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1. Outcomes of hematopoietic stem cell transplantation in 813 pediatric patients with Fanconi anemia

Author

Lum, Su Han, Eikema, Dirk-Jan, Piepenbroek, Brian, Wynn, Robert F., Samarasinghe, Sujith, Dalissier, Arnaud, Kalwak, Krysztof, Ayas, Mouhab, Hamladji, Rose-Marie, Yesilipek, Akif, Dalle, Jean-Hugues, Uckan-Cetinkaya, Duygu, Bierings, Marc, Kupesiz, Alphan, Halahleh, Khalid, Skorobogatova, Elena, Öztürk, Gülyüz, Faraci, Maura, Renard, Cecile, Evans, Pamela, Corbacioglu, Selim, Locatelli, Franco, Dufour, Carlo, Risitano, Antonio, and Peffault de Latour, Régis

Published
2024
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2. Ruxolitinib in treatment-naive or corticosteroid-refractory paediatric patients with chronic graft-versus-host disease (REACH5): interim analysis of a single-arm, multicentre, phase 2 study

Author

Locatelli, Franco, Antmen, Bulent, Kang, Hyoung Jin, Koh, Katsuyoshi, Takahashi, Yoshiyuki, Kupesiz, Alphan, Dias Matos, Maria Gabriela A, Chopra, Yogi, Bhat, Sunil, Im, Ho Joon, Güngör, Tayfun, Lu, Meng-Yao, Stefanelli, Tommaso, Rosko, Christine, St Pierre, Annie, Burock, Karin, Smith, Yvonne, Sinclair, Karen, and Diaz-de-Heredia, Cristina

Published
2024
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3. Thalassemia-free and graft-versus-host-free survival: outcomes of hematopoietic stem cell transplantation for thalassemia major, Turkish experience

Author

Yesilipek, M. Akif, Uygun, Vedat, Kupesiz, Alphan, Karasu, Gulsun, Ozturk, Gulyuz, Ertem, Mehmet, Şaşmaz, İlgen, Daloğlu, Hayriye, Güler, Elif, Hazar, Volkan, Fisgin, Tunç, Sezgin, Gülay, Kansoy, Savaş, Kuşkonmaz, Barış, Akıncı, Burcu, Özbek, Namık, İnce, Elif Ünal, Öztürkmen, Seda, Küpesiz, Funda Tayfun, Yalçın, Koray, Anak, Sema, Bozkurt, Ceyhun, Karakükçü, Musa, Küpeli, Serhan, Albayrak, Davut, Öniz, Haldun, Aksoylar, Serap, Okur, Fatma Visal, Albayrak, Canan, Yenigürbüz, Fatma Demir, Bozkaya, İkbal Ok, İleri, Talia, Gürsel, Orhan, Karagün, Barbaros Şahin, Kintrup, Gülen Tüysüz, Çelen, Suna, Elli, Murat, Aksoy, Basak Adaklı, Yılmaz, Ebru, Tanyeli, Atila, Akyol, Şule Turan, Siviş, Zuhal Önder, Özek, Gülcihan, Uçkan, Duygu, Kartal, İbrahim, Atay, Didem, Akyay, Arzu, Bilir, Özlem Arman, Çakmaklı, Hasan Fatih, Kürekçi, Emin, Malbora, Barış, Akbayram, Sinan, Demir, Hacı Ahmet, Kılıç, Suar Çakı, Güneş, Adalet Meral, Zengin, Emine, Özmen, Salih, and Antmen, Ali Bülent

Published
2022
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4. Hematopoietic stem cell transplantation for adolescents and adults with inborn errors of immunity: an EBMT IEWP study

Author

Aiuti, Alessandro, Maschan, Alexei, Aljurf, Mahmoud, Gedde-Dahl, Tobias, Gurman, Gunhan, Bordon, Victoria, Kriván, Gergely, Locatelli, Franco, Porta, Fulvio, Valcárcel, David, Beguin, Yves, Faraci, Maura, Kröger, Nicolaus, Kulagin, Aleksandr, Shaw, Peter J., Veelken, Joan Hendrik, Diaz de Heredia, Cristina, Fagioli, Franca, Felber, Matthias, Gruhn, Bernd, Holter, Wolfgang, Rössig, Claudia, Sedlacek, Petr, Apperley, Jane, Ayas, Mouhab, Bodova, Ivana, Choi, Goda, Cornelissen, J.J., Sirvent, Anne, Khan, Anjum, Kupesiz, Alphan, Lenhoff, Stig, Ozdogu, Hakan, von der Weid, Nicolas, Rovira, Montserrat, Schots, Rik, Vinh, Donald C., Albert, Michael H., Sirait, Tiarlan, Eikema, Dirk-Jan, Bakunina, Katerina, Wehr, Claudia, Suarez, Felipe, Fox, Maria Laura, Mahlaoui, Nizar, Gennery, Andrew R., Lankester, Arjan C., Beier, Rita, Bernardo, Maria Ester, Bigley, Venetia, Lindemans, Caroline A., Burns, Siobhan O., Carpenter, Ben, Dybko, Jaroslaw, Güngör, Tayfun, Hauck, Fabian, Lum, Su Han, Balashov, Dmitry, Meisel, Roland, Moshous, Despina, Schulz, Ansgar, Speckmann, Carsten, Slatter, Mary A., Strahm, Brigitte, Uckan-Cetinkaya, Duygu, Meyts, Isabelle, Vallée, Tanja C., Wynn, Robert, Neven, Bénédicte, and Morris, Emma C.

Published
2022
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5. Hematopoietic stem cell transplantation for Wiskott-Aldrich syndrome: an EBMT Inborn Errors Working Party analysis

Author

Albert, Michael H., Slatter, Mary A., Gennery, Andrew R., Güngör, Tayfun, Bakunina, Katerina, Markovitch, Benyamin, Hazelaar, Sheree, Sirait, Tiarlan, Courteille, Virginie, Aiuti, Alessandro, Aleinikova, Olga V., Balashov, Dmitry, Bernardo, Maria Ester, Bodova, Ivana, Bruno, Benedicte, Cavazzana, Marina, Chiesa, Robert, Fischer, Alain, Hauck, Fabian, Ifversen, Marianne, Kałwak, Krzysztof, Klein, Christoph, Kulagin, Alexander, Kupesiz, Alphan, Kuskonmaz, Baris, Lindemans, Caroline A., Locatelli, Franco, Lum, Su Han, Maschan, Alexey, Meisel, Roland, Moshous, Despina, Porta, Fulvio, Sauer, Martin G., Sedlacek, Petr, Schulz, Ansgar, Suarez, Felipe, Vallée, Tanja C., Winiarski, Jacek H., Zecca, Marco, Neven, Bénédicte, Veys, Paul, and Lankester, Arjan C.

Published
2022
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6. Allogeneic hematopoietic stem cell transplantation in leukocyte adhesion deficiency type I and III

Author

Bakhtiar, Shahrzad, Salzmann-Manrique, Emilia, Blok, Henric-Jan, Eikema, Dirk-Jan, Hazelaar, Sheree, Ayas, Mouhab, Toren, Amos, Goldstein, Gal, Moshous, Despina, Locatelli, Franco, Merli, Pietro, Michel, Gerard, Öztürk, Gülyüz, Schulz, Ansgar, Heilmann, Carsten, Ifversen, Marianne, Wynn, Rob F., Aleinikova, Olga, Bertrand, Yves, Tbakhi, Abdelghani, Veys, Paul, Karakukcu, Musa, Kupesiz, Alphan, Ghavamzadeh, Ardeshir, Handgretinger, Rupert, Unal, Emel, Perez-Martinez, Antonio, Gokce, Muge, Porta, Fulvio, Aksu, Tekin, Karasu, Gülsün, Badell, Isabel, Ljungman, Per, Skorobogatova, Elena, Yesilipek, Akif, Zuckerman, Tsila, Bredius, Robbert R.G., Stepensky, Polina, Shadur, Bella, Slatter, Mary, Gennery, Andrew R., Albert, Michael H., Bader, Peter, and Lankester, Arjan

Published
2021
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7. Dabigatran etexilate for the treatment of acute venous thromboembolism in children (DIVERSITY): a randomised, controlled, open-label, phase 2b/3, non-inferiority trial

Author

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

Published
2021
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10. A large single‐center cohort of bare lymphocyte syndrome: Immunological and genetic features in Turkey.

Author

Ünsal, Hilal, Caka, Canan, Bildik, Hacer Neslihan, Esenboğa, Saliha, Kupesiz, Alphan, Kuşkonmaz, Barış, Cetinkaya, Duygu Uçkan, van der Burg, Mirjam, Tezcan, İlhan, and Çağdaş, Deniz

Subjects
SEVERE combined immunodeficiency, HEMATOPOIETIC stem cell transplantation, MAJOR histocompatibility complex, INFECTIOUS arthritis, GENETIC counseling, BLOOD coagulation factor XIII
Abstract

Major histocompatibility complex class II (MHC‐II) deficiency or bare lymphocyte syndrome (BLS) is a rare, early‐onset, autosomal recessive, and life‐threatening inborn error of immunity. We aimed to assess the demographic, clinical, laboratory, follow‐up, and treatment characteristics of patients with MHC‐II deficiency, together with their survival. We retrospectively investigated 21 patients with MHC‐II deficiency. Female/male ratio was 1.63. The median age at diagnosis was 16.3 months (5 months–9.7 years). Nineteen patients (90.5%) had parental consanguinity. Pulmonary diseases (pneumonia, chronic lung disease) (81%), diarrhoea (47.6%), and candidiasis (28.6%) were common. Four (19%) had autoimmunity, two developed septic arthritis, and three (14%) developed bronchiectasis in the follow‐up. Three patients (14%) had CMV viraemia, one with bilateral CMV retinitis. Eight (38.1%) had lymphocytopenia, and four (19%) had neutropenia. Serum IgM, IgA, and IgG levels were low in 18 (85.7%), 15 (71.4%), and 11 (52.4%) patients, respectively. CD4+ lymphocytopenia, a reversed CD4+/CD8+ ratio, and absent/low HLA‐DR expressions were detected in 93.3%, 86.7%, and 100% of the patients, respectively. Haematopoietic stem cell transplantation (HSCT) was performed on nine patients, and four died of septicaemia and ARDS after HSCT. The present median age of patients survived is 14 years (1–31 years). Genetic analysis was performed in 10 patients. RFX5 homozygous gene defect was found in three patients (P1, P4 and P8), and RFXANK (P2 and P14) and RFXAP (P18 and P19) heterozygous gene defects were found in each two patients, respectively. This large cohort showed that BLS patients have severe combined immunodeficiency (SCID)‐like clinical findings. Flow cytometric MHC‐II expression study is crucial for the diagnosis, differential diagnosis with SCID, early haematopoietic stem cell transplantation (HSCT), and post‐HSCT follow‐up. Genetic studies are required first for matched family donor evaluation before HSCT and then for genetic counselling. [ABSTRACT FROM AUTHOR]

Published
2024
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11. S245: RUXOLITINIB IN PEDIATRIC PATIENTS WITH TREATMENT-NAIVE OR STEROID-REFRACTORY CHRONIC GRAFT VERSUS HOST DISEASE: PRIMARY FINDINGS FROM THE PHASE II REACH5 STUDY

Author

Locatelli, Franco, primary, Antmen, Bulent, additional, Kang, Hyoung Jin, additional, Koh, Katsuyoshi, additional, Takahashi, Yoshiyuki, additional, Kupesiz, Alphan, additional, Matos, Maria Gabriela, additional, Wall, Donna, additional, Bhat, Sunil, additional, Im, Ho Joon, additional, Gungor, Tayfun, additional, Lu, Meng-Yao, additional, Stefanelli, Tommaso, additional, Rosko, Christine, additional, Pierre, Annie St., additional, Burock, Karin, additional, and Diaz-De-Heredia, Cristina, additional

Published
2023
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12. Clinical Features and HSCT Outcome for SCID in Turkey

Author

Ikinciogullari, Aydan, Cagdas, Deniz, Dogu, Figen, Tugrul, Tuba, Karasu, Gulsum, Haskologlu, Sule, Aksoylar, Serap, Uygun, Vedat, Kupesiz, Alphan, Yildiran, Alisan, Gursel, Orhan, Ates, Can, Elhan, Atilla, Kansoy, Savas, Yesilipek, Akif, Tezcan, Ilhan, and on behalf of Turkish Pediatric Bone Marrow Transplantation Sub Group (TPBMT-SG)

Published
2019
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13. Current use of androgens in bone marrow failure disorders: a report from the Severe Aplastic Anemia Working Party of the European Society of Blood and Marrow Transplantation

Author

Pagliuca, Simona, primary, Kulasekararaj, Austin G., additional, Eikema, Dirk-Jan, additional, Piepenbroek, Brian, additional, Iftikhar, Raheel, additional, Satti, Tariq Mahmood, additional, Griffin, Morag, additional, Laurino, Marica, additional, Kupesiz, Alphan, additional, Bertrand, Yves, additional, Fattizzo, Bruno, additional, Yakoub-Agha, Ibrahim, additional, Aljurf, Mahmoud, additional, Corti, Paola, additional, Massaccesi, Erika, additional, Lioure, Bruno, additional, Calabuig, Marisa, additional, Klammer, Matthias, additional, Unal, Emel, additional, Wu, Depei, additional, Chevallier, Patrice, additional, Forcade, Edouard, additional, Snowden, John A., additional, Ozdogu, Hakan, additional, Risitano, Antonio, additional, and De Latour, Régis Peffault, additional

Published
2023
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14. Outcomes of patients undergoing allogeneic haematopoietic stem cell transplantation for congenital amegakaryocytic thrombocytopenia; a study on behalf of the PDWP of the EBMT

Author

Aldebert, Clémence, Fahd, Mony, Galimard, Jacques-Emmanuel, Ghemlas, Ibrahim A., Zecca, Marco, Silva, Juliana, Mohseny, Alexander, Kupesiz, Alphan, Hamladji, Rose-Marie, Miranda, Nuno, Güngör, Tayfun, Wynn, Robert F., Merli, Pietro, Sundin, Mikael, Faraci, Maura, Diaz-de-Heredia, Cristina, Burkhardt, Birgit, Bordon, Victoria, Angoso, Marie, Bader, Peter, Ifversen, Marianne, Herrera Arroyo, Concepcion, Maximova, Natalia, Riesco, Susana, Stein, Jerry, Dalissier, Arnaud, Locatelli, Franco, Kalwak, Krzysztof, Dalle, Jean-Hugues, and Corbacioglu, Selim

Abstract

Congenital amegakaryocytic thrombocytopenia is a rare, inherited bone marrow failure syndrome. Allogeneic haematopoietic stem cell transplantation (allo-HSCT) is currently the only curative treatment. In this retrospective study, we analysed 66 patients with allo-HSCT, reported in the European Society for Blood and Marrow Transplantation (EBMT) registry. Bone marrow (BM) was the most widely used stem cell source (n= 40; 61%) followed by peripheral blood (PB) (n= 18; 27%), and unrelated umbilical cord blood (UCB) (n= 8; 12%). Most frequently was a HLA-matched graft from related (n= 26; 39%) and unrelated (n= 15; 23%) donors after a myeloablative busulfan-based conditioning regimen. GvHD prophylaxis was mostly cyclosporine and methotrexate (53%). The 6-year cumulative incidence of graft-failure and second transplant were 25% and 17%, respectively. The 6-year disease-free survival (DFS) and overall survival (OS) were 66.9% and 85.6%, respectively. The 6-year transplant-related mortality (TRM) was 8.0%. In conclusion, most patients with CAMT benefit from allo-HSCT, but with many graft failures.

Published
2024
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15. Prophylaxis vs. on‐demand treatment with BAY 81‐8973, a full‐length plasma protein‐free recombinant factor VIII product: results from a randomized trial (LEOPOLD II)

Author

Kavakli, K., Yang, R., Rusen, L., Beckmann, H., Tseneklidou‐Stoeter, D., Maas Enriquez, M., Yang, Renchi, Zhao, Yongqiang, Sun, Jing, Wang, Xuefeng, Wu, Depei, Hlusi, Antonin, Fukutake, Katsuyuki, Hanabusa, Hideji, Fujii, Teruhisa, Ramírez, Oscar Pérez, Alvarado, Blanca Salazar, Serban, Margit, Rusen, Luminita, Uscatescu, Valentina, Truica, Cristina, Kostic, Gordana, Konstantinidis, Nada, Igrutinovic, Zoran, Perina, Farida, Andreeva, Tatiana, Kavakli, Kaan, Antmen, Bulent, Sasmaz, Ilgen, Kupesiz, Alphan, Yesilipek, Mehmet Akif, Peng, Ching‐Tien, French, James, II, Escobar, Miguel, Mahlangu, Johnny, and Pool, Roger

Published
2015
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16. Outcomes of Allogeneic Hematopoietic Stem Cell Transplantation for Congenital Amegakaryocytic Thrombocytopenia, a PDWP/EBMT Study

Author

Aldebert, Clemence, primary, Fahd, Mony, additional, Galimard, Jacques-Emmanuel, additional, Ghemlas, Ibrahim A., additional, Zecca, Marco, additional, Silva, Juliana, additional, Mohseny, Alexander, additional, Kupesiz, Alphan, additional, Hamladji, Rose-Marie, additional, Miranda, Nuno, additional, Gungor, Tayfun, additional, Wynn, Robert F, additional, Merli, Pietro, additional, Sundin, Mikael, additional, Faraci, Maura, additional, Díaz-de-Heredia, Cristina, additional, Burkhardt, Birgit, additional, Bordon, Victoria, additional, Jubert, Charlotte, additional, Bader, Peter, additional, Ifversen, Marianne, additional, Herrera Arroyo, Concepcion, additional, Maximova, Natalia, additional, Riesco, Susana, additional, Stein, Jerry, additional, Dalissier, Arnaud, additional, Locatelli, Franco, additional, Kalwak, Krzysztof, additional, Dalle, Jean-Hugues, additional, and Corbacioglu, Selim, additional

Published
2022
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17. Hematopoietic stem cell transplantation for adolescents and adults with inborn errors of immunity: an EBMT IEWP study

Author

Albert, Michael H., primary, Sirait, Tiarlan, additional, Eikema, Dirk-Jan, additional, Bakunina, Katerina, additional, Wehr, Claudia, additional, Suarez, Felipe, additional, Fox, Maria Laura, additional, Mahlaoui, Nizar, additional, Gennery, Andrew R., additional, Lankester, Arjan C., additional, Beier, Rita, additional, Bernardo, Maria Ester, additional, Bigley, Venetia, additional, Lindemans, Caroline A., additional, Burns, Siobhan O., additional, Carpenter, Ben, additional, Dybko, Jaroslaw, additional, Güngör, Tayfun, additional, Hauck, Fabian, additional, Lum, Su Han, additional, Balashov, Dmitry, additional, Meisel, Roland, additional, Moshous, Despina, additional, Schulz, Ansgar, additional, Speckmann, Carsten, additional, Slatter, Mary A., additional, Strahm, Brigitte, additional, Uckan-Cetinkaya, Duygu, additional, Meyts, Isabelle, additional, Vallée, Tanja C., additional, Wynn, Robert, additional, Neven, Bénédicte, additional, Morris, Emma C., additional, Aiuti, Alessandro, additional, Maschan, Alexei, additional, Aljurf, Mahmoud, additional, Gedde-Dahl, Tobias, additional, Gurman, Gunhan, additional, Bordon, Victoria, additional, Kriván, Gergely, additional, Locatelli, Franco, additional, Porta, Fulvio, additional, Valcárcel, David, additional, Beguin, Yves, additional, Faraci, Maura, additional, Kröger, Nicolaus, additional, Kulagin, Aleksandr, additional, Shaw, Peter J., additional, Veelken, Joan Hendrik, additional, Diaz de Heredia, Cristina, additional, Fagioli, Franca, additional, Felber, Matthias, additional, Gruhn, Bernd, additional, Holter, Wolfgang, additional, Rössig, Claudia, additional, Sedlacek, Petr, additional, Apperley, Jane, additional, Ayas, Mouhab, additional, Bodova, Ivana, additional, Choi, Goda, additional, Cornelissen, J.J., additional, Sirvent, Anne, additional, Khan, Anjum, additional, Kupesiz, Alphan, additional, Lenhoff, Stig, additional, Ozdogu, Hakan, additional, von der Weid, Nicolas, additional, Rovira, Montserrat, additional, Schots, Rik, additional, and Vinh, Donald C., additional

Published
2022
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21. 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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22. Gene therapy in haemophilia: literature review and regional perspectives for Turkey

Author

Kavakli, Kaan, Antmen, Bulent, Okan, Vahap, Sahin, Fahri, Aytac, Selin, Balkan, Can, Berber, Ergul, Kaya, Zuhre, Kupesiz, Alphan, and Zulfikar, Bulent

Subjects
Hemophilia-B, AAV Vectors, Gene Therapy, Hemophilia-A
Abstract

Haemophilia is an X-linked lifelong congenital bleeding disorder that is caused by insufficient levels of factor VIII (FVIII; haemophilia A) or factor IX (FIX; haemophilia B) and characterized by spontaneous and trauma-related bleeding episodes. The cornerstone of the treatment, factor replacement, constitutes several difficulties, including frequent injections due to the short half-life of recombinant factors, intravenous administration and the risk of inhibitor development. While extended half-life factors and subcutaneous novel molecules enhanced the quality of life, initial successes with gene therapy offer a significant hope for cure. Although adeno-associated viral (AAV)-based gene therapy is one of the most emerging approaches for treatment of haemophilia, there are still challenges in vector immunogenicity, potency and efficacy, genotoxicity and persistence. As the approval for the first gene therapy product is coming closer, eligibility criteria for patient selection, multidisciplinary approach for optimal delivery and follow-up and development of new pricing policies and reimbursement models should be concerned. Therefore, this review addresses the unmet needs of current haemophilia treatment and explains the rationale and principles of gene therapy. Limitations and challenges are discussed from a global and national perspective and recommendations are provided to adopt the gene therapies faster and more sufficient for the haemophilia patients in developing countries like Turkey. © The Author(s), 2022.

Published
2022

24. Outcome of autologous hematopoietic stem cell transplantation in children and adolescents with relapsed or refractory Hodgkinʼs lymphoma

Author

Hazar, Volkan, Kesik, Vural, Aksoylar, Serap, Karakukcu, Musa, Ozturk, Gulyuz, Kupesiz, Alphan, Atas, Erman, Oniz, Haldun, Kansoy, Savas, Unal, Ekrem, Tanyeli, Atila, Erbey, Fatih, Elli, Murat, Tacyildiz, Nurdan, Karasu, Gulsun Tezcan, Kocak, Ulker, Anak, Sema, Bengoa, Sebnem Yilmaz, Sezgin, Gulay, Atay, Didem, Unal, Emel, Uygun, Vedat, Kurucu, Nilgun, Kaya, Zuhre, and Yesilipek, Akif

Published
2015
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26. Allogeneic Hematopoietic Stem Cell Transplantation for BCR/ABL1-Negative Myeloproliferative Neoplasms in Children - Retrospective Report on Behalf of I-BFM SCT Committee and EBMT Pediatric Diseases WP

Author

Patrick, Katharine, Gruhn, Bernd, Corbacioglu, Selim, Kupesiz, Alphan, Balduzzi, Adriana, Al-Seraihy, Amal, Wynn, Robert, Veys, Paul, Stepensky, Polina, Robinson, Stephen, Locatelli, Franco, Dalissier, Arnaud, Galimard, Jacques-Emmanuel, Wachowiak, Jacek, Tbakhi, Abdelghani, Diaz, Miguel Angel, Socie, Gerard, Sedlacek, Petr, Abboud, Miguel, Beelen, Dietrich, Krivan, Gergely, ÜNAL, ALİ, Bodova, Ivana, Freycon, Claire, Dalle, Jean-Hugues, Jubert, Charlotte, Ferster, Alina, Ifversen, Marianne, and Hamladji, Rose-Marie

Published
2020

27. Haplo‐identical or mismatched unrelated donor hematopoietic cell transplantation for Fanconi anemia: Results from the Severe Aplastic Anemia Working Party of the EBMT

Author

Zubicaray, Josune, primary, Pagliara, Daria, additional, Sevilla, Julian, additional, Eikema, Dirk‐Jan, additional, Bosman, Paul, additional, Ayas, Mouhab, additional, Zecca, Marco, additional, Yesilipek, Akif, additional, Kansoy, Savas, additional, Renard, Cécile, additional, Dalle, Jean H., additional, Campos, Antonio, additional, Faraci, Maura, additional, Kupesiz, Alphan, additional, Smiers, Frans J. W., additional, Velardi, Andrea, additional, Abecasis, Manuel, additional, Corti, Paola, additional, Fagioli, Franca, additional, González Muñiz, Soledad, additional, Kriván, Gergely, additional, Dufour, Carlo, additional, Risitano, Antonio, additional, Corbacioglu, Selim, additional, and Peffault de Latour, Régis, additional

Published
2021
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29. Dabigatran etexilate for the treatment of acute venous thromboembolism in children (DIVERSITY): a randomised, controlled, open-label, phase 2b/3, non-inferiority trial

Author

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

Published
2021
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30. Allogeneic hematopoietic stem cell transplantation in leukocyte adhesion deficiency type I and III

Author

Bakhtiar, Shahrzad, Salzmann-Manrique, Emilia, Blok, Henric-Jan, Eikema, Dirk-Jan, Hazelaar, Sheree, Ayas, Mouhab, Toren, Amos, Goldstein, Gal, Moshous, Despina, Locatelli, Franco, Merli, Pietro, Michel, Gerard, Öztürk, Gülyüz, Schulz, Ansgar, Heilmann, Carsten, Ifversen, Marianne, Wynn, Rob F., Aleinikova, Olga, Bertrand, Yves, Tbakhi, Abdelghani, Veys, Paul, Karakukcu, Musa, Kupesiz, Alphan, Ghavamzadeh, Ardeshir, Handgretinger, Rupert, Unal, Emel, Perez-Martinez, Antonio, Gokce, Muge, Porta, Fulvio, Aksu, Tekin, Karasu, Gülsün, Badell, Isabel, Ljungman, Per, Skorobogatova, Elena, Yesilipek, Akif, Zuckerman, Tsila, Bredius, Robbert R. G., Stepensky, Polina, Shadur, Bella, Slatter, Mary, Gennery, Andrew R., Albert, Michael H., Bader, Peter, and Lankester, Arjan

Subjects
Clinical Trials and Observations, Leukocyte-Adhesion Deficiency Syndrome, Hematopoietic Stem Cell Transplantation, Graft vs Host Disease, leukocyte adhesion deficiency type 1, leukocyte, periodontitis, surgical procedures, operative, Settore MED/38 - PEDIATRIA GENERALE E SPECIALISTICA, HSCT, Leukocytes, Humans, Retrospective Studies
Abstract

Type I and III leukocyte adhesion deficiencies (LADs) are primary immunodeficiency disorders resulting in early death due to infections and additional bleeding tendency in LAD-III. The curative treatment of LAD-I and LAD-III is allogeneic hematopoietic stem cell transplantation (allo-HSCT). In this retrospective multicenter study, data were collected using the European Society for Blood and Marrow Transplantation registry; we analyzed data from 84 LAD patients from 33 centers, all receiving an allo-HSCT from 2007 to 2017. The 3-year overall survival estimate (95% confidence interval [CI]) was 83% (74-92) for the entire cohort: 84% (75-94) and 75% (50-100) for LAD-I and LAD-III, respectively. We observed cumulative incidences (95% CI) of graft failure (GF) at 3 years of 17% (9%-26%) and grade II to IV acute graft-versus-host disease (aGVHD) at 100 days of 24% (15%-34%). The estimate (95% CI) at 3 years for GF- and GVHD-II to IV-free survival as event-free survival (EFS) was 56% (46-69) for the entire cohort; 58% (46-72) and 56% (23-88) for LAD-I and LAD-III, respectively. Grade II to IV acute GVHD was a relevant risk factor for death (hazard ratio 3.6; 95% CI 1.4-9.1; P = .006). Patients' age at transplant >= 13 months, transplantation from a nonsibling donor, and any serological cytomegalovirus mismatch in donor-recipient pairs were significantly associated with severe acute GVHD and inferior EFS. The choice of busulfan- or treosulfan-based conditioning, type of GVHD prophylaxis, and serotherapy did not impact overall survival, EFS, or aGVHD. An intrinsic inflammatory component of LAD may contribute to inflammatory complications during allo-HSCT, thus providing the rationale for considering anti-inflammatory therapy pretreatment.

Published
2020

35. Prognostic impact of Epstein-Barr virus serostatus in patients with nonmalignant hematological disorders undergoing allogeneic hematopoietic cell transplantation: the study of Infectious Diseases Working Party of the European Society for Blood and Marrow Transplantation

Author

Styczynski, Jan, primary, Tridello, Gloria, additional, Gil, Lidia, additional, Ljungman, Per, additional, Mikulska, Malgorzata, additional, van der Werf, Steffie, additional, Knelange, Nina Simone, additional, Averbuch, Diana, additional, Socié, Gerard, additional, Veelken, Hendrik, additional, Dalle, Jean-Hugues, additional, Aljurf, Mahmoud, additional, Kupesiz, Alphan, additional, Bertrand, Yves, additional, Tbakhi, Abdelghani, additional, Afanasyev, Boris, additional, Lioure, Bruno, additional, Labussière-Wallet, Hélène, additional, Poiré, Xavier, additional, Maertens, Johan, additional, Petersen, Eefke, additional, Chevallier, Patrice, additional, Milpied, Noel, additional, Snowden, John A., additional, Yakoub-Agha, Ibrahim, additional, Cornelissen, Jan, additional, Schaap, Nicolaas, additional, Dufour, Carlo, additional, de Latour, Regis Peffault, additional, Lankester, Arjan, additional, Cesaro, Simone, additional, and _, _, additional

Published
2020
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42. Relapse after allogeneic hematopoietic stem cell transplantation for acute leukemia in children: a survey by the Turkish Pediatric Bone Marrow Study Group of 255 cases

Author

Hazar, Volkan, Karasu, Gulsun Tezcan, Ozturk, Gulyuz, Kupesiz, Alphan, Kansoy, Savas, Ozbek, Namik, Uygun, Vedat, Ileri, Talia, Okur, Fatma Visal, Kocak, Ulker, Kilic, Suar Caki, Akcay, Arzu, Guler, Elif, Gozmen, Salih, Musa Karakukcu, Bayram, Ibrahim, Aksu, Tekin, Yesilipek, Akif, Karagun, Barboros Sahin, Bengoa, Sebnem Yilmaz, Ertem, Mehmet, Uckan, Duygu, Kaya, Zuhre, Fiskin, Tunc, Atay, Didem, Kupesiz, Funda Tayfun, Gursel, Orhan, Yaman, Yontem, Bozkurt, Ceyhun, Gokce, Muge, and Aksoylar, Serap

Subjects
surgical procedures, operative, Turkish Pediatric Bone Marrow Study Group, Allogeneic Hematopoietic, Acute Leukemia, Stem Cell Transplantation
Abstract

European Society for Blood and Marrow Transplantation

Published
2018

43. Juvenile Myelomonocytic Leukemia in Turkey: A Retrospective Analysis of Sixty-five Patients

Author

Yenicesu, Idil, YILMAZ KARAPINAR, DENİZ, Karaman, Serap, Uygun, Vedat, Karasu, Gulsun, Yesilipek, Mehmet Akif, KARAKÜKCÜ, MUSA, ALBAYRAK, DAVUT, ÜNAL, EKREM, ALBAYRAK, CANAN, KOÇ, AHMET, Gunes, Adalet Meral, Ince, Elif Unal, Ileri, Talia, ERTEM, MEHMET, CELKAN, Tülin Tıraje, Ozdemir, Gul Nihal, SEZGİN EVİM, MELİKE, TÜFEKÇİ, ÖZLEM, Tokgoz, Huseyin, Caliskan, Umran, SARPER, NAZAN, Baytan, Birol, ERDURAN, EROL, Atabay, Berna, Vergin, Canan, Kacar, Dilek, Karapinar, Tuba, Oniz, Haldun, KAYA, ZÜHRE, Yarali, Nese, Ozbek, Namik Yasar, Patiroglu, Turkan, Bengoa, Sebnem Yilmaz, Kupesiz, Alphan, ÖREN, HALE, KOÇAK, ÜLKER, and Ege Üniversitesi

Subjects
Male, lcsh:Internal medicine, Turkey, lcsh:RC633-647.5, Biopsy, Infant, Hematopoietic stem cell transplantation, lcsh:Diseases of the blood and blood-forming organs, Combined Modality Therapy, Survival Analysis, Leukemia, Myelomonocytic, Juvenile, Child, Preschool, Juvenile myelomonocytic leukemia, Humans, Female, Public Health Surveillance, Genetic Testing, Symptom Assessment, lcsh:RC31-1245, Research Article, Retrospective Studies
Abstract

WOS: 000426572200004, PubMed ID: 28179213, Objective: This study aimed to define the status of juvenile myelomonocytic leukemia (JMML) patients in Turkey in terms of time of diagnosis, clinical characteristics, mutational studies, clinical course, and treatment strategies. Materials and Methods: Data including clinical and laboratory characteristics and treatment strategies of JMML patients were collected retrospectively from pediatric hematology-oncology centers in Turkey. Results: Sixty-five children with JMML diagnosed between 2002 and 2016 in 18 institutions throughout Turkey were enrolled in the study. The median age at diagnosis was 17 months (min-max: 2-117 months). Splenomegaly was present in 92% of patients at the time of diagnosis. The median white blood cell, monocyte, and platelet counts were 32.9x10(9)/L, 5.4x10(9)/L, and 58.3x10(9)/L, respectively. Monosomy 7 was present in 18% of patients. JMML mutational analysis was performed in 32 of 65 patients (49%) and PTPN11 was the most common mutation. Hematopoietic stem cell transplantation (HSCT) could only be performed in 28 patients (44%), the majority being after the year 2012. The most frequent reason for not performing HSCT was the inability to find a suitable donor. The median time from diagnosis to HSCT was 9 months (min-max: 2-63 months). The 5-year cumulative survival rate was 33% and median estimated survival time was 30 +/- 17.4 months (95% CI: 0-64.1) for all patients. Survival time was significantly better in the HSCT group (log-rank p=0.019). Older age at diagnosis (>2 years), platelet count of less than 40x10(9)/L, and PTPN11 mutation were the factors significantly associated with shorter survival time. Conclusion: Although there has recently been improvement in terms of definitive diagnosis and HSCT in JMML patients, the overall results are not satisfactory and it is necessary to put more effort into this issue in Turkey.

Published
2018

44. Busulfan/Fludarabine- or Treosulfan/Fludarabine-Based Conditioning Regimen for Patients with Wiskott-Aldrich Syndrome – an EBMT Inborn Errors Working Party and Scetide Study

Author

Albert, Michael H., primary, Slatter, Mary, additional, Gennery, Andrew, additional, Gungor, Tayfun, additional, Blok, Henric-Jan, additional, Hazelaar, Sheree, additional, Wang, Junfeng, additional, Courteille, Virginie, additional, Mahlaoui, Nizar, additional, Bernado, Maria Ester, additional, Bodova, Ivana, additional, Bruno, Bénédicte, additional, Bykova, Tatiana, additional, Chiesa, Robert, additional, Fischer, Alain, additional, Formankova, Renata, additional, Kalwak, Krzysztof, additional, Klein, Christoph, additional, Kozlovskaya, Svetlana, additional, Kupesiz, Alphan, additional, Locatelli, Franco, additional, Moshous, Despina, additional, Neven, Benedicte, additional, Porta, Fulvio, additional, Schulz, Ansgar, additional, Sykora, Karl-Walter, additional, Karakukcu, Musa, additional, Winiarski, Jacek, additional, Zecca, Marco, additional, Veys, Paul, additional, and Lankester, Arjan C., additional

Published
2019
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47. Myeloablative conditioning for allo-HSCT in pediatric ALL: FTBI or chemotherapy?—A multicenter EBMT-PDWP study

Author

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
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50. Noninvasive ventilation in cancer children with acute respiratory failure.

Author

Yilmaz, Sema, Yildizdas, Riza Dincer, Dursun, Oguz, Karapinar, Bulent, Kendirli, Tanil, Demirkol, Demet, Citak, Agop, Kupesiz, Alphan, Tekguc, Hakan, Duyu, Muhterem, Yazici, Pinar, Yukselmis, Ufuk, Odek, Caglar, Yaman, Ayhan, Bayraktar, Suleyman, Şık, Guntulu, and Cakir, Fatma Betul

Subjects
NONINVASIVE ventilation, CHILDHOOD cancer, ADULT respiratory distress syndrome, ENDOTRACHEAL tubes, QUALITY of life
Abstract

Objective: To establish the effectiveness of noninvasive ventilation in cancer children with acute respiratory failure. Methods: The data of 33 cancer patients were obtained prospectively from six different pediatric intensive care units in Turkey between the years of 2012 and 2013. Results: The diagnosis was leukemias in 25 (75.8%), lymphomas in 3 (9.1%) and other solid tumors in 5 (15.1%) patients. Pneumonia in 12 (36.3%) and sepsis in 15 (45.4%) patients were seen as the common reasons of respiratory failure. The mean PaO2/FiO2 ratios were (164.22 ± 37.24) and (126.80 ± 42.73) in noninvasive ventilation success and failure group, respectively. Noninvasive ventilation was successful in 18 (54.5%) patients. The failure group consisted of 15 patients required intubation. A total of 14 (42.4%) patients died. The clinical outcome in terms of success and failure was meaningful statistically (P = 0.0 00 1). Conclusions: Our results could encourage the use of noninvasive ventilation in children with cancer who develop acute respiratory failure. It should be considered as a useful therapeutic approach to avoid endotracheal intubation. [ABSTRACT FROM AUTHOR]

Published
2017
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