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. 2014:1:5-18.
doi: 10.1016/j.ymgmr.2013.10.001.

Growth charts for patients with Hunter syndrome

Pravin Patel  1 , Yasuyuki Suzuki  2 , Miho Maeda  1 , Eriko Yasuda  1 , Tsutomu Shimada  1 , Kenji E Orii  3 , Tadao Orii  4 , Shunji Tomatsu  1
Affiliations

Growth charts for patients with Hunter syndrome

Pravin Patel et al. Mol Genet Metab Rep. 2014.

Abstract

Children with mucopolysaccharidosis II (MPS II), also known as Hunter syndrome, an X-linked disorder, suffer from a multisystem dysfunction caused by the accumulation of glycosaminoglycans. However, there has been no systemic report on the growth of patients with MPS II. The purpose of this study is to describe the growth patterns of patients with MPS II and to compare with the patterns of age-matched controls. Data (height, weight, age, etc.) was collected in a longitudinal study of Japanese male patients with MPS II (n = 111). The mean birth length was 50.31 ± 1.42 cm, while the mean birth weight was 3.35 ± 0.39 kg. The mean final height and weight at 18 years and older were 125.63 ± 9.09 cm and 37.18 ± 8.72 kg; corresponding to a difference of -46.40 cm and -25.89 kg lower, when compared with healthy Japanese male controls. The mean birth BMI was 10.84 ± 3.29 kg/m2, while the mean BMI at 18 years was 29.41 ± 6.15 kg/m2. The growth pattern in patients with MPS II was characterized by overgrowth for the first several years, although growth velocity fell below that of the normal healthy controls after one year of age. No statistical difference in height was observed between patients with the attenuated and severe phenotypes in each age class. In conclusion, this report describes the natural history of growth in patients with MPS II, which can help in monitoring the progression of the disease as well as assessing therapeutic efficacy.

Keywords: Growth charts; Height Velocity; Hunter Syndrome; Lysosomal Storage Disorders; Mucopolysaccaridosis type II.

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Figures

Fig. 1
Fig. 1
Comparison of mean height between patients with MPS II and age-matched normal controls. Difference in mean height between patients with MPS II and normal controls is expressed as centimeters. Error bars show ± 1 SD.
Fig. 2
Fig. 2
Growth chart for patients with MPS II from birth to 18 years of age. The dotted line shows the mean height for healthy males. The solid line shows heights for MPS II patients. Arrows point to their respective percentile curve.
Fig. 3
Fig. 3
Comparison of mean height between patients with severe and attenuated phenotype. The dotted line shows the mean height for patients with an attenuated phenotype. The solid line shows the mean height for patients with a severe phenotype.
Fig. 4
Fig. 4
Comparison of the mean height between patients with attenuated and severe phenotypes. From left to right; severe phenotype (newborn), attenuated phenotype (newborn), severe phenotype (18 years old), and attenuated phenotype (18 years old). Error bars show + 1 SD.
Fig. 5
Fig. 5
Growth velocity from birth to 18 years of age in patients with MPS II. The dotted line shows the growth velocity of the normal control. The solid line shows the mean height velocity for patients with MPS II.
Fig. 6
Fig. 6
Comparison of mean weight between patients with MPS II and age-matched normal controls. Difference in mean height between patients with MPS II weight and normal controls is expressed as kilograms. Error bars show ± 1 SD.
Fig. 7
Fig. 7
Weight curve for patients with MPS II from birth to 18 years of age. The dotted line shows the mean height for healthy males. The solid line shows weights for patients with MPS II. Arrows point to their respective percentile curves.
Fig. 8
Fig. 8
Comparison of mean weight between patients with severe and attenuated phenotypes. The dotted line shows the mean weight for patients with an attenuated phenotype. The solid line shows the mean weight for patients with a severe phenotype.
Fig. 9
Fig. 9
Comparison of mean weights between patients with attenuated and severe phenotypes. From left to right; severe phenotype (newborn), attenuated phenotype (newborn), severe phenotype (18 years old), and attenuated phenotype (18 years old). Error bars show + 1 SD. * Shows a significant difference.
Fig. 10
Fig. 10
BMI curve for patients with MPS II from birth to 18 years of age. The dotted line shows the mean BMI for healthy males. The solid line shows BMIs for patients with MPS II. Arrows point to their respective percentile curves.
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