Treatment should be initiated and monitored by physicians who are experienced in the management of patients with rare genetic disorders such as MCT8 deficiency. Posology Dosing of Emcitate should be titrated on an individual patient basis based on the patient’s thyroid hormone levels.
The dose should be increased stepwise approximately every two weeks during a titration period until a maintenance dose has been reached. It is generally recommended to titrate the dose until the serum T3 level is below the midpoint of the normal range for age.
The dose may be further adjusted based on the patient’s response to treatment on clinical features of MCT8 deficiency. 4). TSH and (F)T4 levels may provide further information to guide individual dosing. 3 Adults, adolescents, children, and infants with a body weight of 10 kg or above Dose titration and adjustment The recommended starting dose for patients with a body weight of 10 kg or above is 350 micrograms daily.
A recommended dose titration regimen is shown in Table 1. The daily dose should be gradually increased by 350 micrograms every two weeks until a maintenance dose has been reached. It is generally recommended to titrate the dose until the serum T3 level is below the midpoint of the normal range for age.
Smaller dose escalation steps (half tablets) may be used when a patient is approaching target serum T3 levels, as appropriate. The dose may be further adjusted based on the patient’s response to treatment on clinical features of MCT8 deficiency.
g. morning, midday, evening). Table 1. Recommended dose titration regimen in patients with a body weight of 10 kg or above Titration Total daily dose (micrograms) Number of tablets/day Starting dose 350 1 Week 2 700 2 Week 4 1 050 3 Week 6 1 400 4 Week 8 1 750 5 Week 10 2 100 6 Dose titration should continue in increments of 350 micrograms until a maintenance dose has been reached.
It is not recommended to exceed a daily dose of 80 micrograms/kg in patients with a body weight between 10 and 40 kg; 60 micrograms/kg in patients with a body weight between 40 and 60 kg; and 50 micrograms/kg in patients with a body weight above 60 kg.
Children and infants with a body weight below 10 kg Dose titration and adjustment The recommended starting dose for patients with a body weight below 10 kg is 175 micrograms (a half tablet) daily. A recommended dose titration regimen is shown in Table 2.
The daily dose should be gradually increased by 175 micrograms every two weeks until a maintenance dose has been reached. It is generally recommended to titrate the dose until the serum T3 level is below the midpoint of the normal range for age.
The dose may be further adjusted based on the patient’s response to treatment on clinical features of MCT8 deficiency. g. morning, midday, evening). Table 2. 5 Week 10 1 050 3 Dose titration should continue in increments of 175 micrograms until a maintenance dose has been reached.
It is not recommended to exceed a daily dose of 100 micrograms/kg in patients with a body weight below 10 kg. 4 Maintenance dose The dose of Emcitate is titrated on an individual basis until a maintenance dose has been reached. It is generally recommended to titrate the dose until the serum T3 level is below the midpoint of the normal range for age.
The dose may be further adjusted based on the patient’s response to treatment on clinical features of MCT8 deficiency. 4). Missed or delayed dose If a dose is missed and there are more than 4 hours to the next scheduled dose, it should be taken as soon as possible.
If a dose is missed and the next dose is scheduled within 4 hours, the dose should be omitted and the next dose taken according to the regular schedule. Laboratory tests (T3 measurements) It is recommended to measure individual T3 levels using a liquid chromatography tandem mass spectrometry (LC/MS/MS) method.
Tiratricol cross-reacts with T3 when assessed by immunoassay, which may cause unreliable test results. 4). Hypermetabolic signs and symptoms If hypermetabolic signs and symptoms (such as hyperhidrosis, irritability, anxiety, insomnia, nightmares, hyperthermia, tachycardia, transient elevations in systolic blood pressure (SBP), or diarrhoea) either occur for the first time or worsen, and do not resolve within 2 weeks, the dose should be reduced according to the steps in the dose titration regimen until signs and symptoms resolve (see Table 1 or Table 2).
4). Special populations Hepatic impairment No specific studies have been performed in patients with hepatic impairment. 4). Renal impairment No specific studies have been performed in patients with renal impairment. 4). Method of administration For oral use or administration through gastroenteral feeding tube.
Oral use Emcitate dispersible tablets are […]