• Emanuela Palmerini, Pietro Ruggieri, Andrea Angelini, Stefano Boriani, Domenico Campanacci, Giuseppe M Milano, Marilena Cesari, Anna Paioli, Alessandra Longhi, Massimo E Abate, Guido Scoccianti, Silvia Terzi, Giulia Trovarelli, Alessandro Franchi, Piero Picci, Stefano Ferrari, Martina Piccinni Leopardi, and Michela Pierini.
• 1 Chemotherapy Unit, IRCCS, Istituto Ortopedico Rizzoli, Bologna, Italy.
• Tumori. 2018 Oct 1; 104 (5): 344-351.

Purpose:Aneurysmal bone cyst (ABC) is a rare skeletal tumor usually treated with surgery/embolization. We hypothesized that owing to similarities with giant cell tumor of bone (GCTB), denosumab was active also in ABC.Methods:In this observational study, a retrospective analysis of ABC patients treated with denosumab was performed. Patients underwent radiologic disease assessment every 3 months. Symptoms and adverse events were noted.Results:Nine patients were identified (6 male, 3 female), with a median age of 17 years (range 14-42 years). Primary sites were 6 spine-pelvis, 1 ulna, 1 tibia, and 1 humerus. Patients were followed for a median time of 23 months (range 3-55 months). Patients received a median of 8 denosumab administrations (range 3-61). All symptomatic patients had pain relief and 1 had paresthesia improvement. Signs of denosumab activity were observed after 3 to 6 months of administration: bone formation by computed tomography scan was demonstrated in all patients and magnetic resonance imaging gadolinium contrast media decrease was observed in 7/9 patients. Adverse events were negligible. At last follow-up, all patients were progression-free: 5 still on denosumab treatment, 2 off denosumab were disease-free 11 and 17 months after surgery, and the last 2 patients reported no progression 12 and 24 months after denosumab interruption and no surgery.Conclusions:Denosumab has substantial activity in ABCs, with favorable toxicity profile. We strongly support the use of surgery and/or embolization for the treatment of ABC, but denosumab could have a role as a therapeutic option in patients with uncontrollable, locally destructive, or recurrent disease.

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