Joel Raskin, Anita H Clayton, Susan G Kornstein, George I Papakostas, Yuki Prescott, Kelly Abernathy, John Hall, Michael Ackermann, William Wargin, Greg Rigdon, Valerie Arnold, Roberta Ball, Elan Cohen, Donald J Garcia, Mark DiBuono, Michael Downing, Otto Dueno, Beal Essink, Corinna Gamez, Haig Goenjian, Michael Greenbaum, Paul Gross, Willis Holloway, John Mark Joyce, George Konis, Jelena Kunovac, Mark Lerman, Elia Acevedo-Diaz, Mustafa Rawaf, Leon Rosenberg, Lara Shirikjian, Rishi Kakar, Felipe Suplicy, Drissana Tran, Nick Vatakis, Judith Joseph, James Knutson, Kurian Abraham, Lawrence Ginsberg, Gregory Mattingly, Eric Chavez, Saundra Maass-Robinson, Andrew Sedillo, Benny Barnhart
{"title":"A Phase 2, multicenter, double-blind, randomized, placebo-controlled study of the safety and efficacy of forvisirvat (SP-624) in the treatment of adults with major depressive disorder.","authors":"Joel Raskin, Anita H Clayton, Susan G Kornstein, George I Papakostas, Yuki Prescott, Kelly Abernathy, John Hall, Michael Ackermann, William Wargin, Greg Rigdon, Valerie Arnold, Roberta Ball, Elan Cohen, Donald J Garcia, Mark DiBuono, Michael Downing, Otto Dueno, Beal Essink, Corinna Gamez, Haig Goenjian, Michael Greenbaum, Paul Gross, Willis Holloway, John Mark Joyce, George Konis, Jelena Kunovac, Mark Lerman, Elia Acevedo-Diaz, Mustafa Rawaf, Leon Rosenberg, Lara Shirikjian, Rishi Kakar, Felipe Suplicy, Drissana Tran, Nick Vatakis, Judith Joseph, James Knutson, Kurian Abraham, Lawrence Ginsberg, Gregory Mattingly, Eric Chavez, Saundra Maass-Robinson, Andrew Sedillo, Benny Barnhart","doi":"10.1080/03007995.2025.2574465","DOIUrl":"https://doi.org/10.1080/03007995.2025.2574465","url":null,"abstract":"<p><strong>Objective: </strong>Forvisirvat (SP-624) is an orally-administered epigenetic sirtuin 6 (SIRT6) activator with antidepressant effects in animal models that was well tolerated in three phase 1 trials in healthy adults. This phase 2 clinical study, SP-624-201, was designed to evaluate the safety and efficacy of forvisirvat 20 mg daily for 4 weeks in participants with major depressive disorder.</p><p><strong>Methods: </strong>SP-624-201 (NCT04479852) was a double-blind, placebo-controlled study. Participants were adults who met DSM-5 criteria for moderate to severe major depressive disorder, as confirmed by the Mini International Neuropsychiatric Interview. Participants receiving psychoactive medications or psychoactive supplements including antidepressants and mood stabilizers, were required to discontinue these medications and wait at least five half-lives of the medications before receiving forvisirvat. Primary endpoint was change from baseline to Week 4 in Montgomery Asberg Depression Rating Scale score. Participants were randomized to forvisirvat 20 mg daily (N = 163) or placebo (N = 156).</p><p><strong>Results: </strong>Of the 319 patients enrolled in the study, 319 (70.2%) were White and 211 (66.1%) were female. Mean age across subgroups ranged from 41.4 to 44.4 years. No significant difference in the primary endpoint was observed between treatment groups. However, the first post-hoc analysis conducted found that women treated with forvisirvat experienced significant overall improvement whereas men treated with forvisirvat did not. The difference between sexes was consistent for secondary efficacy measures as well. No serious adverse events were reported for forvisirvat-treated participants. The most frequent treatment-emergent event was headache (forvisirvat: 8.1%, placebo: 11.5%). Six of 163 forvisirvat-treated participants and 5 of 156 participants who received placebo discontinued due to adverse events.</p><p><strong>Conclusions: </strong>The novel epigenetic mechanism of action of forvisirvat, favorable safety profile, and consistent post-hoc efficacy results in women observed in this study support further development of forvisirvat. A phase 2b/3 trial of forvisirvat in major depressive disorder (NCT06254612), to confirm these results, is ongoing.</p>","PeriodicalId":10814,"journal":{"name":"Current Medical Research and Opinion","volume":" ","pages":"1-16"},"PeriodicalIF":2.2,"publicationDate":"2025-10-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145298990","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Sarah A Pollick, Kate L Wilson, Elizabeth C Lloyd, Sarah L Reeves, Megan H Pesch
{"title":"The validity of ICD-based codes to identify pediatric cases of congenital cytomegalovirus.","authors":"Sarah A Pollick, Kate L Wilson, Elizabeth C Lloyd, Sarah L Reeves, Megan H Pesch","doi":"10.1080/03007995.2025.2564340","DOIUrl":"10.1080/03007995.2025.2564340","url":null,"abstract":"<p><strong>Objective: </strong>Administrative claims databases are used to study the care of congenital cytomegalovirus (cCMV), yet the use of International Classification of Diseases, (ICD-9/10) codes for cCMV have not been validated. This study examines the accuracy of ICD-based codes for cCMV infection.</p><p><strong>Methods: </strong>Infants cared for at a quaternary children's hospital (2013-2023) that had an ICD-based diagnosis for cCMV or CMV Infection at ≤90 days of age were included. Medical record data was abstracted. True Positive cases were defined as those with an ICD code AND clinical and laboratory evidence consistent with cCMV. False Positive cases were defined as those with an ICD code without evidence of cCMV. Positive predictive value (PPV) and sensitivity for each diagnostic code at different age cutoffs were calculated within the cohort. Multinomial regression examined characteristics of the infant with odds of being a True Positive case of cCMV.</p><p><strong>Results: </strong>Of the 108 infants with ICD-9/10 codes for cCMV, 35% were false positives. PPV for ICD-9/10-CM codes for cCMV, CMV Infection, and Either code predicting actual cCMV were 0.86, 0.36, and 0.68 at age ≤45 days. PPV was the highest at ≤21 days of age, and for all codes sensitivity increased with patient age. Multinomial logistic regression found the age of the first diagnostic code ≤21 days (vs. >) (OR = 4.11, 95% CI 1.45-12.03), having an ICD-9/10-CM diagnostic code of cCMV (vs. CMV Infection) (OR = 10.87, 95% CI 3.64-32.47), and having Clinical Signs at Birth (vs. none) (OR = 8.4, 95% CI 2.72-25.81) to be associated with greater odds of having a True Positive case of cCMV (vs. Not cCMV).</p><p><strong>Conclusions: </strong>Administrative claims case definitions for cCMV were more likely to be accurate when assigned at a younger age. Studies using case definitions for cCMV that include the presence of codes for either cCMV or CMV Infection may be biased given the high proportion of false positives demonstrated in this study.</p>","PeriodicalId":10814,"journal":{"name":"Current Medical Research and Opinion","volume":" ","pages":"1-10"},"PeriodicalIF":2.2,"publicationDate":"2025-10-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145257623","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Sigal Kaplan, Henok Tadesse Ayele, Dimitri Bennett, Sonia M Grandi, Stephen E Schachterle, Jason C Simeone, Jenny W Sun, Ugochinyere Vivian Ukah
{"title":"Unlocking insights from mother-infant linked data in pharmacoepidemiology: opportunities, challenges, and future directions.","authors":"Sigal Kaplan, Henok Tadesse Ayele, Dimitri Bennett, Sonia M Grandi, Stephen E Schachterle, Jason C Simeone, Jenny W Sun, Ugochinyere Vivian Ukah","doi":"10.1080/03007995.2025.2573652","DOIUrl":"https://doi.org/10.1080/03007995.2025.2573652","url":null,"abstract":"<p><p>Pregnant women and newborns are historically underrepresented in clinical trials, creating critical gaps in evidence on the safety and effectiveness of medications used during pregnancy. Real-world data (RWD) sources offer a promising avenue to address these gaps. To fully realize this potential, it is essential to link maternal and infant records accurately within and across diverse datasets. High-quality mother-infant linkage enables the robust evaluation of maternal medication use and its short- and long-term effects on both maternal and infant health. However, linking maternal and infant healthcare data introduces complex methodological and practical challenges. Achieving accurate linkage is often hindered by factors such as inconsistent personal identifiers, discrepancies in insurance coverage between mother and infant, data incompleteness, algorithmic accuracy, and strict data privacy regulations. Commonly used proxies for linkage (e.g. shared address or healthcare provider) may also be unreliable and can introduce misclassification or duplication.This commentary synthesizes current knowledge on mother-infant data linkage in RWD. It also systematically outlines the key challenges, emerging opportunities, and strategic directions to improve linkage quality and address privacy concerns in the identification of mother-infant dyads to support rigorous pharmacoepidemiologic research on maternal and infant health outcomes.By improving linkage methods and leveraging innovative approaches such as tokenization and validated algorithms, researchers can enhance the reliability of real-world evidence on maternal and infant health outcomes, including long-term follow-up across diverse data sources. Advancing these methodological frontiers is essential to generate evidence that supports safer and more informed treatment decisions for pregnant women and their children.</p>","PeriodicalId":10814,"journal":{"name":"Current Medical Research and Opinion","volume":" ","pages":"1-17"},"PeriodicalIF":2.2,"publicationDate":"2025-10-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145274119","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
S Collings, Amisha Patel, Hannah Gowman, Sarah Law, Aideen Ahern, Robert Pawinski, Robert Wood, Shazia K Afridi
{"title":"The cost of migraine to the NHS in England: a retrospective cohort study using electronic health record data from Clinical Practice Research Datalink (CPRD) and Hospital Episode Statistics (HES).","authors":"S Collings, Amisha Patel, Hannah Gowman, Sarah Law, Aideen Ahern, Robert Pawinski, Robert Wood, Shazia K Afridi","doi":"10.1080/03007995.2025.2571036","DOIUrl":"10.1080/03007995.2025.2571036","url":null,"abstract":"<p><strong>Objectives: </strong>To quantify healthcare resource utilization (HCRU) and associated direct medical costs of migraine among adults in England compared with general population controls.</p><p><strong>Methods: </strong>This retrospective cohort study used linked primary and secondary healthcare data (Clinical Practice Research Datalink Aurum [CPRD] and Hospital Episode Statistics [HES]) in England. Migraine cases (adults newly diagnosed with migraine between 1-Jan-2017 and 31-Mar-2019) and general population controls were matched on age, sex, ethnicity, practice region, Index of Multiple Deprivation 2019, and baseline depression and cardiovascular disease. All-cause HCRU and costs were estimated for cases and compared with controls using Fisher's Exact and Mann-Whitney U tests. Subgroup analyses were conducted by migraine treatment group (acute only, preventative only, acute and preventative, no treatment).</p><p><strong>Results: </strong>A total of 62,143 migraine cases and matched controls were identified, of which 76.2% (<i>n</i> = 47,376) were female and the mean [SD] age at index was 39.8 [15.5] years. All-cause direct medical costs per-person per-year (pppy) were approximately 81% greater (<i>p</i> < 0.0001) in those with migraine (median [Q1, Q3]: £492 [£205, £1,149]) than controls (£272 [£101, £716]). The acute and preventative treatment subgroup had the highest all-cause direct costs pppy (£925 [£430, £2,046]).</p><p><strong>Conclusion: </strong>Overall, patients diagnosed with migraine contribute a substantially larger cost burden on the healthcare system than their matched controls, with costs largely driven by interactions in secondary care. Costs varied depending on types of migraine treatment received and may reflect differences in migraine presentation, such as chronicity and patient needs, and potentially suboptimal management of migraine in primary care.</p>","PeriodicalId":10814,"journal":{"name":"Current Medical Research and Opinion","volume":" ","pages":"1-13"},"PeriodicalIF":2.2,"publicationDate":"2025-10-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145243858","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Boteng Yan, Chaoyan Tang, Shengzhu Huang, Yushuang Wei, Mingjie Xu, Xihui Jin, Xiaoyou Mai, Lingyu Ye, Zengnan Mo, Mingli Li
{"title":"Elevated plasma aldosterone concentrations relate to renal impairment in Chinese southwestern people.","authors":"Boteng Yan, Chaoyan Tang, Shengzhu Huang, Yushuang Wei, Mingjie Xu, Xihui Jin, Xiaoyou Mai, Lingyu Ye, Zengnan Mo, Mingli Li","doi":"10.1080/03007995.2025.2565441","DOIUrl":"10.1080/03007995.2025.2565441","url":null,"abstract":"<p><strong>Background: </strong>Evidence on the biochemical phenotypes of primary aldosteronism and their link to renal function in Chinese populations is limited. This study explored the associations of plasma aldosterone concentration (PAC), plasma renin concentration (PRC), and the aldosterone-to-renin ratio (ARR) with renal function.</p><p><strong>Methods: </strong>In this cross-sectional study of 1700 participants from Southwest China, associations between PAC, PRC, ARR, and renal parameters (serum creatinine [SCR], blood urea nitrogen [BUN], estimated glomerular filtration rate [eGFR]) were analyzed using a generalized linear mixed model. Dose-response relationships were assessed with P-trend and restricted cubic spline (RCS) analyses. A structural equation model evaluated the mediating effect of blood pressure.</p><p><strong>Results: </strong>Higher log-transformed PAC was associated with elevated SCR (β = 0.451, 95% CI: 0.329, 0.573) and BUN (β = 0.370, 95% CI: 0.278, 0.462), and reduced eGFR (β = -0.263, 95% CI: -0.331, -0.194). RCS analyses confirmed linear dose-response relationships. These associations were more pronounced in males than females. Renin-Angiotensin-Aldosterone System (RAS) inhibitor use mitigated the positive association between PAC and BUN. Systolic blood pressure mediated a small proportion of the effect of PAC on SCR (2.4%) and eGFR (2.6%).</p><p><strong>Conclusion: </strong>A continuous spectrum of PAC is associated with renal impairment in this Chinese cohort, with stronger effects observed in males. The use of RAS inhibitors may attenuate this aldosterone-related renal impairment.</p>","PeriodicalId":10814,"journal":{"name":"Current Medical Research and Opinion","volume":" ","pages":"1-9"},"PeriodicalIF":2.2,"publicationDate":"2025-10-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145147984","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Marc Messier, James H Stark, Laura Wagner, Erik Lamberth
{"title":"Antibody response and safety through 12 months after booster vaccination with an investigational Lyme disease vaccine in adults: a plain language summary.","authors":"Marc Messier, James H Stark, Laura Wagner, Erik Lamberth","doi":"10.1080/03007995.2025.2564929","DOIUrl":"https://doi.org/10.1080/03007995.2025.2564929","url":null,"abstract":"","PeriodicalId":10814,"journal":{"name":"Current Medical Research and Opinion","volume":" ","pages":"1-2"},"PeriodicalIF":2.2,"publicationDate":"2025-10-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145238512","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Mallik Greene, Shrey Gohil, Brad Stieber, A Burak Ozbay, Quang A Le, Raja Kakuturu, Joseph W LeMaster, Michael Dore, A Mark Fendrick, Joseph C Anderson, Jordan J Karlitz
{"title":"Utilization of colorectal cancer screening modalities in the United States (2017-2023): a national multi-payer claims database analysis.","authors":"Mallik Greene, Shrey Gohil, Brad Stieber, A Burak Ozbay, Quang A Le, Raja Kakuturu, Joseph W LeMaster, Michael Dore, A Mark Fendrick, Joseph C Anderson, Jordan J Karlitz","doi":"10.1080/03007995.2025.2565442","DOIUrl":"10.1080/03007995.2025.2565442","url":null,"abstract":"<p><strong>Background: </strong>Colorectal cancer (CRC) is the second leading cause of U.S. cancer mortality. This study evaluated the utilization of CRC screening modalities from 2017 to 2023.</p><p><strong>Methods: </strong>This retrospective, cross-sectional study analyzed data from 2017 to 2023 using a national multi-payer claims database, supplemented with laboratory data related to CRC screening. Patients aged 45-75 years, at average risk for CRC, with no prior CRC diagnosis, and who had continuous health insurance enrollment for 24 months (from January 1 of the baseline year to December 31 of the study year) were included. Annual proportions for colonoscopy, multi-target stool DNA (mt-sDNA) test, fecal immunochemical test/fecal occult blood test (FIT/FOBT), and other modalities were assessed, along with sociodemographic factors. Descriptive statistics and chi-square tests were used to assess utilization trends across the years.</p><p><strong>Results: </strong>Colonoscopy remained the most commonly used screening modality, with its share increasing slightly from 53.0% in 2017 to 58.7% in 2023. The mt-sDNA test proportion increased significantly from 2.4% in 2017 to 20.4% in 2023, while the proportion of FIT/FOBT declined significantly, from 44% to 20.4%. Similarly, significant age-related shifts in screening utilization were observed, with colonoscopy proportion increasing from 48.1% to 61.6%, mt-sDNA rising from 0.0% to 24.0%, and FIT/FOBT declining from 50.8% to 14.1% in the 45-49 age group from 2017 to 2023.</p><p><strong>Conclusion: </strong>CRC screening utilization shifted significantly from 2017 to 2023, with increased colonoscopy and mt-sDNA use and a marked decline in FIT/FOBT. Continued monitoring is critical to ensure equitable access to effective modalities.</p>","PeriodicalId":10814,"journal":{"name":"Current Medical Research and Opinion","volume":" ","pages":"1-10"},"PeriodicalIF":2.2,"publicationDate":"2025-10-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145136694","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"The role of serum phosphate control in supporting transplant readiness in patients on dialysis.","authors":"Laura A Williams, Stephen Z Fadem","doi":"10.1080/03007995.2025.2563377","DOIUrl":"10.1080/03007995.2025.2563377","url":null,"abstract":"<p><p>Chronic kidney disease affects over 36 million Americans, with more than 800,000 progressing to end-stage kidney disease (ESKD). Treatment with kidney transplantation offers superior survival, quality of life, and cost-effectiveness compared with dialysis, yet access remains limited. Hyperphosphatemia is a highly prevalent and modifiable complication of ESKD that contributes to cardiovascular disease, mineral bone disorder, and potential pulmonary dysfunction. Additionally, elevated serum phosphate has been linked to transplant graft failure and adverse post-transplant outcomes. Although normal phosphate levels are not formally required for transplant eligibility, they are frequently considered as part of transplant readiness assessments. Persistent gaps between clinical guideline recommendations and real-world serum phosphate control, despite dialysis, dietary restrictions, and phosphate binder therapy, underscore the need for individualized and more effective treatment strategies. Because transplant candidacy may be influenced by serum phosphate levels, phosphate management should be viewed not just as correction of a laboratory abnormality, but as a strategy to improve transplant access, promote health equity, and enhance long-term outcomes. Real-world tracking of transplant readiness metrics by serum phosphate levels will be essential to assessing the impact of new therapies and support transparent and equitable organ allocation. Effective phosphate management may reduce time on dialysis, improve graft and patient survival, and lower healthcare costs. In this context, managing hyperphosphatemia is a clinical and strategic imperative in advancing kidney health.</p>","PeriodicalId":10814,"journal":{"name":"Current Medical Research and Opinion","volume":" ","pages":"1-4"},"PeriodicalIF":2.2,"publicationDate":"2025-09-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145085302","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"2025 National Association of Specialty Pharmacy Annual Meeting & Expo: research presentation abstracts.","authors":"Sheila Arquette, Rebekah H Anguiano","doi":"10.1080/03007995.2025.2529096","DOIUrl":"https://doi.org/10.1080/03007995.2025.2529096","url":null,"abstract":"","PeriodicalId":10814,"journal":{"name":"Current Medical Research and Opinion","volume":"41 sup2","pages":"1"},"PeriodicalIF":2.2,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145058582","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Jason Shafrin, Nadine Zawadzki, Moises Marin, Ivana Audhya, Lauren E Sedita, Natasha Kulkarni, Alexa C Klimchak
{"title":"Caregiver risk preferences for delaying loss of ambulation in Duchenne muscular dystrophy in the United States.","authors":"Jason Shafrin, Nadine Zawadzki, Moises Marin, Ivana Audhya, Lauren E Sedita, Natasha Kulkarni, Alexa C Klimchak","doi":"10.1080/03007995.2025.2550347","DOIUrl":"10.1080/03007995.2025.2550347","url":null,"abstract":"<p><strong>Objectives: </strong>Quantify caregiver risk preferences to inform the \"value of hope\" for Duchenne muscular dystrophy (DMD) therapies affecting time to loss of ambulation (LoA).</p><p><strong>Methods: </strong>Caregivers (medical decision-makers) of patients with DMD were surveyed to evaluate their preferences across 2 therapies with identical expected (average) time to LoA: 1 with variable (i.e. possibly longer or shorter than average) time to LoA and 1 with fixed (i.e. certain) time to LoA. Time to LoA with the fixed therapy was altered to determine the caregiver's indifference point. Study endpoints were (i) the share of caregivers who preferred the variable (vs fixed) time to LoA therapy; and (ii) the length of fixed time to LoA that would result in caregiver indifference between the variable and fixed therapies, calculated using parameter estimation by sequential testing. The base case examined therapy choice for a hypothetical ambulatory DMD patient aged 9 years; sensitivity analyses explored preferences for younger (aged 5) and older (aged 13) patients.</p><p><strong>Results: </strong>Among 103 caregivers surveyed, 72 (69.9%) preferred the variable time to LoA therapy for a hypothetical 9-year-old patient with DMD (<i>p</i> < 0.001). Caregivers were willing to give up 11.5 months (<i>p</i> < 0.001) of certain time to LoA for a chance of longer time to LoA. Caregivers' preference for the variable therapy decreased with hypothetical patient age at treatment initiation, from 72.8% (75/103) for age 5 (<i>p</i> < 0.001) to 60.2% (62/103) for age 13 (<i>p</i> = 0.048).</p><p><strong>Conclusions: </strong>Caregivers of patients with DMD demonstrated risk tolerance (positive value of hope) for therapies that could delay LoA.</p>","PeriodicalId":10814,"journal":{"name":"Current Medical Research and Opinion","volume":" ","pages":"1499-1509"},"PeriodicalIF":2.2,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144945515","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}