Cystinosis Treatment; Depends On the Age, Type, and the Degree of Disease Severity

 

Cystinosis Treatment

In the past, cystinosis treatment was limited to treat metabolic acidosis and replace electrolytes lost in the urine. Today, the wide availability of an effective drug, kidney transplantation, and cysteamine, has dramatically improved the treatment and management of cystinosis. Cystinosis is the most common hereditary cause of renal Fanconi syndrome in children; characterized by accumulation of the amino acid cystine within cells. Excess cystine damages cells and forms crystals that can build up and cause problems in many organs and tissues.

Kidney transplantation is a safe and effective treatment for the kidney failure of individuals with cystinosis. Nephropathic (infantile) cystinosis is the most common and severe form of the cystinosis. Nephropathic cystinosis usually appears in infants and children at a young age. It is a life-long condition, but available treatments, such as kidney transplantation and cysteamine therapy, have allowed people with the disease to live longer. Intermediate cystinosis usually becomes apparent in affected individuals in adolescence.

Intermediate cystinosis is characterized by symptoms similar to nephropathic cystinosis. These symptoms, however, are not prominent till age of 8 to 20 years. If untreated, end-stage renal failure in intermediate cystinosis usually develops at some point between 15 and 25 years of age. Cystinosis is a rare disease characterized by the accumulation of cystine in different organs and tissues of the body, such as brain, eyes, kidneys, liver, muscles, and pancreas. Cystinosis is an inherited disease, meaning that it is passed down through families.

According to the American Kidney Fund, cystinosis occurs in only about 1 in 100,000-200,000 children, so it is not widely known nor understood. Thus, there is an increasing demand for the cystinosis treatment in this region. Several cystinosis treatments are available including the cysteamine therapy, hormonal therapy, and renal replacement therapy, among others; however, no curative treatment is yet available. Moreover, the drug cysteamine is an oral drug given to patients with cystinosis prior to kidney transplantation.


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