Intensive care risk and long-term outcomes in pediatric allogeneic hematopoietic cell transplant recipients

Authors: Zinter, Matt S., Brazauskas, Ruta, Strom, Joelle, Chen, Stella, Bo-Subait, Stephanie, Sharma, Akshay, Beitinjaneh, Amer, Dimitrova, Dimana, Guilcher, Greg, Preussler, Jaime, Myers, Kasiani, Bhatt, Neel S., Ringden, Olle, Hematti, Peiman, Hayashi, Robert J., Patel, Sagar, De Oliveira, Satiro Nakamura, Rotz, Seth, Badawy, Sherif M., Nishihori, Taiga, Buchbinder, David, Hamilton, Betty, Savani, Bipin, Schoemans, Hélène, Sorror, Mohamed, Winestone, Lena, Duncan, Christine, Phelan, Rachel, Dvorak, Christopher C.

Source: In Blood Advances 27 February 2024 8(4):1002-1017

Long-term outcomes after gene therapy for adenosine deaminase severe combined immune deficiency

Authors: Reinhardt, Bryanna ^*, Habib, Omar ^*, Shaw, Kit L., Garabedian, Elizabeth, Carbonaro-Sarracino, Denise A., Terrazas, Dayna, Fernandez, Beatriz Campo, De Oliveira, Satiro, Moore, Theodore B., Ikeda, Alan K., Engel, Barbara C., Podsakoff, Gregory M., Hollis, Roger P., Fernandes, Augustine, Jackson, Connie, Shupien, Sally, Mishra, Suparna, Davila, Alejandra, Mottahedeh, Jack, Vitomirov, Andrej, Meng, Wenzhao, Rosenfeld, Aaron M., Roche, Aoife M., Hokama, Pascha, Reddy, Shantan, Everett, John, Wang, Xiaoyan, Luning Prak, Eline T., Cornetta, Kenneth, Hershfield, Michael S., Sokolic, Robert, De Ravin, Suk See, Malech, Harry L., Bushman, Frederic D., Candotti, Fabio, Kohn, Donald B. ^*

Source: In Blood 14 October 2021 138(15):1304-1316

AT1R Activating Autoantibodies in Hematopoietic Stem Cell Transplantation

Authors: Bradford, Kathryn L., Pearl, Meghan, Kohn, Donald B., Weng, Patricia, Yadin, Ora, Bowles, La Vette, De Oliveira, Satiro N., Moore, Theodore B.

Source: In Biology of Blood and Marrow Transplantation November 2020 26(11):2061-2067

Acoustofluidic sonoporation for gene delivery to human hematopoietic stem and progenitor cells

Authors: Belling, Jason N., Heidenreich, Liv K., Tian, Zhenhua, Mendoza, Alexandra M., Chiou, Tzu-Ting, Gong, Yao, Chen, Natalie Y., Young, Thomas D., Wattanatorn, Natcha, Park, Jae Hyeon, Scarabelli, Leonardo, Chiang, Naihao, Takahashi, Jack, Young, Stephen G., Stieg, Adam Z., De Oliveira, Satiro, Huang, Tony Jun, Weiss, Paul S., Jonas, Steven J.

Source: Proceedings of the National Academy of Sciences of the United States of America, 2020 May . 117(20), 10976-10982.

Access URL: https://www.jstor.org/stable/26931160

Development of Hematopoietic Stem Cell-Engineered Invariant Natural Killer T Cell Therapy for Cancer

Authors: Zhu, Yanni, Smith, Drake J., Zhou, Yang, Li, Yan-Ruide, Yu, Jiaji, Lee, Derek, Wang, Yu-Chen, Di Biase, Stefano, Wang, Xi, Hardoy, Christian, Ku, Josh, Tsao, Tasha, Lin, Levina J., Pham, Alexander T., Moon, Heesung, McLaughlin, Jami, Cheng, Donghui, Hollis, Roger P., Campo-Fernandez, Beatriz, Urbinati, Fabrizia, Wei, Liu, Pang, Larry, Rezek, Valerie, Berent-Maoz, Beata, Macabali, Mignonette H., Gjertson, David, Wang, Xiaoyan, Galic, Zoran, Kitchen, Scott G., An, Dong Sung, Hu-Lieskovan, Siwen, Kaplan-Lefko, Paula J., De Oliveira, Satiro N., Seet, Christopher S., Larson, Sarah M., Forman, Stephen J., Heath, James R., Zack, Jerome A., Crooks, Gay M., Radu, Caius G., Ribas, Antoni, Kohn, Donald B., Witte, Owen N., Yang, Lili

Source: In Cell Stem Cell 3 October 2019 25(4):542-557

Site-Specific Gene Editing of Human Hematopoietic Stem Cells for X-Linked Hyper-IgM Syndrome

Authors: Kuo, Caroline Y., Long, Joseph D., Campo-Fernandez, Beatriz, de Oliveira, Satiro, Cooper, Aaron R., Romero, Zulema, Hoban, Megan D., Joglekar, Alok V., Lill, Georgia R., Kaufman, Michael L., Fitz-Gibbon, Sorel, Wang, Xiaoyan, Hollis, Roger P., Kohn, Donald B.

Source: In Cell Reports 29 May 2018 23(9):2606-2616

Umbilical Cord Blood Transplantation in Children with Acute Leukemia: Impact of Conditioning on Transplantation Outcomes

Authors: Eapen, Mary ^*, Kurtzberg, Joanne, Zhang, Mei-Jie, Hattersely, Gareth, Fei, Mingwei, Mendizabal, Adam, Chan, Ka Wah, De Oliveira, Satiro, Schultz, Kirk R., Wall, Donna, Horowitz, Mary M., Wagner, John E.

Source: In Biology of Blood and Marrow Transplantation October 2017 23(10):1714-1721

Fluorinated Silane-Modified Filtroporation Devices Enable Gene Knockout in Human Hematopoietic Stem and Progenitor Cells.

Authors: Frost, Isaura M., Mendoza, Alexandra M., Chiou, Tzu-Ting, Kim, Philseok, Aizenberg, Joanna, Kohn, Donald B., De Oliveira, Satiro N., Weiss, Paul S., Jonas, Steven J.

Source: ACS Applied Materials & Interfaces; 9/6/2023, Vol. 15 Issue 35, p41299-41309, 11p

Long-Term Outcome of Gene Therapy for X-Linked Severe Combined Immunodeficiency (SCID-X1) Using an Enhancer-Deleted Self-Inactivating Gammaretroviral Vector

Authors: Pai, Sung-Yun, Nagarsheth, Nisha ^*, Prockop, Susan, Armant, Myriam ^*, Kohn, Donald B., Marsh, Rebecca A. ^*, Booth, Claire ^*, Everett, John K. ^*, Bleesing, Jack ^*, Chellapandian, Deepak ^*, Dansereau, Colleen ^*, de Oliveira, Satiro ^*, London, Wendy B., Marinovic, M. Angélica ^*, Moore, Theodore B., Oleastro, Matias ^*, O'Toole, Grainne ^*, Vander Lugt, Mark ^*, Urdinez, Luciano ^*, Walkovich, Kelly J., Walter, Jolan E. ^*, Schambach, Axel ^*, Thrasher, Adrian J. ^*, Bushman, Frederic D. ^*, Williams, David A

Source: In Blood 15 November 2022 140 Supplement 1:10643-10645

Successful engraftment of haploidentical bone marrow with post‐transplantation cyclophosphamide in patients with aplastic anemia.

Authors: Dang, Brian Norman¹ (AUTHOR), De Oliveira, Satiro¹ (AUTHOR), Gray, Ashley¹ (AUTHOR), Bowles, LaVette¹ (AUTHOR), Moore, Theodore Bruce¹ (AUTHOR) tbmoore@mednet.ucla.edu

Source: Pediatric Transplantation. Mar2020, Vol. 24 Issue 2, p1-5. 5p.

Subject Terms: *APLASTIC anemia, *BONE marrow, *HEMATOPOIETIC stem cell transplantation, *TOTAL body irradiation, *STEM cell transplantation

3 - Comparison of Outcomes in Patients with Cerebral Adrenoleukodystrophy (CALD) Receiving Elivaldogene Autotemcel (eli-cel; Lenti-D) Gene Therapy in Clinical Trials Versus Those Receiving Allogeneic Hematopoietic Stem Cell Transplant in a Contemporaneous Comparator Study

Authors: Duncan, Christine N., Kühl, Jörn-Sven, Chiesa, Robert, Boelens, Jaap-Jan, Eichler, Florian, Sevin, Caroline, Dalle, Jean-Hugues, De Oliveira, Satiro N., Amartino, Hernan M., Kapoor, Neena, Prasad, Vinod K., Jones, Simon, Algeri, Mattia, Bunin, Nancy J., Diaz-de-Heredia, Cristina, Thrasher, Adrian J., Fernandes, Juliana Folloni, Smith, Nicholas, Shah, Ami J, Locatelli, Franco, Engelen, Marc, Lindemans, Caroline A., Dietz, Andrew C., Pan, Lin, Sieker, Jakob, Williams, David A., Orchard, Paul J.

Source: In Transplantation and Cellular Therapy March 2022 28(3) Supplement:S2-S4

Evaluation of Mobilization, Apheresis, and Conditioning Regimen and Engraftment in Patients Receiving One-Time Gene Therapy with Elivaldogene Autotemcel (Eli-cel) for Cerebral Adrenoleukodystrophy (CALD)

Authors: Duncan, Christine N., Orchard, Paul J., Eichler, Florian S., Kühl, Jörn-Sven, De Oliveira, Satiro N., Thrasher, Adrian J., Chiesa, Robert, Dalle, Jean-Hugues, Shah, Ami J., Locatelli, Franco, Smith, Nicholas J.C., Fernandes, Juliana Folloni, Amartino, Hernán M., Lindemans, Caroline A., Zhao, Jack, Singh, Ajay, Thakar, Himal L., Prasad, Vinod K., Dietz, Andrew C., Williams, David A.

Source: In Transplantation and Cellular Therapy February 2024 30(2) Supplement:S232-S233

Bone Marrow Failure Associated with Short Telomeres and Digenic Variants of Uncertain Significance in Telomere Biology Genes

Authors: Vadivelan, Akhila, Martinez, Julian, De Oliveira, Satiro, Moore, Theodore, Ikeda, Alan, Aubert, Geraldine, Chang, Vivian Y.

Source: In Blood 2 November 2023 142 Supplement 1:5669-5669

254 - Mediport Use As an Acceptable Standard for CAR T Cell Infusion

Authors: Eylon, Maya, Baggott, Christina, Prabhu, Snehit, John, Samuel, Bhatt, Dhruv, Curran, Kevin J., Karras, Nicole, Phillips, Christine L, Satwani, Prakash, Hermiston, Michelle, Baumeister, Susanne H.C., Talano, Julie, MacMillan, Margaret L., Rossoff, Jenna, Bonifant, Challice, Myers, Doug, Rouce, Rayne Helen, Toner, Keri, Driscoll, Timothy Alan, Katsanis, Emmanuel, Salzberg, Dana, Schiff, Deborah, De Oliveira, Satiro N., Capitini, Christian M., Pacenta, Holly L, Pfeiffer, Thomas, Shah, Niketa C., Huynh, Van, Skiles, Jodi L., Laetsch, Theodore W, Fraint, Ellen, McNerney, Kevin O., Quigg, Troy C., Krueger, Joerg, Fabrizio, Vanessa A, Ligon, Dr. John A, Schultz, Liora Michal

Source: In Transplantation and Cellular Therapy February 2023 29(2) Supplement:S195-S196

3 - Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Interim Results from an Ongoing Phase 1/2 Study

Authors: Booth, Claire, Sevilla, Julian, Rao, Gayatri, Lopez, Maria Chitty, Almarza, Elena, Terrazas, Dayna, Zubicaray, Josune, Gonzalez-Vicent, Marta, Chetty, Kritika, Xu-Bayford, Jinhua, O’Toole, Grainne, Fernandes, Augustine, Kuo, Caroline Y., De Oliveira, Satiro N., Mesa-Núñez, Cristina, Moore, Theodore B., Nicoletti, Eileen, Choi, Grace, Zeini, Miriam, Thrasher, Adrian J., Bueren, Juan, Schwartz, Jonathan, Kohn, Donald B.

Source: In Transplantation and Cellular Therapy February 2023 29(2) Supplement:S2-S3

Interim Results from an Ongoing Phase 1/2 Study of Lentiviral-mediated Ex-vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I)

Authors: Booth, Claire ^*, Sevilla, Julián ^*, Rao, Gayatri R. ^*, Chitty Lopez, Maria ^*, Almarza, Elena ^*, Terrazas, Dayna ^*, Zubicaray, Josune ^*, González Vicent, Marta ^*, Chetty, Kritika ^*, O'Toole, Grainne ^*, Xu-Bayford, Jinhua ^*, Nicoletti, Eileen ^*, Fernandes, Augustine ^*, Kuo, Caroline ^*, de Oliveira, Satiro ^*, Moore, Theodore B., Choi, Grace ^*, Zeini, Miriam ^*, Mesa-Núñez, Cristina ^*, Thrasher, Adrian J. ^*, Bueren, Juan A. ^*, Schwartz, Jonathan D., Kohn, Donald B.

Source: In Blood 15 November 2022 140 Supplement 1:7774-7775

Lentiviral Gene Therapy with Low Dose Conditioning for X-Linked SCID Results in Complete Immune Reconstitution and No Evidence of Clonal Expansion

Authors: Booth, Claire ^*, Kohn, Donald B., Armant, Myriam ^*, Prockop, Susan, Buckland, Karen ^*, Chandra, Sharat ^*, Chandrakasan, Shanmuganathan, Chetty, Kritika ^*, Dansereau, Colleen ^*, de Oliveira, Satiro ^*, Everett, John ^*, León Rico, Diego ^*, London, Wendy B., Xu-Bayford, Jin Hua ^*, Moore, Theodore B., Nagarsheth, Nisha ^*, Parikh, Suhag ^*, Terrazas, Dayna ^*, White, Shanna L ^*, Schambach, Axel ^*, Bushman, Frederic D. ^*, Thrasher, Adrian J. ^*, Williams, David A., Pai, Sung-Yun

Source: In Blood 15 November 2022 140 Supplement 1:7770-7771

Same sibling marrow following cord allogeneic transplantation as therapy for second relapse acute promyelocytic leukemia in a pediatric patient.

Authors: De Oliveira, Satiro N.^1,2, Kao, Roy L.², Pham, Andrew³, Smith, LaMarr Taylor², Kempert, Pamela^1,2, Moore, Theodore B.^1,2

Source: Pediatric Transplantation. Mar2016, Vol. 20 Issue 2, p337-341. 5p.

Subject Terms: *LEUKEMIA, *BONE marrow transplantation, *ALTERNATIVE medicine, *ORGAN donors, *PEDIATRICS

A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Interim Results

Authors: Kohn, Donald B., Booth, Claire, Sevilla, Julián, Rao, Gayatri R, Almarza, Elena, Terrazas, Dayna, Nicoletti, Eileen, Fernandes, Augustine, Kuo, Caroline, de Oliveira, Satiro, Moore, Theodore B., Law, Kenneth M, Beard, Brian C, Choi, Grace, Zeini, Miriam, Mesa-Núñez, Cristina, Thrasher, Adrian J., Bueren, Juan A, Schwartz, Jonathan D

Source: In Blood 23 November 2021 138 Supplement 1:2932-2932

Pediatric hematopoietic cell transplantation: Longitudinal trends in body mass index and outcomes.

Authors: Dang, Brian Norman, Wilhalme, Holly, Ch'ng, James, De Oliveira, Satiro, Bowles, LaVette, Moore, Theodore Bruce

Source: Pediatric Transplantation; Nov2020, Vol. 24 Issue 7, p1-9, 9p

Subject Terms: HEMATOPOIETIC stem cell transplantation, BODY mass index, CHILD patients, WEIGHT gain, YOUNG adults