Abstract
OBJECTIVE: Hematopoietic stem cell transplantation (HSCT) can be a curative treatment for malignant and nonmalignant diseases in children but is associated with significant late effects including growth failure. Growth hormone treatment (GHRx) is offered to improve growth, but limited data are available on its effect on adult height (AH). We aim to evaluate the effectiveness of GHRx. DESIGN: Single-center retrospective study. PATIENTS: Thirty-four patients who had received GHRx for ≥1 year were matched with two controls each, without GHRx, based on sex, indication for HSCT (malignancy, benign haematological disease or immunodeficiency), age at HSCT and conditioning with/without total body irradiation (TBI). All had reached AH. MEASUREMENTS: The primary outcome measure was the difference between AH and predicted AH (PAH) at start of GHRx or the equivalent age in controls (AH-PAH), calculated according to Bailey and Pinneau. RESULTS: GHRx was started at age 12.0 ± 2.6 years; median treatment duration was 3.8 years (range 1.7-9.2). AH-PAH standard deviation score (SDS) was significantly higher in growth hormone (GH) treated boys (-0.5 ± 0.7 SDS) than in controls (-1.5 ± 1.0 SDS, p < .001). Girls also had a higher AH-PAH after GHRx (+0.5 ± 0.6 SDS) compared to controls (-0.2 SDS ±0.7, p < .01). AH remained approximately 2 SDS below target height (TH) in treated and untreated individuals. Among GH-treated children, AH-PAH was higher in those who had received busulfan-based compared to TBI-based conditioning. CONCLUSION: GHRx had a significant positive effect on AH compared to PAH, although AH remained far below TH. Higher AH-PAH was observed in girls and in those conditioned without TBI.