scholarly journals Clinical Trials for Real-World Elderly Patients With Metastatic Soft-Tissue Sarcomas: The Importance of Study Design and Patient-Reported Outcomes

2021 ◽  
Vol 39 (7) ◽  
pp. 860-861
Author(s):  
Eugenie Younger ◽  
Olga Husson ◽  
Winette T. A. van der Graaf
Keyword(s):  
Clinical Trials ◽  
Soft Tissue ◽  
Elderly Patients ◽  
Study Design ◽  
Real World ◽  
Patient Reported Outcomes ◽  
Soft Tissue Sarcomas ◽  
Patient Reported

Problems with the MetaBLIND study: An examination of data on blinding patients in trials with patient-reported outcomes

2021 ◽  
pp. 135910532110593
Author(s):  
Michiel Tack
Keyword(s):  
Clinical Trials ◽  
Epidemiological Study ◽  
Study Design ◽  
Patient Reported Outcomes ◽  
Randomized Trials ◽  
Effect Sizes ◽  
Patient Reported ◽  
The Impact ◽  
Trial Participants

MetaBLIND is the largest meta-epidemiological study on the impact of blinding in randomized trials to date. We examined MetaBLIND data on the impact of blinding patients on patient-reported outcomes. 68 out of 132 included trials tested knowledge recall and had questionable relevance to clinical trials. In 17 out of 18 comparisons, the number of trials in the blinded or nonblinded group was 2 or lower. In several key trials, the blinding status was uncertain. Effect sizes compared in MetaBLIND appear to reflect random differences in study design and setting rather than the impact of blinding trial participants.

scholarly journals PARE0026 WHICH PATIENT-REPORTED OUTCOMES DO RHEUMATOLOGY PATIENTS FIND IMPORTANT TO TRACK DIGITALLY? A REAL-WORLD LONGITUDINAL STUDY IN ARTHRITISPOWER

2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 1298.1-1299
Author(s):  
W. B. Nowell ◽  
C. L. Kannowski ◽  
K. Gavigan ◽  
Z. Cai ◽  
A. Cardoso ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Disease Activity ◽  
Physical Function ◽  
Real World ◽  
Patient Reported Outcomes ◽  
Joint Stiffness ◽  
Summary Score ◽  
Patient Reported ◽  
Rheumatic Conditions ◽  
Over Time

Background:Development of a standardized approach to assess key elements of disease activity in rheumatology clinical trials has been the goal of Outcome Measures in Rheumatology Clinical Trials (OMERACT), American College of Rheumatology (ACR), and European League Against Rheumatism (EULAR).1,2,3The core sets of measures developed include assessments and composite indices incorporating use of patient-reported outcomes (PROs) and clinical measures and clinicians’ assessments to quantify disease activity over time.2PROs are important indicators of disease activity and variability, and they are increasingly used to evaluate treatment effectiveness. Little is known about PROs that patients with rheumatic conditions find most important to convey their experience with their condition and its treatment.Objectives:To examine PROs selected by patients with rheumatic conditions in the ArthritisPower registry to identify symptoms they found most important to track digitally.Methods:Adult US patients within the ArthritisPower registry with rheumatoid arthritis (RA), psoriatic arthritis (PsA), ankylosing spondylitis (AS), systemic lupus erythematosus (SLE), osteoporosis (OP), osteoarthritis (OA), and fibromyalgia syndrome (FMS) were invited via email to participate in this study. Enrolled participants (pts) were prompted to select ≤10 PRO symptom measures they felt were important to track for their condition at baseline via the ArthritisPower app. At 3 subsequent time points (Month [m] 1, m2, m3), pts were given the option to continue tracking their previously selected PRO measures or to add, remove and/or select different measures. At m3, pts completed an exit survey to prioritize ≤5 measures from all measures selected during study participation and to specify other symptoms not available that they would have wanted to track. Measures were rank-ordered based on number of pts rating the item as their 1st, 2nd, 3rd, 4th or 5th choice and weighted by multiplying the rank number by its inverse for a single, weighted summary score for each measure. Values were summed across all pts to produce a summary score for each measure.Results:Among pts who completed initial selection of PRO assessments at baseline (N=253), 184 pts confirmed or changed PRO selections across m1-3. Mean (SD) age of pts was 55.7 (9.2) yrs, 89.3% female, 91.3% White, mean disease duration of 11.6 (10.6) yrs. The majority (64.8%) self-reported OA, followed by RA (48.6%), FMS (40.3%), PsA (26.1%), OP (21.0%), AS (15.8%) and SLE (5.9%), not mutually exclusive, and were similar to the overall ArthritisPower population. The average number of instruments (SD) selected for baseline completion was 7.0 (2.5), 7.1 (2.4) at m1, 7.2 (2.4) at m2, and 7.0 (2.5) at m3. The top 5 PROs ranked by pts overall as most important (weighted summary score) for tracking were Fatigue (71), Physical Function (58), Pain Intensity (50), Pain Interference (49), Duration of Morning Joint Stiffness (41) (Figure 1). Fatigue, Physical Function, and Pain were consistently in the top 5 across diseases while Depression was more frequent among pts with OA, AS and FMS. Pts’ PRO selections showed stability over time except for the RA Flare measure which decreased from 70.5% of RA pts at baseline to 13.6% at m3.Conclusion:The symptoms prioritized by pts included fatigue, physical function, pain, and joint stiffness. Pts‘ choices were consistent over time. These findings provide insights into symptoms rheumatology patients find most important and will be useful to inform design of future patient-centric clinical trials and real-world evidence generation.References:[1]Boers M, et al. J Rheumatol Suppl. 1994;41:86–89.[2]Felson DT, et al. Arthritis Rheum. 1993;36:729–740.[3]Tugwell P, et al. J Rheumatol. 1993;20:555–556.Disclosure of Interests:W. Benjamin Nowell: None declared, Carol L. Kannowski Shareholder of: Eli Lilly and Company, Employee of: Eli Lilly and Company, Kelly Gavigan: None declared, Zhihong Cai Shareholder of: Eli Lilly and Company, Employee of: Eli Lilly and Company, Anabela Cardoso Shareholder of: Eli Lilly and Company, Employee of: Eli Lilly and Company, Theresa Hunter Shareholder of: Eli Lilly and Company, Employee of: Eli Lilly and Company, Shilpa Venkatachalam: None declared, Julie Birt Shareholder of: Eli Lilly and Company, Employee of: Eli Lilly and Company, Jennifer Workman Shareholder of: Eli Lilly and Company, Employee of: Eli Lilly and Company, Jeffrey Curtis Grant/research support from: AbbVie, Amgen, Bristol-Myers Squibb, Corrona, Janssen, Lilly, Myriad, Pfizer, Regeneron, Roche, UCB, Consultant of: AbbVie, Amgen, Bristol-Myers Squibb, Corrona, Janssen, Lilly, Myriad, Pfizer, Regeneron, Roche, UCB

scholarly journals Long-term real-world experience with ipilimumab and non-ipilimumab therapies in advanced melanoma: the IMAGE study

BMC Cancer ◽  
2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Stéphane Dalle ◽  
Laurent Mortier ◽  
Pippa Corrie ◽  
Michal Lotem ◽  
Ruth Board ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Real World ◽  
Patient Reported Outcomes ◽  
Advanced Melanoma ◽  
Image Study ◽  
Patient Reported ◽  
Treatment Naïve ◽  
Systemic Therapies

Abstract Background Ipilimumab has shown long-term overall survival (OS) in patients with advanced melanoma in clinical trials, but robust real-world evidence is lacking. We present long-term outcomes from the IMAGE study (NCT01511913) in patients receiving ipilimumab and/or non-ipilimumab (any approved treatment other than ipilimumab) systemic therapies. Methods IMAGE was a multinational, prospective, observational study assessing adult patients with advanced melanoma treated with ipilimumab or non-ipilimumab systemic therapies between June 2012 and March 2015 with ≥3 years of follow-up. Adjusted OS curves based on multivariate Cox regression models included covariate effects. Safety and patient-reported outcomes were assessed. Results Among 1356 patients, 1094 (81%) received ipilimumab and 262 (19%) received non-ipilimumab index therapy (systemic therapy [chemotherapy, anti–programmed death 1 antibodies, or BRAF ± MEK inhibitors], radiotherapy, and radiosurgery). In the overall population, median age was 64 years, 60% were male, 78% were from Europe, and 78% had received previous treatment for advanced melanoma. In the ipilimumab-treated cohort, 780 (71%) patients did not receive subsequent therapy (IPI-noOther) and 314 (29%) received subsequent non-ipilimumab therapy (IPI-Other) on study. In the non-ipilimumab–treated cohort, 205 (78%) patients remained on or received other subsequent non-ipilimumab therapy (Other-Other) and 57 (22%) received subsequent ipilimumab therapy (Other-IPI) on study. Among 1151 patients who received ipilimumab at any time during the study (IPI-noOther, IPI-Other, and Other-IPI), 296 (26%) reported CTCAE grade ≥ 3 treatment-related adverse events, most occurring in year 1. Ipilimumab-treated and non-ipilimumab–treated patients who switched therapy (IPI-Other and Other-IPI) had longer OS than those who did not switch (IPI-noOther and Other-Other). Patients with prior therapy who did not switch therapy (IPI-noOther and Other-Other) showed similar OS. In treatment-naive patients, those in the IPI-noOther group tended to have longer OS than those in the Other-Other group. Patient-reported outcomes were similar between treatment cohorts. Conclusions With long-term follow-up (≥ 3 years), safety and OS in this real-world population of patients treated with ipilimumab 3 mg/kg were consistent with those reported in clinical trials. Patient-reported quality of life was maintained over the study period. OS analysis across both pretreated and treatment-naive patients suggested a beneficial role of ipilimumab early in treatment. Trial registration ClinicalTrials.gov, NCT01511913. Registered January 19, 2012 – Retrospectively registered, https://clinicaltrials.gov/ct2/show/NCT01511913

2327-PUB: Real-World Patient-Reported Outcomes (PROs) with Insulin Glargine 300 U/ml (Gla-300) in Basal Bolus-Treated People with Type 2 Diabetes (T2DM): The MAGE Study

Diabetes ◽  
2019 ◽  
Vol 68 (Supplement 1) ◽  
pp. 2327-PUB ◽  
Author(s):  
ANN A. VERHAEGEN ◽  
ANDRÉ SCHEEN ◽  
KATHY C. ALEXANDRE ◽  
JACQUES BRUHWYLER ◽  
IDES M. COLIN
Keyword(s):  
Type 2 Diabetes ◽  
Insulin Glargine ◽  
Real World ◽  
Patient Reported Outcomes ◽  
Patient Reported ◽  
Basal Bolus

scholarly journals Potential of patient-reported outcomes as nonprimary endpoints in clinical trials

2013 ◽  
Vol 11 (1) ◽  
pp. 83 ◽  
Author(s):  
Ari Gnanasakthy ◽  
Sandra Lewis ◽  
Marci Clark ◽  
Margaret Mordin ◽  
Carla DeMuro
Keyword(s):  
Clinical Trials ◽  
Patient Reported Outcomes ◽  
Patient Reported

scholarly journals SAT0121 PAIN AND OTHER PATIENT-REPORTED OUTCOMES IN PATIENTS WITH RHEUMATOID ARTHRITIS WHO DID OR DID NOT ACHIEVE TREATMENT RESPONSE BASED ON IMPROVEMENT IN SWOLLEN JOINTS IN TOCILIZUMAB CLINICAL TRIALS

2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 994.2-995
Author(s):  
A. Sebba ◽  
J. Han ◽  
S. Mohan
Keyword(s):  
Rheumatoid Arthritis ◽  
Clinical Trials ◽  
Pain Score ◽  
Patient Reported Outcomes ◽  
Component Score ◽  
Link Type ◽  
Pain Scores ◽  
Sf 36 ◽  
Patient Reported ◽  
Nonresponder Group

Background:Significant improvements in pain and other patient-reported outcomes (PROs) have been shown in large clinical trials in patients with rheumatoid arthritis (RA) who receive tocilizumab (TCZ) compared with placebo (PBO). Recent data suggest that pain in RA may be noninflammatory as well as inflammatory, and improvement in pain scores and other PROs may be seen in patients who do not respond to treatment based on disease activity measures that evaluate inflammation.Objectives:To assess changes in pain scores and other PROs in patients with RA who did or did not achieve ≥ 20% improvement in SJC in TCZ clinical trials.Methods:Data from patients with active RA who received intravenous TCZ 8 mg/kg + MTX or PBO + MTX in 3 Phase III studies (OPTION [NCT00106548], TOWARD [NCT00106574] and LITHE [NCT00109408]) were included. All patients had moderate to severe RA with an inadequate response or intolerance of MTX (OPTION, LITHE) or conventional synthetic disease-modifying antirheumatic drugs (csDMARDs; TOWARD). Changes in pain (visual analog scale [VAS], 0-100 mm), Health Assessment Questionnaire Disability Index (HAQ-DI, 0-3), 36-Item Short Form Survey (SF-36) physical component score (PCS) and mental component score (MCS; 0-50) and Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue score (0-52) from baseline to Week 24 were evaluated. Results were compared between patients receiving TCZ + MTX and those receiving PBO + MTX in both patients who achieved ≥ 20% improvement in SJC (responders) and those who did not (nonresponders). The changes from baseline were analyzed using a mixed model with repeated measures, including the following covariates and interactions: treatment, visit, baseline of endpoint, region, baseline DAS28 and interactions of visit with treatment and baseline of endpoint.Results:Data from 1254 responders (TCZ + MTX, n = 831; PBO + MTX, n = 423) and 620 nonresponders (TCZ + MTX, n = 225; PBO + MTX, n = 395) were included. Patients receiving TCZ + MTX had significantly greater improvement in pain scores and HAQ-DI compared with PBO + MTX in the responder group (–27.19 vs –16.77 and –0.55 vs –0.34, respectively;P< 0.0001 for both) and nonresponder group (–9.59 vs 2.53 and –0.20 vs 0.01;P< 0.0001 for both) at Week 24 (Figure 1). Similar results were seen at Week 16 in the nonresponder group (–11.06 vs –2.38 and –0.23 vs –0.04;P< 0.0001 for both) prior to initiation of rescue treatment. At Week 24 in the responder group, patients receiving TCZ + MTX had significantly greater improvements compared with PBO + MTX in SF-36 PCS and MCS (9.16 vs 5.71 and 6.55 vs 3.79, respectively;P< 0.0001 for both) (Figure 2) and FACIT-Fatigue (8.39 vs 5.11;P< 0.0001). In the nonresponder group, patients receiving TCZ + MTX had significantly greater improvements compared with PBO + MTX in SF-36 PCS at Week 16 (3.81 vs 1.65;P= 0.0006) and Week 24 (4.42 vs 1.01;P< 0.0001) (Figure 2) and FACIT-Fatigue at Week 16 (3.82 vs 1.32;P= 0.0039) and Week 24 (3.90 vs 1.40;P= 0.0111).Conclusion:Patients with RA who received TCZ + MTX had significantly greater improvements in pain score and other PROs than those who received PBO + MTX regardless of whether they achieved ≥ 20% improvement in SJC. Clinical outcome at Week 24 correlated well with PROs, with a relatively larger improvement in pain score and other PROs in the responder group than in the nonresponder group; relative to PBO + MTX, these improvements appear numerically similar in the responder and nonresponder groups with consistently smaller difference between the groups in TCZ-treated arms. The consistent effect of TCZ on PROs in both responder and nonresponder groups warrants further study on the impact of TCZ on sources of pain independent of that caused by joint inflammation.Figure:Acknowledgments:This study was sponsored by Genentech, Inc. Support for third-party writing assistance, furnished by Health Interactions, Inc, was provided by Genentech, Inc.Disclosure of Interests:Anthony Sebba Consultant of: Genentech, Gilead, Lilly, Regeneron Pharmaceuticals Inc., Sanofi, Speakers bureau: Lilly, Roche, Sanofi, Jian Han Shareholder of: Genentech, Inc., Employee of: Genentech, Inc., Shalini Mohan Shareholder of: Genentech, Inc., Employee of: Genentech, Inc.

scholarly journals Longitudinal Analysis of Patient-Reported Outcomes in Clinical Trials: Applications of Multilevel and Multidimensional Item Response Theory

Psychometrika ◽  
2021 ◽  
Author(s):  
Li Cai ◽  
Carrie R. Houts
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Latent Variable ◽  
Patient Reported Outcomes ◽  
Multilevel Models ◽  
Latent Variable Models ◽  
Trial Data ◽  
Regulatory Science ◽  
Measurement Information ◽  
Patient Reported

AbstractWith decades of advance research and recent developments in the drug and medical device regulatory approval process, patient-reported outcomes (PROs) are becoming increasingly important in clinical trials. While clinical trial analyses typically treat scores from PROs as observed variables, the potential to use latent variable models when analyzing patient responses in clinical trial data presents novel opportunities for both psychometrics and regulatory science. An accessible overview of analyses commonly used to analyze longitudinal trial data and statistical models familiar in both psychometrics and biometrics, such as growth models, multilevel models, and latent variable models, is provided to call attention to connections and common themes among these models that have found use across many research areas. Additionally, examples using empirical data from a randomized clinical trial provide concrete demonstrations of the implementation of these models. The increasing availability of high-quality, psychometrically rigorous assessment instruments in clinical trials, of which the Patient-Reported Outcomes Measurement Information System (PROMIS®) is a prominent example, provides rare possibilities for psychometrics to help improve the statistical tools used in regulatory science.

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