The burden of illness in patients with paroxysmal nocturnal hemoglobinuria receiving treatment with the C5-inhibitors eculizumab or ravulizumab: results from a US patient survey

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare and life-threatening disease with symptoms of hemolysis and thrombosis. Current therapies for this complement-mediated disease rely predominantly on inhibition of the C5 complement protein. However, data on treatment responses and quality of life in C5-inhibitor (C5i)-treated PNH patients are scarce. The objective of this study was to determine C5i treatment effects on clinical parameters, PNH symptoms, quality of life, and resource use for PNH patients. This cross-sectional study surveyed 122 individuals in the USA receiving treatment for PNH with C5-targeted monoclonal antibodies, eculizumab (ECU) or ravulizumab (RAV). Despite most patients receiving C5i therapy for ≥ 3 months (ECU 100%, n = 35; RAV 95.4%, n = 83), many patients remained anemic with hemoglobin levels ≤ 12 g/dL in 87.5% (n = 28/32) and 82.9% (n = 68/82) of ECU and RAV recipients, respectively. A majority of patients on ECU (88.6%; n = 31/35) and RAV (74.7%; n = 65/87) reported fatigue symptoms. Among PNH patients receiving C5i therapy for ≥ 12 months, some still reported thrombotic events (ECU, 10.0%, n = 1/10; RAV, 23.5%, n = 4/17) and required transfusions within the past year (ECU, 52.2%, n = 12/23; RAV, 22.6%, n = 7/31). Other patient-reported PNH symptoms included breakthrough hemolysis, shortness of breath, and headaches. Patients reported scores below the average population norms on the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue and European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) scales. Overall, this study found that PNH patients receiving ECU or RAV therapy demonstrated a significant burden of illness, highlighting the need for improved PNH therapies.

Opportunities and Barriers of Telemedicine in Rheumatology: A Participatory, Mixed-Methods Study

Despite all its promises, telemedicine is still not widely implemented in the care of rheumatic and musculoskeletal diseases (RMDs). The aim of this study is to investigate opportunities, barriers, acceptance, and preferences concerning telemedicine among RMD patients and professional stakeholders. From November 2017 to December 2019, a participatory, mixed-methods study was conducted, consisting of (1) expert interviews (n = 27) with RMD patients and professional stakeholders, (2) a national paper-based patient survey (n = 766), and (3) focus groups (n = 2) with patient representatives and rheumatologists. The qualitative findings indicate that patients equate personal contact with physical face-to-face contact, which could be reduced by implementing telemedicine, thus negatively influencing the patient–doctor relationship. Correspondingly “no personal contact with the doctor” is the main reason (64%) why 38% of the surveyed patients refuse to try telemedicine. Professional stakeholders expect telemedicine to contribute to the effective allocation of scarce resources in rheumatology care. The main barriers reported by stakeholders were the scarcity of time resources in RMD care, the absence of physical examinations, and organizational challenges associated with the implementation of telemedicine in RMD care. While the exact integration of telemedicine into routine care has yet to be found, the consequences on the patient-physician relationship must be permanently considered.

Experiences and Perceptions of Older Adults with Lower-Risk Hormone Receptor-Positive Breast Cancer about Adjuvant Radiotherapy and Endocrine Therapy: A Patient Survey

Older patients with lower-risk hormone receptor-positive (HR+) breast cancer are frequently offered both radiotherapy (RT) and endocrine therapy (ET) after breast-conserving surgery (BCS). A survey was performed to assess older patients’ experiences and perceptions regarding RT and ET, and participation interest in de-escalation trials. Of the 130 patients approached, 102 eligible patients completed the survey (response rate 78%). The median age of respondents was 74 (interquartile range 71–76). Most participants (71%, 72/102) received both RT and ET. Patients felt the role of RT and ET, respectively, was to: reduce ipsilateral tumor recurrence (91%, 90/99 and 62%, 61/99) and improve survival (56%, 55/99 and 49%, 49/99). More patients had significant concerns regarding ET (66%, 65/99) than RT (39%, 37/95). When asked which treatment had the most negative effect on their quality of life, the results showed: ET (35%, 25/72), RT (14%, 10/72) or both (8%, 6/72). Participants would rather receive RT (57%, 41/72) than ET (43%, 31/72). Forty-four percent (44/100) of respondents were either, “not comfortable” or “not interested” in participating in potential de-escalation trials. Although most of the adjuvant therapy de-escalation trials evaluate the omission of RT, de-escalation studies of ET are warranted and patient centered.

Telemedicine and patient satisfaction during the COVID-19 pandemic: A case–control study of outpatient pediatric urology patients

Telemedicine has increased during the coronavirus disease 2019 pandemic. Our objective was to determine if patient satisfaction with telemedicine differed from in-person visits in an academic pediatric urology clinic. Following outpatient telemedicine and in-person pediatric urology visits, the validated NRC Health© Patient Survey was used to assess patient experience. Patient satisfaction was assessed on a 10-point scale with scores of 9–10 considered “satisfied” and 1–8 considered “not satisfied.” Satisfaction scores between telemedicine and in-person groups were compared using McNemar’s test, Wilcoxon signed rank test, and conditional logistic regression. Fifty-one patients had urology telemedicine visits during April–August 2020 and completed the NRC Health© Patient Survey. Propensity score matching was used to identify 102 in-person controls between January 2019 and March 2020. Ninety-two percent of telemedicine patients were satisfied compared to 87% of in-person patients (OR 1.7 95% CI [0.53–5.6]). Regression analysis adjusting for matching variables demonstrated that patient satisfaction was higher for telemedicine compared to in-person visits but was not statistically significant (OR 1.5 95% CI [0.43–5.6]). Patient satisfaction with telemedicine was similar to in-person visits in the pediatric urology clinic. Reduced waiting time and convenience associated with telemedicine visits provide an opportunity for telemedicine as a useful modality for pediatric urology.

P-OGC21 Patient perspectives on symptoms of importance and preferences for follow-up after major upper gastro-intestinal cancer surgery

Background Long-lasting symptoms and reductions in quality of life are common after oesophago-gastric surgery. Post-operative follow-up has traditionally focussed on tumour recurrence and survival, but there is a growing need to also identify and treat functional sequelae to improve patients’ recovery. Methods An electronic survey was circulated via a British national charity for patients undergoing oesophago-gastric surgery and their families. Patients were asked about post-operative symptoms they deemed important to their quality of life, as well as satisfaction and preferences for post-operative follow-up. Differences between satisfied and dissatisfied patients with reference to follow-up were assessed. Results Among 362 respondents with a median follow-up of 58 months since surgery (range 3-412), 36 different symptoms were reported as being important to recovery and quality of life after surgery, with a median 13 symptoms per patient. Most (84%) respondents indicated satisfaction with follow-up. Unsatisfied patients were more likely to have received shorter follow-up than 5 years (27% among unsatisfied patient vs. 60% among satisfied patients, p < 0.001and were less likely to have seen a dietitian as part of routine follow-up (37% vs. 58%, p = 0.005). Conclusions This patient survey highlights preferences with regard to follow-up after oesophago-gastrectomy. Longer follow-up and dietician involvement improved patient satisfaction. Patients reported being concerned by a large number of gastrointestinal and non-gastrointestinal symptoms, highlighting the need for multidisciplinary input and a consensus on how to manage the poly-symptomatic patient.

Severity of COVID-19: Causes and Consequences — From Obesity to Chronic Fatigue Syndrome

In circumstances of COVID-19 epidemiological uncertainty, the causes and consequences of the disease remain important issues. The aim of this study was to investigate obesity as a potential predisposition and chronic fatigue syndrome (CFS) as a possible consequence of COVID-19. The study was conducted in two parts: a theoretical part, in which a literature review was performed, and an empirical part, in which COVID-19 patient survey data were analysed. To identify the main findings regarding the relationship between obesity and COVID-19, the literature review was focused on the investigation of systematic reviews and meta-analyses by three databases — Med-line (via PubMed), Cochrane COVID-19 Study Register, and PROSPERO (International prospective register of systematic reviews). The patient survey was performed to investigate the relationship between obesity and severity of the disease, as well as the presence of CFS symptoms in COVID-19 patients in Latvia. The main findings of the literature review showed that obesity increases the risk of hospitalisation, disease severity, clinical complications, poor outcomes, and mortality. The results of the patient survey showed that overweight and obesity were more critical factors for men (males) suffering with COVID-19 than for women (females) in Latvia. The patient group with obesity caused almost half of all hospitalisations. The research data assumed that CFS patients were not a high-risk group for COVID-19, but COVID-19 caused CFS-like symptoms in patients and potentially increased the number of undiagnosed patients. In the context of further epidemiological uncertainty and the possibility of severe post-viral consequences, preventive measures are becoming increasingly important.