Soliris Treats Rare Case of aHUS Due to Scleroderma Renal Crisis

Man, 46, had declined while being given corticosteroids

Teresa Carvalho, MS avatar

by Teresa Carvalho, MS |

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This illustration highlights the kidneys of a person shown from behind while drinking from a glass.

Soliris (eculizumab) safely and effectively treated a 46-year-old man with atypical hemolytic uremic syndrome (aHUS) secondary to a scleroderma renal crisis — a life-threatening complication of an autoimmune disorder that can lead to kidney failure.

His case was described in the study, “Atypical hemolytic-uremic syndrome due to a scleroderma renal crisis treated with eculizumab,” published in the journal Polish Archives of Internal Medicine

aHUS is a type of thrombotic microangiopathy (TMA), a group of diseases characterized by the formation of blood clots in small blood vessels.

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It results from the uncontrolled activation of the complement cascade, a part of the body’s immune system, leading to inflammation and clotting in small blood vessels and giving rise to disease symptoms.

aHUS is rarely associated with systemic scleroderma, an autoimmune disorder characterized by the buildup of scar tissue in the skin and several internal organs, such as the heart, kidney, lungs, and gastrointestinal tract.

Around 5% of patients with systemic scleroderma are reported to develop a renal crisis, a life-threatening complication characterized by a sudden increase in blood pressure and acute kidney failure.

Soliris, a complement inhibitor, is an approved therapy that reduces inflammation and blood clotting, and helps to improve kidney function in people with aHUS.

Scientists in Poland reported the case of a 46-year-old man successfully treated with Soliris after developing aHUS secondary to a scleroderma renal crisis.

The man was admitted to the hospital due to unusual chest pain. Heart scans revealed inflammation of the heart muscle caused by a viral infection.

Further imaging tests suggested eosinophilic granulomatosis with polyangiitis, a rare condition that causes inflammation of small- and medium-sized blood vessels. As such, the patient was started on oral steroids to reduce inflammation, but he continued to decline.

Doctors suspected the patient had developed TMA, which had led to a reduction in red blood cells and platelets counts.

“Corticosteroids were used in our patient causing a deterioration of the general condition. However, the signs of TMA started before the administration of steroids,” the researchers wrote.

He was given freshly frozen plasma, a treatment meant to replace a person’s plasma — the non-cellular parts of blood — and methylprednisolone, a steroid medication.

After second plasma exchange, given due to an increase in markers of kidney injury and TMA signs, his condition continued to worsen. The patient showed an excess amount of water retention, had poorer lab tests results, and higher blood pressure.

The man was then diagnosed with limited systemic scleroderma, which is characterized by skin symptoms largely limited to the face, arms below the elbows, hands, and fingers.

According to researchers, this diagnosis was “very difficult due to a nonobvious course.”

The scientists noted the patient never “noticed” Raynaud’s phenomenon, a condition common to scleroderma patients and characterized by the numbness of fingers and toes in response to cold temperatures or stress. The musculoskeletal symptoms he experienced were attributed to his sports history.

Physicians suspected that he also developed aHUS due to a scleroderma renal crisis, and treated him with Soliris for three months.

After treatment, the man showed a significant improvement in hemoglobin — a protein in red blood cells that is responsible for oxygen transport — and platelet counts, as well as lower activity of lactic dehydrogenase, a marker of tissue damage. No treatment-related side effects were observed.

Genetic testing revealed the presence of mutations in the genes that encode complement factor H (CFH), a key regulator of the complement pathway, and ADAMTS13, a protein involved in blood clotting. Both of these mutations are known to increase the risk of developing severe aHUS.

The man underwent dialysis and, as of this study’s conclusion, is still dependent on it. He is being referred to a kidney transplant waiting list, the scientists noted.

According to the team, studies reporting on the use of Soliris in patients with scleroderma renal crisis are limited by the small number of patients they include, the absence of a control group, and interactions with other medications.

As such, they recognized that “in severe cases of SRC [scleroderma renal crisis] complicated with TMA, eculizumab treatment can be a life-saving drug, however, further studies are needed,” they wrote.