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.

The Economic and Health-related Impact of Crohn's Disease in the United States: Evidence from a Nationally Representative Survey.

BACKGROUND: Approximately 593,000 to 780,000 people in the United States (US) have been diagnosed with Crohn's disease (CD), and an additional 33,000 are diagnosed yearly. Our objective was to estimate CD's impact on medical costs, lost earnings, work and school absences, health status, and health-related quality of life (HRQOL) in the US and to compute current and forecasted national costs. METHODS: We used the nationally representative Medical Expenditure Panel Survey to match 539 respondents with CD to similar respondents without any inflammatory bowel disease (IBD). We estimated annual costs, work and school absences, and self-assessed health status. HRQOL was assessed by the SF-12 Physical Component Summary and Mental Component Summary (PCS and MCS) scores. CD prevalence rates, population counts, and costs were used to forecast total national costs. RESULTS: CD is associated with higher medical costs ($13,446 versus $6029) and lost earnings ($1249 versus $644) and is responsible for $3.48 billion in total national costs (expected to increase to $3.72 billion in 2025). Respondents with CD were more likely to miss work (38% versus 33%) or school (64% versus 33%), less likely to report being in excellent or very good physical health (24% versus 63%), and experienced lower HRQOL measured by the Physical Component Summary (43.4 versus 48.5) and Mental Component Summary (48.6 versus 50.0) than those without IBD. CONCLUSIONS: CD is responsible for increased medical care costs and lower earnings, health status, and HRQOL. These data can serve as benchmarks when examining future CD-related costs and HRQOL.

Prevalence and healthcare costs of obesity-related comorbidities: evidence from an electronic medical records system in the United States.

OBJECTIVE: This study estimated the economic burden of obesity-related comorbidities (ORCs) in the US, at both the person and population levels. METHODS: The Geisinger Health System provided electronic medical records and claims between January 2004 and May 2013 for a sample of 153,561 adults (50% males and 97% white). Adults with < 2 years of data, who were underweight (body mass index (BMI) < 18.5 kg/m(2)), or had diseases causing major weight change (e.g., malignancy) during the study period (i.e., continuous enrollment in health plans) were excluded. A total of 21 chronic conditions, with established association with obesity in the literature, were identified by diagnosis codes and/or lab test results. The total healthcare costs were measured in each year. The association between annual costs and ORCs was assessed by a regression, which jointly considered all the ORCs. The per-person incremental costs of a single comorbidity, without any of the other ORCs, were calculated. The population-level economic burden was the product of each ORC's incremental costs and the annual prevalence of the ORC among 100,000 individuals. The prevalence of ORCs was stratified by obesity status to estimate the economic burden among 100,000 individuals with obesity and among those without. RESULTS: This study identified 56,895 adults (mean age = 47 years; mean BMI = 29.6 kg/m(2)). The annual prevalence of ORCs ranged from 0.5% for pulmonary embolism (PE) to 41.8% for dyslipidemia. The per-person annual incremental costs of a single ORC ranged from $120 for angina to $1665 for PE. Hypertensive diseases (HTND), dyslipidemia, and osteoarthritis were the three most expensive ORCs at the population level; each responsible for ≥$18 million annually among 100,000 individuals. HTND and osteoarthritis were much more costly among individuals with obesity than those without obesity. LIMITATIONS: Data were from a small geographic region. CONCLUSIONS: ORCs are associated with substantial economic burden, especially for those requiring continuous treatments.

The cost-effectiveness of the levonorgestrel-releasing intrauterine system for the treatment of idiopathic heavy menstrual bleeding in the United States.

OBJECTIVES: Heavy menstrual bleeding negatively impacts the health and quality of life of about 18 million women in the United States. Although some studies have established the clinical effectiveness of heavy menstrual bleeding treatments, few have evaluated their cost-effectiveness. Our objective was to evaluate the cost-effectiveness of the levonorgestrel-releasing intrauterine system (LNG-IUS) compared with other therapies for idiopathic heavy menstrual bleeding. METHODS: We developed a model comparing the clinical and economic outcomes (from a US payer perspective) of three broad initial treatment strategies over 5 years: LNG-IUS, oral agents, or surgery. Up to three nonsurgical treatment lines, followed by up to two surgical lines, were allowed; unintended pregnancy was possible, and women could discontinue any time during nonsurgical treatments. Menstrual blood loss of 80 ml or more per cycle determined treatment failure. RESULTS: Initiating treatment with LNG-IUS resulted in the fewest hysterectomies (6 per 1000 women), the most quality-adjusted life-years (3.78), and the lowest costs ($1137) among all the nonsurgical strategies. Initiating treatment with LNG-IUS was also less costly than surgery, resulted in fewer hysterectomies (vs. 9 per 1000 for ablation) but was associated with fewer quality-adjusted life-years gained per patient (vs. 3.80 and 3.88 for ablation and hysterectomy, respectively). Sensitivity analyses confirmed these results. CONCLUSIONS: LNG-IUS resulted in the lowest treatment costs and the fewest number of hysterectomies performed over 5 years compared with all other initial strategies and resulted in the most quality-adjusted life-years gained among nonsurgical options. Initial treatment with LNG-IUS is the least costly and most effective option for women desiring to preserve their fertility.

Revisiting the association between maternal smoking during pregnancy and ADHD.

OBJECTIVE: Studies examining the relationship between maternal smoking during pregnancy and the development of Attention Deficit Hyperactivity Disorder (ADHD) among offspring have yielded mixed results, with some studies suggesting a strong association and others finding no association. These studies have varied in quality of design and measures. The purpose of this study was to evaluate the association between maternal smoking during pregnancy and offspring ADHD, using detailed prospective smoking data and subsequent follow-up data from the Collaborative Perinatal Project (CPP). METHOD: Maternal smoking status was collected throughout pregnancy during the original CPP study. Offspring were followed-up in early adulthood and questioned about ADHD symptoms and diagnosis. Logistic regression was used to model the association between maternal smoking during pregnancy and ADHD. Linear and logistic regression were used to examine clinical characteristics and remission rates associated with ADHD in relation to maternal smoking. RESULTS: No association was found between maternal smoking during pregnancy and offspring ADHD. Further, no differences in age of onset, number of symptoms, or likelihood of remission were found among ADHD subjects with and without a history of maternal smoking during pregnancy. CONCLUSIONS: These findings do not support the hypothesis that maternal smoking during pregnancy is causally related to ADHD. Ongoing research should continue to strive to identify those environmental or genetic factors that may enhance the impact of maternal smoking on ADHD or that may be associated more clearly with the development and potential prevention of ADHD.

Intimate partner violence and patient screening across medical specialties.

OBJECTIVES: The aims of this study were to compare rates of intimate partner violence (IPV) across different medical specialties and health care sites in one metropolitan area, describe demographic characteristics of women with abusive partners, characterize health care provider assessment of IPV, and describe patient characteristics associated with health care assessment for partner violence. METHODS: Women (N = 2,465) completed written surveys about partner violence and health care screening for violence in the waiting rooms of five types of health care settings (obstetrician/gynecologist office, emergency department, primary care office, pediatrics, and addiction recovery) across eight different hospitals in the greater Boston area. RESULTS: The overall survey response rate was 62%. The 12-month prevalence rate of IPV was 14%, with 37% disclosing lifetime prevalence. The highest rates of recent IPV were disclosed in the hospital-based addiction recovery unit (36%) and in emergency departments (17%). Adjusted demographic risk characteristics for IPV included age (younger than 24 years), low income, and unemployment. Health care providers were more likely to discuss IPV with low-income women than with middle- or high-income women but were no more likely to assess violence within the youngest age group. Among women who disclosed abuse to their health care provider, 50% reported receiving direct interventions or services as a result. CONCLUSIONS: Using the same instrument and protocol, different rates of IPV and detection of IPV were found across medical departments, with the highest rates in emergency departments and an addiction recovery program. It is especially important for assessment of IPV to include young women who present to medical departments.

Adverse health behaviors and the detection of partner violence by clinicians.

BACKGROUND: Intimate partner violence (IPV) is a major public health problem in the United States, and victims are commonly encountered in medical settings. Many barriers exist to clinician-initiated screening for IPV. However, smoking and problem drinking are conditions that clinicians commonly screen for and both have been strongly associated with IPV in prior studies. By estimating the predicted probability of 12-month and lifetime IPV for a given patient based on whether she presents with these conditions, our study gives clinicians information that can help them identify patients at risk for IPV. METHODS: A cross-sectional written patient survey was administered to 2386 female patients at 8 different health care settings in the Greater Boston (Mass) metropolitan area. The probabilities of 12-month and lifetime IPV were estimated based on the women's self-report of smoking and drinking behaviors. RESULTS: A woman who neither smoked nor engaged in problem drinking had a 10% probability of IPV in the preceding 12 months and a 39% chance of IPV in her lifetime. Smoking increased the probability to 14% and 49%, respectively. Problem drinking resulted in a doubling of the predicted probability of 12-month IPV to 21%, with a lifetime probability of 43%. When both conditions were present, the effects were additive, with a woman having a 27% probability of experiencing IPV in the preceding 12 months and 54% chance of IPV in her lifetime. CONCLUSIONS: The presence of smoking or problem drinking should raise clinicians' suspicion for IPV. This paradigm should not replace direct questioning about IPV but may aid in the detection of abuse in patient populations.