The value of the patient perspective in understanding the full burden of migraine.
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ABSTRACT:
SUBMITTER: Wells RE
PROVIDER: S-EPMC8352044 | biostudies-literature |
REPOSITORIES: biostudies-literature
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Project description:IntroductionType 2 diabetes (T2D) is extremely burdensome to people with T2D and associated with impaired health-related quality of life. This study explores the impact of T2D and potentially relevant outcomes for new therapies using a unique approach to in-depth qualitative interviews where people with T2D are asked to think about their future with T2D.MethodsA cross-sectional qualitative interview study among people with T2D from the USA and UK. Interviews explored their treatment journey, perceptions of their future with T2D, and the value of achieving normoglycemia (explored through presentation of two vignettes with hypothetical medications that reduced hemoglobin A1c [HbA1c] levels < 7% and < 5.7%).ResultsPatients with T2D (N = 50; US n = 25; UK n = 25) were 66.0% male, had a mean body mass index (BMI) of 30.8 ± 6.3 kg/m2, and had a mean of 13.0 ± 10.0 years since diagnosis. Current diabetes treatments included diet and exercise only (8.0%), oral medications only (62.0%), oral plus injections (24.0%), and insulin only (6.0%). Despite being treated, participants reported over 25 different unmet needs related to their T2D across a broad range of domains. The most common concerns were diet, diabetes-related complications, weight changes, and psychological and emotional issues. A large majority of participants indicated that achieving lower HbA1c values would change their life. When reflecting on the value of improved glycemic control, patients primarily anticipated physical improvements and improved psychological well-being. When presented with two hypothetical treatments, about 70% of participants preferred the < 5.7% treatment option over the < 7% HbA1c treatment option.ConclusionsPeople with T2D have a high disease burden, a broad range of unmet needs, and extremely varied experiences and expectations on the impacts of T2D on their lives and future. Many patients indicated that achieving near normoglycemia would substantially change their lives primarily in terms of their physical and emotional health.
| S-EPMC8155797 | biostudies-literature
Project description:ObjectiveTo better characterize the ways that migraine affects multiple domains of life.BackgroundFurther understanding of migraine burden is needed.MethodsAdults with migraine randomized to mindfulness-based stress reduction or headache education arms (n = 81) in two separate randomized clinical trials participated in semistructured in-person qualitative interviews conducted after the interventions. Interviews queried participants on migraine impact on life and were audio-recorded, transcribed, and summarized into a framework matrix. A master codebook was created until meaning saturation was reached and magnitude coding established code frequency. Themes and subthemes were identified using a constructivist grounded theory approach.ResultsDespite most participants being treated with acute and/or prophylactic medications, 90% (73/81) reported migraine had a negative impact on overall life, with 68% (55/81) endorsing specific domains of life impacted and 52% (42/81) describing impact on emotional health. Six main themes of migraine impact emerged: (1) global negative impact on overall life; (2) impact on emotional health; (3) impact on cognitive function; (4) impact on specific domains of life (work/career, family, social); (5) fear and avoidance (pain catastrophizing and anticipatory anxiety); and (6) internalized and externalized stigma. Participants reported how migraine (a) controls life, (b) makes life difficult, and (c) causes disability during attacks, with participants (d) experiencing a lack of control and/or (e) attempting to push through despite migraine. Emotional health was affected through (a) isolation, (b) anxiety, (c) frustration/anger, (d) guilt, (e) mood changes/irritability, and (f) depression/hopelessness. Cognitive function was affected through concentration and communication difficulties.ConclusionsMigraine has a global negative impact on overall life, cognitive and emotional health, work, family, and social life. Migraine contributes to isolation, frustration, guilt, fear, avoidance behavior, and stigma. A greater understanding of the deep burden of this chronic neurological disease is needed to effectively target and treat what is most important to those living with migraine.
| S-EPMC8428538 | biostudies-literature
Project description:PurposeThe clinical manifestations of sarcoidosis vary widely, depending on the intensity of the inflammation and the organ systems affected. Hence, sarcoidosis patients may suffer from a great variety of symptoms. The aim of this study was to compare the self-reported burden of sarcoidosis patients in Denmark, Germany and the Netherlands, especially the prevalence of fatigue and small fiber neuropathy (SFN)-related symptoms, as well as differences in treatment strategies.MethodsA cross-sectional web-based anonymous survey about complaints was conducted among sarcoidosis patients. Patients were invited to take part through the sarcoidosis patient societies as well as through outpatient sarcoidosis clinics in these countries.ResultsThe questionnaire was completed by 1072 sarcoidosis patients (152 Danish, 532 German and 388 Dutch). Almost all patients reported having sarcoidosis-associated symptoms (organ-related as well as non-specific, non-organ related). Fatigue was reported by almost all respondents (90%), followed by pulmonary symptoms (72.4%). More than 50% of the respondents were being treated with prednisone, which was comparable in all three countries. In contrast, second- and third-line treatment differed substantially between Denmark, Germany and the Netherlands.ConclusionSarcoidosis patients in Denmark, Germany and the Netherlands present with similar self-reported symptoms, organ-related as well as non-specific, non-organ related. Fatigue (90%) and symptoms associated with SFN (86%) were highly prevalent in all three countries.
| S-EPMC6486948 | biostudies-literature
Project description:The aim of this study was to explore the need for a new disease-specific patient reported outcome (PRO) measure for use in clinical trials of drugs designed to target the underlying causes of polycystic ovary syndrome (PCOS), and in the process contribute to our understanding of the symptoms and impacts that define the patient experience with PCOS.Semi-structured interviews were conducted in 20 women diagnosed with PCOS according to the Rotterdam criteria who had not menstruated in the previous month. The relative importance of PCOS symptoms and impact concepts to patients was determined by analyzing the frequency of their expression in the interview transcripts. These insights were compared to clinicians' perceptions of PCOS.Pain- and discomfort-related symptoms accounted for the highest proportion (27.6%) of the 735 patient expressions, although clinicians did not consider pain to be important to patients with PCOS. The most frequently expressed individual symptoms were cramping (70% of patients; 14.7% of concepts), irregular menstruation (95% of patients; 12.2% of concepts), facial hair growth (75% of patients; 10.6% of concepts), heavy bleeding (70% of patients; 8.8% of concepts), infertility (70% of patients; 5.4% of concepts), and bloating (60% of patients; 5.2% of concepts). Cramping, heavy bleeding, and bloating were not identified by clinicians as being important to patients with PCOS. The impacts most frequently reported by patients with PCOS related to emotional well-being (e.g. anxiety/stress) and coping behaviors (e.g. acne medication, hair removal).The only validated PCOS-specific PRO, the PCOSQ, does not capture some key PCOS symptoms and impacts expressed by patients with PCOS, most notably those related to pain and discomfort, bleeding intensity and coping behaviours. Furthermore, some key PCOS symptoms may be under-recognized in the clinic.
| S-EPMC5562990 | biostudies-literature
Project description:ObjectivesWe explore the cost of care of type 2 diabetes mellitus (T2DM) using time-driven activity-based costing (TDABC) and connect that cost to resulting patient health outcomes.DesignWe construct six care pathways varying from low-risk to high-risk patients over a 12-month cycle of care. We collect time, resource and cost data on activities in each care pathway and compute a time-driven estimate of cost. Use of patient outcome data highlights the health outcomes achieved.SettingPrimary, secondary and tertiary care.ParticipantsMedical staff involved in the care of patients with T2DM.Primary and secondary measuresPrimary: resources consumed to provide T2DM care. Secondary: health outcomes for representative patient within each patient category.ResultsBy computing cost of T2DM care and associated complications of chronic kidney disease, active foot disease, moderate risk of active foot disease and myocardial infarction, we show that when patients develop acute complications, significant costs are incurred, as compared with the cost of maintaining a patient at low or moderate risk. Variance analysis further informs decision making by showing the need to have the right personnel doing the right tasks at the right time to control costs.ConclusionsA TDABC approach facilitates an understanding of the drivers of cost in chronic illness care. Our paper highlights the stages in the care pathway where different settings, decision making and a more optimal use of resources could assist with achievement of better patient outcomes.
| S-EPMC8762124 | biostudies-literature
Project description:BackgroundAcute allergic reactions (AARs) occur shortly after exposure to an allergen, and the severity is on a continuum. Systemic corticosteroids (CS) are mainstay treatment of moderate to severe AARs, whereas those at risk of the most severe AARs (ie, anaphylaxis) are also recommended prescription of epinephrine autoinjectors. There is limited research on the impact of AARs not fulfilling the criteria for anaphylaxis. We have characterized a sample with a history of moderate to severe AARs and evaluated their self-reported disease burden (ie, daily life impact, anxiety, and treatment impediments).MethodsSurvey study of adults with experience of AARs treated with CS. Participants recruited from a web-based panel and using social media were asked to complete a questionnaire related to their allergy and experience of AARs. The results were summarized for the whole sample and across subgroups with and without prescription of epinephrine.ResultsThe final study sample included 387 participants (80% women, mean age 41), of which 129 (33%) had at some point been prescribed epinephrine. The most common symptoms were respiratory (80%) and skin (78%) manifestations, and the mean (standard deviation, SD) self-rated severity score (scale from 0 [very mild] to 10 [very severe]) of the most recent AAR was 6.1 (2.0). More than 80% had experience of AARs interrupting daily activities and 50% of AARs that had limited work/studies or participation in leisure activities. Most of the respondents reported some degree of anxiety related to AARs and 43% had feared for their lives. Moreover, difficulties swallowing allergy medicine at an AAR was experienced by 26% and not having the medicine available when needed by 66%. Participants with prescription of epinephrine experienced more severe AARs than those without such prescription (mean [SD] severity 6.8 [2.1] vs 5.8 [1.8], p < 0.0001); however, also those without epinephrine prescription reported considerable anxiety and impact on daily life and to a similar degree as those with prescription.ConclusionsIn this sample, subjects with experience of AARs treated with CS showed a considerable disease burden with anxiety and interruption on daily life, as well as problems related to access to, and swallowing of, medication. Although respondents with epinephrine prescription had more severe disease, a high disease burden was also evident among those without epinephrine. The study increases the knowledge of people with moderate to severe AARs, a patient population that has previously been underrepresented in the research literature.
| S-EPMC10990897 | biostudies-literature
Project description:BackgroundMigraine is a disabling, chronic neurological disease leading to severe headache episodes affecting 13.2% of the Swedish population. Migraine leads to an extensive socio-economic burden in terms of healthcare costs, reduced workforce and quality of life (QoL) but studies of the health-economic consequences in a Swedish context are lacking. The objective of this study is to map the health-economic consequences of migraine in a defined patient population in terms of healthcare consumption, production loss and QoL in Sweden.MethodsThe study is based on data from a web-based survey to members in the Swedish patients' association suffering from migraine. The survey was conducted in May 2018 and included people with migraine aged 18 years or older. The survey included questions on health resource consumption, lost production resulting from migraine-related absenteeism and presenteeism, and QoL as measured by the EuroQol 5 dimensions questionnaire (EQ-5D-5 L) and the Headache Impact Test (HIT-6). The results are presented in yearly costs per patient and losses in quality adjusted life years (QALYs).ResultsThe results are based on answers from 630 individuals with migraine and are presented by number of migraine days per month. The total cost per patient and year increased with the number of migraine days per month (p < 0.001) and varied between approximately €5000 for those with less than 3 migraine days per month and €24,000 per year for those with 21-28 migraine days per month. Production loss represented the main part of the costs, approximately 80%. The average loss in QALYs per year also increased with the monthly number of migraine days (p = 0.023).ConclusionsMigraine leads to significant societal costs and loss of quality of life. There appears to be an unmet need and a potential for both cost savings and QoL benefits connected with a reduction in the number of migraine days.
| S-EPMC6734313 | biostudies-literature
Project description:Acid sphingomyelinase deficiency (ASMD), historically known as Niemann-Pick disease (NPD) types A, A/B, and B, is a rare, progressive, potentially fatal lysosomal storage disease with a spectrum of phenotypes. Little is known about how ASMD symptoms affect the lives of patients and their caregivers. In a cross-sectional qualitative study conducted in the US and UK, and in collaboration with the National Niemann-Pick Disease Foundation (US) and Niemann-Pick UK, we investigated the symptom experience of patients with ASMD types B and A/B and explored how the disease impacts their and their caregivers' lives. The study included 17 adult patients (mean age 38.7 years, 12 female), three caregivers of adults with ASMD, 12 pediatric/adolescent patients with ASMD (mean age 10.5 years, six female), and 12 caregivers of pediatric/adolescent patients with ASMD. The most commonly reported disease manifestations were respiratory (n = 26, 89.7%), abdominal (n = 25, 86.2%), and musculoskeletal symptoms (n = 23, 79.3%); excessive bleeding or bruising (n = 20, 69%); fatigue (n = 20, 69%); gastrointestinal symptoms (n = 18, 62.1%); and headache (n = 15, 51.7%). ASMD was reported to negatively impact patients' physical function (n = 23, 79.3%), self-esteem (n = 18, 62.1%), emotions (n = 16, 55.2%), social function and relationships (n = 16, 55.2%), and personal care (n = 9, 31%). Providing care for individuals with ASMD negatively affected caregivers' emotional well-being (n = 12, 80%), social function (n = 4, 26.7%), relationships (n = 6, 40%), and financial security (n = 7, 46.7%). The physical toll of providing care, the need for lifestyle changes, and the responsibility for making medical decisions added to the burden for caregivers. Alternatively, some caregivers noted that caring for a loved one enhanced their spirituality, providing them with a different outlook on life and a deeper personal resolve. This study showed that ASMD is a substantial burden for patients and caregivers, with long-term physical, emotional, social, and financial impacts. The study confirmed commonly known manifestations of ASMD, especially respiratory problems, but also identified less recognized ones, such as dermatological complications. The data collected and insight gained from this study should enhance clinical care, help evaluate new treatments, and inform health care decision making for patients with ASMD.
| S-EPMC8546120 | biostudies-literature
Project description:BackgroundMigraine is a neurological, primary headache disorder affecting more than 1 billion people worldwide, with a multi-faceted burden that can significantly impact the everyday life of a patient, both during and between attacks. However, studies on patient awareness, burden, and clinical management of migraine in Korea are limited and outdated. The aim of this study was to comprehensively investigate the current difficulties and unmet needs that Korean patients with migraine encounter from their perspective.MethodsA total of 207 patients with episodic or chronic migraine aged between 15 and 76 years, completed a survey designed to cover the following topics: diagnosis, understanding of the disease, treatment experience, disability, and quality of life. Patients were recruited by their neurologists from 11 specialized headache clinics in Korea and completed the survey between 22 July and 19 August 2019. Validated scales such as the Migraine Disability Assessment (MIDAS) questionnaire and Migraine-Specific Quality of Life Questionnaire version 2.1 (MSQv2.1) were used to assess levels of disability and quality of life, respectively, in patients.ResultsOn average, it took 10.1 years from onset of symptoms to diagnosis and a mean of 3.9 hospitals were visited for treatment prior to the patient's current hospital. There was a lack of understanding among respondents about migraine, with 55.6% believing that unilateral headache is a unique feature of migraine compared with other headache disorders. On average, high levels of disability and poor quality of life were reported by patients, as assessed by MIDAS and MSQv2.1, respectively, but only 23.7% had regularly taken preventive medication in the past. Overall satisfaction with previous doctor-patient relationships was reported by 29.5% of respondents, and satisfaction with preventive and acute medications by only 40.8% and 27.1% of the respondents, respectively.ConclusionKorean patients with migraine experience significant disability and reduced quality of life as a result of the disease and have clear unmet needs in terms of diagnosis, understanding of the disease, and disease management including treatment.
| S-EPMC8146656 | biostudies-literature
Project description:BackgroundSurvival for children with sickle cell disease (SCD) has improved significantly. However, patients with SCD still encounter several impediments to accessing adequate healthcare. Rural and medically underserved areas, such as parts of the Midwest, can exacerbate these barriers, separating children with SCD from subspecialists even further. Telemedicine has been a means to close these gaps in care for children with other special healthcare needs, but few studies have discussed how caregivers of children with SCD perceive its use.MethodsThe objective of this study is to understand the experiences of caregivers of pediatric SCD patients in a geographically diverse area in the Midwest in accessing care, and their perspectives of telemedicine. Caregivers of children with SCD completed an 88-item survey via a secured REDCap link either in-person or via secure text. Descriptive statistics (means, medians, ranges, frequencies) were performed for all responses. Univariate chi square tests were performed to analyze associations, particularly with telemedicine responses.ResultsThe survey was completed by 101 caregivers. Nearly 20% of families traveled more than 1 hour to reach the comprehensive SCD center. Other than their SCD provider, caregivers reported their child having at least 2 other healthcare providers. Most barriers caregivers identified were financial or resource based. Almost a quarter of caregivers expressed feeling as though these barriers impacted their and/or their child's mental health. Ease of access to team members, as well as scheduling, were common facilitators of care cited by caregivers. The majority were willing to participate in telemedicine visits, regardless of how far they lived from the SCD center, though many noted aspects requiring adaptation.ConclusionThis cross-sectional study describes barriers to care experienced by caregivers of children with SCD, regardless of proximity to an SCD center, as well as caregiver perceptions of the usefulness and acceptability of telemedicine for SCD care.
| S-EPMC10189684 | biostudies-literature