Growth patterns in patients with mucopolysaccharidosis VII
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 populati...
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| Format: | Article |
| Language: | English |
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Elsevier
2023-09-01
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| Series: | Molecular Genetics and Metabolism Reports |
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| Online Access: | http://www.sciencedirect.com/science/article/pii/S2214426923000332 |
| _version_ | 1827860757675507712 |
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| author | Adriana M. Montaño Agnieszka Różdżyńska-Świątkowska Agnieszka Jurecka Antonio Nino Ramirez Lin Zhang Deborah Marsden Raymond Y. Wang Paul Harmatz |
| author_facet | Adriana M. Montaño Agnieszka Różdżyńska-Świątkowska Agnieszka Jurecka Antonio Nino Ramirez Lin Zhang Deborah Marsden Raymond Y. Wang Paul Harmatz |
| author_sort | Adriana M. Montaño |
| collection | DOAJ |
| description | 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. |
| first_indexed | 2024-03-12T13:27:27Z |
| format | Article |
| id | doaj.art-68199889bc9745e98000b49f745e7638 |
| institution | Directory Open Access Journal |
| issn | 2214-4269 |
| language | English |
| last_indexed | 2024-03-12T13:27:27Z |
| publishDate | 2023-09-01 |
| publisher | Elsevier |
| record_format | Article |
| series | Molecular Genetics and Metabolism Reports |
| spelling | doaj.art-68199889bc9745e98000b49f745e76382023-08-25T04:24:14ZengElsevierMolecular Genetics and Metabolism Reports2214-42692023-09-0136100987Growth patterns in patients with mucopolysaccharidosis VIIAdriana M. Montaño0Agnieszka Różdżyńska-Świątkowska1Agnieszka Jurecka2Antonio Nino Ramirez3Lin Zhang4Deborah Marsden5Raymond Y. Wang6Paul Harmatz7Department of Pediatrics, and Biochemistry and Molecular Biology, School of Medicine, Saint Louis University, St. Louis, MO, USA; Corresponding author at: Doisy Research Center, Department of Pediatrics, Saint Louis University,1100 South Grand Blvd., Room 313, St. Louis, MO 63104, USA.Anthropology Laboratory, Children's Memorial Health Institute, Warsaw, PolandUltragenyx Pharmaceutical Inc., Novato, CA, USAUltragenyx Pharmaceutical Inc., Novato, CA, USAUltragenyx Pharmaceutical Inc., Novato, CA, USAUltragenyx Pharmaceutical Inc., Novato, CA, USADivision of Metabolic Disorders, Children's Hospital of Orange County, USA; Division of Pediatrics, University of California-Irvine School of Medicine, Orange, CA, USAUCSF Benioff Children's Hospital, Oakland, CA, USAObjective: 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.http://www.sciencedirect.com/science/article/pii/S2214426923000332MPS VIIHeightWeight, BMIGrowth |
| spellingShingle | Adriana M. Montaño Agnieszka Różdżyńska-Świątkowska Agnieszka Jurecka Antonio Nino Ramirez Lin Zhang Deborah Marsden Raymond Y. Wang Paul Harmatz Growth patterns in patients with mucopolysaccharidosis VII Molecular Genetics and Metabolism Reports MPS VII Height Weight, BMI Growth |
| title | Growth patterns in patients with mucopolysaccharidosis VII |
| title_full | Growth patterns in patients with mucopolysaccharidosis VII |
| title_fullStr | Growth patterns in patients with mucopolysaccharidosis VII |
| title_full_unstemmed | Growth patterns in patients with mucopolysaccharidosis VII |
| title_short | Growth patterns in patients with mucopolysaccharidosis VII |
| title_sort | growth patterns in patients with mucopolysaccharidosis vii |
| topic | MPS VII Height Weight, BMI Growth |
| url | http://www.sciencedirect.com/science/article/pii/S2214426923000332 |
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