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Growth charts for patients affected with Morquio A disease
Adriana M Montaño1, Shunji Tomatsu, Ana Brusius
1Department of Pediatrics, Saint Louis University, St. Louis, Missouri 63104, USA.
Insights
Children with Morquio A disease experience poor growth and physical handicaps. This study developed growth charts for Morquio A patients, revealing impaired growth velocity after age one, aiding disease monitoring and treatment assessment.
Area of Science:
- Pediatrics
- Genetics
- Metabolic Disorders
Background:
- Morquio A disease causes significant growth impairment and physical disability in children due to systemic bone disease.
- Understanding growth patterns is crucial for managing the condition and its associated health complications.
Purpose of the Study:
- To establish growth curves for patients with Morquio A.
- To analyze the relationship between growth patterns and the physical condition of affected individuals.
- To provide a tool for monitoring disease progression and treatment efficacy.
Main Methods:
- A one-center study involving 354 patients with Morquio A.
- Utilized questionnaire-based longitudinal and cross-sectional data.
- Developed growth charts and assessed physical activity and surgical incidence.
Main Results:
- Mean final heights for males and females were significantly below normal (-7.4 and -7.7 SD, respectively).
- Impaired growth velocity was observed after the first year of age.
- Established normative growth data for Morquio A patients.
Conclusions:
- This study presents the first growth charts specifically for Morquio A patients.
- These charts are valuable for monitoring growth and evaluating therapeutic interventions.
- The findings highlight the critical need for early diagnosis and management of growth issues in Morquio A.
Abstract:
Children with Morquio A disease grow poorly and become physically handicapped because of systemic bone disease. The purpose of this study was to describe observed growth patterns and their relationship with the physical condition of patients with Morquio A. In a one-center study, questionnaire-based longitudinal and cross sectional data were used to develop growth curves, to assess physical activity and to determine the incidence of surgical procedures in 354 patients with Morquio A. Mean birth lengths of boys and girls were 52.6 and 52.1 cm, respectively. The mean final heights for males and females at 18 years and older were 122.4 +/- 21.5 and 113.1 +/- 22.6 cm, respectively. These results corresponded to -7.4 SD for males and -7.7 SD for females compared to the normal healthy controls. Mean birth weights for boys and girls were 3.59 +/- 0.58 and 3.5 +/- 0.7 kg, respectively. The mean body mass index for males and females at over 18 years of age was 24.7 +/- 6.1 and 25.6 +/- 5.4 kg/m(2), respectively. The growth pattern in Morquio A patients was characterized by impaired growth velocity after 1 year of age. This is the first report providing growth charts for patients with Morquio A, which can help with monitoring the disease and assessing the clinical efficacy of treatments.
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