Pyruvate kinase activators in hereditary haemolytic anaemias: current evidence and clinical potential - PubMed
5 hours ago
- #pyruvate kinase activators
- #clinical trials
- #haemolytic anaemias
- Hereditary haemolytic anaemias are the most prevalent genetic disorders globally with significant health impacts.
- Current treatments are limited and mainly supportive.
- Metabolic impairment in red blood cells plays a crucial role in these diseases beyond the primary genetic defect.
- Pyruvate kinase activators enhance glycolysis, increasing ATP production and improving cellular homeostasis.
- The first pyruvate kinase activator has been approved for treating pyruvate kinase deficiency.
- Clinical trials show efficacy in other haemolytic disorders like thalassaemia, sickle cell disease, and red blood cell membrane disorders.
- Pyruvate kinase activators improve haemolytic anaemia and other disease-related outcomes with a favourable safety profile.
- Ongoing research explores further potential indications for pyruvate kinase activators.