Ravulizumab Plus Best Supportive Care in Pediatric Patients with Hematopoietic Stem Cell Transplantation-Associated Thrombotic Microangiopathy: 52-Week Results from a phase 3 Trial

Key Points

• Ravulizumab administration led to significant improvements in key clinical outcomes over 52 weeks.
• The primary endpoint achieved a notable response rate of 75% among treated patients.
• This phase 3 trial evaluated the impacts of ravulizumab combined with supportive care in pediatric patients.
• These findings highlight the necessity for new therapies in managing hematopoietic stem cell transplantation complications.