Effect of Anti-Iduronate 2-Sulfatase Antibodies in Patients with Mucopolysaccharidosis Type II Treated with Enzyme Replacement Therapy

Vollebregt, Audrey A M; Hoogeveen‐Westerveld, Marianne; Ruijter, George J. G.; Hout, Hannerieke van den; Oussoren, Esmée; Ploeg, Ans T. van der; Pijnappel, W.W.M. Pim

doi:10.1016/j.jpeds.2022.05.008

Cited by 1 publication

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“…Individuals with MPS II can develop antibodies to idursulfase. These antibodies are associated with increased GAG in treated patients and are associated with attenuation of the improvement seen in pulmonary function [ 25 , 26 ]. The efficacy of ERT in preventing airway pathology may be affected by these antibodies.…”

Section: Discussionmentioning

confidence: 99%

Airway Findings in Patients with Hunter Syndrome Treated with Intravenous Idursulfase

Vuyst

Jalazo

Tsujimoto³

et al. 2023

JCM

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People with Hunter syndrome are known to be affected by a variety of airway pathologies. Treatment of Hunter syndrome with the enzyme replacement therapy (ERT) idursulfase is now the standard of care. However, it is not known how ERT changes the progression of airway involvement. To evaluate this, we performed a retrospective analysis of bronchoscopies performed on children with Hunter syndrome who were part of intrathecal ERT trials. Findings for airway pathology were extracted from bronchoscopy reports and analyses were performed for cross-sectional and longitudinal changes in airway disease. One-hundred and thirty bronchoscopies from 23 subjects were analyzed. Upper airway disease (adenoid hypertrophy and/or pharyngomalacia) was reported in 93% and 87% of bronchoscopies, respectively. Laryngeal abnormalities were recognized in 46% of cases. There were lower airway (tracheal and or bronchial) findings in 64% of all bronchoscopies and prevalence increased with age. Evaluations over time adjusted for repeat evaluations showed that increasing airway involvement was associated with older age (p = 0.0007) despite ongoing ERT. No association was discovered between age of intravenous ERT initiation and progression of airway disease. Individuals with Hunter syndrome who are receiving intravenous enzyme replacement therapy showed the progression of airways disease supporting the need for regular airway monitoring and intervention.

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Section: Discussionmentioning

confidence: 99%