Improving the Likelihood of Identifying Alpha-1 Antitrypsin Deficiency Among Patients With COPD: A Novel Predictive Model Using Real-World Data.

Background: Despite guideline recommendations, most patients with chronic obstructive pulmonary disease (COPD) do not undergo alpha-1 antitrypsin deficiency (AATD) testing and approximately 90% of people with AATD in the United States remain undiagnosed. This study sought to develop a predictive model using real-world data to improve detection of AATD-positive patients in the general COPD population.

Methods: A predictive model using extreme gradient boosting was developed using the EVERSANA database, including longitudinal, patient-level medical claims, prescription claims, AATD-specific testing data, and electronic health records (EHR).

Post-Hoc Assessment of Cognitive Efficacy in Alzheimer's Disease Using a Latent Growth Mixture Model in AMBAR, a Phase 2B Randomized Controlled Trial.

Background: Disease progression in Alzheimer's Dementia (AD) is typically characterized by accelerated cognitive and functional decline, where heterogeneous trajectories can impact the observed treatment response.

Methods: We hypothesized that unobserved heterogeneity could obscure treatment benefits in AD. The effect of unobserved heterogeneity was empirically quantified within the Alzheimer's Management By Albumin Replacement (AMBAR) phase 2b trial data.

Outcomes of intravenous immunoglobulin treatment of immunocompromised patients with viral respiratory infections.

Background: Limited guidelines exist for treating immunocompromised patients hospitalized for acute viral respiratory infection. Little is known about clinical and economic benefits of intravenous immunoglobulin (IVIG) administration in patients with acute viral respiratory infections.

Objective: To compare clinical and economic outcomes among immunocompromised patients hospitalized with viral respiratory infections who received IVIG with those who did not.

Understanding treatment goals and their application in clinical trial design for patients with Alzheimer disease and caregivers.

Background: As research continues towards improved treatments for Alzheimer disease (AD), there is growing interest in the views and needs from patients and caregivers on AD treatments.

Methods: In this study, we surveyed patients with AD and caregivers to determine the treatment goals that are most important to them. Patients with AD and caregivers were independently recruited in Europe and North America to complete a web-based survey.

Application of a Novel Endpoint Staging Framework: Proof of Concept in the AMBAR Study.

Background: A theoretical endpoint staging framework was previously developed and published, aligning outcomes (i.e., memory) to the stage of Alzheimer's disease (AD) in which a given outcome is most relevant (i.

Toward the optimized assessment of clinical outcomes in studies of novel treatments for Alzheimer's disease.

Introduction: Alzheimer's disease (AD) is characterized by a progressive decline in cognition and daily function, leading to a greater need for caregiver support. Clinical disease is segmented into a preclinical stage, mild cognitive impairment, and mild, moderate, and severe stages of Alzheimer's dementia. Although AD trials enroll participants at various stages of illness, treatment efficacy is often assessed using endpoints based on measures of outcomes that are held fixed across disease stages.

Primary Immune Deficiency: Patients' Preferences for Replacement Immunoglobulin Therapy.

Purpose: Immunoglobulin (Ig) replacement therapy is an important life-saving treatment modality for patients with primary antibody immune deficiency disorders (PAD). IVIG and SCIg are suitable alternatives to treat patients with PAD but vary in key ways. Existing evidence on patient preferences for Ig treatments given the complexities associated with IVIG and SCIg treatment is limited and fails to account for variations in preferences across patients.

Social Restrictions versus Testing Campaigns in the COVID-19 Crisis: A Predictive Model Based on the Spanish Case.

The global COVID-19 spread has forced countries to implement non-pharmacological interventions (NPI) (i.e., mobility restrictions and testing campaigns) to preserve health systems.

Comorbidity Associations with AATD Among Commercially Insured and Medicare Beneficiaries with COPD in the US.

Introduction: Alpha-1 antitrypsin deficiency (AATD) is often not identified in patients with chronic obstructive pulmonary disease (COPD) until advanced stages of disease, despite the availability of genetic testing. While clinical practice guidelines provide recommendations on patients who should be tested, more refined algorithms are needed to identify COPD patients who are likely candidates for AATD testing and to prevent delays in diagnosis and treatment. The objective of this study was to identify comorbid associations with AATD among patients diagnosed with COPD in the United States.

Correction to: The cost-effectiveness of albumin in the treatment of decompensated cirrhosis in Germany, Italy, and Spain.

Following publication of the original article [1], the authors reported that one of the numbers within Fig. 6 contains a mistake.

Differences in Patient Demographics and Healthcare Costs of Patients with PIDD Receiving Intravenous or Subcutaneous Immunoglobulin Therapies in the United States.

Background: Primary immune-deficiency disease (PIDD) is a rare, debilitating disease of the immune system that predisposes the affected individual to infection, autoimmune conditions, and neoplasm. A major component of the cost of treating PIDD is the high price of immunoglobulin drugs, which can be administered via an intravenous (IV) or subcutaneous (SC) route.

Objective: To compare real-world costs for patients with PIDD who are receiving IV immunoglobulin (IVIG) or SC immunoglobulin (SCIG) treatment, from a US payer perspective, using a large claims database.

Immunoglobulin administration for the treatment of CIDP: IVIG or SCIG?

Chronic inflammatory demyelinating polyneuropathy (CIDP) is an acquired neurological disorder characterized clinically by weakness and impaired sensory function evolving over 2 months or more, loss or significant decrease in deep tendon reflexes, and by electrophysiological evidence of peripheral nerve demyelination. Expeditious diagnosis and treatment of CIDP early in the disease course is critical such that irreversible disability can be avoided. Intravenous immunoglobulin (IVIG) is one first-line and maintenance therapy option for CIDP.

Treatment Patterns and Costs of Chronic Inflammatory Demyelinating Polyneuropathy: A Claims Database Analysis.

Background: Corticosteroids, plasma exchange, and intravenous immunoglobulin (IVIG) have been standard-of-care treatments for chronic inflammatory demyelinating polyneuropathy (CIDP) for more than 2 decades. Despite guideline recommendations for best clinical practices, heterogeneity in patient presentation and the course of treatment for CIDP remains. There is limited literature regarding the real-world treatment patterns of and costs associated with CIDP.

The cost-effectiveness of albumin in the treatment of decompensated cirrhosis in Germany, Italy, and Spain.

Background: Albumin is frequently prescribed in cirrhotic patients with acute decompensation. However, the true cost effectiveness of albumin use in cirrhotic patients is still under debate.

Objective: To evaluate the cost-effectiveness of albumin in the treatment of decompensated cirrhosis in Germany, Italy, and Spain.

Two comparative assessments of intravenous immunoglobulin therapy switching patterns in the treatment of chronic inflammatory demyelinating polyneuropathy in the US.

For chronic inflammatory demyelinating polyneuropathy (CIDP) patients, each branded intravenous immunoglobulin (IVIG) treatment differs in production processes, virus elimination, formulation, and composition. Given the limited availability of real-world data comparing IVIGs for CIDP, this study evaluated switching patterns between IVIG products in 2 separate retrospective databases. Two independent analytic teams retrospectively evaluated IVIG treatment-naïve patients with an ICD diagnosis code for CIDP.

Cost analysis of plasma-derived factor VIII/von Willebrand factor versus recombinant factor VIII for treatment of previously untreated patients with severe hemophilia A in the United States.

Background: Inhibitor development to factor VIII (FVIII) hemophilia therapy results in increased complications and substantial economic costs. The SIPPET study, the first randomized controlled trial to compare the immunogenicity of plasma-derived FVIII (pdFVIII)/von Willebrand factor (VWF) and recombinant-DNA-derived FVIII (rFVIII), demonstrated higher inhibitor rates in previously untreated patients (PUPs) treated with rFVIII than in PUPs treated with pdFVIII/VWF.

Objective: To quantify the economic impact of treating PUPs with pdFVIII/VWF vs rFVIII.

Impact of a Health Management Program on Healthcare Outcomes among Patients on Augmentation Therapy for Alpha 1-Antitrypsin Deficiency: An Insurance Claims Analysis.

Introduction: Alpha 1-antitrypsin deficiency (AATD) is a genetic disorder which reduces serum alpha 1-antitrypsin (AAT or alpha1-proteinase inhibitor, A1PI) and increases the risk of chronic obstructive pulmonary disease (COPD). Management strategies include intravenous A1PI augmentation, and, in some cases, a health management program (Prolastin Direct; PD).

Objectives: This study compared clinical and economic outcomes between patients with and without PD program participation.

The dilemma of diabetes in chronic inflammatory demyelinating polyneuropathy.

Purpose: We reviewed the literature on chronic inflammatory demyelinating polyneuropathy (CIDP) in diabetes mellitus (DM) and explored real-world data on the prevalence and treatment of CIDP within DM.

Methods: A literature search of Scopus was performed for the terms chronic inflammatory demyelinating polyradiculoneuropathy, chronic inflammatory demyelinating polyneuropathy, CIDP, and prevalence, incidence, epidemiology, or diabetes; peripheral neuropathy and prevalence or diabetes. We also searched through the reference lists of the resulting publications for additional findings that may have been missed.

Migraine-related healthcare resource use and costs for subjects prescribed fixed-dose combination sumatriptan/naproxen sodium vs. single-entity oral triptans in a managed care population in the USA.

Background: Randomized clinical trials have demonstrated that the efficacy of a fixed-dose single-tablet combination containing sumatriptan and naproxen sodium (S/NS) was greater than either of its individual components. Simplifying drug regimens (e.g.

Sumatriptan/naproxen sodium for the acute treatment of probable migraine without aura: a randomized study.

Objective: Probable migraine is a common, disabling migraine subtype fulfilling all but one of the diagnostic criteria for migraine. This study was conducted to evaluate the efficacy and tolerability of sumatriptan/naproxen sodium for the acute treatment of probable migraine without aura.

Methods: Patients treated a headache of probable migraine without aura when pain was moderate or severe with sumatriptan/naproxen sodium ( N  = 222 intent-to-treat (ITT)) or placebo ( N  = 221 ITT/complete case analysis (a) ) in this randomized, double-blind, parallel-group study.

Consistency of return to normal function, productivity, and satisfaction following migraine attacks treated with sumatriptan/naproxen sodium combination.

Objectives: To assess the consistency of improved functioning, productivity, and medication satisfaction in migraines treated with a single tablet of sumatriptan 85 mg/naproxen sodium 500 mg (S/NS) using an early intervention approach.

Methods: Two randomized, double-blind, placebo-controlled, 4-period crossover, multi-attack, multi-center, outpatient studies of moderate to severe adult migraineurs were conducted to compare S/NS with placebo. Participants recorded outcome assessments in a diary during the 24 hours following study medication.

Impact of NSAID and Triptan use on developing chronic migraine: results from the American Migraine Prevalence and Prevention (AMPP) study.

Objectives: To assess the influence of triptan or nonsteroidal anti-inflammatory drug (NSAID) use on the likelihood of developing chronic migraine (CM) among persons with episodic migraine (EM).

Background: CM is common in tertiary headache care, and relative to EM, CM is associated with a number of deleterious outcomes, including higher headache-related disability, reduced health-related quality of life, and increased direct and indirect costs. Symptomatic medication use has emerged as an important risk factor for the development of CM.

Evaluating migraineurs' preferences for migraine treatment outcomes using a choice experiment.

Objective: The impact of migraines on patients is commonly divided between the level of impairment associated with headache symptoms (headache phase) and the quality-of-life effects immediately following the headache (post-headache phase). Evaluations of migraineurs' productivity losses and health-related quality of life have provided an understanding of the burden associated with the headache and post-headache symptoms, but do not quantify the relative importance of each phase from a patient perspective. In this study, we evaluated migraineurs' willingness to accept trade-offs among symptom severity in the headache and post-headache phases, symptom duration in the headache and post-headache phases, and symptom-free time within a general-preference theoretic framework.

A cross-sectional survey to assess the migraineur's medication decision-making beliefs: determining when a migraine is triptan-worthy.

Objectives: To investigate the factors that influence a migraineur's beliefs regarding oral triptans for the acute treatment of migraines and to provide further insight into patients' decision-making process when faced with migraine.

Methods: A multicenter, cross-sectional, observational study of subjects currently prescribed an oral triptan medication for the acute treatment of migraine headaches. Subjects were recruited from 6 headache clinics and one primary care practice in the United States.