Article
Biochemistry & Molecular Biology
Xiaoqing Xie, Daria Frank, Pradeep Kumar Patnana, Judith Schuette, Yahya Al-Matary, Longlong Liu, Lanying Wei, Martin Dugas, Julian Varghese, Subbaiah Chary Nimmagadda, Cyrus Khandanpour
Summary: The study identified a therapeutic role for curcumin treatment in delaying the expansion of GFI1-36N or -KD, NUP98-HODXD13 leukemic cells and prolonging AML-free survival.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2022)
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)
Article
Oncology
David P. Steensma, Pierre Fenaux, Koen Van Eygen, Azra Raza, Valeria Santini, Ulrich Germing, Patricia Font, Maria Diez-Campelo, Sylvain Thepot, Edo Vellenga, Mrinal M. Patnaik, Jun Ho Jang, Helen Varsos, Jacqueline Bussolari, Esther Rose, Laurie Sherman, Libo Sun, Ying Wan, Souria Dougherty, Fei Huang, Faye Feller, Aleksandra Rizo, Uwe Platzbecker
Summary: Imetelstat treatment shows a meaningful and durable transfusion independence rate in heavily transfused LR-MDS patients, reducing malignant clone count and demonstrating disease modification activity. The most common adverse events are reversible cytopenias.
JOURNAL OF CLINICAL ONCOLOGY
(2021)
Article
Oncology
Jesus D. Gonzalez-Lugo, Suman Kambhampati, Abdulraheem Yacoub, William B. Donnellan, Jesus Berdeja, Prafulla Bhagat, Karen Fehn, Cassady Remy, Sakshi Jasra, Mohammed Kazemi, Kith Pradhan, Mimi Kim, Ioannis Mantzaris, R. Alejandro Sica, Nishi Shah, Mendel Goldfinger, Noah Kornblum, Kira Gritsman, Ira Braunschweig, Ulrich Steidl, Britta Will, Aditi Shastri, Amit Verma
Summary: The study aimed to evaluate the therapeutic effect of Eltrombopag and Lenalidomide in patients with low-risk or intermediate-risk myelo-dysplastic syndromes (MDS). The results showed that both ELT and LEN monotherapy, as well as combination therapy, can improve hematologic parameters in patients and are well tolerated.
CLINICAL CANCER RESEARCH
(2023)
Article
Oncology
Esther Natalie Oliva, Marta Riva, Pasquale Niscola, Valeria Santini, Massimo Breccia, Valentina Giai, Antonella Poloni, Andrea Patriarca, Elena Crisa, Isabella Capodanno, Prassede Salutari, Gianluigi Reda, Nicola Cascavilla, Dario Ferrero, Attilio Guarini, Giovanni Tripepi, Giuseppe Ianni, Emilio Russo, Andrea Castelli, Bruno Fattizzo, Germana Beltrami, Monica Bocchia, Alfredo Molteni, Pierre Fenaux, Ulrich Germing, Alessandra Ricco, Giuseppe A. Palumbo, Stefana Impera, Nicola Di Renzo, Flavia Rivellini, Francesco Buccisano, Aspasia Stamatoullas-Bastard, Anna Marina Liberati, Anna Candoni, Ilaria Maria Delfino, Maria Teresa Arcadi, Patrizia Cufari, Lorenzo Rizzo, Irene Bova, Maria Grazia D'Errigo, Gina Zini, Roberto Latagliata
Summary: This study demonstrates the efficacy and safety of eltrombopag in treating low-risk MDS patients with severe thrombocytopenia. Eltrombopag significantly increases the duration of platelet response and reduces the incidence of clinically significant bleeding.
JOURNAL OF CLINICAL ONCOLOGY
(2023)
Review
Cell Biology
Juan Jose Rodriguez-Sevilla, Vera Adema, Guillermo Garcia-Manero, Simona Colla
Summary: Myelodysplastic syndromes (MDSs) are a group of clonal hematopoietic stem cell disorders characterized by myeloid dysplasia, peripheral blood cytopenias, and increased risk of progression to acute myeloid leukemia (AML). HMA-based therapy is the standard treatment for MDS, but nearly 50% of patients do not respond. For MDS patients who have failed HMA therapy, clinical trials of experimental agents offer their only chance for improved outcomes. New therapies targeting molecular alterations, cell death regulators, signaling pathways, and immune regulatory proteins have been developed for these patients based on a better understanding of the molecular and biological mechanisms underlying MDS pathogenesis.
CELL REPORTS MEDICINE
(2023)
Review
Oncology
Uwe Platzbecker, Anne Sophie Kubasch, Collin Homer-Bouthiette, Thomas Prebet
Summary: Myelodysplastic syndromes (MDS) are a heterogeneous group of myeloid neoplasms characterized by ineffective hematopoiesis, variable cytopenias, and a risk of progression to acute myeloid leukemia. Current treatments for MDS lack a clear standard of care, and there is a need for new, more effective but tolerable strategies. Advances in molecular diagnostics have improved understanding of the genetic abnormalities driving MDS, necessitating complex personalized treatment approaches.
Review
Hematology
Caner Saygin, Hetty E. Carraway
Summary: Myelodysplastic syndromes (MDS) are hematopoietic disorders characterized by ineffective blood cell production, with treatment strategies focusing on improving quality of life for lower-risk MDS and extending survival for higher-risk MDS. Several promising drugs are currently being investigated for MDS treatment.
Article
Oncology
Ernest Nadal, Mansoor Saleh, Santiago Ponce Aix, Maria Ochoa-de-Olza, Sandip Pravin Patel, Scott Antonia, Yumin Zhao, Ivelina Gueorguieva, Michael Man, Shawn T. Estrem, Jiangang Liu, Emin Avsar, Wen Hong Lin, Karim A. Benhadji, Leena Gandhi, Susan C. Guba, Inmaculada Ales Diaz
Summary: In this study, the efficacy of targeting tumor immune suppression using galunisertib with nivolumab was explored in patients with advanced refractory solid tumors and recurrent/refractory NSCLC. The results demonstrated preliminary efficacy and good tolerability of the combination therapy in NSCLC patients.
Review
Pharmacology & Pharmacy
Connie Kang, Yahiya Y. Syed
Summary: Luspatercept, a novel erythroid maturation agent, has shown significant reduction in red blood cell transfusion requirements for patients with MDS or beta-thalassaemia in pivotal phase III trials. It has a generally manageable tolerability profile with adverse events of special interest including thromboembolic events, hypertension, and bone pain. As a result, Luspatercept is an emerging treatment option for adults with transfusion-dependent anaemia due to MDS or beta-thalassaemia.
Article
Hematology
Eugene Brailovski, Qing Li, Ning Liu, Brian Leber, Dina Khalaf, Mitchell Sabloff, Grace Christou, Karen Yee, Lisa Chodirker, Anne Parmentier, Mohammed Siddiqui, Alexandre Mamedov, Liying Zhang, Ying Liu, Craig C. Earle, Matthew C. Cheung, Nicole Mittmann, Rena J. Buckstein, Lee Mozessohn
Summary: Observational studies suggest that statins, metformin, and DPP4i may have an anti-neoplastic effect in patients with myelodysplastic syndromes (MDS), while sulfonylureas may have no benefit or even be detrimental. However, larger prospective studies are needed to confirm these findings.
ANNALS OF HEMATOLOGY
(2022)
Article
Oncology
Nikolay Kalitin, Galina Dudina, Natalia Kostritsa, Anastasiya Sivirinova, Aida Karamysheva
Summary: The expression levels of VEGF and VEGFR genes were found to be elevated in patients with MDS compared to healthy volunteers. Analysis of gene expression in MDS patients stratified by risk groups showed increased expression of VEGF-A, VEGFR1, and VEGFR2 with higher risk. Patients with high levels of VEGFR1 expression had significantly different survival times. Thus, VEGF-A/VEGFR1 expression may be important for risk evaluation in MDS patients.
Article
Medicine, General & Internal
Calogero Vetro, Valeria Di Giacomo, Donato Mannina, Silvana Magrin, Antonio Mule, Maria Enza Mitra, Sergio Siragusa, Andrea Duminuco, Bruno Garibaldi, Maria Cristina Emanuela Vadala, Francesco Di Raimondo, Giuseppe A. Palumbo
Summary: This prospective observational study confirms the efficacy of erythropoietin zeta in the treatment of low-risk myelodysplastic syndrome. The results demonstrate a significant improvement in hemoglobin levels with the use of erythropoietin zeta.
JOURNAL OF CLINICAL MEDICINE
(2022)
Article
Oncology
Tamanna Haque, Felix Lopez Cadenas, Blanca Xicoy, Ana Alfonso-Pierola, Uwe Platzbecker, Irit Avivi, Andrew M. Brunner, Joeerg Chromik, Daniel Morillo, Manish R. Patel, Jose Falantes, Heather A. Leitch, Ulrich Germing, Meir Preis, Laurie Lenox, Josh Lauring, Regina J. Brown, Anna Kalota, Jaydeep Mehta, Friederike Pastore, Junchen Gu, Pankaj Mistry, David Valcarcel
Summary: This study evaluated the efficacy of JNJ-64619178 in patients with lower-risk MDS and found that the drug was well tolerated. However, no significant improvement in clinical outcomes was observed, indicating the need for further research.
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)
Letter
Hematology
Dai Chihara, Luuk Gras, Nienke Zinger, Nicolaus Kroeger, Jiri Mayer, Jakob Passweg, Regis Peffault de Latour, Jenny Byrne, William Krueger, Jan-Paul Bohn, Uwe Platzbecker, Igor Wolfgang Blau, Francesca Bonifazi, Grzegorz Helbig, Andrew McDonald, Martin Mistrik, Mohamad Mohty, Ron Ram, Jaime Sanz, Carlos Vallejo Llamas, Robert J. Kreitman, Patrick J. Hayden, Donal McLornan, Olivier Tournilhac, Michel van Gelder, Ibrahim Yakoub-Agha
Article
Oncology
Thomas Walter Georgi, Lars Kurch, Georg-Nikolaus Franke, Madlen Jentzsch, Sebastian Schwind, Carmen Perez-Fernandez, Naima Petermann, Maximilian Merz, Klaus Metzeler, Gudrun Borte, Sandra Hoffmann, Marco Herling, Timm Denecke, Regine Kluge, Osama Sabri, Uwe Platzbecker, Vladan Vucinic
Summary: Assessing the value of FDG-PET/CT in predicting patient outcomes after CAR-T cell therapy, it was found that PET-1 results were significantly associated with patient prognosis. Patients with CMR had better outcomes, while those without CMR had poorer outcomes.
JOURNAL OF CANCER RESEARCH AND CLINICAL ONCOLOGY
(2023)
Article
Oncology
Raphael Itzykson, Valeria Santini, Sylvain Thepot, Lionel Ades, Cendrine Chaffaut, Aristoteles Giagounidis, Margot Morabito, Nathalie Droin, Michael Luebbert, Rosa Sapena, Stanislas Nimubona, Jean Goasguen, Eric Wattel, Gina Zini, Jose Miguel Torregrosa Diaz, Ulrich Germing, Anna Maria Pelizzari, Sophie Park, Nadja Jaekel, Georgia Metzgeroth, Francesco Onida, Robert Navarro, Andrea Patriarca, Aspasia Stamatoullas, Katharina Goetze, Martin Puttrich, Sandra Mossuto, Eric Solary, Silke Gloaguen, Sylvie Chevret, Fatiha Chermat, Uwe Platzbecker, Pierre Fenaux
Summary: This study compared two treatment methods for chronic myelomonocytic leukemia and found that using DAC significantly reduced the risk of CMML transforming into acute myelomonocytic leukemia.
JOURNAL OF CLINICAL ONCOLOGY
(2023)
Article
Hematology
Saifu Hailu, Samuel Kinde, Michael Cross, Aster Tsegaye, Tsehayneh Kelemu, Daniel Seifu, Dawit Alemayehu, Azeb Tarekegn, Gutema Jabessa, Desalegn Abeje, Markos Abebe, Abdulaziz Sherif, Fisihatsion Tadesse, Uwe Platzbecker, Rawleigh Howe, Amha Gebremedhin
Summary: In low- and middle-income countries, including Ethiopia, access to standard diagnostic and prognostic tools for chronic myeloid leukemia (CML) is limited. This study found that multiplex PCR (mpx-PCR) can serve as a screening tool with high sensitivity and specificity, helping to rule out relapse and non-adherence to medications, thus reducing treatment risks.
ANNALS OF HEMATOLOGY
(2023)
Review
Hematology
Jan Philipp Bewersdorf, Zhuoer Xie, Rafael Bejar, Uma Borate, Jacqueline Boultwood, Andrew M. Brunner, Rena Buckstein, Hetty E. Carraway, Jane E. Churpek, Naval G. Daver, Matteo Giovanni Della Porta, Amy E. DeZern, Pierre Fenaux, Maria E. Figueroa, Steven D. Gore, Elizabeth A. Griffiths, Stephanie Halene, Robert P. Hasserjian, Christopher S. Hourigan, Tae Kon Kim, Rami Komrokji, Vijay K. Kuchroo, Alan F. List, Sanam Loghavi, Ravindra Majeti, Olatoyosi Odenike, Mrinal M. Patnaik, Uwe Platzbecker, Gail J. Roboz, David A. Sallman, Valeria Santini, Guillermo Sanz, Mikkael A. Sekeres, Maximilian Stahl, Daniel T. Starczynowski, David P. Steensma, Justin Taylor, Omar Abdel-Wahab, Mina L. Xu, Michael R. Savona, Andrew H. Wei, Amer M. Zeidan
Summary: Biological events in the pathogenesis of MDS are being characterized and translated into therapeutic strategies. Updates from the iwMDS provide advances in understanding the genetic landscape of MDS, as well as the development of novel therapies targeting specific molecular alterations and the innate immune system. However, more work is needed to develop individualized care for MDS patients.
Article
Biophysics
Arnon Nagler, Myriam Labopin, Ryszard Swoboda, Alexander Kulagin, Andrea Velardi, Jaime Sanz, Helene Labussiere-Wallet, Victoria Potter, Juergen Kuball, Simona Sica, Elena Parovichnikova, Wolfgang Bethge, Natacha Maillard, Uwe Platzbecker, Friedrich Stoelzel, Fabio Ciceri, Mohamad Mohty
Summary: This study evaluated the outcome of second transplantation (HSCT2) for the treatment of primary graft failure (pGF) in 243 patients with acute leukemia. The median age was 44.8 years. 73.7% of patients achieved engraftment after HSCT2. The 5-year nonrelapse mortality was 51.6%, relapse incidence was 18.8%, leukemia-free survival was 29.6%, and overall survival was 30.7%.
BONE MARROW TRANSPLANTATION
(2023)
Article
Oncology
Rama Al Hamed, Maud Ngoya, Jacques-Emmanuel Galimard, Henrik Sengeloev, Tobias Gedde-Dahl, Aleksandr Kulagin, Uwe Platzbecker, Ibrahim Yakoub-Agha, Jenny L. Byrne, Thomas Valerius, Gerard Socie, Nicolaus Kroeger, Didier Blaise, Ali Bazarbachi, Jaime Sanz, Fabio Ciceri, Arnon Nagler, Mohamad Mohty
Summary: This retrospective study investigates the impact of time on allogeneic hematopoietic cell transplantation (allo-HCT) outcomes for patients with acute myeloid leukemia (AML) in second complete remission (CR2). The results show that outcomes of allo-HCT in CR2 have significantly improved over time, with the most favorable outcomes achieved with a matched sibling donor (MSD).
Article
Cell Biology
Syed A. Mian, Celine Philippe, Eleni Maniati, Pantelitsa Protopapa, Tiffany Bergot, Marion Piganeau, Travis Nemkov, Doriana Di Bella, Valle Morales, Andrew J. Finch, Angelo D'Alessandro, Katiuscia Bianchi, Jun Wang, Paolo Gallipoli, Shahram Kordasti, Anne Sophie Kubasch, Michael Cross, Uwe Platzbecker, Daniel H. Wiseman, Dominique Bonnet, Delphine G. Bernard, John G. Gribben, Kevin Rouault-Pierre
Summary: Patients with myelodysplastic syndrome and ring sideroblasts (MDS-RS) suffer from symptomatic anemia due to ineffective erythropoiesis caused by SF3B1 mutations. Mis-splicing of COASY induced by these mutations affects heme biosynthesis and erythropoiesis. Supplementation with COASY substrate may serve as a potential treatment for anemia in MDS-RS patients.
SCIENCE TRANSLATIONAL MEDICINE
(2023)
Article
Oncology
Julia-Annabell Georgi, Sebastian Stasik, Jan-Niklas Eckardt, Sven Zukunft, Marita Hartwig, Christoph Roellig, Jan Moritz Middeke, Uta Oelschlaegel, Utz Krug, Tim Sauer, Sebastian Scholl, Andreas H. Hochhaus, Tim Bruemmendorf, Ralph Naumann, Bjoern Steffen, Hermann Einsele, Markus Schaich, Andreas Burchert, Andreas Neubauer, Kerstin Schaefer-Eckart, Christoph W. Schliemann, Stefan Krause, Mathias Haenel, Richard Noppeney, Ulrich D. Kaiser, Claudia Baldus, Martin Kaufmann, Carsten Mueller-Tidow, Uwe E. Platzbecker, Wolfgang Berdel, Hubert Serve, Gerhard Ehninger, Martin Bornhaeuser, Johannes Schetelig, Frank Kroschinsky, Christian Thiede
Summary: Tandem-duplication mutations of the UBTF gene are associated with pediatric AML patients and inferior outcomes. By screening adult AML and MDS patients, UBTF-TDs were found to be rare but more common in younger patients and associated with MDS-related morphology and lower hemoglobin and platelet levels. These mutations were also associated with specific co-mutations and were stable over the disease course, making them a novel class-defining lesion in both pediatric and younger adult AML patients.
BLOOD CANCER JOURNAL
(2023)
Article
Hematology
Rami S. Komrokji, Luis E. Aguirre, Najla H. Al Ali, Onyee Chan, Zhuoer Xie, Andrew Kuykendall, Kendra Sweet, Jeffrey E. Lancet, Eric Padron, David A. Sallman
Review
Hematology
Luise Fischer, Nora Grieb, Uwe Platzbecker, Vladan Vucinic, Maximilian Merz
Summary: The introduction of CAR T cells has revolutionized the treatment of RRMM. Two CAR T cell products are approved for use in the US and EU, and phase III trials for earlier lines of therapy have been published recently. Despite the success, there are still uncertainties regarding the optimal use of CAR T cells in multiple myeloma. This review discusses the available data and ongoing developments to optimize CAR T cell usage in this disease.
EUROPEAN JOURNAL OF HAEMATOLOGY
(2023)
Article
Hematology
Sowmya Gowdavally, Chrysanthi Tsamadou, Uwe Platzbecker, Elisa Sala, Thomas Valerius, Stefan Klein, Nicolaus Kroeger, Gerald Wulf, Hermann Einsele, Lorenz Thurner, Kerstin Schaefer-Eckart, Sebastian Freitag, Jochen Casper, Mareike Duerholt, Martin Kaufmann, Bernd Hertenstein, Mark Ringhoffer, Sandra Schmeller, Christine Neuchel, Immanuel Rode, Elisa Maria Amann, Anita Richter, Hubert Schrezenmeier, Joannis Mytilineos, Daniel Fuerst
Summary: Previous studies have shown that the presence of activating killer cell immunoglobulin-like receptor (KIR) genes is associated with lower susceptibility to hematologic malignancies in humans. In a large transplantation cohort, researchers investigated the association of KIR2DS4 polymorphisms with disease and the impact of KIR-AA genotype donor KIR2DS4 polymorphisms on hematopoietic stem cell transplantation (HSCT) outcomes. The study found that utilizing grafts from AA/KIR1D donors resulted in improved survival outcomes in 10/10 HLA-matched patients with lymphoid disease.
TRANSPLANTATION AND CELLULAR THERAPY
(2023)
Article
Medicine, Research & Experimental
Jan-Niklas Eckardt, Christoph Roellig, Klaus Metzeler, Peter Heisig, Sebastian Stasik, Julia-Annabell Georgi, Frank Kroschinsky, Friedrich Stoelzel, Uwe Platzbecker, Karsten Spiekermann, Utz Krug, Jan Braess, Dennis Goerlich, Cristina Sauerland, Bernhard Woermann, Tobias Herold, Wolfgang Hiddemann, Carsten Mueller-Tidow, Hubert Serve, Claudia D. Baldus, Kerstin Schaefer-Eckart, Martin Kaufmann, Stefan W. Krause, Mathias Haenel, Wolfgang E. Berdel, Christoph Schliemann, Jiri Mayer, Maher Hanoun, Johannes Schetelig, Karsten Wendt, Martin Bornhaeuser, Christian Thiede, Jan Moritz Middeke
Summary: Researchers use unsupervised machine learning to separate patients with acute myeloid leukemia into four distinct risk subgroups based on genetics, laboratory values, and clinical characteristics. These subgroups show differences in treatment response and patient survival, and the findings are validated in another dataset. This approach can improve outcome prediction and treatment decisions for leukemia patients.
COMMUNICATIONS MEDICINE
(2023)
Article
Medicine, General & Internal
Srdan Verstovsek, Aaron T. Gerds, Alessandro M. Vannucchi, Haifa Kathrin Al-Ali, David Lavie, Andrew T. Kuykendall, Sebastian Grosicki, Alessandra Iurlo, Yeow Tee Goh, Mihaela C. Lazaroiu, Miklos Egyed, Maria Laura Fox, Donal McLornan, Andrew Perkins, Sung -Soo Yoon, Vikas Gupta, Jean -Jacques Kiladjian, Nikki Granacher, Sung-Eun Lee, Luminita Ocroteala, Francesco Passamonti, Claire N. Harrison, Barbara J. Klencke, Sunhee Ro, Rafe Donahue, Jun Kawashima, Ruben Mesa
Summary: This study aimed to compare the clinical effects of momelotinib and danazol in patients with intermediate or high-risk myelofibrosis. The results showed that momelotinib can significantly improve myelofibrosis-associated symptoms, anemia measures, and spleen response compared to danazol, with favorable safety.