Advances in Therapy最新文献

{"title":"Safety of iGlarLixi in Japanese People with Type 2 Diabetes: A Post-marketing Database Study","authors":"Hideaki Kaneto,&nbsp;Makiko Hatanaka,&nbsp;Yukiko Morimoto,&nbsp;Yoko Takahashi,&nbsp;Yasuo Terauchi","doi":"10.1007/s12325-025-03135-5","DOIUrl":"10.1007/s12325-025-03135-5","url":null,"abstract":"<div><h3>Introduction</h3><p>In this post-marketing study in Japan, the occurrence of hospital-treated hypoglycaemia and severe hyperglycaemia requiring inpatient treatment was evaluated in various cohorts of people with type 2 diabetes (T2D) newly switched to iGlarLixi, a titratable, once-daily, fixed-ratio combination of long-acting insulin glargine 100 U/mL (iGlar-100) and a glucagon-like peptide-1 receptor agonist (GLP-1 RA, lixisenatide).</p><h3>Methods</h3><p>In this retrospective, observational study, acute-care hospital data from adults with T2D were analysed from the Medical Data Vision database. In Cohort 1, the incidence rate of hospital-treated hypoglycaemia following newly prescribed iGlarLixi versus iGlar-100 was assessed. Cohort 2 was subdivided to evaluate the incidence rate of hospital-treated hypoglycaemia and severe hyperglycaemia requiring inpatient treatment in people switched to iGlarLixi from either a GLP-1 RA ± oral antidiabetic drugs (OADs) or OADs alone (Cohort 2A) or from a GLP-1 RA and long-acting insulin ± OADs or long-acting insulin ± OADs (Cohort 2B).</p><h3>Results</h3><p>Of the 438 people in the iGlarLixi group and 9295 people in the iGlar-100 group in Cohort 1, who had a median follow-up duration of 52 and 44 days, respectively, there were zero and 0.011 (95% CI 0.006–0.018) events per person-year of hospital-treated hypoglycaemia, respectively. Cohort 2A included 201 people each in the GLP-1 RA ± OADs and OADs alone groups, with a median follow-up duration of 76 and 101 days, respectively, and Cohort 2B included 255 people in the GLP-1 RA and long-acting insulin ± OADs group and 623 people in the long-acting insulin ± OADs group, with a median follow-up duration of 73 and 62 days, respectively; no cases of hospital-treated hypoglycaemia or severe hyperglycaemia requiring inpatient treatment were observed.</p><h3>Conclusion</h3><p>Consistent with clinical trials, this post-marketing database study observed that newly prescribed iGlarLixi has a low risk of serious hypoglycaemia or hyperglycaemia in Japanese people with T2D, irrespective of prior antidiabetic drug treatment.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"42 5","pages":"2168 - 2189"},"PeriodicalIF":3.4,"publicationDate":"2025-03-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-025-03135-5.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143582161","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A Multicenter Single-Arm Study of Switching to Ferric Citrate Hydrate for Iron Deficiency Anemia in Patients Intolerant to Oral Iron: RIO-SWITCH","authors":"Osamu Wada-Hiraike,&nbsp;Aya Maruyama,&nbsp;Yuko Mitobe,&nbsp;Takayuki Iriyama,&nbsp;Mayuyo Mori-Uchino,&nbsp;Yutaka Osuga,&nbsp;RIO-SWITCH Consortium","doi":"10.1007/s12325-025-03123-9","DOIUrl":"10.1007/s12325-025-03123-9","url":null,"abstract":"<div><h3>Introduction</h3><p>Treatment interruptions due to adverse drug reactions (ADRs) are common in iron deficiency anemia (IDA). We evaluated medication completion rates and quality of life (QoL) changes in patients with IDA after starting ferric citrate hydrate (FCH) treatment.</p><h3>Methods</h3><p>This multicenter, open-label, uncontrolled, single-arm comparative study included 30 Japanese female patients with IDA who experienced nausea and/or vomiting (N/V) with previous oral iron preparations. Patients received FCH 500 mg orally daily (&lt; 1000 mg/day). Those with hemoglobin levels ≥ 11.0 g/dl completed the study at week 4; others continued until week 8. The primary endpoint was medication completion rate. Secondary endpoints included medication compliance rate, treatment satisfaction scores, and QoL scores and changes.</p><h3>Results</h3><p>Thirty patients initiated and completed treatment; 24 reached ≥ 11.0 g/dl hemoglobin at week 4 and ended treatment, while 6 continued until week 8. The medication compliance rate was 93.92% ± 8.11% (mean ± standard deviation [SD]), and the completion rate was 100.0% (95% confidence interval 88.4–100.0%). Questionnaire findings revealed that the most severe nausea score decreased from 5.7 ± 2.4 to 1.7 ± 2.1 (mean ± SD), N/V incidence decreased from 100.0 to 63.3%, and patients reporting that N/V did not interfere with daily life increased from 6.7 to 52.6% following the switch to FCH. Twenty-four patients (80.0%) reported a satisfactory experience with FCH versus their previous oral iron preparation. Scores for all eight subscales of the Short-Form 36-Item Health Survey v2 improved, with significant increases in six. Nine ADR events occurred in six patients (20.0%), including nausea in three (10.0%); none were serious or resulted in treatment discontinuation.</p><h3>Conclusion</h3><p>FCH treatment exhibited a satisfactory medication completion rate in patients with IDA. Switching to FCH reduced N/V incidence and improved N/V and QoL severity compared with previous oral iron preparations.</p><h3>Trial Registration</h3><p>jRCTs031210634. Registration date: March 01, 2022.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"42 5","pages":"2150 - 2167"},"PeriodicalIF":3.4,"publicationDate":"2025-03-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-025-03123-9.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143571746","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Efficacy and Safety of Recombinant Factor VIII in Previously Untreated and Previously Treated Children with Hemophilia A: A Systematic Review","authors":"Xiaoqin Feng,&nbsp;Xuan Zhou,&nbsp;Jing Sun,&nbsp;Zhenguo Wang","doi":"10.1007/s12325-025-03110-0","DOIUrl":"10.1007/s12325-025-03110-0","url":null,"abstract":"<div><h3>Introduction</h3><p>Inhibitor development is a primary concern for pediatric patients with hemophilia A (HA) undergoing recombinant factor VIII (rFVIII) therapy, yet relevant research is lacking. We aimed to compare the efficacy and safety of standard (SHL) and extended half-life (EHL) rFVIII products in previously treated (PTPs) and untreated (PUPs) pediatric patients with HA.</p><h3>Methods</h3><p>Following PRISMA guidelines, we searched clinical studies from PubMed, Embase, and Cochrane Library. Data were extracted and a single-arm meta-analysis was performed.</p><h3>Results</h3><p>This systematic review included 16 studies involving 1145 patients. Three studies reported changes in annual bleeding rate (ABR); their results displayed no statistically significant difference in ABR changes in pediatric patients with HA after rFVIII treatment. Ten studies reported inhibitor development, nine focused on PUPs. Here, EHL rFVIII showed a proportion of inhibitors at 27.5% (95% confidence interval [CI] 22.6%; 32.6%), and third-generation SHL rFVIII showed a proportion of inhibitors at 36.4% (27.2%; 46.2%), with a high-titer proportion of 20.9% (12.9%; 30.3%) for the latter. Both SHL rFVIII (octocog alfa) and EHL rFVIII (rurioctocog alfa pegol) presented low proportions of inhibitor development. Octocog alfa exhibited the lowest high-titer inhibitor incidence, marked at 12.7% (5.3%; 24.5%). Eleven studies addressed adverse events (AEs), with octocog alfa showing low reported treatment-related AEs at a proportion of 14.5% (6.5%; 26.7%).</p><h3>Conclusion</h3><p>Our analysis revealed that both octocog alfa and rurioctocog alfa pegol showed low inhibitor development, with octocog alfa having few treatment-related AEs. Regular monitoring for inhibitors during rFVIII therapy is important.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"42 5","pages":"2019 - 2039"},"PeriodicalIF":3.4,"publicationDate":"2025-03-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-025-03110-0.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143565756","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Letter to the Editor Regarding “Predicting Asthma Exacerbations Using Machine Learning Models”","authors":"Zekai Yu","doi":"10.1007/s12325-025-03119-5","DOIUrl":"10.1007/s12325-025-03119-5","url":null,"abstract":"","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"42 5","pages":"2537 - 2538"},"PeriodicalIF":3.4,"publicationDate":"2025-03-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-025-03119-5.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143565757","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Summary of Research: Efficacy of Trastuzumab Deruxtecan in HER2-Expressing Solid Tumors by Enrollment HER2 IHC Status: Post Hoc Analysis of DESTINY-PanTumor02","authors":"Ana Oaknin,&nbsp;Jung-Yun Lee,&nbsp;Vicky Makker,&nbsp;Do-Youn Oh,&nbsp;Susana Banerjee,&nbsp;Antonio González-Martín,&nbsp;Kyung Hae Jung,&nbsp;Iwona Ługowska,&nbsp;Luis Manso,&nbsp;Aránzazu Manzano,&nbsp;Bohuslav Melichar,&nbsp;Salvatore Siena,&nbsp;Daniil Stroyakovskiy,&nbsp;Anitra Fielding,&nbsp;Soham Puvvada,&nbsp;Ann Smith,&nbsp;Funda Meric-Bernstam","doi":"10.1007/s12325-024-03080-9","DOIUrl":"10.1007/s12325-024-03080-9","url":null,"abstract":"<div><p>Summary of the original article, ‘Efficacy of Trastuzumab Deruxtecan in HER2-Expressing Solid Tumors by Enrollment HER2 IHC Status: Post Hoc Analysis of DESTINY-PanTumor02’. Trastuzumab deruxtecan (T-DXd) is an antibody-drug conjugate, which is a chemotherapy with a linker (deruxtecan) joined to an antibody (trastuzumab). Trastuzumab binds to the human epidermal growth factor receptor 2 (HER2) protein on cancer cells, where it releases the chemotherapy to kill these cells. The DESTINY-PanTumor02 clinical study tested the effectiveness of T-DXd for people with various HER2-expressing cancers and the safety of treatment. Previous results from DESTINY-PanTumor02 showed that T-DXd had antitumor activity, and the greatest effects were seen in people with the highest tumor level of HER2 [defined as immunohistochemistry (IHC) 3+]. In this previous analysis, the HER2 expression was measured at a central laboratory. In clinical practice, HER2 expression will likely be measured at a local laboratory, so understanding whether T-DXd has similar effects regardless of how HER2 expression is measured is important. Here, we looked at the effects of T-DXd based on the HER2 test result used to determine a person’s eligibility for the study, which could be measured using a local or central laboratory. In people with IHC 3+ tumors (where HER2 was measured at a local or central laboratory), 51% had a decrease in the size or number of tumors, according to established criteria (referred to as an objective response), while, in people with IHC 2+ tumors, 26% had an objective response. Side effects with T-DXd were consistent with previous studies. These results confirm T-DXd has antitumor effects in HER2-expressing cancers where the HER2 expression is measured by a local or central laboratory.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"42 5","pages":"2015 - 2018"},"PeriodicalIF":3.4,"publicationDate":"2025-03-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-024-03080-9.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143565759","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A Randomised, Double-Blind Trial to Compare the Efficacy, Safety, and Immunogenicity of the Biosimilar Ustekinumab FYB202 with Reference Ustekinumab in Patients with Moderate-to-Severe Plaque Psoriasis","authors":"Kim Papp,&nbsp;Sigrid Balser,&nbsp;Katrin Nopora,&nbsp;Piotr Rewerski,&nbsp;Brigitte Freudensprung,&nbsp;Michael Trieb","doi":"10.1007/s12325-025-03138-2","DOIUrl":"10.1007/s12325-025-03138-2","url":null,"abstract":"<div><h3>Introduction</h3><p>Biosimilars allow more patients access to affordable treatment options and help reduce the financial burden on healthcare systems. This multicentre trial compared the efficacy, safety, and immunogenicity of the approved biosimilar ustekinumab FYB202 with reference ustekinumab.</p><h3>Methods</h3><p>Eligible patients were ≥ 18 years old with stable moderate-to-severe plaque psoriasis for ≥ 6 months and inadequate treatment response to or intolerance of ≥ 1 previous systemic treatment. Patients were randomised (1:1) to double-blind treatment with FYB202 or reference ustekinumab; patients in the reference group who achieved Psoriasis Area and Severity Index (PASI) 75 percent improvement at week 28 were re-randomised to FYB202 or reference product. The primary efficacy endpoint was percent improvement in PASI score from baseline to week 12. Therapeutic equivalence was demonstrated if, depending on the regulatory requirement with respect to the significance level, the two-sided 95% and 90% confidence intervals (CIs) were within the pre-defined equivalence intervals of ± 11% and ± 10%, respectively.</p><h3>Results</h3><p>A total of 392 patients were randomised to FYB202 (<i>n</i> = 197) or reference ustekinumab (<i>n</i> = 195). Baseline characteristics were well balanced between groups. Mean percent improvement in PASI score at week 12 was equivalent between FYB202 and reference ustekinumab with an estimated least-squares mean treatment difference of 3.27% and the two-sided 95% (− 0.90%, 7.44%) and 90% (− 0.22%, 6.77%) CIs fully contained within the pre-defined equivalence margins. Safety and immunogenicity profiles were comparable between groups. Switching from reference product to FYB202 had no clinically relevant effect on efficacy, safety, or immunogenicity.</p><h3>Conclusion</h3><p>FYB202 demonstrated therapeutic equivalence to reference ustekinumab in patients with moderate-to-severe plaque psoriasis.</p><h3>Trial Registration</h3><p>NCT04595409; EudraCT 2019-004364-21.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"42 5","pages":"2135 - 2149"},"PeriodicalIF":3.4,"publicationDate":"2025-03-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-025-03138-2.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143565753","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Characterizing Patient Diversity via Healthcare Access Determinants: A New Approach for Measuring Improvements in Clinical Trial Diversity in the United States","authors":"Jeffrey Yu,&nbsp;Adrian Kielhorn,&nbsp;James Murdoch,&nbsp;Marcus Martin,&nbsp;Eddilisa Martin,&nbsp;Kelly McNeil-Posey,&nbsp;Barbara Mungin,&nbsp;Yiyi Xia,&nbsp;Wendy Erler,&nbsp;Nuwan C. Kurukulasuriya","doi":"10.1007/s12325-025-03140-8","DOIUrl":"10.1007/s12325-025-03140-8","url":null,"abstract":"<div><h3>Introduction</h3><p>Racial and ethnic minorities are frequently under-represented in biomedical research in the United States (US), and the under-representation is amplified in clinical trials in patients with rare diseases. The REthinking MeAsures of DivErsity (REMADE) study was conducted to develop and test a set of questions that may more accurately capture the diversity of patients via socioeconomic, cultural, and ethnic parameters.</p><h3>Methods</h3><p>A web-based survey was developed to assess race, ethnicity/culture, socioeconomic status, disability/mobility, and transportation issues. The survey responses included 5 racial categories as well as 17 cultures, heritages, and/or ethnicities and were multiselect. The survey was tested in US adults from under-represented populations. Survey results were compared with data collected with a pre-survey intake form (PSIF) that utilized historical categories for race and ethnicity.</p><h3>Results</h3><p>Of 219 total survey respondents, 59.8% (131/219) were assigned female sex at birth and 51.1% (112/219) were aged ≥ 18 to &lt; 30 years. Respondents reported being predominantly Black [77.3% [163/211)] or white [19.0% (40/211)] in the PSIF. When respondents were allowed to assign percentages across multiple categories in the survey, only 34.2% (75/219) and 10.5% (23/219) identified as 100% Black or white, respectively. As with race, the REMADE ethnicity/cultural categories revealed greater diversity in the respondent population.</p><h3>Conclusions</h3><p>The REMADE survey results suggest that race and cultural identity are more multidimensional than historical questions/categories were able to capture. These insights, along with those generated on socioeconomic, disability, and transportation issues, will guide initiatives to support fair and equitable representation in clinical trials.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"42 4","pages":"1965 - 1978"},"PeriodicalIF":3.4,"publicationDate":"2025-03-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-025-03140-8.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143536411","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Biologic Treatment Adherence and Persistence in Patients with Palmoplantar Pustulosis: A Real-World, Claims-Based Study","authors":"Steven R. Feldman,&nbsp;Ran Gao,&nbsp;Rhonda L. Bohn,&nbsp;Stephani Gray,&nbsp;Anouk Déruaz-Luyet,&nbsp;Jashin J. Wu","doi":"10.1007/s12325-025-03122-w","DOIUrl":"10.1007/s12325-025-03122-w","url":null,"abstract":"<div><h3>Introduction</h3><p>Palmoplantar pustulosis (PPP) is a debilitating skin condition characterized by pustules, erythema, and scales on the palms and soles. Treatment adherence in psoriatic diseases is suboptimal.</p><h3>Methods</h3><p>A retrospective cohort study, using healthcare claims data from patients aged ≥ 18 years with newly diagnosed PPP (selected between October 1, 2016 and March 31, 2020 from IBM^® MarketScan^® Commercial and the Optum^® Clinformatics^® Data Mart databases), was conducted to investigate adherence to and persistence with biologics in patients with PPP.</p><h3>Results</h3><p>Biologics were dispensed to 114/840 (13.6%) MarketScan and 76/750 (10.1%) Optum patients. Mean proportion of days covered (PDC; range) for biologics was similar between databases (MarketScan, 66% [8–100%]; Optum, 61% [8–99%]), and good adherence (≥ 80% PDC) was infrequent (MarketScan, 42.1%; Optum, 34.2%). Mean (standard deviation) persistence was slightly longer in MarketScan (283 [121] days) versus Optum (275 [133] days). Mean (range) adherence was similar between ages, although persistence was longer in patients aged 18–64 versus ≥ 65 years (279 [132] vs 258 [140] days).</p><h3>Conclusion</h3><p>In patients with PPP, adherence to biologics was poor and persistence was variable; understanding and addressing the reasons behind these observations may improve treatment outcomes.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"42 4","pages":"1994 - 2002"},"PeriodicalIF":3.4,"publicationDate":"2025-03-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-025-03122-w.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143536391","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Survey on the Initiation of Aripiprazole Once-Monthly via a Two-Injection Start in Adult Patients with Schizophrenia: Experience of European Healthcare Professionals","authors":"Andrea Fagiolini,&nbsp;Karolina Leopold,&nbsp;Sofia Pappa,&nbsp;William J. Cottam,&nbsp;Joe Hickey,&nbsp;Olivia Rogerson,&nbsp;Murat Yildirim,&nbsp;Clodagh Beckham","doi":"10.1007/s12325-025-03130-w","DOIUrl":"10.1007/s12325-025-03130-w","url":null,"abstract":"<div><h3>Introduction</h3><p>Aripiprazole once-monthly 400 mg (AOM 400) is a long-acting injectable (LAI) for the maintenance treatment of adults with schizophrenia. The AOM 400 two-injection start initiation regimen (AOM 400-TIS) is an alternative to treatment initiation with one injection of AOM 400 plus 14 days of oral aripiprazole supplementation. This survey investigated the real-world experiences of European healthcare professionals (HCPs) with AOM 400-TIS.</p><h3>Methods</h3><p>Physicians and nurses in Germany, Italy, and the United Kingdom who had prescribed and/or administered AOM 400-TIS ≥ 3 times to patients with schizophrenia were invited to participate in an online survey. The primary objective was to investigate HCPs’ experiences and satisfaction with AOM 400-TIS. Descriptive analysis was performed on data collected between 1 February and 21 March 2024.</p><h3>Results</h3><p>Data from 94 HCPs were analysed. Most were psychiatrists (62.8%) or psychiatric nurses (29.8%) who worked in specialist mental health clinics/centres (59.6%) or hospitals (inpatient, 36.2%; outpatient, 23.4%). Median duration in clinical practice was 21.0 years. Common reasons for initiating AOM 400-TIS were poor adherence (85.1%), relapse (59.6%), and high hospitalisation rates (48.9%). Common prescribing goals for AOM 400-TIS included improving adherence (70.2%), preventing relapses (69.1%), and improving patient quality of life (62.8%). Barriers to AOM 400-TIS were patients not wanting two injections (66.0%), tolerability concerns (30.9%), and safety concerns regarding administration of a high dose on a single day (28.7%). Key factors influencing prescription of AOM 400-TIS were prior treatment adherence (55.3%) and efficacy (48.9%). Most HCPs agreed/strongly agreed that AOM 400-TIS was easy to administer (79.8%) and had a similar safety/tolerability profile to the one-injection start regimen (69.1%), with the majority satisfied with patient outcomes with AOM 400-TIS (84.0%).</p><h3>Conclusions</h3><p>European HCPs reported that AOM 400-TIS was easy to administer, well tolerated, and improved treatment outcomes. Barriers to its use included patient reluctance and perceived safety concerns.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"42 4","pages":"1935 - 1949"},"PeriodicalIF":3.4,"publicationDate":"2025-03-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-025-03130-w.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143536398","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Patient-Reported Outcomes for Patients with Previously Treated Small Cell Lung Cancer Receiving Tarlatamab: Results from the DeLLphi-301 Phase 2 Trial","authors":"Horst-Dieter Hummel,&nbsp;Myung-Ju Ahn,&nbsp;Fiona Blackhall,&nbsp;Martin Reck,&nbsp;Hiroaki Akamatsu,&nbsp;Suresh S. Ramalingam,&nbsp;Hossein Borghaei,&nbsp;Melissa Johnson,&nbsp;Franziska Dirnberger,&nbsp;Kim Cocks,&nbsp;Shuang Huang,&nbsp;Sujoy Mukherjee,&nbsp;Luis Paz-Ares","doi":"10.1007/s12325-025-03136-4","DOIUrl":"10.1007/s12325-025-03136-4","url":null,"abstract":"<div><h3>Introduction</h3><p>Tarlatamab demonstrated a durable response and promising survival outcomes in patients with previously treated small cell lung cancer (SCLC) in the phase 2, open-label DeLLphi-301 trial. Patient-reported outcomes (PROs) were evaluated to assess the benefit-risk profile of tarlatamab.</p><h3>Methods</h3><p>Patients received tarlatamab intravenously every 2 weeks at a dose of 10 mg (regulatory approved dose) or 100-mg until progression or loss of benefit. PROs, including European Organization for Research and Treatment of Cancer 30-item Quality of Life Questionnaire (EORTC-QLQ-C30) and 13-item lung cancer module (LC13), Patient-Reported Outcomes Version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE), and the GP5 question of the Functional Assessment of Cancer Therapy – General Form (FACT-GP5), were collected at Cycle 1 (days 1, 8, 22), Cycle 2 (days 1, 15) and every 6 weeks from Cycle 3 onwards. PROs were summarized descriptively alongside the amount and reason for missing data and analyzed using a mixed model for repeated measures. In addition, median time to deterioration (TTD) for symptom and functional scales was analyzed.</p><h3>Results</h3><p>A total of 100 patients were PRO-evaluable at the selected target dose (10 mg). EORTC-QLQ-C30 and LC13 completion rates (proportion of PRO assessments expected to be completed) were high (&gt; 80%) throughout the study. Least square mean changes from baseline showed a trend towards improvement for the QLQ-C30 subscale of global health status and stabilization for physical functioning. Patients experienced reduced symptom burden for dyspnea which was more pronounced for patients at later cycles (≥ 10 points), and stabilization for chest pain and cough. Median TTD exceeded 6 months for cough and dyspnea and was not estimable for chest pain. Overall, tarlatamab was well tolerated with the majority of patients reporting no bother or a little bit of bother from side effects post baseline. Patient-reported adverse events were generally of mild to moderate severity occurring rarely or occasionally.</p><h3>Conclusion</h3><p>Alongside previously reported antitumor activity, tarlatamab demonstrated a positive benefit–risk profile in previously treated SCLC with favorable PROs across a range of functional outcomes and symptoms, while showing manageable and sustained tolerability.</p><h3>ClinicalTrials.gov Number</h3><p>NCT05060016.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"42 4","pages":"1950 - 1964"},"PeriodicalIF":3.4,"publicationDate":"2025-03-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-025-03136-4.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143536369","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}