- 28 Downloads
An event is serious (based on the ICH definition) when the patient outcome is:
* congenital anomaly
* other medically important event
In a case series of four infants [ages at events onsets not clearly stated], one male and three female infants were described, who developed neutralising antibodies to alglucosidase-α during treatment with recombinant alglucosidase-α for infantile-onset Pompe disease (IPD). Additionally, three of the four patients developed infusion associated reactions (IAR), which manifested as vomiting, fever and tachycardia in one infant and hypotension, tongue and lip swelling, reduced oxygen saturations, tachycardia, fever and rash in one infant during treatment with recombinant alglucosidase-α. Two patients died secondary to alglucosidase-α efficacy attenuation due to high sustained titres of neutralising antibodies (HSAT) [not all durations of treatments to reactions onsets and outcomes...
- Owens P, et al. Infantile-onset Pompe disease: A case series highlighting early clinical features, spectrum of disease severity and treatment response. Journal of Paediatrics and Child Health 54: 1255-1261, No. 11, Nov 2018. Available from: URL: http://doi.org/10.1111/jpc.14070 - Australia