Article
Hematology
Allison Mo, Erica Wood, Jake Shortt, Erin Hu, Zoe McQuilten
Summary: This study investigated the burden of thrombocytopenia, supportive care practices, bleeding complications, and predictors of bleeding in MDS patients at a large Australian hospital network. The results showed that thrombocytopenia is common in MDS patients, and prophylactic platelet transfusions are commonly used for severe cases. Despite the use of platelet transfusions and/or the drug tranexamic acid (TXA), 11% of patients developed major bleeding.
EUROPEAN JOURNAL OF HAEMATOLOGY
(2023)
Article
Medicine, General & Internal
Alessia Campagna, Daniela De Benedittis, Luana Fianchi, Emilia Scalzulli, Lorenzo Rizzo, Pasquale Niscola, Anna Lina Piccioni, Ambra Di Veroli, Stefano Mancini, Nicoletta Villiva, Tiziano Martini, Sara Mohamed, Ida Carmosino, Marianna Criscuolo, Susanna Fenu, Maria Antonietta Aloe Spiriti, Francesco Buccisano, Marco Mancini, Agostino Tafuri, Massimo Breccia, Antonella Poloni, Roberto Latagliata
Summary: Patients with isolated del20q in myelodysplastic syndromes show distinct clinical and prognostic features, including older age, predominantly male, low bone marrow blast percentage, and lower platelet count. In terms of treatment, the majority receive erythropoiesis-stimulating agents, while a small portion progress to acute myeloid leukemia. The median overall survival is 60.6 months, with a 5-year cumulative survival rate of 55.9%.
JOURNAL OF CLINICAL MEDICINE
(2022)
Article
Geriatrics & Gerontology
Silvia Ferrari, Daniela Regazzo, Elisabetta Omenetto, Carla Scaroni, Gianpietro Semenzato, Fabrizio Fabris, Fabrizio Vianello
Summary: Aging is associated with a higher risk of developing malignant diseases, and myelodysplastic syndromes in older subjects are influenced by genetic mutations in genes like RUNX1 and ANKRD26, potentially leading to thrombocytopenia and impaired platelet production.
AGING CLINICAL AND EXPERIMENTAL RESEARCH
(2021)
Article
Oncology
Hong Wang, Jiaqian Qi, Xueqian Li, Tiantian Chu, Huiying Qiu, Chengcheng Fu, Xiaowen Tang, Changgeng Ruan, Depei Wu, Yue Han
Summary: This study conducted a real-world study on 303 patients with myelodysplastic syndrome (MDS) who underwent allogeneic hematopoietic stem cell transplantation (HSCT). The results showed that prolonged isolated thrombocytopenia (PT) was associated with worse overall survival (OS) in MDS patients. Risk factors for PT, such as grade II-IV acute graft-versus-host disease (aGVHD), extensive chronic graft-versus-host disease (GVHD), hemorrhagic cystitis, and CMV activation, were identified. These findings have important implications for patient management and treatment strategies.
FRONTIERS IN ONCOLOGY
(2022)
Article
Medicine, General & Internal
Huan Li, Fang Hu, Robert Peter Gale, Mikkael A. Sekeres, Yang Liang
Summary: Myelodysplastic syndromes (MDS) are a group of blood cancers characterized by dysregulated hematopoiesis and risk of transformation to acute myeloid leukemia. Prognostic systems can predict survival in MDS patients. Treatment goals differ for low-risk and high-risk MDS, with the aim to improve quality of life and prolong survival, respectively. Hematopoietic cell transplantation can cure MDS, but it is not widely used.
NATURE REVIEWS DISEASE PRIMERS
(2022)
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)
Article
Hematology
Alexander Sternberg, Rebecca Boucher, Helen Chantal Coulthard, Manoj Raghavan, Dominic Culligan, Aimee Jackson, Catherine Cargo, Mike Dennis, Marlen Metzner, Jennifer O'Sullivan, Rachel Moore, David Bowen, Paresh Vyas
Summary: Combination therapy with eltrombopag and azacitidine showed promising efficacy and safety in treating high-risk myelodysplastic syndromes, although caution is needed due to potential adverse effects.
BRITISH JOURNAL OF HAEMATOLOGY
(2022)
Review
Oncology
Caner Saygin, Lucy A. Godley
Summary: Myelodysplastic syndrome (MDS) is a bone marrow disease characterized by the inability to produce normal numbers of blood cells. Different types of MDS are driven by distinct biological pathways, with advancements in treatment strategies based on molecular understanding. The recognition of precursor conditions may allow for strategies to delay or prevent MDS development.
Review
Oncology
Yan Jiang, Su-Jun Gao, Benoit Soubise, Nathalie Douet-Guilbert, Zi-Ling Liu, Marie-Berengere Troadec
Summary: Gene variants, particularly TP53 mutations, play a crucial role in the prognosis of myelodysplastic syndromes (MDS), being associated with higher risk categories, resistance to traditional therapies, rapid leukemic transformation, and poor outcomes. Current prognosis classification systems for MDS do not consider gene variants, but the impact is significant on the clinical heterogeneity and prognosis of the disease.
Letter
Oncology
Pauline Durand, Valerie Pottier, Charles Mesguich, Frederic Debordeaux, Estibaliz Lazaro, Jean-Francois Viallard, Etienne Riviere
Summary: This study analyzed the utility of indium-111 platelet scintigraphy (IPS) to better understand the mechanisms and guide treatment of thrombocytopenia in patients with myelodysplastic syndromes (MDS). The results showed that IPS can be a useful tool to identify and treat the increased bleeding risk caused by thrombocytopenia in MDS patients.
EXPERIMENTAL HEMATOLOGY & ONCOLOGY
(2023)
Article
Oncology
Guillermo Garcia-Manero, Valeria Santini, Antonio Almeida, Uwe Platzbecker, Anna Jonasova, Lewis R. Silverman, Jose Falantes, Gianluigi Reda, Francesco Buccisano, Pierre Fenaux, Rena Buckstein, Maria Diez Campelo, Stephen Larsen, David Valcarcel, Paresh Vyas, Valentina Giai, Esther Natalie Oliva, Jake Shortt, Dietger Niederwieser, Moshe Mittelman, Luana Fianchi, Ignazia La Torre, Jianhua Zhong, Eric Laille, Daniel Lopes de Menezes, Barry Skikne, C. L. Beach, Aristoteles Giagounidis
Summary: CC-486 significantly improved RBC-TI rate and induced durable bilineage improvements in patients with LR-MDS and high-risk disease features. However, more early deaths occurred in the CC-486 arm, most related to infections in patients with significant pretreatment neutropenia. Further evaluation of CC-486 in MDS is needed.
JOURNAL OF CLINICAL ONCOLOGY
(2021)
Review
Medicine, General & Internal
Kathrin Nachtkamp, Guido Kobbe, Norbert Gattermann, Ulrich Germing
Summary: Myelodysplastic syndromes (MDS) are malignant diseases arising from hematopoietic stem cells, with an incidence of 4 cases per 100,000 persons per year. They are usually diagnosed when evaluating cytopenia, and the median survival time is three years. This review summarizes the latest research on the diagnosis and treatment of MDS.
DEUTSCHES ARZTEBLATT INTERNATIONAL
(2023)
Article
Medical Laboratory Technology
Juliana Guarente, Christopher Tormey
Summary: Even with the discovery of new treatment modalities, transfusion remains an important aspect of supportive care for patients with MDS. Future studies are awaited to provide an evidence-based approach to RBC and platelet transfusion support for MDS patients, while balancing the benefits and negative effects of transfusion.
CLINICS IN LABORATORY MEDICINE
(2023)
Article
Hematology
Thibault Comont, Mathieu Meunier, Amina Cherait, Clemence Santana, Thomas Cluzeau, Bohrane Slama, Kamel Laribi, Jean-Thomas Giraud, Sophie Dimicoli, Ana Berceanu, Lenaig Le Clech, Pascale Cony-Makhoul, Berangere Gruson, Jose Torregrosa, Laurence Sanhes, Vincent Jachiet, Marie-Agnes Azerad, Ahmad Al Jijakli, Emmanuel Gyan, Clement Gaudin, Jonathan Broner, Claire Guerveno, Thierry Guillaume, Pr Lionel Ades, Odile Beyne-Rauzy, Pierre Fenaux
Summary: This study demonstrates the effectiveness and tolerability of eltrombopag in patients with MDS and CMML, showing significant platelet responses with a relatively long duration. Despite some patients experiencing thrombotic events, the majority responded well to the medication with good treatment tolerance.
BRITISH JOURNAL OF HAEMATOLOGY
(2021)
Article
Multidisciplinary Sciences
Nanfang Huang, Yang Song, Wenhui Shi, Juan Guo, Lingyun Wu, Zheng Zhang, Qi He, Xiao Li, Feng Xu
Summary: DHX9 overexpression in myelodysplastic syndromes (MDS) is associated with poor prognosis and high risk of acute myeloid leukemia (AML) transformation. DHX9 is essential for malignant proliferation of leukemia cells. Suppression of DHX9 increases cell apoptosis and sensitizes cells to chemotherapy. Additionally, DHX9 knockdown inactivates PI3K-AKT and ATR-Chk1 signaling, promotes R-loop accumulation, and leads to R-loop-mediated DNA damage.
Article
Hematology
Filipe Martins, Michael Kruszewski, Ilaria Scarpelli, Jacqueline Schoumans, Olivier Spertini, Michael Luebbert, Sabine Blum
Summary: Myelodysplastic syndromes (MDS) have a variable probability of transforming into acute leukemia, with rare cases of acute lymphoblastic leukemia reported in MDS patients. These atypical cases highlight the possibility of major phenotypic switches occurring during disease progression.
ANNALS OF HEMATOLOGY
(2021)
Article
Hematology
Gerwin Huls, Dana A. Chitu, Thomas Pabst, Saskia K. Klein, Georg Stussi, Laimonas Griskevicius, Peter J. M. Valk, Jacqueline Cloos, Arjan A. van de Loosdrecht, Dimitri Breems, Danielle van Lammeren-Venema, Isabelle van Zeventer, Rinske Boersma, Mojca Jongen-Lavrencic, Martin Fehr, Mels Hoogendoorn, Markus G. Manz, Maaike Sohne, Rien van Marwijk Kooy, Dries Deeren, Marjolein W. M. van der Poel, Marie Cecile Legdeur, Lidwine Tick, Yves Chalandon, Emanuele Ammatuna, Sabine Blum, Bob Lowenberg, Gert J. Ossenkoppele
Review
Hematology
Natacha Dewarrat, Mathilde Gavillet, Anne Angelillo-Scherrer, Olaia Naveiras, Francesco Grandoni, Dimitrios A. Tsakiris, Lorenzo Alberio, Sabine Blum
Summary: Acquired haemophilia A cases in the postpartum setting account for 1%-5% of cases, with characteristics including young age, primigravidae, diagnosis typically 60 days after delivery, and majority having factor VIII <1% and FVIII inhibitor. Treatment often involves fresh frozen plasma, factor concentrate, and/or bypassing agents, with a high percentage of patients requiring immunosuppressive therapy. Subsequent pregnancies have a risk of haemophilia recurrence.
Article
Endocrinology & Metabolism
Josefine Tratwal, Shanti Rojas-Sutterlin, Charles Bataclan, Sabine Blum, Olaia Naveiras
Summary: Bone marrow adipocytes play important roles in the regulation of the hematopoietic microenvironment, with their heterogeneity determining their regulatory capacities in different contexts.
BEST PRACTICE & RESEARCH CLINICAL ENDOCRINOLOGY & METABOLISM
(2021)
Letter
Hematology
Amandine Segot, Gregoire Stalder, Laurence de Leval, Francoise Solly, Jacqueline Schoumans, Valentin Basset, Sabine Blum, Olivier Spertini
AMERICAN JOURNAL OF HEMATOLOGY
(2022)
Editorial Material
Hematology
Natacha Dewarrat, Fabian Bovey, Julie Kaiser, Sabine Blum
INTERNATIONAL JOURNAL OF LABORATORY HEMATOLOGY
(2022)
Article
Multidisciplinary Sciences
Matthew R. Robinson, Marion Patxot, Milos Stojanov, Sabine Blum, David Baud
Summary: Analyzing blood cell count changes during pregnancy, significant differences among women in how these values change were found, with trimester-specific associations with postpartum hemorrhage. These changes have an important role in shaping pregnancy outcomes and identifying women at increased risk of hemorrhage.
SCIENTIFIC REPORTS
(2021)
Letter
Oncology
Sophie Voruz, Sabine Blum, Laurence de Leval, Jacqueline Schoumans, Francoise Solly, Olivier Spertini
Summary: Patients with relapsed/refractory B-ALL often have poor prognosis, requiring a complete molecular response for long-term survival, with immunotherapies being the main effective treatments. However, treatment options are limited when lymphoblasts do not express immunotherapeutic targets.
BIOMARKER RESEARCH
(2021)
Letter
Hematology
Marissa Sjoblom, Haithem Chtioui, Montserrat Fraga, Gregoire Stalder, Francesco Grandoni, Sabine Blum
ANNALS OF HEMATOLOGY
(2022)
Review
Medical Laboratory Technology
Lucine Christe, Luis Veloza, Louis Gros, Bettina Bisig, Sabine Blum, Ekkehard Hewer, Laurence de Leval
Summary: We report a case of EBV-positive large B-cell lymphoma presenting as a pericardial effusion in a patient with chronic myeloid leukemia treated with dasatinib. Despite aggressive cytomorphology, the patient achieved complete remission after R-CHOP treatment.
DIAGNOSTIC CYTOPATHOLOGY
(2022)
Letter
Medicine, Research & Experimental
Gerasimos Tsilimidos, Sabine Blum, Alessandro Aliotta, Alexandre Dumusc, Lorenzo Alberio
CURRENT RESEARCH IN TRANSLATIONAL MEDICINE
(2022)
Review
Oncology
Sabine Blum, Gerasimos Tsilimidos, Helena Bresser, Michael Luebbert
Summary: Myelodysplasic syndromes (MDS) mainly occur in the elderly population, and most patients are too old or frail for intensive treatment. The current gold standard is treatment with hypomethylating agents, but the combination of hypomethylating agents with venetoclax has become the new gold standard for elderly and frail patients unable to undergo intensive treatment.
INTERNATIONAL JOURNAL OF CANCER
(2023)
Article
Hematology
Vasiliki Papadopoulou, Jacqueline Schoumans, Ilaria Scarpelli, Sabine Blum
Summary: The gene encoding for transcription factor ETV6 frequently undergoes mutations in hematologic neoplasms. The role of ETV6 in normal hematopoiesis is unclear, but its loss of function may contribute to tumorigenesis. Deletions at the ETV6-locus (12p13) are rare but recurrent in myeloid neoplasms, while ETV6 translocations are even rarer but seem to have defining consequences on phenotype. The mechanisms underlying these lesions are still unknown.
ACTA HAEMATOLOGICA
(2023)
Article
Hematology
Vasiliki Papadopoulou, Jacqueline Schoumans, Valentin Basset, Francoise Solly, Jerome Pasquier, Sabine Blum, Olivier Spertini
Summary: Objective IDH1/2 mutations, intervening in epigenetic procedures, are frequently encountered in acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS). Knowledge of the genetics, immunophenotypes, and mutational kinetics of IDH1/2-mutated AML can contribute to the understanding of AML clonal architecture and inform therapeutics and monitoring.
Meeting Abstract
Biophysics
Sabine Blum, Yves Chalandon, Myriam Labopin, Jurgen Finke, Tobias Gedde-Dahl, Tarek Ben Othman, Jan J. Cornelissen, Pavel Jindra, Helene Labussiere-Wallet, Matthew Collin, Stig Lenhoff, Guido Kobbe, Dolores Caballero, Arnon Nagler, Mohamad Mohty
BONE MARROW TRANSPLANTATION
(2021)