Efficacy, safety, quality of life, adherence and cost-effectiveness of long-acting growth hormone replacement therapy compared to daily growth hormone in children with growth hormone deficiency: A systematic review and meta-analysis.

We evaluated the efficacy, safety, adherence, quality of life (QoL) and cost-effectiveness of long-acting growth hormone (LAGH) vs daily growth hormone (GH) preparations in the treatment of growth hormone deficiency (GHD) in children. Systematic searches were performed in PubMed, Embase and Web of Science up to July 2022 on randomized and non-randomized studies involving children with GHD receiving LAGH as compared to daily GH. Meta-analyses for efficacy and safety were performed comparing different LAGH/daily GH formulations. From the initial 1393 records, we included 16 studies for efficacy and safety, 8 studies for adherence and 2 studies for QoL. No studies reporting cost-effectiveness were found. Pooled mean differences of mean annualized height velocity (cm/year) showed no difference between LAGH and daily GH: Eutropin Plus® vs Eutropin® [- 0.14 (-0.43, 0.15)], Eutropin Plus® vs Genotropin® [- 0.74 (-1.83, 0.34)], Jintrolong® vs Jintropin AQ® [0.05 (-0.54, 0.65)], Somatrogon vs Genotropin® [- 1.40 (-2.91, 0.10)], TransCon vs Genotropin® [0.93 (0.26, 1.61)]. Also, other efficacy and safety outcomes, QoL and adherence were comparable for LAGH and daily GH. Our results showed that, although most of the included studies had some concerns for risk of bias, regarding efficacy and safety all the LAGH formulations were similar to daily GH. Future high quality studies are needed to confirm these data. Adherence and QoL should be addressed from real-world data studies for both the mid and long term and in a larger population. Cost-effectiveness studies are needed to measure the economic impact of LAGH from the healthcare payer's perspective.

Epidemiology of growth hormone deficiency in children and adolescents: a systematic review.

OBJECTIVE: Growth hormone deficiency (GHD) is the most common pituitary hormone deficiency and is one of the main causes of short stature in children and adolescents. The aim of this study is to evaluate the epidemiology of pediatric GHD worldwide, since no other systematic review has been published so far. METHODS: We searched PubMed, Embase, and Web of Science up to July 2023 to find epidemiological studies involving children with GHD. Two review authors independently screened articles, extracted data and performed the quality assessment. RESULTS: We selected 9 epidemiological studies published from 1974 to 2022. The range of prevalence was 1/1107-1/8,646. A study based on a registry of GH users in the Piedmont region (Italy) reported the highest mean prevalence. In the included studies, the mean incidence ranged from 1/28,800 to 1/46,700 cases per year. One study reported a 20-year cumulative incidence of 127/100,000 for boys and 93/100,000 for girls. Studies were heterogeneous in terms of population (age and GHD etiology) and diagnostic criteria. As for the methodological quality of included studies, all but one study satisfied the majority of the checklist items. CONCLUSIONS: The included studies are mostly European, so the provided estimates cannot be considered global. International multicentre studies are needed to compare epidemiological estimates of GHD among different ethnical groups. Considering the considerable cost of human recombinant GH, the only available therapy to treat GHD, understanding accurate epidemiological estimates of GHD in each country is fundamental for resource allocation.

Partial oesophagectomy for giant leiomyoma of the oesophagus: report of 7 cases.

OBJECTIVES: Oesophageal leiomyoma is the most common benign tumour of the oesophagus. The incidence of leiomyomas larger than 10 cm, defined as giant oesophageal leiomyomas (GELs), has been reported in 17% of all cases. Although computed tomographic scan and endoscopy are usually useful for diagnosis, big and symptomatic masses located in the lower mediastinum remain both a diagnostic and therapeutic challenge. METHODS: We describe our experience in the management of 7 patients (4 males and 3 females, with a mean age of 41 years) with GEL treated in our department. Radical resection was performed in all cases with partial oesophagectomy in order to relieve symptoms and to obtain a definitive diagnosis. RESULTS: There was no perioperative mortality. The minimum diameter of the tumours was 15 cm and the maximum was 30 cm. Definitive histological examination confirmed the diagnosis of leiomyoma in all cases without any sign of malignancy. No major postoperative complications developed. Minor complications included partial abdominal wound dehiscence in 1 case, and retention of secretions requiring bronchoscopy in 2. The mean length of hospital stay was 12 days (ranging between 9 and 14 days). After a mean follow-up of 5.4 years (ranging between 12 and 2 years), no sign of recurrence was observed. CONCLUSIONS: Whereas removal of small oesophageal leiomyomas can be performed by simple enucleation by conventional thoracotomy or video-assisted thoracoscopy, partial oesophagectomy is often necessary for giant lesions. Since it is not possible preoperatively to distinguish GEL from leiomyosarcoma when metastases are absent, partial oesophageal resection is not to be considered an overtreatment and radical resection should always be planned. A gastric tube, in our experience employed as an oesophageal substitute, is effective and could reduce the risk of significant postoperative gastro-oesophageal reflux.