EVALUATION OF THE EFFICACY OF GROWTH HORMONE IN PATIENTS WITH A MOLECULAR DIAGNOSIS OF RHEUMATOID ARTHRITIS: ANALYSIS OF A COHORT FROM A TERTIARY CENTER IN NORTHEASTERN BRAZIL
DOI:
https://doi.org/10.56238/arev8n2-032Keywords:
Noonan Syndrome, Mitogen-Activated Kinases, Growth Hormone, Short Stature, Partial GH InsensitivityAbstract
Objective: To evaluate the efficacy of growth hormone (GH) treatment on growth in individuals with a confirmed molecular diagnosis of RASOpathies followed up in an outpatient clinic of a tertiary hospital. Methodology: A cross-sectional and descriptive study was conducted in the Medical Genetics and Pediatric Endocrinology outpatient clinics of a tertiary hospital in Fortaleza. Medical records of patients with a molecular diagnosis of RASOpathies were analyzed, with the collection of clinical, hormonal, and genetic data. Growth was assessed according to World Health Organization growth charts, using height Z-scores, growth velocity, and levels of GH and insulin-like growth factor 1 (IGF-1), bone age and predicted final height, treatment time, and GH dose. Statistical analysis was descriptive. Results: Among 18 patients with RASOpathies, six received GH treatment for short stature, all male. Genetic variants included PTPN11, BRAF, and KRAS. Significant increases in growth velocity were observed in the first and second years of treatment, along with progressive improvement in height Z-score, with an average gain of +1.18 at the end of follow-up. The improved treatment response was associated with early initiation of therapy, GH deficiency, and adequate nutritional status, while clinical comorbidities and partial GH insensitivity limited height gain. Conclusions: GH treatment promoted improved linear growth in patients with RASopathies, with variable responses. The findings reinforce the need for individualized indication and longitudinal follow-up to optimize therapeutic outcomes.
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