A5043961914- Hemoglobinopathies and Related Disorders
- Iron Metabolism and Disorders
- Complement system in diseases
- Blood groups and transfusion
- Prenatal Screening and Diagnostics
- Erythrocyte Function and Pathophysiology
- Coagulation, Bradykinin, Polyphosphates, and Angioedema
- Metabolism, Diabetes, and Cancer
- Erythropoietin and Anemia Treatment
- Folate and B Vitamins Research
- Renal Diseases and Glomerulopathies
- Genomics and Rare Diseases
- Diabetes Management and Research
- Neonatal Health and Biochemistry
- Blood properties and coagulation
- Platelet Disorders and Treatments
- Chronic Lymphocytic Leukemia Research
- Congenital Ear and Nasal Anomalies
- Metabolism and Genetic Disorders
- Renal Transplantation Outcomes and Treatments
- Biotin and Related Studies
- Immunodeficiency and Autoimmune Disorders
- Connective tissue disorders research
- Cancer, Hypoxia, and Metabolism
- Caveolin-1 and cellular processes
Hospital Clínico San Carlos
2011-2024
Instituto de Investigación Sanitaria del Hospital Clínico San Carlos
2022
Palmetto Hematology Oncology
2019
Universidad Complutense de Madrid
2012
Abstract Ravulizumab every 8 weeks showed non‐inferiority to eculizumab 2 in a 26‐week, phase 3, randomized controlled trial adults with paroxysmal nocturnal hemoglobinuria (PNH) who were clinically stable on (NCT03056040). We report results from the first 26 of extension period which patients continued ravulizumab (n = 96) or switched 95). At week 52, mean (SD) lactate dehydrogenase levels increased 8.8% (29%) ravulizumab‐ravulizumab and 5.8% (27%) eculizumab‐ravulizumab primary evaluation...
To verify with hematimetric data that the diagnosis and clinical grade of β-TI can be established when a triplication alpha genes (αααanti 3.7) heterozygous β-thalassemia coexist.Retrospective study in which 73 patients Caucasian origin participated, who simultaneously showed or quadruplication α β-thalassemia.Screening for most frequent α-thalassemia mutations as well gene was carried out by multiplex PCR followed reverse hybridization commercial Alpha-Globin StripAssay kit confirmed MLPA...
más frecuente en el mundo, con una incidencia de 3 a 80 casos por 100.000 habitantes/año (1) que está aumen-
Background: Since 2007, eculizumab has transformed the management of paroxysmal nocturnal hemoglobinuria (PNH). However, it a treatment burden associated with q2w dosing. Recently, FDA approved ravulizumab for PNH treatment. Ravulizumab administered q8w demonstrated noninferiority to in two phase 3 trials on all primary and key secondary efficacy measures. In view these options, is important consider patient preference determining plan patients (pts). Aims: To assess or treatment, clinical...
Topic: 12. Bone marrow failure syndromes incl. PNH - Clinical Background: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare chronic hematologic disorder characterized by uncontrolled terminal complement activation, major adverse vascular events (MAVEs, including thrombosis) and increased morbidity mortality. Where available, ravulizumab considered the standard of care for patients with PNH, supported pivotal studies adult inhibitor-naïve inhibitor-experienced (studies 301 302,...
Sickle Cell Disease (SCD) is one of the most prevalent autosomal recessive disease, affecting over 600,000 newborns globally each year. In Spain, Spanish Registry Hemoglobinopathies (REHem) has registered 1142 cases. Despite improvements in treatments, prophylaxis, and vaccines, it remains a chronic condition requiring lifelong care. Allogeneic transplantation only cure, contingent upon availability an HLA-compatible donor. SCD considered rare disease some population, resulting from mutation...
Objectives. Check with hematological data that the diagnosis and clinical grade of β-thalassemia intermedia can be established when a triplication genes alpha (αααanti 3.7) heterozygous are coherent. Methods. Retrospective study in which 73 patients Caucasian origin participated, who simultaneously showed tripling or quadrupling α β-thalassemia. Screening for most frequent α-thalassemia mutations, as well gene was carried out by multiplex PCR followed reverse hybridization confirmed MLPA....