Article
Hematology
Justin Loke, Vinit Upasani, Charlotte Gaskell, Sonia Fox, Rachel Fletcher, Catherine Thomas, Louise Hopkins, Anita Kumari, Tina Tang, Emily Yafai, Rebecca Boucher, Victoria Homer, Arpad Toth, Y. L. Tracey Chan, Katie Randall, Tom Rider, Jenny O'Nions, Victoria Drew, Arvind Pillai, Moez Dungarwalla, Duncan Murray, Anjum Khan, Farooq Wandroo, Sally Moore, Pramila Krishnamurthy, Ya-Wen Jessica Huang, Steve Knapper, Jenny Byrne, Rui Zhao, Charles Craddock, Helen Parry, Paul Moss, Simon J. Stanworth, David M. Lowe
Summary: Limited data exist on the efficacy of COVID-19 vaccination in patients with AML/MDS-EB2. A prospective study found detectable antibodies against SARS-COV-2 spike antigen in all post-vaccine samples. T-cell reactivity to SARS-COV-2 spike protein was only seen in a subset of patients, and disease response and age were predictors of poor T-cell response.
BRITISH JOURNAL OF HAEMATOLOGY
(2023)
Review
Oncology
Sargam Kapoor, Grace Champion, Aparna Basu, Anu Mariampillai, Matthew J. Olnes
Summary: Myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML) are hematologic malignancies arising from the bone marrow with poor prognosis. Recent advancements in immune therapies, including immune suppressive therapy and novel treatments like monoclonal antibodies and cellular therapeutics, have shown promise in treating these diseases.
Article
Medicine, General & Internal
Courtney D. Dinardo, Harry P. Erba, Sylvie D. Freeman, Andrew H. Wei
Summary: Progress in acute myeloid leukaemia treatment has seen significant improvements in scientific understanding, prognostication tools, risk assessments, and incorporation of measurable residual disease into risk assessments. Recent updates in classification and recommendations by organizations such as WHO, ICC, and European LeukemiaNet have provided enhanced guidance for prognostic stratification and treatment response assessment. There have also been advancements in treatment options, leading to improved outcomes for both newly diagnosed and relapsed patients.
Article
Medicine, Research & Experimental
Na Man, Gloria Mas, Daniel L. Karl, Jun Sun, Fan Liu, Qin Yang, Miguel Torres-Martin, Hidehiro Itonaga, Concepcion Martinez, Shi Chen, Ye Xu, Stephanie Duffort, Pierre-Jacques Hamard, Chuan Chen, Beth E. Zucconi, Luisa Cimmino, Feng-Chun Yang, Mingjiang Xu, Philip A. Cole, Maria E. Figueroa, Stephen D. Nimer
Summary: This study identified a tumor suppressor role of the acetyltransferase p300 in MDS, with loss of p300 enhancing HSPC proliferation and self-renewal, ultimately increasing leukemogenicity. Mechanistically, loss of p300 altered gene expression and promoted leukemia development, while activating p300 activity countered these effects. This suggests a potential therapeutic application of p300 activators in treating MDS with TET2 mutations.
Review
Medicine, General & Internal
Petter S. Woll, Tetsuichi Yoshizato, Eva Hellstrom-Lindberg, Thoas Fioretos, Benjamin L. Ebert, Sten Eirik W. Jacobsen
Summary: The genetic architecture of cancer has been extensively studied, leading to the identification of new therapeutic targets and the development of promising treatment strategies. However, disease progression, relapse, and treatment-related mortality remain challenging in certain types of cancer such as myelodysplastic syndromes and acute myeloid leukemia. The persistence of rare leukemic stem cells following therapy underscores the resistance mechanisms of these cells and their role in relapse. To improve treatment strategies, there is a need to better understand and characterize the stem cells associated with these diseases at the cellular, molecular, and genetic levels.
JOURNAL OF INTERNAL MEDICINE
(2022)
Review
Biochemistry & Molecular Biology
Thomas Cluzeau, Michael Loschi, Pierre Fenaux, Rami Komrokji, David A. Sallman
Summary: This article reviews the progress in targeting TP53 mutated myelodysplastic syndromes and acute myeloid leukemia, focusing on the use of active agents and future treatment directions. It emphasizes the importance of understanding the clinical characteristics of the disease and the impact of TP53 mutant burden on patient clinical trajectory.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2021)
Review
Cell Biology
Yasmin Abaza, Amer M. Zeidan
Summary: Immune checkpoint inhibitors have had a significant impact on the treatment of solid tumors, but there has been limited progress in myeloid malignancies. The low mutational burden of acute myeloid leukemia is a potential reason for the lack of response to T-cell harnessing ICIs. Targeting agents, such as magrolimab and sabatolimab, in combination with other drugs, have shown promising activity in early clinical trials.
Review
Hematology
Shai Shimony, Richard M. Stone, Maximilian Stahl
Summary: Venetoclax has been approved as a combination therapy drug for newly diagnosed AML patients, and has shown promising initial results in combination therapies for MDS and AML patients. However, further research is needed to determine the exact role of each combination therapy and its long-term effects on patient outcomes.
CURRENT OPINION IN HEMATOLOGY
(2022)
Review
Oncology
Michael Loschi, Pierre Fenaux, Thomas Cluzeau
Summary: TP53-mutated acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) are characterized by poor prognosis. In this review, we analyze the prognosis of these diseases, focusing on the extent of TP53 mutation status and its correlation with cytogenetic complexity. We discuss the potential improvement in outcome based on recent results obtained with new drugs (especially eprenetapopt and magrolimab). The impact of allogeneic hematopoietic stem cell transplantation (aHSCT) and post-transplantation treatment is also emphasized.
Article
Oncology
Chantal Saberian, Noha Abdel-Wahab, Ala Abudayyeh, Hind Rafei, Jacinth Joseph, Gabriela Rondon, Laura Whited, Stephen Gruschkus, Faisal Fa'ak, May Daher, Cristina Knape, Houssein Safa, Mahran Shoukier, Maria E. Suarez-Almazor, Megan Marcotulli, Kaysia Ludford, Alison M. Gulbis, Marina Konopleva, Maro Ohanian, Farhad Ravandi, Guillermo Garcia-Manero, Betul Oran, Uday R. Popat, Rotesh Mehta, Amin M. Alousi, Naval Daver, Richard Champlin, Adi Diab, Gheath Al-Atrash
Summary: This study analyzed the association between GVHD prophylaxis and the frequency of GVHD in patients who received immune checkpoint inhibitors (ICIs) after allogeneic hematopoietic stem cell transplantation. The results indicated that post-transplantation cyclophosphamide (PTCy) was associated with a significantly reduced risk of acute GVHD in this group of patients.
JOURNAL FOR IMMUNOTHERAPY OF CANCER
(2021)
Article
Oncology
Lu Wang, Qi Zhang, Li Ye, Xingnong Ye, Wenli Yang, Hua Zhang, Xinping Zhou, Yanling Ren, Liya Ma, Xiang Zhang, Chen Mei, Gaixiang Xu, Kongfei Li, Yingwan Luo, Lingxu Jiang, Peipei Lin, Shuanghong Zhu, Wei Lang, Yuxia Wang, Chuying Shen, Yueyuan Han, Xiaozhen Liu, Haiyang Yang, Chenxi Lu, Jie Sun, Jie Jin, Hongyan Tong
Summary: Combining all-trans retinoic acid (ATRA) with decitabine (DAC) can improve the treatment outcomes of patients with higher-risk myelodysplastic syndromes (HR-MDS) and elderly acute myeloid leukaemia (AML) by inhibiting Nrf2 activation and antioxidative response induced by DAC, leading to ROS accumulation and cytotoxicity.
BRITISH JOURNAL OF CANCER
(2023)
Review
Biochemistry & Molecular Biology
Caroline M. Putnam, Lahari Kondeti, Meredith B. A. Kesler, Melinda E. Varney
Summary: Modulating the immune system has resulted in the development of novel therapeutics for various diseases, including myeloid malignancies. Dysregulated immune responses, particularly in innate immune and inflammatory pathways, play a crucial role in the pathogenesis of MDS and AML. In addition to using immunotherapeutic interventions, mitigating dysregulation of the immune system remains a priority in treating these malignancies.
BIOCHEMISTRY AND CELL BIOLOGY
(2023)
Article
Medicine, Research & Experimental
Kristen E. Schratz, Valeriya Gaysinskaya, Zoe L. Cosner, Emily A. DeBoy, Zhimin Xiang, Laura Kasch-Semenza, Liliana Florea, Pali D. Shah, Mary Armanios
Summary: The study revealed that patients with germline telomere maintenance defects exhibit diverse somatic adaptive mutations, which may alleviate the telomere crisis that promotes transformation to MDS/AML.
JOURNAL OF CLINICAL INVESTIGATION
(2021)
Review
Oncology
Xingcheng Yang, Ling Ma, Xiaoying Zhang, Liang Huang, Jia Wei
Summary: Myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML) are clonal hematopoietic stem cell diseases that arise from the bone marrow. Dysregulated immune microenvironment, including the molecules PD-1 and PD-L1, play important roles in the pathogenesis of MDS and AML. However, clinical trials using PD-1/PD-L1 inhibitors have reported mixed responses in patients with these diseases.
EXPERIMENTAL HEMATOLOGY & ONCOLOGY
(2022)
Review
Oncology
Kah Keng Wong, Rosline Hassan, Nik Soriani Yaacob
Summary: Decitabine and guadecitabine are hypomethylating agents that enhance anti-tumor immunity in AML and MDS patients by upregulating cancer/testis antigens, stimulating T cells, and inducing immune checkpoint molecules expression. Combining these agents with cancer vaccines or immune checkpoint blockade therapy shows promising efficacy in treating relapsed/refractory AML and high-risk MDS cases.
FRONTIERS IN ONCOLOGY
(2021)
Article
Biophysics
Arnon Nagler, Frederic Baron, Myriam Labopin, Emmanuel Polge, Jordi Esteve, Ali Bazarbachi, Eolia Brissot, Gesine Bug, Fabio Ciceri, Sebastian Giebel, Maria H. Gilleece, Norbert-Claude Gorin, Francesco Lanza, Zinaida Peric, Annalisa Ruggeri, Jaime Sanz, Bipin N. Savani, Christoph Schmid, Roni Shouval, Alexandros Spyridonidis, Jurjen Versluis, Mohamad Mohty
Summary: The survey revealed that detectable measurable residual disease (MRD) is a key prognostic factor in patients with acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML). Most centers assessed MRD using techniques such as PCR and flow cytometry, with assessments conducted every 2-3 months for up to 2 years post-transplant. This survey marks the first step towards including MRD status as a routine registry parameter for acute leukemia.
BONE MARROW TRANSPLANTATION
(2021)
Article
Biophysics
Eolia Brissot, Myriam Labopin, Frederic Baron, Ali Bazarbachi, Gesine Bug, Fabio Ciceri, Jordi Esteve, Sebastian Giebel, Maria H. Gilleece, Norbert-Claude Gorin, Francesco Lanza, Zinaida Peric, Annalisa Ruggeri, Jaime Sanz, Bipin N. Savani, Christoph Schmid, Roni Shouval, Alexandros Spyridonidis, Jurjen Versluis, Arnon Nagler, Mohamad Mohty
BONE MARROW TRANSPLANTATION
(2021)
Article
Oncology
Eva M. Weissinger, Jochen Metzger, Michael Schleuning, Christoph Schmid, Diethelm Messinger, Gernot Beutel, Eva-Maria Wagner-Drouet, Johannes Schetelig, Herrad Baurmann, Andreas Rank, Friedrich Stolzl, Kerstin Schaefer-Eckart, Karin Westphal, Wolfgang Bethge, S. von Harsdorf, Donald W. Bunjes, Daniela Heidenreich, Stefan Klein, Ernst Holler, Hans H. Kreipe, Danny Jonigk, Irina Tueruechanow, Julia Raad, Armin Papkalla, Heiko von der Leyen, Lothar Hambach, Iyas Hamwi, Steve Ehrlich, Jurgen Krauter, Michael Stadler, Arnold Ganser
Summary: The study investigated the effectiveness of preemptive prednisolone treatment in patients with acute graft-versus-host disease, and the results showed no significant difference in the incidence or severity of acute GvHD, and did not increase the relapse rate.
Article
Hematology
Esther Schuler, Eva-Maria Wagner-Drouet, Salem Ajib, Gesine Bug, Martina Crysandt, Sabine Dressler, Andreas Hausmann, Daniela Heidenreich, Klaus Hirschbuehl, Matthias Hoepting, Edgar Jost, Jennifer Kaivers, Stefan Klein, Michael Koldehoff, Lambros Kordelas, Oliver Kriege, Lutz P. Mueller, Christina Rautenberg, Judith Schaffrath, Christoph Schmid, Daniel Wolff, Rainer Haas, Martin Bornhaeuser, Thomas Schroeder, Guido Kobbe
Summary: The retrospective study evaluated the efficacy of Venetoclax combined with hypomethylating agents in treating relapse of myeloid malignancies after alloHSCT. Results showed significant risk of neutropenia and infections, with substantial toxicity. Patients who received the treatment as first salvage therapy or for molecular relapse derived the greatest benefit.
ANNALS OF HEMATOLOGY
(2021)
Article
Biophysics
Klaus Hirschbuehl, Myriam Labopin, Mohamed Houhou, Ludovic Gabellier, Helene Labussiere-Wallet, Bruno Lioure, Dietrich Beelen, Jan Cornelissen, Gerald Wulf, Pavel Jindra, Herve Tilly, Jakob Passweg, Riita Niittyvuopio, Gesine Bug, Christoph Schmid, Arnon Nagler, Sebastian Giebel, Mohamad Mohty
Summary: The study evaluated the efficacy of second- and third-generation TKI in 140 patients with Ph + ALL, showing a response rate of 71% and an overall survival rate of 49% at 2 years and 33% at 5 years. TKI were found to be important compounds for managing active Ph + ALL post alloSCT, with comparable toxicity to pretransplant use.
BONE MARROW TRANSPLANTATION
(2021)
Review
Oncology
Christoph Schmid, Juergen Kuball, Gesine Bug
JOURNAL OF CLINICAL ONCOLOGY
(2021)
Article
Nutrition & Dietetics
R. Toenges, H. Greinix, A. Lawitschka, J. Halter, A. Baumgartner, A. Simon, J. Arends, P. Jaeger, M. Middeke, I. Hilgendorf, S. Klein, E. M. Wagner-Drouet, C. Schmid, G. Bug, D. Wolff
Summary: The study investigates the clinical practice of nutritional treatment after alloHSCT, revealing variability in nutritional approaches and indicating a need for more high-quality data to support evidence-based nutrition for patients.
CLINICAL NUTRITION
(2021)
Article
Oncology
Jordi Esteve, Sebastian Giebel, Myriam Labopin, Tomasz Czerw, Depei Wu, Liisa Volin, Gerard Socie, Ibrahim Yakoub-Agha, Johan Maertens, Jan J. Cornelissen, Arnaud Pigneux, Avichai Shimoni, Rainer Schwerdtfeger, Helene Labussiere-Wallet, Nigel Russell, Anton Schattenberg, Patrice Chevallier, Vladimir Koza, Robin Foa, Christoph Schmid, Zinaida Peric, Mohamad Mohty, Arnon Nagler
Summary: This study analyzed the outcomes of patients with t(4;11) BCP-ALL treated with alloHSCT, finding that a negative MRD status pre-transplant was the strongest beneficial factor. Patients with t(4;11) BCP-ALL had inferior outcomes compared to those with NK BCP-ALL when MRD was detectable.
Editorial Material
Hematology
Christoph Schmid
Summary: The study reported successful generation and safe clinical application of mLSTs derived from healthy stem cell donors, showing low incidence of GVHD after transfusion of mLSTs and long-term remission and clinical responses in patients.
Article
Biophysics
Mohamed A. Kharfan-Dabaja, Myriam Labopin, Ali Bazarbachi, Fabio Ciceri, Juergen Finke, Benedetto Bruno, Martin Bornhaeuser, Tobias Gedde-Dahl, Helene Labussiere-Wallet, Riitta Niittyvuopio, Thomas Valerius, Emanuele Angelucci, Arne Brecht, Dolores Caballero, Juergen Kuball, Victoria Potter, Christoph Schmid, Johanna Tischer, Tsila Zuckerman, Fabio Benedetti, Didier Blaise, Jose Luis Diez-Martin, Jaime Sanz, Annalisa Ruggeri, Eolia Brissot, Bipin N. Savani, Sebastian Giebel, Arnon Nagler, Mohamad Mohty
Summary: The optimal donor choice for a second allo-HCT in relapsed acute lymphoblastic leukemia remains unclear. Comparing outcomes between using HLA-matched unrelated donors (MUD) and haploidentical donors shows no major difference in overall survival for ALL patients needing a second allo-HCT. Peripheral blood stem cells are the most common cell source, with a higher proportion of bone marrow cells used in the haploidentical group.
BONE MARROW TRANSPLANTATION
(2021)
Article
Biophysics
Johanna Waidhauser, Myriam Labopin, Jordi Esteve, Nicolaus Kroger, Jan Cornelissen, Tobias Gedde-Dahl, Gwendolyn Van Gorkom, Jurgen Finke, Montserrat Rovira, Nicolaas Schaap, Eefke Petersen, Dietrich Beelen, Donald Bunjes, Bipin Savani, Christoph Schmid, Arnon Nagler, Mohamad Mohty
Summary: This study analyzed the prognostic value of RUNX1 mutation in acute myeloid leukemia patients after allogeneic stem cell transplantation, and found that in the first complete remission, the presence of RUNX1 mutation did not significantly impact survival rates.
BONE MARROW TRANSPLANTATION
(2021)
Article
Biophysics
Christoph Schmid, Myriam Labopin, Nicolaas Schaap, Hendrik Veelken, Arne Brecht, Michael Stadler, Juergen Finke, Frederic Baron, Matthew Collin, Gesine Bug, Per Ljungman, Didier Blaise, Johanna Tischer, Adrian Bloor, Aleksander Kulagin, Sebastian Giebel, Norbert-Claude Gorin, Jordi Esteve, Fabio Ciceri, Bipin Savani, Arnon Nagler, Mohamad Mohty
Summary: This study analyzed 318 acute leukemia patients who received DLI after alloSCT, showing that preemptive DLI was effective in reducing MRD and increasing chimerism, thus improving survival rates for patients.
BONE MARROW TRANSPLANTATION
(2022)
Article
Oncology
Peter-Martin Bruch, Sascha Dietrich, Herve Finel, Ariane Boumendil, Hildegard Greinix, Thomas Heinicke, Wolfgang Bethge, Dietrich Beelen, Christoph Schmid, Hans Martin, Luca Castagna, Christof Scheid, Kerstin Schaefer-Eckart, Joerg Bittenbring, Juergen Finke, Henrik Sengeloev, Mael Heiblig, Jan Cornelissen, Patrice Chevallier, Mohamad Mohty, Stephen Robinson, Silvia Montoto, Peter Dreger
Summary: This retrospective study analyzed the outcomes of 162 adult patients with BPDCN who underwent a first HCT. The study found that MAC (especially TBI-based) significantly improved the prognosis of alloHCT recipients, and autoHCT could be considered for patients who are not eligible for MAC.
Article
Hematology
Andreas Rank, Christophe Peczynski, Myriam Labopin, Matthias Stelljes, Celestine Simand, Grzegorz Helbig, Jurgen Finke, Stella Santarone, Johanna Tischer, Andrzej Lange, Martin Mistrik, Mohamed Houhou, Christoph Schmid, Arnon Nagler, Mohamad Mohty
Summary: This retrospective analysis evaluated 45 patients with acute myeloid or lymphoblastic leukemia who received a third allogeneic stem cell transplantation (alloSCT3). After the third transplantation, 38 patients engrafted and 26 achieved remission, but the overall survival and progression-free survival rates at 1 year were low, indicating poor outcomes. Factors that may improve patient outcomes include donor changes, selection of an unrelated donor for alloSCT3, and transplantation in more recent years.
TRANSPLANTATION AND CELLULAR THERAPY
(2021)
Article
Hematology
Roni Shouval, Joshua A. Fein, Myriam Labopin, Christina Cho, Ali Bazarbachi, Frederic Baron, Gesine Bug, Fabio Ciceri, Selim Corbacioglu, Jacques-Emmanuel Galimard, Sebastian Glebel, Maria H. Gilleece, Sergio Giralt, Ann Jakubowski, Silvia Montoto, Richard J. O'Reilly, Esperanza B. Papadopoulos, Zinaida Peric, Annalisa Ruggeri, Jaime Sanz, Craig S. Sauter, Bipin N. Savani, Christoph Schmid, Alexandros Spyridonidis, Roni Tamari, Jurjen Versluis, Ibrahim Yakoub-Agha, Miguel Angel Perales, Mohamad Mohty, Arnon Nagler
Summary: The study developed a contemporary disease-risk stratification system that categorized patients into five risk groups based on histology and remission status. The system was able to predict overall survival after allogeneic hematopoietic stem-cell transplantation and may serve as a benchmark for future studies on heterogeneous cohorts.
LANCET HAEMATOLOGY
(2021)