Side Effects of miglustat: A Synthesis of Findings from 16 Studies

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Original Abstract of the Article

Main Research Findings

Miglustat is a medication that has been studied for its potential to treat lysosomal storage disorders, including GM2 gangliosidosis, Gaucher disease, and Niemann-Pick disease type C. These studies have shown that miglustat is generally safe and well-tolerated. However, some side effects have been reported. The main findings of miglustat research are as follows.

  • 2 : Miglustat showed similar pharmacokinetic parameters in all patients with no specific difference between infantile and juvenile forms in infantile and juvenile GM2 gangliosidosis patients for 6- and 24-months, respectively. The major drug-related adverse events (DRAEs) in infantile GM2g patients were abdominal discomfort and flatulence. In the juvenile group, however, the major DRAEs observed were diarrhea and weight loss.
  • 3 : Miglustat has been shown to be effective in patients with adolescent/adult-onset NP-C.
  • 4 : Miglustat has been shown to be an effective oral treatment for symptomatic patients with mild to moderate clinical manifestations for whom ERT is not an option.
  • : Miglustat is an effective treatment for Niemann-Pick disease type C, but it has been associated with side effects.
  • 14 : Miglustat has been investigated as a potential treatment for infantile gangliosidosis, but gastrointestinal side effects have limited its use.
  • 13 : Miglustat has been shown to inhibit the growth and ganglioside content of experimental mouse brain tumors. However, it has also been associated with side effects, including weight loss and intestinal distension.

Reason for Side Effects

Miglustat inhibits the production of glucosylceramide by inhibiting an enzyme called glucosylceramide synthase. Glucosylceramide is a component of cell membranes and is the substance that causes lysosomal storage disorders. The side effects of miglustat are thought to be due to poor digestion and absorption of carbohydrates in the gut, as well as effects on the nervous system.

Common Side Effects

Diarrhea

Diarrhea is the most common side effect of miglustat. It is reported in 2 、 12 、 7 、 1 、 6 、 4 、 11 、 5 、 14 、 13 、 10 . This side effect is thought to be due to miglustat inhibiting digestive enzymes in the gut, leading to poor digestion and absorption of carbohydrates. Reducing the intake of carbohydrates, particularly sucrose and maltose, can improve diarrhea.

Weight Loss

Miglustat can cause weight loss. It is reported in 2 、 7 、 5 、 13 、 10 . Weight loss is thought to be caused by diarrhea and loss of appetite.

Abdominal Discomfort

Miglustat can cause abdominal discomfort and flatulence. It is reported in 2 、 12 、 5 . These symptoms are thought to be caused by excessive gas and fluid buildup in the gut.

Neuropathy

Miglustat may cause neuropathy. It is reported in 2 、 10 . Neuropathy is thought to be caused by miglustat affecting nerve cells. However, it cannot be definitively stated that neuropathy is caused by miglustat. This is because neuropathy may also be caused by the lysosomal storage disorder itself.

Liver Function Abnormality

Miglustat may cause liver function abnormalities. It is reported in 12 . Liver function abnormalities are thought to be caused by miglustat affecting the liver. However, it cannot be definitively stated that liver function abnormalities are caused by miglustat. This is because liver function abnormalities may also be caused by the lysosomal storage disorder itself.

Leukopenia

Miglustat may cause leukopenia. It is reported in 12 . Leukopenia is thought to be caused by miglustat affecting immune cells. However, it cannot be definitively stated that leukopenia is caused by miglustat. This is because leukopenia may also be caused by the lysosomal storage disorder itself.

Cataracts

Miglustat may cause cataracts. It is reported in 12 . Cataracts are thought to be caused by miglustat affecting the lens of the eye. However, it cannot be definitively stated that cataracts are caused by miglustat. This is because cataracts may also be caused by the lysosomal storage disorder itself.

Countermeasures for Side Effects

Countermeasures for Diarrhea

Diarrhea can be improved with dietary or drug therapy. It is reported in 12 、 5 . Dietary therapy includes reducing the intake of carbohydrates such as sucrose and maltose. Drug therapy includes antidiarrheal agents such as loperamide.

Countermeasures for Weight Loss

Weight loss can be improved with dietary and exercise therapy. It is reported in 5 . Dietary therapy includes a high-calorie, high-protein diet. Exercise therapy involves exercising within a reasonable range.

Countermeasures for Abdominal Discomfort

Abdominal discomfort and flatulence can be improved with dietary and drug therapy. It is reported in 5 . Dietary therapy includes eating easily digestible foods, avoiding fatty foods, and chewing thoroughly. Drug therapy includes antacids and digestive enzyme supplements.

Countermeasures for Neuropathy

Neuropathy can be improved by stopping miglustat administration. It is reported in 2 . However, it cannot be definitively stated that neuropathy is caused by miglustat. Therefore, if neuropathy is observed, it is necessary to consult a doctor and receive appropriate treatment.

Countermeasures for Liver Function Abnormality

Liver function abnormalities can be improved by stopping miglustat administration. It is reported in 12 . However, it cannot be definitively stated that liver function abnormalities are caused by miglustat. Therefore, if liver function abnormalities are observed, it is necessary to consult a doctor and receive appropriate treatment.

Countermeasures for Leukopenia

Leukopenia can be improved by stopping miglustat administration. It is reported in 12 . However, it cannot be definitively stated that leukopenia is caused by miglustat. Therefore, if leukopenia is observed, it is necessary to consult a doctor and receive appropriate treatment.

Countermeasures for Cataracts

Cataracts can be treated with surgery. It is reported in 12 . However, it cannot be definitively stated that cataracts are caused by miglustat. Therefore, if cataracts are observed, it is necessary to consult a doctor and receive appropriate treatment.

Comparison Between Studies

Common Points of Studies

Miglustat has been investigated as a treatment for lysosomal storage disorders. These studies have shown that miglustat is generally safe and well-tolerated. However, some side effects have been reported. For example, diarrhea, weight loss, abdominal discomfort, neuropathy, liver function abnormalities, leukopenia, and cataracts have been reported.

Differences Between Studies

The side effects of miglustat vary depending on the study. For example, in 2 , the major side effects in infants were abdominal discomfort and flatulence, while in 3 , it has been shown to be effective in patients with adolescent/adult-onset NP-C. Also, in 4 , it has been shown to be an effective oral treatment for symptomatic patients with mild to moderate clinical manifestations for whom ERT is not an option.

Cautions for Applying Research to Real Life

Miglustat is used as a treatment for lysosomal storage disorders. However, side effects such as diarrhea, weight loss, abdominal discomfort, neuropathy, liver function abnormalities, leukopenia, and cataracts have been reported. Therefore, when taking miglustat, it is necessary to consult a doctor and receive appropriate treatment.

Limitations of Current Research

Although miglustat has been investigated as a treatment for lysosomal storage disorders, there are still many things that are not known. For example, the long-term safety and efficacy of miglustat are not fully understood. Also, methods for reducing the side effects of miglustat have not been sufficiently studied.

Future Research Directions

It is necessary to research the long-term safety and efficacy of miglustat, as well as methods for reducing its side effects. It is also necessary to research other treatments for lysosomal storage disorders that miglustat is effective against.

Conclusion

Miglustat is a promising treatment for lysosomal storage disorders. However, some side effects have been reported. Therefore, when taking miglustat, it is necessary to consult a doctor and receive appropriate treatment.

Literature analysis of 16 papers
Positive Content
12
Neutral Content
1
Negative Content
3
Article Type
2
0
0
5
16
1.

[The dose-dependent effects of oral premedication with midazolam].

Author: BiroP, WeidmannG, PietzschS, AlonE, BruggerP

Midazolam effective amnesia
Midazolam effective sedation
Side effects

Oral midazolam before surgery provided dose-dependent sedation and amnesia, but no significant anxiolysis compared to placebo. 7.5mg midazolam offered the best balance of benefits and side effects. The study highlights the importance of pre-operative communication and patient care by the anesthesiologist.

Clinical TrialA clinical trial is a planned study conducted on humans to evaluate the efficacy and safety of pharmaceuticals, medical devices, and treatment methods. It aims to explore the possibilities of new treatments and improve the health of patients.
English AbstractA summary of a research paper/publication written in English for a wider audience.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.

Language : German

2.

Pharmacokinetics, safety and tolerability of miglustat in the treatment of pediatric patients with GM2 gangliosidosis.

Author: MaegawaGustavo H B, van GiersbergenPaul L M, YangSandra, BanwellBrenda, MorganChristopher P, DingemanseJasper, TifftCynthia J, ClarkeJoe T R

GM2g treatment
Diarrhea
Neuropathy
Weight loss

Miglustat, a substrate reduction therapy for GM2 gangliosidosis, is safe and well-tolerated in both infantile and juvenile patients. While mild to moderate diarrhea is a common side effect, it is manageable with dietary modification.

Clinical TrialA clinical trial is a planned study conducted on humans to evaluate the efficacy and safety of pharmaceuticals, medical devices, and treatment methods. It aims to explore the possibilities of new treatments and improve the health of patients.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Research Support, N.I.H., ExtramuralResearch funding provided by the NIH to external research institutions and researchers.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

3.

Adult Niemann-Pick disease type C in France: clinical phenotypes and long-term miglustat treatment effect.

Author: NadjarYann, Hütter-MoncadaAna Lucia, LatourPhilippe, AyrignacXavier, KaphanElsa, TranchantChristine, CintasPascal, DegardinAdrian, GoizetCyril, LaurencinChloe, MartzolffLionel, TiliketeCaroline, AnheimMathieu, AudoinBertrand, DeramecourtVincent, De GaillarboisThierry Dubard, RozeEmmanuel, LamariFoudil, VanierMarie T, HéronBénédicte

NP-C treatment

This study describes the clinical presentation, evolution, and effect of miglustat treatment in a large cohort of patients with adolescent/adult-onset Niemann-Pick disease type C (NP-C).

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.

Language : English

4.

Guidance on the use of miglustat for treating patients with type 1 Gaucher disease.

Author: WeinrebNeal J, BarrangerJohn A, CharrowJoel, GrabowskiGregory A, MankinHenry J, MistryPramod

Improved hematological
Improved pulmonary
Improved quality of life
Improved skeletal
Improved visceral
Significant side effects

Type 1 Gaucher disease (GD) is a progressive lysosomal storage disorder. Intravenous enzyme replacement (ERT) with imiglucerase is the standard treatment for symptomatic patients. Miglustat, an oral treatment for patients with mild to moderate clinical manifestations, is an alternative treatment option for patients who are not candidates for ERT. Miglustat is associated with significant side effects and responses are slower and less robust than those observed with ERT.

Comparative StudyResearch that compares multiple groups/interventions to determine differences/similarities in outcomes/characteristics.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.

Language : English

5.

Gastrointestinal disturbances and their management in miglustat-treated patients.

Author: BelmatougNadia, BurlinaAlberto, GiraldoPilar, HendrikszChris J, KuterDavid J, MengelEugen, PastoresGregory M

Gastrointestinal disturbances
Weight loss

Miglustat is a treatment for Gaucher disease and Niemann-Pick disease type C. While effective, gastrointestinal disturbances are a common side effect. These disturbances, mainly diarrhoea, are often mild and manageable through dietary modifications, anti-propulsive medications, and dose escalation.

Evaluation StudyResearch that systematically evaluates the effectiveness of a particular program or policy. It measures its outcomes and provides important information for future decision-making.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.

Language : English

6.

N-butyldeoxygalactonojirimycin: a more selective inhibitor of glycosphingolipid biosynthesis than N-butyldeoxynojirimycin, in vitro and in vivo.

Author: AnderssonU, ButtersT D, DwekR A, PlattF M

GSL biosynthesis inhibitor
Gaucher's disease treatment
Side effects

This study compared two inhibitors of glycosphingolipid biosynthesis, N-butyldeoxynojirimycin (NB-DNJ) and N-butyldeoxygalactonojirimycin (NB-DGJ). NB-DGJ was more selective than NB-DNJ and did not cause weight loss or lymphoid organ shrinkage.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

7.

Low-dose N-butyldeoxynojirimycin (OGT 918) for type I Gaucher disease.

Author: HeitnerRene, ElsteinDeborah, AertsJohannes, WeelySonja van, ZimranAri

Reduced chitotriosidase levels
Reduced liver volume
Reduced spleen volume
Diarrhea
Weight loss

This study evaluated the efficacy and safety of low-dose substrate balance therapy with OGT 918 for treating Gaucher disease. OGT 918 was effective in reducing liver and spleen volume and chitotriosidase levels, but did not improve hematological parameters. Higher doses of OGT 918 may be needed for optimal response.

Clinical TrialA clinical trial is a planned study conducted on humans to evaluate the efficacy and safety of pharmaceuticals, medical devices, and treatment methods. It aims to explore the possibilities of new treatments and improve the health of patients.
Clinical Trial, Phase IThe first stage of clinical trials in humans. Evaluates the safety, tolerability, and pharmacokinetics of a new drug/treatment.
Clinical Trial, Phase IIA Phase II trial is a trial that further evaluates the efficacy and safety of a new drug or treatment, whose safety has been confirmed to some extent in a Phase I trial, in a small number of patients.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Multicenter StudyResearch conducted in collaboration with multiple research institutions. It aims to collect a wide range of data and obtain more generalizable results.

Language : English

8.

AAV9 intracerebroventricular gene therapy improves lifespan, locomotor function and pathology in a mouse model of Niemann-Pick type C1 disease.

Author: HughesMichael P, SmithDave A, MorrisLauren, FletcherClaire, ColacoAlexandria, HuebeckerMylene, TordoJulie, PalomarNuria, MassaroGiulia, HenckaertsEls, WaddingtonSimon N, PlattFrances M, RahimAhad A

Lifespan extension
Neurodegeneration prevention
Quality of life improvement
Miglustat side effects

AAV-mediated gene therapy may be a potential therapeutic option for Niemann-Pick type C disease (NP-C). A study in a mouse model of NP-C showed that a single administration of AAV-mediated gene therapy to the brain significantly extended lifespan, improved quality of life, prevented or ameliorated neurodegeneration, and reduced biochemical pathology.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

9.

Substrate reduction therapy in mouse models of the glycosphingolipidoses.

Author: PlattFrances M, JeyakumarMylvaganam, AnderssonUlrika, HeareTanya, DwekRaymond A, ButtersTerry D

Fabry disease treatment
Sandhoff treatment
Tay-Sachs treatment

Substrate reduction therapy uses small molecules to slow the rate of glycolipid biosynthesis. N-butyldeoxynojirimycin (NB-DNJ) shows efficacy in mouse models of Tay-Sachs, Sandhoff and Fabry diseases. A second drug, N-butyldeoxyglactonojirimycin, shows promise for treating storage diseases with neurological involvement.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.

Language : English

10.

Gaucher disease and the clinical experience with substrate reduction therapy.

Author: ZimranAri, ElsteinDeborah

Disease symptom reduction
Gaucher disease treatment
Diarrhoea
Peripheral neuropathy
Tremor
Weight loss

This study evaluated the safety and efficacy of N-butyldeoxynojirimycin (OGT 918) in patients with Gaucher disease. OGT 918 was safe and effective in adult type I patients, and may be a good option for patients unable to receive enzyme replacement therapy.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.

Language : English

11.

Oral maintenance clinical trial with miglustat for type I Gaucher disease: switch from or combination with intravenous enzyme replacement.

Author: ElsteinDeborah, DweckAltoon, AttiasDrorit, Hadas-HalpernIrith, ZevinShoshana, AltarescuGheona, AertsJohannes F M G, van WeelySonja, ZimranAri

Gaucher disease treatment
Organomegaly reduction
Peripheral neuropathy

This trial evaluated miglustat in patients with Gaucher disease type 1 who were clinically stable on enzyme replacement therapy (ERT) with imiglucerase. Miglustat was well tolerated alone or in combination with imiglucerase, and findings suggest it could be an effective maintenance therapy in stabilized patients.

Clinical Trial, Phase IIA Phase II trial is a trial that further evaluates the efficacy and safety of a new drug or treatment, whose safety has been confirmed to some extent in a Phase I trial, in a small number of patients.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

12.

The tolerability and pharmacokinetics of N-butyl-deoxynojirimycin in patients with advanced HIV disease (ACTG 100). The AIDS Clinical Trials Group (ACTG) of the National Institute of Allergy and Infectious Diseases.

Author: TierneyM, PottageJ, KesslerH, FischlM, RichmanD, MeriganT, PowderlyW, SmithS, KarimA, ShermanJ

HIV-1 replication inhibition
Cataracts
Gastrointestinal side effects
Leukopenia
Liver function test elevations
Neutropenia

A phase I trial of N-butyl-deoxynojirimycin, an HIV-1 inhibitor, was stopped after the finding of cataracts in rats. The most common side effects were gastrointestinal, and some patients experienced liver function test abnormalities and leukopenia.

Clinical TrialA clinical trial is a planned study conducted on humans to evaluate the efficacy and safety of pharmaceuticals, medical devices, and treatment methods. It aims to explore the possibilities of new treatments and improve the health of patients.
Clinical Trial, Phase IThe first stage of clinical trials in humans. Evaluates the safety, tolerability, and pharmacokinetics of a new drug/treatment.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Multicenter StudyResearch conducted in collaboration with multiple research institutions. It aims to collect a wide range of data and obtain more generalizable results.

Language : English

14.

Infantile gangliosidoses: Mapping a timeline of clinical changes.

Author: Jarnes UtzJeanine R, KimSarah, KingKelly, ZieglerRichard, SchemaLynn, RedtreeEvelyn S, WhitleyChester B

Gastrointestinal side effects

This article discusses the lack of natural history information and effective treatments for infantile gangliosidoses. Infantile gangliosidoses include GM1 gangliosidosis and GM2 gangliosidosis (Tay-Sachs disease, Sandhoff disease).

Clinical TrialA clinical trial is a planned study conducted on humans to evaluate the efficacy and safety of pharmaceuticals, medical devices, and treatment methods. It aims to explore the possibilities of new treatments and improve the health of patients.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Research Support, N.I.H., ExtramuralResearch funding provided by the NIH to external research institutions and researchers.

Language : English

16.

Identification of Brain-Specific Treatment Effects in NPC1 Disease by Focusing on Cellular and Molecular Changes of Sphingosine-1-Phosphate Metabolism.

Author: GläserAnne, HammerlFranziska, GrälerMarkus H, ColdeweySina M, VölknerChristin, FrechMoritz J, YangFan, LuoJiankai, TönniesEric, von Bohlen Und HalbachOliver, BrandtNicola, HeimesDiana, NeßlauerAnna-Maria, KorenkeGeorg Christoph, Owczarek-LipskaMarta, NeidhardtJohn, RolfsArndt, WreeAndreas, WittMartin, BräuerAnja Ursula

Treatment side effects

This study investigated the role of the sphingosine-1-phosphate (S1P)/S1P receptor (S1PR) axis in Niemann-Pick type C1 (NPC1) pathology. The results suggest that the S1P/S1PR axis is involved in NPC1 pathology, but treatment with 2-hydroxypropyl-beta-cyclodextrin and miglustat showed only weak effects in the mouse brain.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.

Language : English

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