ESPE Abstracts (2018) 89 P-P3-205

ESPE2018 Poster Presentations GH & IGFs P3 (28 abstracts)

Small for Gestational Age (SGA) Patients in Real Life French Clinical Practice: What is the Difference Between Good and Poor Responders to GH Treatment

Marc Nicolino a , Régis Coutant b , Bruno Leheup c , Jean-Pierre Salles d , Evguenia Hacques e & Béatice Villette e


aHôpital Mère-Enfant - Groupement Hospitalier Est, Lyon, France; bCHU, Angers, France; cCHRU - Université de Lorraine, Nancy, France; dHôpital des Enfants, CHU, Toulouse, France; eNovo Nordisk, Paris, France

Objective: Age and height at treatment start, target height, GH dose, and first year treatment response are among known criteria of GH deficiency (GHD) good responders (final adult height [FAH] >−2 standard deviation score [S.D.S.]) to GH treatment (GHT). The authors investigated whether the same criteria are applicable to SGA patients based on real-life ongoing French registry data.

Methods: 291 SGA children treated with Norditropin® were included, 183 were naïve. Naïve completers were stratified in poor and good responders according to FAH ≤−2 S.D.S. or >−2 S.D.S., respectively. The following criteria were addressed and compared: height and weight at birth, target height, age, height and GH dose at treatment initiation, growth velocity (GV) year before GHT, Δ height and GV in the first year of treatment, GH cumulative dose and treatment duration. Analysis was descriptive; Student t-test was used to compare mean quantitative data (S.D.) [min; max] (P value) and Fischer test for the proportion of qualitative data. [Confidence interval 95%] was also calculated.

Results: To date, 51 naïve patients have completed the study, 31 were good responders and 20 poor responders. A significant difference or positive trend was observed for the following mean values in good versus poor responders:

• Height at treatment start (S.D.S.): −2.7 (0.5), [−2.8; −2.5] vs. −3.2 (0.4), [−3.4; −3.0] (P=0.0006)

• Age at treatment start (years): 10.0 (2.5), [9.1; 11.0] vs. 11.4 (2.2), [10.4; 12.5] (P=0.0490)

• GV 1 year before treatment (S.D.S.):- 0.01 (2.1), [−1.1; 1.1] vs. −1.8 (1.8), ([−3.1; −0.4] (P=0.0446)

• Target height (S.D.S.); −1.3 (−1.3), [−1.6; −1.0] vs. −0.8 (1.0), [−1.3; −0.4] (P=0.0857)

• GV the first year of treatment (S.D.S.): 2.33 (1.98), [1.59; 3.07] vs. 1.35 (1.77), [0.52; 2.17] (P=0.0780)

• Δ height ≧+0.5 S.D.S. (% patients): 66.7%, [48.8%; 80.8%] vs. 40%, [21.9%; 61.3%] (P=0.0845)

Good responders were taller and younger at the beginning of treatment with better GV the previous year of treatment. GV in the first year of treatment had a superior positive trend compared to poor responders.

Conclusion: French observational registry data show that some GHD criteria of good response could be applicable to SGA patients treated with Norditropin® and useful for clinical practice. Nevertheless, the observational design of the study and the small sample size of patients could limit the power of analysis. Further investigations with more patients completed the study are needed as well as other observational studies.

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