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Trial protocol : a multicentre randomised trial of first-line treatment pathways for newly diagnosed immune thrombocytopenia : standard steroid treatment versus combined steroid and mycophenolate. The FLIGHT trial
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Pell, Julie, Greenwood, Rosemary, Ingram, Jenny, Wale, Katherine, Thomas, Ian, Kandiyali, Rebecca, Mumford, Andrew, Dick, Andrew, Bagot, Catherine, Cooper, Nichola, Hill, Quentin and Bradbury, Charlotte Ann (2018) Trial protocol : a multicentre randomised trial of first-line treatment pathways for newly diagnosed immune thrombocytopenia : standard steroid treatment versus combined steroid and mycophenolate. The FLIGHT trial. BMJ Open, 8 (10). e024427. doi:10.1136/bmjopen-2018-024427
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Official URL: http://dx.doi.org/10.1136/bmjopen-2018-024427
Abstract
Introduction: Immune thrombocytopenia (ITP) is an autoimmune condition that may cause thrombocytopenia-related bleeding. Current first-line ITP treatment is with high-dose corticosteroids but frequent side effects, heterogeneous responses and high relapse rates are significant problems with only 20% remaining in sustained remission with this approach. Mycophenolate mofetil (MMF) is often used as the next treatment with efficacy in 50%–80% of patients and good tolerability but can take up to 2 months to work.
Objective: To test the hypothesis that MMF combined with corticosteroid is a more effective first-line treatment for immune thrombocytopenia (ITP) than current standard of corticosteroid alone.
Design: Multicentre, UK-based, open-label, randomised controlled trial.
Setting: Haematology departments in secondary care.
Participants: We plan to recruit 120 patients >16 years old with a diagnosis of ITP and a platelet count <30x109/L who require first-line treatment. Patients will be followed up for a minimum of 12 months following randomisation.
Primary outcome: Time from randomisation to treatment failure defined as platelets <30x109/L and a need for second-line treatment.
Secondary outcomes: Side effects, bleeding events, remission rates, time to relapse, time to next therapy, cumulative corticosteroid dose, rescue therapy, splenectomy, socioeconomic costs, patient-reported outcomes (quality of life, fatigue, impact of bleeding, care costs).
Analysis: The sample size of 120 achieves a 91.5% power to detect a doubling of the median time to treatment failure from 5 to 10 months. This will be expressed as an HR with 95% CI, median time to event if more than 50% have had an event and illustrated with Kaplan-Meier curves. Cost-effectiveness will be based on the first 12 months from diagnosis.
| Item Type: | Journal Article | ||||||
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| Divisions: | Faculty of Medicine > Warwick Medical School > Health Sciences > Clinical Trials Unit Faculty of Medicine > Warwick Medical School |
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| Journal or Publication Title: | BMJ Open | ||||||
| Publisher: | BMJ | ||||||
| ISSN: | 2044-6055 | ||||||
| Official Date: | 18 October 2018 | ||||||
| Dates: |
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| Volume: | 8 | ||||||
| Number: | 10 | ||||||
| Article Number: | e024427 | ||||||
| DOI: | 10.1136/bmjopen-2018-024427 | ||||||
| Status: | Peer Reviewed | ||||||
| Publication Status: | Published | ||||||
| Access rights to Published version: | Open Access |
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