Alcohol-dependent patients manifest high rates of co-occurring psychiatric and somatic conditions, which are associated with impaired work productivity and HRQoL. The continued burden of illness observed in these already-diagnosed patients suggests an unmet need in both primary and secondary care.
Introduction: Myasthenia gravis (MG) is a rare, debilitating, chronic disorder caused by the production of pathogenic immunoglobulin G autoantibodies against the neuromuscular junction. A lack of real-world studies in rare diseases reflects a relatively limited understanding of the significant unmet needs and burden of disease for patients. We aimed to provide comprehensive real-world insights into the management and burden of MG from treating physicians in the United States (US). Methods: Data were collected using the Adelphi Real World MG Disease Specific Programme TM , a point-in-time survey of physicians and their patients with MG, in the US between March and July 2020. Physician-reported clinical data, including demographics, comorbidities, symptoms, disease history, treatments, and healthcare resource utilization, were collected. Results: In total, 456 patient record forms were completed by 78 physicians based in the US. At time of survey completion, patient mean age was 54.5 years. Mean time from symptom onset to diagnosis was 9.0 months (n = 357). Ocular symptoms were reported in 71.7% of patients. General fatigue affected 47.1% of patients and over half of those reported the severity as moderate or severe (59.5%, n = 128). Acetylcholinesterase inhibitors and/or steroids were the most frequently prescribed first-line treatment type among patients receiving treatment at time of survey completion and with moderate-to-severe symptoms (77.9%, n = 159/204). High-dose steroids (n = 14) and intravenous immunoglobulin (n = 13) were the most prescribed acute treatments among those receiving an acute treatment at time of survey completion (n = 36), with symptom exacerbations or myasthenic crises being the most common reasons for acute treatment. On average, 2.5 healthcare professionals were involved in patient management and 5.0 consultations were made per patient over the last 12 months. Conclusions: Our findings indicated that, despite treatment, there is a proportion of patients with MG in the US who had a significant need for improved disease management.
Background Myasthenia gravis (MG) is a rare, chronic, debilitating, unpredictable, and potentially life-threatening neuromuscular disease. There is a lack of real-world data on disease management that could be used to further understand and address unmet patient needs and burden. We aimed to provide comprehensive real-world insights in the management of MG in five European countries. Methods Data were collected using the Adelphi Real World Disease Specific Programme™ in MG, a point-in-time survey of physicians and their patients with MG in France, Germany, Italy, Spain, and the United Kingdom (UK). Physician- and patient-reported clinical data were collected, including demographics, comorbidities, symptoms, disease history, treatments, healthcare resource utilization (HCRU), and quality of life outcomes. Results In total, 144 physicians completed 778 patient record forms from March to July 2020 in the UK, and from June to September 2020 in France, Germany, Italy and Spain. Mean patient age at symptom onset was 47.7 years, with a mean time from symptom onset to diagnosis of 332.4 days (10.97 months). At diagnosis, 65.3% of patients were classified as Myasthenia Gravis Foundation of America Class II or above. Mean number of symptoms reported at diagnosis per patient was five, with ocular myasthenia reported in at least 50% of patients. At time of survey completion, the mean number of symptoms reported per patient was five and ocular myasthenia and ptosis were each still present in more than 50% of patients. Acetylcholinesterase inhibitors were the most commonly prescribed chronic treatments in all countries. Of 657 patients treated with chronic treatment at the time of the survey, 62% continued to experience moderate-to-severe symptoms. On average, 3.1 healthcare professionals (HCPs) were involved in patient management, 6.2 consultations were made per patient with any HCP over the last 12 months, and 178 (22.9%) patients were hospitalized in the last 12 months. Overall, HCRU and disease management were similar across all countries. Conclusions Our findings demonstrated the high burden of MG despite current treatment options for patients with MG.
Background: Chorea is recognized as a prototypic motor feature of Huntington’s disease (HD), but its effect on health-related quality of life (HRQoL) has not been fully explored. This study describes the impact of chorea on HRQoL in patients with HD. Objective: To determine the impact of HD-related chorea on employment, self-care activities, activities of daily living, and health-care resource utilization (HCRU). Methods: Data were drawn from the Adelphi HD Disease Specific Programme, a real-world point-in-time survey of 144 neurologists and 427 patients in the United States between July and October 2017. HD patients with and without chorea were identified and examined for differences in employment status, reasons for employment changes, self-care activities, and modifications to cope with involuntary movements. Bivariate tests and inverse probability weighted regression adjustment methods were used to determine differences in outcomes between patients with and without chorea. Results: HD patients with (n=287) and without (n=140) chorea were identified. Patients with chorea were less likely to be employed full-time (16.7% vs 25.7%; P <0.04) and more likely to be on long-term sick leave (17.4% vs 5.0%; P <0.01). The onset of motor symptoms in HD-related chorea patients coincided with a change in employment status (42.7% vs 20.8%; P <0.01). Among those still working (n=145), more than two-fifths of patients with chorea required changes to their workplace and required these changes more frequently (45% vs 17%; P <0.001). HD patients with chorea required aid to help them get around significantly more frequently than those without chorea (55% vs 34%; P <0.001). Discussion: These results demonstrate that HD patients with chorea experienced greater negative impact to employment, self-care activities, and HCRU than patients without chorea experienced. These patients were more likely to stop working due to motor, cognitive, and behavioral symptoms; require modifications in the home and workplace; and need more assistance from caregivers than patients without chorea. Conclusions: Patients with HD-related chorea have greater detriments to emotional, interpersonal, and professional functioning that could be improved by reducing chorea.
Background and Aims Immunoglobulin A nephropathy (IgAN) is the most common form of primary glomerulonephritis worldwide, with an estimated annual incidence of 25 cases per million people. Hematuria (tea coloured urine) and proteinuria (foamy urine) are among the most common clinical manifestations of IgAN, however the burden of such symptoms on diagnosis and disease severity is not fully understood. The aim of this analysis was to better understand patient reported symptoms prior to the first nephrologist consultation and diagnosis of patients with IgAN, in the United States, Europe, Japan, and China. Method Data were drawn from the Adelphi IgAN Disease Specific Programme (DSP), a point-in-time survey of IgAN-treating nephrologists and their consulting patients, conducted in the United States (US), Europe (EU5: France, Germany, Italy, Spain, United Kingdom [UK]), Japan, and China, between June and October 2021. Nephrologists completed structured online patient record forms for successive patients presenting with IgAN. Patients voluntarily completed questionnaires that corresponded with the nephrologist records, with questions about their IgAN on that day regarding demographics, clinical data, and signs and symptoms. Results A total of 991 patients with a nephrologist confirmed IgAN diagnosis completed a self-reported questionnaire. Mean (standard deviation [SD]) patient age was 42.1 (13.8) years, and 57% were male. Patients first noticed their IgAN symptoms at a mean (SD) age of 37.1 (13.1) years. Proteinuria (58%), hematuria (56%), and fatigue (36%) were the top patient-reported symptoms that prompted patients to see a doctor (n = 979). In the EU5, 64% of patients, and in the US 55% of patients, also reported high blood pressure as a sign that prompted them to see a doctor. Patients reported a median (interquartile range [IQR]) time of 12.9 (4.3-25.7) weeks from first experiencing IgAN symptoms to consulting with a doctor, ranging from 8.6 (4.3-25.7) weeks in Europe to 21.4 (4.3-52.1) weeks in Japan. Of those patients who reported a delay between experiencing symptoms and when they visited a doctor, 52% said that they were waiting to see if their symptoms would go away on their own (US: 64%, EU5: 68%, Japan: 34%, China: 49%) and 35% assumed they were just feeling tired or run down (US: 36%, EU5: 22%, Japan 39%, China 39%). Most patients reported first visiting either a family doctor/GP (49%) or a nephrologist (34%) for their IgAN symptoms. In China, 20% of patients reported seeing a urologist for their IgAN symptoms (US 0%, EU5 1%, Japan 6%). Median (IQR) patient-reported time from first doctor visit to IgAN diagnosis was 8.0 (4.0-16.0) weeks. This finding was consistent across geographical regions except in Japan, where median (IQR) duration was 12.0 (4.0-24.0) weeks. Waiting for test results (US: 31%, EU5: 35%, Japan: 34%, China: 68%), waiting to be tested (US: 52%, EU5: 59%, Japan: 37%, China: 54%), and waiting for a referral (US: 48%, EU5: 32%, Japan: 8%, China: 32%) were the top patient-reported reasons for a delay in the patient's IgAN diagnosis. At time of diagnosis, 47% of patients reported ‘mild’, 43% ‘moderate’, and 10% described ‘severe’ IgAN severity (n = 983). 23% of patients in the US reported severe IgAN severity at diagnosis (EU5 14%, Japan 11%, China 7%). Conclusion Despite experiencing symptoms, patients with IgAN waited several weeks before consulting a physician. Of those with a delayed diagnosis, over half described their IgAN as moderate or severe. Greater awareness of symptoms of kidney disease may lead to patients seeking help for their symptoms and getting a confirmed diagnosis more quickly. Improvement of the IgAN patient journey pathway in a health care system is important to expedite timely diagnosis of IgAN and subsequent management of the disease.
Background and Aims Immunoglobulin A nephropathy (IgAN) is the most prevalent form of primary glomerulonephritis worldwide with an estimated annual incidence of 25 cases per million people. Many patients are not diagnosed until they develop kidney dysfunction, such as hematuria and proteinuria. Proteinuria >1g/day is associated with a high risk of progression. The aim of the present study is to describe symptom control and consider treatment satisfaction for patients undergoing treatment for IgAN. Method Data were drawn from the Adelphi IgAN Disease Specific Programme (DSP)™, a point-in-time survey of IgAN-treating nephrologists and their consulting patients conducted in the United States (US), Europe (EU5: France, Germany, Italy, Spain, United Kingdom [UK]), Japan, and China, from June to October 2021. Nephrologists completed structured online patient record forms for successive patients presenting with IgAN including demographics, clinical characteristics and treatment patterns. Patients voluntarily completed questionnaires that corresponded with the nephrologist records, with questions about their IgAN on that day regarding symptoms experienced, disease severity, and treatment satisfaction. Patients who were receiving IgAN treatment at the time of survey are included in the presented analysis. Results The mean (standard deviation [SD]) age of patients (n = 869) was 42 (13.7) years and 57% were male. IgAN symptoms were deemed severe by 10% of patients at the time of survey and 14% before the initiation of their current treatment. Overall, 91% of patients reported having symptoms of IgAN at the time of survey, the most common were: foamy urine, feeling tired or lack of energy, and swelling, with many patients in China reporting hematuria. The top reported most bothersome symptoms were fatigue, swelling, and high blood pressure, with many patients in China also reporting proteinuria. Satisfaction with their current treatment was reported by patients; 61% reported that they were satisfied, 27% said they were neither satisfied or dissatisfied, and 12% said they were dissatisfied with their medication. This dissatisfaction was due to their current medication not working quickly enough (54%, n = 103) or that it had not helped with their IgAN symptoms (45%, n = 103). This was consistent with the physician-reported data; where proteinuria was reported across treatment lines (L), levels remained high amongst patients (mean (SD) proteinuria [g/day] at L1: 2.2 (2.3), n = 629; L2: 1.8 (2.0), n = 257; L3: 1.9 (1.4), n = 95) and estimated glomerular filtration rate (eGFR) continued to fall (mean (SD) eGFR [mL/min/1.73m2] at L1: 77.1 (29.6), n = 615; L2: 72.2 (30.3), n = 246; L3: 66.9 (31.1), n = 96). Conclusion Despite treatment, most patients with IgAN continued to experience symptoms. Many of these symptoms were considered bothersome by patients, including fatigue, swelling and high blood pressure which may have an impact on their everyday lives. These data highlight an unmet need for effective IgAN management and future treatments should aim to target better symptom relief and disease progression.
Background and Aims Although rare (estimated global annual incidence of 25 cases per million people), immunoglobulin A nephropathy (IgAN) is the most common form of primary glomerulonephritis. IgAN is associated with a poor prognosis, with 30% or more of patients with >1g/day of proteinuria progressing to kidney failure within 10 years. As poor prognosis is partly due to delayed diagnosis, this analysis aims to describe aspects of the diagnostic pathway for patients with IgAN. Method Data were drawn from the Adelphi IgAN Disease Specific Programme (DSP)™, a point-in-time survey of IgAN-treating nephrologists and their consulting patients, conducted in the United States (US), Europe (EU5: France, Germany, Italy, Spain, United Kingdom [UK]), Japan, and China, between June and October 2021. Nephrologists completed structured online patient record forms for successive patients presenting with IgAN, including demographics, clinical data, time to diagnosis, reasons for delayed diagnosis, and treatment history. A duration of greater than four weeks between first consultation with a physician and diagnosis was considered a delayed diagnosis. Results A total of 295 nephrologists completed records for 1537 patients which had data characterizing the time from first consultation and diagnosis. Mean (standard deviation (SD)) patient age was 43.0 (15.0) years, and 59% were male. At diagnosis, mean (SD) proteinuria (n = 1254) was 2.3 (2.4). Median time from first physician consultation to confirmed IgAN diagnosis varied by physician speciality. When patients initially consulted a nephrologist regarding their IgAN symptoms, median (interquartile range) duration from first consultation to diagnosis was 2.4 (0.7-5.6) weeks (n = 582). For family doctors/ General Practitioners (GPs)/ Primary Care Physicians (PCPs) (n = 656), this was 6.6 (3.0-13.8) weeks, for internal medicine/ internists (n = 120) and urologists (n = 113) the duration was 4.4 (1.3-9.4 and 1.5-12.5) weeks. Most patients were diagnosed by a nephrologist (US 95%, n = 263; EU5 96%, n = 491; Japan 99%, n = 236; China 98%, n = 547). A delayed diagnosis was found in 56% of patients. Across countries, waiting to conduct tests (41%), waiting for referral to a specialist (34%) and waiting for test results (32%) were the top three reasons for delay, except for Japan where 29% of patients were unable to consult sooner. A smaller proportion of patients who experienced a delay initially consulted with a nephrologist (27%) compared to those who did not experience a delay (52%). 78% of patients in China, diagnosed within 4 weeks, were diagnosed by a nephologist. Of the patients who experienced a delay in diagnosis, a higher proportion initially consulted a family doctor/ GP/ PCP (53%) compared to other physician types. In most cases, a kidney biopsy was used for diagnosis (US 89%, n = 263; EU5 81%, n = 491; Japan 98%, n = 236; China 85%, n = 547) and these were typically performed by a nephologist in most countries (US 59%, n = 233; EU5 81%, n = 397; Japan 95%, n = 231; China 97%, n = 464) except in the US, where 40% of cases were done by a radiologist. However, some patients were unable to undergo a biopsy (6%, n = 1531) and in some cases the physician chose to diagnose the patient using non-invasive methods (8%, n = 1531). Conclusion Most of the patients with IgAN in this survey were diagnosed by a nephrologist using a kidney biopsy. These data suggest that initially consulting a family doctor/ GP/ PCP led to a delayed diagnosis for patients. This may be due to the time taken for onward referral to a nephrologist. China had the highest proportion of patients diagnosed within 4 weeks from the first consultation (57%) and the highest proportion of patients who first visited a nephrologist regarding their symptoms. The results from this study suggest that speeding up referral from PCPs to nephrologists may reduce the amount of time taken to confirm IgAN diagnosis.
Background and Aims C3 glomerulopathy (C3G) is a rare form of glomerulonephritis, with an estimated incidence of 1 – 2 per million per year. C3G is associated with a high risk of disease progression with approximately 50% of patients reaching kidney failure within 10 years of diagnosis. Common signs and symptoms include proteinuria, hematuria, edema and hypertension. The aim of this analysis was to better understand the clinical characteristics of C3G patients from the US, Europe and Asia, at the time of diagnosis. Method An analysis was conducted using interim data from the Adelphi C3G Disease Specific Programme (DSP), a cross-sectional survey of C3G-treating nephrologists in US, EU5 (France, Germany, Italy, Spain, UK), China and Japan (study ongoing since August 2022; interim analysis based on data until November). Nephrologists completed structured forms administered via online links for consecutive patients presenting with C3G. The forms included demographic and clinical information including signs, symptoms, and lab values amongst others. Results In this interim analysis, 88 nephrologists had completed records for 277 patients in this survey, including 95 in US, 120 in EU5, 39 in China and 23 in Japan. Median patient age at diagnosis was 40.9, and 60% were male. 80% had C3 glomerulonephritis (C3GN) and 19% had dense deposit disease (DDD). Median proteinuria at diagnosis was 2.9 g/day, and was ≥1 g/day in 82% of patients. Median eGFR at diagnosis was 50 ml/min/1.73m2 (Table 1). In addition to proteinuria, the main clinical signs and symptoms at diagnosis were hematuria, edema, hypertension and fatigue. Pain, appetite loss and sleep problems were also reported. Physicians described disease severity at diagnosis as moderate in 53% and severe in 31% of patients. Conclusion This study allows evaluation of a rare disease across geographies. C3G patients experience substantial symptomatic and clinical burden at diagnosis. This symptom burden, high proteinuria, and relatively low eGFR is consistent with physician assessment that the disease is moderate or severe by the time of diagnosis. Facilitating early diagnosis of C3G and rapid initiation of treatment could be beneficial for patients in slowing disease progression.
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