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Multicenter Study
. 2017 Nov 6;17(1):717.
doi: 10.1186/s12885-017-3727-1.

Treatment of gastrointestinal stromal tumours in paediatric and young adult patients with sunitinib: a multicentre case series

Piotr Rutkowski  1 , Heather Magnan  2 , Alexander J Chou  2 , Charlotte Benson  3
Affiliations
Multicenter Study

Treatment of gastrointestinal stromal tumours in paediatric and young adult patients with sunitinib: a multicentre case series

Piotr Rutkowski et al. BMC Cancer. .

Abstract

Background: Gastrointestinal stromal tumours (GIST) are rarely encountered mesenchymal tumours of the gastrointestinal tract (1.5 people per 100,000/year) that are even more rarely seen in paediatric patients (1-2% of all cases). The standard treatment for advanced adult GIST is imatinib with sunitinib as a second-line option. Although the efficacy and tolerability of sunitinib in adults with GIST has been established, little is known of the profile of sunitinib in paediatric/young adult patients with GIST given the rarity of this disease.

Methods: Paediatric/young adult patients aged up to 21 years with diagnosis of GIST who were treated with sunitinib were identified from retrospective records from three centres in Europe and the US. Most patients commenced sunitinib in a 6-week cycle, however, dosing could be reduced, delayed, changed to (or initiated with) a continuous schedule. Objective response (Response Evaluation Criteria In Solid Tumours [RECIST]) and adverse events were recorded.

Results: We identified 9 paediatric/young adult patients (aged 11-21 years) with GIST who were treated with sunitinib de novo (n = 1) or following failure of imatinib (n = 8). Progressive disease was previously documented for all patients including 7 patients during imatinib therapy. Baseline patient and tumour profile characteristics showed a distinct profile (notably all were wild-type KIT/PDGFR) compared to that established for adults. Sunitinib treatment was associated with a best response of stable disease for 7 patients, with disease stabilisation lasting from 1 month to >73 months and a median progression free survival time of 15 months. There was some evidence of better disease control for sunitinib when compared to prior imatinib. Most adverse events with sunitinib were manageable and all were consistent with the known profile of the agent.

Conclusion: The ability to draw firm conclusions from this case series is limited by the small number of patients and the use of retrospective data which is largely reflective of the rarity of this condition. However, our findings provide initial evidence of clinical benefit and a generally manageable toxicity profile for sunitinib when administered to paediatric/young adult patients with GIST, most of whom had documented progressive disease during prior imatinib treatment.

Keywords: Case series; GIST; Paediatric; Sunitinib; Young adult.

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Conflict of interest statement

Ethics approval and consent to participate

This study was approved by the ethics committees of (1) Maria Skłodowska-Curie Institute – Oncology Center, Warsaw, Poland; (2) Memorial Sloan Kettering Cancer Center, New York, New York, USA. (3) The Royal Marsden Hospital, London, UK. As this was not an interventional study but a retrospective analysis of a case series, ethical approval was provided from the appropriate Ethics Committee of each participating institution to release this data without patient consent as patient consent was deemed unnecessary.

Consent for publication

Not applicable. All data are anonymized.

Competing interests

PR – Honoraria for lectures (Novartis, Pfizer) and Advisory Board Membership (Novartis, Bayer).

HM – None to declare.

AJC – None to declare.

CB – Travel grants (Pfizer).

Publisher’s Note

Springer Nature remains neutral with regard to jurisdictional claims in published maps and institutional affiliations.

References

    1. Miettinen M, Lasota J. Gastrointestinal stromal tumors: pathology and prognosis at different sites. Semin Diagn Pathol. 2006;23:70–83. doi: 10.1053/j.semdp.2006.09.001. - DOI - PubMed
    1. Gatta G, van der Zwan JM, Casali PG, Siesling S, Dei Tos AP, Kunkler I, et al. Rare cancers are not so rare: the rare cancer burden in Europe. Eur J Cancer. 2011;47(17):2493–2511. doi: 10.1016/j.ejca.2011.08.008. - DOI - PubMed
    1. Pappo AS, Janeway KA. Pediatric gastrointestinal stromal tumors. Hematol Oncol Clin North Am. 2009;23:15–34. doi: 10.1016/j.hoc.2008.11.005. - DOI - PubMed
    1. The ESMO/European Sarcoma Network Working Group Gastrointestinal stromal tumours: ESMO clinical practice guidelines for diagnosis, treatment and follow-up. Ann Oncol. 2014;25(Suppl 3):iii21–iii26. doi: 10.1093/annonc/mdu255. - DOI - PubMed
    1. Verweij J, Casali PG, Zalcberg J, LeCesne A, Reichardt P, Blay JY, et al. Progression-free survival in gastrointestinal stromal tumors with high-dose imatininb: randomised trial. Lancet. 2004;364:1127–1134. doi: 10.1016/S0140-6736(04)17098-0. - DOI - PubMed

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