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Jeff Unger MD Dale C. Allison MD Margit Kaltoft MD Kavitha Lakkole MBA Jayant K. Panda MD Chethana Ramesh MSc Mehmet Sargin MD Elena Smolyarchuk MD Melissa Twine BS Benjamin Wolthers MD Gizem Yarimbas MSc Marouan Zoghbi MD the LIRA-PRIME investigators 《Diabetes, obesity & metabolism》2022,24(2):204-211
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Karakousis Giorgos C. Leong Stanley P. Zager Jonathan S. 《Clinical & experimental metastasis》2022,39(1):257-257
Clinical & Experimental Metastasis - 相似文献
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Andrea Ferrari MD Daniel Orbach MD Michela Casanova MD Max M. van Noesel MD Pablo Berlanga MD Bernadette Brennan MD Nadege Corradini MD Reineke A. Schoot MD Gema L. Ramirez-Villar MD Lisa Lyngsie Hjalgrim MD Rita Alaggio MD Gabriela Guillen Burrieza MD Akmal Safwat MD Alison L. Cameron MD Rick R. van Rijn MD Veronique Minard-Colin MD Ilaria Zanetti BSc Gianni Bisogno MD Julia C. Chisholm MD Johannes H. M. Merks MD 《Cancer》2023,129(16):2542-2552
Background
Limited data exist on the clinical behavior of pediatric non-rhabdomyosarcoma soft tissue sarcomas (NRSTS) with distant metastases at onset, and a clear standard of care has not yet been defined.Methods
This cohort study reports on pediatric adult-type metastatic NRSTS enrolled in two concurrent prospective European studies, i.e., the randomized BERNIE study and the single-arm MTS 2008 study developed by the European paediatric Soft tissue sarcoma Study Group. Treatment programs were originally designed for patients with metastatic rhabdomyosarcoma, i.e., nine courses of multidrug chemotherapy (with or without bevacizumab in the BERNIE study), followed by 12 cycles of maintenance therapy, whereas radiotherapy and/or surgery (on primary tumor and/or metastases) were delayed until after seven courses of chemotherapy had been administered.Results
The study included 61 patients <21 years old treated from July 2008 to December 2016. The lung was the site of metastases in 75% of the cases. All patients received multi-agent chemotherapy, 44% had local therapy to primary tumor, and 18% had treatment of metastases. Median time to progression/relapse was 6 months. A high rate of tumor progression was observed during the initial part of the chemotherapy program. With a median follow-up of 41.5 months (range, 2–111 months), 3-year event-free survival and overall survival were 15.4% (95% confidence interval [CI], 7.6–25.7) and 34.9% (95% CI, 22.7–47.5), respectively. There were no statistically significant differences in outcome depending on the type of treatment administered.Conclusions
The study confirmed the overall poor outcome for patients with metastatic NRSTS, whose treatment remains a challenge.Plain Language Summary
- Pediatric non-rhabdomyosarcoma soft tissue sarcomas form a heterogeneous group of rare tumors.
- Although recent international studies have defined the standard of care for patients with localized disease, limited data are available on the clinical behavior of patients with distant metastases.
- This study on 61 metastatic cases treated on two prospective European protocols confirms that the chances of survival of such patients are often dismal and a standard treatment is still lacking.
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Chang Patrick K. C. Prestidge Clive A. Bremmell Kristen E. 《Pharmaceutical research》2022,39(6):1151-1163
Pharmaceutical Research - Cationic polymers have many advantages as vectors for mediated cellular entry and delivery of siRNA. However, toxicity related to their cationic charge has compromised... 相似文献
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