The Rory Morrison WMUK Registry for Waldenström macroglobulinaemia: The growth of a national registry for a rare disorder.

National registries are used globally to characterise patient demographics, treatment choices and mortality to inform and improve clinical management. Waldenström macroglobulinaemia (WM) is a rare, treatment-responsive B-cell lymphoproliferative disorder with diverse clinical features and variable outcomes. To prospectively chart changes in the management of WM in the UK, the Rory Morrison Registry (RMR) was developed to systematically collect real-world data. Here we describe the development of the RMR, demonstrate its feasibility and describe preliminary observations. The RMR was devised as a collaborative project between patients and clinicians, under the auspices of the UK Charity for WM in 2016. Patients may be registered after the point of diagnosis and those with historic diagnosis were also eligible. Data collection fields were compiled by focus groups of clinicians, patients, industry and commissioning partners. The RMR launched in November 2017 and as of March 2022, there were 22 participating centres and 1305 patients registered. Median follow-up was 6.4 years, five-year overall survival 90.7% (95% confidence interval [CI] 88.4%-92.5%) and 10-year overall survival 79.3% (95% CI 75.7%-82.4%). There has been a clear evolution in treatments including a rapid growth in the use of Bruton's tyrosine kinase inhibitors in relapsed disease since their availability in the UK.

Patient reported outcome measures in Waldenström macroglobulinaemia: A real-world data analysis from the WMUK Rory Morrison Registry.

Waldenström macroglobulinaemia (WM) is an incurable chronic B-cell malignancy, but highly responsive to treatment. Treatments include fixed-duration chemotherapy and continuous oral chemoimmunotherapy. In this expanding field, it is important to have reliable information on the impact of the various therapies on patients' quality of life (QoL). Patient reported outcome measures (PROMs) are increasingly recognised as important to understand patient experience of disease beyond traditional clinical outcome measures. Four QoL questionnaires (EORTC QLQ-C30 [European Organisation for Research and Treatment of Cancer quality of life core questionnaire], BIPQ [Brief Illness Perception Questionnaire], HADS [Hospital Anxiety and Depression Scale], EQ-5D-5L [EuroQoL 5-dimensional descriptive system questionnaire]) are embedded in the UK national WM registry, the Rory Morrison Registry. We reviewed the results from a snapshot of PROMs. As of November 2021, 155 patients completed PROM data with 98% completion rate across all 58 questions. Complete clinical information was available for 52 patients. The majority of QoL questions (69%) failed to elicit a notable median response. Only four questions elicited statistically significant responses when comparing groups, and these were exclusively found in the EuroQoL-5D-5L and HADS questionnaires. Our data suggest that widely used questionnaires may not be suitable for patients with WM. We advocate the development of WM-specific outcome measures to overcome this.

Bortezomib-based therapy is effective and well tolerated in frontline and multiply pre-treated Waldenström macroglobulinaemia including BTKi failures: A real-world analysis.

Waldenström macroglobulinemia (WM) is a rare, incurable low grade lymphoma following a relapsing trajectory. Management strategies have evolved with the introduction of targeted therapy including new classes of Bruton tyrosine kinase inhibitor (BTKi). Treatment may however be limited particularly at relapse by a lack of drug availability and tolerability. We assessed the real-world efficacy and tolerability of bortezomib-containing regimens in patients with WM at frontline and relapse including those with prior BTKi resistance. Forty-one patients were identified with 44 bortezomib-containing regimens administered (n = 12 frontline, n = 32 relapse). Of patients treated at relapse, the median prior lines of therapy was 3 (range 1-7). 24% (10/41) of the cohort were refractory or intolerant to BTKi prior to bortezomib delivery. The median follow-up after bortezomib administration was 34 months (range 0-131). Overall response rate was 88%; 2-year overall survival and progression-free survival were 90% (95% confidence interval [CI] 73-96) and 76% (95% CI 55-87), respectively. Median time-to-next-treatment was 66 months. Neuropathy (grade 1-2) occurred in 24% (8/34) and did not result in treatment cessation in any case. Gastrointestinal disturbance occurred in 7% (3/41). Treatment discontinuations were rare (1/44; 2%), suggesting a manageable safety profile. Major response rate was comparable in those with prior BTKi compared with those without (75% [6/8] vs 84% [27/32], p = 0.61). Bortezomib should be considered as a treatment modality particularly in those who are refractory to BTKi.

'Should I Stay, or Should I Go?' Psychological Distress Predicts Career Change Ideation among Intensive Care Staff in Lithuania and the UK Amid COVID-19 Pandemic.

The COVID-19 pandemic had a significant effect on healthcare globally. Additional pressure created by coronavirus adversely affected the mental health and psychological well-being of healthcare workers, leading many to question their desire and willingness to continue working in healthcare. This study aimed to identify predictors for career change ideation among healthcare professionals in two countries; Lithuania and the United Kingdom amid the coronavirus pandemic. In total, 610 healthcare professionals from Lithuania and the UK (285 and 325, respectively) participated in a survey from May to August 2020. Psychological distress and psychological well-being were measured using the self-report scales "DASS-21" and "WHO-5". Almost half of the sample (49.2%), 59.6% and 40.0% in Lithuanian and the UK, respectively, exhibited career change ideation, the country effect was significant (AOR = 2.21, p < 0.001). Stronger ideation to leave healthcare was predicted by higher levels of depression (AOR = 1.10, p = 0.005), stress (AOR = 1.10, p = 0.007), anxiety surrounding inadequate personal protective equipment (AOR = 2.27, p = 0.009), and lower psychological well-being scores (AOR = 1.10, p = 0.007). We conclude that psychosocial support must be provided for healthcare professionals to prevent burnout and loss of staff amid the pandemic.

Medication Adherence Apps: Review and Content Analysis.

BACKGROUND: Medication adherence is an expensive and damaging problem for patients and health care providers. Patients adhere to only 50% of drugs prescribed for chronic diseases in developed nations. Digital health has paved the way for innovative smartphone solutions to tackle this challenge. However, despite numerous apps available claiming to improve adherence, a thorough review of adherence apps has not been carried out to date. OBJECTIVE: The aims of this study were to (1) review medication adherence apps available in app repositories in terms of their evidence base, medical professional involvement in development, and strategies used to facilitate behavior change and improve adherence and (2) provide a system of classification for these apps. METHODS: In April 2015, relevant medication adherence apps were identified by searching the Apple App Store and the Google Play Store using a combination of relevant search terms. Data extracted included app store source, app price, documentation of health care professional (HCP) involvement during app development, and evidence base for each respective app. Free apps were downloaded to explore the strategies used to promote medication adherence. Testing involved a standardized medication regimen of three reminders over a 4-hour period. Nonadherence features designed to enhance user experience were also documented. RESULTS: The app repository search identified a total of 5881 apps. Of these, 805 fulfilled the inclusion criteria initially and were tested. Furthermore, 681 apps were further analyzed for data extraction. Of these, 420 apps were free for testing, 58 were inaccessible and 203 required payment. Of the 420 free apps, 57 apps were developed with HCP involvement and an evidence base was identified in only 4 apps. Of the paid apps, 9 apps had HCP involvement, 1 app had a documented evidence base, and 1 app had both. In addition, 18 inaccessible apps were produced with HCP involvement, whereas 2 apps had a documented evidence base. The 420 free apps were further analyzed to identify strategies used to improve medication adherence. This identified three broad categories of adherence strategies, reminder, behavioral, and educational. A total of 250 apps utilized a single method, 149 apps used two methods, and only 22 apps utilized all three methods. CONCLUSIONS: To our knowledge, this is the first study to systematically review all available medication adherence apps on the two largest app repositories. The results demonstrate a concerning lack of HCP involvement in app development and evidence base of effectiveness. More collaboration is required between relevant stakeholders to ensure development of high quality and relevant adherence apps with well-powered and robust clinical trials investigating the effectiveness of these interventions. A sound evidence base will encourage the adoption of effective adherence apps, and thus improve patient welfare in the process.