Journal de la recherche osseuse
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Abstrait

Transplant-Associated Thrombotic Microangiopathy (TA-TMA) and Consensus Based Diagnostic and Therapeutic Recommendations: Which TA-TMA Patients to Treat and When?

Cornelio Uderzo C, Sonata Jodele, Mohamed El Missiry, Fabio Ciceri, Alessandro Busca, Andrea Bacigalupo and Selim Corbacioglu

Background: Transplant-associated thrombotic microangiopathy (TA-TMA) is considered one of the most severe complications after hematopoietic stem cell transplantation. Unfortunately, controversial approaches on TA-TMA diagnostic criteria contribute to a delay in both diagnosis and treatment. Recommendations for TA-TMA based in the past on case reports or retrospective studies lack a reasonable level of evidence. One of the most promising drugs for TA-TMA likely induced by endothelium damage is Defibrotide, a polydisperse oligonucleotide. Auto-antibody depleting or complement blocking therapy has also emerged as new strategy to decrease TA-TMA-associated morbidity and mortality.
Methods: A joint study group of experts on TA-TMA met during the 2013 ASH Meeting (New Orleans, USA) and the 2014 EBMT Meeting (Milan, Italy) with the aim of proposing a reliable treatment for this complication. Common diagnostic criteria for TA-TMA have been discussed and are described in the review. Factors influencing the outcome of TA-TMA are also addressed.
Results: The panel agreed that an endothelial damage pathway is involved in the pathogenesis of TA-TMA. With emerging data, an updated version of TA-TMA diagnostic criteria is suggested. High or standard risk patients have been defined according to TA-TMA risk factors and a comprehensive therapeutic strategy for TA-TMA patients has been designed for a possible multicentre study.
Conclusions: The panel focused primarily on high level of awareness about an early TA-TMA diagnosis and treatment before a TA-TMA-induced multi-organ failure. An important consensus was obtained to investigate whether Defibrotide or Eculizumab can improve the outcome of TA-TMA in a multicentre study.