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About Nephropathic Cystinosis
Cystinosis is a rare, genetic condition that affects about 500 to 600 people in the United States and an estimated 2,000 worldwide. Cystinosis symptoms usually appear within a child’s first year of life.
Nephropathic cystinosis is the most severe and most common form of cystinosis, making up 95% of all cases.
Nephropathic cystinosis causes severe damage to kidneys and other organs all over the body.
Nephropathic cystinosis symptoms usually appear within a child’s first year of life.
Cystinosis can affect nearly every organ of the body. Cystinosis treatment can prevent or slow some effects, but existing damage cannot be reversed.
Eyes
Sensitivity to light (photophobia)
childhood and adulthood Blindness
childhood and adulthood
Thyroid
Poorly working thyroid (hypothyroidism)
childhood and adulthood
Pancreas
Diabetes
childhood and adulthood
Kidneys
Fanconi syndrome
infancy, childhood, and adulthood
Kidney failure
childhood and adulthood
Muscles
Muscle weakness and decreased muscle mass (myopathy)
childhood and adulthood
Brain
Visual or learning issues childhood and adulthood
Throat
Trouble swallowing childhood and adulthood
Lungs
Problems breathing adulthood
Male reproductive organs*
Not being able to father children naturally (infertility) adulthood
Bones
Softening or weakening of bones (rickets)
infancy, childhood, and adulthood
Cystinosis has not been shown to cause infertility in women. If you are pregnant, planning on becoming pregnant, or breastfeeding, talk with your doctor about which treatments may be right for you.
Over time, nephropathic cystinosis causes damage to the kidneys. This damage makes the kidneys increasingly unable to absorb essential nutrients and filter out the body’s waste—a disorder known as Fanconi syndrome. In people with Fanconi syndrome, nutrients that would normally be absorbed are passed through the kidneys and are eliminated in pee (urine).
Fanconi syndrome is often the first sign of cystinosis, as cystinosis often presents through Fanconi syndrome and leads to cystinosis diagnosis. Cystinosis can be discovered through a white blood cell cystine level test.
Eventually, the damage caused by cystinosis causes the kidneys to fail completely, which can be treated only with dialysis (an artificial means of getting rid of the body’s waste) and, ultimately, a kidney transplant.
While a transplanted kidney may not be affected by cystinosis, it is important to continue cystine-depleting therapy (CDT) post transplant to help delay or reduce new damage to other organs.
Cystine-depleting therapy (CDT): A treatment that uses cysteamine to reduce the buildup of cystine in cells.
A kidney transplant is not a cure for cystinosis. While a new donor kidney will not be affected, cystine will continue to build up in other parts of the body.
Cystinosis is an inherited disease that is passed from parent to child,
when a gene that doesn’t work right leads to problems with the way cystine is stored in the body. Cystinosis can only develop in children who receive a non-working copy of the cystinosis gene from each parent.
The damage from cystinosis cannot be undone, but it can be delayed or prevented with treatment. The impact of sticking to cystinosis treatment is noticeable, even if people with cystinosis don't notice it themselves. Testing shows how CDTs are working inside the body, and what is happening with cystine levels.
CDTs lower cystine levels, which is why it's important that people with cystinosis take a CDT on time and as prescribed, even if they look and feel fine. Work with the healthcare team to plan a schedule for cystine level testing. Find out more details about cystine level testing.
One study showed that cystine levels increased by more than 50% when the dose was delayed compared to when they took their medicine on time (at 9 hours vs 6 hours).
It’s a lifelong condition, but treatments such as CDT and kidney transplant have allowed people with cystinosis to live longer.
Tell your doctor right away if you develop any of the following symptoms while taking PROCYSBI: headache, buzzing or "whooshing" sound in the ear, dizziness, nausea, double vision, blurry vision, loss of vision, pain behind the eye, or pain with eye movement.
PROCYSBI (cysteamine bitartrate) delayed-release capsules and delayed-release oral granules is a prescription medicine used to treat nephropathic cystinosis in adults and children 1 year of age and older. It is not known if PROCYSBI is safe and effective in children under 1 year of age.
Do not take PROCYSBI if you are allergic to penicillamine or cysteamine.
Before taking PROCYSBI, tell your doctor about all your medical conditions, including if you:
Tell your doctor about all the medicines you take, including prescription and over the counter medicines, vitamins, dietary and herbal supplements. Know the medicines you take. Keep a list of them to show your doctor and pharmacist when you get a new medicine.
PROCYSBI can cause serious side effects, including:
The most common side effects of PROCYSBI include: vomiting, nausea, stomach (abdominal) pain, pink eye, diarrhea, cold, tiredness, flu, headache, problems with body salts or electrolytes, infection of ear, nose or throat, joint pain.
These are not all the possible side effects of PROCYSBI. Call your doctor for medical information about side effects. You may report side effects to FDA at 1-800-FDA-1088.
For additional important safety information, click here for the Patient Package Insert and discuss with your doctor.