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2. Blood and Marrow Transplant Clinical Trials Network State of the Science Symposium 2021: Looking Forward as the Network Celebrates its 20th Year

Author

Heslop, Helen E, Stadtmauer, Edward A, Levine, John E, Ballen, Karen K, Chen, Yi-Bin, DeZern, Amy E, Eapen, Mary, Hamadani, Mehdi, Hamilton, Betty K, Hari, Parameswaran, Jones, Richard J, Logan, Brent R, Kean, Leslie S, Leifer, Eric S, Locke, Frederick L, Maziarz, Richard T, Nemecek, Eneida R, Pasquini, Marcelo, Phelan, Rachel, Riches, Marcie L, Shaw, Bronwen E, Walters, Mark C, Foley, Amy, Devine, Steven M, and Horowitz, Mary M

Subjects
Stem Cell Research, Clinical Trials and Supportive Activities, Transplantation, Clinical Research, Bone Marrow Transplantation, Clinical Trials as Topic, Hematopoietic Stem Cell Transplantation, Humans, Transplants, BMT, Cell Therapy, Clinical Trial
Abstract

In 2021 the BMT CTN held the 4th State of the Science Symposium where the deliberations of 11 committees concerning major topics pertinent to a particular disease, modality, or complication of transplant, as well as two committees to consider clinical trial design and inclusion, diversity, and access as cross-cutting themes were reviewed. This article summarizes the individual committee reports and their recommendations on the highest priority questions in hematopoietic stem cell transplant and cell therapy to address in multicenter trials.

Published
2021

4. A diagnostic classifier for pediatric chronic graft-versus-host disease: results of the ABLE/PBMTC 1202 study

Author

Cuvelier, Geoffrey D. E., Ng, Bernard, Abdossamadi, Sayeh, Nemecek, Eneida R., Melton, Alexis, Kitko, Carrie L., Lewis, Victor A., Schechter, Tal, Jacobsohn, David A., Harris, Andrew C., Pulsipher, Michael A., Bittencourt, Henrique, Choi, Sung Won, Caywood, Emi H., Kasow, Kimberly A., Bhatia, Monica, Oshrine, Benjamin R., Chaudhury, Sonali, Coulter, Donald, Chewning, Joseph H., Joyce, Michael, Savaşan, Süreyya, Pawlowska, Anna B., Megason, Gail C., Mitchell, David, Cheerva, Alexandra C., Lawitschka, Anita, Ostroumov, Elena, and Schultz, Kirk R.

Published
2023
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5. Anakinra for Refractory Cytokine Release Syndrome or Immune Effector Cell-Associated Neurotoxicity Syndrome after Chimeric Antigen Receptor T Cell Therapy

Author

Gazeau, Nicolas, Liang, Emily C., Wu, Qian “Vicky”, Voutsinas, Jenna M., Barba, Pere, Iacoboni, Gloria, Kwon, Mi, Ortega, Juan Luis Reguera, López-Corral, Lucía, Hernani, Rafael, Ortiz-Maldonado, Valentín, Martínez-Cibrian, Nuria, Martinez, Antonio Perez, Maziarz, Richard T., Williamson, Staci, Nemecek, Eneida R., Shadman, Mazyar, Cowan, Andrew J., Green, Damian J., Kimble, Erik, Hirayama, Alexandre V., Maloney, David G., Turtle, Cameron J., and Gauthier, Jordan

Published
2023
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7. Inherited Disorders

Author

DeMartino, Patrick C., Nemecek, Eneida R., Maziarz, Richard T., editor, and Slater, Susan Schubach, editor

Published
2021
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8. Metabolomic identification of α-ketoglutaric acid elevation in pediatric chronic graft-versus-host disease

Author

Subburaj, Divya, Ng, Bernard, Kariminia, Amina, Abdossamadi, Sayeh, Lauener, Madeline, Nemecek, Eneida R., Rozmus, Jacob, Kharbanda, Sandhya, Kitko, Carrie L., Lewis, Victor A., Schechter-Finklestein, Tal, Jacobsohn, David A., Harris, Andrew C., Pulsipher, Michael A., Bittencourt, Henrique, Choi, Sung Won, Caywood, Emi H., Kasow, Kimberly A., Bhatia, Monica, Oshrine, Benjamin R., Coulter, Donald, Chewning, Joseph H., Joyce, Michael, Pawlowska, Anna B., Megason, Gail C., Lawitschka, Anita, Ostroumov, Elena, Klein Geltink, Ramon, Cuvelier, Geoffrey D.E., and Schultz, Kirk R.

Published
2022
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9. Late cardiovascular morbidity and mortality following pediatric allogeneic hematopoietic cell transplantation

Author

Duncan, Christine N, Brazauskas, Ruta, Huang, Jiaxing, Shaw, Bronwen E, Majhail, Navneet S, Savani, Bipin N, Flowers, Mary ED, Battiwalla, Minoo, Beebe, Kristen, Dietz, Andrew C, Dvorak, Christopher C, Giller, Roger, Jacobsohn, David A, Kletzel, Morris, Martin, Paul L, Nemecek, Eneida R, Nuechterlein, Brandon, Talano, Julie-An, Pulsipher, Michael A, and Baker, K Scott

Subjects
Biomedical and Clinical Sciences, Cardiovascular Medicine and Haematology, Clinical Sciences, Hematology, Cancer, Prevention, Transplantation, Heart Disease, Obesity, Pediatric, Cardiovascular, Good Health and Well Being, Adolescent, Adult, Allografts, Cardiovascular Diseases, Child, Child, Preschool, Combined Modality Therapy, Disease-Free Survival, Female, Follow-Up Studies, Hematologic Neoplasms, Hematopoietic Stem Cell Transplantation, Humans, Incidence, Male, Retrospective Studies, Risk Factors, Severity of Illness Index, Survival Rate, Oncology and Carcinogenesis, Immunology, Cardiovascular medicine and haematology, Oncology and carcinogenesis
Abstract

We analyzed late cardiovascular outcomes of 661 patients who survived at least 2 years from hematopoietic cell transplantation for childhood hematologic malignancy between 1995 and 2008. Center for International Blood and Marrow Transplant Research data was supplemented with surveys focused on cardiotoxicity and potential risk factors. The median duration of follow-up was 97 months (range 24-230). 4.2% of survivors experienced at least one of the primary outcomes including coronary artery disease (0.2%), cerebrovascular accident (0.6%), cardiomyopathy (3%), and cardiac-related death (0.5%). Patients who received anthracycline chemotherapy (HR 4.67, p = 0.036) or cranial or chest radiation (HR 5.58, p

Published
2018

10. Outcomes of Measurable Residual Disease in Pediatric Acute Myeloid Leukemia before and after Hematopoietic Stem Cell Transplant: Validation of Difference from Normal Flow Cytometry with Chimerism Studies and Wilms Tumor 1 Gene Expression

Author

Jacobsohn, David A, Loken, Michael R, Fei, Mingwei, Adams, Alexia, Brodersen, Lisa Eidenschink, Logan, Brent R, Ahn, Kwang Woo, Shaw, Bronwen E, Kletzel, Morris, Olszewski, Marie, Khan, Sana, Meshinchi, Soheil, Keating, Amy, Harris, Andrew, Teira, Pierre, Duerst, Reggie E, Margossian, Steven P, Martin, Paul L, Petrovic, Aleksandra, Dvorak, Christopher C, Nemecek, Eneida R, Boyer, Michael W, Chen, Allen R, Davis, Jeffrey H, Shenoy, Shalini, Savasan, Sureyya, Hudspeth, Michelle P, Adams, Roberta H, Lewis, Victor A, Kheradpour, Albert, Kasow, Kimberly A, Gillio, Alfred P, Haight, Ann E, Bhatia, Monica, Bambach, Barbara J, Haines, Hilary L, Quigg, Troy C, Greiner, Robert J, Talano, Julie-An M, Delgado, David C, Cheerva, Alexandra, Gowda, Madhu, Ahuja, Sanjay, Ozkaynak, Mehmet, Mitchell, David, Schultz, Kirk R, Fry, Terry J, Loeb, David M, and Pulsipher, Michael A

Subjects
Biomedical and Clinical Sciences, Cardiovascular Medicine and Haematology, Pediatric, Rare Diseases, Stem Cell Research - Nonembryonic - Human, Clinical Research, Transplantation, Pediatric Research Initiative, Hematology, Regenerative Medicine, Genetics, Stem Cell Research, Cancer, Pediatric Cancer, Detection, screening and diagnosis, 4.2 Evaluation of markers and technologies, Inflammatory and immune system, Adolescent, Adult, Allografts, Child, Child, Preschool, Disease-Free Survival, Female, Flow Cytometry, Hematopoietic Stem Cell Transplantation, Humans, Infant, Infant, Newborn, Leukemia, Myeloid, Acute, Male, Neoplasm, Residual, Transplantation Conditioning, Transplantation, Homologous, Unrelated Donors, WT1 Proteins, Cytogenetics and molecular genetics, Measurable residual disease, Stem cell transplantation, Laboratory hematology, Clinical Sciences, Immunology, Cardiovascular medicine and haematology
Abstract

We enrolled 150 patients in a prospective multicenter study of children with acute myeloid leukemia undergoing hematopoietic stem cell transplantation (HSCT) to compare the detection of measurable residual disease (MRD) by a "difference from normal" flow cytometry (ΔN) approach with assessment of Wilms tumor 1 (WT1) gene expression without access to the diagnostic specimen. Prospective analysis of the specimens using this approach showed that 23% of patients screened for HSCT had detectable residual disease by ΔN (.04% to 53%). Of those patients who proceeded to transplant as being in morphologic remission, 10 had detectable disease (.04% to 14%) by ΔN. The disease-free survival of this group was 10% (0 to 35%) compared with 55% (46% to 64%, P < .001) for those without disease. The ΔN assay was validated using the post-HSCT specimen by sorting abnormal or suspicious cells to confirm recipient or donor origin by chimerism studies. All 15 patients who had confirmation of tumor detection relapsed, whereas the 2 patients with suspicious phenotype cells lacking this confirmation did not. The phenotype of the relapse specimen was then used retrospectively to assess the pre-HSCT specimen, allowing identification of additional samples with low levels of MRD involvement that were previously undetected. Quantitative assessment of WT1 gene expression was not predictive of relapse or other outcomes in either pre- or post-transplant specimens. MRD detected by ΔN was highly specific, but did not identify most relapsing patients. The application of the assay was limited by poor quality among one-third of the specimens and lack of a diagnostic phenotype for comparison.

Published
2018

11. Tabelecleucel for EBV+ PTLD following allogeneic HCT or SOT in a multicenter expanded access protocol

Author

Nikiforow, Sarah, primary, Whangbo, Jennifer S, additional, Reshef, Ran, additional, Tsai, Donald E, additional, Bunin, Nancy J., additional, Abu-Arja, Rolla F, additional, Mahadeo, Kris Michael, additional, Weng, Wen-Kai, additional, Van Besien, Koen, additional, Loeb, David, additional, Nasta, Sunita D., additional, Nemecek, Eneida R., additional, Zhao, Weizhi, additional, Sun, Yan, additional, Galderisi, Faith C., additional, Wahlstrom, Justin, additional, Mehta, Aditi, additional, Gamelin, Laurence Isabelle, additional, Dinavahi, Rajani, additional, and Prockop, Susan E., additional

Published
2024
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13. Transplantation Outcomes for Children with Hypodiploid Acute Lymphoblastic Leukemia

Author

Mehta, Parinda A, Zhang, Mei-Jie, Eapen, Mary, He, Wensheng, Seber, Adriana, Gibson, Brenda, Camitta, Bruce M, Kitko, Carrie L, Dvorak, Christopher C, Nemecek, Eneida R, Frangoul, Haydar A, Abdel-Azim, Hisham, Kasow, Kimberly A, Lehmann, Leslie, Gonzalez Vicent, Marta, Diaz Pérez, Miguel A, Ayas, Mouhab, Qayed, Muna, Carpenter, Paul A, Jodele, Sonata, Lund, Troy C, Leung, Wing H, and Davies, Stella M

Subjects
Biomedical and Clinical Sciences, Cardiovascular Medicine and Haematology, Pediatric Cancer, Pediatric Research Initiative, Pediatric, Patient Safety, Transplantation, Rare Diseases, Hematology, Cancer, Stem Cell Research - Nonembryonic - Human, Childhood Leukemia, Stem Cell Research, Clinical Research, 6.1 Pharmaceuticals, Evaluation of treatments and therapeutic interventions, Good Health and Well Being, Acute Disease, Adolescent, Aneuploidy, Child, Child, Preschool, Female, Graft vs Host Disease, Hematopoietic Stem Cell Transplantation, Humans, Immunosuppressive Agents, Male, Myeloablative Agonists, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Recurrence, Remission Induction, Retrospective Studies, Risk, Siblings, Survival Analysis, Transplantation Conditioning, Transplantation, Homologous, Treatment Outcome, Unrelated Donors, Hypodiploid acute lymphoblastic leukemia, Hematopoietic stem cell transplantation, Clinical Sciences, Immunology, Cardiovascular medicine and haematology
Abstract

Children with hypodiploid acute lymphoblastic leukemia (ALL) have inferior outcomes despite intensive risk-adapted chemotherapy regimens. We describe 78 children with hypodiploid ALL who underwent hematopoietic stem cell transplantation between 1990 and 2010. Thirty-nine (50%) patients had ≤ 43 chromosomes, 12 (15%) had 44 chromosomes, and 27 (35%) had 45 chromosomes. Forty-three (55%) patients underwent transplantation in first remission (CR1) and 35 (45%) underwent transplantation in ≥ second remission (CR2). Twenty-nine patients (37%) received a graft from a related donor and 49 (63%) from an unrelated donor. All patients received a myeloablative conditioning regimen. The 5-year probabilities of leukemia-free survival, overall survival, relapse, and treatment-related mortality for the entire cohort were 51%, 56%, 27%, and 22%, respectively. Multivariate analysis confirmed that mortality risks were higher for patients who underwent transplantation in CR2 (hazard ratio, 2.16; P = .05), with number of chromosomes ≤ 43 (hazard ratio, 2.15; P = .05), and for those who underwent transplantation in the first decade of the study period (hazard ratio, 2.60; P = .01). Similarly, treatment failure risks were higher with number of chromosomes ≤ 43 (hazard ratio, 2.28; P = .04) and the earlier transplantation period (hazard ratio, 2.51; P = .01). Although survival is better with advances in donor selection and supportive care, disease-related risk factors significantly influence transplantation outcomes.

Published
2015

14. Benefits and challenges with diagnosing chronic and late acute GVHD in children using the NIH consensus criteria

Author

Cuvelier, Geoffrey D.E., Nemecek, Eneida R., Wahlstrom, Justin T., Kitko, Carrie L., Lewis, Victor A., Schechter, Tal, Jacobsohn, David A., Harris, Andrew C., Pulsipher, Michael A., Bittencourt, Henrique, Choi, Sung Won, Caywood, Emi H., Kasow, Kimberly A., Bhatia, Monica, Oshrine, Benjamin R., Flower, Allyson, Chaudhury, Sonali, Coulter, Donald, Chewning, Joseph H., Joyce, Michael, Savaşan, Süreyya, Pawlowska, Anna B., Megason, Gail C., Mitchell, David, Cheerva, Alexandra C., Lawitschka, Anita, West, Lori J., Pan, Bo, Al Hamarneh, Yazid N., Halevy, Anat, and Schultz, Kirk R.

Published
2019
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15. Tabelecleucel for EBV+PTLD after allogeneic HCT or SOT in a multicenter expanded access protocol

Author

Nikiforow, Sarah, Whangbo, Jennifer S., Reshef, Ran, Tsai, Donald E., Bunin, Nancy, Abu-Arja, Rolla, Mahadeo, Kris Michael, Weng, Wen-Kai, Van Besien, Koen, Loeb, David, Nasta, Sunita Dwivedy, Nemecek, Eneida R., Zhao, Weizhi, Sun, Yan, Galderisi, Faith, Wahlstrom, Justin, Mehta, Aditi, Gamelin, Laurence, Dinavahi, Rajani, and Prockop, Susan

Abstract

•In this R/R population with high unmet need, patients responding to tabelecleucel demonstrated a clinically meaningful survival benefit.•Tabelecleucel has a favorable safety profile and shows durable clinical benefit in R/R EBV+PTLD after HCT or SOT.

Published
2024
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16. Ibrutinib Treatment of Pediatric Chronic Graft-versus-Host Disease: Primary Results from the Phase 1/2 iMAGINE Study

Author

Carpenter, Paul A., Kang, Hyoung Jin, Yoo, Keon Hee, Zecca, Marco, Cho, Bin, Lucchini, Giovanna, Nemecek, Eneida R., Schultz, Kirk R., Stepensky, Polina, Chaudhury, Sonali, Oshrine, Benjamin, Khaw, Seong Lin, Harris, Andrew C., Verna, Marta, Zubarovskaya, Liudmila, Lee, Yihua, Wahlstrom, Justin, Styles, Lori, Shaw, Peter J., and Dalle, Jean-Hugues

Published
2022
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17. Treosulfan, Fludarabine, and Low-Dose Total Body Irradiation for Children and Young Adults with Acute Myeloid Leukemia or Myelodysplastic Syndrome Undergoing Allogeneic Hematopoietic Cell Transplantation: Prospective Phase II Trial of the Pediatric Blood and Marrow Transplant Consortium

Author

Nemecek, Eneida R., Hilger, Ralf A., Adams, Alexia, Shaw, Bronwen E., Kiefer, Deidre, Le-Rademacher, Jennifer, Levine, John E., Yanik, Gregory, Leung, Wing, Talano, Julie-An, Haut, Paul, Delgado, David, Kapoor, Neena, Petrovic, Aleksandra, Adams, Roberta, Hanna, Rabi, Rangarajan, Hemalatha, Dalal, Jignesh, Chewning, Joseph, Verneris, Michael R., Epstein, Stacy, Burroughs, Lauri, Perez-Albuerne, Evelio D., Pulsipher, Michael A., and Delaney, Colleen

Published
2018
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20. Anakinra for Refractory Cytokine Release Syndrome or Immune Effector Cell-Associated Neurotoxicity Syndrome after Chimeric Antigen Receptor T Cell Therapy

Author

National Institutes of Health (US), National Cancer Institute (US), Gazeau, Nicolas, Liang, Emily C., Wu, Qian “Vicky”, Voutsinas, Jenna M., Barba, Pere, Iacoboni, Gloria, Kwon, Mi, Reguera-Ortega, Juan Luis, López-Corral, Lucía, Hernani, Rafael, Ortiz-Maldonado, Valentín, Martínez-Cibrián, Nuria, Pérez-Martínez, Antonio, Maziarz, Richard T., Williamson, Staci, Nemecek, Eneida R., Shadman, Mazyar, Cowan, Andrew J., Green, Damian J., Kimble, Erik, Hirayama, Alexandre V., Maloney, David G., Turtle, Cameron J., Gauthier, Jordan, National Institutes of Health (US), National Cancer Institute (US), Gazeau, Nicolas, Liang, Emily C., Wu, Qian “Vicky”, Voutsinas, Jenna M., Barba, Pere, Iacoboni, Gloria, Kwon, Mi, Reguera-Ortega, Juan Luis, López-Corral, Lucía, Hernani, Rafael, Ortiz-Maldonado, Valentín, Martínez-Cibrián, Nuria, Pérez-Martínez, Antonio, Maziarz, Richard T., Williamson, Staci, Nemecek, Eneida R., Shadman, Mazyar, Cowan, Andrew J., Green, Damian J., Kimble, Erik, Hirayama, Alexandre V., Maloney, David G., Turtle, Cameron J., and Gauthier, Jordan

Abstract

Chimeric antigen receptor-engineered (CAR)-T cell therapy remains limited by significant toxicities, including cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS). The optimal management of severe and/or refractory CRS/ICANS remains ill-defined. Anakinra has emerged as a promising agent based on preclinical data, but its safety and efficacy in CAR-T therapy recipients are unknown. The primary objective of this study was to evaluate the safety of anakinra to treat refractory CRS and ICANS after CAR-T therapy. The secondary objective was to evaluate the impact of key treatment-, patient-, and disease-related variables on the time to CRS/ICANS resolution and treatment-related mortality (TRM). We retrospectively analyzed the outcomes of 43 patients with B cell or plasma cell malignancies treated with anakinra for refractory CRS or ICANS at 9 institutions in the United States and Spain between 2019 and 2022. Cause-specific Cox regression was used to account for competing risks. Multivariable cause-specific Cox regression was used to estimate the effect of anakinra dose on outcomes while minimizing treatment allocation bias by including age, CAR-T product, prelymphodepletion (pre-LD) ferritin, and performance status. Indications for anakinra treatment were grade ≥2 ICANS with worsening or lack of symptom improvement despite treatment with high-dose corticosteroids (n = 40) and grade ≥2 CRS with worsening symptoms despite treatment with tocilizumab (n = 3). Anakinra treatment was feasible and safe; discontinuation of therapy because of anakinra-related side effects was reported in only 3 patients (7%). The overall response rate (ORR) to CAR-T therapy was 77%. The cumulative incidence of TRM in the whole cohort was 7% (95% confidence interval [CI], 2% to 17%) at 28 days and 23% (95% CI, 11% to 38%) at 60 days after CAR-T infusion. The cumulative incidence of TRM at day 28 after initiation of anakinra therapy was 0% in the high

Published
2023

21. Phase 3 trial of defibrotide for the treatment of severe veno-occlusive disease and multi-organ failure

Author

Richardson, Paul G., Riches, Marcie L., Kernan, Nancy A., Brochstein, Joel A., Mineishi, Shin, Termuhlen, Amanda M., Arai, Sally, Grupp, Stephan A., Guinan, Eva C., Martin, Paul L., Steinbach, Gideon, Krishnan, Amrita, Nemecek, Eneida R., Giralt, Sergio, Rodriguez, Tulio, Duerst, Reggie, Doyle, John, Antin, Joseph H., Smith, Angela, Lehmann, Leslie, Champlin, Richard, Gillio, Alfred, Bajwa, Rajinder, D’Agostino Sr, Ralph B., Massaro, Joseph, Warren, Diane, Miloslavsky, Maja, Hume, Robin L., Iacobelli, Massimo, Nejadnik, Bijan, Hannah, Alison L., and Soiffer, Robert J.

Published
2016
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22. Skeletal Biology and Late Effects Following Allogeneic Transplantation for Pediatric Hematologic Malignancy: A Ptctc and CIBMTR Multicenter Study

Author

Duncan, Christine N., primary, Chen, Nan, additional, London, Wendy B., additional, Kay-Green, Samantha, additional, Anderson, Lynnette, additional, Ahmed, Ibrahim, additional, Auletta, Jeffery James, additional, Bambach, Barbara, additional, Beebe, Kristen L., additional, Chaudhury, Sonali, additional, Lurie, Robert H., additional, Connelly, James A, additional, Dalal, Jignesh D., additional, Davila, Blachy J, additional, Dvorak, Christopher C., additional, Goebel, W Scott, additional, Hanna, Rabi, additional, Hudspeth, Michelle, additional, Huo, Jeffrey S., additional, Kapoor, Neena, additional, Kasow, Kimberly A., additional, Katsanis, Emmanuel, additional, Lalefar, Nahal, additional, Martin, Paul L., additional, Molinari, Lyndsay, additional, Moore, Theodore B., additional, Nemecek, Eneida R., additional, Nuechterlein, Brandon, additional, Oshrine, Benjamin, additional, Qayed, Muna, additional, Shenoy, Shalini, additional, Vander Lugt, Mark T, additional, Shaw, Bronwen E., additional, Pulsipher, Michael A, additional, and Baker, K. Scott, additional

Published
2023
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23. Transplantation Outcomes for Children with Hypodiploid Acute Lymphoblastic Leukemia

Author

Mehta, Parinda A., Zhang, Mei-Jie, Eapen, Mary, He, Wensheng, Seber, Adriana, Gibson, Brenda, Camitta, Bruce M., Kitko, Carrie L., Dvorak, Christopher C., Nemecek, Eneida R., Frangoul, Haydar A., Abdel-Azim, Hisham, Kasow, Kimberly A., Lehmann, Leslie, Gonzalez Vicent, Marta, Diaz Pérez, Miguel A., Ayas, Mouhab, Qayed, Muna, Carpenter, Paul A., Jodele, Sonata, Lund, Troy C., Leung, Wing H., and Davies, Stella M.

Published
2015
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24. Treosulfan-Based Conditioning and Hematopoietic Cell Transplantation for Nonmalignant Diseases: A Prospective Multicenter Trial

Author

Burroughs, Lauri M., Nemecek, Eneida R., Torgerson, Troy R., Storer, Barry E., Talano, Julie-An, Domm, Jennifer, Giller, Roger H., Shimamura, Akiko, Delaney, Colleen, Skoda-Smith, Suzanne, Thakar, Monica S., Baker, K. Scott, Rawlings, David J., Englund, Janet A., Flowers, Mary E.D., Deeg, H. Joachim, Storb, Rainer, and Woolfrey, Ann E.

Published
2014
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25. Hematopoietic Stem Cell Transplantation

Author

Solh, Hassan El, Al-Nasser, Abdallah, Nemecek, Eneida R., Elzouki, Abdelaziz Y., editor, Harfi, Harb A., editor, Nazer, Hisham M., editor, Stapleton, F. Bruder, editor, Oh, William, editor, and Whitley, Richard J., editor

Published
2012
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26. LNK/SH2B3 As a Novel Driver in Juvenile Myelomonocytic Leukemia

Author

Wintering, Astrid, primary, Hecht, Anna, additional, Meyer, Julia, additional, Wong, Eric Brandon, additional, French, Deborah L, additional, Maguire, Jean Ann, additional, Jobaliya, Chintan, additional, Rojas Vasquez, Marta, additional, Desai, Sunil J., additional, Dulman, Robin Yates, additional, Nemecek, Eneida R., additional, Chehab, Farid F, additional, Loh, Mignon L., additional, and Stieglitz, Elliot, additional

Published
2022
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27. Breakthrough CMV Viremia Outcomes of Letermovir Prophylaxis in Haploidentical Stem Cell Transplant with Post-Transplant Cyclophosphamide

Author

Nguyen, Arthur, primary, Bubalo, Joseph S, additional, Saultz, Jennifer N., additional, Hayes-Lattin, Brandon, additional, Chen, Andy, additional, Cook, Rachel J., additional, Gandhi, Arpita, additional, Leonard, Jessica, additional, Maziarz, Richard T., additional, Migdady, Yazan, additional, Nemecek, Eneida R., additional, and Schachter, Levanto, additional

Published
2022
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28. A diagnostic classifier for pediatric chronic graft-versus-host disease: results of the ABLE / PBMTC 1202 study

Author

Cuvelier, Geoffrey D.E., primary, Ng, Bernard, additional, Abdossamadi, Sayeh, additional, Nemecek, Eneida R., additional, Melton, Alexis, additional, Kitko, Carrie L., additional, Lewis, Victor Anthony, additional, Schechter, Tal, additional, Jacobsohn, David A, additional, Harris, Andrew C., additional, Pulsipher, Michael A, additional, Bittencourt, Henrique, additional, Choi, Sung Won, additional, Caywood, Emi H, additional, Kasow, Kimberly A., additional, Bhatia, Monica, additional, Oshrine, Benjamin R, additional, Chaudhury, Sonali, additional, Coulter, Donald, additional, Chewning, Joseph H, additional, Joyce, Michael, additional, Savaşan, Süreyya, additional, Pawlowska, Anna B, additional, Megason, Gail C, additional, Mitchell, David, additional, Cheerva, Alexandra C, additional, Lawitschka, Anita, additional, Ostroumov, Elena, additional, and Schultz, Kirk R, additional

Published
2022
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29. 50 - Skeletal Biology and Late Effects Following Allogeneic Transplantation for Pediatric Hematologic Malignancy: A Ptctc and CIBMTR Multicenter Study

Author

Duncan, Christine N., Chen, Nan, London, Wendy B., Kay-Green, Samantha, Anderson, Lynnette, Ahmed, Ibrahim, Auletta, Jeffery James, Bambach, Barbara, Beebe, Kristen L., Chaudhury, Sonali, Lurie, Robert H., Connelly, James A, Dalal, Jignesh D., Davila, Blachy J, Dvorak, Christopher C., Goebel, W Scott, Hanna, Rabi, Hudspeth, Michelle, Huo, Jeffrey S., Kapoor, Neena, Kasow, Kimberly A., Katsanis, Emmanuel, Lalefar, Nahal, Martin, Paul L., Molinari, Lyndsay, Moore, Theodore B., Nemecek, Eneida R., Nuechterlein, Brandon, Oshrine, Benjamin, Qayed, Muna, Shenoy, Shalini, Vander Lugt, Mark T, Shaw, Bronwen E., Pulsipher, Michael A, and Baker, K. Scott

Published
2023
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30. Treosulfan, Fludarabine, and 2-Gy Total Body Irradiation Followed by Allogeneic Hematopoietic Cell Transplantation in Patients with Myelodysplastic Syndrome and Acute Myeloid Leukemia

Author

Gyurkocza, Boglarka, Gutman, Jonathan, Nemecek, Eneida R., Bar, Merav, Milano, Filippo, Ramakrishnan, Aravind, Scott, Bart, Fang, Min, Wood, Brent, Pagel, John M., Baumgart, Joachim, Delaney, Colleen, Maziarz, Richard T., Sandmaier, Brenda M., Estey, Elihu H., Appelbaum, Frederick R., Storer, Barry E., and Deeg, Hans Joachim

Published
2014
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31. Tisagenlecleucel in Children and Young Adults with B-Cell Lymphoblastic Leukemia

Author

Maude, Shannon L., Laetsch, Theodore W., Buechner, Jochen, Rives, Susana, Boyer, Michael, Bittencourt, Henrique, Bader, Peter, Verneris, Michael R., Stefanski, Heather E., Myers, Gary D., Qayed, Muna, De Moerloose, Barbara, Hiramatsu, Hidefumi, Schlis, Krysta, Davis, Kara L., Martin, Paul L., Nemecek, Eneida R., Yanik, Gregory A., Peters, Christina, Baruchel, Andre, Boissel, Nicolas, Mechinaud, Francoise, Balduzzi, Adriana, Krueger, Joerg, June, Carl H., Levine, Bruce L., Wood, Patricia, Taran, Tetiana, Leung, Mimi, Mueller, Karen T., Zhang, Yiyun, Sen, Kapildeb, Lebwohl, David, Pulsipher, Michael A., and Grupp, Stephan A.

Published
2018
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32. Prospective Trial of Ibrutinib for the Treatment of Pediatric Chronic Graft-Versus-Host Disease

Author

Carpenter, Paul A., primary, Kang, Hyoung Jin, additional, Yoo, Keon Hee, additional, Zecca, Marco, additional, Cho, Bin, additional, Lucchini, Giovanna, additional, Nemecek, Eneida R., additional, Schultz, Kirk R., additional, Stepensky, Polina, additional, Chaudhury, Sonali, additional, Oshrine, Benjamin, additional, Khaw, Seong Lin, additional, Harris, Andrew C., additional, Verna, Marta, additional, Zubarovskaya, Ludmila, additional, Lee, Yihua, additional, Wahlstrom, Justin, additional, Styles, Lori, additional, Shaw, Peter J., additional, and Dalle, Jean-Hugues, additional

Published
2022
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33. Chronic Kidney Disease Is Common in Survivors of Pediatric Allogeneic Hematopoietic Cell Transplantation: A Pediatric Transplantation and Cellular Therapy Consortium Study

Author

Duncan, Christine N., primary, Baker, Scott, additional, London, Wendy B., additional, Kao, Pei-Chi, additional, Chen, Nan, additional, Auletta, Jeffery James, additional, Ahmed, Ibrahim, additional, Anderson, Lynnette, additional, Bambach, Barbara, additional, Beebe, Kristen L., additional, Chaudhury, Sonali, additional, Connelly, James A, additional, Davila Saldana, Blachy J., additional, Dvorak, Christopher C., additional, Eissa, Hesham, additional, Giller, Roger H., additional, Goebel, W Scott, additional, Hanna, Rabi, additional, Hudspeth, Michelle, additional, Huo, Jeffrey S., additional, Kapoor, Neena, additional, Kasow, Kimberly A., additional, Katsanis, Emmanuel, additional, Lalefar, Nahal, additional, Martin, Paul L., additional, Nemecek, Eneida R., additional, Oshrine, Benjamin, additional, Qayed, Muna, additional, Shenoy, Shalini, additional, Vander Lugt, Mark T., additional, Shaw, Bronwen E., additional, Pulsipher, Michael A., additional, and Hingorani, Sangeeta, additional

Published
2022
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34. Safety and Efficacy Comparison of Two Anakinra Dose Regimens for Refractory CRS or Neurotoxicity after CAR T-Cell Therapy

Author

Gazeau, Nicolas, primary, Barba, Pere, additional, Iacoboni, Gloria, additional, Kwon, Mi, additional, Bailen, Rebeca, additional, Reguera, Juan Luis, additional, López-Corral, Lucía, additional, Hernani, Rafael, additional, Ortiz-Maldonado, Valentin, additional, Pérez-Martínez, Antonio, additional, Maziarz, Richard T, additional, Williamson, Staci, additional, Myers, Jessie, additional, Dwinal, Alexandria Hodges, additional, Nemecek, Eneida R., additional, Shadman, Mazyar, additional, Cowan, Andrew J., additional, Green, Damian J., additional, Chow, Victor A., additional, Hirayama, Alexandre V., additional, Maloney, David G., additional, Turtle, Cameron J., additional, and Gauthier, Jordan, additional

Published
2022
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36. Incidence and Outcomes from Transplant-Associated Thrombotic Microangiopathy in Allogeneic Pediatric Hematopoietic Stem Cell Transplant Recipients before and after the Introduction of Eculizumab: A Single Institution Experience from Oregon Health & Science University

Author

Lerner, Kimberly M, primary, Hulme, Rebecca, additional, Shereck, Evan, additional, Maziarz, Richard T, additional, Nemecek, Eneida R., additional, Chang, Bill H., additional, and DeMartino, Patrick, additional

Published
2022
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37. Outcome of Myeloablative Conditioning and Unrelated Donor Hematopoietic Cell Transplantation for Childhood Acute Lymphoblastic Leukemia in Third Remission

Author

Nemecek, Eneida R., Ellis, Kristin, He, Wensheng, Bunin, Nancy J., Bajwa, Rajinder S., Cheerva, Alexandra, Cairo, Mitchell S., Dvorak, Christopher, Duval, Michel, Davies, Stella, Eapen, Mary, Gross, Thomas G., Hussein, Ayad A., MacMillan, Margaret L., Mehta, Parinda A., Pulsipher, Michael A., Seber, Adriana, Woolfrey, Ann E., Frangoul, Haydar A., and Carpenter, Paul A.

Published
2011
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38. Conditioning with Treosulfan and Fludarabine followed by Allogeneic Hematopoietic Cell Transplantation for High-Risk Hematologic Malignancies

Author

Nemecek, Eneida R., Guthrie, Katherine A., Sorror, Mohamed L., Wood, Brent L., Doney, Kristine C., Hilger, Ralf A., Scott, Bart L., Kovacsovics, Tibor J., Maziarz, Richard T., Woolfrey, Ann E., Bedalov, Antonio, Sanders, Jean E., Pagel, John M., Sickle, Eileen J., Witherspoon, Robert, Flowers, Mary E., Appelbaum, Frederick R., and Deeg, H. Joachim

Published
2011
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39. Randomized Phase III BMT CTN Trial of Calcineurin Inhibitor–Free Chronic Graft-Versus-Host Disease Interventions in Myeloablative Hematopoietic Cell Transplantation for Hematologic Malignancies

Author

Luznik, Leo, primary, Pasquini, Marcelo C., additional, Logan, Brent, additional, Soiffer, Robert J., additional, Wu, Juan, additional, Devine, Steven M., additional, Geller, Nancy, additional, Giralt, Sergio, additional, Heslop, Helen E., additional, Horowitz, Mary M., additional, Jones, Richard J., additional, Litzow, Mark R., additional, Mendizabal, Adam, additional, Muffly, Lori, additional, Nemecek, Eneida R., additional, O'Donnell, Lynn, additional, O'Reilly, Richard J., additional, Palencia, Raquel, additional, Schetelig, Johannes, additional, Shune, Leyla, additional, Solomon, Scott R., additional, Vasu, Sumithira, additional, Ho, Vincent T., additional, and Perales, Miguel-Angel, additional

Published
2022
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41. BMT CTN State of the Science Symposium 2021: Looking Forward as the Network Celebrates its 20(th) Year

Author

Heslop, Helen E, Stadtmauer, Edward A, Levine, John E, Ballen, Karen K, Chen, Yi-Bin, DeZern, Amy E, Eapen, Mary, Hamadani, Mehdi, Hamilton, Betty K, Hari, Parameswaran, Jones, Richard J, Logan, Brent R, Kean, Leslie S, Leifer, Eric S, Locke, Frederick L, Maziarz, Richard T, Nemecek, Eneida R, Pasquini, Marcelo, Phelan, Rachel, Riches, Marcie L, Shaw, Bronwen E, Walters, Mark C, Foley, Amy, Devine, Steven M, and Horowitz, Mary M

Subjects
Clinical Trials as Topic, Hematopoietic Stem Cell Transplantation, Humans, Transplants, Article, Bone Marrow Transplantation
Abstract

In 2021 the BMT CTN held the 4th State of the Science Symposium where the deliberations of 11 committees concerning major topics pertinent to a particular disease, modality, or complication of transplant, as well as two committees to consider clinical trial design and inclusion, diversity, and access as cross-cutting themes were reviewed. This article summarizes the individual committee reports and their recommendations on the highest priority questions in hematopoietic stem cell transplant and cell therapy to address in multicenter trials.

Published
2021

42. Safety and Efficacy of Two Anakinra Dose Regimens for Refractory CRS or Icans after CAR T-Cell Therapy

Author

Gazeau, Nicolas, primary, Barba, Pere, additional, Iacoboni, Gloria, additional, Kwon, Mi, additional, Bailen, Rebeca, additional, Reguera, Juan Luis, additional, Corral, Lucía López, additional, Hernani, Rafael, additional, Ortiz-Maldonado, Valentín, additional, Perez-Martinez, Antonio, additional, Maziarz, Richard T., additional, Williamson, Staci, additional, Nemecek, Eneida R., additional, Shadman, Mazyar, additional, Coman, Andrew, additional, Green, Damian J., additional, Chow, Victor A., additional, Hirayama, Alexandre V., additional, Maloney, David G., additional, Turtle, Cameron J., additional, and Gauthier, Jordan, additional

Published
2021
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45. Access and Outcomes of Minority Health Populations Receiving Commercial Anti-CD19 CART Therapy for Non-Hodgkin Lymphoma

Author

Ghilardi, Guido, Williamson, Staci, Pajarillo, Raymone, Paruzzo, Luca, Chen, Linhui, Grady, Connor, Doucette, Abigail, Nemecek, Eneida R, Gabrielli, Giulia, Barta, Stefan K., Svoboda, Jakub, Chong, Elise A., Yelton, Rebecca, Nasta, Dwivedy S., Landsburg, Daniel J., Ugwuanyi, Ositadimma, Chen, Andy, Schachter, Levanto, White, Griffin W, Ballard, Hatcher, Weber, Elizabeth, Curley, Cara, Porter, David L., Garfall, Alfred L., Hwang, Wei-Ting, Guerra, Carmen E, Maziarz, Richard T, Schuster, Stephen J, and Ruella, Marco

Published
2023
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49. A Systematic Framework to Rapidly Obtain Data on Patients with Cancer and COVID-19: CCC19 Governance, Protocol, and Quality Assurance

Author

Abidi, Maheen, primary, Aboulafia, David M., additional, Accordino, Melissa K., additional, Acoba, Jared D., additional, Ahluwalia, Manmeet S., additional, Ahmad, Syed A., additional, Ajmera, Archana, additional, Alimohamed, Saif I., additional, Altman, Jessica, additional, Angevine, Anne H., additional, Bakouny, Ziad, additional, Bar, Michael H., additional, Bardia, Aditya, additional, Barnholtz-Sloan, Jill S., additional, Barrow McCollough, Briana, additional, Bashir, Babar, additional, Batist, Gerald, additional, Bekaii-Saab, Tanios S., additional, Berg, Stephanie, additional, Bernicker, Eric H., additional, Bhutani, Divaya, additional, Bilen, Mehmet A., additional, Bindal, Poorva, additional, Bishnoi, Rohit, additional, Blau, Sibel, additional, Bohachek, Pamela, additional, Boland, Genevieve, additional, Bonnen, Mark, additional, Bouchard, Gabrielle, additional, Bouganim, Nathaniel, additional, Bowles, Daniel W., additional, Busser, Fiona J., additional, Butt, Omar, additional, Cabal, Angelo, additional, Cabalona, Wilhelmina D., additional, Cabebe, Elwyn C., additional, Caimi, Paolo, additional, Campian, Jian L., additional, Carducci, Theresa M., additional, Chen, James L., additional, Cheng, Alex, additional, Chism, David D., additional, Choueiri, Toni K., additional, Clark, Melanie J., additional, Clement, Jessica M., additional, Connors, Jean M., additional, Cook, Erin, additional, Curran, Catherine R., additional, Daher, Ahmad, additional, Dailey, Mark E., additional, Davis, Elizabeth J., additional, Dawsey, Scott J., additional, Deeken, John F., additional, Del Prete, Salvatore A., additional, Demetri, George D., additional, Desai, Aakash, additional, Doroshow, Deborah B., additional, Durbin, Eric B., additional, Egan, Pamela C., additional, Elias, Rawad, additional, Elkrief, Arielle, additional, Elms, Destry J., additional, Elshoury, Amro, additional, Faller, Bryan, additional, Farmakiotis, Dimitrios, additional, Fecher, Leslie A., additional, Feldman, Lawrence E., additional, Ferrario, Cristiano, additional, Fiala, Mark A., additional, Flora, Daniel B., additional, French, Benjamin, additional, Friese, Christopher R., additional, Fu, Julie C., additional, Gadgeel, Shirish M., additional, Gainor, Justin, additional, Galsky, Matthew D., additional, Gantt, Gerald, additional, Garcia, Jorge A., additional, Gartrell, Benjamin A., additional, Gatti-Mays, Margaret E., additional, Gill, David M., additional, Gillaspie, Erin A., additional, Giordano, Antonio, additional, Glace, (Mary) Grace, additional, Glover, Michael J., additional, Goel, Sanjay, additional, Graber, Jerome J., additional, Griffiths, Elizabeth A., additional, Grivas, Petros, additional, Grover, Punita, additional, Gulati, Anthony P., additional, Gulati, Shuchi, additional, Gupta, Shilpa, additional, Gurley, Michael, additional, Hafez, Navid, additional, Halabi, Susan, additional, Halfdanarson, Thorvardur R., additional, Halmos, Balazs, additional, Hausrath, Daniel J., additional, Hawley, Jessica E., additional, Hennessy, Cassandra, additional, Herbst, Roy S., additional, Hershman, Dawn L., additional, Hoppenot, Claire, additional, Hoskins, Kent F., additional, Hoyo-Ulloa, Irma, additional, Hsu, Emily, additional, Hsu, Chih-Yuan, additional, Hwang, Clara, additional, Islam, Jessica Yasmine, additional, Jabbour, Salma K., additional, Jani, Chinmay, additional, Jha, Alokkumar, additional, Jhawar, Sachin R., additional, Johnson, Douglas B., additional, Joshi, Monika, additional, Kasi, Anup, additional, Kelleher, Kaitlin, additional, Kennecke, Hagen F., additional, Khaki, Ali Raza, additional, Khan, Hina, additional, Khan, Mahir, additional, Kharofa, Jordan, additional, Kloecker, Goetz, additional, Knoble, Jeanna L., additional, Kulkarni, Amit A., additional, Kumar, Vaibhav, additional, Lammers, Philip E., additional, Leighton, John C., additional, Lemmon, Christopher A., additional, Lewis, Mark A., additional, Li, Ang, additional, Li, Xuanyi, additional, Liu, Stephen V., additional, Lo, K.M., additional, Loaiza-Bonilla, Arturo, additional, Logan, Barbara B., additional, Loggers, Elizabeth T., additional, de Lima Lopes, Gilberto, additional, Loree, Jonathan M., additional, LoRusso, Patricia, additional, Low, Clarke A., additional, Lustberg, Maryam B., additional, Lyman, Gary H., additional, Lynch, Ryan C., additional, Madhavan, Subha, additional, Mahadevan, Daruka, additional, Mahmood, Sana Z., additional, Mansoor, Abdul-Hai, additional, Marcum, Michelle, additional, Markham, Merry-Jennifer, additional, Mashru, Sandeep H., additional, Masters, Tyler, additional, Mavromatis, Blanche H., additional, McKay, Rana R., additional, McNair, Christopher, additional, McWeeney, Shannon, additional, Menendez, Alvaro G., additional, Menon, Harry, additional, Mesa, Ruben A., additional, Mico, Vasil, additional, Miller, Chaim, additional, Mishra, Sanjay, additional, Monahan, Ryan S., additional, Morgans, Alicia K., additional, Mulcahy, Mary F., additional, Mundt, Daniel, additional, Mushtaq, Sarah, additional, Nagaraj, Gayathri, additional, Nagle, Sarah, additional, Nakasone, Elizabeth S., additional, Nakayama, John M., additional, Nelson, Heather H., additional, Nemecek, Eneida R., additional, Nguyen, Ryan H., additional, Nizam, Amanda, additional, Nohria, Anju, additional, Nuzzo, Pier Vitale, additional, Ohri, Nitin, additional, Olszewski, Adam J., additional, Owenby, Susie, additional, Painter, Corrie A., additional, Palmer, Joshua D., additional, Panagiotou, Orestis A., additional, Park, Cathleen, additional, Pasquinelli, Mary M., additional, Patel, Jaymin M., additional, Patel, Kanishka G., additional, Peddi, Prakash, additional, Pennell, Nathan A., additional, Peters, Solange, additional, Pilar, Christine, additional, Pillainayagam, Clement, additional, Puc, Matthew, additional, Ramirez, Amelie G., additional, Rathmann, Joerg, additional, Ravindranathan, Deepak, additional, Reid, Sonya A., additional, Reuben, Daniel Y., additional, Revankar, Sanjay G., additional, Reynolds, Kerry L., additional, Rho, Young Soo, additional, Rhodes, Terence D., additional, Rice, Robert L., additional, Riess, Jonathan, additional, Rini, Brian I., additional, Rink, Cameron, additional, Rosen, Lane R., additional, Rosenstein, Lori J., additional, Rosovsky, Rachel P., additional, Routy, Bertrand, additional, Rovito, Marc A., additional, Rubinstein, Samuel M., additional, Saif, M. Wasif, additional, Salazar, Mary, additional, Santos Dutra, Miriam, additional, Schapira, Lidia, additional, Schmidt, Andrew L., additional, Schroeder, Brett A., additional, Schwartz, Gary K., additional, Schwartz, Candice, additional, Schweizer, Michael T., additional, Serrano, Oscar K., additional, Shafer, Danielle A., additional, Shah, Pankil K., additional, Shah, Dimpy, additional, Shah, Mansi R., additional, Shah, Sumit A., additional, Shah, Chintan, additional, Shaw, Grace, additional, Shaya, Justin A., additional, Shyr, Yu, additional, Slosky, David A., additional, Smits, Melissa, additional, Solorzano, Carmen C., additional, Stauffer, Karen, additional, Stockerl-Goldstein, Keith E., additional, Stover, Daniel G., additional, Stratton, Jamie, additional, Stratton, Catherine, additional, Streckfuss, Mitrianna, additional, Subbiah, Suki, additional, Tachiki, Lisa, additional, Tadesse, Eyob, additional, Thompson, Michael A., additional, Topaloglu, Umit, additional, Tucker, Matthew D., additional, Van Allen, Eliezer M., additional, Van Loon, Susan, additional, Vega-Luna, Karen, additional, Venepalli, Neeta K., additional, Verma, Amit, additional, Vikas, Praveen, additional, Vinayak, Shaveta, additional, Vinh, Donald C., additional, Wagner, Michael J., additional, Wall, Sarah, additional, Wang, Lucy L., additional, Warner, Jeremy L., additional, Wehbe, Firas H., additional, Weinstein, Paul L., additional, Weiss, Matthias, additional, Weissmann, Lisa B., additional, Wildes, Tanya M., additional, Williams, Nicole, additional, Wise-Draper, Trisha M., additional, Wood, William A., additional, Wu, Julie Tsu-Yu, additional, Wulff-Burchfield, Elizabeth M., additional, Xie, Zhuoer, additional, Xu, Wenxin, additional, Yeh, Albert C., additional, Yu, Irene S., additional, Yu, Peter Paul, additional, Zacks, Rosemary, additional, Zaman, Qamar Ul, additional, Zaren, Howard, additional, Zhang, Tian, additional, Zhou, Alice Y., additional, Zhu, Huili, additional, Zon, Rebecca L., additional, and Zubiri, Leyre, additional

Published
2020
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