Unit:
Κατεύθυνση Θρόμβωση-Αιμορραγία-Ιατρική των μεταγγίσεωνLibrary of the School of Health Sciences
Author:
Patriarcheas Vasileios
Supervisors info:
Γαβριηλάκη Ελένη, Καθηγήτρια, Ιατρική Σχολή, ΑΠΘ
Κόκορη Στυλιανή, Αναπληρώτρια Καθηγήτρια, Ιατρική Σχολή, ΕΚΠΑ
Τσιριγλωτη Παναγιώτης, Καθηγητής, Ιατρική Σχολή, ΕΚΠΑ
Original Title:
Μελέτη του συμπληρώματος σε ασθενείς με θρομβωτική μικροαγγειοπάθεια μετά από αλλογενή μεταμόσχευση αιμοποιητικών κυττάρων
Translated title:
Study of the role of the Complement system, in patients with Transpant- Associated Thrombotic Microangiopathy (HSCT-TMA) after Allogenic Hematopoietic Cell Transplantation»
Summary:
Introduction: Hematopoietic stem cell transplantation-associated thrombotic microangiopathy (HSCT-TMA) constitutes a severe complication of transplantation, associated with high morbidity and mortality. Evidence of complement involvement in the pathogenesis of HSCT-TMA, has led to the use of complement inhibitors as a potential therapeutic approach.
Purpose: To study the complement activation in patients with HSCT-TMA, through measuring soluble terminal complex (sC5b9) and to investigate their response after treatment with the complement inhibitor eculizumab.
Materials and Methods: We prospectively enrolled consecutive adult patients diagnosed with HSCT-TMA (harmonization panel consensus recommended diagnostic criteria). Soluble C5b-9 was measured at diagnosis and after 4 doses of eculizumab ELISA.
Results: We studied 13 patients that developed HSCT-TMA at median 124 post-transplant days (15-2841). Soluble C5b9 levels, significantly decreased after 4 doses of eculizumab in all patients and remained stable thereafter. No drug-related adverse effects were identified. Seven patients received more than 4 doses (6-15). A relapse of TA-TMA was seen in one patient, occurring 7 months after discontinuation. Prompt re-initiation of eculizumab resulted in an immediate response. Overall survival was significantly higher in patients that received eculizumab compared to no eculizumab.
Conclusions: Administering eculizumab to patients with HSCT-TMA leads to a decrease in complement activation and is linked to improved patient outcomes.
Main subject category:
Health Sciences
Keywords:
Complement system, Thrombosis, Thrombotic microangiopathy, Allogeneic stem cell transplantation, Complement inhibition
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Patriarcheas_Vasileios_MSc.pdf
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