Observational studies: goldmines of information on rare diseases

BMC Medicine
R S Benjamin

Abstract

The article by Savina et al. from the large METASARC database of the French Sarcoma Group (BMC Med 15:78, 2017) provides a wealth of information about the natural history and therapy of patients with metastatic soft tissue sarcomas. The information complements - and in some cases surpasses - that obtained from randomized clinical trials, and should not be overlooked because of its retrospective nature. For rare diseases, retrospective data are often more important than data from randomized trials because of the inherent restrictions on sample size. The article provides clear information regarding the different behaviors of different histological types of sarcoma, the importance of localized therapy for metastatic disease, and the critical role of combination chemotherapy in initial treatment to improve survival.Please see related article: https://bmcmedicine.biomedcentral.com/articles/10.1186/s12916-017-0831-7.

References

Nov 4, 1992·JAMA : the Journal of the American Medical Association·UNKNOWN Evidence-Based Medicine Working Group
Jan 1, 1992·Journal of Clinical Oncology : Official Journal of the American Society of Clinical Oncology·J L SpeyerM Meyers
Feb 1, 1982·Annals of Internal Medicine·S S LeghaE J Freireich
Mar 2, 2002·European Journal of Cancer : Official Journal for European Organization for Research and Treatment of Cancer (EORTC) [and] European Association for Cancer Research (EACR)·M Van GlabbekeUNKNOWN EORTC Soft Tissue and Bone Sarcoma Group
Sep 23, 2011·The Lancet Oncology·Patrick SchöffskiUNKNOWN European Organisation for Research and Treatment of Cancer (EORTC) Soft Tissue and Bone Sarcoma Group (STBSG)
Mar 13, 2014·The Lancet Oncology·Ian JudsonUNKNOWN European Organisation and Treatment of Cancer Soft Tissue and Bone Sarcoma Group
Apr 4, 2014·The Lancet Oncology·Robert S Benjamin, J Jack Lee

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Citations

Aug 2, 2019·The Laryngoscope·Ester OrlandiPaolo Bossi
May 8, 2020·Journal of Adolescent and Young Adult Oncology·Eugenie YoungerWinette T A van der Graaf

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Software Mentioned

METASARC

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