Induction therapy with infliximab effective against severe Behçet’s syndrome

A recent groundbreaking phase 2 trial compared the induction therapies for severe Behçet’s syndrome revealed that infliximab is both effective and safe than cyclophosphamide. This randomized controlled trial was conducted between May 2018 and April 2021 focused on patients with major vascular and central nervous system involvement who were particularly a high-risk group within Behçet’s disease spectrum.

The study included a total of 52 patients who met the criteria for Behçet’s syndrome of International Study Group. The participants were randomly assigned to receive either intravenous infliximab (5 mg/kg at weeks 0, 2, 6, 12, and 18) or cyclophosphamide (0.7 g/m² at weeks 0, 4, 8, 12, 16, and 20, with a maximum dose of 1.2 g per infusion). All participants were administered a uniform glucocorticoid regimen throughout the trial.

Infliximab demonstrated a significantly higher rate of complete response which was defined as clinical, biological, and radiological remission coupled with a prednisone dose ≤0.1 mg/kg daily by week 22. Also, 22 out of 27 patients (81%) treated with infliximab achieved complete response when compared to 14 out of 25 patients (56%) in the cyclophosphamide group. The estimated difference was 29.8 percentage points, with a 95% credible interval of 6.6 to 51.7. The posterior probability that at least 70% of infliximab-treated individuals would achieve complete response was 97.4%, while the probability of cyclophosphamide stood at only 6.0%. 

Infliximab also demonstrated a more favorable safety profile. Adverse events were reported in 29.6% (8 out of 27) of patients in the infliximab group compared with 64% (16 out of 25) in the cyclophosphamide group. This resulted in an estimated difference of −32.3 percentage points, with a 95% credible interval ranging from −55.2 to −6.6. The rate of serious adverse events was comparable between the two groups, recorded at 15% for infliximab and 12% for cyclophosphamide.

These findings underline the potential of infliximab as a superior induction therapy for patients with severe forms of Behçet’s syndrome, particularly those with vascular or neurological complications. The higher efficacy rate and fewer adverse events suggest that infliximab could be a preferable option over the traditionally used cyclophosphamide, which had a higher burden of side effects. Overall, the study provides strong evidence to favor infliximab as a first-line treatment for severe Behçet’s syndrome.

Source:

Saadoun, D., Maalouf, G., Vieira, M., Trad, S., Lazaro, E., Sacre, K., Plessier, A., Sené, T., Koné-Paut, I., Noel, N., Mekinian, A., Lambert, M., Ribeiro, E., Mirault, T., Mele, N., Dellal, A., Fain, O., Melki, I., Chiche, L., … Cacoub, P. (2024). Infliximab versus Cyclophosphamide for Severe Behçet’s Syndrome. In NEJM Evidence (Vol. 3, Issue 11). Massachusetts Medical Society. https://doi.org/10.1056/evidoa2300354