Enzyme replacement therapy initiated in adulthood: Findings from the mucopolysaccharidosis VI Clinical Surveillance Program.
Mol Genet Metab
; 127(4): 355-360, 2019 08.
Article
in En
| MEDLINE
| ID: mdl-31324526
ABSTRACT
OBJECTIVE:
To evaluate the impact of galsulfase enzyme replacement therapy (ERT) when initiated in adulthood for patients with mucopolysaccharidosis (MPS) VI.METHODS:
In 2005, the multi-national, MPS VI Clinical Surveillance Program (CSP) was established to collect long-term observational data from routine clinical and laboratory assessments. A sub-analysis was performed in patients who started ERT at ≥16â¯years of age and had received galsulfase for ≥6â¯months. Urinary glycosaminoglycans (uGAG), 6-min walk test (6MWT), 3-min stair climb test (3MSCT), pulmonary function measures, cardiac function, ophthalmology measures, liver and spleen sizes, and safety were evaluated.RESULTS:
Of 223 patients enrolled in the CSP, 51 were included in the sub-analysis. Patients were between 16 and 63â¯years of age at first infusion. From pre-treatment baseline, uGAG level decreased by a mean (±standard deviation [SD]) of 66 (±45)% (Nâ¯=â¯29) after a median follow-up of 7.2â¯years. 6MWT distance decreased slightly by a mean of 17 (±107) meters (Nâ¯=â¯23) after 6.6â¯years. Stairs/min in the 3MSCT increased by a mean of 26 (±33) (Nâ¯=â¯14) after 2.8â¯years. Pulmonary function measures, forced expiratory volume in 1 second and forced vital capacity, increased by a mean of 0.06 (±0.21) L after 7.3â¯years and 0.05 (±0.28) L after 7.2â¯years, respectively (Nâ¯=â¯19 for both measures). Overall, galsulfase was well tolerated, with most adverse events reported being MPS-related clinical manifestations and not related to galsulfase.CONCLUSIONS:
Results of this sub-analysis of the CSP suggest that initiation of galsulfase in adulthood is well tolerated and can possibly stabilize MPS VI in the long term.Key words
Full text:
1
Database:
MEDLINE
Main subject:
Registries
/
Mucopolysaccharidosis VI
/
N-Acetylgalactosamine-4-Sulfatase
/
Enzyme Replacement Therapy
Type of study:
Clinical_trials
/
Diagnostic_studies
/
Observational_studies
/
Screening_studies
Limits:
Adolescent
/
Adult
/
Female
/
Humans
/
Male
/
Middle aged
Language:
En
Journal:
Mol Genet Metab
Journal subject:
BIOLOGIA MOLECULAR
/
BIOQUIMICA
/
METABOLISMO
Year:
2019
Type:
Article