Daratumumab or Active Monitoring for High-Risk Smoldering Multiple Myeloma.

Background: Daratumumab, an anti-CD38 monoclonal antibody, has been approved for the treatment of multiple myeloma. Data are needed regarding the use of daratumumab for high-risk smoldering multiple myeloma, a precursor disease of active multiple myeloma for which no treatments have been approved.

Methods: In this phase 3 trial, we randomly assigned patients with high-risk smoldering multiple myeloma to receive either subcutaneous daratumumab monotherapy or active monitoring.

Meaningful Change Thresholds and Fatigue Severity Points on Patient-Reported Outcomes by the Fatigue Symptoms and Impacts Questionnaire in Patients With Relapsing Multiple Sclerosis.

Objectives: The Fatigue Symptoms and Impacts Questionnaire-Relapsing Multiple Sclerosis (FSIQ-RMS) is a new content-valid, concise, and reliable 20-item patient-reported outcome measure to evaluate the symptoms and impacts of fatigue in patients with relapsing forms of multiple sclerosis. Analyses were performed to derive meaningful change thresholds (MCTs) on patient-reported outcomes as measured by FSIQ-RMS and generate receiver operating characteristic (ROC) curves to determine fatigue severity cut points at baseline and change in severity at post-baseline and supplement the anchor-based MCT results.

Methods: Analyses were based on data from the OPTIMUM trial (NCT02425644).

How can patient preferences be used and communicated in the regulatory evaluation of medicinal products? Findings and recommendations from IMI PREFER and call to action.

Patients have unique insights and are (in-)directly affected by each decision taken throughout the life cycle of medicinal products. Patient preference studies (PPS) assess what matters most to patients, how much, and what trade-offs patients are willing to make. IMI PREFER was a six-year European public-private partnership under the Innovative Medicines Initiative that developed recommendations on how to assess and use PPS in medical product decision-making, including in the regulatory evaluation of medicinal products.

Psychometric evaluation of the Symptoms of Infection with Coronavirus-19 (SIC): results from a cross-sectional study and a phase 3 clinical trial.

Background: The Symptoms of Infection with Coronavirus-19 (SIC) is a 30-item patient-reported outcome (PRO) measure scored by body system composites to assess signs/symptoms of coronavirus disease 2019 (COVID-19). In addition to cross-sectional and longitudinal psychometric evaluations, qualitative exit interviews were conducted to support the content validity of the SIC.

Methods: In a cross-sectional study, adults diagnosed with COVID-19 in the United States completed the web-based SIC and additional PRO measures.

Development of patient- and observer-reported outcome measures to assess COVID-19 signs and symptoms in children and adolescents.

Background: The Symptoms of Infection with Coronavirus-19 (SIC) is a 30-item patient-reported outcome measure to evaluate the presence and severity of COVID-19 signs/symptoms in adults. This study expanded the context of use of the adult SIC among adolescents aged 12-17 years and supported a pediatric adaptation (the Pediatric SIC [PedSIC]) for caregiver assessment of signs/symptoms in children aged < 12 years.

Methods: Draft versions of the PedSIC and reference materials containing sign/symptom definitions for adolescents, based on an assessment of the reading level of SIC items by a professional linguist, were developed to facilitate accurate completion of the SIC by adolescents and observer-report (PedSIC) by caregivers.

U.S. Food and Drug Administration's Patient-Focused Drug Development Initiative: Experience with Integration of Patient-Experience Data in a New Drug Application for Esketamine Nasal Spray Plus a Newly Initiated Oral Antidepressant for Treatment-Resistant Depression.

The Patient-Focused Drug Development initiative of the U.S. Food and Drug Administration (FDA) aims to ensure that the patient experience of disease and treatment is an integral component of the drug development process.

Patient Preferences for Lung Cancer Treatments: A Study Protocol for a Preference Survey Using Discrete Choice Experiment and Swing Weighting.

Advanced treatment options for non-small cell lung cancer (NSCLC) consist of immunotherapy, chemotherapy, or a combination of both. Decisions surrounding NSCLC can be considered as preference-sensitive because multiple treatments exist that vary in terms of mode of administration, treatment schedules, and benefit-risk profiles. As part of the IMI PREFER project, we developed a protocol for an online preference survey for NSCLC patients exploring differences in preferences according to patient characteristics (preference heterogeneity).

Patient Preferences for Lung Cancer Treatment: A Qualitative Study Protocol Among Advanced Lung Cancer Patients.

Lung cancer is the deadliest and most prevalent cancer worldwide. Lung cancer treatments have different characteristics and are associated with a range of benefits and side effects for patients. Such differences may raise uncertainty among drug developers, regulators, payers, and clinicians regarding the value of these treatment effects to patients.

Benefit-Risk Assessment of Esketamine Nasal Spray vs. Placebo in Treatment-Resistant Depression.

This post hoc analysis assessed the benefit-risk profile of esketamine nasal spray + oral antidepressant (AD) induction and maintenance treatment in patients with treatment-resistant depression (TRD). The Benefit-Risk Action Team framework was utilized to assess the benefit-risk profile using data from three induction studies and one maintenance study. Benefits were proportion of remitters or responders in induction studies and proportion of stable remitters or stable responders who remained relapse-free in the maintenance study.

The atopic dermatitis itch scale: development of a new measure to assess pruritus in patients with atopic dermatitis.

Pruritus is the primary symptom of atopic dermatitis (AD). The objective of this study was to develop a patient-reported outcome (PRO) instrument for daily administration in clinical trials to measure AD-related itch in adolescents and adults that would meet the standards described in the US Food and Drug Administration's (FDA's) PRO Guidance. Six focus groups were conducted with 49 patients with AD (32 adults; 17 adolescents).

Evaluating the Effect of Standard of Care Treatment on Burden of Chronic Hepatitis B: A Retrospective Analysis of the United States Veterans Population.

Introduction: This study aimed to characterize chronic hepatitis B (CHB)-infected patients and estimate the association between nucleos(t)ide analogue (NA) persistence and economic outcomes using data from the Veterans Health Administration (VHA) database.

Methods: Patients (at least 18 years of age) with two or more claims for CHB and at least one pharmacy claim for NA were identified using VHA data from 1 April 2013 to 31 March 2018. The index date was the first NA prescription fill date during 1 October 2014 to 31 March 2017.

Physician and patient benefit-risk preferences from two randomized long-acting injectable antipsychotic trials.

Purpose: To quantify clinical trial participants' and investigators' judgments with respect to the relative importance of efficacy and safety attributes of antipsychotic treatments for schizophrenia, and to assess the impact of formulation and adherence.

Methods: Discrete-choice experiment surveys were completed by patients with schizophrenia and physician investigators participating in two phase-3 clinical trials of paliperidone palmitate 3-month long-acting injectable (LAI) antipsychotic. Respondents were asked to choose between hypothetical antipsychotic profiles defined by efficacy, safety, and mode of administration.

Microbiome-Gut-Brain Axis: Probiotics and Their Association With Depression.

To assess the association of probiotics with depression, a large population-based cross-sectional study was conducted. National Health and Nutrition Examination Survey adult participants from 2005 through 2012 were included. Exposure was defined as having consumed any probiotic food or supplement on any of the interview days.

Long-term Anticoagulation With Rivaroxaban for Preventing Recurrent VTE: A Benefit-Risk Analysis of EINSTEIN-Extension.

Background: Short-term anticoagulant treatment for acute DVT or pulmonary embolism (PE) effectively reduces the risk of recurrent disease during the first 6 to 12 months of therapy. Continued anticoagulation often is not instituted because of the perception among physicians that the risk of major bleeding will outweigh the risk of new venous thrombotic episodes.

Methods: The authors performed a benefit-risk analysis by using the randomized EINSTEIN-Extension trial, which compared continued rivaroxaban with placebo in 1,197 patients with symptomatic DVT or PE who had completed 6 to 12 months of anticoagulation and in whom physicians had equipoise with respect to the need for continued anticoagulation.

Associations of body mass index with incident hypertension in American white, American black and Chinese Asian adults in early and middle adulthood: the Coronary Artery Risk Development in Young Adults (CARDIA) study, the Atherosclerosis Risk in Communities (ARIC) study and the People's Republic of China (PRC) study.

The association of body mass index (BMI) with blood pressure may be stronger in Asian than non-Asian populations, however, longitudinal studies with direct comparisons between ethnicities are lacking. We compared the relationship of BMI with incident hypertension over approximately 9.5 years of follow-up in young (24-39 years) and middle-aged (45-64 years) Chinese Asians (n=5354), American Blacks (n=6076) and American Whites (n=13451).

Interactions between obesity, parental history of hypertension, and age on prevalent hypertension: the People's Republic of China Study.

Age, family history, and body mass index (BMI) influence the prevalence of hypertension, but very little is known about the interplay of these factors in Chinese populations. The authors examined this issue in Chinese adults (n = 4104) in the People's Republic of China Study. In young adults (24-39 years), the prevalence of hypertension/1000 persons (95% confidence interval [CI]) at the referent BMI was greater among subjects with a parental history of hypertension (35; 15-54) compared with those without (7; 3-11).

Hip circumference and incident metabolic risk factors in Chinese men and women: the People's Republic of China study.

Background: The associations between adiposity and metabolic risk factors have been suggested to vary across ethnicities. Studies in Caucasians have shown that after adjusting for waist circumference and body mass index (BMI), a larger hip circumference may be protective for metabolic risk factors. To our knowledge, these associations have never been examined in a Chinese population.

Impact of body mass index on incident hypertension and diabetes in Chinese Asians, American Whites, and American Blacks: the People's Republic of China Study and the Atherosclerosis Risk in Communities Study.

Researchers have hypothesized that the impact of body mass index on chronic disease may be greater in Asians than in Whites; however, most studies are cross-sectional and have no White comparison group. The authors compared the associations with body mass index in Chinese Asians (n = 5,980), American Whites (n = 10,776), and American Blacks (n = 3,582) using prospective data from the People's Republic of China Study (1983-1994) and the Atherosclerosis Risk in Communities Study (1987-1998). Slopes of risk differences over body mass index levels were compared among the three ethnic groups in adjusted analyses.