Current therapy and new drugs: a road to personalized treatment of myelodysplastic syndromes

ABSTRACT

Introduction: An accurate diagnostic and prognostic evaluation of patients with myelodysplastic syndromes (MDS) is essential: treatments are different for patients at lower or higher risk of evolution into acute myeloid leukemia (AML). Molecular data are more and more important: gene mutations or microRNA profiling can be used to better stratify MDS patients and address them to a personalized therapeutic approach.

Areas covered: This review focuses on the European MDS diagnosis, prognosis and therapy. The authors, stemming from literature and published data, discuss and describe the current state-of-the art of MDS, focusing on conventional tools, such as morphology and cytogenetics, but also illustrating new molecular data and how targeting these molecules could be important to develop innovative therapeutic approaches.

Expert commentary: Currently, MDS treatments include supportive therapies, iron chelation and erythroid-stimulating agents (ESAs) for lower-risk MDS, Lenalidomide for del(5q) MDS, hypomethylating agents (HMAs) and allogeneic stem cell transplantation (SCT) for higher risk patients. Molecular analyses are acquiring great relevance in MDS diagnosis, prognosis and therapy. Interestingly, PI-PLCbeta1 regulates hematopoietic differentiation, is a predictive marker of response to azacitidine and could be an ideal target for drug design, aiming to regulate MDS pathogenesis and prevent MDS progression to AML.

KEYWORDS:

• Myelodysplastic syndromes
• diagnosis
• prognosis
• therapy
• azacitidine
• Lenalidomide
• Phosholipase C beta1
• gene mutations

Declaration of interest

C. Finelli discloses Celgene: Research Funding; Novartis: Advisory Board; Janssen-Cilag: Advisory Board. 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 work was supported by grants from Italian MIUR-PRIN and Intesa S. Paolo.