NEW STUDY | Pegcetacoplan Shows Potential in Managing CAD and wAIHA
A study published in the journal Blood suggests that pegcetacoplan may serve as a safe and effective treatment option for patients with cold agglutinin disease (CAD) or warm antibody autoimmune hemolytic anemia (wAIHA).
This investigational therapy, which targets complement component C3, has demonstrated the ability to increase haemoglobin levels, reduce haemolysis, and improve fatigue scores, according to the study’s authors.
Although not all patients with CAD or wAIHA require treatment, a significant proportion experience relapse after initial therapies such as rituximab or corticosteroids. Specifically, approximately two-thirds of CAD patients and one-quarter of wAIHA patients may require additional treatment following disease recurrence.
Previous research has indicated that pegcetacoplan could be effective in managing CAD and wAIHA. To further evaluate its safety and efficacy, researchers conducted an open-label phase 2 study (ClinicalTrials.gov Identifier: NCT03226678). The study enrolled 24 patients, including 13 with a primary diagnosis of CAD and 11 with wAIHA. While CAD patients were included regardless of prior treatment history, wAIHA patients were required to have progressed after at least one previous line of therapy. Participants received either 270 mg or 360 mg of pegcetacoplan subcutaneously once daily for 48 weeks.
At the end of the study period (week 48), results indicated promising outcomes. The median increase in haemoglobin levels from baseline was 2.4 g/dL in the CAD cohort and 1.7 g/dL in the wAIHA cohort. Additionally, early improvements in haemolysis and fatigue—assessed using the Functional Assessment of Chronic Illness Therapy scale—were sustained through week 48.
Regarding safety, treatment-emergent adverse events occurred in all CAD patients (100%) and in 91% of wAIHA patients. Serious adverse events were reported in 10 participants, though none were deemed related to pegcetacoplan. However, treatment discontinuation due to adverse events occurred in 15.4% of CAD patients and 27.3% of wAIHA patients.
Encouraged by these findings, particularly in CAD patients, researchers have designed a phase 3 placebo-controlled trial to further evaluate pegcetacoplan’s efficacy. This next-phase study will utilize an adjusted dosing regimen of 1080 mg twice daily, aligning with pharmacokinetic and safety data gathered from the phase 2 trial.
Source: Hematology Advisor
