The current and future role of stem cells in myelodysplastic syndrome therapies

ABSTRACT

Introduction: Allogeneic blood stem cell transplantation (aBSCT) still is the only curative therapy for patients with myelodysplastic syndromes. While it carries the hope for cure for some patients, it may result in severe toxicity and death from complications or recurrent disease in others. Recent developments have improved patient and donor selection as well as technical aspects of the transplant procedure and post-transplant care, including early detection and treatment of relapse.

Areas covered: This review will discuss current stratification tools to identify suitable patients, donors and transplant techniques. In addition, it will cover the prognostic and predictive value of cytogenetics and somatic mutations and elucidate strategies for minimal residual disease detection as well as prophylactic and preemptive treatment of recurrent disease.

Expert commentary: aBSCT will continue to be the most powerful curative treatment option for patients with MDS. Advances in the understanding of disease biology will allow to incorporate molecular alterations in current prognostic scores, tracking of specific mutations during therapy and the use of novel, targeted therapies to augment the graft versus leukemia effect.

KEYWORDS:

• Myelodysplastic syndromes
• MDS
• hematopoietic stem cell transplantation
• minimal residual disease
• treatment of relapse

Declaration of interest

G Kobbe has received research funding from Celgene and Amgen as well as lecture fees and travel support from Celgene, Amgen, Jazz Pharmaceuticals, Medac, Neovii and Janssen-Cilag. T Schroeder has received: research funding from Celgene; lecture fees from Celgene and Janssen-Cilag; travel support from Novartis, Celgene, and Medac; and has served in advisory roles for Celgene; Novartis, and Takeda. U Germing has received: research funding from Celgene and Novartis; and lecture fees from Celgene, Novartis, and Janssen-Cilag. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed. Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Additional information

Funding

This paper was not funded.