Gene therapy for spinal muscular atrophy (SMA) has involved some 40 French infants to date. The onesamnogene abeparvovec (Zolgensma®) was administered in a single intravenous injection. Clinicians in Paris and Nantes report the occurrence in one of them of thrombotic microangiopathy (TMA), a serious and potentially lethal complication directly related to the treatment:
- this child, treated at the age of six months, presented a picture comparable to a haemolytic uraemic syndrome a few days after the gene therapy,
- she did not respond to further treatment of TAM with eculizimab, resulting in a fatal outcome,
- agenetic susceptibility to complement factor abnormalities could be a risk factor in this case.
Early detection and appropriate treatment of MAT is essential.
