Humanistic Burden Research Articles

Humanistic Burden of Rare Kidney Diseases: Understanding the Impact of IgA Nephropathy (IgAN) and FSGS on Patients and Care Partners Study (HONUS): Preliminary Results for FSGS in the United States (US)

Humanistic Burden of Rare Kidney Diseases: Understanding the Impact of IgA Nephropathy (IgAN) and FSGS on Patients and Care Partners Study (HONUS): Preliminary Results for FSGS in the United States (US)

Quality of life and healthcare resource use in patients with bladder pain syndrome: A survey of UK patients

Objectives: The main aim of this study is to characterise the humanistic burden of bladder pain syndrome (BPS) on patients within the United Kingdom and to estimate the key healthcare resource usage and cost of treating these patients. Method: An online survey was administered through two charities. Respondents were asked questions on BPS diagnosis, socio-demographics and health-related quality of life using the O’Leary-Sant Interstitial Cystitis Symptom Index (ICSI) and Problem Index (ICPI), the Pelvic Pain and Urinary/Frequency (PUF) and the EQ-5D questionnaires. Healthcare resource and therapy use, for the previous 6 months, were recorded and used to calculate economic burden. Results: A total of 252 patients completed the survey. Mean ICSI ICPI scores were 12.6 and 11.0, respectively, indicating severe symptoms. Mean PUF score was 21.2, indicating poor health status and a high number of BPS-related symptoms and problems. Mean EQ-5D utility score was 0.541. In the previous 6 months, 172 (68%) saw their general practitioner (GP) and 80 (32%) a primary care nurse, and 165 (65%) had one or more outpatient visits, due to their BPS. Conclusion: This survey adds to our current understanding of BPS in the United Kingdom, highlighting that patients with BPS have poor health-related quality of life and incur high resource use. Level of evidence: Not applicable

Breathing New Life Into Acute Respiratory Care: Proactively Improving Long-Term Outcomes

Admissions due to asthma contribute substantially to the burden faced by emergency departments (ED) worldwide, with a considerable impact arising from the high number of readmissions among patients with severe asthma. Repeated ED readmittance not only places considerable demand on healthcare resources, but also increases the humanistic burden on patients through reduced lung function, decreased quality of life, and increased exposure to systemic corticosteroids (SCS) and oral corticosteroids (OCS). In addition, patients are subject to the increased morbidity and mortality risk, and quality of life deficit associated with repeated asthma exacerbations. Admission to the ED should be seen as an opportunity to break this readmission cycle and prevent further admissions, while offering patient-centric benefits, such as investigation into the underlying causes of disease, and optimisation of care to prevent further exacerbations. Actions that require no additional resource may be taken directly in the ED, including biomarker tests among routine blood tests, or teaching inhaler technique as part of patient education and safety-netting. In addition, patient discharge may be considered as an opportunity for improving guidance implementation and breaking the cycle of readmission. Unlike emergency cardiac care, where >90% of patients are discharged on secondary prevention drugs and 85% of patients are referred to follow-up rehabilitation, guidelines for care following an ED visit for asthma are not always followed. Furthermore, current tools designed to accelerate specialist referral are not always rigorously implemented following an ED visit, meaning that follow-up may be delayed. Finally, further efforts should be made to identify high-risk patients in the community earlier in the disease pathway, allowing timely intervention before further lung function impairment, or the onset of adverse events due to OCS over-exposure. This article summarises an AstraZeneca-sponsored symposium delivered on 12th September 2023, as part of the European Respiratory Society (ERS) International Congress in Milan, Italy. The faculty, consisting of David Price, Head of the Observational and Pragmatic Research Institute, Singapore; Mona Al-Ahmad, Consultant Allergist and Clinical Immunologist at the Ministry of Health in Kuwait; and Mohit Bhutani, Professor of Medicine at the University of Alberta, Edmonton, Canada, each gave a brief presentation on proactive strategies to improve long-term outcomes in acute respiratory care. During panel discussions following each presentation, Anne Marie Marley, Respiratory Nurse Consultant from Belfast Health and Social Care Trust, UK, provided examples of implementing transition of care by bridging hospital and community care settings.

THU394 Disease Burden Of X-Linked Hypophosphatemia Focused On The United States And Canada: A Targeted Literature Review

Abstract Disclosure: Z. Li: Employee; Self; Employee of Kyowa Kirin, Inc., Princeton, NJ, USA. O. Zaidi: Other; Self; Employee of OPEN Health and received funding to complete this research. C. Chukwu: Other; Self; Employee of OPEN Health and received funding to complete this research. H. Heerssen: Other; Self; Employee of Kyowa Kirin, Inc., Princeton, NJ, USA. Y. Zhao: Other; Self; Employee of Kyowa Kirin, Inc., Princeton, NJ, USA. A. Dale: Other; Self; Employee of Kyowa Kirin, Inc., Princeton, NJ, USA. M. Bernauer: Other; Self; Employee of OPEN Health and received funding to complete this research. Introduction: X-linked hypophosphatemia (XLH) is a rare genetic musculoskeletal disease and the most common form of heritable hypophosphatemic rickets. The objective of this review was to summarize the disease burden and treatment patterns of XLH in the United States (US) and Canada. Methods: Publications from January 1, 2015, to June 3, 2022, were searched using the Medline, Embase, and EconLit databases. Observational studies reporting epidemiology, humanistic burden, economic burden, and treatment patterns in the US and Canada were included. Evidence on epidemiology, humanistic burden, and treatment patterns was expanded to other countries as limited US and Canada studies were identified. Results: A total of 1,218 publications were screened; 42 publications were included (6 from US or Canada; 36 did not report location or were from other countries). Epidemiology data (i.e., incidence, prevalence, mortality) were limited for patients with XLH in the US and Canada. The estimated number of people in the US with XLH was <50,000 in 2021. Globally, the incidence of XLH was estimated at 3.9 per 100,000 live births, and the prevalence ranged from 1.4 per 100,000 to 4.8 per 100,000. One United Kingdom-based study estimated an XLH mortality rate of 12.1 per 1,000 person-years. Sixteen publications reported clinical manifestations of XLH, which showed a high level of heterogeneity. Among children with XLH, frequent clinical manifestations (>80% in 1 or more publications) included active rickets, diminished height, gait disturbance, bone or joint pain, and leg bowing. Among adults with XLH, frequent clinical manifestations (>80% in 1 or more publications) included short stature, leg deformity, musculoskeletal pain, fatigue, osteoarthritis, enthesophytes, gait disturbance, dental abscesses, and joint stiffness or restricted range of motion. Humanistic burden data from the US was limited to 2 non-trial publications, which used, respectively, the 36-Item Short Form Survey (SF-36) and the Knee Injury and Osteoarthritis Outcome Score-Physical Function Shortform (KOOS-PS) instruments. These 2 publications reported lower health-related quality of life for patients with XLH compared with the general population. No publication was identified on economic burden, treatment patterns, or XLH guidelines in the US or Canada. A consensus statement from a US panel of experts recommends individualization of monitoring and disease management due to clinical heterogeneity. Conclusions: There are limited published data on disease burden or treatment patterns for XLH in the US or Canada. Further research should consider the epidemiological, humanistic, and economic burden of XLH among pediatric and adult populations in these countries. Presentation: Thursday, June 15, 2023

Economic and Humanistic Burden Among Medicare-Aged Women With Fragility Fracture in the United States.

Describe patient characteristics, health care resource utilization, costs, and humanistic burden of women with Medicare insurance with incident fragility fracture who were admitted to post-acute-care (PAC). Retrospective cohort study using 100% Medicare Fee-for-Service (FFS) data. Community-dwelling female Medicare beneficiaries with incident fragility fracture January 1, 2017, to October 17, 2019, resulting in PAC admission to a skilled nursing facility (SNF), home-health care, inpatient-rehabilitation facility, or long-term acute-care hospital. Patient demographic/clinical characteristics were measured during 1-year baseline. Resource utilization and costs were measured during baseline, PAC event, and PAC follow-up. Humanistic burden was measured among SNF patients with linked Minimum Data Set assessments. Multivariable regression examined predictors of PAC costs after discharge and changes in functional status during SNF stay. A total of 388,732 patients were included. Compared with baseline, hospitalization rates were 3.5, 2.4, 2.6, and 3.1 times higher and total costs 2.7, 2.0, 2.5, and 3.6 times higher for SNF, home-health, inpatient-rehabilitation, and long-term acute-care, respectively, following PAC discharge. Utilization of dual-energy X-ray absorptiometry (DXA) and osteoporosis medications remained low: 8.5% to 13.7% received DXA during baseline vs 5.2% to 15.6% following PAC; 10.2% to 12.0% received osteoporosis medication during baseline vs 11.4% to 22.3% following PAC. Dual eligibility for Medicaid (ie, low income) was associated with 12% higher costs; Black patients had 14% higher costs. Activities of daily living scores improved 3.5 points during SNF stay, but Black patients had 1.22-point lower improvement than White patients. Pain intensity scores showed small improvement (-0.8 points). Women admitted to PAC with incident fracture had high humanistic burden with little improvement in pain and functional status and significantly higher economic burden after discharge compared with baseline. Disparities in outcomes related to social risk factors were observed, with consistently low utilization of DXA and osteoporosis medications even after fracture. Results indicate a need for improved early diagnosis and aggressive disease management to prevent and treat fragility fractures.

P362 Humanistic burden of neurodegenerative lysosomal disorders in the US: insights from caregivers of patients living with GM1 and GM2 gangliosidoses

P362 Humanistic burden of neurodegenerative lysosomal disorders in the US: insights from caregivers of patients living with GM1 and GM2 gangliosidoses

Treatment landscape and burden of disease in metastatic castration-resistant prostate cancer: systematic and structured literature reviews.

Metastatic castration-resistant prostate cancer (mCRPC) is a lethal disease that imposes a major burden on patients and healthcare systems. Three structured literature reviews (treatment guidelines, treatment landscape, and human/clinical/patient burden) and one systematic literature review (economic burden) were conducted to better understand the disease burden and unmet needs for patients with late-stage mCRPC, for whom optimal treatment options are unclear. Embase®, MEDLINE®, MEDLINE® In-Process, the CENTRAL database (structured and systematic reviews), and the Centre for Reviews and Dissemination database (systematic review only) were searched for English-language records from 2009 to 2021 to identify mCRPC treatment guidelines and studies related to the treatment landscape and the humanistic/economic burden of mCRPC in adult men (aged ≥18 years) of any ethnicity. In total, six records were included for the treatment patterns review, 14 records for humanistic burden, nine records for economic burden, three records (two studies) for efficacy, and eight records for safety. Real-world treatment patterns were broadly aligned with treatment guidelines and provided no optimal treatment sequencing beyond second line other than palliative care. Current post-docetaxel treatments in mCRPC are associated with adverse events that cause relatively high rates of treatment discontinuation or disruption. The humanistic and economic burdens associated with mCRPC are high. The findings highlight a lack of treatment options with novel mechanisms of action and more tolerable safety profiles that satisfy a risk-to-benefit ratio aligned with patient needs and preferences for patients with late-stage mCRPC. Treatment approaches that improve survival and health-related quality of life are needed, ideally while simultaneously reducing costs and healthcare resource utilization.

Humanistic and Economic Burden of Patients with Cardiorenal Metabolic Conditions: A Systematic Review.

Diabetes is associated with significant economic burden. Moreover, cardiovascular disease (CVD), including heart failure, and chronic kidney disease (CKD) are common comorbidities, leading to premature mortality. We conducted a systematic review to assess the humanistic and economic burden of cardio-renal-metabolic (CRM) conditions in individuals ≥ 18 years with CVD, CKD, and type 2 diabetes mellitus. We searched Embase® and Medline® databases from 2011 to January 10, 2022 for English publications reporting humanistic and economic burden outcomes from observational studies, real-world evidence, and economic model studies. Intervention and validation studies were excluded. Study quality was assessed using the Newcastle-Ottawa Scale. Abstracts/posters were identified from four conferences (2020-2022). Of 1804 studies identified, 22 (including four conference publications) were selected involving 351,296,930 participants (one modeled the US population); eight reported healthcare resource utilization (HCRU), seven only cost data, six HCRU and cost data, one reported quality-of-life data (11/18 and 7/18 had estimated low and medium risk of bias, respectively). Participants were predominantly ≥ 65 years and identified as having White ethnicity. Higher costs and HCRU were observed in patients with all three conditions compared to those with two or none. Urban/metropolitan and insured patients had higher healthcare expenditure and service utilization compared to uninsured and racial/ethnic minority populations. Comorbidities were associated with increased hospitalizations, higher costs, and more emergency department visits. In general, patients identified as having Black ethnicity had low odds of using healthcare services, possibly due to disparities in healthcare access and distrust in the system. Limitations included no adjustment for inflation and a predominance of retrospective studies. This review showed a greater economic burden for patients with CRM conditions, with a clear trend between increasing numbers of comorbidities and increasing healthcare costs/resource use. Comparisons between countries are complicated and the scarcity of evidence from minority racial and ethnic groups and lack of data from non-US geographies warrant further investigation.

Development of a health economic model to evaluate the cost-effectiveness of roxadustat in treating anemia associated with non-dialysis-dependent chronic kidney disease

BackgroundTreatment for anemia of chronic kidney disease (CKD) largely consists of erythropoiesis-stimulating agents (ESAs) with iron supplementation. Although ESAs are well-established and efficacious, their use has been associated with considerable economic and humanistic burdens. Roxadustat, an oral medication, is a hypoxia-inducible factor prolyl hydroxylase inhibitor that targets multiple causes of CKD and has a similar efficacy and safety profile to ESAs. The cost-effectiveness of this treatment, however, has yet to be investigated.ObjectiveThe study objective was to develop a health economic model to evaluate the cost-effectiveness of roxadustat compared with ESAs for treating anemia of non-dialysis-dependent (NDD) CKD.MethodsA cohort-based model was developed for a hypothetical cohort of 1,000 patients with anemia of NDD CKD, incorporating eight health states, representing the hemoglobin level of each patient. The model was informed by individual patient-level data from the roxadustat global phase 3 clinical trial program. Total and incremental costs as well as quality-adjusted life-years (QALYs) associated with roxadustat versus ESAs were estimated from the perspective of the UK National Health Service. Sensitivity analyses were performed to assess the robustness of the model. Analyses exploring alternative scenarios were also conducted.ResultsOn a per-person basis, over 1,000 simulations, roxadustat was found to be on average less costly (−£32) and more effective (+0.01 QALYs) than ESAs, with a dominant incremental cost-effectiveness ratio. The probability of cost-effectiveness at a £20,000 per QALY willingness-to-pay threshold from the UK perspective was 67%.ConclusionThe model developed may be a useful instrument that, alongside expert clinical opinion, can inform clinical and policy decision-making regarding treatment of anemia of NDD CKD. The model highlights the cost-effectiveness of roxadustat, as well as its potential to have a meaningful impact in reducing the burden of anemia of NDD CKD.

Burden of Medication Overuse in Migraine: A Cross-sectional, Population-Based Study in Five European Countries Using the 2020 National Health and Wellness Survey (NHWS).

Overuse of medication to treat migraine attacks can lead to development of a new type of headache or significant worsening of pre-existing headache, known as medication overuse headache. However, data concerning the burden of medication overuse (MO) in migraine are limited. This study aimed to assess the humanistic burden of MO in individuals with migraine from five European countries. Data are from the 2020 National Health and Wellness Survey-a cross-sectional, population-based survey conducted in France, Germany, Italy, Spain, and the UK. Data were included from adults (≥ 18years) with a self-reported diagnosis of migraine and at least one migraine attack and one headache in the past 30days. MO was defined as (i) use of simple analgesics/over-the-counter medications on ≥ 15days/month; or (ii) use of migraine medication, including combination analgesics, on ≥ 10days/month. Humanistic burden of MO was assessed using the 12-Item Short-Form Health Survey (SF-12v2), EuroQol 5-Dimensions 5-Levels (EQ-5D), Short-Form 6-Dimensions (SF-6D), and Migraine Disability Assessment (MIDAS). The association of MO with humanistic burden was evaluated using generalized linear models adjusted for potential confounders in the full migraine population and in subgroups defined by headache frequency (monthly headache days [MHDs] 1-3, 4-7, 8-14, or ≥ 15). Among individuals with migraine, humanistic burden (SF-12v2, SF-6D, EQ-5D, and MIDAS) was higher in individuals who reported MO (n = 431) versus no MO (n = 3554), even after adjustment for confounding variables (p < 0.001 for all measures). MIDAS and EQ-5D scores were higher in individuals with MO than without, at all levels of headache frequency. For SF-12v2 and SF-6D, differences between groups with/without MO were seen only at lower levels of headache frequency (MHD 1-3 and 4-7). Among people with migraine, those who report MO face a greater humanistic burden than those without MO, irrespective of headache frequency.

P-478 Humanistic, clinical, and economic burden in patients with relapsed or refractory multiple myeloma on second line or higher therapy: a systematic literature review

P-478 Humanistic, clinical, and economic burden in patients with relapsed or refractory multiple myeloma on second line or higher therapy: a systematic literature review

Burden of untreated transthyretin amyloid cardiomyopathy on patients and their caregivers by disease severity: results from a multicenter, non-interventional, real-world study.

The humanistic burden of transthyretin amyloid cardiomyopathy (ATTR-CM) is poorly defined. An international study to comprehensively characterize the burden of ATTR-CM on patients naïve to disease-modifying therapy and their unpaid primary caregivers using study-specific and established surveys (patients: Kansas City Cardiomyopathy Questionnaire Overall Summary [KCCQ-OS], 12-Item Short Form Health Survey [SF-12], Hospital Anxiety and Depression Scale [HADS], Patient-Reported Outcomes Measurement Information System [PROMIS] Fatigue and Dyspnea; caregivers: SF-12, HADS, PROMIS Fatigue, Zarit Burden Interview [ZBI]). All data were summarized descriptively. 208 patient and caregiver pairs were included. 86% of patients were male, median age was 81 years, and 91% (141/155 with genetic testing) had wild-type ATTR-CM. Patient responses characterized the mental and physical burden of ATTR-CM, which was numerically higher among those who were New York Heart Association (NYHA) class III (n = 43) vs. class I/II (n = 156). NYHA class III patients had particularly low KCCQ-OS (36) and SF-12 physical component (27) scores, and 67% had a HADS depression score ≥8. Caregivers (median age 68 years; 85% female; 59% spouse of the patient; median duration of caregiving 1.5 years) reported that NYHA III patients more frequently required help with a range of physical activities than NYHA class I/II patients. 51% of caregivers to NYHA class III patients reported at least a mild-to-moderate burden in the ZBI. A plain language summary of this paper can be found as a supplemental material. Untreated ATTR-CM is a burden to both patients and their caregivers.

The humanistic and economic burden of work-related musculoskeletal pain: a cross-sectional survey of workers in the United Kingdom.

The aim of this study was to evaluate the impact of work-related musculoskeletal (MSK) lower body pain on health-related quality of life (HRQoL) and work productivity in a large sample of workers in the United Kingdom, as well as evaluating the potential economic impact of MSK pain. Participants with self-reported work-related MSK pain were recruited from an online panel maintained by a third party (Qualtrics LLC). Participants completed three validated instruments online: the Brief Pain Inventory (BPI), the Assessment of Quality of Life Instrument (AQoL-4D), and the 6-item Work Productivity and Activity Impairment Questionnaire (WPAI). Sociodemographic details, work patterns and healthcare resource utilisation were also reported. One-way analysis of variance (ANOVA) and t-tests were used to explore differences between variables. Linear regression was applied to determine the impact of work-related MSK pain on HRQoL. All 1035 recruited participants completed the survey (57.4% female; mean age 43.4 years). Participants reported spending all (25.2%) or most (53%) of their time at work on their feet. Mean pain severity was 4.63 (standard deviation: 2.07); mean pain interference was 4.37 (2.49). There was a linear relationship between length of shift, time on feet and pain. Mean AQoL-4D scores were 0.609 (0.254). A mean of 4.12h was lost per week due to pain. Absenteeism (last 7 days) was 9.5% (20.7%), and presenteeism 33.3% (24.9%). An average 1.55 visits were made to family practitioners (total cost: £19,866) and 1 hospital visit (£37,320) due to work-related MSK pain. This study demonstrated that work-related lower body pain has a significant impact in terms of individual HRQoL and as an economic societal burden.

Physician Perceptions of the Clinical, Economic and Humanistic Burden of Disease and Unmet Needs in Myelofibrosis: Preliminary Findings from the MPN Landmark Survey in Indonesia.

A cross-sectional survey was designed and administered between November and December 2021 among practicing Haematologists-Medical Oncologists who are experienced in treating patients with MF. The objectives of the survey were to assess physician's understanding of the overall diagnosis of MF, the disease burden, current treatment practices and remaining unmet needs. Outcomes were analysed descriptively. The survey was completed by 30 respondents. The findings suggest that symptom burden is high and has a significant negative impact on quality-of-life. Treatment burden is also high which can result in high healthcare resource utilisation. Physicians expressed need for novel therapeutic options and improved access and coverage for such options. There is also a need to improve access and coverage for JAK V617F testing locally and local hospital infrastructures should be upgraded to ensure MF is accurately diagnosed. Patient may benefit from information on MPN, which may result in earlier presentation, diagnosis and treatment which can improve outcomes. The findings align with previous international research reporting that symptoms and treatment burden are high, and that novel therapeutic options are needed. Additional patient research might be required to better understand the patient experience of MF and how this can be improved.

Patient Experiences with Clostridioides difficile Infection and Its Treatment: A Systematic Literature Review.

Clostridioides difficile infection (CDI) is a globally recognized cause of morbidity and mortality with devastating effects on health-related quality of life (HRQoL). The objective of this study was to conduct the first systematic literature review (SLR) to assess the humanistic burden of CDI on patient experiences, including HRQoL and related constructs, and attitudes towards treatment alternatives. An SLR was conducted to identify peer-reviewed articles that assessed CDI, including recurrent CDI (rCDI), and patient-reported outcomes or HRQoL. PubMed, Embase, and the Cochrane Collaboration abstracting services were used to conduct literature searches from 2010 to 2021 in the English language. This SLR was conducted in accordance with Preferred Reporting Items for Systematic Reviews and Meta-analyses (PRISMA) criteria. Of 511 identified articles, 21 met study inclusion criteria. The SLR showed CDI has a devastating impact on patients' overall HRQoL that continues well beyond infection clearance. The impact of CDI on physical, emotional, social, and professional well-being rivaled abdominal symptoms of uncontrollable diarrhea, being worse for patients with rCDI. Patients with CDI feel isolated, depressed, lonely, and continue to be frightened of recurrences as well as being contagious to others. Most believe that they will never be free of CDI. CDI and rCDI are debilitating conditions affecting physical, psychological, social, and professional functioning of patients' HRQoL, even long after the event has occurred. The results of this SLR suggest that CDI is a devastating condition in need of better prevention strategies, improved psychological support, and treatments that address the microbiome disruption to break the cycle of recurrence. Additional safe and effective therapies are needed to address this unmet medical need.

HUMANISTIC BURDEN OF INFORMAL CAREGIVERS OF CHILDREN AND YOUNG ADULTS WITH NEWLY DIAGNOSED TYPE 1 DIABETES (T1D): A SYSTEMATIC LITERATURE REVIEW (SLR)

OBJECTIVES: To describe the humanistic and economic burden of informal caregivers of children, adolescents, and young adults newly diagnosed with T1D.&#x0D; METHODS: An SLR was conducted in MEDLINE®, Embase®, and PsycInfo® (search date: December 7, 2021) to identify clinical and observational studies assessing the humanistic burden experienced by informal caregivers (i.e., persons who provide unpaid care) of children, adolescents, and young adults aged 6 to 21 who were diagnosed with T1D within three months.&#x0D; RESULTS: Four prospective cohort studies and one randomized controlled trial (RCT) were included. Caregiver sample size ranged from 59 to 191. Duration of T1D was 2 to 6 weeks. Among parents of children aged ≤11 years, mothers had a moderate posttraumatic stress disorder symptom severity (mean score: 11.9; standard deviation [SD]: 7.4), while fathers experienced mild severity (8.4; SD: 6.2; p &lt; 0.001; Posttraumatic Diagnostic Scale). Mothers experienced clinically significant symptoms (defined as score &gt;5) of anxiety (mean score: 7.5), social dysfunction (7.5), and somatic symptoms (5.1; General Health Questionnaire [GHQ]-28). Fathers showed clinically significant anxiety (5.5) and social dysfunction (7.7; GHQ-28) only. Results from the RCT indicated a mean hypoglycemia fear score of 46.6 out of 108 at diagnosis and 34.9 after 14 weeks, with a higher score indicating greater fear (Hypoglycemia Fear Survey-Parents). Parents of children aged &gt;11 years experienced clinically significant social dysfunction (mean score for mothers/fathers: 7.6/7.1) and anxiety (mothers: 5.4; GHQ-28). Parents experienced moderate to extreme diabetes-specific stress (defined as score ≥5; mean score: 6.3; SD: 1.9) at diagnosis, and no to moderate stress (4.5; SD: 2.1) at 3 months. None of the included studies reported on economic burden.&#x0D; CONCLUSIONS: This SLR highlights parents of children and young adults with newly diagnosed T1D experience caregiver burden, with a greater burden among mothers than fathers. Due to the paucity of evidence, further studies are warranted to better understand the humanistic burden among this group of informal caregivers.

1011-P: Humanistic and Economic Burden of Patients with Cardiorenal Metabolic Conditions—A Systematic Literature Review

Background: A comprehensive understanding of the burden of CRM conditions is needed to guide targeted interventions. Objective: This SLR assessed the humanistic and economic burden of T2D, CVD (including HF), and CKD. Methods: Following PRISMA guidelines, Embase and Medline databases were systematically searched to identify observational, RWE, and economic-model studies in populations ≥18 years old with CKD, T2D, and CVD. Outcomes of interest included humanistic (e.g., health related QoL, functional impairment, QALY) and economic burden. English-language studies from Jan 2011 to Jan 2022 were included. Relevant abstracts/posters also were identified from ACC, AHA, ASN, and ADA from the 2 previous years. Results: Of 22 studies identified (13 US; 9 non-US) and included, 1 Chinese study reported QoL data (depressive symptoms of CKD and T2D), 8 reported healthcare resource utilization (HCRU), 7 reported cost, and 6 reported HCRU and cost. Multimorbidity and advanced stages of CKD were associated with higher medical costs and HCRU for patients with CRM across all studies. Increasing age was associated with more comorbidities, leading to greater economic burden among patients ≥65 years old. Higher costs and HCRU were observed in patients with all 3 conditions (T2D, CVD, CKD) compared to those with fewer comorbidities. Higher healthcare expenditures and HCRU was observed among urban insured compared with uninsured populations. Black patients had lower odds of using healthcare services compared to patients who identified as White, Asian/Pacific Islander, or Hispanic ethnicity. Conclusions: This SLR identified a clear trend of increasing healthcare costs and HCRU commensurate with an increasing number of CRM comorbidities. Further research evaluating the economic and humanistic burden of CRM conditions, especially in patients from racial/ethnic groups, is needed. Disclosure K.Ferdinand: Consultant; Amgen Inc., Boehringer Ingelheim Inc., Novartis, Medtronic, Pfizer Inc. K.C.Norris: None. H.W.Rodbard: Advisory Panel; Merck Sharp &amp; Dohme Corp., Pacira BioSciences, Inc., Research Support; Eli Lilly and Company, Medtronic, Sanofi, Novo Nordisk. J.Trujillo: Advisory Panel; Novo Nordisk, Sanofi. Funding Boehringer Ingelheim and Eli Lilly and Company Diabetes Alliance

396-P: The Humanistic Burden of Type 1 Diabetes in U.S. Adults With or Without Recurrent Severe Hypoglycemic Events—A Qualitative Interview Study

Introduction: Despite advances in technology, many people with type 1 diabetes (T1D) still experience severe hypoglycemic events (SHEs). This study explored the perceived humanistic burden of T1D in adults using continuous glucose monitor (CGM) in the US with or without recurrent SHEs. Methods: In 60-minute semi-structured interviews, adult CGM users with self-reported T1D diagnosis (≥5 yrs.) described the burden of TID on quality of life (QoL). Participants ranked the ‘top 3’ negative impacts of T1D on QoL. Subgroup analyses were conducted in participants with recurrent SHE (rSHE; ≥2 SHEs last yr.) and without recurrent SHE (nSHE; ≤1 SHEs last yr.). Results: Participants (N=31; 42% rSHEs, 58% nSHEs) were 74% female and 81% were married. Mean age of T1D diagnosis was 28 yrs. Median SHEs in the past year in the rSHE group were 3 and in the nSHE group were 0. Both groups had mean HbA1c of 7.1%. rSHE and nSHE participants used hybrid closed loop systems (hCLS) (53.8% vs. 44.4%), multiple daily injections (23.1% vs. 33.3%) and continuous subcutaneous insulin infusion pump without hCLS (23.1% vs. 22.2%), respectively. All participants reported negative impacts on physical health, mental health, sleep, relationships, leisure activities, work/studies, and sex life. The ‘top 3’ ranked impacts for the rSHE cohort were physical health and sleep (tied at first) followed by leisure activities. Impacts to physical health, mental health, and relationships were ‘top 3’ for the nSHE cohort. Conclusions: Recurrent severe hypoglycemia is observed to persist despite use of advanced diabetes technology. Negative impacts on physical health ranked as the primary concern in both rSHE and nSHE cohorts. However, moderate variability in ranked negative impact order was observed between the cohorts. Disclosure A.Boateng-kuffour: None. P.Callahan: Consultant; Rhythm Pharmaceuticals, Inc., Abbott Diabetes, HealthCore Inc., BMS, Employee; Vertex Pharmaceuticals Incorporated, Genesis Research. K.Chandarana: Employee; Vertex Pharmaceuticals Incorporated, Stock/Shareholder; Vertex Pharmaceuticals Incorporated. J.L.Gaglia: Advisory Panel; Dompé, Consultant; Vertex Pharmaceuticals Incorporated, Abata Therapeutics, Avotres Inc., Current Health, Research Support; Avotres Inc., Dompé, Imcyse, Stock/Shareholder; Vertex Pharmaceuticals Incorporated. M.Phinney: None. E.Guzman ortega: Research Support; Vertex Pharmaceuticals Incorporated. R.N.Krupnick: Consultant; Vertex Pharmaceuticals Incorporated. M.Reaney: None. W.H.Polonsky: Consultant; Abbott Diabetes, Boehringer Ingelheim and Eli Lilly Alliance, Eli Lilly and Company, Insulet Corporation, Intuity Medical, Inc., MannKind Corporation, Provention Bio, Inc., Sanofi-Aventis U.S., Vertex Pharmaceuticals Incorporated.

1086-P: Humanistic Burden of Type 1 Diabetes in U.S. Adults across Different Age Cohorts—A Qualitative Interview Study

Introduction: This study explores the perceived humanistic burden of type 1 diabetes (T1D) across different age cohorts of adults (18-35 yrs., 36-64 yrs., and 65+ yrs.) in the US who are using continuous glucose monitors (CGM). Methods: Adult CGM users (≥18 years of age) with self-reported T1D diagnosis (≥ 5 yrs.) were interviewed to evaluate the impact of T1D on quality of life (QoL). Participants ranked the ‘top 3’ negative impacts of T1D on QoL using a “Wheel of Life” visual framework, which helped participants identify and describe the multi-faceted impacts of T1D on QoL. Interview transcripts were thematically analyzed. Subgroup analyses were conducted by age cohort. Results: Overall, 31 participants (18-35 yrs., n=10; 36-64 yrs., n=12; 65+ yrs., n=9) took part in the study. Participants were mostly female (74%) and married (81%), with a mean age at diagnosis of 28 yrs. and a reported mean HbA1c level of 7.1% (range: 5.0% - 9.9%); 42% reported having ≥ 2 severe hypoglycemic events (SHEs) in the past year. Impacts on physical health, mental health, sleep, relationships, leisure activities, work/studies, and sex life were reported by participants as affecting their daily lives. Physical health impacts were most frequently ranked in “top 3” negative impacts across age cohorts. Negative impacts on physical health, mental health and sleep were ranked as “top 3” for both the 18-35 yrs. and the 36-64 yrs. age cohorts, while negative impacts on physical health, leisure activities, and relationships were top ranked by the 65+ yrs. cohort. Conclusions: Despite use of advanced diabetes technologies, the humanistic burden of T1D persists across adults of different age cohorts. Participants in all age cohorts reported physical health as the leading negative impact to QoL, while other perceived negative impacts varied in rank order across age cohorts. Disclosure P. Callahan: Employee; Vertex Pharmaceuticals Incorporated, Genesis Research. Consultant; Rhythm Pharmaceuticals, Inc., Abbott Diabetes, HealthCore Inc., BMS. A. Boateng-Kuffour: None. K. Chandarana: Employee; Vertex Pharmaceuticals Incorporated. Stock/Shareholder; Vertex Pharmaceuticals Incorporated. J.L. Gaglia: Consultant; Vertex Pharmaceuticals Incorporated. Stock/Shareholder; Vertex Pharmaceuticals Incorporated. Consultant; Abata Therapeutics, Avotres Inc. Research Support; Avotres Inc. Advisory Panel; Dompé. Research Support; Dompé, Imcyse. Consultant; Current Health. M. Phinney: None. E. Guzman Ortega: Research Support; Vertex Pharmaceuticals Incorporated. R.N. Krupnick: Consultant; Vertex Pharmaceuticals Incorporated. M. Reaney: None. W.H. Polonsky: Consultant; Abbott Diabetes, Boehringer Ingelheim and Eli Lilly Alliance, Eli Lilly and Company, Insulet Corporation, Intuity Medical, Inc., MannKind Corporation, Provention Bio, Inc., Sanofi-Aventis U.S., Vertex Pharmaceuticals Incorporated.

#4018 CHARACTERISATION OF THE CLINICAL, HUMANISTIC AND ECONOMIC BURDEN OF ANAEMIA IN CHRONIC KIDNEY DISEASE: A SYSTEMATIC LITERATURE REVIEW

Abstract Background and Aims Chronic kidney disease (CKD) is defined as structural kidney damage and/or decreased kidney function (glomerular filtration rate &amp;lt;60mL/min/1.73 m2) persisting for ≥3 months [1]. Anaemia is a frequent CKD comorbidity associated with increased cardiovascular (CV) morbidity, mortality, reduced health-related quality of life (HRQoL) and greater healthcare resource utilisation (HRU) [2]. Our systematic literature review provided an overview of the clinical, humanistic and economic burden of anaemia of CKD. Method A search was performed in MEDLINE, EMBASE, MEDLINE IN-PROCESS, Cochrane Controlled Trials Register and the Cochrane Database of Systematic Reviews from database inception to 10 April 2022, using predefined criteria to identify cohort, cross-sectional, observational and cost studies, economic evaluations, conceptual model studies, systematic and literature reviews (complemented by grey literature and congress abstract searches and back-referencing relevant review papers [2016–2021]). Full-text selected citations were examined by two independent reviewers. Clinical burden parameters included CKD progression, differences in progression between patients with/without CKD anaemia, time to blood transfusion and association of CKD stage/progression with CV outcomes or mortality. Humanistic burden included HRQoL and caregiver burden. Economic burden was determined by HRU and direct/indirect costs. Results Data were extracted from 115 studies (12,109 database records, 79 supplementary items; Figure 1), including patients: pre- or non-dialysis (n = 61), on dialysis (n = 21), mixed non-/on dialysis (n = 19) and unknown dialysis status (n = 14). Incidence of advanced CKD was significantly higher in adult non-dialysis dependent (NDD) patients with anaemia of CKD vs those without (incidence per 1000 person-years: 15 to ∼85 vs 3 to ∼25). Patients with CKD anaemia had a higher risk of disease progression than those without anaemia (n = 21), e.g. non-dialysis stage 1–5 adults with CKD anaemia had ∼double the risk of developing end-stage CKD (hazard ratio [95% confidence interval [CI]] = 2.08 [1.50–2.89]). Median time to dialysis initiation was significantly lower in adult NDD stage 1–5 patients with anaemia of CKD vs those without anaemia (45.1 vs 68.3 months p&amp;lt;0.01). A higher proportion of stage 3 adult NDD patients with anaemia of CKD progressed to stage 4 or 5 over follow-up periods of 1, 2 and 3 years vs those without anaemia (40% vs 20%; 60% vs 25%; 75% vs 30%, respectively). Available data from the 43 studies reporting humanistic burden suggested significantly worse HRQoL in patients with vs without CKD anaemia; e.g. non-dialysis adults with CKD anaemia had worse 36-item kidney disease and QoL scores for symptoms/problems, effect- and burden-of CKD. Comparative caregiver burden data in patients with/without CKD anaemia were limited. Limited available evidence from an economic perspective suggested higher HRU in patients with anaemia of CKD vs those without. Patients with anaemia of CKD has ∼2 times greater risk of hospitalisation due to heart failure vs those without (crude incidence rate ratio [95% CI] = 1.9 [1.6–2.1]). Similarly, patients with anaemia of CKD has significantly higher risk of hospitalisations (adjusted relative risk [ARR] [95% CI] = 1.33 [1.31–1.34]) and emergency department visits (ARR [95% CI] = 1.14 [1.12–1.15]) in a multivariate logistic regressions model. No data were available on change in economic parameters over time or on inter-relationships between humanistic/economic burden parameters. There were no published disease models in CKD anaemia; Figure 2 illustrates identified relationships between clinical, humanistic and economic burden parameters. Conclusion Based on limited moderate-to-good quality data, we found that patients with CKD anaemia face greater clinical, humanistic and economic burdens than those without anaemia, with significantly worse HRQoL and greater HRU. CKD anaemia was also associated with a higher risk of CKD progression.