TY - JOUR
T1 - Atypical Presentations of Pediatric-acquired Thrombotic Thrombocytopenic Purpura
AU - Ziv, Amit
AU - Dardik, Rima
AU - Yacobovich, Joanne
AU - Uziel, Yosef
AU - Haviv, Ruby
AU - Avishai, Einat
AU - Kenet, Gili
AU - Toren, Amos
AU - Barel, Ortal
AU - Barg, Assaf Arie
N1 - Publisher Copyright:
© 2024 Lippincott Williams and Wilkins. All rights reserved.
PY - 2024/8/1
Y1 - 2024/8/1
N2 - Background: Immune thrombotic thrombocytopenic purpura (iTTP) in children is a rare, severe thrombotic microangiopathy. This condition is characterized by microangiopathic hemolytic anemia, severe thrombocytopenia, and organ ischemia due to reduced activity of the von Willebrand factor-cleaving protease ADAMTS13. Methods: A retrospective case series evaluating data collected from the medical files of 4 children diagnosed with iTTP. Results: The presented case series depicts a variety of iTTP presentations: 1 case of primary iTTP, 1 case induced by Shiga toxin, 1 associated with RAS-associated autoimmune leukoproliferative disease (RALD), and 1 initial manifestation of systemic lupus erythematosus (SLE). Notably, 2 patients recovered without undergoing plasma exchange. Conclusion: Early ADAMTS13 testing in children with unexplained hemolysis or thrombocytopenia is crucial. The diverse underlying causes, including infections and autoimmune disorders, underscore the complexity of iTTP in the pediatric population. These cases highlight the necessity for personalized treatment approaches that consider each patient's unique clinical situation and potential alternatives or modifications to conventional therapeutic regimens.
AB - Background: Immune thrombotic thrombocytopenic purpura (iTTP) in children is a rare, severe thrombotic microangiopathy. This condition is characterized by microangiopathic hemolytic anemia, severe thrombocytopenia, and organ ischemia due to reduced activity of the von Willebrand factor-cleaving protease ADAMTS13. Methods: A retrospective case series evaluating data collected from the medical files of 4 children diagnosed with iTTP. Results: The presented case series depicts a variety of iTTP presentations: 1 case of primary iTTP, 1 case induced by Shiga toxin, 1 associated with RAS-associated autoimmune leukoproliferative disease (RALD), and 1 initial manifestation of systemic lupus erythematosus (SLE). Notably, 2 patients recovered without undergoing plasma exchange. Conclusion: Early ADAMTS13 testing in children with unexplained hemolysis or thrombocytopenia is crucial. The diverse underlying causes, including infections and autoimmune disorders, underscore the complexity of iTTP in the pediatric population. These cases highlight the necessity for personalized treatment approaches that consider each patient's unique clinical situation and potential alternatives or modifications to conventional therapeutic regimens.
KW - ADAMTS13
KW - immune thrombotic thrombocytopenic purpura
KW - pediatric
KW - treatment
UR - http://www.scopus.com/inward/record.url?scp=85199814683&partnerID=8YFLogxK
U2 - 10.1097/MPH.0000000000002914
DO - 10.1097/MPH.0000000000002914
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C2 - 38934698
AN - SCOPUS:85199814683
SN - 1077-4114
VL - 46
SP - 306
EP - 310
JO - Journal of Pediatric Hematology/Oncology
JF - Journal of Pediatric Hematology/Oncology
IS - 6
ER -