Growth charts for patients with Hunter syndrome

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.

Therapies for the bone in mucopolysaccharidoses

“…Patients with MPS appear normal at birth although some types of MPS show excessive growth during the first 2-4 years of age [2,3]. MPS varies from severe systemic bone dysplasia to a lesser form of the disease that includes mild bone involvement, depending upon MPS type and clinical phenotype.…”

Impact of enzyme replacement therapy and hematopoietic stem cell therapy on growth in patients with Hunter syndrome

“…Student's t-test was performed on the height, weight, and BMI data for patients according to therapy type to determine the statistical relationship between these two patient groups. These data were also compared with the values in untreated patients with MPS II [19]. The standard control data was taken from established Japanese data sets for healthy male controls issued by the Japanese Ministry of Health, Labor, and Welfare.…”

“…This study was based upon the data obtained from 111 Japanese male patients with MPS II. On average, height and weight measurements were obtained at 8 time points for each patient [19].…”

“…We recently resolved one of the underlying limitations of such studies by establishing growth charts for untreated patients with Hunter syndrome [19].…”

Long-term follow up of post-hematopoietic stem cell transplantation for Hurler syndrome: Clinical biochemical and pathological improvements

“…HSCT also shows some benefits in physical activity and growth. Those studies state that allogeneic HSCT could be potential primary care for MPS I, MPS II [7,8], and MPS VII [9].…”