Atypical Hemolytic Uremic Syndrome Treated With Ravulizumab or Eculizumab: A Claims-Based Evaluation of Health Care Resource Utilization and Clinical Outcomes in the United States.

Rationale And Objective: Ravulizumab and eculizumab have shown efficacy for the treatment of atypical hemolytic uremic syndrome (aHUS), but real-world evidence for ravulizumab is limited owing to its more recent approval. This real-world database study examined outcomes for adult patients switching to ravulizumab from eculizumab and patients treated with individual treatments.

Study Design: A retrospective, observational study using the Clarivate Real World Database.

Clinical characteristics and outcomes of a patient population with atypical hemolytic uremic syndrome and malignant hypertension: analysis from the Global aHUS registry.

Introduction: Atypical hemolytic uremic syndrome (aHUS) is a rare form of thrombotic microangiopathy (TMA) often caused by alternative complement dysregulation. Patients with aHUS can present with malignant hypertension (MHT), which may also cause TMA.

Methods: This analysis of the Global aHUS Registry (NCT01522183) assessed demographics and clinical characteristics in eculizumab-treated and not-treated patients with aHUS, with (n = 71) and without (n = 1026) malignant hypertension, to further elucidate the potential relationship between aHUS and malignant hypertension.

Pregnancy in Women with Atypical Hemolytic Uremic Syndrome.

Background: Pregnancy outcomes in patients with atypical hemolytic uremic syndrome (aHUS) are not well-documented. Here, we present characteristics of and outcomes for patients with aHUS who became pregnant while enrolled in the Global aHUS Registry.

Methods: The observational Global aHUS Registry (NCT01522183), initiated in April 2012, collects demographics, disease history, treatment, and outcomes data for patients with aHUS, regardless of treatment approach.

Pregnancy-triggered atypical hemolytic uremic syndrome (aHUS): a Global aHUS Registry analysis.

Background: Atypical hemolytic uremic syndrome (aHUS) is a rare disease in which uncontrolled terminal complement activation leads to systemic thrombotic microangiopathy (TMA). Pregnancy can trigger aHUS and, without complement inhibition, many women with pregnancy-triggered aHUS (p-aHUS) progress to end-stage renal disease (ESRD) with a high risk of morbidity. Owing to relatively small patient numbers, published characterizations of p-aHUS have been limited, thus the Global aHUS Registry (NCT01522183, April 2012) provides a unique opportunity to analyze data from a large single cohort of women with p-aHUS.

Evaluating the Effectiveness of Apixaban Additional Risk Minimisation Measures Using Surveys in Europe.

Background: Apixaban (ELIQUIS) is a direct oral anticoagulant authorised for multiple indications in the European Economic Area (EEA). Additional risk minimisation measures (aRMMs) to address the risk of bleeding include educational materials comprising a Prescriber Guide and Patient Alert Card.

Objectives: This study evaluated effectiveness of the apixaban Prescriber Guide and Patient Alert Card in terms of healthcare professional (HCP) and patient knowledge of associated bleeding risk, as well as material distribution, utilisation and behaviour.

Functional Assessment of Fatigue and Other Patient-Reported Outcomes in Patients Enrolled in the Global aHUS Registry.

Introduction: Atypical hemolytic uremic syndrome (aHUS) is a progressive and potentially life-threatening disease characterized by complement-mediated thrombotic microangiopathy. Patients with aHUS may experience fatigue, which can negatively impact their lives, but there is a knowledge gap regarding disease burden in these patients.

Methods: In this longitudinal study, patients with aHUS from the Global aHUS Registry who completed patient-reported outcome assessments (Functional Assessment of Chronic Illness Therapy-Fatigue scale [FACIT-Fatigue], general health status, and work status) at ≥2 time points were assessed relative to treatment status: (i) never treated with eculizumab; (ii) on eculizumab at registry enrollment and continued therapy; and (iii) started eculizumab after registry enrollment.

Clinical Characteristics and Outcome of Canadian Patients Diagnosed With Atypical Hemolytic Uremic Syndrome.

Background: Atypical hemolytic uremic syndrome (aHUS) is an extremely rare, heterogeneous disease of uncontrolled activation of the alternative complement pathway that is difficult to diagnose. We have evaluated the Canadian patients enrolled in the Global aHUS Registry to provide a Canadian perspective regarding the diagnosis and management of aHUS and the specific challenges faced.

Objective: To evaluate Canadian patients enrolled in the Global aHUS Registry to provide a Canadian perspective regarding the diagnosis and management of aHUS and the specific challenges faced.

Eculizumab Safety: Five-Year Experience From the Global Atypical Hemolytic Uremic Syndrome Registry.

Introduction: Eculizumab has transformed outcomes for patients with atypical hemolytic uremic syndrome (aHUS). Its efficacy and safety profile was well characterized in the clinical trial program. The long-term safety profile was not previously assessed or compared against nontreated patients in an observational registry setting.

Effectiveness Evaluation of Additional Risk Minimization Measures for Adolescent Use of Aripiprazole in the European Union: Results from a Post-Authorization Safety Study.

Introduction: Two risk minimization (RM) tools-a healthcare professional frequently asked questions (HCP-FAQs) brochure and a patient/caregiver information brochure (PCIB)-were developed for HCPs and for adolescents (aged ≥ 13 years) receiving aripiprazole for bipolar I mania and their caregivers.

Objectives: This study evaluated the effectiveness of these RM tools in improving the awareness and education of HCPs and patients/caregivers.

Method: The RM tools were distributed to HCPs (identified in agreement with the marketing authorization holder [MAH] and local regulatory authorities), who in turn distributed the PCIBs to patients/caregivers.

Which Metrics Are Appropriate to Describe the Value of New Cancer Therapies?

Patients with certain cancers are treated with curative intent, but for others the results are less favorable and different therapeutic approaches are needed. Early data suggest that new therapies, which modulate immune responses to cancers, may have potential for long-term survival in a proportion of cases. Therefore, it is timely to consider whether metrics generally used to describe the medical value of therapies for patients with common solid tumors remain appropriate for therapies with curative potential.

The impact of specific HIV treatment-related adverse events on adherence to antiretroviral therapy: a systematic review and meta-analysis.

Poor adherence to antiretroviral therapies (ARTs) in human immunodeficiency virus (HIV)-infected patients increases the risk of incomplete viral suppression, development of viral resistance, progression to acquired immune deficiency syndrome and death. This study assesses the impact of specific treatment-related adverse events (AEs) on adherence to ART in the adult HIV patient population. A systematic review of studies involving adult HIV-infected patients aged ≥ 16 years that reported an odds ratio (OR) for factors affecting adherence to ART was conducted through a search of the EMBASE(®) and Medline(®) databases.

Socio-economic status and upper aerodigestive tract cancer.

Design: A multicentre case-control study. CASE/CONTROL SELECTION: Cases were defined as those diagnosed with primary squamous cell tumours of the UADT between 2002 and 2005. Diagnoses included malignant cancers of the oral cavity, oropharynx, hypo-pharynx, larynx or oesophagus.

Tea, coffee and oral cancer risk.

Data Sources: Pooled individual-level data from nine case-control studies of head and neck cancers, including 5,139 cases and 9,028 controls.

Study Selection: Nine case-control studies were selected from the International Head and Neck Cancer Epidemiology (INHANCE) consortium pool of 33 studies, which included information on coffee (caffeinated and decaffeinated) and tea drinking and cancer of the oral cavity and pharynx. Seven studies also included information on laryngeal cancer.

The association between cancer treatments and oral diseases.

Data Sources: MEDLINE/PubMed and EMBASE.

Study Selection: The review included papers published between January 1, 1990 and December 31, 2008. The primary outcome was to retrieve all literature containing original data on dental caries, periodontal disease and pre-cancer dental clearance protocols in cancer patients undergoing head and neck radiotherapy, chemotherapy or combined treatment modalities.

Public awareness of oral cancer and associated risk factors is low.

Study Design: A survey was carried out over a three-year period (2004-2007) in Maggie's Cancer Caring Centres or in patients' homes in Glasgow and Edinburgh, Scotland. Participants included young patients diagnosed with oral cancer.

Data Collection And Analysis: Data were collected by interview using a semi-structured interview schedule.

Oral dysplasia and risk of progression to cancer.

Data Sources: Medline, EMbase and Cochrane databases.

Study Selection: Studies were included if they reported data on patients with a histologically-confirmed diagnosis of oral dysplasia. They also had to study at least one outcome measure and one intervention method or clinical risk factor.

Socioeconomic status and head and neck cancer.

Design: This was a population-based case-control study. CASE-CONTROL SELECTION: Eligible patients were aged between 18 and 80 years and had a primary histopathological diagnosis made between April 2002 and December 2004. Diagnosis included malignant cancers of the oral cavity, oropharynx, hypopharynx or larynx.

Diagnostic delay broadly associated with more advanced stage oral cancer.

Data Sources: Medline, Embase, ISI Proceedings and the reference lists of relevant articles were used to find relevant studies.

Study Selection: Studies were included if: they presented original data from observational studies; included patients with a confirmed pathological diagnosis of oral or oropharyngeal squamous cell carcinoma; the outcome of interest was clearly defined as disease stage (TNM classification); the exposure of interest was total diagnostic delay, defined as the period between the patient noticing either the first sign or symptom and definitive diagnosis (data were collected from interviews using a standardised questionnaire and medical records); provided relative risks (RR) and 95% confidence intervals (CI) or provided enough data to allow calculation of these figures.

Data Extraction And Synthesis: Quality assessment was undertaken independently by two reviewers and followed the recommendations of the Meta-analysis of Observational Studies in Epidemiology (MOOSE).