An economic analysis of axitinib and sorafenib for second-line treatment of cytokine-refractory patients with advanced renal cell carcinoma in the United States (US).

2013 ◽  
Vol 31 (15_suppl) ◽  
pp. e15601-e15601
Author(s):  
Ipek Özer-Stillman ◽  
Apoorva Ambavane ◽  
Paul Cislo
Keyword(s):  
Economic Analysis ◽  
The United States ◽  
Phase Iii ◽  
Administrative Claims ◽  
Line Treatment ◽  
Second Line ◽  
Cost Difference ◽  
Life Years ◽  
The Us ◽  
The Cost

e15601 Background: Cytokines are a first-line treatment option for a subset of advanced RCC patients in the US. After progression on cytokines, NCCN guidelines recommend targeted agents, such as axitinib and sorafenib. Subgroup analysis of post-cytokine patients in the phase III AXIS trial found that axitinib increased median progression free survival (PFS) compared with sorafenib (12.0 vs. 6.6 months, p<0.0001), while overall survival (OS) showed no difference (29.4 vs. 27.8 months, p=0.144). An economic analysis for this subgroup was conducted from a US healthcare payer perspective. Methods: A cohort partition model with monthly cycles was constructed to estimate direct medical costs and health outcomes, discounted at 3.0% per annum, over cohort lifetime. Patients were apportioned into 3 health states (progression-free, progressed and dead) based on OS and PFS Kaplan-Meier curves for the post-cytokine subgroup in the AXIS trial. Active treatment was applied until progression, followed by best supportive care (BSC) alone thereafter. The wholesale acquisition costs were based from RedBook. Adverse event (AE) management costs were obtained from published studies. AE rates and utility values were informed by the AXIS trial. Administrative claims data from MarketScan Database were analyzed to estimate costs for BSC and routine care of second-line advanced RCC patients. Results: The total per-patient lifetime costs were estimated to be $242,750 for axitinib and $168,880 for sorafenib and most of the cost difference (84%) was due to the higher total medication cost of axitinib. The cost difference was sensitive to dose intensity and length of treatment. The difference in quality-adjusted life-years (QALY) for axitinib versus sorafenib was minor (1.3 versus 1.2) and the incremental cost-effectiveness ratio (ICER) for axitinib compared with sorafenib was $683,209/QALY. Conclusions: For cytokine-refractory advanced RCC patients, axitinib resulted in an ICER > $650,000/QALY versus sorafenib due to high drug costs and lack of OS benefit, indicating that axitinib may not present good value for money as 2nd line treatment when compared to sorafenib in the US.

The Cost-Effectiveness of Bortezomib for the Initial Treatment of Multiple Myeloma in the United States.

Blood ◽  
2009 ◽  
Vol 114 (22) ◽  
pp. 1379-1379
Author(s):  
Si-Tien Wang ◽  
Hui Huang ◽  
Hongliang Shi ◽  
Mei Sheng Duh ◽  
Kristina Chen ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Health Outcomes ◽  
Incremental Cost ◽  
Research Funding ◽  
The United States ◽  
Line Treatment ◽  
Second Line ◽  
Employment Equity ◽  
Equity Ownership ◽  
The Cost

Abstract Abstract 1379 Poster Board I-401 Background: Data from the phase III VISTA trial demonstrated superiority in terms of clinical effectiveness of bortezomib (Velcadë) plus melphalan and prednisone (VMP) relative to melphalan and prednisone alone (MP) in the initial treatment of patients with multiple myeloma (MM). The aim of this study was to utilize data from the VISTA study and published literature to compare lifetime health outcomes and the cost-effectiveness of these regimens as induction therapy for MM patients ineligible for autologous stem cell transplantation (ASCT). An indirect comparison of VMP versus thalidomide plus MP (MPT) was also conducted using published results from the IFM 99-06 clinical trial for MPT (Facon et al, Lancet 2007). The goal of this study and the derived model was to assess the relative costs and outcomes from these two trials, recognizing the limitations imposed by using data derived from independent studies. Methods: A Markov model from the US payer's perspective was developed. Simulations were performed for hypothetical cohorts of newly diagnosed MM patients with an average age of 70 years at treatment initiation and who were not eligible for ASCT. The model includes seven health states representing periods of treatment response (stable disease/minimal response, partial response, or complete response), treatment-free interval, progressive disease, second-line treatment and death. Monthly transition probabilities were estimated from patient-level VISTA trial data for VMP and MP (with a data cut-off of June 15, 2007), and from the published phase lll IFM 99-06 trial for MPT. Costs included per-protocol drug and medical costs, treatment-related adverse events, second-line treatment, and resource utilization during treatment-free interval and progressive disease. Unit costs of medications and resources were obtained from published literature. All costs were adjusted to 2009 US dollars. State-specific utility estimates were derived from patient-level EQ-5D data from the VISTA trial using US-specific weights. Health outcomes were expressed in life-years (LYs) and quality-adjusted life-years (QALYs). Both cost and health outcomes were discounted at 3%. Incremental cost-effectiveness ratios (ICERs) were calculated for VMP versus MP, and VMP versus MPT, over a lifetime horizon (approximated by 20 years). One-way sensitivity analyses were conducted by running the model with upper and lower values of key parameters to assess the general robustness of model findings and identify key drivers. Results: Model base case results for the incremental cost-effectiveness of VMP relative to MP and MPT are shown in the Table. Comparison of the model's overall survival (OS) projections with the observed differences indicates a conservative approximation of the treatment differences for VMP. The estimated OS was 4.187 years with VMP versus 2.864 years with MP and versus 4.140 years with MPT over a lifetime horizon. Lifetime direct medical costs range from $57,864 for MP to $129,902 for MPT. The cost per LY and QALY gained with VMP compared with MP is $40,051 and $56,109, respectively. VMP is dominant (cost saving and better outcomes) compared with MPT, costing 17.7% less and providing slightly more QALYs on average. One-way sensitivity analyses suggest general robustness of model findings and the key drivers include VMP/MP hazard ratio from second-line treatment to death, and the MPT/MP hazard ratio for treatment discontinuation. Conclusions: In newly diagnosed MM patients ineligible for ASCT, VMP is projected to improve long-term health outcomes, offering a substantial survival benefit compared with MP. The incremental cost-effectiveness of VMP versus MP is within the generally accepted cost-effectiveness range of $50,000 to $100,000 per QALY, suggesting that VMP is cost-effective compared with MP in the United States. Within this cost-effectiveness model, compared with MPT, VMP is dominant, yielding lower costs and better health outcomes. Disclosures: Wang: Milllennium: Research Funding. Huang: Milllennium: Employment, Equity Ownership. Shi: Millennium Pharmaceuticals, Inc.: Employment. Duh: Milllennium: Consultancy, Research Funding. Chen: Milllennium: Research Funding. Chang: Milllennium: Research Funding. Korves: Milllennium: Research Funding. Dhawan: Johnson and Johnson Research Pharmaceuticals: Employment. Cakana: Johnson & Johnson: Employment, Equity Ownership. van de Velde: Johnson & Johnson: Employment, Equity Ownership. Esseltine: Milllennium: Employment, Equity Ownership. Garrison: Milllennium: Consultancy.

scholarly journals Real-World Evidence on Treatment Utilization in Lower-Risk Myelodysplastic Syndromes: Findings from a Medical Record Review in the United States

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 4666-4666
Author(s):  
Ulrich Germing ◽  
Ravi K. Goyal ◽  
Aylin Yucel ◽  
Rohan C. Parikh ◽  
Maria Jimenez ◽  
...  
Keyword(s):  
Board Of Directors ◽  
Lower Risk ◽  
Research Funding ◽  
The United States ◽  
Full Time ◽  
Line Treatment ◽  
Second Line ◽  
First Line ◽  
Advisory Committees ◽  
The Us

Abstract Introduction: Patients diagnosed with myelodysplastic syndromes (MDS) are at an increased risk for developing infections, bleeding events, cardiopulmonary complications, and progressing to acute myeloid leukemia (AML). Patients with lower-risk MDS experience a mean life-year loss of about 6 years, and the main focus of treatment is on management of cytopenias, in particular anemia. Currently, limited real-world evidence exists on prevailing treatment patterns and outcomes in lower-risk MDS in the United States (US). Methods: In this retrospective, non-interventional review of medical records, eligible patients (≥ 18 years of age) with diagnosis of lower-risk MDS (with or without ring sideroblasts and with single-lineage or multilineage dysplasia) between July 1, 2013 and September 30, 2018, were identified by participating clinicians. Patients with prior history of AML or other malignant neoplasms were excluded. This is an ongoing study in the US and interim data are described. Study measures descriptively summarized patient demographics, clinical characteristics at MDS diagnosis, and utilization of medication treatments for the management of anemia. Results: Data from medical records of 50 patients with lower-risk MDS were abstracted by 26 hematologist-oncologists in the US. Participating clinicians had been managing treatment for hematology/oncology patients for an average (standard deviation [SD]) of 16.3 (6.7) years, 53.9% practiced in an academic hospital or cancer center, and 46.2% practiced in a community setting. The mean (SD) age for patients with lower-risk MDS at diagnosis was 64.2 (9.9) years, 64.0% were male, 70.0% were White, and 60% were insured primarily through Medicare. Most patients (54.0%) had Low-risk MDS (as categorized by Revised International Prognostic Scoring System) followed by Very low-risk (28.0%), and Intermediate-risk (18.0%) at initial diagnosis. The most common gene mutations observed were JAK2 (22.0%) and ASXL1 (8.0%). The most common karyotype abnormalities observed were del(5q) (28.0%), −Y (14.0%), and del(7q) (12.0%). During the 12 months before diagnosis of lower-risk MDS, cardiac complications were observed among 18.0% of patients, and 58.0% of patients were either current or former smokers at the time of diagnosis of lower-risk MDS. At the time of data abstraction, mean (SD) follow-up time was 39.6 (21.6) months, and 46 (92.0%) patients had received ≥ 1 line of treatment for MDS-associated anemia. Of these 46 patients, 93.5% received first-line treatment with an erythropoietin-stimulating agent (ESA). In the first-line, most patients received ESA monotherapy (n = 37; 80.4%) followed by ESA-based combination therapy (n = 6; 13.0%), lenalidomide only (n = 2; 4.4%), and azacitidine (n = 1; 2.2%). All 18 patients who received a second-line treatment had received an ESA-based first-line treatment. Second-line treatments received were ESA-based treatment (n = 5; 27.8%), lenalidomide only (n = 5; 27.8%), luspatercept only (n = 4; 22.2%), azacitidine only (n = 3; 16.7%), and filgrastim only (n = 1; 5.6%). Third-line treatment was only observed in 3 patients. Conclusions: In this ongoing study, current analysis for 50 patients with lower-risk MDS in the US showed ESA-based regimens were the most common first-line therapy. ESA-based treatment was again utilized as second-line therapy among some patients who were previously treated with ESA. Disclosures Germing: Janssen: Honoraria; Celgene: Honoraria; Novartis: Honoraria, Research Funding; Jazz Pharmaceuticals: Honoraria; Bristol-Myers Squibb: Honoraria, Other: advisory activity, Research Funding. Goyal: RTI Health Solutions: Other: Full-time employee of RTI Health Solutions, which is business unit of RTI International, a non-profit research organization, which received funding from BMS to conduct this study.. Yucel: BMS: Current Employment, Current holder of individual stocks in a privately-held company. Parikh: RTI-Health Solutions: Other: Full-time employee of RTI-HS, which received funding to conduct this study from BMS.. Jimenez: RTI Health Solutions: Current Employment. Sluga-O'Callaghan: RTI Health Solutions: Current Employment. Tang: Bristol-Myers Squibb: Current Employment, Current equity holder in publicly-traded company. Hughes: BMS: Current Employment. Diez-Campelo: Takeda Oncology: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau; BMS: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau; Novartis: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau.

HALT MBC: HER2 suppression with the addition of lapatinib to trastuzumab in HER2-positive metastatic breast cancer (LPT112515).

2012 ◽  
Vol 30 (15_suppl) ◽  
pp. TPS658-TPS658
Author(s):  
Nancy U. Lin ◽  
Michael A. Danso ◽  
Alice K. David ◽  
Joseph J. Muscato ◽  
Daniel Rayson ◽  
...  
Keyword(s):  
Breast Cancer ◽  
Objective Response ◽  
Pathologic Complete Response ◽  
The United States ◽  
Phase Iii ◽  
Type I ◽  
Preoperative Treatment ◽  
Line Treatment ◽  
Second Line ◽  
Her2 Positive

TPS658 Background: Dual blockade of HER2 with the combination of trastuzumab (T) and lapatinib (L) enhances antitumor activity in HER2-positive breast cancer (BC) preclinical models due to the complementary mechanisms of action of the 2 agents. In patients (pts) with T-treated HER2-positive metastatic BC (MBC), treatment with the combination was associated with longer progression-free (PFS) and overall survival (OS) compared with L alone. In pts with stage II/III BC, preoperative treatment with the combination plus paclitaxel (P) resulted in significantly higher pathologic complete response rates compared with P combined with either agent alone. This evidence supports the concept of dual HER2 blockade as a treatment strategy for HER2-positive MBC. The present study is designed to evaluate whether the addition of L improves PFS among women with HER2-positive MBC receiving T as maintenance therapy. Methods: In this open-label, Phase III study, 280 pts will be stratified by line of treatment (first/second) and hormone receptor status (positive/negative), then randomized 1:1 to receive maintenance treatment with either L (1000 mg qd, continuously) in combination with T (6 mg/kg once every 3 weeks [q3w]) or T (6 mg/kg q3w) alone. Pts will receive study treatment until disease progression, death, discontinuation due to adverse events, or other reasons. The primary endpoint is PFS; secondary endpoints are OS, clinical benefit rate, and safety. Key eligibility criteria include pts with HER2-positive MBC who have completed 12 to 24 weeks of first- or second-line treatment with T plus chemotherapy with an objective response or stable disease at time of chemotherapy discontinuation. Pts with stable brain metastases are eligible if entering the study on second-line treatment. Efficacy endpoints will be analyzed in the ITT population. A total of 193 PFS events is required to detect a 50% increase in median PFS (hazard ratio=0.67) for L plus T compared with T alone (median PFS 27 vs 18 weeks, respectively). The hypothesis will be tested using a 1-sided test with 80% power and a type I error of 0.025. The trial is currently open for accrual in the United States and Canada.

Human epidermal growth factor receptor 2 (HER2) suppression with the addition of lapatinib to trastuzumab in HER2-positive metastatic breast cancer (HALT: LPT112515).

2013 ◽  
Vol 31 (15_suppl) ◽  
pp. TPS664-TPS664
Author(s):  
Nancy U. Lin ◽  
Michael A. Danso ◽  
Alice K. David ◽  
Joseph J. Muscato ◽  
Daniel Rayson ◽  
...  
Keyword(s):  
Breast Cancer ◽  
Objective Response ◽  
The United States ◽  
Phase Iii ◽  
Tolerability Profile ◽  
Type I ◽  
Preoperative Treatment ◽  
Line Treatment ◽  
Second Line ◽  
Her2 Breast Cancer

TPS664 Background: Evidence supports the concept of dual HER2 blockade as a treatment strategy for HER2+ breast cancer (BC). In patients with prior trastuzumab (T)-treated HER2+ metastatic BC (MBC), treatment with T plus lapatinib (L) was associated with longer progression-free survival (PFS) and overall survival (OS) compared with L alone, and had an acceptable safety and tolerability profile. In patients with stage II/III BC, preoperative treatment with T plus L plus paclitaxel (P) resulted in significantly higher pathologic complete response rates compared with P combined with either agent alone. This study is designed to evaluate whether the addition of L improves PFS among women with HER2+ MBC receiving T as maintenance therapy. Methods: In this open-label, Phase III study, 280 patients will be stratified by line of treatment (first/second) and hormone receptor status (positive/negative), then randomized 1:1 to receive maintenance treatment with either L (1000 mg qd, continuously) in combination with T (6 mg/kg once every 3 weeks [q3w]), or T (6 mg/kg q3w) alone until disease progression, death, discontinuation due to adverse events, or other reasons. The primary endpoint is PFS; secondary endpoints are OS, clinical benefit rate, and safety. Eligible patients are females, aged ≥18 years with HER2+ MBC who have completed 12-24 weeks of first-/second-line treatment with T plus chemotherapy with an objective response or stable disease at chemotherapy discontinuation. Patients with stable brain metastases are eligible if entering the study on second-line treatment. Efficacy endpoints will be analyzed in the intent-to-treat population. A total of 193 PFS events is required to detect a 50% increase in median PFS from 18 weeks (T alone) to 27 weeks (L+T) with an associated hazard ratio of 0.667, an 80% power and a 1-sided type I error of 0.025. One interim analysis is planned for futility when ~97 PFS events (50% of required events) have been observed. Safety endpoints will be analyzed in all randomized patients who receive ≥1 dose of study medication. The trial is currently open for accrual in the United States and Canada. Clinical trial information: NCT00968968.

Economic analysis of ceritinib used as second-line versus third-line treatment for patients with ALK-rearranged non-small-cell lung cancer.

2019 ◽  
Vol 37 (15_suppl) ◽  
pp. e18355-e18355
Author(s):  
Bin Wu ◽  
Hongchao Li
Keyword(s):  
Lung Cancer ◽  
Small Cell ◽  
Line Treatment ◽  
Second Line ◽  
Small Cell Lung ◽  
Life Years ◽  
Third Line ◽  
Line Setting ◽  
The Cost ◽  
Third Line Treatment

e18355 Background: Targeted therapy with ceritinib notably improves survival in patients with advanced non–small-cell lung cancer (NSCLC) harboring anaplastic lymphoma kinase (ALK) rearrangement tumors after the failure of chemotherapy and crizotinib. However, the economic outcome of ceritinib used as the second-line versus third-line treatment is still unclear. Methods: A mathematical model was constructed to project 21-day patient transitions. The clinical data were primarily extracted from the ASCEND-5 trial. The health resource consumption data were estimated from the perspective of the Chinese health care system. The cost, quality-adjusted life-years (QALYs) and incremental cost-effectiveness ratio (ICER) was measured in a 10-year time horizon. Sensitivity analyses were conducted to test the impact of uncertainties. Results: Comparing with the 3rd-line strategy, ceritinib 2nd-line strategy achieved the comparable life years and additional 0.06 QALYs with the less cost of $6,694 (Table), which suggested that ceritinib used as 2nd-line treatment is a dominant strategy. The utility of disease-free survival and the cost of ceritinib were the factors that had the considerable impact on the model outcomes. Conclusions: These results indicate that ceritinib used in 2nd-line setting is a more favorable treatment option than in the 3rd-line setting. [Table: see text]

scholarly journals Cost-Effectiveness Analysis of Camrelizumab Versus Chemotherapy as Second-Line Treatment of Advanced or Metastatic Esophageal Squamous Cell Carcinoma

2021 ◽  
Vol 12 ◽  
Author(s):  
Hongfu Cai ◽  
Baohua Xu ◽  
Na Li ◽  
Bin Zheng ◽  
Zhiwei Zheng ◽  
...  
Keyword(s):  
Squamous Cell Carcinoma ◽  
Cost Effectiveness ◽  
Esophageal Squamous Cell Carcinoma ◽  
Incremental Cost ◽  
Cell Carcinoma ◽  
Squamous Cell ◽  
Line Treatment ◽  
Second Line ◽  
Life Years ◽  
The Cost

Background: This study aimed to analyze the cost effectiveness of camrelizumab in the second-line treatment of advanced or metastatic esophageal squamous cell carcinoma in China.Methods: On the basis of the ESCORT clinical trial, a partitioned survival model was constructed to simulate the patient’s lifetime quality-adjusted life years (QALYs), lifetime costs, and incremental cost-effectiveness ratio (ICER). One-way sensitivity and probability sensitivity analyses were performed to test the stability of the model.Results: Treatment of esophageal squamous cell carcinoma with camrelizumab added 0.36 QALYs and resulted in an incremental cost of $1,439.64 compared with chemotherapy, which had an ICER of $3,999 per QALY gained. The ICER was far lower than the threshold of willingness to pay for one time the GDP per capita in China. Sensitivity analysis revealed that the ICERs were most sensitive to the cost of drugs, but the parameters did not have a major effect on the results of the model.Conclusion: Camrelizumab is likely to be a cost-effective option compared with chemotherapy for patients with advanced or metastatic esophageal squamous cell carcinoma. This informs patient selection and clinical path development.

Cost–effectiveness of second-line nivolumab for platinum-treated advanced non-small-cell lung cancer

2020 ◽  
Vol 9 (18) ◽  
pp. 1301-1309
Author(s):  
Longfeng Zhang ◽  
Xiaofang Zeng ◽  
Hongfu Cai ◽  
Na Li ◽  
Maobai Liu ◽  
...  
Keyword(s):  
Lung Cancer ◽  
Cost Effectiveness ◽  
Small Cell Lung Cancer ◽  
Cell Lung Cancer ◽  
Small Cell ◽  
Phase Iii ◽  
Second Line ◽  
Small Cell Lung ◽  
The Cost ◽  
Quality Adjusted Life

Aim: To analyze the economic impact of nivolumab and chemotherapy in patients with non-small-cell lung cancer (NSCLC) who developed disease progression after platinum-containing dual-drug chemotherapy. Materials & methods: The partitioned survival model was used to analyze the cost-utility of two NSCLC treatments by nivolumab and docetaxel. The clinical data resulted from the Phase III clinical trial. The cost parameters were derived from our previous studies, and the utility parameters were derived from the literature. Results: The quality-adjusted life-years of nivolumab and docetaxel were 0.778 and 0.336. The lifetime direct medical expenses of nivolumab and docetaxel were US$44,707.17 and US$12,826.72. The incremental cost–effectiveness ratio was $72,127.71/quality-adjusted life-year. Conclusion: The combination of chemotherapy, nivolumab is not a cost-effective choice in the second-line treatment of NSCLC.

scholarly journals Cost-Effectiveness In The Second-Line Treatment Of Non-Small Cell Lung Cancer (Nsclc) In The Us

2015 ◽  
Vol 18 (7) ◽  
pp. A457-A458 ◽  
Author(s):  
C Graham ◽  
H Knox ◽  
LM Hess ◽  
M Jen ◽  
G Cuyun Carter ◽  
...  
Keyword(s):  
Lung Cancer ◽  
Cost Effectiveness ◽  
Small Cell Lung Cancer ◽  
Cell Lung Cancer ◽  
Small Cell ◽  
Line Treatment ◽  
Second Line ◽  
Small Cell Lung ◽  
The Us

A phase II trial combining tumor-targeting TP53 gene therapy with gemcitabine/nab-paclitaxel as a second-line treatment for metastatic pancreatic cancer.

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. 4139-4139
Author(s):  
Chris Poki Leung ◽  
Minal A. Barve ◽  
Ming-Shiang Wu ◽  
Kathleen F. Pirollo ◽  
James F. Strauss ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Phase Ii ◽  
Phase Ii Trial ◽  
Objective Response ◽  
Tp53 Gene ◽  
Phase Iii ◽  
Published Data ◽  
Line Treatment ◽  
Second Line ◽  
First Line

4139 Background: Nearly all stage IV pancreatic adenocarcinoma (PAC) patients progress after first-line treatment, and second-line options are limited. SGT-53 is an investigational product for tumor-targeted TP53 gene therapy that has completed phase Ia/Ib trials [Senser et al (2013), Mol Ther 21:1096; Pirollo et al (2016) Mol Ther 24:1697]. Methods: Here we provide an interim analysis of a Phase II trial (SGT53-02-1; NCT02340117) combining SGT-53 with gemcitabine/nab-paclitaxel (GEM/ABX). Eligible were first-line patients or those who had progressed after FOLFIRINOX (FFX) and/or gemcitabine-based therapy (second-line). In a 7-week treatment cycle, SGT-53 (3.6 mg DNA) was given once or twice weekly with GEM/ABX (1000 mg/m2/wk and 125 mg/m2/wk, respectively, for 3 of 4 weeks). Progression-free survival (PFS) and objective response rate (ORR) are primary endpoints.Overall survival (OS) and PFS are estimated by Kaplan-Meier analysis. Results: Of all evaluable patients (n=20), best response in 7 patients was determined to be partial response (PR) and 13 had stable disease (SD); none had progressive disease. In the second-line patients (n=11) there were 5 PR and 6 SD after 9 had failed FFX treatment, 3 had failed gemcitabine-based treatment and 1 had failed both. For patients with elevated CA19-9, SGT-53 + GEM/ABX resulted in marked reductions in the tumor marker. Published data for patients with PAC after therapy failure [Mita et al (2019) J Clin Med 8: 761; Portal et al (2015) Br J Cancer 113:989; Wang-Gillam et al (2016) Lancet 387:545] are shown for comparison. Notably, mPFS in our second-line patients was 7.4 months versus 3.1 months for the approved second-line therapy [Wang-Gillam et al (2016)]. This improvement in PFS exceeds the benchmark proposed to predict a clinically meaningful Phase III trial [Rahib et al (2016) Lancet Oncol 2:1209]. Conclusions: Our data suggest a clinically meaningful benefit of adding SGT-53 to GEM/ABX particularly for second-line PAC patients, most of whom had failed prior FFX treatment. Clinical trial information: NCT02340117. [Table: see text]

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