Adaptation of the Patient Benefit Assessment Scale for Hospitalised Older Patients: development, reliability and validity of the P-BAS picture version

Background The Patient Benefit Assessment Scale for Hospitalised Older Patients (P-BAS HOP) is a tool developed to both identify the priorities of the individual patient and to measure the outcomes relevant to him/her, resulting in a Patient Benefit Index (PBI), indicating how much benefit the patient had experienced from the hospitalisation. The reliability and the validity of the P-BAS HOP appeared to be not yet satisfactory and therefore the aims of this study were to adapt the P-BAS HOP and transform it into a picture version, resulting in the P-BAS-P, and to evaluate its feasibility, reliability, validity, responsiveness and interpretability. Methods Process of instrument development and evaluation performed among hospitalised older patients including pilot tests using Three-Step Test-Interviews (TSTI), test-retest reliability on baseline and follow-up, comparing the PBI with Intraclass Correlation Coefficient (ICC), and hypothesis testing to evaluate the construct validity. Responsiveness of individual P-BAS-P scores and the PBI with two different weighing schemes were evaluated using anchor questions. Interpretability of the PBI was evaluated with the visual anchor-based minimal important change (MIC) distribution method and computation of smallest detectable change (SDC) based on ICC. Results Fourteen hospitalised older patients participated in TSTIs at baseline and 13 at follow-up after discharge. After several adaptations, the P-BAS-P appeared feasible with good interviewer’s instructions. The pictures were considered relevant and helpful by the participants. Reliability was tested with 41 participants at baseline and 50 at follow-up. ICC between PBI1 and PBI2 of baseline test and retest was 0.76, respectively 0.73. At follow-up 0.86, respectively 0.85. For the construct validity, tested in 169 participants, hypotheses regarding importance of goals were confirmed. Regarding status of goals, only the follow-up status was confirmed, baseline and change were not. The responsiveness of the individual scores and PBI were weak, resulting in poor interpretability with many misclassifications. The SDC was larger than the MIC. Conclusions The P-BAS-P appeared to be a feasible instrument, but there were methodological barriers for the evaluation of the reliability, validity, and responsiveness. We therefore recommend further research into the P-BAS-P.

Study Designs for Evaluation of Combination Treatment: Focus On Individual Patient Benefit

Combination treatment, i.e., the use of two or more drugs for the same condition, is frequent in medicine if monotherapy yields an insufficient therapeutic response. We here review and challenge clinical study designs and formats of reporting outcomes for the evaluation of the benefit/risk ratio of combination treatment over monotherapy. We demonstrate that benefits of combination treatment at the group level over-estimate the probability of benefit at the single patient level based on outcome simulations under almost any imaginable setting. Based on these findings we propose that studies testing combination treatment should always report on percentages of responders to monotherapy and combination treatment. We provide equations that allow calculation of the percentage of patients truly benefitting from combination (responders to both monotherapies) and that of patients exposed to risk of harm from adverse effects without a reasonable expectation of individual benefit. These considerations are explained based on real clinical data, mostly from the field of functional urology (male lower urinary tract symptoms).

Total Knee Arthroplasty and Intra-Articular Pressure Sensors: Can They Assist Surgeons with Intra-Operative Decisions?

Purpose of Review Soft tissue imbalance, presenting as instability or stiffness, is an important cause of revision total knee arthroplasty (TKA). Traditional methods of determining soft tissue balance of the knee lack precision and are not reliable between operators. Use of intra-operative pressure sensors offers the potential to identify and avoid soft tissue imbalance following TKA. This review aims to summarise the literature supporting the clinical indication for the use of intra-articular pressure sensors during TKA. Recent Findings Analytical validation studies suggest that intra-operative pressure sensors demonstrate ‘moderate’ to ‘good’ intra-observer reliability and ‘good’ to ‘excellent’ interobserver reliability throughout the flexion arc. However, there are important errors associated with measurements when devices are used out-with the stated guidelines and clinicians should be aware of the limitations of these devices in isolation. Current evidence regarding patient benefit is conflicting. Despite positive early results, several prospective studies have subsequently failed to demonstrate significant differences in overall survival, satisfaction, and patient-reported outcome measures within 1 year of surgery. Summary Surgeon-defined soft tissue stability appears to be significantly different from the absolute pressures measured by the intra-operative sensor. Whilst it could be argued that this confirms the need for intra-articular sensor guidance in TKA; the optimal ‘target’ balance remains unclear and the relationship with outcome in patients is not determined. Future research should (1) identify a suitable reference standard for comparison; (2) improve the accuracy of the sensor outputs; and (3) demonstrate that sensor-assisted TKA leads to patient benefit in patient-reported outcome measures and/or enhanced implant survival.

Patients’ Perspectives on Qualitative Olfactory Dysfunction: Thematic Analysis of Social Media Posts

Background The impact of qualitative olfactory disorders is underestimated. Parosmia, the distorted perception of familiar odors, and phantosmia, the experience of odors in the absence of a stimulus, can arise following postinfectious anosmia, and the incidences of both have increased substantially since the outbreak of COVID-19. Objective The aims of this study are to explore the symptoms and sequalae of postinfectious olfactory dysfunction syndrome using unstructured and unsolicited threads from social media, and to articulate the perspectives and concerns of patients affected by these debilitating olfactory disorders. Methods A thematic analysis and content analysis of posts in the AbScent Parosmia and Phantosmia Support group on Facebook was conducted between June and December 2020. Results In this paper, we identify a novel symptom, olfactory perseveration, which is a triggered, identifiable, and usually unpleasant olfactory percept that persists in the absence of an ongoing stimulus. We also observe fluctuations in the intensity and duration of symptoms of parosmia, phantosmia, and olfactory perseveration. In addition, we identify a group of the most common items (coffee, meat, onion, and toothpaste) that trigger distortions; however, people have difficulty describing these distortions, using words associated with disgust and revulsion. The emotional aspect of living with qualitative olfactory dysfunction was evident and highlighted the detrimental impact on mental health. Conclusions Qualitative and unsolicited data acquired from social media has provided useful insights into the patient experience of parosmia and phantosmia, which can inform rehabilitation strategies and ongoing research into understanding the molecular triggers associated with parosmic distortions and research into patient benefit.

Identifying Epidermolysis Bullosa Patient Needs and Perceived Treatment Benefits: An Explorative Study Using the Patient Benefit Index

Epidermolysis bullosa (EB) is a genetic blistering skin condition for which no cure exists. Symptom alleviation and quality of life are therefore central to EB care. This study aimed to gain insight into EB patient needs and benefits from current clinical care. Two questionnaires were administered cross-sectionally to adult EB patients at the Dutch expertise centre for blistering diseases. Patient needs and benefits were analyzed using the patient benefit index survey (PBI-S). Ancillary data were compiled pertaining to self-reported EB severity, pain and pruritus, as well as current and previous treatments. In total, 104 participants were included (response rate 69.8%). Sixty-eight participants comprised the analyzed cohort (n = 36 omitted from analysis). The needs given the highest importance were to get better skin quickly (64.7%) and to be healed of all skin alterations (61.8%). A positive correlation between pain and EB severity and the importance of most needs was observed. Minimal clinically important differences within the PBI-S, relating to reported benefits from clinical care, were reported by 60.3% of the cohort. This study highlights a discrepancy between patient needs and feasible treatment outcomes. Utilizing the PBI-S in conjunction with well-established multidisciplinary care may catalyze the process of tailoring treatments to the needs of individual patients.

Accelerating the transition of clinical science to translational medicine

The SARS-CoV-2 pandemic has shown the importance of medical research in responding to the urgent prevention and health needs to combat the devastating disease, COVID-19, that this β-coronavirus unleashed. Equally, it has demonstrated the importance of interdisciplinary working to translate scientific discovery into public and patient benefit. As we come to adjust to live with this new virus, it is important to look back and see what lessons we have learnt in the way scientific medical discoveries can be more effectively and rapidly moved into public benefit. Clinical Science has had a long and distinguished history with this Journal bearing the same name and being an important contributor to the rapidly increasing use of human pathobiological data to gain mechanistic understanding of disease mechanisms leading to new diagnostic tests and treatments. The recognition that many complex diseases engage multiple causal pathways that may vary from patient to patient, and at different times across the lifecourse, has led to the emergence of stratified or precision medicine in which the right treatment is given to the right patient at the right time and, in doing so, minimise ‘non-responders’ and off-target side effects. Applications of omics technologies, the digitalisation of biology and the applications of machine learning and artificial intelligence (AI) are accelerating disease insights at pace with translation of discoveries into new diagnostic tests and treatments. The future of clinical science, as it morphs into translational medicine, is now creating unique possibilities where even the most intractable diseases are now open to being conquered.

Digital Technology In Psychiatry: Expectations, Barriers And Understanding Of Clinicians (Preprint)

BACKGROUND Digital technology has the potential to transform psychiatry, but adoption has been limited. The proliferation of telepsychiatry during COVID increases urgency of optimizing technology for clinical practice. Understanding clinician attitudes and preferences is crucial to effective implementation and patient benefit. OBJECTIVE Our objective was to elicit clinician perspectives on emerging digital technology. METHODS Clinicians in a large psychiatry department (inpatient and outpatient) were invited to complete an online survey about their attitudes towards digital technology in practice, focusing on implementation, clinical benefits, and expectations about patients’ attitudes. The survey consisted of 23 questions that could be answered on either a 3-point or 5-point Likert scale. We report frequencies and percentages of responses. RESULTS 139 clinicians completed the survey. They represented a variety of years-experience, credentials and diagnostic sub-specialties (response rate of 69.5%). Eighty-three percent (n=116) stated that digital data could improve their practice. Twenty-three percent of responders reported that they had viewed patients’ profiles on social media (n=32). Among anticipated benefits, clinicians rated symptom self-tracking (n=101, 72.7%) as well as clinical intervention support (n=90, 64.7%) as most promising. Among anticipated challenges, clinicians mostly expressed concerns over greater time demand (n=123, 88.5%) and whether digital data would be actionable (n=107, 77%). Ninety-five percent (n=132) of clinicians expected their patients to share digital data. CONCLUSIONS Overall, clinicians reported a positive attitude toward the use of digital data to improve patient outcomes but also highlight significant barriers that implementation need to overcome. Although clinicians’ self-reported attitudes about digital technology may not necessarily translate into behavior, results suggest that technologies that reduce clinician burden and are easily interpretable have the greatest likelihood of uptake.