Reliability of the tunisian pediatric gait, arms, legs, and spine: toward a valid screening tool for tunisian children with musculoskeletal conditions.

BACKGROUND: Pediatric musculoskeletal disorders account for 10% of first-line consultations in Tunisia. Referral delay and deficiencies in musculoskeletal screening raise a challenge to the early diagnosis and management of rheumatic conditions in children. The pGALS (Pediatric Gait Arms Legs Spine) was developed and translated into many languages to overcome these deficiencies. Our study aimed to adapt and validate pGALS to the Tunisian dialect for school-age children. METHODS: Using the Delphi method, we carried out a cross-cultural adaptation of the pGALS to the Tunisian dialect. This consensual version was validated in a cross-sectional study, in two pediatric centers. RESULTS: Ninety-two patients were enrolled, 43 females (46.7%) and 49 males (53.3%), mean age was 9.4 ± 2.6 years. The mean test duration was 3.4 ± 2.3 min, and the acceptability and comprehension of the test were good. Six patients had a musculoskeletal complaint, 19 had positive pGALS, and 14 were diagnosed with musculoskeletal disease. The internal consistency score (Cronbach's α) was 0.852. The sensitivity of the test was 92.8%, the specificity was 92.3%, the positive likelihood ratio was 2.16, and the negative likelihood ratio was 0.01. CONCLUSION: The pGALS test adapted to the Tunisian dialect is a relevant, quick, and valid tool for screening musculoskeletal abnormalities in school-age children.

Denosumab use in osteogenesis imperfecta: an update on therapeutic approaches

Osteogenesis imperfecta (OI) is an inherited skeletal disorder that leads to bone fragility and multiple fractures. Given advances in the genetic understanding of existing phenotypes and newly discovered mutations, therapeutic management of OI has become challenging. Denosumab, a monoclonal antibody that inhibits the interaction between the receptor activator of nuclear factor kappa B ligand (RANKL) and its receptor RANK, has been approved to treat postmenopausal osteoporosis and emerged as an important therapy for malignancies and other skeletal disorders, including pediatric skeletal conditions such as OI. This review summarizes information about denosumab therapy in OI by exploring its mechanisms of action, main indications, and safety and efficacy. Several case reports and small series have been published about the short-term use of denosumab in children with OI. Denosumab was considered a strong drug candidate for OI patients with bone fragility and a high risk of fracture, particularly for patients with the bisphosphonate (BP)-unresponsive OI-VI subtype. The evidence for denosumab's effects in children with OI indicates that it effectively improves bone mineral density but not fracture rates. A decrease in bone resorption markers was observed after each treatment. Safety was assessed by tracking the effects on calcium homeostasis and reporting side effects. No severe adverse effects were reported. Hypercalciuria and moderate hypercalcemia were reported, suggesting that BPs be used to prevent the bone rebound effect. In other words, denosumab can be used as a targeted intervention in children with OI. The posology and administration protocol require more investigation to achieve secure efficiency.