Article
Hematology
Amy E. DeZern, Marianna Zahurak, Heather J. Symons, Kenneth R. Cooke, Carol Ann Huff, Tania Jain, Lode J. Swinnen, Philip H. Imus, Nina D. Wagner-Johnston, Richard F. Ambinder, Mark Levis, Leo Luznik, Javier Bolanos-Meade, Ephraim J. Fuchs, Richard J. Jones, Robert A. Brodsky
Summary: Severe aplastic anemia (SAA) is a marrow failure disorder with high morbidity and mortality. This study found that reduced-intensity conditioning HLA-haploidentical bone marrow transplantation (BMT) with posttransplantation cyclophosphamide (PTCy)-based graft-versus-host (GVHD) prophylaxis resulted in 100% overall survival and minimal GVHD in patients with SAA. This approach not only avoids the adverse effects of immunosuppressive therapy, but also expands access to BMT across all populations.
Review
Biochemistry & Molecular Biology
Xing-An Wang, Ju-Pi Li, Kang-Hsi Wu, Shun-Fa Yang, Yu-Hua Chao
Summary: Aplastic anemia is a rare but life-threatening disease characterized by low blood cell count and reduced cellularity in the bone marrow. Mesenchymal stem cell dysfunction is believed to play a role in the development of the disease. This review provides a comprehensive understanding of the involvement of mesenchymal stem cells in acquired idiopathic aplastic anemia and discusses their clinical application. The pathophysiology of the disease, properties of mesenchymal stem cells, and results of mesenchymal stem cell therapy in animal models are also described. The authors anticipate that more patients with aplastic anemia can benefit from mesenchymal stem cell therapy in the future.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2023)
Review
Oncology
Waseem Nasr, Marie-Dominique Filippi
Summary: Bone marrow failure syndromes (BMFS) are life-threatening disorders characterized by the absence of one or more hematopoietic lineages in the peripheral blood. Inflammation and abnormal mitochondrial functions are common features of BMF and MDS. This review focuses on investigating mitochondria-mediated signaling in acquired and inherited BMF.
FRONTIERS IN ONCOLOGY
(2022)
Review
Oncology
Momen Nassani, Riad El Fakih, Jakob Passweg, Simone Cesaro, Hazzaa Alzahrani, Ali Alahmari, Carmem Bonfim, Raheel Iftikhar, Amal Albeihany, Constantijn Halkes, Syed Osman Ahmed, Carlo Dufour, Mahmoud Aljurf
Summary: Bone marrow failure syndromes are a diverse group of diseases that can now be better classified and treated with the help of advanced diagnostic tools and sequencing techniques. Androgens, a historical group of drugs, have been used for decades to stimulate hematopoiesis and treat bone marrow failure. However, with the availability of more effective treatment options, androgens are now less commonly used. Nonetheless, they can still be considered for bone marrow failure patients who cannot receive standard therapy. This article reviews the use of androgens in bone marrow failure patients and provides recommendations for their optimal use in the current therapeutic landscape.
FRONTIERS IN ONCOLOGY
(2023)
Article
Hematology
Michael R. Grunwald, Mei-Jie Zhang, Hany Elmariah, Mariam H. Johnson, Andrew St Martin, Asad Bashey, Minoo Battiwalla, Christopher N. Bredeson, Edward Copelan, Corey S. Cutler, Biju R. George, Vikas Gupta, Christopher Kanakry, Rohtesh S. Mehta, Filippo Milano, Alberto Mussetti, Ryotaro Nakamura, Taiga Nishihori, Wael Saber, Melhem Solh, Daniel J. Weisdorf, Mary Eapen
Summary: This study compared outcomes of HLA-haploidentical relative and HLA-matched unrelated donor hematopoietic cell transplantation in patients with myelodysplastic syndrome. While the haploidentical relative group had higher relapse rates and lower disease-free survival rates, overall survival rates did not differ between donor types. This was due to lower rates of GVHD in haploidentical relative transplants, although mortality associated with chronic GVHD was higher in the unrelated donor group.
Review
Hematology
Phillip Scheinberg
Summary: The advances in the management of aplastic anaemia (AA) have been successful, with better understanding of the mechanisms underlying the reduction of hematopoietic stem cells (HSCs) and improvements in supportive care. Efforts to increase immunosuppression potency and stimulate HSCs have not achieved better outcomes, but the use of thrombopoietin receptor agonists (Tpo-RA) has been effective in stimulating early HSCs despite high endogenous Tpo levels.
BRITISH JOURNAL OF HAEMATOLOGY
(2021)
Article
Pediatrics
Yufei Zhao, Wenrui Yang, Xin Zhao, Xiangrong Hu, Jing Hu, Xu Liu, Jianping Li, Lei Ye, Youzhen Xiong, Yang Yang, Baohang Zhang, Xiaoxia Li, Xiawan Yang, Yimeng Shi, Guangxin Peng, Yuan Li, Huihui Fan, Kang Zhou, Liping Jing, Li Zhang, Fengkui Zhang
Summary: Adding EPAG to standard IST as the first-line treatment for children with acquired SAA improved the rapidity of hematological response and the complete response rate but did not improve the overall response or event-free survival rates.
FRONTIERS IN PEDIATRICS
(2023)
Article
Oncology
Stefan O. Ciurea, Piyanuch Kongtim, Doris Soebbing, Prashant Trikha, Gregory Behbehani, Gabriela Rondon, Amanda Olson, Qaiser Bashir, Alison M. Gulbis, Kaur Indreshpal, Katayoun Rezvani, Elizabeth J. Shpall, Roland Bassett, Kai Cao, Andrew St Martin, Stevenu Devine, Mary Horowitz, Marcelo Pasquini, Dean A. Lee, Richard E. Champlin
Summary: In this phase I/II clinical trial, high doses of mb-IL21 ex vivo expanded donor-derived NK cells were administered to 25 patients with myeloid malignancies undergoing haploidentical stem-cell transplantation. The results showed a significant decrease in the relapse rate and improved disease-free survival compared to the control group, with a particular benefit in patients without donor-specific anti-HLA antibodies. The administration of expanded NK cells post-transplantation was safe and led to NK cell-dominant immune reconstitution, preserved T-cell reconstitution, and better clinical outcomes.
Editorial Material
Hematology
Susan E. Prockop
Summary: In this article, the authors demonstrate that a standardized treatment approach combined with novel composite end points can establish a standard of care for boys with this rare nonmalignant disorder even in the absence of prospective trials.
Article
Biophysics
Helena Kim, Rafia Ali, Samuel Short, Sarah Kaunfer, Shobana Krishnamurthy, Lavanya Durai, Osman Yilmam, Tushar Shenoy, Audrey E. Monson, Charlotte Thomas, Isabel Park, Dylan Martini, Richard Newcomb, Roman M. Shapiro, Robert J. Soiffer, Zachariah DeFilipp, Rebecca M. Baron, Shruti Gupta, Meghan E. Sise, David E. Leaf
Summary: This study aimed to investigate the incidence, risk factors, and 90-day mortality associated with acute kidney injury treated with kidney replacement therapy (AKI-KRT) in critically ill adult patients who underwent allogeneic hematopoietic stem cell transplantation (allo-HSCT). The study found that lower baseline eGFR, veno-occlusive disease, thrombotic microangiopathy, admission to ICU within 90 days post-transplant, invasive mechanical ventilation, total bilirubin >= 5.0 mg/dl, and arterial pH <7.40 on ICU admission were all associated with a higher risk of AKI-KRT. The short-term mortality rate was exceptionally high among AKI-KRT patients, highlighting the importance of multidisciplinary discussions before initiating kidney replacement therapy.
BONE MARROW TRANSPLANTATION
(2023)
Article
Multidisciplinary Sciences
Taeyoung Kong, Hye Sun Lee, So Young Jeon, Je Sung You, Jong Wook Lee, Hyun Soo Chung, Sung Phil Chung
Summary: Postpartum hemorrhage (PPH) is a major risk for maternal mortality and morbidity due to difficulty in accurately measuring blood loss. The delta neutrophil index (DNI) and shock index (SI) were found to be independent predictors of massive transfusion (MT) requirement in PPH patients, and their combination significantly improved the risk assessment accuracy compared to using DNI or SI alone.
Article
Microbiology
Shiela McCollam, James S. Lewis, Joseph Bubalo, Amber Diaz
Summary: This study aimed to evaluate the efficacy and safety of 300-mg once-monthly intravenous (IV) pentamidine prophylaxis in 702 adult allogeneic hematopoietic stem cell transplant (HSCT) patients. The results showed that IV pentamidine administration effectively prevented Pneumocystis jirovecii pneumonia (PJP), but breakthrough Nocardia and Toxoplasma infections were observed in a certain percentage of patients.
ANTIMICROBIAL AGENTS AND CHEMOTHERAPY
(2022)
Article
Hematology
Antonio Jimenez Jimenez, Krishna Komanduri, Samantha Brown, Trent Wang, Denise Pereira, Mark Goodman, Amer Beitinjaneh, Lazaros Lekakis, Stephanie Chinapen, Sean Devlin, Doris Ponce, Craig Sauter, Miguel-Angel Perales, Brian C. Shaffer
Summary: This study compared the graft-versus-host disease-free, relapse-free survival (GRFS) in patients who underwent allogeneic hematopoietic cell transplantation from an HLA-mismatched unrelated donor using different prophylactic measures. The results showed that the use of posttransplant cyclophosphamide resulted in superior GRFS and overall survival after one year.
Article
Hematology
Sharon Noy-Lotan, Tanya Krasnov, Orly Dgany, Marta Jeison, Asaf D. Yanir, Oded Gilad, Helen Toledano, Shlomit Barzilai-Birenboim, Joanne Yacobovich, Shai Izraeli, Hannah Tamary, Orna Steinberg-Shemer
Summary: Detection of somatic mutations is important for verifying the diagnosis of myelodysplastic syndrome and has revealed a unique spectrum of mutations in pediatric patients, especially in the context of underlying syndromes. Routine somatic panels can help establish the diagnosis and optimal timing for hematopoietic stem cell transplantation, prior to leukemia development, in both inherited and acquired cytopenias.
BRITISH JOURNAL OF HAEMATOLOGY
(2021)
Article
Medicine, General & Internal
Valentina Giudice, Antonio M. Risitano, Carmine Selleri
Summary: Acquired bone marrow failure (BMF) syndromes are considered immune-mediated disorders, with immune cells possibly playing a role in the development of BMF through chronic antigen exposure or cross-reactivity between viral particles and cellular components. The involvement of immunodominant T-cell clones associated with infectious diseases suggests a potential autoimmune response triggered by pathogens or a global immune dysregulation during BMF. These intracellular pathogens may not only be passengers in the development of marrow failure, but also play a central role in initiating an autoimmune response against hematopoietic stem cells.
FRONTIERS IN MEDICINE
(2021)
Editorial Material
Hematology
Mathieu Leclerc, Slim Fourati, Dehbia Menouche, Dominique Challine, Sebastien Maury
LANCET HAEMATOLOGY
(2021)
Letter
Medicine, General & Internal
Rabah Redjoul, Anne Le Bouter, Florence Beckerich, Slim Fourati, Sebastien Maury
Article
Hematology
Howard S. Oster, Simon Crouch, Alexandra Smith, Ge Yu, Bander Abu Shrkihe, Shoham Baruch, Albert Kolomansky, Jonathan Ben-Ezra, Shachar Naor, Pierre Fenaux, Argiris Symeonidis, Reinhard Stauder, Jaroslav Cermak, Guillermo Sanz, Eva Hellstrom-Lindberg, Luca Malcovati, Saskia Langemeijer, Ulrich Germing, Mette Skov Holm, Krzysztof Madry, Agnes Guerci-Bresler, Dominic Culligan, Laurence Sanhes, Juliet Mills, Ioannis Kotsianidis, Corine van Marrewijk, David Bowen, Theo de Witte, Moshe Mittelman
Summary: The study proposes a noninvasive web-based application utilizing gradient boosted models to help exclude or diagnose myelodysplastic syndrome (MDS). By utilizing demographic, clinical, and laboratory variables, the application can accurately predict or exclude MDS in 86% of patients with unexplained anemia. The developed application shows stability and accuracy in predicting MDS and provides physicians with a convenient noninvasive diagnostic tool.
Article
Biophysics
Junya Kanda, Hiromi Hayashi, Annalisa Ruggeri, Fumihiko Kimura, Fernanda Volt, Satoshi Takahashi, Shinichi Kako, Karina Tozatto-Maio, Masamitsu Yanada, Guillermo Sanz, Naoyuki Uchida, Emanuele Angelucci, Seiko Kato, Mohamad Mohty, Edouard Forcade, Masatsugu Tanaka, Jorge Sierra, Takanori Ohta, Riccardo Saccardi, Takahiro Fukuda, Tatsuo Ichinohe, Takafumi Kimura, Vanderson Rocha, Shinichiro Okamoto, Arnon Nagler, Yoshiko Atsuta, Eliane Gluckman
Summary: The study evaluated the impact of GVHD on UCBT outcomes, finding that grade II acute GVHD had a positive impact on survival in the Japanese cohort but a negative impact in the European cohort. Grade III-IV acute GVHD had a negative impact on survival regardless of registries. Additionally, a positive impact of limited chronic GVHD on OS was observed only in the Japanese cohort.
BONE MARROW TRANSPLANTATION
(2022)
Letter
Oncology
Gail J. Roboz, Karen Yee, Amit Verma, Gautam Borthakur, Adolfo de la Fuente Burguera, Guillermo Sanz, Helai P. Mohammad, Ryan G. Kruger, Natalie O. Karpinich, Geraldine Ferron-Brady, Andre Acusta, Heather Del Buono, Therese Collingwood, Marc Ballas, Arindam Dhar, Andrew H. Wei
LEUKEMIA & LYMPHOMA
(2022)
Article
Immunology
Astrid Beerlage, Karoline Leuzinger, Luca Valore, Roby Mathew, Till Junker, Beatrice Drexler, Jakob R. Passweg, Hans H. Hirsch, Joerg Halter
Summary: Patients after allogeneic stem cell transplantation generally have a good response to mRNA vaccines against SARS-CoV-2, but there is still a proportion of patients with inadequate vaccination response, which can be improved after a third vaccination despite immunosuppressive therapy.
TRANSPLANT INFECTIOUS DISEASE
(2022)
Article
Medicine, General & Internal
Mariam Ibanez, Esperanza Such, Alessandro Liquori, Gayane Avestisyan, Rafael Andreu, Ana Vicente, Maria Jose Macian, Mari Carmen Melendez, Mireya Morote-Faubel, Pedro Asensi, Maria Pilar Lloret, Isidro Jarque, Isabel Picon, Alejandro Pacios, Eva Donato, Carmen Mas-Ochoa, Carmen Alonso, Carolina Canigral, Amparo Sempere, Samuel Romero, Marta Santiago, Guillermo F. Sanz, Javier de la Rubia, Leonor Senent, Irene Luna
Summary: This study introduces a comprehensive targeted sequencing solution for detecting common genetic variants in CLL-related genes with high accuracy, specificity, and sensitivity. The strategy can identify somatic variants and copy number variations in genes with predictive and prognostic value, and provide evidence about subclonal events. This Next-Generation Sequencing (NGS) capture-based target assay represents an improvement over current approaches for defining molecular prognostic and predictive variables in CLL patients.
Letter
Hematology
Maximilian Stahl, Omar Abdel-Wahab, Andrew H. Wei, Michael R. Savona, Mina L. Xu, Zhuoer Xie, Justin Taylor, Daniel Starczynowski, Guillermo F. Sanz, David A. Sallman, Valeria Santini, Gail J. Roboz, Mrinal M. Patnaik, Eric Padron, Olatoyosi Odenike, Aziz Nazha, Stephen D. Nimer, Ravindra Majeti, Richard F. Little, Steven Gore, Alan F. List, Vijay Kutchroo, Rami S. Komrokji, Tae Kon Kim, Nina Kim, Christopher S. Hourigan, Robert P. Hasserjian, Stephanie Halene, Elizabeth A. Griffiths, Peter L. Greenberg, Maria Figueroa, Pierre Fenaux, Fabio Efficace, Amy E. DeZern, Matteo G. Della Porta, Naval G. Daver, Jane E. Churpek, Hetty E. Carraway, Andrew M. Brunner, Uma Borate, John M. Bennett, Rafael Bejar, Jacqueline Boultwood, Sanam Loghavi, Jan Philipp Bewersdorf, Uwe Platzbecker, David P. Steensma, Mikkael A. Sekeres, Rena J. Buckstein, Amer M. Zeidan
Article
Hematology
Till Junker, Thomas Volken, Gregor Stehle, Beatrice Drexler, Laura Infanti, Andreas Buser, Jakob Passweg, Stefan Schaub, Michael Dickenmann, Joerg Halter, Andreas Holbro
Summary: This study reports the use of heparin as an alternative anticoagulant for immunoadsorption (IA) in the preparation of ABO blood group-incompatible living donor kidney transplantation (ABOi LDKT). Results showed that IA with heparin was safe and effective, with no major bleeding or significant complications observed. Graft function, graft survival, and overall survival did not significantly differ from standard anticoagulation or ABO compatible recipients.
TRANSFUSION MEDICINE AND HEMOTHERAPY
(2023)
Article
Medicine, General & Internal
Pilar Solves, Javier Marco-Ayala, Miguel Angel Sanz, Ines Gomez-Segui, Aitana Balaguer-Rosello, Ana Facal, Marta Villalba, Juan Montoro, Guillermo Sanz, Javier de la Rubia, Jaime Sanz
Summary: This study compares the transfusion requirements of patients undergoing different modalities of hematopoietic stem cell transplantation (HSCT) over a twelve-year period. The results show that transfusion requirements have significantly increased for matched related donor HSCT, while there were no significant changes for matched unrelated donor and haploidentical transplant HSCT.
JOURNAL OF CLINICAL MEDICINE
(2023)
Article
Hematology
Igor Stojkov, Annette Conrads-Frank, Ursula Rochau, Marjan Arvandi, Karin A. Koinig, Michael Schomaker, Moshe Mittelman, Pierre Fenaux, David Bowen, Guillermo F. Sanz, Luca Malcovati, Saskia Langemeijer, Ulrich Germing, Krzysztof Madry, Agnes Guerci-Bresler, Dominic J. Culligan, Ioannis Kotsianidis, Laurence Sanhes, Juliet Mills, Sibylle Puntscher, Daniela Schmid, Corine van Marrewijk, Alexandra Smith, Fabio Efficace, Theo de Witte, Reinhard Stauder, Uwe Siebert
Summary: This study aimed to identify determinants of low health-related quality of life (HRQoL) in patients recently diagnosed with myelodysplastic syndromes (MDS) for guiding early intervention strategies. Multiple factors, including age, sex, serum ferritin level, comorbidity burden, and Karnofsky performance status, influenced the HRQoL of MDS patients.
Letter
Biophysics
Silvio Ragozzino, Nicolas J. Mueller, Dionysios Neofytos, Jakob Passweg, Antonia Mueller, Michael Medinger, Christian Van Delden, Stavroula Masouridi-Levrat, Yves Chalandon, Sarah Tschudin-Sutter, Nina Khanna, Swiss Transplant Cohort Study
BONE MARROW TRANSPLANTATION
(2023)
Article
Hematology
Simone Dertschnig, Peter Gergely, Jurgen Finke, Urs Schanz, Ernst Holler, Udo Holtick, Gerard Socie, Michael Medinger, Jakob Passweg, Takanori Teshima, Christos Stylianou, Stephan Oehen, Dominik Heim, Christoph Bucher
Summary: Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is the only curative option for patients with acute myelogenous leukemia, but outcomes are limited by leukemia relapse, graft-versus-host disease (GVHD), and abnormal immune reconstitution. Mocravimod, an oral sphingosine-1-phosphate receptor modulator, shows potential in preventing GVHD while maintaining graft-versus-leukemia (GVL) activity. This study evaluated the safety, tolerability, and pharmacokinetics of mocravimod in allo-HSCT recipients and found that it can be safely added to standard treatment regimens without negative impact on engraftment and transplantation outcomes.
TRANSPLANTATION AND CELLULAR THERAPY
(2023)
Article
Oncology
Franziska C. Adam, Jakub Szybinski, Jorg P. Halter, Nathan Cantoni, Friedel Wenzel, Katharina Leonards, Sime Brkic, Jakob R. Passweg, Ivo Touw, Julia E. Maxson, Sara C. Meyer
Summary: This study investigates the role of co-occurring germline and somatic CSF3R mutations in chronic neutrophilic leukemia (CNL) and the impact of clonal evolution on leukemia transformation. The study finds that CNL can transform not only to acute myeloid leukemia (AML) but also to mixed phenotype acute leukemia (MPAL). The molecular evolution is particularly interesting with germline and somatic mutations occurring on the same allele.
Article
Hematology
Aitana Balaguer-Rosello, Jose Luis Pinana, Luis Bataller, Juan Montoro, Samuel Romero, Irene Navarro, Ignacio Lorenzo, Rafael Andreu, Manuel Guerreiro, Cristobal Aguilar, David Gorriz, Lara Dominguez, Rosalia de la Puerta, Ines Gomez, Pilar Solves, Isidro Jarque, Miguel Angel Sanz, Guillermo Sanz, Jaime Sanz
Summary: Central nervous system involvement in EBV-related post-transplant lymphoproliferative disorders after allo-HSCT remains poorly defined, with CNS-PTLDs having a low incidence rate and poor prognosis, mostly presenting with neurological symptoms.
TRANSPLANTATION AND CELLULAR THERAPY
(2021)
