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Predicting ambulatory function at skeletal maturity in children with moderate to severe osteogenesis imperfecta

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Abstract

Maximizing ambulation is a key treatment aim in moderate to severe osteogenesis imperfecta (OI). Here we investigated which early clinical characteristics predicted ambulation function at skeletal maturity. We assessed Bleck ambulation scores in 88 individuals with OI at 5 to 6 years of age and again at final height (at 15 to 24 years of age). At 5 to 6 years of age, 33 (38%) children were non-ambulators, 32 (36%) were fully independent ambulators, and 23 (26%) had intermediate ambulation skills. At skeletal maturity, 58% of the study participants had the same mobility level as at first assessment. The ability to ambulate independently at skeletal maturity was predicted by independent ambulation at 5 to 6 years (odds ratio [OR] 22.6, 95% confidence interval [CI] 4.9–105; P < 0.001), height z score at 5 to 6 years (OR 3.1, CI 1.6–6.3; P = 0.001) and weight z score at 5 to 6 years (OR 0.44, CI 0.19–0.99; P = 0.04).

Conclusion: Independent ambulation at 5 to 6 years was the main determinant of independent ambulation at skeletal maturity. This highlights the importance of maximizing ambulation in children below 5 years of age.

What is Known:

•walking ability varies markedly between OI types. The highest level of mobility was found in OI type I, the lowest in OI type III who require mobility aids; intermediate levels were reported for OI type IV.

• OI type is a key predictor of ultimate ability to ambulate, whereas the timing of developmental milestones was not associated with walking ability

What is New:

• overall key predictors of mobility function at skeletal maturity were mobility status and height z-score at 5-6 years of age

• Childrenwho were non-ambulators at 5 to 6 years of age had a higher chance of having better mobility at skeletal maturity if they had good upper extremity function, as expressed in the PEDI Self Care Score

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Abbreviations

OI:

Osteogenesis imperfecta

PEDI:

Pediatric Evaluation of Disability Inventory

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Acknowledgments

We thank the multi-disciplinary team of Shriners Hospital for Children - Canada who assess, treat and support the families of children with osteogenesis imperfecta.

Funding

This study was supported by the Shriners of North America.

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Authors and Affiliations

  1. Shriners Hospital for Children, 1003 Decarie Boulevard, Montreal, Quebec, Canada

    Kathleen Montpetit, Marie-Elaine Lafrance, Francis H. Glorieux, François Fassier, Reggie Hamdy & Frank Rauch

  2. McGill University, Montreal, Canada

    Francis H. Glorieux, François Fassier, Reggie Hamdy & Frank Rauch

Authors
  1. Kathleen Montpetit
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  2. Marie-Elaine Lafrance
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  3. Francis H. Glorieux
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  4. François Fassier
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  5. Reggie Hamdy
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  6. Frank Rauch
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Contributions

KM and FR: study design, analysis, and production of manuscript. KM and MEL: data collection. FG, FF, and RH: revision and contributed patient data. All authors read and approved the final manuscript.

Corresponding author

Correspondence to Kathleen Montpetit.

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Conflict of interest

The authors declare that they have no conflict of interest.

Ethical approval

This research study was conducted retrospectively from data obtained for clinical purposes. The study was approved by the Institutional Review Board of McGill University.

Informed consent

This research study was conducted retrospectively from data obtained for clinical purposes. The study was approved by the Institutional Review Board of McGill University who determined Informed consent was not applicable.

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Communicated by Peter de Winter

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Cite this article

Montpetit, K., Lafrance, ME., Glorieux, F.H. et al. Predicting ambulatory function at skeletal maturity in children with moderate to severe osteogenesis imperfecta. Eur J Pediatr 180, 233–239 (2021). https://doi.org/10.1007/s00431-020-03754-w

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  • DOI: https://doi.org/10.1007/s00431-020-03754-w

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