Citations 2
Establishing the content validity of patient-reported outcome measures used in neuro-oncology based on the WHO ICF framework: Part of the RANO-PRO initiative
Asogwa, Ogechukwu A , Walbert, Tobias , Arons, David, Blakeley, Jaishri O. , Coomans, Marijke B , Brown, Paul D. , Bulbeck, Helen , Chang, Susan M. , Coens, Corneel , Grant, Robin, Jalali, Rakesh , Koekkoek, Johan A F , Panda, Pankaj Kumar , Leach, Danielle, Mendoza, Tito R. , Nayak, Lakshmi , Oliver, Kathy , Reijneveld, Jaap C. , Taylor, Jennie , Wen, Patrick Y. , Taphoorn, Martin , Walbert, Tobias , Blakeley, Jaishri O , Bulbeck, Helen , Gilbert, Mark R , Grant, Robin , Jalali, Rakesh , Nayak, Lakshmi , Le Rhun, Emilie , Taylor, Jennie W , Weller, Michael , Mendoza, Tito , Koekkoek, Johan A F , Dirven, Linda , Armstrong, Terri S. , Bent, Martin J. van den , Chang, Susan Marina , Gilbert, Mark R. , Leeper, Heather , Rhun, Émilie Le , Rubinstein, Larry , Wen, Patrick Yung
Neuro-Oncology | April 28, 2025 | Volume: 27 | Issue: 9 | Pages: 2399-2414
Abstract Background Instruments to assess patient-reported outcomes (PRO) should generate high-quality evidence. Reliable PRO evidence is essential to policymakers, in conjunction with outcomes such as survival and radiological response, to understand the net clinical benefit of antitumor treatments. This study aimed to establish the content validity of 215 identified PRO measures used in patients with brain tumors. Methods A survey (n = 148 items) was developed reflecting aspects of the WHO International Classification of Functioning, Disability, and Health (ICF) framework. Patients with brain tumors, their proxies, and healthcare professionals (HCPs) were asked to rate each survey item on relevance. An item was considered a relevant issue if ≥25% of the patients or proxies or ≥50% of the HCPs considered that item to be an issue. Next, all items in the identified PRO measures were linked to ICF and relevant items in the survey, and the percentage of content coverage was calculated. Results In total, 114 patients, 71 proxies, and 65 HCPs from different countries completed the survey. Fifty-six of 148 (37.8%) items in the survey were considered relevant. The most important aspects mentioned by both patients and proxies were difficulty concentrating, difficulty remembering, multitasking, and handling stress. Depending on the definition, between 35% and 49% of PRO measures were considered to have sufficient content validity (≥80% coverage). Conclusion The content validity was insufficient in more than half of the identified PRO measures, particularly multidimensional measures. Future research should investigate whether different approaches to PRO assessment better meet the needs of all stakeholders.
Systematic review on the use of patient-reported outcome measures in brain tumor studies: part of the Response Assessment in Neuro-Oncology Patient-Reported Outcome (RANO-PRO) initiative
Dirven, Linda , Vos, Maartje E, Walbert, Tobias , Arons, David, Bent, Martin J. van den , Brown, Paul D. , Chang, Susan M. , Coens, Corneel , Grant, Robin, Jalali, Rakesh , Leach, Danielle, Mendoza, Tito R. , Nayak, Lakshmi , Oliver, Kathy , Reijneveld, Jaap C. , Wen, Patrick Y. , Taphoorn, Martin , Mendoza, Tito , Weller, Michael , van den Bent, Martin J , Armstrong, Terri S. , Blakeley, Jaishri O. , Bulbeck, Helen , Gilbert, Mark R. , Leeper, Heather , Rhun, Émilie Le , Rubinstein, Larry , Wen, Patrick Yung
Neuro-Oncology Practice | February 14, 2021 | Volume: 8 | Issue: 4 | Pages: 417-425
Abstract Background The Response Assessment in Neuro-Oncology Patient-Reported Outcome (RANO-PRO) working group aims to provide guidance on the use of PROs in brain tumor patients. PRO measures should be of high quality, both in terms of relevance and other measurement properties. This systematic review aimed to identify PRO measures that have been used in brain tumor studies to date. Methods A systematic literature search for articles published up to June 25, 2020 was conducted in several electronic databases. Pre-specified inclusion criteria were used to identify studies using PRO measures assessing symptoms, (instrumental) activities of daily living [(I)ADL] or health-related quality of life (HRQoL) in adult patients with glioma, meningioma, primary central nervous system lymphoma, or brain metastasis. Results A total of 215 different PRO measures were identified in 571 published and 194 unpublished studies. The identified PRO measures include brain tumor-specific, cancer-specific, and generic instruments, as well as instruments designed for other indications or multi- or single-item study-specific questionnaires. The most frequently used instruments were the EORTC QLQ-C30 and QLQ-BN20 (n = 286 and n = 247), and the FACT-Br (n = 167), however, the majority of the instruments were used only once or twice (150/215). Conclusion Many different PRO measures assessing symptoms, (I)ADL or HRQoL have been used in brain tumor studies to date. Future research should clarify whether these instruments or their scales/items exhibit good content validity and other measurement properties for use in brain tumor patients.