OP208 Did Health Technology Assessments Make the Wrong Call? Quantitative Bias Analysis: Alectinib versus Ceritinib in Non-Small Cell Lung Cancer

2021 ◽  
Vol 37 (S1) ◽  
pp. 6-6
Author(s):  
Samantha Wilkinson ◽  
Alind Gupta ◽  
Eric Mackay ◽  
Paul Arora ◽  
Kristian Thorlund ◽  
...  
Keyword(s):  
Real World ◽  
Performance Status ◽  
Missing At Random ◽  
Health Technology ◽  
Bias Analysis ◽  
Ecog Performance Status ◽  
Unmeasured Confounding ◽  
Unmeasured Confounder ◽  
Innovative Therapies ◽  
Quantitative Bias Analysis

IntroductionThe German health technology assessment (HTA) rejected additional benefit of alectinib for second line (2L) ALK+ NSCLC, citing possible biases from missing ECOG performance status data and unmeasured confounding in real-world evidence (RWE) for 2L ceritinib that was submitted as a comparator to the single arm alectinib trial. Alectinib was approved in the US and therefore US post-launch RWE can be used to evaluate this HTA decision.MethodsWe compared the real-world effectiveness of alectinib with ceritinib in 2L post-crizotinib ALK+ NSCLC using the nationwide Flatiron Health electronic health record (EHR)-derived de-identified database. Using quantitative bias analysis (QBA), we estimated the strength of (i) unmeasured confounding and (ii) deviation from missing-at-random (MAR) assumptions needed to nullify any overall survival (OS) benefit.ResultsAlectinib had significantly longer median OS than ceritinib in complete case analysis. The estimated effect size (Hazard Ratio: 0.55) was robust to risk ratios of unmeasured confounder-outcome and confounder-exposure associations of <2.4.Based on tipping point analysis, missing baseline ECOG performance status for ceritinib-treated patients (49% missing) would need to be more than 3.4-times worse than expected under MAR to nullify the OS benefit observed for alectinib.ConclusionsOnly implausible levels of bias reversed our conclusions. These methods could provide a framework to explore uncertainty and aid decision-making for HTAs to enable patient access to innovative therapies.

Initial experience of TAS-102 chemotherapy in Australian patients with chemorefractory metastatic colorectal cancer.

2021 ◽  
Vol 39 (3_suppl) ◽  
pp. 71-71
Author(s):  
Azim Jalali ◽  
Hui-Li Wong ◽  
Rachel Wong ◽  
Margaret Lee ◽  
Lucy Gately ◽  
...  
Keyword(s):  
Colorectal Cancer ◽  
Overall Survival ◽  
Febrile Neutropenia ◽  
Metastatic Colorectal Cancer ◽  
Real World ◽  
Performance Status ◽  
Progression Free Survival ◽  
Median Number ◽  
Ecog Performance Status ◽  
Number Of Patients

71 Background: For patients with refractory metastatic colorectal cancer (mCRC) treatment with Trifluridine/Tipiracil, also known as TAS-102, improves overall survival. In Australia, TAS-102 was initially made available locally through patients self-funding, later via an industry sponsored Medicine Access Program (MAP) and then via the Pharmaceutical Benefits Scheme (PBS). This study aims to investigate the efficacy and safety of TAS-102 in real world Australian population. Methods: A retrospective analysis of prospectively collected data from the Treatment of Recurrent and Advanced Colorectal Cancer (TRACC) registry was undertaken. The characteristics and outcomes of patients receiving TAS-102 were assessed and compared to all TRACC patients and those enrolled in the registration study (RECOURSE). Results: Across 13 sites, 107 patients were treated with TAS-102 (non-PBS n = 27, PBS n = 80), The median number of patients per site was 7 (range: 1-17). The median age was 60 years (range: 31-83), compared to 67 for all TRACC patients and 63 for RECOURSE. Comparing registry TAS-102 and RECOURSE patients, 75% vs 100% were ECOG performance status 0-1, 74% vs 79% had initiated treatment more than 18 months from diagnosis of metastatic disease and 39% vs 49% were RAS wild type. Median time on treatment was 10.4 weeks (range: 1.7-32). Median clinician assessed progression-free survival was 3.3 compared to RECIST defined PFS of 2 months in RECOURSE study, while median overall survival was the same at 7.1 months. Two patients (2.3%) had febrile neutropenia and there were no treatment-related deaths in the real-world series, where TAS102 dose at treatment initiation was at clinician discretion. In the RECOURSE study there was a 4% febrile neutropenia rate and one treatment-related death. Conclusions: TRACC registry patients treated with TAS102 were younger than both TRACC patients overall and those from the RECOURSE trial. Less strict application of RECIST criteria and less frequent imaging may have contributed to an apparently longer PFS. However overall survival outcomes achieved with TAS102 in real world patients were comparable to findings from this pivotal trial with an acceptable rate of major adverse events.

scholarly journals Real-World Experience of Pembrolizumab Monotherapy in Patients with Recurrent or Persistent Cervical Cancer: A Korean Multi-Center Retrospective Study (KGOG1041)

Cancers ◽  
2020 ◽  
Vol 12 (11) ◽  
pp. 3188 ◽  
Author(s):  
Min Chul Choi ◽  
Yong-Man Kim ◽  
Jeong-Won Lee ◽  
Yong Jae Lee ◽  
Dong Hoon Suh ◽  
...  
Keyword(s):  
Cervical Cancer ◽  
Retrospective Study ◽  
Antitumor Activity ◽  
Real World ◽  
Performance Status ◽  
Objective Response ◽  
Eastern Cooperative Oncology Group ◽  
Safety Data ◽  
Ecog Performance Status ◽  
Recurrent Cervical Cancer

This study investigated the antitumor activity and safety of pembrolizumab in patients with recurrent cervical cancer in real-world practice. We conducted a multi-center retrospective study of patients with recurrent or persistent cervical cancer treated with pembrolizumab at sixteen institutions in Korea between January 2016 and March 2020. The primary endpoints were the objective response rate (ORR) and safety. Data were available for 117 patients. The median age was 53 years (range, 28–79). Sixty-four (54.7%) patients had an Eastern Cooperative Oncology Group (ECOG) performance status of ≥2. Forty-nine (41.9%) patients were stage ≥III at diagnosis. Eighty-eight (75.2%) patients had squamous cell carcinoma. The median number of prior chemotherapy lines was two (range, 1–6). During the median follow-up of 4.9 months (range, 0.2–35.3), the ORR was 9.4%, with three complete responses and eight partial responses. The median time to response was 2.8 months (range 1.3–13.1), and the median duration of response (DOR) was not reached. In the population of patients with favorable performance status (ECOG ≤1) (n = 53), the ORR was 18.9%, and the median DOR was 8.9 months (range, 7.3–10.4). Adverse events occurred in 55 (47.0%) patients, including eight (6.8%) patients who experienced grade ≥3 events, and two of them were suspicious treatment-related deaths. Pembrolizumab had modest antitumor activity in patients with recurrent cervical cancer comparable to that found in previously reported clinical trials. However, in patients with favorable performance status, pembrolizumab showed effective antitumor activity. Some safety profiles should be carefully monitored during treatment.

scholarly journals Real-world efficacy and safety of liposomal irinotecan plus fluorouracil/leucovorin in patients with metastatic pancreatic adenocarcinoma: a study by the Korean Cancer Study Group

2019 ◽  
Vol 11 ◽  
pp. 175883591987112 ◽  
Author(s):  
Changhoon Yoo ◽  
Hyeon-Su Im ◽  
Kyu-pyo Kim ◽  
Do-Youn Oh ◽  
Kyung-Hun Lee ◽  
...  
Keyword(s):  
Pancreatic Adenocarcinoma ◽  
Real World ◽  
Performance Status ◽  
Objective Response ◽  
Real Life ◽  
Progression Free Survival ◽  
Real World Data ◽  
Ecog Performance Status ◽  
Metastatic Pancreatic Adenocarcinoma ◽  
Liposomal Irinotecan

Background: Liposomal irinotecan (nal-IRI) plus 5-fluorouracil and leucovorin (5-FU/LV) was effective and well-tolerated in patients with metastatic pancreatic adenocarcinoma (mPAC) that progressed on gemcitabine-based therapy in the global NAPOLI-1 trial. Real-world data may further clarify the outcomes and safety profile of nal-IRI + 5-FU/LV in clinical practice. Methods: This retrospective analysis included patients with mPAC who received nal-IRI + 5-FU/LV following gemcitabine-based therapy under a Managed Access Program in Korea. Results: From January 2017 to April 2018, 86 patients across 10 institutions received nal-IRI + 5-FU/LV (median age, 61 years; 60% male; ECOG performance status, 0–1). A total of 35 (41%) and 51 (59%) patients had received less than two and two or more lines of chemotherapy before inclusion, respectively. At a median follow up of 6.4 months, median overall survival (OS) was 9.4 months (95% confidence interval [CI] 7.4–11.4) and median progression-free survival (PFS) was 3.5 months (95% CI 1.3–5.7). Six-month OS and PFS rates were 65.1% and 37.5%, respectively. Objective response and disease control rates were 10% and 55%, respectively. Most common grade 3–4 toxicities were neutropenia (37.2%), nausea (10.5%), vomiting (9.3%), anorexia (8.1%) and diarrhoea (4.7%). Conclusion: Real-life data for Korean patients indicate that, consistent with NAPOLI-1, nal-IRI + 5-FU/LV is effective and well-tolerated in patients with mPAC that progressed on gemcitabine-based therapy.

Real world experience with exemestane and everolimus for advanced HR-positive breast cancer.

2017 ◽  
Vol 35 (15_suppl) ◽  
pp. e12538-e12538
Author(s):  
Ying Wang ◽  
Dan Le ◽  
Pierre Camateros ◽  
Caroline A. Lohrisch
Keyword(s):  
Breast Cancer ◽  
Real World ◽  
Performance Status ◽  
Real Life ◽  
Prior Treatment ◽  
World Population ◽  
Ecog Performance Status ◽  
Dose Modifications ◽  
Dose Reductions ◽  
Positive Breast Cancer

e12538 Background: BOLERO-2 demonstrated a significant progression free survival (PFS) advantage for EvEx compared to Ex alone in the treatment of metastatic hormone receptor positive breast cancer (HP-MBC) following failure with aromatase inhibitor (AI) in menopausal patients. Benefits and harms reported in clinical trials do not always mirror real life, thus we examined the frequency of dose modifications for toxicity and PFS in a real-world population. Methods: Women with HP-MBC started on EvEx between Dec. 2, 2013 and Nov. 1, 2015 in any one of five cancer centres in British Columbia, Canada were identified via the provincial pharmacy database. Clinical and prior treatment details, adverse events, and outcomes were compared to those of the BOLERO-2 study participants. Results: We identified151 patients treated with EvEx: median age was 53 (range 25 – 85), and 60 (40%) were ≤49. ECOG performance status was 0-1 in 122 (81%) and 2-3 in 29 (19%). 135 (89%) received prior treatment for MBC, which included non-steroidal AI in 117 (87%) and chemotherapy in 74 (55%). 94 (62%) received EvEx as 3rd line treatment and beyond. Median follow-up was 714 days (range 10 – 1127); 66 (44%) were alive at time of analysis. Dose reductions, interruptions and discontinuations of everolimus due to toxicity occurred in 32%, 31%, and 30%, respectively, higher than reported in BOLERO-2. Median PFS was 6.1 months (95% CI: 4.9 – 7.7) in our cohort, similar to that of the investigator assessed EvEx arm of BOLERO-2. Majority, 52%, of dose reductions/interruptions occurred within the first month of treatment initiation. Conclusions: We observed similar PFS forEvEx in a real world population compared with BOLERO-2. Dose interruptions and reductions were frequent, but may have enabled patients to tolerate therapy for longer. Discontinuation rates for toxicity were high and may limit the efficacy of the combination in the real world. Recent findings of primary prophylaxis against mucositis may help enhance continuation and dose maintenance (Beck et al. JCO 2016). Alternately, starting at a lower dose of Ev and increasing to tolerance may be worth exploring, as despite higher rates of dose modifications, our PFS rate was similar to that in BOLERO-2.

An observational study of patients with advanced melanoma receiving pembrolizumab in a real-world U.S. community oncology practice.

2017 ◽  
Vol 35 (15_suppl) ◽  
pp. e21015-e21015
Author(s):  
Charles Lance Cowey ◽  
Frank Xiaoqing Liu ◽  
Jenny Black-Shinn ◽  
Kendall Lee Stevinson ◽  
Marley Boyd ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Brain Metastases ◽  
Real World ◽  
Performance Status ◽  
Progression Free Survival ◽  
Advanced Melanoma ◽  
Ecog Performance Status ◽  
Study Results ◽  
Line Of Therapy ◽  
Oncology Practice

e21015 Background: PD-1 monoclonal antibodies are promising immunotherapies approved for treatment of patients (pts) with advanced melanoma. As the first US FDA approved PD-1 antibody, pembrolizumab (pembro) has demonstrated efficacy and safety in clinical trial settings. However, patterns of real world utilization and pt outcomes associated with pembro are limited. Methods: Adult pts with advanced melanoma who initiated pembro between 9/1/ 2014-3/31/2016 were identified retrospectively fromelectronic health records (EHR) of McKesson Specialty Health and followed through 9/ 30/2016. Pts in clinical trials were excluded. Demographic, disease, treatment characteristics and reasons for treatment discontinuation of pembro were abstracted from structured data elements of the EHR with further supplementation of unstructured data within the patient chart (progress notes, radiology scan reports). Overall survival (OS) and physician-reported progression free survival (PFS) from pembro initiation were analyzed using Kaplan Meier analysis. Results: 182 pts, with a median follow-up of 9.9 mos (range = 0.0-25.0), were included. Median age at pembro initiation was 66.0 yrs; 30.8% had an elevated lactate dehydrogenase (LDH); 23.6% had brain metastases and 65.4% had an ECOG performance status of 0 or 1. The most common reason for pembro discontinuation was progression (45.5%) followed by treatment-related toxicity (24.4%). In the overall population, median PFS from pembro initiation was 4.2 mos (95% CI = 3.2-5.3). Median OS was 19.4 mos (14.0-NR) with 12 and 24-month survival probabilities of 61.4% (95% CI = 53.4-68.5) and 43.9% (95% CI = 31.1-55.9). In multivariable analyses, characteristics predictive of worse survival included receipt of pembro at a later line of therapy (HR = 3.36, p = 0.0013 for 3L+), presence of brain metastases (HR = 2.67, p = 0.0007) and elevated LDH (HR = 4.10, p < 0.0001). Conclusions: The study results are consistent with those from pembro clinical trials (KeyNote001) and are in support of the effectiveness of pembro in real world treatment of advanced melanoma. Presence of brain metastases, elevated LDH, and use of pembro 3L+ were associated with worse survival outcome.

REACHAUT: First-line (1L) ribociclib (RIB) + endocrine therapy (ET) in HR+, HER2- metastatic breast cancer (MBC) in the real-world setting.

2019 ◽  
Vol 37 (15_suppl) ◽  
pp. e12527-e12527
Author(s):  
Christian F. Singer ◽  
Leopold Öhler ◽  
Daniel Egle ◽  
Richard Greil ◽  
Edgar Petru ◽  
...  
Keyword(s):  
Median Duration ◽  
Real World ◽  
Performance Status ◽  
Metastatic Breast ◽  
Real World Data ◽  
Ecog Performance Status ◽  
Minimal Impact ◽  
Real World Setting ◽  
Common Prior ◽  
Metastatic Setting

e12527 Background: RIB + aromatase inhibitor (AI) is approved as 1L treatment (tx) for HR+, HER2− MBC. Real-world data on efficacy and safety of RIB+AI are limited. REACHAUT, a prospective, noninterventional trial assessed the safety of RIB+AI in 1L setting in postmenopausal patients (pts) with HR+, HER2− MBC in a real-world setting. First interim analysis results about safety are presented. Methods: 75 postmenopausal pts with HR+, HER2− MBC, QTc < 450 msec, and no prior ET for advanced disease were enrolled at 13 sites. 1L chemotherapy (CT) was allowed. Results: At data cutoff (25-Jan-2019), 61 pts were evaluable for safety (ongoing, n = 44; discontinued, n = 17). Median duration of follow-up was 29 d. Median age at baseline was 65 y ( < 65 y, n = 28; ≥65 y, n = 33); ECOG performance status was 0 (n = 39) and 1 (n = 12). 42.6% had visceral (lung, liver) metastases (mets), while 34.4% had bone only mets. Most common prior tx included CT (29.5% in neoadjuvant/adjuvant/metastatic setting) and ET (41%). Pts received RIB in 1L (93.4%) and second-line (6.6%) setting. In 80.3% pts receiving RIB, the ET partner included letrozole (57.4%), exemestane (11.5%), anastrozole (9.8%). Median duration of RIB exposure was 100 d. Median time to first AE was 14 d. 83.6% pts experienced AEs with mild (63.9%) to moderate (50.8%) severity. Serious AEs were noted in 6.6% pts. Most common AEs were neutropenia (42.6%) and QTc prolongation (24.6%). 4.9% pts had hepatobiliary AEs. Due to AEs, dose adjustments (4.9%) and dose interruptions (19.8%) were needed. No deaths were reported; median PFS was not reached. Subgroup analysis by age ( < 65 y vs ≥65 y) showed that the incidence of AEs was 55.9% vs 44.1%. Neutropenia was reported in 46.4% pts aged < 65 y vs 39.3% pts aged ≥65 y; QT prolongation events were noted in 21.4% pts aged < 65 y vs 27.2% pts aged ≥65 y. Dose adjustments and dose interruptions were needed in 14.3% and 46.4% pts aged < 65 y vs 15.2% and 45.5% pts aged ≥65 y. Conclusions: Overall safety of RIB+AI in routine clinical practice in REACHAUT was consistent with that reported in the MONALEESA-2 study. In real-world setting, pt age ( < 65 y vs ≥65 y) had minimal impact on AEs. Clinical trial information: NIS006622.

Real-world outcomes in patients (pts) with metastatic urothelial carcinoma (mUC) who received first- or second-line immunotherapies (IO) in the United States (US).

2020 ◽  
Vol 38 (6_suppl) ◽  
pp. 457-457
Author(s):  
Marley Boyd ◽  
Srinivas Annavarapu ◽  
Gurjyot K. Doshi ◽  
Kentaro Imai ◽  
Eric Sbar ◽  
...  
Keyword(s):  
Clinical Outcomes ◽  
Real World ◽  
Performance Status ◽  
The United States ◽  
Future Research ◽  
Second Line ◽  
Ecog Performance Status ◽  
Using Data ◽  
Line Of Therapy

457 Background: Benefit of IO (PD1 and PD-L1 inhibitors) for mUC was observed in clinical trials but real-world evidence for benefit and clinical outcomes is limited. Methods: This was a retrospective study of adult pts with mUC who initiated IO regardless of PD-L1 expression in the first- (1L cohort) or second-line (2L cohort) setting between 5/1/2016-1/31/2019 in the US Oncology Network (USON), a network of community oncology practices. Descriptive and Kaplan-Meier analyses to evaluate baseline characteristics, treatment patterns and clinical outcomes were conducted using data from USON’s electronic heath record. Results: Among 393 pts in the 1L cohort, median (range) age at IO initiation was 77 (42, 90+), 74% were male, 69% were White, and 19.1% and 4.1% had ECOG performance status (PS) 2 and 3/4, respectively. Among the 366 pts in the 2L cohort, median (range) age at IO initiation was 70 (29, 90+), 74% were male, 71% were White, and 19.7% and 1.4% had ECOG PS 2 and 3, respectively. Median (range) follow-up durations from IO initiation were 4.2 (0, 34.1; 1L cohort) and 4.1 (0, 31.3; 2L cohort) months (mo), during which time 43.1% (1L cohort) and 44.4% (2L cohort) of pts died. Median overall survival (OS) from IO initiation (95% confidence interval [CI]) was 10.6 (9.7, 13.2) mo for the 1L cohort and 9.4 (7.1, 11.5) mo for the 2L cohort; 1-year survival probabilities (95% CI) were 46.6% (40.1%, 52.8%; 1L cohort) and 43.4% (36.8%, 49.8%; 2L cohort). By the end of the follow-up, 48.1% of 1L pts and 47.8% of 2L pts were alive and did not advance to next line of therapy, and 13.5% of 1L and 13.4% of 2L cohort pts advanced to the next line of therapy. Median (95% CI) treatment durations were 2.6 (2.1, 2.9) and 2.8 (2.2, 3.5) mo for the 1L and 2L cohorts, respectively; 6-mo ongoing treatment probabilities (95% CI) were 26.6% (22.2%, 31.2%; 1L cohort) and 31.4% (26.4%, 36.4%; 2L cohort). Conclusions: OS of pts in the real world receiving 1L and 2L IO appears consistent with clinical trial results, although survival follow-up is limited. A minority of pts received post-IO therapy. Future research should examine influence of pt characteristics and PD-L1 expression on treatment choice and outcomes.

P14.76 Bevacizumab (BEV) alone or in combination with chemotherapy in recurrent Glioblastoma Multiforme (GBM): A real world experience

2021 ◽  
Vol 23 (Supplement_2) ◽  
pp. ii52-ii52
Author(s):  
H I Chalchal ◽  
T Zhu ◽  
C Woitas ◽  
S Ahmed ◽  
O Souied ◽  
...  
Keyword(s):  
Overall Survival ◽  
Glioblastoma Multiforme ◽  
Real World ◽  
Performance Status ◽  
P Value ◽  
Ecog Performance Status ◽  
Number Of Patients ◽  
Significant Difference ◽  
Historic Control ◽  
Recurrent Gbm

Abstract BACKGROUND Patients with glioblastoma multiforme (GBM) have a median survival of about 14 months. In recurrent GBM no active intervention has shown improvement in survival. Clinical trials has shown that bevacizumab (BEV) alone or in combination with chemotherapy is associated with better progression free survival (PFS). The current study aims to assess efficacy of BEV in real-world setting. MATERIAL AND METHODS Population-based retrospective cohort study patients with recurrent GBM diagnosed in the province of Saskatchewan during 2008–2018 and received BEV alone or in combination with chemotherapy were evaluated. Survival was compared with historic control. RESULTS 43 eligible patients with GBM treated with BEV with or without chemotherapy. 25 patients were treated with Bev alone and 18 patients treated with chemotherapy+ BEV. Median age of the patients were noted to be 53 years. 28 male, and 15 female. 80% of patients treated with single agent BEV had a performance status of either 2 or 3 compared to 33% of patient treated with BEV+ chemotherapy. Median PFS was 4.6 months with 95% CI 2.9–6.9. Median Overall survival (OS) from the time of diagnosis was 17.5 month. Median OS from the time of start of BEV was 5.4 months with 95% CI 3.4–6.8. Partial response (PR) was noted in 3 patients (7%) with stable disease (SD) in 6 patients (14%). 33 (77%) had progressive disease (PD). We were unable to confirm response status in one patient (2%). No statistically significant difference in response rate for patients treated with BEV and BEV+ Chemotherapy. From the start of Bev to the best response, 11 patients (30.56%) noted decrease in the dose of steroids, 14 patients (38.89%) dose remained unchanged. 7 patients (19.44%) required increase in the dose of steroids. 4 patients (11.11%) were not on steroids. For 7 patients we did not have the information on use of steroids. PFS was better for patients treated with chemotherapy + BEV with median PFS of 6.9 months, 95% CI 3.2- 22.3 verses BEV alone with median PFS 3.53 months 95% CI 1.4–5.3, P-value 0.0449. The Cox regression model for PFS to test comparing Bev with chemotherapy vs. Bev alone with the co-variables of sex, age, and ECOG performance status (PS). The model showed that patient with higher ECOG PS were noted to have inferior PFS with a Hazard ratio of 1.92 95% CI 1.09–3.37. P value of 0.2. Patient treated with BEV+ chemo had better PFS with a HR of 6.44 95% CI 1.86–22.28. P value of 0.003. CONCLUSION Retrospective real world study confirms that, patients with recurrent GBM, treatment with BEV is associated with similar PFS as reported in literature. Our study showed similar overall survival from the diagnosis compared to historic control. However the Median OS from Start of BEV was noted to be inferior to what is reported in EORTC EH1.3. Better ECOG performance status is associated with better PFS. Higher number of patients with ECOG 2 and 3 received BEV alone.

scholarly journals Real-World Data for Lenvatinib in Hepatocellular Carcinoma (ELEVATOR): A retrospective multicenter study

Liver Cancer ◽  
2022 ◽  
Author(s):  
Sabrina Welland ◽  
Catherine Leyh ◽  
Fabian Finkelmeier ◽  
André Jefremow ◽  
Kateryna Shmanko ◽  
...  
Keyword(s):  
Overall Survival ◽  
Real World ◽  
Median Overall Survival ◽  
Performance Status ◽  
Progression Free Survival ◽  
Inclusion Criteria ◽  
First Line ◽  
Ecog Performance Status ◽  
Free Survival ◽  
First Line Treatment

Background Lenvatinib is approved as first-line treatment for patients with advanced hepatocellular carcinoma (HCC). The efficacy of lenvatinib in Caucasian real-world patients is insufficiently defined. The purpose of this study was to evaluate the efficacy of lenvatinib in a multi-center cohort (ELEVATOR) from Germany and Austria. Methods A retrospective data analysis of 205 patients treated with first-line systemic lenvatinib at 14 different sites was conducted. Overall survival, progression free survival, overall response rate and adverse event rates were assessed and analyzed. Results Patients receiving lenvatinib in the real-world setting reached a median overall survival of 12.8 months, which was comparable to the results reported from the REFLECT study. Median overall survival (mOS) and progression free survival (mPFS) was superior in those patients who met the inclusion criteria of the REFLECT study compared to patients who failed to meet the inclusion criteria (mOS 15.6 vs 10.2 months, HR 0.55, 95% CI 0.38-0.81, p=0.002; mPFS 8.1 vs 4.8 months HR 0.65, 95% CI 0.46-0.91, p=0.0015). For patients with an impaired liver function according to the Albumin-Bilirubin (ALBI) grade, or reduced ECOG performance status ≥2, survival was significantly shorter compared to patients with sustained liver function (ALBI grade 1) and good performance status (ECOG performance status 0), respectively (HR 1.69, 95% CI 1.07-2.66, p=0.023; HR 2.25, 95% CI 1.19-4.23, p=0.012). Additionally, macrovascular invasion (HR 1.55, 95% CI 1.02-2.37, p=0.041) and an AFP ≥200 ng/mL (HR 1.56, 95% CI 1.03-2.34, p=0.034) were confirmed as independent negative prognostic factors in our cohort of patients with advanced HCC. Conclusion Overall, our data confirm the efficacy of lenvatinib as first-line treatment and did not reveal new or unexpected side effects in a large retrospective Caucasian real-world cohort, supporting the use of lenvatinib as meaningful alternative for patients that cannot be treated with IO-based combinations in first-line HCC.

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