TY - JOUR
T1 - Osteogenesis imperfecta in childhood impairment and disability. A prospective study with 4-year follow-up
AU - Engelbert, Raoul H
AU - Uiterwaal, Cuno S
AU - Gerver, Willem-Jan
AU - van der Net, Jan-Jaap
AU - Pruijs, Hans E
AU - Helders, Paul J
PY - 2004
Y1 - 2004
N2 - OBJECTIVES: To study (1). changes in anthropometrics, joint range of motion (ROM), muscle strength, functional ability, caregiver assistance, and level of ambulation in children with osteogenesis imperfecta (OI) and (2). the prediction of clinical characteristics at the level of ambulation at follow-up and the prediction of clinical characteristics on progression or regression at the level of ambulation over time.DESIGN: Prospective study with follow-up of 4 years.SETTING: A children's hospital that serves a nationwide center for treatment and research in children with OI in the Netherlands.PARTICIPANTS: At follow-up, 49 children (24 boys, 25 girls; mean age +/- standard deviation, 11.3+/-3.8y; range, 5.2-19.4y) participated.INTERVENTIONS: Not applicable.MAIN OUTCOME MEASURES: Anthropometry, joint ROM, muscle strength, fracture frequency, intramedullary rodding, level of ambulation, functional ability, and caregiver assistance.RESULTS: In type I OI, total joint ROM decreased significantly over time, especially in the lower extremities, with a significant decrease in generalized joint hypermobility according to Bulbena (median start, 7.5; interquartile range [IQR], 4-9; median end, 6; IQR, 2-7; P<.001). In types III and IV, a severe decrease in total joint ROM was present without significant changes over time. No significant changes in total muscle strength (upper or lower extremities) in the different types of OI were measured at follow-up. In OI type I, a significant increase in self-care (P=.003) and social function (P=.008) was measured; in type III, a significant increase in self-care (P=.003), mobility (P=.004), and social function (P=.005) was measured, with a significant decrease in parental assistance in self-care (P=.02) and mobility (P=.005). In type IV, a significant increase was observed in the self-care (P=.01) and social function domains (P=.02). Type of OI (regression coefficient=-1.0; 95% confidence interval [CI], -1.64 to -0.47) and total muscle strength were the only significant predictors for level of ambulation (regression coefficient=.01; 95% CI,.17-.32). Body weight was significantly lower in the group that progressed in level of ambulation (P=.03), whereas children with a decline in level of ambulation had significantly higher body weight (P=.05).CONCLUSIONS: Ours is the first study with a long-term follow-up that provides information concerning the natural course of developmental outcome parameters of OI in childhood. Joint ROM and muscle strength did not change significantly over time, possibly because of the biomechanical skeletal properties of the different OI types. Functional ability improved significantly over time, but, especially in types III and IV, did not reach normative values, possibly because of a plateau phase in functional ability. Knowledge of the natural course of the disease is essential to interpret the results from intervention studies.
AB - OBJECTIVES: To study (1). changes in anthropometrics, joint range of motion (ROM), muscle strength, functional ability, caregiver assistance, and level of ambulation in children with osteogenesis imperfecta (OI) and (2). the prediction of clinical characteristics at the level of ambulation at follow-up and the prediction of clinical characteristics on progression or regression at the level of ambulation over time.DESIGN: Prospective study with follow-up of 4 years.SETTING: A children's hospital that serves a nationwide center for treatment and research in children with OI in the Netherlands.PARTICIPANTS: At follow-up, 49 children (24 boys, 25 girls; mean age +/- standard deviation, 11.3+/-3.8y; range, 5.2-19.4y) participated.INTERVENTIONS: Not applicable.MAIN OUTCOME MEASURES: Anthropometry, joint ROM, muscle strength, fracture frequency, intramedullary rodding, level of ambulation, functional ability, and caregiver assistance.RESULTS: In type I OI, total joint ROM decreased significantly over time, especially in the lower extremities, with a significant decrease in generalized joint hypermobility according to Bulbena (median start, 7.5; interquartile range [IQR], 4-9; median end, 6; IQR, 2-7; P<.001). In types III and IV, a severe decrease in total joint ROM was present without significant changes over time. No significant changes in total muscle strength (upper or lower extremities) in the different types of OI were measured at follow-up. In OI type I, a significant increase in self-care (P=.003) and social function (P=.008) was measured; in type III, a significant increase in self-care (P=.003), mobility (P=.004), and social function (P=.005) was measured, with a significant decrease in parental assistance in self-care (P=.02) and mobility (P=.005). In type IV, a significant increase was observed in the self-care (P=.01) and social function domains (P=.02). Type of OI (regression coefficient=-1.0; 95% confidence interval [CI], -1.64 to -0.47) and total muscle strength were the only significant predictors for level of ambulation (regression coefficient=.01; 95% CI,.17-.32). Body weight was significantly lower in the group that progressed in level of ambulation (P=.03), whereas children with a decline in level of ambulation had significantly higher body weight (P=.05).CONCLUSIONS: Ours is the first study with a long-term follow-up that provides information concerning the natural course of developmental outcome parameters of OI in childhood. Joint ROM and muscle strength did not change significantly over time, possibly because of the biomechanical skeletal properties of the different OI types. Functional ability improved significantly over time, but, especially in types III and IV, did not reach normative values, possibly because of a plateau phase in functional ability. Knowledge of the natural course of the disease is essential to interpret the results from intervention studies.
KW - Activities of Daily Living
KW - Adolescent
KW - Adult
KW - Anthropometry
KW - Body Weight
KW - Caregivers
KW - Child
KW - Child, Preschool
KW - Disabled Children
KW - Female
KW - Follow-Up Studies
KW - Fracture Fixation, Intramedullary
KW - Fractures, Bone
KW - Humans
KW - Joints
KW - Locomotion
KW - Lower Extremity
KW - Male
KW - Muscle, Skeletal
KW - Osteogenesis Imperfecta
KW - Prospective Studies
KW - Range of Motion, Articular
KW - Self Care
KW - Social Adjustment
KW - Journal Article
M3 - Article
C2 - 15129402
SN - 0003-9993
VL - 85
SP - 772
EP - 778
JO - Archives of physical medicine and rehabilitation
JF - Archives of physical medicine and rehabilitation
IS - 5
ER -