Japan Elaprase® Treatment (JET) study: Idursulfase enzyme replacement therapy in adult patients with attenuated Hunter syndrome (Mucopolysaccharidosis II, MPS II)

Okuyama, Torayuki; Tanaka, Akemi; Suzuki, Yasuyuki; Ida, Hiroyuki; Tanaka, Toju; Cox, Gerald F.; Eto, Yoshikatsu; Orii, Tadao

doi:10.1016/j.ymgme.2009.08.006

Cited by 132 publications

(171 citation statements)

References 26 publications

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“…Inicialmente, foram identificados 374 estudos (Figura 1), mas apenas dois eram ECRs, duploscegos, comparando IDS ao placebo 13,14 , enquanto três preencheram critérios como estudos não randomizados 15,16,17 . Nenhum estudo comparando TRE a outro tratamento foi encontrado e não foram obtidas informações adicionais do registro de ensaios clínicos.…”

Section: Resultados Características Dos Estudos Incluídosunclassified

Eficácia e segurança da terapia com idursulfase em pacientes com mucopolissacaridose tipo II, com e sem comparação com placebo: revisão sistemática e metanálise

et al. 2013

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Section: Resultados Características Dos Estudos Incluídosunclassified

Eficácia e segurança da terapia com idursulfase em pacientes com mucopolissacaridose tipo II, com e sem comparação com placebo: revisão sistemática e metanálise

et al. 2013

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“…Five of ten patients in the JET study had retinal degeneration (Okuyama et al 2009). Although retinal pigmentation had not been documented in PM before the completion of 12 months therapy with idursulfase, the history of restricted visual fields argues that he had pigmentary retinopathy of a long-standing nature.…”

Section: Discussionmentioning

confidence: 97%

“…The oldest phase I/II/ III trial patient was 31 years old, and around half were prepubertal at the time of baseline investigations (Muenzer et al 2006;2007). A more meaningful comparison can be made with the patients in the JET study; an investigation of ten Japanese adults with MPS II following 12 months therapy with idursulfase in whom the mean age was 31 and the oldest patient was 53 (Okuyama et al 2009). Patients in the JET study significantly improved their liver and spleen volumes, normalized urinary GAG's, and improved 6MWT and FVC although these last two measurements were not increased to statistically significant levels.…”

Section: Discussionmentioning

confidence: 99%

Three Adult Siblings with Mucopolysaccharidosis Type II (Hunter Syndrome): A Report on Clinical Heterogeneity and 12 Months of Therapy with Idursulfase

2011

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Mucopolysaccharidosis type II (MPS IIHunter syndrome) is a rare X-linked recessive disease of lysosomal glycosaminoglycan metabolism leading to a systemic storage disorder. We report three adult brothers (aged 46-52 years) with attenuated Hunter syndrome after 12 months of enzyme replacement therapy with idursulfase. Before enzyme replacement therapy, each had serious complications of their disease: in addition to all requiring urgent cervical spinal canal decompressions in middle age, one required emergency aortic and mitral valve surgery, another had stage IV airways disease, and the third had acute glaucoma resulting in unilateral blindness. One brother discontinued therapy after 12 months. The other two brothers reported subjective improvements in energy and exercise tolerance.

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“…Elaprase ® (idursulfase) is another enzyme produced by biotechnological processes used in the treatment of patients with Hunter syndrome (patients are not able to degrade glycosaminoglycans, which gradually accumulates in cells, affecting most organs, causing difficulty breathing and walking) (Okuyama et al, 2010).…”

Section: Enzymesmentioning

confidence: 99%

Drugs obtained by biotechnology processing

Almeida

Amaral

Lobão

2011

Braz. J. Pharm. Sci.

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In recent years, the number of drugs of biotechnological origin available for many different diseases has increased exponentially, including different types of cancer, diabetes mellitus, infectious diseases (e.g. AIDS Virus / HIV) as well as cardiovascular, neurological, respiratory, and autoimmune diseases, among others. The pharmaceutical industry has used different technologies to obtain new and promising active ingredients, as exemplified by the fermentation technique, recombinant DNA technique and the hybridoma technique. The expiry of the patents of the first drugs of biotechnological origin and the consequent emergence of biosimilar products, have posed various questions to health authorities worldwide regarding the definition, framework, and requirements for authorization to market such products.
Nos últimos anos, tem aumentado exponencialmente o número de fármacos de origem biotecnológica ao dispor das mais diversas patologias, entre elas destacam-se, os diferentes tipos de cancêr, as doenças infecciosas (ex. vírus AIDS/HIV), as doenças autoimunes, as doenças cardiovasculares, a Diabetes Mellitus, as doenças neurológicas, as doenças respiratórias, entre outras. A indústria farmacêutica tem recorrido a diferentes tecnologias para a obtenção de novos e promissores princípios ativos, como são exemplo a fermentação, a técnica de DNA Recombinante, a técnica de hidridoma, entre outras. A queda das patentes dos primeiros fármacos de origem biotecnológica e o consequente aparecimento dos produtos biossimilares têm colocado diferentes questões às autoridades de saúde mundiais, sobre a definição, enquadramento e exigências para a autorização de entrada no mercado deste tipo de produtos

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Japan Elaprase® Treatment (JET) study: Idursulfase enzyme replacement therapy in adult patients with attenuated Hunter syndrome (Mucopolysaccharidosis II, MPS II)

Cited by 132 publications

References 26 publications

Eficácia e segurança da terapia com idursulfase em pacientes com mucopolissacaridose tipo II, com e sem comparação com placebo: revisão sistemática e metanálise

Eficácia e segurança da terapia com idursulfase em pacientes com mucopolissacaridose tipo II, com e sem comparação com placebo: revisão sistemática e metanálise

Three Adult Siblings with Mucopolysaccharidosis Type II (Hunter Syndrome): A Report on Clinical Heterogeneity and 12 Months of Therapy with Idursulfase

Drugs obtained by biotechnology processing

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