EBMT 2024 | The diagnosis and management of TA-TMA in pediatric patients

I was able to give two talks. The first was on the diagnosis and management of TA-TMA in pediatrics, and what we went through together is that TMA is associated with poor outcomes, that patients that develop TMA are at higher risk of mortality, as well as complications of transplant, including ICU stays and intubation and pulmonary hypertension and renal failure. We discussed some of the treatments and modalities of treatments, as well as risk stratification of patients, into a high risk, moderate risk and low risk categories. And, went into further details of screening and how we should screen for TMA and how important that is.

Now, this talk was a little bit more granular, a little longer. So, we talked in detail about strategies and screening and risk stratification of stem cell transplant patients. We first started talking about the organs affected by stem cell transplant, which include the lungs and heart and gastrointestinal, kidneys and so forth. And we went into more details of a study we conducted, that was published in 2021, in Blood Advances, that included a multicenter, implementation of standardized screening for TMA and then a retrospective analysis of the data and looked at the different outcomes. And in that, we were able to break down what organs are affected if TMA and then also how to screen for each of those organs, organ dysfunction and what it likely means. Importantly, we talked about intestinal TMA and how the diagnosis can often be missed and that clinicians should work closely with their pathology friends and colleagues into making sure that they don’t miss it. And that the vasculature is thoroughly evaluated in a patient suspected of having TMA.