Effects of switching from imiglucerase to velaglucerase alfa without dose reduction nor wash out in type 1 Gaucher disease

Serratrice, J.; Bengherbia, Monia; Alessandrini, Marine; Grosbois, B.; Camou, Fabrice; Pers, Yves-Marie; Bismuth, Michaël; Marie, I.; Belmatoug, Nadia; Berger, Marc; Gaucher, Comité d'Evaluation et de Traitement de la maladie de

doi:10.1016/j.bcmd.2013.12.004

Cited by 4 publications

(1 citation statement)

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“…In fact, only one infusion-related AE occurred in the 16 patients who switched to velaglucerase alfa over 1.2–4.1 years. Switching from imiglucerase to velaglucerase alfa has been shown to be safe in previous reports, both in patients who transitioned without a break or dose reduction [ 10 , 22 ] and in those who experienced a break or dose adjustment beforehand [ 23 – 25 ].…”

Section: Discussionmentioning

confidence: 99%

Velaglucerase alfa (VPRIV) enzyme replacement therapy in patients with Gaucher disease: Long‐term data from phase III clinical trials

et al. 2015

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Type 1 Gaucher disease is an inherited lysosomal enzyme deficiency with variable age of symptom onset. Common presenting signs include thrombocytopenia, anemia, hepatosplenomegaly, bone abnormalities, and, additionally in children, growth failure. Fifty-seven patients aged 3–62 years at the baseline of two phase III trials for velaglucerase alfa treatment were enrolled in the single extension study. In the extension, they received every-other-week velaglucerase alfa intravenous infusions for 1.2–4.8 years at 60 U/kg, although 10 patients experienced dose reduction. No patient experienced a drug-related serious adverse event or withdrew due to an adverse event. One patient died following a convulsion that was reported as unrelated to the study drug. Only one patient tested positive for anti-velaglucerase alfa antibodies. Combining the experience of the initial phase III trials and the extension study, significant improvements were observed in the first 24 months from baseline in hematology variables, organ volumes, plasma biomarkers, and, in adults, the lumbar spine bone mineral density Z-score. Improvements were maintained over longer-term treatment. Velaglucerase alfa had a good long-term safety and tolerability profile, and patients continued to respond clinically, which is consistent with the results of the extension study to the phase I/II trial of velaglucerase alfa. EudraCT number 2008-001965-27; http://www.clinicaltrials.gov identifier NCT00635427. Am. J. Hematol. 90:584–591, 2015. © 2015 Wiley Periodicals, Inc.

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Section: Discussionmentioning

confidence: 99%

Velaglucerase alfa (VPRIV) enzyme replacement therapy in patients with Gaucher disease: Long‐term data from phase III clinical trials

et al. 2015

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A Systematic Review and Meta-analyses of Longitudinal Studies on Drug Treatments for Gaucher Disease

et al. 2022

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Objective: Gaucher disease (GD) is a rare disorder linked to the absence/deficiency of glucocerebrosidase. GD can be treated by enzyme replacement therapy (ERT) and substrate reduction therapy (SRT). The aim of this systematic review (SR) is to assess the effectiveness of drugs used for GD treatment. Data Sources: Searches were conducted in PubMed and Scopus, in April 2021. The search strategies encompassed the name of the disease and of the drug treatments. Manual search was also conducted. Study Selection and Data Extraction: Observational and interventional longitudinal studies evaluating ERT and SRT for GD were included. Single mean meta-analyses were conducted for each drug using R. Data Synthesis: The initial search retrieved 2246 articles after duplicates were removed. Following screening and eligibility assessment, 68 reports were included. The studies evaluated imiglucerase, velaglucerase alfa, taliglucerase alfa, miglustat, and eliglustat. The results showed that ERT is effective as a treatment in both naïve and experienced patients. Miglustat did not significantly improve blood outcomes in naïve patients and resulted in a decrease in the platelet levels of experienced patients. Eliglustat was mainly assessed for experienced patients and resulted in stable outcome values. Relevance to Patient Care and Clinical Practice: This extensive SR confirms the effectiveness of GD treatments in short- and long-term follow-ups. Conclusions: The results were favorable for all ERTs and for eliglustat. Based on the assessed evidence, miglustat did not achieved expressive results. However, all evidence should be interpreted considering its limitations and does not replace well-conducted randomized trials.

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Joint manifestations revealing inborn metabolic diseases in adults: a narrative review

Loret,

Jacob,

Mammou

et al. 2023

Orphanet J Rare Dis

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Inborn metabolic diseases (IMD) are rare conditions that can be diagnosed during adulthood. Patients with IMD may have joint symptoms and the challenge is to establish an early diagnosis in order to institute appropriate treatment and prevent irreversible damage. This review describes the joint manifestations of IMD that may be encountered in adults. The clinical settings considered were arthralgia and joint stiffness as well as arthritis. Unspecific arthralgias are often the first symptoms of hereditary hemochromatosis, chronic low back pain may reveal an intervertebral disc calcification in relation with alkaptonuria, and progressive joint stiffness may correspond to a mucopolysaccharidosis or mucolipidosis. Gaucher disease is initially revealed by painful acute attacks mimicking joint pain described as “bone crises”. Some IMD may induce microcrystalline arthropathy. Beyond classical gout, there are also gouts in connection with purine metabolism disorders known as “enzymopathic gouts”. Pyrophosphate arthropathy can also be part of the clinical spectrum of Gitelman syndrome or hypophosphatasia. Oxalate crystals arthritis can reveal a primary hyperoxaluria. Destructive arthritis may be indicative of Wilson’s disease. Non-destructive arthritis may be seen in mevalonate kinase deficiency and familial hypercholesterolemia.

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Effects of switching from imiglucerase to velaglucerase alfa without dose reduction nor wash out in type 1 Gaucher disease

Cited by 4 publications

References 5 publications

Velaglucerase alfa (VPRIV) enzyme replacement therapy in patients with Gaucher disease: Long‐term data from phase III clinical trials

Velaglucerase alfa (VPRIV) enzyme replacement therapy in patients with Gaucher disease: Long‐term data from phase III clinical trials

A Systematic Review and Meta-analyses of Longitudinal Studies on Drug Treatments for Gaucher Disease

Joint manifestations revealing inborn metabolic diseases in adults: a narrative review

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