NTRK rearranged sarcoma of the bone. Role for larotrectinib in the neoadjuvant setting of an ultra-rare tumor: a case report

Frontiers in Oncology

Oncology

Stefania Benini¹  Alberto Righi¹  Marco Gambarotti¹  Marco Miceli²  Alberto Bazzocchi²  Stefano Fanti³  Cristina Nanni³  Andrea Marrari⁴  Emanuela Palmerini⁴  Giorgio Frega⁴  Anna Paioli⁴  Toni Ibrahim⁴  Alessandra Longhi⁴  Marilena Cesari⁴  Rossella Hakim⁴  Tommaso Frisoni⁵  Davide Maria Donati⁶ 

[1] Department of Pathology, IRCCS Istituto Ortopedico Rizzoli, Bologna, Italy;Diagnostic and Interventional Radiology, IRCCS Istituto Ortopedico Rizzoli, Bologna, Italy;IRCCS Azienda Ospedaliero-Universitaria di Bologna, Policlinico Di Sant’Orsola, Bologna, Italy;Osteoncology, Soft Tissue and Bone Sarcomas, Innovative Therapy Unit, IRCCS Istituto Ortopedico Rizzoli, Bologna, Italy;Third Orthopaedic Clinic and Traumatology, IRCCS Istituto Ortopedico Rizzoli, Bologna, Italy;Third Orthopaedic Clinic and Traumatology, IRCCS Istituto Ortopedico Rizzoli, Bologna, Italy;Department of Biomedical and Neuromotor Sciences (DIBINEM), University of Bologna, Bologna, Italy;

关键词: neurotrophic tyrosine receptor kinase;    undifferentiated spindle cell sarcoma;    bone sarcoma;    larotrectinib;    entrectinib;    NTRK;   

DOI  :  10.3389/fonc.2023.1252359

 received in 2023-07-03, accepted in 2023-09-04,  发布年份 2023

来源: Frontiers

【 摘 要 】

Background: Neurotrophic tyrosine receptor kinase (NTRK) gene-fusion targeted molecules revolutionized the paradigm of treatment of a limited subgroup of cancers of various histologies. Entrectinib and larotrectinib obtained unprecedented response rates in patients with cancer harboring NTRK rearrangements. This evidence recently led to the agnostic approval of these drugs, and evidence (confirmation) of their activity in a broader disease setting is emerging. Here, we report the case of a patient affected by EML4-NTRK3 rearranged undifferentiated spindle cell bone sarcoma treated with larotrectinib, and we argue (discuss about) the incidence and clinical presentation of NTRK gene-fusion positive bone sarcomas, the potential use of upfront treatment with NTRK inhibitors in neoadjuvant setting, and the role of a multidisciplinary tumor board. Despite the rarity of these rearrangements in patients with primitive bone sarcomas, the therapy with NTRK inhibitors represents a highly effective strategy to be pursued in selected cases even in neoadjuvant settings. The management of these very rare cancers should always be discussed in a multidisciplinary board of reference centers.

【 授权许可】

Unknown   
Copyright © 2023 Palmerini, Frega, Gambarotti, Frisoni, Cesari, Bazzocchi, Miceli, Donati, Fanti, Nanni, Benini, Longhi, Paioli, Marrari, Hakim, Righi and Ibrahim