Patient Perspectives and Experiences of Preventive Treatments and Self-Injectable Devices for Migraine:A Focus Group Study

BACKGROUND: Although several self-injectable preventive treatments for migraine have become available, they are not yet widely used. Thus, understanding patients’ perceptions towards them is limited. OBJECTIVE: This study aimed to inform the design of a preference-elicitation instrument, which is being developed to quantify preventive treatment preferences of people with migraine. METHODS: We conducted a qualitative study involving nine in-person focus groups (three per country) in the United States, the United Kingdom, and Germany. Participants were adults (n = 47) with episodic or chronic migraine who were currently using or had used a prescription preventive treatment for migraine within the previous 5 years. During the focus groups, participants described their experiences of migraine and preventive treatments; handled and simulated self-injection using five different unbranded, fired demonstration auto-injectors and prefilled syringes; and ranked different aspects of preventive treatments by importance. Focus groups were analyzed with a focus on themes that would be feasible or meaningful to include in a subsequent preference-elicitation instrument. RESULTS: Reducing the frequency and severity of migraine attacks was consistently ranked as the most important aspect of preventive treatment. Participants expressed dissatisfaction with available daily oral preventive treatments for migraine they had previously used because they were ineffective or caused intolerable adverse events. Many participants were willing to self-inject a treatment that was effective and tolerable. When presented with devices for self-injecting a preventive treatment for migraine, participants generally preferred autoinjectors over prefilled syringes. Participants especially valued safety features such as the unlocking step and automated needle insertion, and audible and visual dose confirmation increased confidence in autoinjector use. Autoinjector needle protection mechanisms were also appreciated, especially by participants averse to needles, as the needles are not visible. CONCLUSIONS: This study highlights the fact that many people with migraine still lack access to a preventive treatment that is effective and tolerable. In addition to efficacy and safety considerations, treatment decisions may be guided by the mode of administration. In the case of self-injectable preventive treatments, key device characteristics affecting these decisions may be ease of use, comfort, and confidence in self-injection. Insights gained from this study were used to help develop a preliminary set of attributes and levels for a preference-elicitation instrument. SUPPLEMENTARY INFORMATION: The online version contains supplementary material available at 10.1007/s40271-021-00525-z

A systematic review on the impact of leg ulceration on patients' quality of life

<p>Abstract</p> <p>Background</p> <p>A systematic review was conducted to analyse journal articles that describe or measure the impact of leg ulceration on patients' quality of life (QoL) in order to improve the content of an educational programme that aims to enhance self-care agency in leg ulcer patients.</p> <p>Method</p> <p>Original articles published in English and German between 1990 and 2006 were included if the findings were analysed at the level of patients. Articles were excluded if (1) they investigated the impact of specific treatments or settings on QoL or (2) focused mainly on arterial ulcers or diabetic foot ulcers.</p> <p>Results</p> <p>Twenty-four original research articles met the inclusion criteria; 11 studies used a quantitative, 11 studies a qualitative, and 2 used a mixed method approach. The findings were collapsed into 5 core domains. Quantitative studies commonly investigated the parameters of pain, sleep, social isolation, and physical mobility. Patients had significantly more pain, more restrictions regarding social functioning, less vitality, and limitations with respect to emotional roles compared to the respective controls. Other problem areas identified were restrictions in work capacity, recreation, social interaction, psychological well-being, as well as problems caused by treatment regimes. Inconclusive results were obtained regarding pain intensity, physical restrictions, and gender effects.</p> <p>Limitations</p> <p>Numerous original studies neither undertook a differentiation of participants by ulcer aetiology nor did they analyse the results according to gender differences.</p> <p>Conclusion</p> <p>As leg ulceration has an impact on QoL, national guidelines on the treatment of leg ulceration need to more specifically address these far-ranging effects identified in this review.</p

Considering Usual Medical Care in Clinical Trial Design

Liza Dawson and colleagues discuss the scientific and ethical issues associated with choosing clinical trial designs when there is no consensus on what constitutes usual care

The Canadian Bandaging Trial: Evidence-informed leg ulcer care and the effectiveness of two compression technologies

Background: Objective: To determine the relative effectiveness of evidence-informed practice using two high compression systems: four-layer (4LB) and short-stretch bandaging (SSB) in community care of venous leg ulcers. Design and Setting: Pragmatic, multi-centre, parallel-group, open-label, randomized controlled trial conducted in 10 centres. Cognitively intact adults (≥18 years) referred for community care (home or clinic) with a venous ulceration measuring ≥0.7cm and present for ≥1 week, with an ankle brachial pressure index (ABPI) ≥0.8, without medication-controlled Diabetes Mellitus or a previous failure to improve with either system, were eligible to participate.Methods: Consenting individuals were randomly allocated (computer-generated blocked randomization schedule) to receive either 4LB or SSB following an evidence-informed protocol. Primary endpoint: time-to- healing of the reference ulcer. Secondary outcomes: recurrence rates, health-related quality of life (HRQL), pain, and expenditures.Results: 424 individuals were randomized (4LB n = 215; SSB n = 209) and followed until their reference ulcer was healed (or maximum 30 months). An intent-to-treat analysis was conducted on all participants. Median time to ulcer healing in the 4LB group was 62 days [95% confidence interval (CI) 51 to 73], compared with 77 days (95% CI 63 to 91) in the SSB group. The unadjusted Kaplan-Meier curves revealed the difference in the distribution of cumulative healing times was not significantly different between group (log rank χ2 = 0.001, P = 0.98) nor ulcers recurrence (4LB, 10.1%; SSB, 13.3%; p = 0.345). Multivariable Cox Proportional Hazard Modeling also showed no significant between-bandage differences in healing time after controlling for significant covariates (p = 0.77). At 3-months post-baseline there were no differences in pain (no pain: 4LB, 22.7%; SSB, 26.7%; p = 0.335), or HRQL (SF-12 Mental Component Score: 4LB, 55.1; SSB, 55.8; p = 0.615; SF-12 Physical Component Score: 4LB, 39.0; SSB, 39.6; p = 0.675). The most common adverse events experienced by both groups included infection, skin breakdown and ulcer deterioration.Conclusions: The Canadian Bandaging Trial revealed that in the practice context of trained RNs using an evidence-informed protocol, the choice of bandage system (4LB and SSB) does not materially affect healing times, recurrence rates, HRQL, or pain. From a community practice perspective, this is positive news for patient-centred care allowing individual/family and practitioner choice in selecting compression technologies based on circumstances and context.Trial registration: clinicaltrials.gov Identifier: NCT00202267

Evaluation of the psychometric properties of the Early Morning Symptoms of COPD Instrument (EMSCI)

Asha Hareendran,1 Barry J Make,2 Erica Zaiser,1 Esther Garcia Gil3 1Patient Centered Research, Evidera, London, UK; 2Division of Pulmonary, Critical Care and Sleep Medicine, National Jewish Health, Denver, CO, USA; 3Global Medical Affairs, AstraZeneca, Barcelona, Spain Background: Early morning respiratory symptoms impact quality of life and are often the most troublesome for patients with COPD. Reduction in symptoms and their impact are important treatment outcomes for COPD. The Early Morning Symptoms of COPD Instrument (EMSCI) is a daily diary designed to collect patients&rsquo; report of the occurrence, severity, and impact of morning COPD symptoms.Methods: To assess the psychometric properties of the EMSCI, a split-half sample of data from a COPD clinical trial where participants completed the EMSCI daily was used for conducting descriptive statistics, factor analyses, and Rasch model analyses to examine item performance and inform scoring. Once the final scoring algorithm was determined, data from the second split-half sample were used to examine the properties of the EMSCI. Test&ndash;retest reliability was assessed using intraclass correlation coefficient (ICC). Correlations with other study assessments were used to evaluate convergent and known-groups validity.Results: Data from 1,663 patients with COPD aged 40&ndash;93 years were analyzed. Factor analysis and Rasch analysis confirmed a one-factor structure for the 6 individual symptom items. Item analyses supported the generation of 4 scores. All scores demonstrated good test&ndash;retest reliability: 6-item symptom severity (ICC, 0.84); overall morning symptom severity (ICC, 0.84); activity limitation (ICC, 0.85); and rescue medication (ICC, 0.62). Significant correlations between EMSCI scores, St George&rsquo;s Respiratory Questionnaire scores, and EXAcerbations of Chronic pulmonary disease Tool (EXACT)-Respiratory Symptoms scores supported the tool&rsquo;s convergent validity. Significant differences (p&lt;0.0001) in all EMSCI domain scores were found between known-groups based on median split St George&rsquo;s Respiratory Questionnaire and EXACT-Respiratory Symptoms scores.Conclusion: The EMSCI consists of 4 scores: 6-item symptom severity, overall symptom severity, activity limitation, and rescue medication. The EMSCI is a reliable and valid instrument for evaluating patients&rsquo; experience of early morning COPD symptoms. Keywords: respiratory symptoms, daily diary, PRO, psychometric validation, activity limitation, rescue medication&nbsp