Atypical Hemolytic Uremic Syndrome (aHUS) Treatment Options

What is Atypical Hemolytic Uremic Syndrome (aHUS)?

Atypical Hemolytic Uremic Syndrome (aHUS) is a rare disease that affects kidney function. It leads to abnormal blood clots (thrombi) forming in the small blood vessels in the kidneys. Severe medical problems can be caused by these clots if they block or restrict blood flow. Atypical hemolytic-uremic syndrome is primarily characterized by three significant features concerning abnormal clotting. Those three features include hemolytic anemia, kidney failure, and thrombocytopenia. It can happen at any age.

A person with aHUS may experience severe complications as this disease leads the body to develop many blood clots. It ultimately causes the blood to flow slowly to other important organs. These complications include kidney damage, kidney failure, high blood pressure, heart disease, or even heart attack.

Atypical hemolytic uremic syndrome is a complex disorder that can be caused due to various factors. These include environmental, genetic, and immunological factors. There’s no proper cure, but treatments are available to deal with Atypical hemolytic-uremic syndrome. Let’s explore the effective treatments for atypical hemolytic uremic syndrome.

Treatments

Here are the most effective treatments approved by the Food and Drug Administration (FDA).

Soliris (Eculizumab)

Soliris® (Eculizumab) is defined as a monoclonal antibody that is preferred to treat atypical hemolytic uremic syndrome (aHUS). It is given to the patient through intravenous infusion. Doctors may recommend it for 4 weeks pursued by the 5th dose 1 week afterward and then every 2 weeks subsequently. Basically, Soliris is a first-in-class terminal complement inhibitor that is used to target uncontrolled complement activation. It is prescribed for patients with atypical hemolytic uremic syndrome to inhibit complement-mediated TMA. FDA has approved it for the treatment of aHUS. Long-term renal and TMA outcomes can be enhanced and improved in patients with atypical hemolytic uremic syndrome by Soliris. The research shows that Soliris has been effective in patients with aHUS.

The Isaac an Abt MD Professor of Kidney Diseases, Craig B. Langman, M.D., Head of Kidney Diseases, Feinberg School of Medicine, Northwestern University, has stated that Soliris has significantly reduced the TMA process in clinical trials. This process is responsible for renal impairment, thrombosis, angina, and seizures in patients with aHUS. When this is markedly lowered, it shows a significant efficiency in aHUS patients. They further added that this therapy is efficient in restoring kidney function.

The research has shown that Soliris has the tendency to enhance red blood cell count and platelets. It tends to reverse acute kidney damage and may prevent kidney failure. It is effective when taken not too late into a diagnosis.

Atypical hemolytic uremic syndrome is a life-threatening disease that can be transformed significantly with the help of Soliris. It does not just work for the patients with aHUS but is also used to treat Paroxysmal Nocturnal Hemoglobinuria, Generalized Myasthenia Gravis, and Neuromyelitis Optica Spectrum Disorder.

It is necessary for the patient to tell their medical history to their doctor. If you have aHUS and you go to the doctor for treatment, you should tell them about your current condition, because Soliris can cause severe kinds of allergic reactions. So, those who have a history of allergic reactions are less likely to adopt this treatment. Patients who undergo Shiga toxin E. coli hemolytic uremic syndrome (STEC-HUS) can’t be prescribed Soliris.

Basically, Soliris is not a therapy that can be prescribed to everyone. It has limitations of allergy, pregnancy, and other severe diseases. The research has not found any adverse effects of Soliris in pregnant women, though doctors think twice before prescribing it to them.

Ultomiris (Ravulizumab)

Ultomiris® (Ravulizumab) is a humanized monoclonal antibody that is used to treat patients with atypical hemolytic uremic syndrome (aHUS). This therapy inhibits complement protein C5 that proves to be effective in aHUS patients. Many countries, including the USA, have adopted Ultomiris® (Ravulizumab) as a treatment of aHUS.

Basically, Ultomiris and Soliris are not so different because Ravulizumab has been re-engineered by eculizumab. Both Soliris and Ultomiris inhibit the cleavage of the complement factor, which helps them prevent the formation of complex clots within blood vessels. These therapies eventually help extend the terminal elimination half-life. Its maintenance dosage regimen is 4-8 weeks. Ultomiris is also given through an intravenous drip (infusion). The frequency and the dose of the therapy depend upon the age and the body weight of the patient. Adult and pediatric patients can easily tolerate Ultomiris as it resolves TMA and has a similar mechanism to Soliris. However, its duration of action is 4-fold longer. It was eventually considered as a reliable treatment option for patients with atypical hemolytic uremic syndrome.

The research has shown that Ultomiris has proven to be efficient in patients with postpartum aHUS. Doctors may start the treatment with eculizumab and later shift to Ravulizumab. Its safety is enhanced. There are no unexpected safety events seen in the clinical trials.

Patients usually tolerate Ultomiris well, but as every therapy has adverse effects, so does Ultomiris. These are minor adverse effects, including diarrhea, headache, and vomiting. It is one of the most well-known, reliable, and efficient treatments for adults and pediatric patients of atypical hemolytic uremic syndrome.

Living with aHUS

Cases of aHUS are not similar, which makes it difficult for a doctor to understand the exact condition. Those who experience aHUS symptoms should go to a kidney doctor (nephrologist) or a blood doctor (hematologist). They will assess and monitor a patient’s current condition. If you have aHUS, you may need to follow regular blood tests to check your kidney health, red blood cells, and platelet count on a regular basis.