Moving targets and ghosts of the past: outcome measurement in brain tumour therapy

Journal of Clinical Neuroscience : Official Journal of the Neurosurgical Society of Australasia
Stephen J Haines

Abstract

Evaluation of novel therapies for brain tumours should logically consider quality and quantity of patient survival as primary endpoints. The urgency of the problem, however, frequently leads investigators to use surrogate endpoints and historical controls in order to more rapidly evaluate outcome. To examine the impact of the use of surrogate endpoints and historical controls on the evaluation of innovative brain tumour therapy, selective literature review of three content areas (intraarterial chemotherapy for malignant glioma, interstitial brachytherapy for malignant glioma and stereotactic radiosurgery for cerebral metastasis and malignant glioma) was carried out. The impact of surrogate outcome measures and use of historical controls was assessed by comparing the results of trials using these methods and randomised clinical trials. In the evaluation of both intraarterial chemotherapy and interstitial brachytherapy, promising results in early phase trials were not confirmed in randomised clinical trials. This result can be explained by selection bias and predicted by the use of controls carefully selected from large treatment data bases. In the evaluation of stereotactic radiosurgery, early phase trials are promising, but ran...Continue Reading

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Citations

May 18, 2016·The Journal of Comparative Neurology·Christopher S von BartheldSuzana Herculano-Houzel
Oct 29, 2004·Neurosurgery·Arthur J UlmWilliam M Mendenhall
May 17, 2011·PM & R : the Journal of Injury, Function, and Rehabilitation·Susanne M SimmermanJean-Michel Brismée
Dec 6, 2006·Journal of Neuro-oncology·Susan ChangUNKNOWN American Association of Neurological Surgeons and Congress of Neurological Surgeons (AANS/CNS)
Apr 7, 2007·Journal of Neurosurgical Anesthesiology·Shailendra JoshiJohn Pile-Spellman
Sep 15, 2005·Mental Retardation and Developmental Disabilities Research Reviews·Mijna Hadders-Algra
May 20, 2009·Journal of Medical Economics·Simona de Portu, Lorenzo G Mantovani
Dec 21, 2002·Neurosurgery·Stephen J Haines
Mar 24, 2020·Journal of Anatomy·Nikolaus BenkoBrittany Coats
Apr 19, 2008·Journal of the History of the Neurosciences·Levent SarikciogluNurettin Oguz

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