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Table 1 Demographics and baseline characteristics of individual patients

From: Early treatment with laronidase improves clinical outcomes in patients with attenuated MPS I: a retrospective case series analysis of nine sibships

Sibship ID Pt ID Sex Genotype Age at diagnosis (yr) Age at ERT Initiation (T1) (yr) Duration of ERT at T2 (yr)
Group Aa
1 OS F P533R/P533R 8 8.8 5.4
1 YS M P533R/P533R 0.2 0.3 4.5
2 OS M P533R/P533R 1.3 3.3 11.7
2 YS F P533R/P533R Birth 0.4 9.6
3 OS F NA 1 12 8
3 YS F NA 0.4 0.7 8.3
4 OS M G208D/P520R 3.6 5.5 3.5
4 YS M G208D/P520R 0.8 1.3 3.6
5a OS F L490P/L490P 3 5.8 8.6
5a YS1 M L490P/L490P 0.1   0.3   7.6  
  Group A Median (range)
  OS YS OS YS OS YS
N = 5c N = 5 N = 5c N = 5 N = 5c N = 5
3.0 0.2 5.8 0.4 8.0 7.6
(1, 8) (0, 0.8) (3.3, 12) (0.3, 1.3) (3.5, 11.7) (3.6, 9.6)
Group Bb
5bc OS F L490P/L490P 3 5.8 8.6
5b YS2 F L490P/L490P 0.1 2.5 8.8
6 OS1 M NA 9 13 6
6 OS2 M NA 8 11 6
6 YS M NA 0.6 3 7
7 OS F P533R/L564fsx 4.3 4.5 5.5
7 YS F P533R/L564fsx 3.3 3.4 5.6
8 OS M NA 6.8 7 6.0
8 YS M NA 3.3 3.7 5.8
9 OS F NA 7 14 7
9 YS F NA 6.7 8.6 6.4
  Group B Median (range)
  OS YS OS YS OS YS
N = 6 N = 5 N = 6 N = 5 N = 6 N = 5
6.9 3.3 9.0 3.4 6.0 6.4
(3, 9) (0.1, 6.7) (4.5, 14) (2.5, 8.6) (5.5, 8.6) (5.6, 8.8)
  1. F female, M male, NA not available, OS older sibling, YS younger sibling
  2. aGroup A: younger siblings ERT onset ≤1 year of age
  3. bGroup B: younger siblings ERT onset between 2 and 4 years (except #9 at 8.6 year)
  4. cThe older sibling in sibship 5 is listed twice (once in group A and once in Group B) since one of the two younger siblings initiated ERT at 0.3 years of age (sibling 2a, Group A) and the other at 2.5 years of age (sibling 2b, Group B), respectively