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Published on: November 18, 2022
Endocrine complications of pediatric stem cell transplantation
1Department of Pediatrics, Memorial Sloan-Kettering Cancer Center, 1275 York Avenue, NY, NY 10021, USA. sklarc@mskcc.org
Insights
Pediatric stem cell transplant recipients frequently experience endocrine and growth issues. Common problems include short stature, hypothyroidism, gonadal dysfunction, and reduced bone density, impacting long-term health.
Area of Science:
- Pediatric Endocrinology
- Hematology/Oncology
- Transplantation Medicine
Background:
- Endocrine and growth abnormalities are frequent complications in pediatric and adolescent stem cell transplant (SCT) survivors.
- These issues significantly impact quality of life and long-term health outcomes.
- Understanding these sequelae is crucial for comprehensive patient management.
Purpose of the Study:
- To summarize the common endocrine and growth abnormalities observed in pediatric/adolescent SCT recipients.
- To identify risk factors associated with these complications.
- To highlight the prevalence and specific types of endocrine dysfunction.
Main Methods:
- Review of existing literature on endocrine and growth outcomes post-SCT in pediatric/adolescent populations.
- Analysis of reported frequencies of specific endocrine abnormalities.
- Identification of correlations between transplant characteristics and endocrine sequelae.
Main Results:
- Impaired linear growth and short stature are linked to younger age at transplant, total body irradiation (TBI), and chronic graft-versus-host disease (GvHD).
- Primary hypothyroidism affects 10-28% of patients after fractionated TBI; autoimmune hyperthyroidism also occurs.
- Gonadal dysfunction is highly prevalent, with most males experiencing oligo-azoospermia and most females developing primary ovarian failure after TBI or specific chemotherapy regimens.
- Bone mineral density is often reduced, increasing future osteoporosis risk.
Conclusions:
- Stem cell transplantation in pediatric/adolescent populations frequently leads to significant endocrine and growth disturbances.
- Risk factors such as age at transplant, irradiation, and GvHD influence the development of these complications.
- Long-term monitoring and management strategies are essential to address hypothyroidism, gonadal dysfunction, and bone health in survivors.
Abstract:
Abnormalities of endocrine function and growth are common following stem cell transplantation in the pediatric/adolescent population. Impaired linear growth and adult short stature are associated with younger age at transplant, use of TBI and prior cranial irradiation, and development of chronic GvHD. Primary hypothyroidism is the most common abnormality of the thyroid and is observed in 10-28% of cases following fractionated TBI. Autoimmune hyperthyroidism has also been described post-stem cell transplant and most often results from adoptive transfer of abnormal clones of T or B cells from donor to recipient. Gonadal dysfunction is extremely prevalent and includes oligo-azoospermia in the majority of males treated with TBI, and primary ovarian failure in most women treated with TBI or Busulfan/Cyclophosphamide. Leydig cell function, however, is retained in most males treated with standard forms of cytoreduction. Many patients demonstrate reduced bone mineral density and are at risk of developing osteoporosis in the future.
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