Know your cystinosis treatments

Caregiver of child with cystinosis preparing cystine-depleting medicine

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.

FDA-approved cystinosis treatments

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.