ESPE Abstracts (2014) 82 P-D-3-1-823

ESPE2014 Poster Category 3 Growth (14 abstracts)

Three-Years Height Outcome During rhGH Therapy in Severe Short Subjects Affected by Skeletal Dysplasias

F Massart a , L Gnesi a , A Baggiani b & M Miccoli b


aPediatric Unit, Maternal and Infant Department, St Chiara University Hospital of Pisa, Pisa, Italy; bEpidemiology Unit, Department of Experimental Pathology M.B.I.E., University of Pisa, Pisa, Italy

Background: Skeletal dysplasias comprise heterogeneous disorders often characterised by short stature with abnormalities of one or more of epiphysis, metaphysis or diaphysis. Over 200 types of skeletal dysplasias are identified, most of which are autosomal dominantly inherited. Actually, surgery has attempted to correct bone deformities but drug therapy for improving their severe short stature has been rarely attempted.

Objective and hypotheses: Administration of recombinant human GH (rhGH) increases height (Ht) among subjects with achondroplasia and hypochondroplasia, who are the most common short-limb dwarfism. For other skeletal dysplasias, however, the efficacy of rhGH remains substantially unknown for disorder rarity.

Method: After thorough search of pertinent English-language literature, our meta-analysis examined the rhGH therapy efficacy to improve height growth over 3-year period in short stature subjects with skeletal dysplasia. From eligible studies, we excluded data regarding ACH and HCH subjects.

Results: In total sample, mean Ht at rhGH therapy start (rage dosage 0.23–0.46 mg/kg per week) was subnormal (n=79; Ht −3.685 SDS (95% CI −3.899 to −3.472)) in all the studies. Ht scarcely improved during 12 months of rhGH treatment with no catch-up growth (n=79; Ht –3.551 SDS (95% CI –3.737 to –3.366); P<0.0001). Then, Ht trend appeared constant at 24 months (n=39; –3.047 SDS (95% CI –3.439 to –2.655)) and until 36-months (n=21; –2.513 SDS (95% CI –2.702 to –2.323)). Episodic adverse effects were reported.

Conclusion: Although our meta-analysis does not indicate convincing benefits of rhGH treatment in severe short individuals with skeletal dysplasias, larger randomized studies are needed to assess the Ht gain in regard to higher rhGH dosages or longer treatment. Furthermore, careful consideration of indications for rhGH administration as surgery therapy adjuvant is crucial when attempting to treat advanced bone deformities.

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