EBMT NEWSLETTER | March 2016 | Volume 51 - Issue 2

Important dates
Economics and Outcome After Hematopoietic Stem Cell Transplantation: A Retrospective Cohort Study.
Gratwohl A, Sureda A, Baldomero H, Gratwohl M, Dreger P, Kröger N, Ljungman P, McGrath E, Mohty M, Nagler A, Rambaldi A, de Elvira CR, Snowden JA, Passweg J, Apperley J, Niederwieser D, Stijnen T, Brand R; Joint Accreditation Committee (JACIE) of the International Society for Cellular Therapy (ISCT) and the European Society for Blood and Marrow Transplantation (EBMT) and the European Leukemia Net (ELN).
EBioMedicine. 2015 Nov 19;2(12):2101-9. doi: 10.1016/j.ebiom.2015.11.021. eCollection 2015 Dec.

In this international study, led by EBMT a defined cohort of 102,549 patients treated with an allogeneic (N = 37,542; 37%) or autologous (N = 65,007; 63%) hematoptpoietic stem cell ransplantation were analyzed retrospectively. Center patient-volume and center program-duration were significantly and systematically associated with improved survival after allogeneic HSCT (HR 0·87; 0·84-0·91 per 10 patients; p < 0·0001; HR 0·90;0·85-0·90 per 10 years; p < 0·001) and autologous HSCT (HR 0·91;0·87-0·96 per 10 patients; p < 0·001; HR 0·93;0·87-0·99 per 10 years; p = 0·02). The product of Health Care Expenditures by Gross National Income/capita was significantly associated in multivariate analysis with all endpoints (R(2) = 18%; for relapse free survival) after allogeneic HSCT. Data indicate that country- and center-specific economic factors are associated with distinct, significant, systematic, and clinically relevant effects on survival after HSCT. They impact on center expertise in long-term disease and complication management. It is likely that these findings apply to other forms of complex treatments.

High-Dose Chemotherapy and Autologous Hematopoietic Stem Cell Transplantation as Adjuvant Treatment in High-Risk Breast Cancer: Data from the European Group for Blood and Marrow Transplantation Registry.
Martino M, Lanza F, Pavesi L, Öztürk M, Blaise D, Leno Núñez R, Schouten HC, Bosi A, De Giorgi U, Generali D, Rosti G, Necchi A, Ravelli A, Bengala C, Badoglio M, Pedrazzoli P, Bregni M; European Group for Blood and Marrow Transplantation Solid Tumor Working Party.
Biol Blood Marrow Transplant. 2016 Mar;22(3):475-81.
The aim of this retrospective study of the Solid Tumor working party was to assess toxicity and efficacy of adjuvant high-dose chemotherapy (HDC) and autologous hematopoietic stem cell transplantation (AHSCT) in 583 high-risk breast cancer (BC) patients (>3 positive nodes) who were transplanted between 1995 and 2005 in Europe. All patients received surgery before transplant, and 55 patients (9.5%) received neoadjuvant treatment before surgery. Median age was 47.1 years, 57.3% of patients were premenopausal at treatment, 56.5% had endocrine-responsive tumors, 19.5% had a human epidermal growth factor receptor 2 (HER2)-negative tumor, and 72.4% had ≥10 positive lymph nodes at surgery. Seventy-nine percent received a single HDC procedure. Overall transplant-related mortality was 1.9%, at .9% between 2001 and 2005, whereas secondary tumor-related mortality was .9%. With a median follow-up of 120 months, overall survival and disease-free survival rates at 5 and 10 years in the whole population were 75% and 64% and 58% and 44%, respectively. Subgroup analysis demonstrated that rates of overall survival were significantly better in patients with endocrine-responsive tumors, <10 positive lymph nodes, and smaller tumor size. HER2 status did not affect survival probability. Adjuvant HDC with AHSCT has a low mortality rate and provides impressive long-term survival rates in patients with high-risk BC. The authors conclude that this results suggest that this treatment modality should be considered in selected high-risk BC patients and further investigated in clinical trials.

Antilymphocyte Globulin for Prevention of Chronic Graft-versus-Host Disease.
Kröger N, Solano C, Wolschke C, Bandini G, Patriarca F, Pini M, Nagler A, Selleri C, Risitano A, Messina G, Bethge W, Pérez de Oteiza J, Duarte R, Carella AM, Cimminiello M, Guidi S, Finke J, Mordini N, Ferra C, Sierra J, Russo D, Petrini M, Milone G, Benedetti F, Heinzelmann M, Pastore D, Jurado M, Terruzzi E, Narni F, Völp A, Ayuk F, Ruutu T, Bonifazi F.
N Engl J Med. 2016 Jan 7;374(1):43-53.
In this prospective multicenter randomized trial, which was conducted as an EBMT labelled trial of the Chronic Malignancies Working Party a total of 168 patients with acute leukemia in CR who received a peripheral blood stem cell transplantation from HLA identical sibling were enrolled at 27 centers. Patients were randomly assigned in a 1:1 ratio to receive ATG or not receive ATG as part of a myeloablative conditioning regimen, with stratification according to center and risk of disease. Primary endpoint was chronic graft-versus-host disease (GVHD), which is the leading cause of later illness and death after allogeneic hematopoietic stem-cell transplantation. After a median follow-up of 24 months, the cumulative incidence of chronic GVHD was 32.2% in the ATG group and 68.7% in the non-ATG group (P<0.001). The rate of 2-year relapse-free survival was similar in the ATG group and the non-ATG group (59.4% and 64.6%), respectively; P=0.21), as was the rate of overall survival (74.1% and 77.9%, respectively; P=0.46). There were no significant between-group differences in the rates of relapse, infectious complications, acute GVHD, or adverse events. The rate of a composite end point of chronic GVHD-free and relapse-free survival at 2 years was significantly higher in the ATG group than in the non-ATG group (36.6% vs. 16.8%, P=0.005). The authors conclude that the inclusion of ATG resulted in a significantly lower rate of chronic GVHD after allogeneic transplantation than the rate without ATG. The survival rate was similar in the two groups, but the rate of a composite endpoint of chronic GVHD-free survival and relapse-free survival was higher with ATG.
Hemopoietic stem cell transplantation in thalassemia: a report from the European Society for Blood and Bone Marrow Transplantation Hemoglobinopathy Registry, 2000-2010.
Baronciani D, Angelucci E, Potschger U, Gaziev J, Yesilipek A, Zecca M, Orofino MG, Giardini C, Al-Ahmari A, Marktel S, de la Fuente J, Ghavamzadeh A, Hussein AA, Targhetta C, Pilo F, Locatelli F, Dini G, Bader P, Peters C.
Bone Marrow Transplant. 2016 Jan 11.
In this retrospective registry study of the Pediatric Working Party investigated transplantation distribution, demography, activity, policies and outcomes inside the European Group for Blood and Marrow Transplantation (EBMT) of transfusion dependent  patients with thalassemia. They performed a retrospective non-interventional study, extracting data from the EBMT hemoglobinopathy prospective registry database. They included 1493 consecutive patients with thalassemia major transplanted between 1 January 2000 and 31 December 2010. In total, 1359 (91%) transplants were performed on patients <18 years old, 1061 were from a human leukocyte Ag-identical sibling donor. After a median observation time of 2 years, the 2-year overall survival (OS) and event-free survival (EFS; that is, thalassemia-free survival) were 88±1% and 81±1%, respectively. Transplantation from a human leukocyte Ag-identical sibling offered the best results, with OS and EFS of 91±1% and 83±1%, respectively. No significant differences in survival were reported between countries. The threshold age for optimal transplant outcomes was around 14 years, with an OS of 90-96% and an EFS of 83-93% when transplants were performed before this age. Allogeneic HSCT for thalassemia is a curative approach that is employed internationally and produces excellent results.

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Bone 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.
2014 Impact Factor 3.570*
*2014 Journal Citation Reports® Science Edition (Thomson Reuters, 2015)
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Average time to online publication  = 24 days
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