Article
Hematology
Guillermo Garcia-Manero
Summary: The myelodysplastic syndromes (MDS) are a group of myeloid disorders characterized by low blood cell counts and increased risk of developing acute myelogenous leukemia (AML). Diagnosis is based on visual examination of bone marrow samples and additional tests such as karyotyping and flow cytometry. Prognosis can be determined using scoring systems like the Revised International Prognostic Scoring System (IPSS-R).
AMERICAN JOURNAL OF HEMATOLOGY
(2023)
Review
Medicine, Research & Experimental
Fabiana Kalina Marques, Adriano de Paula Sabino
Summary: Myelodysplastic neoplasms are malignancies originating from hematopoietic stem cells, characterized by ineffective hematopoiesis, dysplasia, and increased risk of transformation. Disease-related risk is assessed using a scoring system, and treatment options vary depending on risk factors. Despite progress in understanding molecular mechanisms, a curative treatment has not yet been approved.
BIOMEDICINE & PHARMACOTHERAPY
(2022)
Article
Hematology
Morie A. Gertz
Summary: WM is a lymphoma with IgM monoclonal protein, diagnosed by presence of clonal lymphoplasmacytic cells and MYD88 gene mutation. Predictive characteristics for outcomes include age, hemoglobin level, platelet count, beta(2) microglobulin, LDH, and monoclonal IgM concentrations. Treatment options include rituximab and bendamustine as preferred induction, with various other agents showing activity in relapsed cases of refractory disease.
AMERICAN JOURNAL OF HEMATOLOGY
(2021)
Review
Oncology
Talha Badar, Yazan F. Madanat, Amer M. Zeidan
Summary: The majority of lower-risk myelodysplastic syndromes/neoplasms patients present with anemia. Historically, they were treated with erythropoiesis-stimulating agents (ESA) but with modest responses. Recently, luspatercept has shown better responses compared with ESAs in treatment-naive patients and imetelstat in patients refractory to ESAs. Other evaluated novel compounds have not yet shown meaningful efficacy. More needs to be done to improve outcomes, and participation in clinical trials evaluating novel therapies should be encouraged. Improving prognostication and serial monitoring will help identify high-risk patients for appropriate management.
Review
Medicine, General & Internal
Mikkael A. Sekeres, Justin Taylor
Summary: MDS is a hematopoietic malignancy with bone marrow dysplasia and decreased blood cell numbers. Treatment varies based on disease characteristics, from supportive care to hypomethylating agents for high-risk patients. Hematopoietic cell transplantation is potentially curative for high-risk MDS.
JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION
(2022)
Review
Hematology
Zhuoer Xie, Evan C. Chen, Maximilian Stahl, Amer M. Zeidan
Summary: Accurate risk prognostication is crucial for managing myelodysplastic syndromes (MDS) due to their diverse clinical outcomes. Advances in MDS genomics have provided unprecedented insight into MDS pathobiology, leading to the development of molecular prognostic models. These models, such as the IPSS-M, improve risk prediction but also introduce complexity. This review discusses early MDS prognostic models, recent advancements in MDS genomics, and the challenges and opportunities at the molecular frontier.
Article
Oncology
Sofia Toribio-Castello, Sandra Castano, Angela Villaverde-Ramiro, Esperanza Such, Montserrat Arnan, Francesc Sole, Marina Diaz-Beya, Maria Diez-Campelo, Monica del Rey, Teresa Gonzalez, Jesus Maria Hernandez-Rivas
Summary: This study found that mutations in STAG2, SRSF2 and RUNX1 are independent prognostic factors in isolated +8 MDS, defining the disease's course in terms of overall survival and leukemic transformation. These findings help identify high-risk patients and open new management possibilities for MDS patients with isolated +8.
Review
Oncology
Jan Philipp Bewersdorf, Amer M. Zeidan
Summary: MDS and CMML are two distinct blood cancers with variable symptoms and risks. Management decisions should be individualized and based on validated risk stratification tools. Supportive care is the mainstay for lower-risk patients, while bone marrow transplant is the only curative option for higher-risk patients.
Article
Hematology
Animesh Pardanani
Summary: Systemic mastocytosis (SM) is a disease caused by the clonal proliferation of abnormal mast cells in extra-cutaneous organs, diagnosed primarily based on the presence of abnormal mast cells in the bone marrow. Risk stratification is important for treatment planning, with options including small-molecule inhibitors targeting mutant-KIT for advanced cases. Treatment goals involve symptom control and prevention of complications such as anaphylaxis in indolent cases, while cytoreductive therapy may be necessary for those with advanced disease.
AMERICAN JOURNAL OF HEMATOLOGY
(2021)
Article
Hematology
Mrinal M. Patnaik, Ayalew Tefferi
Summary: Ring sideroblasts (RS) are erythroid precursors with abnormal perinuclear mitochondrial iron accumulation, associated with myeloid neoplasms MDS-RS and MDS/MPN-RS-T; SF3B1 mutations are common in these diseases; treatment options include luspatercept for anemia and consideration of aspirin therapy in MDS/MPN-RS-T.
AMERICAN JOURNAL OF HEMATOLOGY
(2021)
Review
Oncology
Luis E. Aguirre, David A. Sallman, Richard Stone, Rami S. Komrokji
Summary: Risk stratification is crucial for treatment planning in myelodysplastic syndromes. The traditional International Prognostic Scoring System and its revised version rely on laboratory and cytogenetic data to estimate prognosis and guide treatment. However, advancements in DNA sequencing techniques and understanding of clonal dynamics have allowed the identification of molecular markers that were not accounted for in older models. The Molecular International Prognostic Scoring System integrates clinical, cytogenetic, and molecular data to create a more accurate prognostic tool.
Review
Biochemistry & Molecular Biology
Ekaterina Balaian, Manja Wobus, Martin Bornhaeuser, Triantafyllos Chavakis, Katja Sockel
Summary: MDS is a clonal stem cell disorder characterized by genetic alterations, inflammatory processes, and changes in cellular metabolism. These abnormalities lead to dysfunction of HSPCs and niche cells, promoting clone expansion and increasing the risk for AML.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2021)
Review
Medical Laboratory Technology
Akriti G. Jain, Ling Zhang, John M. Bennett, Rami Komrokji
Summary: In this review, the available literature on the presentation and prognosis of patients with MDS and concurrent BMF is discussed, highlighting the importance of considering BMF in treatment decisions. The prognostic impact of BMF should be taken into account when deciding on transplantation, especially for intermediate-risk patients.
ANNALS OF LABORATORY MEDICINE
(2022)
Article
Oncology
Aziz Nazha, Rami Komrokji, Manja Meggendorfer, Xuefei Jia, Nathan Radakovich, Jacob Shreve, C. Beau Hilton, Yasunubo Nagata, Betty K. Hamilton, Sudipto Mukherjee, Najla Al Ali, Wencke Walter, Stephan Hutter, Eric Padron, David Sallman, Teodora Kuzmanovic, Cassandra Kerr, Vera Adema, David P. Steensma, Amy Dezern, Gail Roboz, Guillermo Garcia-Manero, Harry Erba, Claudia Haferlach, Jaroslaw P. Maciejewski, Torsten Haferlach, Mikkael A. Sekeres
Summary: This study developed a personalized prediction model for MDS patients using machine learning techniques and incorporating clinical and genomic data, which showed superior performance in predicting survival and leukemia transformation probabilities compared to established models. The model was validated in external cohorts, demonstrating its potential for dynamic and accurate prognostic predictions at different time points in a patient's disease course.
JOURNAL OF CLINICAL ONCOLOGY
(2021)
Review
Oncology
Jonathan Weiss, John Reneau, Ryan A. A. Wilcox
Summary: The peripheral T-cell lymphomas (PTCL) are rare and heterogeneous, posing challenges for treatment. The most common PTCL subtype in North America, known as not otherwise specified (NOS), remains a significant unmet need. However, advancements in understanding the genetic landscape and ontogeny of PTCL subtypes classified as PTCL, NOS have important therapeutic implications, which will be discussed in this review.
FRONTIERS IN ONCOLOGY
(2023)