Background: Pediatric bone marrow transplantation (BMT) can lead to endocrine dysfunctions due to common pre-operative regimens involving chemo and radiotherapy.
Objective and hypotheses: To evaluate the prevalence an time-of-onset of endocrine dysfunctions after BMT in children and adolescents.
Method: A retrospective cohort-study design was performed. The inclusion criteria were: <18 years of age at the time of their allogenic BMT program, which started in 2010 in our institution. The patients started their follow-up 100 days after BMT (time 0) and every 6 months, when possible. Height (cm), weight (kg), BMI, and respective z scores (NCHS 2000) as well as their pubertal status (Tanner) were obtained. Lab and imaging data for endocrine diseases (GH deficiency; precocious/delayed puberty, thyroid dysfunctions, adrenal diseases, diabetes insipidus, bone diseases, and metabolic syndrome) were collected.
Results: From 45 patients submitted to allogenic BMT, 28 (14F) were referred to endocrine evaluation. Their primary disease was diagnosed at 5.5 (±4.0) years old (range 0.013.5). BMT was performed at 8.7 (±4.3) years (0.817.8). Bone marrow donors were: siblings (15), bone bank (6), umbilicus cord (5), and parents (2). The patients were referred for endocrine evaluation at 10.2 (±4.0) years (2.018.0). The prevalence of endocrine complications were: growth disorders ((5), four with GH deficiency under treatment), hypercolesterolemia (4), hypothyroidism (3), amenorrhea (2), obesity (2), precocious puberty (1), delayed puberty (1), and diabetes mellitus (1). 15 patients are still under follow-up with no endocrine disease detected so far.
Conclusion: These findings emphasize the importance of screening for endocrine complications, particularly growth disorders and metabolic syndrome, in children who have undergone BMT. Children require an early and long follow up so that endocrine complications can be diagnosed and promptly treated.
20 - 22 Sep 2014
European Society for Paediatric Endocrinology