A multidisciplinary group of international experts has examined one of the complications that can arise during systemic gene therapy protocols, particularly in children with spinal muscular atrophy (SMA):
- thrombotic microangiopathy (TMA) is an immune runaway phenomenon linked to complement activation, with potentially dramatic consequences,
- Based on published data and their real-life experience with SMA, the authors highlight the complexity of the immune phenomena involved, and the difficulty of assessing the risk of MAT a priori,
Early detection of TMA, very close monitoring of children receiving viral vector-mediated gene therapy, and the use of eculizumab are the three pillars of treatment.
