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Polish Society of
Endocrinology
Effectiveness of therapy with recombinant human growth hormone (rhGH) in patients with hypoplastic left heart syndrome (HLHS) and short stature
26.10.2025
Aleksandra Furtak, Magdalena Ossowska, Karolina Komorkiewicz, Dominika Januś, Dorota Roztoczyńska, Jerzy B. Starzyk, Anna Wędrychowicz
Abstract
Introduction: Hypoplastic left heart syndrome (HLHS) is among the most severe and complex forms of congenital heart disease. Despite advances in medical and surgical management, patients with HLHS remain at risk for numerous short- and long-term complications, including growth impairment. The aim of this study is to evaluate the effectiveness and safety of recombinant human growth hormone (rhGH) therapy in patients with HLHS and either growth hormone deficiency (GHD) or small for gestational age (SGA) status, treated at a single tertiary pediatric endocrinology center.
Material and methods: We retrospectively studied eight male patients with HLHS and short stature. Patients were qualified for rhGH therapy in accordance with the guidelines of the Polish rhGH treatment program dedicated to children with short stature and GHD or SGA. Five patients with GHD were qualified for rhGH therapy. The median age of the patients was 14.4 years, and the median height standard deviation score (SDS) was –5.82. Patients who did not initiate rhGH therapy had a median age of 13.8 years and a median height SDS of –3.97. To evaluate the effectiveness of rhGH therapy, we assessed patients’ height SDS, bone age, and serum insulin-like growth factor 1(IGF-1) concentrations at baseline (prior to treatment initiation), approximately one year after the start of therapy, and annually thereafter.
Results: During rhGH therapy, the mean height SDS improved from –5.82 to –5.38 at the first follow-up after a mean duration of 1.1 years. Ultimately, the mean height SDS further improved to –4.92 after an average of 2.5 years of treatment. In contrast, patients who did not receive rhGH therapy showed no improvement; their mean height SDS declined from –3.79 to –4.15 over an observation period of 8 to 16 months.
Conclusion: Growth failure in patients with HLHS is likely multifactorial in origin, extending beyond the cardiac defect and its treatment. Short stature in this population warrants thorough evaluation, and in cases of confirmed GHD, rhGH therapy should be considered when feasible. Treatment decisions must be individualized, considering the patient’s overall clinical condition and coexisting comorbidities.