Evaluating methodological quality of Prognostic models Including Patient-reported HeAlth outcomes in oncologY (EPIPHANY): A systematic review protocol

Introduction While there is mounting evidence of the independent prognostic value of patient-reported outcomes (PROs) for overall survival (OS) in patients with cancer, it is known that the conduct of these studies may hold a number of methodological challenges. The aim of this systematic review is to evaluate the quality of published studies in this research area, in order to identify methodological and statistical issues deserving special attention and to also possibly provide evidence-based recommendations. Methods and analysis An electronic search strategy will be performed in PubMed to identify studies developing or validating a prognostic model which includes PROs as predictors. Two reviewers will independently be involved in data collection using a predefined and standardised data extraction form including information related to study characteristics, PROs measures used and multivariable prognostic models. Studies selection will be reported following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines, with data extraction form using fields from the Critical Appraisal and Data Extraction for Systematic Reviews of Prediction Modelling Studies (CHARMS) checklist for multivariable models. Methodological quality assessment will also be performed and will be based on prespecified domains of the CHARMS checklist. As a substantial heterogeneity of included studies is expected, a narrative evidence synthesis will also be provided. Ethics and dissemination Given that this systematic review will use only published data, ethical permissions will not be required. Findings from this review will be published in peer-reviewed scientific journals and presented at major international conferences. We anticipate that this review will contribute to identify key areas of improvement for conducting and reporting prognostic factor analyses with PROs in oncology and will lay the groundwork for developing future evidence-based recommendations in this area of research. Prospero registration number CRD42018099160

Health-related quality of life in lower-risk MDS patients compared with age- and sex-matched reference populations: a European LeukemiaNet study

In myelodysplastic syndromes (MDS), health-related quality of life (HRQoL) represents a relevant patient-reported outcome, which is essential in individualized therapy planning. Prospective data on HRQoL in lower-risk MDS remain rare. We assessed HRQOL by EQ-5D questionnaire at initial diagnosis in 1690 consecutive IPSS-Low/Int-1 MDS patients from the European LeukemiaNet Registry. Impairments were compared with age- and sex-matched EuroQol Group norms. A significant proportion of MDS patients reported moderate/severe problems in the dimensions pain/discomfort (49.5%), mobility (41.0%), anxiety/depression (37.9%), and usual activities (36.1%). Limitations in mobility, self-care, usual activities, pain/discomfort, and EQ-VAS were significantly more frequent in the old, in females, and in those with high co-morbidity burden, low haemoglobin levels, or red blood cells transfusion need (

Chimeric Antigen Receptor T-cell Therapy in Hematologic Malignancies and Patient-reported Outcomes: A Scoping Review

The inclusion of patient-reported outcome (PRO) measures in chimeric antigen receptor (CAR) T-cell therapy research is critical for understanding the impact of this novel approach from a unique patient standpoint. We performed a scoping review to map the available literature on the use of PRO measures in CAR T-cell therapy studies of patients with hematologic malignancies published between January 2015 and July 2022. Fourteen studies were identified, of which 7 (50%) were investigational early-phase trials, 6 (42.9%) were observational studies, and 1 (7.1%) was a pilot study. The EQ-5D and the PROMIS-29 were the 2 most frequently used PRO measures, being included in 6 (42.9%) and 5 (35.7%) studies, respectively. Despite differences in study designs, there seems to be evidence of improvements over time since CAR T-cell infusion in important domains such as physical functioning and fatigue, at least in patients who respond to therapy. Overall, the studies identified in our review have shown the added value of PRO assessment in CAR T-cell therapy research by providing novel information that complements the knowledge on safety and efficacy. However, there are several questions which remain to be answered in future research. For example, limited evidence exists regarding patient experience during important phases of the disease trajectory as only 4 (28.6%) and 5 (35.7%) studies provided information on PROs during the first 2 weeks from CAR T-cell infusion and after the first year, respectively. Time is ripe for a more systematic implementation of high-quality PRO assessment in future clinical trials and in real-life settings of patients treated with CAR T-cell therapy

Similar efficacy outcomes with peripheral blood stem cell versus bone marrow for autologous stem cell transplantation in acute myeloid leukemia: Long-term follow-up of the EORTC-GIMEMA randomized AML-10 trial

we report here the long-term follow-up of the only prospective randomized trial of autologous hematopoietic stem cell transplantation (auto-HSCT) with peripheral blood stem cells (APBSCT) versus auto-HSCT with bone marrow (ABMT) in acute myeloid leukemia (AML) patients in first remission (CR). we observed that among patients alive and still in CR 5 years after planned auto-HSCT, approximately 10% of the patients died in the following 10 years. This stresses the need for long-term close surveillance of AML patients after auto-HSCT. further, long-term follow-up of the trial confirms that APBSCT was comparable to ABMT in term of disease-free-survival and overall survival

Overcoming barriers to the implementation of patient-reported outcomes in cancer clinical trials : The PROMOTION Registry

Acknowledgments We acknowledge the essential contribution of all collaborators of the PROMOTION Registry. This project is funded by a research grant from the European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Group. Also, additional support for the conduct of the study was provided by the Italian Group for Adult Hematologic Diseases (GIMEMA). JMB is supported by the MRC ConDuCT Hub for Trials Methodology Research. We also acknowledge Alessandro Perreca for his invaluable contribution in the data management.Peer reviewedPublisher PD

The prognostic value of health-related quality-of-life data in predicting survival in glioblastoma cancer patients: results from an international randomised phase III EORTC Brain Tumour and Radiation Oncology Groups, and NCIC Clinical Trials Group study

This is one of the few studies that have explored the value of baseline symptoms and health-related quality of life (HRQOL) in predicting survival in brain cancer patients. Baseline HRQOL scores (from the EORTC QLQ-C30 and the Brain Cancer Module (BN 20)) were examined in 490 newly diagnosed glioblastoma cancer patients for the relationship with overall survival by using Cox proportional hazards regression models. Refined techniques as the bootstrap re-sampling procedure and the computation of C-indexes and R2-coefficients were used to try and validate the model. Classical analysis controlled for major clinical prognostic factors selected cognitive functioning (P=0.0001), global health status (P=0.0055) and social functioning (P<0.0001) as statistically significant prognostic factors of survival. However, several issues question the validity of these findings. C-indexes and R2-coefficients, which are measures of the predictive ability of the models, did not exhibit major improvements when adding selected or all HRQOL scores to clinical factors. While classical techniques lead to positive results, more refined analyses suggest that baseline HRQOL scores add relatively little to clinical factors to predict survival. These results may have implications for future use of HRQOL as a prognostic factor in cancer patients

Gratitude among advanced cancer patients and their caregivers: The role of early palliative care

Objective: A cancer diagnosis represents a unique trauma, given its life-threatening, multidimensional, and uncertain nature. Gratitude is a construct representing the emotional state that arises when individuals recognize that a benefit has been received as a result of someone else’s action or a spiritual entity’s intervention. Based on the positive psychological wellbeing, gratitude has been associated with improved health outcomes even in the disease setting. Thus, the models of care that foster gratitude should be adopted in the clinical context. This study aims to explore whether and how gratitude may originate in patients with advanced cancer and their caregivers undergoing early palliative care (EPC). Methods: We analyzed 251 reports from 133 patients and 118 caregivers describing their clinical experience in two EPC units. The sources of gratitude were identified and ranked based on their frequencies. Words expressing gratitude and words referring to communication and spirituality were collected by means of the Linguistic Inquiry and Word Count software and correlated. Results: In total, 123 (92.5%) of 133 patients’ and 97 (82.2%) of 118 caregivers’ reports, respectively, included explicit or implicit expressions of gratitude. Gratitude was associated specifically with successful physical symptom management, emotional support, improved attitude toward death, better information, humanity, and the familiar environment. The use of words of gratitude in patients’ reports was positively correlated with the use of words referring to communication (r =.215, p =.026) and spirituality (r =.612, p &lt;.001). Conclusion: Our results suggest that interventions within the EPC model based on doctor–patient–caregiver communication may allow patients and caregivers to experience a feeling of gratitude, and this may represent a resource to be exploited to improve their physical and psychosocial wellbeing