Real-world Effectiveness of Azacitidine in Treatment-Naive Patients With Higher-risk Myelodysplastic Syndromes.

Introduction: Azacitidine (AZA) is an approved frontline therapy for higher-risk myelodysplastic syndromes (HR-MDS); however, poor survival denotes unmet needs to increase depth/duration of response (DOR).

Methods: This retrospective study with patient chart review evaluated AZA effectiveness in 382 treatment-naive patients with HR-MDS from a US electronic health record (EHR)-derived database. Responses were assessed using International Working Group (IWG) 2006 criteria; real-world equivalents were derived from EHRs.

Clinical Pharmacology Applications of Real-World Data and Real-World Evidence in Drug Development and Approval-An Industry Perspective.

Since the 21st Century Cures Act was signed into law in 2016, real-world data (RWD) and real-world evidence (RWE) have attracted great interest from the healthcare ecosystem globally. The potential and capability of RWD/RWE to inform regulatory decisions and clinical drug development have been extensively reviewed and discussed in the literature. However, a comprehensive review of current applications of RWD/RWE in clinical pharmacology, particularly from an industry perspective, is needed to inspire new insights and identify potential future opportunities for clinical pharmacologists to utilize RWD/RWE to address key drug development questions.

Clinical characteristics and overall survival among acute myeloid leukemia patients with TP53 gene mutation or chromosome 17p deletion.

Approximately 5% to 15% of acute myeloid leukemia (AML) patients have TP53 gene mutations (TP53m), which are associated with very poor outcomes. Adults (≥18 years) with a new AML diagnosis were included from a nationwide, de-identified, real-world database. Patients receiving first-line therapy were divided into three cohorts: venetoclax (VEN) + hypomethylating agents (HMAs; Cohort A), intensive chemotherapy (Cohort B), or HMA without VEN (Cohort C).

Treatment outcomes for newly diagnosed, treatment-naïve TP53-mutated acute myeloid leukemia: a systematic review and meta-analysis.

Background: TP53 mutations, which are present in 5% to 10% of patients with acute myeloid leukemia (AML), are associated with treatment resistance and poor outcomes. First-line therapies for TP53-mutated (TP53m) AML consist of intensive chemotherapy (IC), hypomethylating agents (HMA), or venetoclax combined with HMA (VEN + HMA).

Methods: We conducted a systematic review and meta-analysis to describe and compare treatment outcomes in newly diagnosed treatment-naïve patients with TP53m AML.

Azacitidine Monotherapy in Patients With Treatment-Naïve Higher-risk Myelodysplastic Syndrome: A Systematic Literature Review and Meta-analysis.

Background: The global incidence of myelodysplastic syndromes (MDS) has been estimated as 0.06 to 0.26/100,000.

Integrating real-world data to accelerate and guide drug development: A clinical pharmacology perspective.

Pharmaceutical products in the current accelerated drug development landscape can benefit from tools beyond data generated from randomized control trials. We have seen an abundance of real-world data (RWD) and real-world evidence, driven by the digitalization of healthcare systems and an increased awareness that has inspired a heightened interest in their potential use. Literature review suggest leveraging RWD as a promising tool to answer key questions in the areas of clinical pharmacology and translational science.

A Retrospective Study of Comorbidities and Complications in Elderly Acute Myeloid Leukemia Patients in the United States.

Background: Comorbidities in acute myeloid leukemia (AML) patients have been shown to increase with age. However, few studies have described the disease burden in elderly AML patients, a population generally underrepresented in clinical trials. We aimed to characterize the comorbidities and complications in elderly AML patients.

Assessing Fever Frequency After Pediatric Live Attenuated Versus Inactivated Influenza Vaccination.

Background: Some studies have found a higher frequency of fever with trivalent live attenuated influenza vaccine (LAIV) than with inactivated influenza vaccine (IIV), but quadrivalent LAIV has not been assessed. Understanding fever is important for safety reviews and for parents and providers. In addition, there have been only a limited number of studies in which text messaging was used for vaccine adverse-event (AE) surveillance.

Preparation for global introduction of inactivated poliovirus vaccine: safety evidence from the US Vaccine Adverse Event Reporting System, 2000-12.

Background: Safety data from countries with experience in the use of inactivated poliovirus vaccine (IPV) are important for the global polio eradication strategy to introduce IPV into the immunisation schedules of all countries. In the USA, IPV has been included in the routine immunisation schedule since 1997. We aimed to analyse adverse events after IPV administration reported to the US Vaccine Adverse Event Reporting System (VAERS).

Updated recommendations for the use of typhoid vaccine--Advisory Committee on Immunization Practices, United States, 2015.

These revised recommendations of the Advisory Committee on Immunization Practices update recommendations published in MMWR in 1994 and include updated information on the two currently available vaccines and on vaccine safety. They also include an update on the epidemiology of enteric fever in the United States, focusing on increasing drug resistance in Salmonella enterica serotype Typhi, the cause of typhoid fever, as well as the emergence of Salmonella serotype Paratyphi A, a cause of paratyphoid fever, against which typhoid vaccines offer little or no protection.

Relationship between Guillain-Barré syndrome, influenza-related hospitalizations, and influenza vaccine coverage.

Some studies reported an increased risk of Guillain-Barré syndrome (GBS) within six weeks of influenza vaccination. It has also been suggested that this finding could have been confounded by influenza illnesses. We explored the complex relationship between influenza illness, influenza vaccination, and GBS, from an ecologic perspective using nationally representative data.

Using the exhibited generalization approach to evaluate a carbon monoxide alarm ordinance.

Current interests in enhancing the focus of external validity or transferability call for developing practical evaluation approaches and illustrating their applications in this area for meeting the need. This study takes the challenge by introducing an innovative evaluation approach, named the exhibited generalization approach, and applying it in evaluating the carbon monoxide (CO) alarm ordinance passed by Mecklenburg County, North Carolina. The stakeholders specifically asked evaluators to determine the answers to the following two questions: (1) Does the alarm ordinance work? (2) What generalizable information can the Mecklenburg experience provide to other jurisdictions trying to decide if the alarm ordinance's planning, implementation, adoption, and outcomes are transferable to their communities? This study illustrates how to apply the exhibited generalization approach to provide the stakeholders with answers to these questions.

Cumulative risk of Guillain-Barré syndrome among vaccinated and unvaccinated populations during the 2009 H1N1 influenza pandemic.

Objectives: We sought to assess risk of Guillain-Barré syndrome (GBS) among influenza A (H1N1) 2009 monovalent (pH1N1) vaccinated and unvaccinated populations at the end of the 2009 pandemic.

Methods: We applied GBS surveillance data from a US population catchment area of 45 million from October 15, 2009, through May 31, 2010. GBS cases meeting Brighton Collaboration criteria were included.

Guillain-Barre syndrome, influenza, and influenza vaccination: the epidemiologic evidence.

Guillain-Barré syndrome (GBS) is the most common cause of acute flaccid paralysis worldwide, and is thought to be immune-mediated. It is preceded by upper respiratory or gastrointestinal infection in about two-thirds of cases and is associated with some viral infections, including influenza. GBS has also been associated with the 1976 swine-influenza vaccine.

Association between birthplace and current asthma: the role of environment and acculturation.

Objectives: We evaluated associations between current asthma and birthplace among major racial/ethnic groups in the United States.

Methods: We used multivariate logistic regression methods to analyze data on 102,524 children and adolescents and 255,156 adults in the National Health Interview Survey (2001-2009).

Results: We found significantly higher prevalence (P < .

Number of antigens in early childhood vaccines and neuropsychological outcomes at age 7-10 years.

Purpose: Concerns have been raised that children may be receiving too many immunizations under the recommended schedule in the USA. We used a publicly available dataset to evaluate the association between antibody-stimulating proteins and polysaccharides from early childhood vaccines and neuropsychological outcomes at age 7-10 years.

Methods: Children aged 7-10 years from four managed care organizations underwent standardized tests for domain-specific neuropsychological outcomes: general intellectual function, speech and language, verbal memory, attention and executive function, tics, achievement, visual spatial ability, and behavior regulation.

Racial and ethnic disparities in current asthma and emergency department visits: findings from the National Health Interview Survey, 2001-2010.

Objectives: Racial/ethnic disparities in current asthma prevalence and medical care are a major public health concern. We examined the differences in asthma prevalence and morbidity among major racial/ethnic populations in the US.

Methods: We analyzed data from the 2001-2010 National Health Interview Survey for adults (≥18 years) and children and adolescents (<18 years).

National carbon monoxide poisoning surveillance framework and recent estimates.

Objectives: Unintentional, non-fire-related (UNFR) carbon monoxide (CO) poisoning is a leading cause of poisoning in the United States. A comprehensive national CO poisoning surveillance framework is needed to obtain accurate estimates of CO poisoning burden and guide prevention efforts. This article describes the current national CO poisoning surveillance framework and reports the most recent national estimates.

A review of disaster-related carbon monoxide poisoning: surveillance, epidemiology, and opportunities for prevention.

Objectives: We conducted a systematic literature review to better understand aspects of disaster-related carbon monoxide (CO) poisoning surveillance and determine potentially effective prevention strategies.

Methods: This review included information from 28 journal articles on disaster-related CO poisoning cases occurring between 1991 and 2009 in the United States.

Results: We identified 362 incidents and 1888 disaster-related CO poisoning cases, including 75 fatalities.

Residential carbon monoxide alarm prevalence and ordinance awareness.

Objective: Unintentional carbon monoxide (CO) poisoning is a leading cause of poisoning in the United States. Most poisoning cases occur in residential settings and a working CO alarm may prevent many of these events. The use of a CO alarm is mandated in many parts of the country; however, little is known about the compliance and adoption of such ordinances at the population level.

Environmental lead after Hurricane Katrina: implications for future populations.

Background: As a result of Hurricane Katrina, > 100,000 homes were destroyed or damaged and a significant amount of sediment was deposited throughout the city of New Orleans, Louisiana. Researchers have identified the potential for increased lead hazards from environmental lead contamination of soils.

Objectives: We assessed the distribution of residential soil and dust lead 2 years poststorm and compared soil lead before and after the storm.

Recipients of hyperbaric oxygen treatment for carbon monoxide poisoning and exposure circumstances.

Background: Unintentional carbon monoxide poisoning is preventable. Severe cases are often referred for hyperbaric oxygen treatment. To guide prevention efforts and treatment practices, this study provides some of the most detailed current information about patients with carbon monoxide poisoning who have been treated at hyperbaric facilities across the United States and the circumstances surrounding their exposures.

Factors associated with asthma control among adults in five New England states, 2006-2007.

Background: Despite the National Asthma Education and Prevention Program (NAEPP) guidelines that specify the goals of asthma control and management strategies, the number of patients with uncontrolled asthma remains high, and factors associated with uncontrolled asthma are unknown.

Objective: The aim was to examine the relationship between asthma control and socio-demographic characteristics, health-care access and use, asthma education, and medication use among adults with active asthma residing in New England.

Methods: Data from the 2006-2007 Behavior Risk Factor Surveillance System Adult Asthma Call-Back Survey were analyzed using multinomial logistic regression.

Hospital burden of unintentional carbon monoxide poisoning in the United States, 2007.

Background: Unintentional, non-fire-related (UNFR) carbon monoxide (CO) poisoning is a leading cause of poisoning in the United States, but the overall hospital burden is unknown. This study presents patient characteristics and the most recent comprehensive national estimates of UNFR CO-related emergency department (ED) visits and hospitalizations.

Methods: Data from the 2007 Nationwide Inpatient and Emergency Department Sample of the Hospitalization Cost and Utilization Project were analyzed.

Carbon monoxide poisoning after an ice storm in Kentucky, 2009.

Objectives: Carbon monoxide (CO) poisoning is a leading cause of morbidity and mortality during natural disasters. On January 26-27, 2009, a severe ice storm occurred in Kentucky, causing widespread, extended power outages and disrupting transportation and communications. After the storm, CO poisonings were reported throughout the state.