The Late Effects of Hematopoietic Stem Cell Transplants in Pediatric Patients

Samantha Lai-Ka Lee, Quynh-Nhu Nguyen, Cindy Ho, Simon James, Amreeta Kaur, Angelina Lim, Karin Tiedemann, Margaret Zacharin
The Journal of Clinical Endocrinology & Metabolism, Volume 110, Issue 2, February 2025, Pages e347–e362
https://doi.org/10.1210/clinem/dgae196

Abstract

Context

A rare, large, single-center study covering all long-term health outcomes of pediatric allogeneic hemopoietic stem cell transplant (HSCT) survivors, to provide comprehensive local data and identify gaps and future directions for improved care.

Objective

To document endocrine sequelae and other late effects of all HSCT recipients.

Design

Retrospective review.

Setting

Royal Children’s Hospital Melbourne.

Patients

384 children and adolescents received HSCT; 228 formed the study cohort; 212 were alive at commencement of data accrual.

Intervention

None.

Main Outcome Measures

Incidence of endocrinopathies; fertility, growth, bone and metabolic status; subsequent malignant neoplasms (SMNs).

Results

Gonadotoxicity was more common in females (P < .001). Total body irradiation (TBI) conditioning was more toxic than chemotherapy alone. All females receiving TBI or higher cyclophosphamide equivalent doses developed premature ovarian insufficiency. In males, impaired spermatogenesis + ∕ − testicular endocrine dysfunction was associated with increasing testicular radiation exposure. Preservation of gonadal function was associated with younger age at HSCT. Of sexually active females, 22% reported spontaneous pregnancies. Short stature was common, with GH axis disruption in 30% of these. Of patients exposed to thyroid radiation, 51% developed nodules; 30% were malignant. Metabolic disturbances included hypertension and dyslipidemias, with both excess and underweight reported. Fragility fractures occurred in 6% and avascular necrosis in 6%. Thirteen percent developed SMNs, with the risk continuing to rise throughout follow-up.

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.

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