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#apaperaday: A Mixed-Method Study Exploring Patient-Experienced and Caregiver-Reported Benefits and Side Effects of Corticosteroid Use in Duchenne Muscular Dystrophy

In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: A Mixed-Method Study Exploring Patient-Experienced and Caregiver-Reported Benefits and Side Effects of Corticosteroid Use in Duchenne Muscular Dystrophy

Today’s pick is from Journal of Neuromuscular Diseases by Fischer et al and is selected as a TREAT-NMD Duchenne masterclass which starts today. The paper is on patient and parent perspectives on steroid use. DOI: 10.3233/JND-221617.

Today the Duchenne masterclass will focus on the standards of care and steroid use is part of the Duchenne care standards. The patient perspective is also an important aspect of the masterclass, hence my selection for this paper that reports on a PPMD led study.

The study investigated both the benefits and the side effects of steroid use as perceived by Duchenne patients (teens/adults) and caregivers through interviews (28) and a survey (236 participants). Almost all patients were on chronic steroid use.

The paper contains a LOT of data so I recommend you to read it if you are interested. Some highlights: most mentioned benefits by parents and patients (both ambulatory and non ambulatory) were improved heart and breathing. Ambulatory patients and parents also mentioned longer walking, using of arms, independence and improved balance and less fatigue. The walking aspects were also mentioned by non-ambulatory patients and parents, but they focused more on using arms.

For the side effects weight gain, behavior issues, bone health (and increased fracture risks) and physical appearance (looking young and being short) were mentioned most by parents and patients. Patients also mentioned trouble sleeping.

Risks due to side effects patients and parents worried about were increased fracture risk, developing diabetes, being unable tot manage the behavior and the consequences of weight gain and cataract. Patients and parents generally felt the benefits outweighed the risks and side effects. There was no difference between net benefit for prednisone and deflazacort. None of the patients regretted taking steroids, while only a few parents did.

A patient voiced it very well “There were little options and steroids are damage control”. Generally parents rated the burden of the side effects higher than the patients. Authors also list limitations of the study: it involved small numbers and relied on self reports.

Furthermore, the challenge is that parents and patients do not know how the disease trajectory would have been had they not taken steroids. This was also mentioned by many in the interview.

A nice study that should help clinicians and families who are currently in the stage of starting steroid treatment. The fact that none of the patients regretted taking steroids and that they perceive the benefits outweigh the side effects is very important information.