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Prospects and pitfalls of personalizing therapies for sarcomas: from children, adolescents, and young adults to the elderly.

ABSTRACT: Sarcomas are a heterogeneous class of tumors that affect all ages, from children, adolescents, and young adults to the elderly. Within this panoply of tumor subtypes lies the opportunity to bring to bear a vision of personalized medicine in which the fast-paced evolution from the "one gene, one test, one drug" approach to a comprehensive "panomic," multiplex, multianalyte method coupled with advances in bioinformatics platforms can unravel the biology of this disease. The increasingly enlarging repertoire of novel agents provides innumerable prospects in precision medicine. Personalized therapy covers the entire spectrum of cancer care, from risk factor assessment through prevention, risk reduction, therapy, follow-up after therapy, and survivorship care. Challenges remain in implementing the science of precision medicine in the clinic, including providing comprehensive multidisciplinary care and overcoming regulatory and economic hurdles, which must be facilitated within the collaborative framework of academia, industry, federal regulators, and third-party payers.

SUBMITTER: Subbiah V

PROVIDER: S-EPMC4144039 | biostudies-literature | 2014 Sep

REPOSITORIES: biostudies-literature

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Prospects and pitfalls of personalizing therapies for sarcomas: from children, adolescents, and young adults to the elderly.

Subbiah Vivek V

Current oncology reports 20140901 9

Sarcomas are a heterogeneous class of tumors that affect all ages, from children, adolescents, and young adults to the elderly. Within this panoply of tumor subtypes lies the opportunity to bring to bear a vision of personalized medicine in which the fast-paced evolution from the "one gene, one test, one drug" approach to a comprehensive "panomic," multiplex, multianalyte method coupled with advances in bioinformatics platforms can unravel the biology of this disease. The increasingly enlarging ...[more]

PMID: 25030655

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Project description:Some subsets of pediatric sarcoma patients have very poor survival rates. We sought to determine the feasibility and efficacy of allogeneic hematopoietic stem cell transplantation (alloHSCT) in pediatric sarcoma populations with <25% predicted overall survival (OS). Patients with ultrahigh-risk Ewing's sarcoma family of tumors (ESFT), alveolar rhabdomyosarcoma, or desmoplastic small round cell tumors received EPOCH-fludarabine induction, a cyclophosphamide/fludarabine/melphalan preparative regimen, and HLA matched related peripheral blood stem cells. Thirty patients enrolled; 7 did not undergo alloHSCT because of progressive disease with diminishing performance status during induction. All 23 alloHSCT recipients experienced rapid full-donor engraftment, with no peritransplantation mortality. Five of 23 alloHSCT recipients (22%) remain alive (OS of 30% by Kaplan-Meier analysis at 3 years), including 3 of 7 (42%) transplanted without overt disease (median survival 14.5 versus 29.0 months from alloHSCT for patients transplanted with versus without overt disease, respectively). Among the 28 patients who progressed on the study, the median survival from date of progression was 1.9 months for the 7 who did not receive a transplant compared with 11.4 months for the 21 transplanted (P = .0003). We found prolonged survival after posttransplantation progression with several patients exhibiting indolent tumor growth. We also saw several patients with enhanced antitumor effects from posttransplantation chemotherapy (objective response to pretransplantation EPOCH-F was 24% versus 67% to posttransplantation EOCH); however, this was associated with increased toxicity. This largest reported series of alloHSCT in sarcomas demonstrates that alloHSCT is safe in this population, and that patients undergoing alloHSCT without overt disease show higher survival rates than reported using standard therapies. Enhanced chemo- and radiosensitivity of tumors and normal tissues was observed posttransplantation.

Dataset Information

Prospects and pitfalls of personalizing therapies for sarcomas: from children, adolescents, and young adults to the elderly.

Publications

Prospects and pitfalls of personalizing therapies for sarcomas: from children, adolescents, and young adults to the elderly.

Similar Datasets

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