To get started, know your cystine levels
To determine your THIOLA dosage your doctor may perform a 24-hour urine test. The dosage should be based on the amount of THIOLA required to bring your cystine levels below the line of solubility (generally <250 mg/L).
The recommended starting dosage is different for children and adults.
may begin at 800 mg/day
≥9 years of age:
may begin at 15 mg/kg/day
Your doctor may start you on this recommended dosage, but will adjust as needed based on the amount of THIOLA necessary to reduce your cystine levels.
Did you know?
In a clinical trial, the average dosage of THIOLA per person was approximately 1000 mg/day. Talk to your doctor to make sure your starting dosage is right for you.
How to take THIOLA
Your total THIOLA dose should be divided into smaller doses and taken 3 times per day at least 1 hour before or 2 hours after meals.
The example below shows how a total THIOLA dose might be divided.
Morning (at waking)
Night (before bed)
Always take THIOLA at least 1 hour before or 2 hours after meals, as food can affect how THIOLA is absorbed in your body, potentially lessening the effect of THIOLA.
What to expect once you start THIOLA
For best results, it is important to remember that you should continue making therapeutic lifestyle changes—including drinking more water, reducing your protein and sodium intake, and taking medications to adjust the pH of your urine—while taking THIOLA. Once you start treatment, your doctor will schedule regular visits and ask you to perform 24-hour urine collections to monitor your cystine levels.
After being on THIOLA for 1 month, your doctor will ask you to perform a 24-hour urine test to evaluate your cystine levels and determine necessary dosing adjustments.
Your doctor should ask you to perform a 24-hour urine test every 3 months thereafter to continue monitoring your cystine levels.
As your cystine levels change, your doctor may adjust your THIOLA dose to meet your treatment goal of reducing cystine below the line of solubility (generally <250 mg/L).
Lindsay’s journey to stone-freea
Lindsay, age 23, was able to move from a cystinuria diagnosis to stone-free in less than 1 year by working closely with her doctor and following her management plan, which included being diligent about drinking more fluids, regularly monitoring her urinary cystine level, and finding the right dosage of THIOLA.
Are you ready to become stone-free? Watch Lindsay’s story to learn how she did.
aIndividual patient response. Not all patients will respond similarly to the same medical treatment.
For more information on Lindsay’s journey, as well as cystinuria management tips and insights, download this guide. If you have questions about your own journey to stone-free, bring this guide to your next doctor’s appointment to get the conversation started.
THIOLA® (tiopronin) tablets are a prescription medicine used to help prevent the formation of cystine (kidney) stones in patients who were not successfully treated with dietary changes and increased fluid intake, or patients who have had side effects with the drug d-penicillamine.
Important Safety Information
THIOLA is not for everyone. You should not take THIOLA if you:
- Are pregnant or plan to become pregnant
- Are breastfeeding or plan to breastfeed
- Have a history of blood disorders including aplastic anemia (your bone marrow does not make enough new cells), agranulocytosis (decrease in certain white blood cells), or thrombocytopenia (decrease in the number of platelets)
The safety and effectiveness of THIOLA have not been established in children under 9 years of age, and there are no well-controlled studies in pregnant women. High doses of THIOLA in pregnant laboratory animals have been shown to harm the fetus, so you should talk about these risks with your doctor to determine whether THIOLA is right for you. No long-term animal studies have been performed to see whether THIOLA can cause cancer, so you should discuss these risks with your doctor.
While you are taking THIOLA, your doctor will monitor you closely for signs and symptoms of possible complications. Your doctor will routinely do certain blood and urine tests, and yearly scans of your abdomen to look at the size and appearance of kidney stones. THIOLA should only be used under the close supervision of your doctor.
THIOLA can cause serious side effects or potential complications, and some of these could be fatal. Therefore, it is important to call your doctor right away if you have any side effects.
Side effects associated with THIOLA include a drug-related fever that typically occurs during the first month of treatment. If this occurs, talk with your doctor, who may discontinue treatment until the fever goes away.
You may notice a reduced sense of taste while taking THIOLA, which will eventually go away. Some patients also report wrinkling or thin, fragile skin during long-term treatment.
Other side effects of THIOLA may include an itchy rash that is found on many parts of your body. This typically occurs during the first few months of treatment, and antihistamines can help reduce the itching. The rash will usually disappear once you stop taking THIOLA. Less often, patients who take THIOLA for more than 6 months may develop a rash that is usually located on the upper body and is very itchy. It typically goes away slowly after discontinuing treatment and returns after re-starting treatment.
Some patients may develop a drug hypersensitivity reaction to THIOLA that includes fever, joint pain, and swollen lymph nodes. If this occurs, your doctor may discontinue THIOLA.
THIOLA can cause serious and potentially fatal blood disorders, including aplastic anemia (your bone marrow does not make enough new cells), agranulocytosis (decrease in certain white blood cells), or thrombocytopenia (decrease in the number of platelets). Call your doctor immediately if you have any signs or symptoms such as fever, sore throat, chills, bleeding, or if you are bruising more easily.
Although THIOLA may be less toxic than d-penicillamine, it could potentially cause all of the serious side effects reported for d-penicillamine. No deaths have been reported as a result of THIOLA treatment; however, fatal outcomes have been reported with certain complications of d-penicillamine therapy, including reduced white blood cells, red blood cells, or platelets; Goodpasture's syndrome (an autoimmune disorder that affects the lungs and kidneys); and myasthenia gravis (an autoimmune disorder that causes muscles to weaken). Do not take THIOLA if you have a history of these conditions.
Additional side effects that have been reported during treatment with d-penicillamine and that might occur during THIOLA treatment include: decreased sense of smell, nausea, vomiting, diarrhea or soft stools, loss of appetite, abdominal pain, bloating, gas, sore throat, sores in the mouth, hives, warts, swelling of the throat, difficulty breathing, shortness of breath, fatigue or weakness, muscle or joint pain, swelling in your legs or fluid build-up in the lungs, lung or kidney problems, and blood or high amounts of protein in urine. These side effects are more likely to develop during THIOLA therapy in patients who had previous reactions to d-penicillamine. Talk to your doctor about any unusual side effects.
Seek immediate medical attention and discontinue THIOLA if you notice symptoms such as fever, sore throat, chills, bleeding, easy bruising, coughing up blood, muscle weakness, blistering or raw areas on the skin or mucous membranes, joint pain, swelling of the lymph nodes, or swelling in your legs, as these may be signs of a serious reaction to the drug.
Jaundice (yellow appearance of the skin and whites of the eyes) and abnormal liver function tests have been reported during THIOLA treatment for conditions unrelated to cystine stones.
Call your doctor for medical advice about side effects. You may report negative side effects to Retrophin® Medical Information at 1-877-659-5518, or to the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
Please see full Prescribing Information.