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16. Cardiac and pulmonary late effects do not negatively influence performance status and non-relapse mortality of children surviving five yr after autologous hematopoietic cell transplantation: Report from the EBMT Paediatric Diseases and Late Effects Working Parties

Author

Uderzo, Cornelio, Pillon, Marta, Tridello, Gloria, Dini, Giorgio, Urban, Christian, Corti, Paola, Zintl, Felix, Fagioli, Franca, Messina, Chiara, Verdeguer, Amparo, Faraci, Maura, Fedeli, Sara, Tana, Francesco, Tichelli, André, Passweg, Jakob, and Rovelli, Attilio

Published
2009
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17. Hematopoietic stem cell transplantation for de novo acute megakaryocytic leukemia in first complete remission: a retrospective study of the European Group for Blood and Marrow Transplantation (EBMT)

Author

Garderet, Laurent, Labopin, Myriam, Gorin, Norbert-Claude, Polge, Emmanuelle, Baruchel, André, Meloni, Giovanna, Ortega, Juan, Vossen, Jaak, Bunjes, Donald, Leverger, Guy, Blaise, Didier, Ferrant, Augustin, Brune, Mats, Dore, Eric, Gadner, Helmut, Zintl, Felix, Yaniv, Isaac, Dini, Giorgio, and Frassoni, Francesco

Published
2005
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25. Autologous bone-marrow transplants compared with chemotherapy for children with acute lymphoblastic leukaemia in a second remission: a matched-pair analysis

Author

Borgmann, Anja, Schmid, Hansjorg, Hartmann, Reinhard, Baumgarten, Eckehard, Hermann, Klaus, Klingebiel, Thomas, Ebell, Wolfram, Zintl, Felix, Gadner, Helmut, and Henze, Gunter

Subjects
Lymphoblastic leukemia in children -- Care and treatment, Bone marrow -- Transplantation, Chemotherapy -- Evaluation, Homografts -- Evaluation
Published
1995

30. Successful bone marrow transplantation in a patient with DNA ligase IV deficiency and bone marrow failure

Author

Bechtold Astrid, Neitzel Heidemarie, Tönnies Holger, Varon Raymonda, Zintl Felix, Seidel Joerg, Gruhn Bernd, Hoehn Holger, and Schindler Detlev

Subjects
Medicine
Abstract

Abstract Background DNA Ligase IV deficiency syndrome is a rare autosomal recessive disorder caused by hypomorphic mutations in the DNA ligase IV gene (LIG4). The clinical phenotype shows overlap with a number of other rare syndromes, including Seckel syndrome, Nijmegen breakage syndrome, and Fanconi anemia. Thus the clinical diagnosis is often delayed and established by exclusion. Methods We describe a patient with pre- and postnatal growth retardation and dysmorphic facial features in whom the diagnoses of Seckel-, Dubowitz-, and Nijmegen breakage syndrome were variably considered. Cellular radiosensitivity in the absence of clinical manifestations of Ataxia telangiectasia lead to the diagnosis of DNA ligase IV (LIG4) deficiency syndrome, confirmed by compound heterozygous mutations in the LIG4 gene. At age 11, after a six year history of progressive bone marrow failure and increasing transfusion dependency the patient was treated with matched sibling donor hematopoetic stem cell transplantation (HSCT) using a fludarabine-based conditioning regimen without irradiation. Results The post-transplantation course was uneventful with rapid engraftment leading to complete and stable chimerism. Now at age 16, the patient has gained weight and is in good clinical condition. Conclusion HSCT using mild conditioning without irradiation qualifies as treatment of choice in LIG4-deficient patients who have a matched sibling donor.

Published
2007
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31. Improved Treatment Results in Childhood B-Cell Neoplasms With Tailored Intensification of Therapy: A Report of the Berlin-Frankfurt-Mu¨nster Group Trial NHL-BFM 90

Author

Reiter, Alfred, Schrappe, Martin, Tiemann, Markus, Ludwig, Wolf-Dieter, Yakisan, Elif, Zimmermann, Martin, Mann, Georg, Chott, Andreas, Ebell, Wolfram, Klingebiel, Thomas, Graf, Norbert, Kremens, Bernhard, Mu¨ller-Weihrich, Stefan, Plu¨ss, Hans-Ju¨rgen, Zintl, Felix, Henze, Gu¨nter, and Riehm, Hansjo¨rg

Published
1999
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37. Cardiac and pulmonary late effects do not negatively influence performance status and non-relapse mortality of children surviving five yr after autologous hematopoietic cell transplantation

Author

Uderzo, Cornelio, Pillon, Marta, Tridello, Gloria, Dini, Giorgio, Urban, Christian, Corti, Paola, Zintl, Felix, Fagioli, Franca, Messina, Chiara, Verdeguer, Amparo, Faraci, Maura, Fedeli, Sara, Tana, Francesco, Tichelli, André, Passweg, Jakob, and Rovelli, Attilio

Subjects
ddc:616, Male, Time Factors, Adolescent, Incidence, Hematopoietic Stem Cell Transplantation/adverse effects/methods, Infant, Lung/physiology, Heart/physiology, Respiratory Function Tests, Pediatrics/methods, Treatment Outcome, Child, Preschool, Humans, Female, Prospective Studies, Child
Abstract

The current prospective study dealt with clinical outcome associated with pulmonary and cardiac late effects of AuHCT in children with malignancies. We prospectively evaluated 58 children, utilizing pulmonary function tests and cardiac shortening fraction, performed in pre-AuHCT phase and then annually. The overall five-yr survival was 68%. The five-yr cumulative incidence of lung and cardiac function impairment in survivors was 21% in both cases. None of the patients presented with restrictive or obstructive pulmonary pathology at the last follow-up and performance status for all survivors, ranged from 90% to 100%. The cumulative incidence of non-relapse mortality was 12.6% (range 6.3-25.3%), whereas relapse mortality was 19.7% (range 11.6-33.5). In conclusion, our study shows no significant deterioration in post-AuHCT pulmonary and cardiac function and in particular, no negative impact of lung and heart late effects on performance status and non-relapse mortality.

Published
2009

38. Treatment of chronic granulomatous disease with myeloablative conditioning and an unmodified hemopoietic allograft: a survey of the European experience, 1985-2000

Author

Seger, Reinhard A, Gungor, Tayfun, Belohradsky, Bernd H, Blanche, Stephane, Bordigoni, Pierre, Di Bartolomeo, Paolo, Flood, Terence, Landais, Paul, Müller, Susanna, Ozsahin, Ayse Hulya, Passwell, Justen H, Porta, Fulvio, Slavin, Shimon, Wulffraat, Nico, Zintl, Felix, Nagler, Arnon, Cant, Andrew, and Fischer, Alain

Subjects
Adult, Male, Adolescent, Antibodies, Neoplasm, Antibodies, Monoclonal, Humanized, Transplantation, Homologous/adverse effects, Vidarabine/analogs & derivatives, Genetic Heterogeneity, Cause of Death, Granulomatous Disease, Chronic/genetics/therapy, Humans, Neutrophils/physiology, Graft vs Host Disease/epidemiology/etiology, Child, Hematopoietic Stem Cell Transplantation/adverse effects/statistics & numerical data, Busulfan, Cyclophosphamide, Melphalan, Antilymphocyte Serum, Inflammation, Infection Control, ddc:618, Bone Marrow/drug effects/radiation effects, Antibodies, Monoclonal, Survival Analysis, Europe, surgical procedures, operative, Treatment Outcome, Child, Preschool, Infection/complications, Transplantation Conditioning/adverse effects/statistics & numerical data, Female
Abstract

Treatment of chronic granulomatous disease (CGD) with myeloablative bone marrow transplantation is considered risky. This study investigated complications and survival according to different risk factors present at transplantation. The outcomes of 27 transplantations for CGD, from 1985 to 2000, reported to the European Bone Marrow Transplant Registry for primary immunodeficiencies were assessed. Most transplant recipients were children (n = 25), received a myeloablative busulphan-based regimen (n = 23), and had unmodified marrow allografts (n = 23) from human leukocyte antigen (HLA)-identical sibling donors (n = 25). After myeloablative conditioning, all patients fully engrafted with donor cells; after myelosuppressive regimens, 2 of 4 patients fully engrafted. Severe (grade 3 or 4) graft-versus-host disease (GVHD) disease developed in 4 patients: 3 of 9 with pre-existing overt infection, 1 of 2 with acute inflammatory disease. Exacerbation of infection during aplasia was observed in 3 patients; inflammatory flare at the infection site during neutrophil engraftment in 2: all 5 patients belonged to the subgroup of 9 with pre-existing infection. Overall survival was 23 of 27, with 22 of 23 cured of CGD (median follow-up, 2 years). Survival was especially good in patients without infection at the moment of transplantation (18 of 18). Pre-existing infections and inflammatory lesions have cleared in all survivors (except in one with autologous reconstitution). Myeloablative conditioning followed by transplantation of unmodified hemopoietic stem cells, if performed at the first signs of a severe course of the disease, is a valid therapeutic option for children with CGD having an HLA-identical donor.

Published
2002

39. Hematopoietic stem cell transplantation for de novo acute megakaryocytic leukemia in first complete remission: a retrospective study of the European Group for Blood and Marrow Transplantation (EBMT).

Author

UCL - (MGD) Service d'hématologie, UCL - MD/MINT - Département de médecine interne, Garderet, Laurent, Labopin, Myriam, Gorin, Norbert-Claude, Polge, Emmanuelle, Baruchel, André, Meloni, Giovanna, Ortega, Juan, Vossen, Jaak, Bunjes, Donald, Leverger, Guy, Blaise, Didier, Ferrant, Augustin, Brune, Mats, Dore, Eric, Gadner, Helmut, Zintl, Felix, Yaniv, Isaac, Dini, Giorgio, Frassoni, Francesco, UCL - (MGD) Service d'hématologie, UCL - MD/MINT - Département de médecine interne, Garderet, Laurent, Labopin, Myriam, Gorin, Norbert-Claude, Polge, Emmanuelle, Baruchel, André, Meloni, Giovanna, Ortega, Juan, Vossen, Jaak, Bunjes, Donald, Leverger, Guy, Blaise, Didier, Ferrant, Augustin, Brune, Mats, Dore, Eric, Gadner, Helmut, Zintl, Felix, Yaniv, Isaac, Dini, Giorgio, and Frassoni, Francesco

Abstract

Acute megakaryoblastic leukemia (M7 AML) is a highly aggressive disease. We evaluated outcomes in 57 children (11 with Down syndrome) and 69 adults with M7 AML after first complete remission (CR1) following autologous or HLA-identical allogeneic transplantation. Characteristics of the recipients of autologous transplants (38 children, 37 adults) were, respectively: median age, 1.7 and 46 years; non-total body irradiation (non-TBI) conditioning regimen, 97% and 70%; bone marrow as stem cell source, 74% and 43%. Characteristics of the recipients of allogeneic transplants (19 children, 32 adults) were, respectively: median age, 2.8 and 37 years; non-TBI regimen, 63% and 42%; bone marrow as stem cell source, 95% and 69%. Autologous transplantation benefited children more; the relapse rate was high in adults. Results for autologous transplantation were (children and adults, respectively): engraftment, 90% and 100%; 3-year treatment-related mortality (TRM) rate, 3% and 8%; relapse rate, 45% and 64%; leukemia-free survival (LFS) rate, 52% and 27%; overall survival (OS) rate, 61% and 30%. After allogeneic transplantation, TRM was fairly low in children and adults, and relapse rates were lower than after autologous transplantation. Results for allogeneic transplantation were, respectively: engraftment, 95% and 90%; TRM, 0% and 26%; relapse rate, 34% and 28%; LFS, 66% and 46%; OS, 82% and 43%). We conclude that M7 AML patients in CR1 (except children with Down syndrome, who already have better outcomes) can benefit from transplantation.

Published
2005

45. Risk-adjusted therapy of acute lymphoblastic leukemia can decrease treatment burden and improve survival: treatment results of 2169 unselected pediatric and adolescent patients enrolled in the trial ALL-BFM 95

Author

Möricke, Anja, primary, Reiter, Alfred, additional, Zimmermann, Martin, additional, Gadner, Helmut, additional, Stanulla, Martin, additional, Dördelmann, Michael, additional, Löning, Lutz, additional, Beier, Rita, additional, Ludwig, Wolf-Dieter, additional, Ratei, Richard, additional, Harbott, Jochen, additional, Boos, Joachim, additional, Mann, Georg, additional, Niggli, Felix, additional, Feldges, Andreas, additional, Henze, Günter, additional, Welte, Karl, additional, Beck, Jörn-Dirk, additional, Klingebiel, Thomas, additional, Niemeyer, Charlotte, additional, Zintl, Felix, additional, Bode, Udo, additional, Urban, Christian, additional, Wehinger, Helmut, additional, Niethammer, Dietrich, additional, Riehm, Hansjörg, additional, and Schrappe, Martin, additional

Published
2008
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47. IL23R Arg381Gln Variant in the Donor Confers Strong Protection Against Acute Graft-Versus Host Disease after Hematopoietic Stem Cell Transplantation in Children with Hematological Malignancies.

Author

Gruhn, Bernd, primary, Intek, Juliane, additional, Pfaffendorf, Nadine, additional, Wittig, Susan, additional, Wolff, Ilona, additional, Schiller, Isabella, additional, Voigt, Astrid, additional, Häfer, Ralf, additional, Fuchs, Dietlinde, additional, and Zintl, Felix, additional

Published
2007
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48. Hematopoietic Stem Cell Transplantation (HSCT) after a Myeloablative Conditioning Regimen in Children with Refractory Cytopenia (RC): Results of a Retrospective Analysis from the EWOG-MDS Group.

Author

Locatelli, Franco, primary, Noellke, Peter, primary, Fisher, Alexandra, primary, Bader, Peter, primary, Bernardo, Maria Ester, primary, Bergstraesser, Eva, primary, Hasle, Henrik, primary, van den Heuvel-Eibrink, Marry, primary, Korthof, Elisabeth, primary, Stary, Jan, primary, Strahm, Brigitte, primary, Zintl, Felix, primary, Sedlacek, Petr, primary, Zecca, Marco, primary, and Niemeyer, Charlotte M., primary

Published
2007
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49. Risk-Adjusted Therapy of Acute Lymphoblastic Leukemia Can Decrease Treatment Burden and Improve Survival: Treatment Results of 2169 Unselected Pediatric and Adolescent Patients Enrolled in the Trial ALL-BFM 95.

Author

Möricke, Anja, primary, Reiter, Alfred, additional, Zimmermann, Martin, additional, Gadner, Helmut, additional, Stanulla, Martin, additional, Ludwig, Wolf-Dieter, additional, Harbott, Jochen, additional, Mann, Georg, additional, Niggli, Felix, additional, Henze, Günter, additional, Welte, Karl, additional, Klingebiel, Thomas, additional, Niemeyer, Charlotte, additional, Zintl, Felix, additional, Riehm, Hansjörg, additional, and Schrappe, Martin, additional

Published
2007
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