Background: Hematopoietic stem cell transplantation (HSCT) has become more common in treating malignant and nonmalignant diseases in children. However, HSCT is associated with several late effects that can impair growth, like insufficient growth hormone (GH) secretion, hypogonadism and growth plate damage. Growth hormone treatment (GHRx) is offered but limited data are available on its effect on adult height.
Objective: To evaluate the effectiveness of GHRx after HSCT during childhood.
Patients and methods: In this single center retrospective study, 34 patients were included who had HSCT between 1988 and 2010, received GHRx ≥1 yr and had reached adult height (AH). Each patient was matched with two controls who did not receive GHRx based on: gender, indication for HSCT (malignancy, benign hematological or immune deficiency), age at HSCT and total body irradiation. Predicted AH (PAH) was calculated based on bone age at start of GHRx or at equivalent age in controls. Annual data on growth and puberty were collected until AH.
Results: Boys started GH at 12.6±2.5 yr when height was −1.8±1.1 S.D.S. versus −0.8±1.4 in controls and PAH was −1.8±1.2 versus −0.6±1.6 in controls. After 3.7 yr GHRx (median) (range 1.79.2) they reached AH of −2.3±1.3 S.D.S. versus −2.0±1.2 in controls. The difference between AH and PAH was significantly smaller in GH treated boys (AH-PAH −0.5±0.7 S.D.S.) than in controls (−1.4±1.1 S.D.S., P<0.001). AH was 2.1 S.D.S. below target height (TH) in both groups. Girls started GH at 10.7±2.2 yr when height was −2.2±1.1 S.D.S. versus −0.6±1.1 in controls and PAH was −2.6±1.2 versus −0.8±1.0 in controls. After 4.9 yr GHRx (median) (range 2.48.6) they reached AH of −2.1±1.4 S.D.S. versus −1.1±0.8 in controls. The difference between AH and PAH was significantly different in GH treated girls (+0.5±0.6 S.D.S.) compared to controls (−0.3 S.D.S.±0.7, P<0.001). AH was below TH in both groups (−1.8 versus −1.2 S.D.S.).
Conclusion: GHRx had a positive effect on AH as compared to PAH in children after HSCT. AH was close to PAH in treated boys and above PAH in girls whereas AH was lower than PAH in untreated children, especially in boys. However, in both groups AH was low compared to the general population and far below TH.
27 - 29 Sep 2018
European Society for Paediatric Endocrinology