Table 3.

DS3 score,19 ERT, and dosages at times of renal biopsies

Patient No.Age (yr)/SexTime (yr)DS3 ScoreERTDuration of ERT (mo)Cumulative Enzyme Dose (mg/kg)
PNSRenalCardiacCNSClinical
17/M01210013Beta, 1.0 mg/kg EOW 0–5 yr0
54000465130
211/M01210013Alfa, 0.2 mg/kg EOW 0–5 yr016
311000114027.6
56000669
311/F01010112Beta, 1.0 mg/kg EOW 0–5 yr0
51001260120
412/M060006Alfa, 0.2 mg/kg EOW 0–5 yr026
51000165
516/M−21200012Alfa, 0.2 mg/kg EOW −2 to 0 yr2610.4
0a1115017Alfa, 0.4 mg/kg EOW 0–4 yr4121.6
140509Beta, 1.0 mg/kg EOW 4–5 yr9177.2
54013017(66.8 )
616/M01200012Alfa, 0.2 mg/kg EOW 0–5 yr0
3600064016
5600066526
717/M−21200113Alfa, 0.2 mg/kg EOW −2 to 0y2610.4
0a1100112Alfa, 0.4 mg/kg EOW 0–5 yr4121.6
1600179464
561018(53.6 )
817/M01200012Alfa, 0.2 mg EOW 0–1 yr0
11200012135.2
918/M01210114Beta, 1.0 mg EOW 0–5 yr0
19101111326
591011166132
1023/M01210012Beta, 0.2 mg EOW 0–5 yr0
141004135.2
5410046425.6
1130/M01250017Alfa, 0.2 mg EOW 0–5 yr0
145009135.2
5450096526
1233/M−31207019Alfa, 0.2 mg EOW −3 to 1.5 yr4024.8
0b417012Alfa, 0.2 mg per wk −1.5 to 5 yr9972
5407011(47.2 )
  • Cumulative dose is calculated from initiation of ERT. The cumulative dose since baseline biopsy is given in parentheses (patients 5, 7, 12). Time, time in years related to baseline biopsy (0); PNS, peripheral nervous system; M, male; beta: agalsidase beta; Alfa, agalsidase alfa; EOW, every other week; F, female.

  • a Two years of ERT before baseline.

  • b Three years of ERT before baseline.