الفهرس 
IntroductionOnly 14 pages are availabe for public view
 |  | from | 135 |  |  |
| | | from | 135 | | |
Abstract
S
UMMARY
steogenesis imperfecta is the most common genetic bone fragility disorder in children. Zoledronate, a bisphosphonate used in osteoporosis treatment showed promising results in children with OI. It can serve as a treatment modality directed to achieve normal height with improvement in bone health and quality of life.
This study was conducted on 28 patients with the diagnosis of OI to find out effect of zoledronate on functional mobility and scoliosis degree in children with osteogensis imperfecta. All parameters were evaluated at the Pediatric Endocrinology clinic, Children’s Hospital, Ain Shams University.
In this study intravenous Zoledronate given to our patients with OI improved their heights (by almost 25 cm irrespective of years of treatment), DXA parameters (BMD LS, BMD LS Z score to HA and BMD total body), FMS in addition to less self-reported fractures. Although nearly all DXA parameters increased, yet only BMD total body results showed statistically significant increase.
Our patients were divided according to years of treatment to (≤ 6y) 16 patients (57.1%) and (> 6y) 12 patients (42.9%). We found that patients receiving zoledronate therapy for > 6y had less deformities degree by scoring system, scoliosis degree
O
Summary
84
and number of self-reported fractures as well as more values of height SDS, FMS, BMD LS (g/cm2), BMD LS Z score (HA) and BMD total body.
Spine deformities are generally common in patients with OI and it was seen in 42.9% of our cases. Patients with mild and moderate degree of scoliosis showed a nonsignificant improvement in the degree of scoliosis while patients with severe scoliosis didn’t show any statistically significant difference. Longer follow-up is necessary to monitor the effect of treatment on degree of scoliosis by cobb angle. The degree of scoliosis did not differ when comparing those who received less or more than 6 years of zoledronate therapy.
As a clear objective tool for assessment of mobility following treatment, FMS was used and showed significant increase after therapy.
Zoledronate should be considered in the treatment of children with OI. On the one hand, these observations reinforce the concept that intravenous zoledronate therapy is not a cure for OI but rather an adjunct treatment for rehabilitation and orthopedic therapies. On the other, it is clear that current medical treatment approaches leave significant scope for further reductions in the disease burden carried by children with severe OI