Skip to main content

Maurice Alan Brookhart

Professor in Population Health Sciences
Population Health Sciences

Selected Publications


Characterizing Imbalance in the Tails of the Propensity Score Distribution.

Journal Article Am J Epidemiol · February 5, 2024 Understanding characteristics of patients with propensity scores in the tails of the propensity score (PS) distribution has relevance for inverse-probability-of-treatment-weighted and PS-based estimation in observational studies. Here we outline a method f ... Full text Link to item Cite

Trends in Pediatric Emergency and Inpatient Healthcare Use for Mental and Behavioral Health Among North Carolinians During the Early COVID-19 Pandemic.

Journal Article J Pediatric Infect Dis Soc · December 26, 2023 BACKGROUND: Widespread school closures and health care avoidance during the COVID-19 pandemic led to disruptions in access to pediatric mental health care. METHODS: We conducted a retrospective study of emergency and inpatient administrative claims from pr ... Full text Link to item Cite

Impact of the COVID-19 Pandemic on Pediatric Preventive Health Care Among North Carolina Children Enrolled in Medicaid.

Journal Article J Pediatric Infect Dis Soc · December 26, 2023 BACKGROUND: Children enrolled in private insurance had reduced preventive health care during the coronavirus disease 2019 (COVID-19) pandemic. However, the impact of the pandemic on children enrolled in Medicaid has been minimally described. METHODS: We us ... Full text Link to item Cite

Lessons Learned Using Real-World Data to Emulate Randomized Trials: A Case Study of Treatment Effectiveness for Newly Diagnosed Immune Thrombocytopenia.

Journal Article Clin Pharmacol Ther · December 2021 Regulatory agencies are increasingly considering real-world evidence (RWE) to support label expansions of approved medicines. We conducted a comparative effectiveness study to emulate a proposed randomized trial of romiplostim vs. standard-of-care (SOC) th ... Full text Link to item Cite

Community SARS-CoV-2 Surge and Within-School Transmission.

Journal Article Pediatrics · October 2021 OBJECTIVES: When the severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) pandemic began, experts raised concerns about in-person instruction in the setting of high levels of community transmission. We describe secondary transmission of SARS-CoV-2 ... Full text Link to item Cite

Estimating the Effectiveness of Rotavirus Vaccine Schedules.

Journal Article Epidemiology · July 1, 2021 BACKGROUND: Important questions exist regarding the comparative effectiveness of alternative childhood vaccine schedules; however, optimal approaches to studying this complex issue are unclear. METHODS: We applied methods for studying dynamic treatment reg ... Full text Link to item Cite

Effectiveness and Safety of Romiplostim Among Patients with Newly Diagnosed, Persistent and Chronic Immune Thrombocytopenia in European Clinical Practice.

Journal Article Adv Ther · May 2021 INTRODUCTION: Romiplostim has been approved in Europe since 2009 to treat patients with chronic primary immune thrombocytopenia (ITP). Using real-world data from seven European countries, we measured the effectiveness and safety outcomes within 24 weeks fo ... Full text Link to item Cite

Incidence and Secondary Transmission of SARS-CoV-2 Infections in Schools.

Journal Article Pediatrics · April 2021 BACKGROUND: In an effort to mitigate the spread of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), North Carolina closed prekindergarten through grade 12 public schools to in-person instruction on March 14, 2020. On July 15, 2020, North Carol ... Full text Link to item Cite

Informing Healthcare Decisions with Observational Research Assessing Causal Effect. An Official American Thoracic Society Research Statement.

Journal Article Am J Respir Crit Care Med · January 1, 2021 Rationale: Decisions in medicine are made on the basis of knowledge and reasoning, often in shared conversations with patients and families in consideration of clinical practice guideline recommendations, individual preferences, and individual goals. Obser ... Full text Link to item Cite

Characterizing imbalance in the tails of the propensity score distribution

Conference PHARMACOEPIDEMIOLOGY AND DRUG SAFETY · 2021 Cite

Bias testing, bias correction, and confounder selection using an instrumental variable model.

Journal Article Stat Med · December 20, 2020 Instrumental variable (IV) analysis can be used to address bias due to unobserved confounding when estimating the causal effect of a treatment on an outcome of interest. However, if a proposed IV is correlated with unmeasured confounders and/or weakly corr ... Full text Link to item Cite

Who are we missing? Underrepresentation of data sources used for pharmacoepidemiology research in the United States.

Journal Article Pharmacoepidemiol Drug Saf · November 2020 PURPOSE: Research using healthcare databases often includes patients frequently excluded from clinical trials; yet it is not known whether commonly used data represents the overall population or specific sub-populations of interest. We aimed to examine pop ... Full text Link to item Cite

Using negative control outcomes to assess the comparability of treatment groups among women with osteoporosis in the United States.

Journal Article Pharmacoepidemiol Drug Saf · August 2020 PURPOSE: In contrast to randomized clinical trials, comparative safety and effectiveness assessments of osteoporosis medications in clinical practice may be subject to confounding by indication. We used negative control outcomes to detect residual confound ... Full text Link to item Cite

Mortality in US Hemodialysis Patients Following Exposure to Wildfire Smoke.

Journal Article J Am Soc Nephrol · August 2020 BACKGROUND: Wildfires are increasingly a significant source of fine particulate matter (PM2.5), which has been linked to adverse health effects and increased mortality. ESKD patients are potentially susceptible to this environmental stressor. METHODS: We c ... Full text Link to item Cite

Characterizing Imbalance in the Tails of the Propensity Score Distribution.

Journal Article Am J Epidemiol · February 5, 2024 Understanding characteristics of patients with propensity scores in the tails of the propensity score (PS) distribution has relevance for inverse-probability-of-treatment-weighted and PS-based estimation in observational studies. Here we outline a method f ... Full text Link to item Cite

Trends in Pediatric Emergency and Inpatient Healthcare Use for Mental and Behavioral Health Among North Carolinians During the Early COVID-19 Pandemic.

Journal Article J Pediatric Infect Dis Soc · December 26, 2023 BACKGROUND: Widespread school closures and health care avoidance during the COVID-19 pandemic led to disruptions in access to pediatric mental health care. METHODS: We conducted a retrospective study of emergency and inpatient administrative claims from pr ... Full text Link to item Cite

Impact of the COVID-19 Pandemic on Pediatric Preventive Health Care Among North Carolina Children Enrolled in Medicaid.

Journal Article J Pediatric Infect Dis Soc · December 26, 2023 BACKGROUND: Children enrolled in private insurance had reduced preventive health care during the coronavirus disease 2019 (COVID-19) pandemic. However, the impact of the pandemic on children enrolled in Medicaid has been minimally described. METHODS: We us ... Full text Link to item Cite

Lessons Learned Using Real-World Data to Emulate Randomized Trials: A Case Study of Treatment Effectiveness for Newly Diagnosed Immune Thrombocytopenia.

Journal Article Clin Pharmacol Ther · December 2021 Regulatory agencies are increasingly considering real-world evidence (RWE) to support label expansions of approved medicines. We conducted a comparative effectiveness study to emulate a proposed randomized trial of romiplostim vs. standard-of-care (SOC) th ... Full text Link to item Cite

Community SARS-CoV-2 Surge and Within-School Transmission.

Journal Article Pediatrics · October 2021 OBJECTIVES: When the severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) pandemic began, experts raised concerns about in-person instruction in the setting of high levels of community transmission. We describe secondary transmission of SARS-CoV-2 ... Full text Link to item Cite

Estimating the Effectiveness of Rotavirus Vaccine Schedules.

Journal Article Epidemiology · July 1, 2021 BACKGROUND: Important questions exist regarding the comparative effectiveness of alternative childhood vaccine schedules; however, optimal approaches to studying this complex issue are unclear. METHODS: We applied methods for studying dynamic treatment reg ... Full text Link to item Cite

Effectiveness and Safety of Romiplostim Among Patients with Newly Diagnosed, Persistent and Chronic Immune Thrombocytopenia in European Clinical Practice.

Journal Article Adv Ther · May 2021 INTRODUCTION: Romiplostim has been approved in Europe since 2009 to treat patients with chronic primary immune thrombocytopenia (ITP). Using real-world data from seven European countries, we measured the effectiveness and safety outcomes within 24 weeks fo ... Full text Link to item Cite

Incidence and Secondary Transmission of SARS-CoV-2 Infections in Schools.

Journal Article Pediatrics · April 2021 BACKGROUND: In an effort to mitigate the spread of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), North Carolina closed prekindergarten through grade 12 public schools to in-person instruction on March 14, 2020. On July 15, 2020, North Carol ... Full text Link to item Cite

Informing Healthcare Decisions with Observational Research Assessing Causal Effect. An Official American Thoracic Society Research Statement.

Journal Article Am J Respir Crit Care Med · January 1, 2021 Rationale: Decisions in medicine are made on the basis of knowledge and reasoning, often in shared conversations with patients and families in consideration of clinical practice guideline recommendations, individual preferences, and individual goals. Obser ... Full text Link to item Cite

Characterizing imbalance in the tails of the propensity score distribution

Conference PHARMACOEPIDEMIOLOGY AND DRUG SAFETY · 2021 Cite

Bias testing, bias correction, and confounder selection using an instrumental variable model.

Journal Article Stat Med · December 20, 2020 Instrumental variable (IV) analysis can be used to address bias due to unobserved confounding when estimating the causal effect of a treatment on an outcome of interest. However, if a proposed IV is correlated with unmeasured confounders and/or weakly corr ... Full text Link to item Cite

Who are we missing? Underrepresentation of data sources used for pharmacoepidemiology research in the United States.

Journal Article Pharmacoepidemiol Drug Saf · November 2020 PURPOSE: Research using healthcare databases often includes patients frequently excluded from clinical trials; yet it is not known whether commonly used data represents the overall population or specific sub-populations of interest. We aimed to examine pop ... Full text Link to item Cite

Using negative control outcomes to assess the comparability of treatment groups among women with osteoporosis in the United States.

Journal Article Pharmacoepidemiol Drug Saf · August 2020 PURPOSE: In contrast to randomized clinical trials, comparative safety and effectiveness assessments of osteoporosis medications in clinical practice may be subject to confounding by indication. We used negative control outcomes to detect residual confound ... Full text Link to item Cite

Mortality in US Hemodialysis Patients Following Exposure to Wildfire Smoke.

Journal Article J Am Soc Nephrol · August 2020 BACKGROUND: Wildfires are increasingly a significant source of fine particulate matter (PM2.5), which has been linked to adverse health effects and increased mortality. ESKD patients are potentially susceptible to this environmental stressor. METHODS: We c ... Full text Link to item Cite

At the Crossroads for Intravenous Iron Dosing.

Journal Article J Am Soc Nephrol · July 2020 Full text Link to item Cite

Bleeding After Musculoskeletal Surgery in Hospitals That Switched From Hydroxyethyl Starch to Albumin Following a Food and Drug Administration Warning.

Journal Article Anesth Analg · June 10, 2020 BACKGROUND: While US Food and Drug Administration (FDA) black box warnings are common, their impact on perioperative outcomes is unclear. Hydroxyethyl starch (HES) is associated with increased bleeding and kidney injury in patients with sepsis, leading to ... Full text Link to item Cite

Negative controls to detect uncontrolled confounding in observational studies of mammographic screening comparing participants and non-participants.

Journal Article Int J Epidemiol · June 1, 2020 BACKGROUND: When comparing mammography-screening participants and non-participants, estimates of reduction in breast-cancer mortality may be biased by poor baseline comparability. We used negative controls to detect uncontrolled confounding. METHODS: We de ... Full text Link to item Cite

Use of Low-Density Lipoprotein-Lowering Therapies Before and After PCSK9 Inhibitor Initiation.

Journal Article J Am Heart Assoc · May 5, 2020 Background Proprotein convertase subtilisin/kexin type 9 inhibitors (PCSK9i) are used to reduce low-density lipoprotein (LDL) cholesterol. PCSK9i use after initiation, as well as persistence with or alterations to other LDL-lowering therapy after PCSK9i in ... Full text Link to item Cite

Treatment Patterns Among Adults with Primary Immune Thrombocytopenia Diagnosed in Hematology Clinics in the United States.

Conference Clin Epidemiol · 2020 PURPOSE: Patients with immune thrombocytopenia (ITP) have low platelet counts and an increased risk of bleeding. We described treatment patterns and clinical outcomes in routine practice in the United States (US). PATIENTS AND METHODS: Using electronic hea ... Full text Link to item Cite

Lipid Testing Trends in the US Before and After the Release of the 2013 Cholesterol Treatment Guidelines.

Journal Article Clin Epidemiol · 2020 BACKGROUND: The 2013 ACC/AHA cholesterol treatment guidelines removed the recommendation to treat adults at risk of cardiovascular disease to goal levels of low-density lipoprotein cholesterol (LDL-C). We anticipated that the frequency of LDL-C testing in ... Full text Link to item Cite

Estimating vaccine effects: A comparison of causal methods

Conference PHARMACOEPIDEMIOLOGY AND DRUG SAFETY · 2020 Cite

Impact of an Opioid Safety Initiative on Patients Undergoing Total Knee Arthroplasty: A Time Series Analysis.

Journal Article Anesthesiology · August 2019 BACKGROUND: The Opioid Safety Initiative decreased high-dose prescriptions across the Veterans Health Administration. This study sought to examine the impact of this intervention (i.e., the Opioid Safety Initiative) on pain scores and opioid prescriptions ... Full text Link to item Cite

Nonparametric Bounds for the Risk Function.

Journal Article Am J Epidemiol · April 1, 2019 Nonparametric bounds for the risk difference are straightforward to calculate and make no untestable assumptions about unmeasured confounding or selection bias due to missing data (e.g., dropout). These bounds are often wide and communicate uncertainty due ... Full text Link to item Cite

Psychotherapy Claims Surrounding Pharmacotherapy Initiation in Children and Adolescents with Anxiety Disorders.

Journal Article J Child Adolesc Psychopharmacol · March 2019 OBJECTIVES: Psychotherapy is an effective, recommended treatment for pediatric anxiety disorders. Nevertheless, individuals with mental health conditions often do not receive psychotherapy, with variation across provider types. This study sought to examine ... Full text Link to item Cite

Incidence of mental health hospitalizations, treated self-harm, and emergency room visits following new anxiety disorder diagnoses in privately insured U.S. children.

Journal Article Depress Anxiety · February 2019 BACKGROUND: Anxiety disorders are one of the most common mental illnesses in children and associated with high healthcare utilization. We aimed to estimate 2-year cumulative incidence of mental health-related hospitalizations, treated self-harm, and emerge ... Full text Link to item Cite

Initiation and interruption in intravenous bisphosphonate therapy among patients with multiple myeloma in the United States.

Journal Article Cancer Med · January 2019 BACKGROUND: Prior to 2018, intravenous bisphosphonates (IV BPs) were the only therapies recommended to prevent skeletal-related events for patients diagnosed with multiple myeloma (MM). We examined patterns of IV BP initiation and interruption among patien ... Full text Link to item Cite

Treatment Patterns Among Adults with Newly Diagnosed Primary Immune Thrombocytopenia in the United States

Conference Blood · November 29, 2018 AbstractIntroduction: Immune thrombocytopenia (ITP) is a rare platelet disorder that can lead to an increased tendency to bleed. Recommended first-line therapies include corticosteroids, intravenous immunogl ... Full text Cite

Examining Parental Medication Adherence as a Predictor of Child Medication Adherence in Pediatric Anxiety Disorders.

Journal Article Med Care · June 2018 BACKGROUND: Selective serotonin reuptake inhibitors (SSRIs) are the recommended first-line pharmacotherapy for pediatric anxiety disorders but adherence remains difficult to predict. OBJECTIVES: To estimate SSRI adherence in children with anxiety disorders ... Full text Link to item Cite

Treating Pediatric Anxiety: Initial Use of SSRIs and Other Antianxiety Prescription Medications.

Journal Article J Clin Psychiatry · 2018 OBJECTIVE: Multiple pharmacotherapies for treating anxiety disorders exist, including selective serotonin reuptake inhibitors (SSRIs), the recommended first-line pharmacotherapy for pediatric anxiety. We sought to describe initial antianxiety medication us ... Full text Link to item Cite

Use of bone-modifying agents among breast cancer patients with bone metastasis: evidence from oncology practices in the US.

Journal Article Clin Epidemiol · 2018 PURPOSE: Bone-modifying agents (BMAs) are recommended for women with bone metastasis from breast cancer to prevent skeletal-related events. We examined the usage patterns and identified the factors associated with the use of BMAs (denosumab and intravenous ... Full text Link to item Cite

Safety of Intravenous Iron in Hemodialysis: Longer-term Comparisons of Iron Sucrose Versus Sodium Ferric Gluconate Complex.

Journal Article Am J Kidney Dis · June 2017 BACKGROUND: Controversy exists about any differences in longer-term safety across different intravenous iron formulations routinely used in hemodialysis (HD) patients. We exploited a natural experiment to compare outcomes of patients initiating HD therapy ... Full text Link to item Cite

Comparative outcomes of predominant facility-level use of ferumoxytol versus other intravenous iron formulations in incident hemodialysis patients.

Journal Article Nephrol Dial Transplant · December 2015 BACKGROUND: Ferumoxytol was first approved for clinical use in 2009 solely based on data from trial comparisons with oral iron on biochemical anemia efficacy end points. To compare the rates of important patient outcomes (infection, cardiovascular events a ... Full text Link to item Cite

Association between Initial Fluid Choice and Subsequent In-hospital Mortality during the Resuscitation of Adults with Septic Shock.

Journal Article Anesthesiology · December 2015 BACKGROUND: Currently, guidelines recommend initial resuscitation with intravenous (IV) crystalloids during severe sepsis/septic shock. Albumin is suggested as an alternative. However, fluid mixtures are often used in practice, and it is unclear whether th ... Full text Open Access Link to item Cite

Control Outcomes and Exposures for Improving Internal Validity of Nonrandomized Studies.

Journal Article Health Serv Res · October 2015 OBJECTIVE: Control outcomes and exposures can improve internal validity of nonrandomized studies by assessing residual bias in effect estimates. Control outcomes are those expected to have no treatment effect or the opposite effect of the primary outcome. ... Full text Link to item Cite

Long-term Efficacy of Topical Fluorouracil Cream, 5%, for Treating Actinic Keratosis: A Randomized Clinical Trial.

Conference JAMA Dermatol · September 2015 IMPORTANCE: Topical fluorouracil was demonstrated to be effective in reducing the number of actinic keratoses (AKs) for up to 6 months, but no randomized trials studied its long-term efficacy. OBJECTIVE: To evaluate the long-term efficacy of a single cours ... Full text Link to item Cite

Longer-term outcomes of darbepoetin alfa versus epoetin alfa in patients with ESRD initiating hemodialysis: a quasi-experimental cohort study.

Journal Article Am J Kidney Dis · July 2015 BACKGROUND: Adequately powered studies directly comparing hard clinical outcomes of darbepoetin alfa (DPO) versus epoetin alfa (EPO) in patients undergoing dialysis are lacking. STUDY DESIGN: Observational, registry-based, retrospective cohort study; we mi ... Full text Link to item Cite

Association of Adverse Pregnancy Outcomes With Glyburide vs Insulin in Women With Gestational Diabetes.

Journal Article JAMA Pediatr · May 2015 IMPORTANCE: Glyburide is thought to be safe for use during pregnancy for treatment of gestational diabetes mellitus (GDM). However, there are limited data on the effectiveness of glyburide when compared with insulin as used in a real-world setting. OBJECTI ... Full text Link to item Cite

Trends in glyburide compared with insulin use for gestational diabetes treatment in the United States, 2000-2011.

Journal Article Obstet Gynecol · June 2014 OBJECTIVE: To describe trends and identify factors associated with choice of pharmacotherapy for gestational diabetes (GDM) from 2000-2011 using a healthcare claims database. METHODS: This was a retrospective cohort study of a large nationwide population o ... Full text Link to item Cite

Patient-centered medical homes and oral anticoagulation therapy initiation.

Journal Article Med Care Res Rev · April 2014 Despite evidence-based guidelines, oral anticoagulation therapy (OAT) initiation is low among incident atrial fibrillation (AF) patients. Patient-centered medical homes (PCMHs) may increase access, quality, and value through coordinating care. As such, PCM ... Full text Link to item Cite

A non-experimental study of oral anticoagulation therapy initiation before and after national patient safety goals.

Journal Article BMJ Open · February 12, 2014 OBJECTIVES: The Joint Commission revised its National Patient Safety Goals (NPSGs) to include oral anticoagulation therapy (OAT) in 2008. We sought to examine the effect of including OAT in The Joint Commission's NPSGs on historically low rates of OAT init ... Full text Link to item Cite

Declining rates of work-related overexertion back injuries among union drywall installers in Washington State, 1989-2008: Improved work safety or shifting of care?

Journal Article Am J Ind Med · February 2014 INTRODUCTION: Construction workers are at high risk of work-related musculoskeletal back disorders, and research suggests medical care and costs associated with these conditions may be covered by sources other than workers' compensation (WC). Little is kno ... Full text Link to item Cite

Effects of aggregation of drug and diagnostic codes on the performance of the high-dimensional propensity score algorithm: an empirical example.

Journal Article BMC Med Res Methodol · November 19, 2013 BACKGROUND: The High-Dimensional Propensity Score (hd-PS) algorithm can select and adjust for baseline confounders of treatment-outcome associations in pharmacoepidemiologic studies that use healthcare claims data. How hd-PS performance is affected by aggr ... Full text Link to item Cite

Use of Glyburide vs. Insulin for Treatment of Gestational Diabetes in the US, 2000-2010

Journal Article PHARMACOEPIDEMIOLOGY AND DRUG SAFETY · October 1, 2013 Link to item Cite

New Approaches to Account for Selection Bias and Confounding by Frail Health Status

Conference PHARMACOEPIDEMIOLOGY AND DRUG SAFETY · October 1, 2013 Link to item Cite

Reply to taguri and matsuyama.

Journal Article Stat Med · September 10, 2013 Full text Link to item Cite

An information criterion for marginal structural models.

Journal Article Stat Med · April 15, 2013 Marginal structural models were developed as a semiparametric alternative to the G-computation formula to estimate causal effects of exposures. In practice, these models are often specified using parametric regression models. As such, the usual conventions ... Full text Link to item Cite

Discontinuation and reinitiation patterns of osteoporosis treatment among commercially insured postmenopausal women.

Journal Article Int J Gen Med · 2013 OBJECTIVE: Poor adherence to chronic medications is common and compromises medication effectiveness. We sought to describe longitudinal patterns of osteoporosis medication use. STUDY DESIGN: This was a retrospective observational cohort study using 2005-20 ... Full text Link to item Cite

Implications of M bias in epidemiologic studies: a simulation study.

Journal Article Am J Epidemiol · November 15, 2012 Collider-stratification bias arises from conditioning on a variable (collider) which opens a path from exposure to outcome. M bias occurs when the collider-stratification bias is transmitted through ancestors of exposure and outcome. Previous theoretical w ... Full text Link to item Cite

A SAS macro for a clustered logrank test.

Journal Article Comput Methods Programs Biomed · November 2011 The clustered logrank test is a nonparametric method of significance testing for correlated survival data. Examples of its application include cluster randomized trials where groups of patients rather than individuals are randomized to either a treatment o ... Full text Link to item Cite

Standard Errors & Confidence Limits for Doubly Robust Estimation

Conference PHARMACOEPIDEMIOLOGY AND DRUG SAFETY · August 1, 2011 Link to item Cite

Doubly robust estimation of causal effects.

Journal Article Am J Epidemiol · April 1, 2011 Doubly robust estimation combines a form of outcome regression with a model for the exposure (i.e., the propensity score) to estimate the causal effect of an exposure on an outcome. When used individually to estimate a causal effect, both outcome regressio ... Full text Link to item Cite

The role of the c-statistic in variable selection for propensity score models.

Journal Article Pharmacoepidemiol Drug Saf · March 2011 The applied literature on propensity scores has often cited the c-statistic as a measure of the ability of the propensity score to control confounding. However, a high c-statistic in the propensity model is neither necessary nor sufficient for control of c ... Full text Link to item Cite

The increasing prevalence of atrial fibrillation among hemodialysis patients.

Journal Article J Am Soc Nephrol · February 2011 A half million Americans have ESRD, which puts them at high risk for cardiovascular disease and poor outcomes. Little is known about the epidemiology of atrial fibrillation among patients with ESRD. We analyzed data from annual cohorts (1992 to 2006) of pr ... Full text Link to item Cite