Clinical trials comparing different drug regimens and strategies for the treatment of congenital toxoplasmosis
and its clinical manifestations in the liveborn child in different clinical settings should aim at formally evaluating
the net benefit of existing treatments and at developing new therapeutic options. Currently, there is no ideal drug
for congenital toxoplasmosis; future research should focus on the screening of new active drugs and on their preclinical
and early clinical development, with a focus on pharmacokinetic/dynamic studies and teratogenicity. For
the prenatal treatment of congenital toxoplasmosis, a trial comparing spiramycine to pyrimethamine-sulphadiazine
and placebo would allow a formal estimation of the effect of both drugs in infected pregnant women. In newborn
children, the net benefit of pyrimethamine-sulphadiazine should also be formally assessed. These trials will be
implemented in settings where prenatal screening for
Toxoplasma gondii
is currently implemented. Trials should
be carefully designed to allow for translation to other settings and modelling tools like cost-effectiveness analysis
should be used to provide clinicians and founders with the best available evidence to establish recommendations.
