Uncommon

不常见
  • 文章类型: Journal Article
    背景:应使用真实世界的患者数据对人工智能(AI)症状检查器模型进行训练,以提高其诊断准确性。鉴于目前在临床实践中使用基于AI的症状检查程序,随着时间的推移,他们的表现应该会有所改善。然而,对这些症状检查程序诊断准确性的纵向评估是有限的.
    目的:本研究旨在评估真实世界中使用的基于AI的症状检查程序创建的鉴别诊断列表准确性的纵向变化。
    方法:这是一个单中心,回顾性,观察性研究。在2019年5月1日至2022年4月30日期间,在没有预约的情况下访问了门诊诊所,并且在索引访问后30天内被送往日本社区医院的患者被认为是合格的。我们只包括在索引访视时接受基于AI症状检查的患者,最终在随访期间确诊。最终诊断分为常见或不常见,所有病例均分为典型或非典型.主要结果指标是基于AI的症状检查器创建的鉴别诊断列表的准确性,在症状检查程序创建的10项鉴别诊断列表中定义为最终诊断。为了评估症状检查者3年内诊断准确性的变化,我们使用卡方检验比较了3个时期的主要结果:2019年5月1日至2020年4月30日(第一年);2020年5月1日至2021年4月30日(第二年);2021年5月1日至2022年4月30日(第三年).
    结果:共纳入381例患者。常见疾病包括257例(67.5%),在298例(78.2%)病例中观察到典型表现。总的来说,基于AI的症状检查器创建的鉴别诊断列表的准确性为172(45.1%),在3年内没有差异(第一年:97/219,44.3%;第二年:32/72,44.4%;第三年:43/90,47.7%;P=.85)。症状检查器创建的鉴别诊断列表的准确性在那些患有罕见疾病(30/124,24.2%)和非典型表现(12/83,14.5%)的患者中很低。在多元逻辑回归模型中,常见疾病(P<.001;比值比4.13,95%CI2.50-6.98)和典型表现(P<.001;比值比6.92,95%CI3.62-14.2)与症状检查程序创建的鉴别诊断列表的准确性显著相关.
    结论:由基于AI的症状检查程序开发的鉴别诊断列表的诊断准确性的3年纵向调查,已在现实世界的临床实践中实施,随着时间的推移没有改善。罕见疾病和非典型表现与较低的诊断准确性独立相关。在未来,应该训练症状检查人员来识别不常见的情况。
    BACKGROUND: Artificial intelligence (AI) symptom checker models should be trained using real-world patient data to improve their diagnostic accuracy. Given that AI-based symptom checkers are currently used in clinical practice, their performance should improve over time. However, longitudinal evaluations of the diagnostic accuracy of these symptom checkers are limited.
    OBJECTIVE: This study aimed to assess the longitudinal changes in the accuracy of differential diagnosis lists created by an AI-based symptom checker used in the real world.
    METHODS: This was a single-center, retrospective, observational study. Patients who visited an outpatient clinic without an appointment between May 1, 2019, and April 30, 2022, and who were admitted to a community hospital in Japan within 30 days of their index visit were considered eligible. We only included patients who underwent an AI-based symptom checkup at the index visit, and the diagnosis was finally confirmed during follow-up. Final diagnoses were categorized as common or uncommon, and all cases were categorized as typical or atypical. The primary outcome measure was the accuracy of the differential diagnosis list created by the AI-based symptom checker, defined as the final diagnosis in a list of 10 differential diagnoses created by the symptom checker. To assess the change in the symptom checker\'s diagnostic accuracy over 3 years, we used a chi-square test to compare the primary outcome over 3 periods: from May 1, 2019, to April 30, 2020 (first year); from May 1, 2020, to April 30, 2021 (second year); and from May 1, 2021, to April 30, 2022 (third year).
    RESULTS: A total of 381 patients were included. Common diseases comprised 257 (67.5%) cases, and typical presentations were observed in 298 (78.2%) cases. Overall, the accuracy of the differential diagnosis list created by the AI-based symptom checker was 172 (45.1%), which did not differ across the 3 years (first year: 97/219, 44.3%; second year: 32/72, 44.4%; and third year: 43/90, 47.7%; P=.85). The accuracy of the differential diagnosis list created by the symptom checker was low in those with uncommon diseases (30/124, 24.2%) and atypical presentations (12/83, 14.5%). In the multivariate logistic regression model, common disease (P<.001; odds ratio 4.13, 95% CI 2.50-6.98) and typical presentation (P<.001; odds ratio 6.92, 95% CI 3.62-14.2) were significantly associated with the accuracy of the differential diagnosis list created by the symptom checker.
    CONCLUSIONS: A 3-year longitudinal survey of the diagnostic accuracy of differential diagnosis lists developed by an AI-based symptom checker, which has been implemented in real-world clinical practice settings, showed no improvement over time. Uncommon diseases and atypical presentations were independently associated with a lower diagnostic accuracy. In the future, symptom checkers should be trained to recognize uncommon conditions.
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  • 文章类型: Journal Article
    前列腺特异性膜抗原(PSMA)PET/CT越来越多地用作评估前列腺癌(PCa)的混合成像模式。我们报告了一例50岁的男性,其活检证实为高危PCa,其中在68Ga-PSMA-11PET/CT上发现了多个示踪剂,除了多灶性前列腺原发性,广泛的节点,和骨骼转移。此病例强调肾周筋膜是PCa中罕见的转移部位。
    Prostate-specific membrane antigen (PSMA) PET/CT is being increasingly utilized as a hybrid imaging modality for the evaluation of prostate cancer (PCa). We report a case of a 50-year-old man with biopsy-proven high-risk PCa in which multiple tracer avid perirenal fascia deposits were identified on 68Ga-PSMA-11 PET/CT, in addition to multi-focal prostatic primary, extensive nodal, and skeletal metastases. This case highlights that perirenal fascia is an uncommon metastatic site in PCa.
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  • 文章类型: Case Reports
    我们介绍了一例40岁的白人男性,其既往有滥用多种物质(可卡因和甲基苯丙胺)的病史,向急诊科(ED)就诊,抱怨间歇性咳嗽并伴有胸部不适和呼吸急促2周。最初的生命体征表现为临界性心动过速(每分钟98次),呼吸急促(37次/分钟),缺氧(室内空气中氧饱和度为89%),他的身体检查非常平淡无奇。包括计算机断层扫描血管造影(CTA)在内的初步检查显示,A型主动脉夹层伴有胸部和腹部受累,患者入院。该患者在移植后切除了升主动脉,体外循环,使用复合假体和左右冠状动脉重建和再植入的主动脉根部置换术,并在复杂的住院过程中幸存下来。这个案例证明了娱乐性药物使用之间存在的经典关联,特别是可卡因和安非他明等兴奋剂,和急性主动脉夹层(AAD)。然而,这种临界亚急性的表现,在多物质使用的背景下进行无痛解剖引发了进一步的问题,因为罕见的AAD通常在高风险人群中发现,如结缔组织疾病(马凡氏综合征,Ehlers-Danlos综合征,Loeys-Dietz综合征),二叶主动脉瓣,慢性高血压,或先前的主动脉病理学。因此,我们建议临床医生强烈考虑将罕见的AAD作为已知或高度怀疑多物质滥用的患者的鉴别诊断的一部分。
    We present a case of a 40-year-old Caucasian male with past medical history of polysubstance abuse (cocaine and methamphetamine), who presented to the emergency department (ED) complaining of intermittent cough with associated chest discomfort and shortness of breath for 2 weeks. Initial vital signs demonstrated borderline tachycardia (98 beats per minute), tachypnea (37 times per minutes), and hypoxia (oxygen saturation 89% on room air), and his physical exam was grossly unremarkable. A preliminary workup including a computed tomography angiography (CTA) revealed a type A aortic dissection with both thoracic and abdominal involvement for which the patient was admitted. This patient had resection of the ascending aorta with graft placement, cardiopulmonary bypass, aortic root replacement using composite prosthesis and left and right coronary reconstruction and reimplantation and survived a complicated hospital course. This case demonstrates the classic association known to exist between recreational drug use, specifically stimulants such as cocaine and amphetamines, and acute aortic dissection (AAD). However, such a presentation of borderline subacute, painless dissection in the setting of polysubstance use raises further questions, since uncommon AAD is typically found in higher-risk populations such as those with connective tissue disorders (Marfan syndrome, Ehlers-Danlos syndrome, Loeys-Dietz syndrome), bicuspid aortic valve, chronic hypertension, or previous aortic pathology. We therefore suggest clinicians strongly consider uncommon AAD as part of their differential diagnosis in patients with known or highly suspected polysubstance abuse.
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  • 文章类型: Journal Article
    男性乳腺癌(MBC)是一种罕见的恶性肿瘤,占男性所有恶性肿瘤的不到1%。然而,MBC的临床病理特征与女性乳腺癌并不完全相似,它的治疗符合女性乳腺癌方案。
    为了回顾性分析MBC的分布趋势,介绍,治疗,和结果。
    回顾性分析了1991年至2020年的106例MBC患者。进行人口统计学和临床病理数据以及治疗变量的频率分布分析。
    演讲的中位年龄为57岁;范围从30到86岁。任一侧几乎同样受到1.2:1的R:L比率的影响。平均投诉时间为26.2个月(范围1-240个月)。18例患者有妇科乳房发育史,13例良性前列腺肥大,14例高血压需要药物治疗。大多数患者是吸烟者(72/106)和酗酒者(43/106)。5例患者报告阳性家族史。21例患者在就诊时患有转移性疾病,并接受姑息治疗。第二阶段为36.8%,第三阶段为43.4%,和IV期19.8%的患者。节点阳性为63.2%。病理总是(90.5%)浸润性导管癌。85.8%的病人接受了放疗,72.6%的患者接受化疗,47.2%的患者接受了激素治疗。中位总生存期(OS)为78个月。5年和10年的OS分别为78%和58%。
    尽管MBC可能在早期阶段出现,存在局部晚期疾病的患者。辅助/新辅助化疗和辅助放疗的根治性手术仍然是金标准。必须开展癌症教育运动,以发现早期疾病并从根本上治疗疾病。
    UNASSIGNED: Male breast cancer (MBC) is one of the rare malignancies that account for less than 1% of all malignancies in males. However, the clinicopathological characteristics of MBC are not entirely similar to female breast cancer; but still, it is treated in line with the female breast cancer protocols.
    UNASSIGNED: To retrospectively analyse trends in MBC as to its distribution, presentation, treatment, and outcome.
    UNASSIGNED: A total of 106 patients with MBC from 1991 to 2020 were analysed retrospectively. Frequency distribution analysis of the demographic and clinicopathological data and treatment variables was done.
    UNASSIGNED: Median age of presentation was 57 years; ranging from 30 to 86 years. Either of the sides was almost equally affected with an R: L ratio of 1.2:1. The average duration of complaint was 26.2 months (range 1-240 months). History of gynaecomastia was noted in 18 patients, significant benign prostate hypertrophy in 13, and hypertension needing medical treatment in 14 patients. The majority of the patients were smokers (72/106) and alcoholics (43/106). Five patients reported positive family history. 21 patients had metastatic disease at presentation and received palliative treatment. Stage II was seen in 36.8%, stage III in 43.4%, and stage IV in 19.8% of patients. Node positives were 63.2%. Pathology was invariably (90.5%) infiltrative ductal carcinoma. Radiation was administered in 85.8% of the patients, chemotherapy in 72.6% of patients, and hormonal treatment was given in 47.2% of patients. The median overall survival (OS) was 78 months. OS at 5 and 10 years was 78% and 58% respectively.
    UNASSIGNED: Despite the possibility of MBC being apparent at an early stage, patients present with locally advanced disease. Radical surgery with adjuvant/neoadjuvant chemotherapy and adjuvant radiotherapy remains the gold standard. Cancer education campaigns must be run to catch the early disease and to radically treat the disease.
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  • 文章类型: Case Reports
    一名43岁的男性使用丙戊酸(VA)治疗癫痫发作2年,出现右侧中度胸腔积液。胸水分析显示有42%嗜酸性粒细胞的渗出性积液。没有血胸的证据,气胸,恶性肿瘤,和寄生虫感染。怀疑与毒品有关的事件,VA已停产。三周后,患者在胸片上显示胸腔积液消退,临床症状有所改善。VA是一种流行的药物,用于各种疾病,如癫痫发作,偏头痛,和精神分裂症。关于VA引起的胸腔积液的文献很少。虽然这是一种罕见的现象,临床医生应该意识到这种可能性,以避免错误诊断。
    A 43-year-old male using valproic acid (VA) for 2 years for seizure disorder presented with right-sided moderate pleural effusion. Pleural fluid analysis revealed exudative effusion with 42% eosinophils. There was no evidence of haemothorax, pneumothorax, malignancy, and parasitic infections. Suspecting a drug-related event, VA was discontinued. The patient showed clinical improvement with resolution of pleural effusion on chest radiograph three weeks later. VA is a popular drug used for variety of disorders like seizures, migraines, and schizophrenia. There is a paucity of literature on VA-induced pleural effusion. Though a rare phenomenon, clinicians should be aware of such a possibility to avoid erroneous diagnosis.
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  • 文章类型: Journal Article
    未经证实:不常见的表皮生长因子受体(EGFR)突变由分子改变的异质群体组成,关于阿法替尼治疗后EGFR突变罕见的非小细胞肺癌(NSCLC)患者结局的现有临床数据有限.此汇总分析的目的是调查罕见EGFR突变(um-EGFRms)患者的临床病理特征以及阿法替尼治疗后的治疗反应和生存结果。
    UNASSIGNED:我们在NCBIPubMed数据库中进行了文献检索,以确定相关文章,并基于70项研究进行了此汇总分析。患者临床特征之间的关系,EGFR突变类型和阿法替尼治疗的反应使用单变量卡方分析,采用Kaplan-Meier法进行生存分析.
    UNASSIGNED:共有99名患者的数据被纳入汇总分析。阿法替尼治疗的客观缓解率(ORR)为53.5%,中位无进展生存期(mPFS)为9.0个月。对于接受一线阿法替尼治疗的患者,ORR和中位PFS分别为73.5%和15.6个月,分别,均优于接受二线或后期治疗的患者(ORR:37.0%,p<0.001;mPFS:6.0个月,p=0.001)。此外,单一um-EGFRm的患者在阿法替尼治疗后比多个um-EGFRm的患者更有可能具有良好的反应和预后益处(ORR:63.3%vs38.5%,p=0.017;mPFS:15.6个月vs6.0个月,p=0.010)。此外,单个um-EGFRm是更好的治疗反应和更好的PFS的独立预测因素。亚组分析表明,患者携带主要um-EGFRms(即,L861Q,G719X,和S768I)表现出最佳的治疗反应和预后(ORR:74.1%,mPFS:15.6个月),相比之下,包含19del/L858R的多个um-EGFRms的患者的治疗反应和预后最差(ORR:23.5%,mPFS:5.6个月)。
    未经评估:um-EGFRms患者在阿法替尼治疗后表现出良好但不一致的反应和生存结果,与突变模式和共生伴侣突变基因密切相关。在某些情况下,使用阿法替尼治疗um-EGFRm患者可能被认为是一种有效的治疗选择。但这一建议需要进一步的临床研究进行验证.
    UNASSIGNED: Uncommon epidermal growth factor receptor (EGFR) mutations consist of a heterogeneous population of molecular alterations, and the available clinical data on the outcomes of patients with non-small-cell lung cancer (NSCLC) harboring uncommon EGFR mutations following afatinib treatment are limited. The purpose of this pooled analysis was to investigate the clinicopathological features of patients with uncommon EGFR mutations (um-EGFRms) along with their treatment response and survival outcomes following afatinib treatment.
    UNASSIGNED: We performed a literature search in the NCBI PubMed database to identify relevant articles and conducted this pooled analysis based on 70 studies. The relationships between patient clinical characteristics, EGFR mutation type and the response to afatinib treatment were analyzed using univariate chi-square analysis, and survival analysis was performed using the Kaplan-Meier method.
    UNASSIGNED: Data from a total of 99 patients were included in the pooled analysis. The objective response rate (ORR) to treatment with afatinib was53.5%, with a median progression-free survival (mPFS) of 9.0 months. For patients administered first-line afatinib treatment, the ORR and median PFS were 73.5% and 15.6 months, respectively, which were both superior to those of patients treated with second- or later-line treatments (ORR:37.0%, p < 0.001; mPFS: 6.0months, p = 0.001). Moreover, patients with a single um-EGFRm were more likely to have a favorable response and prognosis benefit after treatment with afatinib than patients with multiple one (ORR: 63.3% vs 38.5%, p=0.017; mPFS: 15.6 months vs 6.0 months,p=0.010). Moreover, single um-EGFRm were independent predictive factors for better treatment response and superior PFS. Subgroup analysis indicated that patients harboring major um-EGFRms (i.e., L861Q, G719X, and S768I) exhibited the best treatment responses and prognoses (ORR: 74.1%, mPFS: 15.6 months), by contrast, patients harboring multiple um-EGFRms comprising 19del/L858R had the worst treatment responses and prognoses (ORR: 23.5%, mPFS: 5.6months).
    UNASSIGNED: Patients with um-EGFRms exhibit favorable but inconsistent responses and survival outcomes following afatinib treatment, which closely related to the mutation pattern and cooccurring partner mutant genes. Administering afatinib for the treatment of patients with um-EGFRm might be considered an effective treatment option in some circumstances, but this recommendation requires further clinical studies for verification.
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  • 文章类型: Journal Article
    背景:在这个汇总分析中,目的是研究罕见表皮生长因子受体(EGFR)(ucm-EGFRms)患者的临床病理特征,以及奥希替尼治疗后的治疗反应和生存率.方法:采用单因素卡方分析临床特征,EGFR突变类型,和治疗反应,应用Kaplan-Meier法进行生存分析。应用单因素logistic回归模型和Cox比例风险模型比较亚组疗效和预后。结果:共有72例NSCLC患者被纳入该汇总分析。奥希替尼治疗的客观缓解率(ORR)为57.0%,平均PFS为7.1个月。28例患者接受奥希替尼作为一线治疗,ORR为67.9%,高于接受奥希替尼作为二线或后期治疗的患者,他们的反应率为50%,然而,差异无统计学意义(p=0.139).然而,接受奥希替尼一线治疗的患者比接受二线或后期治疗的患者有更显著的PFS获益(mPFS:16.8个月vs6.0个月HR:2.453,95CI:1.285~4.682,p=0.004).亚组分析显示,非外显子20ins,ucm-EGFRm在奥希替尼治疗后显示出优异的疗效优势和良好的生存获益,ORR为68.8%,mPFS为15.1个月。相比之下,含有T790M的多个ucm-EGFRm的患者表现出奥希替尼治疗的最差结局,ORR为47.6%,mPFS仅为3.6个月,分别。结论:um-EGFRms患者在奥希替尼治疗后表现出良好但不一致的反应和生存结果,这与突变模式和共生伴侣突变基因密切相关。在某些情况下,服用奥希替尼治疗um-EGFRm患者可能被认为是一种有效的治疗选择。
    Background: In this pooled analysis, the aim was to investigate the clinicopathological characteristics of patients with uncommon epidermal growth factor receptor (EGFR) (ucm-EGFRms) along with their treatment responses and survival following osimertinib treatment. Methods: Univariate chi-square analysis was conducted to analyze the correlation between clinical characteristics, EGFR mutation type, and treatment response, and the Kaplan-Meier method was applied for survival analysis. Univariate logistic regression model and Cox proportional hazards model were performed to compare the efficacy and prognosis in subgroup analysis. Results: Seventy-two NSCLC patients in total were included in this pooled analysis. The objective response rate (ORR) for osimertinib treatment was 57.0%, with a median PFS of 7.1 months. Twenty-eight patients received osimertinib as first-line therapy with an ORR of 67.9%, which was higher than that in patients who received osimertinib as second- or later-line therapy, and their response rate was 50%, nevertheless, no statistically significant differences were found (p = 0.139). However, patients who received first-line osimertinib showed a more significant PFS benefit than those who received second- or later-line therapy (mPFS: 16.8 months vs 6.0 months HR: 2.453, 95%CI: 1.285-4.682, p =0.004). Subgroup analysis showed that patients with a single, non-ex20ins, ucm-EGFRm displayed a superior efficacy advantage and favorable survival benefit following osimertinib treatment, with an ORR of 68.8% and an mPFS at 15.1 months. By contrast, patients with a multiple ucm-EGFRm that contain T790M exhibited the worst outcome of osimertinib treatment, with an ORR of 47.6% and an mPFS of only 3.6 months, respectively. Conclusion: Patients with um-EGFRms exhibit favorable but inconsistent responses and survival outcomes following osimertinib treatment, which is closely related to the mutation pattern and cooccurring partner mutant genes. Administering osimertinib for the treatment of patients with um-EGFRm might be considered an effective treatment option in some circumstances.
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  • 文章类型: Journal Article
    隐源性卒中的病因复杂且异质性。一些不常见的病因没有被充分认识,其中一些主要影响女性。这些病因大多与激素水平密切相关,生殖因素,凝血功能,女性的药物。此外,一旦诊断出隐源性中风,女性往往有更糟糕的结果。因此,及时的病因识别和治疗对于良好的恢复至关重要。本文的目的是回顾探索隐源性卒中罕见的女性主要病因的进展。这些病因分为动脉,心脏,和静脉来源。动脉血管收缩性狭窄,内膜损伤,内膜发育异常可引起脑缺血或动脉-动脉脑栓塞。心肌收缩功能障碍,心壁损伤,发育异常可诱发心内血栓形成并导致心脏栓塞。此外,皮质静脉血栓形成和通过心内或心外通道的隐匿性静脉血栓栓塞也是女性隐源性卒中的原因。由于知识的缺乏,在临床实践中,上述病因很少被评估。这些病因的低发病率可导致漏诊。该综述将为隐源性卒中的病因诊断提供新的临床线索,并将有助于改善女性人群卒中的管理和二级预防。在未来,还需要更多的研究来探讨隐源性卒中的病因和预防策略。
    The etiologies of cryptogenic stroke are complex and heterogeneous. A number of uncommon etiologies are not fully recognized, some of which predominantly affect females. Most of these etiologies are closely related to the hormonal level, reproductive factors, coagulation function, and medications of females. Moreover, once cryptogenic stroke is diagnosed, females tend to have worse outcomes. Therefore, prompt etiological recognition and treatment are crucial for good recovery. The aim of this article is to review advances in exploring uncommon female-predominant etiologies of cryptogenic stroke. These etiologies are categorized into arterial, cardiac, and venous sources. Arterial vasoconstrictive narrowing, intimal injury, and intimal developmental abnormality can cause brain ischemia or artery-to-artery cerebral embolism. Myocardial contraction dysfunction, cardiac wall injury, and developmental abnormality can induce intracardiac thrombosis and lead to cardiac embolism. In addition, cortical venous thrombosis and occult venous thromboembolism via intracardiac or extracardiac channels also account for cryptogenic stroke in females. Due to the lack of knowledge, in clinical practice, the above etiologies are seldom assessed. The low incidence rate of these etiologies can lead to missed diagnosis. This review will provide novel clinical clues for the etiological diagnosis of cryptogenic stroke and will help to improve the management and secondary prevention of stroke in the female population. In the future, more studies are needed to explore the etiology and prevention strategies of cryptogenic stroke.
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  • 文章类型: Journal Article
    背景:达科替尼是第二代药物,不可逆表皮生长因子受体酪氨酸激酶抑制剂(EGFR-TKI)。ARCHER-1050表明,与吉非替尼相比,这种药物可以改善EGFR突变敏感的晚期非小细胞肺癌患者的无进展生存期和总生存期。然而,目前尚不清楚达克替尼对18-21号外显子罕见EGFR突变致敏患者是否有效.这项研究的目的是研究达克替尼在这些患者中的安全性和有效性。
    方法:这是一个单臂,prospective,开放标签和II期试验。样本量将根据以下参数通过minimax两阶段设计方法计算:α=0.075,1-β=0.9,P0=0.20,P1=0.45和10%的辍学率。总共将包括30名符合条件的患者。患者将接受达克替尼(45毫克/天)的连续口服治疗,直至疾病进展,撤回同意,或不可接受的毒性,以先发生者为准。主要终点是根据RECIST1.1版的客观缓解率(ORR),由研究者评估。第二个终点是疾病控制率(DCR),PFS,操作系统,和安全。
    结论:我们进行单臂,II期研究旨在研究达克替尼在具有敏感罕见EGFR突变的晚期NSCLC患者中的安全性和有效性。DANCE研究的结果将提供有关这些患者的疗效和安全性的新数据。
    背景:NCT04504071。
    BACKGROUND: Dacomitinib is a second-generation, irreversible epidermal growth factor receptor tyrosine kinase inhibitor (EGFR-TKI). ARCHER-1050 showed that this agent can improve progression-free survival and overall survival in advanced non-small cell lung cancer patients with sensitive EGFR mutation compared to gefitinib. However, it is unclear whether dacomitinib is effective in patients with sensitizing uncommon EGFR mutations in exon 18-21. The aim of this study is to investigate the safety and efficacy of dacomitinib in these patients.
    METHODS: This is a single arm, prospective, open label and phase II trial. Sample size will be calculated by a minimax two-stage design method based on the following parameters: α = 0.075, 1-β = 0.9, P0 = 0.20, P1 = 0.45 and a dropout rate of 10%. A total of 30 eligible patients will be included. Patients will receive continuous oral therapy with dacomitinib (45 mg/day) until disease progression, withdrawal of consent, or unacceptable toxicity, whichever occurs first. The primary endpoint is objective response rate (ORR) per RECIST version 1.1, as assessed by investigators\' review. The second endpoint is disease control rate (DCR), PFS, OS, and safety.
    CONCLUSIONS: We conduct a single arm, phase II study to investigate the safety and efficacy of dacomitinib in advanced NSCLC patients with sensitizing uncommon EGFR mutations. The results of the DANCE study will provide new data regarding efficacy and safety of these patients.
    BACKGROUND: NCT04504071.
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  • 文章类型: Case Reports
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