EBMT 2018 | A new endpoint that accurately recognizes the long-term outcomes of allo-HCT in refractory GvHD

An oral abstract was presented at the 44^th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT), Lisbon, Portugal, on Wednesday 21 March 2018, from Saitama Medical Center, Jichi Medical University, Division of Hematology, Saitama, Japan.

Graft-versus-host disease-free survival (GRFS) has been a key measure to estimate transplant outcomes, hence it characterizes disease-free survival as well as survival without major morbidity related to GvHD. GRFS definites GvHD as a permanent therapy failure event, despite of the fact that GvHD might response to treatment. As a result, GRFS may overestimates the effect of GvHD on the outcome of allo-HCT.

This study aimed to identify a new endpoint that accurately recognizes the long-term outcomes of allo-HCT including life expectancy, disease remission, and quality of life (QOL).

Methods:

• QOL was compared after allo-HCT according to the status of chronic GvHD using a nationwide cross-sectional questionnaire study
• Two new composite endpoints were implemented, current GRFS (cGRFS) and refractory GRFS (rGRFS) with the use of a single-center cohort (n = 315)
• cGRFS was defined as survival without disease relapse/progression or active chronic GVHD at a given time after allo-HCT

Key findings:

• Only active cGVHD had an inferior impact on QOL after allo-HCT
• The two cGRFS curves obtained using two altered methods were overlaid and both showed better outcome to conventional GRFS
• The curves of rGRFS and cGRFS were superimposed

This study showed that cGRFS and rGRFS more accurately reflect long-term transplant outcomes than standard GRFS. “Particularly, rGRFS can be easily calculated and analyzed with widely-used statistical approaches including proportional hazard modeling. Therefore, we propose rGRFS as a more effective and simple endpoint for assessing the long-term transplant success.”