抗PD-1抗体联合治疗晚期肝细胞癌的真实世界研究

目的探讨真实世界中以抗PD-1抗体为基础的疗法在晚期肝细胞癌治疗中的疗效、不良反应及可能影响疗效的因素。方法收集55例接受以PD-1抗体为基础治疗的晚期肝细胞癌患者,回顾性分析其临床特点、疗效及不良反应,并进行随访。结果客观有效率为21.8%,疾病控制率为76.4%。治疗过程中不良反应整体发生率为81.8%,其中3~4级不良反应发生率为14.5%,免疫相关不良反应发生率为58.2%,其中3~4级免疫相关不良反应发生率为3.6%,无治疗相关死亡。55例患者中位无进展生存期为5.0月(95%CI:3.9~6.1),中位生存期11.4月(95%CI:6.5~16.3)。应用抗PD-1抗体前患者肝功能Child-Pugh评分和体能状态ECOG评分是影响治疗有效率和生存时间的主要因素;多因素分析也表明治疗前患者的体能状态ECOG评分和肝功能Child-Pugh评分是影响患者生存的独立预后因素(P<0.001,P=0.034)。结论真实世界中以PD-1抗体为基础的治疗在晚期肝细胞肝癌患者中是安全有效的,其中治疗前患者的体能状态ECOG评分和肝功能Child-Pugh评分是影响患者生存期的独立预后因素。     

经导管肝动脉化疗栓塞联合索拉非尼治疗中晚期肝细胞癌的临床观察

目的 观察经导管肝动脉化疗栓塞(TACE)联合索拉非尼治疗中晚期肝细胞癌的有效性和安全性.方法 40例已接受过TACE治疗的中晚期肝细胞癌患者口服索拉非尼单药治疗,400mg,2次/d,直至病情进展或出现不可耐受的毒性反应.按照实体瘤疗效评价标准(RECIST)评价疗效,按照美国国立癌症研究所常见毒性事件标准(NCI-CTCAE)评价不良反应.结果 40例中晚期肝细胞癌患者中,获得完全缓解1例,部分缓解7例,疾病稳定19例,疾病进展13例,疾病控制率为67.5%.全组患者的生存时间为1～18个月,1年生存率为54.0%.主要不良反应为手足皮肤反应,其次是腹泻和血小板计数降低.结论 TACE联合索拉非尼治疗中晚期肝细胞癌是有效和安全的.     

多靶点分子靶向治疗在晚期肝细胞肝癌治疗中的应用研究

目的评价多靶点分子靶向治疗药物甲磺酸索拉非尼治疗晚期／进展期肝细胞肝癌的临床疗效和不良反应。方法2007年4月至2008年11月，中国医学科学院肿瘤医院收治的37例晚期／进展期肝细胞肝癌患者，采用多靶点分子靶向治疗药物甲磺酸索拉非尼（多吉美）治疗，起始剂量400mg，口服，每日2次，治疗过程中根据不良反应发生情况调整用量。每6周、12周全面复查1次，评价肝功能变化、治疗效果和不良反应。用SPSS13．0软件统计中位至疾病进展时间和中位总生存时间。结果至2009年2月，37名患者均达到临床评价要求，无中途停药。治疗相关不良反应发生率为84．8％，其中主要不良反应为：手足皮肤反应、腹泻和高血压。2例Ⅲ级手足皮肤反应患者经调整用药剂量后症状缓解，Ⅰ级不良反应和Ⅱ级不良反应患者经过对症处理后症状缓解，未影响治疗。37名患者治疗前及治疗12周后的ALT、TBIL和ALB无明显变化。疾病进展11例，其中死亡8例。经统计学分析，该组患者中位至疾病进展时间66周，中位生存时间72周。结论无法接受局部治疗，或接受局部治疗后肿瘤进展的晚期／进展期肝细胞肝癌患者，使用甲磺酸索拉非尼治疗，无严重不良反应发生，能显著延长无病生存时间和总生存时间。提前干预不良反应的发生是确保疗效的重要手段之一。     

索拉非尼治疗晚期肝细胞癌１０例临床观察

目的　观察索拉非尼治疗国人晚期肝癌的临床疗效及不良反应。方法　１０例晚期肝细胞癌患者口服索拉非尼４００ｍｇ,每日２次,至少用药６周以上评价疗效。结果　１０例患者均可评价疗效,获得ＣＲ０例,ＰＲ２例,ＳＤ３例,ＰＤ５例,中位ＴＴＰ为４.５个月。主要不良反应为皮疹、腹泻、恶心、呕吐以及手足皮肤反应。结论　索拉非尼治疗国人晚期肝癌患者的疗效较好,不良反应可以耐受。     

以索拉非尼为基础综合治疗晚期原发性肝细胞肝癌的临床研究(附15例分析)

目的探讨以索拉非尼为基础的综合治疗在临床治疗晚期原发性肝细胞肝癌中的初步疗效。方法回顾性分析2008年11月至2009年7月间确诊的原发性肝细胞肝癌,无法接受手术根治性治疗而接受以索拉非尼为基础的综合治疗的患者的相关临床数据。结果本研究共15例患者入组,平均生存时间(10.93±5.548)个月。随访至2010年1月,根据BCLC分期,B级患者的平均生存时间为(11.89±6.153)个月,平均疾病进展时间为(6.56±2.242)个月,存活率88.9%;BCLC分期达C、D级患者的平均生存时间为(9.50±4.637)个月,平均疾病进展时间为(4.50±2.345)个月,存活率33.3%。在15例患者中,4例患者为单用索拉非尼治疗,其余患者均至少联合应用一种其他治疗方式。在BCLC-C/D级的患者中,3例(50%)接受索拉非尼单一治疗,平均生存时间8个月,接受联合治疗的患者,平均生存时间1 1个月。结论索拉非尼治疗可延长晚期原发性肝细胞肝癌的总生存期,与其他治疗联合应用可能存在协同作用,进一步延长进展期/终末期患者的总生存期。     

索拉非尼靶向治疗晚期肾癌的临床疗效与安全性分析

目的 探讨索拉非尼对治疗晚期肾癌的临床治疗效果与安全性.方法 选取56例晚期肾癌患者作为研究对象,使用索拉非尼作为其一线单独用药,口服索拉非尼400mg,2次/d,观察患者的治疗效果以及不良反应.结果 完全缓解0例、部分缓解9例、42例患者病情稳定、5例患者疾病进展,客观反应率为16.1 (9/56),疾病控制率达到91.1％ (51/56).结论 索拉非尼对处于晚期肾癌患者有良好的治疗效果,不良反应深度较轻,具有良好的安全性和耐受性.     

索拉非尼联合经导管肝动脉化疗栓塞治疗无远处转移的晚期肝细胞癌

目的　观察多靶点分子靶向治疗药物索拉非尼联合经导管肝动脉化疗栓塞（ＴＡＣＥ）治疗不伴远处转移的晚期或进展期肝细胞癌的疗效和不良反应。方法　２００７年４月至２００９年９月,中国医学科学院肿瘤医院收治４５例不伴有远处转移的晚期或进展期肝细胞癌患者,口服索拉非尼治疗,其中１８例联合ＴＡＣＥ（１～５次）,２７例单用索拉非尼。索拉非尼起始剂量４００ｍｇ,每日２次,治疗过程中根据不良反应发生情况调整用量。每２个月评价疗效和不良反应,并随访中位至疾病进展时间（ＴＴＰ）和中位总生存时间（ＯＳ）。结果　至２００９年１２月,４０例患者达到临床评价要求（联合ＴＡＣＥ１８例,单用索拉非尼２２例）。两组不良反应发生率无显著差异,主要治疗相关不良反应为手足皮肤反应、腹泻和高血压。两组患者均无４级严重不良反应。索拉非尼联合ＴＡＣＥ组中位ＴＴＰ为１０.０个月,中位ＯＳ１６.０个月;单用索拉非尼组中位ＴＴＰ为４.５个月,中位ＯＳ５.３个月。两组ＯＳ和ＴＴＰ差异有统计学意义（Ｐ＜０.０１）。结论　病变局限在肝内且不合并远处转移的晚期或进展期肝细胞癌患者,口服索拉非尼联合ＴＡＣＥ不增加并发症发生率,且生存预后改善。     

吉西他滨联合多西紫杉醇方案治疗晚期肝癌的临床观察

目的评价吉西他滨联合多西紫杉醇治疗晚期肝细胞癌患者的疗效和不良反应。方法收集我科2006年2月-2010年2月间均诊断为晚期肝细胞癌患者42例。化疗方案剂量及方法设定：多西紫杉醇30 mg/m²,吉西他滨800 mg/m²在d1、d8天应用,静脉滴注。21天为 1周期,2周期后评价疗效。结果全组42例患者均可评价疗效。其中未见完全缓解患者,部分缓解率为21.4%(9/42),稳定47.6%(20/42),进展31.0%(13/42)。中位进展时间4.1月(95％CI：2.14~7.26月),中位生存时间9.2月(95％CI：4.25~18.12月)。无治疗相关死亡,主要不良反应为外周血粒细胞下降、血小板减少、轻度贫血、消化道反应、疲劳、外周神经毒性及偶发的腹泻和皮疹。结论吉西他滨联合多西他赛治疗晚期肝细胞癌具有显著的抗肿瘤作用,化疗期间出现血液学不良反应,食欲下降、乏力是普遍现象,需要临床重视。早期实施有效的干预可明显减少不良反应的发生率。     

索拉非尼在晚期肾癌治疗中所致高血压及其处理

目的:观察索拉非尼治疗晚期肾癌期间高血压的发生及其处理,评估高血压对索拉非尼疗效的预测作用.方法:对30例晚期肾癌患者给予索拉非尼治疗,观察不良反应高血压的发生情况,对高血压进行分级并采取相应的降压治疗措施,观察索拉非尼所致高血压对降压治疗的反应,并采用单因素分析评估高血压对索拉非尼疗效的预测作用.结果:30例晚期肾癌患者服用索拉非尼后,发生高血压的中位时间为15d (6～58d),高血压发生率为30.0％ (9/30),其中大多数(8/9,88.9％)为1～2级.降压治疗后血压控制稳定,未出现高血压危象及相关心脏事件.无患者因严重高血压导致索拉非尼治疗减量或中断.发生高血压对索拉非尼疗效的预测无统计学意义(x2=0.635,P=0.637).结论:索拉非尼治疗晚期肾癌的高血压发生率较高,但以轻至中度为主,降压治疗效果较好.高血压对索拉非尼疗效无预测作用.     

微波消融术联合索拉非尼治疗晚期肝细胞癌的临床研究

目的 比较微波消融术(MWA)联合索拉非尼与索拉非尼单药治疗晚期肝细胞癌(HCC)的临床疗效及不良反应.方法 回顾性分析57例晚期原发性HCC患者的病历和随访资料,其中25例患者接受MWA联合索拉非尼治疗(联合组),32例患者接受索拉非尼单药治疗(单药组),观察终点为两组患者的治疗疗效、无进展生存时间(PFS)、总生存时间(0S)及不良反应.结果 联合组和单药组客观缓解率分别为16.0％和3.1％,差异无统计学意义(χ2=1.521,P=0.217).联合组的疾病控制率为80.0％,明显高于单药组的50.0％,差异有统计学意义(χ2=5.429,P=0.020).联合组的中位PFS显著长于单药组(6.0个月:3.2个月,x2 =7.675,P=0.006),而联合组与单药组的中位OS差异无统计学意义(11.5个月:8.5个月,x2=2.480,P=0.115).联合组和单药组3～4级不良反应发生率分别为44.0％和34.4％,差异无统计学意义(x2=0.549,P=0.459).结论 联合组与单药组比较虽OS无明显延长,但联合组PFS长于单药组,且没有增加严重不良反应发生风险.     

Our experience of the treatment with sorafenib for unresectable hepatocellular carcinoma

We report here the experience of the treatment with sorafenib for advanced hepatocellular carcinoma (HCC) in our department. Forty patients received the therapy of sorafenib until April 2011. Twenty seven unresectable advanced HCC, 7 lung metastasis, 6 bone metastasis, 3 abdominal lymph node metastasis, and 2 peritoneal dissemination were included. The median duration of sorafenib treatment was 197 days. Grade 3 adverse event occurred in 9 patients (22.5%), and grade 4 adverse event occurred in 1 patient (3%). The response rate and disease control rate were 5% and 55%, respectively (CR 2, PR 0, SD 20, PD 9). The median overall survival was 15.2 months, and median recurrence-free survival was 3.7 months. These results suggested that a prevention of adverse events would lead to a continued treatment with sorafenib, and could expect to have a prolonged survival in patients with advanced HCC.     

Evaluation of the mRECIST and α-Fetoprotein Ratio for Stratification of the Prognosis of Advanced-Hepatocellular-Carcinoma Patients Treated with Sorafenib

Objective: To compare the assessment of response and prognosis of patients to sorafenib treatment by the Response Evaluation Criteria in Solid Tumors (RECIST), modified RECIST (mRECIST), α-fetoprotein (AFP) and des-γ-carboxy prothrombin (DCP). Methods: Sixty-six patients with advanced hepatocellular carcinoma (HCC) treated with sorafenib were enrolled in this retrospective study. The response to treatment was evaluated by RECIST, mRECIST and changes in AFP and DCP. Results: The median survival time of all patients was 8.6 months. The median time to radiological progression was 3.3 months. The response rates [complete response (CR) + partial response (PR)] by RECIST and mRECIST were 3.0 and 9.0%, respectively, while the disease control rates [CR + PR + stable disease (SD)] were 50 and 50%, respectively. Assessment by mRECIST of overall survival provided a better stratification of the patients according to the response to treatment (p = 0.009) than RECIST (p = 0.09). Assessment of overall survival by a change in AFP ratio of ≤1 at 8 weeks was better than that of >1 at 8 weeks (p = 0.002). The DCP ratio was not useful for assessment of overall survival. Multivariate analysis identified mRECIST response (CR + PR + SD; p = 0.001), AFP ratio at 8 weeks (≤1; p = 0.046) and Child-Pugh A before treatment (p = 0.012) as significant and independent determinants of survival. The combination of AFP ratio at 8 weeks, assessment by mRECIST and Child-Pugh score before treatment allows stratification of prognosis of patients treated with sorafenib. Conclusion: The combination of mRECIST and AFP ratio is useful for the assessment of prognosis of patients with advanced HCC treated with sorafenib.     

Retrospective analysis of clinical results in eight patients with advanced hepatocellular carcinoma with lung metastases treated by TS-1

Advanced hepatocellular carcinoma (HCC) with distant metastases, in particular to the lung, has a poor prognosis. This study was undertaken to evaluate the effectiveness of TS-1 as chemotherapy in advanced HCC with lung metastases. Between January 2004 and October 2005, 8 patients with advanced HCC with lung metastasis were enrolled. All patients received systemic chemotherapy with TS-1. The drug was administered at a dose of 80 mg/m(2)/day for four weeks, followed by a two-week rest, repeated every six weeks until disease progression, unacceptable toxicity, or the patient's refusal. Median age of the patients was 59 years (range, 44 to 79 years). All patients were in Child-Pugh class A. A total of 22 cycles were administered to each patient (range, 1 to 5). No complete or partial responses were observed. There were two patients (25%) with decreasing tumor marker. Median progression-free survival was 79.5 days (range, 29 to 225). The median overall survival was 257 days (95% confidence interval, 191 to 323 days). TS-1 as chemotherapy was well tolerated when administered in the schedule used in this study. Some patients achieved stable disease and clinical benefits, though this regimen has limited activity in HCC with lung metastases. Randomized controlled trials are necessary to clarify survival benefits in patients with advanced HCC with lung metastasis.     

Sorafenib With and Without Transarterial Chemoembolization for Advanced Hepatocellular Carcinoma With Main Portal Vein Tumor Thrombosis: A Retrospective Analysis

Background.

The survival benefit of combining sorafenib and transarterial chemoembolization (TACE) therapy compared with sorafenib monotherapy for patients with advanced hepatocellular carcinoma (HCC) and main portal vein tumor thrombosis (MPVTT) is unclear.

Methods.

Between January 2009 and June 2013, 183 consecutive patients with advanced HCC (Barcelona Clinic Liver Cancer stage C) and MPVTT were retrospectively reviewed. Of these, 89 patients with advanced HCC and MPVTT were enrolled in this study: 45 were treated with combination therapy (sorafenib-TACE group), and the other 44 treated with sorafenib monotherapy (sorafenib group).

Results.

The mean number of TACE sessions per patient was 2.6 (range: 1–5). The median duration of sorafenib in the sorafenib-TACE group and sorafenib group was 5.6 months and 5.4 months, respectively. The disease control rate was similar between the two groups. Median time to progression was 3.0 months (95% confidence interval [CI]: 2.2, 3.7) in the sorafenib-TACE group, and 3.0 months (95% CI: 2.1, 3.8) in the sorafenib group (p = .924). Median overall survival was 7.0 months (95% CI: 6.1, 7.8) and 6.0 months (95% CI: 4.7, 7.3) in the sorafenib-TACE group and the sorafenib group, respectively (p = .544). The adverse events related to sorafenib were comparable between the two groups. Twenty-one adverse events of grade 3–4 related to TACE occurred in 12 patients (26.7%), and 2 of them died (4.4%).

Conclusion.

This study demonstrated no advantage of combination therapy over sorafenib monotherapy. Considering the patients’ morbidity after TACE, sorafenib monotherapy is appropriate for managing patients with advanced HCC and MPVTT.

Implications for Practice:

For patients with advanced hepatocellular carcinoma (HCC) and main portal vein tumor thrombosis (MPVTT), no benefit was seen in this study in terms of disease control rate, time to progression, and overall survival for patients receiving sorafenib and transarterial chemoembolization compared with those receiving sorafenib monotherapy. Considering the patients’ morbidity after combination therapy, monotherapy is appropriate for managing patients with advanced HCC and MPVTT.     

Thalidomide in advanced hepatocellular carcinoma with optional low-dose interferon-alpha2a upon progression

PURPOSE: To evaluate thalidomide in advanced hepatocellular carcinoma (HCC) and to evaluate combined thalidomide and low-dose interferon-alpha2a (IFN-alpha2a) after tumor progression on thalidomide. Systemic therapy is minimally effective in HCC and tumor angiogenesis is a potential therapeutic target. PATIENTS AND METHODS: Patients with unresectable HCC were eligible if they had preserved hepatic and renal function. The initial thalidomide dosage was 200 mg daily and was adjusted for toxicity. Upon progression, patients could continue thalidomide with additional low-dosage (one million units twice daily) IFN-alpha2a. RESULTS: Thirty-eight enrolled patients were predominantly hepatitis C virus infected (53%), Child-Pugh class A (79%), and Eastern Cooperative Oncology Group performance status 0-1 (92%); 60% had extrahepatic metastasis. Confirmed disease control was seen in seven patients (18%) and included one complete and one partial response (5% response rate). The median progression-free survival was 2.1 months, and median overall survival was 5.5 months. Tumor invasion of the portal vein or vena cava, large (>10 cm) tumor, and younger age were associated with shorter overall survival. Toxicity included fatigue in 74% of patients. Six patients stopped therapy because of side effects, including two patients (5%) with grade 4 arteriothrombotic events. Five patients continued thalidomide upon progression with the addition of IFN-alpha2a; there was no disease control and 80% had grade 3 toxicity. CONCLUSIONS: Thalidomide is not well tolerated and confers limited disease control in advanced HCC. Combination thalidomide and low-dose IFN-alpha2a is neither safe nor efficacious in this population.     

Somatostatin receptor expression, tumour response, and quality of life in patients with advanced hepatocellular carcinoma treated with long-acting octreotide

Octreotide may extend survival in hepatocellular carcinoma (HCC). Forty-one per cent of HCCs have high-affinity somatostatin receptors. We aimed to determine the feasibility, safety, and activity of long-acting octreotide in advanced HCC; to identify the best method for assessing somatostatin receptor expression; to relate receptor expression to clinical outcomes; and to evaluate toxicity. Sixty-three patients with advanced HCC received intramuscular long-acting octreotide 20 mg monthly until progression or toxicity. Median age was 67 years (range 28-81 years), male 81%, Child-Pugh A 83%, and B 17%. The aetiologies of chronic liver disease were alcohol (22%), viral hepatitis (44%), and haemochromatosis (6%). Prior treatments for HCC included surgery (8%), chemotherapy (2%), local ablation (11%), and chemoembolisation (6%). One patient had an objective partial tumour response (2%, 95% CI 0-9%). Serum alpha-fetoprotein levels decreased more than 50% in four (6%). Median survival was 8 months. Thirty four of 61 patients (56%) had receptor expression detected by scintigraphy; no clear relationship with clinical outcomes was identified. There were few grade 3 or 4 toxicities: hyperglycaemia (8%), hypoglycaemia (2%), diarrhoea (5%), and anorexia (2%). Patients reported improvements in some symptoms, but no major changes in quality of life were detected. Long-acting octreotide is safe in advanced HCC. We found little evidence of anticancer activity. A definitive randomised trial would identify whether patients benefit from this treatment in other ways.     

Topotecan plus ifosfamide in patients with platinum refractory advanced/metastatic non-small cell lung cancer: a phase II trial

A number of second line treatments have been proposed in patients with advanced pretreated non-small cell lung cancer (NSCLC). However, either single agents or two or three drug combinations achieved very poor results with no superiority of any combination over monotherapy. We have treated 42 patients (30 males) affected by advanced/metastatic NSCLC progressing during front line cisplatin-based chemotherapy with a combination of topotecan (1.2 mg/m2) plus ifosfamide (1200 mg/m2) for 3 consecutive days every 3 weeks. The median age was 63 years (range 43-76); cell types were: squamous carcinoma (n=17), adenocarcinoma (n=16), large cell carcinoma (n=3), broncho-alveolar carcinoma (n=2) and undifferentiated carcinoma (n=4). All patients were treated with a platinum containing chemotherapy: 39 patients with cisplatin, 2 patients with carboplatin and 1 patient with oxaliplatin, respectively. The ECOG PS was 0 in 8 patients (19%), 1 in 11 patients (26%), and 2 in 23 patients (55%). The median number of courses administered was 3 (range 1-8). Grade 3-4 neutropenia was the dose limiting toxicity, observed in 36% of patients. Moreover, grade 3-4 anemia and thrombocytopenia were observed in 17% and in 12% of patients, respectively. One PS 2 patient died of grade 4 hematological toxicity after the first cycle. No complete response was observed. Six (14.2%) subjects obtained a partial response (PR). In addition, 1 (2.4%) minimal response (MR) plus 14 (34%) stable diseases (SD) and 21 (51%) progressive diseases (PD) were observed. Median time to disease progression and median survival were 9 weeks (range 1-13) and 26 weeks (range 1-91+), respectively. The 1-year survival rate was 14%. Combination of topotecan and ifosfamide demonstrated antitumor activity in patients with relapsing or refractory NCSLC with a modest side effect profile and an overall disease control (PR + MR + SD) of 50.7%. Nevertheless, the still low response rate and the shortness of median survival indicates the need for more effective second line treatments in this disease.     

索拉非尼治疗不能手术的原发性肝细胞癌的疗效及预后因素

目的 研究索拉非尼治疗不能手术的原发性肝细胞癌(HCC)的疗效以及预后影响因素.方法 2005年12月至2009年3月间,50例肝功能分级为Child-Pugh A级的不能手术的原发性HCC患者连续口服索拉非尼治疗,索拉非尼用法为400 mg/次,每日2次.每6～8周复查CT或MRI,根据实体瘤疗效评价标准(RECIST)进行疗效评价,根据美国国立癌症研究所常见毒性分级标准评价不良反应,观察患者的总生存时间(OS)和疾病进展时间(TTP).结果 50例患者均可评价疗效,其中疾病稳定(SD)28例,疾病稳定率为56.0%;疾病进展(PD)22例,占44.0%;无完全缓解(CR)和部分缓解(PR)患者.中位随访时间为15个月,随访期间共有17例患者死亡,全组患者的中位TTP为4个月,中位OS为14个月.索拉非尼治疗不能手术的原发性HCC患者的不良反应为皮肤损害、腹泻、高血压、脱发、骨髓抑制和肝功能损害等,大多为Ⅰ～Ⅱ级,经对症处理或调整用药剂量后多可恢复.单因素分析结果显示,有无远处转移是影响HCC患者TTP的主要因素.结论 索拉非尼可有效治疗不能手术的原发性HCC,有无远处转移是影响HCC患者TTP的主要因素.     

Efficacy and safety of immune checkpoint inhibitor monotherapy in pretreated elderly patients with non-small cell lung cancer

Immune checkpoint inhibitors (ICIs) are an effective subsequent-line treatment for patients with advanced non-small cell lung cancer (NSCLC). However, it remains unclear whether the efficacy and safety of subsequent-line ICI monotherapy in elderly patients (aged ≥ 75 years) are similar to that in non-elderly patients. Therefore, we aimed to investigate the efficacy and safety of ICI monotherapy in pretreated elderly patients with NSCLC. Between January 2016 and February 2018, 131 elderly patients with advanced NSCLC who received subsequent-line ICI monotherapy at 13 Japanese institutions were enrolled in this study. Baseline characteristics, the efficacy of ICI treatment, and adverse events were evaluated. Ninety-eight men and 33 women (median age 77 [range 75–87] years) were enrolled. Among those who received subsequent-line ICI monotherapy, the overall response, disease control rates, median progression-free survival (PFS), and overall survival (OS) were 27.4%, 61.8%, 4.5 months, and 16.0 months, respectively. Adverse events such as anorexia, fatigue, pneumonitis, and hypothyroidism were observed. There were two treatment-related deaths due to pneumonitis and thrombocytopenia. Subsequent-line ICI monotherapy in patients with good performance status (PS), receiving steroids for immune-related adverse events (irAEs), and exhibiting partial response (PR) was associated with improved PFS, as well as OS in patients with good PS and PR. Subsequent-line ICI monotherapy in elderly patients, with previously treated NSCLC, was effective, safe and showed outcomes equivalent to those in non-elderly patients. Immunotherapy provides a survival benefit for elderly patients, who exhibit its efficacy and a favorable general condition.