ArticleBiologics : targets & therapy2009
Review of the safety and efficacy of imiglucerase treatment of Gaucher disease.
Article in Biologics : targets & therapy, 2009. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 papers, 1 of them a synthesis that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
19 citing papers in PubMed, 1 synthesis or guideline pooled it, 46 citations in OpenAlex.
- Gaucher disease: a systematic review and meta-analysis of bone complications and their response to treatment.Journal of inherited metabolic disease · 2010Pooled it
- A Feasibility Open-Labeled Clinical Trial Using a Second-Generation Artificial-Intelligence-Based Therapeutic Regimen in Patients with Gaucher Disease Treated with Enzyme Replacement Therapy.Journal of clinical medicine · 2024Article
- Cardiac Manifestations in a Group of Romanian Patients with Gaucher Disease Type 1 (a Monocentric Study).Diagnostics (Basel, Switzerland) · 2021Article
- Allogeneic hematopoietic stem cell transplantation for treating severe lung involvement in Gaucher disease.Molecular genetics and metabolism reports · 2020Article
- Gaucher disease in Montenegro - genotype/phenotype correlations: Five cases report.World journal of clinical cases · 2019Article
- Case report of unexpected gastrointestinal involvement in type 1 Gaucher disease: comparison of eliglustat tartrate treatment and enzyme replacement therapy.BMC medical genetics · 2017Article
- 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
- An overview of Compassionate Use Programs in the European Union member states.Intractable & rare diseases research · 2016Review
- 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
- Hemorrhagic aspects of Gaucher disease.Rambam Maimonides medical journal · 2014Article
- Long-term clinical outcomes in type 1 Gaucher disease following 10 years of imiglucerase treatment.Journal of inherited metabolic disease · 2013Article
- Evaluation of miglustat as maintenance therapy after enzyme therapy in adults with stable type 1 Gaucher disease: a prospective, open-label non-inferiority study.Orphanet journal of rare diseases · 2012Article
- Gaucher disease and the synucleinopathies: refining the relationship.Orphanet journal of rare diseases · 2012Review
- Generation of a Chinese hamster ovary cell line producing recombinant human glucocerebrosidase.Journal of biomedicine & biotechnology · 2012Article
- Gaucher disease and cancer: concept and controversy.International journal of cell biology · 2011Article
- Echocardiographic Assessment of Left Ventricular Function in Type 1 Gaucher's Disease.Advances in hematology · 2010Article
- The Clinical Efficacy of Imiglucerase versus Eliglustat in Patients with Gaucher's Disease Type 1: A Systematic Review.Journal of research in pharmacy practiceReview
- Gaucher disease in a family from Maranhão.Revista brasileira de hematologia e hemoterapiaArticle
- Clinical manifestations and management of Gaucher disease.Clinical cases in mineral and bone metabolism : the official journal of the Italian Society of Osteoporosis, Mineral Metabolism, and Skeletal DiseasesReview
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Most patients who suffer from symptomatic Gaucher disease will benefit from enzyme replacement therapy (ERT) with imiglucerase. The safety profile is excellent, only a small percentage of those exposed developing antibodies; similarly, very few patients require pre-medication for allergic reactions. Within 3 to 5 years of imiglucerase therapy, best documented at doses of 30 to 60 units/kg/infusion, hepatosplenomegaly can be expected to be reduced so that the liver volume will be maintained at 1 to 1.5 times normal (30% to 40% reduction from advent of ERT) and spleen volume to </= 2 to 8 times normal (50% to 60% reduction from advent of ERT). For anemic and thrombocytopenic patients, with 2 to 5 years of imiglucerase, hemoglobin levels are expected to be >/= 11 g/dL for women and children and >/= 12 g/dL for men; and platelet counts in patients with an intact spleen, depending on the baseline value, should approximately be doubled. Bone crises and bone pain but not irreversible skeletal damage will improve in most patients. Nonetheless, some features and some symptomatic patients apparently do not respond equally well and/or perhaps inadequately. The benefit for patients with the neuronopathic forms is primarily in improved visceral and hematological signs and symptoms. There are still several unresolved issues, the high per-unit cost being an important one, which have spurred the development of biosimilar enzymes as well as chaperone therapies currently in clinical trials.
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Registered trials
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.