Vamshi Rao, MD, attending physician of Neurology at Ann and Robert H. Lurie Children’s Hospital of Chicago discusses how the pandemic has impacted access to spinal muscular atrophy (SMA) treatments.
Vamshi Rao, MD, is an attending physician of Neurology at Ann and Robert H. Lurie Children’s Hospital of Chicago and assistant professor of Pediatrics (Neurology and Epilepsy) at Northwestern University Feinberg School of Medicine.
Has the COVID-19 [coronavirus disease 2019] pandemic impacted access to either newborn screening for spinal muscular atrophy [SMA] type 1 or to treatments?
Rao: We've been very fortunate that COVID-19 overall has not impacted the access to treatments and newborn screening. I can speak for the state of Illinois because that is where I practice. Newborn screening was introduced as a pilot somewhere between May and July 2020 and rapidly took over as a permanent program after that. All of the children that are being born in the state of Illinois are definitely going through newborn screening and COVID-19 has not affected that. As far as access to treatment, access to treatment at a particular institution for a therapy that requires children to come to the hospital, such as Spinraza [nusinersen] that is given through lumbar puncture, or on a *17 hour power glycosyltransferase* that is given through IV [intravenous injection] requires children to come to the hospital.
Guidelines that required COVID-19 precautions were in place in most people places, and guidelines have to be practiced. But at the same time, a very risk-benefit analysis was done in terms of exposure vs children getting treatment and I'm happy to say that, at least at our institution, as we really looked at these guidelines [with] a committee that was formed to assess whether children were going to come in for their regular treatments, and clinical trials, it was found that the risk of not getting the medication vs the risk of getting COVID was definitely in favor of decline with the underlying estimate if they did not get their medication. So all these children were allowed to have their disease modifying therapies given to them at our hospital.
I definitely think that access was affected at some institutions and I do think access was affected around the country, in places where a lot of travel was involved in whether kids were coming from rural communities, or coming from very far off, mountainous regions to their nearest center that was giving them these drugs. Overall, I do think there were places that where access was affected, but as an SMA community, our goal was to always administer the drug despite all odds, of course, keeping all the safety and precautions in place.