A5077739374- Complement system in diseases
- Renal Diseases and Glomerulopathies
- Iron Metabolism and Disorders
- Blood groups and transfusion
- Renal Transplantation Outcomes and Treatments
- Adenosine and Purinergic Signaling
- Hemoglobinopathies and Related Disorders
- Oral Health Pathology and Treatment
- Smoking Behavior and Cessation
- Health Systems, Economic Evaluations, Quality of Life
- Porphyrin Metabolism and Disorders
- HIV/AIDS Research and Interventions
- HIV/AIDS drug development and treatment
- Head and Neck Cancer Studies
- Adolescent and Pediatric Healthcare
- Acute Myeloid Leukemia Research
- Diagnosis and Treatment of Venous Diseases
- Coffee research and impacts
- Stuttering Research and Treatment
- Chemotherapy-induced organ toxicity mitigation
- HIV-related health complications and treatments
- Oral health in cancer treatment
- Pharmaceutical studies and practices
- Otitis Media and Relapsing Polychondritis
- Erythropoietin and Anemia Treatment
AstraZeneca (United States)
2021-2024
Alexion Pharmaceuticals (United States)
2018-2024
PAREXEL International (United Kingdom)
2014-2015
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 not previously assessed or compared against nontreated an observational registry setting.The Global aHUS Registry recruits diagnoses of aHUS. This analysis includes baseline characteristics targeted events from adult pediatric who were "ever treated" versus "never eculizumab first 5 years registry,...
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 inhibition, many women with pregnancy-triggered (p-aHUS) progress end-stage renal (ESRD) high risk of morbidity. Owing relatively small patient numbers, published characterizations p-aHUS have been limited, thus the Global Registry (NCT01522183, April 2012) provides unique opportunity...
Abstract Introduction Atypical haemolytic uremic syndrome (aHUS) is a rare form of thrombotic microangiopathy (TMA) associated with complement dysregulation; aHUS may be other ‘triggers’ or ‘clinical conditions’. This study aimed to characterize this patient population using data from the Global Registry, largest collection real‐world on patients aHUS. Methods Patients enrolled in Registry between April 2012 and June 2021 recorded aHUS‐associated triggers clinical conditions prior/up onset...
Abstract 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 malignant hypertension (MHT), which may also cause TMA. Methods This analysis the Global Registry (NCT01522183) assessed demographics and clinical characteristics in eculizumab-treated not-treated patients aHUS, ( n = 71) without 1026) hypertension, to further elucidate potential relationship...
Atypical hemolytic uremic syndrome (aHUS) is a progressive rare disease that, if untreated, can result in severe organ damage and death. Ravulizumab, next-generation terminal complement inhibitor, provides immediate, complete, sustained C5 inhibition. Real-world data patients with aHUS who switched to ravulizumab from eculizumab are lacking.
Two risk minimization (RM) tools—a healthcare professional frequently asked questions (HCP-FAQs) brochure and a patient/caregiver information (PCIB)—were developed for HCPs adolescents (aged ≥ 13 years) receiving aripiprazole bipolar I mania their caregivers. This study evaluated the effectiveness of these RM tools in improving awareness education patients/caregivers. The were distributed to (identified agreement with marketing authorization holder [MAH] local regulatory authorities), who...
IntroductionAtypical 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 knowledge gap regarding burden in these patients.MethodsIn this longitudinal study, patients from the Global Registry who completed patient-reported outcome assessments (Functional Assessment of Chronic Illness...
Pregnancy outcomes in patients with atypical hemolytic uremic syndrome (aHUS) are not well-documented. Here, we present characteristics of and for aHUS who became pregnant while enrolled the Global Registry.The observational Registry (NCT01522183), initiated April 2012, collects demographics, disease history, treatment, data aHUS, regardless treatment approach. This descriptive analysis includes from evaluable pregnancy supplemented pharmacovigilance information; number pregnancies,...
Ravulizumab and eculizumab have shown efficacy for the treatment of atypical hemolytic uremic syndrome (aHUS), but real-world evidence ravulizumab is limited owing to its more recent approval. This database study examined outcomes adult patients switching from treated with individual treatments.A retrospective, observational using Clarivate Real World Database.US health-insurance billing data (January 2012 March 2021) aged 18 years or older ≥1 diagnosis relevant aHUS, claim ravulizumab, no...
Atypical hemolytic uremic syndrome (aHUS) is an extremely rare, heterogeneous disease of uncontrolled activation the alternative complement pathway that difficult to diagnose. We have evaluated Canadian patients enrolled in Global aHUS Registry provide a perspective regarding diagnosis and management specific challenges faced.To evaluate faced.The observational, noninterventional, multicenter study has prospectively retrospectively collected data from all ages with investigator-made clinical...
Abstract Background and Aims Atypical haemolytic uraemic syndrome (aHUS) is a progressive rare disease caused by complement dysregulation that can result in severe organ damage death. Ravulizumab, next-generation terminal inhibitor, was designed via targeted modification of eculizumab to enhance antibody recycling attenuate target-mediated drug disposition achieve an extended half-life. Ravulizumab first approved for the treatment aHUS 2019. Real-world evidence with ravulizumab patients...
Background Thrombotic microangiopathy (TMA) associated with hematopoietic stem cell transplantation (HSCT-TMA) is a serious post-transplant complication. Diagnosis difficult due to overlapping symptoms other conditions and lack of universally adopted diagnostic criteria. Methods This retrospective, observational study investigated HSCT-TMA incidence between July 2009–August 2020 using the TriNetX US Electronic Medical Record database. Patients who underwent autologous or allogeneic HSCT...