Article
Hematology
Suhag Parikh, Joel A. Brochstein, Einat Galamidi, Aurelie Schwarzbach, Joanne Kurtzberg
Summary: The use of omidubicel for ex vivo expansion of unrelated cord blood can improve engraftment in pediatric patients undergoing myeloablative HSCT for SCD, resulting in faster neutrophil engraftment and high long-term engraftment rates. However, the incidence of acute GVHD is high and needs to be addressed to optimize outcomes for patients with SCD.
Review
Medicine, General & Internal
Diana Vanegas, Laura Nino-Quiroga, Mauricio Chaparro, Bernardo Camacho-Rodriguez, Marcela Estupinan, Ana-Maria Perdomo-Arciniegas
Summary: The translated article discusses the main issues associated with using unrelated umbilical cord blood and haploidentical grafts as alternative donors, emphasizing that selecting the best donor depends on various factors.
FRONTIERS IN MEDICINE
(2021)
Review
Hematology
Arpita P. Gandhi, Laura F. Newell, Richard T. Maziarz
Summary: Umbilical cord blood transplantation is an important alternative for patients without matched related or unrelated donors. However, its use is limited due to lower cell dose, immature immune system, and increased risks of infection.
THERAPEUTIC ADVANCES IN HEMATOLOGY
(2023)
Review
Cell & Tissue Engineering
Yufeng Xi, Guang Yue, Shuqiang Gao, Rong Ju, Yujia Wang
Summary: Perinatal brain injury is a major cause of death and disability in children. Transplantation of human umbilical cord blood mononuclear cells has shown significant efficacy and safety in animal and clinical trials for the treatment of perinatal brain injury.
STEM CELL RESEARCH & THERAPY
(2022)
Review
Cell & Tissue Engineering
Gabriela Sanchez-Petitto, Katayoun Rezvani, May Daher, Hind Rafei, Partow Kebriaei, Elizabeth J. Shpall, Amanda Olson
Summary: Umbilical cord blood transplantation is an attractive alternative source of hematopoietic stem cells, with unique immune tolerance and engraftment kinetics. It has a lower risk of GvHD and comparable clinical outcomes to bone marrow transplantation. However, limitations in cell numbers and delayed engraftment pose challenges for its wider application. Research is being conducted to address these limitations and explore the potential of cord blood in treating various disorders.
STEM CELLS TRANSLATIONAL MEDICINE
(2023)
Article
Pediatrics
Amber Gibson, Dristhi Ragoonanan, Priti Tewari, Demetrios Petropoulos, Nidra Rodriguez, Courtney DiNardo, Kris M. Mahadeo, Sajad Khazal
Summary: This study reported a case of atypical DC with a germline missense mutation in the PMS2 gene, where the patient was successfully treated with umbilical cord blood transplant using a non-myeloablative regimen.
PEDIATRIC TRANSPLANTATION
(2022)
Article
Hematology
John E. Wagner, Karen K. Ballen, Mei-Jie Zhang, Mariam Allbee-Johnson, Chatchada Karanes, Filippo Milano, Michael R. Verneris, Mary Eapen, Claudio G. Brunstein
Summary: This study evaluated outcomes in patients with hematological malignancy treated with myeloablative conditioning prior to haplo- or umbilical cord blood (UCB) HSCT. The study found that overall survival after haplo-HSCT with PTCy was comparable to that after UCB regardless of HLA match group, but UCB recipients had lower relapse rates and higher nonrelapse mortality.
Article
Agriculture, Dairy & Animal Science
Mailin Gan, Lin Liu, Shunhua Zhang, Zongyi Guo, Ya Tan, Jia Luo, Qiong Yang, Hongmei Pan, Xuewei Li, Jinyong Wang, Linyuan Shen, Li Zhu
Summary: Differential expression of porcine umbilical cord blood miRNA may have differences in immunity and angiogenesis, with great potential in the treatment of human diseases due to their ability for cross-species transmission. miRNA in cord blood may be beneficial for muscle-related diseases and liver- and brain-related diseases, providing insights for potential applications in mother-fetal communication.
Article
Clinical Neurology
Ping Wang, Xiaonan Du, Quanli Shen, Wenjin Jiang, Chen Shen, Hongsheng Wang, Shuizhen Zhou, Yi Wang, Xiaowen Qian, Xiaowen Zhai
Summary: The study analyzed the efficacy of unrelated umbilical cord blood transplantation (UCBT) in the treatment of hereditary leukodystrophy. The results showed that UCBT can improve survival rates and stabilize neurological function in patients with hereditary leukodystrophy.
FRONTIERS IN NEUROLOGY
(2022)
Article
Biophysics
Stephanie Vairy, Isabelle Louis, Marie-France Vachon, Johanne Richer, Pierre Teira, Sonia Cellot, Edith Villeneuve, Elie Haddad, Michel Duval, Henrique Bittencourt
Summary: Intrabone cord blood transplantation (IB UCBT) is safe and well-tolerated in children with malignant and non-malignant hematologic diseases, providing faster hematologic recovery compared to traditional IV UCBT. A clinical trial showed a 6-year event-free survival rate of 66.7% and an overall survival rate of 80% with IB UCBT.
BONE MARROW TRANSPLANTATION
(2021)
Article
Medicine, General & Internal
Francesca Fumagalli, Valeria Calbi, Maria Grazia Natali Sora, Maria Sessa, Cristina Baldoli, Paola Maria Rancoita, Francesca Ciotti, Marina Sarzana, Maddalena Fraschini, Alberto Andrea Zambon, Serena Acquati, Daniela Redaelli, Vanessa Attanasio, Simona Miglietta, Fabiola De Mattia, Federica Barzaghi, Francesca Ferrua, Maddalena Migliavacca, Francesca Tucci, Vera Gallo, Ubaldo Del Carro, Sabrina Canale, Ivana Spiga, Laura Lorioli, Salvatore Recupero, Elena Sophia Fratini, Francesco Morena, Paolo Silvani, Maria Rosa Calvi, Marcella Facchini, Sara Locatelli, Ambra Corti, Stefano Zancan, Gigliola Antonioli, Giada Farinelli, Michela Gabaldo, Jesus Garcia-Segovia, Laetitia C. Schwab, Gerald F. Downey, Massimo Filippi, Maria Pia Cicalese, Sabata Martino, Clelia Di Serio, Fabio Ciceri, Maria Ester Bernardo, Luigi Naldini, Alessandra Biffi, Alessandro Aiuti
Summary: This study investigated the safety and efficacy of atidarsagene autotemcel (arsa-cel) in patients with metachromatic leukodystrophy (MLD). The results showed that arsa-cel treatment can preserve cognitive function and motor development, and slow down demyelination and brain atrophy in children with early-onset MLD.
Article
Cell & Tissue Engineering
Meijuan Tu, Aijie Huang, Lijuan Ning, Baolin Tang, Chunli Zhang, Guangyu Sun, Xiang Wan, Kaidi Song, Wen Yao, Ping Qiang, Yue Wu, Xiaoyu Zhu
Summary: The study established a predictive model combining clinical and nutritional factors to predict overall survival in UCBT recipients. High-risk score, calf skinfold thickness, and low albumin level were associated with survival post-UCBT.
STEM CELL RESEARCH & THERAPY
(2023)
Article
Oncology
Xin-Yu Li, Li-Ping Zhan, Dian-Dian Liu, Xia-Wei Han, Han Chen, Zheng-Zhou Wu, Yin Wang, Li-Ping Que, Xiao-Jun Wu, Su Liu, Kai-Mei Wang, Shao-Liang Huang, Jian-Pei Fang, Ke Huang, Hong-Gui Xu
Summary: Using posttransplant cyclophosphamide (PTCY) as a method for controlling peri-engraftment syndrome (PES) and prophylaxis against graft-versus-host disease (GvHD) in single-unit umbilical cord blood transplantation (UCBT) for children with acute leukemia is safe and effective. Higher doses of PTCY may be more suitable for GvHD prophylaxis in UCBT patients.
Article
Hematology
Navneet S. Majhail, Beckley Miller, Rebecca Dean, Rocio Manghani, Heayoung Shin, Smitha Sivaraman, Richard T. Maziarz
Summary: This study found that patients treated with omidubicel-onlv had faster hematopoietic recovery, shorter hospital stays, and reduced healthcare resource utilization compared to patients receiving standard umbilical cord blood treatment.
TRANSPLANTATION AND CELLULAR THERAPY
(2023)
Article
Obstetrics & Gynecology
Anup C. Katheria, Erin Clark, Bradley Yoder, Georg M. Schmolzer, Brenda Hiu Yan Law, Walid El-Naggar, David Rittenberg, Sheetal Sheth, Mohamed A. Mohamed, Courtney Martin, Farha Vora, Satyan Lakshminrusimha, Mark Underwood, Jan Mazela, Joseph Kaempf, Mark Tomlinson, Yvonne Gollin, Kevin Fulford, Yvonne Goff, Paul Wozniak, Katherine Baker, Wade Rich, Ana Morales, Michael Varner, Debra Poeltler, Yvonne Vaucher, Judith Mercer, Neil Finer, Laure El Ghormli, Madeline Murguia Rice
Summary: This study found that umbilical cord milking did not reduce admission to the neonatal intensive care unit for nonvigorous infants born between 35 and 42 weeks' gestation. However, infants in the umbilical cord milking arm had higher hemoglobin, received less delivery room cardiorespiratory support, had a lower incidence of moderate-to-severe hypoxic-ischemic encephalopathy, and received less therapeutic hypothermia.
AMERICAN JOURNAL OF OBSTETRICS AND GYNECOLOGY
(2023)
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
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
Ulrich Jaeger, Constantine S. Tam, Peter Borchmann, Joseph P. McGuirk, Marianne Johansen, Edmund K. Waller, Samantha Jaglowski, Charalambos Andreadis, Stephen R. Foley, Jason R. Westin, Isabelle Fleury, P. Joy Ho, Stephan Mielke, Takanori Teshima, Gilles Salles, Stephen J. Schuster, Fiona He, Richard T. Maziarz, Sebastian Mayer, Shinichi Makita, Marie J. Kersten, Monalisa Ghosh, Nina Wagner-Johnston, Koji Kato, Paolo Corradini, Hideki Goto, Silvia Colicino, Abhijit Agarwal, Chiara Lobetti-Bodoni, Michael R. Bishop
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
Ulrich Jaeger, Nina Worel, Joseph P. McGuirk, Peter A. Riedell, Isabelle Fleury, Yan Du, Xia Han, David Pearson, Santiago Redondo, Edmund K. Waller
Summary: In the PORTIA trial, it was feasible to add pembrolizumab to tisagenlecleucel for adult patients with r/r DLBCL who had& GE;2 prior lines of therapy and had an Eastern Cooperative Oncology Group performance status of& LE;1. The combination showed a manageable safety profile and had potential efficacy benefits when pembrolizumab was given the day before tisagenlecleucel.
Article
Biochemistry & Molecular Biology
Alvin Wen Choong Chua, Dianyang Guo, Jia Chi Tan, Frances Ting Wei Lim, Chee Tian Ong, Jeyakumar Masilamani, Tony Kiat Hon Lim, William Ying Khee Hwang, Ivor Jiun Lim, Jinmiao Chen, Toan Thang Phan, Xiubo Fan
Summary: This study investigated the therapeutic efficacy and working mechanisms of human umbilical cord lining mesenchymal stromal cells (CL-MSCs) in lupus-prone mice. The results showed that CL-MSCs improved the survival and kidney function of mice by reducing lupus nephritis activity and chronicity, and lymphocyte infiltration. Additionally, CL-MSCs reduced urinary albumin-to-creatinine ratio and the production of anti-dsDNA and isotype antibodies. Mechanistically, CL-MSCs suppressed splenic neutrophils and monocytes/macrophages, reduced proinflammatory cytokines, and modulated immunomodulatory pathways. These findings highlight the potential of CL-MSCs as a novel therapy for systemic lupus erythematosus.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2023)
Article
Hematology
Shakthi T. Bhaskar, Vivek G. Patel, David L. Porter, Stephen J. Schuster, Loretta J. Nastoupil, Miguel-Angel Perales, Ana Alarcon Tomas, Michael R. Bishop, Joseph P. Mcguirk, Richard T. Maziarz, Andy I. Chen, Veronika Bachanova, Joseph E. Maakaron, Peter A. Riedell, Olalekan O. Oluwole
Summary: Chimeric antigen receptor (CAR) T-cell therapy has revolutionized the treatment of aggressive relapsed or refractory large B-cell lymphoma (LBCL). It was found that patients who developed clinically evident cytokine release syndrome (CRS) had clinical outcomes similar to those of patients without CRS.
Article
Hematology
Kitsada Wudhikarn, Ana Alarcon Tomas, Jessica R. Flynn, Sean M. Devlin, Jamie Brower, Veronika Bachanova, Loretta J. Nastoupil, Joseph P. McGuirk, Richard T. Maziarz, Olalekan O. Oluwole, Stephen J. Schuster, David L. Porter, Michael R. Bishop, Peter A. Riedell, Miguel-Angel Perales, Cell Therapy Consortium
Summary: CD19 CAR T-cell therapy is effective in inducing sustained remissions in patients with R/R DLBCL, but can lead to significant toxicities. Treating patients in complete remission at the time of CAR T-cell infusion is feasible, safe, and associated with favorable disease control. Further exploration in larger clinical trials is needed.
Article
Oncology
Yucai Wang, Preetesh Jain, Frederick L. Locke, Matthew J. Maurer, Matthew J. Frank, Javier L. Munoz, Saurabh Dahiya, Amer M. Beitinjaneh, Miriam T. Jacobs, Joseph P. Mcguirk, Julie M. Vose, Andre Goy, Charalambos Andreadis, Brian T. Hill, Kathleen A. Dorritie, Olalekan O. Oluwole, Abhinav Deol, Jonas Paludo, Bijal Shah, Trent Wang, Rahul Banerjee, David B. Miklos, Aaron P. Rapoport, Lazaros Lekakis, Armin Ghobadi, Sattva S. Neelapu, Yi Lin, Michael L. Wang, Michael D. Jain
Summary: This study evaluated the efficacy and toxicity of Brexucabtagene autoleucel (brexu-cel) for relapsed/refractory mantle cell lymphoma (MCL) in the standard-of-care setting. The results showed that the efficacy and toxicity of brexu-cel were consistent with those reported in the ZUMA-2 trial. Tumor-intrinsic features of MCL, and possibly recent bendamustine exposure, may be associated with inferior efficacy outcomes.
JOURNAL OF CLINICAL ONCOLOGY
(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.
Article
Biophysics
Joseph P. McGuirk, Leland Metheny, Luis Pineiro, Mark Litzow, Scott D. Rowley, Batia Avni, Roni Tamari, Hillard M. Lazarus, Jacob M. Rowe, Michal Sheleg, Daniel Rothenstein, Nitsan Halevy, Tsila Zuckerman
Summary: PLX-R18, a placental-derived mesenchymal-like cell product, promotes hematopoietic cell regeneration, maturation, and differentiation, and enhances their migration to peripheral blood. In this study, patients with incomplete hematopoietic recovery post-HCT were treated with escalating doses of PLX-R18. The results showed sustained increases in peripheral blood counts, reduced need for platelet and red blood cell transfusions, and no significant adverse events, indicating the potential of PLX-R18 in improving incomplete hematopoietic recovery post-HCT.
BONE MARROW TRANSPLANTATION
(2023)
Article
Oncology
Jurjen Versluis, Wael Saber, Harrison K. Tsai, Christopher J. Gibson, Laura W. Dillon, Asmita Mishra, Joseph Mcguirk, Richard T. Maziarz, Peter Westervelt, Pranay Hegde, Devdeep Mukherjee, Michael J. Martens, Brent Logan, Mary Horowitz, Christopher S. Hourigan, Ryotaro Nakamura, Corey Cutler, R. Coleman Lindsley
Summary: This study evaluated the impact of gene mutations on the efficacy of allogeneic hematopoietic cell transplantation (HCT) in patients with myelodysplastic syndrome. The results showed that HCT improved overall survival (OS) in patients with TP53 mutations, regardless of TP53 allelic status. Additionally, high-risk patients without TP53 mutations had favorable outcomes when a donor was available.
JOURNAL OF CLINICAL ONCOLOGY
(2023)
Article
Oncology
Christopher J. J. Ferreri, Michelle A. T. Hildebrandt, Hamza Hashmi, Leyla O. O. Shune, Joseph P. P. McGuirk, Douglas W. W. Sborov, Charlotte B. B. Wagner, M. Hakan Kocoglu, Aaron Rapoport, Shebli Atrash, Peter M. M. Voorhees, Jack Khouri, Danai Dima, Aimaz Afrough, Gurbakhash Kaur, Larry D. D. Anderson, Gary Simmons, James A. A. Davis, Nilesh Kalariya, Lauren C. C. Peres, Yi Lin, Murali Janakiram, Omar Nadeem, Melissa Alsina, Frederick L. L. Locke, Surbhi Sidana, Doris K. K. Hansen, Krina K. K. Patel, Omar Alexis Castaneda Puglianini
Summary: Most patients with multiple myeloma experience disease relapse after treatment with a B-cell maturation antigen-targeted therapy (BCMA-TT). Limited data is available on clinical outcomes for patients treated with sequential BCMA-TT. This study analyzed outcomes for patients infused with idecabtagene vicleucel, finding that patients with prior BCMA-TT exposure had lower response rates, shorter duration of response, and shorter progression-free survival compared to those without prior BCMA-TT exposure. However, all patients who received a prior anti-BCMA CAR T responded well to ide-cel.
BLOOD CANCER JOURNAL
(2023)