To evaluate the effectiveness of growth hormone (GH) therapy and identify specific clinical factors associated with GH response in childhood hematopoietic cell transplantation (HCT) survivors diagnosed with short stature.
Key Findings:
GH-treated patients showed significantly greater improvement in final height SDS and ΔSDS compared to untreated patients, indicating the effectiveness of GH therapy.
Growth responses to GH therapy were heterogeneous, suggesting variability in treatment effectiveness.
Total body irradiation (TBI), HCT before age 5, and chronic graft-versus-host disease were linked to poorer height outcomes, highlighting the impact of these factors.
Factors influencing GH responsiveness varied by transplant type, with absence of TBI and female sex being favorable in allogeneic HCT, indicating the need for tailored approaches.
GH therapy did not increase the risk of slipped capital femoral epiphysis, secondary malignancies, or relapse of the primary disease, supporting its safety.
Interpretation:
GH therapy is effective in improving height outcomes in childhood HCT survivors, but responses are influenced by transplant-related factors and long-term complications, necessitating personalized treatment strategies.
Limitations:
The study is retrospective and may be subject to selection bias, which could affect the generalizability of the findings.
The sample size for GH-treated patients was limited, potentially impacting the robustness of the conclusions.
Conclusion:
Optimizing GH therapy and developing targeted strategies for patients expected to respond poorly are critical challenges to improve height outcomes after childhood HCT.
