Thrombotic Thrombocytopenic Purpura in Pediatric Patients DOI Creative Commons
Niki Shrestha,

Ebruphiyo Okpako,

Robert W. Maitta

et al.

Biomedicines, Journal Year: 2025, Volume and Issue: 13(5), P. 1038 - 1038

Published: April 25, 2025

Thrombotic thrombocytopenia purpura is a serious disease that can involve complex symptomatology, prolonged hospitalization, and high risk of mortality if treatment delayed. This rare, but it even rarer among pediatric patients. Even though was first described 100 years ago, the earliest documented case patient. The last three decades have seen discovery pathological mechanisms responsible for its clinical presentation. Symptoms/signs characteristic microangiopathic hemolytic anemia with significant characterize vast majority Its pathology centers on accumulation ultra-large von Willebrand factor multimers due to an enzyme deficiency prevents their breakdown. Currently, in patients, two forms are known: congenital mutation enzyme’s gene immune-mediated depletion or neutralization secondary autoantibody formation. With advent therapeutic plasma exchanges, immunosuppression, and, more recently, TTP-specific nanobody, there reason optimism does not necessarily equate bad outcome. Thus, aim this review contrast patients while presenting them context pathologic mechanisms, diagnosis, treatment.

Language: Английский

Thrombotic Thrombocytopenic Purpura in Pediatric Patients DOI Creative Commons
Niki Shrestha,

Ebruphiyo Okpako,

Robert W. Maitta

et al.

Biomedicines, Journal Year: 2025, Volume and Issue: 13(5), P. 1038 - 1038

Published: April 25, 2025

Thrombotic thrombocytopenia purpura is a serious disease that can involve complex symptomatology, prolonged hospitalization, and high risk of mortality if treatment delayed. This rare, but it even rarer among pediatric patients. Even though was first described 100 years ago, the earliest documented case patient. The last three decades have seen discovery pathological mechanisms responsible for its clinical presentation. Symptoms/signs characteristic microangiopathic hemolytic anemia with significant characterize vast majority Its pathology centers on accumulation ultra-large von Willebrand factor multimers due to an enzyme deficiency prevents their breakdown. Currently, in patients, two forms are known: congenital mutation enzyme’s gene immune-mediated depletion or neutralization secondary autoantibody formation. With advent therapeutic plasma exchanges, immunosuppression, and, more recently, TTP-specific nanobody, there reason optimism does not necessarily equate bad outcome. Thus, aim this review contrast patients while presenting them context pathologic mechanisms, diagnosis, treatment.

Language: Английский

Citations

0