Press Release

medac announces initial results of the phase III trial on conditioning therapy with treosulfan prior to allogeneic haematopoietic stem cell transplantation at the 2017 ASH Annual Meeting

Myelodysplastic syndromes (MDS) and acute myeloid leukaemia (AML) are the most common malignant diseases of bone marrow in adults. The prognosis of some patient populations is still poor. In addition, survival rate falls further with increasing age.1

For these risk groups of AML and MDS patients, allogeneic haematopoietic stem cell transplantation (HSCT) is the only potentially curative treatment option.2 However, before receiving healthy donor cells, the patients’ diseased bone marrow cells must first be destroyed. Amongst others, this is done by using what is known as conditioning therapy, by radiation and/or by administering chemotherapeutic agents. This therapy is absolutely essential, but is associated with a high burden and occasionally toxic or even life-threatening side effects for the affected patients.3

There is consequently a great need for effective and, at the same time, less toxic conditioning therapies. This was the inspiration for the largest to date, prospective, randomised, multicentre phase III trial4 in predominantly elderly and, in some cases, comorbid AML and MDS patients requiring allogeneic HSCT. The comparative trial tested a treosulfan-based conditioning regimen as an alternative to the established, reduced-intensity busulfan therapy. The trial was prematurely terminated at the end of last year because the primary objective had already been achieved. Initial results based on the data of 476 patients are now available and will be presented at the 59th Annual Meeting of the American Society of Hematology (ASH) in Atlanta. The abstract will be presented by Prof. Dietrich W. Beelen, University of Duisburg/Essen, the trial leader and representative of the 30 European transplantation centres that are actively participating in the trial.

Session 732: Clinical Allogeneic Transplantation: Results: GVH and GVL
Room: Georgia World Congress Center, Building C, Level 2, C211-C213
Date: Sunday 10 December 2017
Time: 4:30-6:00 PM

Abstract No. 521
Final Results of a Prospective Randomized Multicenter Phase III Trial Comparing Treosulfan/Fludarabine to Reduced Intensity Conditioning with Busulfan/Fludarabine Prior to Allogeneic Hematopoietic Stem Cell Transplantation in Elderly or Comorbid Patients with Acute Myeloid Leukemia or Myelodysplastic Syndrome
First author: Prof. Dietrich W. Beelen, University of Duisburg/Essen
Time: 5:30 PM

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1Juliusson G et al. Age and acute myeloid leukemia: real world data on decision to treat and outcomes from the Swedish Acute Leukemia Registry. Blood. 2009;113(18):4179-87.
2Niederwieser D et al. Hematopoietic stem cell transplantation activity worldwide in 2012 and a SWOT analysis of the Worldwide Network for Blood and Marrow Transplantation Group including the global survey. Bone Marrow Transplant. 2016;51(6):778-5.
3Mohty B, Mohty M. Long-term complications and side effects after allogeneic hematopoietic stem cell transplantation: an update. Blood Cancer J. 2011;1(4):e16. Clinical Phase III Trial to Compare Treosulfan-based Conditioning Therapy With Busulfan-based Reduced-intensity Conditioning (RIC) Prior to Allogeneic Haematopoietic Stem Cell Transplantation in Patients With AML or MDS Considered Ineligible to Standard Conditioning Regimens. NCT0082239. URL: (Stand: 13.11.2017).