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


Gürlek Gökçebay D., Gürsoy G., Kanbur M., O. K. Bozkaya İ., Arman Bilir O., Ozbek N. Y.

Clinical Transplantation, vol.40, no.3, 2026 (SCI-Expanded, Scopus) identifier identifier

  • Publication Type: Article / Article
  • Volume: 40 Issue: 3
  • Publication Date: 2026
  • Doi Number: 10.1111/ctr.70504
  • Journal Name: Clinical Transplantation
  • Journal Indexes: Science Citation Index Expanded (SCI-EXPANDED), Scopus, EMBASE, MEDLINE
  • Keywords: children, endocrine, late effects, stem cell transplantation
  • Hacettepe University Affiliated: Yes

Abstract

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