Thrombotic Microangiopathy In Pediatric Patients

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About this Research Topic

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Background

Thrombotic microangiopathy (TMA) is characterized by non-immune hemolytic anemia and thrombocytopenia and laboratory findings of elevated serum lactate dehydrogenase and indirect bilirubin and low haptoglobin levels. TMA is diagnosed based on histopathological evaluation of tissue biopsy samples, specifically renal biopsy specimens.

Pediatric TMA includes hemolytic uremic syndrome secondary to Shiga toxin-producing Escherichia coli infection (STEC-HUS), complement-mediated HUS secondary to complement dysregulation (hereditary or acquired), thrombotic thrombocytopenic purpura (TTP), drug-induced TMA, TMA associated with collagen diseases, hematopoietic stem cell transplantation, and rare metabolic diseases.

In recent years, the development of innovative molecular targeted drugs, which serve as anticomplement agents, has resulted in significant advances in the treatment of complement-mediated HUS, which occurs secondary to complement dysregulation.

Accurate diagnosis and effective treatment of TMA are important to improve prognosis and protect renal function. It is important that pediatricians accurately identify the early signs and symptoms of TMA and perform appropriate clinical evaluation for prompt diagnosis and treatment. Progressive TMA leads to worsening of the patient's condition and can result in permanent organ injury and may be associated with fatal outcomes; therefore, early diagnosis is essential to facilitate prompt treatment.

Studies have reported the effectiveness of early administration of complement inhibitors in patients with complement-mediated HUS, and significant progress has occurred in this field in recent years. Genetic analysis is a useful method for evaluation of complement-related factors. Therefore, it is necessary to develop a novel testing method that can accurately determine the etiology of TMA based on the patient's symptoms and clinical course, to enable early treatment initiation.

The purpose of this research is to investigate the mechanisms underlying TMA, the characteristic clinical course, effective treatment strategies, as well as appropriate laboratory tests and diagnostic methods. This information will be useful to select more effective therapeutic approaches to pediatric TMA and improve the prognosis of this patient population. We intend to accumulate data from both basic and clinical research papers; we will search the appropriate databases for Original Research, Reviews, Mini-Reviews, Case Reports, and Perspective articles that discuss the following topics:
1. Pathophysiology of TMA
2. Clinical features, interesting cases, and effective treatment strategies used in patients with STEC-HUS/complement-mediated HUS/pediatric TTP
3. Recent advances in complement dysregulation, testing methods
4. TMA associated with hematopoietic stem cell transplantation

Keywords: Thrombotic microangiopathy, Hemolytic uremic syndrome, Shiga toxin-producing Escherichia coli, complement-mediated HUS

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