Long-Term Endocrine Effects of Hematopoietic Stem Cell Transplantation in Children: A Reappraisal

Hematopoietic stem cell transplantation (HSCT) is widely used in both malignant and non-malignant diseases in children. This study aimed to evaluate long-term endocrine complications in pediatric HSCT survivors.

Children who underwent HSCT between April 2010 and October 2014 were retrospectively assessed. Data included demographics, growth and nutritional status, thyroid function, bone health, pubertal development, and gonadal function.

Seventy-five patients (45 males, 30 females; mean current age 18.8 ± 3.9 years) were included. The mean follow-up duration after HSCT was 9.7 ± 1.2 years, and at least one endocrine disorder was identified in 40 patients (53.3%). At the last follow-up, 23 patients (30.6%) were underweight, and 17 (18.6%) had short stature. Growth impairment was more frequent in patients with non-malignant diseases. Hypothyroidism was observed in 6 patients (8%), and low bone mineral density (BMD)/osteoporosis in 17 (22.6%). Short stature, malnutrition, low BMD, and vitamin D deficiency were more prevalent among those who underwent HSCT at ≥10 years of age. Hypogonadism was detected in 12 females (40%) and 11 males (24%) and showed no association with age at HSCT, pubertal stage, primary diagnosis, or conditioning regimen.

These findings underscore the importance of long-term endocrine surveillance in HSCT survivors, particularly in those transplanted at ≥10 years of age.