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5. The type II RAF inhibitor tovorafenib in relapsed/refractory pediatric low-grade glioma: the phase 2 FIREFLY-1 trial

Author

Kilburn, Lindsay B., Khuong-Quang, Dong-Anh, Hansford, Jordan R., Landi, Daniel, van der Lugt, Jasper, Leary, Sarah E. S., Driever, Pablo Hernáiz, Bailey, Simon, Perreault, Sébastien, McCowage, Geoffrey, Waanders, Angela J., Ziegler, David S., Witt, Olaf, Baxter, Patricia A., Kang, Hyoung Jin, Hassall, Timothy E., Han, Jung Woo, Hargrave, Darren, Franson, Andrea T., Yalon Oren, Michal, Toledano, Helen, Larouche, Valérie, Kline, Cassie, Abdelbaki, Mohamed S., Jabado, Nada, Gottardo, Nicholas G., Gerber, Nicolas U., Whipple, Nicholas S., Segal, Devorah, Chi, Susan N., Oren, Liat, Tan, Enrica E. K., Mueller, Sabine, Cornelio, Izzy, McLeod, Lisa, Zhao, Xin, Walter, Ashley, Da Costa, Daniel, Manley, Peter, Blackman, Samuel C., Packer, Roger J., and Nysom, Karsten

Published
2024
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9. Ruxolitinib in treatment-naive or corticosteroid-refractory paediatric patients with chronic graft-versus-host disease (REACH5): interim analysis of a single-arm, multicentre, phase 2 study

Author

Locatelli, Franco, Antmen, Bulent, Kang, Hyoung Jin, Koh, Katsuyoshi, Takahashi, Yoshiyuki, Kupesiz, Alphan, Dias Matos, Maria Gabriela A, Chopra, Yogi, Bhat, Sunil, Im, Ho Joon, Güngör, Tayfun, Lu, Meng-Yao, Stefanelli, Tommaso, Rosko, Christine, St Pierre, Annie, Burock, Karin, Smith, Yvonne, Sinclair, Karen, and Diaz-de-Heredia, Cristina

Published
2024
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10. A phase 3 randomized trial of mavorixafor, a CXCR4 antagonist, for WHIM syndrome

Author

Badolato, Raffaele, Alsina, Laia, Azar, Antoine, Bertrand, Yves, Bolyard, Audrey A., Dale, David, Deyà-Martínez, Àngela, Dickerson, Kathryn E., Ezra, Navid, Hasle, Henrik, Kang, Hyoung Jin, Kiani-Alikhan, Sorena, Kuijpers, Taco W., Kulagin, Alexander, Langguth, Daman, Levin, Carina, Neth, Olaf, Olbrich, Peter, Peake, Jane, Rodina, Yulia, Rutten, Caroline E., Shcherbina, Anna, Tarrant, Teresa K., Vossen, Matthias G., Wysocki, Christian A., Belschner, Andrea, Bridger, Gary J., Chen, Kelly, Dubuc, Susan, Hu, Yanping, Jiang, Honghua, Li, Sunny, MacLeod, Rick, Stewart, Murray, Taveras, Arthur G., Yan, Tina, and Donadieu, Jean

Published
2024
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11. Real-world data of long-term survival in patients with T-cell lymphoma who underwent stem cell transplantation

Author

Baek, Dong Won, Moon, Joon Ho, Lee, Jae Hoon, Kang, Ka-Won, Lee, Ho Sup, Eom, Hyeon-Seok, Lee, Enuyoung, Lee, Ji Hyun, Lee, Jeong-Ok, Park, Seong Kyu, Kim, Seok Jin, Yoo, Keon Hee, Yoon, Sung-Soo, Koh, Youngil, Kang, Hyoung Jin, Won, Jong-Ho, Lyu, Chuhl Joo, Hahn, Seung Min, Lee, Jung-Hee, Park, Joon Seong, Jo, Jae-Cheol, Mun, Yeung-Chul, Yang, Deok-Hwan, Song, Ga-Young, Lim, Sung-Nam, and Sohn, Sang Kyun

Published
2023
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12. International retrospective study of allogeneic hematopoietic cell transplantation for activated PI3K-delta syndrome.

Author

Dimitrova, Dimana, Nademi, Zohreh, Maccari, Maria Elena, Ehl, Stephan, Uzel, Gulbu, Tomoda, Takahiro, Okano, Tsubasa, Imai, Kohsuke, Carpenter, Benjamin, Ip, Winnie, Rao, Kanchan, Worth, Austen JJ, Laberko, Alexandra, Mukhina, Anna, Néven, Bénédicte, Moshous, Despina, Speckmann, Carsten, Warnatz, Klaus, Wehr, Claudia, Abolhassani, Hassan, Aghamohammadi, Asghar, Bleesing, Jacob J, Dara, Jasmeen, Dvorak, Christopher C, Ghosh, Sujal, Kang, Hyoung Jin, Markelj, Gašper, Modi, Arunkumar, Bayer, Diana K, Notarangelo, Luigi D, Schulz, Ansgar, Garcia-Prat, Marina, Soler-Palacín, Pere, Karakükcü, Musa, Yilmaz, Ebru, Gambineri, Eleonora, Menconi, Mariacristina, Masmas, Tania N, Holm, Mette, Bonfim, Carmem, Prando, Carolina, Hughes, Stephen, Jolles, Stephen, Morris, Emma C, Kapoor, Neena, Koltan, Sylwia, Paneesha, Shankara, Steward, Colin, Wynn, Robert, Duffner, Ulrich, Gennery, Andrew R, Lankester, Arjan C, Slatter, Mary, and Kanakry, Jennifer A

Subjects
Humans, Treatment Outcome, Hematopoietic Stem Cell Transplantation, Transplantation, Homologous, Retrospective Studies, Graft Rejection, Adolescent, Adult, Aged, Middle Aged, Child, Child, Preschool, Female, Male, Young Adult, Phosphatidylinositol 3-Kinases, Kaplan-Meier Estimate, Class I Phosphatidylinositol 3-Kinases, Primary Immunodeficiency Diseases, MTOR Inhibitors, Primary immunodeficiency, activated phosphoinositide 3-kinase delta syndrome, allogeneic hematopoietic cell transplantation, graft failure, lymphoproliferation, mTOR inhibitor, serotherapy, Rare Diseases, Cancer, Transplantation, Regenerative Medicine, Hematology, allogeneic hemato-poietic cell transplantation, Immunology, Allergy
Abstract

BackgroundActivated phosphoinositide 3-kinase delta syndrome (APDS) is a combined immunodeficiency with a heterogeneous phenotype considered reversible by allogeneic hematopoietic cell transplantation (HCT).ObjectivesThis study sought to characterize HCT outcomes in APDS.MethodsRetrospective data were collected on 57 patients with APDS1/2 (median age, 13 years; range, 2-66 years) who underwent HCT.ResultsPre-HCT comorbidities such as lung, gastrointestinal, and liver pathology were common, with hematologic malignancy in 26%. With median follow-up of 2.3 years, 2-year overall and graft failure-free survival probabilities were 86% and 68%, respectively, and did not differ significantly by APDS1 versus APDS2, donor type, or conditioning intensity. The 2-year cumulative incidence of graft failure following first HCT was 17% overall but 42% if mammalian target of rapamycin inhibitor(s) (mTORi) were used in the first year post-HCT, compared with 9% without mTORi. Similarly, 2-year cumulative incidence of unplanned donor cell infusion was overall 28%, but 65% in the context of mTORi receipt and 23% without. Phenotype reversal occurred in 96% of evaluable patients, of whom 17% had mixed chimerism. Vulnerability to renal complications continued post-HCT, adding new insights into potential nonimmunologic roles of phosphoinositide 3-kinase not correctable through HCT.ConclusionsGraft failure, graft instability, and poor graft function requiring unplanned donor cell infusion were major barriers to successful HCT. Post-HCT mTORi use may confer an advantage to residual host cells, promoting graft instability. Longer-term post-HCT follow-up of more patients is needed to elucidate the kinetics of immune reconstitution and donor chimerism, establish approaches that reduce graft instability, and assess the completeness of phenotype reversal over time.

Published
2022

13. Author Correction: The type II RAF inhibitor tovorafenib in relapsed/refractory pediatric low-grade glioma: the phase 2 FIREFLY-1 trial

Author

Kilburn, Lindsay B., Khuong-Quang, Dong-Anh, Hansford, Jordan R., Landi, Daniel, van der Lugt, Jasper, Leary, Sarah E. S., Driever, Pablo Hernáiz, Bailey, Simon, Perreault, Sébastien, McCowage, Geoffrey, Waanders, Angela J., Ziegler, David S., Witt, Olaf, Baxter, Patricia A., Kang, Hyoung Jin, Hassall, Timothy E., Han, Jung Woo, Hargrave, Darren, Franson, Andrea T., Yalon Oren, Michal, Toledano, Helen, Larouche, Valérie, Kline, Cassie, Abdelbaki, Mohamed S., Jabado, Nada, Gottardo, Nicholas G., Gerber, Nicolas U., Whipple, Nicholas S., Segal, Devorah, Chi, Susan N., Oren, Liat, Tan, Enrica E. K., Mueller, Sabine, Cornelio, Izzy, McLeod, Lisa, Zhao, Xin, Walter, Ashley, Da Costa, Daniel, Manley, Peter, Blackman, Samuel C., Packer, Roger J., and Nysom, Karsten

Published
2024
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14. Apixaban versus no anticoagulation for the prevention of venous thromboembolism in children with newly diagnosed acute lymphoblastic leukaemia or lymphoma (PREVAPIX-ALL): a phase 3, open-label, randomised, controlled trial

Author

O'Brien, Sarah H, Rodriguez, Vilmarie, Lew, Glen, Newburger, Jane W, Schultz, Corinna L, Orgel, Etan, Derr, Kimberly, Ranalli, Mark A, Esbenshade, Adam J, Hochberg, Jessica, Kang, Hyoung Jin, Dinikina, Yulia, Mills, Donna, Donovan, Mark, Dyme, Joshua L, Favatella, Nicholas A, and Mitchell, Lesley G

Published
2024
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17. The long-term efficacy and safety of nilotinib in pediatric patients with CML: a 5-year update of the DIALOG study

Author

Hijiya, Nobuko, Maschan, Alexey, Rizzari, Carmelo, Shimada, Hiroyuki, Dufour, Carlo, Goto, Hiroaki, Kang, Hyoung Jin, Guinipero, Terri, Karakas, Zeynep, Bautista, Francisco, Ducassou, Stéphane, Yoo, Keon Hee, Zwaan, Christian Michel, Millot, Frédéric, Patterson, Briana C., Samis, Jill, Izquierdo, Miguel, Titorenko, Ksenia, Li, Sai, and Sosothikul, Darintr

Published
2023
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24. Outcomes of intracranial non-germinomatous germ cell tumors: a retrospective Asian multinational study on treatment strategies and prognostic factors

Author

Hong, Kyung Taek, Han, Jung Woo, Fuji, Hiroshi, Byun, Hwa Kyung, Koh, Kyung-Nam, Wong, Ru Xin, Lee, Hsin-Lun, Yoon, Hong In, Lee, Joo Ho, Phi, Ji Hoon, Kim, Seung-Ki, Kim, Dong-Seok, Lyu, Chuhl Joo, Choi, Jung Yoon, Kang, Hyoung Jin, Chen, Yi-Wei, Lee, Yi-Yen, Im, Ho Joon, Ra, Young-Shin, Do Ahn, Seung, Low, Sharon Yin Yee, Looi, Wen Shen, Park, Hyeon Jin, Suh, Yang-Gun, Suh, Chang-Ok, Wang, Kyu-Chang, Tan, Enrica Ee Kar, Wong, Tai-Tong, and Kim, Joo-Young

Published
2022
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26. Ibrutinib plus RICE or RVICI for relapsed/refractory mature B-cell non-Hodgkin lymphoma in children and young adults: SPARKLE trial

Author

Burke, G. A. Amos, Vinti, Luciana, Kabickova, Edita, Beishuizen, Auke, Tacyildiz, Nurdan, Uyttebroeck, Anne, Kang, Hyoung Jin, Luisi, Flavio, Minard-Colin, Véronique, Burkhardt, Birgit, Tamegnon, Monelle, Sun, Steven, Curtis, Madeliene, Deshpande, Sanjay, Nottage, Kerri, Howes, Angela, Srinivasan, Srimathi, Bhojwani, Deepa, Norris, Robin, and Cairo, Mitchell

Published
2023
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29. A phase 2 study of nilotinib in pediatric patients with CML: long-term update on growth retardation and safety

Author

Hijiya, Nobuko, Maschan, Alexey, Rizzari, Carmelo, Shimada, Hiroyuki, Dufour, Carlo, Goto, Hiroaki, Kang, Hyoung Jin, Guinipero, Terri, Karakas, Zeynep, Bautista, Francisco, Ducassou, Stéphane, Yoo, Keon Hee, Zwaan, Christian Michel, Millot, Frédéric, Patterson, Briana, Samis, Jill, Aimone, Paola, Allepuz, Alex, Titorenko, Ksenia, and Sosothikul, Darintr

Published
2021
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30. A Study to Evaluate the Effectiveness and Safety of Prephase Steroid Treatment before Remission Induction Chemotherapy in Patients with Pediatric Acute Lymphoblastic Leukemia Using Common Data Model-Based Real-World Data: A Retrospective Observational Study

Author

Choi,Yoona, Kim,Bo Kyung, Won,Jung-Hyun, Yoo,Jae Won, Choi,Wona, Jung,Surin, Kim,Jae Yoon, Choi,In Young, Chung,Nack-Gyun, Lee,Jae Wook, Choi,Jung Yoon, Kang,Hyoung Jin, Lee,Howard, Choi,Yoona, Kim,Bo Kyung, Won,Jung-Hyun, Yoo,Jae Won, Choi,Wona, Jung,Surin, Kim,Jae Yoon, Choi,In Young, Chung,Nack-Gyun, Lee,Jae Wook, Choi,Jung Yoon, Kang,Hyoung Jin, and Lee,Howard

Abstract

Yoona Choi,1,2,* Bo Kyung Kim,3,4,* Jung-Hyun Won,2,5 Jae Won Yoo,6 Wona Choi,7 Surin Jung,7 Jae Yoon Kim,7,8 In Young Choi,7 Nack-Gyun Chung,6 Jae Wook Lee,6 Jung Yoon Choi,3,4 Hyoung Jin Kang,3,4,9 Howard Lee1,2,5,10,11 1Department of Applied Bioengineering, Graduate School of Convergence Science and Technology, Seoul National University, Seoul, Republic of Korea; 2Center for Convergence Approaches in Drug Development, Graduate School of Convergence Science and Technology, Seoul National University, Seoul, Republic of Korea; 3Department of Pediatrics, Seoul National University Hospital, Seoul, Republic of Korea; 4Seoul National University Cancer Research Institute, Seoul, Republic of Korea; 5Department of Molecular Medicine and Biopharmaceutical Sciences, Graduate School of Convergence Science and Technology, Seoul National University, Seoul, Republic of Korea; 6Department of Pediatrics, Seoul St. Mary’s Hospital, College of Medicine, the Catholic University of Korea, Seoul, Republic of Korea; 7Department of Medical Informatics, College of Medicine, the Catholic University of Korea, Seoul, Republic of Korea; 8Department of Biomedicine & Health Sciences, the Catholic University of Korea, Seoul, Republic of Korea; 9Department of Pediatrics, Seoul National University College of Medicine, Seoul, Republic of Korea; 10Advanced Institutes of Convergence Technology, Suwon, Republic of Korea; 11Department of Clinical Pharmacology and Therapeutics, Seoul National University Hospital, Seoul, Republic of Korea*These authors contributed equally to this workCorrespondence: Howard Lee, Department of Clinical Pharmacology and Therapeutics, Seoul National University Hospital, 101 Daehak-ro, Jongno-gu, Seoul, 03080, Republic of Korea, Email howardlee@snu.ac.kr Hyoung Jin Kang, Department of Pediatrics, Seoul National University College of Medicine, 101 Daehak-ro, Jongno-gu, Seoul, 03080, Republic of Korea, Email kanghj@snu.ac.krBackground: Rapid reduct

Published
2024

31. The type II RAF inhibitor tovorafenib in relapsed/refractory pediatric low-grade glioma: the phase 2 FIREFLY-1 trial

Author

Kilburn, Lindsay B; https://orcid.org/0000-0003-1478-3619, Khuong-Quang, Dong-Anh; https://orcid.org/0000-0001-6305-7790, Hansford, Jordan R; https://orcid.org/0000-0001-7733-383X, Landi, Daniel, van der Lugt, Jasper; https://orcid.org/0000-0002-8186-338X, Leary, Sarah E S; https://orcid.org/0000-0003-0225-6184, Driever, Pablo Hernáiz; https://orcid.org/0000-0003-3135-3872, Bailey, Simon; https://orcid.org/0000-0003-4763-4329, Perreault, Sébastien, McCowage, Geoffrey, Waanders, Angela J, Ziegler, David S; https://orcid.org/0000-0001-7451-7916, Witt, Olaf, Baxter, Patricia A, Kang, Hyoung Jin; https://orcid.org/0000-0003-1009-6002, Hassall, Timothy E, Han, Jung Woo; https://orcid.org/0000-0001-8936-1205, Hargrave, Darren; https://orcid.org/0000-0001-8219-9807, Franson, Andrea T; https://orcid.org/0000-0002-5361-7683, Yalon Oren, Michal, Toledano, Helen, Larouche, Valérie; https://orcid.org/0000-0003-4082-7267, Kline, Cassie; https://orcid.org/0000-0001-7765-7690, Abdelbaki, Mohamed S, Jabado, Nada; https://orcid.org/0000-0003-2485-3692, Gottardo, Nicholas G; https://orcid.org/0000-0002-1082-6776, Gerber, Nicolas U; https://orcid.org/0000-0002-1783-631X, Whipple, Nicholas S, Segal, Devorah; https://orcid.org/0000-0002-9740-1286, Chi, Susan N, et al, Kilburn, Lindsay B; https://orcid.org/0000-0003-1478-3619, Khuong-Quang, Dong-Anh; https://orcid.org/0000-0001-6305-7790, Hansford, Jordan R; https://orcid.org/0000-0001-7733-383X, Landi, Daniel, van der Lugt, Jasper; https://orcid.org/0000-0002-8186-338X, Leary, Sarah E S; https://orcid.org/0000-0003-0225-6184, Driever, Pablo Hernáiz; https://orcid.org/0000-0003-3135-3872, Bailey, Simon; https://orcid.org/0000-0003-4763-4329, Perreault, Sébastien, McCowage, Geoffrey, Waanders, Angela J, Ziegler, David S; https://orcid.org/0000-0001-7451-7916, Witt, Olaf, Baxter, Patricia A, Kang, Hyoung Jin; https://orcid.org/0000-0003-1009-6002, Hassall, Timothy E, Han, Jung Woo; https://orcid.org/0000-0001-8936-1205, Hargrave, Darren; https://orcid.org/0000-0001-8219-9807, Franson, Andrea T; https://orcid.org/0000-0002-5361-7683, Yalon Oren, Michal, Toledano, Helen, Larouche, Valérie; https://orcid.org/0000-0003-4082-7267, Kline, Cassie; https://orcid.org/0000-0001-7765-7690, Abdelbaki, Mohamed S, Jabado, Nada; https://orcid.org/0000-0003-2485-3692, Gottardo, Nicholas G; https://orcid.org/0000-0002-1082-6776, Gerber, Nicolas U; https://orcid.org/0000-0002-1783-631X, Whipple, Nicholas S, Segal, Devorah; https://orcid.org/0000-0002-9740-1286, Chi, Susan N, and et al

Abstract

BRAF genomic alterations are the most common oncogenic drivers in pediatric low-grade glioma (pLGG). Arm 1 (n = 77) of the ongoing phase 2 FIREFLY-1 (PNOC026) trial investigated the efficacy of the oral, selective, central nervous system-penetrant, type II RAF inhibitor tovorafenib (420 mg m$^{-}$$^{2}$ once weekly; 600 mg maximum) in patients with BRAF-altered, relapsed/refractory pLGG. Arm 2 (n = 60) is an extension cohort, which provided treatment access for patients with RAF-altered pLGG after arm 1 closure. Based on independent review, according to Response Assessment in Neuro-Oncology High-Grade Glioma (RANO-HGG) criteria, the overall response rate (ORR) of 67% met the arm 1 prespecified primary endpoint; median duration of response (DOR) was 16.6 months; and median time to response (TTR) was 3.0 months (secondary endpoints). Other select arm 1 secondary endpoints included ORR, DOR and TTR as assessed by Response Assessment in Pediatric Neuro-Oncology Low-Grade Glioma (RAPNO) criteria and safety (assessed in all treated patients and the primary endpoint for arm 2, n = 137). The ORR according to RAPNO criteria (including minor responses) was 51%; median DOR was 13.8 months; and median TTR was 5.3 months. The most common treatment-related adverse events (TRAEs) were hair color changes (76%), elevated creatine phosphokinase (56%) and anemia (49%). Grade ≥3 TRAEs occurred in 42% of patients. Nine (7%) patients had TRAEs leading to discontinuation of tovorafenib. These data indicate that tovorafenib could be an effective therapy for BRAF-altered, relapsed/refractory pLGG. ClinicalTrials.gov registration: NCT04775485 .

Published
2024

33. Pembrolizumab in paediatric patients with advanced melanoma or a PD-L1-positive, advanced, relapsed, or refractory solid tumour or lymphoma (KEYNOTE-051): interim analysis of an open-label, single-arm, phase 1–2 trial

Author

Geoerger, Birgit, Kang, Hyoung Jin, Yalon-Oren, Michal, Marshall, Lynley V, Vezina, Catherine, Pappo, Alberto, Laetsch, Theodore W, Petrilli, Antonio S, Ebinger, Martin, Toporski, Jacek, Glade-Bender, Julia, Nicholls, Wayne, Fox, Elizabeth, DuBois, Steven G, Macy, Margaret E, Cohn, Susan L, Pathiraja, Kumudu, Diede, Scott J, Ebbinghaus, Scot, and Pinto, Navin

Published
2020
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38. Phase 2 study of nilotinib in pediatric patients with Philadelphia chromosome–positive chronic myeloid leukemia

Author

Hijiya, Nobuko, Maschan, Alexey, Rizzari, Carmelo, Shimada, Hiroyuki, Dufour, Carlo, Goto, Hiroaki, Kang, Hyoung Jin, Guinipero, Terri, Karakas, Zeynep, Bautista, Francisco, Ducassou, Stéphane, Yoo, Keon Hee, Zwaan, Christian Michel, Millot, Frédéric, Aimone, Paola, Allepuz, Alex, Quenet, Sara, Hourcade-Potelleret, Florence, Hertle, Sabine, and Sosothikul, Darintr

Published
2019
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41. Advances in hematopoietic stem cell transplantation in the Asia-Pacific region: the second report from APBMT 2005–2015

Author

Iida, Minako, Kodera, Yoshihisa, Dodds, Anthony, Ho, Aloysius Yew Leng, Nivison-Smith, Ian, Akter, Mafruha Rumana, Wu, Tong, Lie, Albert Kwok Wai, Ghavamzadeh, Ardeshir, Kang, Hyoung Jin, Ong, Tee Chuan, Gyi, Aye Aye, Farzana, Tasneem, Baylon, Honorata, Gooneratne, Lallindra, Tang, Jih-Luh, Bunworasate, Udomsak, Huynh, Van Man, Srivastava, Alok, Okamoto, Shinichiro, and Atsuta, Yoshiko

Published
2019
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43. Harmonization of Busulfan Plasma Exposure Unit (BPEU): A Community-Initiated Consensus Statement

Author

McCune, Jeannine S., Quinones, Christine M., Ritchie, James, Carpenter, Paul A., van Maarseveen, Erik, Yeh, Rosa F., Anasetti, Claudio, Boelens, Jaap J., Hamerschlak, Nelson, Hassan, Moustapha, Kang, Hyoung Jin, Kanda, Yoshinobu, Paci, Angelo, Perales, Miguel-Angel, Shaw, Peter J., Seewaldt, Victoria L., Savani, Bipin N., Hsieh, Angela, Poon, Betsy, Mohty, Mohamad, Pulsipher, Michael A., Pasquini, Marcelo, and Dupuis, L. Lee

Published
2019
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45. A Study to Evaluate the Effectiveness and Safety of Prephase Steroid Treatment before Remission Induction Chemotherapy in Patients with Pediatric Acute Lymphoblastic Leukemia Using Common Data Model-Based Real-World Data: A Retrospective Observational Study

Author

Choi, Yoona, Kim, Bo Kyung, Won, Jung-Hyun, Yoo, Jae Won, Choi, Wona, Jung, Surin, Kim, Jae Yoon, Choi, In Young, Chung, Nack-Gyun, Lee, Jae Wook, Choi, Jung Yoon, Kang, Hyoung Jin, and Lee, Howard

Subjects
REMISSION induction, INDUCTION chemotherapy, LYMPHOBLASTIC leukemia, CHILD patients, ACUTE leukemia
Abstract

Background: Rapid reduction of leukemic cells in the bone marrow during remission induction chemotherapy (RIC) can lead to significant complications such as tumor lysis syndrome (TLS). We investigated whether prephase steroid treatment before RIC could decrease TLS incidence and improve overall survival in pediatric patients with acute lymphoblastic leukemia (ALL). Methods: Data were extracted from the Common Data Model databases in two tertiary-care hospitals in Seoul, South Korea. Patients were classified into the treated or untreated group if they had received RIC with prephase steroid treatment ≥ 7 days before RIC in 2012– 2021 or not, respectively. Stabilized Inverse Probability of Treatment Weighting (sIPTW) was applied to ensure compatibility between the treated and untreated groups. The incidence of TLS within 14 days of starting RIC, overall survival (OS), and the incidence of adverse events of special interest were the primary endpoints. Multiple sensitivity analyses were performed. Results: Baseline characteristics were effectively balanced between the treated (n=308.4) and untreated (n=246.6) groups after sIPTW. Prephase steroid treatment was associated with a significant 88% reduction in the risk of TLS (OR 0.12, 95% CI: 0.03– 0.41). OS was numerically greater in the treated group than in the untreated group although the difference was not statistically significant (HR 0.64, 95% CI 0.25– 1.64). The treated group experienced significantly elevated risks for hyperbilirubinemia and hyperglycemia. The reduction in TLS risk by prephase steroid treatment was maintained in all of the sensitivity analyses. Conclusion: Prephase steroid treatment for ≥ 7 days before RIC in pediatric patients with ALL reduces the risk of TLS, while careful monitoring for toxicities is necessary. If adequately analyzed, real-world data can provide crucial effectiveness and safety information for proper management of pediatric patients with ALL, for whom prospective randomized studies may be difficult to perform for ethical and practical reasons. [ABSTRACT FROM AUTHOR]

Published
2024
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46. The type II RAF inhibitor tovorafenib in relapsed/refractory pediatric low-grade glioma: the phase 2 FIREFLY-1 trial

Author

Kilburn, Lindsay B., primary, Khuong-Quang, Dong-Anh, additional, Hansford, Jordan R., additional, Landi, Daniel, additional, van der Lugt, Jasper, additional, Leary, Sarah E. S., additional, Driever, Pablo Hernáiz, additional, Bailey, Simon, additional, Perreault, Sébastien, additional, McCowage, Geoffrey, additional, Waanders, Angela J., additional, Ziegler, David S., additional, Witt, Olaf, additional, Baxter, Patricia A., additional, Kang, Hyoung Jin, additional, Hassall, Timothy E., additional, Han, Jung Woo, additional, Hargrave, Darren, additional, Franson, Andrea T., additional, Yalon Oren, Michal, additional, Toledano, Helen, additional, Larouche, Valérie, additional, Kline, Cassie, additional, Abdelbaki, Mohamed S., additional, Jabado, Nada, additional, Gottardo, Nicholas G., additional, Gerber, Nicolas U., additional, Whipple, Nicholas S., additional, Segal, Devorah, additional, Chi, Susan N., additional, Oren, Liat, additional, Tan, Enrica E. K., additional, Mueller, Sabine, additional, Cornelio, Izzy, additional, McLeod, Lisa, additional, Zhao, Xin, additional, Walter, Ashley, additional, Da Costa, Daniel, additional, Manley, Peter, additional, Blackman, Samuel C., additional, Packer, Roger J., additional, and Nysom, Karsten, additional

Published
2023
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48. CTNI-37. CLINICAL ACTIVITY OF RAF INHIBITOR TOVORAFENIB ACCORDING TO PRIOR MAPK INHIBITOR TREATMENT IN THE REGISTRATIONAL PEDIATRIC LOW-GRADE GLIOMA ARM OF THE PHASE 2 FIREFLY-1 (PNOC026) STUDY

Author

Khuong-Quang, Dong-Anh, primary, Nysom, Karsten, additional, Landi, Daniel, additional, Ziegler, David, additional, Driever, Pablo Hernáiz, additional, Leary, Sarah, additional, Bailey, Simon, additional, van der Lugt, Jasper, additional, Perreault, Sébastien, additional, Waanders, Angela, additional, Baxter, Patricia, additional, Witt, Olaf, additional, Hargrave, Darren, additional, McCowage, Geoffrey, additional, Hansford, Jordan, additional, Toledano, Helen, additional, Oren, Liat, additional, Tan, Enrica E K, additional, Gerber, Nicolas, additional, Kang, Hyoung Jin, additional, Larouche, Valérie, additional, Abdelbaki, Mohamed S, additional, Cornelio, Izzy, additional, Kim, Yeonhee, additional, Walter, Ashley, additional, Manley, Peter, additional, and Kilburn, Lindsay, additional

Published
2023
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