Article Text
PDF
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.
Ethics and dissemination Ethical approval from NRES Committee South West (IRAS number 225959). EudraCT Number: 2017-001171-23. Results will be submitted for publication in peer-reviewed journals.
Trial registration number NCT03156452
- immune thrombocytopenia
- itp
- prednisolone
- mycophenolate
- dexamethasone
- corticosteroid
This is an open access article distributed in accordance with the Creative Commons Attribution 4.0 Unported (CC BY 4.0) license, which permits others to copy, redistribute, remix, transform and build upon this work for any purpose, provided the original work is properly cited, a link to the licence is given, and indication of whether changes were made. See: https://creativecommons.org/licenses/by/4.0/.
Statistics from Altmetric.com
Footnotes
Contributors CAB is chief investigator for the Flight trial and was responsible for writing the protocol with clinical input from NC, QH and CB. RG is the trial statistician who has contributed to the trial design and writing of protocol. JI contributed to trial design and leads the patient advisory group input. RK is the trial health economist and provided input to the trial design and protocol. JP and IT (Cardiff CTR) and KW (sponsor representative) have also provided contribution to writing the protocol. AM and AD have provided mentorship to the chief investigator.
Funding The FLIGHT trial is independent research funded by the National Institute for Health Research (NIHR) under its Research for Patient Benefit (RfPB) Programme (Grant Reference Number PB-PG-0815- 20016).
Disclaimer The views expressed are those of the authors and not necessarily those of the NHS, the NIHR or the Department of Health.
Competing interests None declared.
Patient consent Not required.
Ethics approval Ethical approval from NRES Committee South West (IRAS number 225959).
Provenance and peer review Not commissioned; externally peer reviewed.