ArticleOrphanet journal of rare diseases2012
Evaluation of miglustat as maintenance therapy after enzyme therapy in adults with stable type 1 Gaucher disease: a prospective, open-label non-inferiority study.
Article in Orphanet journal of rare diseases, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT00319046 (Open-label, Non Comparative, Multi-center Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Oral Miglustat as a Maintenance Therapy After a Switch From Enzyme Replacement Therapy in Adult Patients With Stable Type 1 Gaucher Disease), which is not on this map. Cited by 20 papers.
What it found
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The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Open-label, Non Comparative, Multi-center Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Oral Miglustat as a Maintenance Therapy After a Switch From Enzyme Replacement Therapy in Adult Patients With Stable Type 1 Gaucher Disease
Who cites it
20 citing papers in PubMed, 48 citations in OpenAlex.
- Trial
- Lucerastat, an iminosugar with potential as substrate reduction therapy for glycolipid storage disorders: safety, tolerability, and pharmacokinetics in healthy subjects.Orphanet journal of rare diseases · 2017Trial
- Trial
- Gaucher disease: the hematologist's perspective of a multisystemic disorder.Annals of hematology · 2026Review
- The Two Substrate Reduction Therapies for Type 1 Gaucher Disease Are Not Equivalent. Comment on Hughes et al. Switching between Enzyme Replacement Therapies and Substrate Reduction Therapies in Patients with Gaucher Disease: Data from the Gaucher Outcome Survey (GOS).Journal of clinical medicine · 2023Article
- An Unexpected Finding of Hepatosplenomegaly in a Pediatric Patient.Clinical pediatrics · 2022Article
- Engineering monocyte/macrophage-specific glucocerebrosidase expression in human hematopoietic stem cells using genome editing.Nature communications · 2020Article
- Impact of the organic cation transporter 2 inhibitor cimetidine on the single-dose pharmacokinetics of the glucosylceramide synthase inhibitor lucerastat in healthy subjects.European journal of clinical pharmacology · 2020Article
- Genome Editing for Mucopolysaccharidoses.International journal of molecular sciences · 2020Review
- A health state utility valuation study to assess the impact of treatment mode of administration in Gaucher disease.Orphanet journal of rare diseases · 2018Review
- Combined miglustat and enzyme replacement therapy in two patients with type 1 Gaucher disease: two case reports.Journal of medical case reports · 2018Article
- Treatment of profound thrombocytopenia in a patient with Gaucher disease type 1: Is there a role for substrate reduction therapy.Molecular genetics and metabolism reports · 2017Article
- Stability is maintained in adults with Gaucher disease type 1 switched from velaglucerase alfa to eliglustat or imiglucerase: A sub-analysis of the eliglustat ENCORE trial.Molecular genetics and metabolism reports · 2016Article
- Successful switch from enzyme replacement therapy to miglustat in an adult patient with type 1 Gaucher disease: a case report.Journal of medical case reports · 2016Article
- Enzyme Replacement or Substrate Reduction? A Review of Gaucher Disease Treatment Options.Hospital pharmacy · 2016Article
- Biochemical response to substrate reduction therapy versus enzyme replacement therapy in Gaucher disease type 1 patients.Orphanet journal of rare diseases · 2016Article
- Observational cohort study of the natural history of Niemann-Pick disease type C in the UK: a 5-year update from the UK clinical database.BMC neurology · 2015Observational
- Switching from imiglucerase to miglustat for the treatment of French patients with Gaucher disease type 1: a case series.Journal of medical case reports · 2015Article
- Cervical lymph node diseases in children.GMS current topics in otorhinolaryngology, head and neck surgery · 2014Review
- Gaucher disease: Progress and ongoing challenges.Molecular genetics and metabolismReview
Corrections and comments
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Authors and funding
8 authors at 5 institutions in 5 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundPrevious studies have provided equivocal data on the use of miglustat as maintenance therapy in Gaucher disease type 1. We report findings from a clinical trial evaluating the effects of miglustat treatment in patients with stable type 1 Gaucher disease after enzyme therapy.
methodsAdult type 1 Gaucher disease patients stabilized during at least 3 years of previous enzyme therapy were included in this 2-year, prospective, open-label non-inferiority study. The primary endpoint was percent change from baseline in liver volume. Secondary endpoints included changes in spleen volume, hemoglobin concentration and platelet count.
resultsForty-two patients were enrolled (mean±SD age, 45.1±12.7 years; previous enzyme therapy duration 9.5±4.0 years). Median (range) exposure to miglustat 100 mg t.i.d. was 658 (3-765) days. Twenty-one patients discontinued treatment prematurely; 13 due to adverse events, principally gastrointestinal. The upper 95% confidence limit of mean percent change in liver volume from baseline to end of treatment was below the non-inferiority margin of 10% (-1.1%; 95%CI -6.0, 3.9%). Mean (95%CI) changes in spleen volume, hemoglobin concentration and platelet count were 102 (24,180) mL, -0.95 (-1.38, -0.53) g/dL and -44.1 (-57.6, -30.7) ×109/L, respectively.
conclusionsThe primary efficacy endpoint was met; overall there was no change in liver volume during 24 months of miglustat therapy. Several patients showed a gradual deterioration in some disease manifestations, suggesting that miglustat could maintain clinical stability, but not in all patients. Miglustat demonstrated a predictable profile of safety and tolerability that was consistent with that reported in previous clinical trials and experience in clinical practice.
trial registrationClinicaltrials.gov identifier NCT00319046.
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.