Long Term Effects of Pediatric Hematopoietic Stem Cell Transplant on Endocrine Function

Noman Ahmad; Ali Alghamdi; Mrouge Sobaihi; Mohamed Bayoumy; Nabil Aleysae; Muhammad Shahzad; Abdulatef Ahmed; Hesham Aboelghar; Abdulmajid Almahbosh; Marwa Elhadidy; Emily Heaphy; Saleh Shaheen; Maha Alzubaidi; Ali Alharbi

Background: Advances in the treatment of childhood and adolescence cancer treatment have led to significant increase in survival rate. Current 5-survival rate of childhood cancer is nearly 80%. Hematopoietic stem cell transplant (HSCT) is treatment of choice in many clinical conditions including malignant and non-malignant hematological diseases, solid tumors and immunodeficiency diseases. Children receiving HSCT are prepared with different pre-transplant conditioning regimens. These regimens expose the recipients to potent cytotoxic chemotherapy and sometimes to fractionated or non-fractionated total body irradiation (TBI). Adverse effects on endocrine function and bone health are the most commonly found sequelae affecting 20%-50% subjects.

Method: We did a retrospective chart review of children age less than 18 years at the time of HSCT at King Faisal Specialist Hospital and research Centre Jeddah. They received conditioning regimen prior to HSCT either myeloablative (MAC) or reduced intensity (RIC) regimen and in few case no conditioning regimen was given. All participants were followed for a median period of 5 years. Endocrinology assessment carried out periodically including thyroid, adrenal, pituitary glands function and gonadal assessment upon expected physiological age of puberty.

Results: 141 children (75 males and 66 females) had HSCT at median age of 7 years. 128 patients had allogenic while13 patients had autologous HSCT. The conditioning regimen include MAC in 110 (78%), RIC in 28 (19.9%) and no condition in three (2.1%) children. Only 17 children received radiation; 13 had fractionated and four had non-fractionated. Nearly half of the patients (65/141, 46%) developed one of endocrine dysfunction. Girls are more effected than boys (48 vs 17). Growth hormone deficiency in 4 children (1 male and 3 females), adrenal insufficiency in 13 children (6 males and 7 females), thyroid deficiency in 23 children (13 males and 10 females) and gonadal failure in 36 children all females. Endocrine outcomes are compared against the primary disease and conditioning regimen. Gonadal failure has significant association with MAC (P-value 0.0002) however; other endocrine dysfunctions do not have any significant association with conditioning regimen or underlying disease.

Conclusion: Current treatment strategies for childhood cancers and hematological diseases have significantly improved long-term survival. However, this survival is associated with treatment associated adverse effects, which have an impact not only to physical health but also on psychosocial wellbeing of survivor. Our data suggests further improvement in conditioning regimens to avoid endocrine dysfunction.

ESPE Abstracts

P1-539