Thrombotic microangiopathy in children: Redefining hemolytic uremic syndrome, thrombotic thrombocytopenic purpura and related disorders
Thrombotic microangiopathy (TMA) is a triad of microangiopathic hemolytic anemia, thrombocytopenia, and end-organ ischemic damage. The primary mechanism involved is the occurrence of microthrombi due to deficient activity of ADAMTS13 (A Disintegrin And Metalloprotease with ThromboSpondin type 1 repe...
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| Format: | Article |
| Language: | English |
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Elsevier
2024-03-01
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| Series: | Pediatric Hematology Oncology Journal |
| Subjects: | |
| Online Access: | http://www.sciencedirect.com/science/article/pii/S2468124524000056 |
| _version_ | 1797244911907504128 |
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| author | Mamta Manglani Pranoti Kini |
| author_facet | Mamta Manglani Pranoti Kini |
| author_sort | Mamta Manglani |
| collection | DOAJ |
| description | Thrombotic microangiopathy (TMA) is a triad of microangiopathic hemolytic anemia, thrombocytopenia, and end-organ ischemic damage. The primary mechanism involved is the occurrence of microthrombi due to deficient activity of ADAMTS13 (A Disintegrin And Metalloprotease with ThromboSpondin type 1 repeats, member 13). The most common types of TMA in children are Shiga toxin-producing Escherichia coli-associated hemolytic uremic syndrome (STEC-HUS) followed by complement-mediated (CM) TMA, Streptococcus pneumoniae-associated hemolytic uremic syndrome (Sp-HUS) and hereditary thrombotic thrombocytopenic purpura (hTTP) and other rare causes. Since the outcomes are dismal if appropriate treatment is not promptly initiated, there is a need to have a high clinical suspicion. Additionally, urgently performing ADAMTS13 functional activity and autoantibody levels can help differentiate hTTP, immune thrombotic thrombocytopenic purpura (iTTP), and CM-TMA. The etiological differentiation is crucial as eculizumab is a specific therapy with exceedingly good results in CM-TMA. While plasma exchanges are required for iTTP, besides corticosteroids and/or rituximab, plasma infusions suffice for hTTP. This review focuses on the commonly encountered congenital and acquired types of TMA in children and their varied presentations while briefly touching upon the rarer disorders causing TMA. |
| first_indexed | 2024-04-24T19:18:32Z |
| format | Article |
| id | doaj.art-60a9745ee91747b2829473ab503950ae |
| institution | Directory Open Access Journal |
| issn | 2468-1245 |
| language | English |
| last_indexed | 2024-04-24T19:18:32Z |
| publishDate | 2024-03-01 |
| publisher | Elsevier |
| record_format | Article |
| series | Pediatric Hematology Oncology Journal |
| spelling | doaj.art-60a9745ee91747b2829473ab503950ae2024-03-26T04:26:56ZengElsevierPediatric Hematology Oncology Journal2468-12452024-03-01914553Thrombotic microangiopathy in children: Redefining hemolytic uremic syndrome, thrombotic thrombocytopenic purpura and related disordersMamta Manglani0Pranoti Kini1Corresponding author. MCGM – Comprehensive Thalassemia Care, Pediatric Hematology-Oncology and BMT Centre, CCI Compound, Opp Samarpan Exotica, Borivali (E), Mumbai, 400066, India.; MCGM – Comprehensive Thalassemia Care, Pediatric Hematology-Oncology and BMT Centre, Borivali (E), Mumbai, 400066, IndiaMCGM – Comprehensive Thalassemia Care, Pediatric Hematology-Oncology and BMT Centre, Borivali (E), Mumbai, 400066, IndiaThrombotic microangiopathy (TMA) is a triad of microangiopathic hemolytic anemia, thrombocytopenia, and end-organ ischemic damage. The primary mechanism involved is the occurrence of microthrombi due to deficient activity of ADAMTS13 (A Disintegrin And Metalloprotease with ThromboSpondin type 1 repeats, member 13). The most common types of TMA in children are Shiga toxin-producing Escherichia coli-associated hemolytic uremic syndrome (STEC-HUS) followed by complement-mediated (CM) TMA, Streptococcus pneumoniae-associated hemolytic uremic syndrome (Sp-HUS) and hereditary thrombotic thrombocytopenic purpura (hTTP) and other rare causes. Since the outcomes are dismal if appropriate treatment is not promptly initiated, there is a need to have a high clinical suspicion. Additionally, urgently performing ADAMTS13 functional activity and autoantibody levels can help differentiate hTTP, immune thrombotic thrombocytopenic purpura (iTTP), and CM-TMA. The etiological differentiation is crucial as eculizumab is a specific therapy with exceedingly good results in CM-TMA. While plasma exchanges are required for iTTP, besides corticosteroids and/or rituximab, plasma infusions suffice for hTTP. This review focuses on the commonly encountered congenital and acquired types of TMA in children and their varied presentations while briefly touching upon the rarer disorders causing TMA.http://www.sciencedirect.com/science/article/pii/S2468124524000056Thrombotic microangiopathy (TMA)Hemolytic uremic syndrome (HUS)Thrombotic thrombocytopenia purpura (TTP)ADAMTS13Upshaw schulman syndrome (USS) |
| spellingShingle | Mamta Manglani Pranoti Kini Thrombotic microangiopathy in children: Redefining hemolytic uremic syndrome, thrombotic thrombocytopenic purpura and related disorders Pediatric Hematology Oncology Journal Thrombotic microangiopathy (TMA) Hemolytic uremic syndrome (HUS) Thrombotic thrombocytopenia purpura (TTP) ADAMTS13 Upshaw schulman syndrome (USS) |
| title | Thrombotic microangiopathy in children: Redefining hemolytic uremic syndrome, thrombotic thrombocytopenic purpura and related disorders |
| title_full | Thrombotic microangiopathy in children: Redefining hemolytic uremic syndrome, thrombotic thrombocytopenic purpura and related disorders |
| title_fullStr | Thrombotic microangiopathy in children: Redefining hemolytic uremic syndrome, thrombotic thrombocytopenic purpura and related disorders |
| title_full_unstemmed | Thrombotic microangiopathy in children: Redefining hemolytic uremic syndrome, thrombotic thrombocytopenic purpura and related disorders |
| title_short | Thrombotic microangiopathy in children: Redefining hemolytic uremic syndrome, thrombotic thrombocytopenic purpura and related disorders |
| title_sort | thrombotic microangiopathy in children redefining hemolytic uremic syndrome thrombotic thrombocytopenic purpura and related disorders |
| topic | Thrombotic microangiopathy (TMA) Hemolytic uremic syndrome (HUS) Thrombotic thrombocytopenia purpura (TTP) ADAMTS13 Upshaw schulman syndrome (USS) |
| url | http://www.sciencedirect.com/science/article/pii/S2468124524000056 |
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