Myelodysplastic Syndromes in the Postgenomic Era and Future Perspectives for Precision Medicine

Chanias, Ioannis; Stojkov, Kristina; Stehle, Gregor Th.; Daskalakis, Michael; Simeunovic, Helena; Njue, Linet Muthoni; Schnegg-Kaufmann, Annatina S.; Porret, Naomi A.; Ramanjaneyulu, Allam; Tata, Nageswara Rao; Benz, Rudolf; Ruefer, Axel; Schmidt, Adrian; Adler, Marcel; Rovo, Alicia; Balabanov, Stefan; Stuessi, Georg; Bacher, Vera Ulrike; Bonadies, Nicolas (2021). Myelodysplastic Syndromes in the Postgenomic Era and Future Perspectives for Precision Medicine. Cancers, 13(13) MDPI AG 10.3390/cancers13133296

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Myelodysplastic syndromes (MDS) represent a heterogeneous group of clonal disorders caused by sequential accumulation of somatic driver mutations in hematopoietic stem and progenitor cells (HSPCs). MDS is characterized by ineffective hematopoiesis with cytopenia, dysplasia, inflammation, and a variable risk of transformation into secondary acute myeloid leukemia. The advent of next-generation sequencing has revolutionized our understanding of the genetic basis of the disease. Nevertheless, the biology of clonal evolution remains poorly understood, and the stochastic genetic drift with sequential accumulation of genetic hits in HSPCs is individual, highly dynamic and hardly predictable. These continuously moving genetic targets pose substantial challenges for the implementation of precision medicine, which aims to maximize efficacy with minimal toxicity of treatments. In the current postgenomic era, allogeneic hematopoietic stem cell transplantation remains the only curative option for younger and fit MDS patients. For all unfit patients, regeneration of HSPCs stays out of reach and all available therapies remain palliative, which will eventually lead to refractoriness and progression. In this review, we summarize the recent advances in our understanding of MDS pathophysiology and its impact on diagnosis, risk-assessment and disease monitoring. Moreover, we present ongoing clinical trials with targeting compounds and highlight future perspectives for precision medicine.

Item Type:

Journal Article (Review Article)

Division/Institute:

04 Faculty of Medicine > Pre-clinic Human Medicine > BioMedical Research (DBMR)
04 Faculty of Medicine > Department of Haematology, Oncology, Infectious Diseases, Laboratory Medicine and Hospital Pharmacy (DOLS) > Clinic of Haematology and Central Haematological Laboratory
04 Faculty of Medicine > Pre-clinic Human Medicine > BioMedical Research (DBMR) > Unit Childrens Hospital > Forschungsgruppe Hämatologie (Erwachsene)

UniBE Contributor:

Chanias, Ioannis; Stojkov, Kristina; Daskalakis, Michael; Simeunovic, Helena; Njue, Linet Muthoni; Schnegg-Kaufmann, Annatina; Porret, Naomi; Ramanjaneyulu, Allam; Tata, Nageswara Rao; Rovó, Alicia; Bacher, Vera Ulrike and Bonadies, Nicolas

Subjects:

600 Technology > 610 Medicine & health

ISSN:

2072-6694

Publisher:

MDPI AG

Language:

English

Submitter:

Pierrette Durand Lüthi

Date Deposited:

05 Jul 2021 15:55

Last Modified:

05 Jul 2021 15:55

Publisher DOI:

10.3390/cancers13133296

PubMed ID:

34209457

BORIS DOI:

10.48350/157275

URI:

https://boris.unibe.ch/id/eprint/157275

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