Cystinosis Treatment
Know your cystinosis treatments
Making careful treatment choices may help people with cystinosis manage their condition today and prevent issues tomorrow. There are medicines that may help delay or limit cystinosis damage called cystine-depleting therapies (CDTs) and treatments that may help with other symptoms and effects related to cystinosis. Talk with your healthcare team to create cystinosis treatment plans that may work.
What is cystine-depleting therapy?
CDTs are medicines that work to lower the levels of cystine in the cells. Keeping cystine levels low can help limit or possibly delay damage to the body.
There are a couple of CDTs. They work to remove cystine from the body or the eyes.
* Oral treatments do not reduce or remove cystine in the eye
Kidney dialysis and transplant
Cystinosis harms the kidneys, making it difficult for the body to absorb substances it needs and causing large losses of fluid through peeing (urination). Eventually, the kidneys may be unable to remove waste from the blood, leading to kidney failure. As the kidneys fail, dialysis or a kidney transplant is needed. Dialysis is a temporary treatment that does the job of the kidneys by removing waste from the blood.
After the transplant, medicines are used to help keep the body’s immune system from rejecting the transplanted kidney.
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Tracking cystine levels
It’s one of the most important things to do to manage cystinosis.
Goals of managing cystinosis
The main goal of managing cystinosis is to slow the progression of the disease by having a plan that works.
Living with cystinosis
Tips for facing the daily challenges of cystinosis from others with the disease.
Goals of managing cystinosis
Managing your cystinosis is key to living the fullest life possible. The best way to do this is to take steps that may delay or limit cystinosis from damaging the body. And the only way to possibly delay or limit damage is to keep your cystine levels low.
One of the hardest things about managing cystinosis is staying motivated to prevent future health issues. This is particularly true when you feel fine. In fact, you can't feel the cystine building up in your cells. Instead, cystine buildup slowly damages the body and may lead to more serious issues over time. That's why it's important to keep cystine levels low—not just now and then but all the time. Making cystinosis management goals and sticking to them may help.
Achieving goals
Define and stick to your cystinosis management goals by working with your doctor and healthcare team. Start by:
Learning about your disease
- Discover what cystinosis is and what it does to your body
- Find out about cystine and why it's so important to keep cystine levels low
- Understand the disease well enough to explain it to others in simple terms. Understand more
Knowing your treatment options
- Speak to your doctor about what treatment options are available to you and how they work. Find out more
Creating a treatment plan
- Make sure you take your medicine as prescribed to manage your cystine levels
Sticking to your treatment schedule
- Take your medicines every day, on time, and as instructed by your doctor
- Remind yourself by setting alarms on your smartphone, downloading a treatment schedule app, or keeping a calendar
Tracking your progress
- Check cystine levels with testing. This is the only way you and your doctor will know how well your cystine levels are managed. Find out how
Support is key
You know it's important to manage cystinosis every day, but you also know how challenging it is. Fortunately, others living with cystinosis may help you stick to your goals. People like you understand the challenges of cystinosis better than anyone else.
Tracking cystine levels
What is a cystine level test?
Cystine level testing measures the amount of cystine in white blood cells (WBCs). Along with carefully following a treatment plan, routine testing is key. Testing is the only way to know a person’s cystine level.
Why cystine level testing is important
Tracking cystine levels over time helps people and their doctors know how well their treatment is working. Knowing a person’s cystine level will help a doctor decide if a dose needs to be adjusted. Having the correct dose is the only way to help delay or limit damage to the body.
Two cystine tests. Two target levels.
There are two types of cystine level tests. They measure cystine levels differently.
| Type of WBC cystine level tests | Mixed leukocytes test | Granulocytes test |
| Target cystine level range | Below 1.0 nmol ½ cystine/mg protein | Below 1.9 nmol ½ cystine/mg protein |
| When to get the test | The doctor will order WBC cystine level testing as they feel is appropriate. | |
It's important to work with a doctor to decide which cystine level test is right for you. Always know which test is being taken: results mean different things with different tests. The same test should be taken each time to help compare test results over time.
Timing matters
It's important to have cystine levels tested at the right time to get useful test results. Ask your doctor what time of day to get your cystine levels tested.
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Finding your cystinosis treatment
Have a meaningful discussion with your doctor about your treatment options.
Goals of managing cystinosis
The main goal of managing cystinosis is to slow the progression of the disease by having a plan that works.
Living with cystinosis
Tips for facing the daily challenges of cystinosis from others with the disease.
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