Summary of four EBMT recent studies published in peer-reviewed journalsAutologous stem-cell transplantation in treatment-refractory Crohn's disease: an analysis of pooled data from the ASTIC trial.
Lindsay JO, Allez M, Clark M, Labopin M, Ricart E, Rogler G, Rovira M, Satsangi J, Farge D, Hawkey CJ; ASTIC trial group.; European Society for Blood and Marrow Transplantation Autoimmune Disease Working Party.; European Crohn's and Colitis Organisation.
Lancet Gastroenterol Hepatol. 2017 Jun;2(6):399-406. Epub 2017 Apr 6.
The randomised controlled ASTIC trial on autologous HCT compared with mobilisation followed by conventional therapy in patients with refractory Crohn’s disease did not meet its very stringent primary endpoint (steroid-free clinical remission for 3 months with no endoscopic or radiological evidence of intestinal inflammation) [Hawkey CJ, et al. JAMA 2015]. The new analysis presented now assesses the results of all HCT procedures in patients enrolled in the ASTIC trial, 23 randomised to HCT first line and 17 from the conventional therapy arm who were eventually transplanted one year later. The analysis also uses endpoints that are traditional for clinical trials in Crohn's disease, and identifies independent factors that may predict benefit or harm. In this context, three-month steroid-free clinical remission one year after HCT was observed in 38% (95% CI 22-55) and complete endoscopic healing in 50% (95% CI 34-66) of patients. Factors with an independent impact on the primary outcome were short disease duration (OR 0.64 per year; p=0·048) and low baseline Crohn's Disease Activity Index (OR 0.82 per 10 units; p=0·031). Serious adverse events, most commonly infection, occurred in 23 of 40 patients with available data. Smoking and perianal disease at baseline were independent factors associated with the number of serious adverse events (OR 3.07 and 3.97, respectively). This analysis confirms that when assessed using traditional endpoints for conventional therapy in Crohn's disease, HCT resulted in clinical and endoscopic benefit. This study also shows that despite the relatively high burden of adverse events, HCT could be targeted to patients most likely to benefit without serious adverse events, based on the prognostic factors identified.
Use of haploidentical stem cell transplantation continues to increase: the 2015 European Society for Blood and Marrow Transplant activity survey report.
Passweg JR, Baldomero H, Bader P, Bonini C, Duarte RF, Dufour C, Gennery A, Kröger N, Kuball J, Lanza F, Montoto S, Nagler A, Snowden JA, Styczynski J, Mohty M.
Bone Marrow Transplant. Epub 2017 Mar 13.
The annual activity survey of the EBMT) has become over the years a key instrument to inform in Europe and affiliated countries about status of HCT and novel cell therapies with hematopoietic cells for non-hematopoietic use, as well as on the use of non-hematopoietic stem and progenitor cells. This year’s report provides useful novel information. First, it focuses on the continued growth of haploidentical HCT (a 291% increase from 2005 to 2015), which is seen across lymphoid and myeloid indications, both with bone marrow and peripheral blood as cell source, and including patients with advanced forms of the disease at transplant. Second, the report provides important information on the increase in transplant activity in the past decade per country according to World Bank income group gross national income per capita in USD in 2015. Higher income countries had already achieved a high level of transplant activity in 2005, and although they continue to have markedly higher transplant rates, the percentage increase in this period is highest for middle income countries for both allogeneic and autologous HCT. Third, the survey reports on the activity on other cellular therapies from 330 teams in 35 countries, including 2940 patients receiving donor lymphocyte infusions, 510 patients receiving mesenchymal stromal cells for GVHD treatment and graft enhancement, and 173 patients who received adoptive T cell therapy for infections or disease-relapse.
Dose-Reduced Versus Standard Conditioning Followed by Allogeneic Stem-Cell Transplantation for Patients With Myelodysplastic Syndrome: A Prospective Randomized Phase III Study of the EBMT (RICMAC Trial).
Kröger N, Iacobelli S, Franke GN, Platzbecker U, Uddin R, Hübel K, Scheid C, Weber T, Robin M, Stelljes M, Afanasyev B, Heim D, Deliliers GL, Onida F, Dreger P, Pini M, Guidi S, Volin L, Günther A, Bethge W, Poiré X, Kobbe G, van Os M, Brand R, de Witte T.
J Clin Oncol. Epub 2017 May 2.
The importance of the intensity of conditioning for allogeneic HCT remains a matter of debate, in particular for patients with myelodysplastic syndromes or secondary acute myeloid leukemia. Most analyses have traditionally come from retrospective studies, with unavoidable caveats. Here, the EBMT reports an important multicentre, prospective, randomised, open-label phase III clinical trial comparing busulfan-based dose-reduced versus standard myeloablative conditioning for allogeneic HCT in these patients (RICMAC Trial). A total of 129 patients were enrolled and randomly assigned in a 1:1 ratio, stratified according to donor type, patient age, and blast count. Overall, the study confirms prospectively that all main outcomes, including acute and chronic GVHD, non-relapse mortality and relapse rate are comparable between RIC and MAC. Thus, RIC provides a two-year relapse-free survival (62%) and overall survival (76%) similar to MAC (58% and 63%, respectively) in patients with myelodysplastic syndromes or acute myeloid leukemia.
Read the RICMAC press release entitled "Results of an EBMT phase III randomized trial on dose-reduced versus standard conditioning followed by allogeneic stem cell transplantation for patients with myelodysplastic syndrome".
JACIE accreditation for blood and marrow transplantation: past, present and future directions of an international model for healthcare quality improvement.
Snowden JA, McGrath E, Duarte RF, Saccardi R, Orchard K, Worel N, Kuball J, Chabannon C, Mohty M.
Bone Marrow Transplant. 2017 Mar 27. [Epub ahead of print]
Almost 20 years ago, the EBMT established the Joint Accreditation Committee of the International Society for Cellular Therapy and EBMT (JACIE), which in collaboration with the Foundation for the Accreditation of Cellular Therapy has become an internationally harmonised and recognised model for healthcare quality improvement in our complex and multidisciplinary field. The accreditation system is based on agreed quality standards and implemented by teams of voluntary inspectors. From the perspective of these two decades, this special report describes key JACIE elements such as its adoption across Europe, with over 530 voluntary accreditation inspections (62% first time; 38% reaccreditation) in 25 countries, representing 40% of transplant centres in Europe, the evidence showing its association with clinical benefits and positive impact on survival outcomes, as well as its incorporation into the regulatory framework for delivery of HCT and other haematopoietic cellular therapies in several countries. In addition, the manuscript explores the potential benefits of such an international model relative to national quality standards, as well as its potential disadvantages. Finally, from this historical perspective, the authors provide future directions with a focus on the development of Europe-wide risk-adjusted outcome benchmarking through the EBMT registry as well as on the further extension of the model beyond Europe, including access to the quality system for HCT programmes in in low- and middle-income economies via a ‘first-step’ process.
View all the EBMT publications on the website.
Bone Marrow Transplantation journal featured articlesBone Marrow Transplantation publishes high quality, peer reviewed original research and reviews that address all aspects of basic biology and clinical use of haemopoietic cell transplantation.
The journal also covers all aspects of the research and treatment of transplant-related complications and consequences including quality of life and psychological issues. Basic research studies on topics of relevance are also covered.
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*2016 Journal Citation Reports® Science Edition (Thomson Reuters, 2017)