With recent approvals in Japan, identifying optimal first-line therapy for metastatic castration-sensitive prostate cancer (mCSPC) is critical. We evaluated real-world outcomes of androgen-deprivation therapy (ADT) plus androgen receptor signaling inhibitors (ARSIs) as first-line therapies using prostate-specific antigen (PSA) responses and treatment duration. This retrospective study used the Medical Data Vision database (May 2020–April 2024). The study population included males with mCSPC, with two cohorts: cohort 1 (with PSA data); cohort 2 (all eligible patients). Primary endpoint: cumulative incidence of ≥ 90
The MiniMed™ 780G (MM780G) advanced hybrid closed-loop system has been shown to help improve glycemic control compared with continuous glucose monitoring plus multiple daily injections of insulin (MDI) in people with type 1 diabetes (T1D). The long-term health economic outcomes with MM780G versus MDI + CGM in people with T1D in Australia and HbA1c levels above target were investigated. The analysis was performed using the IQVIA Core Diabetes Model. Clinical input data were sourced from a randomized controlled trial with HbA1c reductions of 1.54
Well-powered, prospective, registry-based clinical studies present the opportunity for broad safety surveillance of bioscaffolds used in soft tissue reconstruction but remain limited in the field. The Myriad™ Augmented Soft Tissue Reconstruction Registry (“MASTRR Registry”) was established as an ongoing multicenter prospective registry to evaluate the real-world safety and performance of ovine forestomach matrix (OFM)-based devices in complex soft tissue reconstruction. The interim safety analysis included prospective subject data from the first 411 subjects and 474 defects undergoing reconstruction with OFM-based devices from 10 participating sites. The analysis included subjects from trauma, colorectal, burn, and limb salvage specialties. All adverse events (AEs) were categorized based on severity, causality in relation to the investigational device, and correlated to the surgical procedure and relationship to the index defect. Subjects represented a medically complex population with substantial comorbidity burden and challenging defect characteristics. Most defects were chronic (59.8
According to recent data 21% of hematology fellowship applications in Italy remained unfilled [1]. Similarly, medical specialties such as surgery, pathology, radiotherapy, and emergency medicine have seen a significant reduction in the number of applicants. Coupled with the chronic shortage of nursing staff, the outlook for hematology as a specialty is concerning: within a few years, Italy risks lacking key healthcare professionals to treat patients with hematological malignancies.
Fostamatinib, an oral spleen tyrosine kinase (SYK) inhibitor, prevents antibody-mediated platelet destruction and has proven efficacy and safety in immune thrombocytopenia (ITP). However, evidence from real-world use in earlier treatment lines is limited. Here the objective was to evaluate the effectiveness and safety of fostamatinib as second- or third-line therapy in adult patients with ITP in Spain. This multicenter study included 72 adult patients treated with fostamatinib across 22 Spanish centers, assessed retrospectively and prospectively. Demographic, clinical, and laboratory data, treatment responses, and adverse events (AEs) were analyzed. Median age was 67 years, and 55.6
Efgartigimod is a human immunoglobulin G1 (IgG) antibody Fc fragment that reduces levels of circulating IgG, including pathogenic autoantibodies, by blocking neonatal Fc receptor. The original 1000-mg fixed-dose formulation of efgartigimod coformulated with recombinant human hyaluronidase PH20 for subcutaneous injection (PH20 SC) is supplied in a single-dose vial and administered with a separate syringe (V + S). To increase patient convenience, a 5-ml prefilled syringe (PFS) of efgartigimod PH20 SC has been developed. These studies examined the pharmacokinetics, injection speed, and usability of the efgartigimod PH20 SC PFS. Bioequivalence of efgartigimod PH20 SC administered via PFS or V + S was assessed in an open-label study in healthy participants. Feasibility, safety, and tolerability of injecting efgartigimod PH20 SC at different injection speeds ranging from 20 to 60 s was examined in a separate open-label study in healthy participants. Finally, two human factor (HF) validation studies investigated usability of the efgartigimod PH20 SC PFS by participants with generalized myasthenia gravis (gMG) or chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) and lay caregivers in a simulated at-home environment. Pharmacokinetic bioequivalence was established between efgartigimod PH20 SC administered by PFS and V + S. No meaningful differences were observed between mean fluid leakage/backflow volume at the injection site between the injection speed groups, and no meaningful differences in tolerability across injection speeds occurred. All participants in the HF studies successfully used the efgartigimod PH20 SC PFS and associated instructional materials without training to deliver the full injection volume in an average of 30 s. These studies support the bioequivalence, safety, and feasibility of an efgartigimod PH20 SC PFS. Both participants with gMG or CIDP and lay caregivers successfully administered the full dose of the efgartigimod PH20 SC PFS. Rapid-administration efgartigimod PH20 SC PFS is safe and feasible for both patients with autoimmune diseases and lay caregivers. ClinicalTrials.gov identifier: NCT05817435. Efgartigimod is a medication that reduces immunoglobulin G, a protein that can attack the body by mistake in some autoimmune diseases. Previously, efgartigimod injected under the skin (efgartigimod PH20 SC) was only available in a vial, and health care providers needed to fill a separate syringe to give the medication to a patient. Now, an efgartigimod PH20 SC prefilled syringe is available, which lets patients give themselves their medication more easily. The goals of these studies were to look at: (1) how the body processes efgartigimod PH20 SC once it is injected from either the prefilled syringe or a vial and syringe, (2) how safe it was to quickly inject efgartigimod PH20 SC (between 20–60 s), and (3) whether patients with generalized myasthenia gravis or chronic inflammatory demyelinating polyradiculoneuropathy (both autoimmune diseases) and their caregivers could safely use the prefilled syringe at home. These studies found that the same amount of efgartigimod PH20 SC enters the body whether it is injected using the prefilled syringe or a vial and syringe. Efgartigimod PH20 SC was also safely injected over 20, 30, 45, or 60 s. Lastly, untrained participants with autoimmune diseases or caregivers were able to use the instructions given and inject all the medication from the prefilled syringe without error. A prefilled syringe of efgartigimod PH20 SC may provide patients with certain autoimmune diseases and their caregivers with a quick, convenient, and flexible option for receiving their medication in a health care facility or at home.
The primary objective of this study was to report real-world outcomes of the travoprost intracameral implant (iDose TR) as standalone surgery or combined with minimally invasive glaucoma surgery (MIGS). The secondary objective was to compare efficacy across subgroups stratified by surgical category, severity, and race/ethnicity. This was a retrospective nonrandomized single-center consecutive case series of iDose TR implantations, with or without MIGS. Inclusion criteria were primary open-angle glaucoma, mild-to-severe stage, and minimum 3 months’ follow-up. Eyes with visually significant cataract undergoing combined surgery also underwent concomitant phacoemulsification. Efficacy endpoints were mean and proportional analyses of IOP and medication burden (at baseline vs 3 months). The study included 85 eyes. iDose TR was a standalone procedure in 40 eyes (47.1
This is a summary of the original research article “Brentuximab Vedotin and Nivolumab in Combination with Chemotherapy for Nonbulky, Early-Stage Classical Hodgkin Lymphoma.” The phase 2 SGN35-027 study (NCT03646123) is a multiple-part clinical trial of brentuximab vedotin (BV), with nivolumab, doxorubicin, and dacarbazine (AN + AD), in classical Hodgkin lymphoma (cHL). Here, we present the efficacy and safety of AN + AD from part C of this study in patients with nonbulky, early-stage cHL. At the time of this analysis, 154 patients had received ≥ 1 dose of AN + AD and 98
Trastuzumab deruxtecan (T-DXd) is a human epidermal growth factor receptor 2 (HER2)-targeting antibody–drug conjugate that has demonstrated encouraging efficacy and a manageable safety profile in the second-line or later setting for non-small cell lung cancer (NSCLC) harboring HER2 mutations. While T-DXd was approved for treating patients with locally advanced or metastatic HER2-mutant NSCLC in China, real-world data on its use in Chinese clinical practice are lacking. This study will collect real-world data on T-DXd to evaluate its effectiveness and safety in Chinese patients with HER2-mutant metastatic NSCLC, thereby providing additional evidence to the oncology community in China. RERUN is a prospective, multicenter, observational cohort study conducted at approximately 30 sites in China. Approximately 150 adult patients (≥ 18 years) with pathologically documented unresectable and/or metastatic non-squamous NSCLC harboring any known activating HER2 mutation are currently being enrolled. The follow-up period will last approximately 6 months after the last patient is enrolled, when sufficient progression-free survival (PFS) maturity (approximately 60
Abrocitinib is approved for moderate-to-severe atopic dermatitis (AD) treatment. Real-world abrocitinib usage data are limited. The aim of this study was to characterize patients initiating abrocitinib in clinical settings in the United States. Retrospective, observational study of patients aged ≥ 12 years with AD using linked claims (index date: abrocitinib initiation) and electronic health records in the OMNY Health real-world data platform (January 1, 2017–October 31, 2025). Baseline demographics, characteristics, comorbidities, medication history, and healthcare resource utilization were evaluated. Abrocitinib dose changes ≤ 12 months post-index, early persistence (prescription refill within 60 days of index), and AD severity changes from baseline were assessed. Overall, 401 patients initiated abrocitinib (8.5
Cholestatic pruritus is common in primary biliary cholangitis (PBC), adversely affecting quality of life. This study aimed to establish the within-patient meaningful change threshold (MCT) for the Worst Itch Numerical Rating Scale (WI-NRS) in patients with PBC and pruritus. Determining MCTs for patient-reported outcomes is crucial for interpreting treatment efficacy in clinical trials. Given the high impact of pruritus on sleep in PBC, an MCT was also estimated for the Sleep Interference NRS (SI-NRS), which assesses pruritus-related sleep interference. Analyses were performed on blinded data from the Phase 3 GLISTEN study of linerixibat in patients with PBC and moderate-to-severe pruritus (NCT04950127). Anchor-based methods included the Patient Global Impression of Severity (PGI-S) and Change (PGI-C) as primary and supportive anchors, respectively. Correlations between weekly averages of daily WI-NRS and SI-NRS change scores and the anchors were assessed. A ‘1-point’ improvement in PGI-S and ‘moderately improved’ on PGI-C were considered key reference groups for MCT determination. MCTs were evaluated in patients who reported changes in their itch and/or sleep were meaningful, as indicated by PGI-C item 2. Cumulative distribution function curves and distribution-based methods provided supporting evidence. Data from 238 patients were included. Among patients reporting meaningful change in itch at Week 24, mean (standard deviation) change in WI-NRS was − 3.0 (1.7) for the ‘1-point’ PGI-S improvers and − 2.9 (2.0) for the ‘moderately improved’ PGI-C group. Corresponding values for SI-NRS were − 2.6 (2.1) and − 2.4 (2.1). Distribution-based estimates for the change in WI-NRS and SI-NRS each ranged from 0.42 to 0.85. This study demonstrated that a reduction of ≥ 3 points in WI-NRS and ≥ 2.5 points in SI-NRS weekly scores represent clinically meaningful improvement thresholds for patients with PBC and moderate-to-severe pruritus. These results support use of WI-NRS and SI-NRS in future PBC trials evaluating new treatments for pruritus. People living with primary biliary cholangitis (PBC), a liver disease, often experience severe itching (also known as pruritus) that can disrupt sleep and reduce quality of life. To measure symptoms in PBC, numerical rating scales from 0 to 10 are commonly used. Two important scales are the Worst Itch Numerical Rating Scale, which measures the severity of itch, and the Sleep Interference Numerical Rating Scale, which measures how much itching affects sleep. This study looked at what level of improvement on these scales are considered meaningful by patients with PBC. Researchers analyzed data from 238 patients enrolled in a clinical trial that tested a treatment called linerixibat for itch relief in patients with PBC. They used several methods to determine what changes on the Worst Itch Numerical Rating Scale and Sleep Interference Numerical Rating Scale truly mattered to patients. The study found that a reduction of at least 3 points on the Worst Itch Numerical Rating Scale for itch severity, and 2.5 points on the Sleep Interference Numerical Rating Scale for sleep interference due to itch, were considered meaningful improvements by patients. These results were confirmed by several analyses and patient feedback. In summary, this research shows that, when people with PBC report their worst itch or sleep problems improving by these amounts on the Worst Itch Numerical Rating Scale and the Sleep Interference Numerical Rating Scale, it likely represents a real and important benefit in their daily lives. These findings will help researchers design better clinical trials and ensure that new treatments make a meaningful difference for patients.
SMART technological advancements help diagnose, treat, and monitor various diseases at the earliest stages. It presents an opportunity to maintain the key components of conventional obesity management programming while reducing costs and provider time inputs. Various machine learning models have helped predict the risks of obesity and metabolic syndrome. Additionally trained convolutional neural networks can now automatically segment and quantify different adipose tissue compartments. Various multicenter series and randomized clinical trials showed clinically meaningful total and excess weight loss at 6–24 months after minimally invasive robotic bariatric surgical procedures with an acceptable safety profile and shorter recovery compared with surgical sleeve gastrectomy. Virtual reality cue exposure therapy (VR-CET), a Website interactive voice response system, and various mobile applications for tracking diet and activity are the most advanced technological uses in the field of obesity management. The technological advancements should be adaptable, affordable, accessible, accurate, intuitive, sustainable and scalable. The conventional face-to-face interactions in obesity management have yielded short-term dividends, whereas technology-driven methods have the potential to produce long-lasting effects. This narrative review presented the different types of technologies used for obesity management and their outcomes in recent times.
The Tracking Treatment Pathways in Adult Patients with Hyperkalemia (TRACK) study demonstrated that clinical practice guidelines for hyperkalemia are inconsistently implemented and a conservative approach to management is often preferred. We report additional analyses focusing on routine clinical practice in the USA. TRACK was an observational prospective cohort study (July 2022–December 2024) including adults in the USA with a recent index hyperkalemia episode (serum potassium [K+] > 5.0 mmol/L ≤ 21 days of enrollment). Primary and secondary data were obtained from participants, their respective healthcare professionals (HCPs), and medical records. Clinical characteristics, hyperkalemia management decisions and rationales, and treatment outcomes over 12 months were evaluated. The final cohort included 229 participants, of whom 66.4
We aimed to achieve consensus on optimal bridging therapy (BT) selection for patients with relapsed/refractory multiple myeloma (RRMM) undergoing chimeric antigen receptor T-cell (CAR-T) therapy. We conducted a double-blind, three-round modified Delphi panel among U.S.-based hematologist-oncologists/hematologists using CAR-T therapy for RRMM. Panelists (N = 15; all managing –60–100 patients with RRMM within the past year; > 5 years of practice: 86.7
Atypical antipsychotics (AAs) are first-line treatments for bipolar I disorder (BP-I). While cariprazine is approved in the US for the treatment of both BP-I manic/mixed and depressive episodes, most other AAs are only approved for one affective pole but are utilized across the disease spectrum. For example, aripiprazole is only approved for BP-I manic/mixed episodes. This real-world analysis compared the rates of healthcare resource utilization (HRU)-defined manic and depressive events among patients treated with cariprazine versus aripiprazole. A retrospective observational cohort study was conducted using IQVIA® PharMetrics® Plus Database (5/28/2018–6/30/2022). Adults diagnosed with BP-I with ≥ 2 dispensings for cariprazine or aripiprazole were included. Cohorts were balanced via inverse probability of treatment weighting. Manic and depressive events were identified using a claims-based algorithm and reported per person-year (PPY) by setting of care [inpatient, emergency department (ED), and outpatient] and overall (any setting). Manic and depressive events during the ≥ 3-month on-treatment period were compared between cohorts via rate ratios (RRs) with P values and 95
Whether preoperative advanced therapy increases postoperative complications in inflammatory bowel disease (IBD) is contested. We conducted a cross-class systematic review distinguishing crude from confounding-adjusted estimates. Five databases were searched to May 2026 for studies of patients with IBD undergoing abdominal surgery. Random-effects meta-analysis estimated comparative odds ratios (ORs) with 95
Type 2 diabetes (T2D) is often accompanied by complications and comorbidities. This retrospective study used a large medical claims database to examine the annualized, direct costs associated with T2D-related comorbidities and complications among US adults diagnosed with T2D. It also examined excess costs associated with substandard glycemic control (glycated hemoglobin [HbA1c] ≥ 7
DMB-3115 is a biosimilar to ustekinumab (Stelara®). Although a single-dose study conducted in Europe showed that the pharmacokinetics of DMB-3115 was equivalent to that of ustekinumab, another single-dose study in Japan suggested that the maximum serum concentration (Cmax) of DMB-3115 might be slightly lower than that of ustekinumab in the Japanese population. Thus, we conducted a post hoc analysis of randomized controlled studies. The single-dose DMB-3115-1 and DMB-3115-3 studies enrolled healthy participants. The DMB-3115-2 study enrolled patients with moderate-to-severe chronic plaque psoriasis. It comprised 2 periods (Weeks 0–28 and 28–52), and we used the data through Week 12, during which patients received the assigned treatments at Weeks 0 and 4. Across all studies, participants were randomized to receive subcutaneous DMB-3115 or Stelara. We conducted regression analysis to investigate the relationships between the Cmax and efficacy outcomes using the data of participants receiving DMB-3115 or Stelara in the DMB-3115-2 study. We also conducted population pharmacokinetic (PK) analysis using the data of participants receiving DMB-3115 in the DMB-3115-1, -2, and -3 studies. Based on its results, we simulated the Cmax in the Japanese population. In the regression analysis, the combined Cmax of DMB-3115 and ustekinumab was not statistically associated with the percent change in Psoriasis Area and Severity Index (PASI) score from baseline to Week 12 or PASI-75 achievement (at least 75
DMB-3115 is a biosimilar to ustekinumab (Stelara®). We conducted a randomized controlled study to determine the similarity of bioavailability between DMB-3115 and Stelara (distributed in Japan) in healthy Japanese participants. Healthy Japanese male participants aged 18 to 55 years were randomly assigned in a 1:1 ratio to receive a single subcutaneous injection (45 mg) of DMB-3115 or Stelara. Participants were followed for 112 days. The primary pharmacokinetic (PK) endpoints were area under the concentration–time curve from time zero to the final sampling time point t (AUCt) and maximum serum concentration (Cmax). This study was conducted between April 11, 2022 and November 22, 2022. A total of 150 participants (75 in each group) received the study drug and were included in the safety analysis, whereas 147 participants (74 in the DMB-3115 group and 73 in the Stelara group) with properly measured PK data were included in the PK analysis. The 90
Ashwagandha supplementation has been associated with reduced self-reported stress and anxiety, although most trials have used doses of 250–600 mg daily for 6–8 weeks. This study examined the effects of a low-dose ashwagandha extract (Ashwa.30™) on perceived stress, mood, fatigue, and stress reactivity in stressed adults. In this 28-day, randomised, double-blind, placebo-controlled trial, 60 adults aged 18–65 years with self-reported stress were randomly allocated to receive ashwagandha 30 mg/day or placebo. Outcomes included the Depression, Anxiety, and Stress Scale, daily mood ratings, and the Chalder Fatigue Scale. The Socially Evaluated Cold-Pressor Test (SECPT) was conducted on Days 7 and 28, with subjective stress and fatigue, cardiovascular and electrodermal measures, skin temperature, and salivary cortisol and chromogranin A assessed. Compared to the placebo, ashwagandha intake was associated with greater reductions in self-reported stress in the full analysis set (primary analysis; p = 0.046). The per protocol set analysis was directionally consistent but not statistically significant. Mean daily stress ratings were also lower in the ashwagandha group (p = 0.043). During the Day 28 SECPT, the ashwagandha group reported lower stress ratings (p = 0.044), and salivary cortisol concentrations remained relatively stable in the ashwagandha group but increased in the placebo group, suggesting attenuation of cortisol reactivity (p = 0.047). However, no statistically significant between-group differences were detected for other secondary and physiological outcomes. An ashwagandha extract (Ashwa.30™) administered at 30 mg daily was associated with reduced self-reported stress in the Full Analysis Set and attenuation of SECPT-related salivary cortisol reactivity. These preliminary findings support further investigation in larger, adequately powered trials. Australian and New Zealand Clinical Trials Registry: (ACTRN12625000129482) https://www.anzctr.org.au/ACTRN12625000129482p.aspx .
