With the recent FDA approval of narsoplimab for transplant-associated thrombotic microangiopathy, the field is moving from recognition to intervention. Dr Eleni Gavriilaki discusses the evolving role of complement inhibition, endothelial-directed strategies, and why earlier-career researchers will be central to advancing transplant medicine.
“What we now understand is that TA-TMA is not simply another complication of transplantation; it is a distinct thrombotic microangiopathy with unique biological drivers”
Once considered a poorly defined and often fatal complication of allogeneic transplantation, transplant-associated thrombotic microangiopathy is increasingly being understood as a biologically distinct syndrome driven in part by complement dysregulation and endothelial injury. Speaking at the European Hematology Association (EHA) 2026 Congress, Dr Eleni Gavriilaki (Aristotle University of Thessaloniki, Greece) outlines how advances in complement biology have helped distinguish TA-TMA from severe GVHD and informed the development of targeted therapies. With narsoplimab now approved and ravulizumab data emerging, she argues the next challenge is refining patient selection, improving access, and broadening investigation into endothelial-targeted interventions.
touchHEMATOLOGY coverage from EHA 2026
What do you see as the biggest unmet need in managing TA-TMA today?
Transplant-associated thrombotic microangiopathy (TA-TMA) has been a major focus of my work for more than a decade. When we first started studying it, it was significantly under-recognized. Many clinicians did not actively diagnose it because there were no effective treatments available. That has changed. What we now understand is that TA-TMA is not simply another complication of transplantation; it is a distinct thrombotic microangiopathy with unique biological drivers.
The challenge is that it often emerges in very complex clinical settings. Patients may already have severe graft-versus-host disease, infections, or multiple organ dysfunction. In that context, TA-TMA can be difficult to identify. I often describe it as the ‘tip of the iceberg’.
One of the key questions has been why some patients with severe inflammatory complications develop TA-TMA while others do not. Our work, and that of others, suggests genetic susceptibility and complement dysregulation are important factors. Complement activation appears to be one of the defining features separating TA-TMA from GVHD.
The unmet need now is precision: understanding which patients will benefit most, and when to intervene.
How close are monoclonal antibodies and complement inhibitors to becoming standard of care?
We are much closer than we were even a few years ago. The approval of narsoplimab is an important milestone. It targets the lectin pathway of complement activation and represents the first approved therapy specifically for TA-TMA. This is highly significant because it validates the biology we have been studying for years. It also gives clinicians a targeted therapeutic option in a setting where treatment has historically been largely supportive.
As experts in the field, we now need to use these agents in clinical practice and understand their limitations. Safety has been reassuring so far, which is critical in this very fragile patient population. The main issue is access. Availability is still limited in many parts of the world, and this remains a major barrier.
We are also awaiting adult data for ravulizumab. Pediatric studies have been encouraging, and if adult outcomes are similarly positive, that could expand access substantially because ravulizumab is already more widely available globally.
Ultimately, I think we are entering a phase where these therapies will become integrated into standard management, but treatment will need to be individualized.
Where could endothelial-targeted therapies have the greatest impact in transplantation medicine?
Endothelial injury is central to many transplant complications. TA-TMA is one of the clearest examples, but it is also relevant to other syndromes, both inside and outside transplantation.
The difficulty is that endothelial dysfunction is challenging to measure directly. That is why we often focus on syndromes of endothelial injury rather than trying to assess the endothelium itself. At the moment, we do not have therapies that directly target endothelial injury. What we have are treatments that interrupt the downstream consequences; the vicious cycle of complement activation, coagulation, and inflammation. If you can interrupt that cycle, the endothelium may recover.
I think there is significant room to explore additional approaches. Statins are a good example. They are widely used for dyslipidaemia, but they also have endothelial-protective effects. These have been relatively understudied in transplantation. As we improve survival and gain better disease control, it will become increasingly important to investigate preventive and endothelial-stabilizing strategies.
What is most important in supporting the next generation of transplant researchers?
Mentorship is fundamental. Through Young EHA, we have developed programmes focused on education, mentorship, and research collaboration, all designed to support early-career clinicians and scientists. We are also building partnerships with sister organisations such as the European Society for Blood and Marrow Transplantation (EBMT), particularly in transplantation and CAR T-cell therapy. These collaborations create opportunities for younger colleagues to build networks, exchange ideas, and develop research skills. Importantly, they help younger researchers find their voice.
Why is it so important to spotlight early-career professionals in hematology?
Because every generation needs support. Many of us benefited from senior leaders who created space for younger voices, and it is important that we continue that. Hematology is a field where innovation directly changes patient outcomes. That is very motivating for young physicians and scientists. But to sustain that innovation, we need visible role models, structured mentorship, and opportunities for recognition. Initiatives that highlight early-career work are not just beneficial for individuals; they are essential for the future of the specialty.
This content has been developed independently by Touch Medical Media for touchHEMATOLOGY. Views expressed are the speaker’s own and do not necessarily reflect the views of Touch Medical Media. This article was created by the editorial team utilizing AI as an editorial tool (ChatGPT 5.5 [Large language model]. https://chat.openai.com/chat.) The content was developed and edited by human editors. No funding was received in the publication of this article.
Cite: Refining the management of transplant-associated thrombotic microangiopathy: A Q&A with Dr Eleni Gavriilaki. touchHEMATOLOGY. 2nd July 2026.
Disclosure: Eleni Gavriilaki has no financial or non-financial conflicts of interest to declare in relation to this article.
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