The Late Effects of Hematopoietic Stem Cell Transplants in Pediatric Patients: A 25-Year Review
Authors' conclusion
We confirm gonadal dysfunction, multiple endocrine and metabolic abnormalities, thyroid cancer, and SMNs as common sequelae of HSCT and identify gaps in management-particularly the need for informed fertility counseling and pretreatment fertility preservation, evaluation, and management of bone health-and underline the need for early lifestyle modification, long-term surveillance, and prospective planned studies aimed at reducing complication risk.
Study context
- Population
- Children and adolescents who received allogeneic hemopoietic stem cell transplant (HSCT) at Royal Children's Hospital Melbournestudy cohort of 384 HSCT recipients
- Participants
- 228 children
- Outcome
- Incidence of endocrinopathies; fertility, growth, bone and metabolic status; subsequent malignant neoplasms (SMNs)
Methodological detailssingle-center
- Single-center
Main finding
As reported in the abstract
Late effects of HSCT
- Gonadotoxicity more common in females
- P < .001
- Spontaneous pregnancies in sexually active females
- 22%
- Short stature common; GH axis disruption among these
- 30%
- Thyroid nodules in patients exposed to thyroid radiation
- 51%
- Malignant nodules
- 30%
- Fragility fractures
- 6%
- Avascular necrosis
- 6%
- Subsequent malignant neoplasms (SMNs), risk continuing to rise throughout follow-up
- Thirteen percent
Observational data: associations, not effects.
Original publication
The Late Effects of Hematopoietic Stem Cell Transplants in Pediatric Patients: A 25-Year Review. J Clin Endocrinol Metab. Published 27 March 2024. doi:10. 1210/ clinem/ dgae196
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