Cost-effectiveness analyses of new cancer treatments in real-world settings (e.g., post-clinical trials) inform healthcare decision makers about their healthcare investments for patient populations. The results of these analyses are often, though not...
Background/Objectives: Brolucizumab is a humanized single-chain antibody fragment with a molecular weight of approximately 26 kilodaltons (scFv, ~26 kDa) targeting all VEGF-A isoforms. Intravitreal brolucizumab (6 mg) is FDA-approved for neovascular...
Cost-effectiveness analysis (CE Analysis) provides evidence about the incremental gains in patient outcomes costs from new treatments and interventions in cancer care. The utilization of “real-world” data allows these analyses to better r...
Despite methodological limitations, real-world studies might support clinicians by broadening the knowledge of antipsychotics’ (APs) effectiveness and tolerability in different clinical scenarios and complement clinical trials. We conducted an...
(1) Background: Data on combination or sequential treatment of spinal muscular atrophy (SMA) with disease-modifying drugs (DMDs) are missing and the latter field is poorly understood. The currently available data of patients on risdiplam previously t...
Background: The long-term follow-up of clinical trials of novel first-line (1L) therapies for chronic lymphocytic leukemia (CLL) demonstrates 6–10-year progression-free survival. We describe the effectiveness of 1L CLL treatments in real-world...
Background/Objectives: Evidence is needed on the real-world outcomes of upadacitinib in patients with ulcerative colitis. This systematic review and meta-analysis evaluated the real-world effectiveness of upadacitinib for active UC. Methods: The prim...
1 August 2023
There is evidence of the efficacy of collaborative health interventions with pharmacies and primary care providers but little of its real-world effectiveness. We aimed to assess the effectiveness and discuss the design and challenges of hypertension...
Background: Hepatocellular carcinoma (HCC) accounts for approximately 90% of liver cancer cases. Sorafenib, the first drug to demonstrate survival benefits for advanced HCC, was validated through the SHARP randomized clinical trial (RCT). While RCTs...
Background: Invasive mucormycosis (IM) poses a substantial morbidity and mortality burden among immunocompromised patients. Objectives: We aim to compare the real-world effectiveness and safety of isavuconazole with those of amphotericin B in patient...
25 June 2020
Background: Morbidity and mortality associated with non-valvular atrial fibrillation (NVAF) imposes a substantial economic burden on the UK healthcare system. Objectives: An existing Markov model was adapted to assess the real-world cost-effectivenes...
(1) Background: Evidence on the outcomes of ustekinumab treatment in real-world Crohn’s disease (CD) patients is needed. Our aim was to evaluate the effectiveness and safety of ustekinumab in CD, reported by observational studies. (2) Methods:...
17 June 2020
Introduction: This study is one of the first real-world cost-effectiveness analyses of one-year adjuvant trastuzumab used in HER2-positive early female breast cancer in comparison to chemotherapy alone. It is just the second one in Europe, the first...
CT-P13 is an infliximab biosimilar approved for indications including ankylosing spondylitis (AS); the approved maintenance regimen is 5 mg/kg infused every 6–8 weeks. In clinical practice, modifications to infliximab dose and/or infusion interval ca...
Overall, it is estimated that more than 3,500,000 patients have received Bevacizumab as part of systemic oncologic treatment. Bevacizumab and its biosimilars are currently marketed in over 130 countries. Given the wide usage of Bevacizumab in current...
(1) Background: To investigate the real-world effectiveness and safety profile of nusinersen in Croatian paediatric and adult spinal muscular atrophy (SMA) patients. (2) Methods: A retrospective and anonymous collection of relevant demographic and cl...
Lorlatinib is the only targeted therapy approved in Canada to treat patients with anaplastic lymphoma kinase (ALK)-positive non-small cell lung cancer (NSCLC) whose tumor has progressed despite treatment with second-generation ALK tyrosine kinase inh...
While clinical trials have shown the efficacy of using dupilumab and upadacitinib to treat atopic dermatitis (AD), long-term real-world data remain limited. This retrospective cohort study, utilizing data from the Polish National Health Fund Registry...
Background: Nivolumab, an anti–programmed cell death 1 immuno-oncology therapy, is approved as an adjuvant treatment for patients with completely resected stage III or stage IV melanoma. PRESERV MEL (Prospective and REtrospective Study of nivol...
Elexacaftor/tezacaftor/ivacaftor (ETI) is a cystic fibrosis (CF) transmembrane conductance regulator modulator, which has shown efficacy in people with CF (pwCF) carrying the F508del (F) variant, both in homozygosity and heterozygosity with a minimal...
Background: New oral anticoagulant agents (NOACs) are valid alternatives for vitamin K antagonists (VKA) in patients with non-valvular atrial fibrillation (NVAF) for stroke prevention. In clinical practice, NOACs users may differ from patients enroll...
The preventive effect of HPV vaccines against anogenital and oropharyngeal cancers has been proven in both clinical trials and real-world data. We reviewed the published evidence about the long-term efficacy and effectiveness of the HPV vaccine in av...
Voretigene neparvovec (VN) is the first available gene therapy for patients with biallelic RPE65-mediated inherited retinal dystrophy who have sufficient viable retinal cells. PERCEIVE is an ongoing, post-authorization, prospective, multicenter, regi...
Background: Nintedanib is an oral multitarget tyrosine kinase inhibitor approved for the treatment of patients with idiopathic pulmonary fibrosis (IPF). Recent evidence demonstrated that nintedanib reduced functional disease progression also in subje...
The perioperative and short-term oncological outcomes of robotic-assisted rectal surgery (RRS) are unclear. This retrospective observational study enrolled patients with rectal adenocarcinoma undergoing RRS from three high-volume institutions in Taiw...
Randomized controlled trials (RCTs) have been regarded as the gold standard for evaluating the efficacy of treatments for chronic pain and are the foundation for regulatory approval and guideline development. However, their restrictive design and dep...
17 March 2020
The maintenance of patients with renal transplant typically involves two or more drugs to prevent rejection and prolong graft survival. The calcineurin inhibitors (CNI) are the most commonly recommended medicines in combinations with others. While im...
Objective: To perform a systematic review of real-world outcomes for anti-CGRP-mAbs. Methods: Following the PRISMA guidelines, we searched PubMed for real-world data of erenumab, galcanezumab, fremanezumab, or eptinezumab in patients with migraines....
Specific adult populations known to be at high risk for human papillomavirus (HPV)-related disease, such as men who have sex with men, are inconsistently included in national immunization programs. No compilation of the evidence on the real-world imp...
The efficacy of vaccines against coronavirus disease 2019 (COVID-19) has now been well established in phase III clinical trials. However, clinical studies based on real-world data remain critical to assess vaccines effectiveness (VE), especially in s...
Most studies on the real-world effectiveness and safety of dolutegravir/lamivudine (DTG/3TC) have been conducted in Western countries, and Asian reports are lacking. We evaluated the effectiveness and safety of DTG/3TC in Korean adult people living w...
Whilst there is considerable evidence to support the efficacy of physical activity and dietary interventions in disease and death prevention, translation of knowledge into practice remains inadequate. We aimed to examine the uptake, retention, accept...
12 May 2020
Impairment in real-world functioning remains one of the most problematic challenges that people with schizophrenia have to face. Various psychosocial interventions have proven to be effective in promoting recovery and improving functioning in schizop...
No evidence is available on the head-to-head comparison of clinical outcomes of patients treated with immune checkpoint inhibitors (ICIs) for advanced non-small cell lung cancer (NSCLC) in a real-world setting. We aimed to compare the effectiveness a...
This study evaluated the real-world effectiveness of belimumab (BLM) in the treatment of systemic lupus erythematosus (SLE) patients with moderate to high disease activity. This retrospective cohort study enrolled 129 Japanese patients with moderate...
Trastuzumab is a milestone in the treatment of human epidermal growth factor receptor 2 positive (HER2+) breast cancer (BC), in both the early and metastatic settings. Over the last two decades, clinical trials have established the good safety profil...
Background: Both novel hormonal therapies and docetaxel are approved for treatment of metastatic prostate cancer (mPC; in castration sensitive or refractory settings). Present knowledge gaps include lack of real-world data on treatment patterns in pa...
Since the authorization of the first COVID-19 vaccines in December 2020, multiple studies using real-world data (RWD) have been published to assess their effectiveness/safety profile. This systematic review aimed to characterize the methods and outco...
Chronic obstructive pulmonary disease (COPD) is a major health burden in India with limited real-world data on triple inhaler therapy. This prospective, open-label, multi-center, single-arm, phase 4 study (October 2023–August 2024) assessed the...
Background: Immune checkpoint inhibitors (ICIs) such as pembrolizumab (Pem) have demonstrated clinical benefits in esophageal cancer. Cisplatin, 5-fluorouracil, and Pem combination (CF plus Pem) has emerged as a promising first-line regimen. However,...
Background: data regarding the effectiveness and safety of sacubitril/valsartan in heart failure and reduced ejection fraction (HFrEF) patients with chronic kidney disease (CKD) are scarse. Objective: to evaluate the effectiveness and safety of sacub...
Background: There is a scarcity of evidence regarding the real-world effectiveness of coronavirus disease 2019 (COVID-19) vaccines. This was the first study to evaluate the effectiveness of four types of vaccines against asymptomatic and symptomatic...
Background/Objectives: Interstitial lung disease (ILD) is one of the most severe complications of rheumatoid arthritis (RA). Real-world data on antifibrotic treatment are needed. Our objective was to evaluate the real-world effectiveness and tolerabi...
Background and Objectives: Ofatumumab (OFA) is the first fully human anti-CD20 monoclonal antibody approved for the treatment of RRMS classified as a high-efficacy treatment agent. Real-world evidence is essential for evaluating the effectiveness and...
Objective: The aim was to characterize the real-world use of GLP-1 receptor agonists (GLP-1 RAs) and/or SGLT2 inhibitors in kidney transplant recipients (KTRs) with diabetes and to compare the clinical management, safety, and effectiveness between pa...
This prospective, observational study examined the real-world use of carfilzomib across 11 European countries in adults with relapsed/refractory multiple myeloma (RRMM) who received at least one prior line of therapy. Carfilzomib and dexamethasone (K...
Objective: The effectiveness and safety of belimumab in Chinese lupus patients with different disease activities were investigated in a real-world setting. Method: Patients who received 10 mg/kg belimumab intravenously on weeks 0, 2, and 4, and then...
Background: Small cell lung cancer (SCLC) is a malignant carcinoma characterized by high proliferative rate and early metastatization with limited treatment options and poor prognosis. The approval of ICIs has established a new standard of care for e...
Background and Aims: Inflammatory bowel disease (IBD) requires effective treatment options. Upadacitinib, a Janus kinase 1 (JAK1) inhibitor, has shown effectiveness in trials for Crohn’s disease (CD) and ulcerative colitis (UC). This study eval...
Background: Ixekizumab, an IL-17A inhibitor, is an effective treatment for moderate-to-severe plaque psoriasis. Although clinical trials support the use of an induction phase for optimal results, real-world evidence comparing induction versus mainten...
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