Background Botulinum neurotoxin type A (BoNT-A) is an effective pharmacological treatment for the management of cervical dystonia (CD) that requires repeated administration at variable intervals. We explored patient perceptions of the impact of CD and the waning of BoNT-A therapeutic effects. Methods An internet-based survey was conducted through Carenity, a global online patient community, from May to September 2019. Eligible respondents were adults with CD who had ≥ 2 previous BoNT-A injections. Results 209 respondents (81% females; mean age of 49.7 years) met the screening criteria. The mean BoNT-A injection frequency was 3.9 injections/year. The mean reported onset of BoNT-A therapeutic effect was 11.7 days and the time to peak effect was 4.5 weeks. Symptom re-emergence between injections was common (88%); the time from injection to symptom re-emergence was 73.6 days (~ 10.5 weeks). Treatment was not reported to completely abolish symptoms, even at peak effect. However, symptom severity was rated (0 = no symptoms; 10 = very strong symptoms) as lowest at the peak of treatment effects (mean scores ~ 3/10), increasing as the effects of treatment start waning (~ 5.5/10) and was strongest one day before the next session (~ 7–8/10). The impact of CD on quality of life followed the same ‘rollercoaster’ pattern. Conclusions This survey highlights the burden of CD symptoms, even in patients undergoing regular treatment. Symptom re-emergence is common and has significant impact on daily activities and quality of life. Greater awareness of the therapeutic profile of BoNT-A treatment should lead to better informed therapeutic discussions and planning.

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Background While the lung cancer (LC) treatment landscape has rapidly evolved in recent years, easing symptom burden and treatment side effects remain central considerations in disease control. Objective The aim of this study was to assess the relative importance of dimensions of LC care to patients, and to explore the disease burden, including socioeconomic aspects not commonly covered in patient-reported outcomes instruments. Methods A questionnaire was sent to patients with LC and their caregivers to rate the value of a diverse set of quality of life dimensions in care, to evaluate communication between health care professionals (HCPs) and patients, and to explore the economic impact on respondents. The survey included questions on the dimensions of care covered by patient-reported outcomes instruments for quality-of-life evaluation (Functional Assessment of Cancer Therapy–Lung scale, EQ-5D, the European Organization for Research and Treatment of Cancer’s Core Quality of Life questionnaire, and the European Organization for Research and Treatment of Cancer’s Core Quality of Life in lung cancer 13-item questionnaire), as well as the International Consortium for Health Outcomes Measurement (ICHOM) standard set of patient-centered outcomes for LC. The survey respondents were participants on Carenity’s patient community platform, living either in France, the United Kingdom, Germany, Italy, or Spain. Results The survey included 150 respondents (115 patients and 35 caregivers). “Physical well-being” and “end-of-life care” (median scores of 9.6, IQR 7.7-10, and 9.7, IQR 8.0-10, on a 10-point scale) were rated highest among the different value dimensions assessed. “Physical well-being and functioning” was the dimension most frequently discussed with health care professionals (82/150, 55%), while only (17/100, 17%) reported discussing “end-of-life care.” After diagnosis, 43% (49/112) of patients younger than 65 years stopped working. Among respondents who indicated their monthly household income before and after diagnosis, 55% (38/69) reported a loss of income. Conclusions Our results showed the relevance of a broad range of aspects of care for the quality of life of patients with LC. End-of-life care was the dimension of care rated highest by patients with LC, irrespective of stage at diagnosis; however, this aspect is least frequently discussed with HCPs. The results also highlight the considerable socioeconomic impact of the disease, despite insurance coverage of direct costs.

The article Patient perspectives on the therapeutic profile of botulinum neurotoxin type A in cervical dystonia, written by Cynthia Comella, Joaquim J. Ferreira, Emilie Pain, Marion Azoulai and Savary Om, was originally published electronically on the publisher’s internet portal on 16 September 2020 without open access. With the author(s)’ decision to opt for Open Choice the copyright of the article changed on 25 September 2020 to © The Authors 2020 and this article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made

Objectives: To understand the economic and humanistic burden attributed to myasthenia gravis (MG). Methods: We conducted a literature search (January 2009 to April 2019) in the biomedical database Embase (including Medline), focusing on English language observational studies and economic models evaluating patients with MG in the United States. Key search terms focused on costs, healthcare resource utilization, and quality of life (QoL). Results: A total of 18 studies were identified that included economic or humanistic burden data. Median length of hospitalization in patients with MG ranged from 2 days to 8 days, with costs per admission, using National Inpatient Sample data, ranging from a median of $16,000 in 1 study to a mean of nearly $99,000 in another analysis. Patient factors including MG crisis, presence of respiratory failure, and complex medication regimens (including multiple immunosuppressive and conventional therapies) worsened the burden, increasing length of stay from a median of 4 -6 days to 6 -10 days. Costs of admission increased from $16,000 to $26,600 for patients hospitalized for MG (not MG crisis), up to almost $54,000 for patients experiencing an MG crisis and receiving plasma exchange. Among patients treated with multiple immunosuppressants, presence of continued functional disability contributed to a worse QoL. One study found that QoL was worse in women than men, but the difference was eliminated for women who had undergone a thymectomy. Conclusions: The economic and humanistic burden associated with MG is considerable, particularly for patients with severe complications, including MG crisis. Understanding the risk factors associated with MG-related complications, and the potential impact on treatment modalities, is crucial to improve the medical management of these patients. Additionally, there is a clear unmet need in this patient population for new treatment options to improve disease course and prevent complications.