The clinical outcomes and healthcare resource utilization in IgG4-related disease: a claims-based analysis of commercially insured adults in the United States.

OBJECTIVES: IgG4-related disease (IgG4-RD) can affect nearly any organ and is often treated with glucocorticoids, which contribute to organ damage and toxicity. Comorbidities and healthcare utilization in IgG4-RD are poorly understood. METHODS: We conducted a cohort study using claims data from a US managed care organization. Incident IgG4-RD cases were identified using a validated algorithm; general population comparators were matched by age, sex, race/ethnicity and index date. The frequency of 21 expert-defined clinical outcomes associated with IgG4-RD or its treatment and healthcare-associated visits and costs were assessed 12 months before and 36 months after the index date (date of earliest IgG4-RD-related claim). RESULTS: There were 524 cases and 5240 comparators. Most cases received glucocorticoids prior to (64.0%) and after (85.1%) the index date. Nearly all outcomes, many being common glucocorticoid toxicities, occurred more frequently in cases vs comparators. During follow-up, the largest differences between cases and comparators were seen for gastroesophageal reflux disease (prevalence difference: +31.2%, P < 0.001), infections (+17.3%, P < 0.001), hypertension (+15.5%, P < 0.01) and diabetes mellitus (+15.0%, P < 0.001). The difference in malignancy increased during follow-up from +8.8% to +12.5% (P < 0.001). Some 17.4% of cases used pancreatic enzyme replacement therapy during follow-up. Over follow-up, cases were more often hospitalized (57.3% vs 17.2%, P < 0.01) and/or had an emergency room visit (72.0% vs 36.7%, P < 0.01); all costs were greater in cases than comparators. CONCLUSIONS: Patients with IgG4-RD are disproportionately affected by adverse outcomes, some of which may be preventable or modifiable with vigilant clinician monitoring. Glucocorticoid-sparing treatments may improve these outcomes.

Healthcare Service Use Patterns Among Patients with Acid Sphingomyelinase Deficiency Type B: A Retrospective US Claims Analysis.

INTRODUCTION: Acid sphingomyelinase deficiency (ASMD) is a rare lysosomal storage disease. Patients with ASMD type B experience multiple morbidities, potentially leading to early mortality. Before the 2022 approval of olipudase alfa for non-neuronopathic ASMD manifestations, only symptom management was offered. Data on healthcare services used by patients with ASMD type B are limited. This analysis used medical claims data to evaluate real-world healthcare service use by patients with ASMD type B in the United States of America (USA). METHODS: The IQVIA Open Claims patient-level database (2010-2019) was cross-examined. Two patient cohorts were identified: the primary analysis cohort, which included patients with at least two claims associated with ASMD type B (ICD-10 code E75.241) and more total claims with ASMD type B than any other ASMD types, and the sensitivity analysis cohort, which included patients with a high probability of having ASMD type B identified using a validated machine-learning algorithm. Claims for ASMD-associated healthcare services were recorded, including outpatient visits, emergency department (ED) visits, and inpatient hospitalizations. RESULTS: The primary analysis cohort included 47 patients; a further 59 patients made up the sensitivity analysis cohort. Patient characteristics and healthcare service use were similar in both cohorts and were consistent with established characteristics of ASMD type B. Overall, 70% of the primary analysis cohort from this study were aged < 18 years, and the liver, spleen, and lungs were the most frequently affected organs. Cognitive, developmental, and/or emotional problems and respiratory/lung disorders caused most outpatient visits; respiratory/lung disorders accounted for most ED visits and hospitalizations. CONCLUSION: This retrospective analysis of medical claims data identified patients with ASMD type B who had characteristics typical of this condition. A machine-learning algorithm detected further cases with a high probability of having ASMD type B. High use of ASMD-related healthcare services and medications was observed in both cohorts.

Acid sphingomyelinase deficiency (ASMD) type B, historically known as Niemann­Pick type B, is a rare illness. People with acid sphingomyelinase deficiency type B experience damage to many organs of the body (such as the liver and lungs), which may lead to early death. Until recently, no treatment has been available, and people were only treated for their symptoms. Now, a treatment called olipudase alfa has been approved in Europe, Japan, and the USA. People with ASMD type B may need lots of tests, surgeries, medications, and physician visits; however, we do not know how often these healthcare services are used. This study used medical claims to find out more about the healthcare services used by people with ASMD type B. To find as many people with ASMD type B as possible, we identified two groups of people. The first group included people with diagnosis codes for ASMD type B, the other group was identified as having a high likelihood of ASMD type B. The people in each group were similar in age and the illnesses/symptoms they had. The liver, spleen, and lungs were the most frequently damaged organs, and most physician visits were for mental, developmental, and/or emotional problems, and breathing or lung diseases. Breathing or lung disease and bleeding problems caused the most emergency department visits and hospitalizations. Overall, the use of healthcare services was high in people with ASMD type B. This study shows the need for specific treatments for people with ASMD.

Uterine fibroids and incidence of depression, anxiety and self-directed violence: a cohort study.

BACKGROUND: Depression and anxiety are prevalent among women with uterine fibroids (UF). The rate of mental health diagnoses in women with UF has not been studied. METHODS: Women aged 18-50 years with diagnosed UF were identified in the Optum Clinformatics commercial insurance claims database (OptumInsight, Eden Prairie, Minnesota) from 1 May 2000 to 31 March 2020 (n=313 754) and were matched 1:2 on age and calendar time to women without (n=627 539). Cox proportional hazards models estimated HRs and 95% CIs between UF and diagnosed depression, anxiety and self-directed violence, adjusting for demographics and comorbidities. Among women with diagnosed UF, the association between hysterectomy and mental health outcomes was estimated. RESULTS: After adjusting for confounders, women with diagnosed UF had a higher rate of depression (HR: 1.12; 95% CI 1.10 to 1.13), anxiety (HR: 1.12; 95% CI 1.10 to 1.13) and self-directed violence (HR: 1.46; 95% CI 1.29 to 1.64) than women without. Among women with pain symptoms and heavy menstrual bleeding, the HR comparing women with diagnosed UF to women without was 1.21 (95% CI 1.18 to 1.25) for depression, 1.18 (95% CI 1.15 to 1.21) for anxiety and 1.68 (95% CI 1.35 to 2.09) for self-directed violence. Among women with diagnosed UF, the HR comparing women who underwent a hysterectomy to women who did not was 1.22 (95% CI 1.17 to 1.27) for depression, 1.13 (95% CI 1.09 to 1.17) for anxiety and 1.86 (95% CI 1.39 to 2.49) for self-directed violence. CONCLUSIONS: Rates of depression, anxiety and self-directed violence were higher among women with diagnosed UF, particularly among those who experienced pain symptoms or who underwent hysterectomy.

Midlife Cardiovascular Health and Robust Versus Frail Late-Life Status: The Atherosclerosis Risk in Communities Study.

BACKGROUND: We examined the relationship of midlife cardiovascular health (CVH) with late-life robustness among men and women and the impact of survivorship bias on sex differences in robustness. METHODS: Prospective analysis of 15 744 participants aged 45-64 (visit 1 median age: 54 years, 55% female, 27% Black) in 1987-1989 from the population-based Atherosclerosis Risk in Communities Study. CVH was operationalized according to the Life's Simple 7 (LS7) metric of health behaviors (smoking, weight, physical activity, diet, cholesterol, blood pressure, and glucose); each behavior was scored as ideal (2 points), intermediate (1 point), or poor (0 points) and summed. Late-life robust/prefrail/frailty was defined at visit 5 (2011-2013). Multinomial regression estimated relative prevalence ratios (RPRs) of late-life robustness/prefrailty/frailty/death across overall midlife LS7 score and components, for the full visit 1 sample. Separate analyses considered visit 5 survivors-only. RESULTS: For each 1-unit greater midlife LS7 score, participants had a 37% higher relative prevalence of being robust versus frail (overall RPR = 1.37 [95% confidence interval {CI}: 1.30-1.44]; women = 1.45 [1.36-1.54]; men = 1.24 [1.13-1.36]). Among the full visit 1 sample, women had a similar 1-level higher robustness category prevalence (RPR = 1.35 [95% CI: 1.32-1.39]) than men (RPR = 1.31 [95% CI: 1.27-1.35]) for every 1-unit higher midlife LS7 score. Among survivors, men were more likely to be robust than women at lower LS7 levels; differences were attenuated and not statistically different at higher midlife LS7 levels. CONCLUSIONS: Midlife CVH is positively associated with robustness in late life among men and women. Accounting for mortality in part explains documented sex differences in robustness across all levels of LS7.

Clinical features and diagnosis of multiple myeloma: a population-based cohort study in primary care.

OBJECTIVES: Patients with multiple myeloma (MM) experience significant delays in diagnosis due to non-specific symptomatology. The aim of this study was to characterise the frequency and timing of clinical features in the primary care setting prior to MM diagnosis. DESIGN: Population-based cohort study. SETTING: Electronic health records data of approximately 17 million patients (2006-2016) within the UK Clinical Practice Research Datalink. PARTICIPANTS: Patients aged ≥18 years with newly diagnosed MM (NDMM), no history of solid tumours and ≥2 years registration in a primary care practice prior to MM diagnosis. MAIN OUTCOME MEASURES: Clinical features and symptoms including bone pain, skeletal-related events (SREs), investigation and confirmation of MM diagnostic CRAB criteria (hyperCalcaemia, Renal impairment, Anaemia, Bone lesions) during the 2 years prior to MM diagnosis; time between symptom manifestation and/or relevant investigation and diagnosis of MM. RESULTS: Among 2646 patients with NDMM, 47.5% had a bone pain record during the 2-year period prior to MM diagnosis, mainly affecting the back. Regardless of baseline bone pain, investigations for serum calcium level were used in 36.4% of patients prior to MM diagnosis, followed by haemoglobin (65.6%) or renal function (74.1%). Median (Q1, Q3) time from first-recorded bone pain to MM diagnosis was 220 (80, 476) days. Median (Q1, Q3) time from first-recorded hypercalcaemia, renal impairment or anaemia to MM diagnosis was 23 (12, 46), 58 (17, 254) and 73 days (28, 232), respectively. An imaging investigation or referral for imaging was recorded for 60.0% of patients with bone pain/SRE and 32% without. CONCLUSIONS: Nearly half of patients diagnosed with NDMM presented with bone pain approximately 7 months prior to MM diagnosis. Investigations to evaluate all CRAB criteria, including targeted imaging, were underused. Early recognition of myeloma clinical features and optimised use of investigations in primary care may potentially expedite MM diagnosis.

Adherence to Chronic Kidney Disease Screening Guidelines Among Patients With Type 2 Diabetes in a US Administrative Claims Database.

OBJECTIVE: To examine the screening rates for kidney damage and function among patients with type 2 diabetes (T2D) and chronic kidney disease stage at diabetes diagnosis using a US administrative claims database. PATIENTS AND METHODS: This cohort study used a claims database enriched with laboratory results data. Patients with T2D (defined as 1 inpatient or 2 outpatient claims for diabetes), aged 18 years or older, and with at least 1 year of follow-up enrollment were identified. Patients with type 1 diabetes, kidney disease, or other related conditions at baseline were excluded. We estimated screening rates using laboratory orders for serum creatinine and estimated glomerular filtration rate (eGFR) measurement and urine albumin to creatinine ratio (UACR). Chronic kidney disease severity was reported using the Kidney Disease: Improving Global Outcomes classification based on laboratory results. RESULTS: A total of 1,881,447 patients with T2D were eligible for analysis. Mean ± SD age was 63.1±13.1 years; 947,150 patients (50.3%) were male. Serum creatinine tests were ordered within 14 days of the index date among 290,722 patients of 622,915 (46.7%) patients with newly-recognized T2D. Overall, 1,595,964 patients (84.8%) had at least one serum creatinine test ordered during the 1-year follow-up period. Fewer patients received a UACR test during follow-up (814,897 [43.3%]). Less than half of all patients with T2D received a laboratory test order for both serum creatinine and urine albumin measurements during the follow-up period. CONCLUSION: Physicians treating patients with diabetes are selectively adhering to chronic kidney disease screening guidelines, as indicated by high rates of eGFR testing, but less frequent UACR testing. Despite recommendations to monitor both eGFR and UACR, less than half of patients were screened for albuminuria during the 1-year follow-up.

Real-world reproducibility study characterizing patients newly diagnosed with multiple myeloma using Clinical Practice Research Datalink, a UK-based electronic health records database.

PURPOSE: We evaluated the reproducibility of a study characterizing newly-diagnosed multiple myeloma (MM) patients within an electronic health records (EHR) database using different analytic tools. METHODS: We reproduced the findings of a descriptive cohort study using an iterative two-phase approach. In Phase I, a common protocol and statistical analysis plan (SAP) were implemented by independent investigators using the Aetion Evidence Platform® (AEP), a rapid-cycle analytics tool, and SAS statistical software as a gold standard for statistical analyses. Using the UK Clinical Practice Research Datalink (CPRD) dataset, the study included patients newly diagnosed with MM within primary care setting and assessed baseline demographics, conditions, drug exposure, and laboratory procedures. Phase II incorporated analysis revisions based on our initial comparison of the Phase I findings. Reproducibility of findings was evaluate by calculating the match rate and absolute difference in prevalence between the SAS and AEP study results. RESULTS: Phase I yielded slightly discrepant results, prompting amendments to SAP to add more clarity to operational decisions. After detailed specification of data and operational choices, exact concordance was achieved for the number of eligible patients (N = 2646), demographics, comorbidities (i.e., osteopenia, osteoporosis, cardiovascular disease [CVD], and hypertension), bone pain, skeletal-related events, drug exposure, and laboratory investigations in the Phase II analyses. CONCLUSIONS: In this reproducibility study, a rapid-cycle analytics tool and traditional statistical software achieved near-exact findings after detailed specification of data and operational choices. Transparency and communication of the study design, operational and analytical choices between independent investigators were critical to achieve this reproducibility.

Dietary Protein Sources and Risk for Incident Chronic Kidney Disease: Results From the Atherosclerosis Risk in Communities (ARIC) Study.

OBJECTIVE: Dietary protein restriction is recommended for patients with moderate to severe renal insufficiency. Long-term data on the relationship between dietary protein sources and risk for incident kidney disease in individuals with normal kidney function are largely missing. This study aimed to assess the association between dietary protein sources and incident chronic kidney disease (CKD). DESIGN: Prospective cohort. SETTING: Atherosclerosis Risk in Communities study participants from 4 US communities. SUBJECTS: A total of 11,952 adults aged 44-66 years in 1987-1989 who were free of diabetes mellitus, cardiovascular disease, and had an estimated glomerular filtration rate (eGFR) ≥ 60 mL/minute/1.73 m2. MAIN OUTCOME MEASURE: A 66-item food frequency questionnaire was used to assess food intake. CKD stage 3 was defined as a decrease in eGFR of ≥25% from baseline resulting in an eGFR of less than 60 mL/minute/1.73 m2; CKD-related hospitalization; CKD-related death; or end-stage renal disease. Hazard ratios (HRs) and 95% confidence intervals (CIs) were estimated using Cox proportional hazards regression. RESULTS: During a median follow-up of 23 years, there were 2,632 incident CKD cases. Red and processed meat consumption was associated with increased CKD risk (HRQ5 vs. Q1: 1.23, 95% CI: 1.06-1.42, ptrend = 0.01). In contrast, higher dietary intake of nuts, legumes, and low-fat dairy products was associated with lower CKD risk (nuts: HRQ5 vs. Q1: 0.81, 95% CI: 0.72-0.92, ptrend <0.001; low-fat dairy products: HRQ5 vs. Q1: 0.75, 95% CI: 0.65-0.85, ptrend <0.001; legumes: HRQ5 vs. Q1: 0.83, 95% CI: 0.72-0.95, ptrend = 0.03). CONCLUSION: There were varied associations of specific dietary protein sources with risk of incident CKD; with red and processed meat being adversely associated with CKD risk; and nuts, low-fat dairy products, and legumes being protective against the development of CKD.