ESPE Abstracts (2016) 86 RFC6.4

Growth Hormone (GH) Treatment in Skeletal Dysplasias - Short-term Results in Prepubertal Children Reported in KIGS

Lars Hagenäsa, Anders Lindbergb, Cecilia Camacho-Hübnerc & Raoul Roomanc

aPediatric Endocrinology, Karolinska Hospital, Stockholm, Sweden; bPfizer Health AB, Sollentuna, Sweden; cPfizer Inc., Endocrine Care, New York, USA, 4University of Antwerp, Antwerpen, Belgium

Background: A total of 83,803 patients who received rhGH therapy were enrolled in KIGS (Pfizer International Growth Database) including 748 patients diagnosed with a specified or unspecified skeletal dysplasia. The most prevalent diagnoses were hypochondroplasia (n=238: Female=111, Male=127), achondroplasia (n=113: F=51, M=62) and Leri-Weill dyschondrosteosis, LWD (n=88: F=59, M=29).

Objective: To analyse the first year response in height and body proportions to rhGH treatment (GH Tx) in prepubertal patients with hypochondroplasia (Hch), achondroplasia (Ach) and LWD.

Methods: For descriptive statistics median, 10 th and 90 th percentiles are presented.

Results: Only data from children who remained prepubertal during the 1st year of treatment were analysed. The Table summarizes the findings. The percentage of children with a 1st yr Ht gain >0.5 SDS were for Hch, Ach and LWD 54%, 29% and 77%, respectively. Serious Adverse Events reported for the 3 groups were: Hch=2: oral discomfort and appendectomy; Ach=5: gastrointestinal pain, femur fracture, shunt occlusion, headache and hydrocephalus; LWD=2: scoliosis and limb asymmetry.

Table 1. (for abstract RFC6.4)
nmedian (p10 to p90)nmedian (p10 to p90)nmedian (p10 to p90)
Age at start1107.8 (3.5 to 10.9)565.3 (1.9 to 10.4)309.1 (4.8 to 11.9)
Height SDS
At start110−3.8 (−4.7 to −2.9)56−5.5 (−7.2 to −4.1)30−2.8 (−3.9 to −2.0)
First year gain1100.5 (0.2 to 1.1)560.4 (0.3 to 1.2)300.6 (0.4 to 1.1)
Sitting height & relative sitting height SDS*
First year change in sitting height370.6 (−0.1 to 1.1)260.4 (−0.3 to 1.6)130.7 (0.0 to 1.0)
Relative sitting height at start445.2 (2.6 to 7.9)2613.8 (9.5 to 18.6)153.3 (2.4 to 4.1)
First year change in relative sitting height370.1 (−1.6 to 1.0)261.0 (−1.7 to 3.5)130.1 (−1.1 to 0.6)
GH dose (μg/kg per day)11036 (24 to 53)5633 (17 to 46)3039 (23 to 51)
*Relative sitting height=sitting height/height; SDS calculations based on Rader (height) and Gerver (sitting height) references.

Conclusion: Response to GH Tx was modest in Hch and LWD but poor in Ach prepubertal children. Body disproportion in Hch and LWD was unchanged by GH Tx but possibly increased in Ach.

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