Growth Impairment after Busulphan/Cyclophosphamide-Based Conditioning for Hematopoietic Stem Cell Transplantation

Strategic Insights -

Children undergoing hematopoietic stem cell transplantation (SCT) after busulphan/cyclophosphamide-based conditioning regimens may experience unexplained disturbances of growth, particularly if they have not received gonadotoxic therapy prior to the treatment. The growth impairment may be attributed to insufficient growth hormone secretion. Additionally, thyroid function may be disrupted in some patients, with 10% developing compensated primary hypothyroidism, and gonadal function may also be affected, with high-dose busulphan exposure leading to gonadal failure in a significant number of cases.

Key Takeaways:

  • 35% of children without growth-limiting disorders experience unexplained disturbances of growth after SCT, with 10/23 cases in patients treated for hematological malignancies.
  • Insufficient growth hormone (GH) secretion was diagnosed in four patients out of 10 who had their GH secretion status evaluated.
  • Compensated primary hypothyroidism was found in 10 (19%) patients, with thyroid function being evaluable in 52 patients.
  • Gonadal failure occurred in 7 of the 14 children receiving high-dose busulphan (16-20 mg/kg), with the majority not exposed to gonadotoxic therapy prior to Bu/Cy.
  • Subclinical hyperparathyroidism was found in 16 of the 49 evaluable patients.
  • The study suggests that growth impairment may be the result of insufficient GH secretion.

Statistics:

  • 35% (17/48) of children without growth-limiting disorders experienced unexplained disturbances of growth after SCT.
  • 19% (10/52) of patients developed compensated primary hypothyroidism.
  • 7 (49%) of 14 children receiving high-dose busulphan (16-20 mg/kg) developed gonadal failure.
  • 16 of the 49 (33%) evaluable patients developed subclinical hyperparathyroidism.

Sources:

  • Bakker, B., et al. "Disturbances of growth and endocrine function after busulphan-based conditioning for haematopoietic stem cell transplantation during infancy and childhood." Bone Marrow Transplant 33.10 (2004): 1049-1056.