Growth hormone improves short stature in children with Shwachman-Diamond syndrome

Agnieszka Bogusz-Wójcik; Honorata Kołodziejczyk; Elżbieta Moszczyńska; Maja Klaudel-Dreszler; Grzegorz Oracz; Joanna Pawłowska; Mieczysław Szalecki

doi:10.5114/pedm.2021.105298

eISSN: 2083-8441
ISSN: 2081-237X

Pediatric Endocrinology Diabetes and Metabolism

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abstract:

Original paper

Growth hormone improves short stature in children with Shwachman-Diamond syndrome

Agnieszka Bogusz-Wójcik

¹

,

Honorata Kołodziejczyk

²

,

Elżbieta Moszczyńska

¹

,

Maja Klaudel-Dreszler

³

,

Grzegorz Oracz

³

,

Joanna Pawłowska

³

,

Mieczysław Szalecki

^{1, 4}

Department of Endocrinology and Diabetology, The Children’s Memorial Health Institute, Warsaw, Poland
Anthropology Department, The Children’s Memorial Health Institute, Warsaw, Poland
Department of Gastroenterology, Hepatology, Feeding Disorders and Paediatrics, The Children’s Memorial Health Institute, Warsaw, Poland
Department of Medical Sciences, Collegium Medicum, Poland

Pediatr Endocrinol Diabetes Metab 2021; 27 (2): 87–92

DOI: https://doi.org/10.5114/pedm.2021.105298

Online publish date: 2021/04/20

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Introduction
Shwachman-Diamond syndrome (SDS) is a rare, autosomal recessive multisystemic disorder characterized by pancreatic insufficiency and bone marrow failure. Short stature is a recognized feature of SDS syndrome; however, systemic data concerning recombinant human growth hormone (rGH) treatment are limited. Aim of the study: To assess the effect of rGH treatment in patients with SDS.

Material and methods
Retrospective data were collected from patients with SDS and growth hormone deficiency (GHD) treated with rGH in the Children’s Memorial Health Institute in Warsaw. The annual growth velocity (GV) and height standard deviation score (SD) were compared for up to 2 years of rGH treatment.

Results
Six SDS patients (M : F = 1 : 5) treated with rGH were identified. The median age of starting rGH therapy was 7.5 years, with a mean baseline height SD of –4.06 (range: –6.3 to –2.3 SD). The height SD significantly improved to –3.3 (p = 0.002) and then –3.03 (p = 0.002), following 1 and 2 years of treatment, respectively. The average GV for the patients prior to starting treatment was 4.9 cm/year (range: 3.1–6.5 cm/year), which significantly improved to 7.6 cm/year (range: 5.7–9.6 cm/year) after 1 year of rGH treatment (p = 0.020) and to 6.7 cm/year at the end of 2 years.

Conclusions
Our study has shown that rGH treatment significantly improves the height SDS and GV of patients with SDS and GHD without any side effects. Further research is required to analyse the long-term effect of rGH therapy in patients with SDS.

keywords:

Growth hormone improves short stature in children with Shwachman-Diamond syndrome

Agnieszka Bogusz-Wójcik 1 , Honorata Kołodziejczyk 2 , Elżbieta Moszczyńska 1 , Maja Klaudel-Dreszler 3 , Grzegorz Oracz 3 , Joanna Pawłowska 3 , Mieczysław Szalecki 1, 4

growth hormone, short stature, Shwachman-Diamond syndrome

Agnieszka Bogusz-Wójcik

¹

,

Honorata Kołodziejczyk

²

,

Elżbieta Moszczyńska

¹

,

Maja Klaudel-Dreszler

³

,

Grzegorz Oracz

³

,

Joanna Pawłowska

³

,

Mieczysław Szalecki

^{1, 4}