Summary Duchenne muscular dystrophy (DMD) is a severe, progressive disease that affects 1 in 3600–6000 live male births. Although guidelines are available for various aspects of DMD, comprehensive ...clinical care recommendations do not exist. The US Centers for Disease Control and Prevention selected 84 clinicians to develop care recommendations using the RAND Corporation–University of California Los Angeles Appropriateness Method. The DMD Care Considerations Working Group evaluated assessments and interventions used in the management of diagnostics, gastroenterology and nutrition, rehabilitation, and neuromuscular, psychosocial, cardiovascular, respiratory, orthopaedic, and surgical aspects of DMD. These recommendations, presented in two parts, are intended for the wide range of practitioners who care for individuals with DMD. They provide a framework for recognising the multisystem primary manifestations and secondary complications of DMD and for providing coordinated multidisciplinary care. In part 1 of this Review, we describe the methods used to generate the recommendations, and the overall perspective on care, pharmacological treatment, and psychosocial management.
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Recently, the number of projects has grown connected with the application of high-temperature superconductors (HTSC) with exceptionally high current-carrying characteristics in strong magnetic ...fields. However, the thickness of the RBa
2
Cu
3
O
7–
x
(R = REE, Y) superconducting layer is only 1–2% of the thickness of HTSC tape. Increasing the current-carrying characteristics owing to the thickness of the conducting layer is a promising approach. The fundamental problem of crystallite formation of
a-
oriented crystallites prevents the development of this approach. Such crystallites do not carry superconducting current along the substrate tape and interfere with the growth of
c-
oriented crystallites. This review presents existing methods of suppression of
a
-oriented growth, the basis of the creation of HTSC materials, and prospects of obtaining HTSC materials with high current-carrying characteristics.
Introduction
This study establishes research priorities for Medically Not Yet Explained Symptoms (MNYES). A significant number of patients suffer from these symptoms, also known as MUS, that are ...likely to cause work disability and impact on quality of life. Research into MNYES in general has been poorly funded over the years, has been primarily researcher-led, and was sometimes controversial.
Objectives
To identify research priorities from the perspective of patients, caregivers and clinicians, following the James Lind Alliance (JLA) priority setting partnership (PSP) method.
Methods
The PSP Steering Group termed these symptoms Medically Not Yet Explained Symptoms (MNYES). This was an operational definition not intended to add to or replace other definitions already in use, that was constructed to embrace the views of all stakeholders. The nomenclature MNYES was chosen to indicate our incomplete understanding of these conditions. This could pertain to biological, psychological and social factors, as well as factors involving the trajectory of patients through various healthcare settings.
The study involved five key stages: defining the appropriate term for the conditions under study by the PSP Steering Group; gathering questions on MNYES from patients, caregivers and clinicians in a publicly accessible survey; checking these research questions against existing evidence; interim prioritisation in a second survey; and a final multi-stakeholder consensus meeting to determine the top 10 unanswered research questions using the modified nominal group methodology.
Results
Over 700 responses from UK patients, caregivers and clinicians were identified in two surveys from a broad range of medical specialities and primary care. Patients prioritised research questions regarding diagnosis and aetiology; clinicians and caregivers prioritised outcomes and treatment, relatively.
The top 10 unanswered research questions cover the domains of: treatment; the role of clinicians; symptoms and outcomes; and recovery.
Conclusions
This JLA PSP may well be the first attempt at capturing the thoughts of a wide group of medical professionals, patients and caregivers in one place with the aim eventually of standardising care and reducing unhelpful variability in the management of MNYES. Following the JLA approach is a strength of the study. The choice of the term MNYES conveys a message of hope, which responds to a need identified by patients, carers and clinicians alike for vigorous research in this domain. The research priorities are expected to generate much-needed, relevant and impactful research into MNYES. Better funding possibilities for MNYES are urgently needed.
Disclosure of Interest
None Declared
Background
Primary and secondary non-response to anti-tumor necrosis factor (TNF) therapy is common in patients with Crohn’s disease (CD), yet limited research has compared the effectiveness of ...subsequent biological therapy.
Objective
We sought to compare the effectiveness of vedolizumab and ustekinumab in anti-TNF-experienced patients with CD, focusing on patient-prioritized patient-reported outcomes (PROs).
Methods
We conducted a prospective, internet-based cohort study nested within IBD Partners. We identified anti-TNF-experienced patients initiating with CD vedolizumab or ustekinumab and analyzed PROs reported approximately 6 months later (minimum 4 months, maximum 10 months). Co-primary outcomes were Patient-Reported Outcome Measurement Information System (PROMIS) domains of Fatigue and Pain Interference. Secondary outcomes included patient-reported short Crohn’s disease activity index (sCDAI), treatment persistence, and corticosteroid use. Inverse probability of treatment weighting (IPTW) was used to control for a number of potential confounders and incorporated into linear and logistic regression models for continuous and categorical outcomes, respectively.
Results
Overall, 141 vedolizumab and 219 ustekinumab initiators were included in our analysis. After adjustment, we found no differences between treatment groups in our primary outcomes of Pain Interference or Fatigue or the secondary outcome of sCDAI. However, vedolizumab was associated with lower treatment persistence (OR 0.4, 95% CI 0.2–0.6) and higher corticosteroid use at follow-up assessment (OR 1.7, 95% CI 1.1–2.6).
Discussion
Among anti-TNF experienced patients with CD, Pain Interference or Fatigue was not significantly different 4–10 months after starting ustekinumab or vedolizumab. However, reduced steroid use and increased persistence suggest superiority of ustekinumab for non-PRO outcomes.