Novel therapies in low- and high-risk myelodysplastic syndrome.

Abstract

Introduction: Myelodysplastic syndromes (MDS) comprise a heterogeneous group of myeloid neoplasms with diverse clinical courses. The revised version of the international prognostic scoring system (IPSS-R) provides risk stratification into 5 different groups. Areas covered: For lower-risk patients, red blood cell transfusions and iron chelation are the backbone of supportive care. In addition, erythropoiesis-stimulating agents (ESA) are used to ameliorate anemia. Lenalidomide is approved for the treatment of lower-risk patients with del(5q) who are transfusion-dependent. Patients with higher-risk disease should be offered allogeneic stem cell transplantation whenever possible. If they are unfit for transplantation or an appropriate donor cannot be found, hypomethylating agents may be used. Expert opinion: New therapeutic options for lower-risk patients include thrombopoietin analogues, the TGF-beta family ligand trapping drug Luspatercept, and the telomerase inhibitor Imetelstat. Combinations of hypomethylating agents (HMA) with other compounds, and inhibitors of bcl2, such as venetoclax are being developed for higher-risk patients. Finally, hypomethylating agents in combination with donor lymphocytes may lead to long-term remission following molecular or hematological relapse after allogeneic SCT.

• Authors: Ulrich Germing, Thomas Schroeder, Jennifer Kaivers, Andrea Kündgen, Guido Kobbe, Norbert Gattermann
• Journal: Expert review of hematology (Expert Rev Hematol) Vol. 12 Issue 10 Pg. 893-908 (10 2019) ISSN: 1747-4094 [Electronic] England
• PMID: 31353975 (Publication Type: Journal Article, Review)

Chemical References

• Antineoplastic Agents
• Bridged Bicyclo Compounds, Heterocyclic
• Hematinics
• Immunoglobulin Fc Fragments
• Iron Chelating Agents
• Oligonucleotides
• Recombinant Fusion Proteins
• Sulfonamides
• Thalidomide
• Decitabine
• Thrombopoietin
• luspatercept
• Activin Receptors, Type II
• Lenalidomide
• imetelstat
• Azacitidine
• venetoclax

Topics

• Activin Receptors, Type II (therapeutic use)
• Anemia (diagnosis, metabolism, pathology, therapy)
• Antineoplastic Agents (therapeutic use)
• Azacitidine (therapeutic use)
• Bridged Bicyclo Compounds, Heterocyclic (therapeutic use)
• Decitabine (therapeutic use)
• Erythrocyte Transfusion (adverse effects, methods)
• Hematinics (therapeutic use)
• Humans
• Immunoglobulin Fc Fragments (therapeutic use)
• Iron Chelating Agents (therapeutic use)
• Lenalidomide (therapeutic use)
• Myelodysplastic Syndromes (diagnosis, metabolism, pathology, therapy)
• Oligonucleotides (therapeutic use)
• Prognosis
• Recombinant Fusion Proteins (therapeutic use)
• Risk Assessment
• Stem Cell Transplantation
• Sulfonamides (therapeutic use)
• Thalidomide (therapeutic use)
• Thrombopoietin (therapeutic use)
• Transplantation, Homologous