Post-Conference Perspectives: Advances in the Treatment of HSCT-TMA - Episode 5

Future Directions For the Treatment of Transplant TMA


Vincent T. Ho, MD: The question is in regard to what the next 5 years will bring in terms of the treatment of transplant TMA [thrombotic microangiopathy]. I think we’re in an exciting time right now. For the last 2 decades, nobody has paid attention to TMA because there was no effective therapy. With the advent of complement blockers and with a better understanding of the pathophysiology of this disease, there’s now renewed interested in this realm.

There is still a lot of work that needs to be done in terms of tying down good diagnostic criteria as well as finding good biomarkers to help us diagnose the disease. We now know there are certain risk factors for this disease. For example, genetic testing of patients to look for predisposing genes might help us target certain populations to screen and to use prophylaxis. Once we are more cognizant of looking for this condition during transplant, we’re more likely to diagnose it earlier in the course by implementing more stringent screening; for example, screening for hypertension and screening for proteinuria. Being vigilant to the diagnosis will allow us to diagnose this condition earlier. Having tests to look for complements will help us decide who might be the best candidates for intervention with complement blockers or with a drug like defibrotide.

I think there’s a lot of interest in this disease because there are now potentially effective treatments. This will be a major step forward because it will help improve our transplant-related mortality. Much of the mortality after transplant from graft-vs-host disease probably intertwines with morbidity from thrombotic microangiopathy. In my experience, this tends to go hand in hand with graft-vs-host disease, and graft-vs-host disease is 1 of the most common complications of transplantation.

Transcript Edited for Clarity