preferential treatment rachel sabatini sga

Treatment with rhGH has been approved in 2009 by the Italian Medicines Agency (AIFA) for the treatment of children with short stature born SGA and is then reimbursed by the Italian National Health System (Servizio Sanitario Nazionale – SSN) according to the Note #39 on the use of drugs. Although considerably lower than the hypothetical one, this prevalence far exceeds the estimated prevalence of GHD (1:4000–1:10,000).Īlthough rhGH treatment is not mandatory in every child born SGA without catch-up growth, and parents need to be informed about the variability of response to therapy, we believe that in Italy many short children born SGA are still not properly identified and therefore rhGH treatment is not offered as an option.Īim of this article is to describe the situation of rhGH prescriptions in short children born SGA in Italy and to provide practical tools for pediatricians to identify children eligible for rhGH treatment according to Italian regulation. Only one Japanese study verified the prevalence of children with short stature born SGA by studying an entire cohort of nearly 30,000 children born during a three-year period and re-evaluated at 3 years of life: the prevalence was found to be 0.06% (1:1800) (notably greater in preterm births < 34 SGA, 0.39%, 1:256). With the limits of a mechanistic calculus, considering a minimum prevalence of 2% children born SGA for weight and/or length, the hypothetical prevalence of short children born SGA at the age of 2 years would be 0.24% (1:417).

Assessment of the GH-IGF-I axis may be required if growth velocity is persistently reduced and signs of GH deficiency or hypopituitarism are present.

Moreover, SGA children with GH deficiency need higher than replacement dose of rhGH for optimum growth response (0,035 mg/kg/day), probably because of some IGF-I resistance. SGA children do not need to be deficient in growth hormone (GH) in order to qualify for, or benefit from, rhGH therapy: in fact, the vast majority of SGA children demonstrate GH levels in the normal range, but appear to have low normal circulating IGF-I concentrations. Although long-term treatment with rhGH can increase adult height, since SGA children are increasingly recognized as a heterogeneous group in which multiple mechanisms of growth retardation and metabolic disturbances could be causative, it has to be kept in mind – and shared with parents prior to treatment – that the response to rhGH therapy is highly variable and additional studies are needed to identify the responders. Recombinant human growth hormone (rhGH) is an approved and effective treatment for short children born SGA. Previous studies found that 8–12% of SGA children will have a short stature at 2 years of life and these children have a higher risk of short stature later in life. Ĭatch-up growth is more pronounced during the first 6 months and is usually completed in the first 2 years of life (although preterm born SGA catch-up growth is completed also beyond the first 2 years of life, beyond age 6 years and sometimes in adolescence). Actually, some population studies have shown different prevalence: 3.1% in Finland out of 1,390,165 singletons, 5.5% in Sweden out of 3650 healthy full-term children (37–43 gestational weeks) (of which 1.6% SGA only for weight, 2.4% SGA only for length, 1.5% SGA both for weight and length), 3.5% in Japan on 27,228 children (3.4% in term children, 5.5% in preterm children 1.2% SGA only for weight, 1.5% SGA only for length, 0.8% SGA both in weight and length). To born small for gestational age (SGA) is considered the main reason for short stature in 10% of short adults.Ĭhildren born SGA should be 2% of the population by definition (− 2 SDS correspond to the 2nd percentile).

Preferential treatment rachel sabatini sga