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1. Profile of Luspatercept in the Treatment of Anemia in Adults with Non-Transfusion-Dependent β-Thalassemia (NTDT): Design, Development and Potential Place in Therapy

Author

Musallam KM, Taher AT, Kattamis A, Kuo KH, Sheth S, and Cappellini MD

Subjects
anemia, ineffective erythropoiesis, iron, management, thalassemia, transfusion., Therapeutics. Pharmacology, RM1-950
Abstract

Khaled M Musallam,1 Ali T Taher,2 Antonis Kattamis,3 Kevin HM Kuo,4 Sujit Sheth,5 Maria Domenica Cappellini6 1Thalassemia Center, Burjeel Medical City, Abu Dhabi, United Arab Emirates; 2Department of Internal Medicine, American University of Beirut Medical Center, Beirut, Lebanon; 3First Department of Pediatrics, National and Kapodistrian University of Athens, Athens, Greece; 4Division of Hematology, University of Toronto, Toronto, ON, Canada; 5Division of Hematology and Oncology, Department of Pediatrics, Weill Cornell Medicine, New York, NY, USA; 6Department of Clinical Sciences and Community, University of Milan, Ca’ Granda Foundation IRCCS Maggiore Policlinico Hospital, Milan, ItalyCorrespondence: Maria Domenica Cappellini, Department of Clinical Sciences and Community, University of Milan, Ca’ Granda Foundation IRCCS Maggiore Policlinico Hospital, Milan, 20122, Italy, Tel + 39 02 5503 3752, Email maria.cappellini@unimit.itAbstract: Over the past decade, evidence has been mounting on the detrimental clinical sequelae of untreated anemia in patients with non-transfusion-dependent β-thalassemia (NTDT). There are no pharmacologic agents that are specifically approved for the management of anemia in NTDT, and available options such as splenectomy, transfusion therapy, and hydroxyurea each come with their own shortcomings, especially for long-term use. Luspatercept is an erythroid maturation agent that has been evaluated in a Phase 2, randomized trial and showed a significant benefit in raising hemoglobin level by at least 1 g/dL in adults with NTDT and a baseline hemoglobin level ≤ 10 g/dL. These data led to luspatercept’s approval by the European Commission for the treatment of anemia in adults with NTDT and presents the first evidence-based approach for a novel agent that is able to ameliorate anemia in this patient population.Keywords: anemia, ineffective erythropoiesis, iron, management, thalassemia, transfusion

Published
2023

2. Recommendations for diagnosis, treatment, and prevention of iron deficiency and iron deficiency anemia.

Author

Iolascon, Achille, Andolfo, Immacolata, Russo, Roberta, Sanchez, Mayka, Busti, Fabiana, Swinkels, Dorine, Aguilar Martinez, Patricia, Bou-Fakhredin, Rayan, Muckenthaler, Martina, Unal, Sule, Porto, Graça, Ganz, Tomas, Kattamis, Antonis, De Franceschi, Lucia, Cappellini, Maria, Munro, Malcolm, and Taher, Ali

Abstract

Iron is an essential nutrient and a constituent of ferroproteins and enzymes crucial for human life. Generally, nonmenstruating individuals preserve iron very efficiently, losing less than 0.1% of their body iron content each day, an amount that is replaced through dietary iron absorption. Most of the iron is in the hemoglobin (Hb) of red blood cells (RBCs); thus, blood loss is the most common cause of acute iron depletion and anemia worldwide, and reduced hemoglobin synthesis and anemia are the most common consequences of low plasma iron concentrations. The term iron deficiency (ID) refers to the reduction of total body iron stores due to impaired nutrition, reduced absorption secondary to gastrointestinal conditions, increased blood loss, and increased needs as in pregnancy. Iron deficiency anemia (IDA) is defined as low Hb or hematocrit associated with microcytic and hypochromic erythrocytes and low RBC count due to iron deficiency. IDA most commonly affects women of reproductive age, the developing fetus, children, patients with chronic and inflammatory diseases, and the elderly. IDA is the most frequent hematological disorder in children, with an incidence in industrialized countries of 20.1% between 0 and 4 years of age and 5.9% between 5 and 14 years (39% and 48.1% in developing countries). The diagnosis, management, and treatment of patients with ID and IDA change depending on age and gender and during pregnancy. We herein summarize what is known about the diagnosis, treatment, and prevention of ID and IDA and formulate a specific set of recommendations on this topic.

Published
2024

3. Efficacy and safety of iron-chelation therapy with deferoxamine, deferiprone, and deferasirox for the treatment of iron-loaded patients with nontransfusion-dependent thalassemia syndromes

Author

Taher AT, Porter JB, Kattamis A, Viprakasit V, and Cappellini MD

Subjects
Clinical iron overload, Iron chelation, non-transfusion-dependent thalassemia, Therapeutics. Pharmacology, RM1-950
Abstract

Ali T Taher,1 John B Porter,2 Antonis Kattamis,3 Vip Viprakasit,4 M Domenica Cappellini51Department of Internal Medicine, American University of Beirut, Beirut, Lebanon; 2Department of Haematology, University College London, London, UK; 3First Department of Pediatrics, University of Athens, Athens, Greece; 4Department of Pediatrics and Thalassemia Center, Siriraj Hospital, Mahidol University, Bangkok, Thailand; 5Department of Internal Medicine, Università di Milano, Ca Granda Foundation IRCCS, Milan, ItalyAs the scientific steering committee for THALASSA (an assessment of Exjade in nontransfusion-dependent thalassemia [NTDT]), we read with interest the review by Kontoghiorghe and Kontoghiorghes entitled “Efficacy and safety of iron-chelation therapy with deferoxamine, deferiprone, and deferasirox for the treatment of iron-loaded patients with non-transfusion-dependent thalassemia syndromes” published in January 2016.1 While this review provides a detailed overview of available iron chelators for the treatment of NTDT patients, there remain some factual inaccuracies and misrepresentations of data related to deferasirox. Therefore, we believe that the current article may be misleading to readers of Drug Design, Development and Therapy.Author’s replyGeorge J KontoghiorghesPostgraduate Research Institute of Science, Technology, Environment and Medicine, Limassol, CyprusThere are many murky areas and marketing, legal, ethical, and other conflicts in the pharmaceutical industry, some of which involve physicians and academics. These activities and related ethical issues affect the safety and treatment of millions of patients.1–11 Irregular and sometimes illegal activities for new patented drugs carried out by pharmaceutical companies, such as secrecy agreements with academics/academic institutions, can lead to biased reporting of the results of clinical trials and cover ups or underreporting of toxic side effects, as well as doctor’s bribes, irregularities in drug pricing, corruption of the drug regulatory authorities, influential medical journals and patient organizations, etc.1–11 The commercial influence and drug marketing tactics by pharmaceutical companies in the case of deferasirox affect the safety and long-term survival of thousands of thalassemia and other categories of transfused patients.11–14View the original paper by Kontoghiorghe and Kontoghiorghes.

Published
2016

4. IMPROVEMENTS IN FATIGUE AND 6-MINUTE WALK TEST IN ADULTS WITH ALPHA- OR BETA-NON–TRANSFUSION-DEPENDENT THALASSEMIA: THE PHASE 3 ENERGIZE TRIAL OF MITAPIVAT

Author

KH Kuo, H Al-Samkari, Y Aydinok, M Besser, S Gheuens, G Luna, A Glenthj, AS Goh, A Kattamis, SR Loggetto, KM Musallam, P Ricchi, E Salido-Fiérrez, S Sheth, V Viprakasit, MD Cappellini, and AT Taher

Subjects
Diseases of the blood and blood-forming organs, RC633-647.5
Abstract

Background: Thalassemia, a group of inherited disorders characterized by ineffective erythropoiesis and chronic hemolytic anemia, is associated with wide-ranging impacts on health-related quality of life (HRQoL), such as impaired physical functioning and fatigue. Anemia has been associated with increased symptom burden and poor HRQoL in patients (pts) with non–transfusion-dependent thalassemia (NTDT). There are no available oral disease-modifying therapies that have been shown to improve HRQoL in β-thalassemia and no agents are approved for α-thalassemia. In a phase 2 study of pts with NTDT, improvements in hemoglobin were observed with mitapivat, a first-in-class, oral, allosteric activator of pyruvate kinase, and it has the potential to improve HRQoL. Aims: To evaluate the impact of mitapivat vs placebo on fatigue, physical function, and other thalassemia symptoms in adults with α- or β-NTDT in ENERGIZE, a phase 3, double-blind, randomized, placebo-controlled, global trial. Methods: Adults (≥18 years) were randomized 2:1 to mitapivat 100 mg twice daily or placebo for 24 weeks (wks). NTDT was defined as ≤5 red blood cell (RBC) units transfused in the 24 wks before randomization and no RBC transfusions ≤8 wks before informed consent or during screening. The Functional Assessment of Chronic Illness Therapy–Fatigue Scale (FACIT-Fatigue), 6-Minute Walk Test (6MWT), and Patient Global Impression of Change (PGIC) of Fatigue, Thalassemia Symptoms, and Walking Capacity were among the outcomes assessed. Changes from baseline (BL) for FACIT-Fatigue (Wks 12–24) and 6MWT (Wk 24), and the results of PGIC-Fatigue, (Wks 12–24), PGIC-Thalassemia Symptoms, and PGIC-Walking Capacity (both Wk 24) were summarized. The clinically meaningful within-person change (MWPC) threshold for FACIT-Fatigue was estimated to be a ≥4.5-point change from BL in average score from Wks 12–24, using an anchor-based method. Results: 194 pts were randomized (mitapivat n = 130; placebo n = 64); BL characteristics were similar between treatment arms. Mitapivat demonstrated a statistically significant improvement compared with placebo in change from BL to Wk 12–24 average FACIT-Fatigue score; least-squares mean (LSM) change from BL was 4.85 for mitapivat vs 1.46 for placebo (LSM difference (95% CI): 3.40 (1.21, 5.59); 2-sided p < 0.0026), and 36.2% of pts in the mitapivat arm achieved the MWPC threshold of ≥4.5 vs 21.9% in the placebo arm. For the 6MWT, LSM change from BL to Wk 24 was 30.48 m for mitapivat and 7.11 m for placebo (LSM difference (95% CI): 23.36 m (6.90, 39.83)). The observed frequency of pts with improvements (reporting feeling much/a little better) in PGIC-Fatigue was higher for pts in the mitapivat arm than the placebo arm at Wk 12 (63.1% vs 23.4%), Wk 16 (69.2% vs 23.4%), Wk 20 (62.3% vs 28.1%), and Wk 24 (60.8% vs 31.3%). Improvements in PGIC-Thalassemia Symptoms and PGIC-Walking Capacity were also reported in a higher frequency of pts in the mitapivat arm than in the placebo arm at Wk 24 (67.7% vs 32.8%; 55.4% vs 28.1%, respectively). Summary/Conclusion: Mitapivat is the first oral, disease-modifying, investigational therapy with which meaningful improvements in aspects of HRQoL, including fatigue and walking capacity, were observed in a clinical trial that enrolled both pts with α-NTDT and pts with β-NTDT.

Published
2024
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5. ENERGIZE: A GLOBAL PHASE 3 STUDY OF MITAPIVAT DEMONSTRATING EFFICACY AND SAFETY IN ADULTS WITH ALPHA- OR BETA-NON–TRANSFUSION-DEPENDENT THALASSEMIA

Author

AT Taher, H Al-Samkari, Y Aydinok, M Besser, G Luna, S Gheuens, A Glenthj, AS Goh, A Kattamis, SR Loggetto, KM Musallam, P Ricchi, E Salido-Fiérrez, S Sheth, V Viprakasit, MD Cappellini, and KH Kuo

Subjects
Diseases of the blood and blood-forming organs, RC633-647.5
Abstract

Background: In thalassemia, ATP production in erythroid cells is too low to meet the demand of oxidative stress and ensuing cellular damage; this leads to ineffective erythropoiesis (IE) and chronic hemolytic anemia. Guidelines for non–transfusion-dependent thalassemia (NTDT) recommend raising hemoglobin (Hb) by ≥1 g/dL to reduce morbidities from IE and anemia. No oral disease-modifying therapies are approved for the treatment of β-thalassemia, and no agents are approved for α-thalassemia. Mitapivat is a first-in-class, oral, activator of pyruvate kinase that increases ATP production. Mitapivat may reduce metabolic stress, addressing the underlying pathophysiology across the full range of thalassemias, with the potential to reduce complications and improve health-related quality of life (HRQoL). Aims: To assess the efficacy and safety of mitapivat vs placebo (pbo) in adults with α- or β-NTDT in ENERGIZE (NCT04770753), a phase 3, double-blind, randomized, pbo-controlled, global trial. Methods: Adults (≥18 years) with α- or β-NTDT and baseline (BL) Hb ≤10 g/dL were randomized 2:1 to mitapivat 100 mg twice daily or pbo for 24 weeks (wks). NTDT was defined as ≤5 red blood cell (RBC) units transfused 24 wks before randomization and no RBC transfusions ≤8 wks before informed consent or during screening. The primary endpoint was Hb response: ≥1.0 g/dL increase in average Hb concentration over Wks 12–24 compared with BL. Key secondary endpoints were changes from BL in average Hb concentration and Functional Assessment of Chronic Illness Therapy–Fatigue Scale (FACIT-Fatigue) score over Wks 12–24. Safety and markers of hemolysis and erythropoiesis were among the secondary endpoints. Results: 194 patients (pts) were randomized (mitapivat n = 130; pbo n = 64); 94.8% completed the 24-wk trial. Mean age was 41.2 years, mean BL Hb was 8.3 g/dL, 86.6% received no transfusions in the 24 wks before randomization, and 32.0% had α-NTDT. BL characteristics were similar between treatment arms. Mitapivat demonstrated statistically significant improvements vs pbo for Hb response (42.3% vs 1.6%, respectively; 2-sided p < 0.0001), and for changes from BL in Wks 12–24 average Hb (least-squares mean [LSM] difference (95% CI): 0.96 g/dL (0.78, 1.15); 2-sided p < 0.0001) and Wks 12–24 average FACIT-Fatigue score (LSM difference (95% CI): 3.40 (1.21, 5.59); 2-sided p < 0.0026). Results favored mitapivat across all prespecified subgroups. Improvements in several markers of hemolysis and erythropoiesis were also observed, consistent with the proposed mechanism of mitapivat. The proportion of pts with treatment-emergent adverse events (TEAEs) of any grade was similar across treatment arms (mitapivat 82.9%; pbo 79.4%). The most common TEAEs (≥10% of pts) with mitapivat were headache, initial insomnia, nausea, and upper respiratory tract infection. Among mitapivat-treated pts, 6.2% had serious TEAEs (none considered treatment related) and 3.1% had TEAEs leading to treatment discontinuation; none occurred with pbo. Summary/Conclusion: Mitapivat significantly increased Hb and improved fatigue vs pbo; improvements were observed across all prespecified subgroups. Mitapivat was generally well tolerated with a low treatment discontinuation rate. These data are the first proof of efficacy of a disease-modifying therapy across the full range of NTDT (α- and β-thalassemia). Mitapivat may represent a new oral treatment option addressing both pathophysiology and HRQoL in thalassemia.

Published
2024
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7. Recommendations for diagnosis, treatment, and prevention of iron deficiency and iron deficiency anemia

Author

Achille Iolascon, Immacolata Andolfo, Roberta Russo, Mayka Sanchez, Fabiana Busti, Dorine Swinkels, Patricia Aguilar Martinez, Rayan Bou‐Fakhredin, Martina U. Muckenthaler, Sule Unal, Graça Porto, Tomas Ganz, Antonis Kattamis, Lucia De Franceschi, Maria Domenica Cappellini, Malcolm G. Munro, Ali Taher, and from EHA‐SWG Red Cell and Iron

Subjects
Diseases of the blood and blood-forming organs, RC633-647.5
Abstract

Abstract Iron is an essential nutrient and a constituent of ferroproteins and enzymes crucial for human life. Generally, nonmenstruating individuals preserve iron very efficiently, losing less than 0.1% of their body iron content each day, an amount that is replaced through dietary iron absorption. Most of the iron is in the hemoglobin (Hb) of red blood cells (RBCs); thus, blood loss is the most common cause of acute iron depletion and anemia worldwide, and reduced hemoglobin synthesis and anemia are the most common consequences of low plasma iron concentrations. The term iron deficiency (ID) refers to the reduction of total body iron stores due to impaired nutrition, reduced absorption secondary to gastrointestinal conditions, increased blood loss, and increased needs as in pregnancy. Iron deficiency anemia (IDA) is defined as low Hb or hematocrit associated with microcytic and hypochromic erythrocytes and low RBC count due to iron deficiency. IDA most commonly affects women of reproductive age, the developing fetus, children, patients with chronic and inflammatory diseases, and the elderly. IDA is the most frequent hematological disorder in children, with an incidence in industrialized countries of 20.1% between 0 and 4 years of age and 5.9% between 5 and 14 years (39% and 48.1% in developing countries). The diagnosis, management, and treatment of patients with ID and IDA change depending on age and gender and during pregnancy. We herein summarize what is known about the diagnosis, treatment, and prevention of ID and IDA and formulate a specific set of recommendations on this topic.

Published
2024
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8. Use of HSC-targeted LNP to generate a mouse model of lethal α-thalassemia and treatment via lentiviral gene therapy

Author

Chappell, Maxwell E., Breda, Laura, Tricoli, Lucas, Guerra, Amaliris, Jarocha, Danuta, Castruccio Castracani, Carlo, Papp, Tyler E., Tanaka, Naoto, Hamilton, Nolan, Triebwasser, Michael P., Ghiaccio, Valentina, Fedorky, Megan T., Gollomp, Kandace L., Bochenek, Veronica, Roche, Aoife M., Everett, John K., Cook, Emma J., Bushman, Frederic D., Teawtrakul, Nattiya, Glentis, Stavros, Kattamis, Antonis, Mui, Barbara L., Tam, Ying K., Weissman, Drew, Abdulmalik, Osheiza, Parhiz, Hamideh, and Rivella, Stefano

Published
2024
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9. Retrospective comparative analysis of two medical evacuation systems for Ukrainian patients affected by war

Author

Adyrov, Mykhaylo V, Alanbousi, Inna, Alexander, Sarah Weeks, Apel, Anna, Avula, Meghana, Bal, Wioletta Anna, Balwierz, Walentyna Aniela, Basset-Salom, Luisa, Bastardo Blanco, Daniel, Bauer, Karolina Jadwiga, Bayazitov, Ildar T, Berlanga, Pablo, Bhakta, Nickhill Hitesh, Bieniek, Katarzyna Anna, Bien, Ewa Iwona, Blackwood, Christopher Andrew, Blair, Sally Jane, Bodak, Khrystyna Ihorivna, Bordeianu, Irina, Bouffet, Eric Eric, Braganca, Joao Maria, Bucurenci, Mihaela Silvia, Budny, Elżbieta Beata, Budzyn, Andrii, Bumgardner, Christopher Carl, Burditt, Raina Nichole, Burnside Clapp, Victoria, Bykov, Viacheslav Valeriyovych, Cañete, Adela, Carnelli, Monica, Cela, Elena, Cepowska, Zuzanna, Chaber, Radoslaw, Cherner-Drieux, Anna, Chubata, Mariya, Clough, Heidi M, Czauderna, Piotr Stefan, Czernicka - Siwecka, Jolanta, Czyzewski, Krzysztof, Dalle, Jean-Hugues, Dashchakovska, Olha, de Koning, Linda A, Dembowska-Baginska, Bozenna Malgorzata, Derwich, Katarzyna, Dirksen, Uta, Dommett, Rachel, Dorosh, Olha Ihorivna, dos Reis Farinha, Nuno Jorge, Drabko, Katarzyna Anna, Dragomir, Monica Desiree, Dutkiewicz, Malgorzata, Dworzak, Michael, Dyma, Sergii Vitaliiovych, Earl, Julian, Eggert, Angelika, English, Martin William, Farren, Becky S, Fedyk, Nataliia Yuriina, Fernández-Teijeiro, Ana, Ferneza, Severyn Romanovych, Foster, Whitney Baer, Fox Irwin, Leeanna Elizabeth, Gałązkowski, Robert Maciej, Ganieva, Galyna, Garanzha, Vasylyna Andriivna, Gelman, Marina S, Godzinski, Jan Krzysztof, Goeres, Anne Françoise, Golban, Rodica, Graetz, Dylan Elizabeth, Greiner, Jeanette, Griksaitis, Michael J, Gupta, Sumit, Hampel, Michal Andrzej, Hastings, Sara Grace, Heenen, Delphine Liliane, Hill, Marcela C, Holiuk, Ihor, Holter, Wolfgang, Hough, Rachael Emma, Hutnik, Lukasz Marek, Irga-Jaworska, Ninela, Istomin, Oleksandr Andriyovych, Ignatova, Anna, Janczar, Szymon Lech, Kacharian, Arman, Kalwak, Krzysztof, Karolczyk, Grażyna Małgorzata, Karpenko, Nataliia Mikolaivna, Katsubo, Halyna Oleksandrivna, Kattamis, Antonis, Kazanowska, Bernarda Jadwiga, Kentsis, Alex, Ketteler, Petra, Kienesberger, Anita, Kiselev, Roman, Kizyma, Zoryana Petrivna, Kliuchkivska, Khrystyna, Klymniuk, Hryhorii Ivanovych, Kolenova, Alexandra, Kolodrubiec, Julia, Kostiuk, Yuliia, Kowalik, Tomasz, Kozlova, Olena Igorivna, Kozubenko, Vladyslav, Kraal, Kathelijne, Kramar, Tetyana Oleksandrivna, Krawczuk-Rybak, Maryna Maryna, Kulemzina, Irina, Kurkowska, Paulina, Kuzyk, Andriy S., Ladenstein, Ruth Lydia, Laguna, Pawel Jozef, Lassaletta, Alvaro, Lehmberg, Kai, Leontieva, Oksana, Liashenko, Serhii, Loizou, Loizos G., Lucchetta, Sonia Anna, Lupo, Matthew William, Lysytsia, Lesya, Lysytsia, Oleksandr, Machnik, Katarzyna Anna, Massimino, Maura, Mainland, Jeff A, Matczak, Katarzyna, Matysiak, Michal Jacek, Mayeur, Pierre, Miller, Beth Anne, Minervina, Anastasia A, Mishkova, Volha, Mizia-Malarz, Agnieszka Joanna, Morales La Madrid, Andres, Moreira, Daniel C, Moreno, Lucas, Moskvin, Vadim P, Mukkada, Sheena Teresa, Muszyńska-Rosłan, Katarzyna Maria, Mykychak, Iryna Volodymyrivna, Niemeyer, Charlotte, Nelson, Akoya Janae', Nogovitsyna, Yuliya, Ociepa, Tomasz, Oltolini, Stefano, Onipko, Nataliia, Pappas, Andrew, Patel, Amit B, Patrahau, Alina Alina, Pauley, Jennifer L., Pavlenko, Yehor Mikhailovich, Pavlovych, Andrij Oleksandrovych, Peregud-Pogorzelski, Jarosław Władyslaw, Perek-Polnik, Marta, Pérez, Vanesa, Pérez-Martínez, Antonio, Pikman, Yana, Pitozzi, Graziano Pitozzi, Portugal, Rui Gentil, Posternak, Victoria Vita, Pleshkan, Viktoriya, Prete, Arcangelo, Pritchard-Jones, Kathy, Raciborska, Anna, Radaelli, Alessandra, Reeves, Tegan Jemma, Reinhardt, Dirk, Reshetnyak, Andrey V, Rider, Andrew Jacob, Rizzari, Carmelo, Rizzi, Damiano Damiano, Rodriguez Hermosillo, Karen Gabriela, Ronenko, Olena Volodymyrivna, Rostkowska, Aneta Olga, Rudko, Liudmyla Yaroslavivna, Sakaan, Firas Mohamed, Sakhar, Nadezhda Aleksandrovna, Salman, Zeena S, Savva, Natallia N., Scaccaglia, Davide, Schaeffer, Elizabeth Hawthorne, Schneider, Carina Ursula, Scobie, Nicole, Semeniuk, Olena Volodymyrivna, Shevchyk, Roksoliana, Shuler, Ana I., Shvets, Stanislav, Sniderman, Liz, Skoczen, Szymon Pawel, Smeal, William John, Sokolowski, Igor, Sonkin, Anna Alexandra, Spolinyak, Andriy, Spota, Andrea, Sramkova, Lucie, Stepanjuk, Alla Ivanivna, Sterba, Jaroslav, Strahm, Brigitte, Styczynski, Jan, Svintsova, Olha, Synyuta, Andriy V, Szczepanski, Tomasz, Szczucinski, Pawel Kukiz, Szmyd, Bartosz Miroslaw, Tasso Cereceda, Maria, Teliuk, Alina, Tomanek, Iwona, Topping, Phoebe, Torrent, Montserrat, Trelińska, Joanna, Troyanovska, Olha Orestivna, Tsurkan, Lyudmila G., Tsymbalyuk-Voloshyn, Iryna, Tyupa, Sergiy Ihorovych, Urasinski, Tomasz Franciszek, Urbanek Dądela, Agnieszka, Vasilieva, Nataliia Jroslavivna, Vasilyeva, Aksana, Verdú-Amorós, Jaime, Vilcu-Bajurean, Natalia, Vinitsky, Leo, Vivtcharenko, Victoria, Vovk, Nelia, Volpe, Giovanni, Vorobel, Oksana Ivanivna, Wachowiak, Jacek Tadeusz, Wasiak, Marcin Slawomir, Wiedower, Lance Allan, Wobst, Natalia, Wuenschel, Lena Isolde, Wysocki, Mariusz Stanislaw, Yurieva, Marina, Zagurska, Anastasiia, Zakharenko, Stanislav S, Zakharenko, Aelita V, Zapotochna, Khrystyna, Zawitkowska, Joanna Emilia, Zecca, Marco, Mueller, Alexandra, Salek, Marta, Oszer, Aleksandra, Evseev, Dmitry, Yakimkova, Taisiya, Wlodarski, Marcin, Vinitsky, Anna, Kizyma, Roman, Pogorelyy, Mikhail, Zuber, Maria, Escalante, Juan, Lipska, Elzbieta, Fendler, Wojciech, Nowicka, Zuzanna, Szyszka, Adam, Rodriguez-Galindo, Carlos, Wise, Paul H., Agulnik, Asya, and Mlynarski, Wojciech

Published
2024
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10. Central Nervous System Fungal Diseases in Children with Malignancies: A 16-Year Study from the Infection Working Group of the Hellenic Society of Pediatric Hematology Oncology

Author

Loizos Petrikkos, Maria Kourti, Kondylia Antoniadi, Tatiana-Sultana Tziola, Angeliki-Eleni Sfetsiori, Vasiliki Antari, Sofia Savoukidou, Georgia Avgerinou, Maria Filippidou, Eugenia Papakonstantinou, Sophia Polychronopoulou, Emmanuel Hatzipantelis, Dimitrios Doganis, Antonios Kattamis, Vassilios Papadakis, Emmanuel Roilides, and Athanasios Tragiannidis

Subjects
central nervous system, invasive fungal infection, children, malignancies, outcome, brain abscess, Biology (General), QH301-705.5
Abstract

We analyzed data on pediatric invasive fungal diseases of the central nervous system (CNS-IFDs) reported by five of a total of eight Pediatric Hematology-Oncology Departments in Greece for 16 years (2007–2022). A total of twelve patients (11 boys, median age: 9.5 years, range: 2–16) were reported suffering from CNS-IFDs. The underlying malignancy was acute lymphoblastic leukemia in 9/12 and acute myeloid leukemia, Ewing sarcoma, and rhabdomyosarcoma in one each. Eleven patients presented with CNS-related symptoms (i.e., seizures, headache, cerebral palsy, ataxia, hallucination, seizures, blurred vision, amaurosis). All patients had pathological MRI findings. Multifocal fungal disease was observed in 6/12 patients. Nine proven and three probable CNS-IFD cases were diagnosed. Causative pathogens in proven cases were Aspergillus spp. and Candida albicans (n = 2 each), Mucor spp., Rhizopus arrhizus, Absidia spp., Fusarium oxysporum and Cryptococcus neoformans (n = 1 each). Causative pathogens in probable cases were Aspergillus spp. (n = 2) and Candida spp. (n = 1). All patients received appropriate antifungal therapy (median duration: 69.5 days, range 19–364). Two patients underwent additional surgical treatment. Six patients were admitted to the Intensive Care Unit due to complications. Three patients (25%) died, two due to IFD and one due to an underlying disease. Early recognition and prompt intervention of CNS-IFDs may rescue the patients and improve overall survival.

Published
2024
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11. Worldwide survey of T2* cardiovascular magnetic resonance in Thalassaemia

Author

El-Beshlawy Amal, Rangelova Mirella, Aydinok Yesim, Almeida Ana, Hazirolan Tuncay, Perifanis Vassilios, Michos Andreas, Vini Demetra, Drossou Marouso, Ladis Vassilis, Kattamis Antonis, Forni Gianluca, Danjou Fabrice, Galanello Renzo, Walker Malcolm, Porter John B, Anderson Lisa, Kirk Paul, He Taigang, Carpenter John-Paul, Elalfy Mohsen, Alnasser Ibrahim, Daar Shahina, Fernandes Juliano, and Pennell Dudley J

Subjects
Diseases of the circulatory (Cardiovascular) system, RC666-701
Published
2011
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14. Diagnosis, treatment, and surveillance of Diamond-Blackfan anaemia syndrome: international consensus statement

Author

Wlodarski, Marcin W, Vlachos, Adrianna, Farrar, Jason E, Da Costa, Lydie M, Kattamis, Antonis, Dianzani, Irma, Belendez, Cristina, Unal, Sule, Tamary, Hannah, Pasauliene, Ramune, Pospisilova, Dagmar, de la Fuente, Josu, Iskander, Deena, Wolfe, Lawrence, Liu, Johnson M, Shimamura, Akiko, Albrecht, Katarzyna, Lausen, Birgitte, Bechensteen, Anne Grete, Tedgard, Ulf, Puzik, Alexander, Quarello, Paola, Ramenghi, Ugo, Bartels, Marije, Hengartner, Heinz, Farah, Roula A, Al Saleh, Mahasen, Hamidieh, Amir Ali, Yang, Wan, Ito, Etsuro, Kook, Hoon, Ovsyannikova, Galina, Kager, Leo, Gleizes, Pierre-Emmanuel, Dalle, Jean-Hugues, Strahm, Brigitte, Niemeyer, Charlotte M, Lipton, Jeffrey M, and Leblanc, Thierry M

Published
2024
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15. CAN WE PREDICT INCIPIENT DIABETES MELLITUS IN PATIENTS WITH TRANSFUSION DEPENDENT β-THALASSEMIA (β-TDT) REFERRED WITH A HISTORY OF PREDIABETES?

Author

Vincenzo De Sanctis, Ashraf Soliman, Shahina Daar, Ploutarchos Tzoulis, and Christos Kattamis

Subjects
Transfusion dependent β-thalassemia, OGTT, pancreatic β-cell function, insulin sensitivity/resistance, risk factors, incipient diabetes., Diseases of the blood and blood-forming organs, RC633-647.5
Abstract

Background: Prediabetes and diabetes mellitus (DM) are complications in adult patients with transfusion dependent β-thalassemia (β-TDT), with their incidence increasing with age. Objective: This retrospective observational study describes the glycemic trajectories and evaluates predictive indices of β-cell function and insulin sensitivity/resistance in β-TDT patients with prediabetes, both in a steady state and during 3-h oral glucose tolerance test (OGTT), in order to identify patients at high risk for incipient diabetes. Setting: The study was mainly conducted at the Pediatric and Adolescent Outpatient Clinic, Quisisana Hospital, Ferrara (Italy) in collaboration with thalassemia referring centers across Italy. Patients: The study included 11 β-TDT (aged 15.11- 31.10 years) with history of prediabetes. Methods: The ADA criteria for the diagnosis of glucose dysregulation were adopted. Investigations included evaluation of plasma glucose levels and insulin secretion, analysis of glycemic trajectories and indices of β-cell function and insulin sensitivity/resistance assessed in steady state and during OGTT. Results: The duration of progression from prediabetes to DM, expressed in years, showed a positive direct correlation with corrected insulin response (CIR-30 = r: 0.7606, P: 0.0065), insulinogenic index (IGI 0-120 = r: 0.6121, P:0.045), oral disposition index (oDI = r: 0.7119, P:0.013), insulin growth factor-1 (IGF-1= r: 0.6246, P: 0.039) and an inverse linear correlation with serum ferritin (SF = r: -0.7197, P: 0.012). Conclusions: Progressive β-cell failure, peripheral resistance to the action of insulin and reduction of oDI were the principal factors responsible for the progression from prediabetes to incipient DM.

Published
2024
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16. IMMUNOPHENOTYPE IN SAMD9/9L SYNDROME: CORRELATIONS WITH MORPHOLOGY

Author

Charikleia Kelaidi, Marianna Tzanoudaki, Loizos Petrikkos, Eleni Dana, Natalia Tourkantoni, Iordanis Pelagiadis, Kondylia Antoniadi, Eleni-Dikaia Ioannidou, Maria Kourti, Helen Kosmidis, Eftychia Stiakaki, Marina Oikonomou, Evgenios Goussetis, Ioulia Peristeri, Antonis Kattamis, Aikaterini Bountali, Kalliopi Manola, Kalliopi Stefanaki, and Sophia Polychronopoulou

Subjects
Neoplasms. Tumors. Oncology. Including cancer and carcinogens, RC254-282
Published
2023
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17. Sickle cell disease landscape and challenges in the EU: the ERN-EuroBloodNet perspective

Author

Mañú Pereira, María del Mar, Colombatti, Raffaella, Alvarez, Federico, Bartolucci, Pablo, Bento, Celeste, Brunetta, Angelo Loris, Cela, Elena, Christou, Soteroula, Collado, Anna, de Montalembert, Mariane, Dedeken, Laurence, Fenaux, Pierre, Galacteros, Frédéric, Glenthøj, Andreas, Gutiérrez Valle, Victoria, Kattamis, Antonis, Kunz, Joachim, Lobitz, Stephan, McMahon, Corrina, Pellegrini, Mariangela, Reidel, Sara, Russo, Giovanna, Santos Freire, Miriam, van Beers, Eduard, Kountouris, Petros, and Gulbis, Béatrice

Published
2023
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20. Luspatercept for the treatment of anaemia in non-transfusion-dependent β-thalassaemia (BEYOND): a phase 2, randomised, double-blind, multicentre, placebo-controlled trial

Author

Buaboonnam, Jassada, Ekwattanakit, Supachai, Khunhapinant, Archrob, Loka, Efthalia, Moraki, Maria, Flevari, Pagona, Dimopoulou, Maria, Bartzi, Vasiliki, Daadaa, Hisham, El Hasbani, Georges, Koussa, Suzanne, Tartaglione, Immacolata, Ammendola, Federica, Scianguetta, Saverio, Puglia, Marta, Ferrara, Ilaria, Ferrero, Giovanni, Gaglioti, Carmen, Longo, Filomena, Turrini, Silvia, Voi, Vincenzo, Cassinerio, Elena, De, Anna, Graziadei, Giovanna, Marcon, Alessia, Migone De Amicis, Margherita, Motta, Irene, Cinque, Patrizia, Pannone, Bruno, Ricchi, Paolo, Balocco, Manuela, Carrara, Paola, Della Rovere, Francesco, Lamagna, Martina, Pinto, Valeria, Quintino, Sabrina, Eleftheriou, Perla, Garbowski, Maciej, de Kreuk, Arne, Carson, Susan, Denton, Christopher, Hofstra, Tom, Veluswamy, Sayany, Wood, John, Badawy, Sherif, Bercovitz, Rachel, Bhat, Rukhmi, Calamaras, Diane, Liem, Robert, Mack, Astrid, Taher, Ali T, Cappellini, Maria Domenica, Kattamis, Antonis, Voskaridou, Ersi, Perrotta, Silverio, Piga, Antonio G, Filosa, Aldo, Porter, John B, Coates, Thomas D, Forni, Gian Luca, Thompson, Alexis A, Musallam, Khaled M, Backstrom, Jay T, Esposito, Oriana, Giuseppi, Ana Carolina, Kuo, Wen-Ling, Miteva, Dimana, Lord-Bessen, Jennifer, Yucel, Aylin, Zinger, Tatiana, Shetty, Jeevan K, and Viprakasit, Vip

Published
2022
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21. Outcome of Children and Adolescents With Relapsed/Refractory/Progressive Malignancies Treated With Molecularly Informed Targeted Drugs in the Pediatric Precision Oncology Registry INFORM

Author

Heipertz, Anna-Elisa, Pajtler, Kristian W., Pfaff, Elke, Schramm, Kathrin, Blattner-Johnson, Mirjam, Milde, Till, Jones, Barbara C., Zuliani, Cecilia, Hutter, Caroline, Lohi, Olli, Kattamis, Antonis, Dachowska-Kalwak, Iwona, Nilsson, Anna, Gerber, Nicolas U., Langenberg, Karin P.S., Goemans, Bianca, Zwaan, C. Michel, Molenaar, Jan J., Jäger, Natalie, Dirksen, Uta, Witt, Ruth, Pfister, Stefan M., Jones, David T.W., Kopp-Schneider, Annette, Witt, Olaf, and van Tilburg, Cornelis M.

Published
2023
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22. Brachytherapy for Pediatric Patients at Gustave Roussy Cancer Campus: A Model of International Cooperation for Highly Specialized Treatments

Author

Chargari, Cyrus, Haie-Meder, Christine, Espenel, Sophie, Garcia, Max-Adrien, Ben-Arush, Myriam, Bolle, Stéphanie, Borjesson, Anna, Cesen, Maja, Lago, Rita Costa, Defachelles, Anne-Sophie, De Moerloose, Barbara, Devalck, Christine, Edslev, Pernille, Farinha, Nuno Jorge, Francotte, Nadine, Glosli, Heidi, Gouy, Sebastien, Burrieza, Gabriela Guillen, Helfre, Sylvie, Irtan, Sabine, Kattamis, Antonis, Lacerda, Ana, Levy, Antonin, Hjalgrim, Lisa Lyngsie, Mansuy, Ludovic, Mascard, Eric, Moalla, Salma, Orbach, Daniel, Owens, Cormac, Philippe-Chomette, Pascale, Pizer, Barry, Pluchart, Claire, Renard, Marleen, Rognlien, Anne Gro Wesenberg, Rome, Angélique, Sarnacki, Sabine, Safwat, Akmal, Schiavetti, Amalia, Serre, Jill, Verite, Cécile, Weid, Nicolas Von Der, Wysocki, Mariusz, Valteay-Couanet, Dominique, Deutsch, Eric, Minard-Colin, Véronique, Martelli, Hélène, and Guérin, Florent

Published
2022
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24. Drug sensitivity profiling of 3D tumor tissue cultures in the pediatric precision oncology program INFORM

Author

Peterziel, Heike, Jamaladdin, Nora, ElHarouni, Dina, Gerloff, Xenia F., Herter, Sonja, Fiesel, Petra, Berker, Yannick, Blattner-Johnson, Mirjam, Schramm, Kathrin, Jones, Barbara C., Reuss, David, Turunen, Laura, Friedenauer, Aileen, Holland-Letz, Tim, Sill, Martin, Weiser, Lena, Previti, Christopher, Balasubramanian, Gnanaprakash, Gerber, Nicolas U., Gojo, Johannes, Hutter, Caroline, Øra, Ingrid, Lohi, Olli, Kattamis, Antonis, de Wilde, Bram, Westermann, Frank, Tippelt, Stephan, Graf, Norbert, Nathrath, Michaela, Sparber-Sauer, Monika, Sehested, Astrid, Kramm, Christof M., Dirksen, Uta, Kallioniemi, Olli, Pfister, Stefan M., van Tilburg, Cornelis M., Jones, David T. W., Saarela, Jani, Pietiäinen, Vilja, Jäger, Natalie, Schlesner, Matthias, Kopp-Schneider, Annette, Oppermann, Sina, Milde, Till, Witt, Olaf, and Oehme, Ina

Published
2022
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26. Central Nervous System Fungal Diseases in Children with Malignancies: A 16-Year Study from the Infection Working Group of the Hellenic Society of Pediatric Hematology Oncology.

Author

Petrikkos, Loizos, Kourti, Maria, Antoniadi, Kondylia, Tziola, Tatiana-Sultana, Sfetsiori, Angeliki-Eleni, Antari, Vasiliki, Savoukidou, Sofia, Avgerinou, Georgia, Filippidou, Maria, Papakonstantinou, Eugenia, Polychronopoulou, Sophia, Hatzipantelis, Emmanuel, Doganis, Dimitrios, Kattamis, Antonios, Papadakis, Vassilios, Roilides, Emmanuel, and Tragiannidis, Athanasios

Subjects
CENTRAL nervous system diseases, ACUTE myeloid leukemia, JUVENILE diseases, BRAIN abscess, MYCOSES
Abstract

We analyzed data on pediatric invasive fungal diseases of the central nervous system (CNS-IFDs) reported by five of a total of eight Pediatric Hematology-Oncology Departments in Greece for 16 years (2007–2022). A total of twelve patients (11 boys, median age: 9.5 years, range: 2–16) were reported suffering from CNS-IFDs. The underlying malignancy was acute lymphoblastic leukemia in 9/12 and acute myeloid leukemia, Ewing sarcoma, and rhabdomyosarcoma in one each. Eleven patients presented with CNS-related symptoms (i.e., seizures, headache, cerebral palsy, ataxia, hallucination, seizures, blurred vision, amaurosis). All patients had pathological MRI findings. Multifocal fungal disease was observed in 6/12 patients. Nine proven and three probable CNS-IFD cases were diagnosed. Causative pathogens in proven cases were Aspergillus spp. and Candida albicans (n = 2 each), Mucor spp., Rhizopus arrhizus, Absidia spp., Fusarium oxysporum and Cryptococcus neoformans (n = 1 each). Causative pathogens in probable cases were Aspergillus spp. (n = 2) and Candida spp. (n = 1). All patients received appropriate antifungal therapy (median duration: 69.5 days, range 19–364). Two patients underwent additional surgical treatment. Six patients were admitted to the Intensive Care Unit due to complications. Three patients (25%) died, two due to IFD and one due to an underlying disease. Early recognition and prompt intervention of CNS-IFDs may rescue the patients and improve overall survival. [ABSTRACT FROM AUTHOR]

Published
2024
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27. Open versus Transcutaneous (Ultrasound-Guided and Based on Anatomic Landmarks) Tunneled Venous Access to the Right Internal Jugular Vein in Children: A Prospective Single-Center Study

Author

Niki Kouna, George Noutsos, Christina Koufopoulou, Dimitrios Panagopoulos, and Antonis Kattamis

Subjects
open surgical technique, percutaneous insertion via external landmarks, ultrasound-guided insertion, thrombosis, infection, catheter malposition, Medicine
Abstract

Background: The purpose of this study was to compare the immediate and long-term complications that are associated with the utilized techniques for the insertion of indwelling central venous catheters, that is the open surgical technique, the ultrasound-guided technique, and the transcutaneous technique based on external anatomical landmarks in the right internal jugular vein, to a pediatric population. Methods: This was a prospective randomized trial based on a pediatric patient population under 16 years of age of a tertiary pediatric-oncological hospital. The procedure was performed by a medical team with varying experience regarding the percutaneous and open insertion methods. We studied the outcome of our procedure, based on the immediate and delayed complication rate, as well as the needed time in order to complete the procedure and mean duration of line use. Results: The patients that were inserted in our protocol were divided into three subgroups based on the selected technique for the insertion of the central venous catheter. A total number of 88 insertions (25.4%) (out of 346) were based on the technique that was using external anatomical landmarks, 121 insertions were based on the ultrasound-guided transcutaneous technique (34.9%), whereas in 137 cases (39.5%) the open surgical technique was preferred. All cases that were related to catheter re-insertion were excluded from our study. We performed a statistical analysis regarding the catheter dwell time between the three subgroups of patients and no significant difference was recorded. Moreover, the development of thrombosis was investigated, and we noted that a higher percentage of this complication was related to the transcutaneous external landmark and open surgical technique. Also, the incidence of infection was taken into consideration, which manifested an increased incidence when the transcutaneous technique based on external landmarks was used. Conclusions: Ultrasound-guided percutaneous insertion was considered to be a safe and effective technique for the insertion of central venous catheters. Our study also demonstrated a decrease in operating times when performed by operators with increasing expertise, increased preservation of the diameter of the venous lumen, and no increase in complication rates when the ultrasound-guided technique was selected.

Published
2023
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28. Genotypic and Clinical Analysis of a Thalassemia Major Cohort: An Observational Study

Author

A, Tsartsalis, Lambrou, George I., Samartzi, Athanasia, Vlachou, Eugenia, Papassotiriou, Ioannis, Geronikolou, Styliani A., Kanaka-Gantenbein, Christina, Chrousos, George P., Kattamis, Antonis, Crusio, Wim E., Series Editor, Dong, Haidong, Series Editor, Radeke, Heinfried H., Series Editor, Rezaei, Nima, Series Editor, Steinlein, Ortrud, Series Editor, Xiao, Junjie, Series Editor, and Vlamos, Panayiotis, editor

Published
2021
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31. Gliomatosis cerebri in children: A poor prognostic phenotype of diffuse gliomas with a distinct molecular profile

Author

Nussbaumer, Gunther, primary, Benesch, Martin, additional, Grabovska, Yura, additional, Mackay, Alan, additional, Castel, David, additional, Grill, Jacques, additional, Alonso, Marta M, additional, Antonelli, Manila, additional, Bailey, Simon, additional, Baugh, Joshua N, additional, Biassoni, Veronica, additional, Blattner Johnson, Mirjam, additional, Broniscer, Alberto, additional, Carai, Andrea, additional, Colafati, Giovanna Stefania, additional, Colditz, Niclas, additional, Corbacioglu, Selim, additional, Crampsie, Shauna, additional, Entz-Werle, Natacha, additional, Eyrich, Matthias, additional, Friker, Lea L, additional, Frühwald, Michael C, additional, Garrè, Maria Luisa, additional, Gerber, Nicolas U, additional, Giangaspero, Felice, additional, Gil-da-Costa, Maria J, additional, Graf, Norbert, additional, Hargrave, Darren, additional, Hauser, Peter, additional, Herrlinger, Ulrich, additional, Hoffmann, Marion, additional, Hulleman, Esther, additional, Izquierdo, Elisa, additional, Jacobs, Sandra, additional, Karremann, Michael, additional, Kattamis, Antonis, additional, Kebudi, Rejin, additional, Kortmann, Rolf-Dieter, additional, Kwiecien, Robert, additional, Massimino, Maura, additional, Mastronuzzi, Angela, additional, Miele, Evelina, additional, Morana, Giovanni, additional, Noack, Claudia M, additional, Pentikainen, Virve, additional, Perwein, Thomas, additional, Pfister, Stefan M, additional, Pietsch, Torsten, additional, Roka, Kleoniki, additional, Rossi, Sabrina, additional, Rutkowski, Stefan, additional, Schiavello, Elisabetta, additional, Seidel, Clemens, additional, Štěrba, Jaroslav, additional, Sturm, Dominik, additional, Sumerauer, David, additional, Tacke, Anna, additional, Temelso, Sara, additional, Valentini, Chiara, additional, van Vuurden, Dannis, additional, Varlet, Pascale, additional, Veldhuijzen van Zanten, Sophie E M, additional, Vinci, Maria, additional, von Bueren, André O, additional, Warmuth-Metz, Monika, additional, Wesseling, Pieter, additional, Wiese, Maria, additional, Wolff, Johannes E A, additional, Zamecnik, Josef, additional, Morales La Madrid, Andrés, additional, Bison, Brigitte, additional, Gielen, Gerrit H, additional, Jones, David T W, additional, Jones, Chris, additional, and Kramm, Christof M, additional

Published
2024
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32. Expression of DNA Repair Genes in Ewing Sarcoma

Author

KYRIAZOGLOU, ANASTASIOS, primary, MOUTAFI, MYRTO, additional, ZOGRAFOS, ELENI, additional, KONTELES, VASSILIS, additional, SOFIANIDIS, GEORGIOS, additional, MAHAIRA, LOUISA, additional, PAPAKOSTA, ALEXANDRA, additional, TOURKANTONI, NATALIA, additional, PATERELI, AMALIA, additional, STEFANAKI, KALLIOPI, additional, TZOTZOLA, VASILIKI, additional, MPAKA, MARGARITA, additional, POLYCHRONOPOULOU, SOFIA, additional, DIMITRIADIS, EFTHYMIOS, additional, and KATTAMIS, ANTONIS, additional

Published
2024
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33. Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia

Author

Locatelli, Franco, primary, Lang, Peter, additional, Wall, Donna, additional, Meisel, Roland, additional, Corbacioglu, Selim, additional, Li, Amanda M., additional, de la Fuente, Josu, additional, Shah, Ami J., additional, Carpenter, Ben, additional, Kwiatkowski, Janet L., additional, Mapara, Markus, additional, Liem, Robert I., additional, Cappellini, Maria Domenica, additional, Algeri, Mattia, additional, Kattamis, Antonis, additional, Sheth, Sujit, additional, Grupp, Stephan, additional, Handgretinger, Rupert, additional, Kohli, Puja, additional, Shi, Daoyuan, additional, Ross, Leorah, additional, Bobruff, Yael, additional, Simard, Christopher, additional, Zhang, Lanju, additional, Morrow, Phuong Khanh, additional, Hobbs, William E., additional, and Frangoul, Haydar, additional

Published
2024
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35. 120P Discovery of prognostic biomarkers in Ewing sarcoma

Author

Kyriazoglou, A., primary, Moutafi, M., additional, Zografos, E., additional, Konteles, V., additional, Sofianidis, G., additional, Mahaira, L., additional, Papakosta, A., additional, Tourkantoni, N., additional, Patereli, A., additional, Stefanaki, K., additional, Tzotzola, V., additional, Baka, M., additional, Polychronopoulou, S., additional, Dimitriadis, E., additional, and Kattamis, A., additional

Published
2024
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36. Global characteristics and outcomes of SARS-CoV-2 infection in children and adolescents with cancer (GRCCC): a cohort study

Author

Ribelles, A Juan, Balduzzi, Adriana, Elhaddad, Alaa, Casanovas, Alejandra, Garcia Velazquez, Alejandra, Laptsevich, Aliaksandra, Chang, Alicia, F. Sampaio, Alessandra Lamenha, González Prieto, Almudena, Lassaletta, Alvaro, Suarez M, Amaranto, Alcasabas, Ana Patricia, Colita, Anca, Morales La Madrid, Andres, Samudio, Angélica, Tondo, Annalisa, Colombini, Antonella, Kattamis, Antonis, Lopez Facundo, N Araceli, Bhattacharyya, Arpita, Alimi, Aurélia, Phulpin, Aurélie, Vakrmanova, Barbora, Aksoy, Basak A, Brethon, Benoit, Kobuin, Jator Brian, Nolasco Monteiro, Carla, Paillard, Catherine, Vezina, Catherine, Ceyhun, Bozkurt, Hentea, Cristiana, Meazza, Cristina, Ortiz-Morales, Daniel, Solorzano, Roque Daniel, Arce Cabrera, Daniela, Zama, Daniele, Ghosh, Debjani, Ramírez-Rivera, Diana, Calle Jara, Doris A, Janic, Dragana, Rey Helo, Elianneth, Gouache, Elodie, Guerrero Quiroz, Enmanuel, Lopez, Enrique, Thebault, Eric, Maradiegue, Essy, de Berranger, Eva, Ebeid, Fatma S E, Galaverna, Federica, Antillon-Klussmann, Federico, Espinoza Chacur, Felipe, Negro, Fernando Daniel, Carraro, Francesca, Compagno, Francesca, Barriga, Francisco, Tamayo Pedraza, Gabriela, Sanchez Fernandez, Gissela, Naidu, Gita, Tokuc, Gülnur, Alias, Hamidah, B Segocio, Hannah Grace, Boudiaf, Houda, Asetre Luna, Imelda, Maia, Iris, Astigarraga, Itziar, Maza, Ivan, Montoya Vásquez, Jacqueline E, Jazbec, Janez, Lazic, Jelena, Beck Dean, Jeniffer, Rouger-Gaudichon, Jeremie, Contreras González, Johanny Carolina, Huerta Aragonés, Jorge, Fuster, José L, Quintana, Juan, Palma, Julia, Svojgr, Karel, Quintero, Karina, Malic Tudor, Karolina, Georgantzi, Kleopatra, P Schultz, Kris Ann, Ureña Horno, Laura, Fraquelli, Lidia, Meneghello, Linda, Shalaby, Lobna, Macias Mora, Lola L, A Renner, Lorna, Nunes Silva, Luciana, Sisinni, Luisa, Hammad, Mahmoud, Fernández Sanmartín, M, Zubieta A, C Marcela, Drozdowski, María Constanza, Kourti, Maria, Palladino, Marcela María, Miranda Madrazo, Maria R, Poiree, Marilyne, Popova, Marina, Melgar, Mario, Baragaño, Marta, Avilés-Robles, Martha J, Provenzi, Massimo, Mendes Lins, Mecneide, Fatih Orhan, Mehmet, Villarroel, Milena, Jerónimo, Mónica, Varas Palma, Mónica, Rafie Raza, Muhammad, M Justin, Mulindwa, Shaheen, Najma, Domínguez-Pinilla, Nerea, Whipple, Nicholas S, André, Nicolas, Hrusak, Ondrej, Velasco Puyó, Pablo, Zacasa Vargas, Pamela, Olate Mellado, Paola, Yola Gassant, Pascale, Diaz Romero, Paulina, De Santis, Raffaella, Kebudi, Rejin, Boranbayeva, Riza, Vasquez, Roberto, Segura, Romel A., Rosado, Roy Enrique, Gómez, Sandra, Raimbault, Sandra, Gunasekera, Sanjeeva, Makkeyah, Sara M, Buyukkapu Bay, Sema, M Gómez, Sergio, Bouttefroy, Séverine, Islam, Shahnoor, Abouelnaga, Sherif, Torres, Silvio Fabio, Cesaro, Simone, Nunes, Sofia, Rouxinol, Soraia, Bhaumik, Sucharita, Saliyeva, Symbat, Inostroza, Tamara, Velasquez, Thelma, Hnin, Tint Myo, Norén-Nyström, Ulrika, Baretta, Valentina, Jimenez-Antolinez, Yajaira Valentine, Pérez Alonso, Vanesa, Ayer Miller, Vanessa, Gandemer, Virginie, Lotero, Viviana, Mishkova, Volha, Gómez-García, Wendy, Margaryan, Yeva, Syed, Yumna, Mukkada, Sheena, Bhakta, Nickhill, Chantada, Guillermo L, Chen, Yichen, Vedaraju, Yuvanesh, Faughnan, Lane, Homsi, Maysam R, Muniz-Talavera, Hilmarie, Ranadive, Radhikesh, Metzger, Monika, Friedrich, Paola, Agulnik, Asya, Jeha, Sima, Lam, Catherine, Dalvi, Rashmi, Hessissen, Laila, Moreira, Daniel C, Santana, Victor M, Sullivan, Michael, Bouffet, Eric, Caniza, Miguela A, Devidas, Meenakshi, Pritchard-Jones, Kathy, and Rodriguez-Galindo, Carlos

Published
2021
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37. PATZ1 fusions define a novel molecularly distinct neuroepithelial tumor entity with a broad histological spectrum

Author

Alhalabi, Karam T., Stichel, Damian, Sievers, Philipp, Peterziel, Heike, Sommerkamp, Alexander C., Sturm, Dominik, Wittmann, Andrea, Sill, Martin, Jäger, Natalie, Beck, Pengbo, Pajtler, Kristian W., Snuderl, Matija, Jour, George, Delorenzo, Michael, Martin, Allison M., Levy, Adam, Dalvi, Nagma, Hansford, Jordan R., Gottardo, Nicholas G., Uro-Coste, Emmanuelle, Maurage, Claude-Alain, Godfraind, Catherine, Vandenbos, Fanny, Pietsch, Torsten, Kramm, Christof, Filippidou, Maria, Kattamis, Antonis, Jones, Chris, Øra, Ingrid, Mikkelsen, Torben Stamm, Zapotocky, Michal, Sumerauer, David, Scheie, David, McCabe, Martin, Wesseling, Pieter, Tops, Bastiaan B. J., Kranendonk, Mariëtte E. G., Karajannis, Matthias A., Bouvier, Nancy, Papaemmanuil, Elli, Dohmen, Hildegard, Acker, Till, von Hoff, Katja, Schmid, Simone, Miele, Evelina, Filipski, Katharina, Kitanovski, Lidija, Krskova, Lenka, Gojo, Johannes, Haberler, Christine, Alvaro, Frank, Ecker, Jonas, Selt, Florian, Milde, Till, Witt, Olaf, Oehme, Ina, Kool, Marcel, von Deimling, Andreas, Korshunov, Andrey, Pfister, Stefan M., Sahm, Felix, and Jones, David T. W.

Published
2021
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38. Evaluation of endocrine complications in beta-thalassemia intermedia (β-TI): a cross-sectional multicenter study

Author

Karimi, Mehran, Zarei, Tahereh, Haghpanah, Sezaneh, Azarkeivan, Azita, Kattamis, Christos, Ladis, Vassilis, Kattamis, Antonios, Kilinc, Yurdanur, Daar, Shahina, Alyaarubi, Saif, Khater, Doaa, Wali, Yasser, Elshinawy, Mohamed, Almadhani, Ali, Yassin, Mohamed, Soliman, Ashraf T., Canatan, Duran, Obiedat, Maha, Al-Rimawi, Hala, Mariannis, Demetris, Christodoulides, Constantinos, Christou, Soteroula, Tzoulis, Ploutarchos, Campisi, Saveria, Di Maio, Salvatore, and De Sanctis, Vincenzo

Published
2020
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39. Dabigatran etexilate for the treatment of acute venous thromboembolism in children (DIVERSITY): a randomised, controlled, open-label, phase 2b/3, non-inferiority trial

Author

Nurmeev, Ildar, Safina, Asiya, Zapletal, Ondrej, Brandão, Leonardo R, Kuhn, Tomas, Votava, Tomas, Felgenhauer, Judy, Sharathkumar, Anjali, Svirin, Pavel, Amid, Ali, Halton, Jacqueline, Gorbatikov, Kirill, Saracco, Paola, Kiss, Csongor, Halimeh, Susan, Reschke, Madlen, Wulff, Beate, David, Michele, Novak, Zbynek, Trunina, Inna, Albisetti, Manuela, Frisk, Tony, Glosli, Heidi, Groll, Andreas, Lvova, Olga, Sasmaz, Ilgen, Sosothikul, Darintr, Zilinskaite, Virginija, Cockrell, Erin, Digtyar, Valeriy, Hadacova, Ivana, Palmu, Sauli, Pawar, Anjali, Annichino Bizzacchi, Joyce Maria, Caliskan, Umran, Celkan, Tiraje, Dmytriiev, Dmytro, Druzgal, Colleen Harkins, Onelda Elena, Graciela, Kattamis, Antonis, Kavakli, Ramazan Kaan, Male, Christoph, Ozdemir, Nihal, Van Damme, An, Zvereva, Tatiana, Aarli, Aanen, Paredes Aguilera, Rogelio Alejandro, Aytac, Selin, Carneiro, Jorge, Chistolini, Antonio, Mazzucconi, Maria Gabriela, Corrales-Medina, Fernando, Couturaud, Francis, Croteau, Stacey E, Trenor III, Cameron, Damgaard, Michael, Dixon, Natalia, Galustyan, Anna, Hak, Jiri, Hoffmann, Marianne, Kupesiz, Alphan, Labarque, Veerle, van Geet, Christel, Lin, Ming-Chih, Fu, Yun-Ching, Loggetto, Sandra, Mondelaers, Veerle, Odri-Komazec, Irena, Revel-Vilk, Shoshana, Sevilla, Julian, Fuzzato Silva, Luciano, Kerr Saraiva, José, Montes Tapia, Fernando Felix, Woods-Swafford, Wendy, Luciani, Matteo, Bomgaars, Lisa, Chalmers, Elizabeth, Mitchell, Lesley G, Tartakovsky, Igor, Simetzberger, Monika, Huang, Fenglei, Sun, Zhichao, Kreuzer, Jörg, Gropper, Savion, Reilly, Paul, and Brueckmann, Martina

Published
2021
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40. Gliomatosis cerebri in children:A poor prognostic phenotype of diffuse gliomas with a distinct molecul profile

Author

Nussbaumer, Gunther, Benesch, Martin, Grabovska, Yura, Mackay, Alan, Castel, David, Grill, Jacques, Alonso, Marta M., Antonelli, Manila, Bailey, Simon, Baugh, Joshua N., Biassoni, Veronica, Blattner-Johnson, Mirjam, Broniscer, Alberto, Carai, Andrea, Colafati, Giovanna Stefania, Colditz, Niclas, Corbacioglu, Selim, Crampsie, Shauna, Entz-Werle, Natacha, Eyrich, Matthias, Friker, Lea L., Frühwald, Michael C., Garrè, Maria Luisa, Gerber, Nicolas U., Giangaspero, Felice, Gil-Da-Costa, Maria J., Graf, Norbert, Hargrave, Darren, Hauser, Peter, Herrlinger, Ulrich, Hoffmann, Marion, Hulleman, Esther, Izquierdo, Elisa, Jacobs, Sandra, Karremann, Michael, Kattamis, Antonis, Kebudi, Rejin, Kortmann, Rolf Dieter, Kwiecien, Robert, Massimino, Maura, Mastronuzzi, Angela, Miele, Evelina, Morana, Giovanni, Noack, Claudia M., Pentikainen, Virve, Perwein, Thomas, Pfister, Stefan M., Pietsch, Torsten, Roka, Kleoniki, Rossi, Sabrina, Rutkowski, Stefan, Schiavello, Elisabetta, Seidel, Clemens, Štěrba, Jaroslav, Sturm, Dominik, Sumerauer, David, Tacke, Anna, Temelso, Sara, Valentini, Chiara, van Vuurden, Dannis, Varlet, Pascale, Veldhuijzen van Zanten, Sophie E.M., Vinci, Maria, von Bueren, André O., Warmuth-Metz, Monika, Wesseling, Pieter, Wiese, Maria, Wolff, Johannes E.A., Zamecnik, Josef, La Madrid, Andrés Morales, Bison, Brigitte, Gielen, Gerrit H., Jones, David T.W., Jones, Chris, Kramm, Christof M., Nussbaumer, Gunther, Benesch, Martin, Grabovska, Yura, Mackay, Alan, Castel, David, Grill, Jacques, Alonso, Marta M., Antonelli, Manila, Bailey, Simon, Baugh, Joshua N., Biassoni, Veronica, Blattner-Johnson, Mirjam, Broniscer, Alberto, Carai, Andrea, Colafati, Giovanna Stefania, Colditz, Niclas, Corbacioglu, Selim, Crampsie, Shauna, Entz-Werle, Natacha, Eyrich, Matthias, Friker, Lea L., Frühwald, Michael C., Garrè, Maria Luisa, Gerber, Nicolas U., Giangaspero, Felice, Gil-Da-Costa, Maria J., Graf, Norbert, Hargrave, Darren, Hauser, Peter, Herrlinger, Ulrich, Hoffmann, Marion, Hulleman, Esther, Izquierdo, Elisa, Jacobs, Sandra, Karremann, Michael, Kattamis, Antonis, Kebudi, Rejin, Kortmann, Rolf Dieter, Kwiecien, Robert, Massimino, Maura, Mastronuzzi, Angela, Miele, Evelina, Morana, Giovanni, Noack, Claudia M., Pentikainen, Virve, Perwein, Thomas, Pfister, Stefan M., Pietsch, Torsten, Roka, Kleoniki, Rossi, Sabrina, Rutkowski, Stefan, Schiavello, Elisabetta, Seidel, Clemens, Štěrba, Jaroslav, Sturm, Dominik, Sumerauer, David, Tacke, Anna, Temelso, Sara, Valentini, Chiara, van Vuurden, Dannis, Varlet, Pascale, Veldhuijzen van Zanten, Sophie E.M., Vinci, Maria, von Bueren, André O., Warmuth-Metz, Monika, Wesseling, Pieter, Wiese, Maria, Wolff, Johannes E.A., Zamecnik, Josef, La Madrid, Andrés Morales, Bison, Brigitte, Gielen, Gerrit H., Jones, David T.W., Jones, Chris, and Kramm, Christof M.

Abstract

Background: The term gliomatosis cerebri (GC), a radiology-defined highly infiltrating diffuse glioma, has been abandoned since molecular GC-associated features could not be established. Methods: We conducted a multinational retrospective study of 104 children and adolescents with GC providing comprehensive clinical and (epi-)genetic characterization. Results: Median overall survival (OS) was 15.5 months (interquartile range, 10.9–27.7) with a 2-year survival rate of 28%. Histopathological grading correlated significantly with median OS: CNS WHO grade II: 47.8 months (25.2–55.7); grade III: 15.9 months (11.4–26.3); grade IV: 10.4 months (8.8–14.4). By DNA methylation profiling (n = 49), most tumors were classified as pediatric-type diffuse high-grade glioma (pedHGG), H3-/IDH-wild-type (n = 31/49, 63.3%) with enriched subclasses pedHGG_RTK2 (n = 19), pedHGG_A/B (n = 6), and pedHGG_MYCN (n = 5), but only one pedHGG_RTK1 case. Within the pedHGG, H3-/IDH-wild-type subgroup, recurrent alterations in EGFR (n = 10) and BCOR (n = 9) were identified. Additionally, we observed structural aberrations in chromosome 6 in 16/49 tumors (32.7%) across tumor types. In the pedHGG, H3-/IDH-wild-type subgroup TP53 alterations had a significant negative effect on OS. Conclusions: Contrary to previous studies, our representative pediatric GC study provides evidence that GC has a strong predilection to arise on the background of specific molecular features (especially pedHGG_ RTK2, pedHGG_A/B, EGFR and BCOR mutations, chromosome 6 rearrangements).

Published
2024

43. Evaluation of the efficacy and safety of deferiprone compared with deferasirox in paediatric patients with transfusion-dependent haemoglobinopathies (DEEP-2): a multicentre, randomised, open-label, non-inferiority, phase 3 trial

Author

Maggio, Aurelio, Kattamis, Antonis, Felisi, Mariagrazia, Reggiardo, Giorgio, El-Beshlawy, Amal, Bejaoui, Mohamed, Sherief, Laila, Christou, Soteroula, Cosmi, Carlo, Della Pasqua, Oscar, Del Vecchio, Giovanni Carlo, Filosa, Aldo, Cuccia, Liana, Hassab, Hoda, Kreka, Manika, Origa, Raffaella, Putti, Maria Caterina, Spino, Michael, Telfer, Paul, Tempesta, Bianca, Vitrano, Angela, Tsang, Yu Chung, Zaka, Ariana, Tricta, Fernando, Bonifazi, Donato, and Ceci, Adriana

Published
2020
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44. Transfusion Independence after Exagamglogene Autotemcel in Patients with Transfusion-Dependent β-Thalassemia

Author

Mapara, Markus Y., primary, Locatelli, Franco, additional, Lang, Peter, additional, Corbacioglu, Selim, additional, Li, Amanda, additional, Fuente, Josu de la, additional, Wall, Donna A., additional, Meisel, Roland, additional, Shah, Ami J., additional, Liem, Robert, additional, Carpenter, Ben, additional, Kwiatkowski, Janet L., additional, Cappellini, Maria Domenica, additional, Kattamis, Antonis, additional, Sheth, Sujit, additional, Grupp, Stephan A., additional, Kohli, Puja, additional, Shi, Daoyuan, additional, Bobruff, Yael, additional, Ross, Leorah, additional, Simard, Christopher, additional, Zhang, Lanju, additional, Morrow, Phuong Khanh, additional, Hobbs, William, additional, and Frangoul, Haydar, additional

Published
2024
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45. Antibiotic use in Greek Pediatric Hematology-Oncology and Bone Marrow Transplantation units

Author

Mantadakis, Elpis, primary, Kopsidas, Ioannis, additional, Coffin, Susan, additional, Dimitriou, Gabriel, additional, Gkentzi, Despoina, additional, Hatzipantelis, Emmanuel, additional, Kaisari, Aikaterini, additional, Kattamis, Antonis, additional, Kourkouni, Eleni, additional, Papachristidou, Smaragda, additional, Papakonstantinou, Eugenia, additional, Polychronopoulou, Sophia, additional, Roilides, Emmanuel, additional, Spyridis, Nikos, additional, Tsiodras, Sotirios, additional, Tsolia, Maria, additional, Tsopela, Grammatiki-Christina, additional, Zaoutis, Theoklis, additional, and Tragiannidis, Athanasios, additional

Published
2024
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46. Increased age-associated B cells in patients with acquired aplastic anemia correlate with IFN-γ

Author

Solomou, Elena E., primary, Kattamis, Antonis, additional, Symeonidis, Argyris, additional, Sirinian, Chaido, additional, Salamaliki, Christina, additional, Tzanoudaki, Marianna, additional, Diamantopoulos, Panagiotis, additional, Plakoula, Eva, additional, Palasopoulou, Maria, additional, Giannakoulas, Nikolaos, additional, Kontandreopoulou, Christina-Nefeli, additional, Kollia, Panagoula, additional, Viniou, Nora-Athina, additional, Galanopoulos, Athanasios, additional, Liossis, Stamatis-Nick, additional, and Vassilopoulos, George, additional

Published
2024
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49. Development of a centralised triage centre for children with cancer and blood disorders in response to the humanitarian crisis in Ukraine

Author

Salek, Marta, primary, Mueller, Alexandra, additional, Alanbousi, Inna, additional, Cepowska, Zuzanna, additional, Dutkiewicz, Malgorzata, additional, Earl, Julian, additional, Evseev, Dmitry, additional, Kizyma, Roman, additional, Kliuchkivska, Khrystyna, additional, Kolodrubiec, Julia, additional, Matczak, Katarzyna, additional, Nogovitsyna, Yuliya, additional, Oszer, Aleksandra, additional, Pogorelyy, Mikhail, additional, Raciborska, Anna, additional, Rasul, Suheir, additional, Sokolowski, Igor, additional, Sopilnyak, Andriy, additional, Vinitsky, Anna, additional, Wlodarski, Marcin W, additional, Wobst, Natalia, additional, Yakimkova, Taisiya, additional, Rodriguez-Galindo, Carlos, additional, Wise, Paul H, additional, Mlynarski, Wojciech, additional, Agulnik, Asya, additional, Alexander, Sarah Weeks, additional, Apel, Anna, additional, Adyrov, Mykhaylo V, additional, Avula, Meghana, additional, Bal, Wioletta Anna, additional, Balwierz, Walentyna Aniela, additional, Basset-Salom, Luisa, additional, Bastardo Blanco, Daniel, additional, Bauer, Karolina Jadwiga, additional, Bayazitov, Ildar T, additional, Berlanga, Pablo, additional, Bhakta, Nickhill Hitesh, additional, Bieniek, Katarzyna Anna, additional, Bien, Ewa Iwona, additional, Blackwood, Christopher Andrew, additional, Blair, Sally Jane, additional, Bodak, Khrystyna Ihorivna, additional, Bordeianu, Irina, additional, Bouffet, Eric, additional, Braganca, Joao Maria, additional, Bucurenci, Mihaela Silvia, additional, Budny, Elżbieta Beata, additional, Budzyn, Andrii, additional, Bumgardner, Christopher Carl, additional, Burditt, Raina Nichole, additional, Burnside Clapp, Victoria, additional, Bykov, Viacheslav Valeriyovych, additional, Cañete, Adela, additional, Carnelli, Monica, additional, Cela, Elena, additional, Chaber, Radoslaw, additional, Cherner-Drieux, Anna, additional, Chubata, Mariya, additional, Clough, Heidi M, additional, Czauderna, Piotr Stefan, additional, Czernicka - Siwecka, Jolanta, additional, Czyzewski, Krzysztof, additional, Dalle, Jean-Hugues, additional, Dashchakovska, Olha, additional, de Koning, Linda A, additional, Dembowska-Baginska, Bozenna Malgorzata, additional, Derwich, Katarzyna, additional, Dirksen, Uta, additional, Dommett, Rachel, additional, Dorosh, Olha Ihorivna, additional, dos Reis Farinha, Nuno Jorge, additional, Drabko, Katarzyna Anna, additional, Dragomir, Monica Desiree, additional, Dworzak, Michael, additional, Dyma, Sergii Vitaliiovych, additional, Eggert, Angelika, additional, English, Martin William, additional, Farren, Becky S, additional, Fedyk, Nataliia Yuriina, additional, Fernández-Teijeiro, Ana, additional, Ferneza, Severyn Romanovych, additional, Foster, Whitney Baer, additional, Fox Irwin, Leeanna Elizabeth, additional, Gałązkowski, Robert Maciej, additional, Ganieva, Galyna, additional, Garanzha, Vasylyna Andriivna, additional, Gelman, Marina S, additional, Godzinski, Jan Krzysztof, additional, Goeres, Anne Françoise, additional, Golban, Rodica, additional, Graetz, Dylan Elizabeth, additional, Greiner, Jeanette, additional, Griksaitis, Michael J, additional, Gupta, Sumit, additional, Hampel, Michal Andrzej, additional, Hastings, Sara Grace, additional, Heenen, Delphine Liliane, additional, Hill, Marcela C, additional, Holiuk, Ihor, additional, Holter, Wolfgang, additional, Hough, Rachael Emma, additional, Hutnik, Lukasz Marek, additional, Irga-Jaworska, Ninela, additional, Istomin, Oleksandr Andriyovych, additional, Ignatova, Anna, additional, Janczar, Szymon Lech, additional, Kacharian, Arman, additional, Kalwak, Krzysztof, additional, Karolczyk, Grażyna Małgorzata, additional, Karpenko, Nataliia Mikolaivna, additional, Katsubo, Halyna Oleksandrivna, additional, Kattamis, Antonis, additional, Kazanowska, Bernarda Jadwiga, additional, Kentsis, Alex, additional, Ketteler, Petra, additional, Kienesberger, Anita, additional, Kiselev, Roman, additional, Kizyma, Zoryana Petrivna, additional, Klymniuk, Hryhorii Ivanovych, additional, Kolenova, Alexandra, additional, Kostiuk, Yuliia, additional, Kowalik, Tomasz, additional, Kozlova, Olena Igorivna, additional, Kozubenko, Vladyslav, additional, Kraal, Kathelijne, additional, Kramar, Tetyana Oleksandrivna, additional, Krawczuk-Rybak, Maryna Maryna, additional, Kulemzina, Irina, additional, Kurkowska, Paulina, additional, Kuzyk, Andriy S., additional, Ladenstein, Ruth Lydia, additional, Laguna, Pawel Jozef, additional, Lassaletta, Alvaro, additional, Lehmberg, Kai, additional, Leontieva, Oksana, additional, Liashenko, Serhii, additional, Loizou, Loizos G., additional, Lucchetta, Sonia Anna, additional, Lupo, Matthew William, additional, Lysytsia, Lesya, additional, Lysytsia, Oleksandr, additional, Machnik, Katarzyna Anna, additional, Massimino, Maura, additional, Mainland, Jeff A, additional, Matysiak, Michal Jacek, additional, Mayeur, Pierre, additional, Miller, Beth Anne, additional, Minervina, Anastasia A, additional, Mishkova, Volha, additional, Mizia-Malarz, Agnieszka Joanna, additional, Morales La Madrid, Andres, additional, Moreira, Daniel C, additional, Moreno, Lucas, additional, Moskvin, Vadim P, additional, Mukkada, Sheena Teresa, additional, Muszyńska-Rosłan, Katarzyna Maria, additional, Mykychak, Iryna Volodymyrivna, additional, Niemeyer, Charlotte, additional, Nelson, Akoya Janae', additional, Ociepa, Tomasz, additional, Oltolini, Stefano, additional, Onipko, Nataliia, additional, Pappas, Andrew, additional, Patel, Amit B, additional, Patrahau, Alina Alina, additional, Pauley, Jennifer L., additional, Pavlenko, Yehor Mikhailovich, additional, Pavlovych, Andrij Oleksandrovych, additional, Peregud-Pogorzelski, Jarosław Władysław, additional, Perek-Polnik, Marta, additional, Pérez, Vanesa, additional, Pérez-Martínez, Antonio, additional, Pikman, Yana, additional, Pitozzi, Graziano Pitozzi, additional, Portugal, Rui Gentil, additional, Posternak, Victoria Vita, additional, Pleshkan, Viktoriya, additional, Prete, Arcangelo, additional, Pritchard-Jones, Kathy, additional, Radaelli, Alessandra, additional, Reeves, Tegan Jemma, additional, Reinhardt, Dirk, additional, Reshetnyak, Andrey V, additional, Rider, Andrew Jacob, additional, Rizzari, Carmelo, additional, Rizzi, Damiano Damiano, additional, Rodriguez Hermosillo, Karen Gabriela, additional, Ronenko, Olena Volodymyrivna, additional, Rostkowska, Aneta Olga, additional, Rudko, Liudmyla Yaroslavivna, additional, Sakaan, Firas Mohamed, additional, Sakhar, Nadezhda Aleksandrovna, additional, Salman, Zeena S, additional, Savva, Natallia N, additional, Scaccaglia, Davide, additional, Schaeffer, Elizabeth Hawthorne, additional, Schneider, Carina Ursula, additional, Scobie, Nicole, additional, Semeniuk, Olena Volodymyrivna, additional, Shevchyk, Roksoliana, additional, Shuler, Ana, additional, Shvets, Stanislav, additional, Sniderman, Liz, additional, Skoczen, Szymon Pawel, additional, Smeal, William John, additional, Sonkin, Anna Alexandra, additional, Spota, Andrea, additional, Sramkova, Lucie, additional, Stepanjuk, Alla Ivanivna, additional, Sterba, Jaroslav, additional, Strahm, Brigitte, additional, Styczynski, Jan, additional, Svintsova, Olha, additional, Synyuta, Andriy V, additional, Szczepanski, Tomasz, additional, Szczucinski, Paweł Kukiz, additional, Szmyd, Bartosz Miroslaw, additional, Tasso Cereceda, Maria, additional, Teliuk, Alina, additional, Tomanek, Iwona, additional, Topping, Phoebe, additional, Torrent, Montserrat, additional, Trelińska, Joanna, additional, Troyanovska, Olha Orestivna, additional, Tsurkan, Lyudmila G., additional, Tsymbalyuk-Voloshyn, Iryna, additional, Tyupa, Sergiy Ihorovych, additional, Urasinski, Tomasz Franciszek, additional, Urbanek Dądela, Agnieszka, additional, Vasilieva, Nataliia Jroslavivna, additional, Vasilyeva, Aksana, additional, Verdú-Amorós, Jaime, additional, Vilcu-Bajurean, Natalia, additional, Vinitsky, Leo, additional, Vivtcharenko, Victoria, additional, Vovk, Nelia, additional, Volpe, Giovanni, additional, Vorobel, Oksana Ivanivna, additional, Wachowiak, Jacek Tadeusz, additional, Wasiak, Marcin Sławomir, additional, Wiedower, Lance Allan, additional, Wuenschel, Lena Isolde, additional, Wysocki, Mariusz Stanislaw, additional, Yurieva, Marina, additional, Zagurska, Anastasiia, additional, Zakharenko, Stanislav S, additional, Zakharenko, Aelita V, additional, Zapotochna, Khrystyna, additional, Zawitkowska, Joanna Emilia, additional, and Zecca, Marco, additional

Published
2023
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50. IMMUNOPHENOTYPE IN SAMD9/9L SYNDROME: CORRELATIONS WITH MORPHOLOGY

Author

Kelaidi, Charikleia, primary, Tzanoudaki, Marianna, additional, Petrikkos, Loizos, additional, Dana, Eleni, additional, Tourkantoni, Natalia, additional, Pelagiadis, Iordanis, additional, Antoniadi, Kondylia, additional, Ioannidou, Eleni-Dikaia, additional, Kourti, Maria, additional, Kosmidis, Helen, additional, Stiakaki, Eftychia, additional, Oikonomou, Marina, additional, Goussetis, Evgenios, additional, Peristeri, Ioulia, additional, Kattamis, Antonis, additional, Bountali, Aikaterini, additional, Manola, Kalliopi, additional, Stefanaki, Kalliopi, additional, and Polychronopoulou, Sophia, additional

Published
2023
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