Table 3.
US from GD patients at follow-up.
| Patient | Age (y) |
Time on treatment (months) |
Steatosis | Hepatomegaly | Cholelithiasis | BMI | Ferritin (ng/dL) |
MetS during treatmenta | Liver biopsy during treatment |
|---|---|---|---|---|---|---|---|---|---|
| 1A | – | T(7) | – | No | – | 17.4 | 308 | No | No |
| 1B | 20 | I(168) | No | No | No | 28.6 | 485 | No | No |
| 2 | 20 | I(110) T(1) V(81) | Yesb | Yes | Yes | 21.7 | 254 | No | No |
| 3 | – | – | – | – | – | – | – | – | – |
| 4A | – | – | – | No | No | 24.4 | 312.2 | No | No |
| 4B | 36 | I(19) T(1) I(89) | No | Yes | Yes | 25.9 | 57.3 | No | No |
| 5 | 22 | I(177) | No | No | No | 22.7 | 84.7 | No | No |
| 6 | 23 | T(33) | No | No | No | 22.4 | 172.6 | No | No |
| 7 | 17 | I(68) | No | No | No | 19.2 | 885.3 | No | No |
| 8 | 25 | I(156) | Yesb | Yes | No | 20.5 | 508.3 | No | No |
| 9 | 26 | I(112) | No | No | No | 22.7 | 94.6 | No | No |
| 10 | 25 | I(200) | No | Yes | No | 24.7 | 547 | No | No |
| 11A | 26 | I(174) | Yes | Yes | Yes | 18.8 | 242.6 | No | No |
| 11B | 28 | I(146) | No | Yes | No | 27.8 | 285.9 | No | No |
| 12 | 25 | I(126) | No | Yes | No | 23.1 | 546.3 | No | No |
| 13 | 27 | I(120) T(1) I(6) E(41) I(8) E(9) | Noc | No | Yes | 28.8 | 611.6 | No | Yes |
| 14 | 23 | I(189) | No | Yes | No | 24.9 | 619.9 | No | No |
| 15 | 29 | I(216) | Yesb | Yes | No | 26.6 | 311.1 | No | No |
| 16 | 25 | A(31) I(222) | No | No | No | 20.6 | 569.8 | No | No |
| 17 | 29 | I(109) T(2) I(9) T(55) | No | No | No | 30.6 | 325.8 | No | No |
| 18 | 33 | M(7) I(111) | No | Yes | Yes | 25.3 | 278.8 | Yes | No |
| 19A | 45 | I(67) | Yesb | No | Yes | 32 | 66.5 | No | No |
| 19B | 34 | I(71) | No | No | No | 27.8 | 585.3 | No | No |
| 19C | 51 | I(6) T(1) I(80) | Yesb | Yes | No | 28.3 | 1084 | Yes | No |
| 20 | 36 | I(207) | No | No | Yes | 25.6 | 574.2 | No | No |
| 21 | 32 | A(25) I(28) T(3) I(4) | No | No | No | 22.7 | 415.7 | No | No |
| 22A | 36 | I(5) T(65) | No | Yes | No | 26.8 | 463.8 | No | No |
| 22B | 40 | I(22) T(2) I(14) E(70) | Yes | No | No | 30 | 32 | Yes | No |
| 23 | 38 | I(35) | Yesb | No | Yes | 30.7 | 427.2 | No | No |
| 24 | 44 | I(102) T(3) I(29) T(38) | No | Yes | No | 28.7 | 247.9 | No | No |
| 25A | 48 | M(11) T(1) I(55) | Yes | No | Yes | 25.8 | 611.9 | No | Yes |
| 25B | 51 | M(30) T(10) | Yes | No | Yes | 31.9 | 1053 | No | Yes |
| 26A | 52 | I(80) | No | No | No | 24.1 | 480.6 | No | No |
| 26B | 58 | I(76) | Yes | No | No | 32.2 | 1457 | Yes | Yes |
| 26C | 62 | I(66) | No | No | No | 21.6 | 624.8 | No | No |
| 27 | 61 | I(124) T(2) I(8) T(71) | Yes | No | No | 26.6 | 1052 | Yes | No |
| 28 | 62 | I(34) T(2) I(88) | No | No | No | 22.3 | 543.2 | No | Yes |
| 29A | 65 | I(10) T(2) I(9) T(81) | Yes | No | Yes | 34.5 | 670 | Yes | No |
| 29B | 65 | I(19) T(2) I(8) T(14) | Yes | Yes | Yes | 29.7 | 686 | Yes | No |
| 30 | 67 | I(12) M(25) I(47) | No | Yes | No | 30.5 | 1103 | Yes | Yes |
| 31 | – | – | – | – | – | – | – | – | – |
y = years-old; BMI = body mass index; I = imiglucerase; T = taliglucerase alfa; V= = velaglucerase alfa; E = eliglustat; A = alglucerase; M = miglustat.
a
Metabolic syndrome is defined as the presence of at least three of the following: obesity, high triglycerides level, increased blood pressure, and elevated fasting blood glucose (reduced HDL level was not considered as a criterium because it is a feature of GD).
b
Steatosis regressed within two years of US detection.
c
Steatosis at liver biopsy only.