ABSTRACT
PURPOSE: The Gross Motor Function Measure (GMFM) is a criterion-referenced evaluative measure designed to detect change over time for children diagnosed with cerebral palsy (CP). Reliability of this measure has not been tested for children with osteogenesis imperfecta (OI). The purpose of this study was to determine the intra- and interrater reliabilities of the GMFM for use with children diagnosed with OI. METHOD: One physical therapist administered and scored the GMFM for 19 children with OI who were followed at the Shriners Hospital for Children. The assessments were videotaped, then viewed and scored by five physical therapists, including the author, at least six weeks later. Intra- and interrater reliabilities were assessed using intraclass correlation coefficients (ICCs). Kappa statistics were calculated for items demonstrating more disagreement than the majority. RESULTS: The ICCs for intrarater reliability of the five dimensions and total score were 0.99. The ICCs for interrater reliability were 0.98 for the lying and rolling dimension and 0.99 for the other dimensions and total score. Kappa statistics for items demonstrating more disagreement than the majority ranged from 0.552 to 1.00. CONCLUSIONS: This study provides evidence of the reliability of the GMFM for children with OI when scored by pediatric physical therapists familiar with the measure. The videotape provided a consistent situation because each therapist did not directly interact with each child, but rather rated a videotaped session of the child's performance.
ABSTRACT
Severe osteogenesis imperfecta (OI) is a hereditary disorder characterized by increased bone fragility and progressive bone deformity. Cyclical pamidronate infusions improve clinical outcome in children older than 3 yr of age with severe OI. Because earlier treatment may have potential to prevent deformities and improve functional prognosis in young children, we studied nine severely affected OI patients under 2 yr of age (2.3-20.7 months at entry) for a period of 12 months. Pamidronate was administered i.v. in cycles of 3 consecutive days. Patients received four to eight cycles during the treatment period, with cumulative doses averaging 12.4 mg/kg. Clinical changes were evaluated regularly during treatment, and radiological changes were assessed after 6-12 months of treatment. The control group consisted of six age-matched, severely affected OI patients, who had not received pamidronate treatment. During treatment bone mineral density (BMD) increased between 86-227%. The deviation from normal, as indicated by the z-score, diminished from -6.5 +/- 2.1 to -3.0 +/- 2.1 (P < 0.001). In the control group the BMD z-score worsened significantly. Vertebral coronal area increased in all treated patients (11.4 +/- 3.4 to 14.9 +/- 1.8 cm2; P < 0.001), but decreased in the untreated group (P < 0.05). In the treated patients, fracture rate was lower than in control patients (2.6 +/- 2.5 vs. 6.3 +/- 1.6 fractures/year; P < 0.01). No adverse side-effects were noted, apart from the well known acute phase reaction during the first infusion cycle. Pamidronate treatment in severely affected OI patients under 3 yr of age is safe, increases BMD, and decreases fracture rate.
