Objective:

To assess the impact of growth hormone (GH) therapy on final adult height in patients who developed short stature following hematopoietic cell transplantation (HCT) in childhood.

Approach:

• Study Design: Nationwide, multicenter retrospective cohort study using medical records from 55 institutions in the Japan Children’s Cancer Group.
• Patient Selection: Eligible patients were those diagnosed with hematologic malignancies or solid tumors, underwent HCT at age 15 or younger, were aged 15 to 39 years at registration, and had short stature defined as height SDS of −2.0 or lower.
• Data Collection: Final adult height data were collected for 171 patients, including 58 who received GH therapy and 113 who did not, with a median follow-up of 18.2 years.

Key Findings:

• GH therapy was associated with a 0.94-SDS greater improvement in height SDS compared to those who did not receive GH.
• Factors positively influencing height outcomes included absence of total body irradiation (TBI), initiation of GH therapy at age 10 or older, and female sex.
• TBI was identified as a strong negative predictor of height outcomes and GH responsiveness.
• Responses to GH therapy were heterogeneous, with some patients showing transient responses.

Interpretation:

The study indicates that GH therapy can improve final height in patients with short stature post-HCT, but responses are influenced by various transplant-related factors.

Limitations:

• Retrospective design may introduce residual confounding.
• Non-standardized criteria for GH therapy initiation, dosing, and duration across institutions.
• Selection bias due to inclusion of only patients referred for GH evaluation.
• Lack of data on parental height and genetic growth potential.

Conclusion:

GH therapy is associated with improved final height in this population, but responses vary significantly and are influenced by transplant-related factors.

Sources:

• Frontiers in Endocrinology