ESPE Abstracts

Hypoplastic left heart syndrome (HLHS) is one of the most severe congenital heart defects. Despite medical progress, HLHS patients remain at risk of many complications of this cardiac defect and cardiac surgery, including growth failure. Some of them can be treated with recombinant human growth hormone (rhGH) fulfilling criteria of GH–deficiency (GHD) or having a history of intrauterine growth restriction or small for gestational age (SGA). We assessed the effectiveness of rhGH therapy in patients with HLHS treated in our single center in the years 2019-2024. Eight male HLHS patients with short stature were qualified for rhGH therapy: six GHD-patients and two SGA patients. Ultimately, rhGH therapy was administered to five of them with GHD. Patient’s age at the origin of rhGH therapy was 7.7–17.6, mean 14 years; bone age was delayed of 2.6-9.5, mean 5.7 years. Patients IGF-1 levels were below -2 SD in two cases and in lower normal ranges in 3 cases. Their maximum release of GH in two stimulation tests was 4.78 ng/ml. The older patients had hypoplastic sella turcica, four patients presented with hypogonadism, two with hypothyroidism. Four patients had enteropathy treated with glucocorticoids, of which two patients had osteoporosis treated with bisphosphonates. Patients who did not receive rhGH were at age (13.1-15.2, mean 14 years): two patients with SGA (born weight: -2.47 SD and -2 SD) without GHD and one GHD patient with maximum release of GH in tests 4.80 ng/ml. After one year of rhGH therapy at an average dose of 0.29 mg/kg/week (0.24-0.37) we observed improvement of growth velocity in all patients. Standard deviations (SDs) of their height changed from (-4.66; -7.05; -5.11; -8.73; -3.55) to (respectively: -3.41; -6.35; -4.71; -8.0; -3.99). The best improvement was observed in patient without enteropathy treated with steroids, the worse in those treated with bisphosphonates. We did not observed side effects of rhGH. One patient interrupted therapy after 5 months because of worsening of his cardiac function. In patients without rhGH therapy after 8-16 months of observation, their SDs changed from (-2.42; -3.8; -5.16) to (respectively: -3.39; -3.22, -5.83). Growth failure in HLHS patients can have multifactorial etiology not only associated with cardiac defect and its treatment. Each short stature should be properly diagnosed and in cases of GHD, therapy with rhGH should be implemented if it is possible. Each improvement of height improve the quality of life those patients.