Until relatively recently, most doctors thought Alport syndrome was an untreatable disease. However, experiments carried out in mice with Alport syndrome demonstrate that several different kinds of medication could slow down loss of kidney function. Preliminary studies in people with Alport syndrome have also provided evidence that early treatment delays kidney fibrosis. This means that diagnosing Alport syndrome as early in life as possible in order to get patients on medication is important.
At this time, there is no FDA approved treatment for Alport syndrome and gene therapy and/or gene editing is not available. The current goal of all treatment is to help slow progression of Alport syndrome so that patients keep their “native” kidneys as long as possible.
Blood-pressure lowering medicines are often prescribed for this reason, even if patients don’t have high blood pressure. ACE and ARB medications; Lisinopril, Ramipril and Losartan – are proven to slow the spill of protein into the urine, slowing the scarring of the kidneys. Current research is in process to more fully understand the disease, identify more treatment options, and a potential cure. Several human clinical trials are in progress as well..
Importance of Diagnosis and Treatment (All Ages)
Medication Information (All Ages)
Click here to learn about medications used to treat Alport syndrome.
The Alport syndrome Research Collaborative, an international group of medical professionals who treat Alport patients, developed clinical practice recommendations aimed at standardizing care for children with Alport syndrome in 2012. A PDF version of the recommendations can be accessed here.
In November 2020, the 2012 Alport syndrome Research Collaborative recommendations were updated by Drs. Clifford Kashtan and Oliver Gross and published in Pediatric Nephrology (please note this is a subscription-locked article).
In February 2021, Dr. Clifford Kashtan graciously prepared a layman’s summary of the November 2020 updated pediatric treatment recommendations. Click here to read Dr. Kashtan’s summary. PDF de las pautas de tratamiento pediátrico actualizadas del Dr. Kashtan.
On February 24, 2021 ASF hosted a live Q&A with pediatric nephrologist Dr. Bradley Warady to discuss care for Alport youth. Click here to view a closed-captioned recording of the event.
It is very important for people with Alport syndrome to be examined regularly by a nephrologist so that effects of kidney disease, such as hypertension (high blood pressure), can be identified early and treated. Regular evaluations of hearing and vision are also important.