scholarly journals PS1456 FAVORABLE OVERALL SURVIVAL OF IMETELSTAT-TREATED RELAPSED/REFRACTORY MYELOFIBROSIS PATIENTS COMPARED WITH CLOSELY MATCHED REAL WORLD DATA

HemaSphere ◽  
2019 ◽  
Vol 3 (S1) ◽  
pp. 669-670 ◽  
Author(s):  
A. Kuykendall ◽  
Y. Wan ◽  
J. Mascarenhas ◽  
J.-J. Kiladjian ◽  
A. Vannucchi ◽  
...  
Keyword(s):  
Overall Survival ◽  
Real World ◽  
Real World Data ◽  
World Data

Socioeconomic Status Is an Independent Prognostic Factor for Overall Survival in Patients With Multiple Myeloma: Real-World Data From a Cohort of 223 Patients

2020 ◽  
Vol 20 (10) ◽  
pp. 704-711
Author(s):  
Stergios Intzes ◽  
Marianthi Symeonidou ◽  
Konstantinos Zagoridis ◽  
Zoe Bezirgiannidou ◽  
Aikaterini Pentidou ◽  
...  
Keyword(s):  
Multiple Myeloma ◽  
Socioeconomic Status ◽  
Overall Survival ◽  
Prognostic Factor ◽  
Real World ◽  
Independent Prognostic Factor ◽  
Real World Data ◽  
World Data

The rate of tumor growth, g, as a biomarker for overall survival (OS) in prostate cancer (PC) in clinical trials as well as in real-world data from the Veterans Administration Medical Centers (VAMCs).

2019 ◽  
Vol 37 (15_suppl) ◽  
pp. 5074-5074
Author(s):  
Harshraj Leuva ◽  
Mengxi Zhou ◽  
Julia Wilkerson ◽  
Keith Sigel ◽  
Ta-Chueh Hsu ◽  
...  
Keyword(s):  
Prostate Cancer ◽  
Clinical Trial ◽  
Overall Survival ◽  
Clinical Trials ◽  
Tumor Growth ◽  
Real World ◽  
Clinical Trial Data ◽  
Trial Data ◽  
Real World Data ◽  
World Data

5074 Background: Novel assessments of efficacy are needed to improve determination of treatment outcomes in clinical trials and in real-world settings. Methods: Cancer treatments usually lead to concurrent regression and growth of the drug-sensitive and drug-resistant fractions of a tumor, respectively. We have exploited novel methods of analysis that assess these two simultaneous processes and have estimated rates of tumor growth ( g) and regression ( d) in over 30,000 patients (pts) with diverse tumors. Results: In prostate cancer (PC) we have analyzed both clinical trial and real-world data from Veterans. Using clinical trial data from 6819 pts enrolled in 15 treatment arms we have established separately and by combining all the data that g correlates highly (p<0.0001) with overall survival (OS) – slower g associated with better OS. In PC, abiraterone (ABI) and docetaxel (DOC) are superior to placebo, prednisone and mitoxantrone. ABI (median g =0.0017) is superior to DOC ( g=0.0021) in first line (p=0.0013); and ABI in 2nd line ( g=0.0034) is inferior to ABI in 1st line ( g=0.0017; p<0.0001). Finally, using combined clinical trial data as a benchmark we could assess the efficacy of novel therapies in as few as 30-40 patients. Amongst 7457 Veterans, the median g on a taxane ( g=0.0022) was similar to that from clinical trials ( g=0.0012). Although only 258 Veterans received cabazitaxel (CAB), g values for CAB ( g=0.0018) and DOC ( g=0.0023) were indistinguishable (p=0.3) consistent with their identical mechanism of action. Finally, outcomes with DOC in African American (AA) ( g=0.00212) and Caucasian ( g=0.00205) Veterans were indistinguishable (p=0.9) and comparable across all VAMCs. Conclusions: The rate of tumor growth, g, is an excellent biomarker for OS both in clinical trials and in real-world settings. g allows comparisons between trials and for large trial data sets to be used as benchmarks of efficacy. Real-world outcomes in the VAMCs are similar to those in clinical trials. In the egalitarian VAMCs DOC efficacy in PC is comparable in AA and Caucasian Veterans -- indicating inferior outcomes reported in AAs are likely due to differential health care access, not differences in biology.

Using real world data to examine outcomes in immunotherapy-treated patients with metastatic non-small cell lung cancer.

2020 ◽  
Vol 38 (15_suppl) ◽  
pp. e21715-e21715
Author(s):  
Sierra Luciano ◽  
Christian Haudenschild ◽  
Seth Kuranz
Keyword(s):  
Overall Survival ◽  
Real World ◽  
Small Cell ◽  
Advanced Stage ◽  
Small Cell Lung ◽  
Real World Data ◽  
Treatment Pathways ◽  
Time To Treatment ◽  
World Data

e21715 Background: Important questions have been raised about whether real world data (RWD) can be relied upon to support clinical and regulatory decision-making. The aims of this study were to assess overall survival (OS), time-to-treatment (TTT), follow-up time, and treatment pathways in metastatic non-small cell lung cancer (mNSCLC) patients treated with programmed cell death protein 1 inhibitors (PD-1i). Methods: Two mNSCLC cohorts were identified in a US based EMR network. To explore treatment pathways, patients had to have an advanced (stage 3/4) diagnosis of NSCLC confirmed by a tumor registry record. A line of treatment (LOT) was defined as a PD-1i or chemotherapy taken within 30 days. OS, time to treatment, and follow-up time were also calculated. In another cohort patients had to have at least one PD-1i (pembrolizumab, nivolumab, durvalumab, or atezolizmab) and an oncology treatment within 3 months of an advanced stage diagnosis. OS was calculated using Kaplan-Meier analysis and stratified by time after nivolumab approval, quarter of PD-1i initiation, and line of therapy of first PD-1i. Patient characteristics were defined by ICD, CPT, LOINC, and RxNorm terminology. Results: Median overall survival was found to be 213 days (IQR 109, 425.25). In the treatment pathways analysis, 58.3% of patients started on a non-PD-1i chemotherapy as an initial line of treatment for mNSCLC. PD-1i was the most common second line treatment and 41.4% of patients who started on a non-PD-1i therapy switched to a PD-1i as their second line therapy. Median time from advanced diagnosis to PD-1i inhibitor initiation (9.6 months (IQR 3.45, 21.45)) and median structured follow-up times from advanced diagnosis (21.87 months (IQR 11.94, 38.97)) and from inhibitor initiation (8.71 months (3.06, 17.26)) were comparable to results found in other RWD. Conclusions: Overall survival, time to treatment, and other outcomes were consistent with comparable RWD sources, (Stewart M, 2019; Sean K 2018) regardless of treatment timeframe. We demonstrated that our real world evidence based approach provides a robust method for analyzing clinical outcomes, supporting the validity of real world data to complement clinical trials and inform clinical decisions.

Overall survival of cetuximab administered every two weeks versus weekly in real-world data of U.S. patients with metastatic colorectal cancer.

2020 ◽  
Vol 38 (4_suppl) ◽  
pp. 23-23 ◽  
Author(s):  
Chris Pescott ◽  
Michael Batech ◽  
Emmanuelle Boutmy ◽  
Philippe Ronga ◽  
Francois-Xavier Lamy
Keyword(s):  
Colorectal Cancer ◽  
Overall Survival ◽  
Metastatic Colorectal Cancer ◽  
Real World ◽  
Cox Model ◽  
Small Sample Size ◽  
Small Sample ◽  
Patient Death ◽  
Real World Data ◽  
World Data

23 Background: Cetuximab (CET) administered weekly (q1w) at 250 mg/m², after an initial dose of 400 mg/m², is approved in combination with chemotherapy (CT) for the treatment of (K) RAS wild-type metastatic colorectal cancer (mCRC). The use of CET 500 mg/m2 administered with CT every 2 weeks (q2w) is according to US clinical practice guidelines and observed routinely. In this study, we compared q2w vs q1w regimens on overall survival (OS) in a presumed first-line (1L) treatment subcohort and present updated data on the noninferiority of q2w vs q1w in line-agnostic (1L+) treatment using US real-world data. Methods: Using IBM MarketScan, a large US insurance claims database, we classified a cohort of mCRC patients treated between 07/2010 and 12/2016 with CET+CT as q1w or q2w based on observed infusion patterns. Absence of mCRC-related treatment claims preceding CET initiation date (defined as the index date) qualified as CET treated in 1L. A validated algorithm was used to determine patient death. Confounding was accounted for using high-dimensional propensity scoring (hdPS) with inverse probability of treatment weights. OS was compared using Cox proportional hazards regression. Imbalanced confounders after hdPS were added to the Cox model. In 1L+, noninferiority of the q2w regimen was tested with a margin hazard ratio (HR) of 1.25. However, noninferiority could not be tested in 1L due to the small sample size; a test for difference was used instead. Results: Of 2,730 CET-exposed mCRC patients (updated), 1,779 (65.2%) and 951 (34.8%) were classified in q1w and q2w groups, respectively, among which 557 (31.3%) and 316 (33.2%) received CET in 1L. The HR (95% CI) for OS of q2w vs q1w in 1L was 1.10 (0.92-1.31; crude), and 1.05 (0.86-1.29; adjusted; p for difference: 0.625). In 1L+, crude and adjusted HRs were 1.05 (0.94-1.18) and 0.94 (0.85-1.03), respectively, rejecting the inferiority hypothesis at p < 0.001. Conclusions: Only a third of patients received CET in 1L in this study. OS was statistically noninferior in q2w vs q1w in 1L+, and adjusted results in 1L suggest no differences between both treatment schedules. However, more data would be needed to formally test the noninferiority hypothesis in 1L.

scholarly journals Ramucirumab plus paclitaxel as second-line treatment in patients with advanced gastric or gastroesophageal junction adenocarcinoma: a nationwide real-world outcomes in Korea study (KCSG-ST19-16)

2021 ◽  
Vol 13 ◽  
pp. 175883592110428
Author(s):  
Hye Sook Han ◽  
Bum Jun Kim ◽  
Hee-Jung Jee ◽  
Min-Hee Ryu ◽  
Se Hoon Park ◽  
...  
Keyword(s):  
Overall Survival ◽  
Confidence Interval ◽  
Real World ◽  
Gastroesophageal Junction ◽  
Locally Advanced ◽  
Line Treatment ◽  
Second Line ◽  
First Line ◽  
Real World Data ◽  
World Data

Background: Ramucirumab as monotherapy or in combination with paclitaxel is a second-line treatment option recommended for patients with locally advanced unresectable or metastatic gastric or gastroesophageal junction (GEJ) adenocarcinoma. However, real-world data from large study cohorts focused on ramucirumab plus paclitaxel in gastric cancer are limited. Methods: The study population comprised all patients with gastric or GEJ cancer who received ramucirumab plus paclitaxel in South Korea between 1 May 2018 and 31 December 2018. We included patients with advanced gastric or GEJ adenocarcinoma and disease progression after first-line platinum and fluoropyrimidine-containing combination chemotherapy. Results: In total, 1063 patients were included in the present study. The objective response rate and disease control rate were 15.1% and 57.7%, respectively. The median progression-free survival was 4.03 months (95% confidence interval, 3.80–4.27) and the median overall survival was 10.03 months (95% confidence interval, 9.33–10.73). Grade 3 or higher treatment-related adverse events with incidence of ⩾5% were neutropenia (35.1%) and anemia (10.5%). Based on multivariable analysis, overall survival was negatively associated with Eastern Cooperative Oncology Group performance status ⩾2, weight loss ⩾10% in the previous 3 months, GEJ of primary tumor, poor or unknown histologic grade, number of metastatic sites ⩾3, presence of peritoneal metastasis, no prior gastrectomy, and time to second-line since first-line treatment <6 months. Conclusion: Our large-scale, nationwide, real-world data analysis of an unselected real-world population adds evidence for the efficacy and safety of second-line ramucirumab plus paclitaxel in patients with locally advanced unresectable or metastatic gastric or GEJ adenocarcinoma.

scholarly journals Validate Real-World Data-based Endpoint Measures of Cancer Treatment Outcomes

2021 ◽  
Author(s):  
Qian Li ◽  
Hansi Zhang ◽  
Zhaoyi Chen ◽  
Yi Guo ◽  
Thomas George ◽  
...  
Keyword(s):  
Overall Survival ◽  
Clinical Trials ◽  
Data Quality ◽  
Treatment Outcomes ◽  
Real World ◽  
Gold Standard ◽  
Surrogate Marker ◽  
Real World Data ◽  
World Data ◽  
Real World Evidence

Recently, there is a growing interest in using real-world data (RWD) to generate real-world evidence (RWE) that complements clinical trials. Nevertheless, to quantify the treatment effects, it is important to develop meaningful RWD-based endpoints. In cancer trials, two real-world endpoints are particularly of interest: real-world overall survival (rwOS) and real-world time to next treatment (rwTTNT). In this work, we identified ways to calculate these real-world endpoints with structured EHR data, and validated these endpoints against the gold-standard measurements of these endpoints derived from linked EHR and TR data. In addition, we also examined and reported the data quality issues especially the inconsistency between the EHR and TR data. Using survival model, our result showed that patients (1) without subsequent chemotherapy or (2) with subsequent chemotherapy and longer rwTTNT, would have longer rwOS, showing the validity of using rwTTNT as a real-world surrogate marker for measuring cancer endpoints.

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