Effects of Standing on Non-Ambulatory Children With Neuromuscular Conditions
Condition: Osteopenia; Spinal Muscular Atrophy; Cerebral Palsy; Muscular Dystrophy; Spina Bifida; Rett Syndrome
MeSH terms: Muscular Dystrophies; Bone Diseases, Metabolic; Cerebral Palsy; Muscular Atrophy; Muscular Atrophy, Spinal; Rett Syndrome; Spinal Dysraphism; Atrophy
Study type: Interventional

Summary: 
      Children with neuromuscular disabilities and limited ambulation are at significant risk for
      decreased bone mineral density (BMD) and increased incidence of fracture. This is caused, in
      part, by low levels of load experienced by the skeleton due to a child's functional
      limitations. Low BMD has been shown to be predictive of fracture, and in fact, fractures
      usually occur without significant trauma in children with neuromuscular conditions. The
      discomfort and distress from fractures in this population are considerable, and the
      associated costs to the family and healthcare system are substantial. Numerous interventions
      have been devoted to improving BMD in these children. Stationary assisted standing devices
      are widely used and represent the standard-of-care. However, evidence supporting this
      approach is limited due to inadequate study designs with insufficient numbers of patients.

      This study will use load-sensing platforms in patients with neuromuscular conditions.
      Successful completion of this pilot study will assist in the development of a future
      multicenter clinical trial to definitively determine relationships, if any, between passive
      standing and measures of BMD, fracture incidence, pulmonary function, and health-related
      quality-of-life measures in children with a variety of neuromuscular disabilities (e.g.,
      spinal muscular atrophy, cerebral palsy, muscular dystrophy, spina bifida, Rett syndrome).

      Hypothesis: Assisted standing treatment program will gradually increase their duration of
      standing by up to 75% after the baseline phase.
    

Detailed description: 
      Children with neuromuscular disabilities and limited ambulation are at significant risk for
      decreased bone mineral density (BMD) and increased incidence of fracture. This is caused, in
      part, by low levels of load experienced by the skeleton due to a child's functional
      limitations. Low BMD has been shown to be predictive of fracture, and in fact, fractures
      usually occur without significant trauma in children with neuromuscular conditions such as
      cerebral palsy, spinal muscular atrophy, or other muscular dystrophies. The discomfort and
      distress from fractures in this population are considerable, and the associated costs to the
      family and healthcare system are substantial. Numerous interventions have been devoted to
      improving BMD in these children. Stationary assisted standing devices are widely used and
      represent the standard-of-care. However, evidence supporting this approach is limited due to
      inadequate study designs with insufficient numbers of patients.

      Various modifications are added to assisted-standing devices to allow children with
      neuromuscular impairments to achieve standing postures. These customizations lead to
      load-sharing with the standing device, and consequently, a decrease in the amount of load
      passing through the lower extremities. Previous investigators developed custom-made
      load-measuring sensors to quantify the amount of load borne by the lower extremities while in
      passive standers, and found that the actual load varied from 23-102% of the child's body
      weight. These load-measuring sensors were specifically developed for restricted laboratory
      testing, rather than recording the load magnitude and duration of standing in any brand of
      standing device during daily use at home, school or therapy.

      We previously developed load-sensing platforms that accurately measure loads experienced by
      the lower extremities of children with cerebral palsy in passive standers. These platforms
      can be incorporated into any stander design, are able to be used on a routine basis at a
      child's home, school or therapy, and are able to record the duration of weight-bearing to
      monitor compliance.

      This study will use these load-sensing platforms in patients with neuromuscular conditions.
      Successful completion of this pilot study will assist in the development of a future
      multicenter clinical trial to definitively determine relationships, if any, between passive
      standing and measures of BMD, fracture incidence, pulmonary function, and health-related
      quality-of-life measures in children with a variety of neuromuscular disabilities (e.g.,
      spinal muscular atrophy, cerebral palsy, muscular dystrophy, spina bifida, Rett syndrome).
    

Intervention name(s): Assisted Standing Treatment Program
Intervention type(s): Other
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: Osteopenia; spinal muscular atrophy; cerebral palsy; muscular dystrophy; spina bifida; Rett Syndrome