Impact of graft function on health status and quality of life in 112 very long-term survivors who received an HSCT for Inborn Errors of Immunity, a prospective study of the CEREDIH

Hematopoietic stem cell transplantation (HSCT) for Inborn Errors of Immunity (IEI) survival outcome has improved considerably and the indications have broadened. Subsequently, the issue of long-term quality of live (HRQoL) has become crucial. Our study focuses on the health and HRQoL of post-HSCT survivors . We conducted a multicenter prospective follow-up program enrolling IEI patients included in the CEREDIH (French IEI Reference Centers) registry, transplanted during childhood, before 2009. Answers from self-reported French Childhood Immune Deficiency Long-term Cohort (F-CILC) and 36-item Short Form (SF-36) questionnaires were compiled. 112 survivors were included with a time median from HSCT of 15 years (range :5-37), 55% were transplanted for a combined immunodeficiency. We show that in patients evaluated prospectively at least 5 years after HSCT, 55% are still affected by a poor or very poor health status. Poor and very poor health status were correlated with an abnormal graft function defined as host or mixed chimerism and/or an abnormal CD3+ count and/or a diagnosis of chronic GVHD (Poor health: OR 2,6 CI 95%: 1,1-5,9 p:0,028 ; Very poor health : OR 3,6 CI 95%: 1,1-13, p:0,049 ). Poor health directly linked to a poorer HRQoL. Significant improvements made in graft procedures translated in better survival, but we show that about half of the survivors are affected by an altered health with a correlation to both abnormal graft function and impaired HRQoL. Future prospective studies will be needed to measure the impact of those improvements on long-term health status and HRQoL.