tbuckley/cabot-search huggingface dataset:
Development of urine metabolomic biomarkers for ifosfamide-induced encephalopathy (IIE) in soft tissue sarcoma patients.
Authors: Garrett Kinnebrew, David A. Liebner, Audra Phillips, Colin W. Stets, Gabriel Tinoco, Ewy Mathe, James Lin Chen
Journal: Journal of Clinical Oncology (2020)

e23534 Background: In the treatment of sarcomas, high doses of ifosfamide in combination with other chemotherapeutic agents remains a backbone of cytoreductive therapy. Unfortunately, IIE remains a prevalent treatment side effect with a reported prevalence of 10-20%. Although mild hypoalbuminemia has previously been implicated as a risk factor, its risk contribution is modest. There are no clinically significant biomarkers which may help identify IIE patents. Given the correlation with hypoalbuminemia to renal function, we hypothesized that urinary metabolites may serve as potential early biomarkers for IIE. Here we report our interim biomarker findings as part of our sarcoma metabolomic biocollection protocol. Methods: All sarcoma patients that received ifosfamide were identified (2011-2020) through a sarcoma registry (OSU-14242). Development of IIE was obtained through chart review. For consented patients undergoing adriamycin and ifosfamide treatment, urine samples were obtained over the course of 5 days and two treatment cycles (OSU IRB: 2014C0181). Urine samples were subjected to targeted metabolomic analysis for known ifosfamide metabolites and untargeted metabolomic profiling. LC-MS was performed on a Sciex TripleTOF 5600 and data preprocessing with XCMS. For each metabolite, mean abundance and slope, as determined by linear regression across all time points, was compared to development of IIE coded as a binary variable. Results: A total of 70 sarcoma patients were identified, of whom 11 developed IIE for a prevalence of 15.7%. Baseline values at the start of ifosfamide treatment were a mean albumin of 3.7 g/dL (+/- 0.38) and a mean serum creatinine of 0.79 mg/dL (+/- 0.26). Both values are within our “normal range”. For seven of these patients, of whom one developed IIE, we successfully collected additional metabolomic profiling. From these data, 6,030 features (representing metabolites or metabolite fragments) were measured. Time course plots of drug metabolites for which pure standards were run showed an increase of expected chemotherapy-related metabolites at the beginning of each cycle, with inter- and intra-patient variability. IIE was associated with 70 metabolite features (by mean abundance, FDR &lt; 5%). Metabolite identification of putative features based on tandem mass spectrometry is in process. Conclusions: This pilot study indicates that urine metabolomic analysis is feasible and may identify IIE biomarkers. We expect to confirm these results once ongoing specimen analysis is complete.
Awareness rate of child health management in parents of children aged 0 - 36 months
Authors: Xiao-bing Fu, Ping Shi
Journal: BMJ (2012)

A questionnaire survey on awareness of child health management was conducted in 238 family members ( 236 were parents) of children aged 0 - 36 months; among whom 92 of children aged 0 to 6 months,85 of children aged 7 to 12 months,33 of children aged 13 to 18 months,16 of children aged 19 to 24 months and 12 of children aged 24 to 36 months.52.1％ of the surveyees had college education level.The awareness about medical examination in last half year in female subjects was higher than males ( P ＜ 0.05).There were no differences in other health management knowledge.The results showed that the average times of medical examination was 2.8 ± 1.9 in last half year; the age for supplement of meat in food was 7.4 ± 2.4 mouths; supplement of meat,animal liver and eggs was more than 7 time a week.The subjects with college education or above paid more attention to the children's growth,risk for rickets and anemia than other peoples.Pure breast feeding accounted for only 37.0％ (88/238).The study indicates that strengthening the knowledge and awareness is necessary to improve the children health management.

Key words: 
Children health services; Questionnaires
Efficacy and safety of camrelizumab combined with FOLFOX as neoadjuvant therapy for patients with resectable locally advanced gastric and gastroesophageal junction adenocarcinoma who received D2 radical gastrectomy.
Authors: Yuzhou Zhao, Guangsen Han, Jing Zhuang, Zhimeng Li, Gangcheng Wang, Baodong Li, Jinbang Wang, Yanhui Gu, Pengfei Ma, Junli Zhang, Yanghui Cao, Chenyu Liu, Sen Li, Xijie Zhang
Journal: Journal of Clinical Oncology (2020)

e16549 Background: Neoadjuvant chemotherapy for patients with locally advanced gastric and gastroesophageal junction adenocarcinoma (GC/GEJC) can improve the overall survival without increasing operation risk. Nowadays, immunotherapy has become a new promising neoadjuvant treatment. Therefore, we intended to evaluate the safety and efficacy of camrelizumab (anti-PD-1 antibody) combined with FOLFOX as the neoadjuvant therapy for patients with locally advanced GC/GEJC who received D2 radical gastrectomy. Methods: Patients who were diagnosed as resectable locally advanced GC/GEJC received the neoadjuvant treatment of camrelizumab and FOLFOX every 2 weeks for 4 cycles. Imaging evaluation was performed in 2-4 weeks after neoadjuvant therapy. Patients who had no progression disease (PD) were recruited. Eligible patients underwent gastrectomy with D2 lymph node dissection through laparotomy or laparoscopic surgery. The primary end points were safety and R0 resection rate. Results: From July 24 2019 to January 31 2020, 15 patients were recruited. The mean age was 57 years. A total of 10(67%) were males and 5(33%) were females. According to AJCC 8 th , cT3 and cT4 were confirmed in 7(47%) patients and 8(53%) patients, N1 and N2 in 7(47%) patients and 8(53%) patients, respectively. During operation, intraperitoneal metastases were found in 2 patients. Of the 13 surgeries, only 2 were laparoscopic and the others were laparotomy. The surgical procedures included Roux-en-Y (9, 69.2%), Billroth II (1, 7.7%) and jejunum interposition (3, 23.1%). Thirteen patients underwent gastrectomy with D2 lymph node dissection and all of them were confirmed R0 resection by postoperative pathology results. The mean lymph node yield was 44.1±13.2 nodes, positive lymph node yield was 1.8±2.8 nodes. Duration time of surgery was 186.5±45.5 minutes, mean blood loss was 219.2±109 ml during the operation. Mean hospital stays were 13.2±2.4 days. Only 1 patient experienced grade 3 pneumonia. Neither serious intraoperative complications nor immune-related adverse events both prior and post operation were observed. There was no treatment-related death. Conclusions: Camrelizumab combined with FLOFOX as neoadjuvant treatment for patients with locally advanced GC/GEJC showed acceptable toxicity and promising efficacy with low complications and mortality. Clinical trial information: NCT03939962 .
Abstract 12318: A Cost-effectiveness Analysis of Ventricular Assist Devices (VAD) for the Treatment of Systolic Heart Failure in Pediatric Patients
Authors: Bryant Priromprintr, Nancy Song, Douglas K Owens, Joshua Salomon, Seo-Ho Cho, Sony Ton, Diana Y Zhao, Christopher S Almond, David N Rosenthal, Jeremy D Goldhaber-Fibert, Andrew Shin
Journal: Circulation (2019)

Introduction: Costs for treating pediatric heart failure are substantial and increasing with utilization of ventricular assist devices (VAD) as a bridge to cardiac transplant. Given the potential h...
Clinical features of acute coronary syndrome patients with prior ischemic stroke
Authors: Fang Wang, Hongbin Mei, Jun Ji, Bing Xu, Jing Zhang, Zhimin Qian, Yunan Gong, Jiayang Liang, Shenghu He
Journal: BMJ (2019)

From November 2014 to July 2017, 637 patients with acute coronary syndrome (ACS) were included in the analysis, among whom there were 48 cases with prior ischemic stroke (7.5%). The risk factors, history, severity of coronary artery disease, medication status, and incidence of adverse cardiovascular and cerebrovascular events (cardiac death, re-infarction, heart failure, stroke) were analyzed. Compare with patients without prior ischemic stroke (control group) patients with prior ischemic stroke (study group) had lower rates in administration of beta blockers [50.00%(24/28) vs. 69.78%(411/589), χ2=8.02, P 0.05); and there was no significant difference in major adverse cardiovascular events between two groups (P>0.05). In the future, more studies are needed for clinical management of this group of patients.


Key words: 
Acute coronary syndrome; Ischemic stroke; Adverse cardiovascular events
主动脉夹层误诊误治四例
Authors: 林晓文, 汪春华, 汪春红, 张静
Journal: BMJ (2008)

一、临床资料 患者男,57岁.因持续性胸骨后刀割样疼痛7 d于2007年7月4日人院.既往高血压病史11年,血压最高达199/120 mm Hg(1 mm Hg=0.133 kPa),未系统治疗。
中性粒细胞缺乏症感染大肠埃希菌58例分析
Authors: 于洪艳, 吴绍丽
Journal: BMJ (2006)

对58例中性粒细胞缺乏症感染大肠埃希菌病例的临床资料进行分析,应用MicroScanA/S-4全自动微生物分析仪鉴定菌株,NC21药敏板进行药敏试验,并对结果作综合分析.结果示滥用头孢三代抗生素增加大肠埃希菌耐药性;对大肠埃希菌耐药率低的抗菌药物是:亚胺培南(1.7%),氧派嗪青霉素/他唑巴坦(10.3%),丁胺卡那霉素(19.0%),头孢西丁(19.0%),头孢他啶(22.4%).提示中性粒细胞缺乏时应缩短粒细胞缺乏期,合理应用抗菌药物,以减少大肠埃希菌感染,降低其耐药率。
Advances of neural protective treatment in Parkinson desease
Authors: 李淑华, 陈海波
Journal: BMJ (2008)

帕金森病(Parkinson disease,PD)是由于进行性中脑黑质多巴胺能神经元变性脱失,导致纹状体多巴胺含量降低所致的神经系统变性疾病.主要表现为静止性震颤、运动减少、肌强直以及姿势平衡障碍。
Prevalence of iron deficiency in cancer patients: The French prospective CARENFER study.
Authors: Elisabeth Luporsi, Aurelien Carnot, Vincent Massard, Sophie Morin, Bruno Chauffert, Anthony Turpin, Jean-Noël Trochu, Patrice Cacoub
Journal: Journal of Clinical Oncology (2020)

e15617 Background: Despite the potential deleterious consequences of iron deficiency (ID) in patients with cancer, under-diagnosis is frequent and existence of ID based on both serum ferritin concentration and transferrin coefficient saturation (TSAT) index is rarely determined. The CARENFER Study aimed to assess prospectively the prevalence of ID, anemia and iron deficiency anemia (IDA) in cancer patients. Methods: The study was conducted in France from May to July 2019, as a prospective cross-sectional survey carried out in 15 oncology units. The analysis focused on 1221 patients with different types of solid malignant tumors. Serum ferritin and iron concentrations, TSAT index and hemoglobin (Hb) level were determined. Based on ESMO 2018 Guidelines, ID was defined as ferritin &lt; 100 μg/L or TSAT &lt; 20%, and then categorized as either absolute ID (ferritin &lt; 100 μg/L) or functional ID (ferritin ≥ 100μg/L and TSAT &lt; 20%). Anemia was defined as Hb level ≤ 11 g/dL. Iron deficiency anemia (IDA) was defined according to ESMO 2018 Guidelines. The prevalence of ID, anemia and IDA was estimated using Agresti-Coull 95% confidence interval. Results: Patient’s median age was 63 years (55% women and 45% men). 89.4% of patients were currently under treatment for their cancer, mainly by chemotherapy (75.4%). The proportion of cancer patients with ferritin level &lt; 100 μg/L was 20.5% [18.3-22.9] and 50.6% [47.8-53.4] of patients had a TSAT index &lt; 20%. Overall, ID was found in 57.9% [55.1-60.6] of patients, with absolute ID accounting for 36% of all ID cases. 36.0% of patients were anemic and among them, IDA was reported in 21.8% [19.6-24.2] of patients. The type of cancer treatment did not influence the ID status as ID was also present in 57.4% of untreated cancer patient. Conclusions: This study highlights the high prevalence of ID in cancer patients, whether or not concomitantly associated with anemia or treatment. These results emphasize the need to better management of ID in cancer thereby optimizing overall patient care. Clinical trial information: NCT03924271.
A phase II, single-arm study of denosumab in multiple myeloma patients with renal insufficiency.
Authors: Elizabeth O'Donnell, Andrew Jenho Yee, Omar Nadeem, Brea Lipe, Jacob Laubach, Ehsan Malek, Andrew Branagan, Robb S. Friedman, Paul G. Richardson, Ajay K. Nooka, Noopur S. Raje
Journal: Journal of Clinical Oncology (2020)

8520 Background: Bone destruction is a devastating consequence of multiple myeloma (MM). An unmet medical need exists for the management of skeletal complications in MM patients with renal insufficiency. Despite the effectiveness of intravenous bisphosphonates, their use is limited in situations where renal dysfunction is present. Denosumab is a fully human monoclonal IgG2 antibody to RANKL that has been shown to be non-inferior to zoledronic acid for SRE rates in MM (Raje, et al. Lancet Oncol). More importantly, it does not have the same renal toxicity as the bisphosphonates. We hypothesize that denosumab can be safely and effectively administered in MM patients with renal insufficiency to improve bone health. This may fill a gap in clinical care for bone-directed therapy in this patient population. Methods: This multi-center study will include forty adult patients with newly diagnosed or relapsed MM with a creatinine clearance of less than 30 mL/min (NCT02833610). All patients will receive a subcutaneous injection of denosumab 120 mg Q4W for a total of 12 cycles. In addition, it is required that all subjects receive daily vitamin D and calcium supplementation at standard doses (at least 2000 mg calcium citrate and1000 IU of vitamin D), unless documented hypercalcemia develops on study. The primary objective is to assess the effect of denosumab 120 mg Q4W on serum c-terminal telopeptide (sCTX). Secondary objectives include evaluation of safety and tolerability of denosumab in patients with renal insufficiency, incidence of hypocalcemia, effect on bone mineral density, effect on urinary n-terminal telopeptide, and proportion of patients who have a documented skeletal-related event. Results: At the time of this analysis, a total of 20 out of a planned 40 patients have been enrolled, including 9 women, 11 men. Of those treated, 8 patients have completed the planned 12 cycles of therapy. Hypocalcemia was observed in 7 (35%) patients. Four patients (20%) experienced grade 3 hypocalcemia, 3 (15%) grade 2 or less. Two patients developed osteonecrosis of the jaw, one (5%) grade 1 and one (5%) grade 4 event. Data for the analysis of the primary endpoint of sCTX levels will be presented. Conclusions: Investigation of denosumab in MM patients with renal insufficiency defined as a creatinine clearance less than 30 mL/min is ongoing. Early data support that denosumab can be safely administered in this population. However, incidence of hypocalcemia despite aggressive prophylactic calcium dosing underscores the importance of the trial to inform safe practice in this more fragile population. Clinical trial information: NCT02833610 .
Serum levels of uric acid in the population of health check-up
Authors: Qing Zhang, Kun Song, Hong-lin Zhao
Journal: BMJ (2010)

Total 11 008 subjects underwent routine health check-up at Tianjin General Hospital in 2008. The results showed that the rate of hyperuricemia was 11.56% ( 1272/11 008 ) in this population, 17. 17% ( 1216/7082 ) for male, and 1.43% (56/3926) for female (P ＜0. 05). The serum uric acid levels were positively correlates with body mass index ( r = 0. 346), waist circumference ( r = 0. 449), systolic blood pressure ( r = 0. 193 ), diastolic blood pressure ( r = 0. 240), total cholesterol levels ( r = 0. 240) and triglyceride ester levels ( r = 0. 284 ). The factors including overweight, hypertension, hyperlipidemia and hyperglycosemia were clustered in subjects with hyperuricemia.

Key words: 
Hyperuricemia;  Health surverys
Advances in etiological study of non-arterial inflammatory ischemic optic neuropathy (NAION)
Authors: Jiaqi Bi, Mengyu Su, Jing Ning, Ying Qi, Ping Qi, Xinyuan Cao, Daofei Qu, Yufu Liu
Journal: BMJ (2018)

Non-arterial Inflammatory ischemic optic neuropathy (NAION) is a common ocular disease in middle and old ages. Symptoms of optic nerve dysfunction in NAION are caused by cerebral ischemia, which leads to optic atrophy. Recent studies have focused on the early interventions targeting NAION′s risk factors to protect the optic nerve and retinal ganglion cells. This article reviews recent progress in studies on the etiology and risk factors of NAION and potential therapies that may help to preserve optic nerve function in NAION.


Key words: 
Optic neuropathy, ischemic; Risk factors; Pathologic processes
慢性阻塞性肺疾病的早期诊断
Authors: 张荣葆, 何权瀛
Journal: BMJ (2004)

慢性阻塞性肺疾病(COPD)是一种主要的慢性致残和致死性疾病,近年来全球的患病人数在不断增加[1].根据全球疾病负担的研究,到21世纪的上半叶,COPD将成为全球第5位致残和第3位致死性疾病.对COPD的早期诊断和早期干预非常重要.理想的情况是及早发现COPD危险人群,对其进行密切随访,早期干预,以预防疾病发生或至少延缓疾病的发展.然而,实际上COPD的早期诊断常常被忽视。
Pharmacogenetics findings in Brazilian cancer patients.
Authors: Luis Eduardo Werneck De Carvalho, Jonathan Souza Sarraf, Taynah Cascaes Puty, Juliana Ramos Chaves, Thiago Farias Câmara, Nise Hitomi Yamaguchi, Fernando Adami
Journal: Journal of Clinical Oncology (2020)

e13607 Background: Pharmacogenetic is the study of genetic modifications influence on drugs action. Recent studies have suggested that some genetic polymorphisms may have an influence on patient outcomes. Thus, the objective of this thesis is to evaluate the frequency of genetic polymorphisms in cancer patients. Methods: Cross-sectional study at the Oncology Center of São Paulo. The reports presented a SNP evaluation (saliva - PCR-Multiplex, PCR-RFLP or capillary sequencing - Genotypic variants: 1 allele, 2 alleles and no allelic alteration). Polymorphs were validated according to the localized gene and its relationship to drugs using PharmGKB, NCBI SNP Database, NCBI Gene Database and DrugBank Database. Results: Sample mean aging was 49.92 years (SD = 22.73), with breast cancer (n = 26 /% = 26.3) and stage IV (n = 51 /% = 51.6). Among the evaluated platinum components, it is possible to verify that the presence of polymorphisms was more frequent. Of these, all had the highest frequency of polymorphisms in only 1 allele (xpc__rs2228001 = 25.4%; ercc1_rs11615 = 21.2%; xrcc3_rs861539 = 22.2%; mthfr_rs1801133 = 44.4%). This was repeated when evaluating the Cyclophosphamide, Fluoropyrimidines, Taxanes, Anastrozole and Epirubicin-related polymorphisms. When we evaluated the Tamoxifen-related genes, the polymorphisms absence was more frequent (cyp2d6_rs3892097 = 26.3%; cyp3a4_rs2740574 = 61.6%). Evaluating all medications the highest frequency was polymorphism in only 1 allele. Consequently, for the irinotecan drug evaluation, two polymorphisms were evaluated and presence of polymorphism (xrcc3_rs861539 = 58%), mostly in only 1 allele (44%). Conclusions: Therefore we can evaluate that the allelic frequency variability of the polymorphs observed, despite the great variability, shows most evaluated individuals present some type of genotypic modification, being the majority in only 1 allele.
如何在临床中实践循证医学
Authors: 周亚友
Journal: BMJ (2012)

通读本期文章,感触颇深.其一,几乎所有栏目均有与全科医学直接相关的内容,充分体现了本刊为全科医生服务的宗旨,对基层全科医生工作有较强的针对性和启发;其二,既有不少热心关注全科医学发展的专科医生之作,更有许多从事全科医学教育培训和一线全科医生的研究成果;其三,既有理论研究新进展的阐述,又有临床思路、方法的推介.真可谓开卷有益。
Validation of targeted next-generation sequencing for RAS mutation detection in FFPE colorectal cancer tissues: comparison with Sanger sequencing and ARMS-Scorpion real-time PCR
Authors: 高洁, 吴焕文, 汪莉, 张卉, 段焕利, 陆俊良, 梁智勇
Journal: BMJ (2016)

目的
通过与Sanger测序与扩增阻滞突变系统（ARMS）技术对比，验证靶向新一代测序平台Ion Torrent PGM在甲醛固定石蜡包埋（FFPE）结直肠癌组织标本KRAS 2号外显子及扩展RAS突变检测中的应用。


研究地点
中国北京。


研究对象
随机选取51例中国北京协和医院病理科存档的FFPE结直肠癌标本（36例男性，15例女性）。在检测前，这些标本的病理诊断及肿瘤组织含量均经资深病理医师确认。


方法
分别利用PGM平台、Sanger测序技术以及Therascreen KRAS突变检测试剂盒检测51例FFPE结直肠癌标本中RAS突变情况，评估3种方法之间的一致性，进而通过检测梯度稀释的FFPE细胞系（突变状态已知）来探讨PGM平台的检测敏感性。


结果
应用PGM平台, 51例标本中13例（25.5%）检出了KRAS 2号外显子突变。针对KRAS 2号外显子突变检测，PGM平台与Sanger测序之间的一致性达100%(51/51) [κ=1.000, 95％可信区间（CI）: 1~1]；PGM平台与Therascreen检测试剂盒之间的一致性为98.04% (50/51) (κ=0.947, 95% CI: 0.844~1)，仅有1例不一致的病例，其KRAS 2号外显子突变为c.37G>T突变，该突变未被Therascreen检测试剂盒覆盖。梯度稀释实验结果显示，PGM平台能够检出突变频率低至1％的KRAS突变。重要的是，在51例标本中，PGM平台还检出了10例KRAS 2号外显子以外区域的RAS突变。此外，PGM平台还能够同时检出RAS以外的其他结直肠癌相关基因的突变。


结论
靶向新一代测序平台兼具特异性与敏感性，适合应用于KRAS 2号外显子突变的临床常规检测。此外，该平台节省样本与时间、性价比高，在FFPE结直肠癌标本的扩展RAS突变检测以及其他结直肠癌相关基因突变检测方面具有重要的临床应用前景。
视网膜检影镜在儿童眼屈光状态评估中的应用
Authors: 李丽红, 许江涛, 邓敏, 钟江涛, 肖亦爽, 郝玉星, 马艳玲, 孙青, 周莹
Journal: BMJ (2007)

儿童眼病的诊断、治疗、预防和康复工作中,检查是评估儿童视功能的惟一线索,一个普通的眼科检查内容包括:视力、眼位、眼球运动、裂隙灯、直接或间接眼底镜检查.然而对保健机构的医生和社区医生来说,要完成这些检查是很困难的,尤其对一个生长发育期的儿童来说,视力表视力有时只能做参考,有时就根本不可能完成检查,往往使一些患者延误治疗时机。
Donafenib versus sorafenib as first-line therapy in advanced hepatocellular carcinoma: An open-label, randomized, multicenter phase II/III trial.
Authors: Feng Bi, Shukui Qin, Shanzhi Gu, Yuxian Bai, Zhendong Chen, Zishu Wang, Jieer Ying, Yinying Lu, Zhiqiang Meng, Hongming Pan, Ping Yang, Helong Zhang, Xi Chen, Aibing Xu, Xiufeng Liu, Qiu Meng, Liqing Wu, Feng Chen
Journal: Journal of Clinical Oncology (2020)

4506 Background: Sorafenib is still the standard first-line therapy for advanced hepatocellular carcinoma (HCC). Donafenib, a novel multikinase inhibitor, showed potential benefits in a previous phase Ib study in HCC. Methods: In this open-label, randomized phase II/III trial (ZGDH3), patients with unresectable or metastatic HCC, a Child-Pugh liver function score ≤ 7, and no prior systemic therapy were enrolled from 37 sites across China and randomized (1:1) to receive oral donafenib (0.2 g) or sorafenib (0.4 g) twice daily until intolerable toxicity or disease progression. The primary endpoint was overall survival (OS). Efficacy analysis was primarily based on the full analysis set (FAS). Results: Between March 2016 and April 2018, 668 patients were randomized (donafenib, 334; sorafenib, 334) and included in the intention-to-treat (ITT) set, of whom 659 were analysed by FAS (328 vs 331). Donafenib was associated with a significantly longer median OS than sorafenib in both FAS (12.1 months vs 10.3 months, hazard ratio 0.831, 95% confidence interval 0.699–0.988, p = 0.0363) and ITT (12.0 months vs 10.1 months, 0.839, 0.706–0.996, p = 0.0446). There were no significant differences in median progression-free survival (3.7 months vs 3.6 months, p = 0.2824), objective response rate (4.6% vs 2.7%, p = 0.2448), and disease control rate (30.8% vs 28.7%, p = 0.5532). Grade 3 or worse adverse events (AEs) occurred in 191 (57.4%) and 224 (67.5%) patients ( p = 0.0082), respectively, and AEs of special interest and those leading to treatment interruption occurred in 287 (86.2%) vs 309 (93.1%, p = 0.0049) and 101 (30.3%) vs 141 (42.5%, p = 0.0013). A numerically lower number of patients reported serious AEs (55 [16.5%] vs 67 [20.2%], p = 0.2307) with donafenib. Common AEs with donafenib included hand-foot skin reaction (50.5%), aspartate aminotransferase increased (40.5%), blood bilirubin increased (39.0%), platelet count decreased (37.8%), and diarrhea (36.6%). Conclusions: Donafenib significantly improves OS over sorafenib with favourable safety and tolerability. Donafenib is a promising superior first-line therapy for advanced HCC. Funding: Zelgen. Clinical trial information: NCT02645981 .