Thrombotic Thrombocytopenic Purpura in Pediatric Patients
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, th...
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MDPI AG
2025-04-01
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| Online Access: | https://www.mdpi.com/2227-9059/13/5/1038 |
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| author | Niki Shrestha Ebruphiyo Okpako Robert W. Maitta |
| author_facet | Niki Shrestha Ebruphiyo Okpako Robert W. Maitta |
| author_sort | Niki Shrestha |
| collection | DOAJ |
| description | 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. |
| format | Article |
| id | doaj-art-c42c0e504ee9432bb536cfacab6c2b1b |
| institution | DOAJ |
| issn | 2227-9059 |
| language | English |
| publishDate | 2025-04-01 |
| publisher | MDPI AG |
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| series | Biomedicines |
| spelling | doaj-art-c42c0e504ee9432bb536cfacab6c2b1b2025-08-20T03:14:32ZengMDPI AGBiomedicines2227-90592025-04-01135103810.3390/biomedicines13051038Thrombotic Thrombocytopenic Purpura in Pediatric PatientsNiki Shrestha0Ebruphiyo Okpako1Robert W. Maitta2Department of Pathology, University Hospitals Cleveland Medical Center, Case Western Reserve University, Cleveland, OH 44106, USADepartment of Pathology, University Hospitals Cleveland Medical Center, Case Western Reserve University, Cleveland, OH 44106, USADepartment of Pathology, University Hospitals Cleveland Medical Center, Case Western Reserve University, Cleveland, OH 44106, USAThrombotic 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.https://www.mdpi.com/2227-9059/13/5/1038thrombotic thrombocytopenic purpuracongenitalimmune-mediatedTTPsteroidstherapeutic plasma exchange |
| spellingShingle | Niki Shrestha Ebruphiyo Okpako Robert W. Maitta Thrombotic Thrombocytopenic Purpura in Pediatric Patients Biomedicines thrombotic thrombocytopenic purpura congenital immune-mediated TTP steroids therapeutic plasma exchange |
| title | Thrombotic Thrombocytopenic Purpura in Pediatric Patients |
| title_full | Thrombotic Thrombocytopenic Purpura in Pediatric Patients |
| title_fullStr | Thrombotic Thrombocytopenic Purpura in Pediatric Patients |
| title_full_unstemmed | Thrombotic Thrombocytopenic Purpura in Pediatric Patients |
| title_short | Thrombotic Thrombocytopenic Purpura in Pediatric Patients |
| title_sort | thrombotic thrombocytopenic purpura in pediatric patients |
| topic | thrombotic thrombocytopenic purpura congenital immune-mediated TTP steroids therapeutic plasma exchange |
| url | https://www.mdpi.com/2227-9059/13/5/1038 |
| work_keys_str_mv | AT nikishrestha thromboticthrombocytopenicpurpurainpediatricpatients AT ebruphiyookpako thromboticthrombocytopenicpurpurainpediatricpatients AT robertwmaitta thromboticthrombocytopenicpurpurainpediatricpatients |