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3,088 results on '"Litzow, Mark R"'

1. Measurable residual mutated IDH1 before allogeneic transplant for acute myeloid leukemia

Author

Gui, Gege, Ravindra, Niveditha, Hegde, Pranay S., Andrew, Georgia, Mukherjee, Devdeep, Wong, Zoë, Auletta, Jeffery J., El Chaer, Firas, Chen, Evan C., Chen, Yi-Bin, Corner, Adam, Devine, Steven M., Iyer, Sunil G., Jimenez Jimenez, Antonio Martin, De Lima, Marcos J. G., Litzow, Mark R., Kebriaei, Partow, Saber, Wael, Spellman, Stephen R., Zeger, Scott L., Page, Kristin M., Dillon, Laura W., and Hourigan, Christopher S. more...

Published
2025
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2. Measurable residual FLT3 tyrosine kinase domain mutations before allogeneic transplant for acute myeloid leukemia

Author

Hegde, Pranay S., Andrew, Georgia, Gui, Gege, Ravindra, Niveditha, Mukherjee, Devdeep, Wong, Zoë C., Auletta, Jeffery J., El Chaer, Firas, Corner, Adam, Devine, Steven M., Jimenez Jimenez, Antonio Martin, De Lima, Marcos J. G., Litzow, Mark R., Kebriaei, Partow, Saber, Wael, Spellman, Stephen R., Zeger, Scott L., Page, Kristin M., Dillon, Laura W., and Hourigan, Christopher S. more...

Published
2025
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3. Investigation of inherited noncoding genetic variation impacting the pharmacogenomics of childhood acute lymphoblastic leukemia treatment

Author

Bhattarai, Kashi Raj, Mobley, Robert J., Barnett, Kelly R., Ferguson, Daniel C., Hansen, Baranda S., Diedrich, Jonathan D., Bergeron, Brennan P., Yoshimura, Satoshi, Yang, Wenjian, Crews, Kristine R., Manring, Christopher S., Jabbour, Elias, Paietta, Elisabeth, Litzow, Mark R., Kornblau, Steven M., Stock, Wendy, Inaba, Hiroto, Jeha, Sima, Pui, Ching-Hon, Cheng, Cheng, Pruett-Miller, Shondra M., Relling, Mary V., Yang, Jun J., Evans, William E., and Savic, Daniel more...

Published
2024
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5. Comparing venetoclax in combination with hypomethylating agents to hypomethylating agent-based therapies for treatment naive TP53-mutated acute myeloid leukemia: results from the Consortium on Myeloid Malignancies and Neoplastic Diseases (COMMAND)

Author

Badar, Talha, Nanaa, Ahmad, Atallah, Ehab, Shallis, Rory M., Guilherme, Sacchi de Camargo Correia, Goldberg, Aaron D., Saliba, Antoine N., Patel, Anand, Bewersdorf, Jan P., DuVall, Adam S., Bradshaw, Danielle, Abaza, Yasmin, Murthy, Guru Subramanian Guru, Palmisiano, Neil, Zeidan, Amer M., Kota, Vamsi, and Litzow, Mark R. more...

Published
2024
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6. Entospletinib with decitabine in acute myeloid leukemia with mutant TP53 or complex karyotype: A phase 2 substudy of the Beat AML Master Trial

Author

Duong, Vu H, Ruppert, Amy S, Mims, Alice S, Borate, Uma, Stein, Eytan M, Baer, Maria R, Stock, Wendy, Kovacsovics, Tibor, Blum, William, Arellano, Martha L, Schiller, Gary J, Olin, Rebecca L, Foran, James M, Litzow, Mark R, Lin, Tara L, Patel, Prapti A, Foster, Matthew C, Redner, Robert L, Al‐Mansour, Zeina, Cogle, Christopher R, Swords, Ronan T, Collins, Robert H, Vergilio, Jo‐Anne, Heerema, Nyla A, Rosenberg, Leonard, Yocum, Ashley O, Marcus, Sonja, Chen, Timothy, Druggan, Franchesca, Stefanos, Mona, Gana, Theophilus J, Shoben, Abigail B, Druker, Brian J, Burd, Amy, Byrd, John C, Levine, Ross L, and Boyiadzis, Michael M more...

Subjects
Biomedical and Clinical Sciences, Oncology and Carcinogenesis, Childhood Leukemia, Pediatric, Rare Diseases, Hematology, Cancer, Clinical Research, Orphan Drug, Aging, Clinical Trials and Supportive Activities, Pediatric Cancer, 6.1 Pharmaceuticals, Humans, Decitabine, Tumor Suppressor Protein p53, Leukemia, Myeloid, Acute, Karyotype, Treatment Outcome, Antineoplastic Combined Chemotherapy Protocols, acute myeloid leukemia, decitabine, entospletinib, hypomethylating agents, tumor protein p53, Public Health and Health Services, Oncology & Carcinogenesis, Oncology and carcinogenesis, Public health
Abstract

BackgroundPatients with acute myeloid leukemia (AML) who have tumor protein p53 (TP53) mutations or a complex karyotype have a poor prognosis, and hypomethylating agents are often used. The authors evaluated the efficacy of entospletinib, an oral inhibitor of spleen tyrosine kinase, combined with decitabine in this patient population.MethodsThis was a multicenter, open-label, phase 2 substudy of the Beat AML Master Trial (ClinicalTrials.gov identifier NCT03013998) using a Simon two-stage design. Eligible patients aged 60 years or older who had newly diagnosed AML with mutations in TP53 with or without a complex karyotype (cohort A; n = 45) or had a complex karyotype without TP53 mutation (cohort B; n = 13) received entospletinib 400 mg twice daily with decitabine 20 mg/m2 on days 1-10 every 28 days for up to three induction cycles, followed by up to 11 consolidation cycles, in which decitabine was reduced to days 1-5. Entospletinib maintenance was given for up to 2 years. The primary end point was complete remission (CR) and CR with hematologic improvement by up to six cycles of therapy.ResultsThe composite CR rates for cohorts A and B were 13.3% (95% confidence interval, 5.1%-26.8%) and 30.8% (95% confidence interval, 9.1%-61.4%), respectively. The median duration of response was 7.6 and 8.2 months, respectively, and the median overall survival was 6.5 and 11.5 months, respectively. The study was stopped because the futility boundary was crossed in both cohorts.ConclusionsThe combination of entospletinib and decitabine demonstrated activity and was acceptably tolerated in this patient population; however, the CR rates were low, and overall survival was short. Novel treatment strategies for older patients with TP53 mutations and complex karyotype remain an urgent need. more...

Published
2023

7. DNTT-mediated DNA damage response drives inotuzumab ozogamicin resistance in B-cell acute lymphoblastic leukemia

Author

Escherich, Carolin S., Moriyama, Takaya, Li, Zhenhua, Hsiao, Yu-Chih, Yang, Wenjian, Li, Yizhen, Reyes, Noemi, Walker, Megan, Budhraja, Amit, Bhatara, Sheetal, Diaz-Flores, Ernesto, Stock, Wendy, Paietta, Elisabeth, Konopleva, Marina Y., Kornblau, Steven M., Litzow, Mark R., Inaba, Hiroto, Pui, Ching-Hon, Opferman, Joseph T., Loh, Mignon L., Yu, Jiyang, O’Brien, Maureen M., Evans, William E., and Yang, Jun J. more...

Published
2025
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8. Measurable residual disease and posttransplantation gilteritinib maintenance for patients with FLT3-ITD–mutated AML

Author

Levis, Mark J., Hamadani, Mehdi, Logan, Brent R., Jones, Richard J., Singh, Anurag K., Litzow, Mark R., Wingard, John R., Papadopoulos, Esperanza B., Perl, Alexander E., Soiffer, Robert J., Ustun, Celalettin, Oshima, Masumi Ueda, Uy, Geoffrey L., Waller, Edmund K., Vasu, Sumithira, Solh, Melhem, Mishra, Asmita, Muffly, Lori S., Kim, Hee-Je, Stelljes, Matthias, Najima, Yuho, Onozawa, Masahiro, Thomson, Kirsty, Nagler, Arnon, Wei, Andrew H., Marcucci, Guido, Chen, Caroline, Hasabou, Nahla, Rosales, Matt, Hill, Jason, Gill, Stanley C., Nuthethi, Rishita, King, Denise, Mendizabel, Adam, Devine, Steven M., Horowitz, Mary M., and Chen, Yi-Bin more...

Published
2025
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10. A randomized phase III study of standard versus high-dose cytarabine with or without vorinostat for AML

Author

Garcia-Manero, Guillermo, Podoltsev, Nikolai A., Othus, Megan, Pagel, John M., Radich, Jerald P., Fang, Min, Rizzieri, David A., Marcucci, Guido, Strickland, Stephen A., Litzow, Mark R., Savoie, M. Lynn, Medeiros, Bruno C., Sekeres, Mikkael A., Lin, Tara L., Uy, Geoffrey L., Powell, Bayard L., Kolitz, Jonathan E., Larson, Richard A., Stone, Richard M., Claxton, David, Essell, James, Luger, Selina M., Mohan, Sanjay R., Moseley, Anna, Appelbaum, Frederick R., and Erba, Harry P. more...

Published
2024
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11. A Multicenter Analysis of Allogeneic Transplant Outcomes in Adults with Philadelphia-Like B-Cell Acute Lymphoblastic Leukemia in First Complete Remission

Author

Rahman, Zaid Abdel, Othman, Tamer, Saliba, Rima M., Vanegas, Yenny Alejandra Moreno, Mohty, Razan, Ledesma, Celina, Rondon, Gabriela, Jain, Nitin, Jabbour, Elias, Pullarkat, Vinod, Alkhateeb, Hassan B, Kantarjian, Hagop M., Greipp, Patricia T., Nakamura, Ryotaro, Kharfan-Dabaja, Mohamed A., Champlin, Richard E., Forman, Stephen J., Shpall, Elizabeth J., Litzow, Mark R., Foran, James M., Aldoss, Ibrahim, Koller, Paul B., and Kebriaei, Partow more...

Published
2024
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12. Health-related quality of life with gilteritinib vs placebo posttransplant for FLT3-ITD+ acute myeloid leukemia

Author

Hamilton, Betty K., Pandya, Bhavik J., Ivanescu, Cristina, Elsouda, Dina, Hamadani, Mehdi, Chen, Yi-Bin, Levis, Mark J., Ueda Oshima, Masumi, Litzow, Mark R., Soiffer, Robert J., Ustun, Celalettin, Perl, Alexander E., Singh, Anurag K., Geller, Nancy, Hasabou, Nahla, Rosales, Matt, Cella, David, Corredoira, Laura, Pestana, Carolina, Horowitz, Mary M., and Logan, Brent more...

Published
2024
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13. Disparities in trial enrollment and outcomes of Hispanic adolescent and young adult acute lymphoblastic leukemia

Author

Muffly, Lori, Yin, Jun, Jacobson, Sawyer, Wall, Anna, Quiroz, Elisa, Advani, Anjali S, Luger, Selina M, Tallman, Martin S, Litzow, Mark R, Foster, Matthew C, Erba, Harry P, Appelbaum, Frederick R, Larson, Richard A, Keegan, Theresa HM, and Stock, Wendy more...

Subjects
Clinical Research, Rare Diseases, Pediatric Cancer, Clinical Trials and Supportive Activities, Cancer, Pediatric, Hematology, Adolescent, Clinical Trials as Topic, Healthcare Disparities, Hispanic or Latino, Humans, Incidence, Patient Participation, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Registries, United States, Young Adult
Abstract

In this secondary analysis of Hispanic adolescents and young adults (AYA) with acute lymphoblastic leukemia (ALL) treated on Cancer and Leukemia Group B (CALGB) 10403, we evaluated outcomes and geographic enrollment patterns relative to US population data. We used demographic, clinical, and survival data on AYAs enrolled on CALGB 10403 (N = 295, 2007-2012). Surveillance, Epidemiology, and End Results registries provided overall survival (OS) for US AYA ALL by ethnicity/race. North American Association of Cancer Registries provided AYA ALL incidence overall and proportion among Hispanics by US state. Of AYAs enrolled on CALGB 10403, 263 (89%) reported ethnicity/race: 45 (17%) Hispanic, 172 (65%) non-Hispanic White (NHW), 25 (10%) non-Hispanic Black (NHB), and 21 (8%) other. Compared with NHWs, Hispanic and NHB patients had lower household income, and Hispanic patients were more likely to harbor high-risk CRLF2 aberrations. Relative to US estimates, where Hispanic patients represented 46% of newly diagnosed AYA ALL patients and experienced inferior OS compared with NHW (P < .001), Hispanic AYAs on CALGB 10403 did as well as NHW patients (3 year OS, 75% vs 74%; P = NS). Hispanic patients also had higher rates of protocol completion (P = .05). Enrollments on CALGB 10403 differed relative to the distribution of Hispanic AYA ALL in the United States: enrollment was highest in the Midwest; t and only 15% of enrollees were from states with a high proportion of Hispanic AYA ALL patients. In summary, Hispanic patients treated on CALGB 10403 did as well as NHWs and better than population estimates. Geographical misalignment between trial sites and disease epidemiology may partially explain the lower-than-expected enrollment of Hispanic AYA ALL patients. more...

Published
2022

14. Haploidentical vs. sibling, unrelated, or cord blood hematopoietic cell transplantation for acute lymphoblastic leukemia

Author

Wieduwilt, Matthew J, Metheny, Leland, Zhang, Mei-Jie, Wang, Hai-Lin, Estrada-Merly, Noel, Marks, David I, Al-Homsi, A Samar, Muffly, Lori, Chao, Nelson J, Rizzieri, David, Gale, Robert Peter, Gadalla, Shahinaz M, Cairo, Mitchell S, Mussetti, Alberto, Gore, Steven D, Bhatt, Vijaya Raj, Patel, Sagar S, Michelis, Fotios V, Inamoto, Yoshihiro, Badawy, Sherif M, Copelan, Edward, Palmisiano, Neil, Kharfan-Dabaja, Mohamed A, Lazarus, Hillard M, Ganguly, Siddhartha, Bredeson, Christopher N, Perez, Miguel Angel Diaz, Cassaday, Ryan, Savani, Bipin N, Ballen, Karen Kuhn, Martino, Rodrigo, Wirk, Baldeep, Bacher, Ulrike, Aljurf, Mahmoud, Bashey, Asad, Murthy, Hemant S, Yared, Jean A, Aldoss, Ibrahim, Farhadfar, Nosha, Liu, Hongtao, Abdel-Azim, Hisham, Waller, Edmund K, Solh, Melhem, Seftel, Matthew, van der Poel, Marjolein WM, Grunwald, Michael Richard, Liesveld, Jane L, Kamble, Rammurti T, McGuirk, Joseph P, Munker, Reinhold, Cahn, Jean-Yves, Lee, Jong Wook, Freytes, Cesar O, Krem, Maxwell, Winestone, Lena E, Gergis, Usama, Nathan, Sunita, Olsson, Richard F, Verdonck, Leo F, Sharma, Akshay, Ringden, Olle, Friend, Brian D, Cerny, Jan, Choe, Hannah K, Chhabra, Saurabh, Nishihori, Taiga, Seo, Sachiko, George, Biju, Baxter-Lowe, Lee Ann, Hildebrandt, Gerhard C, de Lima, Marcos, Litzow, Mark R, Kebriaei, Partow, Hourigan, Christopher S, Abid, Muhammad Bilal, Weisdorf, Daniel J, and Saber, Wael more...

Subjects
Rare Diseases, Cancer, Pediatric, Pediatric Cancer, Stem Cell Research - Nonembryonic - Human, Orphan Drug, Hematology, Stem Cell Research, Transplantation, Good Health and Well Being, Fetal Blood, Hematopoietic Stem Cell Transplantation, Humans, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Retrospective Studies, Siblings, Unrelated Donors
Abstract

The role of haploidentical hematopoietic cell transplantation (HCT) using posttransplant cyclophosphamide (PTCy) for acute lymphoblastic leukemia (ALL) is being defined. We performed a retrospective, multivariable analysis comparing outcomes of HCT approaches by donor for adults with ALL in remission. The primary objective was to compare overall survival (OS) among haploidentical HCTs using PTCy and HLA-matched sibling donor (MSD), 8/8 HLA-matched unrelated donor (MUD), 7 /8 HLA-MUD, or umbilical cord blood (UCB) HCT. Comparing haploidentical HCT to MSD HCT, we found that OS, leukemia-free survival (LFS), nonrelapse mortality (NRM), relapse, and acute graft-versus-host disease (aGVHD) were not different but chronic GVHD (cGVHD) was higher in MSD HCT. Compared with MUD HCT, OS, LFS, and relapse were not different, but MUD HCT had increased NRM (hazard ratio [HR], 1.42; P = .02), grade 3 to 4 aGVHD (HR, 1.59; P = .005), and cGVHD. Compared with 7/8 UD HCT, LFS and relapse were not different, but 7/8 UD HCT had worse OS (HR, 1.38; P = .01) and increased NRM (HR, 2.13; P ≤ .001), grade 3 to 4 aGVHD (HR, 1.86; P = .003), and cGVHD (HR, 1.72; P ≤ .001). Compared with UCB HCT, late OS, late LFS, relapse, and cGVHD were not different but UCB HCT had worse early OS (≤18 months; HR, 1.93; P < .001), worse early LFS (HR, 1.40; P = .007) and increased incidences of NRM (HR, 2.08; P < .001) and grade 3 to 4 aGVHD (HR, 1.97; P < .001). Haploidentical HCT using PTCy showed no difference in survival but less GVHD compared with traditional MSD and MUD HCT and is the preferred alternative donor HCT option for adults with ALL in complete remission. more...

Published
2022

17. Survival of TP53-mutated acute myeloid leukemia patients receiving allogeneic stem cell transplantation after first induction or salvage therapy: results from the Consortium on Myeloid Malignancies and Neoplastic Diseases (COMMAND)

Author

Badar, Talha, Atallah, Ehab, Shallis, Rory, Saliba, Antoine N., Patel, Anand, Bewersdorf, Jan P., Grenet, Justin, Stahl, Maximilian, Duvall, Adam, Burkart, Madelyn, Palmisiano, Neil, Bradshaw, Danielle, Kubiak, Michal, Dinner, Shira, Goldberg, Aaron D., Abaza, Yasmin, Murthy, Guru Subramanian Guru, Kota, Vamsi, and Litzow, Mark R. more...

Published
2023
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18. Epigenomic mapping reveals distinct B cell acute lymphoblastic leukemia chromatin architectures and regulators

Author

Barnett, Kelly R., Mobley, Robert J., Diedrich, Jonathan D., Bergeron, Brennan P., Bhattarai, Kashi Raj, Monovich, Alexander C., Narina, Shilpa, Yang, Wenjian, Crews, Kristine R., Manring, Christopher S., Jabbour, Elias, Paietta, Elisabeth, Litzow, Mark R., Kornblau, Steven M., Stock, Wendy, Inaba, Hiroto, Jeha, Sima, Pui, Ching-Hon, Mullighan, Charles G., Relling, Mary V., Pruett-Miller, Shondra M., Ryan, Russell J.H., Yang, Jun J., Evans, William E., and Savic, Daniel more...

Published
2023
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20. Prospective evaluation of genome sequencing to compare conventional cytogenetics in acute myeloid leukemia

Author

Pitel, Beth A., Zepeda-Mendoza, Cinthya, Sachs, Zohar, Tang, Hongwei, Shivaram, Suganti, Sharma, Neeraj, Smadbeck, James B., Smoley, Stephanie A., Pearce, Kathryn E., Luoma, Ivy M., Cook, Joselle, Litzow, Mark R., Hoppman, Nicole L., Viswanatha, David, Xu, Xinjie, Ketterling, Rhett P., Greipp, Patricia T., Peterson, Jess F., and Baughn, Linda B. more...

Published
2023
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21. Real-world experience with ponatinib therapy in chronic phase chronic myeloid leukemia: impact of depth of response on survival and prior exposure to nilotinib on arterial occlusive events

Author

Abdelmagid, Maymona G., Al-Kali, Aref, Litzow, Mark R., Begna, Kebede H., Hogan, William J., Patnaik, Mirinal S., Hashmi, Shahrukh K., Elliott, Michelle A., Alkhateeb, Hassan, Karrar, Omer S., Fleti, Farah, Elnayir, Mohammed H., Rivera, Candido E., Murthy, Hemant S., Foran, James M., Kharfan-Dabaja, Mohamed A., Badar, Talha, Viswanatha, David S., Reichard, Kaaren K., Gangat, Naseema, and Tefferi, Ayalew more...

Published
2023
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22. Abnormal karyotype is an independent predictor of inferior survival in Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)

Author

Abdallah, Mostafa, McCullough, Kristen, Ilyas, Rimal, Begna, Kebede H., Al-Kali, Aref, Litzow, Mark R., Hogan, William J., Mangaonkar, Abhishek, Alkhateeb, Hassan, Shah, Mithun V., Elliott, Michelle A., Foran, James M., Badar, Talha, Palmer, Jeanne M., Yi, Cecilia Arana, Sproat, Lisa, Pardanani, Animesh, Patnaik, Mrinal M., Olteanu, Horatiu, Ketterling, Rhett P., Tefferi, Ayalew, and Gangat, Naseema more...

Published
2023
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24. Surrogates of Endothelial Injury Predict Survival After Post-transplant Cyclophosphamide

Author

Baranwal, Anmol, Langer, Kimberly J., Kharfan-Dabaja, Mohamed A., Ayala, Ernesto, Foran, James, Murthy, Hemant, Roy, Vivek, Iqbal, Madiha, Palmer, Jeanne, Sproat, Lisa Z., Chhabra, Saurabh, Khera, Nandita, Durani, Urshila, Hefazi, Mehrdad, Mangaonkar, Abhishek, Shah, Mithun V., Litzow, Mark R., Hogan, William J., and Alkhateeb, Hassan B. more...

Published
2025
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25. DNA polymerase θ protects leukemia cells from metabolically induced DNA damage

Author

Vekariya, Umeshkumar, Toma, Monika, Nieborowska-Skorska, Margaret, Le, Bac Viet, Caron, Marie-Christine, Kukuyan, Anna-Mariya, Sullivan-Reed, Katherine, Podszywalow-Bartnicka, Paulina, Chitrala, Kumaraswamy N., Atkins, Jessica, Drzewiecka, Malgorzata, Feng, Wanjuan, Chan, Joe, Chatla, Srinivas, Golovine, Konstantin, Jelinek, Jaroslav, Sliwinski, Tomasz, Ghosh, Jayashri, Matlawska-Wasowska, Ksenia, Chandramouly, Gurushankar, Nejati, Reza, Wasik, Mariusz, Sykes, Stephen M., Piwocka, Katarzyna, Hadzijusufovic, Emir, Valent, Peter, Pomerantz, Richard T., Morton, George, Childers, Wayne, Zhao, Huaqing, Paietta, Elisabeth M., Levine, Ross L., Tallman, Martin S., Fernandez, Hugo F., Litzow, Mark R., Gupta, Gaorav P., Masson, Jean-Yves, and Skorski, Tomasz more...

Published
2023
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27. Clinical utilization of Chimeric Antigen Receptor T-cells (CAR-T) in B-cell acute lymphoblastic leukemia (ALL)–an expert opinion from the European Society for Blood and Marrow Transplantation (EBMT) and the American Society for Blood and Marrow Transplantation (ASBMT)

Author

Kansagra, Ankit J, Frey, Noelle V, Bar, Merav, Laetsch, Theodore W, Carpenter, Paul A, Savani, Bipin N, Heslop, Helen E, Bollard, Catherine M, Komanduri, Krishna V, Gastineau, Dennis A, Chabannon, Christian, Perales, Miguel A, Hudecek, Michael, Aljurf, Mahmoud, Andritsos, Leslie, Barrett, John A, Bachanova, Veronika, Bonini, Chiara, Ghobadi, Armin, Gill, Saar I, Hill, Joshua A, Kenderian, Saad, Kebriaei, Partow, Nagler, Arnon, Maloney, David, Liu, Hien D, Shah, Nirali N, Kharfan-Dabaja, Mohamed A, Shpall, Elizabeth J, Mufti, Ghulam J, Johnston, Laura, Jacoby, Elad, Bazarbachi, Ali, DiPersio, John F, Pavletic, Steven Z, Porter, David L, Grupp, Stephan A, Sadelain, Michel, Litzow, Mark R, Mohty, Mohamad, and Hashmi, Shahrukh K more...

Subjects
Biomedical and Clinical Sciences, Oncology and Carcinogenesis, Immunology, Cancer, Pediatric, Childhood Leukemia, Immunization, Genetics, Transplantation, Biotechnology, Gene Therapy, Pediatric Cancer, Pediatric Research Initiative, Hematology, Orphan Drug, Rare Diseases, Development of treatments and therapeutic interventions, 5.2 Cellular and gene therapies, Good Health and Well Being, Clinical Sciences, Cardiovascular medicine and haematology, Oncology and carcinogenesis
Abstract

On August 30, 2017, the U.S. Food and Drug Administration (US-FDA) approved tisagenlecleucel (KYMRIAH, Novartis, Basel, Switzerland), a synthetic bioimmune product of anti-CD19 chimeric antigen receptor-T cells (CAR-T), for the treatment of children and young adults with relapsed/refractory B-cell acute lymphoblastic leukemia (B-ALL). With this new era of personalized cancer immunotherapy, multiple challenges are present ranging from implementation of a CAR-T program to safe delivery of the drug, long-term toxicity monitoring and disease assessments. To address these issues, experts representing the American Society for Blood and Marrow Transplant (ASBMT), the European Group for Blood and Marrow Transplantation (EBMT), the International Society of Cell and Gene Therapy (ISCT), and the Foundation for the Accreditation of Cellular Therapy (FACT), formed a global CAR-T task force to identify and address key questions pertinent for hematologists and transplant physicians regarding the clinical use of anti CD19 CAR-T therapy in patients with B-ALL. This article presents an initial roadmap for navigating common clinical practice scenarios that will become more prevalent now that the first commercially available CAR-T product for B-ALL has been approved. more...

Published
2019

28. Related peripheral blood stem cell donors experience more severe symptoms and less complete recovery at one year compared to unrelated donors

Author

Pulsipher, Michael A, Logan, Brent R, Kiefer, Deidre M, Chitphakdithai, Pintip, Riches, Marcie L, Rizzo, J Douglas, Anderlini, Paolo, Leitman, O’Susan F, Kobusingye, Hati, Besser, RaeAnne M, Miller, John P, Drexler, Rebecca J, Abdel-Mageed, Aly, Ahmed, Ibrahim A, Akard, Luke P, Artz, Andrew S, Ball, Edward D, Bayer, Ruthee-Lu, Bigelow, Carolyn, Bolwell, Brian J, Broun, E Randolph, Delgado, David C, Duckworth, Katharine, Dvorak, Christopher C, Hahn, Theresa E, Haight, Ann E, Hari, Parameswaran N, Hayes-Lattin, Brandon M, Jacobsohn, David A, Jakubowski, Ann A, Kasow, Kimberly A, Lazarus, Hillard M, Liesveld, Jane L, Linenberger, Michael, Litzow, Mark R, Longo, Walter, Magalhaes-Silverman, Margarida, McCarty, John M, McGuirk, Joseph P, Mori, Shahram, Parameswaran, Vinod, Prasad, Vinod K, Rowley, Scott D, Rybka, Witold B, Sahdev, Indira, Schriber, Jeffrey R, Selby, George B, Shaughnessy, Paul J, Shenoy, Shalini, Spitzer, Thomas, Tse, William T, Uberti, Joseph P, Vusirikala, Madhuri, Waller, Edmund K, Weisdorf, Daniel J, Yanik, Gregory A, Navarro, Willis H, Horowitz, Mary M, Switzer, Galen E, Confer, Dennis L, and Shaw, Bronwen E more...

Subjects
Biomedical and Clinical Sciences, Clinical Sciences, Transplantation, Patient Safety, Regenerative Medicine, Clinical Research, Neurosciences, Stem Cell Research - Nonembryonic - Human, Chronic Pain, Pain Research, Stem Cell Research, Adolescent, Adult, Female, Humans, Living Donors, Male, Middle Aged, Peripheral Blood Stem Cell Transplantation, Peripheral Blood Stem Cells, Prospective Studies, Quality of Life, Unrelated Donors, Young Adult, Cardiorespiratory Medicine and Haematology, Immunology, Cardiovascular medicine and haematology
Abstract

Unlike unrelated donor registries, transplant centers lack uniform approaches to related donor assessment and deferral. To test whether related donors are at increased risk for donation-related toxicities, we conducted a prospective observational trial of 11,942 related and unrelated donors aged 18-60 years. Bone marrow (BM) was collected at 37 transplant and 78 National Marrow Donor Program centers, and peripheral blood stem cells (PBSC) were collected at 42 transplant and 87 unrelated donor centers in North America. Possible presence of medical comorbidities was verified prior to donation, and standardized pain and toxicity measures were assessed pre-donation, peri-donation, and one year following. Multivariate analyses showed similar experiences for BM collection in related and unrelated donors; however, related stem cell donors had increased risk of moderate [odds ratios (ORs) 1.42; P more...

Published
2019

29. Effect of Aging and Predonation Comorbidities on the Related Peripheral Blood Stem Cell Donor Experience: Report from the Related Donor Safety Study.

Author

Pulsipher, Michael A, Logan, Brent R, Chitphakdithai, Pintip, Kiefer, Deidre M, Riches, Marcie L, Rizzo, J Douglas, Anderlini, Paolo, Leitman, Susan F, Varni, James W, Kobusingye, Hati, Besser, RaeAnne M, Miller, John P, Drexler, Rebecca J, Abdel-Mageed, Aly, Ahmed, Ibrahim A, Akard, Luke P, Artz, Andrew S, Ball, Edward D, Bayer, Ruthee-Lu, Bigelow, Carolyn, Bolwell, Brian J, Broun, E Randolph, Bunin, Nancy J, Delgado, David C, Duckworth, Katharine, Dvorak, Christopher C, Hahn, Theresa E, Haight, Ann E, Hari, Parameswaran N, Hayes-Lattin, Brandon M, Jacobsohn, David A, Jakubowski, Ann A, Kasow, Kimberly A, Lazarus, Hillard M, Liesveld, Jane L, Linenberger, Michael, Litzow, Mark R, Longo, Walter, Magalhaes-Silverman, Margarida, McCarty, John M, McGuirk, Joseph P, Mori, Shahram, Prasad, Vinod K, Rowley, Scott D, Rybka, Witold B, Sahdev, Indira, Schriber, Jeffrey R, Selby, George B, Shaughnessy, Paul J, Shenoy, Shalini, Spitzer, Thomas, Tse, William T, Uberti, Joseph P, Vusirikala, Madhuri, Waller, Edmund K, Weisdorf, Daniel J, Yanik, Gregory A, Navarro, Willis H, Horowitz, Mary M, Switzer, Galen E, Shaw, Bronwen E, and Confer, Dennis L more...

Subjects
Humans, Peripheral Blood Stem Cell Transplantation, Comorbidity, Adolescent, Adult, Aged, Middle Aged, Blood Donors, Female, Male, Young Adult, Peripheral Blood Stem Cells, BM collection toxicities, Donor safety, PBSC collection toxicities, Stem cell transplantation, Transplantation, Pain Research, Stem Cell Research - Nonembryonic - Human, Clinical Research, Chronic Pain, Stem Cell Research, Aging, Clinical Sciences, Immunology
Abstract

The development of reduced-intensity approaches for allogeneic hematopoietic cell transplantation has resulted in growing numbers of older related donors (RDs) of peripheral blood stem cells (PBSCs). The effects of age on donation efficacy, toxicity, and long-term recovery in RDs are poorly understood. To address this we analyzed hematologic variables, pain, donation-related symptoms, and recovery in 1211 PBSC RDs aged 18 to 79 enrolled in the Related Donor Safety Study. RDs aged > 60 had a lower median CD34+ level before apheresis compared with younger RDs (age > 60, 59 × 106/L; age 41 to 60, 81 × 106/L; age 18 to 40, 121 × 106/L; P < .001). This resulted in older donors undergoing more apheresis procedures (49% versus 30% ≥ 2 collections, P < .001) and higher collection volumes (52% versus 32% > 24 L, P < .001), leading to high percentages of donors aged > 60 with postcollection thrombocytopenia more...

Published
2019

31. Hepatotoxicity induced by arsenic trioxide: clinical features, mechanisms, preventive and potential therapeutic strategies.

Author

Wen, Jun, Li, Aiwen, Wang, Ziliang, Guo, Xiaoxiao, Zhang, Gaoling, Litzow, Mark R., and Liu, Qiuju

Abstract

Arsenic trioxide (ATO) has shown substantial efficacy in the treatment of patients with acute promyelocytic leukemia, and the utilization of ATO as a potential treatment for other tumors is currently being investigated; thus, its clinical application is becoming more widespread. However, the toxicity of ATO has prevented many patients from receiving this highly beneficial treatment. The clinical features, mechanisms, and preventive measures for ATO hepatotoxicity, as well as potential curative strategies, are discussed in this review. This review not only discusses existing drugs for the treatment of hepatotoxicity but also focuses on potential future therapeutic agents, providing forward-looking guidance for the clinical use of small molecule extracts, trace elements, antidiabetic drugs, and vitamins. [ABSTRACT FROM AUTHOR] more...

Published
2025
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32. Mayo Genetic Risk Models for Newly Diagnosed Acute Myeloid Leukemia Treated With Venetoclax + Hypomethylating Agent.

Author

Gangat, Naseema, Elbeih, Azeem, Ghosoun, Nour, McCullough, Kristen, Aperna, Fnu, Johnson, Isla M., Abdelmagid, Maymona, Al‐Kali, Aref, Alkhateeb, Hassan B., Begna, Kebede H., Elliott, Michelle, Mangaonkar, Abhishek, Matin, Aasiya, Saliba, Antoine N., Hefazi Torghabeh, Mehrdad, Litzow, Mark R., Hogan, William, Shah, Mithun, Patnaik, Mrinal M., and Pardanani, Animesh more...

Published
2025
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33. Randomized Phase III SIERRA Trial of 131 I-Apamistamab Before Allogeneic Hematopoietic Cell Transplantation Versus Conventional Care for Relapsed/Refractory AML.

Author

Gyurkocza, Boglarka, Nath, Rajneesh, Seropian, Stuart, Choe, Hannah, Litzow, Mark R., Abboud, Camille, Koshy, Nebu, Stiff, Patrick, Tomlinson, Benjamin, Abhyankar, Sunil, Foran, James, Hari, Parameswaran, Chen, George, Al-Kadhimi, Zaid, Kebriaei, Partow, Sabloff, Mitchell, Orozco, Johnnie J., Jamieson, Katarzyna, Silverman, Margarida, and Van Besien, Koen more...

Published
2025
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36. Clinical Utilization of Chimeric Antigen Receptor T Cells in B Cell Acute Lymphoblastic Leukemia: An Expert Opinion from the European Society for Blood and Marrow Transplantation and the American Society for Transplantation and Cellular Therapy

Author

Kansagra, Ankit J, Frey, Noelle V, Bar, Merav, Laetsch, Theodore W, Carpenter, Paul A, Savani, Bipin N, Heslop, Helen E, Bollard, Catherine M, Komanduri, Krishna V, Gastineau, Dennis A, Chabannon, Christian, Perales, Miguel A, Hudecek, Michael, Aljurf, Mahmoud, Andritsos, Leslie, Barrett, John A, Bachanova, Veronika, Bonini, Chiara, Ghobadi, Armin, Gill, Saar I, Hill, Joshua, Kenderian, Saad, Kebriaei, Partow, Nagler, Arnon, Maloney, David, Liu, Hien D, Shah, Nirali N, Kharfan-Dabaja, Mohamed A, Shpall, Elizabeth J, Mufti, Ghulam J, Johnston, Laura, Jacoby, Elad, Bazarbachi, Ali, DiPersio, John F, Pavletic, Steven Z, Porter, David L, Grupp, Stephan A, Sadelain, Michel, Litzow, Mark R, Mohty, Mohamad, and Hashmi, Shahrukh K more...

Subjects
Biomedical and Clinical Sciences, Oncology and Carcinogenesis, Immunology, Transplantation, Childhood Leukemia, Pediatric Cancer, Pediatric, Biotechnology, Orphan Drug, Genetics, Pediatric Research Initiative, Cancer, Rare Diseases, Gene Therapy, Immunization, Hematology, Development of treatments and therapeutic interventions, 5.2 Cellular and gene therapies, Good Health and Well Being, Antigens, CD19, Child, Critical Pathways, Drug Approval, Expert Testimony, Humans, Immunotherapy, Adoptive, Practice Patterns, Physicians', Precursor B-Cell Lymphoblastic Leukemia-Lymphoma, Receptors, Antigen, T-Cell, Societies, Medical, United States, Young Adult, Chimeric antigen receptor, Leukemia, T cell, Clinical Sciences, Cardiovascular medicine and haematology
Abstract

On August 30, 2017 the US Food and Drug Administration approved tisagenlecleucel (Kymriah; Novartis, Basel, Switzerland), a synthetic bioimmune product of anti-CD19 chimeric antigen receptor T cells (CAR-T), for the treatment of children and young adults with relapsed/refractory B cell acute lymphoblastic leukemia (B-ALL). With this new era of personalized cancer immunotherapy, multiple challenges are present, ranging from implementation of a CAR-T program to safe delivery of the drug, long-term toxicity monitoring, and disease assessments. To address these issues experts representing the American Society for Blood and Marrow Transplant, the European Society for Blood and Marrow Transplantation, the International Society of Cell and Gene Therapy, and the Foundation for the Accreditation of Cellular Therapy formed a global CAR-T task force to identify and address key questions pertinent for hematologists and transplant physicians regarding the clinical use of anti CD19 CAR-T therapy in patients with B-ALL. This article presents an initial roadmap for navigating common clinical practice scenarios that will become more prevalent now that the first commercially available CAR-T product for B-ALL has been approved. more...

Published
2019

37. Core‐binding factor acute myeloid leukemia with t(8;21): Risk factors and a novel scoring system (I‐CBFit)

Author

Ustun, Celalettin, Morgan, Elizabeth, Moodie, Erica EM, Pullarkat, Sheeja, Yeung, Cecilia, Broesby‐Olsen, Sigurd, Ohgami, Robert, Kim, Young, Sperr, Wolfgang, Vestergaard, Hanne, Chen, Dong, Kluin, Philip M, Dolan, Michelle, Mrózek, Krzysztof, Czuchlewski, David, Horny, Hans‐Peter, George, Tracy I, Kristensen, Thomas Kielsgaard, Ku, Nam K, Yi, Cecilia Arana, Møller, Michael Boe, Marcucci, Guido, Baughn, Linda, Schiefer, Ana‐Iris, Hilberink, JR, Pullarkat, Vinod, Shanley, Ryan, Kohlschmidt, Jessica, Coulombe, Janie, Salhotra, Amandeep, Soma, Lori, Cho, Christina, Linden, Michael A, Akin, Cem, Gotlib, Jason, Hoermann, Gregor, Hornick, Jason, Nakamura, Ryo, Deeg, Joachim, Bloomfield, Clara D, Weisdorf, Daniel, Litzow, Mark R, Valent, Peter, Huls, Gerwin, Perales, Miguel‐Angel, and Borthakur, Gautam more...

Subjects
Biomedical and Clinical Sciences, Cardiovascular Medicine and Haematology, Clinical Sciences, Oncology and Carcinogenesis, Cancer, Pediatric Research Initiative, Rare Diseases, Pediatric Cancer, Clinical Research, Childhood Leukemia, Pediatric, Stem Cell Research - Nonembryonic - Human, Stem Cell Research, Genetics, Hematology, Adolescent, Adult, Aged, Aged, 80 and over, Child, Child, Preschool, Chromosomes, Human, Pair 21, Chromosomes, Human, Pair 8, Core Binding Factors, Female, Humans, Leukemia, Myeloid, Acute, Male, Middle Aged, Risk Factors, Severity of Illness Index, Translocation, Genetic, Young Adult, acute myeloid leukemia, core-binding factor, disease-free survival, KIT mutation, predictive value, relapse, scoring system, Biochemistry and Cell Biology, Oncology and carcinogenesis
Abstract

BackgroundAlthough the prognosis of core-binding factor (CBF) acute myeloid leukemia (AML) is better than other subtypes of AML, 30% of patients still relapse and may require allogeneic hematopoietic cell transplantation (alloHCT). However, there is no validated widely accepted scoring system to predict patient subsets with higher risk of relapse.MethodsEleven centers in the US and Europe evaluated 247 patients with t(8;21)(q22;q22).ResultsComplete remission (CR) rate was high (92.7%), yet relapse occurred in 27.1% of patients. A total of 24.7% of patients received alloHCT. The median disease-free (DFS) and overall (OS) survival were 20.8 and 31.2 months, respectively. Age, KIT D816V mutated (11.3%) or nontested (36.4%) compared with KIT D816V wild type (52.5%), high white blood cell counts (WBC), and pseudodiploidy compared with hyper- or hypodiploidy were included in a scoring system (named I-CBFit). DFS rate at 2 years was 76% for patients with a low-risk I-CBFit score compared with 36% for those with a high-risk I-CBFit score (P more...

Published
2018

39. Measurable residual disease does not preclude prolonged progression-free survival in CLL treated with ibrutinib

Author

Wang, Xin Victoria, Hanson, Curtis A., Tschumper, Renee C., Lesnick, Connie E., Braggio, Esteban, Paietta, Elisabeth M., O'Brien, Susan, Barrientos, Jacqueline C., Leis, Jose Francisco, Zhang, Cong Christine, Coutre, Steven E., Barr, Paul M., Cashen, Amanda F., Mato, Anthony R., Singh, Avina K., Mullane, Michael P., Erba, Harry, Stone, Richard, Litzow, Mark R., Tallman, Martin S., Shanafelt, Tait D., and Kay, Neil E. more...

Published
2021
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40. Degradation of Janus kinases in CRLF2-rearranged acute lymphoblastic leukemia

Author

Chang, Yunchao, Min, Jaeki, Jarusiewicz, Jamie A., Actis, Marisa, Yu-Chen Bradford, Shanshan, Mayasundari, Anand, Yang, Lei, Chepyala, Divyabharathi, Alcock, Lisa J., Roberts, Kathryn G., Nithianantham, Stanley, Maxwell, Dylan, Rowland, Lauren, Larsen, Randolph, Seth, Aman, Goto, Hiroaki, Imamura, Toshihiko, Akahane, Koshi, Hansen, Baranda S., Pruett-Miller, Shondra M., Paietta, Elisabeth M., Litzow, Mark R., Qu, Chunxu, Yang, Jun J., Fischer, Marcus, Rankovic, Zoran, and Mullighan, Charles G. more...

Published
2021
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41. Tipifarnib as maintenance therapy did not improve disease-free survival in patients with acute myelogenous leukemia at high risk of relapse: Results of the phase III randomized E2902 trial

Author

Luger, Selina M., Wang, Victoria X., Rowe, Jacob M., Litzow, Mark R., Paietta, Elisabeth, Ketterling, Rhett P., Lazarus, Hillard, Rybka, Witold B., Craig, Michael D., Karp, Judith, Cooper, Brenda W., Makary, Adel Z., Kaminer, Lynne S., Appelbaum, Frederick R., Larson, Richard A., and Tallman, Martin S. more...

Published
2021
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45. Molecular classification improves risk assessment in adult BCR-ABL1–negative B-ALL

Author

Paietta, Elisabeth, Roberts, Kathryn G., Wang, Victoria, Gu, Zhaohui, Buck, Georgina A.N., Pei, Deqing, Cheng, Cheng, Levine, Ross L., Abdel-Wahab, Omar, Cheng, Zhongshan, Wu, Gang, Qu, Chunxu, Shi, Lei, Pounds, Stanley, Willman, Cheryl L., Harvey, Richard, Racevskis, Janis, Barinka, Jan, Zhang, Yanming, Dewald, Gordon W., Ketterling, Rhett P., Alejos, David, Lazarus, Hillard M., Luger, Selina M., Foroni, Letizia, Patel, Bela, Fielding, Adele K., Melnick, Ari, Marks, David I., Moorman, Anthony V., Wiernik, Peter H., Rowe, Jacob M., Tallman, Martin S., Goldstone, Anthony H., Mullighan, Charles G., and Litzow, Mark R. more...

Published
2021
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47. Extensive virologic and immunologic characterization in an HIV-infected individual following allogeneic stem cell transplant and analytic cessation of antiretroviral therapy: A case study.

Author

Cummins, Nathan W, Rizza, Stacey, Litzow, Mark R, Hua, Stephane, Lee, Guinevere Q, Einkauf, Kevin, Chun, Tae-Wook, Rhame, Frank, Baker, Jason V, Busch, Michael P, Chomont, Nicolas, Dean, Patrick G, Fromentin, Rémi, Haase, Ashley T, Hampton, Dylan, Keating, Sheila M, Lada, Steven M, Lee, Tzong-Hae, Natesampillai, Sekar, Richman, Douglas D, Schacker, Timothy W, Wietgrefe, Stephen, Yu, Xu G, Yao, Joseph D, Zeuli, John, Lichterfeld, Mathias, and Badley, Andrew D more...

Subjects
Leukocytes, Mononuclear, Humans, HIV, HIV-1, HIV Infections, Anti-Retroviral Agents, Stem Cell Transplantation, Viral Load, Phylogeny, Middle Aged, Male, Precursor Cell Lymphoblastic Leukemia-Lymphoma, General & Internal Medicine, Medical and Health Sciences
Abstract

BackgroundNotwithstanding 1 documented case of HIV-1 cure following allogeneic stem cell transplantation (allo-SCT), several subsequent cases of allo-SCT in HIV-1 positive individuals have failed to cure HIV-1 infection. The aim of our study was to describe changes in the HIV reservoir in a single chronically HIV-infected patient on suppressive antiretroviral therapy who underwent allo-SCT for treatment of acute lymphoblastic leukemia.Methods and findingsWe prospectively collected peripheral blood mononuclear cells (PBMCs) by leukapheresis from a 55-year-old man with chronic HIV infection before and after allo-SCT to measure the size of the HIV-1 reservoir and characterize viral phylogeny and phenotypic changes in immune cells. At day 784 post-transplant, when HIV-1 was undetectable by multiple measures-including PCR measurements of both total and integrated HIV-1 DNA, replication-competent virus measurement by large cell input quantitative viral outgrowth assay, and in situ hybridization of colon tissue-the patient consented to an analytic treatment interruption (ATI) with frequent clinical monitoring. He remained aviremic off antiretroviral therapy until ATI day 288, when a low-level virus rebound of 60 HIV-1 copies/ml occurred, which increased to 1,640 HIV-1 copies/ml 5 days later, prompting reinitiation of ART. Rebounding plasma HIV-1 sequences were phylogenetically distinct from proviral HIV-1 DNA detected in circulating PBMCs before transplantation. The main limitations of this study are the insensitivity of reservoir measurements, and the fact that it describes a single case.Conclusionsallo-SCT led to a significant reduction in the size of the HIV-1 reservoir and a >9-month-long ART-free remission from HIV-1 replication. Phylogenetic analyses suggest that the origin of rebound virus was distinct from the viruses identified pre-transplant in the PBMCs. more...

Published
2017

49. Secondary cytogenetic abnormalities in core-binding factor AML harboring inv(16) vs t(8;21)

Author

Han, Se young, Mrózek, Krzysztof, Voutsinas, Jenna, Wu, Qian, Morgan, Elizabeth A., Vestergaard, Hanne, Ohgami, Robert, Kluin, Philip M., Kristensen, Thomas Kielsgaard, Pullarkat, Sheeja, Møller, Michael Boe, Schiefer, Ana-Iris, Baughn, Linda B., Kim, Young, Czuchlewski, David, Hilberink, Jacobien R., Horny, Hans-Peter, George, Tracy I., Dolan, Michelle, Ku, Nam K., Arana Yi, Cecilia, Pullarkat, Vinod, Kohlschmidt, Jessica, Salhotra, Amandeep, Soma, Lori, Bloomfield, Clara D., Chen, Dong, Sperr, Wolfgang R., Marcucci, Guido, Cho, Christina, Akin, Cem, Gotlib, Jason, Broesby-Olsen, Sigurd, Larson, Melissa, Linden, Michael A., Deeg, H. Joachim, Hoermann, Gregor, Perales, Miguel-Angel, Hornick, Jason L., Litzow, Mark R., Nakamura, Ryotaro, Weisdorf, Daniel, Borthakur, Gautam, Huls, Gerwin, Valent, Peter, Ustun, Celalettin, and Yeung, Cecilia C.S. more...

Published
2021
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50. Impact of Age on Pharmacogenomics and Treatment Outcomes of B-Cell Acute Lymphoblastic Leukemia.

Author

Yoshimura, Satoshi, Li, Zhenhua, Gocho, Yoshihiro, Yang, Wenjian, Crews, Kristine R., Lee, Shawn H.R., Roberts, Kathryn G., Mullighan, Charles G., Relling, Mary V., Yu, Jiyang, Yeoh, Allen E.J., Loh, Mignon L., Saygin, Caner, Litzow, Mark R., Jeha, Sima, Karol, Seth E., Inaba, Hiroto, Pui, Ching-Hon, Konopleva, Marina, and Jain, Nitin more...

Published
2024
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