Growth hormone therapy shows variable effectiveness in helping childhood stem cell transplant survivors achieve normal height

A study by the Japanese Children's Cancer Group published in Frontiers in Endocrinology in 2026 shows that growth hormone therapy improves final height outcomes in childhood hematopoietic stem cell transplantation survivors with short stature. However, treatment responses vary widely and are strongly influenced by transplant-related factors like total body irradiation and chronic graft-versus-host disease. Growth improvement often continues for over five years, unlike in classical growth hormone deficiency.

Growth hormone therapy effectiveness

• ▪A study published in Frontiers in Endocrinology in 2026 found that growth hormone therapy was associated with improved final height outcomes in childhood hematopoietic stem cell transplantation survivors with short stature.
• ▪In the Japanese Children's Cancer Group study, growth improvement in childhood hematopoietic stem cell transplantation survivors often continued for five years and up to final adult height after starting growth hormone therapy.

Post-transplant growth impairment

• ▪Out of 268 patients enrolled in the Japanese Children's Cancer Group study of childhood transplant survivors, 171 had available final height data, with 58 receiving growth hormone therapy and 113 not receiving it.
• ▪Growth impairment and short stature are common long-term complications in children who undergo hematopoietic stem cell transplantation.

Total body irradiation effects

• ▪Total body irradiation, a form of radiotherapy used to destroy or suppress a recipient's immune system before hematopoietic stem cell transplantation, was identified as the strongest negative predictor of height.
• ▪Growth outcomes in childhood hematopoietic stem cell transplantation survivors receiving growth hormone therapy were strongly influenced by transplant-related factors such as total body irradiation and chronic graft-versus-host disease.

Treatment response variability

• ▪Researchers observed differences in growth hormone therapy response depending on whether the hematopoietic stem cell transplant was allogeneic, using donor stem cells, or autologous, using the patient's own stem cells.
• ▪Treatment responses to growth hormone therapy among childhood hematopoietic stem cell transplantation survivors with short stature were highly variable.

Predictive factors for outcomes

• ▪The Japanese Children's Cancer Group study collected patient characteristics including age at diagnosis, age at transplant, sex, diagnosis, transplant types, and comorbidities to analyze growth outcomes.
• ▪Researchers from the Japanese Children's Cancer Group aim to identify patients at risk for poor growth outcomes to develop more personalized growth hormone treatment strategies for transplant survivors.

Long-term survivorship care

• ▪Improving long-term health and quality of life has become a major goal of survivorship care as the number of childhood transplant survivors increases.
• ▪The Japanese Children's Cancer Group study aimed to determine if growth hormone therapy improves final height outcomes to help children achieve their full growth potential and enjoy a better quality of life in adulthood.

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