Graft Versus Host Disease Clinical Trials: Is it Time for Patients Centered Outcomes to Be the Primary Objective?

Abstract

Purpose of review: Graft versus host disease (GVHD) is a common complication following Hematopoietic Cell Transplant, and is associated with a high symptom burden, reduced functional status and impaired quality of life (QOL). QOL is best assessed by Patient-Reported Outcomes (PRO). Numerous clinical trials for prevention and treatment of GVHD are available. This review aims to understand the landscape of PRO inclusion in clinical trials for GVHD over the last decade. Recent findings: Consensus bodies, including experts in GVHD, PRO and clinical trials have made recommendations for a standardized approach for the inclusion of PRO in clinical trials including as primary outcomes, however, these have yet to be implemented in a consistent manner in practice. Summary: Consistently applying consensus recommendation in chronic GVHD will ensure PROs are appropriately included in clinical trials. Development of validated measures in acute GVHD and composite outcomes for all GVHD trials are required.

Systematic Review of Patient-Reported Outcome Measures in Graft-versus-Host Disease

“…The comprehensive psychometric testing of PROMs should incorporate the cultural, religious, and spiritual perspective lacking in most available PROMs and investigate patient factors such as personality, which may influence the use of these instruments [13]. As Shaw [11] identified, PROMs should be more consistently incorporated where appropriate into GVHD clinical trials, and further testing and possibly development of a new PROM tool for use in the aGVHD setting should be considered. The Internet of Things healthcare ecosystem is expanding and will offer increased opportunities for hematologists to gain insights that improve the ability to tailor treatment and enhance their understanding and approach to personalized management strategies.…”

“…PROMs additionally enable the evaluation of a wide range of issues that go beyond the clinician's perspective of GVHD severity [8]. There are, however, challenges to collecting PROMs in GVHD patients, including age; gender; cultural and linguistic barriers [9]; the need to integrate PROMs into routine clinical workflow without increased workload; organizational issues such as staffing and data protection [10]; inconsistent use of PROMs in clinical trials [11]; lack of normal/reference values for some instruments that make interpretation difficult [12]; impact of external factors and personality on the outcomes, which can be variable over time [13]; and the need to minimize patient burden, particularly when patients are unwell [11]. Furthermore, PROMs require baseline evaluation to understand the relevance of changes over time [10], and the optimal timing of questionnaire collection is an important consideration [14].…”

Graft-Versus-Host Disease Associated Post-operative Complications After Pelvic Reconstructive Surgery: A Case Report

“…It is classified into acute (aGVHD) and chronic (cGVHD). Chronic GVHD (cGVHD) is a multi-system disease that significantly deteriorates functional status, quality of life, and survival (5).The incidence of symptomatic cGVHD requiring medication is 40% to 70% .However, there are reasons that symptomatic genital cGVHD is considered an underdiagnosed and underestimated cGVHD (6). The risk of pelvic organ prolapse is extremely rare in GVHD patients possibly due to the high risk of vaginal stenosis.…”

B Lymphocytes Are the Target of Mesenchymal Stem Cells Immunoregulatory Effect in a Murine Graft-versus-Host Disease Model

“…Clinically significant aGvHD (grade II or higher) developed in 20–65% of patients. Mortality due to this complication accounts for approximately 50% 1,2 .…”