Neurofibromatosis Type 1 Clinical Trial
Official title:
Multi-center Project: Spinal Abnormalities in Neurofibromatosis Type1 (NF1) Patients
We propose to establish a multi-center study to investigate the outcome of scoliosis and
spinal abnormalities in patients with NF1.
The three specific aims of this study are:
Specific Aim 1 - To assess health status and health-related quality of life (HRQL) in
children and adolescents with NF1 and scoliosis. We hypothesize that children and adolescents
with NF1 and scoliosis will experience an additional burden of morbidity due to scoliosis and
a downward trajectory of health status and HRQL over time.
Specific Aim 2 - To assess the natural history and short-term response to therapy in a cohort
of children with NF1 and scoliosis prospectively diagnosed during the course of the four-year
study period. We hypothesize that some NF1 patients with idiopathic scoliosis will modulate
to the dystrophic form. We also hypothesize that NF1 patients with earlier presentation are
more likely to have or modulate to the dystrophic form.
Specific Aim 3 - To assess biochemical markers of bone metabolism in NF1 individuals. We
hypothesize that NF1 individuals will have statistically significant differences in
biochemical markers of bone metabolism compared to controls. We also hypothesize that NF1
individuals with scoliosis will have differences in biochemical markers of bone metabolism
compared to NF1 individuals without scoliosis.
The three specific aims of this study are:
Specific Aim 1 - To assess health status and health-related quality of life (HRQL) in
children and adolescents with NF1 and scoliosis. We hypothesize that children and adolescents
with NF1 and scoliosis will experience an additional burden of morbidity due to scoliosis and
a downward trajectory of health status and HRQL over time.
Specific Aim 2 - To assess the natural history and short-term response to therapy in a cohort
of children with NF1 and scoliosis prospectively diagnosed during the course of the four-year
study period. We hypothesize that some NF1 patients with idiopathic scoliosis will modulate
to the dystrophic form. We also hypothesize that NF1 patients with earlier presentation are
more likely to have or modulate to the dystrophic form.
Specific Aim 3 - To assess biochemical markers of bone metabolism in NF1 individuals. We
hypothesize that NF1 individuals will have statistically significant differences in
biochemical markers of bone metabolism compared to controls. We also hypothesize that NF1
individuals with scoliosis will have differences in biochemical markers of bone metabolism
compared to NF1 individuals without scoliosis.
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