Background Treatment options for Duchenne muscular dystrophy remain limited, although consensus treatment guidelines recommend corticosteroid use. Methods This retrospective analysis assessed corticosteroid use in ambulatory and nonambulatory US males with Duchenne, age 35 and under, or Becker muscular dystrophy, who enrolled in The Duchenne Registry from 2007 to 2016 (formerly DuchenneConnect). Results The mean (SD) age of corticosteroid use initiation was 5.9 (2.5) years, and deflazacort use (54%) was slightly more common than prednisone/prednisolone (46%). Among all responses from those with Duchenne, 63% were currently on corticosteroids, 12% were no longer on corticosteroids, and 25% had never been on corticosteroids. Among those who were nonambulatory, 49% were currently on corticosteroids, 28% had discontinued corticosteroids, and 23% had never used corticosteroids. Primary reasons for never initiating therapy were that corticosteroids were not prescribed or recommended and concerns about side effects. Corticosteroid use was maximal at age 8 (84% on corticosteroids) and gradually declined from age 10 to 19. The primary reasons for corticosteroid discontinuation were problems with side effects (65%) or not enough benefit (28%). Average doses of corticosteroids were below recommended doses. In the 159 responses with Becker muscular dystrophy, 20% were currently using corticosteroids. Conclusions Recognizing the self-selected nature of participation, it appears that a considerable proportion of US participants registered with The Duchenne Registry were either not on corticosteroids or not on recommended doses despite consensus recommendations. Side effects were a consideration in initiating and discontinuing treatment. These data reinforce the need for additional treatment options for those affected by Duchenne.

The 79 female and 147 male patients constituting the population with cystic fibrosis (CF) aged 16 years and older attending The Hospital for Sick Children were asked to complete the Cornell Medical Index (CMI) and Tennessee Self-Concept Scale (TSCS); 64 female (81%) and 112 male (76%) subjects participated. Analysis of CMI results showed 43% of female subjects to have moderate to severe emotional disturbance compared to 19% of male subjects. This female : male ratio for severity of emotional disturbance is found in ostensibly healthy groups, but the percentages of disturbance approach values for medical patient populations. The frequency of emotional disability is greater in those more than 20 than in those 16-19 years old. The TSCS results portray a generally normal self-concept except for scores of positive physical self and psychosis for patients aged 20 years and older; these scores approach psychiatric values, suggesting that some reality distortion facilitates emotional adjustment to adult life with CF. The TSCS and CMI results correlate significantly, indicating a connection between self-concept and emotional status. However, TSCS and CMI scores do not correlate with measures of disease severity except for correlations between lung function and physical self-concept in older male patients. These results suggest that psychologic functioning is independent of the degree of physical impairment in older patients with CF, with the long-surviving male patients more realistically appraising the limitations their disease imposes and utilizing denial and minimization to a lesser degree. Demographic data on the clinic population reveal that most patients aged 16 years and older cope with their intellectual, developmental, and socioeconomic tasks commensurate with normal age expectations.

Eighty-five 12- to 15-year-old adolescents regularly attending the cystic fibrosis (CF) clinic of The Hospital for Sick Children were asked to complete the Children's Health Locus of Control and the Tennessee Self Concept Scale. Their parents were requested to complete the Child Behavior Checklist. Thirty-four males (72%) and 28 females (74%) participated in the study. This study found that adolescents with CF are able to maintain a good self concept, be socially competent, and perceive that they are in control of their health while showing an increase in behavior problems. Females rely heavily on denial and are more behaviorally compliant, whereas boys use less denial but show more behavior problems. Males appear to integrate having a physical disorder into their self concept, whereas females do not. The findings demonstrate a difference in mechanisms of coping with cystic fibrosis between male and female adolescents with CF, which may contribute to the decline in physical status in females and better survival of males.