Israeli study evaluates actual results of growth hormone treatment
Available research suggests that the success of GH treatment depends on the age at which treatment is started, the quality of family adherence to treatment, the indication and gender. The 2 most common reasons for GH treatment are GH deficiency (GHD) and idiopathic short stature (ISS). Other indications are Turner syndrome (TS), Prader-Willi syndrome (PWS), chronic renal failure (CRF), offspring for gestational age (SGA) and Noonan syndrome.
This large retrospective database study included data on 2379 children starting GH treatment at Maccabi Healthcare Services (MHS) between January 2004 and December 2014. The MHS database includes up to 20 years of data on 2 million people. members and represents a 25% sample of the Israeli population.
The database integrates data from electronic patient records (EMRs), which includes growth parameters, indication for treatment, and socio-economic factors.
The objectives of the study were to assess the prescribing regimens, adherence and results of treatment with GH in patients followed from the start of treatment and for at least 12 months.
Most of the patients starting GH treatment were men (62.1%) with a mean [SD] age 9.8 [3.1] years. The height standard deviation score (SDS) was -2.36 [0.65] and the time from the diagnosis of small size to the first purchase of GH was 4.8 [3.3] years.
The results also showed that the ISS accounted for the majority of diagnoses leading to treatment with GH (m = 1615, or nearly 68%) and GHD accounted for 611 cases, or 25.7%.
The mean duration of treatment was 3.5 [0.95] years and almost 80% of children have been treated for more than 3 years.
After 3 years, the mean SDS height gain was 1.09 [0.91] for GHD and 0.96 [0.57] for the ISS (P = .0004).
Adult height (15 years for girls and 17 years for boys) was recorded for 624 patients (26.2%) with better results for GHD than for ISS (-1.0 [0.82] against -1.28 [0.93], respectively; P = .0002).
Good treatment adherence, assessed during follow-up, was defined as the proportion of days covered (PDC)> 80%. Membership declined over time, investigators found. Just over 78% of the group had good adhesion in the first year; the rate fell to 68.1% in the third year of treatment.
Recombinant GH therapy in this study took the form of daily injections, and since adherence was defined as PDC, it could not take into account the possibility that parents or guardians would not give the injection. some days.
Interestingly, most of the patients were from high socioeconomic backgrounds (SES) (61.3% for ISS and 59.7% for GHD). This differed from the general pediatric MHS population, where 23% belong to the highest SES tertile and 14.4% belong to the lowest tertile.
GH treatment is slanted with respect to SES levels, the authors said, and is unlikely to be solely due to cost concerns, as the cost of treatment in Israel is heavily subsidized by health maintenance organizations.
Previous studies have indicated that parents seeking assessment and treatment for their children’s short stature tend to have higher levels of education and income, and that “referral decisions are strongly influenced by level. of parental concern and the attitudes of physicians, ”the researchers noted.
Clinicians should be aware that GH deficiency issues may be underestimated in patients with lower SES tertiles, they said.
Strengths of the study included the fact that it was based on high-quality standardized data with a large sample size and a long follow-up period.
“Proper referral, diagnosis and follow-up care may lead to better treatment outcomes with GH therapy” the researchers said.
Ben-Ari T, Chodick G, Shalev V, Goldstein D, Gomez R, Landau Z. Real-world treatment models and growth hormone treatment outcomes in children in Israel over the past decade (2004 -2015). Pediatrician before Published online August 20, 2021. doi: 10.3389 / fped.2021.711979