evidence (HEE) has been used in clinical practice guidelines (CPGs) for cancer to facilitate the rational allocation of health resources. However, in certain guideline development organizations, HEE is not yet utilized as a formal decision-making criterion. This study aimed to compare the discrepancies in the utilization of health economics as evidence in CRC CPGs across different countries and review specific features of economic evidence concerning the guidelines' applicability. <b>Materials & methods:</b> A systematic review was conducted using databases including Medline, Embase, CNKI, WanFang, and other guidelines databases to identify CPGs for CRC published in English or Chinese from January 2017 to September 2023. Data on the incorporation and application of HEE were extracted, and the method and quality of cost-effectiveness analysis (CEA) studies were evaluated. Descriptive analyses were used to summarize the results. <b>Results:</b> Out of 53 CPGs from 14 countries, most originated from the USA (n = 17 of 53 [32%]) and Canada (n = 9 of 53 [17%]). Sixty-eight percent (36/53) considered cost justification, and 57% (30/53) incorporated health economics studies as evidence. The included HEE cited in CPGs ranged from 1990 to 2021 and were not aligned with the countries in which the guidelines were issued. Among these CEA studies, 52% (26/50) were related to screening strategies, and 32% (16/50) pertained to treatment measures. The Markov model was the most frequently used (n = 27 of 50 [54%]). Based on the CHEQUE tool, the methodological quality of these CEA studies was inadequate in areas such as multiple data sources, approaches to select data sources, assessing the quality of data, and relevant equity or distribution. <b>Conclusion:</b> In summary, 57% of guidelines incorporated health economics studies as evidence, with a variation between different countries. The included HEE still had deficiencies in methodology and reporting quality. In the future, it is suggested that health economics research should use a standardized methodology and reporting approach to assist in clinical decision making.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240226"},"PeriodicalIF":1.9,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11963387/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143449213","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":"Matching-adjusted indirect comparisons of efficacy outcomes between etrasimod and ozanimod for moderately to severely active ulcerative colitis.","authors":"Vipul Jairath, Tim Raine, Thomas P Leahy, Ravi Potluri, Karolina Wosik, David Gruben, Joseph C Cappelleri, Peter Hur, Lauren Bartolome","doi":"10.57264/cer-2024-0193","DOIUrl":"10.57264/cer-2024-0193","url":null,"abstract":"<p><p><b>Aim:</b> Etrasimod and ozanimod are selective sphingosine 1-phosphate receptor modulators targeting the S1P_1,4,5, and S1P_1,5 receptors, respectively, for the treatment of patients with moderately to severely active ulcerative colitis (UC). No head-to-head trial data exist between the two treatments. We compared these treatments indirectly using key efficacy outcomes from pivotal trials with induction and maintenance phase data adjusting for differences in clinical trial design and populations. <b>Materials & methods:</b> Individual patient data for etrasimod were matched to published aggregate data of ozanimod by key baseline characteristics. An anchored matching-adjusted indirect comparison (MAIC) was conducted for the induction period. An unanchored MAIC was utilized during the maintenance period due to differences in placebo arms between trials as a result of differing trial designs. Matching characteristics measured at baseline were age, sex, corticosteroid use, duration of UC, biologic exposure, modified Mayo score, and presence of left-sided colitis. Outcomes were clinical response and clinical remission for the induction period, and clinical response and clinical remission among induction phase responders for the maintenance period. Two sensitivity analyses were conducted. The first matched on prior TNFi exposure rather than biologic exposure, the second sensitivity analysis included an induction only etrasimod trial (ELEVATE UC 12). <b>Results:</b> There were no significant differences between etrasimod and ozanimod at the end of the induction period for clinical response and clinical remission, respectively (relative risk [RR] 0.98 [95% confidence interval (CI): 0.76-1.33], RR: 1.25 [95% CI: 0.71-2.92]). At the end of maintenance, etrasimod demonstrated improved outcomes compared with ozanimod for both clinical response (RR: 1.18 [95% CI: 1.05-1.30]) and clinical remission among induction phase responders (RR: 1.33 [95% CI: 1.12-1.55]). In the sensitivity analysis that matched on prior TNFi exposure rather than biologic exposure, there were no notable differences compared with the primary analyses. In the sensitivity analysis pooling ELEVATE UC 12 and ELEVATE UC 52 data, results were similar for clinical response (RR: 0.90 [95% CI: 0.75-1.10]) but etrasimod showed reduced efficacy for clinical remission (RR: 0.72 [95% CI: 0.50-1.12]) compared with the primary analysis, though overall remained not significantly different from ozanimod. <b>Conclusion:</b> MAIC results suggest that patients receiving etrasimod have similar induction results but are more likely to have clinical response and clinical remission at the end of the maintenance phase compared with patients receiving ozanimod. Despite the approach to ensure similarity between the trials by weighting, residual imbalance is possible, and results should be interpreted in the context of the assumptions.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240193"},"PeriodicalIF":1.9,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11963346/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143483330","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":"Access in all areas? A roundup of developments in market access and health technology assessment: part 6.","authors":"Annie Jullien Pannelay, Ramiro E Gilardino, Sreeram V Ramagopalan","doi":"10.57264/cer-2024-0239","DOIUrl":"10.57264/cer-2024-0239","url":null,"abstract":"<p><p>In this update, we discuss an article covering the implementation challenges of the upcoming European Union Health Technology Assessment regulation, particularly focusing on the complexity of population, intervention, comparator and outcomes requirements across member states; a user guide to applying generalized cost-effectiveness analysis for broader value assessment and finally highlight an ongoing debate surrounding National Institute for Health and Care Excellence's severity modifier implementation.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240239"},"PeriodicalIF":1.9,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11864082/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142949708","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":"Selumetinib for children with neurofibromatosis type 1 and plexiform neurofibromas that can't be removed by surgery, and impact on how the condition affects caregivers: a plain language summary.","authors":"Yulia Dinikina, Marina Dorofeeva, Fatima Nakhusheva","doi":"10.57264/cer-2024-0184","DOIUrl":"10.57264/cer-2024-0184","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;What is this summary about?: &lt;/strong&gt;Neurofibromatosis type 1 (also called NF1) is a rare genetic condition. It causes a range of symptoms that develop from childhood onwards and worsen over time. Some children with NF1 develop non-cancerous nerve tumors called plexiform neurofibromas. Plexiform neurofibromas can grow large and compress nearby tissues. This can cause severe pain, reduced movement, vision and hearing loss, and other medical problems. Some children can have plexiform neurofibromas removed surgically. Most children have tumors that cannot be removed by surgery (known as inoperable tumors). Children with inoperable plexiform neurofibromas can receive a medicine called selumetinib. This plain language summary includes important findings from two selumetinib studies in children with NF1 and inoperable plexiform neurofibromas: The SPRINT selumetinib studies are part of a clinical study program that looked at how well selumetinib works in treating children with symptomatic, inoperable plexiform neurofibromas. The SPRINT studies program included the first studies of this medicine done in children, called phase 1 and phase 2 studies. For the phase 2 study, some children had severe symptoms and some children did not. The group of children with severe symptoms is called group 1, and their results are included in this summary. The researchers monitored the participating children for up to 5 years in a long-term study to better understand how the treatment works over time. The NF1 caregivers experience study is a related study where caregivers shared their experiences of caring for children with NF1and plexiform neurofibromas.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;What were the results?: &lt;/strong&gt;A total of 74 children took part in the SPRINT phase 1 and phase 2 (group 1) study. Their ages ranged from 3 to 18.5 years, and their average age was 10.3 years. After more than 4 years of treatment, around 70% of the children (52 out of 74) had smaller tumors. For most children, the responses lasted beyond 1 year. There was a significant and lasting reduction in the intensity of the children';s tumor pain, noticeable as early as 2 months after starting the treatment. After 12 months, children reported their pain dropped from an average score of 2.2 to 0.6 and stayed low at 0.58 over 4 years. There was also an improvement in how much their pain affected the children's ability to do daily tasks. Some children had side effects related to selumetinib, although these were generally manageable. Results from the NF1 caregivers experience study showed caregivers of children with plexiform neurofibromas face significant impacts in physical, psychological, economic, and social aspects. These effects often result in a loss of productivity and difficulties with daily activity.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;What do the results of the study mean?: &lt;/strong&gt;Children with NF1 who have symptomatic, inoperable plexiform neurofibromas can benefit from selumetinib treatment. Selumetinib is general","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240184"},"PeriodicalIF":1.9,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11864077/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143006398","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}