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The role of allogeneic hematopoietic stem cell transplantation for patients with Myelodysplastic Syndromes

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Prof. Theo de Witte

9 January 2018

Transplantation with blood stem cells from donors for patients with Myelodysplastic Syndromes (MDS) has been introduced into clinical practice about 35 years ago. Initially, this innovative and revolutionary treatment has been pioneered in young and fit patients with advanced stages of MDS. Many patients died prematurely due to complications of the transplantation procedure despite the young age of patients and relapse rates were high due to the advanced stages in most patients. Nevertheless, it became more and more clear that a rewarding minority of the transplanted patients became long term survivors. We learned quite a lot from our experience during the early exploratory phase.

In the beginning, only matched family members could be used as a donor which implicated that a donor was only available in about 30% of the transplantation candidates. We could extend the number of available donors with unrelated donors thanks to better tissue typing techniques and the creation of large (inter)national donor banks. Nowadays, almost all transplantation candidates will have a suitable donor identified within a relatively short search period. We learned that the most potent curative feature of transplantation was mediated by the transplanted donor immune cells, which exert a strong graft versus tumour (MDS) effect. Therefore, we could reduce the intensity of the classical transplant conditioning towards a less toxic level. This development allowed us to extend the indication for transplantation to older and less fit patients with rewarding results.

A group of 32 leading experts from the European LeukemiaNet and from the United States has analysed the available data on transplantation in MDS and formulated recommendations how and when to advice a patient with MDS to undergo a transplantation. The basis for these recommendations were the published data and the expert opinion on about 100 clinical scenario’s which were developed to discuss the expert opinions on the various clinical and biological issues.

The experts agreed that transplantation was the treatment of choice for patients with high risk MDS as defined by the revised International Prognostic Scoring System, provided that patients are fit enough to undergo this demanding treatment. The intriguing question concerns the advice how to prepare for transplantation? Should a patient receive cytoreductive treatment prior to the transplantation conditioning? In practice, most patients do receive treatment with hypomethylating agents or intensive chemotherapy, but the value has not been proven by appropriate, clinical studies.

My personal advice to a patient with high risk MDS is to go for transplantation as soon as possible if an agreement for transplantation has been established by the patient and the treating physicians.

The longer the delay to transplantation, the higher the risk for complications and relapse of MDS after transplantation.

Patients with earlier stages of MDS should consider a transplantation when the available treatment interventions have failed or when additional poor-risk factors have been identified over time. Poor-risk factors include progression of the disease, detoriation of the blood counts, the development of transfusion dependency, and last but not least detection of poor-risk molecular mutations.

Details of the recommendations can be found in the official publication of the recommendations formulated by the expert panel (1). The expert panel is developing an interactive website which will support both patients and their treating physicians to reach a well-balanced decision concerning transplantation. We hope to launch the interactive tool early in 2018 – keep an eye on this site for more information.

On behalf of the expert panel and the MDS-Right team,

Theo de Witte

1. de Witte T, Bowen D, Robin M, et al. Allogeneic hematopoietic stem cell transplantation for MDS and CMML: recommendations from an international expert panel. Blood. 2017;129(13):1753-1762.


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