Review
Oncology
Russell Lewis, Jan Philipp Bewersdorf, Amer M. Zeidan
Summary: For patients with LR-MDS, the goal is to alleviate symptoms and reduce transfusion burden associated with anemia. In addition to traditional treatments like supportive transfusions and ESAs, there are other therapeutic options available, such as luspatercept and lenalidomide, with novel agents like imetelstat and roxadustat in development.
CANCER MANAGEMENT AND RESEARCH
(2021)
Review
Hematology
Virginia O. Volpe, Rami S. Komrokji
Summary: Myelodysplastic syndromes (MDS) are characterized by bone-marrow failure, with anemia as the most common cytopenia in low-risk MDS patients. Current treatment options for MDS have limited durable responses and significant adverse effects, with chronic transfusions carrying risks. Luspatercept, a recently approved therapy for LR-MDS with ring sideroblasts, has shown improvement in transfusion independence with a well-tolerated safety profile.
THERAPEUTIC ADVANCES IN HEMATOLOGY
(2021)
Review
Oncology
Michael P. Randall, Amy E. DeZern
Summary: Myelodysplastic syndromes (MDSs) are a diverse group of hematologic neoplasms with different natural courses and prognoses. Treatment for low-risk MDS focuses on improving quality of life by addressing cytopenias instead of urgently modifying the disease to prevent acute myeloid leukemia. This can involve transfusion support, iron chelation, growth factors such as luspatercept, lenalidomide for del(5q) disease, and low-dose hypomethylating agents. Recent insights into the genetic mutations driving MDS have prompted a reconsideration of low-risk disease definition, leading to the identification of a subset of low-risk MDS patients who may benefit from more aggressive treatment, such as hematopoietic stem cell transplantation.
Review
Hematology
Maria Domenica Cappellini, Ali T. Taher, Amit Verma, Farrukh Shah, Olivier Hermine
Summary: Hematologic disorders like myelodysplastic syndromes and beta-thalassemia are characterized by ineffective erythropoiesis and anemia, usually managed through regular blood transfusions. However, treatments targeting the underlying mechanisms of ineffective erythropoiesis are limited, leading to a burden on patients, caregivers, and healthcare systems. This article provides an overview of the role of ineffective erythropoiesis in these disorders, discusses unmet needs in targeting it, and describes current management strategies and emerging treatments.
Article
Integrative & Complementary Medicine
Teng Fan, Xiaomin Feng, Asumi Yokota, Weiyi Liu, Yuting Tang, Xiaomei Yan, Haiyan Xiao, Yue Wang, Zhongyang Deng, Pan Zhao, Mingjing Wang, Hongzhi Wang, Rou Ma, Xiaomei Hu, Gang Huang
Summary: Traditional Chinese Medicine (TCM) such as Arsenic dispensing powder (ADP) has shown superior efficacy in treating MDS patients by improving anemia and prolonging overall survival. ADP promotes erythropoiesis in both normal and MDS clones through the downregulation of HIF1A and upregulation of GATA factors, suggesting it as a promising drug for MDS treatment.
AMERICAN JOURNAL OF CHINESE MEDICINE
(2021)
Article
Multidisciplinary Sciences
Yen K. Lieu, Zhaoqi Liu, Abdullah M. Ali, Xin Wei, Alex Penson, Jian Zhang, Xiuli An, Raul Rabadan, Azra Raza, James L. Manley, Siddhartha Mukherjee
Summary: SF3B1 is a frequently mutated RNA splicing factor in cancer, and its mutations are strongly associated with severe anemia in MDS patients. MAP3K7 dysfunction is identified as a key mechanism underlying SF3B1 mutation-induced anemia.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
(2022)
Review
Hematology
Felicitas Schulz, Kathrin Nachtkamp, Annika Kasprzak, Norbert Gattermann, Rainer Haas, Ulrich Germing
Summary: Luspatercept shows promise as an alternative to ESAs for a subset of transfusion-dependent patients with lower risk MDS. Its favorable safety profile and convenient subcutaneous administration every 3 weeks are more conducive to patients' quality of life than chronic red blood cell transfusion therapy.
EXPERT REVIEW OF HEMATOLOGY
(2021)
Article
Pharmacology & Pharmacy
Peng Xu, Raymond S. M. Wong, Wojciech Krzyzanski, Xiaoyu Yan
Summary: The hormone erythroferrone (ERFE) may serve as a predictive factor for erythropoiesis, with its dynamics showing a correlation with the erythropoietic effects of erythropoiesis-stimulating agents (ESAs) treatment. The early increase of ERFE levels appears to be a predictor of the hemoglobin response.
FRONTIERS IN PHARMACOLOGY
(2022)
Article
Urology & Nephrology
Jose Portoles, Maria Luisa Serrano Salazar, Olga Gonzalez Pena, Sandra Gallego Dominguez, Manel Vera Rivera, Jara Caro Espada, Alba Herreros Garcia, Maria Antonia Munar Vila, Maria Jose Espigares Huete, Haridian Sosa Barrios, Vicente Paraiso, Loreto Mariscal de Gante, Maria Auxiliadora Bajo, Antonia Gueorguieva Mijaylova, Elena Pascual Pajares, Nuria Areste Fosalba, Laura Espinel, Fernando Tornero Molina, Soledad Pizarro Sanchez, Mayra Ortega Diaz, Aleix Cases, Borja Quiroga
Summary: This study describes the current management of anemia in peritoneal dialysis (PD) patients, revealing that many prescriptions do not adhere to guidelines and hemoglobin targets are set too high. It also shows that iron therapy, especially intravenous, is underused. Improvement strategies such as structured dissemination of guidelines and clinical routes for intravenous iron administration to PD patients are needed. Specific studies on anemia in PD patients are necessary for personalized prescriptions and targets.
CLINICAL KIDNEY JOURNAL
(2023)
Review
Oncology
Chen Wang, David. A. A. Sallman
Summary: Lower risk myelodysplastic syndromes have a low risk of transformation into acute myeloid leukemia and are characterized by an indolent disease course. The revised International Prognostic Scoring System is used to identify these patients, with future use of its molecular version expected to change treatment strategies. Symptomatic control is the main goal of care, with anemia being the most common indication for disease-specific therapies. Erythropoiesis-stimulating agents and lenalidomide monotherapy are the standard upfront treatments, with other therapeutic options available after treatment failure.
CURRENT TREATMENT OPTIONS IN ONCOLOGY
(2023)
Article
Hematology
Sai Pan, De-Long Zhao, Ping Li, Xue-Feng Sun, Jian-Hui Zhou, Kang-Kang Song, Yong Wang, Li-Ning Miao, Zhao-Hui Ni, Hong-Li Lin, Fu-You Liu, Ying Li, Ya Ni He, Nian-Song Wang, Cai-Li Wang, Ai-Hua Zhang, Meng-Hua Chen, Xiao-Ping Yang, Yue-Yi Deng, Feng-Min Shao, Shu-Xia Fu, Jing-Ai Fang, Guang-Yan Cai, Xiang-Mei Chen
Summary: The study revealed that Chinese hemodialysis patients administered high dosages of erythropoiesis-stimulating agents had a significantly increased risk of mortality, and patients with a higher degree of resistance to these agents also faced a higher risk of all-cause mortality.
BLOOD PURIFICATION
(2022)
Article
Medicine, General & Internal
Jina Lim, Chung Jo Yu, Hoon Yu, Sang Jin Ha
Summary: The study showed that ESA can improve endothelial dysfunction, left ventricular hypertrophy, and left atrial volume in patients with non-dialysis CKD. This suggests that ESA may be considered as adjunctive therapy for reducing cardiovascular risk in these patients.
Article
Chemistry, Multidisciplinary
Tao Wang, Weiqi Zhang, Wanshu Chen, Jie Meng, Qinglin Hu, Jian Liu, Tao Wen, Bing Han, Haiyan Xu
Summary: In this study, a novel function of As4S4 nanoparticles in promoting terminal erythropoiesis in MDS patients was revealed. These nanoparticles triggered the phosphorylation of eIF2α, leading to an increase in global protein synthesis and mitigated oxidative stress in bone marrow cells. Overall, ee-As4S4 hold significant potentials for the effective differentiation of erythroblasts in refractory anemia.
Article
Cell Biology
Ana Cristina Goncalves, Raquel Alves, Ines Baldeiras, Joana Jorge, Barbara Marques, Artur Paiva, Barbara Oliveiros, Emilia Cortesao, Jose Manuel Nascimento Costa, Ana Bela Sarmento-Ribeiro
Summary: Oxidative stress is correlated with the development of various cancers, including myelodysplastic syndromes (MDS), and treatment resistance. This study found that lower-risk MDS patients who responded to ESA treatment had lower oxidative stress levels, with plasmatic peroxides levels being a good predictive biomarker for ESA treatment response.
FRONTIERS IN CELL AND DEVELOPMENTAL BIOLOGY
(2021)
Article
Oncology
Jingyong Tan, Sitong Du, Xueyan Zang, Kaiyue Ding, Yelena Ginzburg, Huiyong Chen
Summary: This study conducted a meta-analysis to evaluate the efficacy of iron supplementation combined with erythropoiesis-stimulating agents (ESA) in managing chemotherapy-induced anemia (CIA). The results demonstrated that the combination therapy was more effective in improving anemia compared to iron supplementation alone, regardless of whether oral or intravenous iron was used.
INTERNATIONAL JOURNAL OF CANCER
(2022)