Medical Costs Related to Enzyme Replacement Therapy for Mucopolysaccharidosis Types I, II, and VI in Brazil: A Multicenter Study

Bitencourt, Fernanda Hendges de; Vieira, Taiane Alves; Steiner, Carlos Eduardo; Neto, Jordão Corrêa; Boy, Raquel; Schwartz, Ida Vanessa Döederlein

doi:10.1016/j.vhri.2015.08.002

Cited by 7 publications

(9 citation statements)

References 23 publications

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“…There are currently no adequate cost-utility studies with an appropriate time horizon and considering the health system perspective—particularly for countries with universal health systems. Other studies conducted by our group show that patients on ERT undergo fewer medical interventions, which can lead to a reduction in direct medical costs to the publicly funded health care system [ 37 , 42 ].…”

Section: Discussionmentioning

confidence: 99%

“…Although we were not able to perform quantitative measurement of all summarized results, our study also suggests that ERT can affect ROM, with positive effects on shoulder flexion. Interventions such as physical therapy, when provided, may contribute to improved joint mobility, and it is thus advisable that such interventions be reported in all studies that analyze this outcome [ 37 ]. However, none of included studies has mentioned this aspect and this lack of information have been reported elsewhere [ 37 ].…”

Section: Discussionmentioning

confidence: 99%

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Efficacy and safety of intravenous laronidase for mucopolysaccharidosis type I: A systematic review and meta-analysis

et al. 2017

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Mucopolysaccharidosis type I (MPS I) is an autosomal recessive disease caused by deficient activity of alpha-L-iduronidase. Intravenous (IV) enzyme replacement therapy (ERT) with laronidase is currently used for treating patients with MPS I.ObjectiveTo evaluate the efficacy and safety of IV laronidase for MPS I.MethodsA systematic literature review was performed by searching the ClinicalTrials.gov, MEDLINE/PubMed, EMBASE, LILACS, and Cochrane Library databases, limited to clinical trials published until December 31, 2016. The first inclusion criterion was being a randomized controlled trial (RCT). If < five RCTs were identified, open-label and nonrandomized trials, controlled or uncontrolled (quasi-experimental), including ≥ five patients, and evaluating relevant outcomes defined a priori, would also be included. For meta-analysis, primary inferences were based on random-effects models. Assessment of article quality was performed in accordance with the GRADE criteria. The Cochrane Risk of Bias tool was used to examine the risk of bias for RCTs.ResultsThe selection phase retrieved 632 articles. During the first phase of selection, 158 had the abstract or full text read for assessment of eligibility, of which nine (two RCTs) were included for qualitative synthesis. Four papers were included in the meta-analysis, which was performed for the following outcomes: occurrence of treatment-emergent or infusion-related adverse events (65%; 95%CI 53, 76), mild in most cases; development of IgG antibodies to laronidase (88%; 95%CI 67, 100); apnea-hypopnea index (not significant—NS), urinary glycosaminoglycans (GAGs) [mean change -65.5 μg/mg creatinine (95%CI -68.8, -62.3)], liver size [mean change -31.03% (95%CI -36.1, -25.9)], left ventricular mass index (LVMI) [mean change -1.8 (95%CI -2.32, -0.25)], and distance covered in the 6-minute walk test (NS). Among the outcomes not included in meta-analysis, we found evidence for benefit of laronidase only on shoulder flexion.ConclusionsOur findings suggest that IV laronidase effectively reduces urinary GAGs excretion, hepatomegaly and LVMI, and can improve shoulder flexion in MPS I patients. Laronidase appears to be safe in the studied population.

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Efficacy and safety of intravenous laronidase for mucopolysaccharidosis type I: A systematic review and meta-analysis

et al. 2017

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“…In addition, organomegaly decreases, left ventricular hypertrophy and improves the range of joint mobility. [22] For the treatment of otorhinolaryngological failures, the efficacy of ERT in airway permeable maintenance, the reduction of obstruction (tonsillectomy and adenoidectomy) has been reported [23] and the effectiveness in reducing respiratory arrest related to the dream [24]; also to present improvement of the recurrent infections, rhinorrhea, and general respiratory status, expressive and significant improvement of the hearing loss, tympanometric curve pattern, sleep disorders, macroglossia and tonsils and adenoid hypertrophy. [13].…”

Section: Introductionmentioning

confidence: 99%

Otorhinolaryngological Findings in Patients from Southwestern Colombia with Clinical, Enzymatic and Molecular Diagnosis of Mucopolysaccharidosis II, IV-A and VI

Giraldo

Terranova

Soto

2020

J. inborn errors metab. screen.

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Mucopolysaccharidosis is characterized by excessive accumulation of glycosaminoglycan sulfate in organs and tissues. Otorhinolaryngological and upper respiratory tract pathologies are among the earliest clinical manifestations. We realized a retrospective study of clinical and otorhinolaryngologic findings of 35 patients diagnosed with MPS type II, IV-A and VI of the Colombian southwest. As a result, we found that 64% of the patients evaluated had hearing loss, 11.3% had hypertrophy of the tonsils,17.10% short neck and macroglossia. Additionally, 47.8% of the patients presented otitis media. 20% received treatment with hearing aids. no patient reported otosclerosis or tinittus. In patients with different types of MPS, there is a high frequency and progressive tendency to suffer audiological losses and recurrent infections, so it is important an opportune diagnosis, permanent monitoring and adequate therapy to avoid the repercussion of the pathology in the quality of life of patients.

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“…Current therapy options for MPS patients include enzyme replacement therapy (ERT) and hematopoietic stem cell transplantation (HSCT) [2]. ERT was approved for MPS I, II, IVA, VI, and VII by the Food and Drug Administration; however, several limitations are observed: (1) weekly or biweekly infusions for 5-6 h, (2) high cost [10], (3) rapid clearance (a short half-life time, 35-60 min) [11], and (4) limited impact on CNS involvement and skeletal dysplasia [11,12]. HSCT for a patient with MPS I started in 1981 [13].…”

Section: Introductionmentioning

confidence: 99%

Evading the AAV Immune Response in Mucopolysaccharidoses

Piechnik

Sawamoto

Ohnishi

et al. 2020

IJMS

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The humoral immune response elicited by adeno-associated virus (AAV)-mediated gene therapy for the treatment of mucopolysaccharidoses (MPS) poses a significant challenge to achieving therapeutic levels of transgene expression. Antibodies targeting the AAV capsid as well as the transgene product diminish the production of glycosaminoglycan (GAG)-degrading enzymes essential for the treatment of MPS. Patients who have antibodies against AAV capsid increase in number with age, serotype, and racial background and are excluded from the clinical trials at present. In addition, patients who have undergone AAV gene therapy are often excluded from the additional AAV gene therapy with the same serotype, since their acquired immune response (antibody) against AAV will limit further efficacy of treatment. Several methods are being developed to overcome this immune response, such as novel serotype design, antibody reduction by plasmapheresis and immunosuppression, and antibody evasion using empty capsids and enveloped AAV vectors. In this review, we examine the mechanisms of the anti-AAV humoral immune response and evaluate the strengths and weaknesses of current evasion strategies in order to provide an evidence-based recommendation on evading the immune response for future AAV-mediated gene therapies for MPS.

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Medical Costs Related to Enzyme Replacement Therapy for Mucopolysaccharidosis Types I, II, and VI in Brazil: A Multicenter Study

Cited by 7 publications

References 23 publications

Efficacy and safety of intravenous laronidase for mucopolysaccharidosis type I: A systematic review and meta-analysis

Efficacy and safety of intravenous laronidase for mucopolysaccharidosis type I: A systematic review and meta-analysis

Otorhinolaryngological Findings in Patients from Southwestern Colombia with Clinical, Enzymatic and Molecular Diagnosis of Mucopolysaccharidosis II, IV-A and VI

Evading the AAV Immune Response in Mucopolysaccharidoses

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