Two children die after an anti Sma treatment with vomiting, weakness and a seriously compromised general liver picture up to an extreme outcome
Two kids they die after one treatment anti Sma and “under accusation” the drug ends up Zolgensma with a note Aifa indicating adverse symptoms after use.
The fact is that those two little ones died after being treated with Zolgensma, the drug used against Sma, Spinal Muscular Atrophya genetic disorder that causes motor neuron degeneration basis of the brain.
Anti Sma drug, two children die
From what we learn the two very small victims, of 4 and 28 monthsdied due to “acute liver failure occurring after drug administration”. Drug that Aifa had approved in 2021 and which is paid by the National Health Service “for all children affected by the pathology and under 13.5 kg in weight”.
Fanpage reminds you that at present about 3,000 children are treated with this very expensive drug. The Aifa website reports “common characteristics” in the two cases of death: “Asymptomatic increase in liver aminotransferases within the first 1-2 weeks after the infusion; and then vomiting, weakness and a second increase in liver aminotransferases”.
High liver toxicity
And then a full-blown hepatotoxicity, or the liver toxicitywhich “often manifests as abnormal liver function or increased aminotransferases.”
Aifa therefore recommends a prophylactic regimen “with corticosteroids and liver function monitoring for at least 3 months after drug infusion”. This includes weekly monitoring “for the first month and throughout the corticosteroid tapering period, followed by bi-weekly monitoring for an additional month and, if clinically indicated, at other time points as well.” The two children died 6-7 weeks after the infusion, during the period “gradual reduction of corticosteroid dose”.