Table 3. Lipid profiles improved in all patients after olipudase alfa treatment.
All five patients demonstrated improvements in both pro- and anti-atherogenic lipid markers. Notably, all patients had elevation of the pro-atherogenic VLDL-C at baseline and subsequent reduction after 26 weeks of olipudase alfa. All patients demonstrated improvement in anti-atherogenic markers after 26 weeks of olipudase alfa. Patients 1 and 2 were on stable doses of lipid lowering therapies (statins) prior to, and during the course of the trial. Therefore, changes observed in these patients during the course of the trial occurred with the addition of enzyme replacement therapy. Red fill indicates values above normal reference ranges; yellow fill indicates values below normal reference ranges.
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|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Patient Number |
Total Cholesterol mmol/L |
LDL-C mmol/L |
VLDL-C mmol/L |
ApoB100 g/L |
Triglycerides mmol/L |
HDL-C mmol/L |
ApoA-I g/L |
|||||||
| Baseline | 26 weeks |
Baseline | 26 weeks |
Baseline | 26 weeks |
Baseline | 26 weeks |
Baseline | 26 weeks |
Baseline | 26 weeks | Baseline | 26 weeks | |
| 1 | 4.70 | 2.30 | 3.38 | 1.34 | 0.88 | 0.23 | 1.41 | 0.54 | 2.20 | 0.80 | 0.32 | 0.59 | 0.64 | 0.70 |
| 2 | 3.83 | 3.78 | 2.59 | 2.72 | 0.88 | 0.49 | 0.96 | 1.01 | 1.55 | 1.16 | 0.36 | 0.57 | 0.64 | 0.85 |
| 3 | 5.26 | 4.22 | 3.32 | 2.93 | 0.98 | 0.21 | 0.92 | 0.80 | 1.14 | 0.89 | 0.96 | 1.09 | 1.30 | 1.32 |
| 4 | 4.66 | 3.55 | 2.69 | 2.33 | 1.66 | 0.80 | 1.32 | 0.90 | 4.35 | 1.73 | 0.31 | 0.41 | 0.60 | 0.71 |
| 5 | 3.63 | 2.98 | 2.25 | 1.74 | 0.80 | 0.62 | 0.59 | 0.58 | 1.38 | 0.73 | 0.57 | 0.91 | 0.90 | 1.00 |
= above normal range
= below normal range
ApoA-I = apolipoprotein A-I; Apo B100 = apolipoprotein B100; HDL=high-density lipoprotein; LDL=low-density lipoprotein; VLDL=very low-density lipoprotein.
Total cholesterol normal range: US <5.18 mmol/L; UK 0-3.9 mmol/L
HDL normal range: US male >0.78; US female >0.907 mmol/L; UK >1.2 mmol/L
LDL normal range: US <3.34 mmol/L; UK 0-2 mmol/L
VLDL normal range: US <0.52 mmol/L; UK 0.09-0.71 mmol/L
Triglycerides normal range: <1.7 mmol/L
Apo B normal range: US male 0.55-1.4 g/L; US female 0.55 −1.25 g/L; UK 0.52-1.09 g/L
ApoA-I normal range: US male 0.94-1.78 g/L; US female 1.01-1.99; UK male 0.87-2.64 g/L