scholarly journals Clinical outcome assessments in neuro-oncology: a regulatory perspective

2015 ◽  
Vol 3 (1) ◽  
pp. 4-9 ◽  
Author(s):  
Joohee Sul ◽  
Paul G. Kluetz ◽  
Elektra J. Papadopoulos ◽  
Patricia Keegan
Keyword(s):  
Clinical Outcome ◽  
Neurological Diseases ◽  
Objective Response ◽  
Progression Free Survival ◽  
Patient Centered ◽  
Outcome Assessments ◽  
Regulatory Perspective ◽  
Clinical Outcome Assessments ◽  
And Function ◽  
The Impact

Abstract Overall survival, progression-free survival, and to a lesser extent objective response rate, have long been the most widely accepted endpoints used to evaluate clinical benefit in oncology trials. More recently, clinical outcome assessments (COAs) that measure the impact of disease and treatment on patients′ symptoms and function have been recognized as having potential to be an integral component of the risk/benefit analysis of new therapies. Although COAs have been used to evaluate cognitive and physical functioning in neurological diseases, assessing patient-centered outcomes in individuals with malignant brain tumors presents unique challenges. The approach to developing appropriate instruments to measure COAs in neuro-oncology should include identifying areas requiring new tools, reviewing existing tools that may be suitable or adapted for use in clinical trials, and engaging early with regulatory agencies to standardize a set of well-defined and reliable instruments to quantify important patient-centered outcomes.

scholarly journals Opportunities and challenges of incorporating clinical outcome assessments in brain tumor clinical trials

2018 ◽  
Vol 6 (2) ◽  
pp. 81-92 ◽  
Author(s):  
Emanuela Molinari ◽  
Tito R Mendoza ◽  
Mark R Gilbert
Keyword(s):  
Clinical Trials ◽  
Brain Tumor ◽  
Clinical Outcome ◽  
Clinical Trial Design ◽  
Well Being ◽  
Successful Implementation ◽  
Outcome Assessments ◽  
Tumor Studies ◽  
Clinical Outcome Assessments ◽  
The Impact

Abstract Regulatory agencies have progressively emphasized the importance of assessing broader aspects of patient well-being to better define therapeutic gain. As a result, clinical outcome assessments (COAs) are increasingly used to evaluate the impact, both positive and negative, of cancer treatments and in some instances have played a major factor in the regulatory approval of drugs. Challenges remain, however, in the routine incorporation of these measures in cancer clinical trials, particularly in brain tumor studies. Factors unique to brain tumor patients such as cognitive decline and language dysfunction may hamper their successful implementation. Study designs often relegated these outcome measures to exploratory endpoints, further compromising data completion. New strategies are needed to maximize the complementary information that COAs could add to clinical trials alongside more traditional measures such as progression-free and overall survival. The routine incorporation of COAs as either primary or secondary objectives with attention to minimizing missing data should define a novel clinical trial design. We provide a review of the approaches, challenges, and opportunities for incorporating COAs into brain tumor clinical research, providing a perspective for integrating these measures into clinical trials.

scholarly journals Blood Tumor Mutational Burden as a Predictive Biomarker in Patients With Advanced Non-Small Cell Lung Cancer (NSCLC)

2021 ◽  
Vol 11 ◽  
Author(s):  
Yuhui Ma ◽  
Quan Li ◽  
Yaxi Du ◽  
Jingjing Cai ◽  
Wanlin Chen ◽  
...  
Keyword(s):  
Clinical Outcome ◽  
Advanced Nsclc ◽  
Objective Response ◽  
Progression Free Survival ◽  
Predictive Biomarker ◽  
Mutational Burden ◽  
Tumor Mutational Burden ◽  
Nsclc Patients ◽  
The Impact ◽  
The Relationship

This study was designed to investigate the impact of blood tumor mutational burden (bTMB) on advanced NSCLC in Southwest China. The relationship between the tTMB estimated by next-generation sequencing (NGS) and clinical outcome was retrospectively analyzed in tissue specimens from 21 patients with advanced NSCLC. Furthermore, the relationship between the bTMB estimated by NGS and clinical outcome was retrospectively assessed in blood specimens from 70 patients with advanced NSCLC. Finally, 13 advanced NSCLC patients were used to evaluate the utility of bTMB assessed by NGS in differentiating patients who would benefit from immunotherapy. In the tTMB group, tTMB ≥ 10 mutations/Mb was related to inferior progression-free survival (PFS) (hazard ratio [HR], 0.30; 95% CI, 0.08-1.17; log-rank P = 0.03) and overall survival (OS) (HR, 0.30; 95% CI, 0.08-1.16; log-rank P = 0.03). In the bTMB group, bTMB ≥ 6 mutations/Mb was associated with inferior PFS (HR, 0.32; 95% CI, 0.14-1.35; log-rank P < 0.01) and OS (HR, 0.31; 95% CI, 0.14-0.7; log-rank P < 0.01). In the immunotherapy section, bTMB ≥ 6 mutations/Mb was related to superior PFS (HR, 0.32; 95% CI, 0.14-1.35; log-rank P < 0.01) and objective response rates (ORRs) (bTMB < 6: 14.2%; 95% CI, 0.03-1.19; bTMB ≥ 6: 83.3%; 95% CI, 0.91-37.08; P = 0.02). These findings suggest that bTMB is a validated predictive biomarker for determining the clinical outcome of advanced NSCLC patients and may serve as a feasible predictor of the clinical benefit of immunotherapies (anti-PD-1 antibody) in the advanced NSCLC population in Yunnan Province.

THE IMPACT OF ELECTRONIC CLINICAL OUTCOME ASSESSMENTS (ECOA) ON ALZHEIMER’S DISEASE CLINICAL TRIAL DATA QUALITY

2017 ◽  
Vol 13 (7) ◽  
pp. P935
Author(s):  
H. Todd Feaster ◽  
Todd M. Solomon ◽  
Danielle Abi-Saab ◽  
Annamarie Vogt ◽  
Jordan M. Barbone ◽  
...  
Keyword(s):  
Alzheimer’S Disease ◽  
Alzheimer's Disease ◽  
Clinical Trial ◽  
Clinical Outcome ◽  
Data Quality ◽  
Clinical Trial Data ◽  
Trial Data ◽  
Outcome Assessments ◽  
Clinical Outcome Assessments ◽  
The Impact

scholarly journals Development of a Conceptual Framework of Patient-reported Outcomes in Light Chain Amyloidosis: A Qualitative Study

2021 ◽  
Author(s):  
Anita D'Souza ◽  
Judith Myers ◽  
Rachel Cusatis ◽  
Angela Dispenzieri ◽  
Muriel Finkel ◽  
...  
Keyword(s):  
Clinical Outcome ◽  
Conceptual Understanding ◽  
Light Chain ◽  
Patient Reported Outcomes ◽  
Symptom Burden ◽  
Al Amyloidosis ◽  
Outcome Assessments ◽  
Patient Reported ◽  
Clinical Outcome Assessments ◽  
The Impact

Abstract Background Light chain (AL) amyloidosis is a plasma cell neoplasm associated with high early mortality and severe morbidity that can cause severe disability. We explored the impact of AL amyloidosis on symptoms and well-being from the perspectives of patients and health care providers who regularly care for AL patients. We intended to develop a conceptual understanding of patient-reported outcomes in AL amyloidosis to identify the context of use and concept of interest for a clinical outcome assessments tool in this disease.Method Twenty patients and 10 professionals were interviewed. Patient interviews captured the spectrum of amyloidosis experience including time from diagnosis, type of organ involvement, and presence and type of treatment received. Interviews with professionals included physicians, advanced practice providers, registered nurse, and a patient advocate; these interviews covered similar topics. Results The impact of AL amyloidosis on patients’ life was multidimensional, with highly subjective perceptions of normality and meaning. Four major themes from patients and experts included diagnosis of AL amyloidosis, living with AL amyloidosis, symptom burden, and social roles. Barriers to patient-reported outcomes data collection in patients were additionally explored from experts. The themes provide a comprehensive understanding of the important experiences of symptom burden and its impact on daily life from AL amyloidosis patients’ and from the perspectives of professionals who care for patients with AL amyloidosis. Conclusion These findings further the conceptual understanding and identification of a preliminary model of concept of interest for development of a clinical outcome assessments tool for AL amyloidosis.

Clinical outcome assessments of motor status in patients undergoing brain tumor surgery

2021 ◽  
Vol 201 ◽  
pp. 106420
Author(s):  
Mayla Santana Correia ◽  
Iuri Santana Neville ◽  
Cesar Cimonari de Almeida ◽  
Cintya Yukie Hayashi ◽  
Luana Talita Diniz Ferreira ◽  
...  
Keyword(s):  
Brain Tumor ◽  
Clinical Outcome ◽  
Tumor Surgery ◽  
Brain Tumor Surgery ◽  
Outcome Assessments ◽  
Clinical Outcome Assessments

Association of statins and nivolumab activity in patients with metastatic renal cell carcinoma (mRCC): Results from the phase II nivoren—GETUG AFU 26 trial.

2021 ◽  
Vol 39 (6_suppl) ◽  
pp. 359-359
Author(s):  
Emeline Colomba ◽  
Ronan Flippot ◽  
Cécile Dalban ◽  
Sylvie Negrier ◽  
Christine Chevreau ◽  
...  
Keyword(s):  
Phase Ii ◽  
Immune Modulation ◽  
Performance Status ◽  
Objective Response ◽  
Progression Free Survival ◽  
Population Based ◽  
Metabolic Reprogramming ◽  
Oncogenic Signaling ◽  
Grade 3 ◽  
The Impact

359 Background: Statins are HMG-CoA inhibitors that regulate several mechanisms involved in tumor growth, including mitochondrial metabolism, activation of oncogenic signaling pathways, and immune modulation. Population-based studies showed that statin intake may be negatively associated with RCC onset. The impact of statins on response to immunotherapy in mRCC is unknown. Herein we study the association between statin administration and outcomes in patients with mRCC treated with nivolumab in the NIVOREN-GETUG AFU 26 phase II trial (NCT03013335). Methods: Patients with mRCC who failed previous VEGFR inhibitors were included. We assessed nivolumab activity, including objective response rate (ORR), progression-free survival (PFS), and overall survival (OS) according to statin intake at baseline. Toxicity was assessed using CTCAE v4.0. Results: Overall,133 patients were treated with statins at baseline among 702 evaluable for concomitant therapies (19%). Among them, median age was 68 (49-90), 84% were male, 85% had a performance status ≥ 80%, 42% were overweight and 20% obese. Patients treated with statins had mostly good (23%) or intermediate (58%) IMDC risk, 64% had grade 3 or 4 tumors, and nivolumab was given in a third line setting or more in 55%. Median follow-up was 23.9 months (95%CI 23.0-24.5) in the overall cohort. The ORR was 26% in patients treated with statins, PFS 5.0 months (CI95% 3.0 – 5.5), OS 27.9 months (CI95% 19.4-30.3). Outcomes of patients with or without statins did not differ significantly. Similar rates of grade 3-5 TRAE were reported in patients with (20%) or without (18%) statin intake. Conclusions: This is the first study to evaluate statin intake and outcomes with nivolumab in patients with mRCC. Despite numerically higher ORR, statins were not significantly associated with improved outcomes. These data require other analyzes considering other factors such as BMI and other comorbidities. Further studies may help better understand the interplay between immunity and metabolic reprogramming in RCC.

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