Review
Medicine, General & Internal
Mariana Nassif Kerbauy, Leonardo Javier Arcuri, Sergio Leonardo Favareto, Ana Carolina Pires de Rezende, Nelson Hamerschlak
Summary: Total body irradiation (TBI) is an important part of the conditioning regimen in hematopoietic cell transplantation. However, higher doses of TBI can result in more significant toxicities. To overcome this, total marrow irradiation and total marrow and lymphoid irradiation have been developed to deliver targeted radiotherapy while sparing organs. Studies have shown that these techniques can be safely administered with different chemotherapy conditioning regimens, especially in situations such as multiple myeloma, high-risk hematologic malignancies, relapsed or refractory leukemias, and elderly or frail patients, with low transplant-related mortality rates.
FRONTIERS IN MEDICINE
(2023)
Article
Biophysics
Alice Pievani, Valentina Granata, Giacomo Desantis, Laura Antolini, Sara Ornaghi, Antonio Galleu, Andrea Biondi, Bernhard Gentner, Francesco Dazzi, Marta Serafini
Summary: Monocytes are effective at improving engraftment in HSPC transplantation, particularly in cases of limited cell doses such as CB transplantation and HSC gene therapy. Co-culturing monocytes increases survival and stemness of CB-CD34(+) cells. Soluble factors and direct-cell contact interactions play critical roles in the HSC-monocyte crosstalk.
BONE MARROW TRANSPLANTATION
(2022)
Article
Hematology
Satoshi Yamasaki, Shohei Mizuno, Makoto Iwasaki, Sachiko Seo, Naoyuki Uchida, Miyakoshi Shigesaburo, Nobuaki Nakano, Kazuya Ishiwata, Yasufumi Uehara, Tetsuya Eto, Ken Takase, Toshiro Kawakita, Masatsugu Tanaka, Masashi Sawa, Yuta Katayama, Yuichiro Nawa, Onizuka Makoto, Tatsuo Ichinohe, Yoshiko Atsuta, Junya Kanda, Masamitsu Yanada
Summary: This study analyzed the applications and factors affecting unrelated donor bone marrow transplantation (UR-BMT), unrelated donor cord blood stem cell transplantation (UR-CBT), and haploidentical peripheral blood stem cell transplantation (Haplo-PBSCT) in older patients with acute myeloid leukemia (AML) in Japan. The results showed that overall survival (OS) was superior in the UR-BMT group compared to the other groups. However, in non-complete remission (non-CR) patients, Karnofsky performance status (KPS) <80 at HCT and poor-risk cytogenetics were identified as independent predictors of worse OS. Therefore, KPS <80 may serve as an alternative indicator for non-CR AML patients with poor-risk cytogenetics in the selection of HCT, alternative treatments, or best supportive therapy, and optimal KPS is crucial for the success of HCT.
ANNALS OF HEMATOLOGY
(2023)
Article
Oncology
Rebecca A. Harrison, Noha Sharafeldin, Jennie L. Rexer, Brennan Streck, Melissa Petersen, Ashley M. Henneghan, Shelli R. Kesler
Summary: Hematopoietic stem cell transplant (HSCT) is crucial in treating hematologic cancers, but can lead to cognitive impairment and other challenges for survivors. Understanding the impact on brain health is essential for clinicians working with HSCT survivors, as it can affect various aspects of their lives. Efforts to identify and manage cognitive dysfunction and related symptoms are important for managing the long-term effects of HSCT.
Review
Oncology
Aneta Sevcikova, Ivana Fridrichova, Nataliia Nikolaieva, Lenka Kalinkova, Radoslav Omelka, Monika Martiniakova, Sona Ciernikova
Summary: Hematologic malignancies can arise at any stage of hematopoiesis and miRNAs play a key role in their development by regulating gene expression. This review summarizes the current knowledge of dysregulated miRNA expression in hematological malignancies and discusses their clinical utility as biomarkers for diagnosis, prognosis, and treatment response. The potential role of miRNAs in hematopoietic stem cell transplantation and post-transplant complications is also explored. The therapeutical potential of miRNA-based approaches, including antagomiRs, mimetics, and circRNAs, is outlined. Overall, miRNAs have the potential to improve the diagnosis and outcomes of hematologic malignancies.
Article
Oncology
Leo Luznik, Marcelo C. Pasquini, Brent Logan, Robert J. Soiffer, Juan Wu, Steven M. Devine, Nancy Geller, Sergio Giralt, Helen E. Heslop, Mary M. Horowitz, Richard J. Jones, Mark R. Litzow, Adam Mendizabal, Lori Muffly, Eneida R. Nemecek, Lynn O'Donnell, Richard J. O'Reilly, Raquel Palencia, Johannes Schetelig, Leyla Shune, Scott R. Solomon, Sumithira Vasu, Vincent T. Ho, Miguel-Angel Perales
Summary: The study found that CNI-free approaches, such as ex vivo CD34 selection or in vivo post-transplant cyclophosphamide, can reduce the incidence of chronic GVHD after hematopoietic cell transplantation. However, these approaches did not improve patients' relapse-free survival. Tacrolimus and methotrexate with bone marrow graft remain an effective GVHD prophylaxis strategy.
JOURNAL OF CLINICAL ONCOLOGY
(2022)
Review
Biophysics
Hermioni L. Amonoo, Lauren E. Harnedy, Sophie C. Staton, Regina M. Longley, Elizabeth Daskalakis, Areej El-Jawahri, Jeff C. Huffman
Summary: While peer support has been widely used and associated with clinical outcomes in patients with solid malignancies, there is limited research on the use of peer support among patients with hematologic malignancies and/or hematopoietic stem cell transplantation (HSCT) recipients. Few studies have shown a positive association between peer support and better health outcomes, particularly in physical symptoms improvement. However, more empirical-based peer support interventions are needed for the HSCT population.
BONE MARROW TRANSPLANTATION
(2022)
Review
Microbiology
Tony Cundell, J. Wade Atkins, Anna F. Lau
Summary: In the past two decades, the rapid advancement of technology has allowed for the widespread use of cell and gene therapy products in treating various diseases. This study reviewed literature from 2003 to 2021 to identify trends in microbial contamination of hematopoietic stem cells (HSCs) derived from different sources. The study also discussed regulatory context, testing expectations, clinical risks, and current practices for manufacturing and testing of HSCs.
JOURNAL OF CLINICAL MICROBIOLOGY
(2023)
Article
Hematology
Laveena Munshi, Michael Darmon, Marcio Soares, Peter Pickkers, Philippe Bauer, Anne-Pascale Meert, Ignacio Martin-Loeches, Thomas Staudinger, Frederic Pene, Massimo Antonelli, Andreas Barratt-Due, Alexandre Demoule, Victoria Metaxa, Virginie Lemiale, Fabio Taccone, Djamel Mokart, Elie Azoulay, Sangeeta Mehta
Summary: Research suggests that patients with acute respiratory failure requiring ICU admission after autologous or allogeneic hematopoietic cell transplantation have similar hospital mortality rates as those with hematologic malignancy not treated with HCT.
TRANSPLANTATION AND CELLULAR THERAPY
(2021)
Article
Biophysics
M. Akif Yesilipek, Vedat Uygun, Alphan Kupesiz, Gulsun Karasu, Gulyuz Ozturk, Mehmet Ertem, Ilgen Sasmaz, Hayriye Daloglu, Elif Guler, Volkan Hazar, Tunc Fisgin, Gulay Sezgin, Savas Kansoy, Baris Kuskonmaz, Burcu Akinci, Namik Ozbek, Elif Unal Ince, Seda Ozturkmen, Funda Tayfun Kupesiz, Koray Yalcin, Sema Anak, Ceyhun Bozkurt, Musa Karakukcu, Serhan Kupeli, Davut Albayrak, Haldun Oniz, Serap Aksoylar, Fatma Visal Okur, Canan Albayrak, Fatma Demir Yenigurbuz, Ikbal Ok Bozkaya, Talia Ileri, Orhan Gursel, Barbaros Sahin Karagun, Gulen Tuysuz Kintrup, Suna Celen, Murat Elli, Basak Adakli Aksoy, Ebru Yilmaz, Atila Tanyeli, Sule Turan Akyol, Zuhal Onder Sivi, Gulcihan Ozek, Duygu Uckan, Ibrahim Kartal, Didem Atay, Arzu Akyay, Ozlem Arman Bilir, Hasan Fatih Cakmakl, Emin Kurekci, Baris Malbora, Sinan Akbayram, Haci Ahmet Demir, Suar Caki Kilic, Adalet Meral Gunes, Emine Zengin, Salih Ozmen, Ali Bulent Antmen
Summary: The study analyzed national data on hematopoietic stem cell transplantation for thalassemia major patients in Turkey, finding that transplanting before the age of 7 with a matched donor can improve outcomes, and that different time points and donor types have varying impacts on transplant results.
BONE MARROW TRANSPLANTATION
(2022)
Article
Multidisciplinary Sciences
Helene Bjoerg Kristensen, Thomas Levin Andersen, Andrea Patriarca, Klaus Kallenbach, Birgit MacDonald, Tanja Sikjaer, Charlotte Ejersted, Jean-Marie Delaisse
Summary: The dormancy and formation of hematopoietic stem cells and progenitors are guided by signals from the bone marrow microenvironment. Research on the microenvironment of murine hematopoietic stem cells is more comprehensive, while knowledge on murine progenitor microenvironments and even more so in humans is lacking. Characterizing these microenvironments is crucial for understanding hematopoiesis and developing treatments for bone marrow malignancies, but challenging due to difficulties in detecting hematopoietic stem cells in the complex bone marrow landscape.
Article
Hematology
Robert Chiesa, Jaap Jan Boelens, Christine N. Duncan, Jorn-Sven Kuhl, Caroline Sevin, Neena Kapoor, Vinod K. Prasad, Caroline A. Lindemans, Simon A. Jones, Hernan M. Amartino, Mattia Algeri, Nancy Bunin, Cristina Diaz-de-Heredia, Daniel J. Loes, Esther Shamir, Alison Timm, Elizabeth McNeil, Andrew C. Dietz, Paul J. Orchard
Summary: A study found that allogeneic hematopoietic stem cell transplantation can stabilize neurologic function and improve survival in patients with early cerebral adrenoleukodystrophy. The donor source, donor match, conditioning regimen, and disease stage all have an impact on the outcomes of the transplantation. However, there are associated risks such as transplant-related mortality, graft failure, and graft-versus-host disease.
Review
Nutrition & Dietetics
Siyuan Sun, Yingxue Han, Yumei Lei, Yifei Yu, Yanbin Dong, Juan Chen
Summary: This review comprehensively summarizes the intrinsic factors involved in the regulation of hematopoietic stem cells (HSCs), including RNA-binding protein, modulators in epigenetics, and enhancer-promotor-mediated transcription. It also explores their roles in bone marrow transplantation therapies and autoimmune diseases. Additionally, it discusses the effects of high-fat diets and nutrients (such as vitamins, amino acids, probiotics, and prebiotics) on HSC regulation, providing valuable insights for future HSC research.
Review
Hematology
Zachariah DeFilipp, Mehrdad Hefazi, Yi-Bin Chen, Bruce R. Blazar
Summary: This article discusses the application of transplantation in the treatment of congenital or acquired nonmalignant blood diseases. Unlike patients with hematologic malignancies, the priority for nonmalignant blood disease patients is to avoid graft-versus-host disease, and emerging therapeutic methods hold promise for advancing transplantation care.
Article
Pediatrics
Jian Hua Li, Shu Wen Sun, Yuan Ai, Xue Yang, Yi Ping Zhu
Summary: Glanzmann thrombasthenia (GT) is a rare platelet disorder caused by an anomaly of the platelet membrane glycoprotein GPIIb/IIIa. Diagnosis relies on platelet function analysis, flow cytometry, and gene detection, while allogeneic hematopoietic stem cell transplantation (allo-HSCT) is the only radical treatment for GT.
FRONTIERS IN PEDIATRICS
(2022)
Article
Cell & Tissue Engineering
Mathew G. Angelos, Juan E. Abrahante, Robert H. Blum, Dan S. Kaufman
Article
Cell & Tissue Engineering
Ye Li, David L. Hermanson, Branden S. Moriarity, Dan S. Kaufman
Review
Hematology
Mathew G. Angelos, Dan S. Kaufman
CURRENT OPINION IN HEMATOLOGY
(2018)
Article
Hematology
Davide Bernareggi, Somayeh Pouyanfard, Dan S. Kaufman
EXPERIMENTAL HEMATOLOGY
(2019)
Review
Immunology
Michelle L. Saetersmoen, Quirin Hammer, Bahram Valamehr, Dan S. Kaufman, Karl-Johan Malmberg
SEMINARS IN IMMUNOPATHOLOGY
(2019)
Review
Clinical Neurology
Anastasie M. Dunn-Pirio, Benjamin M. Heyman, Dan S. Kaufman, Revere P. Kinkel
CURRENT TREATMENT OPTIONS IN NEUROLOGY
(2019)
Article
Hematology
Huang Zhu, Robert H. Blum, Ryan Bjordahl, Svetlana Gaidarova, Paul Rogers, Tom Tong Lee, Ramzey Abujarour, Gregory B. Bonello, Jianming Wu, Pei-Fang Tsai, Jeffrey S. Miller, Bruce Walcheck, Bahram Valamehr, Dan S. Kaufman
Article
Cell & Tissue Engineering
Fahad Kidwai, Byron W. H. Mui, Deepika Arora, Kulsum Iqbal, Madison Hockaday, Luis Fernandez de Castro Diaz, Natasha Cherman, Daniel Martin, Vamsee D. Myneni, Moaz Ahmad, Katarzyna Futrega, Sania Ali, Randall K. Merling, Dan S. Kaufman, Janice Lee, Pamela G. Robey
Article
Cell & Tissue Engineering
Huang Zhu, Robert H. Blum, Davide Bernareggi, Eivind Heggernes Ask, Zhengming Wu, Hanna Julie Hoel, Zhipeng Meng, Chengsheng Wu, Kun-Liang Guan, Karl-Johan Malmberg, Dan S. Kaufman
Article
Immunology
Benjamin H. Goldenson, Huang Zhu, YunZu Michele Wang, Naveen Heragu, Davide Bernareggi, Alessa Ruiz-Cisneros, Andres Bahena, Eivind Heggernes Ask, Hanna Julie Hoel, Karl-Johan Malmberg, Dan S. Kaufman
FRONTIERS IN IMMUNOLOGY
(2020)
Review
Biochemistry & Molecular Biology
William C. Hahn, Joel S. Bader, Theodore P. Braun, Andrea Califano, Paul A. Clemons, Brian J. Druker, Andrew J. Ewald, Haian Fu, Subhashini Jagu, Christopher J. Kemp, William Kim, Calvin J. Kuo, Michael McManus, Gordon B. Mills, Xiulei Mo, Nidhi Sahni, Stuart L. Schreiber, Jessica A. Talamas, Pablo Tamayo, Jeffrey W. Tyner, Bridget K. Wagner, William A. Weiss, Daniela S. Gerhard
Summary: Characterization of cancer genomes has provided insight into somatically altered genes across tumors, but the function of most cancer alleles remains mysterious and does not impact therapy for most patients. Approaches to understand the function and circuitry of cancer genes provide complementary approaches to elucidate both oncogene and non-oncogene dependencies. Emerging work indicates that therapeutic targets engendered by non-oncogene dependencies are more diverse than recurrently mutated genes, providing new opportunities for clinical translation.
Editorial Material
Cell & Tissue Engineering
Benjamin H. Goldenson, Dan S. Kaufman
Summary: The study demonstrates that expression of modified CD16a and IL-15 receptor combined with knockout of CD38 can enhance NK cell-mediated activity against leukemia and multiple myeloma.
Article
Allergy
Lori Broderick, Gwendolyn M. Clay, Robert H. Blum, Yang Liu, Rachael McVicar, Fabio Papes, Laela M. Booshehri, Ian G. Cowell, Caroline A. Austin, Christopher D. Putnam, Dan S. Kaufman
Summary: The study examines the impact of Hoffman syndrome-associated mutations in TOP2B on the development and function of NK cells. Using a knockin murine model and patient-derived iPSCs, the researchers investigated NK-cell development in mouse bone marrow and spleen, and assessed the immunophenotype, gene expression, and cytotoxic activity of NK cells in both murine and human models.
JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY
(2022)
Article
Cell & Tissue Engineering
Somayeh Pouyanfard, Nairika Meshgin, Luisjesus S. Cruz, Karin Diggle, Hamidreza Hashemi, Timothy Pham, Manuel Fierro, Pablo Tamayo, Andrea Fanjul, Tatiana Kisseleva, Dan S. Kaufman
Summary: The study shows that human iPSC-derived macrophages can effectively reduce gene expression and pathological markers of liver fibrosis, offering a promising cell therapy approach to ameliorate fibrosis.
Article
Cell Biology
Frank Cichocki, Ryan Bjordahl, Svetlana Gaidarova, Sajid Mahmood, Ramzey Abujarour, Hongbo Wang, Katie Tuininga, Martin Felices, Zachary B. Davis, Laura Bendzick, Raedun Clarke, Laurel Stokely, Paul Rogers, Moyar Ge, Megan Robinson, Betsy Rezner, David L. Robbins, Tom T. Lee, Dan S. Kaufman, Bruce R. Blazar, Bahram Valamehr, Jeffrey S. Miller
SCIENCE TRANSLATIONAL MEDICINE
(2020)