Article

AIM: To explore variation in clinical course and steroid treatment in Duchenne
muscular dystrophy (DMD) by ethnic origin and socio-economic status. METHOD: In
this longitudinal cohort study, clinical outcome was defined as age at loss of
ambulation (LOA). Ages are presented as months for accurate calculation. Steroid
use was reviewed against national guidelines. Kaplan-Meier survival analysis was
used to determine probabilities over time of LOA. Log-rank test was used to
evaluate comparisons between ethnic and socio-economic groups. RESULTS: From 2005
to 2014, 71 children were newly diagnosed with DMD. Complete data were available
on 69, including 33 of white British heritage and 23 of South Asian heritage.
Mean age at diagnosis (without known family history) was 45.7 months; white
British ethnicity 42.1 months (range 14-86mo), South Asian ethnicity 50.2 months
(range 5-98mo). Twenty-four males lost ambulation. Those of South Asian heritage
lost ambulation earlier (mean LOA 105.8mo [8y 10mo]) than those of white British
heritage (mean LOA 117.8mo [9y 10mo]): log-rank test score 0.012 (p<0.05). Those
most deprived did worse: mean age at LOA 130.0 months (10y 10mo) for the top 20
per cent and 102.5 months (8y 6mo) in the lower 20 per cent: log-rank test score
0.035 (p<0.05). The most socially deprived were diagnosed earlier and started
steroids earlier. Of those of South Asian heritage, 18 per cent declined
steroids, compared with 9 per cent of white British heritage. Also, 44 per cent
of those of South Asian heritage stopped steroids compared with 17 per cent of
those of white British heritage. INTERPRETATION: Patients from South Asian and
deprived backgrounds had earlier LOA. Genetic disease modifiers are likely to be
implicated, but social and cultural factors influence access to treatment.
CI - (c) 2017 Mac Keith Press.

Langue : ANGLAIS