Drug discoveries & therapeutics最新文献

{"title":"Metabolic and endocrinological effects of weekly growth hormone replacement therapy with somapacitan in patients with adult growth hormone deficiency after switching from daily growth hormone replacement therapy: A real-world exploratory cohort study.","authors":"Ichiro Abe, Keita Kubo, Yuya Fujita, Kentaro Ochi, Toshiaki Wada, Ryo Mitsuoka, Kaori Takeshita, Ayuko Higashi, Tamotsu Kato, Tadachika Kudo, Hideaki Shimada, Osamu Imakyure, Makiko Abe, Shigeaki Mukoubara, Kunihisa Kobayashi","doi":"10.5582/ddt.2026.01003","DOIUrl":"10.5582/ddt.2026.01003","url":null,"abstract":"<p><p>The metabolic and endocrinological effects in patients with adult growth hormone deficiency (AGHD) who switched from daily growth hormone (GH) replacement therapy to weekly GH replacement therapy with somapacitan were evaluated and observed over a long follow-up period. Patients were included only if their medical treatments, aside from GH replacement therapy, remained unchanged. Metabolic and endocrinological parameters were assessed at the time of switching, and at 1- and 2-year follow-up after switching from daily GH replacement therapy to weekly GH replacement therapy with somapacitan. The results showed that the body mass index (BMI), fasting plasma glucose (FPG), aspartate transaminase (AST), and triglyceride (TG) levels at 1 year after switching significantly improved compared with those at the time of switching (each P < 0.025). At 2 years, the homeostasis model assessment of insulin resistance (HOMA-IR), systolic blood pressure (SBP), diastolic blood pressure (DBP), and alanine aminotransferase (ALT), as well as BMI, FPG, TG, and AST significantly improved compared with those observed at the time of switching (each P < 0.025). In addition, improvement in HOMA-IR at 2 years after switching was significantly associated with improvement in AST. Switching to GH replacement therapy did not affect endocrinological parameters. These findings, which were revealed by the present real-world exploratory cohort study, indicate that weekly GH replacement therapy with somapacitan may confer more beneficial effects than daily GH replacement therapy.</p>","PeriodicalId":520606,"journal":{"name":"Drug discoveries & therapeutics","volume":" ","pages":"221-227"},"PeriodicalIF":0.0,"publicationDate":"2026-07-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148255540","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Analytical method validation and feasibility of salivary pregabalin measurement in Japanese volunteers: A pilot study.","authors":"Hina Itoi, Yoshito Gando, Takeo Yasu, Masayuki Kobayashi","doi":"10.5582/ddt.2026.01015","DOIUrl":"10.5582/ddt.2026.01015","url":null,"abstract":"<p><p>This pilot study evaluated whether salivary pregabalin concentrations reflect plasma levels in Japanese volunteers to support non‑invasive therapeutic drug monitoring (TDM). Four healthy adults received a single 75‑mg orally disintegrating tablet; unstimulated saliva and venous blood were collected 1 hour post‑dose using a standardized passive‑drool protocol with pre‑collection rinsing. Pregabalin was quantified by a high-performance liquid chromatography with fluorescence detection. The assay met bioanalytical performance criteria across both matrices (excellent linearity, recovery > 94.2%, precision ≤ 10%, stability within 5.2%). At 1 hour, median concentrations were 1.96 μg/mL (plasma) and 0.466 μg/mL (saliva). In paired analysis (n = 4), saliva and plasma showed a positive trend (r = 0.838, p = 0.298). Given the small sample size, these results are considered exploratory and demonstrate the feasibility of the analytical approach rather than providing definitive clinical evidence. Under standardized collection conditions, salivary pregabalin concentrations appear to yield clinically interpretable estimates of systemic exposure, warranting validation in larger, multi‑time‑point cohorts to establish actionable saliva‑to‑plasma conversion thresholds and evaluate clinical utility.</p>","PeriodicalId":520606,"journal":{"name":"Drug discoveries & therapeutics","volume":" ","pages":"269-272"},"PeriodicalIF":0.0,"publicationDate":"2026-07-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148298658","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Low rate of pharmacists accessing renal function laboratory values: A cross-sectional study using electronic medical records of a Japanese community pharmacy.","authors":"Shingo Kondo, Nozomi Ito, Mari Maese, Yuko Okamoto, Hiroki Iwata, Noriko Kobayashi, Katsunori Yamaura","doi":"10.5582/ddt.2025.01136","DOIUrl":"10.5582/ddt.2025.01136","url":null,"abstract":"<p><p>This study aimed to assess the availability of renal function laboratory values in Japanese community pharmacies and examine their association with the prescription of drugs requiring dosage adjustment. In this cross-sectional observational study, data were obtained from the electronic medical records of a community pharmacy in Japan. A total of 389 eligible patients (aged ≥ 18 years) who visited the pharmacy in April 2024 were included. The primary outcome measures were the proportion of participants with available renal function laboratory values and those prescribed medications requiring dosage adjustment. A total of 128 patients (32.9%) were prescribed at least one medication requiring dosage adjustment, most commonly vitamin A and D preparations (32 cases) and antidiabetic agents (31 cases). Renal function laboratory values were available for 40 patients (10.3%). Compared to those not prescribed such drugs, patients receiving medications requiring renal-based dosage adjustments included a significantly higher proportion of older adults (70/128, 54.7% vs 98/261, 37.5%, p = 0.001) and patients with available laboratory values (20/128, 15.6% vs 20/261, 7.7%, p = 0.015). No patients were identified with the \"Triple Whammy,\" which is known to significantly increase the risk of renal function deterioration. Community pharmacists endeavor to obtain renal function laboratory values for older adults, who are more likely to be prescribed medications requiring dosage adjustment. However, the proportion of cases with obtained laboratory values remains low. Pharmacists are expected to actively recommend renal function testing to physicians. Furthermore, they should contribute to safe pharmacotherapy by utilizing comprehensive patient information.</p>","PeriodicalId":520606,"journal":{"name":"Drug discoveries & therapeutics","volume":" ","pages":"211-220"},"PeriodicalIF":0.0,"publicationDate":"2026-07-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148255548","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Classical traditional Chinese medicine formulas for inflammatory bowel disease: therapeutic evidence and mechanistic insights.","authors":"Lichao Yang, Zhixian Jiang, Qi Sun, Lianwen Yuan, Wei Tang","doi":"10.5582/ddt.2026.01010","DOIUrl":"10.5582/ddt.2026.01010","url":null,"abstract":"<p><p>Inflammatory bowel disease (IBD) is a digestive system disorder characterized by chronic recurrent inflammation, primarily including ulcerative colitis (UC) and Crohn's disease (CD). Although modern precision medicine therapies, represented by biologics and small-molecule drugs, have reshaped the IBD treatment landscape, clinical practice still faces challenges such as primary non-response, secondary loss of response, risks of severe opportunistic infections, and an increasingly heavy health-economic burden. Traditional Chinese Medicine (TCM), as a unique medical system with millennia of clinical experience and guided by its distinct theoretical framework of \"holistic concept\" and \"treatment based on syndrome differentiation,\" shows great potential in IBD management. In recent years, advances in evidence-based medicine and modern biotechnology have propelled TCM research in IBD from empirical treatment toward precision medicine. TCM exerts its effects through multi-target and multi-pathway mechanisms, demonstrating unique advantages particularly in immunomodulation, intestinal barrier repair, and gut microbiota regulation. Furthermore, modern research, from holistic perspectives such as the \"gut-lung axis\" and \"gut-brain axis,\" scientifically interprets the modern biological connotations of traditional TCM theories, providing new understanding for TCM's application in IBD. Concurrently, this article discusses the revolutionary progress of nanodrug delivery systems and multi-omics technologies in enhancing the bioavailability of TCM components, reducing toxicity, and elucidating complex mechanisms. It aims to construct a modern scientific framework for TCM in treating IBD through the dual macroscopic and microscopic perspectives of systems biology and translational medicine, offering insights for achieving precision therapy, whole-life-cycle management of IBD, and complementary integration of Chinese and Western medicine.</p>","PeriodicalId":520606,"journal":{"name":"Drug discoveries & therapeutics","volume":" ","pages":"104-118"},"PeriodicalIF":0.0,"publicationDate":"2026-05-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147795145","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cytoderm metabolic-labeling SCMLP-TB for pulmonary tuberculosis diagnosis: a preliminary diagnostic accuracy study.","authors":"Guangyan Liang, Guiqin Dai, Xiaorong Hu, Deliang Liu, Zhiqiang Lin, Mengru Yang, Zhuojun He, Peifen Chen, Yipeng Liu, Xinyun Jia, Xiafei Dai, Pengfei Zhao, Mingbin Zheng, Yang Zhou, Hongzhou Lu","doi":"10.5582/ddt.2025.01123","DOIUrl":"10.5582/ddt.2025.01123","url":null,"abstract":"<p><p>Tuberculosis (TB) remains a significant global health issue. Early diagnosis is crucial, yet current diagnostic technologies are limited by suboptimal sensitivity. Thus, we developed a novel tuberculosis metabolic labeling probe (single cell metabolic labeling probe for tuberculosis, SCMLP-TB) and evaluated its diagnostic performance. In this retrospective study of 70 suspected TB patients, we calculated the sensitivity and specificity of SCMLP-TB and compared it with culture and Xpert MTB/RIF (Xpert) using the final clinical diagnosis as reference standard. Eligible participants were classified as confirmed TB (CT), clinically diagnosed TB (CDxT), or non-TB cases based on the diagnosis criteria for pulmonary tuberculosis (WS 288-2017). Of the 70 participants, 40 (57.0%) were diagnosed with TB, including 30 CT cases and 10 CDxT cases. The overall diagnostic sensitivity and specificity of SCMLP-TB were 97.5% and 96.7%, respectively. Notably, SCMLP-TB identified 10 CDxT cases missed by both culture and Xpert. The overall diagnostic sensitivity of culture and Xpert was 62.5% and 72.5%, respectively, while both showed a specificity of 100.0%, demonstrating that SCMLP-TB was more sensitive than culture and Xpert. Besides, the fluorescence intensity from TB patients was significantly higher than non-TB patients. The fluorescence intensity showed a significant negative correlation with the time to positivity (TTP) of culture, which suggested that SCMLP-TB could also serve as an indicator of bacterial loads in patients' samples. Consequently, SCMLP-TB demonstrated a promising tool for the sensitive and ultra-fast diagnosis of pulmonary TB suspects, particularly for paucibacillary pulmonary TB.</p>","PeriodicalId":520606,"journal":{"name":"Drug discoveries & therapeutics","volume":" ","pages":"147-154"},"PeriodicalIF":0.0,"publicationDate":"2026-05-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147641276","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Trends in the prescription of constipation medications in Japan (fiscal years 2019-2023): A nationwide baseline study prior to the 2023 clinical guidelines.","authors":"Hiroyuki Tanaka, Toshihiro Ishii","doi":"10.5582/ddt.2025.01135","DOIUrl":"10.5582/ddt.2025.01135","url":null,"abstract":"<p><p>Constipation is a common gastrointestinal disorder that markedly affects health and quality of life. In Japan, magnesium-based and contact laxatives have long been widely prescribed. Since the launch of lubiprostone in November 2012, several new agents with novel mechanisms have been introduced, and their use has increased. However, to our knowledge, no nationwide study has examined their use. This study aimed to analyze nationwide trends and regional differences in the prescription of medications for constipation. We used data from the Japanese National Health Insurance Claims and Specific Health Checkup Database Open Data from fiscal years (FYs) 2019 to 2023, covering the period when all currently available constipation medications were on the market in Japan, and prior to the inclusion of the drug selection flowchart in the Evidence-Based Clinical Guidelines for Chronic Constipation 2023. Prescription trends were evaluated using defined daily doses per 1,000 inhabitants per day (DID), and regional variations were assessed using standardized claim ratios (SCRs). The DID for contact laxatives decreased during the study period, whereas the DID for other constipation medications increased. Magnesium oxide showed the smallest regional variation, whereas polyethylene glycol and linaclotide showed the largest variations in FY 2019 and FY 2023, respectively. Throughout the 5-year period, the highest SCRs for contact laxatives were observed in Akita, Iwate, and Aomori prefectures. Our findings provide a comprehensive nationwide picture of evolving constipation treatment patterns and regional variations in Japan, establishing baseline data for evaluating the impact of guideline dissemination and evidence accumulation on clinical practice.</p>","PeriodicalId":520606,"journal":{"name":"Drug discoveries & therapeutics","volume":" ","pages":"199-204"},"PeriodicalIF":0.0,"publicationDate":"2026-05-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147694536","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Artificial intelligence (AI)-assisted ultrasound in clinical trials: Endpoint automation, decentralized monitoring, and regulatory readiness.","authors":"Kenji Karako, Jianjun Gao","doi":"10.5582/ddt.2026.01020","DOIUrl":"10.5582/ddt.2026.01020","url":null,"abstract":"<p><p>Ultrasound is among the most widely used imaging modalities in clinical trials, and yet its dependence on operator skill and equipment settings has historically limited the reproducibility of ultrasound-based endpoints in multi-center studies. Artificial intelligence (AI) now addresses this limitation across two complementary dimensions: automated measurement algorithms that quantify cardiac function, organ volume, and vascular parameters with reproducibility approaching or, in select settings, exceeding that of trained human readers and real-time acquisition guidance systems that enable clinicians with no formal sonography training to perform diagnostic-level examinations, making remote and decentralized assessment increasingly feasible. This narrative review synthesizes current evidence and regulatory developments across three interconnected domains. First, use of automated ultrasound to ascertain endpoints has advanced from single-institution validation to prospective and randomized evidence, with deep learning measurement of the left ventricular ejection fraction demonstrating formal equivalence to expert readers across multiple echocardiographic parameters and AI-first workflows shortening the time to diagnosis in a blinded non-inferiority trial. Second, AI-guided ultrasound acquisition by nurses and other non-expert operators has achieved a high rate of diagnostic acceptability in cardiac and pulmonary ultrasound, laying the groundwork for use of ultrasound-based endpoints in decentralized clinical trial designs, as reflected in Food and Drug Administration (FDA) guidance on decentralized trial elements. Third, the regulatory frameworks governing AI-enabled medical devices - including the U.S. FDA's Predetermined Change Control Plan guidance, the EU Artificial Intelligence Act, and internationally harmonized good machine learning practice relevant to Japan and other jurisdictions - increasingly emphasize overlapping principles such as specification of prospective performance, post-marketing oversight, and transparent reporting. Addressing remaining challenges in domain generalization across vendors, subgroup fairness, and algorithm change management during ongoing trials will be essential for AI-assisted ultrasound to fulfill its potential as a robust, scalable endpoint in clinical research worldwide.</p>","PeriodicalId":520606,"journal":{"name":"Drug discoveries & therapeutics","volume":" ","pages":"91-103"},"PeriodicalIF":0.0,"publicationDate":"2026-05-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147725427","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Modern research on formulated granules of traditional Chinese medicines and Japanese Kampo medicines: A narrative review.","authors":"Wenyuan Li","doi":"10.5582/ddt.2026.01012","DOIUrl":"10.5582/ddt.2026.01012","url":null,"abstract":"<p><p>The modernization of traditional medicine follows diverse pathways. This paper aims to provide a narrative review of the manufacturing processes, research on active ingredients, and models for the clinical use of formulated granules of traditional Chinese medicines (TCM) and Japanese Kampo medicines in the context of their modernization by comparing advances in research on the two and their challenges. By searching databases such as PubMed, Web of Science, and China National Knowledge Infrastructure (CNKI), this review included relevant literature published up to 2026. After screening, a total of 48 relevant articles were included in the review. In TCM, formulated granules evolved from the need to modernize herbal medicines through multi-stage extraction and granulation. This enhances convenience while preserving medicinal properties. TCM enables flexible syndrome differentiation and it is working to industrially produce and standardize herbal medicines while refining quality standards. Japanese Kampo, based on fixed classical formulations and covered by National Health Insurance, has developed a highly standardized, evidence-based model. Research in China broadly investigates numerous formulations using omics technologies to explore their material basis and network pharmacology. Research in Japan focuses on in-depth analysis of \"pathways to efficacious formulations\" for select classic formulations. Faced with the challenge of providing modern evidence, granules can produce real-world data on TCM. This comparative analysis offers insights into the development of quality granules and their use internationally and it explores prospects for Sino-Japanese collaboration in complementary manufacturing processes and fundamental research.</p>","PeriodicalId":520606,"journal":{"name":"Drug discoveries & therapeutics","volume":" ","pages":"119-127"},"PeriodicalIF":0.0,"publicationDate":"2026-05-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147794406","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Chemotherapy combined targeted therapy has a potential benefit on the survival of advanced intrahepatic cholangiocarcinoma.","authors":"Jian Yang, Tengqian Tang, Rui Liao, Yan He, Hua Zhang","doi":"10.5582/ddt.2026.01007","DOIUrl":"10.5582/ddt.2026.01007","url":null,"abstract":"<p><p>The treatment strategies and outcomes for advanced intrahepatic cholangiocarcinoma (ICC) are restricted. The treatment modalities and effectiveness of local interventional chemotherapy and systemic chemotherapy remain indeterminate. The objective of this study is to explore and assess the influence of different chemotherapy methods on the prognosis of patients with advanced ICC. A retrospective investigation was carried out at two research centers. The recruited patients were divided into the systemic chemotherapy cohort and the local chemotherapy cohort (Transarterial Chemoembolization, TACE). The primary endpoint of this study was overall survival (OS), while the secondary endpoints encompassed progression free survival (PFS), response rate (RR), and adverse events (AE). From January 2014 to January 2024, a total of 124 patients were included. Systemic/local chemotherapy combined with targeted therapy exhibited superior survival performance compared to chemotherapy alone. Additionally, patients with lesions confined to the liver and large tumors (> 6 cm) obtained better survival benefits from systemic chemotherapy. There was no significant disparity in grade 3 or more severe adverse events between the two groups. Whether it is systemic chemotherapy or TACE, combining them with targeted therapy can confer significant therapeutic advantages to patients with advanced ICC. ICC patients with a higher tumor burden may attain better therapeutic outcomes by selecting systemic chemotherapy.</p>","PeriodicalId":520606,"journal":{"name":"Drug discoveries & therapeutics","volume":" ","pages":"155-164"},"PeriodicalIF":0.0,"publicationDate":"2026-05-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147795198","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Efficacy and safety of filgotinib versus tocilizumab in active rheumatoid arthritis: A randomized, open-label, multicenter study with clinical and musculoskeletal ultrasound evaluation (TRANSFORM study).","authors":"Toshimasa Shimizu, Shin-Ya Kawashiri, Tomohiro Koga, Rieko Kiya, Michiko Morita, Rina Kawasaki, Shohei Kuroda, Shigeki Tashiro, Shuntaro Sato, Moemi Yabe, Kenta Misaki, Shunichiro Hanai, Daiki Nakagomi, Michihiro Ogasawara, Naoto Tamura, Rina Watanabe, Hiroshi Kanazawa, Tatsuya Atsumi, Yukitaka Ueki, Tadashi Okano, Takahisa Suzuki, Hirokazu Takaoka, Hiroaki Hamada, Toshihiko Hidaka, Shunsuke Furuta, Naoki Hosogaya, Hiroshi Yamamoto, Atsushi Kawakami","doi":"10.5582/ddt.2026.01008","DOIUrl":"10.5582/ddt.2026.01008","url":null,"abstract":"<p><p>Janus kinase (JAK) and interleukin-6 (IL-6) inhibitors are therapeutic options for patients with rheumatoid arthritis (RA) with inadequate response to conventional synthetic disease-modifying antirheumatic drugs (csDMARDs); however, no randomized controlled trial has compared their efficacy and safety. Since both act through the JAK-signal transducer and activator of transcription pathway, a comparative evaluation is warranted. We conducted a prospective, randomized, open-label trial at 55 centers in Japan, randomizing patients with active RA despite csDMARD therapy in a 1:1 ratio to receive 200 mg/day filgotinib or subcutaneous tocilizumab as monotherapy; the primary endpoint was American College of Rheumatology (ACR) 50 at week 12, and secondary endpoints included clinical disease activity indices, musculoskeletal ultrasonography scores, patient-reported outcomes, and serum biomarkers through 52 weeks. Twenty-six patients were enrolled (13 per group) before study termination due to insufficient recruitment, and descriptive analyses were performed. At week 12, ACR50 was achieved in 38.5% (5/13) patients in the filgotinib group and 46.2% (6/13) in the tocilizumab group (risk difference: -7.69%; 95% confidence interval: -42.26 to 28.8). Both groups showed early and sustained improvements in disease activity from week 2. The improvement in patient global assessment scores was greater with filgotinib at week 2 but the difference diminished thereafter, and serum IL-6 level decreased with filgotinib but increased with tocilizumab. Four serious adverse events occurred with filgotinib, including infections and cardiac events. Because this study was underpowered and the analysis was descriptive, larger studies are needed to confirm these findings and define optimal use. (The study was registered in the Japan Registry of Clinical Trials (https://jrct.niph.go.jp) as jRCTs071200107 and in ClinicalTrials.gov as NCT05090410.).</p>","PeriodicalId":520606,"journal":{"name":"Drug discoveries & therapeutics","volume":" ","pages":"175-189"},"PeriodicalIF":0.0,"publicationDate":"2026-05-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147794238","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}