Thrombotic microangiopathy: a formidable complication of gene therapy

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.

 

Thrombotic Microangiopathy as an Emerging Complication of Viral Vector-Based Gene Therapy. Schwotzer N, El Sissy C, Desguerre I, et al. Kidney Int Rep. 2024 Apr.