Working plan for the use of patient-reported outcome measures in adults with brain tumours: a Response Assessment in Neuro-Oncology (RANO) initiative - 28/03/18

Doi : 10.1016/S1470-2045(18)30004-4

Linda Dirven, PhD ^a,^b, Terri S Armstrong, PhD ^c, Jaishri O Blakeley, MD ^d, Paul D Brown, MD ^e, Robin Grant, MD ^f, Rakesh Jalali, MD ^g, Heather Leeper, MD ^h, Tito Mendoza, MD ⁱ, Lakshmi Nayak, MD ^j, Jaap C Reijneveld, MD ^l,^m, Emilie Le Rhun, MD ^n,^o,^p, Tobias Walbert, MD ^q, Michael Weller, ProfMD ^r, Patrick Y Wen, ProfMD ^j,^k, Martin J B Taphoorn, ProfMD ^a,^b,⁎

^a Department of Neurology, Leiden University Medical Center, Leiden, Netherlands

^b Department of Neurology, Haaglanden Medical Center, The Hague, Netherlands

^c Center for Cancer Research, National Cancer Institute, Bethesda, MD, USA

^d Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, MD, USA

^e Department of Radiation Oncology, Mayo Clinic, Rochester, MN, USA

^f Edinburgh Centre for Neuro-Oncology, Western General Hospital, Edinburgh, UK

^g Neuro-Oncology Disease Management Group, Tata Memorial Centre, Mumbai, Maharashtra, India

^h Department of Neurology, NorthShore University Health System, Evanston, IL, USA

ⁱ Department of Symptom Research, The University of Texas MD Anderson Cancer Center, Houston, TX, USA

^j Center for Neuro-Oncology, Dana-Farber Cancer Institute, Boston, MA, USA

^k Department of Neurology, Brigham and Women’s Hospital, Boston, MA, USA

^l Department of Neurology and Brain Tumor Center Amsterdam, VU University Medical Center, Amsterdam, Netherlands

^m Department of Neurology, Academic Medical Center, Amsterdam, Netherlands

ⁿ Department of Neurosurgery, University Hospital, Lille, France

^o Department of Medical Oncology, Oscar Lambret Center, Lille, France

^p Inserm, U-1192, University of Lille, Lille, France

^q Department of Neurology and Neurosurgery, Henry Ford Health System, Detroit, MI, USA

^r Department of Neurology, University Hospital and University of Zurich, Zurich, Switzerland

^* Correspondence to: Prof Martin J B Taphoorn, Haaglanden Medical Center, Department of Neurology, PO Box 2191, 2501 VC The Hague, Netherlands Haaglanden Medical Center Department of Neurology PO Box 2191 The Hague VC 2501 Netherlands

Summary

The Response Assessment in Neuro-Oncology-Patient-Reported Outcome (RANO-PRO) working group is an international multidisciplinary collaboration that provides guidance on the use of patient-reported outcome (PRO) measures in clinical trials and practice for adult patients with brain tumours. Findings from both PROs and traditional outcome measures, such as survival, and clinical or radiological response, are essential to inform the research community, policy makers, physicians, and patients in the treatment decision-making process. Previous initiatives in oncology have focused on guidelines concerning the collection, analysis, interpretation, and reporting of PRO data. However, we recommend the application of appropriate PRO instruments, with respect to its content and measurement properties (ie, research question, content validity, and other measurement properties), in brain tumour research. PROs should be well defined and reliable to generate high-quality evidence, and our recommendations on the use of specific PRO measures could help to improve the quality of PRO evidence derived from neuro-oncological studies, and might add a new dimension in how the value of therapeutics is assessed in patients with brain tumours. In this Policy Review, we present the RANO-PRO working plan for the use of PROs in adults with brain tumours.

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