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 and Hiroshima University shows that growth hormone therapy improves final height outcomes in childhood stem cell transplant survivors with short stature. However, responses vary widely and are strongly influenced by transplant-related factors like total body irradiation and chronic graft-versus-host disease. Unlike classical growth hormone deficiency, height gains often continue for over five years, highlighting the need for personalized long-term care.

Growth hormone therapy effectiveness

• ▪In a study of 268 childhood hematopoietic stem cell transplantation survivors, 58 of the 171 patients with available final height data received growth hormone therapy, while 113 did not.
• ▪Growth improvement in childhood hematopoietic stem cell transplantation survivors often continued for many years after starting growth hormone therapy, with height gains continuing to increase at 5 years and at final adult height.
• ▪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.

Post-transplant growth impairment

• ▪Hematopoietic stem cell transplantation is a curative treatment for some life-threatening cancers and nonmalignant disorders in children, but it commonly causes late effects such as growth impairment and short stature.
• ▪Growth impairment is one of the most common long-term complications in children who undergo hematopoietic stem cell transplantation.

Total body irradiation effects

• ▪Total body irradiation is a form of radiotherapy used before hematopoietic stem cell transplantation to eradicate cancer cells and destroy or suppress the recipient's immune system.
• ▪Total body irradiation was identified as the strongest negative predictor of height in childhood hematopoietic stem cell transplantation survivors who underwent growth hormone therapy.

Treatment response variability

• ▪The pattern of growth improvement in childhood hematopoietic stem cell transplantation survivors receiving growth hormone therapy differs from the pattern typically observed in children with classical growth hormone deficiency.
• ▪Treatment responses to growth hormone therapy in childhood hematopoietic stem cell transplantation survivors were highly variable, with some patients showing limited benefit and others showing long-term improvement.

Predictive factors for outcomes

• ▪Growth outcomes after growth hormone therapy in childhood hematopoietic stem cell transplantation survivors are strongly influenced by transplant-related factors, including chronic graft-versus-host disease.
• ▪Researchers observed differences in response to growth hormone therapy depending on whether the transplant was allogeneic, using donor stem cells, or autologous, using the patient's own stem cells.

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 hematopoietic stem cell transplantation survivors increases.
• ▪Researchers from the Japanese Children's Cancer Group and Hiroshima University aim to identify patients at risk for poor growth outcomes to develop more personalized treatment strategies.

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