Mycophenolate Mofetil for First-Line Treatment of Immune Thrombocytopenia.

Adolescent Adult Aged Drug Therapy, Combination Fatigue / chemically induced Female Glucocorticoids / adverse effects Hemorrhage / etiology Humans Immunosuppressive Agents / adverse effects Male Middle Aged Mycophenolic Acid / adverse effects Platelet Count Purpura, Thrombocytopenic, Idiopathic / complications Quality of Life Young Adult

Journal

The New England journal of medicine
ISSN: 1533-4406
Titre abrégé: N Engl J Med
Pays: United States
ID NLM: 0255562

Informations de publication

Date de publication:
02 09 2021
Historique:
entrez: 1 9 2021
pubmed: 2 9 2021
medline: 18 9 2021
Statut: ppublish

Résumé

Immune thrombocytopenia is a rare autoimmune disorder with associated bleeding risk and fatigue. Recommended first-line treatment for immune thrombocytopenia is high-dose glucocorticoids, but side effects, variable responses, and high relapse rates are serious drawbacks. In this multicenter, open-label, randomized, controlled trial conducted in the United Kingdom, we assigned adult patients with immune thrombocytopenia, in a 1:1 ratio, to first-line treatment with a glucocorticoid only (standard care) or combined glucocorticoid and mycophenolate mofetil. The primary efficacy outcome was treatment failure, defined as a platelet count of less than 30×10 A total of 120 patients with immune thrombocytopenia underwent randomization (52.4% male; mean age, 54 years [range 17 to 87]; mean platelet level, 7×10 The addition of mycophenolate mofetil to a glucocorticoid for first-line treatment of immune thrombocytopenia resulted in greater response and a lower risk of refractory or relapsed immune thrombocytopenia, but with somewhat decreased quality of life. (Funded by the U.K. National Institute for Health Research; FLIGHT ClinicalTrials.gov number, NCT03156452; EudraCT number, 2017-001171-23.).

Sections du résumé

BACKGROUND
Immune thrombocytopenia is a rare autoimmune disorder with associated bleeding risk and fatigue. Recommended first-line treatment for immune thrombocytopenia is high-dose glucocorticoids, but side effects, variable responses, and high relapse rates are serious drawbacks.
METHODS
In this multicenter, open-label, randomized, controlled trial conducted in the United Kingdom, we assigned adult patients with immune thrombocytopenia, in a 1:1 ratio, to first-line treatment with a glucocorticoid only (standard care) or combined glucocorticoid and mycophenolate mofetil. The primary efficacy outcome was treatment failure, defined as a platelet count of less than 30×10
RESULTS
A total of 120 patients with immune thrombocytopenia underwent randomization (52.4% male; mean age, 54 years [range 17 to 87]; mean platelet level, 7×10
CONCLUSIONS
The addition of mycophenolate mofetil to a glucocorticoid for first-line treatment of immune thrombocytopenia resulted in greater response and a lower risk of refractory or relapsed immune thrombocytopenia, but with somewhat decreased quality of life. (Funded by the U.K. National Institute for Health Research; FLIGHT ClinicalTrials.gov number, NCT03156452; EudraCT number, 2017-001171-23.).

Identifiants

DOI: 10.1056/NEJMoa2100596 PMID: 34469646
pubmed: 34469646
doi: 10.1056/NEJMoa2100596
doi:

Substances chimiques

Glucocorticoids 0
Immunosuppressive Agents 0
Mycophenolic Acid HU9DX48N0T

Banques de données

ClinicalTrials.gov
['NCT03156452']
EudraCT
['2017-001171-23']

Types de publication

Journal Article Multicenter Study Randomized Controlled Trial Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

885-895

Subventions

Organisme : National Institute on Handicapped Research
ID : PB-PG-0815-20016

Commentaires et corrections

Type : CommentIn
Type : CommentIn
Type : CommentIn
Type : CommentIn

Informations de copyright

Copyright © 2021 Massachusetts Medical Society.

Auteurs

Charlotte A Bradbury (CA)

From the Faculty of Translational Health Sciences (C.A.B.), University of Bristol (J.I., K.B., R.K.), and the Bristol Haematology and Oncology Centre (C.A.B.) and the Research Design Service (R.G.), University Hospitals Bristol and Weston NHS Foundation Trust, Bristol, the Centre for Trials Research, Cardiff University (J.P., I.T.), and the Department of Haematology, Cardiff and Vale University Health Board (R.R.), Cardiff, Leeds Teaching Hospitals NHS Trust, Leeds (Q.H.), Glasgow Royal Infirmary, Glasgow (C.B.), the Department of Immunology and Inflammation, Imperial College London, London (N.C.), East Kent Hospitals University NHS Foundation Trust, Canterbury (G.E.), and Newcastle upon Tyne Hospitals NHS Trust, Newcastle upon Tyne (K.T.) - all in the United Kingdom.
ORCID: 0000-0001-5248-8165

Julie Pell (J)

From the Faculty of Translational Health Sciences (C.A.B.), University of Bristol (J.I., K.B., R.K.), and the Bristol Haematology and Oncology Centre (C.A.B.) and the Research Design Service (R.G.), University Hospitals Bristol and Weston NHS Foundation Trust, Bristol, the Centre for Trials Research, Cardiff University (J.P., I.T.), and the Department of Haematology, Cardiff and Vale University Health Board (R.R.), Cardiff, Leeds Teaching Hospitals NHS Trust, Leeds (Q.H.), Glasgow Royal Infirmary, Glasgow (C.B.), the Department of Immunology and Inflammation, Imperial College London, London (N.C.), East Kent Hospitals University NHS Foundation Trust, Canterbury (G.E.), and Newcastle upon Tyne Hospitals NHS Trust, Newcastle upon Tyne (K.T.) - all in the United Kingdom.

Quentin Hill (Q)

From the Faculty of Translational Health Sciences (C.A.B.), University of Bristol (J.I., K.B., R.K.), and the Bristol Haematology and Oncology Centre (C.A.B.) and the Research Design Service (R.G.), University Hospitals Bristol and Weston NHS Foundation Trust, Bristol, the Centre for Trials Research, Cardiff University (J.P., I.T.), and the Department of Haematology, Cardiff and Vale University Health Board (R.R.), Cardiff, Leeds Teaching Hospitals NHS Trust, Leeds (Q.H.), Glasgow Royal Infirmary, Glasgow (C.B.), the Department of Immunology and Inflammation, Imperial College London, London (N.C.), East Kent Hospitals University NHS Foundation Trust, Canterbury (G.E.), and Newcastle upon Tyne Hospitals NHS Trust, Newcastle upon Tyne (K.T.) - all in the United Kingdom.

Catherine Bagot (C)

From the Faculty of Translational Health Sciences (C.A.B.), University of Bristol (J.I., K.B., R.K.), and the Bristol Haematology and Oncology Centre (C.A.B.) and the Research Design Service (R.G.), University Hospitals Bristol and Weston NHS Foundation Trust, Bristol, the Centre for Trials Research, Cardiff University (J.P., I.T.), and the Department of Haematology, Cardiff and Vale University Health Board (R.R.), Cardiff, Leeds Teaching Hospitals NHS Trust, Leeds (Q.H.), Glasgow Royal Infirmary, Glasgow (C.B.), the Department of Immunology and Inflammation, Imperial College London, London (N.C.), East Kent Hospitals University NHS Foundation Trust, Canterbury (G.E.), and Newcastle upon Tyne Hospitals NHS Trust, Newcastle upon Tyne (K.T.) - all in the United Kingdom.

Nichola Cooper (N)

From the Faculty of Translational Health Sciences (C.A.B.), University of Bristol (J.I., K.B., R.K.), and the Bristol Haematology and Oncology Centre (C.A.B.) and the Research Design Service (R.G.), University Hospitals Bristol and Weston NHS Foundation Trust, Bristol, the Centre for Trials Research, Cardiff University (J.P., I.T.), and the Department of Haematology, Cardiff and Vale University Health Board (R.R.), Cardiff, Leeds Teaching Hospitals NHS Trust, Leeds (Q.H.), Glasgow Royal Infirmary, Glasgow (C.B.), the Department of Immunology and Inflammation, Imperial College London, London (N.C.), East Kent Hospitals University NHS Foundation Trust, Canterbury (G.E.), and Newcastle upon Tyne Hospitals NHS Trust, Newcastle upon Tyne (K.T.) - all in the United Kingdom.

Jenny Ingram (J)

From the Faculty of Translational Health Sciences (C.A.B.), University of Bristol (J.I., K.B., R.K.), and the Bristol Haematology and Oncology Centre (C.A.B.) and the Research Design Service (R.G.), University Hospitals Bristol and Weston NHS Foundation Trust, Bristol, the Centre for Trials Research, Cardiff University (J.P., I.T.), and the Department of Haematology, Cardiff and Vale University Health Board (R.R.), Cardiff, Leeds Teaching Hospitals NHS Trust, Leeds (Q.H.), Glasgow Royal Infirmary, Glasgow (C.B.), the Department of Immunology and Inflammation, Imperial College London, London (N.C.), East Kent Hospitals University NHS Foundation Trust, Canterbury (G.E.), and Newcastle upon Tyne Hospitals NHS Trust, Newcastle upon Tyne (K.T.) - all in the United Kingdom.

Katie Breheny (K)

From the Faculty of Translational Health Sciences (C.A.B.), University of Bristol (J.I., K.B., R.K.), and the Bristol Haematology and Oncology Centre (C.A.B.) and the Research Design Service (R.G.), University Hospitals Bristol and Weston NHS Foundation Trust, Bristol, the Centre for Trials Research, Cardiff University (J.P., I.T.), and the Department of Haematology, Cardiff and Vale University Health Board (R.R.), Cardiff, Leeds Teaching Hospitals NHS Trust, Leeds (Q.H.), Glasgow Royal Infirmary, Glasgow (C.B.), the Department of Immunology and Inflammation, Imperial College London, London (N.C.), East Kent Hospitals University NHS Foundation Trust, Canterbury (G.E.), and Newcastle upon Tyne Hospitals NHS Trust, Newcastle upon Tyne (K.T.) - all in the United Kingdom.

Rebecca Kandiyali (R)

From the Faculty of Translational Health Sciences (C.A.B.), University of Bristol (J.I., K.B., R.K.), and the Bristol Haematology and Oncology Centre (C.A.B.) and the Research Design Service (R.G.), University Hospitals Bristol and Weston NHS Foundation Trust, Bristol, the Centre for Trials Research, Cardiff University (J.P., I.T.), and the Department of Haematology, Cardiff and Vale University Health Board (R.R.), Cardiff, Leeds Teaching Hospitals NHS Trust, Leeds (Q.H.), Glasgow Royal Infirmary, Glasgow (C.B.), the Department of Immunology and Inflammation, Imperial College London, London (N.C.), East Kent Hospitals University NHS Foundation Trust, Canterbury (G.E.), and Newcastle upon Tyne Hospitals NHS Trust, Newcastle upon Tyne (K.T.) - all in the United Kingdom.

Rachel Rayment (R)

From the Faculty of Translational Health Sciences (C.A.B.), University of Bristol (J.I., K.B., R.K.), and the Bristol Haematology and Oncology Centre (C.A.B.) and the Research Design Service (R.G.), University Hospitals Bristol and Weston NHS Foundation Trust, Bristol, the Centre for Trials Research, Cardiff University (J.P., I.T.), and the Department of Haematology, Cardiff and Vale University Health Board (R.R.), Cardiff, Leeds Teaching Hospitals NHS Trust, Leeds (Q.H.), Glasgow Royal Infirmary, Glasgow (C.B.), the Department of Immunology and Inflammation, Imperial College London, London (N.C.), East Kent Hospitals University NHS Foundation Trust, Canterbury (G.E.), and Newcastle upon Tyne Hospitals NHS Trust, Newcastle upon Tyne (K.T.) - all in the United Kingdom.

Gillian Evans (G)

From the Faculty of Translational Health Sciences (C.A.B.), University of Bristol (J.I., K.B., R.K.), and the Bristol Haematology and Oncology Centre (C.A.B.) and the Research Design Service (R.G.), University Hospitals Bristol and Weston NHS Foundation Trust, Bristol, the Centre for Trials Research, Cardiff University (J.P., I.T.), and the Department of Haematology, Cardiff and Vale University Health Board (R.R.), Cardiff, Leeds Teaching Hospitals NHS Trust, Leeds (Q.H.), Glasgow Royal Infirmary, Glasgow (C.B.), the Department of Immunology and Inflammation, Imperial College London, London (N.C.), East Kent Hospitals University NHS Foundation Trust, Canterbury (G.E.), and Newcastle upon Tyne Hospitals NHS Trust, Newcastle upon Tyne (K.T.) - all in the United Kingdom.

Kate Talks (K)

From the Faculty of Translational Health Sciences (C.A.B.), University of Bristol (J.I., K.B., R.K.), and the Bristol Haematology and Oncology Centre (C.A.B.) and the Research Design Service (R.G.), University Hospitals Bristol and Weston NHS Foundation Trust, Bristol, the Centre for Trials Research, Cardiff University (J.P., I.T.), and the Department of Haematology, Cardiff and Vale University Health Board (R.R.), Cardiff, Leeds Teaching Hospitals NHS Trust, Leeds (Q.H.), Glasgow Royal Infirmary, Glasgow (C.B.), the Department of Immunology and Inflammation, Imperial College London, London (N.C.), East Kent Hospitals University NHS Foundation Trust, Canterbury (G.E.), and Newcastle upon Tyne Hospitals NHS Trust, Newcastle upon Tyne (K.T.) - all in the United Kingdom.

Ian Thomas (I)

From the Faculty of Translational Health Sciences (C.A.B.), University of Bristol (J.I., K.B., R.K.), and the Bristol Haematology and Oncology Centre (C.A.B.) and the Research Design Service (R.G.), University Hospitals Bristol and Weston NHS Foundation Trust, Bristol, the Centre for Trials Research, Cardiff University (J.P., I.T.), and the Department of Haematology, Cardiff and Vale University Health Board (R.R.), Cardiff, Leeds Teaching Hospitals NHS Trust, Leeds (Q.H.), Glasgow Royal Infirmary, Glasgow (C.B.), the Department of Immunology and Inflammation, Imperial College London, London (N.C.), East Kent Hospitals University NHS Foundation Trust, Canterbury (G.E.), and Newcastle upon Tyne Hospitals NHS Trust, Newcastle upon Tyne (K.T.) - all in the United Kingdom.

Rosemary Greenwood (R)

From the Faculty of Translational Health Sciences (C.A.B.), University of Bristol (J.I., K.B., R.K.), and the Bristol Haematology and Oncology Centre (C.A.B.) and the Research Design Service (R.G.), University Hospitals Bristol and Weston NHS Foundation Trust, Bristol, the Centre for Trials Research, Cardiff University (J.P., I.T.), and the Department of Haematology, Cardiff and Vale University Health Board (R.R.), Cardiff, Leeds Teaching Hospitals NHS Trust, Leeds (Q.H.), Glasgow Royal Infirmary, Glasgow (C.B.), the Department of Immunology and Inflammation, Imperial College London, London (N.C.), East Kent Hospitals University NHS Foundation Trust, Canterbury (G.E.), and Newcastle upon Tyne Hospitals NHS Trust, Newcastle upon Tyne (K.T.) - all in the United Kingdom.

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Classifications MeSH

questionsmedicales.fr › Personnes › Tranches d'âge › Adolescent › Mycophenolate Mofetil for First-Line Treatment...
questionsmedicales.fr › Personnes › Tranches d'âge › Adulte › Mycophenolate Mofetil for First-Line Treatment...
questionsmedicales.fr › Personnes › Tranches d'âge › Adulte › Sujet âgé › Mycophenolate Mofetil for First-Line Treatment...
questionsmedicales.fr › Thérapeutique › Traitement médicamenteux › Association de médicaments › Mycophenolate Mofetil for First-Line Treatment...
questionsmedicales.fr › États, signes et symptômes pathologiques › Signes et symptômes › Fatigue › Mycophenolate Mofetil for First-Line Treatment...
questionsmedicales.fr › Actions chimiques et utilisations › Actions pharmacologiques › Effets physiologiques des médicaments › Hormones, substituts d'hormones et antagonistes d'hormones › Hormones › Glucocorticoïdes › Mycophenolate Mofetil for First-Line Treatment...
questionsmedicales.fr › États, signes et symptômes pathologiques › Processus pathologiques › Hémorragie › Mycophenolate Mofetil for First-Line Treatment...
questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Primates › Haplorhini › Catarrhini › Hominidae › Humains › Mycophenolate Mofetil for First-Line Treatment...
questionsmedicales.fr › Actions chimiques et utilisations › Actions pharmacologiques › Effets physiologiques des médicaments › Facteurs immunologiques › Immunosuppresseurs › Mycophenolate Mofetil for First-Line Treatment...
questionsmedicales.fr › Personnes › Tranches d'âge › Adulte › Adulte d'âge moyen › Mycophenolate Mofetil for First-Line Treatment...
questionsmedicales.fr › Lipides › Acides gras › Acide mycophénolique › Mycophenolate Mofetil for First-Line Treatment...
questionsmedicales.fr › Phénomènes physiologiques respiratoires et circulatoires › Phénomènes physiogiques du sang › Hémogramme › Numération des plaquettes › Mycophenolate Mofetil for First-Line Treatment...
questionsmedicales.fr › États, signes et symptômes pathologiques › Signes et symptômes › Manifestations cutanées › Purpura › Purpura thrombopénique › Purpura thrombopénique idiopathique › Mycophenolate Mofetil for First-Line Treatment...
questionsmedicales.fr › Environnement et santé publique › Santé publique › Mesures épidémiologiques › Démographie › État de santé › Qualité de vie › Mycophenolate Mofetil for First-Line Treatment...
questionsmedicales.fr › Personnes › Tranches d'âge › Adulte › Jeune adulte › Mycophenolate Mofetil for First-Line Treatment...