Continuing corticosteroid treatment into adulthood was associated with slower lung-function decline in people with Duchenne muscular dystrophy (DMD), according to a new study.
“The results of this study provide further evidence to support continuing [corticosteroid] treatment into late adolescence and adulthood” for people with DMD, the researchers wrote.
The study, “The Effect of Corticosteroids on Pulmonary Function in a Large Cohort of Late Adolescents and Adults With Duchenne Muscular Dystrophy,” was published in Muscle & Nerve.
Steroid benefits in adults with DMD remain uncertain
DMD is a genetic disorder that causes muscles to become damaged over time. As people with DMD move through adolescence and into adulthood, weakness in the muscles used for breathing can worsen, causing breathing difficulties and leading some people to need ventilation to support their breathing.
Corticosteroids are anti-inflammatory medications shown to help slow disease progression and preserve muscle and lung function in people with DMD. They are therefore routinely given to children with the disorder. However, evidence supporting their continued use into adulthood is limited, and treatment becomes less common with age, possibly reflecting uncertainty about the balance of benefits and risks in older patients.
Scientists in the U.K. wanted to determine whether continued corticosteroid use was associated with better lung function in older teens and adults. They reviewed medical records from three adult DMD centers and compared outcomes among 208 males, ages 16 to 40.5. Of these, 113 were receiving corticosteroids at the time of the study, 43 had used them for more than one year but stopped before moving to adult care, and 52 had never used corticosteroids or had used them for no more than one year.
The researchers found that patients who continued taking corticosteroids started noninvasive ventilation (NIV) at an older median age. NIV is breathing support delivered through a mask rather than a surgically placed airway. The median age at NIV initiation was 19.17 years in the continued-treatment group, compared with 16 years in the group that stopped treatment and 17.33 years in the group with minimal or no prior corticosteroid use. The overall difference among the groups was statistically significant.
The continued-treatment group also lost the ability to walk at a median age about two years later than the other groups: 12.67 years, compared with 10.67 years in the group that stopped treatment and 10 years in the group with minimal or no prior use. This difference was statistically significant. At the last assessment, five people, or 4.6%, in the continued-treatment group could still walk; no one in the other two groups remained ambulatory.
Continued treatment linked to better lung function
Other measures showed a similar pattern. Patients who continued corticosteroids generally reached key lung-function milestones at older ages, and their breathing function was better preserved at a given age than in the other groups. Each additional year of preserved ability to walk was associated with better lung function.
“Our data show better preserved respiratory function and a significantly older age at NIV initiation in individuals who continued [corticosteroids] compared with those who either stopped or were [corticosteroid]-naïve,” the researchers concluded.
Dose information was available for 36 of the 113 people who continued corticosteroids, and all 36 were receiving doses below recommended levels. This suggests “that continuing [corticosteroids] at lower-than-recommended doses may still positively influence [long-term lung function] outcomes in late adolescence and adulthood,” they wrote.
Although the findings suggest that continuing corticosteroids may help preserve lung function, the study was retrospective and uncontrolled, so it cannot prove that continued treatment caused the better outcomes. It also could not assess the long-term adverse effects of corticosteroid use in adulthood. As such, the researchers noted that “important questions remain, including when the risks of continued treatment outweigh the benefits, and which [corticosteroid] type, dose, or regimen optimally balances efficacy and safety as muscle function declines.”
“Until further research provides guidance, clinicians must make individualized decisions, weighing potential benefits against adverse effects in the context of declining muscle function, and exercise informed judgment when considering [corticosteroid] withdrawal,” the scientists wrote.
The post Continued steroid use tied to slower lung decline in adults with DMD appeared first on Muscular Dystrophy News.