scholarly journals Response-shift effects in neuromyelitis optica spectrum disorder: estimating response-shift-adjusted scores using equating

2021 ◽  
Author(s):  
Carolyn E. Schwartz ◽  
Roland B. Stark ◽  
Brian D. Stucky ◽  
Yuelin Li ◽  
Bruce D. Rapkin
Keyword(s):  
Clinical Trial ◽  
Neuromyelitis Optica ◽  
Clinical Trial Data ◽  
Response Shift ◽  
Trial Data ◽  
Spectrum Disorder ◽  
Neuromyelitis Optica Spectrum Disorder ◽  
Score Distribution ◽  
Shared Variance ◽  
The Impact

Abstract Background In our companion paper, random intercept models (RIMs) investigated response-shift effects in a clinical trial comparing Eculizumab to Placebo for people with neuromyelitis optica spectrum disorder (NMOSD). RIMs predicted Global Health using the EQ-5D Visual Analogue Scale item (VAS) to encompass broad criteria that people might consider. The SF36™v2 mental and physical component scores (MCS and PCS) helped us detect response shift in VAS. Here, we sought to “back-translate” the VAS into the MCS/PCS scores that would have been observed if response shift had not been present. Methods This secondary analysis utilized NMOSD clinical trial data evaluating the impact of Eculizumab in preventing relapses (n = 143). Analyses began by equating raw scores from the VAS, MCS, and PCS, and computing scores that removed response-shift effects. Correlation analysis and descriptive displays provided a more comprehensive examination of response-shift effects. Results MCS and PCS crosswalks with VAS equated the scores that include and exclude response-shift effects. These two sets of scores had low shared variance for MCS for both groups, suggesting that corresponding mental health constructs were substantially different. The shared variance contrast for physical health was distinct only for the Placebo group. The larger MCS response-shift effects were found at end of study for Placebo only and were more prominent at extremes of the MCS score distribution. Conclusions Our results reveal notable treatment group differences in MCS but not PCS response shifts, which can explain null results detected in previous work. The method introduced herein provides a way to provide further information about response-shift effects in clinical trial data.

scholarly journals Response-shift effects in neuromyelitis optica spectrum disorder: a secondary analysis of clinical trial data

2020 ◽  
Author(s):  
Carolyn E. Schwartz ◽  
Roland B. Stark ◽  
Brian D. Stucky
Keyword(s):  
Clinical Trial ◽  
Random Effects ◽  
Neuromyelitis Optica ◽  
Treatment Effects ◽  
Secondary Analysis ◽  
Response Shift ◽  
Small Sample ◽  
Spectrum Disorder ◽  
Equation Modeling ◽  
Neuromyelitis Optica Spectrum Disorder

Abstract Background Researchers have long posited that response-shift effects may obfuscate treatment effects. The present work investigated possible response-shift effects in a recent clinical trial testing a new treatment for Neuromyelitis Optica Spectrum Disorder (NMOSD). This pivotal trial provided impressive support for the drug Eculizumab in preventing relapse, but less strong or null results as the indicators became more subjective or evaluative. This pattern of results suggests that response-shift effects are present. Methods This secondary analysis utilized data from a randomized, double-blind trial evaluating the impact of Eculizumab in preventing relapses in 143 people with NMOSD. Treatment arm and then relapse status were hypothesized ‘catalysts’ of response shift in two series of analyses. We devised a “de-constructed” version of Oort structural-equation modeling using random-effects modeling for use in small samples. This method begins by testing an omnibus response-shift hypothesis and then, pending a positive result, implements a series of random-effects models to elucidate specific response-shift effects. Results In the omnibus test, the ‘standard quality-of-life (QOL) model’ captured substantially less well the experience of placebo as compared to Eculizumab group. Recalibration and reconceptualization response-shift effects were detected. Detected relapse-related response shifts included recalibration, reprioritization, and reconceptualization. Conclusions Trial patients experienced response shifts related to treatment- and relapse-related experiences. Published trial results likely under-estimated Eculizumab vs. Placebo differences due to recalibration and reconceptualization, and relapse effects due to recalibration, reprioritization, and reconceptualization. This novel random-effects- model application builds on response-shift theory and provides a small-sample method for better estimating treatment effects in clinical trials.

scholarly journals Setting the IMPACT (IMProve Access to Clinical Trial data) Observatory baseline

2018 ◽  
Vol 28 (1) ◽  
Author(s):  
Mersiha Mahmić-Kaknjo ◽  
Josip Šimić ◽  
Karmela Krleža-Jerić
Keyword(s):  
Clinical Trial ◽  
Clinical Trial Data ◽  
Trial Data ◽  
The Impact ◽  
Improve Access

scholarly journals The impact of patient-reported outcome (PRO) data from clinical trials: a systematic review and critical analysis

2019 ◽  
Vol 17 (1) ◽  
Author(s):  
Samantha Cruz Rivera ◽  
Derek G. Kyte ◽  
Olalekan Lee Aiyegbusi ◽  
Anita L. Slade ◽  
Christel McMullan ◽  
...  
Keyword(s):  
Systematic Review ◽  
Clinical Trial ◽  
Clinical Trials ◽  
Case Studies ◽  
Real World ◽  
Clinical Trial Data ◽  
Research Impact ◽  
Trial Data ◽  
Patient Reported ◽  
The Impact

Abstract Background Patient-reported outcomes (PROs) are commonly collected in clinical trials and should provide impactful evidence on the effect of interventions on patient symptoms and quality of life. However, it is unclear how PRO impact is currently realised in practice. In addition, the different types of impact associated with PRO trial results, their barriers and facilitators, and appropriate impact metrics are not well defined. Therefore, our objectives were: i) to determine the range of potential impacts from PRO clinical trial data, ii) identify potential PRO impact metrics and iii) identify barriers/facilitators to maximising PRO impact; and iv) to examine real-world evidence of PRO trial data impact based on Research Excellence Framework (REF) impact case studies. Methods Two independent investigators searched MEDLINE, EMBASE, CINAHL+, HMIC databases from inception until December 2018. Articles were eligible if they discussed research impact in the context of PRO clinical trial data. In addition, the REF 2014 database was systematically searched. REF impact case studies were included if they incorporated PRO data in a clinical trial. Results Thirty-nine publications of eleven thousand four hundred eighty screened met the inclusion criteria. Nine types of PRO trial impact were identified; the most frequent of which centred around PRO data informing clinical decision-making. The included publications identified several barriers and facilitators around PRO trial design, conduct, analysis and report that can hinder or promote the impact of PRO trial data. Sixty-nine out of two hundred nine screened REF 2014 case studies were included. 12 (17%) REF case studies led to demonstrable impact including changes to international guidelines; national guidelines; influencing cost-effectiveness analysis; and influencing drug approvals. Conclusions PRO trial data may potentially lead to a range of benefits for patients and society, which can be measured through appropriate impact metrics. However, in practice there is relatively limited evidence demonstrating directly attributable and indirect real world PRO-related research impact. In part, this is due to the wider challenges of measuring the impact of research and PRO-specific issues around design, conduct, analysis and reporting. Adherence to guidelines and multi-stakeholder collaboration is essential to maximise the use of PRO trial data, facilitate impact and minimise research waste. Trial registration Systematic Review registration PROSPERO CRD42017067799.

PO157 The impact of neuromyelitis optica spectrum disorder on employment

2017 ◽  
Vol 88 (Suppl 1) ◽  
pp. A54.3-A54
Author(s):  
Kerry Mutch ◽  
Sam Linaker ◽  
Dan Whittam ◽  
Shahd Hamid ◽  
Anu Jacob
Keyword(s):  
Neuromyelitis Optica ◽  
Spectrum Disorder ◽  
Neuromyelitis Optica Spectrum Disorder ◽  
The Impact

The impact of immunotherapy education on GI cancers.

2021 ◽  
Vol 39 (3_suppl) ◽  
pp. 479-479
Author(s):  
Kinjal Parikh ◽  
Katie Lucero ◽  
Charlotte Warren ◽  
Emily Sherene Van Laar ◽  
Patrick Kugel ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Checkpoint Inhibitors ◽  
Statistical Significance ◽  
Clinical Trial Data ◽  
Trial Data ◽  
Educational Activities ◽  
Cancer Subtypes ◽  
Gi Cancers ◽  
The Impact

479 Background: Gastrointestinal (GI) cancers are a heterogeneous group of cancers with varying underlying pathophysiology and distinct treatment paradigms. Immunotherapy (IO) is unique in each of the subtypes and biomarkers utility varies. With the expansion of IO in each cancer subtypes, education remains essential to optimize patient outcomes through integration of the latest evidence-based data at point of care. Through the partnership between Medscape Oncology and the Society for Immunotherapy of Cancer, 2 educational activities were designed to increase the knowledge and competence of oncologists surrounding the role of IO in patients with advanced GI cancers. Methods: The 2 educational activities included a text based online activity with 3 chapters focused on gastroesophageal cancers, colorectal cancers, and hepatocellular carcinoma (HCC) and a 30-minute online, video discussion with 3 faculty and synchronized slides on HCC. Educational effectiveness was assessed with repeated paired pre/post assessment where learners served as their own controls. A chi-square test was used to identify statistical significance in proportion of correct responses. The first activity launched 11/27/2019 and the second activity launched on 5/8/20. Data were collected and reported through 8/25/2020. Results: A total of 8433 learners, including 1543 oncologists, participated from 11/2019 through 8/2020. Participation in education resulted in significant relative improvements among oncologist learners on IO in GI cancers in (n = 641): 110%: role or eligibility of immune checkpoint inhibitors (ICIs) (p < .001) 38%: clinical trial data of ICIs (p < .001) Subsequent education on unresectable HCC demonstrated a significant relative improvement in both knowledge and competence for oncologist learners (n = 902): 19%: regarding clinical trial data in unresectable HCC (p = .073) 19%; competence identifying role of ICIs in unresectable HCC (p < .05) 59%: competence managing irAEs in unresectable HCC (p < .001). Conclusions: These 2 online CME-certified educational activities resulted in statistically significant gains in oncologist knowledge surrounding the use of IO in advanced GI cancers Follow-up education on HCC demonstrated the value and benefit of multi-modal and sequential activities on improving competence among oncologists caring for patients with unresectable HCC There remains a need for continuous education as more oncologists utilize IO in their practice while the understanding and availability of clinical data continues to expand and evolve in the varying GI cancer subtypes. More than 50% of learners continued to demonstrate a need in understanding the clinical trial data or role of IO in metastatic GI cancers with more than 40% of the learners demonstrating continued need on clinical trial data or role of IO in HCC specifically.

Comparison of the efficacy of azathioprine and rituximab in neuromyelitis optica spectrum disorder: a randomized clinical trial

2017 ◽  
Vol 264 (9) ◽  
pp. 2003-2009 ◽  
Author(s):  
Zahra Nikoo ◽  
Shervin Badihian ◽  
Vahid Shaygannejad ◽  
Nasrin Asgari ◽  
Fereshteh Ashtari
Keyword(s):  
Clinical Trial ◽  
Randomized Clinical Trial ◽  
Neuromyelitis Optica ◽  
Spectrum Disorder ◽  
Neuromyelitis Optica Spectrum Disorder

scholarly journals The impact of the COVID-19 pandemic on hospitalizations and plasmapheresis therapy in multiple sclerosis and neuromyelitis optica spectrum disorder: a nationwide analysis from Germany

2021 ◽  
Vol 14 ◽  
pp. 175628642110306
Author(s):  
Daniel Richter ◽  
Simon Faissner ◽  
Dirk Bartig ◽  
Lars Tönges ◽  
Kerstin Hellwig ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Neuromyelitis Optica ◽  
Demyelinating Disease ◽  
Substantial Reduction ◽  
Spectrum Disorder ◽  
Administrative Database ◽  
Main Diagnosis ◽  
Neuromyelitis Optica Spectrum Disorder ◽  
Cross Sectional ◽  
The Impact

Background: Many countries worldwide reported side effects of the coronavirus disease 2019 (COVID-19) pandemic that have influenced the care of patients with other diseases in both acute and elective settings. Patients with multiple sclerosis (MS) and neuromyelitis optica spectrum disorder (NMOSD) represent the major patient population suffering from an autoimmune inflammatory demyelinating disease of the central nervous system. We aimed to analyze MS and NMOSD hospitalizations, the application of plasmapheresis therapy, and the dynamic during different periods of the COVID-19 pandemic in Germany. Methods: We conducted a nationwide retrospective cross-sectional study using the administrative database of all hospitalized patients with the main diagnosis of MS and NMOSD, including the information on the application of plasmapheresis therapy. We included full-year data from 1463 hospitals of all MS and NMOSD patients hospitalized in 2019 and 2020 in Germany ( n = 87,453). We compared case numbers and plasmapheresis therapy rates of the different pandemic periods in 2020 with the corresponding periods in 2019. Results: We observed a substantial decline of MS and NMOSD patients’ hospitalizations during the different pandemic periods, with the most remarkable decline during the first wave of the pandemic (First diagnosis of MS: −16.8%; relapsing-remitting MS: −34.0%; secondary progressive MS: −48.9%; primary progressive MS: −43.8%; NMOSD: −19.2%). Treatment rates with plasmapheresis increased for MS and NMOSD patients in 2020 compared to 2019 (1.8% versus 1.6%, p = 0.003; 14.0% versus 9.3%, p < 0.001), with a substantial increase during the first wave of the pandemic, especially in NMOSD patients (19.7% versus 8.4%, p < 0.001). Conclusion: There was a marked decline of MS and NMOSD patients’ hospitalizations during the different pandemic periods in 2020, with the most substantial reduction during the pandemic’s first wave and in progressive MS patients. MS and NMOSD patients who needed rescue relapse treatment continued to receive plasmapheresis therapy in Germany.

scholarly journals 488 The impact of education on novel concepts in metastatic melanoma: triplet therapy

2020 ◽  
Vol 8 (Suppl 3) ◽  
pp. A524-A524
Author(s):  
Kinjal Parikh ◽  
Sara Fagerlie ◽  
Patrick Kugel ◽  
Richard Caracio ◽  
Ryan Sullivan
Keyword(s):  
Clinical Trial ◽  
Checkpoint Inhibitors ◽  
Panel Discussion ◽  
Clinical Trial Data ◽  
Advanced Melanoma ◽  
Trial Data ◽  
Educational Activity ◽  
List Type ◽  
The Impact ◽  
Post Assessment

BackgroundAdvanced melanoma treatment selection is guided by BRAF-mutation status and patient and disease-specific factors. Historically, oncologists decided between targeted therapy or immune checkpoint inhibitors (ICI). However, given the differences in onset of activity, response durability, and adverse events combination BRAF/MEK inhibitors and ICI (triplet therapy) are being evaluated to optimize outcomes. With several trials due to report, oncologists need education to stay up-to-date on the available data and contextualize this potential treatment option.MethodsAn online continuing education (CME) activity consisting of a multi-media 30-minute video panel discussion explored the rationale, available clinical trial data, and future directions of triplet therapy for the treatment of advanced BRAF-mutated melanoma. Educational effect was assessed using a repeated pairs pre-assessment/post-assessment study design and compared the pre- and post-assessment responses. A chi-square test was used to identify differences between pre- and post-assessment responses. Effect size was calculated using Cramer’s V test by determining the strength of the association between the activity and the outcomes (V = 0.16–0.26 is considerable and V > 0.26 is extensive). P values were calculated and those < 0.05 were considered statistically significant. Data from oncologist participants were collected between 12/23/2019 through 2/26/20.ResultsParticipation in education resulted in statistically significant improvements and noticeable educational effect for oncologists (n=49; p < 0.05, V =0.136). • 39% of pre-assessment questions were correctly answered increasing to 52% post-assessment • 15% of oncologists had a measurable improvement in confidence regarding the rationale for use of triplet therapy in advanced melanoma• Significant improvement in knowledge regarding clinical trial data in triplet therapy was observed (35% vs. 55%; p < 0.05, V = 0.205)ConclusionsThis online, interactive, expert-led, CME-certified educational activity resulted in significant gains in oncologist knowledge and confidence regarding triplet therapy in the management of melanoma. These results demonstrate the effectiveness of on-demand education but also highlight an ongoing need for education on this topic as further data becomes available.AcknowledgementsThis educational initiative was supported through educational grants from Novartis Pharmaceuticals Corporation and GenentechReferenceSullivan RJ, Salama AKS. Managing Melanoma: Emerging Concepts of Triplet Therapy. https://www.medscape.org/viewarticle/923003

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