Recombinant GH(Hamad General Hospital)

Recombinant human growth hormone (GH) therapy shows variable growth responses in patients with growth hormone deficiency (GHD), highlighting the need for reliable biomarkers to predict individual sensitivity.

This study investigated circulating microRNAs (miRNAs) involved in growth plate regulation during GH therapy in prepubertal children with GHD, aiming to establish a miRNA panel correlating with GH responsiveness.

Sixteen patients were treated with daily recombinant GH (0.033 mg/kg) for 6 months. Two participants were excluded for protocol noncompliance, and one withdrew due to the spontaneous onset of puberty. Circulating levels of six miRNAs (miR‐22‐3p, miR‐30c‐5p, miR‐140‐5p, miR‐340‐5p, miR‐494‐3p, and miR‐16‐5p) were measured via digital PCR at baseline and at 1, 3, and 6 months of therapy. Clinical data (height, weight, growth velocity) and hormone levels were collected simultaneously.

All miRNAs displayed a characteristic response pattern: significant increases at 1 and 6 months, with a transient decline at 3 months. Despite these changes, no significant correlations were identified between miRNA levels and growth velocity, height SDS, or IGF‐1 levels. The study included treatment‐naïve patients and those with prior GH therapy following a 30‐day wash‐out period. While the treatment‐naïve group exhibited greater height‐SDS gains (p = 0.008), no differences in miRNA expression were observed between groups, nor was there a correlation between miRNA levels and height‐SDS increments.

While this miRNA panel identified treatment‐responsive miRNAs, it did not correlate with growth outcomes. Increasing the sample size and incorporating additional miRNAs could enhance its clinical utility for predicting GH treatment sensitivity in GHD patients.

NCT05946915.

Growth hormone insensitivity syndrome (GHIS) is a rare genetic disorder characterized by short stature due to the body's inability to effectively utilize growth hormone (GH). This case report describes a patient with concurrent hypothyroidism and GHIS. This patient is an 11-year-old female presented with short stature; general examination suggested a prominent forehead and a depressed nasal bridge. Laboratory evaluations revealed elevated thyroid-stimulating hormone (TSH) levels alongside low levels of triiodothyronine (T3) and thyroxine (T4), indicating hypothyroidism. Additionally, elevated GH levels and significantly reduced insulin-like growth factor 1 (IGF-1) levels confirmed the diagnosis of GHIS. The patient was managed with thyroid hormone replacement therapy and recombinant GH. This dual therapeutic approach will lead to improvements in both thyroid function and growth parameters. This case underscores the importance of recognizing and addressing coexisting endocrine disorders in patients with GHIS to optimize their growth and developmental outcomes. Early diagnosis and a comprehensive treatment strategy are essential for managing such complex cases effectively.