Current Therapy of the Patients with MDS: Walking towards Personalized Therapy
Myelodysplastic syndromes (MDS) are characterized by ineffective hematopoiesis, dysplasia and peripheral cytopenias. Nowadays, MDS therapy is selected based on risk. The goals of therapy are different in low-risk and high-risk patients. In low-risk MDS, the goal is to decrease transfusion needs and...
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| Format: | Article |
| Language: | English |
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MDPI AG
2021-05-01
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| Series: | Journal of Clinical Medicine |
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| Online Access: | https://www.mdpi.com/2077-0383/10/10/2107 |
| _version_ | 1797534176392511488 |
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| author | Maria Luisa Palacios-Berraquero Ana Alfonso-Piérola |
| author_facet | Maria Luisa Palacios-Berraquero Ana Alfonso-Piérola |
| author_sort | Maria Luisa Palacios-Berraquero |
| collection | DOAJ |
| description | Myelodysplastic syndromes (MDS) are characterized by ineffective hematopoiesis, dysplasia and peripheral cytopenias. Nowadays, MDS therapy is selected based on risk. The goals of therapy are different in low-risk and high-risk patients. In low-risk MDS, the goal is to decrease transfusion needs and to increase the quality of life. Currently, available drugs for newly diagnosed low-risk MDS include growth factor support, lenalidomide and immunosuppressive therapy. Additionally, luspatercept has recently been added to treat patients with MDS with ring sideroblasts, who are not candidates or have lost the response to erythropoiesis-stimulating agents. Treatment of high-risk patients is aimed to improve survival. To date, the only currently approved treatments are hypomethylating agents and allogeneic stem cell transplantation. However, the future for MDS patients is promising. In recent years, we are witnessing the emergence of multiple treatment combinations based on hypomethylating agents (pevonedistat, magrolimab, eprenetapopt, venetoclax) that have proven to be effective in MDS, even those with high-risk factors. Furthermore, the approval in the US of an oral hypomethylating agent opens the door to exclusively oral combinations for these patients and their consequent impact on the quality of life of these patients. Relapsed and refractory patients remain an unmet clinical need. We need more drugs and clinical trials for this profile of patients who have a dismal prognosis. |
| first_indexed | 2024-03-10T11:26:53Z |
| format | Article |
| id | doaj.art-73484d3a66194fa1bed1096a74f39a6c |
| institution | Directory Open Access Journal |
| issn | 2077-0383 |
| language | English |
| last_indexed | 2024-03-10T11:26:53Z |
| publishDate | 2021-05-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Journal of Clinical Medicine |
| spelling | doaj.art-73484d3a66194fa1bed1096a74f39a6c2023-11-21T19:36:47ZengMDPI AGJournal of Clinical Medicine2077-03832021-05-011010210710.3390/jcm10102107Current Therapy of the Patients with MDS: Walking towards Personalized TherapyMaria Luisa Palacios-Berraquero0Ana Alfonso-Piérola1Hematology and Hemotherapy Department, Clínica Universidad de Navarra, 31008 Pamplona, SpainHematology and Hemotherapy Department, Clínica Universidad de Navarra, 31008 Pamplona, SpainMyelodysplastic syndromes (MDS) are characterized by ineffective hematopoiesis, dysplasia and peripheral cytopenias. Nowadays, MDS therapy is selected based on risk. The goals of therapy are different in low-risk and high-risk patients. In low-risk MDS, the goal is to decrease transfusion needs and to increase the quality of life. Currently, available drugs for newly diagnosed low-risk MDS include growth factor support, lenalidomide and immunosuppressive therapy. Additionally, luspatercept has recently been added to treat patients with MDS with ring sideroblasts, who are not candidates or have lost the response to erythropoiesis-stimulating agents. Treatment of high-risk patients is aimed to improve survival. To date, the only currently approved treatments are hypomethylating agents and allogeneic stem cell transplantation. However, the future for MDS patients is promising. In recent years, we are witnessing the emergence of multiple treatment combinations based on hypomethylating agents (pevonedistat, magrolimab, eprenetapopt, venetoclax) that have proven to be effective in MDS, even those with high-risk factors. Furthermore, the approval in the US of an oral hypomethylating agent opens the door to exclusively oral combinations for these patients and their consequent impact on the quality of life of these patients. Relapsed and refractory patients remain an unmet clinical need. We need more drugs and clinical trials for this profile of patients who have a dismal prognosis.https://www.mdpi.com/2077-0383/10/10/2107myelodysplastic syndrometreatmentluspaterceptazacitidinepevonedistatmagrolimab |
| spellingShingle | Maria Luisa Palacios-Berraquero Ana Alfonso-Piérola Current Therapy of the Patients with MDS: Walking towards Personalized Therapy Journal of Clinical Medicine myelodysplastic syndrome treatment luspatercept azacitidine pevonedistat magrolimab |
| title | Current Therapy of the Patients with MDS: Walking towards Personalized Therapy |
| title_full | Current Therapy of the Patients with MDS: Walking towards Personalized Therapy |
| title_fullStr | Current Therapy of the Patients with MDS: Walking towards Personalized Therapy |
| title_full_unstemmed | Current Therapy of the Patients with MDS: Walking towards Personalized Therapy |
| title_short | Current Therapy of the Patients with MDS: Walking towards Personalized Therapy |
| title_sort | current therapy of the patients with mds walking towards personalized therapy |
| topic | myelodysplastic syndrome treatment luspatercept azacitidine pevonedistat magrolimab |
| url | https://www.mdpi.com/2077-0383/10/10/2107 |
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