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Short-term Assessment of HSCT Effects on the Hypothalamus-Pituitary Axis in Pediatric Thalassemic Patients

Amir Ali Hamidieh1, Fariba Mohseni2, Maryam Behfar3

  • 1Pediatric Stem Cell Transplant Department Children's Medical Center, Tehran University of Medical Sciences, Tehran, Iran.

Archives of Iranian Medicine
|April 18, 2018
PubMed

Insights

Hematopoietic stem cell transplantation (HSCT) in pediatric beta thalassemia major patients showed no significant overall effect on pituitary-hypothalamus axis disorders within 3 months. Endocrine function monitoring is crucial post-HSCT for these patients.

Area of Science:

  • Pediatric Endocrinology
  • Hematology
  • Stem Cell Transplantation

Background:

  • Beta thalassemia major (BTM) is a severe genetic blood disorder.
  • Hematopoietic stem cell transplantation (HSCT) is a primary treatment for BTM.
  • HSCT may lead to endocrine system complications in pediatric patients.

Purpose of the Study:

  • To assess endocrine complications following HSCT in pediatric patients with BTM.
  • To evaluate changes in pituitary-hypothalamus axis function up to 3 months post-HSCT.

Main Methods:

  • Hormone levels (prolactin, LH, FSH, T4, T3, TSH, IGF-1, testosterone/estradiol) were measured in 20 pediatric BTM patients before and 1, 3 months after HSCT.
  • Cosyntropin and clonidine tests were performed to assess adrenal and growth hormone axes.

Main Results:

  • Pre-HSCT endocrine issues included delayed puberty, hypogonadotropic hypogonadism, GH deficiency, low IGF1, short stature, hypocortisolism, hypothyroidism, and panhypopituitarism.
  • After 3 months, hypogonadotropic hypogonadism, low IGF1, hypothyroidism, and panhypopituitarism were observed.
  • HSCT impacted T3, estradiol, and TSH levels; corticosteroid use affected GH, T3, and sex hormone levels.

Conclusions:

  • HSCT did not demonstrate a significant overall positive or negative effect on pituitary-hypothalamus axis disorders in pediatric BTM patients within the 3-month study period.
  • The study's small sample size and short duration limit definitive conclusions.
  • Further long-term studies are needed to fully understand the endocrine impact of HSCT in BTM.
Abstract

Keywords:
Endocrine disordersHSCTHypothalamusPituitaryThalassaemia

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