Crohn’s disease is a chronic inflammatory condition of the gastrointestinal tract causing pain and diarrhoea that can mean life-long ill health, impaired quality of life and reduced life expectancy. About one in 200 people in developed countries suffer from Crohn disease or ulcerative colitis. Immunosuppressive drugs are the standard treatment for Crohn’s but some patients do not respond to it, or lose response to it over time.
Patient case reports had suggested that blood stem cell transplantation (HSCT) may help some patients so an international randomised clinical trial was set up by the Nottingham Digestive Diseases Centre (NDDC) to test this hypothesis. The results are published in the Journal of the American Medical Association.
The researchers randomly assigned 45 patients with Crohn’s disease who were not responsive to treatment or suitable for surgery to autologous (the use of one’s own cells) blood stem cell treatment or a control treatment in groups of 23 and 22. Patients were aged between 18 and 50 years and all were given standard Crohn’s treatment as needed. The trial involved 11 European transplant units from July 2007 to September 2011 with follow-up until March 2013.
‘No significant difference’
Leading the trial, Professor Chris Hawkey of the Nottingham Digestive Diseases Centre, said: “We found there was no statistically significant difference between the groups in the proportion of patients who met our highly stringent definition of sustained disease remission after treatment — only two in the stem cell transplant group (8.7 per cent) and one in the control group (4.5 per cent). However, there was a significant difference among patients able to discontinue active treatment in the last three months of participation in the trial — 61 per cent in the stem cell group and 23 per cent in the control, and objective signs of disease disappeared in about a quarter of patients.
“There were 76 serious adverse events in patients undergoing stem cell therapy and 38 in the control group. Because very few patients achieved sustained disease remission, we conclude that HSCT is unlikely to alter the natural history of Crohn disease, and our findings argue against extension of HSCT to a wider group of patients outside of future additional trials.”
Professor Dominique Farge Bancel, Saint Louis Hospital in Paris and Chair of the European Blood and Marrow Transplantation Society, added: “Although autologous HSCT therapy showed more toxicity in early stages of the follow-up in Crohn’s patients, long term results after HSCT treatment may shed new lights on the expected benefit, as observed in Scleroderma, another autoimmune disease where the proof of a HSCT superiority over conventional therapy was obtained only after long term follow-up at the price of higher early toxicity.”
The research concludes that further study of HSCT in patients with Crohn’s disease which does not respond to standard treatment is needed. It recommends that future trials should examine the benefit of maintenance immunosuppressive therapy after HSCT and also an investigation of whether patients may regain responsiveness to treatments which were previously ineffective for them. As toxicity is the most significant barrier to HSCT in patients with Crohn’s, more work is also needed to identify factors that predict either the risk of adverse effects or response to treatment to enhance the use of stem cell therapy in clinical practice.
The trial was a collaborative project conducted by leading entities in the fields of Bone Marrow Transplantation and Gastroenterology: the European Society for Blood and Marrow Transplantation (EBMT) and the European Crohn’s and Colitis Organisation (ECCO) and funded by the Broad Medical Foundation and the Nottingham Digestive Diseases Centre.
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Notes to editors: The University of Nottingham has 43,000 students and is ‘the nearest Britain has to a truly global university, with a “distinct” approach to internationalisation, which rests on those full-scale campuses in China and Malaysia, as well as a large presence in its home city.’ (Times Good University Guide 2016). It is also one of the most popular universities in the UK among graduate employers and the winner of ‘Research Project of the Year’ at the Times Higher Education Awards 2014. It is ranked in the world’s top 75 by the QS World University Rankings 2015/16, and 8th in the UK by research power according to the Research Excellence Framework 2014. It has been voted the world’s greenest campus for three years running, according to Greenmetrics Ranking of World Universities.
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