scholarly journals PCN16 External Control Analysis of Overall Survival in BRAFV600 Mutated Metastatic NSCLC: Comparing Single-Arm Dabrafenib+Trametinib Clinical Trial Outcomes with Real-World Standards of Care

2020 ◽  
Vol 23 ◽  
pp. S423
Author(s):  
H. Kanakamedala ◽  
W.H. Wu ◽  
I.S. Souef ◽  
F. Asad Zadeh Vosta Kolaei ◽  
S. Zhang ◽  
...  
2020 ◽  
Vol 16 (35) ◽  
pp. 2939-2948
Author(s):  
Martin Boegemann ◽  
Katrin Schlack ◽  
Michael Rink ◽  
Stephan Bernhardt ◽  
Michael Moran ◽  
...  

Aim: Examine the effects of baseline hypertension (HTN) and statin or proton pump inhibitor (PPI) use on sunitinib treatment outcomes in STAR-TOR, a real-world registry. Materials & methods: Presence or absence of HTN and use or nonuse of statins or PPIs were determined at registry entry. End points included overall survival (OS) and progression-free survival (PFS). Results: Data were from 557 patients. Presence or absence of HTN did not affect OS or PFS. PFS (median [95% CI]) was longer in statin users (9.4 [6.5–13.6] months) versus nonusers (6.9 [5.7–8.2] months) (p = 0.0442). OS was shorter in PPI users (20.2 [14.9–28.3] months) versus nonusers (25.7 [22.7–33.0] months) (p = 0.0212). Conclusion: Comorbidities and comedications may affect real-world sunitinib treatment outcomes. Clinical Trial Registration: NCT00700258 ( ClinicalTrials.gov )


2019 ◽  
Vol 37 (15_suppl) ◽  
pp. 5074-5074
Author(s):  
Harshraj Leuva ◽  
Mengxi Zhou ◽  
Julia Wilkerson ◽  
Keith Sigel ◽  
Ta-Chueh Hsu ◽  
...  

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.


2020 ◽  
Vol 23 ◽  
pp. S422
Author(s):  
J.F.H. Eijsink ◽  
M.H. Schöttler ◽  
M.N.M.T. Al Khayat ◽  
J.W.B. de Groot ◽  
C. Boersma ◽  
...  

2016 ◽  
Vol 34 (15_suppl) ◽  
pp. 6564-6564
Author(s):  
Jason Shafrin ◽  
Ningqi Hou ◽  
Ron Brookmeyer ◽  
Desi Peneva ◽  
Seanna Vine ◽  
...  

2019 ◽  
Vol 19 (10) ◽  
pp. e115-e116
Author(s):  
Robert Cornell ◽  
Hari Parameswaran ◽  
Shijie Tang ◽  
Alyssa Barnstead ◽  
Noa Biran ◽  
...  

2021 ◽  
Vol 6 (1) ◽  
Author(s):  
Fawaz A ◽  
◽  
Shim I ◽  
Tilley D ◽  
Kelaney MR ◽  
...  

Background: Pazopanib is an oral multitarget tyrosine kinase inhibitor that is currently approved for the treatment of select subtypes of advanced Soft Tissue Sarcoma (STS) in patients who have progressed on prior anthracyclinebased chemotherapy regimens. In this study, we examine data from multiple centers to assess the efficacy of pazopanib in practice outside of a clinical trial setting. Methods: A retrospective chart analysis was conducted for pre-treated, advanced soft tissue sarcoma patients who began treatment with pazopanib in Alberta, Canada and Cairo, Egypt (2012-2018). Results: In total, 39 predominantly male (56.4%) patients received pazopanib. The median age was 51, 67% of whom had an ECOG of one or less. The predominant sarcoma subtype was leiomyosarcoma (30.8%), and all patients had received at least one prior line of systemic therapy. Thirtytwo of the 39 patients (82%) were initially given the full dose of 800mg with a median time on treatment of 116 days. Seven of the 39 (18%) patients required a dose reduction while on treatment. A majority (94.9%) of patients ultimately discontinued pazopanib treatment for reasons including death (21.6%), disease progression (62.2%), and toxicity (16.4%). The median progression-free and overall survival for these patients was 4.1 months (95%CI, 3.6-4.5) and 8.4 months (95% CI, 4.3-12.5), respectively. Conclusion: Pazopanib is an efficient and generally well-tolerated oral systemic therapy for the treatment of advanced, pre-treated, non-adipocytic soft tissue sarcoma. These results show the efficacy of pazoponib outside of a clinical trial setting.


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