Immune tolerance after long-term enzyme-replacement therapy among patients who have mucopolysaccharidosis I

Kakavanos, Revecca; Turner, Cathy; Hopwood, John J.; Kakkis, Emil; Brooks, Doug A.

doi:10.1016/s0140-6736(03)13311-9

Cited by 94 publications

(77 citation statements)

References 27 publications

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“…7,8 These trials typically involved subjects with less severe clinical phenotypes of MPS I (i.e., Scheie and Hurler-Scheie syndromes), and suggested that antibodies that develop against laronidase are nonneutralizing and do not effect efficacy. 12 Similar to these data, in our series of 12 patients with Hurler syndrome, infusionassociated reactions were infrequent and mild. Three patients had follow-up data on antibody titers after initiation of ERT; all three developed positive titers against the enzyme.…”

Section: Discussionsupporting

confidence: 86%

Safety and efficacy of enzyme replacement therapy in combination with hematopoietic stem cell transplantation in Hurler syndrome

Grewal

Wynn

Abdenur

et al. 2005

Genetics in Medicine

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Purpose: Hurler syndrome is a debilitating genetic disease with a typical life span of 5 to 8 years. Early hematopoietic stem cell transplantation (HSCT) mitigates disease symptoms and improves survival. However, morbidity and mortality associated with HSCT can limit its success. We describe the initial experience with combined use of enzyme replacement therapy (ERT, laronidase) and HSCT in Hurler syndrome. Methods: Thirteen transplants were performed in 12 patients. ERT was given at a standard dose of 0.58 mg/kg per week. Transplant conditioning regimen and donor graft source were determined by institutional protocol. Results: The median age at initiation of ERT was 12 months (range, 8 to 18 months). The median duration of pre-HSCT ERT was 12 weeks (range, 4 to 28). All but 1 patient tested showed decrease in urinary GAG excretion during ERT. ERT infusion-related

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Section: Discussionsupporting

confidence: 86%

Safety and efficacy of enzyme replacement therapy in combination with hematopoietic stem cell transplantation in Hurler syndrome

Grewal

Wynn

Abdenur

et al. 2005

Genetics in Medicine

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“…Preexisting antibodies of various specificities have been described including those with reactivity to PEG, glycans, microbial (bacterial, fungal, etc.) toxins, viral proteins, and cytokines (13)(14)(15)(16)(17)(18). Naturally occurring antibodies to neoepitopes that are revealed after conformational change of a biotherapeutic with reactivity to the hinge region and/or other internal residues of the IgG have also been reported (19)(20)(21).…”

Section: Classification (Definition) Of Pre-existing Drug-reactive Anmentioning

confidence: 99%

Recommendations for the Assessment and Management of Pre-existing Drug-Reactive Antibodies During Biotherapeutic Development

Xue

Clements-Egan

Amaravadi

et al. 2017

AAPS J

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Abstract.Anti-drug antibodies (ADA) pose a potential risk to patient safety and efficacy and are routinely monitored during clinical trials. Pre-existing drug-reactive antibodies are present in patients without prior drug exposure and are defined by their ability to bind to a component of the drug. These pre-existing drug-reactive antibodies are frequently observed and could represent an adaptive immune response of an individual who has been previously exposed to antigens with structural similarities to the biotherapeutic. Clinical consequences of these antibodies can vary from no impact to adverse effects on patient safety, exposure, and efficacy, and are highly dependent on biotherapeutic modality, disease indications, and patient demographics. This paper describes how the immunogenicity risk assessment of a biotherapeutic integrates the existence of pre-existing drug-reactive antibodies, and provides recommendations for risk-based strategies to evaluate treatment-emergent ADA responses.

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“…In fact, immune tolerance developed for each of those patients during a 2-year period. 7 Although the rate of seroconversion has been reported to be much lower for patients receiving replacement enzyme for treatment of Gaucher disease (Ͻ20%), 8 several patients with Gaucher disease have demonstrated clinical deterioration after the development of neutralizing antibodies. Several strategies to provide replacement enzyme while overcoming deleterious antibody effects have been investigated in this setting, including treatment with cytotoxic agents, plasmapheresis, and increasing the amount or frequency of enzyme infusion.…”

mentioning

confidence: 99%

Nephrotic Syndrome Complicating α-Glucosidase Replacement Therapy for Pompe Disease

et al. 2004

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Immune tolerance after long-term enzyme-replacement therapy among patients who have mucopolysaccharidosis I

Cited by 94 publications

References 27 publications

Safety and efficacy of enzyme replacement therapy in combination with hematopoietic stem cell transplantation in Hurler syndrome

Safety and efficacy of enzyme replacement therapy in combination with hematopoietic stem cell transplantation in Hurler syndrome

Recommendations for the Assessment and Management of Pre-existing Drug-Reactive Antibodies During Biotherapeutic Development

Nephrotic Syndrome Complicating α-Glucosidase Replacement Therapy for Pompe Disease

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