Where significant CNS changes are observed on ultrasound (ventriculomegaly, microcephaly, cerebellar hypoplasia), it is highly likely that these will be associated with significant sequelae.
The development and evaluation of gene-specific therapy (e.g., cost benefit of vitamin A, antiusherin antibiotics, or medication to reduce retinitis pigmentosa sequelae) will be possible.
The information including the type, date(s) of onset and resolution (if applicable), severity of influence, relationship with grouping, treatment and outcome (sequelae) will be recorded in the Case Report Forms.
Among patients with a good clinical outcome, it is impossible to separate those who will have a complete recovery (restitutio ad integrum) from those whose quality of life will be hampered by significant neurological sequelae.
Further, targeted therapies need to be identified such that normal developing brain tissue will be spared, thereby avoiding the disabling sequelae which are, unfortunately, commonplace in survivors.
Primary infections in pregnant women result in fetal infection 40%50%% of the time; 10% of infected infants will be symptomatic at birth, and neurologic sequelae will develop in nearly all of them.
Some AEs, such as bacteriuria, urinary tract infections, crystalluria and diarrhoea were only observed among subjects treated with tralokinumab, although these did not result in either early withdrawal or sequelae; further studies will be necessary to determine whether these events are related to blockade of IL-13.
In the near future, the aim will be to prevent these disorders and their sequelae through early detection and early intervention (for example genetic therapy).
Follow-up studies will be required to determine if long-term sequelae are indeed different between non-severe and severe clinical presentations.
