Genotype characterization and delayed loss of ambulation by glucocorticoids in a large cohort of patients with Duchenne muscular dystrophy

Table 3 Effects of glucocorticoid treatment on median age at loss of ambulation in the different genotypes

DMD mutation cox	GC treatment for less than 1 month or never			Continuous GC treatment for 12 months or longer
DMD mutation cox	Total No. of participants (No. with LOA)	Median age, years, at LOA (95% CI)	HR ^a	Total No. of participant (No. with LOA)	Median age, years, at LOA (95% CI)	p Value	HR (95% CI)
Other deletions	139 (36)	10.530 (10.177–10.883)	1	142 (46)	12.090 (11.642–12.538)	0.0003^b	0.436 (0.277–0.685)
Nonsense mutations	73 (12)	10.900 (9.293–12.507)	1	84 (19)	13.290 (11.482–15.098)	0.024^b	0.418 (0.196–0.891)
Exon 44 amenable skipping	32 (6)	11.580 (9.189–13.971)	1	36 (9)	13.650 (12.987–14.313)	0.003^b	0.155 (0.045–0.535)
Exon 45 amenable skipping	42 (13)	10.100 (8.883–11.317)	1	46 (14)	11.710 (9.292–14.128)	0.007^b	0.327 (0.145–0.739)
Exon 51 amenable skipping	65 (11)	10.010 (9.372–10.648)	1	63 (18)	11.150 (9.82–12.318)	0.032^b	0.419 (0.189–0.927)
Exon 53 amenable skipping	51 (14)	10.000 (9.221–10.779)	1	81 (21)	11.510 (10.514–12.506)	0.001^b	0.266 (0.125–0.566)

CI confidence interval, DMD dystrophin gene, GC glucocorticoid corticosteroid, HR hazard ratio, LOA loss of ambulation
^aAn HR of 1 is assigned to the GC treatment for less than 1 month or never, as it is used as a reference in the Cox regression model
^bSignificant (the p value represents the comparison of GC-treated versus GC-untreated patients in each row)

ISSN: 1750-1172