Published by: MedSci
Release Date: January 15, 2026
Location: Shanghai, China
The "Top 10 Chinese Medical Research Studies of 2025" have been officially announced. Looking back at 2025, Chinese scientists have achieved fruitful results in the medical field; these achievements not only demonstrate the steady improvement of China's scientific research strength but also inject significant momentum into global medical development.
MedSci has authoritatively released the annual top ten medical research progress reports for eight consecutive years. This year, through an open and transparent online selection mechanism, the "Top 10 Most High-Profile Chinese Medical Research Studies of 2025" were selected, covering oncology, cardiovascular, neurology, hematology, endocrinology, pediatrics & rare diseases, respiratory, and Traditional Chinese Medicine (TCM).
In this year's final selection, the top two vote-getters from each clinical specialty entered the ultimate round. A total of 16 heavyweight studies were screened, and the top 10 by vote count were selected as the "Top 10 Most Chinese Medical Research Studies of 2025." (Note: The ranking is based on the number of online votes; in the event of a tie, the order is determined by the voting sequence number.)— 01 —
NEJM: Teams led by Zhang Li, Fang Wenfeng, and Yang Yunpeng Release the "China Solution" for Targeted Resistance in Lung Cancer; Sacituzumab Tirumotecan for EGFR-TKI Resistant NSCLC
Tao C, Fang W, Yang Y, et al. Sacituzumab Govitecan for EGFR-TKI-Resistant EGFR-Mutant NSCLC. N Engl J Med. 2025;393(17):1613-1623. doi:10.1056/NEJMoa2512071
lJournal: N Engl J Med
lCorresponding/Key Authors: Zhang Li, Fang Wenfeng, Yang Yunpeng
lAffiliation: Sun Yat-sen University Cancer Center
The OptiTROP-Lung04 study, led by the teams of Zhang Li, Fang Wenfeng, and Yang Yunpeng from Sun Yat-sen University Cancer Center, is the world's first Phase III randomized controlled trial focusing on EGFR-mutation-positive non-squamous NSCLC after EGFR-TKI resistance. EGFR-TKI resistance is a major bottleneck in lung cancer treatment; previously, no monotherapy significantly improved overall survival, and this study fills that gap, enrolling 376 patients from China and the Asia-Pacific region.
The study showed that the median progression-free survival (PFS) in the Sacituzumab Tirumotecan group reached 8.3 months, significantly superior to the 4.3 months in the platinum-based doublet chemotherapy group (HR=0.49, 95% CI: 0.39-0.62, P<0.0001), reducing the risk of disease progression by 51%. The median overall survival (OS) has not yet been reached, compared to 17.4 months in the chemotherapy group (HR=0.60, 95% CI: 0.44-0.82, P=0.001), reducing the risk of death by 40%. The objective response rate (ORR) was 60.6% vs. 43.1% (P=0.002). Regarding safety, the incidence of Grade 3 or higher treatment-related adverse events was lower than that of the chemotherapy group, primarily consisting of myelosuppression and gastrointestinal reactions, which were controllable and manageable.
This study confirms the significant survival benefit of TROP2 ADC in the EGFR-TKI resistant population, becoming the first monotherapy regimen to improve both PFS and OS. Based on this, China's NMPA has approved the relevant indications, and it has been included in the CSCO Lung Cancer Guidelines as a Class 1A recommendation, providing a "Chinese Innovative Drug Solution" for these refractory patients and driving the upgrade of the global EGFR-mutant lung cancer treatment landscape.— 02 —
BMJ: Teams led by Zhang Li and Fang Wenfeng Break the Deadlock of EGFR-mutant NSCLC Resistance with Sacituzumab Tirumotecan
Fang W, Li X, Wang Q, et al. Sacituzumab tirumotecan versus docetaxel for previously treated EGFR-mutated advanced non-small cell lung cancer: multicentre, open label, randomised controlled trial. BMJ. 2025;389:e085680. Published 2025 Jun 5. doi:10.1136/bmj-2025-085680
lJournal: BMJ
lCorresponding/Key Authors: Zhang Li, Fang Wenfeng
lAffiliation: Sun Yat-sen University Cancer Center
An open-label, randomized, multicenter trial (OptiTROP-Lung03), led by Professors Zhang Li and Fang Wenfeng of Sun Yat-sen University Cancer Center, aimed to evaluate the efficacy and safety of Sacituzumab Tirumotecan versus docetaxel in patients with locally advanced or metastatic EGFR-mutant NSCLC who failed EGFR-TKI and platinum-based chemotherapy.
The study confirmed: As of December 31, 2024, per Blinded Independent Review Committee (BIRC) assessment, the ORR for the Sacituzumab Tirumotecan group was as high as 45%, while the docetaxel group was only 16%, a difference of 29% (P<0.001). Regarding median PFS, the Sacituzumab Tirumotecan group reached 6.9 months compared to 2.8 months for docetaxel, with a significant 70% reduction in the risk of disease progression or death (HR 0.30, P<0.001). In terms of OS, neither group reached median OS, but the Sacituzumab Tirumotecan group already showed significant OS improvement with a 51% reduction in death risk (HR 0.49, P=0.007); after cross-over correction, the OS advantage was even more pronounced (HR 0.36), with a 64% reduction in death risk.
The success of Sacituzumab Tirumotecan not only brings a much-needed new treatment option with high efficacy and low toxicity to patients with advanced EGFR-mutant NSCLC but also provides a valuable "China Solution" for global anti-tumor drug R&D through its unique mechanism of action and clinical advantages. This breakthrough is both a crystallization of the collective efforts of the global oncology community and a concentrated demonstration of the Chinese research team’s innovation capabilities and international academic influence rooted in clinical needs.— 03 —
NEJM: Teams led by Ji Linong, Jiang Hongwei, and Qian Lei Release Chinese Original Weight Loss Drug Mazdutide, with Efficacy Comparable to Semaglutide
Ji L, Jiang H, Bi Y, et al. Once-Weekly Mazdutide in Chinese Adults with Obesity or Overweight. N Engl J Med. 2025;392 (22):2215-2225. doi:10.1056/NEJMoa2411528
lJournal: NEJM
lCorresponding/Key Authors: Ji Linong, Jiang Hongwei, Qian Lei
lAffiliation: Peking University People's Hospital
Professor Ji Linong of Peking University People's Hospital led the GLORY-1 Phase 3 double-blind, placebo-controlled trial, focusing on obese or overweight Chinese adults to evaluate the weight loss efficacy and safety of once-weekly Mazdutide injections. The study randomized adults aged 18-75 with a BMI ≥28 kg/m² or 24-28 kg/m² with at least one weight-related comorbidity into 4 mg Mazdutide, 6 mg Mazdutide, or placebo groups in a 1:1:1 ratio for 48 weeks of treatment.
Results showed that after 32 weeks of treatment, Mazdutide achieved clinically meaningful weight loss, with effects comparable to the "blockbuster" drug Semaglutide; the drug was well-tolerated, with the most common adverse events being nausea, diarrhea, and vomiting, mostly mild to moderate. Mazdutide is the world's first GCG/GLP-1 dual receptor agonist to apply for marketing authorization. This study marks that clinical research on China's self-developed innovative weight-loss drugs has entered the international forefront.— 04 —
JAMA Pediatrics: Mao Jianhua's Team Releases New Evidence for Treating Frequently Relapsing and Steroid-Dependent Nephrotic Syndrome in Children; Tacrolimus Superior in Both Efficacy and Safety
Wang J, Liu F, Yan W, et al. Tacrolimus or Mycophenolate Mofetil for Frequently Relapsing or Steroid-Dependent Nephrotic Syndrome: A Randomized Clinical Trial. JAMA Pediatr. Published online May 12, 2025. doi:10.1001/jamapediatrics.2025.0765
lJournal: JAMA Pediatrics
lCorresponding/Key Author: Mao Jianhua
lAffiliation: Children's Hospital, Zhejiang University School of Medicine
Children with frequently relapsing or steroid-dependent nephrotic syndrome require continuous steroid maintenance and face high relapse rates; clinicians often struggle between choosing "Tacrolimus vs. Mycophenolate Mofetil (MMF)." Professor Mao Jianhua of the Children's Hospital, Zhejiang University School of Medicine, led 12 top-tier domestic hospitals in a randomized clinical trial to provide evidence-based support for treatment. The study randomized 270 children aged 2-18 into Tacrolimus and MMF groups, combined with a tapered glucocorticoid regimen, and followed them for one year.
Results showed that the 1-year relapse-free survival rate in the Tacrolimus group was 1.86 times higher than that of the MMF group, and the median relapse-free survival was extended by over 60 days (323.99 days vs. 263.21 days). The annual relapse rate was slashed by nearly 60%, and cumulative steroid dosage was reduced by 35%, with no significant differences in safety indicators between the two groups. This study provides a high-evidence-level answer for the treatment of pediatric frequently relapsing and steroid-dependent nephrotic syndrome, establishing Tacrolimus as the preferred option, which is expected to rewrite global treatment guidelines and optimize clinical medication strategies.— 05 —
J Hematol Oncol: Jin Fengyan's Team Breaks the Shackles of pPCL Risk Stratification! New Staging System (PSS) Enables Precision Stratification
Tian M, An G, Fu W, et al. Development and validation of a prognostic staging system for primary plasma cell leukemia. J Hematol Oncol. 2025;18 (1):72. Published 2025 Jul 15. doi:10.1186/s13045-025-01723-0
lJournal: Journal of Hematology & Oncology
lCorresponding/Key Author: Jin Fengyan
lAffiliation: The First Hospital of Jilin University
A retrospective study conducted by Jin Fengyan’s team at the First Hospital of Jilin University, in collaboration with 26 centers nationwide, addressed the challenge of precise prognostic stratification for primary plasma cell leukemia (pPCL). pPCL is the most aggressive subtype of multiple myeloma with the poorest prognosis; existing staging systems fail to meet clinical needs, making individualized treatment difficult to implement.
Utilizing multi-center real-world data, the research innovatively constructed a pPCL-specific staging system (PSS) based on three variables easily obtained during routine clinical diagnosis (LDH, thrombocytopenia, and cytogenetic abnormalities). This system breaks through the limitations of existing risk stratification models, more accurately predicts patient survival and prognosis, and provides a crucial tool for formulating targeted clinical treatment strategies, possessing significant potential for clinical promotion.— 06 —
Allergy: Teams led by Zhang Qinxiu, Wang Deyun, and Wang Qi formulate the World’s First international TCM prevention and treatment Guidelines for Allergic Rhinitis.
Fu QW, Liu P, Ruan Y, et al. International Evidence-Based Guidelines for Traditional Chinese Medicine Management of Allergic Rhinitis. Allergy. Published online September 13, 2025. doi:10.1111/all.70057.
lJournal: Allergy
lCorresponding/Key Authors: Zhang Qinxiu, Wang Deyun, Wang Qi
lAffiliation: Hospital of Chengdu University of Traditional Chinese Medicine
Allergic rhinitis is a common allergic disease where TCM prevention and treatment offer unique advantages, yet there has been a lack of internationally recognized evidence-based guidelines. Led by Zhang Qinxiu’s team from the Hospital of Chengdu University of TCM, more than 60 experts from 23 institutions across China, Singapore, Japan, and Canada collaborated on the guideline's development. Based on "theory-experiment-clinical" closed-loop research and using evidence-based medical standards, the guideline systematically integrates global evidence on TCM management of allergic rhinitis.
This is the first time Allergy has published a TCM guideline. It has been included in the "International Promotion Project of TCM Standards" by the World Federation of Chinese Medicine Societies. Multilingual translation and global training will be launched, with plans to establish 10 clinical demonstration centers along "Belt and Road" countries. This guideline marks the official international debut of research results for TCM in treating allergic rhinitis, providing an "Oriental Solution" for global treatment and advancing the internationalization of TCM.— 07 —
Lancet Respiratory Medicine: Teams led by Zhi Lili, Li Xu, and Jin Peng Warn of Thunderstorm Asthma Risks in China, Calling for a Monitoring and Early Warning System
Li X, Jin P, Zhi L. Thunderstorm asthma in China: an emerging clinical and public health concern. Lancet Respir Med. Published online November 28, 2025. doi:10.1016/S2213-2600 (25) 00396-0
lJournal: Lancet Respiratory Medicine
lCorresponding/Key Authors: Zhi Lili, Li Xu, Jin Peng
lAffiliation: The First Affiliated Hospital of Shandong First Medical University, The Second Hospital of Shandong University
The allergy research team at the First Affiliated Hospital of Shandong First Medical University systematically reviewed reported thunderstorm asthma events in China from 2018 to 2025 and published a commentary in a Lancet sub-journal. The study pointed out that thunderstorm asthma in China is trending from sporadic to recurrent, with incidence highly concentrated during the Artemisia pollen season in Northern China. While there is a large base of highly sensitized individuals, the monitoring system and emergency response capabilities do not yet match the risk, lacking cross-departmental information sharing and rapid warning systems.
The article emphasizes that China is in a critical window for building a prevention and control system and must take action early to avoid repeating the large-scale event seen in Australia in 2016. This work provides Chinese evidence for the global respiratory health field and enhances China's international voice in the study of environmental-triggered emergencies.— 08 —
NEJM: Lou Min's Team Releases Findings on Extending the Thrombolysis Window for Posterior Circulation Stroke to 24 Hours
Yan S, Zhou Y, Lansberg MG, et al. Alteplase for Posterior Circulation Ischemic Stroke at 4.5 to 24 Hours. N Engl J Med. 2025;392(13):1288-1296. doi:10.1056/NEJMoa2413344
lJournal: N Engl J Med
lCorresponding/Key Author: Lou Min
lAffiliation: Second Affiliated Hospital, Zhejiang University School of Medicine
The EXPECTS study, led by Lou Min’s team at the Second Affiliated Hospital of Zhejiang University, aimed to evaluate the efficacy and safety of intravenous thrombolysis in patients with posterior circulation ischemic stroke (PCS) within 4.5 to 24 hours of onset. Due to atypical symptoms and delayed presentation, many PCS patients miss the traditional 4.5-hour window; there is an urgent clinical need for imaging-based evidence for extended-window treatment. The EXPECTS study included 372 PCS patients with salvageable brain tissue confirmed by perfusion imaging to systematically evaluate this strategy.
The study showed that the 90-day functional independence rate (mRS 0–2) in the alteplase group was significantly higher than in the standard treatment group (40% vs. 26%, P=0.004), indicating clear clinical benefit from extended-window thrombolysis. Although the risk of symptomatic intracranial hemorrhage increased slightly, it did not lead to increased mortality. Under strict imaging screening, alteplase retained the ability to improve tissue survival and neurological function between 4.5 and 24 hours, providing solid randomized evidence for extended-window IV thrombolysis.
In conclusion, even beyond the traditional window, PCS patients selected by imaging can still achieve significant functional benefits from IV thrombolysis. This result is expected to drive updates in stroke treatment guidelines, providing opportunities for more patients with delayed presentation, particularly in areas where endovascular thrombectomy resources are not immediately available.— 09 —
WJP: Song Hongmei's Team Finds Tofacitinib Provides a New Treatment Option for Persistent Rash in Juvenile Dermatomyositis
Chen ZY, Zhang TN, Li J, Zhang ZJ, Song HM. Tofacitinib: a promising agent for the treatment of persistent rashes in juvenile dermatomyositis. World J Pediatr. 2025;21 (4):419-422. doi:10.1007/s12519-025-00901-x
lJournal: World Journal of Pediatrics
lCorresponding/Key Author: Song Hongmei
lAffiliation: Peking Union Medical College Hospital, CAMS
Juvenile dermatomyositis (JDM) is a chronic pediatric autoimmune disease where standard treatments can control myositis symptoms but struggle to improve persistent rash. Song Hongmei's team at Peking Union Medical College Hospital conducted a study, evaluating for the first time the efficacy of Tofacitinib for persistent rash in JDM refractory to conventional therapy.
The study detailed treatment experiences of 8 patients with refractory JDM using Tofacitinib. Results showed that Tofacitinib significantly improved skin symptoms, allowing patients to reduce or even discontinue glucocorticoids without significant adverse reactions. This study confirms the efficacy and safety of Tofacitinib for persistent rash in JDM, providing a potential new option for refractory clinical cases, though subsequent prospective studies are needed to further verify its effects on preventing relapse and calcinosis.— 10 —
JAMA: Lou Min's Team Releases the HOPE Study, Extending the Stroke IV Thrombolysis Window to 24 Hours via Perfusion Imaging Selection
Zhou Y, He Y, Campbell BCV, et al. Alteplase for Acute Ischemic Stroke at 4.5 to 24 Hours: The HOPE Randomized Clinical Trial. JAMA. 2025;334(9):788-797. doi:10.1001/jama.2025.12063
lJournal: JAMA
lCorresponding/Key Author: Lou Min
lAffiliation: Second Affiliated Hospital, Zhejiang University School of Medicine
The HOPE study, led by Professor Lou Min’s team at the Second Affiliated Hospital of Zhejiang University, evaluated the efficacy and safety of IV alteplase in acute ischemic stroke patients within 4.5 to 24 hours using perfusion imaging screening. Traditional views suggest high risk and limited benefit after 4.5 hours; however, as the concept shifts from "time window" to "tissue window," more patients can be confirmed via imaging to still have salvageable brain tissue. This study, conducted across 26 stroke centers in China, fills a critical gap in high-quality RCT evidence, enrolling 372 patients with salvageable tissue and no initial plan for endovascular thrombectomy.
Results showed that the 90-day functional independence rate with alteplase was significantly higher than standard treatment (40% vs. 26%, aRR 1.52; P=0.004), with an absolute benefit of 13.98%. While the incidence of symptomatic intracranial hemorrhage within 36 hours was slightly higher (3.8% vs. 0.51%), the 90-day mortality was 11% in both groups (no difference), indicating that extending the window under imaging screening is safe and controllable. The median age was 72, and baseline NIHSS mostly reflected mild-to-moderate strokes, providing representative heterogeneity.
In summary, provided that perfusion imaging confirms salvageable tissue, patients with acute ischemic stroke can still achieve significant functional improvement from IV alteplase 4.5 to 24 hours after onset, without an observed increase in long-term mortality risk. This finding reflects a shift in stroke treatment philosophy toward a "tissue window" based on tissue viability, offering a new intervention for patients who miss the 4.5-hour window and providing key clinical evidence for extended-window thrombolysis strategies.
This article presents only a portion of the heavyweight research achievements published by Chinese scientists in 2025. We believe that in 2026, our scientists will create further brilliance and achieve even more significant research milestones!
撰文 | 梅斯医学
编辑 | 阿拉斯加宝
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