Table 3 Blood Phe levels in treating and monitoring children with PAH deficiency
n (%) | |
|---|---|
Blood Phe level (consistently elevated) to prompt initiation of a Phe-restricted diet (n = 19) | |
≥ 360 μmol/L (≥6 mg/dL) | 14 (74) |
360–420 μmol/L (6–7 mg/dL) | 1 (5) |
≥ 420 μmol/L (≥7 mg/dL) | 1 (5) |
≥ 480 μmol/L (≥8 mg/dL) | 2 (10) |
≥ 600 μmol/L (≥10 mg/dL) | 1 (5) |
Optimal target range of blood Phe, by age | |
0–12 months of age (n = 19) | |
120–360 μmol/L (2–6 mg/dL) | 19 (100) |
> 1–2 years of age (n = 19) | |
120–360 μmol/L (2–6 mg/dL) | 19 (100) |
> 2–10 years of age (n = 19) | |
120–360 μmol/L (2–6 mg/dL) | 19 (100) |
> 10–18 years of age (n = 18) | |
120–360 μmol/L (2–6 mg/dL) | 14 (78) |
120–600 μmol/L (2–10 mg/dL) | 2 (11) |
320–600 μmol/L (5.33–10 mg/dL) | 2 (11) |
Lowest acceptable average level of blood Phe as a long-term treatment goal, by age | |
0–12 months of age (n = 18) | |
100 μmol/L | 1 (6) |
120 μmol/L | 17 (94) |
> 1–2 years of age (n = 17) | |
120 μmol/L | 16 (94) |
150 μmol/L | 1 (6) |
> 2–10 years of age (n = 17) | |
120 μmol/L | 16 (94) |
150 μmol/L | 1 (6) |
> 10–18 years of age (n = 18) | |
100 μmol/L | 1 (6) |
120 μmol/L | 16 (89) |
200 μmol/L | 1 (6) |
Would you be comfortable with a patient’s steady Phe level of < 120 μmol/L (n = 19) | |
Yes | 6 (32) a |
No | 13(68) |
Recommend maintaining higher-end therapeutic range blood Phe and more liberal natural protein intake (n = 19) | |
For most/nearly all patients | 5 (26) |
For some patients | 9 (47) |
Rarely/never | 5 (26) |
Recommend maintaining lower-end therapeutic range blood Phe levels and more restricted natural protein intake (n = 19) | |
For most/nearly all patients | 4 (21) |
For some patients | 4 (21) |
Rarely/never | 11 (58) |