Version May 2024
Homocystinuria: Oral: 3 g twice daily. Dosages of up to 20 g/day have been necessary to control homocysteine levels in some patients.
Note: Dosage in all patients can be gradually increased until plasma total homocysteine is undetectable or present only in small amounts. One in vitro study indicated minimal benefit from exceeding a twice daily dosing schedule and a 150 mg/kg/day dosage.
There are no dosage adjustments provided in the manufacturer’s labeling.
There are no dosage adjustments provided in the manufacturer’s labeling.
Refer to adult dosing.
(For additional information see "Betaine (anhydrous, not equivalent to betaine hydrochloride): Pediatric drug information")
Homocystinuria: Note: The overall role of betaine in management of homocystinuria and effectiveness is variable and dependent upon several factors, including specific enzyme deficiency, genetic mutation(s), and clinical condition; dose should be individualized.
Infants and Children <3 years: Oral: 50 mg/kg/dose twice daily; increase at weekly intervals in 50 mg/kg/day increments until plasma total homocysteine is undetectable or present in small amounts. Note: Minimal additional benefit has been observed with dosages >150 to 200 mg/kg/day or exceeding a twice daily dosing schedule (Morris 2017); however, case reports in infants suggest more frequent dosing may be necessary in some patients (Schiff 2011; Ucar 2010).
Children ≥3 years and Adolescents: Oral: 3,000 mg twice daily; increase gradually until plasma total homocysteine is undetectable or present in small amounts; in some patients, doses up to 20 g/day have been needed to control homocysteine plasma concentrations. Note: Minimal benefit has been observed with dosages >20 g/day or exceeding a twice daily dosing schedule.
There are no dosage adjustments provided in the manufacturer’s labeling.
There are no dosage adjustments provided in the manufacturer’s labeling.
The following adverse drug reactions are derived from product labeling unless otherwise specified.
Frequency not defined:
Dermatologic: Abnormal skin odor
Gastrointestinal: Diarrhea, dysgeusia, gastrointestinal distress, nausea
Nervous system: Psychological disorder
Postmarketing:
Dermatologic: Alopecia, urticaria
Gastrointestinal: Anorexia, glossitis, stomach discomfort, vomiting
Genitourinary: Urinary incontinence
Nervous system: Agitation, brain edema (associated with hypermethioninemia) depression, irritability, personality disorder, sleep disorder
There are no contraindications listed in the manufacturer’s labeling.
Special populations:
• Cystathionine beta-synthase (CBS) deficiency: Use caution in patients with CBS deficiency; treatment with betaine may cause large increases of plasma methionine concentrations which may cause cerebral edema.
Dosage form specific issues:
• Appropriate use: Betaine anhydrous, a prescription medication indicated for the treatment of homocystinuria and betaine hydrochloride, a nutritional supplement, are not to be used interchangeably (ISMP 2016).
Excipient information presented when available (limited, particularly for generics); consult specific product labeling.
Powder, Oral, as anhydrous:
Cystadane: 1 g/scoop (180 g)
Generic: 1 g/scoop (180 g)
Tablet, Oral, as hydrochloride:
Generic: 300 mg
Yes
Ordering information is available at https://www.cystadane.com/physicians/prescribing-cystadane/.
For oral administration. Administer without regard to food immediately after reconstitution.
Oral: Administer without regard to food immediately after reconstitution; do not use if powder does not completely dissolve or gives a colored solution.
Homocystinuria: Treatment of homocystinuria including deficiencies or defects in cystathionine beta-synthase (CBS), 5,10-methylene tetrahydrofolate reductase (MTHFR), and cobalamin cofactor metabolism (CBL).
Betaine anhydrous may be confused with betaine hydrochloride (a nutritional supplement)
Betaine may be confused with Betadine
Cystadane may be confused with cysteamine, cysteine
None known.
There are no known significant interactions.
The risk of adverse pregnancy outcomes may be increased in females with untreated homocystinuria (Langendonk 2012; Levin 2016; Levy 2002). Although betaine is an endogenous substance, information related to the use of betaine supplementation for treating homocystinuria in pregnancy is limited (Langendonk 2012; Levy 2002; Liu 2015; Pierre 2006; Stabler 2017; Vilaseca 2004; Wilcken 1997; Yap 2001). In general, females with inherited metabolic disease should achieve adequate metabolic control prior to conception (Langendonk 2012).
It is not known if betaine is present in breast milk.
According to the manufacturer, the decision to breastfeed during therapy should consider the risk of infant exposure, the benefits of breastfeeding to the infant, and benefits of treatment to the mother. Case reports related to the use of breastfeeding during betaine therapy are limited (Liu 2015). In general, females with inherited metabolic disease should ensure adequate energy intake if breastfeeding (Langendonk 2012).
May be mixed with water, juice, milk, formula, or with food. Betaine is a metabolite of choline and is present in small amounts in foods such as beets, spinach, cereals, and seafood.
Total plasma homocysteine levels to determine therapeutic response. In patients with elevated plasma methionine (eg, CBS deficiency), monitor plasma methionine (maintain <1000 micromol/L).
Betaine is an endogenous metabolite of choline. Betaine acts as a methyl group donor in the remethylation of homocysteine to methionine. Homocystinuria is an inborn error of metabolism in which elevated plasma homocysteine levels can lead to intellectual disability, ocular abnormalities, osteoporosis, premature atherosclerosis and thromboembolic disease. Remethylation is one of the two divergent pathways in the metabolism of homocysteine. The second pathway involves transulfuration of homocysteine to produce cysteine. A number of enzymes and cofactors are also involved in these pathways.
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