Ultomiris Used for 1st Time to Manage aHUS During Kidney Transplant

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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Ultomiris (ravulizumab) was reportedly used for the first time to control atypical hemolytic uremic syndrome (aHUS) in a patient who received a kidney transplant from a living donor, according to a group of doctors in Germany.

“We provide first evidence for the efficacy of [Ultomiris] to prevent recurrence of aHUS in a genetically high-risk situation before and after [kidney] transplantation,” the team wrote.

Their report, “Ravulizumab in Preemptive Living Donor Kidney Transplantation in Hereditary Atypical Hemolytic Uremic Syndrome,” was published in the journal Transplantation Direct.

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The team described the case of a 33-year-old woman with aHUS and kidney failure. The patient had originally been diagnosed with kidney disease of unknown cause in 2004. Nearly 15 years later, in 2018, she became pregnant for the first time, and during her pregnancy, she developed acute kidney failure.

As a result, the baby was born prematurely, and the patient underwent a kidney biopsy. Kidney tissue analysis revealed signs of thrombotic microangiopathy, a type of blood vessel damage characteristic of aHUS.

Genetic testing revealed that the patient carried a mutation in the CFH gene. Mutations in this gene are known to be associated with aHUS and are generally linked with a more severe disease course.

Once the diagnosis of aHUS was confirmed, the patient quickly started treatment with Soliris (eculizumab), an approved treatment for aHUS that works by blocking the activity of part of the immune system called the complement cascade, which is overactive in people with the disease.

Ultomiris is a newer medication that has a similar mechanism to Soliris, but is modified to allow for less frequent dosing. In light of the patient’s busy schedule — she had a new baby, and was also completing studies at university — she transitioned from Soliris to Ultomiris.

Subsequently, the patient developed renal anemia — when kidney dysfunction prevents the body from making enough oxygen-carrying red blood cells — and metabolic acidosis, an acidity imbalance in the body. Due to these problems, she was advised to undergo a kidney transplant.

In April 2021, the patient received a kidney donated by her 30-year-old husband.

Six months after the transplant, lab tests showed no evidence of kidney dysfunction. The patient continued on Ultomiris, without any change in infusion schedules, when she received the transplant.

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“Given concerns of safety or on drug interactions, clinicians might hesitate to use [Ultomiris] in patients who progress to end-stage kidney disease and are candidates for living donation transplantation. Hence, patients might be deprived of the benefit from the extended half-life of this drug,” the team wrote.

Soliris and Ultomiris are both marketed by Alexion Pharmaceuticals, now part of AstraZeneca, which was not involved in this study.