Endocrine complications after hematopoietic stem cell transplantation during childhood - results from a close follow-up in a cohort of 152 patients

Context: Haematopoietic stem cell transplantation (HSCT) is a therapeutic option for numerous haematologic diseases and solid tumours. Increasing indications for HSCT and reduction in associated mortality have been raising the number of paediatric HSCT survivors and their long-term toxicities. Objective: To characterize the endocrine disorders developed after HSCT. Design and Patients: Retrospective analysis of 152 patients submitted to HSCT in paediatric age with at least 24 months of follow-up at our endocrine late-effects clinics. Results: Patients were followed up for 9.9 (interquartile range [IQR]: 12.2) years. The median age at HSCT was 7.5 (IQR: 9) years. At least one endocrine complication was observed in 65.1% of the patients. Primary hypogonadism was detected in 34.2%. Female gender (p <.001), HSCT > 10 years old (p =.01) and chemotherapy before HSCT (p <.001) were identified as risk factors for developing gonadal dysfunction. Growth hormone deficiency (GHD) occurred in 23.0% with a mean stature Z-score at diagnosis of −1.8 ± 1.4. GHD was associated with cranial (p <.001) and HSCT < 10 years old (p ≤ 0.001). Patients who were exposed to total body irradiation (TBI) were at higher risk for primary hypothyroidism (22.3%) (p =.01), thyroid nodules (17.1%) (p <.001), thyroid carcinoma (5.3%) (p <.001), dyslipidaemia (19.1%) (p <.001) and disturbance of carbohydrate metabolism (19.1%) (p <.001). Conclusion: At least one endocrine complication was diagnosed in 65.1% of patients, with gonadal dysfunction being the most prevalent. The conditioning regimen with TBI was a risk factor for the development of several endocrine disorders. This study is one of the largest series evaluating the endocrine disorders among survivors of paediatric HSCT and intends to reinforce the importance of routine follow-up of these patients.