Article
Immunology
Alexandra Laberko, Anna Mukhina, Elena Machneva, Olga Pashchenko, Tatiana Bykova, Larisa Vahonina, Gleb Bronin, Yulia Skvortsova, Elena Skorobogatova, Irina Kondratenko, Larisa Fechina, Anna Shcherbina, Ludmila Zubarovskaya, Dmitry Balashov, Alexander Rumiantsev
Summary: This study collected and analyzed data on HSCT activity in IEI patients in Russia. The results showed that the number of IEI patients receiving HSCT has increased significantly over the years, and there has been a change in the distribution of IEI diagnoses. The study suggests the need for additional transplant beds for IEI in Russia.
JOURNAL OF CLINICAL IMMUNOLOGY
(2023)
Review
Health Care Sciences & Services
Heather R. Wolfe, Mitchell E. Horwitz, Lindsay A. M. Rein
Summary: Primary myelofibrosis is a BCR-ABL1 negative myeloproliferative neoplasm characterized by clonal proliferation of myeloid cells. Allogenic hematopoietic stem cell transplantation may provide a safe and effective curative option for a broader range of primary myelofibrosis patients in the future.
JOURNAL OF PERSONALIZED MEDICINE
(2022)
Article
Hematology
Masatomo Kuno, Satoshi Yamasaki, Nobuharu Fujii, Yasushi Ishida, Takahiro Fukuda, Keisuke Kataoka, Naoyuki Uchida, Yuta Katayama, Maho Sato, Daishi Onai, Toshihiro Miyamoto, Shuichi Ota, Satoshi Yoshioka, Takahide Ara, Akira Hangaishi, Yoshiko Hashii, Makoto Onizuka, Tatsuo Ichinohe, Yoshiko Atsuta, Yoshihiro Inamoto
Summary: The study compared myeloid neoplasms (MNs) following allogeneic hematopoietic cell transplantation (HCT) versus autologous HCT using a Japanese HCT registry database. The cumulative incidence of MNs was lower after allogeneic HCT than after autologous HCT, and those following allogeneic HCT developed in younger patients and sooner after HCT. Further research is needed to understand the mechanisms of MN development following allogeneic HCT.
AMERICAN JOURNAL OF HEMATOLOGY
(2022)
Article
Medicine, Research & Experimental
Christopher J. Severyn, Benjamin A. Siranosian, Sandra Tian-Jiao Kong, Angel Moreno, Michelle M. Li, Nan Chen, Christine N. Duncan, Steven P. Margossian, Leslie E. Lehmann, Shan Sun, Tessa M. Andermann, Olga Birbrayer, Sophie Silverstein, Carol G. Reynolds, Soomin Kim, Niaz Banaei, Jerome Ritz, Anthony A. Fodor, Wendy B. London, Ami S. Bhatt, Jennifer S. Whangbo
Summary: This pilot study examined the impact of gut decontamination (GD) on gut microbiome composition and the incidence of acute graft-versus-host disease (aGVHD) in patients undergoing allogeneic hematopoietic cell transplantation (HCT). The study found that GD did not affect gut microbiome diversity or clinical outcomes, but it may protect against gut-derived bloodstream infections (BSI) by decreasing the prevalence or abundance of gut pathogens.
Review
Oncology
Li Xuan, Qifa Liu
Summary: Relapse is still the main reason for treatment failure in AML patients undergoing allo-HSCT. Evidence suggests that maintenance therapy post-transplantation, particularly targeted drugs like hypomethylating agents, FLT3 inhibitors, and isocitrate dehydrogenase inhibitors, could benefit AML patients, especially those at high risk.
JOURNAL OF HEMATOLOGY & ONCOLOGY
(2021)
Review
Cell Biology
Paula Germino-Watnick, Malikiya Hinds, Anh Le, Rebecca Chu, Xiong Liu, Naoya Uchida
Summary: Autologous hematopoietic stem cell (HSC)-targeted gene therapy provides a one-time cure for various genetic diseases including sickle cell disease (SCD) and beta-thalassemia. This review discusses the methods of gene addition and gene editing in HSC-targeted gene therapy for SCD.
Article
Immunology
Meng Shan, Danya Shen, Tiemei Song, Wenyan Xu, Huiying Qiu, Suning Chen, Yue Han, Xiaowen Tang, Miao Miao, Aining Sun, Depei Wu, Yang Xu
Summary: The diagnostic value of procalcitonin and the prognostic role of PCT clearance were evaluated in neutropenic patients after allogeneic hematopoietic stem cell transplantation. It was found that procalcitonin can be used to detect documented and bloodstream infection, and the inability to decrease procalcitonin by more than 80% within 5-7 days after fever onset predicted poor 100-day survival. A prognostic nomogram combining procalcitonin clearance and clinical parameters effectively predicted the 100-day survival of febrile patients.
FRONTIERS IN IMMUNOLOGY
(2022)
Article
Oncology
Vera Dalla Via, Anne Stelmes, Mike Recher, Christian Arranto, Till Junker, Francesca Matteazzi, Dominik Heim, Jakob R. Passweg, Michael Medinger, Jana van den Berg
Summary: This study presents three cases of adult patients with PID who received reduced-intensity allogenic hematopoietic cell transplantation. The patients were successfully engrafted, but infectious complications remained a major concern.
ANTICANCER RESEARCH
(2022)
Review
Immunology
Guancui Yang, Xiang Wang, Shiqin Huang, Ruihao Huang, Jin Wei, Xiaoqi Wang, Xi Zhang
Summary: Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is the only curative treatment for myeloid malignancies, but relapse and graft-versus-host disease (GvHD) still impact patient survival. Epigenetic agents, such as hypomethylating agents (HMAs), have been explored to reduce relapse risk and prevent GvHD, and combining epigenetic therapy with HSCT may optimize the transplantation process and prevent treatment failure.
FRONTIERS IN IMMUNOLOGY
(2022)
Article
Allergy
Dimana Dimitrova, Zohreh Nademi, Maria Elena Maccari, Stephan Ehl, Gulbu Uzel, Takahiro Tomoda, Tsubasa Okano, Kohsuke Imai, Benjamin Carpenter, Winnie Ip, Kanchan Rao, Austen J. J. Worth, Alexandra Laberko, Anna Mukhina, Benedicte Neven, Despina Moshous, Carsten Speckmann, Klaus Warnatz, Claudia Wehr, Hassan Abolhassani, Asghar Aghamohammadi, Jacob J. Bleesing, Jasmeen Dara, Christopher C. Dvorak, Sujal Ghosh, Hyoung Jin Kang, Gasper Markelj, Arunkumar Modi, Diana K. Bayer, Luigi D. Notarangelo, Ansgar Schulz, Marina Garcia-Prat, Pere Soler-Palacin, Musa Karakukcu, Ebru Yilmaz, Eleonora Gambineri, Mariacristina Menconi, Tania N. Masmas, Mette Holm, Carmem Bonfim, Carolina Prando, Stephen Hughes, Stephen Jolles, Emma C. Morris, Neena Kapoor, Sylwia Koltan, Shankara Paneesha, Colin Steward, Robert Wynn, Ulrich Duffner, Andrew R. Gennery, Arjan C. Lankester, Mary Slatter, Jennifer A. Kanakry
Summary: This study retrospectively collected data on 57 patients with activated phosphoinositide 3-kinase delta syndrome (APDS) who underwent hematopoietic cell transplantation (HCT) and evaluated the outcomes. The study found that graft failure, graft instability, and poor graft function requiring unplanned donor cell infusion were major barriers to successful HCT. The use of mammalian target of rapamycin inhibitor (mTORi) post-transplantation may confer an advantage to residual host cells, promoting graft instability.
JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY
(2022)
Article
Immunology
Vera Portillo, Stavroula Masouridi-Levrat, Lena Royston, Sabine Yerly, Manuel Schibler, Maria Mappoura, Sarah Morin, Federica Giannotti, Anne-Claire Mamez, Christian van Delden, Yves Chalandon, Dionysios Neofytos
Summary: Positive CMV serology in allogeneic hematopoietic cell transplant recipients (allo-HCTRs) may be false-positive due to transfusion-associated passive immunity. Reclassifying CMV status can help accurately assess the risk of CMV infection in these patients.
CLINICAL INFECTIOUS DISEASES
(2023)
Article
Microbiology
Carmine Liberatore, Francesca Farina, Raffaella Greco, Fabio Giglio, Daniela Clerici, Chiara Oltolini, Maria Teresa Lupo Stanghellini, Federica Barzaghi, Paolo Vezzulli, Elena Orsenigo, Consuelo Corti, Fabio Ciceri, Jacopo Peccatori
Summary: Despite the introduction of mold-active antifungal prophylaxis, breakthrough invasive fungal infections remain a possible complication for hematological patients and hematopoietic stem-cell transplantation recipients. Management of these infections is challenging and requires individualized antifungal treatment based on risk factors, ongoing prophylaxis, and local epidemiology. Early broad-spectrum preemptive therapy is advisable to cover against rare fungal strains and atypical infection sites.
Article
Medicine, General & Internal
He Zhang, Yunxing Xue, Tuo Pan, Xiyu Zhu, Hoshun Chong, Can Xu, Fudong Fan, Hailong Cao, Bomin Zhang, Jun Pan, Qing Zhou, Gang Yang, Jiaxian Wang, Dong-Jin Wang
Summary: Cell-based transplantation using hiPSC-CMs is a promising strategy for treating HF, but its safety and efficacy in patients are still uncertain. This study aims to evaluate the safety and efficacy of epicardial injection of hiPSC-CMs during CABG surgery in patients with advanced HF.
Article
Oncology
Juan Carlos Hernandez-Boluda, Arturo Pereira, Nicolaus Kroeger, Dietrich Beelen, Marie Robin, Martin Bornhaeuser, Emanuele Angelucci, Antonin Vitek, Igor Wolfgang Blau, Riitta Niittyvuopio, Juergen Finke, Jan J. Cornelissen, Jakob Passweg, Peter Dreger, Eefke Petersen, Lothar Kanz, Jaime Sanz, Tsila Zuckerman, Nienke Zinger, Simona Iacobelli, Patrick Hayden, Tomasz Czerw, Donal McLornan, Ibrahim Yakoub-Agha
Summary: This study aimed to evaluate survival determinants in myelofibrosis patients undergoing allo-HCT and describe factors predicting post-HCT complications. Factors associated with increased mortality included age, Karnofsky Performance Status, graft failure, aGVHD, and disease progression/relapse. Risk of aGVHD and relapse incidence varied with donor type and conditioning regimen, with GVHD having a complex impact on relapse and survival.
Review
Biochemistry & Molecular Biology
Riccardo Masetti, Daniele Zama, Davide Leardini, Edoardo Muratore, Silvia Turroni, Patrizia Brigidi, Andrea Pession
Summary: The gut microbiome plays a significant role in hematopoietic stem cell transplantation, with its metabolites mediating interactions between intestinal microbial communities and the host. New data offer a fresh perspective on the role of microbiome-derived metabolites in transplant settings.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2021)
Article
Hematology
Michael A. Pulsipher, Kwang W. Ahn, Nancy J. Bunin, Nahal Lalefar, Eric Anderson, Allyson Flower, Mitchell S. Cairo, Julie-An Talano, Sonali Chaudhury, Carrie L. Kitko, Jamie L. Duke, Dimitrios Monos, Wing Leung, Christopher C. Dvorak, Hisham Abdel-Azim
Summary: A prospective multicenter study was conducted to evaluate the use of TCR-alpha beta/CD19-depleted haploidentical donors in hematopoietic cell transplantation for children with acute leukemia and MDS. The study showed improved outcomes in terms of disease-free survival and overall survival, especially in younger patients, those treated with reduced toxicity conditioning, and children with minimal residual disease before transplantation.
Article
Oncology
Leena Chehab, David R. Doody, Adam J. Esbenshade, Gregory M. T. Guilcher, Christopher C. Dvorak, Brian T. Fisher, Beth A. Mueller, Eric J. Chow, Jenna Rossoff
Summary: Infections pose a significant risk for childhood cancer patients during therapy, but little is known about the risk of infection in long-term survivors. A retrospective observational study in Washington State found that survivors of childhood cancer had a higher incidence of hospitalized infections compared to individuals without cancer. The risk remained elevated even years after the initial diagnosis.
JOURNAL OF CLINICAL ONCOLOGY
(2023)
Article
Allergy
Roxane Labrosse, Ines Boufaied, Benoite Bourdin, Saideep Gona, Haley E. Randolph, Brent R. Logan, Sara Bourbonnais, Chloe Berthe, Wendy Chan, Rebecca H. Buckley, Roberta E. Parrott, Geoffrey D. E. Cuvelier, Neena Kapoor, Sharat Chandra, Blachy J. Davila Saldana, Hesham Eissa, Fred D. Goldman, Jennifer Heimall, Richard O'Reilly, Sonali Chaudhury, Edward A. Kolb, Shalini Shenoy, Linda M. Griffith, Michael Pulsipher, Donald B. Kohn, Luigi D. Notarangelo, Sung-Yun Pai, Morton J. Cowan, Christopher C. Dvorak, Ellie Haddad, Jennifer M. Puck, Luis B. Barreiro, Helene Decaluwe
Summary: Severe combined immunodeficiency (SCID) patients may experience CD4+ T-cell lymphopenia and T-cell exhaustion after transplantation, leading to incomplete immune reconstitution and poor long-term outcomes.
JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY
(2023)
Article
Pediatrics
William R. Otto, Christopher C. Dvorak, Craig L. K. Boge, Luis Ostrosky-Zeichner, Adam J. Esbenshade, Michael L. Nieder, Sarah Alexander, William J. Steinbach, Ha Dang, Doojduen Villaluna, Lu Chen, Micah Skeens, Theoklis E. Zaoutis, Lillian Sung, Brian T. Fisher
Summary: Fungitell (R) BDG screening is not effective in diagnosing IFD in the post-HCT period, mainly due to high false-positive rates. Fungitell (R) BDG surveillance testing should not be performed in children during the early post-HCT period while receiving antifungal prophylaxis.
PEDIATRIC TRANSPLANTATION
(2023)
Article
Allergy
Christopher C. Dvorak, Elie Haddad, Jennifer Heimall, Elizabeth Dunn, Morton J. Cowan, Sung-Yun Pai, Neena Kapoor, Lisa Forbes Satter, Rebecca H. Buckley, Richard J. O'Reilly, Sharat Chandra, Jeffrey J. Bednarski, Olatundun Williams, Ahmad Rayes, Theodore B. Moore, Christen L. Ebens, Blachy J. Davila Saldana, Aleksandra Petrovic, Deepak Chellapandian, Geoffrey D. E. Cuvelier, Mark T. Vander Lugt, Emi H. Caywood, Shanmuganathan Chandrakasan, Hesham Eissa, Frederick D. Goldman, Evan Shereck, Victor M. Aquino, Kenneth B. Desantes, Lisa M. Madden, Holly K. Miller, Lolie Yu, Larisa Broglie, Alfred Gillio, Ami J. Shah, Alan P. Knutsen, Jeffrey P. Andolina, Avni Y. Joshi, Paul Szabolcs, Malika Kapadia, Caridad A. Martinez, Roberta E. Parrot, Kathleen E. Sullivan, Susan E. Prockop, Roshini S. Abraham, Monica S. Thakar, Jennifer W. Leiding, Donald B. Kohn, Michael A. Pulsipher, Linda M. Griffith, Luigi D. Notarangelo, Jennifer M. Puck
Summary: Shearer et al in 2014 established criteria for the diagnosis and classification of severe combined immunodeficiency (SCID), but due to advancements in screening and genetic sequencing, revision of the criteria was necessary. The PIDTC 2022 Definitions provide a more precise description of SCID and its subtypes, facilitating analysis of SCID characteristics and outcomes.
JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY
(2023)
Meeting Abstract
Immunology
Lena Winestone, Brent R. Logan, Xuerong Liu, Rebecca H. Buckley, Richard J. O'Reilly, Linda M. Griffith, Jennifer Puck, Christopher C. Dvorak, Morton J. Cowan, Elie Haddad
CLINICAL IMMUNOLOGY
(2023)
Meeting Abstract
Immunology
Monica Thakar, Brent R. Logan, Jennifer Puck, Elizabeth Dunn, Rebecca Buckley, Morton Cowan, Sung-Yun Pai, Jennifer Heimall, Michael Pulsipher, Linda Griffith, Elie Haddad, Christopher C. Dvorak, Luigi Notarangelo
CLINICAL IMMUNOLOGY
(2023)
Article
Medicine, General & Internal
Monica S. Thakar, Brent R. Logan, Jennifer M. Puck, Elizabeth A. Dunn, Rebecca H. Buckley, Morton J. Cowan, Richard J. O'Reilly, Neena Kapoor, Lisa Forbes Satter, Sung-Yun Pai, Jennifer Heimall, Sharat Chandra, Christen L. Ebens, Deepak Chellapandian, Olatundun Williams, Lauri M. Burroughs, Blachy Davila Saldana, Ahmad Rayes, Lisa M. Madden, Shanmuganathan Chandrakasan, Jeffrey J. Bednarski, Kenneth B. Desantes, Geoffrey D. E. Cuvelier, Pierre Teira, Alfred P. Gillio, Hesham Eissa, Alan P. Knutsen, Frederick Goldman, Victor M. Aquino, Evan B. Shereck, Theodore B. Moore, Emi H. Caywood, Mark T. Vander Lugt, Jacob Rozmus, Larisa Broglie, Lolie C. Yu, Ami J. Shah, Jeffrey R. Andolina, Xuerong Liu, Roberta E. Parrott, Jasmeen Dara, Susan Prockop, Caridad A. Martinez, Malika Kapadia, Soma C. Jyonouchi, Kathleen E. Sullivan, Jack J. Bleesing, Sonali Chaudhury, Aleksandra Petrovic, Michael Keller, Troy C. Quigg, Suhag Parikh, Shalini Shenoy, Christine Seroogy, Tamar Rubin, Helene Decaluwe, John M. Routes, Troy R. Torgerson, Jennifer W. Leiding, Michael A. Pulsipher, Donald B. Kohn, Linda M. Griffith, Elie Haddad, Christopher C. Dvorak, Luigi D. Notarangelo
Summary: This study found that population-based newborn screening plays a crucial role in early detection and prompt treatment of SCID. The overall 5-year survival rate has significantly increased with the implementation of newborn screening, and the promotion of this screening method can benefit public health programs globally.
Editorial Material
Oncology
Robert Peter Gale, Wolfgang Hinterberger, Neal S. Young, Andrew R. Gennery, Christopher C. Dvorak, Kyle M. Hebert, Michael Heim, Larisa Broglie, Mary Eapen
Article
Oncology
Natasha N. Frederick, James L. Klosky, Lillian Meacham, Gwendolyn P. Quinn, Joanne F. Kelvin, Brooke Cherven, David R. Freyer, Christopher C. Dvorak, Julienne Brackett, Sameeya Ahmed-Winston, Elyse Bryson, H. Irene Su, Eric J. Chow, Jennifer Levine
Summary: This study investigated the fertility discussion practices in 220 Children's Oncology Group member institutions. It found that fertility discussions were not routine and males were more likely to be offered appropriate options compared to females. There were no specific criteria for offering fertility preservation options for females, while there were clearer criteria for males.
JCO ONCOLOGY PRACTICE
(2023)
Letter
Biophysics
Tristan E. Knight, Kwang Woo Ahn, Kyle M. Hebert, Rasha Atshan, Donna A. Wall, Kanhatai Chiengthong, Troy C. Lund, Tim Prestidge, Hemalatha G. Rangarajan, Christopher C. Dvorak, Jeffery J. Auletta, Michael Kent, Hasan Hashem, Julie-An Talano, Seth J. Rotz, Ellen Fraint, Kasiani C. Myers, Wing Leung, Akshay Sharma, Neel S. Bhatt, Timothy A. Driscoll, Lolie C. Yu, Kirk R. Schultz, Muna Qayed, Larisa Broglie, Mary Eapen, Gregory A. Yanik
BONE MARROW TRANSPLANTATION
(2023)
Article
Oncology
Michelle L. L. Schoettler, Christopher E. E. Dandoy, Anora Harris, Marilynn Chan, Keiko M. M. Tarquinio, Sonata Jodele, Muna Qayed, Benjamin Watkins, Pradip Kamat, Toni Petrillo, Jeremy Obordo, Christine S. S. Higham, Christopher C. C. Dvorak, Adrianna Westbrook, Matt S. S. Zinter, Kirsten M. M. Williams
Summary: This study evaluated the effect of different treatments on the outcomes of DAH patients and analyzed data from consecutive HCT patients from January 2018 to August 2022. The study found that using steroids, inhaled tranexamic acid, and inhaled recombinant activated factor VII can reduce the risk of death. Prospective studies are needed to validate these findings.
FRONTIERS IN ONCOLOGY
(2023)
Article
Hematology
Tristan E. Knight, Kwang Woo Ahn, Kyle M. Hebert, Rasha Atshan, Donna A. Wall, Kanhatai Chiengthong, Seth J. Rotz, Ellen Fraint, Hemalatha G. Rangarajan, Jeffery J. Auletta, Akshay Sharma, Carrie L. Kitko, Hasan Hashem, Kirsten M. Williams, Baldeep Wirk, Christopher C. Dvorak, Kasiani C. Myers, Michael A. Pulsipher, Anne B. Warwick, Nahal Rose Lalefar, Kirk R. Schultz, Muna Qayed, Larisa Broglie, Mary Eapen, Gregory A. Yanik
Summary: Consolidation with autologous hematopoietic stem cell transplantation (HSCT) has improved survival for patients with central nervous system tumors (CNSTs). This study analyzed the relationship between the CD34(+) dose and clinical outcomes, and found that increasing CD34(+) cell dose was associated with significantly improved overall survival (OS), progression-free survival (PFS), and lower relapse rates in children undergoing autologous HSCT for CNSTs.
TRANSPLANTATION AND CELLULAR THERAPY
(2023)
Review
Immunology
Donald B. Kohn
Summary: ADA SCID is a severe inborn immune deficiency disease that can be effectively treated using gene therapy. Continuous improvements in treatment methods have led to significant immune restoration and stable engraftment of gene-corrected hematopoietic stem cells in most ADA SCID patients. However, achieving sustained availability and access to this therapy remains challenging.
IMMUNOLOGICAL REVIEWS
(2023)
Meeting Abstract
Oncology
Jordan Milner, Julie-An Talano, Lauren Harrison, Janet Ayello, Allyson Flower, Yaya Chu, Olivia Rigot, Bryon D. Johnson, Yimei Li, Dean Anthony Lee, Rolla Abu-Arja, Julia Chu, Christopher C. Dvorak, Lynn C. O'Donnell, Yongping Wang, Nancy J. Bunin, Mitch S. Cairo
PEDIATRIC BLOOD & CANCER
(2022)