FLIGHT Study, V1.0
Research type
Research Study
Full title
A multicentre randomised trial of First Line treatment pathways for newly diagnosed Immune Thrombocytopenia: Standard steroid treatment versus combined steroid and mycophenolate.\n\n
IRAS ID
225959
Contact name
Charlotte Bradbury
Contact email
Sponsor organisation
University Hospitals Bristol NHS Foundation Trust
Eudract number
2017-001171-23
Duration of Study in the UK
3 years, 0 months, 2 days
Research summary
Summary of Research
This is a study of two treatment pathways [Standard steroid treatment versus combined steroid and Mycophenolate (MMF)] for subjects with newly diagnosed Immune Thrombocytopenia (ITP). ITP is an illness that causes bruising and bleeding due to a low platelet count (blood cells essential for normal clotting). Patients are first given high dose steroids but most suffer side effects (e.g. difficulty sleeping, weight gain, moods swings, high blood pressure and diabetes). In addition, the majority of patients become ill again when the steroids are stopped - only about 20% stay well long term. ITP is relatively rare, non-cancerous in nature and the rare impact on survival of ITP have prevented it from being a priority for research funding, with first line treatment being unsatisfactory and unchallenged for decades. This underestimates the profound adverse impact an ITP diagnosis and its treatment has on individual patients, many of whom are young. \n\nMMF is often used as the next stage treatment for ITP and it works well. However, it can take up to 2 months to work during which patients continue to be at risk of bleeding, bruising, fatigue and usually need more steroids which they find intolerable. They are required to come to hospital for weekly blood tests and for many this impacts on work. We want to find out whether it would benefit more patients if everyone takes MMF at diagnosis instead of current practice (waiting for the illness to come back). We plan to test this by comparing the current way we treat patients to a new way with patients given MMF right at the start of their treatment. 120 patients from 20 different hospitals will be asked to take part and half will be randomly chosen for the new pathway. \nSummary of Results
Background. Immune Thrombocytopenia (ITP) is an illness causing bruising
and bleeding due to a low platelet count (blood cells essential for normal
clotting). It is a rare auto-immune disease, is non-cancerous and the
occasional impact on survival has prevented it being a research funding
priority.# The FLIGHT trial was the first independently funded UK
multicentre clinical trial for ITP and represented a big step forward for
research in this disease. The trial duration was 3 years and we aimed to
recruit 120 participants in a 12-month period with a minimum 12-month
follow up. There was an optional laboratory study for participants to give a
blood sample to study predictive bio-markers of treatment response and
illness outcomes. The trial question was to compare mycophenolate (MMF)
plus steroids as a first treatment with current treatment of high dose
steroids in newly diagnosed ITP participants. Essentially, to find out whether
MMF would delay disease relapse and participants would stay well for
longer if they took MMF at diagnosis instead of what currently happens,
which is waiting for the illness to return. Who was recruited: We recruited
123 participants from 43 hospital sites, of which 3 participants were
excluded, one participant tested positive for HIV, one lost to follow up and
one decided to go for private care shortly after consenting. Participants
were 16 years and over, with a low platelet count (less than 30) and
required first treatment for their ITP. Participants were excluded if they
tested positive for HIV, had common variable immunodeficiency, were
pregnant, breastfeeding or unwilling to follow contraception advice if they
were randomly selected to receive MMF treatment. What was measured.
The key measurement was the time it took participants to reach treatment
failure (treatment that no longer works), from the date they were
randomised and received their first treatment. We also recorded treatment
side effects, whether participants had any bleeding events and their quality
of life, measured by questionnaires completed at hospital visits. Results. Of
the 120 ITP participants consented, recruited and randomised, the age
range was 17-87 years, with an average age of 54 years, and 52% were
male. Compared to participants receiving steroids alone, those also taking
MMF were less likely to fail treatment, more likely to respond and less likely
to relapse. There were similar rates between the two participant groups of
bleeding events, rescue treatments, hospital admissions and treatment side
effects (including infection). However, some aspects of quality of life over
the 12-month follow-up period may have been slightly shown to be worse
in those participants assigned to the MMF plus steroids group, including
fatigue. Conclusion. This is the first randomised trial using MMF to treat ITP
soon after diagnosis and it was an effective and well tolerated first
treatment option alongside a short course of steroids (even with the
inclusion of elderly participants). Therefore, this combination of treatment
may be considered for some patients in whom early disease control is
particularly important. It was unclear why some aspects of quality of life
may have been worse in the MMF group, and this is an important reminder
that researchers should continue to explore and examine these measures of
patient experience when testing new treatment strategiesREC name
South West - Central Bristol Research Ethics Committee
REC reference
17/SW/0127
Date of REC Opinion
12 Jun 2017
REC opinion
Further Information Favourable Opinion