A5039428639- Muscle Physiology and Disorders
- Health Systems, Economic Evaluations, Quality of Life
- Renal cell carcinoma treatment
- Cancer Genomics and Diagnostics
- Economic and Financial Impacts of Cancer
- Chronic Myeloid Leukemia Treatments
- Lung Cancer Treatments and Mutations
- Advanced Breast Cancer Therapies
- Cancer Treatment and Pharmacology
- Chronic Lymphocytic Leukemia Research
- Myeloproliferative Neoplasms: Diagnosis and Treatment
- Psoriasis: Treatment and Pathogenesis
- Amyloidosis: Diagnosis, Treatment, Outcomes
- Renal and related cancers
- Dermatology and Skin Diseases
- Children's Physical and Motor Development
- Hematopoietic Stem Cell Transplantation
- Cardiac pacing and defibrillation studies
- Parathyroid Disorders and Treatments
- CAR-T cell therapy research
- Muscle activation and electromyography studies
- Pneumonia and Respiratory Infections
- Pharmaceutical Economics and Policy
- Rheumatoid Arthritis Research and Therapies
- Statistical Methods in Clinical Trials
Analysis Group (United States)
2016-2025
Cambridge Collaborative (United States)
2016-2025
Boston University
2015-2023
Great Ormond Street Hospital
2023
University College London
2023
Leiden University Medical Center
2022
Children's National
2020
Agostino Gemelli University Polyclinic
2020
Istituti di Ricovero e Cura a Carattere Scientifico
2020
KU Leuven
2020
A better understanding of the factors that contribute to heterogeneous outcomes and lifetime disease burden in hypertrophic cardiomyopathy (HCM) is critically needed improve patient management outcomes. The Sarcomeric Human Cardiomyopathy Registry (SHaRe) was established provide scale data required address these issues, aggregating longitudinal datasets curated by eight international HCM specialty centers.
Quantification of disease severity supports the development evidence-based treatments. Assessments to capture clinical improvement in hidradenitis suppurativa (HS) can be improved.This study aimed validate Hidradenitis Suppurativa Clinical Response (HiSCR), which is defined as a ≥ 50% reduction inflammatory lesion count (sum abscesses and nodules, AN), no increase or draining fistulas HS when compared with baseline meaningful endpoint for treatment.Patients 3 ANs at Phase II adalimumab trial...
Background: The DANISH study (Danish Study to Assess the Efficacy of ICDs [Implantable Cardioverter Defibrillators] in Patients With Non-Ischemic Systolic Heart Failure on Mortality) did not demonstrate an overall effect all-cause mortality with ICD implantation. However, prespecified subgroup analysis suggested a possible age-dependent association between implantation and survival benefit seen only youngest patients. nature this relationship age outcome primary prevention patients...
Purpose: To evaluate the performance characteristics of computed tomographic (CT) urography for detection bladder cancer in patients at risk disease. Materials and Methods: Institutional review board approval was obtained this retrospective HIPAA-compliant medical records 2600 consecutive undergoing CT urography. Of these, 838 urograms 779 (449 men, mean age 62 years, range 27–92 years; 330 women, 56 18–86 years) evaluated hematuria or a history urothelial cancer, who had undergone...
Background The randomized phase 3 ELOQUENT‐2 study (NCT01239797) evaluated the efficacy and safety of elotuzumab plus lenalidomide dexamethasone (ELd) versus (Ld) in relapsed/refractory multiple myeloma (RRMM), to date, has longest follow‐up any monoclonal antibody patients with RRMM. Methods In this extended 4‐year trial, coprimary endpoints progression‐free survival (PFS) overall response rate as well secondary endpoint were assessed. absence head‐to‐head trials comparing Ld‐based triplet...
The purpose of this study was to evaluate in-hospital outcomes among patients with a history heart failure (HF) hospitalized coronavirus disease-2019 (COVID-19).
To assess associations between hypoglycaemia and risk of accidents resulting in hospital visits among people with type 2 diabetes receiving antidiabetes drugs without insulin.People who were not treated insulin identified from a US-based employer claims database (1998-2010). Following initiation an drug, the occurrence was compared with, without, for using multivariable Cox proportional hazard models adjusted demographics, comorbidities, prior treatments medical service use. Additional...
Functional variability among boys with Duchenne muscular dystrophy (DMD) is well recognised and complicates interpretation of clinical studies. We hypothesised that DMD could be clustered into groups sharing similar trajectories ambulatory function over time, as measured by the North Star Ambulatory Assessment (NSAA) total score. also explored associations other variables such age, functional abilities, genotype. Using NorthStar Clinical Network database, 395 patients >1 NSAA assessment were...
To estimate projected US-based cost and time burden for patients with myelofibrosis anemia treated momelotinib compared danazol.
The risk of gastrointestinal (GI) bleeding limits the use antiplatelet and anticoagulant drugs. Risk factors for GI in post- myocardial infarction (MI) patients have not been well defined. We sought to identify following MI. VALsartan In Acute iNfarcTion trial (VALIANT) enrolled 14 703 post-MI with left ventricular dysfunction and/or heart failure followed them a median 24.7 months. present secondary analysis, times from baseline first were identified VALIANT serious adverse event database....
Abstract Background Though tobramycin inhalation solution has been used for over a decade to improve lung function and reduce exacerbations in patients with cystic fibrosis (CF), its effects on mortality have not well‐described. This study aimed assess the association between use of inhaled CF chronic Pseudomonas aeruginosa (PA) infection. Methods Longitudinal logistic regression was current‐year reported subsequent‐year meeting recommended criteria United States Cystic Fibrosis Foundation's...
To evaluate the World Health Organization's (WHO) Bleeding Scale in two studies of eltrombopag adults with chronic immune thrombocytopenia (ITP).Validated scales assessing bleeding ITP are lacking. Data from long-term, phase 3 clinical trials (RAISE: NCT00370331; EXTEND: NCT00351468) that assessed were analyzed to performance WHO Scale.In RAISE, effect size (0.71), standardized response (0.75), and responsiveness statistics (0.57) moderate for bruising assessments. In EXTEND, (0.62) (0.59)...
nab-Paclitaxel plus gemcitabine (nab-P + G) and FOLFIRINOX (FFX) are among the most common first-line (1L) therapies for metastatic adenocarcinoma of pancreas (MPAC), but real-world data on their comparative effectiveness limited.
Abstract Chimeric antigen receptor T-cell (CAR-T) therapy yields durable responses in patients with relapsed/refractory diffuse large B-cell lymphoma (r/r DLBCL). Cytokine release syndrome (CRS) is a CAR-T therapy–related adverse event. To date, clinical trials of different products have not been aligned on CRS grading scales and management algorithms. We assessed concordance between the Penn, Lee, American Society for Transplantation Cellular Therapy (ASTCT) systems by retrospectively...
High variability in patients' changes 6 minute walk distance (6MWD) over time has complicated clinical trials of treatment efficacy Duchenne muscular dystrophy (DMD). We assessed whether boys with DMD could be grouped into classes that shared similar ambulatory function trajectories as measured by 6MWD. Ambulatory aged 5 years or older genetically confirmed who were enrolled a natural history study at 11 care centers throughout Italy included. For each boy, standardized assessments 6MWD...