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1. Gemtuzumab ozogamicin for relapsed or primary refractory acute myeloid leukemia in children—the Polish Pediatric Leukemia and Lymphoma Study Group experience

Author

Pawinska-Wasikowska, Katarzyna, primary, Czogala, Malgorzata, additional, Skoczen, Szymon, additional, Surman, Marta, additional, Rygielska, Monika, additional, Ksiazek, Teofila, additional, Pac, Agnieszka, additional, Wieczorek, Aleksandra, additional, Skalska-Sadowska, Jolanta, additional, Samborska, Magdalena, additional, Wachowiak, Jacek, additional, Chaber, Radoslaw, additional, Tomaszewska, Renata, additional, Szczepanski, Tomasz, additional, Zielezinska, Karolina, additional, Urasinski, Tomasz, additional, Moj-Hackemer, Malgorzata, additional, Kalwak, Krzysztof, additional, Kozlowska, Marta, additional, Irga-Jaworska, Ninela, additional, Balwierz, Walentyna, additional, and Bukowska-Strakova, Karolina, additional

Published
2023
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2. Biologic and clinical features of childhood gamma delta T-ALL: identification of STAG2/LMO2 γδ T-ALL as an extremely high risk leukemia in the very young

Author

Kimura, Shunsuke, primary, Polonen, Petri, additional, Montefiori, Lindsey, additional, Park, Chun Shik, additional, Iacobucci, Ilaria, additional, Yeoh, Allen EJ, additional, Attarbaschi, Andishe, additional, Moore, Andrew S., additional, Brown, Anthony, additional, Manabe, Atsushi, additional, Buldini, Barbara, additional, Freeman, Burgess B., additional, Chen, Chelsey, additional, Cheng, Cheng, additional, Kean Hui, Chiew, additional, Li, Chi-Kong, additional, Pui, Ching-Hon, additional, Qu, Chunxu, additional, Tomizawa, Daisuke, additional, Teachey, David T., additional, Varotto, Elena, additional, M Paietta, Elisabeth, additional, Arnold, Elizabeth D., additional, Locatelli, Franco, additional, Escherich, Gabriele, additional, Elisa Muhle, Hannah, additional, Marquart, Hanne Vibeke, additional, de Groot-Kruseman, Hester A., additional, Rowe, Jacob M, additional, Stary, Jan, additional, Trka, Jan, additional, Choi, John Kim, additional, Meijerink, Jules P.P., additional, Yang, Jun J., additional, Takita, Junko, additional, Pawinska-Wasikowska, Katarzyna, additional, Roberts, Kathryn G., additional, Han, Katie, additional, Caldwell, Kenneth J., additional, Schmiegelow, Kjeld, additional, Crews, Kristine R., additional, Eguchi, Mariko, additional, Schrappe, Martin, additional, Zimmerman, Martin, additional, Takagi, Masatoshi, additional, Maybury, Mellissa, additional, Svaton, Michael, additional, Reiterova, Michaela, additional, Kicinski, Michal, additional, Prater, Mollie S., additional, Kato, Motohiro, additional, Reyes, Noemi, additional, Spinelli, Orietta, additional, Thomas, Paul, additional, Mazilier, Pauline, additional, Gao, Qingsong, additional, Masetti, Riccardo, additional, Kotecha, Rishi S, additional, Pieters, Rob, additional, Elitzur, Sarah, additional, Luger, Selina M., additional, Mitchell, Sharnise, additional, Pruett-Miller, Shondra M., additional, Shen, Shuhong, additional, Jeha, Sima, additional, Kohrer, Stefan, additional, Kornblau, Steven M, additional, Skoczen, Szymon, additional, Miyamura, Takako, additional, Vincent, Tiffaney L, additional, Imamura, Toshihiko, additional, Conter, Valentino, additional, Tang, Yanjing, additional, Liu, Yen-Chun, additional, Chang, Yunchao, additional, Gu, Zhaohui, additional, Cheng, Zhongshan, additional, Yinmei, Zhou, additional, Inaba, Hiroto, additional, and Mullighan, Charles G., additional

Published
2023
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3. Gemtuzumab ozogamicin for relapsed or primary refractory acute myeloid leukemia in children--the Polish Pediatric Leukemia and Lymphoma Study Group experience.

Author

Pawinska-Wasikowska, Katarzyna, Czogala, Malgorzata, Skoczen, Szymon, Surman, Marta, Rygielska, Monika, Ksiazek, Teofila, Pac, Agnieszka, Wieczorek, Aleksandra, Skalska-Sadowska, Jolanta, Samborska, Magdalena, Wachowiak, Jacek, Chaber, Radoslaw, Tomaszewska, Renata, Szczepanski, Tomasz, Zielezinska, Karolina, Urasinski, Tomasz, Moj-Hackemer, Malgorzata, Kalwak, Krzysztof, Kozlowska, Marta, and Irga-Jaworska, Ninela

Subjects
ACUTE myeloid leukemia, CHILDHOOD cancer, HEMATOPOIETIC stem cell transplantation, HEPATIC veno-occlusive disease, LIVER enzymes, BONE marrow
Abstract

Background: Gemtuzumab ozogamicin (GO), one of the first targeted drugs used in oncology, consists of an anti-cluster of differentiation 33 (CD33) monoclonal antibody bound to a derivative of cytotoxic calicheamicin. After the drug withdrawn in 2010 due to a significantly higher rate of early deaths, GO regained approval in 2017 for the treatment of newly diagnosed, refractory, or relapsed acute myeloid leukemia (AML) in adults and children over 15 years of age. The objective of the study was a retrospective analysis of clinical characteristics, treatment outcomes, and GO toxicity profile in children with primary refractory or relapsed (R/R) AML treated in Poland from 2008 to 2022. Methods: Data were collected through the Polish Registry of Acute Myeloid Leukemia. From January 2008 to December 2022, 35 children with R/R AML were treated with GO in seven centers of the Polish Pediatric Leukemia and Lymphoma Study Group. Results: Most of the children (30 of 35) received only one GO cycle in combination with various chemotherapy cycles (IDA-FLA, DOXO-FLA, FLA, FLAG, and others). Eighteen children (51%) achieved complete remission (CR), 14 did not respond to treatment, and three progressed. GO therapy was followed by allogeneic hematopoietic stem cell transplantation (allo-HSCT) in 18 children in CR. The 5-year overall survival (OS) after GO therapy was 37.1% ± 8.7% for the total cohort. There was a trend toward a superior outcome in patients with strong expression of CD33 expression (over 50% positive cells) compared with that in patients with lower expression of CD33 (OS, 41.2% ± 11.9% versus 27.8% ± 13.2%; p = 0.5; 5-year event-free survival, 35.4% ± 11.6% versus 25.7% ± 12.3%; p = 0.5, respectively). Children under 15 years have better outcome (OS, 34.9% ± 10.4% versus 30% ± 14.5%, p = 0.3). The most common adverse events were bone marrow aplasia, fever of unknown origin, infections, and elevated liver enzyme elevation. Sinusoidal obstruction syndrome occurred in two children. Conclusions: The use of GO in severely pretreated children, including those under 15 years of age, with previous failure of AML treatment is a feasible and effective bridging therapy to allo-HSCT with an acceptable toxicity profile. [ABSTRACT FROM AUTHOR]

Published
2024
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6. Go with the Flow—Early Assessment of Measurable Residual Disease in Children with Acute Lymphoblastic Leukemia Treated According to ALL IC-BFM2009

Author

Pawinska-Wasikowska, Katarzyna, primary, Bukowska-Strakova, Karolina, additional, Surman, Marta, additional, Rygielska, Monika, additional, Sadowska, Beata, additional, Ksiazek, Teofila, additional, Klekawka, Tomasz, additional, Wieczorek, Aleksandra, additional, Skoczen, Szymon, additional, and Balwierz, Walentyna, additional

Published
2022
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8. Case Report: Cytoreductive Surgery and Hyperthermic Intraperitoneal Chemotherapy Application in Intraperitoneally Disseminated Inflammatory Myofibroblastic Tumor and in the Youngest Patient in the World: New Indication and Modification of Technique

Author

Garnier, Hanna, primary, Murawski, Maciej, additional, Jastrzebski, Tomasz, additional, Pawinska-Wasikowska, Katarzyna, additional, Balwierz, Walentyna, additional, Sinacka, Katarzyna, additional, Gorecki, Wojciech, additional, Izycka-Swieszewska, Ewa, additional, and Czauderna, Piotr, additional

Published
2021
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9. Incidence of Retinoblastoma Has Increased: Results from 40 European Countries

Author

Stacey, Andrew W., primary, Bowman, Richard, additional, Foster, Allen, additional, Kivelä, Tero T., additional, Munier, Francis L., additional, Cassoux, Nathalie, additional, Fabian, Ido Didi, additional, Harby, Lamis Al, additional, Portabella, Silvia Alarcón, additional, Alia, Donjeta B., additional, All-Eriksson, Charlotta, additional, Antonino, Romanzo, additional, Astbury, Nicholas J., additional, Balaguer, Julia, additional, Balwierz, Walentyna, additional, Barranco, Honorio, additional, Bascaran, Covadonga, additional, Popovic, Maja Beck, additional, Biewald, Eva M., additional, Bobrova, Nadia, additional, Bornfeld, Norbert, additional, Brichard, Bénédicte G., additional, Blum, Sharon, additional, Capra, Michael, additional, Castela, Guilherme, additional, Català-Mora, Jaume, additional, Chantada, Guillermo L., additional, Chernodrinska, Violeta S., additional, Cieslik, Krzysztof, additional, Comsa, Codruta, additional, Correa Llano, Maria G., additional, Csóka, Monika, additional, De Potter, Patrick, additional, Desjardins, Laurence, additional, Dragomir, Monica D., additional, Fernández-Teijeiro, Ana, additional, Aldana, David García, additional, Gregersen, Pernille A., additional, Gomel, Nir, additional, Hadjistilianou, Theodora, additional, Hederova, Stanislava, additional, Hummlen, Marlies, additional, Husakova, Kristina, additional, Ida, Russo, additional, Ilic, Vesna R., additional, Jenkinson, Helen, additional, Kapelushnik, Noa, additional, Kardava, Tamar, additional, Keren-Froim, Naama, additional, Kepak, Tomas, additional, Khotenashvili, Zaza, additional, Klett, Artur, additional, Krivaitiene, Dalia, additional, Latinović, Slobodanka, additional, Lumbroso, Livia, additional, Lysytsia, Lesia, additional, Maka, Erika, additional, Begue, Nieves Martín, additional, Midena, Edoardo, additional, Moll, Annette C., additional, Murgoi, Gabriela, additional, Naumenko, Larisa, additional, Neroev, Vladimir, additional, Nikitovic, Marina, additional, Olechowski, Andrzej, additional, Papyan, Ruzanna, additional, Parrozzani, Raffaele, additional, Parulekar, Manoj V., additional, Pawinska-Wasikowska, Katarzyna, additional, Perić, Sanja, additional, Pochop, Pavel, additional, Polyakov, Vladimir G., additional, Reddy, M. Ashwin, additional, Ritter-Sovinz, Petra, additional, Saakyan, Svetlana, additional, Sagoo, Mandeep S., additional, San Román Pacheco, Sonsoles, additional, Seregard, Stefan, additional, Silva, Sónia, additional, Sorochynska, Tetyana, additional, Stathopoulos, Christina, additional, Kranjc, Branka Stirn, additional, Svojgr, Karel, additional, Tamamyan, Gevorg, additional, Tandili, Alketa, additional, Tateshi, Bekim, additional, Pompe, Manca Tekavcic, additional, Urbak, Steen F., additional, Ushakova, Tatiana L., additional, Valeina, Sandra, additional, van Hoefen Wijsard, Milo, additional, Veleva-Krasteva, Nevyana V., additional, Viksnins, Maris, additional, Wackernagel, Werner, additional, Dod, Charlotte Wolley, additional, Yarovaya, Vera A., additional, Yarovoy, Andrey A., additional, Zhilyaeva, Katsiaryna, additional, and Zondervan, Marcia, additional

Published
2021
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10. Development of treatment and clinical results in childhood acute myeloid leukemia in Poland

Author

Balwierz, Walentyna, Pawinska-Wasikowska, Katarzyna, Klekawka, Tomasz, Czogala, Malgorzata, Matysiak, Michal, Fic-Sikorska, Barbara, Adamkiewicz-Drozynska, Elzbieta, Maciejka-Kapuscinska, Lucyna, Chybicka, Alicja, Potocka, Kinga, Wachowiak, Jacek, Skalska-Sadowska, Jolanta, Kowalczyk, Jerzy, Wojcik, Beata, Wysocki, Mariusz, Koltan, Sylwia, Krawczuk-Rybak, Maryna, Muszynska-Roslan, Katarzyna, Mlynarski, Wojciech, Stolarska, Malgorzata, Urasinski, Tomasz, Kamienska, Elzbieta, Szczepanski, Tomasz, Tomaszewska, Renata, Sobol, Grazyna, Mizia-Malarz, Agnieszka, Karolczyk, Grazyna, Podhorecka, Joanna, Wieczorek, Maria, Karpinska-Derda, Irena, Badowska, Wanda, and Moryl-Bujakowska, Angelina

Published
2013
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11. STAG2/LMO2 Gamma-Delta (γδ) T-ALL: Identification and Characterization of an Extremely High Risk Group of T-ALL in the Very Young

Author

Kimura, Shunsuke, Pölönen, Petri, Montefiori, Lindsey, Caldwell, Kenneth, Iacobucci, Ilaria, Chen, Chelsey, Brown, Anthony, Han, Katie, Liu, Yen-Chun, Chang, Yunchao, Cheng, Zhongshan, Yinmei, Zhou, Park, Chun Shik, Mitchell, Sharnise, Reyes, Noemi, Yeoh, Allen, Attarbaschi, Andishe, Moore, Andrew, Manabe, Atsushi, Buldini, Barbara, Chiew, Kean Hui, Li, Chi Kong, Pui, Ching-Hon, Qu, Chunxu, Tomizawa, Daisuke, Paietta, Elisabeth, Locatelli, Franco, Escherich, Gabriele, Qingsong, Gao, Muhle, Hannah Elisa, Marquart, Hanne Vibeke, de Groot-Kruseman, Hester A., Rowe, Jacob M., Stary, Jan, Trka, Jan, Choi, John Kim, Meijerink, Jules P.P., Takita, Junko, Pawinska-Wasikowska, Katarzyna, Schmiegelow, Kjeld, Eguchi, Mariko, Schrappe, Martin, Zimmermann, Martin, Takagi, Masatoshi, Maybury, Melissa, Svaton, Michael, Reiterova, Michaela, Kicinski, Michal, Kato, Motohiro, Spinelli, Orietta, Mazilier, Pauline, Thomas, Paul G., Masetti, Riccardo, Kotecha, Rishi Sury, Pieters, Rob, Elitzur, Sarah, Luger, Selina M, Shen, Shuhong, Jeha, Sima, Kornblau, Steven M., Skoczen, Szymon, Miyamura, Takako, Vincent, Tiffaney, Imamura, Toshihiko, Conter, Valentino, Tang, Yanjing, Gu, Zhaohui, Roberts, Kathryn G., Teachey, David T., Crews, Kristine R, Cheng, Cheng, Yang, Jun J., Inaba, Hiroto, and Mullighan, Charles G.

Published
2023
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12. Adjuvant therapy of histopathological risk factors of retinoblastoma in Europe: A survey by the European Retinoblastoma Group (EURbG)

Author

Dittner‐Moormann, Sabine, primary, Reschke, Madlen, additional, Abbink, Floor C. H., additional, Aerts, Isabelle, additional, Atalay, Hatice Tuba, additional, Fedorovna Bobrova, Nadezhda, additional, Biewald, Eva, additional, Brecht, Ines B., additional, Caspi, Shani, additional, Cassoux, Nathalie, additional, Castela, Guilherme, additional, Diarra, Yelena, additional, Duncan, Catriona, additional, Ebinger, Martin, additional, Garcia Aldana, David, additional, Hadjistilianou, Doris, additional, Kepák, Tomáš, additional, Klett, Artur, additional, Kiratli, Hayyam, additional, Maka, Erika, additional, Opocher, Enrico, additional, Pawinska‐Wasikowska, Katarzyna, additional, Rascon, Jelena, additional, Russo, Ida, additional, Rutynowska‐Pronicka, Olga, additional, Sábado Álvarez, Constantino, additional, Pacheco, Sonsoles San Roman, additional, Svojgr, Karel, additional, Timmermann, Beate, additional, Vishnevskia‐Dai, Vicktoria, additional, Eggert, Angelika, additional, Ritter‐Sovinz, Petra, additional, Bechrakis, Nikolaos E., additional, Jenkinson, Helen, additional, Moll, Annette, additional, Munier, Francis L., additional, Popovic, Maja Beck, additional, Chantada, Guillermo, additional, Doz, François, additional, and Ketteler, Petra, additional

Published
2021
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14. Travel burden and clinical presentation of retinoblastoma: analysis of 1024 patients from 43 African countries and 518 patients from 40 European countries

Author

Fabian, Ido Didi, primary, Stacey, Andrew W, additional, Foster, Allen, additional, Kivelä, Tero T, additional, Munier, Francis L., additional, Keren-Froim, Naama, additional, Gomel, Nir, additional, Cassoux, Nathalie, additional, Sagoo, Mandeep S, additional, Reddy, M Ashwin, additional, Harby, Lamis Al, additional, Zondervan, Marcia, additional, Bascaran, Covadonga, additional, Abdallah, Elhassan, additional, Abdullahi, Shehu U, additional, Boubacar, Sahadatou Adamou, additional, Ademola-Popoola, Dupe S, additional, Adio, Adedayo, additional, Aghaji, Ada E, additional, Portabella, Silvia Alarcón, additional, Alfa Bio, Amadou I, additional, Ali, Amany M, additional, Alia, Donjeta B, additional, All-Eriksson, Charlotta, additional, Almeida, Argentino A, additional, Alsawidi, Khalifa M, additional, Antonino, Romanzo, additional, Astbury, Nicholas J, additional, Atsiaya, Rose, additional, Balaguer, Julia, additional, Balwierz, Walentyna, additional, Barranco, Honorio, additional, Popovic, Maja Beck, additional, Benmiloud, Sarra, additional, Guebessi, Nissrine Bennani, additional, Berete, Rokia C, additional, Biddulph, Shelley J, additional, Biewald, Eva M, additional, Blum, Sharon, additional, Bobrova, Nadia, additional, Boehme, Marianna, additional, Bornfeld, Norbert, additional, Bouda, Gabrielle C, additional, Bouguila, Hédi, additional, Boumedane, Amaria, additional, Brichard, Bénédicte G, additional, l, Michael Capra, additional, Castela, Guilherme, additional, Català-Mora, Jaume, additional, Chantada, Guillermo L, additional, Chernodrinska, Violeta S, additional, Chiwanga, Faraja S, additional, Cieslik, Krzysztof, additional, Comsa, Codruta, additional, Correa Llano, Maria G, additional, Csóka, Monika, additional, Da Gama, Isac V, additional, Davidson, Alan, additional, Potter, Patrick De, additional, Desjardins, Laurence, additional, Dragomir, Monica D, additional, Bruyn, Magritha Du, additional, Kettani, Asmaa El, additional, Elbahi, Amal M, additional, Elgalaly, Dina, additional, Elhaddad, Alaa M, additional, Ali Elhassan, Moawia M, additional, Elzembely, Mahmoud M, additional, Essuman, Vera A, additional, Evina, Ted Grimbert A, additional, Fasina, Oluyemi, additional, Fernández-Teijeiro, Ana, additional, Gandiwa, Moira, additional, Aldana, David García, additional, Geel, Jennifer A, additional, Gizachew, Zelalem, additional, Gregersen, Pernille A, additional, Guedenon, Koffi M, additional, Hadjistilianou, Theodora, additional, Hassan, Sadiq, additional, Hederova, Stanislava, additional, Hessissen, Laila, additional, Hordofa, Diriba F, additional, Hummlen, Marlies, additional, Husakova, Kristina, additional, Ida, Russo, additional, Ilic, Vesna R, additional, Jenkinson, Helen, additional, Amani Kabesha, Theophile B, additional, Kabore, Rolande L, additional, Kalinaki, Abubakar, additional, Kapelushnik, Noa, additional, Kardava, Tamar, additional, Kemilev, Pavlin Kroumov, additional, Kepak, Tomas, additional, Khotenashvili, Zaza, additional, Klett, Artur, additional, Kosh Komba Palet, Jess Elio, additional, Krivaitiene, Dalia, additional, Kruger, Mariana, additional, Kyara, Alice, additional, Lachmann, Eva S, additional, Latinović, Slobodanka, additional, Lecuona, Karin, additional, Lukamba, Robert M, additional, Lumbroso, Livia, additional, Lysytsia, Lesia, additional, Maka, Erika, additional, Makan, Mayuri, additional, Manda, Chatonda, additional, Begue, Nieves Martín, additional, Matende, Ibrahim O, additional, Matua, Marchelo, additional, Mayet, Ismail, additional, Mbumba, Freddy B, additional, Mengesha, Aemero A, additional, Midena, Edoardo, additional, Mndeme, Furahini G, additional, Mohamedani, Ahmed A, additional, Moll, Annette C, additional, Moreira, Claude, additional, Msina, Mchikirwa S, additional, Msukwa, Gerald, additional, Muma, Kangwa I, additional, Murgoi, Gabriela, additional, Musa, Kareem O, additional, Mustak, Hamzah, additional, Muyen, Okwen M, additional, Naidu, Gita, additional, Naumenko, Larisa, additional, Ndoye Roth, Paule Aïda, additional, Neroev, Vladimir, additional, Nikitovic, Marina, additional, Nkanga, Elizabeth D, additional, Nkumbe, Henry, additional, Nyaywa, Mutale, additional, Obono-Obiang, Ghislaine, additional, Oguego, Ngozi C, additional, Olechowski, Andrzej, additional, Oscar, Alexander Hugo, additional, Osei-Bonsu, Peter, additional, Painter, Sally L, additional, Paintsil, Vivian, additional, Paiva, Luisa, additional, Papyan, Ruzanna, additional, Parrozzani, Raffaele, additional, Parulekar, Manoj, additional, Pawinska-Wasikowska, Katarzyna, additional, Perić, Sanja, additional, Philbert, Remezo, additional, Pochop, Pavel, additional, Polyakov, Vladimir G, additional, Pompe, Manca T, additional, Pons, Jonathan J, additional, Raobela, Léa, additional, Renner, Lorna A, additional, Reynders, David, additional, Ribadu, Dahiru, additional, Riheia, Mussagy M, additional, Ritter-Sovinz, Petra, additional, Saakyan, Svetlana, additional, Said, Azza MA, additional, Román Pacheco, Sonsoles San, additional, Scanlan, Trish A, additional, Schoeman, Judy, additional, Seregard, Stefan, additional, Sherief, Sadik T, additional, Cheikh, Sidi Sidi, additional, Silva, Sónia, additional, Sorochynska, Tetyana, additional, Ssali, Grace, additional, Stathopoulos, Christina, additional, Kranjc, Branka Stirn, additional, Stones, David K, additional, Svojgr, Karel, additional, Sylla, Fatoumata, additional, Tamamyan, Gevorg, additional, Tandili, Alketa, additional, Tateshi, Bekim, additional, Theophile, Tuyisabe, additional, Traoré, Fousseyni, additional, Tyau-Tyau, Harba, additional, Umar, Ali B, additional, Urbak, Steen F, additional, Ushakova, Tatiana L, additional, Valeina, Sandra, additional, Hoefen Wijsard, Milo van, additional, Veleva-Krasteva, Nevyana V, additional, Viksnins, Maris, additional, Wackernagel, Werner, additional, Waddell, Keith, additional, Wade, Patricia D, additional, Wali Nigeria, Amina H, additional, Wime, Amelia DC, additional, Dod, Charlotte Wolley, additional, Yanga, Jenny M, additional, Yarovaya, Vera A, additional, Yarovoy, Andrey A, additional, Zein, Ekhtelbenina, additional, Sharabi, Shirley, additional, Zhilyaeva, Katsiaryna, additional, Ziko, Othman AO, additional, and Bowman, Richard, additional

Published
2020
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15. High Frequency of Fusion Gene Transcript Resulting From t(10;11)(p12;q23) Translocation in Pediatric Acute Myeloid Leukemia in Poland

Author

Ksiazek, Teofila, primary, Czogala, Malgorzata, additional, Kaczowka, Przemyslaw, additional, Sadowska, Beata, additional, Pawinska-Wasikowska, Katarzyna, additional, Bik-Multanowski, Mirosław, additional, Sikorska-Fic, Barbara, additional, Matysiak, Michał, additional, Skalska-Sadowska, Jolanta, additional, Wachowiak, Jacek, additional, Rodziewicz-Konarska, Anna, additional, Chybicka, Alicja, additional, Muszynska-Rosłan, Katarzyna, additional, Krawczuk-Rybak, Maryna, additional, Grabowski, Dominik, additional, Kowalczyk, Jerzy, additional, Maciejka-Kemblowska, Lucyna, additional, Adamkiewicz-Drozynska, Elzbieta, additional, Mlynarski, Wojciech, additional, Tomaszewska, Renata, additional, Szczepanski, Tomasz, additional, Pohorecka, Joanna, additional, Karolczyk, Grazyna, additional, Mizia-Malarz, Agnieszka, additional, Mycko, Katarzyna, additional, Badowska, Wanda, additional, Zielezinska, Karolina, additional, Urasinski, Tomasz, additional, Karpinska-Derda, Irena, additional, Woszczyk, Mariola, additional, Ciebiera, Małgorzata, additional, Lejman, Monika, additional, Skoczen, Szymon, additional, and Balwierz, Walentyna, additional

Published
2020
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16. Retrospective Analysis of the Treatment Outcome in Myeloid Leukemia of Down Syndrome in Polish Pediatric Leukemia and Lymphoma Study Group From 2005 to 2019

Author

Czogala, Malgorzata, primary, Pawinska-Wasikowska, Katarzyna, additional, Ksiazek, Teofila, additional, Sikorska-Fic, Barbara, additional, Matysiak, Michal, additional, Skalska-Sadowska, Jolanta, additional, Wachowiak, Jacek, additional, Rodziewicz-Konarska, Anna, additional, Chybicka, Alicja, additional, Myszynska-Roslan, Katarzyna, additional, Krawczuk-Rybak, Maryna, additional, Grabowski, Dominik, additional, Kowalczyk, Jerzy, additional, Maciejka-Kemblowska, Lucyna, additional, Adamkiewicz-Drozynska, Elzbieta, additional, Bobeff, Katarzyna, additional, Mlynarski, Wojciech, additional, Tomaszewska, Renata, additional, Szczepanski, Tomasz, additional, Pohorecka, Joanna, additional, Chodala-Grzywacz, Agnieszka, additional, Karolczyk, Grazyna, additional, Mizia-Malarz, Agnieszka, additional, Mycko, Katarzyna, additional, Badowska, Wanda, additional, Zielezinska, Karolina, additional, Urasinski, Tomasz, additional, Nykiel, Magdalena, additional, Woszczyk, Mariola, additional, Ciebiera, Malgorzata, additional, Chaber, Radosław, additional, Skoczen, Szymon, additional, and Balwierz, Walentyna, additional

Published
2020
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17. Travel burden and clinical presentation of retinoblastoma: analysis of 1024 patients from 43 African countries and 518 patients from 40 European countries

Author

Fabian, Ido Didi, Stacey, Andrew W, Foster, Allen, Kivela¨, Tero T, Munier, Francis L., Keren-Froim, Naama, Gomel, Nir, Cassoux, Nathalie, Sagoo, Mandeep S, Reddy, M Ashwin, Harby, Lamis Al, Zondervan, Marcia, Bascaran, Covadonga, Abdallah, Elhassan, Abdullahi, Shehu U, Boubacar, Sahadatou Adamou, Ademola-Popoola, Dupe S, Adio, Adedayo, Aghaji, Ada E, Portabella, Silvia Alarcón, Alfa Bio, Amadou I, Ali, Amany M, Alia, Donjeta B, All-Eriksson, Charlotta, Almeida, Argentino A, Alsawidi, Khalifa M, Antonino, Romanzo, Astbury, Nicholas J, Atsiaya, Rose, Balaguer, Julia, Balwierz, Walentyna, Barranco, Honorio, Popovic, Maja Beck, Benmiloud, Sarra, Guebessi, Nissrine Bennani, Berete, Rokia C, Biddulph, Shelley J, Biewald, Eva M, Blum, Sharon, Bobrova, Nadia, Boehme, Marianna, Bornfeld, Norbert, Bouda, Gabrielle C, Bouguila, Hédi, Boumedane, Amaria, Brichard, Bénédicte G, l, Michael Capra, Castela, Guilherme, Català-Mora, Jaume, Chantada, Guillermo L, Chernodrinska, Violeta S, Chiwanga, Faraja S, Cieslik, Krzysztof, Comsa, Codruta, Correa Llano, Maria G, Csóka, Monika, Da Gama, Isac V, Davidson, Alan, Potter, Patrick De, Desjardins, Laurence, Dragomir, Monica D, Bruyn, Magritha Du, Kettani, Asmaa El, Elbahi, Amal M, Elgalaly, Dina, Elhaddad, Alaa M, Ali Elhassan, Moawia M, Elzembely, Mahmoud M, Essuman, Vera A, Evina, Ted Grimbert A, Fasina, Oluyemi, Fernández-Teijeiro, Ana, Gandiwa, Moira, Aldana, David García, Geel, Jennifer A, Gizachew, Zelalem, Gregersen, Pernille A, Guedenon, Koffi M, Hadjistilianou, Theodora, Hassan, Sadiq, Hederova, Stanislava, Hessissen, Laila, Hordofa, Diriba F, Hummlen, Marlies, Husakova, Kristina, Ida, Russo, Ilic, Vesna R, Jenkinson, Helen, Amani Kabesha, Theophile B, Kabore, Rolande L, Kalinaki, Abubakar, Kapelushnik, Noa, Kardava, Tamar, Kemilev, Pavlin Kroumov, Kepak, Tomas, Khotenashvili, Zaza, Klett, Artur, Kosh Komba Palet, Jess Elio, Krivaitiene, Dalia, Kruger, Mariana, Kyara, Alice, Lachmann, Eva S, Latinović, Slobodanka, Lecuona, Karin, Lukamba, Robert M, Lumbroso, Livia, Lysytsia, Lesia, Maka, Erika, Makan, Mayuri, Manda, Chatonda, Begue, Nieves Martín, Matende, Ibrahim O, Matua, Marchelo, Mayet, Ismail, Mbumba, Freddy B, Mengesha, Aemero A, Midena, Edoardo, Mndeme, Furahini G, Mohamedani, Ahmed A, Moll, Annette C, Moreira, Claude, Msina, Mchikirwa S, Msukwa, Gerald, Muma, Kangwa I, Murgoi, Gabriela, Musa, Kareem O, Mustak, Hamzah, Muyen, Okwen M, Naidu, Gita, Naumenko, Larisa, Ndoye Roth, Paule Ai¨da, Neroev, Vladimir, Nikitovic, Marina, Nkanga, Elizabeth D, Nkumbe, Henry, Nyaywa, Mutale, Obono-Obiang, Ghislaine, Oguego, Ngozi C, Olechowski, Andrzej, Oscar, Alexander Hugo, Osei-Bonsu, Peter, Painter, Sally L, Paintsil, Vivian, Paiva, Luisa, Papyan, Ruzanna, Parrozzani, Raffaele, Parulekar, Manoj, Pawinska-Wasikowska, Katarzyna, Perić, Sanja, Philbert, Remezo, Pochop, Pavel, Polyakov, Vladimir G, Pompe, Manca T, Pons, Jonathan J, Raobela, Léa, Renner, Lorna A, Reynders, David, Ribadu, Dahiru, Riheia, Mussagy M, Ritter-Sovinz, Petra, Saakyan, Svetlana, Said, Azza MA, Román Pacheco, Sonsoles San, Scanlan, Trish A, Schoeman, Judy, Seregard, Stefan, Sherief, Sadik T, Cheikh, Sidi Sidi, Silva, Sónia, Sorochynska, Tetyana, Ssali, Grace, Stathopoulos, Christina, Kranjc, Branka Stirn, Stones, David K, Svojgr, Karel, Sylla, Fatoumata, Tamamyan, Gevorg, Tandili, Alketa, Tateshi, Bekim, Theophile, Tuyisabe, Traoré, Fousseyni, Tyau-Tyau, Harba, Umar, Ali B, Urbak, Steen F, Ushakova, Tatiana L, Valeina, Sandra, Hoefen Wijsard, Milo van, Veleva-Krasteva, Nevyana V, Viksnins, Maris, Wackernagel, Werner, Waddell, Keith, Wade, Patricia D, Wali Nigeria, Amina H, Wime, Amelia DC, Dod, Charlotte Wolley, Yanga, Jenny M, Yarovaya, Vera A, Yarovoy, Andrey A, Zein, Ekhtelbenina, Sharabi, Shirley, Zhilyaeva, Katsiaryna, Ziko, Othman AO, and Bowman, Richard

Abstract

BackgroundThe travel distance from home to a treatment centre, which may impact the stage at diagnosis, has not been investigated for retinoblastoma, the most common childhood eye cancer. We aimed to investigate the travel burden and its impact on clinical presentation in a large sample of patients with retinoblastoma from Africa and Europe.MethodsA cross-sectional analysis including 518 treatment-nai¨ve patients with retinoblastoma residing in 40 European countries and 1024 treatment-nai¨ve patients with retinoblastoma residing in 43 African countries.ResultsCapture rate was 42.2% of expected patients from Africa and 108.8% from Europe. African patients were older (95% CI −12.4 to −5.4, p<0.001), had fewer cases of familial retinoblastoma (95% CI 2.0 to 5.3, p<0.001) and presented with more advanced disease (95% CI 6.0 to 9.8, p<0.001); 43.4% and 15.4% of Africans had extraocular retinoblastoma and distant metastasis at the time of diagnosis, respectively, compared to 2.9% and 1.0% of the Europeans. To reach a retinoblastoma centre, European patients travelled 421.8 km compared to Africans who travelled 185.7 km (p<0.001). On regression analysis, lower-national income level, African residence and older age (p<0.001), but not travel distance (p=0.19), were risk factors for advanced disease.ConclusionsFewer than half the expected number of patients with retinoblastoma presented to African referral centres in 2017, suggesting poor awareness or other barriers to access. Despite the relatively shorter distance travelled by African patients, they presented with later-stage disease. Health education about retinoblastoma is needed for carers and health workers in Africa in order to increase capture rate and promote early referral.

Published
2021
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18. Global Retinoblastoma Presentation and Analysis by National Income Level

Author

Fabian, Ido Didi, Abdallah, Elhassan, Abdullahi, Shehu U., Abdulqader, Rula A., Adamou Boubacar, Sahadatou, Ademola-Popoola, Dupe S., Adio, Adedayo, Afshar, Armin R., Aggarwal, Priyanka, Aghaji, Ada E., Ahmad, Alia, Akib, Marliyanti N. R., Al Harby, Lamis, Al Ani, Mouroge H., Alakbarova, Aygun, Portabella, Silvia Alarcón, Al-Badri, Safaa A. F., Alcasabas, Ana Patricia A., Al-Dahmash, Saad A., Alejos, Amanda, Alemany-Rubio, Ernesto, Alfa Bio, Amadou I., Alfonso Carreras, Yvania, Al-Haddad, Christiane, Al-Hussaini, Hamoud H. Y., Ali, Amany M., Alia, Donjeta B., Al-Jadiry, Mazin F., Al-Jumaly, Usama, Alkatan, Hind M., All-Eriksson, Charlotta, Al-Mafrachi, Ali A. R. M., Almeida, Argentino A., Alsawidi, Khalifa M., Al-Shaheen, Athar A. S. M., Al-Shammary, Entissar H., Amiruddin, Primawita O., Antonino, Romanzo, Astbury, Nicholas J., Atalay, Hatice T., Atchaneeyasakul, La-ongsri, Atsiaya, Rose, Attaseth, Taweevat, Aung, Than H., Ayala, Silvia, Baizakova, Baglan, Balaguer, Julia, Balayeva, Ruhengiz, Balwierz, Walentyna, Barranco, Honorio, Bascaran, Covadonga, Beck Popovic, Maja, Benavides, Raquel, Benmiloud, Sarra, Bennani Guebessi, Nissrine, Berete, Rokia C., Berry, Jesse L., Bhaduri, Anirban, Bhat, Sunil, Biddulph, Shelley J., Biewald, Eva M., Bobrova, Nadia, Boehme, Marianna, Boldt, H.C., Bonanomi, Maria Teresa B. C., Bornfeld, Norbert, Bouda, Gabrielle C., Bouguila, Hédi, Boumedane, Amaria, Brennan, Rachel C., Brichard, Bénédicte G., Buaboonnam, Jassada, Calderón-Sotelo, Patricia, Calle Jara, Doris A., Camuglia, Jayne E., Cano, Miriam R., Capra, Michael, Cassoux, Nathalie, Castela, Guilherme, Castillo, Luis, Català-Mora, Jaume, Chantada, Guillermo L., Chaudhry, Shabana, Chaugule, Sonal S., Chauhan, Argudit, Chawla, Bhavna, Chernodrinska, Violeta S., Chiwanga, Faraja S., Chuluunbat, Tsengelmaa, Cieslik, Krzysztof, Cockcroft, Ruellyn L., Comsa, Codruta, Correa, Zelia M., Correa Llano, Maria G., Corson, Timothy W., Cowan-Lyn, Kristin E., Csóka, Monika, Cui, Xuehao, Da Gama, Isac V., Dangboon, Wantanee, Das, Anirban, Das, Sima, Davanzo, Jacquelyn M., Davidson, Alan, De Potter, Patrick, Delgado, Karina Q., Demirci, Hakan, Desjardins, Laurence, Diaz Coronado, Rosdali Y., Dimaras, Helen, Dodgshun, Andrew J., Donaldson, Craig, Donato Macedo, Carla R., Dragomir, Monica D., Du, Yi, Du Bruyn, Magritha, Edison, Kemala S., Eka Sutyawan, I. Wayan, El Kettani, Asmaa, Elbahi, Amal M., Elder, James E., Elgalaly, Dina, Elhaddad, Alaa M., Elhassan, Moawia M. Ali, Elzembely, Mahmoud M., Essuman, Vera A., Evina, Ted Grimbert A., Fadoo, Zehra, Fandiño, Adriana C., Faranoush, Mohammad, Fasina, Oluyemi, Fernández, Delia D. P. G., Fernández-Teijeiro, Ana, Foster, Allen, Frenkel, Shahar, Fu, Ligia D., Fuentes-Alabi, Soad L., Gallie, Brenda L., Gandiwa, Moira, Garcia, Juan L., García Aldana, David, Gassant, Pascale Y., Geel, Jennifer A., Ghassemi, Fariba, Girón, Ana V., Gizachew, Zelalem, Goenz, Marco A., Gold, Aaron S., Goldberg-Lavid, Maya, Gole, Glen A., Gomel, Nir, Gonzalez, Efren, Gonzalez Perez, Graciela, González-Rodríguez, Liudmira, Garcia Pacheco, Henry N., Graells, Jaime, Green, Liz, Gregersen, Pernille A., Grigorovski, Nathalia D. A. K., Guedenon, Koffi M., Gunasekera, D. Sanjeeva, Gündüz, Ahmet K., Gupta, Himika, Gupta, Sanjiv, Hadjistilianou, Theodora, Hamel, Patrick, Hamid, Syed A., Hamzah, Norhafizah, Hansen, Eric D., Harbour, J. William, Hartnett, M. Elizabeth, Hasanreisoglu, Murat, Hassan, Sadiq, Hassan, Shadab, Hederova, Stanislava, Hernandez, Jose, Hernandez, Lorelay Marie Carcamo, Hessissen, Laila, Hordofa, Diriba F., Huang, Laura C., Hubbard, G. B., Hummlen, Marlies, Husakova, Kristina, Hussein Al-Janabi, Allawi N., Ida, Russo, Ilic, Vesna R., Jairaj, Vivekaraj, Jeeva, Irfan, Jenkinson, Helen, Ji, Xunda, Jo, Dong Hyun, Johnson, Kenneth P., Johnson, William J., Jones, Michael M., Kabesha, Theophile B. Amani, Kabore, Rolande L., Kaliki, Swathi, Kalinaki, Abubakar, Kantar, Mehmet, Kao, Ling-Yuh, Kardava, Tamar, Kebudi, Rejin, Kepak, Tomas, Keren-Froim, Naama, Khan, Zohora J., Khaqan, Hussain A., Khauv, Phara, Kheir, Wajiha J., Khetan, Vikas, Khodabande, Alireza, Khotenashvili, Zaza, Kim, Jonathan W., Kim, Jeong Hun, Kiratli, Hayyam, Kivelä, Tero T., Klett, Artur, Komba Palet, Jess Elio Kosh, Krivaitiene, Dalia, Kruger, Mariana, Kulvichit, Kittisak, Kuntorini, Mayasari W., Kyara, Alice, Lachmann, Eva S., Lam, Carol P. S., Lam, Geoffrey C., Larson, Scott A., Latinovic, Slobodanka, Laurenti, Kelly D., Le, Bao Han A., Lecuona, Karin, Leverant, Amy A., Li, Cairui, Limbu, Ben, Long, Quah Boon, López, Juan P., Lukamba, Robert M., Lumbroso, Livia, Luna-Fineman, Sandra, Lutfi, Delfitri, Lysytsia, Lesia, Magrath, George N., Mahajan, Amita, Majeed, Abdul Rahim, Maka, Erika, Makan, Mayuri, Makimbetov, Emil K., Manda, Chatonda, Martín Begue, Nieves, Mason, Lauren, Mason, John O., Matende, Ibrahim O., Materin, Miguel, Mattosinho, Clarissa C. D. S., Matua, Marchelo, Mayet, Ismail, Mbumba, Freddy B., McKenzie, John D., Medina-Sanson, Aurora, Mehrvar, Azim, Mengesha, Aemero A., Menon, Vikas, Mercado, Gary John V. D., Mets, Marilyn B., Midena, Edoardo, Mishra, Divyansh K. C., Mndeme, Furahini G., Mohamedani, Ahmed A., Mohammad, Mona T., Moll, Annette C., Montero, Margarita M., Morales, Rosa A., Moreira, Claude, Mruthyunjaya, Prithvi, Msina, Mchikirwa S., Msukwa, Gerald, Mudaliar, Sangeeta S., Muma, Kangwa I., Munier, Francis L., Murgoi, Gabriela, Murray, Timothy G., Musa, Kareem O., Mushtaq, Asma, Mustak, Hamzah, Muyen, Okwen M., Naidu, Gita, Nair, Akshay Gopinathan, Naumenko, Larisa, Ndoye Roth, Paule Aïda, Nency, Yetty M., Neroev, Vladimir, Ngo, Hang, Nieves, Rosa M., Nikitovic, Marina, Nkanga, Elizabeth D., Nkumbe, Henry, Nuruddin, Murtuza, Nyaywa, Mutale, Obono-Obiang, Ghislaine, Oguego, Ngozi C., Olechowski, Andrzej, Oliver, Scott C. N., Osei-Bonsu, Peter, Ossandon, Diego, Paez-Escamilla, Manuel A., Pagarra, Halimah, Painter, Sally L, Paintsil, Vivian, Paiva, Luisa, Pal, Bikramjit P., Palanivelu, Mahesh Shanmugam, Papyan, Ruzanna, Parrozzani, Raffaele, Parulekar, Manoj, Pascual Morales, Claudia R., Paton, Katherine E., Pawinska-Wasikowska, Katarzyna, Pe'er, Jacob, Peña, Armando, Peric, Sanja, Pham, Chau T. M., Philbert, Remezo, Plager, David A., Pochop, Pavel, Polania, Rodrigo A., Polyakov, Vladimir G., Pompe, Manca T., Pons, Jonathan J., Prat, Daphna, Prom, Vireak, Purwanto, Ignatius, Qadir, Ali O., Qayyum, Seema, Qian, Jiang, Rahman, Ardizal, Rahman, Salman, Rahmat, Jamalia, Rajkarnikar, Purnima, Ramanjulu, Rajesh, Ramasubramanian, Aparna, Ramirez-Ortiz, Marco A., Raobela, Léa, Rashid, Riffat, Reddy, M. Ashwin, Reich, Ehud, Renner, Lorna A., Reynders, David, Ribadu, Dahiru, Riheia, Mussagy M., Ritter-Sovinz, Petra, Rojanaporn, Duangnate, Romero, Livia, Roy, Soma R., Saab, Raya H., Saakyan, Svetlana, Sabhan, Ahmed H, Sagoo, Mandeep S., Said, Azza M. A., Saiju, Rohit, Salas, Beatriz, San Román Pacheco, Sonsoles, Sánchez, Gissela L., Sayalith, Phayvanh, Scanlan, Trish A., Schefler, Amy C., Schoeman, Judy, Sedaghat, Ahad, Seregard, Stefan, Seth, Rachna, Shah, Ankoor S., Shakoor, Shawkat A., Sharma, Manoj K., Sherief, Sadik T., Shetye, Nandan G., Shields, Carol L., Siddiqui, Sorath Noorani, Sidi Cheikh, Sidi, Silva, Sónia, Singh, Arun D., Singh, Niharika, Singh, Usha, Singha, Penny, Sitorus, Rita S., Skalet, Alison H., Soebagjo, Hendrian D., Sorochynska, Tetyana, Ssali, Grace, Stacey, Andrew W., Staffieri, Sandra E., Stahl, Erin D., Stathopoulos, Christina, Stirn Kranjc, Branka, Stones, David K., Strahlendorf, Caron, Suarez, Maria Estela Coleoni, Sultana, Sadia, Sun, Xiantao, Sundy, Meryl, Superstein, Rosanne, Supriyadi, Eddy, Surukrattanaskul, Supawan, Suzuki, Shigenobu, Svojgr, Karel, Sylla, Fatoumata, Tamamyan, Gevorg, Tan, Deborah, Tandili, Alketa, Tarrillo Leiva, Fanny F., Tashvighi, Maryam, Tateshi, Bekim, Tehuteru, Edi S., Teixeira, Luiz F., Teh, Kok Hoi, Theophile, Tuyisabe, Toledano, Helen, Trang, Doan L., Traoré, Fousseyni, Trichaiyaporn, Sumalin, Tuncer, Samuray, Tyau-Tyau, Harba, Umar, Ali B., Unal, Emel, Uner, Ogul E., Urbak, Steen F., Ushakova, Tatiana L., Usmanov, Rustam H., Valeina, Sandra, van Hoefen Wijsard, Milo, Varadisai, Adisai, Vasquez, Liliana, Vaughan, Leon O., Veleva-Krasteva, Nevyana V., Verma, Nishant, Victor, Andi A., Viksnins, Maris, Villacís Chafla, Edwin G., Vishnevskia-Dai, Vicktoria, Vora, Tushar, Wachtel, Antonio E., Wackernagel, Werner, Waddell, Keith, Wade, Patricia D., Wali, Amina H., Wang, Yi-Zhuo, Weiss, Avery, Wilson, Matthew W., Wime, Amelia D. C., Wiwatwongwana, Atchareeya, Wiwatwongwana, Damrong, Wolley Dod, Charlotte, Wongwai, Phanthipha, Xiang, Daoman, Xiao, Yishuang, Yam, Jason C., Yang, Huasheng, Yanga, Jenny M., Yaqub, Muhammad A, Yarovaya, Vera A., Yarovoy, Andrey A., Ye, Huijing, Yousef, Yacoub A., Yuliawati, Putu, Zapata López, Arturo M., Zein, Ekhtelbenina, Zhang, Chengyue, Zhang, Yi, Zhao, Junyang, Zheng, Xiaoyu, Zhilyaeva, Katsiaryna, Zia, Nida, Ziko, Othman A. O., Zondervan, Marcia, and Bowman, Richard

Abstract

IMPORTANCE: Early diagnosis of retinoblastoma, the most common intraocular cancer, can save both a child’s life and vision. However, anecdotal evidence suggests that many children across the world are diagnosed late. To our knowledge, the clinical presentation of retinoblastoma has never been assessed on a global scale. OBJECTIVES: To report the retinoblastoma stage at diagnosis in patients across the world during a single year, to investigate associations between clinical variables and national income level, and to investigate risk factors for advanced disease at diagnosis. DESIGN, SETTING, AND PARTICIPANTS: A total of 278 retinoblastoma treatment centers were recruited from June 2017 through December 2018 to participate in a cross-sectional analysis of treatment-naive patients with retinoblastoma who were diagnosed in 2017. MAIN OUTCOMES AND MEASURES: Age at presentation, proportion of familial history of retinoblastoma, and tumor stage and metastasis. RESULTS: The cohort included 4351 new patients from 153 countries; the median age at diagnosis was 30.5 (interquartile range, 18.3-45.9) months, and 1976 patients (45.4%) were female. Most patients (n = 3685 [84.7%]) were from low- and middle-income countries (LMICs). Globally, the most common indication for referral was leukocoria (n = 2638 [62.8%]), followed by strabismus (n = 429 [10.2%]) and proptosis (n = 309 [7.4%]). Patients from high-income countries (HICs) were diagnosed at a median age of 14.1 months, with 656 of 666 (98.5%) patients having intraocular retinoblastoma and 2 (0.3%) having metastasis. Patients from low-income countries were diagnosed at a median age of 30.5 months, with 256 of 521 (49.1%) having extraocular retinoblastoma and 94 of 498 (18.9%) having metastasis. Lower national income level was associated with older presentation age, higher proportion of locally advanced disease and distant metastasis, and smaller proportion of familial history of retinoblastoma. Advanced disease at diagnosis was more common in LMICs even after adjusting for age (odds ratio for low-income countries vs upper-middle–income countries and HICs, 17.92 [95% CI, 12.94-24.80], and for lower-middle–income countries vs upper-middle–income countries and HICs, 5.74 [95% CI, 4.30-7.68]). CONCLUSIONS AND RELEVANCE: This study is estimated to have included more than half of all new retinoblastoma cases worldwide in 2017. Children from LMICs, where the main global retinoblastoma burden lies, presented at an older age with more advanced disease and demonstrated a smaller proportion of familial history of retinoblastoma, likely because many do not reach a childbearing age. Given that retinoblastoma is curable, these data are concerning and mandate intervention at national and international levels. Further studies are needed to investigate factors, other than age at presentation, that may be associated with advanced disease in LMICs.

Published
2020
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19. STAG2/LMO2Gamma-Delta (γδ) T-ALL: Identification and Characterization of an Extremely High Risk Group of T-ALL in the Very Young

Author

Kimura, Shunsuke, Pölönen, Petri, Montefiori, Lindsey, Caldwell, Kenneth, Iacobucci, Ilaria, Chen, Chelsey, Brown, Anthony, Han, Katie, Liu, Yen-Chun, Chang, Yunchao, Cheng, Zhongshan, Yinmei, Zhou, Park, Chun Shik, Mitchell, Sharnise, Reyes, Noemi, Yeoh, Allen, Attarbaschi, Andishe, Moore, Andrew, Manabe, Atsushi, Buldini, Barbara, Chiew, Kean Hui, Li, Chi Kong, Pui, Ching-Hon, Qu, Chunxu, Tomizawa, Daisuke, Paietta, Elisabeth, Locatelli, Franco, Escherich, Gabriele, Qingsong, Gao, Muhle, Hannah Elisa, Marquart, Hanne Vibeke, de Groot-Kruseman, Hester A., Rowe, Jacob M., Stary, Jan, Trka, Jan, Choi, John Kim, Meijerink, Jules P.P., Takita, Junko, Pawinska-Wasikowska, Katarzyna, Schmiegelow, Kjeld, Eguchi, Mariko, Schrappe, Martin, Zimmermann, Martin, Takagi, Masatoshi, Maybury, Melissa, Svaton, Michael, Reiterova, Michaela, Kicinski, Michal, Kato, Motohiro, Spinelli, Orietta, Mazilier, Pauline, Thomas, Paul G., Masetti, Riccardo, Kotecha, Rishi Sury, Pieters, Rob, Elitzur, Sarah, Luger, Selina M, Shen, Shuhong, Jeha, Sima, Kornblau, Steven M., Skoczen, Szymon, Miyamura, Takako, Vincent, Tiffaney, Imamura, Toshihiko, Conter, Valentino, Tang, Yanjing, Gu, Zhaohui, Roberts, Kathryn G., Teachey, David T., Crews, Kristine R, Cheng, Cheng, Yang, Jun J., Inaba, Hiroto, and Mullighan, Charles G.

Abstract

Background

Published
2023
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20. Development of treatment and clinical results in childhood acute myeloid leukemia in Poland

Author

Balwierz, Walentyna, primary, Pawinska-Wasikowska, Katarzyna, additional, Klekawka, Tomasz, additional, Czogala, Malgorzata, additional, Matysiak, Michal, additional, Fic-Sikorska, Barbara, additional, Adamkiewicz-Drozynska, Elzbieta, additional, Maciejka-Kapuscinska, Lucyna, additional, Chybicka, Alicja, additional, Potocka, Kinga, additional, Wachowiak, Jacek, additional, Skalska-Sadowska, Jolanta, additional, Kowalczyk, Jerzy, additional, Wojcik, Beata, additional, Wysocki, Mariusz, additional, Koltan, Sylwia, additional, Krawczuk-Rybak, Maryna, additional, Muszynska-Roslan, Katarzyna, additional, Mlynarski, Wojciech, additional, Stolarska, Malgorzata, additional, Urasinski, Tomasz, additional, Kamienska, Elzbieta, additional, Szczepanski, Tomasz, additional, Tomaszewska, Renata, additional, Sobol, Grazyna, additional, Mizia-Malarz, Agnieszka, additional, Karolczyk, Grazyna, additional, Podhorecka, Joanna, additional, Wieczorek, Maria, additional, Karpinska-Derda, Irena, additional, Badowska, Wanda, additional, and Moryl-Bujakowska, Angelina, additional

Published
2012
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21. Improvement of the Treatment Results in Childhood Acute Myeloid Leukemia in Poland from 1983 to 2017

Author

Balwierz, Walentyna, Czogala, Malgorzata, Pawinska-Wasikowska, Katarzyna, Wachowiak, Jacek, Skalska-Sadowska, Jolanta, Michał Matysiak, Szczepanski, Tomasz, Tomaszewska, Renata, Krawczuk-Rybak, Maryna, Muszynska-Roslan, Katarzyna, Kowalczyk, Jerzy R., Wisniewska-Slusarz, Hanna, Mlynarski, Wojciech, Chybicka, Alicja, Rodziewicz, Anna, Urasinski, Tomasz, Adamkiewicz-Drozynska, Elzbieta, Maciejka-Kemblowska, Lucyna, Karolczyk, Grazyna, Karpinska-Derda, Irena, Przybyszewski, Borys, Malarz-Mizia, Agnieszka, Koltan, Sylwia, and Chaber, Radoslaw

22. Improvement of the Treatment Results in Childhood Acute Myeloid Leukemia in Poland from 1983 to 2017

Author

Balwierz, Walentyna, Czogala, Malgorzata, Pawinska-Wasikowska, Katarzyna, Wachowiak, Jacek, Skalska-Sadowska, Jolanta, Matysiak, Michal, Szczepanski, Tomasz, Tomaszewska, Renata, Krawczuk-Rybak, Maryna, Muszynska-Roslan, Katarzyna, Kowalczyk, Jerzy R., Wisniewska-Slusarz, Hanna, Mlynarski, Wojciech, Chybicka, Alicja, Rodziewicz, Anna, Urasinski, Tomasz, Adamkiewicz-Drozynska, Elzbieta, Maciejka-Kemblowska, Lucyna, Karolczyk, Grazyna, Karpinska-Derda, Irena, Przybyszewski, Borys, Malarz-Mizia, Agnieszka, Koltan, Sylwia, and Chaber, Radoslaw

Abstract

Introduction: Acute myeloid leukemia (AML), heterogeneous group of malignant neoplasm of hematopoietic system, comprises of 15% of all leukemias in children. Standardized AML-BFM based protocols of the treatment of AML introduced in Poland from 1983 enabled successive improvement of survival rates. Before 1983 only 10% of children with AML in our country had chance to survive.

Published
2017
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23. Biologic and Clinical Analysis of Childhood Gamma Delta T-ALL Identifies LMO2/STAG2 Rearrangements as Extremely High Risk.

Author

Kimura S, Park CS, Montefiori LE, Iacobucci I, Pölönen P, Gao Q, Arnold ED, Attarbaschi A, Brown A, Buldini B, Caldwell KJ, Chang Y, Chen C, Cheng C, Cheng Z, Choi J, Conter V, Crews KR, de Groot-Kruseman HA, Deguchi T, Eguchi M, Muhle HE, Elitzur S, Escherich G, Freeman BB 3rd, Gu Z, Han K, Horibe K, Imamura T, Jeha S, Kato M, Chiew KH, Khan T, Kicinski M, Köhrer S, Kornblau SM, Kotecha RS, Li CK, Liu YC, Locatelli F, Luger SM, Paietta EM, Manabe A, Marquart HV, Masetti R, Maybury M, Mazilier P, Meijerink JPP, Mitchell S, Miyamura T, Moore AS, Oshima K, Pawinska-Wasikowska K, Pieters R, Prater MS, Pruett-Miller SM, Pui CH, Qu C, Reiterova M, Reyes N, Roberts KG, Rowe JM, Sato A, Schmiegelow K, Schrappe M, Shen S, Skoczeń S, Spinelli O, Stary J, Svaton M, Takagi M, Takita J, Tang Y, Teachey DT, Thomas PG, Tomizawa D, Trka J, Varotto E, Vincent TL, Yang JJ, Yeoh AEJ, Zhou Y, Zimmermann M, Inaba H, and Mullighan CG

Subjects
Humans, Child, Preschool, Male, Female, Precursor T-Cell Lymphoblastic Leukemia-Lymphoma genetics, Precursor T-Cell Lymphoblastic Leukemia-Lymphoma pathology, Infant, Child, Cell Cycle Proteins genetics, Cell Cycle Proteins metabolism, Gene Rearrangement, Proto-Oncogene Proteins, LIM Domain Proteins genetics, Adaptor Proteins, Signal Transducing genetics, Adaptor Proteins, Signal Transducing metabolism
Abstract

Acute lymphoblastic leukemia expressing the gamma delta T-cell receptor (γδ T-ALL) is a poorly understood disease. We studied 200 children with γδ T-ALL from 13 clinical study groups to understand the clinical and genetic features of this disease. We found age and genetic drivers were significantly associated with outcome. γδ T-ALL diagnosed in children under 3 years of age was extremely high-risk and enriched for genetic alterations that result in both LMO2 activation and STAG2 inactivation. Mechanistically, using patient samples and isogenic cell lines, we show that inactivation of STAG2 profoundly perturbs chromatin organization by altering enhancer-promoter looping, resulting in deregulation of gene expression associated with T-cell differentiation. High-throughput drug screening identified a vulnerability in DNA repair pathways arising from STAG2 inactivation, which can be targeted by poly(ADP-ribose) polymerase inhibition. These data provide a diagnostic framework for classification and risk stratification of pediatric γδ T-ALL. Significance: Patients with acute lymphoblastic leukemia expressing the gamma delta T-cell receptor under 3 years old or measurable residual disease ≥1% at end of induction showed dismal outcomes and should be classified as having high-risk disease. The STAG2/LMO2 subtype was enriched in this very young age group. STAG2 inactivation may perturb chromatin conformation and cell differentiation and confer vulnerability to poly(ADP-ribose) polymerase inhibition., (©2024 American Association for Cancer Research.)

Published
2024
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24. Biologic and clinical features of childhood gamma delta T-ALL: identification of STAG2/LMO2 γδ T-ALL as an extremely high risk leukemia in the very young.

Author

Kimura S, Polonen P, Montefiori L, Park CS, Iacobucci I, Yeoh AE, Attarbaschi A, Moore AS, Brown A, Manabe A, Buldini B, Freeman BB, Chen C, Cheng C, Kean Hui C, Li CK, Pui CH, Qu C, Tomizawa D, Teachey DT, Varotto E, Paietta EM, Arnold ED, Locatelli F, Escherich G, Elisa Muhle H, Marquart HV, de Groot-Kruseman HA, Rowe JM, Stary J, Trka J, Choi JK, Meijerink JPP, Yang JJ, Takita J, Pawinska-Wasikowska K, Roberts KG, Han K, Caldwell KJ, Schmiegelow K, Crews KR, Eguchi M, Schrappe M, Zimmerman M, Takagi M, Maybury M, Svaton M, Reiterova M, Kicinski M, Prater MS, Kato M, Reyes N, Spinelli O, Thomas P, Mazilier P, Gao Q, Masetti R, Kotecha RS, Pieters R, Elitzur S, Luger SM, Mitchell S, Pruett-Miller SM, Shen S, Jeha S, Köhrer S, Kornblau SM, Skoczeń S, Miyamura T, Vincent TL, Imamura T, Conter V, Tang Y, Liu YC, Chang Y, Gu Z, Cheng Z, Yinmei Z, Inaba H, and Mullighan CG

Abstract

Purpose: Gamma delta T-cell receptor-positive acute lymphoblastic leukemia (γδ T-ALL) is a high-risk but poorly characterized disease., Methods: We studied clinical features of 200 pediatric γδ T-ALL, and compared the prognosis of 93 cases to 1,067 protocol-matched non-γδ T-ALL. Genomic features were defined by transcriptome and genome sequencing. Experimental modeling was used to examine the mechanistic impacts of genomic alterations. Therapeutic vulnerabilities were identified by high throughput drug screening of cell lines and xenografts., Results: γδ T-ALL in children under three was extremely high-risk with 5-year event-free survival (33% v. 70% [age 3-<10] and 73% [age ≥10], P =9.5 x 10 -5 ) and 5-year overall survival (49% v. 78% [age 3-<10] and 81% [age ≥10], P =0.002), differences not observed in non-γδ T-ALL. γδ T-ALL in this age group was enriched for genomic alterations activating LMO2 activation and inactivating STAG2 inactivation ( STAG2/LMO2 ). Mechanistically, we show that inactivation of STAG2 profoundly perturbs chromatin organization by altering enhancer-promoter looping resulting in deregulation of gene expression associated with T-cell differentiation. Drug screening showed resistance to prednisolone, consistent with clinical slow treatment response, but identified a vulnerability in DNA repair pathways arising from STAG2 inactivation, which was efficaciously targeted by Poly(ADP-ribose) polymerase (PARP) inhibition, with synergism with HDAC inhibitors. Ex-vivo drug screening on PDX cells validated the efficacy of PARP inhibitors as well as other potential targets including nelarabine., Conclusion: γδ T-ALL in children under the age of three is extremely high-risk and enriched for STAG2/LMO2 ALL. STAG2 loss perturbs chromatin conformation and differentiation, and STAG2/LMO2 ALL is sensitive to PARP inhibition. These data provide a diagnostic and therapeutic framework for pediatric γδ T-ALL., Support: The authors are supported by the American and Lebanese Syrian Associated Charities of St Jude Children's Research Hospital, NCI grants R35 CA197695, P50 CA021765 (C.G.M.), the Henry Schueler 41&9 Foundation (C.G.M.), and a St. Baldrick's Foundation Robert J. Arceci Innovation Award (C.G.M.), Gabriella Miller Kids First X01HD100702 (D.T.T and C.G.M.) and R03CA256550 (D.T.T. and C.G.M.), F32 5F32CA254140 (L.M.), and a Garwood Postdoctoral Fellowship of the Hematological Malignancies Program of the St Jude Children's Research Hospital Comprehensive Cancer Center (S.K.). This project was supported by the National Cancer Institute of the National Institutes of Health under the following award numbers: U10CA180820, UG1CA189859, U24CA114766, U10CA180899, U10CA180866 and U24CA196173., Disclaimer: The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health. The funding agencies were not directly involved in the design of the study, gathering, analysis and interpretation of the data, writing of the manuscript, or decision to submit the manuscript for publication.

Published
2023
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