Intravenous immunoglobulin and intravenous methylprednisolone as optimal induction treatment in chronic inflammatory demyelinating polyradiculoneuropathy: protocol of an international, randomised, double-blind, placebo-controlled trial (OPTIC)
Abstract Background International guidelines recommend either intravenous immunoglobulin (IVIg) or corticosteroids as first-line treatment for chronic inflammatory demyelinating polyradiculoneuropathy (CIDP). IVIg treatment usually leads to rapid improvement and is generally safe, but does not seem...
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BMC
2021-02-01
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| Online Access: | https://doi.org/10.1186/s13063-021-05083-1 |
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| author | S. R. M. Bus L. Zambreanu A. Abbas Y. A. Rajabally R. D. M. Hadden R. J. de Haan C. A. J. M. de Borgie M. P. Lunn I. N. van Schaik F. Eftimov on behalf of the OPTIC study group |
| author_facet | S. R. M. Bus L. Zambreanu A. Abbas Y. A. Rajabally R. D. M. Hadden R. J. de Haan C. A. J. M. de Borgie M. P. Lunn I. N. van Schaik F. Eftimov on behalf of the OPTIC study group |
| author_sort | S. R. M. Bus |
| collection | DOAJ |
| description | Abstract Background International guidelines recommend either intravenous immunoglobulin (IVIg) or corticosteroids as first-line treatment for chronic inflammatory demyelinating polyradiculoneuropathy (CIDP). IVIg treatment usually leads to rapid improvement and is generally safe, but does not seem to lead to long-term remissions. Corticosteroids act more slowly and are associated with more side effects, but may induce long-term remissions. The hypothesis of this study is that combined IVIg and corticosteroid induction treatment will lead to more frequent long-term remissions than IVIg treatment alone. Methods An international, randomised, double-blind, placebo-controlled trial, in adults with ‘probable’ or ‘definite’ CIDP according to the EFNS/PNS 2010 criteria. Three groups of patients are included: (1) treatment naïve, (2) known CIDP patients with a relapse after > 1 year without treatment, and (3) patients with CIDP who improved within 3 months after a single course of IVIg, who subsequently deteriorate at any interval without having received additional treatment. Patients are randomised to receive 7 courses of IVIg and 1000 mg intravenous methylprednisolone (IVMP) (in sodium chloride 0.9%) or IVIg and placebo (sodium chloride 0.9%), every 3 weeks for 18 weeks. IVIg treatment consists of a loading dose of 2 g/kg (over 3–5 days) followed by 6 courses of IVIg 1/g/kg (over 1–2 days). The primary outcome is remission at 1 year, defined as improvement in disability from baseline, sustained between week 18 and week 52 without further treatment. Secondary outcomes include changes in disability, impairment, pain, fatigue, quality of life, care use and costs and (long-term) safety. Discussion In case of superiority of the combined treatment, patients will experience the advantages of two proven efficacious treatments, namely rapid improvement due to IVIg and long-term remission due to corticosteroids. Long-term remission would reduce the need for maintenance IVIg treatment and may decrease health care costs. Additionally, we expect that the combined treatment leads to a higher proportion of patients with improvement as some patients who do not respond to IVIg will respond to corticosteroids. Risks of short and long-term additional adverse events of the combined treatment need to be assessed. Trial registration ISRCTN registry ISRCTN15893334 . Prospectively registered on 12 February 2018. |
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| institution | Directory Open Access Journal |
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| language | English |
| last_indexed | 2024-12-20T07:37:09Z |
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| spelling | doaj.art-86106c2f745f4ee7aa575fbdc28612b92022-12-21T19:48:15ZengBMCTrials1745-62152021-02-0122111810.1186/s13063-021-05083-1Intravenous immunoglobulin and intravenous methylprednisolone as optimal induction treatment in chronic inflammatory demyelinating polyradiculoneuropathy: protocol of an international, randomised, double-blind, placebo-controlled trial (OPTIC)S. R. M. Bus0L. Zambreanu1A. Abbas2Y. A. Rajabally3R. D. M. Hadden4R. J. de Haan5C. A. J. M. de Borgie6M. P. Lunn7I. N. van Schaik8F. Eftimov9on behalf of the OPTIC study groupDepartment of Neurology, Amsterdam Neuroscience, Amsterdam UMC, University of AmsterdamDepartment of Neurology, National Hospital for Neurology and Neurosurgery, Centre for Neuromuscular DiseaseDepartment of Neurology, University Hospitals of Birmingham, Regional Neuromuscular ServiceDepartment of Neurology, University Hospitals of Birmingham, Regional Neuromuscular ServiceDepartment of Neurology, King’s College HospitalClinical Research Unit, Amsterdam UMC, University of AmsterdamClinical Research Unit, Amsterdam UMC, University of AmsterdamDepartment of Neurology, National Hospital for Neurology and Neurosurgery, Centre for Neuromuscular DiseaseDepartment of Neurology, Amsterdam Neuroscience, Amsterdam UMC, University of AmsterdamDepartment of Neurology, Amsterdam Neuroscience, Amsterdam UMC, University of AmsterdamAbstract Background International guidelines recommend either intravenous immunoglobulin (IVIg) or corticosteroids as first-line treatment for chronic inflammatory demyelinating polyradiculoneuropathy (CIDP). IVIg treatment usually leads to rapid improvement and is generally safe, but does not seem to lead to long-term remissions. Corticosteroids act more slowly and are associated with more side effects, but may induce long-term remissions. The hypothesis of this study is that combined IVIg and corticosteroid induction treatment will lead to more frequent long-term remissions than IVIg treatment alone. Methods An international, randomised, double-blind, placebo-controlled trial, in adults with ‘probable’ or ‘definite’ CIDP according to the EFNS/PNS 2010 criteria. Three groups of patients are included: (1) treatment naïve, (2) known CIDP patients with a relapse after > 1 year without treatment, and (3) patients with CIDP who improved within 3 months after a single course of IVIg, who subsequently deteriorate at any interval without having received additional treatment. Patients are randomised to receive 7 courses of IVIg and 1000 mg intravenous methylprednisolone (IVMP) (in sodium chloride 0.9%) or IVIg and placebo (sodium chloride 0.9%), every 3 weeks for 18 weeks. IVIg treatment consists of a loading dose of 2 g/kg (over 3–5 days) followed by 6 courses of IVIg 1/g/kg (over 1–2 days). The primary outcome is remission at 1 year, defined as improvement in disability from baseline, sustained between week 18 and week 52 without further treatment. Secondary outcomes include changes in disability, impairment, pain, fatigue, quality of life, care use and costs and (long-term) safety. Discussion In case of superiority of the combined treatment, patients will experience the advantages of two proven efficacious treatments, namely rapid improvement due to IVIg and long-term remission due to corticosteroids. Long-term remission would reduce the need for maintenance IVIg treatment and may decrease health care costs. Additionally, we expect that the combined treatment leads to a higher proportion of patients with improvement as some patients who do not respond to IVIg will respond to corticosteroids. Risks of short and long-term additional adverse events of the combined treatment need to be assessed. Trial registration ISRCTN registry ISRCTN15893334 . Prospectively registered on 12 February 2018.https://doi.org/10.1186/s13063-021-05083-1Chronic inflammatory demyelinating polyradiculoneuropathyCIDPRandomised controlled trialRCTCorticosteroidsMethylprednisolone |
| spellingShingle | S. R. M. Bus L. Zambreanu A. Abbas Y. A. Rajabally R. D. M. Hadden R. J. de Haan C. A. J. M. de Borgie M. P. Lunn I. N. van Schaik F. Eftimov on behalf of the OPTIC study group Intravenous immunoglobulin and intravenous methylprednisolone as optimal induction treatment in chronic inflammatory demyelinating polyradiculoneuropathy: protocol of an international, randomised, double-blind, placebo-controlled trial (OPTIC) Trials Chronic inflammatory demyelinating polyradiculoneuropathy CIDP Randomised controlled trial RCT Corticosteroids Methylprednisolone |
| title | Intravenous immunoglobulin and intravenous methylprednisolone as optimal induction treatment in chronic inflammatory demyelinating polyradiculoneuropathy: protocol of an international, randomised, double-blind, placebo-controlled trial (OPTIC) |
| title_full | Intravenous immunoglobulin and intravenous methylprednisolone as optimal induction treatment in chronic inflammatory demyelinating polyradiculoneuropathy: protocol of an international, randomised, double-blind, placebo-controlled trial (OPTIC) |
| title_fullStr | Intravenous immunoglobulin and intravenous methylprednisolone as optimal induction treatment in chronic inflammatory demyelinating polyradiculoneuropathy: protocol of an international, randomised, double-blind, placebo-controlled trial (OPTIC) |
| title_full_unstemmed | Intravenous immunoglobulin and intravenous methylprednisolone as optimal induction treatment in chronic inflammatory demyelinating polyradiculoneuropathy: protocol of an international, randomised, double-blind, placebo-controlled trial (OPTIC) |
| title_short | Intravenous immunoglobulin and intravenous methylprednisolone as optimal induction treatment in chronic inflammatory demyelinating polyradiculoneuropathy: protocol of an international, randomised, double-blind, placebo-controlled trial (OPTIC) |
| title_sort | intravenous immunoglobulin and intravenous methylprednisolone as optimal induction treatment in chronic inflammatory demyelinating polyradiculoneuropathy protocol of an international randomised double blind placebo controlled trial optic |
| topic | Chronic inflammatory demyelinating polyradiculoneuropathy CIDP Randomised controlled trial RCT Corticosteroids Methylprednisolone |
| url | https://doi.org/10.1186/s13063-021-05083-1 |
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