Growth patterns in patients with mucopolysaccharidosis VII

Adriana M Montaño; Agnieszka Różdżyńska-Świątkowska; Agnieszka Jurecka; Antonio Nino Ramirez; Lin Zhang; Deborah Marsden; Raymond Y Wang; Paul Harmatz

doi:10.1016/j.ymgmr.2023.100987

Growth patterns in patients with mucopolysaccharidosis VII

Mol Genet Metab Rep. 2023 Jun 26:36:100987. doi: 10.1016/j.ymgmr.2023.100987. eCollection 2023 Sep.

Authors

Adriana M Montaño¹, Agnieszka Różdżyńska-Świątkowska², Agnieszka Jurecka³, Antonio Nino Ramirez³, Lin Zhang³, Deborah Marsden³, Raymond Y Wang^{4

5}, Paul Harmatz⁶

Affiliations

¹ Department of Pediatrics, and Biochemistry and Molecular Biology, School of Medicine, Saint Louis University, St. Louis, MO, USA.
² Anthropology Laboratory, Children's Memorial Health Institute, Warsaw, Poland.
³ Ultragenyx Pharmaceutical Inc., Novato, CA, USA.
⁴ Division of Metabolic Disorders, Children's Hospital of Orange County, USA.
⁵ Division of Pediatrics, University of California-Irvine School of Medicine, Orange, CA, USA.
⁶ UCSF Benioff Children's Hospital, Oakland, CA, USA.

Abstract

Objective: This study assessed growth patterns in patients with mucopolysaccharidosis (MPS) VII before enzyme replacement therapy.

Methods: Height, weight, and body mass index (BMI) measurements and Z-scores from patients from three clinical studies were compared with those from CDC healthy population growth charts. Relationships with age/sex and history of non-immune hydrops fetalis (NIHF) were assessed by linear regression and ANOVA, respectively.

Results: Among 20 enrolled patients with MPS VII, height Z-scores were near normal until 1 year of age but declined thereafter, particularly among males. There was no consistent pattern in weight Z-score. BMI Z-scores were above normal and increased slightly with age among males and were slightly below normal among females. Male patients with a history of NIHF had greater declines in height and weight Z-scores over time versus males without history of NIHF. There was no clear effect of NIHF history on height and weight Z-scores in female patients.

Conclusions: In patients with MPS VII, declines in height Z-score began early in life, particularly among males, while changes in BMI varied by sex. Patients with MPS VII and a history of NIHF had greater declines in height Z-score with age than did patients without a history of NIHF.Clinical trial registration: This retrospective analysis included patients enrolled in an open-label phase 2 study (UX003-CL203; ClinicalTrials.gov, NCT02418455), a randomized, placebo-controlled, blind-start phase 3 study (UX003-CL301; ClinicalTrials.gov, NCT02230566), or its open-label, long-term extension (UX003-CL202; ClinicalTrials.gov, NCT02432144). Requests for individual de-identified participant data and the clinical study report from this study are available to researchers providing a methodologically sound proposal that is in accordance with the Ultragenyx data sharing commitment. To gain access, data requestors will need to sign a data access and use agreement. Data will be shared via secured portal. The study protocol and statistical analysis plan for this study are available on the relevant clinical trial registry websites with the tabulated results.

Keywords: Growth; Height; MPS VII; Weight, BMI.

Associated data

ClinicalTrials.gov/NCT02230566
ClinicalTrials.gov/NCT02432144
ClinicalTrials.gov/NCT02418455