Preprint Review Version 1 Preserved in Portico This version is not peer-reviewed

Desmoplastic Small Round Cell Tumor: A Review of Main Molecular Abnormalities and Emerging Therapy

Celso Abdon Mello
* ,
Fernando Augusto Batista Campos
,
Tiago Goss Santos
ORCID logo ,
Maria Leticia Gobo Silva
,
Giovana Tardin Torrezan
ORCID logo ,
Felipe D´Almeida Costa
,
Maria Nirvana Formiga
,
Ulisses Nicolau
,
Antonio Geraldo Nascimento
,
Cassia Silva
,
Suely Nakagawa
ORCID logo ,
Maria Paula Curado
,
Ademar Lopes
,
Samuel Aguiar
Version 1 : Received: 10 December 2020 / Approved: 11 December 2020 / Online: 11 December 2020 (15:53:20 CET)

A peer-reviewed article of this Preprint also exists.

Mello, C.A.; Campos, F.A.B.; Santos, T.G.; Silva, M.L.G.; Torrezan, G.T.; Costa, F.D.; Formiga, M.N.; Nicolau, U.; Nascimento, A.G.; Silva, C.; Curado, M.P.; Nakagawa, S.A.; Lopes, A.; Aguiar, S., Jr. Desmoplastic Small Round Cell Tumor: A Review of Main Molecular Abnormalities and Emerging Therapy. Cancers 2021, 13, 498. Mello, C.A.; Campos, F.A.B.; Santos, T.G.; Silva, M.L.G.; Torrezan, G.T.; Costa, F.D.; Formiga, M.N.; Nicolau, U.; Nascimento, A.G.; Silva, C.; Curado, M.P.; Nakagawa, S.A.; Lopes, A.; Aguiar, S., Jr. Desmoplastic Small Round Cell Tumor: A Review of Main Molecular Abnormalities and Emerging Therapy. Cancers 2021, 13, 498.

Abstract

Desmoplastic small round cell tumor (DSRCT) is an extremely rare, aggressive sarcoma affecting adolescents and young adults with male predominance. Generally, it originates from serosal surface of abdominal cavity. The hallmark characteristic of DSRCT is the EWSR1-WT1 gene fusion. This translocation up-regulates the expression of PDGFRα, VEGF and other proteins related to tumor and vascular cell proliferation. Current management of DSRCT includes a combination of chemotherapy, radiation and aggressive cytoreductive surgery plus intra-peritoneal hyperthermic chemotherapy (HIPEC). Despite advances in multimodal therapy, outcomes remain poor since the majority of patients present disease recurrence and die within 3 years. The dismal survival makes DSRCT an orphan disease with urgent need of new drugs. The treatment of advanced and recurrent disease with tyrosine kinase inhibitors, such as pazopanib, sunitinib, and mTOR inhibitors have been evaluated in small studies. Recent works using comprehensive molecular profiling of DSRCT identified potential therapeutic targets. In this review, we aim to describe the current studies conducted to better understand DSRCT biology and to explore the new therapeutic strategies under investigation in preclinical models and in early phase clinical trials.

Keywords

desmoplastic small round cell tumor; treatment; prognosis; surgery; radiotherapy; chemotherapy; tyrosine kinase receptor; target therapy; rare disease

Subject

Medicine and Pharmacology, Immunology and Allergy

Copyright: This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.

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