Ependymal tumors are rare CNS tumors and may occur at any age, but their proportion among primary brain tumors is highest in children and young adults. Thus, the level of evidence of diagnostic and therapeutic interventions is higher in the pediatric compared with the adult patient population.The diagnosis and disease staging is performed by craniospinal MRI. Tumor classification is achieved by histological and molecular diagnostic assessment of tissue specimens according to the World Health Organization (WHO) classification 2016. Surgery is the crucial initial treatment in both children and adults. In pediatric patients with intracranial ependymomas of WHO grades II or III, surgery is followed by local radiotherapy regardless of residual tumor volume. In adults, radiotherapy is employed in patients with anaplastic ependymoma WHO grade III, and in case of incomplete resection of WHO grade II ependymoma. Chemotherapy alone is reserved for young children <12 months and for adults with recurrent disease when further surgery and irradiation are no longer feasible. A gross total resection is the mainstay of treatment in spinal ependymomas, and radiotherapy is reserved for incompletely resected tumors. Nine subgroups of ependymal tumors across different anatomical compartments (supratentorial, posterior fossa, spinal) and patient ages have been identified with distinct genetic and epigenetic alterations, and with distinct outcomes. These findings may lead to more precise diagnostic and prognostic assessments, molecular subgroup-adapted therapies, and eventually new recommendations pending validation in prospective studies.

https://www.zora.uzh.ch/id/eprint/153708/1/Ruda_2017.pdf

EANO guidelines for the diagnosis and treatment of ependymal tumors.

Mapping from health status measures onto generic preference-based measures is becoming a common solution when health state utility values are not directly available for economic evaluation. However the accuracy and reliability of the models employed is largely untested, and there is little evidence of their suitability in patient datasets. This paper examines whether mapping approaches are reliable and accurate in terms of their predictions for a large and varied UK patient dataset. SF-36 dimension scores are mapped onto the EQ-5D index using a number of different model specifications. The predicted EQ-5D scores for subsets of the sample are compared across inpatient and outpatient settings and medical conditions. This paper compares the results to those obtained from existing mapping functions. Our results suggest that models mapping the SF-36 onto the EQ-5D have similar predictions across inpatient and outpatient setting and medical conditions. However, the models overpredict for more severe EQ-5D states; this problem is also present in the existing mapping functions.

Mapping SF-36 onto the EQ-5D index: how reliable is the relationship?

PURPOSE\nRadiation Therapy Oncology Group trial 0525 tested whether dose-intensifying temozolomide versus standard chemoradiotherapy improves overall survival (OS) or progression-free survival (PFS) in newly diagnosed glioblastoma. Tests of neurocognitive function (NCF) and symptoms (using the MD Anderson Symptom Inventory-Brain Tumor module; MDASI-BT) and of quality of life (European Organisation for the Research and Treatment of Cancer Quality of Life Questionnaire [EORTC QLQ] -C30/BN20) examined the net clinical benefit (NCB) of therapy.\n\n\nPATIENTS AND METHODS\nNCF tests (Hopkins Verbal Learning Test-Revised, Trail Making Test, and Controlled Oral Word Association), MDASI-BT, and EORTC QLQ-C30/BN20 were completed in a subset of patients. Multivariate Cox proportional hazard regression modeling determined the prognostic value of baseline and early change from baseline to cycle 1 for OS and PFS. Two-sample proportional test statistic was used to evaluate differences between treatments (dose-dense v standard-dose) on NCB measures from baseline to cycle 4 in stable patients.\n\n\nRESULTS\nOverall, 182 patients participated in the study. Baseline NCF tests and the physical functioning quality of life scale were associated with OS and PFS. Baseline to cycle 1 in all NCB components were associated with OS and PFS. There was greater deterioration in the dose-dense arm from baseline to cycle 4 in the Global Health and Motor Function subscales (EORTC QLQ-C30/BN20) as well as in overall symptom burden, overall symptom interference, and activity-related symptom interference subscales (MDASI-BT). There were no between-arm differences in NCF.\n\n\nCONCLUSION\nLongitudinal collection of NCB measures is feasible in cooperative group studies and provides an added dimension to standard outcome measures. Greater adverse symptom burden and functional interference, as well as decreased global health and motor function were observed in patients randomly assigned to the dose-dense arm. Baseline and early change in NCB measures were associated with decreased rates of survival.

Net clinical benefit analysis of radiation therapy oncology group 0525

Mapping is an increasingly common method used to predict instrument-specific preference-based health-state utility values (HSUVs) from data obtained from another health-related quality of life (HRQoL) measure. There have been several methodological developments in this area since a previous review up to 2007. To provide an updated review of all mapping studies that map from HRQoL measures to target generic preference-based measures (EQ-5D measures, SF-6D, HUI measures, QWB, AQoL measures, 15D/16D/17D, CHU-9D) published from January 2007 to October 2018. A systematic review of English language articles using a variety of approaches: searching electronic and utilities databases, citation searching, targeted journal and website searches. Full papers of studies that mapped from one health measure to a target preference-based measure using formal statistical regression techniques. Undertaken by four authors using predefined data fields including measures, data used, econometric models and assessment of predictive ability. There were 180 papers with 233 mapping functions in total. Mapping functions were generated to obtain EQ-5D-3L/EQ-5D-5L-EQ-5D-Y (n = 147), SF-6D (n = 45), AQoL-4D/AQoL-8D (n = 12), HUI2/HUI3 (n = 13), 15D (n = 8) CHU-9D (n = 4) and QWB-SA (n = 4) HSUVs. A large number of different regression methods were used with ordinary least squares (OLS) still being the most common approach (used ≥ 75% times within each preference-based measure). The majority of studies assessed the predictive ability of the mapping functions using mean absolute or root mean squared errors (n = 192, 82%), but this was lower when considering errors across different categories of severity (n = 92, 39%) and plots of predictions (n = 120, 52%). The last 10 years has seen a substantial increase in the number of mapping studies and some evidence of advancement in methods with consideration of models beyond OLS and greater reporting of predictive ability of mapping functions.

/pdf/an-updated-systematic-review-of-studies-mapping-or-cross-4vu1llrkgg.pdf

An Updated Systematic Review of Studies Mapping (or Cross-Walking) Measures of Health-Related Quality of Life to Generic Preference-Based Measures to Generate Utility Values.

Background Laser Ablation of Abnormal Neurological Tissue using Robotic NeuroBlate System (LAANTERN) is an ongoing multicenter prospective NeuroBlate (Monteris Medical) LITT (laser interstitial thermal therapy) registry collecting real-world outcomes and quality-of-life (QoL) data. Objective To compare 12-mo outcomes from all subjects undergoing LITT for intracranial tumors/neoplasms. Methods Demographics, intraprocedural data, adverse events, QoL, hospitalizations, health economics, and survival data are collected; standard data management and monitoring occur. Results A total of 14 centers enrolled 223 subjects; the median follow-up was 223 d. There were 119 (53.4%) females and 104 (46.6%) males. The median age was 54.3 yr (range 3-86) and 72.6% had at least 1 baseline comorbidity. The median baseline Karnofsky Performance Score (KPS) was 90. Of the ablated tumors, 131 were primary and 92 were metastatic. Most patients with primary tumors had high-grade gliomas (80.9%). Patients with metastatic cancer had recurrence (50.6%) or radiation necrosis (40%). The median postprocedure hospital stay was 33.4 h (12.7-733.4). The 1-yr estimated survival rate was 73%, and this was not impacted by disease etiology. Patient-reported QoL as assessed by the Functional Assessment of Cancer Therapy-Brain was stabilized postprocedure. KPS declined by an average of 5.7 to 10.5 points postprocedure; however, 50.5% had stabilized/improved KPS at 6 mo. There were no significant differences in KPS or QoL between patients with metastatic vs primary tumors. Conclusion Results from the ongoing LAANTERN registry demonstrate that LITT stabilizes and improves QoL from baseline levels in a malignant brain tumor patient population with high rates of comorbidities. Overall survival was better than anticipated for a real-world registry and comparative to published literature.

Laser Ablation of Abnormal Neurological Tissue Using Robotic NeuroBlate System (LAANTERN): 12-Month Outcomes and Quality of Life After Brain Tumor Ablation.

BACKGROUND
Outcomes projects can be a catalyst for determining disease- and treatment-related consequences for patients with rare tumors. The Adult Ependymoma Outcomes (AEO) survey uses self-reported experience to evaluate how this tumor affects patient groups throughout the illness trajectory.

METHODS
Patients completed the AEO survey via a Web-based portal. The survey included questions on treatment, tumor recurrence, and current health status; the MD Anderson Symptom Inventory Brain Tumor and Spine Tumor modules; and the Medical Outcomes Study 36-Item Short Form Health Survey (SF-36).

RESULTS
The sample included 264 participants (57% female) with a median age of 46 years (range, 18-77 years). Radiation treatment was commonly used for patients who had brain involvement (χ2(1) = 20.7; P < .001), underwent a partial resection (43%; χ2(3) = 15.4; P < .001), or had a grade 3 tumor (41%; χ2(2) = 18.8; P < .001). Recurrence occurred in a small group (29%), with grade 1 tumor patients 2.6 times more likely and grade 3 tumor patients 2.5 times more likely to experience recurrence than those with grade 2 tumors. Spine tumor patients had a higher symptom burden (mean, 2.8; scale, 0-10) than brain tumor patients (t(247) = −4.0), and they reported more moderate to severe symptoms (rating ≥ 5; 29%) than their counterparts (18%). Within the physical health portion of the SF-36, spine tumor patients reported worse health with respect to bodily pain (t(249) = 6.8; P < .001), physical functioning (t(252) = 4.1; P < .001), and vitality (t(202.2) = 3.0; P < .003).

CONCLUSIONS
These results demonstrate the feasibility of implementing outcomes projects that report on the clinical and demographic characteristics of a rare patient population, and they underscore the importance of outcomes data in understanding disease-related issues. Cancer 2016. © 2016 American Cancer Society.

Clinical presentation and outcomes for adult ependymoma patients.

Background Patients with glioma are highly symptomatic and often have functional limitations from the time of diagnosis. Measuring health status may have value in determining impact of disease. This study provided a description of health status and utility scores in glioma patients throughout the illness trajectory using the EQ-5D (a functional measure of general health status). Furthermore, it evaluated the information provided by the MD Anderson Symptom Inventory-Brain Tumor (MDASI-BT; a measure of symptom burden and interference) in describing health-related quality of life as assessed by the EQ-5D. Methods Glioma patients completed the EQ-5D and MDASI-BT. Disease and clinical details were collected by medical record review. Linear regression evaluated whether MDASI-BT scores adequately predict patient health outcomes measured by the EQ-5D. Results The sample included 100 patients (65% male, 78% with a glioblastoma, median age 52 [range, 20-75], 56% in active treatment). Seventy-two percent of patients reported functional limitations in at least 1 area. Extreme cases reported inability to perform usual activities (8%) and significant anxiety/depression (5%). The MDASI-BT neurologic factor and activity-related interference (walking/activity/work) explained 52% of the variability in the EQ-5D in this patient population while adjusting for the effect of tumor grade, recurrence status, and performance status. Conclusions The majority of glioma patients reported at least 1 functional limitation on the EQ-5D. Over half of the variance in the EQ-5D was explained by the MDASI-BT, performance status, tumor grade, and recurrence status. The resultant model demonstrates the significant contribution of symptom burden on health status in glioma patients.

Relationship between symptom burden and health status: analysis of the MDASI-BT and EQ-5D.

2018Background: Fatigue is a consistently reported severe symptom among patients with gliomas throughout the disease trajectory. Clinical variables and genomic pathways associated with fatigue in g...

Alvina A. Acquaye

Papers

Clinical presentation and outcomes for adult ependymoma patients.

Relationship between symptom burden and health status: analysis of the MDASI-BT and EQ-5D.

Investigation of risk factors associated with fatigue in glioma patients.