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51. Hematopoietic cell transplantation in severe combined immunodeficiency: The SCETIDE 2006-2014 European cohort

Author

Lankester, Arjan C., primary, Neven, Benedicte, additional, Mahlaoui, Nizar, additional, von Asmuth, Erik G.J., additional, Courteille, Virginie, additional, Alligon, Mikael, additional, Albert, Michael H., additional, Serra, Isabelle Badell, additional, Bader, Peter, additional, Balashov, Dmitry, additional, Beier, Rita, additional, Bertrand, Yves, additional, Blanche, Stephane, additional, Bordon, Victoria, additional, Bredius, Robbert G., additional, Cant, Andrew, additional, Cavazzana, Marina, additional, Diaz-de-Heredia, Cristina, additional, Dogu, Figen, additional, Ehlert, Karoline, additional, Entz-Werle, Natacha, additional, Fasth, Anders, additional, Ferrua, Francesca, additional, Ferster, Alina, additional, Formankova, Renata, additional, Friedrich, Wilhelm, additional, Gonzalez-Vicent, Marta, additional, Gozdzik, Jolanta, additional, Güngör, Tayfun, additional, Hoenig, Manfred, additional, Ikinciogullari, Aydan, additional, Kalwak, Krzysztof, additional, Kansoy, Savas, additional, Kupesiz, Alphan, additional, Lanfranchi, Arnalda, additional, Lindemans, Caroline A., additional, Meisel, Roland, additional, Michel, Gerard, additional, Miranda, Nuno A.A., additional, Moraleda, Jose, additional, Moshous, Despina, additional, Pichler, Herbert, additional, Rao, Kanchan, additional, Sedlacek, Petr, additional, Slatter, Mary, additional, Soncini, Elena, additional, Speckmann, Carsten, additional, Sundin, Mikael, additional, Toren, Amos, additional, Vettenranta, Kim, additional, Worth, Austen, additional, Yeşilipek, Mehmet A., additional, Zecca, Marco, additional, Porta, Fulvio, additional, Schulz, Ansgar, additional, Veys, Paul, additional, Fischer, Alain, additional, and Gennery, Andrew R., additional

Published
2022
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53. Incidence of Secondary Malignancies after Total Body Irradiation-Based Allogeneic HSCT in Children with ALL – Long-Term Follow-up from the Prospective ALL-SCT 2003 Trial

Author

Eichinger, Anna, primary, Glogova, Evgenia, additional, Poetschger, Ulrike, additional, Bader, Peter, additional, Basu, Oliver, additional, Beier, Rita, additional, Burkhardt, Birgit, additional, Classen, Carl-Friedrich, additional, Claviez, Alexander, additional, Corbacioglu, Selim, additional, Deubzer, Hedwig, additional, Greil, Johann, additional, Gruhn, Bernd, additional, Gungor, Tayfun, additional, Kafa, Kinan, additional, Lang, Peter, additional, Lange, Bjoern Soenke, additional, Mueller, Ingo, additional, Sauer, Martin G., additional, Schlegel, Paul-Gerhard, additional, Schulz, Ansgar, additional, Stachel, Klaus Daniel, additional, Strahm, Brigitte R., additional, Wawer, Angela, additional, Kühl, Jörn-Sven, additional, Meisel, Roland, additional, Peters, Christina, additional, and Albert, Michael H., additional

Published
2022
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54. CTLA-4 Insufficiency due to a Novel CTLA-4 Deletion, Identified through Copy Number Variation Analysis.

Author

Olfe, Lisa, von Hardenberg, Sandra, Hofmann, Winfried, Auber, Bernd, Baumann, Ulrich, Beier, Rita, Adriawan, Ignatius Ryan, Atschekzei, Faranaz, Witte, Torsten, and Sogkas, Georgios

Subjects
CYTOTOXIC T lymphocyte-associated molecule-4, SINGLE nucleotide polymorphisms, JUVENILE idiopathic arthritis, DNA copy number variations, NUCLEOTIDE sequencing
Abstract

Background: The diagnostic yield of next-generation sequencing (NGS) technologies in the diagnosis of monogenic inborn errors of immunity (IEI) remains limited, rarely exceeding 30%. Monoallelic pathogenic germline variants in cytotoxic T lymphocyte-associated antigen-4 (CTLA-4) result in variable immunodeficiency and immune dysregulation. The genetic diagnosis of CTLA-4 insufficiency can affect follow-up procedures and may lead to consideration of treatment with CTLA-4-Ig. Objectives: The aim of the study was to identify the genetic cause of familial immunodeficiency and immune dysregulation in cases where single nucleotide variant analysis of short-read NGS data yielded no diagnostic result. Methods: Analysis of copy number variants (CNVs) was applied on short-read NGS data. Results: We identified a novel monoallelic deletion-insertion variant in CTLA-4 (c.445_568-544delinsTTTGCGATTG) resulting in familial autoimmunity. This is the second larger scale variant in CTLA-4, which despite consistently reduced expression of CTLA-4 displayed variable expressivity, ranging from typical juvenile idiopathic arthritis to common variable immunodeficiency-like immunodeficiency. Conclusions: Our report suggests the significance of integration of CNV analysis in routine evaluation of NGS, which may increase its diagnostic yield in IEI. [ABSTRACT FROM AUTHOR]

Published
2023
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55. Molecular response to treatment redefines all prognostic factors in children and adolescents with B-cell precursor acute lymphoblastic leukemia: results in 3184 patients of the AIEOP-BFM ALL 2000 study

Author

Conter, Valentino, Bartram, Claus R., Valsecchi, Maria Grazia, Schrauder, André, Panzer-Grümayer, Renate, Möricke, Anja, Aricò, Maurizio, Zimmermann, Martin, Mann, Georg, De Rossi, Giulio, Stanulla, Martin, Locatelli, Franco, Basso, Giuseppe, Niggli, Felix, Barisone, Elena, Henze, Günter, Ludwig, Wolf-Dieter, Haas, Oskar A., Cazzaniga, Giovanni, Koehler, Rolf, Silvestri, Daniela, Bradtke, Jutta, Parasole, Rosanna, Beier, Rita, van Dongen, Jacques J.M., Biondi, Andrea, and Schrappe, Martin

Published
2010
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58. Publisher Correction : Clinical evolution, genetic landscape and trajectories of clonal hematopoiesis in SAMD9/SAMD9L syndromes (Nature Medicine, (2021), 27, 10, (1806-1817), )

Author

Sahoo, Sushree S., Pastor, Victor B., Goodings, Charnise, Voss, Rebecca K., Kozyra, Emilia J., Szvetnik, Amina, Noellke, Peter, Dworzak, Michael, Starý, Jan, Locatelli, Franco, Masetti, Riccardo, Schmugge, Markus, De Moerloose, Barbara, Catala, Albert, Kállay, Krisztián, Turkiewicz, Dominik, Hasle, Henrik, Buechner, Jochen, Jahnukainen, Kirsi, Ussowicz, Marek, Polychronopoulou, Sophia, Smith, Owen P., Fabri, Oksana, Barzilai, Shlomit, de Haas, Valerie, Baumann, Irith, Schwarz-Furlan, Stephan, Moerloose, Barbara De, Kallay, Krisztián, Smith, Owen, Haas, Valérie De, Gohring, Gudrun, Niemeyer, Charlotte, Nebral, Karin, Simonitsch-Kluppp, Ingrid, Paepe, Pascale De, Van Roy, Nadine, Campr, Vit, Zemanova, Zuzana, Clasen-Linde, Erik, Plesner, Tine, Schlegelberger, Brigitte, Rudelius, Martina, Manola, Kalliopi, Stefanaki, Kalliopi, Csomor, Judit, Andrikovics, Hajnalka, Betts, David, O’Sullivan, Maureen, Zohar, Yaniv, Jeison, Marta, Vito, Rita De, Pasquali, Francesco, Maldyk, Jadwiga, Haus, Olga, Alaiz, Helena, Kjollerstrom, Paula, Lemos, Luis Mascarenhas de, Bodova, Ivana, Čermák, Martin, Plank, Lukas, Gazic, Barbara, Kavcic, Marko, Podgornik, Helena, Ros, Margarita Llavador, Cervera, Jose, Gengler, Carole, Tchinda, Joelle, Beverloo, Berna, Leguit, Roos, Niewisch, Marena R., Sauer, Martin G., Burkhardt, Birgit, Lang, Peter, Bader, Peter, Beier, Rita, Müller, Ingo, Albert, Michael H., Meisel, Roland, Schulz, Ansgar, Cario, Gunnar, Panda, Pritam K., Wehrle, Julius, Hirabayashi, Shinsuke, Derecka, Marta, Durruthy-Durruthy, Robert, Göhring, Gudrun, Yoshimi-Noellke, Ayami, Ku, Manching, Lebrecht, Dirk, Erlacher, Miriam, Flotho, Christian, Strahm, Brigitte, Niemeyer, Charlotte M., and Wlodarski, Marcin W.

Subjects
Medizin
Abstract

Korrektur zu 10.1038/s41591-021-01511-6

Published
2021

59. Hematopoietic stem cell transplantation in children and adolescents with GATA2-related myelodysplastic syndrome

Author

EWOG-MDS, Bortnick, Rachel, Wlodarski, Marcin W., Haas, Valerie de, Moerloose, Barbara de, Dworzak, Michael, Hasle, Henrik, Masetti, Riccardo, Starý, Jan, Turkiewicz, Dominik, Ussowicz, Marek, Kozyra, Emilia J., Albert, Michael, Bader, Peter, Bordon, Victoria, Cario, Gunnar, Beier, Rita, Schulte, Johannes Hubertus, Bresters, Dorine, Müller, Ingo, Pichler, Herbert, Sedlacek, Petr, Sauer, Martin Günther, Zecca, Marco, Göhring, Gudrun, Yoshimi, Ayami, Nöllke, Peter, Erlacher, Miriam, Locatelli, Franco, Niemeyer, Charlotte, Strahm, Brigitte, EWOG-MDS, Bortnick, Rachel, Wlodarski, Marcin W., Haas, Valerie de, Moerloose, Barbara de, Dworzak, Michael, Hasle, Henrik, Masetti, Riccardo, Starý, Jan, Turkiewicz, Dominik, Ussowicz, Marek, Kozyra, Emilia J., Albert, Michael, Bader, Peter, Bordon, Victoria, Cario, Gunnar, Beier, Rita, Schulte, Johannes Hubertus, Bresters, Dorine, Müller, Ingo, Pichler, Herbert, Sedlacek, Petr, Sauer, Martin Günther, Zecca, Marco, Göhring, Gudrun, Yoshimi, Ayami, Nöllke, Peter, Erlacher, Miriam, Locatelli, Franco, Niemeyer, Charlotte, and Strahm, Brigitte

Abstract

GATA2 deficiency is a heterogeneous multi-system disorder characterized by a high risk of developing myelodysplastic syndrome (MDS) and myeloid leukemia. We analyzed the outcome of 65 patients reported to the registry of the European Working Group (EWOG) of MDS in childhood carrying a germline GATA2 mutation (GATA2mut) who had undergone hematopoietic stem cell transplantation (HSCT). At 5 years the probability of overall survival and disease-free survival (DFS) was 75% and 70%, respectively. Non-relapse mortality and relapse equally contributed to treatment failure. There was no evidence of increased incidence of graft-versus-host-disease or excessive rates of infections or organ toxicities. Advanced disease and monosomy 7 (−7) were associated with worse outcome. Patients with refractory cytopenia of childhood (RCC) and normal karyotype showed an excellent outcome (DFS 90%) compared to RCC and −7 (DFS 67%). Comparing outcome of GATA2mut with GATA2wt patients, there was no difference in DFS in patients with RCC and normal karyotype. The same was true for patients with −7 across morphological subtypes. We demonstrate that HSCT outcome is independent of GATA2 germline mutations in pediatric MDS suggesting the application of standard MDS algorithms and protocols. Our data support considering HSCT early in the course of GATA2 deficiency in young individuals.

Published
2021

60. Salvage HLA-haploidentical hematopoietic stem cell transplantation with post-transplant cyclophosphamide for graft failure in non-malignant disorders

Author

Albert, Michael H; https://orcid.org/0000-0001-9143-3263, Sirin, Mehtap, Hoenig, Manfred; https://orcid.org/0000-0001-9165-1690, Hauck, Fabian, Schuetz, Catharina, Bhattacharyya, Rajat, Stepensky, Polina, Jacoby, Elad; https://orcid.org/0000-0003-1411-8942, Güngör, Tayfun; https://orcid.org/0000-0002-3261-1186, Beier, Rita, Schulz, Ansgar; https://orcid.org/0000-0002-7798-7996, Albert, Michael H; https://orcid.org/0000-0001-9143-3263, Sirin, Mehtap, Hoenig, Manfred; https://orcid.org/0000-0001-9165-1690, Hauck, Fabian, Schuetz, Catharina, Bhattacharyya, Rajat, Stepensky, Polina, Jacoby, Elad; https://orcid.org/0000-0003-1411-8942, Güngör, Tayfun; https://orcid.org/0000-0002-3261-1186, Beier, Rita, and Schulz, Ansgar; https://orcid.org/0000-0002-7798-7996

Abstract

Graft failure requires urgent salvage HSCT, but there is no universally accepted approach for this situation. We investigated T-cell replete haploidentical HSCT with post-transplantation cyclophosphamide following serotherapy-based, radiation-free, reduced intensity conditioning in children with non-malignant disorders who had rejected their primary graft. Twelve patients with primary or secondary graft failure received T-cell replete bone marrow grafts from haploidentical donors and post-transplantation cyclophosphamide. The recommended conditioning regimen comprised rituximab 375 mg/m2, alemtuzumab 0.4 mg/kg, fludarabine 150 mg/m2, treosulfan 20–24 g/m2 and cyclophosphamide 29 mg/kg. After a median follow-up of 26 months (7–95), eleven of twelve patients (92%) are alive and well with complete donor chimerism in ten. Neutrophil and platelet engraftment were observed in all patients after a median of 18 days (15–61) and 39 days (15–191), respectively. Acute GVHD grade I was observed in 1/12 patients (8%) and mild chronic GVHD in 1/12 patients (8%). Viral reactivations and disease were frequent complications at 75% and 42%, respectively, but no death from infectious causes occurred. In summary, this retrospective analysis demonstrates that a post-transplantation cyclophosphamide-based HLA-haploidentical salvage HSCT after irradiation-free conditioning results in excellent engraftment and overall survival in children with non-malignant diseases.

Published
2021

61. Interleukin-10 Receptor Signaling in Innate Immune Cells Regulates Mucosal Immune Tolerance and Anti-Inflammatory Macrophage Function

Author

Shouval, Dror S., Biswas, Amlan, Goettel, Jeremy A., McCann, Katelyn, Conaway, Evan, Redhu, Naresh S., Mascanfroni, Ivan D., Al Adham, Ziad, Lavoie, Sydney, Ibourk, Mouna, Nguyen, Deanna D., Samsom, Janneke N., Escher, Johanna C., Somech, Raz, Weiss, Batia, Beier, Rita, Conklin, Laurie S., Ebens, Christen L., Santos, Fernanda G.M.S., Ferreira, Alexandre R., Sherlock, Mary, Bhan, Atul K., Müller, Werner, Mora, Rodrigo J., Quintana, Francisco J., Klein, Christoph, Muise, Aleixo M., Horwitz, Bruce H., and Snapper, Scott B.

Published
2014
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63. Treosulfan vs busulfan conditioning for allogeneic bmt in children with nonmalignant disease: a randomized phase 2 trial

Author

Sykora, Karl-Walter, Beier, Rita, Schulz, Ansgar, Cesaro, Simone, Greil, Johann, Gozdzik, Jolanta, Sedlacek, Petr, Bader, Peter, Schulte, Johannes, Zecca, Marco, Locatelli, Franco, Gruhn, Bernd, Reinhardt, Dirk, Styczynski, Jan, Piras, Simona, Fagioli, Franca, Bonanomi, Sonia, Caniglia, Maurizio, Li, Xieran, Baumgart, Joachim, Kehne, Jochen, Mielcarek-Siedziuk, Monika, and Kalwak, Krzysztof

Abstract

Optimal conditioning prior to allogeneic hematopoietic stem cell transplantation for children with non-malignant diseases is subject of ongoing research. This prospective, randomized, phase 2 trial compared safety and efficacy of busulfan with treosulfan based preparative regimens. Children with non-malignant diseases received fludarabine and either intravenous (IV) busulfan (4.8 to 3.2 mg/kg/day) or IV treosulfan (10, 12, or 14 g/m2/day). Thiotepa administration (2 × 5 mg/kg) was at the investigator’s discretion. Primary endpoint was freedom from transplantation (treatment)-related mortality (freedom from TRM), defined as death between Days -7 and +100. Overall, 101 patients (busulfan 50, treosulfan 51) with at least 12 months follow-up were analyzed. Freedom from TRM was 90.0% (95% CI: 78.2%, 96.7%) after busulfan and 100.0% (95% CI: 93.0%, 100.0%) after treosulfan. Secondary outcomes (transplantation-related mortality [12.0% versus 3.9%]) and overall survival (88.0% versus 96.1%) favored treosulfan. Graft failure was more common after treosulfan (n = 11), than after busulfan (n = 2) while all patients were rescued by second procedures except one busulfan patient. CTCAE Grade III adverse events were similar in both groups. This study confirmed treosulfan to be an excellent alternative to busulfan and can be safely used for conditioning treatment in children with non-malignant disease.

Published
2023
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67. Sickle cell disease in Germany: Results from a national registry

Author

Kunz, Joachim B., Lobitz, Stephan, Grosse, Regine, Oevermann, Lena, Hakimeh, Dani, Jarisch, Andrea, Cario, Holger, Beier, Rita, Schenk, Daniela, Schneider, Dominik, Gross-Wieltsch, Ute, Prokop, Aram, Heine, Sabine, Khurana, Claudia, Erlacher, Miriam, Duerken, Matthias, Linke, Christina, Fruehwald, Michael, Corbacioglu, Selim, Claviez, Alexander, Metzler, Markus, Ebinger, Martin, Full, Hermann, Wiesel, Thomas, Eberl, Wolfgang, Reinhard, Harald, Tagliaferri, Laura, Allard, Pierre, Karapanagiotou-Schenkel, Irini, Rother, Lisa-Marie, Beck, Dorothea, Le Cornet, Lucian, Kulozik, Andreas E., Kunz, Joachim B., Lobitz, Stephan, Grosse, Regine, Oevermann, Lena, Hakimeh, Dani, Jarisch, Andrea, Cario, Holger, Beier, Rita, Schenk, Daniela, Schneider, Dominik, Gross-Wieltsch, Ute, Prokop, Aram, Heine, Sabine, Khurana, Claudia, Erlacher, Miriam, Duerken, Matthias, Linke, Christina, Fruehwald, Michael, Corbacioglu, Selim, Claviez, Alexander, Metzler, Markus, Ebinger, Martin, Full, Hermann, Wiesel, Thomas, Eberl, Wolfgang, Reinhard, Harald, Tagliaferri, Laura, Allard, Pierre, Karapanagiotou-Schenkel, Irini, Rother, Lisa-Marie, Beck, Dorothea, Le Cornet, Lucian, and Kulozik, Andreas E.

Abstract

Background Limited data on the prevalence and medical care of sickle cell disease (SCD) in Germany are available. Here, we make use of a patient registry to characterize the burden of disease and the treatment modalities for patients with SCD in Germany. Procedure A nationwide German registry for patients with SCD documents basic data on diagnosis and patient history retrospectively at the time of registration. A prospective annual documentation provides more details on complications and treatment of SCD. For the current analyses, data of 439 patients were available. Results Most patients had homozygous SCD (HbSS 75.1%, HbS/beta-thalassemia 13.2%, and HbSC 11.3%). The median age at diagnosis was 1.9 years (interquartile range, 0.6-4.4 years), most patients were diagnosed when characteristic symptoms occurred. Sepsis and stroke had affected 3.2% and 4.2% of patients, respectively. During the first year of observation, 48.3% of patients were admitted to a hospital and 10.1% required intensive care. Prophylactic penicillin was prescribed to 95.6% of patients with homozygous SCD or HbS/beta thalassemia below the age of six and hydroxycarbamide to 90.4% of patients above the age of two years. At least one annual transcranial Doppler ultrasound was documented for 74.8% of patients between 2 and 18 years. Conclusion With an estimated number of at least 2000, the prevalence of SCD in Germany remains low. Prospectively, we expect that the quality of care for children with SCD will be further improved by an earlier diagnosis after the anticipated introduction of a newborn screening program for SCD.

Published
2020

69. Risk factors for mixed chimerism in children with hemophagocytic lymphohistiocytosis after reduced toxicity conditioning

Author

Wustrau, Katharina, primary, Greil, Johann, additional, Sykora, Karl‐Walter, additional, Albert, Michael H., additional, Burkhardt, Birgit, additional, Lang, Peter, additional, Meisel, Roland, additional, Wössmann, Wilhelm, additional, Beier, Rita, additional, Schulz, Ansgar, additional, Bader, Peter, additional, Chada, Martin, additional, Kühl, Jörn‐Sven, additional, Schlegel, Paul‐Gerhardt, additional, Speckmann, Carsten, additional, Gruhn, Bernd, additional, Seidel, Markus, additional, Wawer, Angela, additional, Ozga, Ann‐Kathrin, additional, Janka, Gritta, additional, Ehl, Stephan, additional, Müller, Ingo, additional, and Lehmberg, Kai, additional

Published
2020
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71. Kinetics of particle uptake in the domes of Peyer's patches

Author

Beier, Rita and Gebert, Andreas

Subjects
Swine -- Physiological aspects, Cells -- Physiological aspects, Phagocytes -- Physiological aspects, Lysosomes -- Physiological aspects, Biological sciences
Abstract

A study was conducted to examine the kinetics of particle uptake in the Peyer's patches of pigs. Lyophilized baker's yeast was suspended in phosphate-buffered saline while tissue samples were fixed in a solution composed of formaldehyde, glutaraldehyde and CalCl2. Results indicated that particles migrate down to and across the basal lamina within 2.5 to 4-h. Findings also showed that phagocytosis, lysosomal degradation and antigen presentation were supported by subepithelial phagocytes.

Published
1998

74. 92 - Incidence of Secondary Malignancies after Total Body Irradiation-Based Allogeneic HSCT in Children with ALL – Long-Term Follow-up from the Prospective ALL-SCT 2003 Trial

Author

Eichinger, Anna, Glogova, Evgenia, Poetschger, Ulrike, Bader, Peter, Basu, Oliver, Beier, Rita, Burkhardt, Birgit, Classen, Carl-Friedrich, Claviez, Alexander, Corbacioglu, Selim, Deubzer, Hedwig, Greil, Johann, Gruhn, Bernd, Gungor, Tayfun, Kafa, Kinan, Lang, Peter, Lange, Bjoern Soenke, Mueller, Ingo, Sauer, Martin G., Schlegel, Paul-Gerhard, Schulz, Ansgar, Stachel, Klaus Daniel, Strahm, Brigitte R., Wawer, Angela, Kühl, Jörn-Sven, Meisel, Roland, Peters, Christina, and Albert, Michael H.

Published
2022
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77. Risk factors for mixed chimerism after stem cell transplantation with treosulfan or melphalan based conditioning regimens in children and adolescents with primary hemophagocytic lymphohistocytosis

Author

Wustrau, Katharina, Albert, Michael H., Bader, Peter, Beier, Rita, Burkhardt, Birgit, Chada, Martin, Greil, Johann, Gruhn, Bernd, Kühl, Jörn-Sven, Lang, Peter, Meisel, Roland, Schulz, Ansgar, Seidel, Markus, Sykora, Karl-Walter, Wawer, Angela, Wössmann, Wilhelm, Ozga, Ann-Kathrin, Ehl, Stephan, Müller, Ingo, and Lehmberg, Kai

Subjects
Medizin
Published
2018

79. Reduced-Intensity Delayed Intensification in Standard-Risk Pediatric Acute Lymphoblastic Leukemia Defined by Undetectable Minimal Residual Disease: Results of an International Randomized Trial (AIEOP-BFM ALL 2000)

Author

Schrappe, Martin, Bleckmann, Kirsten, Zimmermann, Martin, Biondi, Andrea, Möricke, Anja, Locatelli, Franco, Cario, Gunnar, Rizzari, Carmelo, Attarbaschi, Andishe, Valsecchi, Maria Grazia, Bartram, Claus R, Barisone, Elena, Niggli, Felix, Niemeyer, Charlotte, Testi, Anna Maria, Mann, Georg, Ziino, Ottavio, Schäfer, Beat, Panzer-Grümayer, Renate, Beier, Rita, Parasole, Rosanna, Göhring, Gudrun, Ludwig, Wolf-Dieter, Casale, Fiorina, Schlegel, Paul-Gerhardt, Basso, Giuseppe, Conter, Valentino, Schrappe, Martin, Bleckmann, Kirsten, Zimmermann, Martin, Biondi, Andrea, Möricke, Anja, Locatelli, Franco, Cario, Gunnar, Rizzari, Carmelo, Attarbaschi, Andishe, Valsecchi, Maria Grazia, Bartram, Claus R, Barisone, Elena, Niggli, Felix, Niemeyer, Charlotte, Testi, Anna Maria, Mann, Georg, Ziino, Ottavio, Schäfer, Beat, Panzer-Grümayer, Renate, Beier, Rita, Parasole, Rosanna, Göhring, Gudrun, Ludwig, Wolf-Dieter, Casale, Fiorina, Schlegel, Paul-Gerhardt, Basso, Giuseppe, and Conter, Valentino

Abstract

Purpose Delayed intensification (DI) is an integral part of treatment of childhood acute lymphoblastic leukemia (ALL), but it is associated with relevant toxicity. Therefore, standard-risk patients of trial AIEOP-BFM ALL 2000 (Combination Chemotherapy Based on Risk of Relapse in Treating Young Patients With ALL) were investigated with the specific aim to reduce treatment intensity. Patients and Methods Between July 2000 and July 2006, 1,164 patients (1 to 17 years of age) with standard-risk ALL (defined as the absence of high-risk cytogenetics and undetectable minimal residual disease on days 33 and 78) were randomly assigned to either experimental reduced-intensity DI (protocol III; P-III) or standard DI (protocol II; P-II). Cumulative drug doses of P-III were reduced by 30% for dexamethasone and 50% for vincristine, doxorubicin, and cyclophosphamide, which shortened the treatment duration from 49 to 29 days. The study aimed at noninferiority of reduced-intensity P-III; analyses were performed according to treatment given. Results For P-III and P-II, respectively, the 8-year rate of disease-free survival (± SE) was 89.2 ± 1.3% and 92.3 ± 1.2% ( P = .04); cumulative incidence of relapse, 8.7 ± 1.2% and 6.4 ± 1.1% ( P = .09); and overall survival, 96.1 ± 0.8% and 98.0 ± 0.6% ( P = .06). Patients with ETV6-RUNX1-positive ALL and patients 1 to 6 years of age performed equally well in both arms. The incidence of death during remission was comparable, which indicates equivalent toxicity. The 8-year cumulative incidence rate of secondary malignancies was 1.3 ± 0.5% and 0.6 ± 0.4% for P-III and P-II, respectively ( P = .37). Conclusion Although the criteria used for the standard-risk definition in this trial identified patients with exceptionally good prognosis, reduction of chemotherapy was not successful mainly because of an increased rate of relapse. The data suggest that treatment reduction is feasible in specific subgroups, which underlines the biologic heterogeneity

Published
2018

80. Hematopoietic stem cell transplantation in children and adolescents with GATA2-related myelodysplastic syndrome

Author

Bortnick, Rachel, Wlodarski, Marcin, de Haas, Valerie, De Moerloose, Barbara, Dworzak, Michael, Hasle, Henrik, Masetti, Riccardo, Starý, Jan, Turkiewicz, Dominik, Ussowicz, Marek, Kozyra, Emilia, Albert, Michael, Bader, Peter, Bordon, Victoria, Cario, Gunnar, Beier, Rita, Schulte, Johannes, Bresters, Dorine, Müller, Ingo, Pichler, Herbert, Sedlacek, Petr, Sauer, Martin G., Zecca, Marco, Göhring, Gudrun, Yoshimi, Ayami, Noellke, Peter, Erlacher, Miriam, Locatelli, Franco, Niemeyer, Charlotte M., and Strahm, Brigitte

Abstract

GATA2 deficiency is a heterogeneous multi-system disorder characterized by a high risk of developing myelodysplastic syndrome (MDS) and myeloid leukemia. We analyzed the outcome of 65 patients reported to the registry of the European Working Group (EWOG) of MDS in childhood carrying a germline GATA2mutation (GATA2mut) who had undergone hematopoietic stem cell transplantation (HSCT). At 5 years the probability of overall survival and disease-free survival (DFS) was 75% and 70%, respectively. Non-relapse mortality and relapse equally contributed to treatment failure. There was no evidence of increased incidence of graft-versus-host-disease or excessive rates of infections or organ toxicities. Advanced disease and monosomy 7 (−7) were associated with worse outcome. Patients with refractory cytopenia of childhood (RCC) and normal karyotype showed an excellent outcome (DFS 90%) compared to RCC and −7 (DFS 67%). Comparing outcome of GATA2mutwith GATA2wtpatients, there was no difference in DFS in patients with RCC and normal karyotype. The same was true for patients with −7 across morphological subtypes. We demonstrate that HSCT outcome is independent of GATA2germline mutations in pediatric MDS suggesting the application of standard MDS algorithms and protocols. Our data support considering HSCT early in the course of GATA2 deficiency in young individuals.

Published
2021
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82. Reduced-Intensity Delayed Intensification in Standard-Risk Pediatric Acute Lymphoblastic Leukemia Defined by Undetectable Minimal Residual Disease: Results of an International Randomized Trial (AIEOP-BFM ALL 2000)

Author

Schrappe, Martin, primary, Bleckmann, Kirsten, additional, Zimmermann, Martin, additional, Biondi, Andrea, additional, Möricke, Anja, additional, Locatelli, Franco, additional, Cario, Gunnar, additional, Rizzari, Carmelo, additional, Attarbaschi, Andishe, additional, Valsecchi, Maria Grazia, additional, Bartram, Claus R., additional, Barisone, Elena, additional, Niggli, Felix, additional, Niemeyer, Charlotte, additional, Testi, Anna Maria, additional, Mann, Georg, additional, Ziino, Ottavio, additional, Schäfer, Beat, additional, Panzer-Grümayer, Renate, additional, Beier, Rita, additional, Parasole, Rosanna, additional, Göhring, Gudrun, additional, Ludwig, Wolf-Dieter, additional, Casale, Fiorina, additional, Schlegel, Paul-Gerhardt, additional, Basso, Giuseppe, additional, and Conter, Valentino, additional

Published
2018
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83. Reduced Intensity Delayed Intensification in Standard-Risk Patients Defined By Minimal Residual Disease in Childhood Acute Lymphoblastic Leukemia: Results of an International Randomized Trial in 1164 Patients (Trial AIEOP-BFM ALL 2000)

Author

Schrappe, Martin, primary, Zimmermann, Martin, additional, Möricke, Anja, additional, Mann, Georg, additional, Valsecchi, Maria Grazia, additional, Bartram, Claus R., additional, Biondi, Andrea, additional, Panzer-Grümayer, Renate, additional, Beier, Rita, additional, Cario, Gunnar, additional, Locatelli, Franco, additional, Basso, Giuseppe, additional, Niggli, Felix, additional, Aricò, Maurizio, additional, and Conter, Valentino, additional

Published
2016
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84. Classical Hodgkin lymphoma-type PTLD after solid organ transplantation in children: a report on 17 patients treated according to subsequent GPOH-HD treatment schedules

Author

Kampers, Johanna, primary, Orjuela-Grimm, Manuela, additional, Schober, Tilmann, additional, Schulz, Thomas F., additional, Stiefel, Martina, additional, Klein, Christoph, additional, Körholz, Dieter, additional, Mauz-Körholz, Christine, additional, Kreipe, Hans, additional, Beier, Rita, additional, and Maecker-Kolhoff, Britta, additional

Published
2016
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85. Unique and shared signaling pathways cooperate to regulate the differentiation of human CD4+ T cells into distinct effector subsets

Author

Ma, Cindy S., primary, Wong, Natalie, additional, Rao, Geetha, additional, Nguyen, Akira, additional, Avery, Danielle T., additional, Payne, Kathryn, additional, Torpy, James, additional, O’Young, Patrick, additional, Deenick, Elissa, additional, Bustamante, Jacinta, additional, Puel, Anne, additional, Okada, Satoshi, additional, Kobayashi, Masao, additional, Martinez-Barricarte, Ruben, additional, Elliott, Michael, additional, Sebnem Kilic, Sara, additional, El Baghdadi, Jamila, additional, Minegishi, Yoshiyuki, additional, Bousfiha, Aziz, additional, Robertson, Nic, additional, Hambleton, Sophie, additional, Arkwright, Peter D., additional, French, Martyn, additional, Blincoe, Annaliesse K., additional, Hsu, Peter, additional, Campbell, Dianne E., additional, Stormon, Michael O., additional, Wong, Melanie, additional, Adelstein, Stephen, additional, Fulcher, David A., additional, Cook, Matthew C., additional, Stepensky, Polina, additional, Boztug, Kaan, additional, Beier, Rita, additional, Ikincioğullari, Aydan, additional, Ziegler, John B., additional, Gray, Paul, additional, Picard, Capucine, additional, Boisson-Dupuis, Stéphanie, additional, Phan, Tri Giang, additional, Grimbacher, Bodo, additional, Warnatz, Klaus, additional, Holland, Steven M., additional, Uzel, Gulbu, additional, Casanova, Jean-Laurent, additional, and Tangye, Stuart G., additional

Published
2016
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86. Dyskeratosis congenita (DC) bei zwei Kindern: Seltene Erkrankungen und ungewöhnliche Komplikationen erfordern interdisziplinäre Nachsorge

Author

Stratmann, Christine, Kratz, Christian, Bettoni, Claudia, Wieland, Ivonne, Koch, Corinna, Rose, Martina, Drube, Jens, Schubert, Kai-Peter, Sykora, Karl-Walter, Göhring, Gudrun, Beier, Rita, and Grigull, Lorenz

Subjects
ddc: 610, 610 Medical sciences, Medicine
Abstract

Einleitung: Die Dyskeratosis congenita (DC) ist eine multisystemische Erkrankung die klinisch durch die mucocutane Trias aus Nageldystrophie, oraler Leukoplakie und Pigmenstörungen der Haut sowie zunehmende Knochenmarkinsuffizienz imponiert. Als genetische Ursachen sind bisher verschiedene Mutationen[for full text, please go to the a.m. URL], 62. Jahrestagung der Norddeutschen Gesellschaft für Kinder- und Jugendmedizin (NDGKJ)

Published
2013

87. Mo1210 Loss of IL-10 Receptor Signaling in Patients With Infantile IBD Results in Aberrant Th17 Responses and Enhanced T-Cell Proliferation

Author

Shouval, Dror S., primary, Griffith, Alexandra, additional, Bousvaros, Athos, additional, Weiss, Batia, additional, Somech, Raz, additional, Loizides, Anthony, additional, Beier, Rita, additional, Santos, Fernanda G., additional, Sherlock, Mary, additional, Muise, Aleixo, additional, Klein, Christoph, additional, and Snapper, Scott B., additional

Published
2015
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88. Response-Adapted Sequential Immuno-Chemotherapy of Post-Transplant Lymphoproliferative Disorders in Pediatric Solid Organ Transplant Recipients: Results from the Prospective Ped-PTLD 2005 Trial

Author

Maecker-Kolhoff, Britta, primary, Beier, Rita, additional, Zimmermann, Martin, additional, Schlegelberger, Brigitte, additional, Baumann, Ulrich, additional, Mueller, Carsten, additional, Pape, Lars, additional, Reiter, Alfred, additional, Rossig, Claudia, additional, Schubert, Stephan, additional, Toenshoff, Burkhard, additional, Wingen, Anne-Margret, additional, Meissner, Barbara, additional, Kebelmann-Betzing, Christian, additional, Henze, Guenter, additional, Kreipe, Hans H., additional, and Klein, Christoph, additional

Published
2014
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89. Hodgkin’s Disease / Hodgkin-PTLD after Solid Organ Transplantation in Children: A Report on 16 Patients Treated According to Subsequent Gpoh-HD Treatment Schedules

Author

Kampers, Johanna, primary, Beier, Rita, additional, Orjuela-Grimm, Manuela A., additional, Schober, Tilmann, additional, Schulz, Thomas, additional, Stiefel, Martina, additional, Klein, Christoph, additional, Mauz-Koerholz, Christine, additional, Kreipe, Hans H., additional, and Maecker-Kolhoff, Britta, additional

Published
2014
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91. Classical Hodgkin lymphoma-type PTLD after solid organ transplantation in children: a report on 17 patients treated according to subsequent GPOH-HD treatment schedules.

Author

Kampers, Johanna, Orjuela-Grimm, Manuela, Schober, Tilmann, Schulz, Thomas F., Stiefel, Martina, Klein, Christoph, Körholz, Dieter, Mauz-Körholz, Christine, Kreipe, Hans, Beier, Rita, and Maecker-Kolhoff, Britta

Subjects
LYMPHOPROLIFERATIVE disorders, HODGKIN'S disease, COMPLICATIONS from organ transplantation, CANCER chemotherapy, IMMUNOHISTOCHEMISTRY, HISTOLOGY
Abstract

Post-transplant lymphoproliferative disease (PTLD) is a severe complication after solid organ transplantation (SOT). Classical Hodgkin lymphoma-type (HL-) PTLD is a rare subtype, and systematic data on treatment and prognosis are lacking. We report on 17 pediatric patients with classical HL-PTLD. HL-PTLD developed late at a median of 8.1 years after SOT. It was commonly EBV-positive (16/17) and expressed both CD30 (all tumors) and CD20 (8/17 tumors). Patients were treated with chemotherapy +/− involved field radiotherapy (IF-RT) according to the respective GPOH-HD protocol tailored by stage and LDH. Overall survival at 2 and 5 years was 86% with 81% of patients surviving event-free. Six patients had additional rituximab treatment; in two it was given as upfront monotherapy and in four was given concurrently with their chemotherapy. Rituximab monotherapy did not lead to long-term remission. In conclusion, treatment of HL-PTLD with classical HL chemotherapy is effective and tolerable. New treatment modalities such as CD30-targeted or EBV-specific agents may diminish toxicity. [ABSTRACT FROM PUBLISHER]

Published
2017
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92. 785 Anti-Inflammatory Macrophage Function Requires IL10 Receptor Signaling and Regulates Mucosal Homeostasis in Mice and Humans

Author

Shouval, Dror S., primary, Biswas, Amlan, additional, Goettel, Jeremy A., additional, McCann, Katelyn, additional, Conaway, Evan, additional, Samsom, Janneke N., additional, Escher, Johanna C., additional, Weiss, Batia, additional, Somech, Raz, additional, Beier, Rita, additional, Conklin, Laurie, additional, Ebens, Christen L., additional, Bhan, Atul K., additional, Klein, Christoph, additional, Muise, Aleixo, additional, Horwitz, Bruce, additional, and Snapper, Scott B., additional

Published
2014
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93. Gene Therapy for Wiskott-Aldrich Syndrome—Long-Term Efficacy and Genotoxicity

Author

Braun, Christian Jörg, primary, Boztug, Kaan, additional, Paruzynski, Anna, additional, Witzel, Maximilian, additional, Schwarzer, Adrian, additional, Rothe, Michael, additional, Modlich, Ute, additional, Beier, Rita, additional, Göhring, Gudrun, additional, Steinemann, Doris, additional, Fronza, Raffaele, additional, Ball, Claudia Regina, additional, Haemmerle, Reinhard, additional, Naundorf, Sonja, additional, Kühlcke, Klaus, additional, Rose, Martina, additional, Fraser, Chris, additional, Mathias, Liesl, additional, Ferrari, Rudolf, additional, Abboud, Miguel R., additional, Al-Herz, Waleed, additional, Kondratenko, Irina, additional, Maródi, László, additional, Glimm, Hanno, additional, Schlegelberger, Brigitte, additional, Schambach, Axel, additional, Albert, Michael Heinrich, additional, Schmidt, Manfred, additional, von Kalle, Christof, additional, and Klein, Christoph, additional

Published
2014
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94. Prediction of outcome by early bone marrow response in childhood acute lymphoblastic leukemia treated in the ALL-BFM 95 trial: differential effects in precursor B-cell and T-cell leukemia

Author

Lauten, Melchior, Möricke, Anja, Beier, Rita, Zimmermann, Martin, Stanulla, Martin, Meissner, Barbara, Odenwald, Edelgard, Attarbaschi, Andishe, Niemeyer, Charlotte, Niggli, Felix, Riehm, Hansjörg, Schrappe, Martin, Lauten, Melchior, Möricke, Anja, Beier, Rita, Zimmermann, Martin, Stanulla, Martin, Meissner, Barbara, Odenwald, Edelgard, Attarbaschi, Andishe, Niemeyer, Charlotte, Niggli, Felix, Riehm, Hansjörg, and Schrappe, Martin

Abstract

BACKGROUND: In the ALL-BFM 95 trial for treatment of acute lymphoblastic leukemia, response to a prednisone pre-phase (prednisone response) was used for risk stratification in combination with age and white blood cell count at diagnosis, response to induction therapy and specific genetic high-risk features. DESIGN AND METHODS: Cytomorphological marrow response was prospectively assessed on Day 15 during induction, and its prognostic value was analyzed in 1,431 patients treated on ALL-BFM 95. RESULTS: The 8-year probabilities of event-free survival were 86.1%, 74.5%, and 46.4% for patients with M1, M2, and M3 Day 15 marrows, respectively. Compared to prednisone response, Day 15 marrow response was superior in outcome prediction in precursor B-cell and T-cell leukemia with, however, a differential effect depending on blast lineage. Outcome was poor in T-cell leukemia patients with prednisone poor-response independent of Day 15 marrow response, whereas among patients with prednisone good-response different risk groups could be identified by Day 15 marrow response. In contrast, prednisone response lost prognostic significance in precursor B-cell leukemia when stratified by Day 15 marrow response. Age and white blood cell count retained their independent prognostic effect. CONCLUSIONS: Selective addition of Day 15 marrow response to conventional stratification criteria applied on ALL-BFM 95 (currently in use in several countries as regular chemotherapy protocol for childhood acute lymphoblastic leukemia) may significantly improve risk-adapted treatment delivery. Even though cutting-edge trial risk stratification is meanwhile dominated by minimal residual disease evaluation, an improved conventional risk assessment, as presented here, could be of great importance to countries that lack the technical and/or financial resources associated with the application of minimal residual disease analysis.

Published
2012

95. Hematopoietic Stem Cell Gene Therapy For Wiskott- Aldrich Syndrome

Author

Witzel, Maximilian GW, primary, Braun, Christian J., primary, Boztug, Kaan, primary, Paruzynski, Anna, primary, Albert, Michael H., primary, Schwarzer, Adrian, primary, Modlich, Ute, primary, Beier, Rita, primary, Göhring, Gudrun, primary, Naundorf, Sonja, primary, Kühlcke, Klaus, primary, Rose, Martina, primary, Fraser, Christopher, primary, Mathias, Liesl, primary, Ferrari, Rudolf, primary, Abboud, Miguel R., primary, Al-Herz, Waleed, primary, Kondratenko, Irina, primary, Maródi, László, primary, Schlegelberger, Brigitte, primary, Baum, Christopher, primary, Schmidt, Manfred, primary, von Kalle, Christof, primary, and Klein, Christoph, primary

Published
2013
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96. Pediatric Fanconi Anemia With Secondary AML: A Retrospective Outcome Report From The German AML-BFM Group

Author

Beier, Rita, primary, Hoseini, Sayed, additional, Maecker-Kolhoff, Britta, additional, Schneppenheim, Reinhard, additional, von Neuhoff, Christine, additional, Ebell, Wolfram, additional, Schmid, Hansjörg, additional, Bader, Peter, additional, Ehlert, Karoline, additional, Sykora, Karl-Walter, additional, Creutzig, Ursula, additional, Kratz, Christian, additional, Chao, Mwe Mwe, additional, Reinhardt, Dirk, additional, and Sauer, Martin G., additional

Published
2013
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98. G6PC3 Deficiency Associated with Congenital Neutropenia and Enterocolitis

Author

Diestelhorst, Jana, primary, Kotlarz, Daniel, additional, Appaswamy, Giridharan, additional, Beier, Rita, additional, Krawitz, Peter M, additional, Robinson, Peter N, additional, Hecht, Jochen, additional, Puchalka, Jacek, additional, Schäffer, Alejandro A., additional, Pfeifer, Dietmar, additional, Baumann, Ulrich, additional, Pfister, Eva-Doreen, additional, Brandes, Gudrun, additional, Palla, Gabriella, additional, Jacobs, Roland, additional, Kreipe, Hans H., additional, Mundlos, Stefan, additional, and Klein, Christoph, additional

Published
2011
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99. The Phenotype of Human STK4 Deficiency

Author

Abdollahpour, Hengameh, primary, Appaswamy, Giridharan, additional, Beier, Rita, additional, Schäffer, Alejandro A., additional, Gertz, E. Michael, additional, Kreipe, Hans H., additional, Pfeifer, Dietmar, additional, Grimbacher, Bodo, additional, Lohrmann, Sabine, additional, Sherkat, Roya, additional, and Klein, Christoph, additional

Published
2011
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100. Efficacy of Gene Therapy for Wiskott-Aldrich-Syndrome

Author

Braun, Christian J., primary, Boztug, Kaan, additional, Schmidt, Manfred, additional, Albert, Michael H, additional, Schwarzer, Adrian, additional, Paruzynski, Anna, additional, Modlich, Ute, additional, Beier, Rita, additional, Göhring, Gudrun, additional, Naundorf, Sonja, additional, Kühlcke, Klaus, additional, Rose, Martina, additional, Fraser, Christopher, additional, Mathias, Liesl A., additional, Ferrari, Rudolf, additional, Abboud, Miguel R., additional, Al-Herz, Waleed, additional, Kondratenko, Irina, additional, Maródi, László, additional, Schlegelberger, Brigitte, additional, Baum, Christopher, additional, von Kalle, Christof, additional, and Klein, Christoph, additional

Published
2011
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