Ultomiris as effective as Soliris for PNH: Real-world analysis
Approved treatments help control destruction of red blood cells
Ultomiris (ravulizumab-cwvz) is as effective or better than Soliris (eculizumab) at keeping paroxysmal nocturnal hemoglobinuria (PNH) under control, a real-world case series shows.
“Our real-world data reflect routine clinical practice, supporting the notion that [Ultomiris] may provide enhanced disease stability in high-risk patients,” researchers wrote.
The results were described in the study, “Disease Activity After Switching From Eculizumab to Ravulizumab in Patients With Paroxysmal Nocturnal Haemoglobinuria: Real World Data From Denmark and Finland,” which was published in eJHaem. It was supported by a research grant from Alexion, which markets Soliris and Ultomiris.
Most patients had classical PNH
In PNH, the excessive activation of the complement system, a group of immune proteins, leads to the destruction of red blood cells in a process known as hemolysis. Hemolysis can occur inside or outside blood vessels and lead to a range of PNH symptoms.
Soliris and Ultomiris are approved PNH treatments that help control hemolysis by blocking the activation of the complement system.
In this study, the researchers shared their real-world experience with 20 individuals with PNH who switched from Soliris to Ultomiris. Fourteen patients were being treated across four hospitals in Denmark, and six were being followed at a single hospital in Finland.
Participants were mainly women (70%), with a median age of 34.4 years at diagnosis. Most had classical PNH (70%), characterized by preserved bone marrow function, while 30% had PNH associated with aplastic anemia, a condition in which the bone marrow fails to produce enough blood cells and that sometimes precedes or occurs alongside PNH.
They received Soliris for a median of 4.6 years before switching to Ultomiris. Soliris was typically administered at 900 mg every two weeks, while Ultomiris was given every eight weeks (roughly two months) at a dose based on body weight.
4 patients required fewer blood transfusions after switch
The treatment’s efficacy was evaluated based on the need for blood transfusions and episodes of breakthrough hemolysis (BTH), meaning events of increased hemolysis in patients undergoing treatment with complement inhibitors.
In 11 patients, the disease was well managed on either treatment, with no transfusions or BTH episodes occurring in the 12 months before and after the switch. Following the switch to Ultomiris, four patients required fewer blood transfusions, and three participants had significantly reduced BTH episodes. For example, one patient went from needing 26 transfusions to just one after switching treatments, while another saw the number of BTH episodes drop from 37 to none.
Most participants also had similar levels of hemolysis markers, including lactate dehydrogenase (LDH) and reticulocytes, or immature red blood cells, before and after switching. Three patients had lower levels of LDH after the switch.
This case series aligns with previous findings that [Ultomiris] provides at least comparable disease control compared with [Soliris] in most patients with PNH.
Only one patient showed improved disease control during treatment with Soliris, with a smaller decline in LDH levels and an increase in the number of blood transfusions following the switch to Ultomiris.
The researchers also observed that Ultomiris effectively reduced transfusion requirements and BTH episodes in patients with a high burden of these features.
“This case series aligns with previous findings that [Ultomiris] provides at least comparable disease control compared with [Soliris] in most patients with PNH,” the researchers wrote.
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