ESPE Abstracts (2018) 89 P-P3-200

ESPE2018 Poster Presentations GH & IGFs P3 (28 abstracts)

Small for Gestational Age (SGA) Patients with Premature Treatment Discontinuation: Their Journey in French Real-life Settings

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

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

Objective: Premature GH treatment discontinuation of SGA is usually linked to safety or ineffectiveness. However, this population is poorly addressed compared to those with final adult height (FAH). Authors investigated the journey of SGA prematurely discontinued Norditropin® treatment in a French real-life.

Methods: Observational prospective ongoing study: 291 Norditropin®-treated SGA. Annual FU up to FAH. Descriptive analyses made on the criteria addressed and compared prematurely discontinued with the study completers: target height, age at treatment initiation, GH dose at inclusion, treatment duration, % of patients discontinued temporarily minimum once, Δ height between inclusion and treatment stop, height at last visit, % of patients >−2 SDS, last GH dose, age at last visit.

Results: Ninety patients reached FAH, including 51 naïve (56.6%); 69 (23.71%) discontinued prematurely, including 37 naïve (53.6%); 23 lost to FU; 109 ongoing. Significant difference was observed for the following mean values in prematurely discontinued versus completers, respectively (years):

• Age at treatment initiation: 8.9 [8.0; 9.7] vs. 9.5 [5.4; 11.2] (P=0.0579)

• Age at last visit: 13.1 [9.3; 15.2] vs 15.4 [14.4; 16.4] (P=<0.0001)

• Treatment duration: 3.0 [2.1; 3.9] vs 4.8 [3.7; 6.2]. (P=<0.0001)

First year of FU the Δ height of prematurely discontinued was comparable with completers. Second year growth slows considerably and continues to decline. Fourth year 42% continue treatment versus 83% among study completers.

One year between two visits.

Height at last visit was −1.6 [−2.2; −1.1] for study completers and −1.8 [−2.4; −1.3] for prematurely discontinued (P=0.1252). The most common reason for premature treatment discontinuation was related to adverse event or questioning on safety of GH treatment for 13 patients, poor adherence and treatment fatigue concerned 11 patients, 8 subjects were satisfied with obtained stature.

Table 1 Height gain SDS between each visit, mean values.
Completers N=900.50 [0.38;0.61] (N=86)0.27 [0.21;0.34] (N=87)0.15 [0.08;0.22] (N=84)0.03 [−0.02;0.09] (N=75)
Premature discontinuation N=690.40 [0.32;0.48] (N=59)0.17 [0.08;0.25] (N=63)0.07 [−0.01;0.16] (N=52)0.00 [−0.09;0.10] (N=29)

Conclusion: French real-life data show that around one fifth of SGA patients treated with GH stopped treatment prematurely at a median the third year with the main reasons being safety issues, poor adherence and satisfaction of obtained height. An increased understanding of reasons for premature treatment discontinuation is needed. The link between first year height gain and good long-term statural response should be investigated further.

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