Mortality in GH-treated (Tx) patients (pts) enrolled in the global genetics and neuroendocrinology of short stature international study (GeNeSIS)
C. Child1, A. Zimmermann2, C. Quigley2, R. Rosenfeld3, L. Robison4 & W. Blum5
Preliminary data from the French SAGhE study of 6928 pts with isolated idiopathic GH deficiency (IsIGHD), idiopathic short stature (ISS) or born small for gestational age (SGA) who started GH treatment during childhood (19851996) & were followed up in 2009, suggested increased mortality vs the French general population (pop; standardized mortality ratio (SMR): 1.3, 95% confidence interval (CI): 1.11.6; 116 403 person-years (PY))1.
To assess mortality in a global pediatric observational study, deaths in GeNeSIS pts (all diagnoses, incl. organic & syndromic) were identified; for reference to SAGhE data, mortality rates were calculated for GH-Tx pts with IsIGHD, ISS & SGA. SMR/95% CI were calculated using age- & sex-specific pop rates from CDC (USA) & WHO (other countries).
Among 18 147 pts (all diagnoses) 33 deaths were reported (30/17 692 GH-Tx, 3/455 non-GH-Tx) during median (Q1, Q3) follow-up of 2.0 (0.9, 3.8) years. Deaths were due to acute illness (16), recurrence of pre-existing intracranial neoplasms (8, incl. the 3 non-GH-Tx pts), second cancers (2, both irradiated), accidents (3), underlying conditions (2) & unknown causes (2). 5 deaths were in pts with IsIGHD, ISS & SGA (Table).
The total death rate in GH-Tx pts with IsIGHD, ISS & SGA in GeNeSIS was similar to the 2007 US age adjusted mortality rate for children aged 514 years (rate: 15.3/100 000 PY)2. SMRs for each diagnosis individually & combined were not elevated.
There was no evidence of increased mortality in the GH-Tx IsIGHD, ISS or SGA pts in GeNeSIS. However, our analysis in pts during GH treatment is not directly comparable with SAGhE. Other limitations include comparison with general pop data (lacking untreated controls) & limited follow-up time.
References: 1 Carel J-C et al. 2011 Endocr Rev 32 LB-5.
2 Xu J et al. 2010 National Vital Statistics Reports 58(19).
Declaration of interest: The authors declare that there is a conflict of interest.
Funding: This work was supported, however funding details are unavailable.