Although enzyme replacement therapy (ERT) has become an alternative for treating Hunter's syndrome (MPS II) in the last 10 years, evidence on its efficacy and safety is limited. This review analyzed 22 studies on ERT for MPS II. It found that ERT significantly improved 6-minute walk tests, vital lung capacity, left ventricular mass index and ejection fraction. ERT also showed benefits for urinary glycosaminoglycans, spleen and liver size, quality of life and articular mobility, but less clear effects on growth, cognitive impairment and sleep apnea. Adverse effects occurred in 7-8% of patients and were not related to antibodies, usually appearing in the first 3 months. One study found home
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Ldn2020 pw
1. 21%
68%
12%
GOOD FAIR POOR
Fig 4. Non-clinical trial studies quality
evaluation (https://www.nhlbi.nih.gov/
health-topics/study-quality-assessment-
tools).
Urinary Glycosaminoglycans Huge reduction in all the included publications(26/26).
Spleen and liver size Huge reduction in all the included publications (18/18).
Mortality
Burton, B., Jego, V., Mikl, J. & Jones, S. Survival in idursulfase
and untreated patients with mucopolysaccharidosis type II:
Data from the Hunter Outcome Survey (HOS).
J. Inherit. Metab. Dis. 40, 867–874 (2017).
Quality of life Benefit in all the studies. Better in attenuated phenotype. Few and small studies.
Cardiac valvules Estabilizing effect.
Articular mobility Modest benefitial effect.
Growth Small benefit.
Cognitive impairment No clear effect (Some benefit in attenuated phenotype). /
Aopnea/Hypopnea index Unclear effect.
113. Enzymatic replacement therapy in Hunter’s
syndrome (MPS II): A systematic review with narrative
synthesis and meta-analysis.
Introduction
*Esteban-Giner MJ1, Wikman-Jorgensen PE2, 3, López-Amorós A4, Jorge Peris-García2, Pedro Jesús Esteve-Atiénzar2, Cañizares-Navarro R2, Robert X2, Seguí-Ripoll JM2,Giner-Galvañ V2, 5, 6.
1Rare Diseases Unit. Department of General Internal Medicine. Hospital Mare de Déu dels Lliris. Alcoy (Alicante). Spain. 2Rare Diseases Unit. Department of General Internal Medicine. Hospital Universitario de San Juan. San Juan de
Alicante (Alicante). Spain. 3Fundación para el Fomento de la Investigación Sanitaria y Biomédica de la Comunitat Valenciana (FISABIO). Valencia. Spain. 4Department of General Internal Medicine. Hospital Universitario de Elda.
Elda (Alicante). Spain.5Departamento de Medicina Clínica. Facultad de Medicina. Universidad Miguel Hernández. Elche (Alicante). Spain. 6Liga para la investigación Traslacional en Enfermedades Raras de la Comunidad
Valenciana. Valencia. Spain. *Corresponding authors: wikman_phi@gva.es / giner_vicgal@gva.es
Objective
Results
Although during the last 10 years the enzyme replacement therapy (ERT) has become
an alternative for the treatment of patients with Hunter´s disease (HD) after the
publication of some clinical trials and observational studies. Nevertheless, the
information regarding efficacy and safety is scarce and mainly based on the pivotal
trials. This scarcity is especially strong for adults and severe forms of HD.
To evaluate the efficacy of ERT in HD in terms of the impact on clinical and safety
variables.
Methodology
Bibliographic search
o Bibliographic sources: PubMed, Embase, Cochrane Central.
o Key words:"Therapeutics" AND "Iduronate Sulfatase" AND "Mucopolysaccharidosis II".
o Exclusion criteria: none.
o Date of the search: Until September 30, 2018.
o Selection process (Fig 1): The initial found entries were screened by title and
abstract. The final decision to include an article was based on the full text.
Analysis of the quality of the evidence
o Quality of the evidence: Evaluation was undertaken using the recommendations of
the Cochrane Collaboration for clinical trials, and the recommendations of the
National Library of Health for the rest of the study types. The complete applied
protocol is available through e-mail to the first corresponding author.
Fig 1. Flow chart of the bibliographic review.
Fig 2. Overall risk of bias of the clinical trials.
Fig 3. Risk of bias of the included clinical trials.
Metanalyzed results
Six-minute walking test (Walked meters)
Vital Forced Capacity (%)
Left ventricular mass index (g/m2)
Left ventricular Ejection Fraction (%)
Non-Metanalyzed results
Safety
¡ 22 studies were included: 8 clinical trials, 8 observational studies, 4 clinical case
series and 2 individual clinical cases.
¡ Serious adverse effects (AE) were presented by 7-8 % of patients with mild/severe
AE occurring in the comparison group with a similar frequency (4 clinical trials).
¡ AE are not related to IgG anti-IDS antibodies, and they ussually appears during
the first 3 months of ERT.
¡ One observational study (n 671 patient/week for 6 months) demonstrated that
IDSα administered at home was as secure as at hospital and improve patient’s
compliance.
VGG 2020
: Benefitial effect. The clearer the benefit, the higher the number of this symbol for the analyzed parameter. : No clear effect.