Identifying thresholds for meaningful improvements in NTDT-PRO scores to support conclusions about treatment benefit in clinical studies of patients with non-transfusion-dependent beta-thalassaemia: analysis of pooled data from a phase 2, double-blind, placebo-controlled, randomised trial.

Objectives: To estimate thresholds for defining meaningful within-patient improvement from baseline to weeks 13-24 and interpreting meaningfulness of between-group difference for the non-transfusion-dependent beta-thalassaemia patient-reported outcome (NTDT-PRO) tiredness/weakness (T/W) and shortness of breath (SoB) scores. A secondary objective was to determine the symptom severity threshold for the NTDT-PRO T/W domain to identify patients with symptomatic T/W.

Design: Pooled blinded data from the phase 2, double-blind, placebo-controlled, randomised BEYOND trial in NTDT (NCT03342404) were used.

Characterizing Vascular Wall and Lumen Caliber in Eyes with Diabetic Retinopathy Based on Adaptive Optics Scanning Laser Ophthalmoscopy.

Unlabelled: (200/200) Purpose: Our aim was to evaluate structural alterations of retinal arterioles due to type 1 diabetes (T1D) and/or diabetic retinopathy (DR) under AOSLO imaging.

Methods: Each study eye underwent mydriasis and AOSLO imaging in a single-visit study. The instrument's arrangement of four offset aperture images provided two orthogonal split-detector images and enabled isotropic analysis of the arteriolar boundaries.

Artificial Intelligence in Medical Education and Mentoring in Rehabilitation Medicine.

Artificial intelligence emerges as a transformative force, offering novel solutions to enhance medical education and mentorship in the specialty of physical medicine and rehabilitation. Artificial intelligence is a transformative technology that is being adopted in nearly every industry. In medicine, the use of artificial intelligence in medical education is growing.

Age, Race, Ethnicity, and Sex of Participants in Clinical Trials Focused on Chronic Pain.

There is limited data on equitable inclusion in chronic pain trials. We aimed to 1) identify the frequency of reporting age, race, ethnicity, and sex in clinical trials targeting chronic pain, and 2) compare sociodemographic representation to the United States (US) population. We examined US-based intervention trials for chronic pain initiated between 2007 and 2021 and registered on ClinicalTrials.

Transforming the Future of Surgeon-Scientists.

Objective: To create a blueprint for surgical department leaders, academic institutions, and funding agencies to optimally support surgeon-scientists.

Background: Scientific contributions by surgeons have been transformative across many medical disciplines. Surgeon-scientists provide a distinct approach and mindset toward key scientific questions.

Analysis of Race and Ethnicity Among United States Medical Board Leadership.

The inequitable representation of women and members of racial and ethnic minority groups in leadership positions within academic medicine is an ongoing challenge with practical and realistic solutions. The purpose of this study was to assess the race and ethnicity of individuals in leadership positions among the 24 Member Boards of Directors (Boards) of the American Board of Medical Specialties (ABMS). We performed a cross-sectional analysis of the race and ethnicity patterns for individuals holding leadership positions among the 24 Boards of the ABMS as of March 1, 2022.

Six Practical Strategies to Mentor and Sponsor Women in Academic Medicine.

This article focuses on the importance of mentorship and sponsorship for women in academic medicine, including trainees and faculty, and emphasizes the need for flexible and expanded definitions. Both the benefits and potential harms associated with sponsorship are described. There are 6 actionable strategies illustrated that may be added to a multidimensional mentoring model in order to better support women in medicine.

Antibodies against the Ebola virus soluble glycoprotein are associated with long-term vaccine-mediated protection of non-human primates.

The 2013 Ebola epidemic in Central and West Africa heralded the emergence of wide-spread, highly pathogenic viruses. The successful recombinant vector vaccine against Ebola (rVSVΔG-ZEBOV-GP) will limit future outbreaks, but identifying mechanisms of protection is essential to protect the most vulnerable. Vaccine-induced antibodies are key determinants of vaccine efficacy, yet the mechanism by which vaccine-induced antibodies prevent Ebola infection remains elusive.

Gender Representation Among United States Medical Board Leadership.

To assess the gender composition of the American Board of Medical Specialties' (ABMS) member boards and evaluate the equitable inclusion of women and a subset of women physicians. The gender of individuals on 24 boards as of March 1, 2022, was assessed. Two benchmarks-parity (50:50 representation) and equity (compared to the proportion of practicing physicians in each medical specialty)-were utilized to determine if women are equitably represented on medical boards.

Intratumoral administration of CD1c (BDCA-1) and CD141 (BDCA-3) myeloid dendritic cells in combination with talimogene laherparepvec in immune checkpoint blockade refractory advanced melanoma patients: a phase I clinical trial.

Background: Intratumoral (IT) myeloid dendritic cells (myDCs) play a pivotal role in initiating antitumor immune responses and relicensing of anti-tumor cytotoxic T lymphocytes within the tumor microenvironment. Talimogene laherparepvec (T-VEC) induces immunogenic cell death, thereby providing maturation signals and enhancing the release of tumor antigens that can be captured and processed by CD1c (BDCA-1) / CD141 (BDCA-3) myDCs, in order to reinvigorate the cancer-immunity cycle.

Methods: In this phase I trial, patients with advanced melanoma who failed standard therapy were eligible for IT injections of ≥1 non-visceral metastases with T-VEC on day 1 followed by IT injection of CD1c (BDCA-1) myDCs +/- CD141 (BDCA-3) myDCs on day 2.

Efficacy of anti-PD-1 and ipilimumab alone or in combination in acral melanoma.

Background: Acral melanoma is a rare melanoma subtype with poor prognosis. Importantly, these patients were not identified as a specific subgroup in the landmark melanoma trials involving ipilimumab and the anti-programmed cell death protein-1 (PD-1) agents nivolumab and pembrolizumab. There is therefore an absence of prospective clinical trial evidence regarding the efficacy of checkpoint inhibitors (CPIs) in this population.

Factors Associated With Burnout in Physical Medicine and Rehabilitation Residents in the United States.

Objectives: The aims of the study were to determine the prevalence of burnout in physical medicine and rehabilitation residents in the United States and to identify the personal- and program-specific characteristics most strongly associated with residents reporting burnout.

Design: This was a cross-sectional survey of US physical medicine and rehabilitation residents. Emotional exhaustion, depersonalization, and burnout were assessed using two validated items from the Maslach Burnout Inventory.

Adagrasib in Non-Small-Cell Lung Cancer Harboring a Mutation.

Background: Adagrasib, a KRAS inhibitor, irreversibly and selectively binds KRAS, locking it in its inactive state. Adagrasib showed clinical activity and had an acceptable adverse-event profile in the phase 1-1b part of the KRYSTAL-1 phase 1-2 study.

Methods: In a registrational phase 2 cohort, we evaluated adagrasib (600 mg orally twice daily) in patients with -mutated non-small-cell lung cancer (NSCLC) previously treated with platinum-based chemotherapy and anti-programmed death 1 or programmed death ligand 1 therapy.

Population pharmacokinetic/pharmacodynamic joint modeling of ixazomib efficacy and safety using data from the pivotal phase III TOURMALINE-MM1 study in multiple myeloma patients.

Ixazomib is an oral proteasome inhibitor approved in combination with lenalidomide and dexamethasone for the treatment of relapsed/refractory multiple myeloma (MM). Approval in the United States, Europe, and additional countries was based on results from the phase III TOURMALINE-MM1 (C16010) study. Here, joint population pharmacokinetic/pharmacodynamic time-to-event (TTE) and discrete time Markov models were developed to describe key safety (rash and diarrhea events, and platelet counts) and efficacy (myeloma protein [M-protein] and progression-free survival [PFS]) outcomes observed in TOURMALINE-MM1.

Avatrombopag for chemotherapy-induced thrombocytopenia in patients with non-haematological malignancies: an international, randomised, double-blind, placebo-controlled, phase 3 trial.

Background: Chemotherapy-induced thrombocytopenia is common and causes chemotherapy dose reductions or treatment delays, bleeding, and suboptimal oncological outcomes. We aimed to evaluate avatrombopag, a thrombopoietin receptor agonist that increases platelet counts, in patients with non-haematological cancer and platelet counts lower than 50 ×10 cells per L.

Methods: In this randomised, double-blind, placebo-controlled, phase 3 study, patients aged 18 years or older at 71 hospitals or cancer treatment centres in China, Hungary, Poland, Russia, Serbia, Ukraine, and the USA and with ovarian, bladder, or lung cancer receiving chemotherapy who had severe thrombocytopenia were randomly assigned (2:1) to oral avatrombopag 60 mg or oral placebo once daily given 5 days before and after chemotherapy, with randomisation stratified by number of chemotherapy drugs used.

Investigation of Health Behavior on Burnout Scores in Women Physicians who Self-Identify as Runners: A Cross-Sectional Survey Study.

As the proportion of women in the physician workforce increases, burnout in this population warrants further investigation. Exercise is an often-proposed strategy to combat burnout. Evaluating physical activity across a cohort of women physicians can assess associations of health behaviors with burnout.

Do Patients with Paget's Disease Have Worse Outcomes following Primary Total Knee Arthroplasty?

Well-powered studies evaluating the effects of Paget's disease on patient outcomes following primary total knee arthroplasty (TKA) are limited. The objective of this study was to determine whether Paget's disease patients undergoing primary TKA have higher rates of complications. A query of an administrative database was performed identifying Paget's disease patients undergoing primary TKA as the study cohort.