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1. Busulfan–fludarabine- or treosulfan–fludarabine-based conditioning before allogeneic HSCT from matched sibling donors in paediatric patients with sickle cell disease: A study on behalf of the EBMT Paediatric Diseases and Inborn Errors Working Parties

Author

Cseh, A, Galimard, J, de la Fuente, J, Isgro, A, Zecca, M, Garwer, B, Biffi, A, Aljurf, M, Sundin, M, Belendez, C, Locatelli, F, Balduzzi, A, Lawson, S, Sengeloev, H, Ifversen, M, Saccardi, R, Wynn, R, Lankester, A, Corbacioglu, S, Peters, C, Cseh A., Galimard J. -E., de la Fuente J., Isgro A., Zecca M., Garwer B., Biffi A., Aljurf M., Sundin M., Belendez C., Locatelli F., Balduzzi A., Lawson S., Sengeloev H., Ifversen M., Saccardi R., Wynn R., Lankester A. C., Corbacioglu S., Peters C., Cseh, A, Galimard, J, de la Fuente, J, Isgro, A, Zecca, M, Garwer, B, Biffi, A, Aljurf, M, Sundin, M, Belendez, C, Locatelli, F, Balduzzi, A, Lawson, S, Sengeloev, H, Ifversen, M, Saccardi, R, Wynn, R, Lankester, A, Corbacioglu, S, Peters, C, Cseh A., Galimard J. -E., de la Fuente J., Isgro A., Zecca M., Garwer B., Biffi A., Aljurf M., Sundin M., Belendez C., Locatelli F., Balduzzi A., Lawson S., Sengeloev H., Ifversen M., Saccardi R., Wynn R., Lankester A. C., Corbacioglu S., and Peters C.

Published
2024

5. Analysis of Uncertainty in Calibration Curves

Author

Machado, V., Sundin, M. S., MAGJAREVIC, Ratko, Editor-in-chief, Ładyzynsk, Piotr, Series editor, Ibrahim, Fatimah, Series editor, Lacković, Igor, Series editor, Rock, Emilio Sacristan, Series editor, Braidot, Ariel, editor, and Hadad, Alejandro, editor

Published
2015
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6. Ethylene Oxide Residues in Gelfoam

Author

Machado, V., Sundin, M. S., MAGJAREVIC, Ratko, Editor-in-chief, Ładyzynsk, Piotr, Series editor, Ibrahim, Fatimah, Series editor, Lacković, Igor, Series editor, Rock, Emilio Sacristan, Series editor, Braidot, Ariel, editor, and Hadad, Alejandro, editor

Published
2015
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8. Busulfan–fludarabine- or treosulfan–fludarabine-based conditioning before allogeneic HSCT from matched sibling donors in paediatric patients with sickle cell disease: A study on behalf of the EBMT Paediatric Diseases and Inborn Errors Working Parties

Author

Cseh, A., Galimard, J. -E., de la Fuente, J., Isgro, A., Zecca, M., Garwer, B., Biffi, A., Aljurf, M., Sundin, M., Belendez, C., Locatelli, Franco, Balduzzi, A., Lawson, S., Sengeloev, H., Ifversen, M., Saccardi, R., Wynn, R., Lankester, A. C., Corbacioglu, S., Peters, C., Locatelli F. (ORCID:0000-0002-7976-3654), Cseh, A., Galimard, J. -E., de la Fuente, J., Isgro, A., Zecca, M., Garwer, B., Biffi, A., Aljurf, M., Sundin, M., Belendez, C., Locatelli, Franco, Balduzzi, A., Lawson, S., Sengeloev, H., Ifversen, M., Saccardi, R., Wynn, R., Lankester, A. C., Corbacioglu, S., Peters, C., and Locatelli F. (ORCID:0000-0002-7976-3654)

Abstract

How important is choice of conditioning regimen in allogeneic haematopoietic stem cell transplantation (HSCT) for sickle cell disease (SCD)? We compared HSCT outcomes by conditioning regimen in paediatric patients with SCD from the EBMT registry. In 2010-2020, 251 patients aged <18 years underwent a first matched sibling donor (MSD) HSCT with conditioning based on busulfan-fludarabine (bu-flu; n = 89) or treosulfan-fludarabine (treo-flu; n = 162). In the bu-flu and treo-flu groups, 51.7% and 99.4% of patients, respectively, received thiotepa. Median follow-up was 2.7 years. Two-year overall survival (OS) was 98.7% (95% confidence interval [CI]: 90.9-99.8) with bu-flu and 99.3% (95% CI: 95.2-99.9) with treo-flu (p = 0.63). Grade III-IV acute graft-versus-host disease (GVHD) at 100 days was 2.4% (95% CI: 0.4-7.5) and 0.6% (0.1%-3.2%) for bu-flu and treo-flu respectively (p = 0.25). The 2-year incidence of extensive chronic GVHD was 1.5% (95% CI: 0.1-7.3) with bu-flu and 8.0% (95% CI: 4.1-13.3) with treo-flu (p = 0.057). These multinational data confirm the excellent curative capacity of MSD HSCT with myeloablative conditioning. Both conditioning regimens yielded excellent OS, low rates of acute and chronic GVHD, and low rates of graft failure.

Published
2023

9. Correction: Myeloablative conditioning for allo-HSCT in pediatric ALL: FTBI or chemotherapy?—A multicenter EBMT-PDWP study (Bone Marrow Transplantation, (2020), 55, 8, (1540-1551), 10.1038/s41409-020-0854-0)

Author

Willasch A. M., Willasch, A, Peters, C, Sedlacek, P, Dalle, J, Kitra-Roussou, V, Yesilipek, A, Wachowiak, J, Lankester, A, Prete, A, Hamidieh, A, Ifversen, M, Buechner, J, Krivan, G, Hamladji, R, Diaz-de-Heredia, C, Skorobogatova, E, Michel, G, Locatelli, F, Bertaina, A, Veys, P, Dupont, S, Or, R, Gungor, T, Aleinikova, O, Sufliarska, S, Sundin, M, Rascon, J, Kaare, A, Nemet, D, Fagioli, F, Klingebiel, T, Styczynski, J, Bierings, M, Nagy, K, Abecasis, M, Afanasyev, B, Ansari, M, Vettenranta, K, Alseraihy, A, Chybicka, A, Robinson, S, Bertrand, Y, Kupesiz, A, Ghavamzadeh, A, Campos, A, Pichler, H, Dalissier, A, Labopin, M, Corbacioglu, S, Balduzzi, A, Galimard, J, Bader, P, Willasch A. M., Peters C., Sedlacek P., Dalle J. -H., Kitra-Roussou V., Yesilipek A., Wachowiak J., Lankester A., Prete A., Hamidieh A. A., Ifversen M., Buechner J., Krivan G., Hamladji R. -M., Diaz-de-Heredia C., Skorobogatova E., Michel G., Locatelli F., Bertaina A., Veys P., Dupont S., Or R., Gungor T., Aleinikova O., Sufliarska S., Sundin M., Rascon J., Kaare A., Nemet D., Fagioli F., Klingebiel T. E., Styczynski J., Bierings M., Nagy K., Abecasis M., Afanasyev B., Ansari M., Vettenranta K., Alseraihy A., Chybicka A., Robinson S., Bertrand Y., Kupesiz A., Ghavamzadeh A., Campos A., Pichler H., Dalissier A., Labopin M., Corbacioglu S., Balduzzi A., Galimard J. -E., Bader P., Willasch A. M., Willasch, A, Peters, C, Sedlacek, P, Dalle, J, Kitra-Roussou, V, Yesilipek, A, Wachowiak, J, Lankester, A, Prete, A, Hamidieh, A, Ifversen, M, Buechner, J, Krivan, G, Hamladji, R, Diaz-de-Heredia, C, Skorobogatova, E, Michel, G, Locatelli, F, Bertaina, A, Veys, P, Dupont, S, Or, R, Gungor, T, Aleinikova, O, Sufliarska, S, Sundin, M, Rascon, J, Kaare, A, Nemet, D, Fagioli, F, Klingebiel, T, Styczynski, J, Bierings, M, Nagy, K, Abecasis, M, Afanasyev, B, Ansari, M, Vettenranta, K, Alseraihy, A, Chybicka, A, Robinson, S, Bertrand, Y, Kupesiz, A, Ghavamzadeh, A, Campos, A, Pichler, H, Dalissier, A, Labopin, M, Corbacioglu, S, Balduzzi, A, Galimard, J, Bader, P, Willasch A. M., Peters C., Sedlacek P., Dalle J. -H., Kitra-Roussou V., Yesilipek A., Wachowiak J., Lankester A., Prete A., Hamidieh A. A., Ifversen M., Buechner J., Krivan G., Hamladji R. -M., Diaz-de-Heredia C., Skorobogatova E., Michel G., Locatelli F., Bertaina A., Veys P., Dupont S., Or R., Gungor T., Aleinikova O., Sufliarska S., Sundin M., Rascon J., Kaare A., Nemet D., Fagioli F., Klingebiel T. E., Styczynski J., Bierings M., Nagy K., Abecasis M., Afanasyev B., Ansari M., Vettenranta K., Alseraihy A., Chybicka A., Robinson S., Bertrand Y., Kupesiz A., Ghavamzadeh A., Campos A., Pichler H., Dalissier A., Labopin M., Corbacioglu S., Balduzzi A., Galimard J. -E., and Bader P.

Abstract

The article “Myeloablative conditioning for allo-HSCT in pediatric ALL: FTBI or chemotherapy?—A multicenter EBMT-PDWP study,” written by Andre Manfred Willasch, Christina Peters, Petr Sedlácek, Jean-Hugues Dalle, Vassiliki Kitra-Roussou, Akif Yesilipek, Jacek Wachowiak, Arjan Lankester, Arcangelo Prete, Amir Ali Hamidieh, Marianne Ifversen, Jochen Buechner, Gergely Kriván, Rose-Marie Hamladji, Cristina Diaz-de-Heredia, Elena Skorobogatova, Gérard Michel, Franco Locatelli, Alice Bertaina, Paul Veys, Sophie Dupont, Reuven Or, Tayfun Güngör, Olga Aleinikova, Sabina Sufliarska, Mikael Sundin, Jelena Rascon, Ain Kaare, Damir Nemet, Franca Fagioli, Thomas Erich Klingebiel, Jan Styczynski, Marc Bierings, Kálmán Nagy, Manuel Abecasis, Boris Afanasyev, Marc Ansari, Kim Vettenranta, Amal Alseraihy, Alicja Chybicka, Stephen Robinson, Yves Bertrand, Alphan Kupesiz, Ardeshir Ghavamzadeh, Antonio Campos, Herbert Pichler, Arnaud Dalissier, Myriam Labopin, Selim Corbacioglu, Adriana Balduzzi, Jacques-Emmanuel Galimard, Peter Bader, on behalf of the EBMT Paediatric Diseases Working Party, was originally published online first without Open Access. After publication in volume 55, issue 8, page 1540–1551, the author decided to opt for Open Choice and to make the article an Open Access publication. Therefore, the copyright of the article has been changed to © The Author(s) 2020 and the article is forthwith distributed under the terms of the Creative Commons Attribution 4.0 InternationalS License, which permits use, sharing, adaptation, distribution, and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. The images or other third-party material in this article are included in the article’s Creative Commons license, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Comm

Published
2021

14. Correction: Myeloablative conditioning for allo-HSCT in pediatric ALL: FTBI or chemotherapy?—A multicenter EBMT-PDWP study (Bone Marrow Transplantation, (2020), 55, 8, (1540-1551), 10.1038/s41409-020-0854-0)

Author

Willasch, A. M., Peters, C., Sedlacek, P., Dalle, J. -H., Kitra-Roussou, V., Yesilipek, A., Wachowiak, J., Lankester, A., Prete, A., Hamidieh, A. A., Ifversen, M., Buechner, J., Krivan, G., Hamladji, R. -M., Diaz-de-Heredia, C., Skorobogatova, E., Michel, G., Locatelli, F., Bertaina, A., Veys, P., Dupont, S., Or, R., Gungor, T., Aleinikova, O., Sufliarska, S., Sundin, M., Rascon, J., Kaare, A., Nemet, D., Fagioli, F., Klingebiel, T. E., Styczynski, J., Bierings, M., Nagy, K., Abecasis, M., Afanasyev, B., Ansari, M., Vettenranta, K., Alseraihy, A., Chybicka, A., Robinson, S., Bertrand, Y., Kupesiz, A., Ghavamzadeh, A., Campos, A., Pichler, H., Dalissier, A., Labopin, M., Corbacioglu, S., Balduzzi, A., Galimard, J. -E., and Bader, P.

Subjects
Settore MED/38 - PEDIATRIA GENERALE E SPECIALISTICA, conditioning regimens
Published
2021

15. Outcomes of Unmanipulated Haploidentical Transplantation Using Post-Transplant Cyclophosphamide (PT-Cy) in Pediatric Patients With Acute Lymphoblastic Leukemia

Author

Ruggeri, A., Galimard, J. -E., Paina, O., Fagioli, F., Tbakhi, A., Yesilipek, A., Navarro, J. M. F., Faraci, M., Hamladji, R. -M., Skorobogatova, E., Al-Seraihy, A., Sundin, M., Herrera, C., Rifon, J., Dalissier, A., Locatelli, Franco, Rocha, V., Corbacioglu, S., Locatelli F. (ORCID:0000-0002-7976-3654), Ruggeri, A., Galimard, J. -E., Paina, O., Fagioli, F., Tbakhi, A., Yesilipek, A., Navarro, J. M. F., Faraci, M., Hamladji, R. -M., Skorobogatova, E., Al-Seraihy, A., Sundin, M., Herrera, C., Rifon, J., Dalissier, A., Locatelli, Franco, Rocha, V., Corbacioglu, S., and Locatelli F. (ORCID:0000-0002-7976-3654)

Abstract

HLA-haploidentical transplantation (haplo-HCT) using post-transplantation-cyclophosphamide (PT-Cy) is a feasible procedure in children with malignancies. However, large studies on Haplo-HCT with PT-Cy for childhood acute lymphoblastic leukemia (ALL) are lacking. We analyzed haplo-HCT outcomes in 180 children with ALL. Median age was 9 years, and median follow-up was 2.7 years. Disease status was CR1 for 24%, CR2 for 45%, CR+3 for 12%, and active disease for 19%. All patients received PT-Cy day +3 and +4. Bone marrow (BM) was the stem cell source in 115 patients (64%). Cumulative incidence of 42-day engraftment was 88.9%. Cumulative incidence of day-100 acute graft-versus-host disease (GVHD) grade II-IV was 28%, and 2-year chronic GVHD was 21.9%. At 2 years, cumulative incidence of nonrelapse mortality (NRM) was 19.6%. Cumulative incidence was 41.9% for relapse and 25% for patients in CR1. Estimated 2-year leukemia free survival was 65%, 44%, and 18.8% for patients transplanted in CR1, CR2, CR3+ and 3% at 1 year for active disease. In multivariable analysis for patients in CR1 and CR2, disease status (CR2 [hazard ratio {HR} = 2.19; P = .04]), age at HCT older than 13 (HR = 2.07; P = .03) and use of peripheral blood stem cell (PBSC) (HR = 1.98; P = .04) were independent factors associated with decreased overall survival. Use of PBSC was also associated with higher NRM (HR = 3.13; P = .04). Haplo-HCT with PT-Cy is an option for children with ALL, namely those transplanted in CR1 and CR2. Age and disease status remain the most important factors for outcomes. BM cells as a graft source is associated with improved survival.

Published
2021

17. The PedPAD study: boys predominate in the hypogammaglobulinaemia registry of the ESID online database

Author

Schatorjé, E. J. H., Gathmann, B., van Hout, R. W. N. M., de Vries, E., Alsina, L, Baumann, U, Belohradsky, B H, Bienemann, K, Boardman, B, Borte, M, Bredius, R G, Brodszki, N, Caracseghi, F, Ciznar, P, de Vries, E, Driessen, G J, Dückers, G, Duppenthaler, A, Farmaki, E, Galal, N, Gennery, A, Gonzalez-Granado, L I, Hlavackova, E, Hoernes, M, Kilic, S S, Krüger, R, Kuijpers, T W, Kütükcüler, N, Llobet, P, Marques, L, van Montfrans, J M, Papadopoulou-Alataki, E, Paschenko, O, Pasic, S, Pietrogrande, M C, Pignata, C, Reda, S M, Reisli, I, Roesler, J, Santos, J L, Schölvinck, E H, Schulze, Ilka, Seidel, M G, Shcherbina, A, Sundin, M, Szaflarska, A, Velbri, S, Warnatz, K, and Warris, A

Published
2014
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19. Hematopoietic stem cell transplantation for CD40 ligand deficiency : Results from an EBMT/ESID-IEWP-SCETIDE-PIDTC study

Author

Ferrua, F., Galimberti, S., Courteille, V., Slatter, M.A., Booth, C., Moshous, D., Neven, B., Blanche, S., Cavazzana, M., Laberko, A., Shcherbina, A., Balashov, D., Soncini, E., Porta, F., Al-Mousa, H., Al-Saud, B., Al-Dhekri, H., Arnaout, R., Formankova, R., Bertrand, Y., Lange, A., Smart, J., Wolska-Kusnierz, B., Aquino, V.M., Dvorak, C.C., Fasth, A., Fouyssac, F., Heilmann, C., Hoenig, M., Schuetz, C., Kelecic, J., Bredius, R.G.M., Lankester, A.C., Lindemans, C.A., Suarez, F., Sullivan, K.E., Albert, M.H., Kalwak, K., Barlogis, V., Bhatia, M., Bordon, V., Czogala, W., Alonso, L., Dogu, F., Gozdzik, J., Ikinciogullari, A., Krivan, G., Ljungman, P., Meyts, I., Mustillo, P., Smith, A.R., Speckmann, C., Sundin, M., Keogh, S.J., Shaw, P.J., Boelens, J.J., Schulz, A.S., Sedlacek, P., Veys, P., Mahlaoui, N., Janda, A., Davies, E.G., Fischer, A., Cowan, M.J., Gennery, A.R., SCETIDE, PIDTC, EBMT, ESID IEWP, Ferrua, F, Galimberti, S, Courteille, V, Slatter, M, Booth, C, Moshous, D, Neven, B, Blanche, S, Laberko, A, Shcherbina, A, Balashov, D, Soncini, E, Porta, F, Al-Mousa, H, Al-Saud, B, Al-Dhekri, H, Arnaout, R, Formankova, R, Bertrand, Y, Lange, A, Smart, J, Wolska-Kusnierz, B, Aquino, V, Dvorak, C, Fasth, A, Fouyssac, F, Heilmann, C, Hoenig, M, Schuetz, C, Kelečić, J, Bredius, R, Lankester, A, Lindemans, C, Suarez, F, Sullivan, K, Albert, M, Kałwak, K, Barlogis, V, Bhatia, M, Bordon, V, Czogala, W, Alonso, L, Dogu, F, Gozdzik, J, Ikinciogullari, A, Kriván, G, Ljungman, P, Meyts, I, Mustillo, P, Smith, A, Speckmann, C, Sundin, M, Keogh, S, Shaw, P, Boelens, J, Schulz, A, Sedlacek, P, Veys, P, Mahlaoui, N, Janda, A, Davies, E, Fischer, A, Cowan, M, and Gennery, A

Subjects
0301 basic medicine, Oncology, medicine.medical_specialty, medicine.medical_treatment, Immunology, Hematopoietic stem cell transplantation, X-Linked Combined Immunodeficiency Diseases, primary immunodeficiency, Donor lymphocyte infusion, 03 medical and health sciences, 0302 clinical medicine, Internal medicine, X-linked hyper-IgM syndrome, Humans, Medicine, Immunology and Allergy, Risk factor, Child, Prospective cohort study, business.industry, Infant, Newborn, Infant, medicine.disease, Treatment Outcome, 030104 developmental biology, medicine.anatomical_structure, Graft-versus-host disease, surgical procedures, operative, Supportive psychotherapy, Child, Preschool, hematopoietic stem cell transplantation, Primary immunodeficiency, Bone marrow, CD40 ligand, business, 030215 immunology
Abstract

BACKGROUND: CD40 ligand (CD40L) deficiency, an X-linked primary immunodeficiency, causes recurrent sinopulmonary, Pneumocystis and Cryptosporidium species infections. Long-term survival with supportive therapy is poor. Currently, the only curative treatment is hematopoietic stem cell transplantation (HSCT). OBJECTIVE: We performed an international collaborative study to improve patients' management, aiming to individualize risk factors and determine optimal HSCT characteristics. METHODS: We retrospectively collected data on 130 patients who underwent HSCT for CD40L deficiency between 1993-2015. We analyzed outcome and variables' relevance with respect to survival and cure. RESULTS: Overall survival (OS), event-free survival (EFS), and disease-free survival (DFS) were 78.2%, 58.1%, and 72.3% 5 years after HSCT. Results were better in transplantations performed in 2000 or later and in children less than 10 years old at the time of HSCT. Pre-existing organ damage negatively influenced outcome. Sclerosing cholangitis was the most important risk factor. After 2000, superior OS was achieved with matched donors. Use of myeloablative regimens and HSCT at 2 years or less from diagnosis associated with higher OS and DFS. EFS was best with matched sibling donors, myeloablative conditioning (MAC), and bone marrow-derived stem cells. Most rejections occurred after reduced-intensity or nonmyeloablative conditioning, which associated with poor donor cell engraftment. Mortality occurred mainly early after HSCT, predominantly from infections. Among survivors who ceased immunoglobulin replacement, T-lymphocyte chimerism was 50% or greater donor in 85.2%. CONCLUSION: HSCT is curative in patients with CD40L deficiency, with improved outcome if performed before organ damage development. MAC is associated with better OS, EFS, and DFS. Prospective studies are required to compare the risks of HSCT with those of lifelong supportive therapy. ispartof: JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY vol:143 issue:6 pages:2238-2253 ispartof: location:United States status: published

Published
2019

21. Myeloablative conditioning for allo-HSCT in pediatric ALL: FTBI or chemotherapy?—A multicenter EBMT-PDWP study

Author

Willasch, A. M., Peters, C., Sedlacek, P., Dalle, J. -H., Kitra-Roussou, V., Yesilipek, A., Wachowiak, J., Lankester, A., Prete, A., Hamidieh, A. A., Ifversen, M., Buechner, J., Krivan, G., Hamladji, R. -M., Diaz-de-Heredia, C., Skorobogatova, E., Michel, G., Locatelli, Franco, Bertaina, A., Veys, P., Dupont, S., Or, R., Gungor, T., Aleinikova, O., Sufliarska, S., Sundin, M., Rascon, J., Kaare, A., Nemet, D., Fagioli, F., Klingebiel, T. E., Styczynski, J., Bierings, M., Nagy, K., Abecasis, M., Afanasyev, B., Ansari, M., Vettenranta, K., Alseraihy, A., Chybicka, A., Robinson, S., Bertrand, Y., Kupesiz, A., Ghavamzadeh, A., Campos, A., Pichler, H., Dalissier, A., Labopin, M., Corbacioglu, S., Balduzzi, A., Galimard, J. -E., Bader, P., Locatelli F. (ORCID:0000-0002-7976-3654), Willasch, A. M., Peters, C., Sedlacek, P., Dalle, J. -H., Kitra-Roussou, V., Yesilipek, A., Wachowiak, J., Lankester, A., Prete, A., Hamidieh, A. A., Ifversen, M., Buechner, J., Krivan, G., Hamladji, R. -M., Diaz-de-Heredia, C., Skorobogatova, E., Michel, G., Locatelli, Franco, Bertaina, A., Veys, P., Dupont, S., Or, R., Gungor, T., Aleinikova, O., Sufliarska, S., Sundin, M., Rascon, J., Kaare, A., Nemet, D., Fagioli, F., Klingebiel, T. E., Styczynski, J., Bierings, M., Nagy, K., Abecasis, M., Afanasyev, B., Ansari, M., Vettenranta, K., Alseraihy, A., Chybicka, A., Robinson, S., Bertrand, Y., Kupesiz, A., Ghavamzadeh, A., Campos, A., Pichler, H., Dalissier, A., Labopin, M., Corbacioglu, S., Balduzzi, A., Galimard, J. -E., Bader, P., and Locatelli F. (ORCID:0000-0002-7976-3654)

Abstract

Although most children with acute lymphoblastic leukemia (ALL) receive fractionated total body irradiation (FTBI) as myeloablative conditioning (MAC) for allogeneic hematopoietic stem cell transplantation (allo-HSCT), it is an important matter of debate if chemotherapy can effectively replace FTBI. To compare outcomes after FTBI versus chemotherapy-based conditioning (CC), we performed a retrospective EBMT registry study. Children aged 2–18 years after MAC for first allo-HSCT of bone marrow (BM) or peripheral blood stem cells (PBSC) from matched-related (MRD) or unrelated donors (UD) in first (CR1) or second remission (CR2) between 2000 and 2012 were included. Propensity score weighting was used to control pretreatment imbalances of the observed variables. 3.054 patients were analyzed. CR1 (1.498): median follow-up (FU) after FTBI (1.285) and CC (213) was 6.8 and 6.1 years. Survivals were not significantly different. CR2 (1.556): median FU after FTBI (1.345) and CC (211) was 6.2 years. Outcomes after FTBI were superior as compared with CC with regard to overall survival (OS), leukemia-free survival (LFS), relapse incidence (RI), and nonrelapse mortality (NRM). However, we must emphasize the preliminary character of the results of this retrospective “real-world-practice” study. These findings will be prospectively assessed in the ALL SCTped 2012 FORUM trial.

Published
2020

22. Myeloablative conditioning for allo-HSCT in pediatric ALL: FTBI or chemotherapy?—A multicenter EBMT-PDWP study

Author

Willasch, A, Peters, C, Sedláček, P, Dalle, J, Kitra-Roussou, V, Yesilipek, A, Wachowiak, J, Lankester, A, Prete, A, Hamidieh, A, Ifversen, M, Buechner, J, Kriván, G, Hamladji, R, Diaz-de-Heredia, C, Skorobogatova, E, Michel, G, Locatelli, F, Bertaina, A, Veys, P, Dupont, S, Or, R, Güngör, T, Aleinikova, O, Sufliarska, S, Sundin, M, Rascon, J, Kaare, A, Nemet, D, Fagioli, F, Klingebiel, T, Styczynski, J, Bierings, M, Nagy, K, Abecasis, M, Afanasyev, B, Ansari, M, Vettenranta, K, Alseraihy, A, Chybicka, A, Robinson, S, Bertrand, Y, Kupesiz, A, Ghavamzadeh, A, Campos, A, Pichler, H, Dalissier, A, Labopin, M, Corbacioglu, S, Balduzzi, A, Galimard, J, Bader, P, Willasch, Andre Manfred, Peters, Christina, Sedláček, Petr, Dalle, Jean-Hugues, Kitra-Roussou, Vassiliki, Yesilipek, Akif, Wachowiak, Jacek, Lankester, Arjan, Prete, Arcangelo, Hamidieh, Amir Ali, Ifversen, Marianne, Buechner, Jochen, Kriván, Gergely, Hamladji, Rose-Marie, Diaz-de-Heredia, Cristina, Skorobogatova, Elena, Michel, Gérard, Locatelli, Franco, Bertaina, Alice, Veys, Paul, Dupont, Sophie, Or, Reuven, Güngör, Tayfun, Aleinikova, Olga, Sufliarska, Sabina, Sundin, Mikael, Rascon, Jelena, Kaare, Ain, Nemet, Damir, Fagioli, Franca, Klingebiel, Thomas Erich, Styczynski, Jan, Bierings, Marc, Nagy, Kálmán, Abecasis, Manuel, Afanasyev, Boris, Ansari, Marc, Vettenranta, Kim, Alseraihy, Amal, Chybicka, Alicja, Robinson, Stephen, Bertrand, Yves, Kupesiz, Alphan, Ghavamzadeh, Ardeshir, Campos, Antonio, Pichler, Herbert, Dalissier, Arnaud, Labopin, Myriam, Corbacioglu, Selim, Balduzzi, Adriana, Galimard, Jacques-Emmanuel, Bader, Peter, Willasch, A, Peters, C, Sedláček, P, Dalle, J, Kitra-Roussou, V, Yesilipek, A, Wachowiak, J, Lankester, A, Prete, A, Hamidieh, A, Ifversen, M, Buechner, J, Kriván, G, Hamladji, R, Diaz-de-Heredia, C, Skorobogatova, E, Michel, G, Locatelli, F, Bertaina, A, Veys, P, Dupont, S, Or, R, Güngör, T, Aleinikova, O, Sufliarska, S, Sundin, M, Rascon, J, Kaare, A, Nemet, D, Fagioli, F, Klingebiel, T, Styczynski, J, Bierings, M, Nagy, K, Abecasis, M, Afanasyev, B, Ansari, M, Vettenranta, K, Alseraihy, A, Chybicka, A, Robinson, S, Bertrand, Y, Kupesiz, A, Ghavamzadeh, A, Campos, A, Pichler, H, Dalissier, A, Labopin, M, Corbacioglu, S, Balduzzi, A, Galimard, J, Bader, P, Willasch, Andre Manfred, Peters, Christina, Sedláček, Petr, Dalle, Jean-Hugues, Kitra-Roussou, Vassiliki, Yesilipek, Akif, Wachowiak, Jacek, Lankester, Arjan, Prete, Arcangelo, Hamidieh, Amir Ali, Ifversen, Marianne, Buechner, Jochen, Kriván, Gergely, Hamladji, Rose-Marie, Diaz-de-Heredia, Cristina, Skorobogatova, Elena, Michel, Gérard, Locatelli, Franco, Bertaina, Alice, Veys, Paul, Dupont, Sophie, Or, Reuven, Güngör, Tayfun, Aleinikova, Olga, Sufliarska, Sabina, Sundin, Mikael, Rascon, Jelena, Kaare, Ain, Nemet, Damir, Fagioli, Franca, Klingebiel, Thomas Erich, Styczynski, Jan, Bierings, Marc, Nagy, Kálmán, Abecasis, Manuel, Afanasyev, Boris, Ansari, Marc, Vettenranta, Kim, Alseraihy, Amal, Chybicka, Alicja, Robinson, Stephen, Bertrand, Yves, Kupesiz, Alphan, Ghavamzadeh, Ardeshir, Campos, Antonio, Pichler, Herbert, Dalissier, Arnaud, Labopin, Myriam, Corbacioglu, Selim, Balduzzi, Adriana, Galimard, Jacques-Emmanuel, and Bader, Peter

Abstract

Although most children with acute lymphoblastic leukemia (ALL) receive fractionated total body irradiation (FTBI) as myeloablative conditioning (MAC) for allogeneic hematopoietic stem cell transplantation (allo-HSCT), it is an important matter of debate if chemotherapy can effectively replace FTBI. To compare outcomes after FTBI versus chemotherapy-based conditioning (CC), we performed a retrospective EBMT registry study. Children aged 2-18 years after MAC for first allo-HSCT of bone marrow (BM) or peripheral blood stem cells (PBSC) from matched-related (MRD) or unrelated donors (UD) in first (CR1) or second remission (CR2) between 2000 and 2012 were included. Propensity score weighting was used to control pretreatment imbalances of the observed variables. 3.054 patients were analyzed. CR1 (1.498): median follow-up (FU) after FTBI (1.285) and CC (213) was 6.8 and 6.1 years. Survivals were not significantly different. CR2 (1.556): median FU after FTBI (1.345) and CC (211) was 6.2 years. Outcomes after FTBI were superior as compared with CC with regard to overall survival (OS), leukemia-free survival (LFS), relapse incidence (RI), and nonrelapse mortality (NRM). However, we must emphasize the preliminary character of the results of this retrospective "real-world-practice" study. These findings will be prospectively assessed in the ALL SCTped 2012 FORUM trial.

Published
2020

28. Combined analysis of the ORCHID and OCAY studies: Does eradication of Helicobacter pylori lead to sustained improvement in functional dyspepsia symptoms? (Dyspepsia Management)

Author

Zanten, S.J.O. Veldhuyzen van, Talley, N.J., Blum, A.L., Bolling-Sternevald, E., Sundin, M., and Junghard, O.

Subjects
Helicobacter infections -- Drug therapy -- Care and treatment, Indigestion -- Care and treatment -- Drug therapy, Health, Drug therapy, Care and treatment
Abstract

Currently, one of the most important unresolved questions concerning Helicobacter pylori is whether eradication of the organism leads to a sustained improvement in symptoms in patients diagnosed with functional (non-ulcer) [...]

Published
2002

31. Gonadal Function after Busulfan Compared with Treosulfan in Children and Adolescents Undergoing Allogeneic Hematopoietic Stem Cell Transplant

Author

Faraci, M., Diesch, T., Labopin, M., Dalissier, A., Lankester, A., Gennery, A., Sundin, M., Uckan-Cetinkaya, D., Bierings, M., Peters, A.M.J., Garwer, M., Schulz, A., Michel, G., Giorgiani, G., Gruhn, B., Locatelli, F., Giardino, S., Uyttebroeck, A., Rialland, F., Itala-Remes, M., Dreger, P., Shaw, P.J., Bordon, V., Schlegel, P.G., Mellgren, K., Moraleda, J.M., Patrick, K., Schneider, P., Jubert, C., Lawitschka, A., Salooja, N., Basak, G.W., Corbacioglu, S., Duarte, R., Bader, P., and Pediat Transplant Complications Wo

Subjects
Treo, Adult, Male, Pediatrics, medicine.medical_specialty, Cyclophosphamide, Adolescent, Puberty, Precocious, Treosulfan, medicine, Humans, pubertal stage, Child, Gonads, Busulfan, Retrospective Studies, Transplantation, business.industry, Late effect, Hematopoietic Stem Cell Transplantation, gonadal damage, Hematology, Allografts, Settore MED/38 - PEDIATRIA GENERALE E SPECIALISTICA, Prepubertal stage, Child, Preschool, Female, busulfan, treosulfan, Transplantation Conditioning, medicine.symptom, business, Chemoradiotherapy, medicine.drug
Abstract

Gonadal impairment is an important late effect with a significant impact on quality of life of transplanted patients. The aim of this study was to compare gonadal function after busulfan (Bu) or treosulfan (Treo) conditioning regimens in pre- and postpubertal children. This retrospective, multicenter study included children transplanted in pediatric European Society for Blood and Marrow Transplantation (EBMT) centers between 1992 and 2012 who did not receive gonadotoxic chemoradiotherapy before the transplant. We evaluated 137 patients transplanted in 25 pediatric EBMT centers. Median age at transplant was 11.04 years (range, 5 to 18); 89 patients were boys and 48 girls. Eighty-nine patients were prepubertal at transplant and 48 postpubertal. One hundred eighteen children received Bu and 19 Treo. A higher proportion of girls treated with Treo in the prepubertal stage reached spontaneous puberty compared with those treated with Bu (P = .02). Spontaneous menarche was more frequent after Treo than after Bu (P < .001). Postpubertal boys and girls treated with Treo had significantly lower luteinizing hormone levels (P = .03 and P = .04, respectively) compared with the Bu group. Frequency of gonadal damage associated with Treo was significantly lower than that observed after Bu. These results need to be confirmed in a larger population.

Published
2019

34. Hematopoietic stem cell transplantation for CD40 ligand deficiency: Results from an EBMT/ESID-IEWP-SCETIDE-PIDTC study

Author

Ferrua, F, Galimberti, S, Courteille, V, Slatter, M, Booth, C, Moshous, D, Neven, B, Blanche, S, Laberko, A, Shcherbina, A, Balashov, D, Soncini, E, Porta, F, Al-Mousa, H, Al-Saud, B, Al-Dhekri, H, Arnaout, R, Formankova, R, Bertrand, Y, Lange, A, Smart, J, Wolska-Kusnierz, B, Aquino, V, Dvorak, C, Fasth, A, Fouyssac, F, Heilmann, C, Hoenig, M, Schuetz, C, Kelečić, J, Bredius, R, Lankester, A, Lindemans, C, Suarez, F, Sullivan, K, Albert, M, Kałwak, K, Barlogis, V, Bhatia, M, Bordon, V, Czogala, W, Alonso, L, Dogu, F, Gozdzik, J, Ikinciogullari, A, Kriván, G, Ljungman, P, Meyts, I, Mustillo, P, Smith, A, Speckmann, C, Sundin, M, Keogh, S, Shaw, P, Boelens, J, Schulz, A, Sedlacek, P, Veys, P, Mahlaoui, N, Janda, A, Davies, E, Fischer, A, Cowan, M, Gennery, A, Ferrua, Francesca, Galimberti, Stefania, Courteille, Virginie, Slatter, Mary Anne, Booth, Claire, Moshous, Despina, Neven, Benedicte, Blanche, Stephane, Laberko, Alexandra, Shcherbina, Anna, Balashov, Dmitry, Soncini, Elena, Porta, Fulvio, Al-Mousa, Hamoud, Al-Saud, Bandar, Al-Dhekri, Hasan, Arnaout, Rand, Formankova, Renata, Bertrand, Yves, Lange, Andrzej, Smart, Joanne, Wolska-Kusnierz, Beata, Aquino, Victor M, Dvorak, Christopher C, Fasth, Anders, Fouyssac, Fanny, Heilmann, Carsten, Hoenig, Manfred, Schuetz, Catharina, Kelečić, Jadranka, Bredius, Robbert G M, Lankester, Arjan C, Lindemans, Caroline A, Suarez, Felipe, Sullivan, Kathleen E, Albert, Michael H, Kałwak, Krzysztof, Barlogis, Vincent, Bhatia, Monica, Bordon, Victoria, Czogala, Wojciech, Alonso, Laura, Dogu, Figen, Gozdzik, Jolanta, Ikinciogullari, Aydan, Kriván, Gergely, Ljungman, Per, Meyts, Isabelle, Mustillo, Peter, Smith, Angela R, Speckmann, Carsten, Sundin, Mikael, Keogh, Steven John, Shaw, Peter John, Boelens, Jaap Jan, Schulz, Ansgar S, Sedlacek, Petr, Veys, Paul, Mahlaoui, Nizar, Janda, Ales, Davies, E Graham, Fischer, Alain, Cowan, Morton J, Gennery, Andrew Richard, Ferrua, F, Galimberti, S, Courteille, V, Slatter, M, Booth, C, Moshous, D, Neven, B, Blanche, S, Laberko, A, Shcherbina, A, Balashov, D, Soncini, E, Porta, F, Al-Mousa, H, Al-Saud, B, Al-Dhekri, H, Arnaout, R, Formankova, R, Bertrand, Y, Lange, A, Smart, J, Wolska-Kusnierz, B, Aquino, V, Dvorak, C, Fasth, A, Fouyssac, F, Heilmann, C, Hoenig, M, Schuetz, C, Kelečić, J, Bredius, R, Lankester, A, Lindemans, C, Suarez, F, Sullivan, K, Albert, M, Kałwak, K, Barlogis, V, Bhatia, M, Bordon, V, Czogala, W, Alonso, L, Dogu, F, Gozdzik, J, Ikinciogullari, A, Kriván, G, Ljungman, P, Meyts, I, Mustillo, P, Smith, A, Speckmann, C, Sundin, M, Keogh, S, Shaw, P, Boelens, J, Schulz, A, Sedlacek, P, Veys, P, Mahlaoui, N, Janda, A, Davies, E, Fischer, A, Cowan, M, Gennery, A, Ferrua, Francesca, Galimberti, Stefania, Courteille, Virginie, Slatter, Mary Anne, Booth, Claire, Moshous, Despina, Neven, Benedicte, Blanche, Stephane, Laberko, Alexandra, Shcherbina, Anna, Balashov, Dmitry, Soncini, Elena, Porta, Fulvio, Al-Mousa, Hamoud, Al-Saud, Bandar, Al-Dhekri, Hasan, Arnaout, Rand, Formankova, Renata, Bertrand, Yves, Lange, Andrzej, Smart, Joanne, Wolska-Kusnierz, Beata, Aquino, Victor M, Dvorak, Christopher C, Fasth, Anders, Fouyssac, Fanny, Heilmann, Carsten, Hoenig, Manfred, Schuetz, Catharina, Kelečić, Jadranka, Bredius, Robbert G M, Lankester, Arjan C, Lindemans, Caroline A, Suarez, Felipe, Sullivan, Kathleen E, Albert, Michael H, Kałwak, Krzysztof, Barlogis, Vincent, Bhatia, Monica, Bordon, Victoria, Czogala, Wojciech, Alonso, Laura, Dogu, Figen, Gozdzik, Jolanta, Ikinciogullari, Aydan, Kriván, Gergely, Ljungman, Per, Meyts, Isabelle, Mustillo, Peter, Smith, Angela R, Speckmann, Carsten, Sundin, Mikael, Keogh, Steven John, Shaw, Peter John, Boelens, Jaap Jan, Schulz, Ansgar S, Sedlacek, Petr, Veys, Paul, Mahlaoui, Nizar, Janda, Ales, Davies, E Graham, Fischer, Alain, Cowan, Morton J, and Gennery, Andrew Richard

Abstract

Background: CD40 ligand (CD40L) deficiency, an X-linked primary immunodeficiency, causes recurrent sinopulmonary, Pneumocystis and Cryptosporidium species infections. Long-term survival with supportive therapy is poor. Currently, the only curative treatment is hematopoietic stem cell transplantation (HSCT). Objective: We performed an international collaborative study to improve patients' management, aiming to individualize risk factors and determine optimal HSCT characteristics. Methods: We retrospectively collected data on 130 patients who underwent HSCT for CD40L deficiency between 1993-2015. We analyzed outcome and variables' relevance with respect to survival and cure. Results: Overall survival (OS), event-free survival (EFS), and disease-free survival (DFS) were 78.2%, 58.1%, and 72.3% 5 years after HSCT. Results were better in transplantations performed in 2000 or later and in children less than 10 years old at the time of HSCT. Pre-existing organ damage negatively influenced outcome. Sclerosing cholangitis was the most important risk factor. After 2000, superior OS was achieved with matched donors. Use of myeloablative regimens and HSCT at 2 years or less from diagnosis associated with higher OS and DFS. EFS was best with matched sibling donors, myeloablative conditioning (MAC), and bone marrow–derived stem cells. Most rejections occurred after reduced-intensity or nonmyeloablative conditioning, which associated with poor donor cell engraftment. Mortality occurred mainly early after HSCT, predominantly from infections. Among survivors who ceased immunoglobulin replacement, T-lymphocyte chimerism was 50% or greater donor in 85.2%. Conclusion: HSCT is curative in patients with CD40L deficiency, with improved outcome if performed before organ damage development. MAC is associated with better OS, EFS, and DFS. Prospective studies are required to compare the risks of HSCT with those of lifelong supportive therapy.

Published
2019

40. Spermatogonial quantity in human prepubertal testicular tissue collected for fertility preservation prior to potentially sterilizing therapy

Author

Stukenborg, J. -B., Alves-Lopes, J. P., Kurek, M., Albalushi, H., Reda, A., Keros, V., Töhönen, V., Bjarnason, R., Romerius, P., Sundin, M., Nyström, U. Noren, Langenskiöld, C., Vogt, H., Henningsohn, L., Mitchell, R. T., Söder, O., Petersen, C., Jahnukainen, K., Stukenborg, J-B [0000-0002-2839-1870], Alves-Lopes, JP [0000-0003-3042-6988], Reda, A [0000-0001-7053-7979], Apollo - University of Cambridge Repository, Læknadeild (HÍ), Faculty of Medicine (UI), Heilbrigðisvísindasvið (HÍ), School of Health Sciences (UI), Háskóli Íslands, University of Iceland, Children's Hospital, Clinicum, University of Helsinki, and HUS Children and Adolescents

Subjects
Male, SICKLE-CELL-DISEASE, fertility preservation, CHILDHOOD, CHILDREN, Reproduktionsmedicin och gynekologi, Anemia, Sickle Cell, Cohort Studies, 3123 Gynaecology and paediatrics, Obstetrics, Gynecology and Reproductive Medicine, Neoplasms, ADOLESCENTS, alkylating agents, Testis, Chemotherapy, childhood cancer, Humans, Hydroxyurea, Child, Antineoplastic Agents, Alkylating, Krabbamein, CANCER-TREATMENT, Cryopreservation, Cancer och onkologi, spermatogonial quantity, Radiotherapy, TRANSPLANTATION, Fertility Preservation, Spermatogonia, sickle cell disease, Cancer and Oncology, Case-Control Studies, Child, Preschool, WORKING PARTY, Ófrjósemi, EBMT
Abstract

Publisher's version (útgefin grein), STUDY QUESTION: Does chemotherapy exposure (with or without alkylating agents) or primary diagnosis affect spermatogonial quantity in human prepubertal testicular tissue? SUMMARY ANSWER: Spermatogonial quantity is significantly reduced in testes of prepubertal boys treated with alkylating agent therapies or with hydroxyurea for sickle cell disease. WHAT IS KNOWN ALREADY: Cryopreservation of spermatogonial stem cells, followed by transplantation into the testis after treatment, is a proposed clinical option for fertility restoration in children. The key clinical consideration behind this approach is a sufficient quantity of healthy cryopreserved spermatogonia. However, since most boys with malignancies start therapy with agents that are not potentially sterilizing, they will have already received some chemotherapy before testicular tissue cryopreservation is considered. STUDY DESIGN, SIZE, DURATION: We examined histological sections of prepubertal testicular tissue to elucidate whether chemotherapy exposure or primary diagnosis affects spermatogonial quantity. Quantity of spermatogonia per transverse tubular cross-section (S/T) was assessed in relation to treatment characteristics and normative reference values in histological sections of paraffin embedded testicular tissue samples collected from 32 consecutive boy patients (aged 6.3 ± 3.8 [mean ± SD] years) between 2014 and 2017, as part of the NORDFERTIL study, and in 14 control samples (from boys aged 5.6 ± 5.0 [mean ± SD] years) from an internal biobank. PARTICIPANTS/MATERIALS, SETTING, METHODS: Prepubertal boys in Sweden, Finland and Iceland who were facing treatments associated with a very high risk of infertility, were offered the experimental procedure of testicular cryopreservation. Exclusion criteria were testicular volumes >10 ml and high bleeding or infection risk. There were 18 patients with a diagnosis of malignancy and 14 patients a nonmalignant diagnosis. While 20 patients had the testicular biopsy performed 1-45 days after chemotherapy, 12 patients had not received any chemotherapy. In addition, 14 testicular tissue samples of patients with no reported testicular pathology, obtained from the internal biobank of the Department of Pathology at Karolinska University Hospital, were included as control samples in addition to reference values obtained from a recently published meta-analysis. The quantity of spermatogonia was assessed by both morphological and immunohistochemical analysis. MAIN RESULTS AND THE ROLE OF CHANCE: The main finding was a significant reduction in spermatogonial cell counts in boys treated with alkylating agents or with hydroxyurea for sickle cell disease. The mean S/T values in boys exposed to alkylating agents (0.2 ± 0.3, n = 6) or in boys with sickle cell disease and exposed to hydroxyurea (0.3 ± 0.6, n = 6) were significantly lower (P = 0.003 and P = 0.008, respectively) than in a group exposed to non-alkylating agents or in biobank control samples (1.7 ± 1.0, n = 8 and 4.1 ± 4.6, n = 14, respectively). The mean S/T values of the testicular tissue samples included in the biobank control group and the patient group exposed to nonalkylating agents were within recently published normative reference values. LIMITATIONS, REASONS FOR CAUTION: Normal testicular tissue samples included in this study were obtained from the internal biobank of Karolinska University Hospital. Samples were considered normal and included in the study if no testicular pathology was reported in the analysed samples. However, detailed information regarding previous medical treatments and testicular volumes of patients included in this biobank were not available. WIDER IMPLICATIONS OF THE FINDINGS: This study summarizes, for the first time, spermatogonial quantity in a prepubertal patient cohort just before and after potentially sterilizing treatments. Boys facing cancer and cytotoxic therapies are regarded as the major group who will benefit from novel fertility preservation techniques. There are no previous reports correlating spermatogonial quantity to cumulative exposure to alkylating agents and anthracyclines (non-alkylating agents) and no information about the timing of cytotoxic exposures among this particular patient cohort. For prepubertal boys in whom fertility preservation is indicated, testicular tissue should be obtained before initiation of chemotherapy with alkylating agents, whilst for those with sickle cell disease and treated with hydroxyurea, this approach to fertility preservation may not be feasible., This study was supported by grants from The Swedish Childhood Cancer Foundation (PR2016-0124; TJ2016-0093; PR2015-0073, TJ2015-0046) (K.J. and J.-B.S.), the Jane and Dan Olssons Foundation (2016-33) (J.-B.S.), the Finnish Cancer Society (K.J.), the Foundation for Paediatric Research (J.-B.S), Kronprinsessan Lovisas Förening För Barnasjukvård/Stiftelsen Axel Tielmans Minnesfond, Samariten Foundation (J.-B.S.), the Väre Foundation for Paediatric Cancer Research (K.J.), and Swedish Research Council (2012-6352) (O.S.). R.T.M. was supported by a Wellcome Trust Fellowship (09822). J.P.A.-L. and M.K. were supported by the ITN Marie Curie program ‘Growsperm’ (EU-FP7-PEOPLE-2013-ITN 603568).

Published
2018

43. One-step superhydrophobic coating using hydrophobized cellulose nanofibrils

Author

Reverdy, C., Belgacem, N., Moghaddam, M. S., Sundin, M., Swerin, Agne, Bras, J., Reverdy, C., Belgacem, N., Moghaddam, M. S., Sundin, M., Swerin, Agne, and Bras, J.

Abstract

Superhydrophobic surfaces have high potential in self-cleaning and anti-fouling applications. We developed a one-step superhydrophobic coating formulation containing sodium oleate (NaOl), hydrophobized precipitated calcium carbonate and biobased cellulose nanofibrils (CNFs) hydrophobized with either alkyl ketene dimer (AKD) or amino propyl trimethoxy silane (APMS) as a binder to fix and distribute the particles. Coatings were made on paperboard and the wetting behavior of the surface was assessed. Static, advancing and receding contact angles with water as well as roll-off and water shedding angle were compared to coatings made with styrene butadiene latex as binder instead of CNFs. Modifications with alkyl ketene dimer showed most promising results for a viable process in achieving superhydrophobic paperboard but required reformulation of the coating with optimized and reduced amount of NaOl to avoid surfactant-induced wetting via excess NaOl. A static water contact angle of 150° was reached for the CNF-AKD. The use of CNFs enables the improvement of coating quality avoiding cracking with the use of nanocellulose as a renewable binder., QC 20180514

Published
2018
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44. Change of apheresis device decreased the incidence of severe acute graft-versus-host disease among patients after allogeneic stem cell transplantation with sibling donors

Author

Wang, T., Remberger, M., Nygell, U. Axdorph, Sundin, M., Björklund, A., Mattsson, J., Uhlin, Michael, Watz, E., Wang, T., Remberger, M., Nygell, U. Axdorph, Sundin, M., Björklund, A., Mattsson, J., Uhlin, Michael, and Watz, E.

Abstract

BACKGROUND: The composition of the graft used for allogeneic hematopoietic stem cell transplantation (HSCT) is important for the treatment outcome. Different apheresis devices may yield significant differences in peripheral blood stem cell graft cellular composition. We compared stem cell grafts produced by Cobe Spectra (Cobe) and Spectra Optia (Optia) with use of the mononuclear cell (MNC) protocol, and evaluated clinical outcome parameters such as graft-versus-host disease (GvHD), transplant-related mortality (TRM), relapse, and overall survival. STUDY DESIGN AND METHODS: During 5 years, 31 Cobe Spectra and 40 Spectra Optia grafts were analyzed for CD34, CD3, CD4, CD8, CD19, and CD56 cell content. Clinical outcome parameters were correlated and compared between the two patient groups using different apheresis devices. RESULTS: Optia grafts contained fewer lymphocytes compared to Cobe (p<0.001). Optia grafts had a significantly lower incidence of acute GvHD Grades II through IV (Cobe 45% vs. Optia 23%; p=0.039) and TRM (16% vs. 2.5%; p<0.05) but higher chronic GvHD (32% vs. 67%; p=0.005) compared to Cobe grafts. Finally, the multivariate analysis showed a significant correlation among the different apheresis devices and both acute GvHD II through IV and severe chronic GvHD. The multivariate analysis also showed a significant correlation between the CD3+ cell dose and the incidence of severe acute GvHD. CONCLUSION: Optia-obtained grafts yielded a lower acute GvHD Grades II-IV and TRM risk, but had no impact on relapse or overall survival in this study. Understanding and further improvement of peripheral blood stem cell (PBSC) apheresis techniques may be used in the future to personalize HSCT by, for example, fine-tuning the GvHD incidence., QC 20180719

Published
2018
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46. Spermatogonial quantity in human prepubertal testicular tissue collected for fertility preservation prior to potentially sterilizing therapy

Author

Stukenborg, J -B, primary, Alves-Lopes, J P, additional, Kurek, M, additional, Albalushi, H, additional, Reda, A, additional, Keros, V, additional, Töhönen, V, additional, Bjarnason, R, additional, Romerius, P, additional, Sundin, M, additional, Norén Nyström, U, additional, Langenskiöld, C, additional, Vogt, H, additional, Henningsohn, L, additional, Mitchell, R T, additional, Söder, O, additional, Petersen, C, additional, and Jahnukainen, K, additional

Published
2018
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48. Impact of Conditioning Regimen on Outcomes for Children with Acute Myeloid Leukemia Undergoing Transplantation in First Complete Remission. An Analysis on Behalf of the Pediatric Disease Working Party of the European Group for Blood and Marrow Transplantation

Author

Lucchini, G., Labopin, M., Beohou, E., Dalissier, A., Dalle, J. H., Cornish, J., Zecca, M., Samarasinghe, S., Gibson, B., Locatelli, Franco, Bertrand, Y., Abdel-Rahman, F., Socie, G., Sundin, M., Lankester, A., Sedlacek, P., Hamladji, R. M., Heilmann, C., Afanasyev, B., Hough, R., Peters, C., Bader, P., Veys, P., Locatelli F. (ORCID:0000-0002-7976-3654), Lucchini, G., Labopin, M., Beohou, E., Dalissier, A., Dalle, J. H., Cornish, J., Zecca, M., Samarasinghe, S., Gibson, B., Locatelli, Franco, Bertrand, Y., Abdel-Rahman, F., Socie, G., Sundin, M., Lankester, A., Sedlacek, P., Hamladji, R. M., Heilmann, C., Afanasyev, B., Hough, R., Peters, C., Bader, P., Veys, P., and Locatelli F. (ORCID:0000-0002-7976-3654)

Abstract

Hematopoietic stem cell transplantation (HSCT) represents the cornerstone of treatment in pediatric high-risk and relapsed acute myeloid leukemia (AML). The aim of the present study was to compare outcomes of pediatric patients with AML undergoing HSCT using 3 different conditioning regimens: total body irradiation (TBI) and cyclophosphamide (Cy); busulfan (Bu) and Cy; or Bu, Cy, and melphalan (Mel). In this retrospective study, registry data for patients > 2 and <18 years age undergoing matched allogeneic HSCT for AML in first complete remission (CR1) in 204 European Group for Blood and Marrow Transplantation centers between 2000 and 2010 were analyzed. Data were available for 631 patients; 458 patients received stem cells from a matched sibling donor and 173 from a matched unrelated donor. For 440 patients, bone marrow was used as stem cell source, and 191 patients received peripheral blood stem cells. One hundred nine patients received TBICy, 389 received BuCy, and 133 received BuCyMel as their preparatory regimen. Median follow-up was 55 months. Patients receiving BuCyMel showed a lower incidence of relapse at 5 years (14.7% versus 31.5% in BuCy versus 30% in TBICy, P < .01) and higher overall survival (OS) (76.6% versus 64% versus 64.5%, P = .04) and leukemia-free survival (LFS) (74.5% versus 58% versus 61.9%, P < .01), with a comparable nonrelapse mortality (NRM) (10.8% versus 10.5% versus 8.1%, P = .79). Acute graft-versus-host disease (GVHD) grades III and IV but not chronic GVHD, was higher in patients receiving BuCyMel. Older age at HSCT had an adverse impact on NRM and the use of peripheral blood as stem cell source was associated with increased chronic GVHD and NRM as well as lower LFS and OS. Among pediatric patients receiving HSCT for AML in CR1, the use of BuCyMel conditioning proved superior to TBICy and BuCy in reducing relapse and improving LFS.

Published
2017

49. Vasopressin Versus Norepinephrine in Patients With Vasoplegic Shock After Cardiac Surgery

Author

Hajjar, L. A., primary, Vincent, J. L., additional, Barbosa Gomes Galas, F. R., additional, Rhodes, A., additional, Landoni, G., additional, Osawa, E. A., additional, Melo, R. R., additional, Sundin, M. R., additional, Grande, S. M., additional, Gaiotto, F. A., additional, Pomerantzeff, P. M., additional, Dallan, L. O., additional, Franco, R. A., additional, Nakamura, R. E., additional, Lisboa, L. A., additional, de Almeida, J. P., additional, Gerent, A. M., additional, Souza, D. H., additional, Gaiane, M. A., additional, Fukushima, J. T., additional, Park, C. L., additional, Zambolim, C., additional, Rocha Ferreira, G. S., additional, Strabelli, T. M., additional, Fernandes, F. L., additional, Camara, L., additional, Zeferino, S., additional, Santos, V. G., additional, Piccioni, M. A., additional, Jatene, F. B., additional, Costa Auler, J. O., additional, and Filho, R. K., additional

Published
2017
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