CureSCi Metadata Catalog – Making sickle cell studies findable

Objectives: To adopt the FAIR principles (Findable, Accessible, Interoperable, Reusable) to enhance data sharing, the Cure Sickle Cell Initiative (CureSCi) MetaData Catalog (MDC) was developed to make Sickle Cell Disease (SCD) study datasets more Findable by curating study metadata and making them available through an open-access web portal. Methods: Study metadata, including study protocol, data collection forms, and data dictionaries, describe information about study patient-level data. We curated key metadata of 16 SCD studies in a three-tiered conceptual framework of category, subcategory, and data element using ontologies and controlled vocabularies to organize the study variables. We developed the CureSCi MDC by indexing study metadata to enable effective browse and search capabilities at three levels: study, Patient-Reported Outcome (PRO) Measures, and data element levels. Results: The CureSCi MDC offers several browse and search tools to discover studies by study level, PRO Measures, and data elements. The “Browse Studies,” “Browse Studies by PRO Measures,” and “Browse Studies by Data Elements” tools allow users to identify studies through pre-defined conceptual categories. “Search by Keyword” and “Search Data Element by Concept Category” can be used separately or in combination to provide more granularity to refine the search results. This resource helps investigators find information about specific data elements across studies using public browsing/search tools, before going through data request procedures to access controlled datasets. The MDC makes SCD studies more Findable through browsing/searching study information, PRO Measures, and data elements, aiding in the reuse of existing SCD data.

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End points for sickle cell disease clinical trials: renal and cardiopulmonary, cure, and low-resource settings

Blood Advances ◽

10.1182/bloodadvances.2019000883 ◽

2019 ◽

Vol 3 (23) ◽

pp. 4002-4020 ◽

Cited By ~ 3

Author(s):

Ann T. Farrell ◽

Julie Panepinto ◽

Ankit A. Desai ◽

Adetola A. Kassim ◽

Jeffrey Lebensburger ◽

...

Keyword(s):

Sickle Cell Disease ◽

Sickle Cell ◽

Patient Reported Outcome ◽

Cell Disease ◽

End Points ◽

Low Resource Settings ◽

Low Resource ◽

Patient Reported ◽

The Us ◽

American Society

Abstract To address the global burden of sickle cell disease and the need for novel therapies, the American Society of Hematology partnered with the US Food and Drug Administration to engage the work of 7 panels of clinicians, investigators, and patients to develop consensus recommendations for clinical trial end points. The panels conducted their work through literature reviews, assessment of available evidence, and expert judgment focusing on end points related to patient-reported outcome, pain (non–patient-reported outcomes), the brain, end-organ considerations, biomarkers, measurement of cure, and low-resource settings. This article presents the findings and recommendations of the end-organ considerations, measurement of cure, and low-resource settings panels as well as relevant findings and recommendations from the biomarkers panel.

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A pain relieving reimbursement program? Effects of a value-based reimbursement program on patient reported outcome measures

BMC Health Services Research ◽

10.1186/s12913-020-05578-8 ◽

2020 ◽

Vol 20 (1) ◽

Cited By ~ 1

Author(s):

Thérèse Eriksson ◽

Hans Tropp ◽

Ann-Britt Wiréhn ◽

Lars-Åke Levin

Keyword(s):

Outcome Measures ◽

Patient Reported Outcome Measures ◽

Patient Reported Outcome ◽

Lumbar Spine Surgery ◽

A Value ◽

Level Data ◽

Patient Reported ◽

Before And After ◽

Cherry Picking ◽

Inpatient And Outpatient Care

Abstract Background Value-based reimbursement programs have become increasingly common. However, little is known about the effect of such programs on patient reported outcomes. Thus, the aim of this study was to analyze the effect of introducing a value-based reimbursement program on patient reported outcome measures and to explore whether a selection bias towards less complicated patients occurred. Methods This is a retrospective observational study with a before and after design based on the introduction of a value-based reimbursement program in Region Stockholm, Sweden. We analyzed patient level data from inpatient and outpatient care of patients undergoing lumbar spine surgery during 2006–2015. Patient reported outcome measures used was Global Assessment, EQ-5D-3L and Oswestry Disability Index. The case-mix of surgically treated patients was analyzed using medical and socioeconomic factors. Results The value-based reimbursement program did not have any effect on targeted or non-targeted patient reported outcome measures. Moreover, the share of surgically treated patients with risk factors such as having comorbidities and being born outside of Europe increased after the introduction. Hence, the value-based reimbursement program did not encourage discrimination against sicker patients. However, the income was higher among patients surgically treated after the introduction of the value-based reimbursement. This indicates that a value-based reimbursement program may contribute to increased inequalities in access to healthcare. Conclusions The value-based reimbursement program did not have any effect on patient reported outcome measures. Our study contributes to the understanding of the effects of a value-based reimbursement program on patient reported outcome measures and to what extent cherry-picking arises.

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PD45-10 VALIDATION STUDY: PATIENT REPORTED OUTCOME MEASURE (PROM) FOR PENILE CURVATURE SURGERY.

The Journal of Urology ◽

10.1016/j.juro.2016.02.2110 ◽

2016 ◽

Vol 195 (4S) ◽

Author(s):

Angus Campbell ◽

RIchard Akiboye ◽

Saheel Mukhtar ◽

Matthew Jackson ◽

Tet Yap ◽

...

Keyword(s):

Validation Study ◽

Outcome Measure ◽

Patient Reported Outcome ◽

Study Patient ◽

Penile Curvature ◽

Patient Reported Outcome Measure ◽

Patient Reported

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A systematic review of the evidence base for vidian neurectomy in managing rhinitis

The Journal of Laryngology & Otology ◽

10.1017/s0022215116008008 ◽

2016 ◽

Vol 130 (S4) ◽

pp. S7-S28 ◽

Cited By ~ 19

Author(s):

T Marshak ◽

W K Yun ◽

C Hazout ◽

R Sacks ◽

R J Harvey

Keyword(s):

Systematic Review ◽

Outcome Measures ◽

Original Data ◽

Patient Reported Outcome Measures ◽

Evidence Base ◽

Patient Reported Outcome ◽

Study Patient ◽

Secondary Outcome ◽

Vidian Neurectomy ◽

Patient Reported

AbstractBackground:Vidian neurectomy has been proposed as a surgical option for rhinitis refractory to medical treatment. However, the evidence base for its benefit remains controversial.Methods:A systematic review was performed. Studies reporting original data on patients with rhinitis treated by vidian neurectomy were included. Patient-reported outcome measures were the primary outcome investigated; specific peri-operative morbidities were the secondary outcome.Results:A total of 1012 articles fulfilled the search criteria, 32 of which were included in the study. Patient-reported outcome measures were compared before and after surgery in eight studies. There were 529 patients represented in these trials. Significant improvement in rhinorrhoea was reported in all eight studies. Temporary dry eyes was reported in 24.63 per cent of cases (272 out of 1104 cases). There was no report of cranial nerve deficit or eye movement disturbance.Conclusion:Endoscopic vidian neurectomy does have a role in the surgical management of refractory rhinitis, particularly in patients with non-allergic rhinitis, but a well-designed cohort trial would be advantageous to clarify long-term outcomes.

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Study Data Element Mapping: Feasibility of Defining Common Data Elements Across COVID-19 Studies

10.1101/2020.05.19.20106641 ◽

2020 ◽

Author(s):

Priscilla Mathewson ◽

Ben Gordon ◽

Kay Snowley ◽

Clara Fennessy ◽

Alastair Denniston ◽

...

Keyword(s):

Outcome Measures ◽

Study Data ◽

Data Element ◽

Common Data Elements ◽

Specific Data ◽

The United Kingdom ◽

Recorded Data ◽

Data Elements ◽

Selection Of ◽

Element Mapping

Background: Numerous clinical studies are now underway investigating aspects of COVID-19. The aim of this study was to identify a selection of national and/or multicentre clinical COVID-19 studies in the United Kingdom to examine the feasibility and outcomes of documenting the most frequent data elements common across studies to rapidly inform future study design and demonstrate proof-of-concept for further subject-specific study data element mapping to improve research data management. Methods: 25 COVID-19 studies were included. For each, information regarding the specific data elements being collected was recorded. Data elements collated were arbitrarily divided into categories for ease of visualisation. Elements which were most frequently and consistently recorded across studies are presented in relation to their relative commonality. Results: Across the 25 studies, 261 data elements were recorded in total. The most frequently recorded 100 data elements were identified across all studies and are presented with relative frequencies. Categories with the largest numbers of common elements included demographics, admission criteria, medical history and investigations. Mortality and need for specific respiratory support were the most common outcome measures, but with specific studies including a range of other outcome measures. Conclusion: The findings of this study have demonstrated that it is feasible to collate specific data elements recorded across a range of studies investigating a specific clinical condition in order to identify those elements which are most common among studies. These data may be of value for those establishing new studies and to allow researchers to rapidly identify studies collecting data of potential use hence minimising duplication and increasing data re-use and interoperability

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Patient-Reported Outcomes Before and After Primary and Revision Total Ankle Arthroplasty

Foot & Ankle International ◽

10.1177/1071100718794956 ◽

2018 ◽

Vol 40 (1) ◽

pp. 34-41 ◽

Cited By ~ 9

Author(s):

James R. Lachman ◽

Jania A. Ramos ◽

Samuel B. Adams ◽

James A. Nunley ◽

Mark E. Easley ◽

...

Keyword(s):

Clinical Outcomes ◽

High Volume ◽

Clinical Results ◽

Patient Reported Outcome ◽

Total Ankle Arthroplasty ◽

Study Patient ◽

Ankle Arthroplasty ◽

Patient Reported ◽

Before And After

Background: Studies examining the clinical outcomes of revision total ankle arthroplasty (TAA) are sparse. Revision TAA surgery has become more common with availability of revision implants and refinement of bone-conserving primary implants. In this study, patient-reported results and clinical outcomes were analyzed for a cohort of patients who underwent both primary and revision TAA at a single high-volume institution. Methods: We retrospectively reviewed prospectively collected data on 29 patients with failed primary total ankle arthroplasty. Cases of isolated polyethylene exchange, infection, or extra-articular realignment procedures were excluded. Patient-reported outcome (PRO) measures and clinical results were reviewed in this longitudinal study. Results: Fifteen patients (51.7%) underwent revision of just the talar and polyethylene components while 13 patients (44.8%) underwent revision of all components. The most common cause was talar subsidence (51.7%). The average time to revision was 3.9 years with a follow-up of 3.2 years after revision, and 3 (10.3%) revision arthroplasties required further surgery; 2 required conversion to arthrodesis and 1 required second revision TAA. Improvements in PROs were better after primary than revision TAA. Conclusions: Clinical and patient-reported results of revision ankle arthroplasty after metal component failure improved significantly but never reached the improvements seen after primary ankle arthroplasty. In our series, 10.3% of revision TAAs required a second revision TAA or arthrodesis surgery. Levels of Evidence: Therapeutic Level III, comparative series.

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The influence of patient engagement on the design and implementation of a clinical trial to improve cancer care delivery.

Journal of Clinical Oncology ◽

10.1200/jco.2018.36.30_suppl.223 ◽

2018 ◽

Vol 36 (30_suppl) ◽

pp. 223-223

Author(s):

Sarah Barger ◽

Sean D Sullivan ◽

Gary H. Lyman ◽

Dawn L. Hershman ◽

Ari Bell-Brown ◽

...

Keyword(s):

Clinical Trial ◽

Care Delivery ◽

Patient Reported Outcome ◽

Study Patient ◽

Advisory Group ◽

Patient Centeredness ◽

Patient Centered ◽

Implementation Challenges ◽

Patient Reported ◽

Study Team

223 Background: We have engaged 10 patient partners in the development and implementation of S1415CD, a five-year pragmatic clinical trial currently in year 3 assessing the effectiveness of a guideline-based colony stimulating factor standing order intervention (NCT02728596). Patient partners serve as part of a 21-person External Stakeholder Advisory Group (ESAG), which also includes providers, payers and guidelines experts. This abstract explores the influence of patient partners on the design, tools and implementation of S1415CD Methods: Patient partners advise the study team on protocol development, patient-facing materials and implementation challenges over four teleconferences each year, annual in-person meetings and targeted email communication. All patient partner input from 2014-2017 was tracked, collected and reviewed for impact on the trial. Results: Input from patient partners led to the refinement of the study’s patient-reported outcome (PRO) survey questions, the creation of a highly utilized patient brochure, and the formation of talking points for clinic staff to help explain the study. Patient partners in conjunction with high performing sites helped develop strategies for sites with lower patient accrual to optimize the approach and consent of study participants. Conclusions: The sustained engagement of patient partners in S1415CD ensured patient-centeredness in trial design and guided the development of PRO surveys and relevant, high quality patient-facing materials. Drawing on experiential knowledge and insights from their roles as caregivers and advocates, patient partners provided valuable feedback that influenced patient approach and engagement in the study. Embedding patient partners in the research continuum has catalyzed critical discussions and problem solving among the patient partners and study team, which has led to patient-centered solutions to study challenges. Clinical trial information: NCT02728596.

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