Mortality is not increased in rhGH-treated patients when adjusting for birth characteristics.
Artikel i vetenskaplig tidskrift, 2016

Objective: This study aimed to investigate whether reported high mortality in childhood recombinant human GH (rhGH)-treated patients was related to birth-characteristics and/or rhGH treatment. Design and Setting: We sought to develop a mortality model of the Swedish general population born between 1973 and 2010, using continuous-hazard functions adjusting for birth characteristics, sex, age intervals, and calendar year to estimate standardized mortality ratio (SMR) and to apply this model to assess expected deaths in Swedish rhGH-treated patients with idiopathic isolated GH deficiency (IGHD), idiopathic short stature (155) or born small for gestational age (SGA). Participants:The general population: Swedish Medical Birth Register (1973-2010: 1 880 668 males; 1 781 131 females) and Cause of Death Register (1985-2010). Intervention Population: Three thousand eight hundred forty-seven patients starting rhGH treatment between 1985 and 2010 and followed in the National GH Register and/or in rhGH trials diagnosed with IGHD (n = 1890), ISS (n = 975), or SGA (n=982). Main Outcome Measures: Death. Results: Using conventional models adjusting for age, sex, and calendar-year, the SMR was 1.43 (95% confidence interval, 0.89-2.19), P = .14, observed/expected deaths 21/14.68. The rhGH population differed (P < .001) from the general population regarding birth weight, birth length, and congenital malformations. Application of an Advanced Model: When applying the developed mortality model of the general population, the ratio of observed/expected deaths in rhGH-treated patients was 21/21.99; SMR = 0.955 (0.591-1.456)P = .95. Model Comparison: Expected number of deaths were 14.68 (14.35-14.96) using the conventional model, and 21.99 (21.24-22.81) using the advanced model, P < .001, which had at all ages a higher gradient of risk per SD of the model, 24% (range, 18-42%; P < .001). Conclusions: Compared with the general Swedish population, the ratio of observed/expected deaths (21/21.99) was not increased in childhood rhGH-treated IGHD, ISS, and SGA patients when applying an advanced sex-specific mortality model adjusting for birth characteristics.

Författare

Kerstin Albertsson-Wikland

Göteborgs universitet

Anton Mårtensson

Göteborgs universitet

Lars Sävendahl

Karolinska universitetssjukhuset

Aimon Niklasson

Göteborgs universitet

Peter Bang

Linköpings universitet

Jovanna Dahlgren

Göteborgs universitet

Jan Gustafsson

Uppsala universitet

Berit Kriström

Umeå universitet

Svante Norgren

Karolinska universitetssjukhuset

Nils-Gunnar Pehrsson

Statistiska Konsultgruppen

Anders Odén

Chalmers, Matematiska vetenskaper, matematisk statistik

Göteborgs universitet

The Journal of clinical endocrinology and metabolism

1945-7197 (eISSN)

Vol. 101 5 2149-2159

Ämneskategorier

Pediatrik

DOI

10.1210/jc.2015-3951

PubMed

26918292

Mer information

Senast uppdaterat

2018-02-28