CYSTADANE acts as a methyl donor, providing an alternate remethylation pathway to convert excess homocysteine to methionine in patients with homocystinuria.1,2,3,4,5
RANGE OF PERCENT DECREASE IN PLASMA CONCENTRATIONS1
Clinical Study Design
A meta-analysis of published study data* was performed, which included a double-blind, placebo controlled, crossover study of 6 patients and 16 additional case studies1:
*No formal statistical analyses were performed.
*Homocystine is formed nonenzymatically from two molecules of homocysteine1
CYSTADANE is generally well tolerated.1
Hypermethioninemia and cerebral edema in patients with CBS deficiency were the most serious adverse reactions reported. To keep plasma methionine levels below 1,000 μmol/L, some patients may require dietary modification and, if necessary, a reduction in CYSTADANE dose.1
To determine additional adverse reactions, data was collected through a retrospective survey from 41 physicians who had treated 111 patients with CYSTADANE. Adverse events were retrospectively recalled and were not collected systematically.1
Most common adverse reactions (>2%) were nausea and gastrointestinal distress.1
In patients with low plasma methionine concentrations due to MTHFR deficiency or cbl defect, CYSTADANE has been demonstrated to increase plasma methionine and S-adenosylmethionine (SAM) concentrations.1
Patients have taken CYSTADANE for many years without evidence of tolerance. There has been no demonstrated correlation between CYSTADANE concentrations and homocysteine concentrations.1
Recommended dosage: 6 grams per day in divided doses of 3 grams twice daily1
Recommended starting dosage: 100 mg/kg/day divided in twice daily doses, and then increased weekly by 50 mg/kg increments1
Therapy with CYSTADANE should be directed by physicians knowledgeable in the management of patients with genetic disorders that cause homocystinuria1
CYSTADANE is available in plastic bottles containing 180 grams of betaine anhydrous. Each bottle comes with a plastic child-resistant cap and a polypropylene measuring scoop. One level 1.7 mL scoop equals 1 gram of betaine anhydrous powder.1
CYSTADANE should only be taken as directed.1
Shake the bottle of CYSTADANE lightly, then remove the cap.1
Use the scoop provided to measure the number of scoops as prescribed by your doctor.* One level scoop equals 1 gram of powdered betaine.1
Mix the powder with 4 to 6 ounces (120 to 180 mL) of water, juice, milk, or formula until completely dissolved, or mix the powder with food.1
Immediately eat or drink the CYSTADANE mixture.1
Replace the bottle cap tightly to protect CYSTADANE from moisture.1
Store the bottle at room temperature.1
*Over time the doctor may adjust the amount of CYSTADANE taken based on tests of blood homocysteine levels and other factors.1
Downloadable information for your patients and families to learn more about the genetic disorders that cause homocystinuria and instructions for how to take CYSTADANE.
The following educational and advocacy groups provide information about rare genetic disorders, including the different types of homocystinuria. You can explore these groups online.
HCU Network America
The mission of HCU Network America is to help people with homocystinuria (HCU) and related disorders manage their disease and to find a cure.
Organic Acidemia Association
The Organic Acidemia Association is a patient advocacy organization that provides support and information for people with inherited metabolic disorders. Homocystinuria caused by several cobalamin defects—cblC, cblD, cblF, cblJ, and cblX—is included as a part of the group’s advocacy activities.
NORD - National Organization for Rare Disorders
NORD is patient advocacy organization dedicated to helping people with rare diseases. NORD provides information on homocystinuria due to CBS deficiency.
Cystadane is a methylating agent indicated in pediatric and adult patients for the treatment of homocystinuria to decrease elevated homocysteine blood concentrations. Included within the category of homocystinuria are: