GROWTH-HORMONE THERAPY IN SILVER-RUSSELL-SYNDROME - 5 YEARS EXPERIENCE OF THE AUSTRALIAN AND NEW-ZEALAND GROWTH DATABASE (OZGROW)

Data were analysed on 33 children (22 males) with Silver Russell syndr ome created with growth hormone for periods up to 5 years. Baseline da ta (medians) at commencement of growth hormone (GH) therapy were age 6 .7 years, bone age delay 1.7 years, height standard deviation score (S DS)-3.2, weight SDS -3.1, and growth velocity 5.7 cm/year. All were pr epubertal. Median birth weight SDS for gestational age was -3.2. GH wa s commenced at 14 IU/m(2) per week and subsequently adjusted according to response. Growth velocity and growth velocity SDS for chronologica l age (CA) improved over baseline and grains in height SDS for CA were 1.0, 1.5 and 1.8 SD over 3, 4 and 5 years respectively (P < 0.001). N o significant increase in height SDS for bone age was observed. Increa sed GH doses were required after the 1st year to maintain growth rates . Mean bone age advancement was 3.1 years after 3 years of treatment, and 6.0 years after 5 years treatment. Younger age was a predictor of the growth response over the 1st year. Predictors of response after 3 years were catch-up growth, low weight SDS at birth and low height SDS for CA. Age at onset of puberty was normal, but height at onset of pu berty was lower than normal means. Conclusion We have demonstrated sig nificant improvement in growth in Silver Russell syndrome after 3 year s of GH therapy, however data on estimated mature height and final hei ght are insufficient to conclude final outcomes. Further follow up is required to assess the long-term benefit.