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

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

Research output: Contribution to journalArticle

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.

Original languageEnglish
Pages (from-to)344-351
Number of pages8
JournalTumori
Volume104
Issue number5
DOIs
Publication statusE-pub ahead of print - Aug 8 2018

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Aneurysmal Bone Cysts
Denosumab
Giant Cell Tumor of Bone
Ulna
Paresthesia
Humerus
Gadolinium
Pelvis
Tibia
Osteogenesis
Contrast Media
Observational Studies
Spine
Therapeutics

Keywords

  • Aneurysmal bone cyst
  • bone tumor
  • denosumab
  • receptor activator of nuclear factor-kappa B ligand

Cite this

Denosumab in patients with aneurysmal bone cysts: A case series with preliminary results. / Palmerini, Emanuela; Ruggieri, Pietro; Angelini, Andrea; Boriani, Stefano; Campanacci, Domenico; Milano, Giuseppe M; Cesari, Marilena; Paioli, Anna; Longhi, Alessandra; Abate, Massimo E; Scoccianti, Guido; Terzi, Silvia; Trovarelli, Giulia; Franchi, Alessandro; Picci, Piero; Ferrari, Stefano.

In: Tumori, Vol. 104, No. 5, 08.08.2018, p. 344-351.

Research output: Contribution to journalArticle

Palmerini, E, Ruggieri, P, Angelini, A, Boriani, S, Campanacci, D, Milano, GM, Cesari, M, Paioli, A, Longhi, A, Abate, ME, Scoccianti, G, Terzi, S, Trovarelli, G, Franchi, A, Picci, P & Ferrari, S 2018, 'Denosumab in patients with aneurysmal bone cysts: A case series with preliminary results', Tumori, vol. 104, no. 5, pp. 344-351. https://doi.org/10.1177/0300891618784808
Palmerini, Emanuela ; Ruggieri, Pietro ; Angelini, Andrea ; Boriani, Stefano ; Campanacci, Domenico ; Milano, Giuseppe M ; Cesari, Marilena ; Paioli, Anna ; Longhi, Alessandra ; Abate, Massimo E ; Scoccianti, Guido ; Terzi, Silvia ; Trovarelli, Giulia ; Franchi, Alessandro ; Picci, Piero ; Ferrari, Stefano. / Denosumab in patients with aneurysmal bone cysts: A case series with preliminary results. In: Tumori. 2018 ; Vol. 104, No. 5. pp. 344-351.
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T1 - Denosumab in patients with aneurysmal bone cysts: A case series with preliminary results

AU - Palmerini, Emanuela

AU - Ruggieri, Pietro

AU - Angelini, Andrea

AU - Boriani, Stefano

AU - Campanacci, Domenico

AU - Milano, Giuseppe M

AU - Cesari, Marilena

AU - Paioli, Anna

AU - Longhi, Alessandra

AU - Abate, Massimo E

AU - Scoccianti, Guido

AU - Terzi, Silvia

AU - Trovarelli, Giulia

AU - Franchi, Alessandro

AU - Picci, Piero

AU - Ferrari, Stefano

PY - 2018/8/8

Y1 - 2018/8/8

N2 - 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.

AB - 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.

KW - Aneurysmal bone cyst

KW - bone tumor

KW - denosumab

KW - receptor activator of nuclear factor-kappa B ligand

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JO - Tumori

JF - Tumori

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