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Cited by 32Sarcoma Diagnosis and TreatmentBone Tumor Diagnosis and TreatmentsOvarian cancer diagnosis and treatment

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Off-label use of targeted therapies in osteosarcomas: data from the French registry OUTC’S (Observatoire de l’Utilisation des Thérapies Ciblées dans les Sarcomes)

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Penel-Page, Mathilde, Ray-Coquard, Isabelle, Larcade, Julie, Girodet, Magali, Bouclier, Laure, Rogasik, Muriel, Corradini, Nadège, Entz-Werle, Natacha, Brugieres, Laurence, Domont, Julien, Lervat, Cyril, Piperno-Neumann, Sophie, Pacquement, Helène, Bay, Jacques-Olivier, Gentet, Jean-Claude, Thyss, Antoine, Chaigneau, Loic, Narciso, Bérangère, Cornille, Helène, Blay, Jean-Yves, Marec-Bérard, Perrine·2015·BMC Cancer
DOI 10.1186/s12885-015-1894-5PMC PMC4635968
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Abstract

BACKGROUND: The objective of this study is to explore the off-label use of targeted therapies (TTs) for patients with osteosarcoma registered within the French Sarcoma Group--Bone Tumor Study Group (GSF-GETO) national registry. METHODS: All patients with an osteosarcoma, registered between January 1, 2009 and July 15, 2013 were analyzed. RESULTS: Twenty-nine patients with refractory relapsed osteosarcomas received 33 treatment lines of TTs. The median age at the beginning of treatment was 19 years (range 9-72). The median number of previous lines of chemotherapy was 3 (range 1-8). Before inclusion, 3 patients were in second complete remission, 26 were in progression for metastatic relapse. Twenty-three patients received sirolimus (in combination with cyclophosphamide for 18); 5, sunitinib; 4, sorafenib; and one, pazopanib. Stable disease was observed for 45.5% of patients (95% Confidence Interval (CI) [20-52.8]). The median Progression-Free Survival (PFS) was 3 months (95% CI [2-5.4]) for patients treated by sirolimus and 1.8 months (95% CI [1.3-2.8]) for patients receiving multi-targeted tyrosine kinase inhibitors; 6-month PFS 15%. The median Overall Survival (OS) was 6.8 months (95% CI [4.7-12.1]), and one-year OS was 24%. In a multivariate analysis, PFS was superior for patients receiving sirolimus compared to other TTs (Hazard Ratio (HR) = 2.7, 95% CI [1.05-7.1]). No toxic death was reported. Grade 3 and 4 toxicities were observed in 27 and 6% of cases respectively. CONCLUSION: Off-label TTs, especially sirolimus, reported benefit in the treatment of refractory osteosarcomas with an acceptable toxicity profile, including in pediatric population.

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  • Sunitinib has been explored off-label for other types of bone sarcoma, including osteosarcoma, showing some activity based on retrospective analyses.

    “it has been explored off‐label for other types of BS, including osteosarcoma, showing some activity based on retrospective analyses. 47”

    From Nivolumab and sunitinib in patients with advanced bone sarcomas: A multicenter, single‐arm, phase 2 trial (2025) · cites this paper as [47]

  • There are no controlled trials to date assessing sunitinib as monotherapy in bone sarcoma.

    “there are no controlled trials to date assessing sunitinib as monotherapy in BS”

    From Nivolumab and sunitinib in patients with advanced bone sarcomas: A multicenter, single‐arm, phase 2 trial (2025) · cites this paper as [47]

    The Discussion (page unavailable) lists preclinical sunitinib work and phase I and II studies, but does not state that no controlled trial of sunitinib monotherapy in bone sarcoma existed. The Results report sunitinib use in the registry, which does not establish that broader absence; searches for controlled trials and monotherapy found no decisive passage. (LLM, conf=0.72)

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Metadata

Authors
Penel-Page, M., Ray-Coquard, I., Larcade, J., Girodet, M., Bouclier, L., Rogasik, M., Corradini, N., Entz-Werle, N., Brugieres, L., Domont, J., Lervat, C., Piperno-Neumann, S., Pacquement, H., Bay, J.-O., Gentet, J.-C., Thyss, A., Chaigneau, L., Narciso, B., Cornille, H., Blay, J.-Y., & Marec-Bérard, P.
Affiliations
  • Lyon 1 Université (FR)
  • Centre Léon Bérard (FR)
  • Hôpital Mère-Enfant (FR)
  • Centre Oscar Lambret (FR)
  • Institut Curie (FR)
  • Centre Jean Perrin (FR)
  • Hôpital de la Timone (FR)
  • Centre Antoine Lacassagne (FR)
  • Centre Hospitalier Universitaire de Besançon (FR)
  • Centre Hospitalier Universitaire de Tours (FR)
ORCID
Venue
BMC Cancer
Publisher
Springer Science and Business Media LLC
Published
2015-11-05
Volume
15
Issue
1
Pages
854-854
ISSN
1471-2407 (ISSN-L)
DOI
10.1186/s12885-015-1894-5
PMC
PMC4635968
OpenAlex
W2125474267
Cited by
32
References
35
Citations by year
2025: 1 · 2024: 2 · 2023: 4 · 2022: 4 · 2021: 5 · 2020: 4 · 2019: 2 · 2018: 4 · 2017: 2 · 2016: 4
FWCI
2.64
Type
journal article
Language
en
Open access
Licence
cc-by
Locations
  • BMC Cancer (publishedVersion, cc-by)
  • PubMed Central (publishedVersion, cc-by)
  • PubMed (publishedVersion)
  • HAL (submittedVersion)
Funders
  • Pfizer France
  • Institut National du Cancer (NetSarc, InCa
Topics
  • Health Sciences › Medicine › Oncology › Sarcoma Diagnosis and Treatment
  • Health Sciences › Medicine › Orthopedics and Sports Medicine › Bone Tumor Diagnosis and Treatments
  • Health Sciences › Medicine › Oncology › Ovarian cancer diagnosis and treatment
Keywords
osteosarcoma, off-label drug use, targeted therapy, rapamycin, sunitinib, sorafenib, pazopanib, tyrosine kinase inhibitors, progression-free survival, overall survival, treatment toxicity, pediatric patients
MeSH terms
Adolescent, Adult, Aged, Antineoplastic Agents, Antineoplastic Combined Chemotherapy Protocols, Child, Female, Follow-Up Studies, France, Humans, Male, Middle Aged, Neoplasm Recurrence, Local, Neoplasm Staging, Registries, Osteosarcoma, Treatment Outcome, Retreatment, Young Adult, Off-Label Use, Molecular Targeted Therapy, Neoplasm Grading

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