Final height and hormonal function after bone marrow transplantation in children

Area of Science:

• Pediatric endocrinology
• Hematology
• Oncology

Background:

• Bone marrow transplantation (BMT) is a life-saving procedure for various hematologic malignancies and severe aplastic anemia.
• Conditioning regimens for BMT, particularly those involving total body irradiation (TBI), can have long-term effects on growth and endocrine function.
• Understanding these long-term sequelae is crucial for optimizing patient care and outcomes.

Purpose of the Study:

• To evaluate the final height and hormonal function in long-term survivors of BMT.
• To compare outcomes between patients conditioned with TBI and chemotherapy versus those treated with chemotherapy alone.

Main Methods:

• Two groups of pediatric patients undergoing BMT were analyzed: Group 1 (n=16) received TBI (7.5-12 Gy) and cyclophosphamide for hematologic malignancies. Group 2 (n=14) received chemotherapy only for severe aplastic anemia.
• Final height, growth patterns (prepubertal and pubertal), and endocrine function (thyroid, adrenal, gonadal, growth hormone) were assessed.

Main Results:

• Group 1 patients exhibited significantly reduced final height compared to their target height and height at BMT, primarily due to blunted pubertal growth. Group 2 patients achieved normal final height.
• Thyroid, adrenal, and growth hormone functions were normal in all patients. However, Group 1 patients showed increased follicle-stimulating hormone and luteinizing hormone levels post-BMT, indicating gonadal disturbances, with partial recovery in some.
• Group 1 boys had normal luteinizing hormone and spontaneous puberty, while Group 2 patients had minimal gonadotropin disturbances.

Conclusions:

• Childhood BMT conditioned with chemotherapy and TBI (≥7.5 Gy) compromises final height due to impaired pubertal growth.
• BMT without TBI did not result in height loss.
• The combination of chemotherapy and TBI often leads to irreversible gonadal dysfunction.