Diagnosis, quality of life, and treatment of patients with Hunter syndrome in the French healthcare system: a retrospective observational study

Table 4 CGI-I results for patients with severe and attenuated phenotypes and for the total population for 2 time periods

	Severe (n = 33)			Attenuated (n = 16)			Total population (N = 52) ^e
Period 1: From baseline (ERT onset) versus 1 y later	n (%)	Mean age at start of ERT (y)	Mean age at diagnosis (y)	n(%)	Mean age at start of ERT (y)	Mean age at diagnosis (y)		N (%)
Significant improvement^a	10 (30.3)	4.0	2.6	7 (43.8)	11.1	4.4	Improvement^f	44 (84.6)
No change/little improvement or worsening^b	21 (63.6)	6.8	2.4	9 (56.2)	19.5	6.5	No change	4 (7.7)
Significant worsening^c	2 (6.1)	6.9	2.7	0 (0)	–	–	Worsening^g	2 (3.8)
P value^d		0.1181	0.9448		0.1248	0.2235
Period 2: From end of first year ERT versus present	n (%)	Mean age at start of ERT (y)	Mean age at diagnosis (y)	n (%)	Mean age at start of ERT (y)	Mean age at diagnosis (y)
Significant improvement^a	3 (9.1)	3.1	2.1	7 (43.8)	11.0	4.9	Improvement^f	24 (46.2)
No change/little improvement or worsening^b	17 (51.5)	5.9	2.7	7 (43.8)	21.6	6.8	No change	8 (15.4)
Significant worsening^c	13 (39.4)	6.2	1.7	2 (12.5)	12.6	3.7	Worsening^g	18 (34.6)
P value^d		0.2619	0.0638		0.1710	0.6233

^a“Very much improved” and “much improved”.
^b“Minimally improved”, “no change”, and “minimally worse”.
^c“Much worse” and “very much worse”.
^dWilcoxon test.
^eTwo patients in the total population were not evaluable each during Period 1 and Period 2.
^f“Very much improved”, “much improved”, and “minimally improved”.
^g“Minimally worse”, “much worse”, “very much worse”.

ISSN: 1750-1172