EBMT NEWSLETTER | March 2015 | Volume 46 - Issue 1

Important dates
Current outcome of HLA identical sibling vs. unrelated donor transplants in severe aplastic anemia: an EBMT analysis.
Bacigalupo A, Socié G, Hamladji RM, Aljurf M, Maschan A, Kyrcz-Krzemien S, Cybicka A, Sengelov H, Unal A, Beelen D, Locasciulli A, Dufour C, Passweg JR, Oneto R, Signori A, Marsh JC.
Haematologica. 2015 Jan 23. pii: haematol.2014.115345. [Epub ahead of print]

The potential implications of using an unrelated donor transplant in patients with acquired aplastic anemia in the need of an allogeneic stem cell transplantation are discussed in this retrospective analysis of the Aplastic Anemia Working Party of the European Group for Blood and Marrow Transplantation. 940 identical sibling transplants were compared with 508 unrelated donor transplantations. As expected, unrelated transplants shared some negative clinical features such as longer time between diagnosis and transplantation, to have a CMV negative donor / recipient status, to receive anti-thymocyte globulin in the conditioning regimen and to use peripheral blood stem cells instead of bone marrow. As expected the incidence of clinically significant acute and chronic graft versus host disease was higher in the unrelated population of patients but non-relapse mortality was not significantly different between both groups of patients in spite of the fact that the use of peripheral blood stem cells was the strongest predictor for this outcome. At the end of the day, the long- term outcome of patients with aplastic anemia being treated with anunrelated donor stem cell transplantation was not significantly different than that of HLA identical sibling transplants. A note of caution has to be made with the use of peripheral blood as stem cell source in this clinical setting.
DOCK8 Deficiency: Clinical and Immunological Phenotype and Treatment Options - a Review of 136 Patients.
Aydin SE, Kilic S, Aytekin C, Kumar A, Porras O, Kainulainen L, Kostyuchenko L, Genel F, Kütükcüler N, Karaca N, Gonzalez-Granado L, Abbott J, Al-Zahrani D, Rezaei N, Baz Z, Thiel J, Ehl S, Marodi L, Orange JS, Sawalle-Belohradsky J, Keles S, Holland SM, Sanal Ö, Ayvaz DC, Tezcan I, Al-Mousa H, Alsum Z, Hawwari A, Metin A, Matthes-Martin S, Hönig M, Schulz A, Picard C, Barlogis V, Gennery A, Ifversen M, van Montfrans J, Kuijpers T, Bredius R, Dückers G, Al-Herz W, Pai SY, Geha R, Notheis G, Schwarze CP, Tavil B, Azik F, Bienemann K, Grimbacher B, Heinz V, Gaspar HB, Aydin R, Hagl B, Gathmann B, Belohradsky BH, Ochs HD, Chatila T, Renner ED, Su H, Freeman AF, Engelhardt K, Albert MH; On behalf of the inborn errors working party of EBMT.
J Clin Immunol. 2015 Jan 28. [Epub ahead of print]

An international survey on behalf the Inborn Errors Working Party of EBMT  aiming to describe the natural course, long term prognosis and optimal treatment of a group of 136 patients diagnosed of hyper-IgE syndrome with combined immunodeficiency. This disorder has an autosomal recessive inheritance and results from mutations in DOCK8. The clinical picture was characterized by recurrent infections from bacterial, viral and fungal origin. With a median follow up of all the patients of 11.3 years, overall survival (censored at hematopoietic stem cell transplantation) was of 87%, 47% and 33% at 10, 20 and 30 years of age. Event free survival was 44%, 18% and 4% at the same three time points. Interestingly, malignancies were diagnosed in 17% of the patients at a median age of 12 years, life-threatening infectious episodes were observed in 58% of the patients and non infectious cerebral events occurred in 10% of the population. Treatment options for this group of patients include conservative measures such as antiviral and antibacterial prophylaxis, immunoglobulin replacement and hematopoietic stem cell transplantation. The authors conclude that stem cell transplantation should be strongly considered as a therapeutic alternative in this disease due to its severity and adverse prognosis in the long term. This study shows that collaborative work under the leadership of an EBMT Working Party can provide useful data on extremely rare disorders such as the one described in this manuscript.
The EBMT/EMCL consensus project on the role of autologous and allogeneic stem cell transplantation in mantle cell lymphoma.
Robinson S, Dreger P, Caballero D, Corradini P, Geisler C, Ghielmini M, Le Gouill S, Kimby E, Rule S, Vitolo U, Dreyling M, Hermine O.
Leukemia. 2015 Feb;29(2):464-73. doi: 10.1038/leu.2014.223. Epub 2014 Jul 18.

Consensus documents elaborated with the use of scientific methods such as the RAND-modified Delphi consensus procedure are currently being used to try to establish the role of certain treatment strategies when the strength of the evidence is not adequate enough. The Lymphoma Working Party of the European Society for Blood and Marrow transplantation has undertaken this effort for the second time in the setting of Mantle Cell Lymphoma. In this sense, the group of experts listed above considered that autologous stem cell transplantation was the standard of care for patients in complete remission / partial remission after first line induction chemotherapy containing both Rituximab and ara-C. Autologous transplantation should not be omitted in the so called ‘low risk’ group of patients and there was no evidence supporting the use of maintenance with Rituximab after transplantation. No consensus was reached in other aspects such as the role of autologous transplant in patients in relapse after a non-transplant strategy, the role of total body irradiation in the conditioning regimen, the value of positron emission tomography as well as minimal residual monitoring after transplantation, in vivo purging with Rituximab and preemptive Rituximab after transplantation. Allogeneic approaches should be considered for patients relapsing after an autologous transplant with the use of reduced intensity conditioning protocols. The role of allogeneic transplant as first transplant as well as its role in relapsed disease following non-transplant strategies still needs to be defined.

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