|Title||Transplant-Associated Thrombotic Microangiopathy (TA-TMA) and Consensus Based Diagnostic and Therapeutic Recommendations: Which TA-TMA Patients to Treat and When?|
|Publication Type||Journal Article|
|Year of Publication||2014|
|Authors||Uderzo, C, Jodele, S, El Missiry, M, Ciceri, F, Busca, A, Bacigalupo, A, Corbacioglu, S|
|Publication Name||Journal of Bone Marrow Research|
|Pagination||1 - 7|
Background: Transplant-associated thrombotic microangiopathy (TA-TMA) is considered one of the most severecomplications after hematopoietic stem cell transplantation. Unfortunately, controversial approaches on TA-TMAdiagnostic criteria contribute to a delay in both diagnosis and treatment. Recommendations for TA-TMA based in thepast on case reports or retrospective studies lack a reasonable level of evidence. One of the most promising drugsfor TA-TMA likely induced by endothelium damage is Defibrotide, a polydisperse oligonucleotide. Auto-antibodydepleting or complement blocking therapy has also emerged as new strategy to decrease TA-TMA-associatedmorbidity and mortality.Methods: A joint study group of experts on TA-TMA met during the 2013 ASH Meeting (New Orleans, USA) andthe 2014 EBMT Meeting (Milan, Italy) with the aim of proposing a reliable treatment for this complication. Commondiagnostic criteria for TA-TMA have been discussed and are described in the review. Factors influencing theoutcome of TA-TMA are also addressed.Results: The panel agreed that an endothelial damage pathway is involved in the pathogenesis of TA-TMA. Withemerging data, an updated version of TA-TMA diagnostic criteria is suggested. High or standard risk patients havebeen defined according to TA-TMA risk factors and a comprehensive therapeutic strategy for TA-TMA patients hasbeen designed for a possible multicentre study.Conclusions: The panel focused primarily on high level of awareness about an early TA-TMA diagnosis andtreatment before a TA-TMA-induced multi-organ failure. An important consensus was obtained to investigatewhether Defibrotide or Eculizumab can improve the outcome of TA-TMA in a multicentre study.
|Short Title||J Bone Marrow Res|