Growth hormone therapy can improve adult height in children who have undergone stem cell transplantation, according to a Japan-wide study published on Aug. 7. The study found that while growth hormone therapy is beneficial, prior radiation treatment and transplant-related complications can reduce its effectiveness.
Hematopoietic stem cell transplantation is used to treat certain life-threatening cancers and nonmalignant disorders in children. Although it is considered a curative procedure, advances in medicine have increased the number of long-term survivors, which has also revealed late effects such as growth impairment and short stature among childhood survivors.
A research team from the Japanese Children's Cancer Group, led by Hiroshima University, conducted a nationwide retrospective cohort study involving childhood hematopoietic stem cell transplantation survivors diagnosed with post-transplant short stature. The findings were published in the journal Frontiers in Endocrinology on May 20. Of the 268 patients enrolled, final height data was available for 171; among these, 58 received growth hormone therapy while 113 did not. The researchers collected additional patient information including age at diagnosis and transplant, sex, type of transplant, comorbidities, and other factors for statistical analysis.
"GH therapy was associated with improved final height outcomes in childhood HCT survivors with short statures," 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 (GVHD). Our findings suggest that GH therapy is beneficial, but individualized long-term management is essential." Shimomura also said that some patients continued to see improvements many years after starting growth hormone therapy—a pattern different from what is typically seen in children with classical growth hormone deficiency.
The strongest negative predictor of height was total body irradiation prior to transplantation. Researchers observed differences depending on whether transplants involved donor or self-derived cells—suggesting distinct biological mechanisms underlying growth impairment after transplantation.
"Our next goal is to better understand why some patients respond well to GH therapy while others show limited benefit," Shimomura concludes. "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." The study was carried out by committees within the Japanese Children's Cancer Group and supported by Hiroshima University Hospital Research Support Grant.
