A Multicenter Retrospective Analysis Stressing Importance of Long-Term Follow Up after Haematopoietic Cell Transplantation for β-Thalassaemia

Biol. of Blood and Marrow Transpl






Allogeneic haematopoietic stem cell transplantation (HSCT) is the only established curative therapy for β-thalassaemia. Patients who opt for this treatment are faced with potential late effects that may lead to chronic health conditions. Long-term follow-up after HSCT has the potential to identify late complications of HSCT, allowing for implementation of standardized surveillance strategies and interventions to optimize outcomes and further increase survival rates. While long-term outcomes of HSCT for β-thalassaemia have been reported in literature, these are scarce and limited to single center experiences. An article published in the June 13 issue of the Biology of Blood and Marrow Transplantationreports late effects and long-term health among 176 pediatric patients affected by β-thalassaemia who survived 1 year and beyond after HSCT with a median follow-up of 7 years. This is an original report on long-term follow-up after HSCT for β-thalassaemia using data from multiple international transplant centres. Outcomes of interest included engraftment, chimerism, graft rejection, graft-vs-host disease, ferritin, growth velocity and measures of organ function. Although the study reported high survival among transplant recipients, late organ toxicity and growth impairment was frequently described in those greater than 7 years of age. The incidence of late effects highlighted the need of uniform, careful and systematic follow-up care as to correctly identify and promptly treat any complications that may arise after HSCT. The study also highlighted the need to systematically document the long-term outcomes of HSCT in data registries in order to optimize the tracking and management of post-HSCT health among transplant survivors.

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