Document Type
Article
Publication Date
4-25-2025
Abstract
Thrombotic thrombocytopenia purpura is a serious disease that can involve complex symptomatology, prolonged hospitalization, and a high risk of mortality if treatment is delayed. This disease is rare, but it is even rarer among pediatric patients. Even though it was first described 100 years ago, the earliest documented case was a pediatric patient. The last three decades have seen the discovery of the pathological mechanisms responsible for its clinical presentation. Symptoms/signs characteristic of microangiopathic hemolytic anemia with significant thrombocytopenia characterize the vast majority of patients. Its pathology centers on the accumulation of ultra-large von Willebrand factor multimers due to an enzyme deficiency that prevents their breakdown. Currently, in pediatric patients, two forms of the disease are known: congenital due to a mutation in the enzyme’s gene and immune-mediated due to enzyme depletion or neutralization secondary to autoantibody formation. With the advent of therapeutic plasma exchanges, immunosuppression, and, more recently, a TTP-specific nanobody, there is reason for optimism that the disease does not necessarily equate to a bad outcome. Thus, the aim of this review is to contrast the congenital and immune-mediated forms of the disease in pediatric patients while presenting them in the context of their pathologic mechanisms, diagnosis, and treatment.
Keywords
congenital, immune-mediated, mutation, steroids, therapeutic plasma exchange, thrombocytopenia, thrombotic thrombocytopenic purpura, ttp
Language
English
Publication Title
Biomedicines
Rights
© 2025 The Author(s). This is an Open Access work distributed under the terms of the Creative Commons Attribution License (https://creativecommons.org/licenses/by/4.0/) which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.
Creative Commons License
This work is licensed under a Creative Commons Attribution 4.0 International License.
Recommended Citation
Shrestha, N., Okpako, E., & Maitta, R. W. (2025). Thrombotic Thrombocytopenic Purpura in Pediatric Patients. Biomedicines, 13(5), 1038. https://doi.org/10.3390/biomedicines13051038
Manuscript Version
Final Publisher Version