Growth hormone therapy helps childhood stem cell transplant survivors grow taller, but responses vary widely
· Medical Xpressedited by Lisa Lock, reviewed by Robert Egan
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Hematopoietic stem cell transplantation (HCT) is a treatment for some life-threatening cancers and nonmalignant disorders. As HCT is a curative treatment, it is used to treat children where necessary. It has historically been a dangerous procedure, but advances in medicine have increased the number of long-term survivors. This increase has also revealed late effects in childhood survivors, most notably growth impairment and short stature, which are common after HCT.
A multi-institution research team from the Japanese Children's Cancer Group, led by Hiroshima University, studied the long-term effectiveness and response patterns of growth hormone (GH) therapy in children who underwent HCT and were subsequently diagnosed with short stature. Their findings were published in Frontiers in Endocrinology.
"Growth impairment is one of the most common long-term complications in children who undergo HCT," said Maiko Shimomura, assistant professor at Hiroshima University Hospital and first and corresponding author of the study. "We aimed to determine whether GH therapy improves final height outcomes in childhood HCT survivors and to identify clinical factors associated with treatment response. This is important because the number of childhood transplant survivors is increasing, and improving long-term health and quality of life has become a major goal of survivorship care."
The researchers conducted a nationwide, multicenter retrospective cohort study of childhood HCT survivors with post-transplant short stature. Height outcomes were evaluated using final adult height standard deviation scores (SDS).
Of the 268 patients enrolled in the study, 171 had available final height data. Of these, 58 had received GH therapy, while the remaining 113 had not. Other patient characteristics, such as age at diagnosis, age at HCT, sex, diagnosis, types of HCT and comorbidities, were also collected, and all data were statistically analyzed.
"GH therapy was associated with improved final height outcomes in childhood HCT survivors with short stature," Shimomura said. "However, treatment responses were highly variable. Growth outcomes were strongly influenced by transplant-related factors such as total body irradiation (TBI) and chronic graft-versus-host disease. Our findings suggest that GH therapy is beneficial, but individualized long-term management is essential."
One particularly interesting finding was that growth improvement often continued for many years after GH therapy was started, and height gains continued to increase at 5 years and even at final adult height in some patients. This is a marked difference from the pattern typically observed in children with classical GH deficiency.
The strongest negative predictor of height was TBI, a form of radiotherapy that serves to destroy or suppress the recipient's immune system. TBI is often used before HCT to eradicate cancer cells and increase the success of HCT.
The researchers also observed differences in response to GH therapy depending on whether the transplant was allogeneic—stem cells taken from a donor—or autologous—stem cells taken from the patient themselves before HCT. This suggests that distinct biological mechanisms underlie growth impairment.
"Our next goal is to better understand why some patients respond well to GH therapy while others show limited benefit," Shimomura said. "We hope to identify patients at risk for poor growth outcomes and develop more personalized treatment strategies. Ultimately, our goal is not only to help children survive transplantation, but also to help them achieve their full growth potential and enjoy a better quality of life throughout adulthood."
More information
Maiko Shimomura et al, Growth hormone therapy after hematopoietic cell transplantation in childhood: a nationwide survey and longitudinal cohort study, Frontiers in Endocrinology (2026). DOI: 10.3389/fendo.2026.1831711
Key medical concepts
Short StatureHematopoietic Stem Cell TransplantationWhole-Body Irradiation
Clinical categories
PediatricsEndocrinologyChildren's healthOncology Provided by Hiroshima University Who's behind this story?
Lisa Lock
BA art history, MA material culture. Former museum editor, paramedic, and transplant coordinator. Editing for Science X since 2021. Full profile →
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Citation: Growth hormone therapy helps childhood stem cell transplant survivors grow taller, but responses vary widely (2026, August 7) retrieved 7 August 2026 from https://medicalxpress.com/news/2026-08-growth-hormone-therapy-childhood-stem.html This document is subject to copyright. Apart from any fair dealing for the purpose of private study or research, no part may be reproduced without the written permission. The content is provided for information purposes only.