June 1, 2023

Sue Apkon, MD, Chief of Pediatric Rehabilitation Medicine, Children’s Hospital Colorado, discusses a real-world study of patients with Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) who switched from prednisone to deflazacort.

The switch study focused on a group of boys with duchenne and becker muscular dystrophy which is the most common neuromuscular condition in kids one of the standard treatments for this group of kids is the use of corticosteroids and this study we embarked on to better understand the experience of switching from one of the corticosteroids prednisone to another

One called the flasa court to back up corticosteroids have become standard of care um and have been really for well over two decades we know that boys on a corticosteroid will have the ability to walk for longer periods of time so typically in this condition boys lose the ability to walk between 10 and 12 years of age but when they’re on steroids they walk for

A couple to three years longer they maintain upper body strength for longer periods of time and it has positive impacts on the spine breathing and even the heart and so we believe that boys um should be on corticosteroids so the switch study was um really to give us an idea of the real world experience so the experience of physicians throughout the community

So not in a a rigorous clinical trial but the real world experience of people switching from prednisone to deflasic court we wanted to know why um uh children were being switched from the prednisone to the deflas report how they did with that switch and that included their outcomes from a functional standpoint but also from an adverse event standpoint and so

The study is a retrospective chart review we reached out to neurologists in the community who were caring for boys with duchenne or back or muscular dystrophy and we had them review their records we looked at the diagnosis of whether they boy had duchenne or back or muscular dystrophy how when they started corticosteroids how long they had been on prednisone

At what age they switched to the plasmacort and for how long they had been onto flag support and again most importantly how they did with that switch so we had 55 neurologists that completed uh this study and there were 92 boys 60 um two of them had duchenne muscular dystrophy and 30 of them had better muscular dystrophy and better muscular dystrophy is a more

Mild form of duchenne the boys walk longer and in general um have a less aggressive phenotype the reason for the change uh as uh identified through this study was to slow the progression of the disease was one of the main reasons and another main reason that the boys were switched from prednisone to deflatoport was tolerability one thing i didn’t mention and

You may be aware is that uh corticosteroid prednisone um which is the most common one we use can really lead to a lot of side effects weight gain behavioral changes impacting sleep short stature and so the tolerability in our study was one of those factors that peop that that prompted um the switched from prednisone to deflasa court when we looked at uh the

Physician’s perspective on the um the impact of the change uh in general um the physicians or neurologists felt that they were either somewhat or very effective in terms of the switch so that was a really good thing to see we also looked there’s some practices some neurologists who use something called the cgi or the clinical global impression so that means

If i am seeing a child over time how is that child doing um are they uh are they stable in their course are they getting better are they a lot better are they getting worse are they getting a lot worse and so the cgi is a tool that we use to track outcomes over time and so we looked at the cgi results of boys as they were switching from prednisone to flasa court

Um and in all boys on cortical steroids most patients disease either had improved or stabilized on steroids and for those who made the switch to de flasa court there was a shift to the left or a shift to improvement so there was a greater improvement on deflation court compared to those on premise and the side effect profile of those adverse events that we

See and when boys are on prednisone were less obvious when they switched over to deplosment so those are sort of the general results of the study um you know for me as a clinician it was great to see um you know something that i’m seeing uh clinically also that that boys seem to be benefiting from the switch from prednisone to the flask with you know more

Stabilization or improvement and fewer side effects

Transcribed from video
Switching from Prednisone to Deflazacort To Treat Duchenne or Becker Muscular Dystrophy By CheckRare