Treatment

There is no cure for aHUS. In fact, there is no standard treatment, as each case is different. The short term strategy is to be supportive and reactive to whatever symptoms are taking place.

Once an aHUS patient’s symptoms are analyzed, a treatment plan can be developed. Nephrologists (doctors specializing in kidney function) treat patients with medications that prevent blood clotting, as well as blood transfusions, plasma infusions (plasma is the liquid part of the blood), and plasmapheresis (a blood filtering process) in order to replace the missing or defective complement proteins.

Blood transfusions:

Blood transfusions are almost always the first line of management, aHUS patients are typically admitted to the hospital with anemia (a condition in which the body does not have enough healthy red blood cells). A patient may receive packed red blood cells, whole blood, and/or platelets in order to stabilize their symptoms. These products help to return the blood to a more normal level for a brief time, but do not treat the disease.

Plasma therapies:

Plasma therapies are the most traditional way to treat patients with aHUS. Plasma from healthy donors is transfused into an aHUS patient through a process called plasma infusion. In difficult cases, plasmapheresis (or plasma exchange) may be used instead. In plasmapheresis, blood is withdrawn from the patient and the damaged plasma is removed from the blood and replaced with donor plasma. The blood, with all its red and white blood cells, is then transfused back into the patient. A single plasmapheresis treatment can take one to three hours to complete, with a single plasma infusion taking as little as 30 minutes to several hours.

No clinical studies have shown that plasma exchange therapy is safe or effective in treating aHUS. Plasma exchange/infusion does not treat the underlying disease, is not a cure, and symptoms may return over time.

Dialysis:

aHUS can cause a patient’s kidneys to permanently or temporarily stop functioning. If the patient’s kidneys cannot perform properly, dialysis can be used as an interim replacement for kidneys. Dialysis uses a special machine to remove waste and excess water from the blood, providing an artificial replacement for lost kidney function. Though it is necessary to sustain life, dialysis can be dangerous and risky. Kidney transplants are not a viable option for these patients until a treatment or cure can be found that puts the disease in remission so that the new kidney(s) won’t be damaged.

Medications:

Recently, Eculizumab (brand name: Soliris) which blocks complement activation was introduced as a treatment option for aHUS.  Treatment trials using eculizumab in adolescent and adult patients with aHUS has shown to be highly effective and safe (when appropriate protection against certain infections was applied) in stopping aHUS and improving reported quality of life of patients. Eculizumab has been approved for aHUS in Canada, the United States, the United Kingdom, the European Union and Japan. Unlike historical plasma therapies, this new treatment option has potential to change the course of the disease, and for the first time offers a real opportunity for transplant in those aHUS patients on chronic dialysis

Talk with your doctor about a management plan that includes frequent follow-up and monitoring of your disease.