Canakinumab treatment in a young girl with refractory chronic recurrent multifocal osteomyelitis associated with pyoderma gangrenosum

Background Chronic recurrent multifocal osteomyelitis (CRMO) is a bone inflammatory disorder characterized by osteolytic, usually multiple, symmetric lesions. Diagnosis is one of exclusion, and no standardized therapies are available. Presumed deregulation of the interleukin (IL)-1 beta axis, as observed in 2 monogenic autoinflammatory conditions such as Majeed syndrome (LPIN2 mutations) and deficiency of IL-1 receptor antagonist (IL1RN mutations) with CRMO-like bone involvement, suggests the blockade of IL-1 as potentially useful also in this condition, even if scarce data are available. Case presentation We report the case of a 13-year-old girl affected by a multidrug-resistant and pyoderma gangrenosum-complicated CRMO treated with canakinumab, a human monoclonal antibody targeting IL-1 beta. Conclusion In this young patient pyoderma gangrenosum and CRMO showed a rapid and satisfactory response to canakinumab, although over time a decreased efficacy in controlling bone disease was observed.