Publications

Background: Schnitzler syndrome is an adult-onsetautoinflammatory disease characterized by urticarialexanthema and monoclonal gammopathy accompanied bysystemic symptoms such as fever, bone, and muscle pain. Up tonow, approved treatment options are not available.

Objective: We assessed effects of the anti–IL-1bmAbcanakinumab on the clinical signs and symptoms of Schnitzlersyndrome.

Methods: In this phase II, randomized placebo-controlledmulticenter study, 20 patients with active disease enrolled in 4German study centers. Patients were randomly assigned to receive single subcutaneous canakinumab 150 mg or placebo injections for 7 days, followed by a 16-week open-label phase with canakinumab injections on confirmed relapse of symptoms.The primary end point was the proportion of patients with complete clinical response evaluated by physician global assessment at day 7. Key secondary end points included changesin patient-reported disease activity (Schnitzler activity score),inflammation markers (C-reactive protein and serum amyloidA), and quality-of-life assessments (Dermatology Life QualityIndex and 36-item short form health survey).

Results: The proportion of patients with complete clinical response at day 7 was significantly higher (P5.001) in the canakinumab-treated group (n55 of 7) than in the placebo group (n50 of 13). Levels of inflammation markers C-reactiveprotein and serum amyloid A and quality-of-life scores were significantly reduced in canakinumab-treated but not in placebo-treated individuals. Positive effects continued up to16 weeks. Adverse events were manageable and includedrespiratory tract infections, gastrointestinal symptoms, andhypertension.

Conclusions: In this first placebo-controlled study,canakinumab was effective in patients with Schnitzlersyndrome, and thus canakinumab may be further evaluated as atherapeutic option for this rare disease. (J Allergy ClinImmunol 2017;139:1311-20.)