Denosumab in patients with aneurysmal bone cysts: A case series with preliminary results

Abstract

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.

Aneurysmal bone cyst: results of an off label treatment with Denosumab

“…Palmerini et al reported 9 patients with Denosumab treatment [18]. It is not clear from their publication how many of the patients had any kind of prior therapy.…”

“…To our best knowledge, 2 case series (with 9 patients each) have previously been published [18][19][20] with an additional 11 cases having been published as individual case reports [20][21][22][23][24][25][26][27][28][29].…”

Recurrent Aneurysmal Bone Cyst of the Distal Fibula Treated with Denosumab and Curettage

“…There have been several case reports highlighting the efficacy of denosumab in the spine and sacrum, but very few have reported on outcomes in long bones. Using denosumab for a recurrent ABC of the distal radius, Pauli et al [22] were able to consolidate a locally aggressive and destructive lesion into a resectable lesion and implement limb-preserving surgery in an otherwise unresectable lesion. More recently, two cases of ABC in long bones, one of which was recurrent, have been treated with denosumab for an average of 7 months followed by curettage and bone grafting with histologic compete response to treatment with a median of 23-month follow-up [22].…”

“…Using denosumab for a recurrent ABC of the distal radius, Pauli et al [22] were able to consolidate a locally aggressive and destructive lesion into a resectable lesion and implement limb-preserving surgery in an otherwise unresectable lesion. More recently, two cases of ABC in long bones, one of which was recurrent, have been treated with denosumab for an average of 7 months followed by curettage and bone grafting with histologic compete response to treatment with a median of 23-month follow-up [22]. Kurucu et al [23] treated nine cases of ABC with denosumab for an average of 12 months, of which two eventually needed surgery.…”

“…However, current trends are for treatment lasting 3-4 months prior to surgical intervention for GCT. We chose to present this case given the over 3-year follow-up which is longer than previously reported in [12, 22, 23]. Although segments of the fibula are resectable, resection of the distal fibula can lead to lifelong ankle instability, even with reconstruction, so we chose to preserve it.…”