The primary objective of this study is to evaluate the quality of life and caregiver burden issues related to disability and adaptive behavior in children with neuromuscular disease (see 'Center for Neuromuscular Diseases').
In order to do this the study will focus on the using patient-based measures. Patient-based outcome measure is a short-hand term used to refer to a collection of questionnaires, interviews and other related ways of evaluating health from the patient's perspective. These instruments are completed by the patients themselves or sometimes when necessary, by their caregivers. The Irving Study hopes to compare the patient based outcome measures to the traditional, physician based assessments. From this comparison, we can explore the relative weaknesses and strengths of each of these instruments to orthopaedic intervention in this unique population.
The end goal of this study is to develop the first ever Disease Specific Instrument (DSI) for this population. A Disease Specific Instrument is an instrument (for example, a questionnaire) that has been developed to measure the patient's perceptions of a specific disease or health problem. The advantage to developing a Disease Specific Instrument is that they provide an assessment of a particular dimension of health that is often more detailed than that provided by generic instruments that attempt to cover broader aspects of health. Many instruments, which include measures of physical functioning as well as psychological well-being, have been widely used. Therefore, there is a wide range of data available for comparing and interpreting results.
Who are Eligible?
All children and adolescents (ages 5-18 years) with a diagnosis that involves a neurological basis for their musculoskeletal condition will be eligible to participate in this study. Some common neuromuscular conditions include cerebral palsy, muscular dystrophy, and spinal muscular atrophy (See 'Center for Neuromuscular Disorders' for more Information). Patients who require surgery will be followed for a 2 year period.
Study Procedures/Recruitment
Eligible patients and their caregivers will be asked if they are willing to participate in this study. After obtaining informed consent, they will be enrolled into the study at the point of contact by the consulting pediatric orthopaedic surgeon and/or a study coordinator. This initial visit with the surgeon will only involve patient enrollment into the study. Once informed consent has been obtained at this visit, patients and their caregivers will be asked to come back for their study related visit.
Surgical Patients
Patients who require surgery will be asked to come in for a preoperative evaluation and three postoperative evaluations (6months, 1year, 2 years after surgery).
Non-Surgical Patients
Patients who do not require surgery will be asked to come in for a baseline evaluation.
What happens at each of the study visits?
After scheduling a convenient time between the study patients and the researchers, the study patients will be asked to come back in for their study evaluation. This involves participation of both the caregiver and the study patient at the Neurological Institute located on 168th and Fort Washington Street (just two blocks from Babies Hospital). At the neurological institute the study patient will meet with Dr. Krasinski and her research team for a physical therapy assessment of the patient.
While the patient is undergoing their physical therapy assessment, the caregivers will be administered a set of questionnaires by the research coordinator. These questionnaires collect information about:
Quality of Life
For example: During the last week, how happy has your child been with what clothes or shoes he/she can wear?
Caregiver Burden
For example: Given your child's health condition, how able are you to take care of other family members like you want to?
Motor Skills
For example: Is your child able to walk down stairs with alternating feet, without assistance?
The time needed to complete all study questionnaires and the physical therapy assessment at each study time point is approximately 1 to 1.5 hours.
Purpose
Surgical Patients