Advances in Therapy

Cardiac arrest (CA) is a critical public health issue affecting more than half a million Americans annually. The main determinant of outcome post-CA is hypoxic-ischemic brain injury (HIBI), and temperature control is currently the only evidence-based, guideline-recommended intervention targeting secondary brain injury. Temperature control is a key component of a post-CA care bundle; however, conflicting evidence challenges its wide implementation across the vastly heterogeneous population of CA survivors. Here, we critically appraise the available literature on temperature control in HIBI, detail how the evidence has been integrated into clinical practice, and highlight the complications associated with its use and the timing of neuroprognostication after CA...

INTRODUCTION: Nasopharyngeal carcinoma (NPC) responds well to radiotherapy but recurrence and metastasis are common. Currently, there is no widely used biomarker for accurately predicting the recurrence and metastasis of NPC. In this study, we aimed to evaluate the prognostic ability of Epstein-Barr virus (EBV) capsid antigen (VCA-IgA) kinetics by assessing the dynamic changes of VCA-IgA levels in the pre- and post-treatment plasma of patients with NPC and have proposed a prognostic model for clinical use...

INTRODUCTION: Patients with triple-class-exposed relapsed/refractory multiple myeloma (TCE-RRMM) have a poor prognosis and limited treatment options. Teclistamab, a B-cell maturation antigen × CD3 bispecific antibody, was studied in patients with TCE-RRMM in the single-arm MajesTEC-1 study. To assess the relative effectiveness of teclistamab versus real-world physician's choice of therapy (RWPC), adjusted comparisons were performed using individual patient data from MajesTEC-1 and LocoMMotion, a prospective study of patients with TCE-RRMM...

INTRODUCTION: Bardet-Biedl syndrome (BBS) is a rare genetic disease associated with hyperphagia, a pathologic insatiable hunger, due to impaired signaling in the melanocortin-4 receptor (MC4R) pathway. The impact of hyperphagia on the lives of patients with BBS and their families has not been fully characterized. METHODS: Patients with BBS or their caregivers who participated in clinical trials of the MC4R agonist setmelanotide (NCT03013543 and NCT03746522) were included in this qualitative study...

INTRODUCTION: Direct oral anticoagulants (DOACs) are essential in ischemic stroke/systemic embolism (SE) prevention among patients with nonvalvular atrial fibrillation (NVAF). This study compared the risk of ischemic stroke/SE among patients with NVAF who discontinued DOACs following the first fill ("one-and-done") relative to patients who continued DOACs beyond the first fill ("continuers"). METHODS: De-identified data from Symphony Health, an ICON plc Company, PatientSource® , April 1, 2017 to October 31, 2020, were used to identify adults with NVAF initiated on DOACs (index date)...

Non-steroidal anti-inflammatory drugs (NSAIDs) are among the most common class of drugs utilized for a variety of disorders, including headaches, pain states, fever, and other common conditions. In recent years, a link between NSAIDs and adverse effects has been identified, including renal, heart, and liver disease, bleeding, and increased mortality. NSAID-mediated renal disease is associated with interference with the cyclooxygenase enzyme. Literature evaluating NSAID renal effects has indicated that a number of factors are associated with acute and chronic kidney injury (AKI)...

INTRODUCTION: Retinitis pigmentosa (RP) is an inherited progressive disease, characterized by a loss of photoreceptors, and is the second leading cause of visual impairment in Japan. RP is currently incurable and can result in complete blindness, with affected patients typically experiencing a gradual loss of light sensitivity, visual field, and visual acuity. Identification of any unmet medical needs of patients with this condition requires an understanding of the impacts of RP; in this study, we surveyed Japanese patients with RP to investigate the quality of life and economic impacts of visual impairment...

INTRODUCTION: The objective of this study was to evaluate the cost-effectiveness of lisocabtagene maraleucel (liso-cel) versus other available chimeric antigen receptor T-cell therapies, including axicabtagene ciloleucel (axi-cel) and tisagenlecleucel (tisa-cel), in patients who had received at least two prior therapies from a United States (US) commercial third-party payer perspective. METHODS: To capture this heterogeneity in survival outcomes, we used mixture cure models to extrapolate progression-free survival (PFS) and overall survival (OS)...

INTRODUCTION: The efficacy and safety of upadacitinib in atopic dermatitis have been defined in clinical trials, but long-term real-life experience, essential for clinical decision-making, is still limited. We aimed to assess the effectiveness and tolerance of upadacitinib in a real-life cohort of adults and adolescents with severe atopic dermatitis in whom previous systemic therapies largely failed. METHODS: Retrospective cohort study collecting data from adults and adolescents treated with upadacitinib 15 or 30 mg per day between July 2021 to August 2022...

INTRODUCTION: Dose escalation is one of the treatment approaches studied and suggested in advanced therapies for Crohn's disease (CD) and ulcerative colitis (UC). This study aimed to identify and characterize the dosing escalation patterns of advanced therapies in CD and UC. METHODS: Two systematic literature reviews (SLRs) were conducted in accordance with Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines. MEDLINE® , Embase® , and Cochrane Library were searched for articles published between January 2011 and October 2021 and limited to non-interventional studies in English language...

INTRODUCTION: Prior studies have demonstrated guselkumab improves disease activity and patient-reported outcomes (PROs) among patients with moderate-to-severe plaque psoriasis. However, the real-world effectiveness of guselkumab across different subgroups [e.g., body mass index (BMI) categories] remains an area of active research. METHODS: This study included patients enrolled in the CorEvitas Psoriasis Registry between July 18, 2017 and March 10, 2020 who had moderate-to-severe psoriasis [Investigator's Global Assessment (IGA) score ≥ 3], initiated guselkumab at a registry visit (index date), and had a follow-up registry visit after persistent guselkumab therapy for 9-12 months...

INTRODUCTION: This study describes the epidemiological, clinical, patient-reported and economic burden of inflammatory bowel disease (IBD), including Crohn's disease (CD) and ulcerative colitis (UC), in Spain. METHODS: A systematic review was performed of observational studies reporting the epidemiological, clinical, patient-reported and economic burden of IBD in the Spanish population, from 2011 to 2021. Original articles and conference abstracts published in English or Spanish were eligible...

INTRODUCTION: The objective of this systematic literature review was to evaluate the available literature concerning the clinical, economic, and patient-reported benefits of insulin pen platforms, including connected insulin pens/caps/sleeves and insulin platforms, as well as mobile apps capable of receiving near real-time insulin dosing information. METHODS: Medline and Embase databases and the Cochrane Library were searched for published literature between January 2015 and May 2021, and manual searches for conference abstracts from 2018 to May 2021 were performed...

Nintedanib is a tyrosine kinase inhibitor approved for the treatment of idiopathic pulmonary fibrosis (IPF) and other progressive fibrosing interstitial lung diseases. Placebo-controlled trials showed that the adverse event profile of nintedanib was characterised mainly by gastrointestinal events, particularly diarrhoea. We review the data from all published real-world studies of the safety of nintedanib in patients with IPF. These real-world data were consistent with the safety profile observed in clinical trials and described in the product label...

The projected increase in life expectancy over the next few decades is expected to result in a rise in age-related diseases, including cancer. Head and neck cancer (HNC) is a worldwide health problem with high rates of morbidity and mortality. In this report, we have critically reviewed the literature reporting the management of older patients with HNC. Older adults are more prone to complications and toxicities secondary to HNC treatment, especially those patients who are frail or have comorbidities. Thus, this population should be screened prior to treatment for such predispositions to maximize medical management of comorbidities...

INTRODUCTION: Pharmacodynamic and pharmacokinetic studies in animal experiments and a phase 1 study suggested remimazolam tosylate as an effective and safe sedation/anesthetic agent. However, the effects and safety dose of remimazolam for light sedation in intensive care unit (ICU) patients are not clear and should be confirmed in a phase 2 study. METHODS: Sixty ICU patients requiring sedation treatment and undergoing mechanical ventilation will be enrolled and randomly assigned to a high dose group (HD group, 30 cases) and a low dose group (LD group, 30 cases) in a 1:1 ratio...

INTRODUCTION: Brentuximab vedotin versus physician's choice of methotrexate (MTX) or bexarotene (BEX) significantly improved progression-free survival (PFS) (median PFS, 16.7 vs. 3.5 months) and delayed time to subsequent treatment (8.4 vs. 3.7 months), with similar overall survival in patients with CD30-expressing mycosis fungoides (MF) or primary cutaneous anaplastic large cell lymphoma (pcALCL), two types of cutaneous T-cell lymphomas. We assessed the cost-effectiveness of brentuximab vedotin versus MTX or BEX from a Canadian healthcare payer perspective in the indicated population...

INTRODUCTION: Hypertension is the leading cause of morbidity and mortality in sub-Saharan Africa (SSA). Current guidelines recommend using two or more antihypertensive agents in single pill combinations (SPCs) to treat hypertension, but data from African patients that support these recommendations are lacking. We assessed the effectiveness and tolerance of three SPCs in lowering blood pressure (BP) amongst hypertensive patients in Douala. METHOD: All patients included in the hypertension registry of the Douala General Hospital and the Douala Cardiovascular Center between January 2010 and May 2020, and receiving a two-drug SPCs (renin-angiotensin system inhibitors (RAASi) + diuretics (DIU), calcium channel blockers (CCB) + RAASi, or DIU + CCB) were tracked from baseline through 16 weeks...

INTRODUCTION: In patients with moderate to severe Crohn's disease (CD), intravenous induction and subcutaneous maintenance dosing with risankizumab was efficacious and well tolerated. Long-term management of CD via self-administration of risankizumab using an on-body injector (OBI) may improve treatment adherence through convenience and ease of use. METHODS: Within the FORTIFY maintenance study, 46 patients from the United States (US) sites participated in an open-label extension Substudy and received 180 mg or 360 mg risankizumab delivered subcutaneously via OBI [360 mg (2...

INTRODUCTION: Limb girdle muscular dystrophies (LGMDs) are a group of rare and heterogeneous disorders involving progressive wasting of shoulder and pelvic girdle musculature. This study aimed to generate qualitative evidence on patient and caregiver experiences with symptoms and impacts of LGMD on overall function and daily life for sarcoglycanopathy subtypes 2C/R5, 2D/R3, and 2E/R4. METHODS: Twenty-three individuals with LGMD with (n = 5) or without (n = 18) a caregiver participated in 60-minute semi-structured video interviews...