ESPE Abstracts

Introduction: The conventional approach to the treatment of Beta- thalassemia major (BTM) is based on the correction of hemoglobin status through regular blood transfusions and iron chelation therapy for iron overload. Allogeneic hematopoietic stem cell transplantation (HCT) remains the only currently available technique that has curative potential. No previous study compared the long-term growth and endocrinopathy changes among large cohorts of BTM patients on conventional treatment vs those treated with HCT.

Aim: The goal of this study was to find out the prevalence of long-term growth and endocrine changes after HCT in patients with BTM and compare these data with those occurring in BTM patients on conventional treatment.

Methods: We recorded, summarized, and calculated the prevalence of different growth and endocrine abnormalities in published studies (Pubmed, Google scholar) on children and adolescents with BTM in the past 30 years (n=527). These data were compared to published data on 2 large cohorts of BTM children and adolescents who were on conventional therapy (n=11744 and n=3817).

Results: Considerable growth and endocrine abnormalities were detected after BMT in patients with BTM. (~ 41% had growth impairment, ~37% had GH deficiency, ~30 % had gonadal abnormalities and ~10 % had thyroid anomalies). In comparison to prevalence data from 2 large cohorts of children on Conv therapy, our analysis showed clearly that BMT cannot correct the endocrinological complications and therefore highly supported the set-up of simple universal measures that allow early identification and proper and timely management of these consequences. (table 1)

Conclusion: In Children with BTM who had BMT, annual assessments of growth, pubertal development, sexual, and reproductive function are recommended based on the high prevalence of growth and endocrinopathy after exposure to conditioning regimens.