scholarly journals The ALPHA Project: Establishing consensus and prioritisation of global community recommendations to address major challenges in lupus diagnosis, care, treatment and research

2021 ◽  
Vol 8 (1) ◽  
pp. e000433
Author(s):  
Karin Tse ◽  
Sanjyot Sangodkar ◽  
Lauren Bloch ◽  
Kathleen Arntsen ◽  
Sang-Cheol Bae ◽  
...  
Keyword(s):  
Drug Development ◽  
Access To Care ◽  
Clinical Care ◽  
Media Access ◽  
Global Consensus ◽  
Outcomes Measures ◽  
Care Treatment ◽  
Adults And Children ◽  
Community Recommendations

The Addressing Lupus Pillars for Health Advancement (ALPHA) Project is a global consensus effort to identify, prioritise and address top barriers in lupus impacting diagnosis, care, treatment and research. To conduct this process, the ALPHA Project convened a multistakeholder Global Advisory Committee (GAC) of lupus experts and collected input from global audiences, including patients. In phase I, the ALPHA Project used expert interviews and a global survey of lupus experts to identify and categorise barriers into three overarching pillars: drug development, clinical care and access to care. In phase II, reported here, the GAC developed recommended actionable solutions to address these previously identified barriers through an in-person stakeholder meeting, followed by a two-round scoring process. Recommendations were assessed for feasibility, impact and timeline for implementation (FIT), where potential FIT component values were between 1 and 3 and total scores were between 3 and 9. Higher scores represented higher achievability based on the composite of the three criteria. Simplifying and standardising outcomes measures, including steroid sparing as an outcome (drug development) and defining the lupus spectrum (clinical care) ranked as the highest two priority solutions during the GAC meeting and received high FIT scores (7.67 and 7.44, respectively). Leveraging social media (access to care) received the highest FIT score across all pillars (7.86). Cross-cutting themes of many solutions include leveraging digital technology and applying specific considerations for special populations, including paediatrics. Implementing the recommendations to address key barriers to drug development, clinical care and access to care is essential to improving the quality of life of adults and children with lupus. Multistakeholder collaboration and guidance across existing efforts globally is warranted.

scholarly journals Global consensus building and prioritisation of fundamental lupus challenges: the ALPHA project

2019 ◽  
Vol 6 (1) ◽  
pp. e000342 ◽  
Author(s):  
Susan Manzi ◽  
Sandra Raymond ◽  
Karin Tse ◽  
Yaritza Peña ◽  
Annick Anderson ◽  
...  
Keyword(s):  
Drug Development ◽  
Clinical Care ◽  
Treatment Options ◽  
Clinical Trial Design ◽  
Expert Interviews ◽  
Long Term Outcomes ◽  
Global Consensus ◽  
Autoimmune Conditions ◽  
The Continuum

ObjectiveLupus is a complex, heterogeneous autoimmune disease that has yet to see significant progress towards more timely diagnosis, improved treatment options for short-term and long-term outcomes, and appropriate access to care. The Addressing Lupus Pillars for Health Advancement (ALPHA) project is the first step in establishing global consensus and developing concrete strategies to address the challenges limiting progress.MethodsA Global Advisory Committee of 13 individuals guided the project and began barrier identification. Seventeen expert interviews were conducted to further characterise key barriers. Transcripts were analysed using Nvivo and a codebook was created containing a list of thematic ‘nodes’ (topics) and their descriptions. Findings were used to develop a final survey instrument that was fielded to a diverse, international stakeholder audience to achieve broad consensus.ResultsExpert interviews identified lupus heterogeneity as the primary barrier hindering advancement. Subsequent barriers were categorised into three areas: (1) Drug development. (2) Clinical care. (3) Access and value. The global survey received 127 completed responses from experts across 20 countries. Respondents identified barriers as high priority including the lack of biomarkers for clinical and drug development use, flawed clinical trial design, lack of access to clinicians familiar with lupus, and obstacles to effective management of lupus due to social determinants of care. Respondents also identified 30 autoimmune conditions that may be lupus-related based on overlapping features, shared autoantibodies and pathophysiology.ConclusionsALPHA is a comprehensive initiative to identify and prioritise the continuum of challenges facing people with lupus by engaging a global audience of lupus experts. It also explored views on lupus as a spectrum of related diseases. Conclusions from this effort provide a framework to generate actionable approaches to the identified high-priority barriers.

scholarly journals IGDA. 9: Linking diagnosis to care – treatment planning

2003 ◽  
Vol 182 (S45) ◽  
pp. s58-s59 ◽  
Author(s):  
Keyword(s):  
Quality Of Life ◽  
Treatment Planning ◽  
Clinical Care ◽  
Diagnostic Interview ◽  
Care Treatment ◽  
Clinical Disorders ◽  
Comprehensive Diagnosis ◽  
Clinical Problems

Clinical care starts with the first diagnostic interview. Therapeutic planning and prognosis should be based on competently conducted and documented comprehensive diagnosis, i.e. a standardised multi-axial formulation covering clinical disorders, disabilities, contextual problems and quality of life, as well as an idiographic or personalised formulation articulating the perspectives of the clinician with those of the patient and family on contextualised clinical problems, the patient's positive factors, and expectations about restoration and promotion of health.

Gastroesophageal Reflux Disease: A Gastroenterologist's Perspective

2011 ◽  
Vol 21 (3) ◽  
pp. 89-99
Author(s):  
Michael F. Vaezi
Keyword(s):  
Gastroesophageal Reflux Disease ◽  
Gastroesophageal Reflux ◽  
Reflux Disease ◽  
Best Practice ◽  
Clinical Care ◽  
Diagnostic Testing ◽  
Diagnostic Tools ◽  
Swallowing Function ◽  
Atypical Symptoms

Gastroesophageal reflux disease (GERD) is a commonly diagnosed condition often associated with the typical symptoms of heartburn and regurgitation, although it may present with atypical symptoms such as chest pain, hoarseness, chronic cough, and asthma. In most cases, the patient's reduced quality of life drives clinical care and diagnostic testing. Because of its widespread impact on voice and swallowing function as well as its social implications, it is important that speech-language pathologists (SLPs) understand the nature of GERD and its consequences. The purpose of this article is to summarize the nature of GERD and GERD-related complications such as GERD-related peptic stricture, Barrett's esophagus and adenocarcinoma, and laryngeal manifestations of GERD from a gastroenterologist's perspective. It is critical that SLPs who work with a multidisciplinary team understand terminology, diagnostic tools, and treatment to ensure best practice.

Treatment of painful bone metastases in prostate and breast cancer patients with the therapeutic radiopharmaceutical rhenium-188-HEDP

2016 ◽  
Vol 55 (05) ◽  
pp. 188-195 ◽  
Author(s):  
Floor Overbeek ◽  
John de Klerk ◽  
Pieternel Pasker-de Jong ◽  
Alexandra van den Berk ◽  
Rob ter Heine ◽  
...  
Keyword(s):  
Bone Metastases ◽  
Clinical Benefit ◽  
Response Rate ◽  
Clinical Care ◽  
Pain Response ◽  
Pain Palliation ◽  
Breast Cancer Patients ◽  
Routine Clinical Care ◽  
Painful Bone Metastases

Summary Aim: Rhenium-188-HEDP (188Re-HEDP) is an effective radiopharmaceutical for the palliative treatment of osteoblastic bone metastases. However, only limited data on its routine use are available and its effect on quality of life (QoL) has not been studied. Therefore, we evaluated the clinical benefit of 188Re-HEDP in routine clinical care. Patients and methods: Prostate or breast cancer patients with painful bone metastases receiving 188Re-HEDP as a routine clinical procedure were eligible for evaluation. Clinical benefit was assessed in terms of efficacy and toxicity. Pain palliation and QoL were monitored using the visual analogue scale (VAS), corrected for opioid intake, and the EORTC QLQ-C30 Global health status/QoL-scale. Thrombocyte and leukocyte nadirs were used to assess haematological toxicity. Results: 45 and 47 patients were evaluable for pain palliation and QoL, respectively. After a single injection of 188Re-HEDP, the overall pain response rate was 69% and mean VAS-scores decreased relevantly and significantly (p < 0.05). Repeated treatment resulted in similar pain response. The overall QoL response rate was 68% and mean Global health status/QoL-scores increased relevantly and significantly. Haematological side effects were mild and transient. Conclusion: The clinically relevant response on pain and quality of life and the limited adverse events prove clinical benefit of treatment with 188Re-HEDP and support its use in routine clinical care. Its effectiveness appears comparable to that of external beam radiotherapy.

Quality of Life for Children with Persistent Sinonasal Symptoms

2003 ◽  
Vol 128 (1) ◽  
pp. 17-26 ◽  
Author(s):  
David J. Kay ◽  
Richard M. Rosenfeld
Keyword(s):  
Quality Of Life ◽  
Clinical Care ◽  
Longitudinal Change ◽  
Good Test ◽  
Routine Clinical Care ◽  
Before And After ◽  
The Mean ◽  
The Individual ◽  
Sinonasal Symptoms

OBJECTIVE: The goal was to validate the SN-5 survey as a measure of longitudinal change in health-related quality of life (HRQoL) for children with persistent sinonasal symptoms. DESIGN AND SETTING: We conducted a before and after study of 85 children aged 2 to 12 years in a metropolitan pediatric otolaryngology practice. Caregivers completed the SN-5 survey at entry and at least 4 weeks later. The survey included 5 symptom-cluster items covering the domains of sinus infection, nasal obstruction, allergy symptoms, emotional distress, and activity limitations. RESULTS: Good test-retest reliability ( R = 0.70) was obtained for the overall SN-5 score and the individual survey items ( R ≥ 0.58). The mean baseline SN-5 score was 3.8 (SD, 1.0) of a maximum of 7.0, with higher scores indicating poorer HRQoL. All SN-5 items had adequate correlation ( R ≥ 0.36) with external constructs. The mean change in SN-5 score after routine clinical care was 0.88 (SD, 1.19) with an effect size of 0.74 indicating good responsiveness to longitudinal change. The change scores correlated appropriately with changes in related external constructs ( R ≥ 0.42). CONCLUSIONS: The SN-5 is a valid, reliable, and responsive measure of HRQoL for children with persistent sinonasal symptoms, suitable for use in outcomes studies and routine clinical care.

scholarly journals Access to care and worsening eating disorder symptomatology in youth during the COVID-19 pandemic

2021 ◽  
Vol 9 (1) ◽  
Author(s):  
Rebecca Spigel ◽  
Jessica A. Lin ◽  
Carly E. Milliren ◽  
Melissa Freizinger ◽  
Julia A. Vitagliano ◽  
...  
Keyword(s):  
Quality Of Care ◽  
Eating Disorder ◽  
Access To Care ◽  
Treatment Quality ◽  
Related Factors ◽  
Web Based ◽  
Face To Face ◽  
Recovery Study ◽  
And Behaviors

Abstract Background Shelter-in-place orders and social distancing guidelines, in response to the COVID-19 pandemic, have limited traditional face-to-face interactions and led to many clinical providers transitioning to the use of videoconferencing platforms. The present study aims to assess how the COVID-19 pandemic has impacted adolescents’/young adults’ (AYA) eating disorder (ED)-related care, and how access to, changes in, perceived disruptions to, and quality of care are associated with ED thoughts and behaviors. Methods AYA enrolled in the RECOVERY study, a pre-existing web-based longitudinal study, and completed a COVID-19-specific survey (n = 89). We examined bivariate associations of four markers of care: i) access to care, ii) changes in care, iii) perceived disruption to care, and iv) quality of care. Using multiple logistic regression, we examined the associations of pandemic-related markers of care with changes in ED thoughts and behaviors. We excluded those not engaged in treatment pre-pandemic (n = 16). Results In the remaining 73 participants, reported access to care was high, with 92% of respondents continuing care with at least one ED provider during the pandemic; however, 47% stopped some treatment during the pandemic. Nearly one-third (32%) perceived a disruption in treatment. Quality of care remained high with 67% reporting care to be better than or as good as pre-pandemic. Respondents acknowledged heightened symptomatology: 81% reported increased ED thoughts and 81% reported increased ED behaviors due to COVID-19-related factors. However, none of the markers of care described were significantly associated with ED thoughts or behaviors in regression analyses adjusting for demographic variables and baseline characteristics, except our quality of care measure which was approaching significance (p = 0.07). Conclusions Our findings show the majority of AYA who had care prior to the pandemic continued receiving some element of their multi-disciplinary ED treatment and perceived their care as high quality. None of the markers of care described were statistically associated with increased ED thoughts and behaviors.

scholarly journals OP0277-PARE METABERN, THE EUROPEAN REFERENCE NETWORK FOR RARE HEREDITARY DISEASES: STRUCTURE, OBJECTIVES, METABOLIC RMDS AND THE ROLES OF EUROPEAN PATIENT ADVOCACY GROUPS (EPAGS)

2021 ◽  
Vol 80 (Suppl 1) ◽  
pp. 168.2-168
Author(s):  
L. Wagner ◽  
S. Sestini ◽  
C. Brown ◽  
A. Finglas ◽  
R. Francisco ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Health Care ◽  
Social Science ◽  
Metabolic Pathways ◽  
Metabolic Disorders ◽  
Clinical Care ◽  
Single Point ◽  
Link Type ◽  
The Impact

Background:Inborn metabolic disorders (IMDs) currently encompass more than 1,500 diseases with new ones still to be identified1. Each of them is characterised by a genetic defect affecting a metabolic pathway. Only few of them have curative treatments, that target the respective metabolic pathway. Commonly, treatment examples include diet, substrate reduction therapies, enzyme replacement therapies, gene therapy and biologicals, enabling IMD-patient now to survive to adulthood. About 30 % of all IMDs involve the musculoskeletal system and are here referred to as rare metabolic RMDs. Generally, IMDs are very heterogenous with respect to symptoms and severity, often being systemic and affecting more children than adults. Thus, challenges include certified advanced training of adult metabolic experts, standardised transition plans, social support and development of therapies for diseases that do not have any cure yet.Objectives:Introduction of MetabERN, its structure and objectives, highlighting on the unique features and challenges of metabolic RMDs and describing the involvement of patient representation in MetabERN.Methods:MetabERN is stratified in 7 subnetworks (SNW) according to the respective metabolic pathways and 9 work packages (WP), including administration, dissemination, guidelines, virtual counselling framework, research/clinical trials, continuity of care, education and patient involvement. The patient board involves a steering committee and single point of contacts for each subnetwork and work package, respectively2. Projects include identifying the need of implementing social science to assess the psycho-socio-economic burden of IMDs, webinars on IMDs and their transition as well as surveys on the impact of COVID-193 on IMD-patients and health care providers (HCPs), social assistance for IMD-patients and analysing the transition landscape within Europe.Results:The MetabERN structure enables bundling of expertise, capacity building and knowledge transfer for faster diagnosis and better health care. Rare metabolic RMDs are present in all SNWs that require unique treatments according to their metabolic pathways. Implementation of social science to assess the psycho-socio-economic burden of IMDs is still underused. Involvement of patient representatives is essential for a holistic healthcare not only focusing on clinical care, but also on the quality of life for IMD-patients. Surveys identified unmet needs of patient care, patients having little information on national support systems and structural deficits of healthcare systems to ensure HCP can provide adequate clinical care during transition phases. These results are collected by MetabERN and forwarded to the Directorate-General for Health and Food Safety (DG SANTE) of the European Commission (EC) to be addressed further.Conclusion:MetabERN offers an infrastructure of virtual healthcare for patients with IMDs. Thus, in collaboration with ERN ReCONNET, MetabERN can assist in identifying rare metabolic disorders of RMDs to shorten the odyssey of diagnosis and advise on their respective therapies. On the other hand, MetabERN can benefit from EULAR’s longstanding experience regarding issues affecting the quality of life, all RMD patients are facing, such as pain, stiffness, fatigue, rehabilitation, maintaining work and disability claims.References:[1]IEMbase - Inborn Errors of Metabolism Knowledgebase http://www.iembase.org/ (accessed Jan 29, 2021).[2]MetabERN: European Refence Network for Hereditary Metabolic Disorders https://metab.ern-net.eu/ (accessed Jan 29, 2021).[3]Lampe, C.; Dionisi-Vici, C.; Bellettato, C. M.; Paneghetti, L.; van Lingen, C.; Bond, S.; Brown, C.; Finglas, A.; Francisco, R.; Sestini, S.; Heard, J. M.; Scarpa, M.; MetabERN collaboration group. The Impact of COVID-19 on Rare Metabolic Patients and Healthcare Providers: Results from Two MetabERN Surveys. Orphanet J. Rare Dis.2020, 15 (1), 341. https://doi.org/10.1186/s13023-020-01619-x.Acknowledgements:The authors thank the MetabERN collaboration group, the single point of contacts (SPOC) of the MetabERN patient board and the Transition Project Working Group (TPWG)Disclosure of Interests:None declared

scholarly journals A Physical Activity Intervention Feasibility Study for Kidney Inpatients: A Basic Research Protocol

2021 ◽  
Vol 8 ◽  
pp. 205435812098705
Author(s):  
Kathryn Wytsma-Fisher ◽  
Stefan Mustata ◽  
Theresa Cowan ◽  
Manuel Ester ◽  
S. Nicole Culos-Reed
Keyword(s):  
Quality Of Life ◽  
Physical Activity ◽  
Physical Function ◽  
Kidney Failure ◽  
Standard Care ◽  
Physical Activity Intervention ◽  
Clinical Care ◽  
Activity Promotion ◽  
Cardiovascular Morbidity And Mortality

Background: Low physical activity levels and poor physical functioning are strongly associated with poor clinical outcomes and mortality in adult kidney failure patients, regardless of treatment modality. Compared with the general population, individuals with chronic kidney disease are physically inactive, have reduced physical abilities and difficulties performing routine daily tasks, lower health-related quality of life, and higher cardiovascular morbidity and mortality. In addition, frail kidney failure patients have higher hospitalization and mortality rates as compared with other kidney failure patients. Evidence suggests that assessment and recommendations for physical activity should be part of standard care for kidney failure patients. Structured exercise can improve physical function and quality of life in frail older adults and may be used specifically for management of frailty in kidney failure. However, research is needed to determine best practices for implementation of physical function measurements and physical activity promotion in standard kidney failure care. Objective: The proposed Move More study will assess the feasibility of a physical activity intervention offered to the kidney failure inpatients in Calgary, Alberta. Specifically, this study is designed to examine the effects of an early physical activity/mobility intervention led by a kinesiologist, and supported by the clinical care team including physiotherapists (PT) and nurse clinicians. Methods: The Move More study is a single-arm pilot intervention examining feasibility and optimal improvement in real-world conditions. Kidney failure inpatients at the Foothills Medical Centre will be recruited to participate. Patients will receive an individualized in-hospital physical activity/mobility intervention. Frailty and physical function will be assessed at baseline and postintervention prior to hospital discharge. The goal is to recruit 24 to 36 patients. Conclusions: Evidence needed to support the inclusion of mobility and physical activity as part of standard care will be gathered, with knowledge gained used to help direct future physical activity programming for kidney failure inpatients.

scholarly journals Standardized Outcomes Measures in Physical Therapy Practice for Treatment and Rehabilitation of Cerebral PALSY: A Systematic Review

2021 ◽  
Vol 11 (7) ◽  
pp. 604
Author(s):  
Maria Dolores Apolo-Arenas ◽  
Aline Ferreira de Araújo Jerônimo ◽  
Alejandro Caña-Pino ◽  
Orlando Fernandes ◽  
Joana Alegrete ◽  
...  
Keyword(s):  
Cerebral Palsy ◽  
Physical Therapy ◽  
Gait Training ◽  
Physical Therapy Modalities ◽  
Spastic Diplegia ◽  
Evaluation Procedures ◽  
Outcomes Measures ◽  
Prisma Statement ◽  
Spastic Hemiplegia

Cerebral palsy (CP) treatment includes physical therapy and various complementary therapies to the standard clinical treatment. However, there are not many reviews that focus on the methods used and evaluation procedures. This study aims to analyze which tools are most suitable for the evaluation and methodology of patients with CP treated with physical therapy. Following the PRISMA statement, through a PICOS strategy, PubMed/MEDLINE, Web of Science (WOS), Scopus, Science Direct, and Scielo were searched with the following terms: cerebral palsy AND (physical therapy modalities OR therapeutics) AND outcome assessment. The methodological quality of the RCTs was assessed with the Evidence Project risk of bias tool. Thirty-seven RCTs and six RCT protocols, comprising 1359 participants with different types of CP: spastic hemiplegia/paresis, spastic diplegia/paresis, and spastic CP, met the inclusion criteria, uncovering 21 variables measured through 77 different instruments and several interventions. The therapies most widely used in CP are gaming or technology-assisted therapies, aerobic training, hippotherapy, music therapy, gait training, and aquatic exercises. This study provides an overview of what the authors used in the neurorehabilitation field through procedure evaluation and checking the technological advance that began to be used.

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