Who to screen for calreticulin mutations? An audit of real-life practice and review of current evidence

Introduction and aims Treatment for childhood and adolescent brain tumours is often intensive, with significant neurocognitive and psycho-social late effects ( Zeltzer et al., 2009 ). This feasibility Study aimed to inform the development of a cognitive rehabilitation and psycho-social group intervention for Teenage and Young Adult (TYA) survivors of brain tumours. Methods A group-based intervention incorporated ideas from the current evidence base, including psychoeducation and compensatory strategy training, with a focus on real-life goals and improving quality of life. Participants ( N = 19, 13–24 years) were recruited from the University College London Hospital TYA Oncology Service. Participants had received treatment for a malignant brain tumour and had completed their treatment at least 1 year prior to participation. Four group-based, whole-day interventions ran every 3 months throughout a year. Feasibility criteria were established to answer questions about acceptability of the intervention and recruitment. Results Qualitative and quantitative feedback from all four groups demonstrated acceptability and suitability of the intervention with regards to the content, structure and delivery. Recruitment presented more of a challenge with 35% fewer referrals than expected. Discussion and conclusion Feedback suggests that the intervention is suitable and acceptable, whilst limitations include numbers of referrals and referral pathways. Future directions are discussed.

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P282 Outcome for PsA biologic switchers following primary inefficacy of TNFi therapy in a secondary care cohort: sequential TNFi versus switching mode of action

Rheumatology ◽

10.1093/rheumatology/keaa111.275 ◽

2020 ◽

Vol 59 (Supplement_2) ◽

Author(s):

Meena Naja ◽

Liliana R Santos ◽

Muhammad Shipa ◽

Greenwood Mandy ◽

Madhura Castelino

Keyword(s):

Mode Of Action ◽

Real Life ◽

Initial Therapy ◽

Current Evidence ◽

First Line ◽

Test Analysis ◽

Drug Survival ◽

Clinical Biomarkers ◽

Switching Mode ◽

Drop Outs

Abstract Background TNFi are the most used first line of biologic disease modifying anti-rheumatic drugs (bDMARDs) in PsA. In primary loss of response (PLR) to TNFi, there is no current evidence that directs the choice of second line bDMARDs. Our aim in this work was to compare drug survival for those who switched to a second TNFi versus alternative agents in real life. Methods Data was analysed retrospectively from a cohort of 400 PsA patients followed through from 2002-2019. Statistical analysis was carried out with descriptive statistics and t-test analysis using SPSS version 22. Results Out of 400 patients, 220 (55.0%) were started on bDMARD treatment. Of these 220, 212 (96.5%) were started on TNFi as initial therapy. PLR was seen in 42 of these patients (19.8%). The median drug survival of initial TNFi therapy was 7.1 months (interquartile range [IQR] 3.6 - 53.4 months). Of the 42 patients with PLR: 32 (76.2%) were switched to a second TNFi; 6 (14.3%) were switched to ustekinumab and 4 (9.5%) were switched to secukinumab. 21 of the 32 patients switched to a second TNFi were subsequently switched onto a third biologic due to treatment failure (65.6%). The median drug survival of the second TNFi in this group was 7.7 months (IQR 3.0 - 26.3 months). 3 out of the 6 (50%) patients who were switched to ustekinumab were then subsequently switched, this was due to primary inefficacy in 66.7% and adverse events in 33.3%. The median drug survival of ustekinumab in this group was 10.0 months (IQR 0.0 - 16.0 months). All 4 patients switched to secukinumab continue on this treatment with no drop outs, giving a median drug survival 12.3 months (IQR 6.5 - 19.5 months). Conclusion Our data suggests that patients with PLR to TNFi in PsA who switched mode of action to IL-17 inhibitor appeared to have better drug survival than subsequent TNFi or IL-12/23 inhibitors. Exploring the clinical biomarkers for those with successful switch to non-TNFi bDMARD in a larger cohort would help with targeting the most appropriate individuals and early disease control. Disclosures M. Naja None. L. R. Santos None. M. Shipa None. G. Mandy None. M. Castelino None.

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Oxford Case Histories in General Surgery

10.1093/med/9780198749813.001.0001 ◽

2018 ◽

Keyword(s):

General Surgery ◽

Real Life ◽

Surgical Care ◽

Diagnostic Information ◽

Current Evidence ◽

Evidence Based ◽

Case Histories ◽

The Core ◽

Discussion Format ◽

Case Based

Oxford Case Histories in General Surgery aims to bring the different subspecialties of general surgery to life for its readers by adopting a case-based discussion format around real-life cases. It is most relevant to those who are just starting out in general surgery, including medical students, surgical care practitioners, foundation doctors, and those entering core surgical training. Each case presents a clinical vignette comprising focussed and relevant clinical and diagnostic information followed by a cases-based discussion that covers relevant clinical material pertinent to the core surgical element of the Intercollegiate Surgical Curriculum. The case-based discussion format is an important learning tool, as it allows focussed application of textbook knowledge to clinical practice and incorporates that with current evidence-based approaches to clinical and surgical management.

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Switching to riociguat: a potential treatment strategy for the management of CTEPH and PAH

Pulmonary Circulation ◽

10.1177/2045894019837849 ◽

2020 ◽

Vol 10 (1) ◽

pp. 204589401983784

Author(s):

Raymond L. Benza ◽

Paul A. Corris ◽

Hossein-Ardeschir Ghofrani ◽

Manreet Kanwar ◽

Vallerie V. McLaughlin ◽

...

Keyword(s):

Life Experience ◽

Soluble Guanylate Cyclase ◽

Real Life ◽

Chronic Thromboembolic Pulmonary Hypertension ◽

Cyclic Guanosine Monophosphate ◽

Initial Therapy ◽

Guanosine Monophosphate ◽

Current Evidence ◽

Off Label ◽

Insufficient Response

Currently, five classes of drug are approved for the treatment of pulmonary arterial hypertension (PAH): phosphodiesterase 5 inhibitors (PDE5i); endothelin receptor antagonists; prostacyclin analogs; the IP receptor agonist selexipag; and the soluble guanylate cyclase (sGC) stimulator riociguat. For patients with inoperable or persistent/recurrent chronic thromboembolic pulmonary hypertension (CTEPH), riociguat is currently the only approved pharmacotherapy. Despite the development of evidence-based guidelines on appropriate use of specific drugs, in clinical practice patients are often prescribed PAH-targeted therapies off label or at inadequate doses. PDE5i are the most often prescribed class of drugs as initial therapy, either alone or in combination with other drug classes. However, a proportion of patients receiving PAH therapies do not reach or maintain treatment goals. As PDE5i and riociguat target different molecules in the nitric oxide-sGC-cyclic guanosine monophosphate (NO-sGC-cGMP) signaling pathway, for patients with PAH without an initial or sustained response to PDE5i, there is a biological rationale for switching to riociguat. However, robust data from randomized controlled trials on the safety and efficacy of switching are lacking, as is formal guidance for clinicians. Here we review studies of sequential combination therapy, and trial data and case studies that have investigated switching between PAH-approved therapies, particularly from PDE5i to riociguat in patients with PAH with an insufficient response to PDE5i, and in patients with CTEPH who were receiving off-label treatment. These studies summarize the current evidence and practical real-life experience on the concept of switching treatments.

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Eye Gaze And Perceptual Adaptation To Audiovisual Degraded Speech

10.31234/osf.io/65849 ◽

2020 ◽

Author(s):

Briony Banks ◽

Emma Gowen ◽

Kevin Munro ◽

patti adank

Keyword(s):

Visual Cues ◽

Eye Gaze ◽

Real Life ◽

Audio Signal ◽

Current Evidence ◽

Control Group ◽

Speech Comprehension ◽

Perceptual Adaptation ◽

Degraded Speech ◽

Over Time

Visual cues from a speaker’s face may improve perceptual adaptation to degraded speech over time, but current evidence is limited. We aimed to replicate results from previous studies and extend them to more demanding speech stimuli (sentences), to better represent real-life, challenging speech comprehension. In addition, we investigated whether particular eye gaze patterns towards the speaker’s mouth were related to adaptation, hypothesising that listeners who looked more at the speaker’s mouth would show greater adaptation. A group of listeners were presented with noise-vocoded sentences in audiovisual format while a control group were presented with the audio signal only, presented congruently with a still image of the speaker’s face. Results of previous adaptation studies were partially replicated: the audiovisual group had better recognition throughout and adapted slightly more rapidly, but both groups showed an equal amount of improvement overall (after exposure to 90 sentences). Longer fixations on the speaker’s mouth in the audiovisual group were related to better overall accuracy, although evidence for this relationship was relatively weak. An exploratory analysis further showed that the duration of fixations to the speaker’s mouth decreased over time. The results suggest that the benefits from visual cues to adaptation to unfamiliar speech vary more than previously thought. Longer fixations on a speaker’s mouth may play a role in successfully decoding these cues, but more evidence is needed to fully establish how patterns of eye gaze are related to audiovisual speech recognition.

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Incidence of anogenital warts: epidemiological risk factors and real-life impact of human papillomavirus vaccination

International Journal of STD & AIDS ◽

10.1177/0956462420958577 ◽

2020 ◽

pp. 095646242095857

Author(s):

Georgios Tyros ◽

Styliani Mastraftsi ◽

Stamatis Gregoriou ◽

Electra Nicolaidou

Keyword(s):

Risk Factors ◽

Human Papillomavirus ◽

Hpv Vaccination ◽

Real Life ◽

Current Evidence ◽

Evidence Based ◽

Anogenital Warts ◽

Psychological Burden ◽

Increased Risk ◽

The Impact

Anogenital warts (AGWs) rank among the most frequent sexually transmitted infections in young adults. They are benign lesions, but they pose a significant economic cost to health care systems and a substantial psychological burden on patients, who need evidence-based counselling. Human papillomavirus (HPV) vaccination has shown very high protection rates against AGWs in clinical trials and real-world settings but vaccination coverage remains low in many countries. The aim of this review is to summarize the current evidence on the risk factors for AGW development and to present the available real-life data on the impact of HPV vaccination on AGW incidence. An increased number of lifetime sexual partners, a new sexual partner in the last 12 months, smoking, and immunosuppression have been associated with increased risk for AGWs. HPV vaccination has led to a dramatic decline in AGW incidence in populations that have achieved high vaccination rates. These conclusions can contribute to primary prevention of AGWs and evidence-based counselling of AGW patients.

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Reduction of Fear: Towards a Unifying Theory

Canadian Psychiatric Association Journal ◽

10.1177/070674377301800103 ◽

1973 ◽

Vol 18 (1) ◽

pp. 9-12 ◽

Cited By ~ 23

Author(s):

Isaac M. Marks

Keyword(s):

Prolonged Exposure ◽

Real Life ◽

Current Evidence ◽

Therapeutic Principle ◽

Phobic Disorders ◽

Short Acting ◽

New Methods ◽

Avoidance Responses

In recent years many new methods which alleviate phobic disorders have been introduced. These include desensitization, operant shaping, flooding (implosion), prolonged exposure, paradoxical intention, modelling, cognitive rehearsal and intravenous short-acting sedatives. Different theories have been invoked to explain the action of these procedures, and these are often contradictory. Current evidence suggests that the same therapeutic principle is responsible for the efficacy of most of these methods, this being the continued exposure to the phobic situation until anxiety and avoidance responses are extinguished. This exposure is greatly facilitated when carried out in real life rather than in phantasy. The conditions for successful exposure are explored and other possible therapeutic elements are discussed.

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Evidence for the Application of Sepsis Bundles in 2021

Seminars in Respiratory and Critical Care Medicine ◽

10.1055/s-0041-1733899 ◽

2021 ◽

Vol 42 (05) ◽

pp. 706-716

Author(s):

Erika P. Plata-Menchaca ◽

Juan Carlos Ruiz-Rodríguez ◽

Ricard Ferrer

Keyword(s):

Quality Of Care ◽

Real Life ◽

Current Evidence ◽

Sepsis Management ◽

Severe Condition ◽

Sepsis Bundles ◽

Risk Of Death ◽

Significant Survival ◽

Evidence Based Treatments

AbstractSepsis represents a severe condition that predisposes patients to a high risk of death if its progression is not ended. As with other time-dependent conditions, the performance of determinant interventions has led to significant survival benefits and quality-of-care improvements in acute emergency care. Thus, the initial interventions in sepsis are a cornerstone for prognosis in most patients. Even though the evidence supporting the hour-1 bundle is perfectible, real-life application of thoughtful and organized sepsis care has improved survival and quality of care in settings promoting compliance to evidence-based treatments. Current evidence for implementing the Surviving Sepsis Campaign bundles for early sepsis management is moving forward to better approaches as more substantial evidence evolves.

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Breathing exercises for asthma

Breathe ◽

10.1183/20734735.008414 ◽

2014 ◽

Vol 10 (4) ◽

pp. 312-322 ◽

Cited By ~ 7

Author(s):

Mike Thomas ◽

Anne Bruton

Keyword(s):

Quality Of Life ◽

Real Life ◽

Psychological State ◽

Current Evidence ◽

List Type ◽

Breathing Control ◽

Breathing Exercises ◽

Breathing Training ◽

Patient Reported

Educational aimsTo summarise the evidence of the role of breathing control approaches in the management of asthmaTo provide information on the content of evidence-based breathing exercises programmesSummaryAsthma is a complex, multi-dimensional condition that affects patients in many ways. Having asthma is inherently stressful and psychological problems are common and associated with poor asthma outcomes. Although most patients in clinical trials can achieve high levels of control with optimised pharmacotherapy, in “real-life” practice, poor control is common, with over-reliance on rescue bronchodilator medication and ongoing symptoms and quality-of-life impairment. Many patients are interested in non-pharmacological treatments to improve asthma control, particularly breathing control exercises but, until recently, the evidence base has been inadequate. The place of breathing exercises has been controversial, partly because some proponents have made exaggerated, implausible claims of effectiveness. Recent evidence, however, has resulted in endorsement of breathing exercises as add-on treatment in asthma in systematic reviews and guidelines.This review summarises the current evidence of effectiveness of breathing exercises programmes as an adjuvant treatment to pharmacological strategies for people with asthma. The types of breathing training programmes used and the content of effective programmes are discussed. We conclude that patients whose asthma continues to cause symptoms and quality-of-life impairment, despite adequate pharmacological treatment, or who have high bronchodilator use, should be offered access to an effective breathing training programme as a part of holistic, integrated asthma care.Key pointsAsthma is frequently poorly controlled despite effective modern medicationPsychological factors can be as important as physiological ones in affecting symptom perception and disease impactBreathing exercises can improve patient-reported outcomes and psychological stateBreathing exercises should be offered to all asthma patients with symptoms or impaired quality of life despite standard treatment

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Managing Urolithiasis with Thulium Fiber Laser: Updated Real-Life Results—A Systematic Review

Journal of Clinical Medicine ◽

10.3390/jcm10153390 ◽

2021 ◽

Vol 10 (15) ◽

pp. 3390

Author(s):

Olivier Traxer ◽

Mariela Corrales

Keyword(s):

Clinical Trials ◽

Fiber Laser ◽

Full Text ◽

Gold Standard ◽

Real Life ◽

Current Evidence ◽

Clinical Experiences ◽

Ablation Efficiency ◽

Thulium Fiber Laser ◽

Endoscopic Lithotripsy

Thirty-three years ago, pulsed lasers marked the beginning of a new era in endoscopic lithotripsy, and the one that was highlighted because of its potential was the Holmium: YAG laser, which became and still is the gold standard in endourology. Recently, a new laser technology has been accepted for clinical use in lithotripsy: the thulium fiber laser (TFL), showing appealing characteristics not seen before in several preclinical studies. A review of the literature was performed and all relevant in vitro studies and clinical trials until April 2021 were selected. The search came back with 27 clinical experiences (7 full-text clinical trials and 20 peer-reviewed abstracts) and 33 laboratory studies (18 full-text articles and 15 peer-reviewed abstracts). The clinical experiences confirmed the clinical safety of using the wide parameter range of the TFL. This technology demonstrated the performance at a higher ablation speed, the higher ablation efficiency, and the better dust quality of the TFL, as well as reduced stone retropulsion, thus helping to maintain an optimal visibility. No thermal or radiation damage was found. Given the current evidence, we may be facing the future gold standard laser in endoscopic lithotripsy.

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