Evaluating ravulizumab for the treatment of children and adolescents with paroxysmal nocturnal hemoglobinuria.
Justin J YooSatheesh ChonatPublished in: Expert review of hematology (2022)
The discovery of eculizumab, a monoclonal antibody against complement protein 5, has revolutionized the PNH landscape, with decreased hemolysis and risk of thrombosis, improved quality-of-life, and has become the standard of care. Ravulizumab, a longer-acting C5-inhibitor with 4 times the half-life of eculizumab, was recently approved for pediatric patients with PNH. Ravulizumab is effective, safe, and has the potential to improve quality of life further. In addition, ongoing clinical trials using second-generation complement inhibitors may provide promising new interventions in PNH.
Keyphrases
- monoclonal antibody
- clinical trial
- healthcare
- palliative care
- blood pressure
- small molecule
- physical activity
- pulmonary embolism
- obstructive sleep apnea
- quality improvement
- protein protein
- high throughput
- pain management
- randomized controlled trial
- single cell
- young adults
- sleep quality
- red blood cell
- open label
- amino acid
- childhood cancer
- myasthenia gravis