The treatment of cystinosis includes taking a drug, either Cystagon or Procysbi, that must be taken daily, either 4 or 2 times a day. Some people may choose to stop taking the drug because of side effects. This is called poor compliance. Cystagon or Procysbi both have side effects. They taste and smell bad and when people use them they may have bad breath or develop a body odor. Children, particularly older children, may stop taking a drug because they are embarrassed or their classmates may make unkind comments. Children or teenagers who struggle with compliance should talk to their parents, physicians and a psychologist to work through these issues.
Sometimes people stop taking a drug because it’s working well and they feel better. They may start to think that they do not need the drug anymore or as often as their doctors recommend. However, cystine will keep building up in the cells throughout life, even if you start to feel better. Stopping one of these drugs will lead to even more cystine build up. These drugs must be taken every day. Even if someone feels better and their symptoms improve, they need to continue to follow their treatment plan as developed by their physician and medical team in order to continue feeling better.
Cystinosis support groups have information about how to handle compliance problems. The Cystinosis Foundation offers a booklet called Nephropathic Cystinosis: A Guide for Parents, Patients and Healthcare Professionals. The Cystinosis Research Networks offers booklet called, Cystinosis Parent Handbook.
