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328 results on '"Yesilipek, Akif"'

1. Allogeneic Hematopoietic Stem Cell Transplantation in Immunodeficiency—Centromeric Instability—Facial Dysmorphism (ICF) Syndrome: an EBMT/ESID Inborn Errors Working Party Study

Author

Berghuis, Dagmar, Mehyar, Lubna S., Abu-Arja, Rolla, Albert, Michael H., Barnum, Jessie L., von Bernuth, Horst, Elfeky, Reem, Lewalle, Philippe, Laberko, Alexandra, Ghosh, Sujal, Slatter, Mary A., Weemaes, Corry M. R., Yesilipek, Akif, Sirait, Tiarlan, Neven, Bénédicte, Gennery, Andrew R., and Lankester, Arjan C.

Published
2024
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2. 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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3. Low rate of nonrelapse mortality in under-4-year-olds with ALL given chemotherapeutic conditioning for HSCT: a phase 3 FORUM study

Author

Bader, Peter, Pötschger, Ulrike, Dalle, Jean-Hugues, Moser, Laura M., Balduzzi, Adriana, Ansari, Marc, Buechner, Jochen, Güngör, Tayfun, Ifversen, Marianne, Krivan, Gergely, Pichler, Herbert, Renard, Marleen, Staciuk, Raquel, Sedlacek, Petr, Stein, Jerry, Heusel, Jan Robert, Truong, Tony, Wachowiak, Jacek, Yesilipek, Akif, Locatelli, Franco, and Peters, Christina

Published
2024
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4. Influence of invasive aspergillosis during acute leukaemia treatment on survival after allogeneic stem cell transplantation: a prospective study of the EBMT Infectious Diseases Working Party

Author

Penack, Olaf, Tridello, Gloria, Salmenniemi, Urpu, Martino, Rodrigo, Khanna, Nina, Perruccio, Katia, Fagioli, Franca, Richert-Przygonska, Monika, Labussière-Wallet, Hélène, Maertens, Johan, Jubert, Charlotte, Aljurf, Mahmoud, Pichler, Herbert, Kriván, Gergely, Kunadt, Desiree, Popova, Marina, Gabriel, Melissa, Calore, Elisabetta, Blau, Igor Wolfgang, Benedetti, Fabio, Itäla-Remes, Maija, de Kort, Elizabeth, Russo, Domenico, Faraci, Maura, Ménard, Anne-Lise, Borne, Peter von dem, Poiré, Xavier, Yesilipek, Akif, Gozdzik, Jolanta, Yeğin, Zeynep Arzu, Yañez, Lucrecia, Facchini, Luca, Van Gorkom, Gwendolyn, Thurner, Lorenz, Kocak, Ulker, Sampol, Antònia, Zuckerman, Tsila, Bierings, Marc, Mielke, Stephan, Ciceri, Fabio, Wendel, Lotus, Knelange, Nina, Mikulska, Malgorzata, Averbuch, Dina, Styczynski, Jan, Camara, Rafael de la, and Cesaro, Simone

Published
2024
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6. Low Density Granulocytes and Dysregulated Neutrophils Driving Autoinflammatory Manifestations in NEMO Deficiency

Author

Surucu Yilmaz, Naz, Bilgic Eltan, Sevgi, Kayaoglu, Basak, Geckin, Busranur, Heredia, Raul Jimenez, Sefer, Asena Pinar, Kiykim, Ayca, Nain, Ercan, Kasap, Nurhan, Dogru, Omer, Yucelten, Ayse Deniz, Cinel, Leyla, Karasu, Gulsun, Yesilipek, Akif, Sozeri, Betul, Kaya, Goksu Gokberk, Yilmaz, Ismail Cem, Baydemir, Ilayda, Aydin, Yagmur, Cansen Kahraman, Deniz, Haimel, Matthias, Boztug, Kaan, Karakoc-Aydiner, Elif, Gursel, Ihsan, Ozen, Ahmet, Baris, Safa, and Gursel, Mayda

Published
2022
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7. Busulfan–fludarabine- or treosulfan–fludarabine-based myeloablative conditioning for children with thalassemia major

Author

Lüftinger, Roswitha, Zubarovskaya, Natalia, Galimard, Jacques-Emmanuel, Cseh, Annamaria, Salzer, Elisabeth, Locatelli, Franco, Algeri, Mattia, Yesilipek, Akif, de la Fuente, Josu, Isgrò, Antonella, Alseraihy, Amal, Angelucci, Emanuele, Smiers, Frans J., La La Nasa, Giorgia, Zecca, Marco, Fisgin, Tunc, Unal, Emel, Kleinschmidt, Katharina, Peters, Christina, Lankester, Arjan, and Corbacioglu, Selim

Published
2022
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9. Long-term follow-up of IPEX syndrome patients after different therapeutic strategies: An international multicenter retrospective study.

Author

Barzaghi, Federica, Amaya Hernandez, Laura Cristina, Neven, Benedicte, Ricci, Silvia, Kucuk, Zeynep Yesim, Bleesing, Jack J, Nademi, Zohreh, Slatter, Mary Anne, Ulloa, Erlinda Rose, Shcherbina, Anna, Roppelt, Anna, Worth, Austen, Silva, Juliana, Aiuti, Alessandro, Murguia-Favela, Luis, Speckmann, Carsten, Carneiro-Sampaio, Magda, Fernandes, Juliana Folloni, Baris, Safa, Ozen, Ahmet, Karakoc-Aydiner, Elif, Kiykim, Ayca, Schulz, Ansgar, Steinmann, Sandra, Notarangelo, Lucia Dora, Gambineri, Eleonora, Lionetti, Paolo, Shearer, William Thomas, Forbes, Lisa R, Martinez, Caridad, Moshous, Despina, Blanche, Stephane, Fisher, Alain, Ruemmele, Frank M, Tissandier, Come, Ouachee-Chardin, Marie, Rieux-Laucat, Frédéric, Cavazzana, Marina, Qasim, Waseem, Lucarelli, Barbarella, Albert, Michael H, Kobayashi, Ichiro, Alonso, Laura, Diaz De Heredia, Cristina, Kanegane, Hirokazu, Lawitschka, Anita, Seo, Jong Jin, Gonzalez-Vicent, Marta, Diaz, Miguel Angel, Goyal, Rakesh Kumar, Sauer, Martin G, Yesilipek, Akif, Kim, Minsoo, Yilmaz-Demirdag, Yesim, Bhatia, Monica, Khlevner, Julie, Richmond Padilla, Erick J, Martino, Silvana, Montin, Davide, Neth, Olaf, Molinos-Quintana, Agueda, Valverde-Fernandez, Justo, Broides, Arnon, Pinsk, Vered, Ballauf, Antje, Haerynck, Filomeen, Bordon, Victoria, Dhooge, Catharina, Garcia-Lloret, Maria Laura, Bredius, Robbert G, Kałwak, Krzysztof, Haddad, Elie, Seidel, Markus Gerhard, Duckers, Gregor, Pai, Sung-Yun, Dvorak, Christopher C, Ehl, Stephan, Locatelli, Franco, Goldman, Frederick, Gennery, Andrew Richard, Cowan, Mort J, Roncarolo, Maria-Grazia, Bacchetta, Rosa, and Primary Immune Deficiency Treatment Consortium (PIDTC) and the Inborn Errors Working Party (IEWP) of the European Society for Blood and Marrow Transplantation (EBMT)

Subjects
Primary Immune Deficiency Treatment Consortium (PIDTC) and the Inborn Errors Working Party (IEWP) of the European Society for Blood and Marrow Transplantation, Humans, Genetic Diseases, X-Linked, Diabetes Mellitus, Type 1, Immune System Diseases, Diarrhea, Disease-Free Survival, Hematopoietic Stem Cell Transplantation, Survival Rate, Retrospective Studies, Follow-Up Studies, Mutation, Adolescent, Adult, Child, Child, Preschool, Infant, Female, Male, Forkhead Transcription Factors, Allografts, Immunosuppression Therapy, FOXP3, IPEX, Treg cells, enteropathy, genetic autoimmunity, hematopoietic stem cell transplantation, immunosuppression, neonatal diabetes, primary immune deficiency, rapamycin, Clinical Research, Stem Cell Research, Regenerative Medicine, Pediatric, Genetics, Transplantation, Aetiology, 2.1 Biological and endogenous factors, Immunology, Allergy
Abstract

BackgroundImmunodysregulation polyendocrinopathy enteropathy x-linked (IPEX) syndrome is a monogenic autoimmune disease caused by FOXP3 mutations. Because it is a rare disease, the natural history and response to treatments, including allogeneic hematopoietic stem cell transplantation (HSCT) and immunosuppression (IS), have not been thoroughly examined.ObjectiveThis analysis sought to evaluate disease onset, progression, and long-term outcome of the 2 main treatments in long-term IPEX survivors.MethodsClinical histories of 96 patients with a genetically proven IPEX syndrome were collected from 38 institutions worldwide and retrospectively analyzed. To investigate possible factors suitable to predict the outcome, an organ involvement (OI) scoring system was developed.ResultsWe confirm neonatal onset with enteropathy, type 1 diabetes, and eczema. In addition, we found less common manifestations in delayed onset patients or during disease evolution. There is no correlation between the site of mutation and the disease course or outcome, and the same genotype can present with variable phenotypes. HSCT patients (n = 58) had a median follow-up of 2.7 years (range, 1 week-15 years). Patients receiving chronic IS (n = 34) had a median follow-up of 4 years (range, 2 months-25 years). The overall survival after HSCT was 73.2% (95% CI, 59.4-83.0) and after IS was 65.1% (95% CI, 62.8-95.8). The pretreatment OI score was the only significant predictor of overall survival after transplant (P = .035) but not under IS.ConclusionsPatients receiving chronic IS were hampered by disease recurrence or complications, impacting long-term disease-free survival. When performed in patients with a low OI score, HSCT resulted in disease resolution with better quality of life, independent of age, donor source, or conditioning regimen.

Published
2018

12. 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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13. Low rate of nonrelapse mortality in under-4-year-olds with ALL given chemotherapeutic conditioning for HSCT: a phase 3 FORUM study

Author

Bader, Peter; https://orcid.org/0000-0003-4554-0265, Pötschger, Ulrike, Dalle, Jean-Hugues; https://orcid.org/0000-0002-8406-0793, Moser, Laura M, Balduzzi, Adriana; https://orcid.org/0000-0002-5879-0610, Ansari, Marc; https://orcid.org/0000-0002-9649-6498, Buechner, Jochen; https://orcid.org/0000-0001-5848-4501, Güngör, Tayfun; https://orcid.org/0000-0002-3261-1186, Ifversen, Marianne, Krivan, Gergely, Pichler, Herbert; https://orcid.org/0000-0001-5107-5123, Renard, Marleen, Staciuk, Raquel, Sedlacek, Petr; https://orcid.org/0000-0001-7895-2886, Stein, Jerry, Heusel, Jan Robert; https://orcid.org/0000-0002-5753-0817, Truong, Tony, Wachowiak, Jacek, Yesilipek, Akif, Locatelli, Franco; https://orcid.org/0000-0002-7976-3654, Peters, Christina; https://orcid.org/0000-0003-0369-8515, Bader, Peter; https://orcid.org/0000-0003-4554-0265, Pötschger, Ulrike, Dalle, Jean-Hugues; https://orcid.org/0000-0002-8406-0793, Moser, Laura M, Balduzzi, Adriana; https://orcid.org/0000-0002-5879-0610, Ansari, Marc; https://orcid.org/0000-0002-9649-6498, Buechner, Jochen; https://orcid.org/0000-0001-5848-4501, Güngör, Tayfun; https://orcid.org/0000-0002-3261-1186, Ifversen, Marianne, Krivan, Gergely, Pichler, Herbert; https://orcid.org/0000-0001-5107-5123, Renard, Marleen, Staciuk, Raquel, Sedlacek, Petr; https://orcid.org/0000-0001-7895-2886, Stein, Jerry, Heusel, Jan Robert; https://orcid.org/0000-0002-5753-0817, Truong, Tony, Wachowiak, Jacek, Yesilipek, Akif, Locatelli, Franco; https://orcid.org/0000-0002-7976-3654, and Peters, Christina; https://orcid.org/0000-0003-0369-8515

Abstract

Allogeneic hematopoietic stem cell transplantation (HSCT) is highly effective for treating pediatric high-risk or relapsed acute lymphoblastic leukemia (ALL). For young children, total body irradiation (TBI) is associated with severe late sequelae. In the FORUM study (NCT01949129), we assessed safety, event-free survival (EFS), and overall survival (OS) of 2 TBI-free conditioning regimens in children aged <4 years with ALL. Patients received fludarabine (Flu), thiotepa (Thio), and either busulfan (Bu) or treosulfan (Treo) before HSCT. From 2013 to 2021, 191 children received transplantation and were observed for ≥6 months (median follow-up: 3 years). The 3-year OS was 0.63 (95% confidence interval [95% CI], 0.52-0.72) and 0.76 (95% CI, 0.64-0.84) for Flu/Thio/Bu and Flu/Thio/Treo (P = .075), respectively. Three-year EFS was 0.52 (95% CI, 0.41-0.61) and 0.51 (95% CI, 0.39-0.62), respectively (P = .794). Cumulative incidence of nonrelapse mortality (NRM) and relapse at 3 years were 0.06 (95% CI, 0.02-0.12) vs 0.03 (95% CI: <0.01-0.09) (P = .406) and 0.42 (95% CI, 0.31-0.52) vs 0.45 (95% CI, 0.34-0.56) (P = .920), respectively. Grade >1 acute graft-versus-host disease (GVHD) occurred in 29% of patients receiving Flu/Thio/Bu and 17% of those receiving Flu/Thio/Treo (P = .049), whereas grade 3/4 occurred in 10% and 9%, respectively (P = .813). The 3-year incidence of chronic GVHD was 0.07 (95% CI, 0.03-0.13) vs 0.05 (95% CI, 0.02-0.11), respectively (P = .518). In conclusion, both chemotherapeutic conditioning regimens were well tolerated and NRM was low. However, relapse was the major cause of treatment failure. This trial was registered at www.clinicaltrials.gov as #NCT01949129.

Published
2024

14. The role of haematopoietic stem cell transplantation for sickle cell disease in the era of targeted disease-modifying therapies and gene editing

Author

Amrolia, Persis, Ansari, Marc, Balduzzi, Adriana, Dalassier, Arnaud, Dalle, Jean-Hugues, Hereda Diaz, Cristina, Feuchtinger, Tobias, Locatelli, Franco, Lucchini, Giovanna, Galimard, Jaques-Emmanuel, Gonzalez Vincent, Marta, Handgretinger, Rupert, Kleinschmidt, Katharina, Lawitschka, Anita, Perez Martinez, Antonio, Peters, Christina, Rocha, Vanderson, Ruggeri, Annalisa, Sedlacek, Petr, Svec, Peter, Toporski, Jacek, Yesilipek, Akif, de la Fuente, Josu, Gluckman, Eliane, Makani, Julie, Telfer, Paul, Faulkner, Lawrence, and Corbacioglu, Selim

Published
2020
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15. The impact of donor type on the outcome of pediatric patients with very high risk acute lymphoblastic leukemia. A study of the ALL SCT 2003 BFM-SG and 2007-BFM-International SG

Author

Dalle, Jean-Hugues, Balduzzi, Adriana, Bader, Peter, Pieczonka, Anna, Yaniv, Isaac, Lankester, Arjan, Bierings, Marc, Yesilipek, Akif, Sedlacek, Petr, Ifversen, Marianne, Svec, Peter, Toporski, Jacek, Gungor, Taifun, Wachowiak, Jacek, Glogova, Evgenia, Poetschger, Ulrike, and Peters, Christina

Published
2021
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17. Supportive care during pediatric hematopoietic stem cell transplantation: beyond infectious diseases. A report from workshops on supportive care of the Pediatric Diseases Working Party (PDWP) of the European Society for Blood and Marrow Transplantation (EBMT)

Author

Nava, Tiago, Ansari, Marc, Dalle, Jean-Hugues, de Heredia, Christina Diaz, Güngör, Tayfun, Trigoso, Eugenia, Falkenberg, Ulrike, Bertaina, Alice, Gibson, Brenda, Jarisch, Andrea, Balduzzi, Adriana, Boenig, Halvard, Krivan, Gergely, Vettenranta, Kim, Matic, Toni, Buechner, Jochen, Kalwak, Krzysztof, Lawitschka, Anita, Yesilipek, Akif, Lucchini, Giovanna, Peters, Christina, Turkiewicz, Dominik, Niinimäki, Riitta, Diesch, Tamara, Lehrnbecher, Thomas, Sedlacek, Petr, Hutt, Daphna, Dalissier, Arnaud, Wachowiak, Jacek, Yaniv, Isaac, Stein, Jerry, Yalçin, Koray, Sisinni, Luisa, Deiana, Marco, Ifversen, Marianne, Kuhlen, Michaela, Meisel, Roland, Bakhtiar, Shahrzad, Cesaro, Simone, Willasch, Andre, Corbacioglu, Selim, and Bader, Peter

Published
2020
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18. Transplantation in Children and Adolescents with Acute Lymphoblastic Leukemia from a Matched Donor versus an HLA-Identical Sibling: Is the Outcome Comparable? Results from the International BFM ALL SCT 2007 Study

Author

Balduzzi, Adriana, Dalle, Jean-Hugues, Wachowiak, Jacek, Yaniv, Isaac, Yesilipek, Akif, Sedlacek, Petr, Bierings, Marc, Ifversen, Marianne, Sufliarska, Sabina, Kalwak, Krzysztof, Lankester, Arjan, Toporski, Jacek, Di Maio, Lucia, Glogova, Evgenia, Poetschger, Ulrike, and Peters, Christina

Published
2019
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19. Allogeneic Stem Cell Transplantation from HLA-Mismatched Donors for Pediatric Patients with Acute Lymphoblastic Leukemia Treated According to the 2003 BFM and 2007 International BFM Studies: Impact of Disease Risk on Outcomes

Author

Dalle, Jean-Hugues, Balduzzi, Adriana, Bader, Peter, Lankester, Arjan, Yaniv, Isaac, Wachowiak, Jacek, Pieczonka, Anna, Bierings, Marc, Yesilipek, Akif, Sedlaçek, Petr, Ifversen, Marianne, Sufliarska, Sabina, Toporski, Jacek, Glogova, Evgenia, Poetschger, Ulrike, and Peters, Christina

Published
2018
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20. 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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21. Comparison of tacrolimus vs. cyclosporine in pediatric hematopoietic stem cell transplantation for thalassemia.

Author

Zhumatayev, Suleimen, Yalcin, Koray, Celen, Safiye Suna, Karaman, Irem, Daloglu, Hayriye, Ozturkmen, Seda, Uygun, Vedat, Karasu, Gulsun, and Yesilipek, Akif

Subjects
HEPATIC veno-occlusive disease, STEM cell transplantation, HEMATOPOIETIC stem cell transplantation, TACROLIMUS, CYCLOSPORINE, THALASSEMIA, CHILD patients, GRAFT versus host disease
Abstract

Objectives: Graft‐versus‐host disease (GvHD) is one of the leading causes of morbidity and mortality in patients undergoing allogeneic HSCT, and effective prevention of GvHD is critical for the success of the HSCT procedure. Calcineurin inhibitors (CNI) have been used for decades as the backbone of GvHD prophylaxis. In this study, the efficacy and safety of Cyclosporine A (CsA) and tacrolimus (TCR) were compared in pediatric HSCT for thalassemia. Materials and Methods: This is a retrospective analysis of 129 pediatric patients who underwent HSCT with the diagnosis of thalassemia at Medicalpark Göztepe and Antalya Hospitals between January 2017 and December 2020. Results: Despite the GvHD prophylaxis, grade II–IV acute GvHD developed in 29 patients. Of these patients, 12 had only gut, 10 had only skin, 6 had combined gut and skin, and one had only liver GvHD. Fifteen of these 29 patients were in the CsA group, and 14 of them were in the TCR group. There was no significant difference between the groups in terms of acute GvHD occurrence, GvHD stage, or involvement sites. In terms of CNI‐related toxicity, neurotoxicity in 15 (CsA n = 9, TCR n = 6) and nephrotoxicity in 18 (CsA n = 4, TCR n = 14) patients were observed. While there was no difference between the two groups in terms of neurotoxicity, more nephrotoxicity developed in patients using TCR (p =.013). There was no significant difference between the groups in terms of engraftment syndrome, veno‐occlusive disease, CMV reactivation, PRES, or graft rejection. Conclusion: Regarding GvHD, there was no difference in efficacy between TCR and CsA usage. Patients taking TCR experienced noticeably higher nephrotoxicity in terms of adverse effects. This difference should be considered according to the patient's clinical situation while choosing a CNI. [ABSTRACT FROM AUTHOR]

Published
2024
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22. Different Kinetics and Risk Factors for Isolated Extramedullary Relapse after Allogeneic Hematopoietic Stem Cell Transplantation in Children with Acute Leukemia

Author

Hazar, Volkan, Öztürk, Gülyüz, Yalçın, Koray, Uygun, Vedat, Aksoylar, Serap, Küpesiz, A., Ok Bozkaya, İkbal, Karagün, Barbaros Şahin, Bozkurt, Ceyhun, İleri, Talia, Atay, Didem, Koçak, Ülker, Karasu, Gülsün Tezcan, Yeşilipek, Akif, Gökçe, Müge, Kansoy, Savaş, Kintrup, Gülen Tüysüz, Karakükcü, Musa, Okur, Fatma Visal, Ertem, Mehmet, Kaya, Zühre, Gürsel, Orhan, Yaman, Yöntem, Özbek, Namık, Antmen, Bülent, Tüfekçi, Özlem, Albayrak, Canan, Adaklı Aksoy, Başak, Sezgin, Gülay, Albayrak, Davut, Evim, Melike Sezgin, Zengin, Emine, and Pekpak, Esra

Published
2021
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23. Outcomes of Unmanipulated Haploidentical Transplantation Using Post-Transplant Cyclophosphamide (PT-Cy) in Pediatric Patients With Acute Lymphoblastic Leukemia

Author

Ruggeri, Annalisa, Galimard, Jacques-Emmanuel, Paina, Olesya, Fagioli, Franca, Tbakhi, Abdelghani, Yesilipek, Akif, Navarro, José Maria Fernandez, Faraci, Maura, Hamladji, Rose-Marie, Skorobogatova, Elena, Al-Seraihy, Amal, Sundin, Mikael, Herrera, Concepcion, Rifón, Jose, Dalissier, Arnaud, Locatelli, Franco, Rocha, Vanderson, and Corbacioglu, Selim

Published
2021
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24. Hematopoietic stem cell transplantation in children with mucopolysaccharidosis IVA: single center experience

Author

Yalcin, Koray, Uygun, Vedat, Ozturk Hismi, Burcu, Celen, Suna, Ozturkmen, Seda, Zhumatayev, Suleimen, Daloglu, Hayriye, Karasu, Gülsün, and Yesilipek, Akif

Abstract

Mucopolysaccharidosis IVA (MPS IVA; Morquio syndrome) is a lysosomal storage disorder and features systemic skeletal dysplasia that is caused by defective Nacetylgalactosamine-6-sulfate sulfatase (GALNS). Although there are convincing data for hematopoietic stem cell transplantation (HSCT) in certain types of MPS, the studies are limited for MPS IVA and more data is still pending to show the efficacy/safety of HSCT. This study included 3 girls and 7 boys, with a median age of 75,5 months (35–186 months), who underwent allogeneic HSCT for severe MPS IVA between February 12, 2021, and March 10, 2023. Enzyme levels, height growth, the most involved organs (ear, eye, and heart), and the activities of daily living (ADL) scoring system were monitored to assess the benefit of HSCT. In a median follow-up of 20 months (9–34 months), there is no severe transplant-related adverse event was observed. In all cases, normal enzyme levels were reached after HSCT. During the short follow-up period, our cases showed an increase in stature and improvement in daily activity functions. Here we present the data of our HSCT experience in MPS IVA with promising results regarding both safety and efficacy. Although there are signs of amelioration with HSCT, we need more data and long-term follow-up to comment properly on the benefits of HSCT in MPS IVA.

Published
2024
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25. Stem cell transplantation in severe congenital neutropenia: an analysis from the European Society for Blood and Marrow Transplantation

Author

Fioredda, Francesca, Iacobelli, Simona, van Biezen, Anja, Gaspar, Bobby, Ancliff, Phil, Donadieu, Jean, Aljurf, Mahmoud, Peters, Christina, Calvillo, Michaela, Matthes-Martin, Susanne, Morreale, Giuseppe, van 't Veer-Tazelaar, Nelleke, de Wreede, Liesbeth, Al Seraihy, Amal, Yesilipek, Akif, Fischer, Alain, Bierings, Marc, Ozturk, Gulyuz, Smith, Owen, Veys, Paul, Ljungman, Per, Peffault de Latour, Régis, Sánchez de Toledo Codina, José, Or, Reuven, Ganser, Arnold, Afanasyev, Boris, Wynn, Robert, Kalwak, Krzysztof, Marsh, Judith, and Dufour, Carlo

Published
2015
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26. Correction: ABO incompatibile graft management in pediatric transplantation

Author

Balduzzi, Adriana, Bönig, Halvard, Jarisch, Andrea, Nava, Tiago, Ansari, Marc, Cattoni, Alessandro, Prunotto, Giulia, Lucchini, Giovanna, Krivan, Gergely, Matic, Toni, Kalwak, Krzyzstof, Yesilipek, Akif, Ifversen, Marianne, Svec, Peter, Buechner, Jochen, Vettenranta, Kim, Meisel, Roland, Lawitschka, Anita, Peters, Christina, Gibson, Brenda, Dalissier, Arnaud, Corbacioglu, Selim, Willasch, André, Dalle, Jean-Hugues, and Bader, Peter

Published
2021
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27. Pre‐transplantation vitamin D deficiency increases acute graft‐versus‐host disease after hematopoietic stem cell transplantation in thalassemia major patients

Author

Daloğlu, Hayriye, Uygun, Vedat, Ozturkmen, Seda, Yalcın, Koray, Karasu, Gulsu, Yesilipek, Akif, İstinye Üniversitesi, Tıp Fakültesi, Dahili Tıp Bilimleri Bölümü, Uygun, Vedat, and AGH-4534-2022

Subjects
Transplantation, Acute Graft Versus Host Disease, Hematopoetic Stem Cell Transplantation, Vitamin D, Children, Thalassemia Major
Abstract

Background: Although there are many studies on the role of vitamin D deficiency (VDD) in hematopoetic stem cell transplantation (HSCT), outcomes have often reported conflicting results because of the heterogeneity of the patients in the studies. Methods: We investigated the association between VDD prior to HSCT and outcomes after HSCT in a relatively homogenous group of patients with thalassemia major (TM) who received identical treatment for TM before transplantation, and the same conditioning regimen and GVHD prophylaxis during and after transplantation. All patients, including the patients with normal vitamin D3 levels received 400 to 800 IU per day of vitamin D for the first 6 months after HSCT. Results: Pre-HSCT VDD increased the frequency of aGVHD after transplantation, particularly in HSCTs performed with PBSC for the stem cell source. Pre-transplant low vitamin D3 levels had no association with transplant outcomes such as engraftment, viral infections, alloimmunization, chronic GvHD, total days of hospitalization, and success in terms of transfusion independence. Conclusions: Low vitamin D3 levels before HSCT carry a significant risk for aGVHD. All patients with TM should be screened for VDD before HSCT, and every effort should be made to supplement vitamin D before the transplant in VDD patients 36461145

Published
2022

28. Correction: Supportive care during pediatric hematopoietic stem cell transplantation: beyond infectious diseases. A report from workshops on supportive care of the Pediatric Diseases Working Party (PDWP) of the European Society for Blood and Marrow Transplantation (EBMT)

Author

Nava, Tiago, Ansari, Marc, Dalle, Jean-Hugues, de Heredia, Christina Diaz, Güngör, Tayfun, Trigoso, Eugenia, Falkenberg, Ulrike, Bertaina, Alice, Gibson, Brenda, Jarisch, Andrea, Balduzzi, Adriana, Boenig, Halvard, Krivan, Gergely, Vettenranta, Kim, Matic, Toni, Buechner, Jochen, Kalwak, Krzysztof, Lawitschka, Anita, Yesilipek, Akif, Lucchini, Giovanna, Peters, Christina, Turkiewicz, Dominik, Niinimäki, Riitta, Diesch, Tamara, Lehrnbecher, Thomas, Sedlacek, Petr, Hutt, Daphna, Dalissier, Arnaud, Wachowiak, Jacek, Yaniv, Isaac, Stein, Jerry, Yalçin, Koray, Sisinni, Luisa, Deiana, Marco, Ifversen, Marianne, Kuhlen, Michaela, Meisel, Roland, Bakhtiar, Shahrzad, Cesaro, Simone, Willasch, Andre, Corbacioglu, Selim, and Bader, Peter

Published
2020
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29. Superior Graft-Versus-Leukemia Effect in Matched Unrelated Donor Versus HLA-Identical Sibling Pediatric Recipients Transplanted for Acute Lymphoblastic Leukemia within the Forum Study

Author

Dalle, Jean-Hugues, primary, Bader, Peter, additional, Yesilipek, Akif, additional, Locatelli, Franco, additional, Palma, Julia, additional, Wachowiak, Jacek, additional, Pichler, Herbert, additional, Ifversen, Marianne, additional, Kriván, Gergely, additional, Buechner, Jochen, additional, Díaz-de-Heredia, Cristina, additional, Bierings, Marc, additional, Staciuk, Raquel, additional, Gungor, Tayfun, additional, Toporski, Jacek, additional, Balduzzi, Adriana, additional, Poetschger, Ulrike, additional, Peters, Christina, additional, and Sedlacek, Petr, additional

Published
2022
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30. Outcome of Haematopoietic Cell Transplantation in 813 Children with Fanconi Anaemia: A Study on Behalf of the EBMT Severe Aplastic Anaemia Working Party and Paediatric Disease Working Party

Author

Lum, Su Han, primary, Samarasinghe, Sujith, additional, Eikema, Dirk-Jan, additional, Piepenbroek, Brian, additional, Dalissier, Arnaud, additional, Ayas, Mouhab, additional, Mousavi, Ashrafsadat, additional, Hamladji, Rose-Marie, additional, Yesilipek, Akif, additional, Dalle, Jean-Hugues, additional, Kansoy, Savas, additional, Locatelli, Franco, additional, Çetinkaya, Duygu, additional, Bierings, Marc, additional, Kupesiz, O. Alphan, additional, Tbakhi, Abdelghani, additional, Skorobogatova, Elena, additional, Öztürk, Gülyüz, additional, Faraci, Maura, additional, Bertrand, Yves, additional, Evans, Pamela, additional, Carbacioglu, Selim, additional, Dufour, Carlo, additional, Risitano, Antonio, additional, Wynn, Robert F, additional, and Peffault de Latour, Régis, additional

Published
2022
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31. Relapse Is the Most Common Treatment Failure Post HSCT in Children with ALL below 4 Years of Age Given a Chemo-Based Conditioning Regimen. Results from the Prospective Multinational Forum-Trial

Author

Peters, Christina, primary, Poetschger, Ulrike, additional, Dalle, Jean-Hugues, additional, Yesilipek, Akif, additional, Stein, Jerry, additional, Pichler, Herbert, additional, Balduzzi, Adriana, additional, Locatelli, Franco, additional, Sedlacek, Petr, additional, Wachowiak, Jacek, additional, Güngör, Tayfun, additional, Ifversen, Marianne, additional, Renard, Marleen, additional, Truong, Tony H, additional, Staciuk, Raquel, additional, Shaw, Peter J., additional, Kriván, Gergely, additional, Büchner, Jochen, additional, and Bader, Peter, additional

Published
2022
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37. 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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39. Combined cord blood and bone marrow transplantation from the same human leucocyte antigen-identical sibling donor for children with malignant and non-malignant diseases

Author

Tucunduva, Luciana, Volt, Fernanda, Cunha, Renato, Locatelli, Franco, Zecca, Marco, Yesilipek, Akif, Caniglia, Maurizio, Güngör, Tayfun, Aksoylar, Serap, Fagioli, Franca, Bertrand, Yves, Addari, Maria Carmen, de la Fuente, Josu, Winiarski, Jacek, Biondi, Andrea, Sengeloev, Henrik, Badell, Isabel, Mellgren, Karin, de Heredia, Cristina Díaz, Sedlacek, Petr, Vora, Ajay, Rocha, Vanderson, Ruggeri, Annalisa, and Gluckman, Eliane

Published
2015
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42. Supportive Care During Pediatric Hematopoietic Stem Cell Transplantation: Prevention of Infections. A Report From Workshops on Supportive Care of the Paediatric Diseases Working Party (PDWP) of the European Society for Blood and Marrow Transplantation (EBMT)

Author

Ifversen, Marianne, primary, Meisel, Roland, additional, Sedlacek, Petr, additional, Kalwak, Krzysztof, additional, Sisinni, Luisa, additional, Hutt, Daphna, additional, Lehrnbecher, Thomas, additional, Balduzzi, Adriana, additional, Diesch, Tamara, additional, Jarisch, Andrea, additional, Güngör, Tayfun, additional, Stein, Jerry, additional, Yaniv, Isaac, additional, Bonig, Halvard, additional, Kuhlen, Michaela, additional, Ansari, Marc, additional, Nava, Tiago, additional, Dalle, Jean-Hugues, additional, Diaz-de-Heredia, Cristina, additional, Trigoso, Eugenia, additional, Falkenberg, Ulrike, additional, Hartmann, Mihaela, additional, Deiana, Marco, additional, Canesi, Marta, additional, Broggi, Chiara, additional, Bertaina, Alice, additional, Gibson, Brenda, additional, Krivan, Gergely, additional, Vettenranta, Kim, additional, Matic, Toni, additional, Buechner, Jochen, additional, Lawitschka, Anita, additional, Peters, Christina, additional, Yesilipek, Akif, additional, Yalçin, Koray, additional, Lucchini, Giovanna, additional, Bakhtiar, Shahrzad, additional, Turkiewicz, Dominik, additional, Niinimäki, Riitta, additional, Wachowiak, Jacek, additional, Cesaro, Simone, additional, Dalissier, Arnaud, additional, Corbacioglu, Selim, additional, Willasch, Andre Manfred, additional, and Bader, Peter, additional

Published
2021
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43. ABO incompatibile graft management in pediatric transplantation

Author

Balduzzi, Adriana, Bönig, Halvard, Jarisch, Andrea, Nava, Tiago, Ansari Djaberi, Marc Georges, Cattoni, Alessandro, Prunotto, Giulia, Lucchini, Giovanna, Krivan, Gergely, Matic, Toni, Kalwak, Krzyzstof, Yesilipek, Akif, Ifversen, Marianne, Svec, Peter, Buechner, Jochen, Vettenranta, Kim, Meisel, Roland, Lawitschka, Anita, Peters, Christina, Gibson, Brenda, Dalissier, Arnaud, Corbacioglu, Selim, Willasch, André, Dalle, Jean-Hugues, Bader, Peter, EBMT Pediatric Diseases Working Party, Balduzzi, A, Bönig, H, Järisch, A, Nava, T, Ansari, M, Cattoni, A, Prunotto, G, Lucchini, G, Krivan, G, Matic, T, Kalwak, K, Yesilipek, A, Ifversen, M, Svec, P, Büchner, J, Vettenranta, K, Meisel, R, Lawitschka, A, Peters, C, Gibson, B, Dalissier, A, Corbacioglu, S, Willasch, A, Dalle, J, Bader, P, HUS Children and Adolescents, Lastentautien yksikkö, Children's Hospital, and Helsinki University Hospital Area

Subjects
medicine.medical_specialty, Erythrocytes, BONE-MARROW, medicine.medical_treatment, Pure red cell aplasia, Hematopoietic stem cell transplantation, Hemolysis, Isohemagglutinin, ABO Blood-Group System, ABO incompatibility, 03 medical and health sciences, 0302 clinical medicine, TRANSFUSION, 3123 Gynaecology and paediatrics, ABO blood group system, medicine, Humans, Child, Bone Marrow Transplantation, Transplantation, ddc:618, PLASMA, business.industry, Hematology, medicine.disease, 3. Good health, Surgery, REDUCTION, medicine.anatomical_structure, pediatric, Tolerability, Blood Group Incompatibility, 030220 oncology & carcinogenesis, hematopoietic stem cell transplantation, Erythropoiesis, PROGENITOR-CELL TRANSPLANTATION, Bone marrow, DEPLETION, business, 030215 immunology
Abstract

Up to 40% of donor-recipient pairs in SCT have some degree of ABO incompatibility, which may cause severe complications. The aim of this study was to describe available options and survey current practices by means of a questionnaire circulated within the EBMT Pediatric Diseases Working Party investigators. Major ABO incompatibility (donor's RBCs have antigens missing on the recipient's cell surface, towards which the recipient has circulating isohemagglutinins) requires most frequently an intervention in case of bone marrow grafts, as immediate or delayed hemolysis, delayed erythropoiesis and pure red cell aplasia may occur. RBC depletion from the graft (82%), recipient plasma-exchange (14%) were the most common practices, according to the survey. Graft manipulation is rarely needed in mobilized peripheral blood grafts. In case of minor incompatible grafts (donor has isohemagglutinins directed against recipient RBC antigens), isohemagglutinin depletion from the graft by plasma reduction/centrifugation may be considered, but acute tolerability of minor incompatible grafts is rarely an issue. According to the survey, minor ABO incompatibility was either managed by means of plasma removal from the graft, especially when isohemagglutinin titer was above a certain threshold, or led to no intervention at all (41%). Advantages and disadvantages of each method are discussed.

Published
2021

44. ABO incompatibile graft management in pediatric transplantation

Author

Balduzzi, A, Bönig, H, Järisch, A, Nava, T, Ansari, M, Cattoni, A, Prunotto, G, Lucchini, G, Krivan, G, Matic, T, Kalwak, K, Yesilipek, A, Ifversen, M, Svec, P, Büchner, J, Vettenranta, K, Meisel, R, Lawitschka, A, Peters, C, Gibson, B, Dalissier, A, Corbacioglu, S, Willasch, A, Dalle, J, Bader, P, Balduzzi, Adriana, Bönig, Halvard, Järisch, Andrea, Nava, Tiago, Ansari, Marc, Cattoni, Alessandro, Prunotto, Giulia, Lucchini, Giovanna, Krivan, Gergely, Matic, Toni, Kalwak, Krzyzstof, Yesilipek, Akif, Ifversen, Marianne, Svec, Peter, Büchner, Jochen, Vettenranta, Kim, Meisel, Roland, Lawitschka, Anita, Peters, Christina, Gibson, Brenda, Dalissier, Arnaud, Corbacioglu, Selim, Willasch, André, Dalle, Jean-Hugues, Bader, Peter, Balduzzi, A, Bönig, H, Järisch, A, Nava, T, Ansari, M, Cattoni, A, Prunotto, G, Lucchini, G, Krivan, G, Matic, T, Kalwak, K, Yesilipek, A, Ifversen, M, Svec, P, Büchner, J, Vettenranta, K, Meisel, R, Lawitschka, A, Peters, C, Gibson, B, Dalissier, A, Corbacioglu, S, Willasch, A, Dalle, J, Bader, P, Balduzzi, Adriana, Bönig, Halvard, Järisch, Andrea, Nava, Tiago, Ansari, Marc, Cattoni, Alessandro, Prunotto, Giulia, Lucchini, Giovanna, Krivan, Gergely, Matic, Toni, Kalwak, Krzyzstof, Yesilipek, Akif, Ifversen, Marianne, Svec, Peter, Büchner, Jochen, Vettenranta, Kim, Meisel, Roland, Lawitschka, Anita, Peters, Christina, Gibson, Brenda, Dalissier, Arnaud, Corbacioglu, Selim, Willasch, André, Dalle, Jean-Hugues, and Bader, Peter

Abstract

Up to 40% of donor-recipient pairs in SCT have some degree of ABO incompatibility, which may cause severe complications. The aim of this study was to describe available options and survey current practices by means of a questionnaire circulated within the EBMT Pediatric Diseases Working Party investigators. Major ABO incompatibility (donor’s RBCs have antigens missing on the recipient’s cell surface, towards which the recipient has circulating isohemagglutinins) requires most frequently an intervention in case of bone marrow grafts, as immediate or delayed hemolysis, delayed erythropoiesis and pure red cell aplasia may occur. RBC depletion from the graft (82%), recipient plasma-exchange (14%) were the most common practices, according to the survey. Graft manipulation is rarely needed in mobilized peripheral blood grafts. In case of minor incompatible grafts (donor has isohemagglutinins directed against recipient RBC antigens), isohemagglutinin depletion from the graft by plasma reduction/centrifugation may be considered, but acute tolerability of minor incompatible grafts is rarely an issue. According to the survey, minor ABO incompatibility was either managed by means of plasma removal from the graft, especially when isohemagglutinin titer was above a certain threshold, or led to no intervention at all (41%). Advantages and disadvantages of each method are discussed.

Published
2021

45. Supportive care during pediatric hematopoietic stem cell transplantation: prevention of infections. A report from workshops on supportive care of the Paediatric Diseases Working Party (PDWP) of the European Society for Blood and Marrow Transplantation (EBMT)

Author

Ifversen, Marianne, Meisel, Roland, Sedlacek, Petr, Kalwak, Krzysztof, Sisinni, Luisa, Hutt, Daphna, Lehrnbecher, Thomas, Balduzzi, Adriana, Diesch, Tamara, Jarisch, Andrea, Güngör, Tayfun, Stein, Jerry, Yaniv, Isaac, Bönig, Halvard-Björn, Kuhlen, Michaela, Ansari, Marc, Nava, Tiago, Dalle, Jean-Hugues, Diaz-de-Heredia, Cristina, Trigoso, Eugenia, Falkenberg, Ulrike, Hartmann, Mihaela, Deiana, Marco, Canesi, Marta, Broggi, Chiara, Bertaina, Alice, Gibson, Brenda, Krivan, Gergely, Vettenranta, Kim, Matic, Toni, Büchner, Jochen, Lawitschka, Anita, Peters, Christina, Yesilipek, Akif, Yalçin, Koray, Lucchini, Giovanna, Bakhtiar, Shahrzad, Turkiewicz, Dominik, Niinimäki, Riitta, Wachowiak, Jacek, Cesaro, Simone, Dalissier, Arnaud, Corbacioglu, Selim, Willasch, Andre Manfred, Bader, Peter, Ifversen, Marianne, Meisel, Roland, Sedlacek, Petr, Kalwak, Krzysztof, Sisinni, Luisa, Hutt, Daphna, Lehrnbecher, Thomas, Balduzzi, Adriana, Diesch, Tamara, Jarisch, Andrea, Güngör, Tayfun, Stein, Jerry, Yaniv, Isaac, Bönig, Halvard-Björn, Kuhlen, Michaela, Ansari, Marc, Nava, Tiago, Dalle, Jean-Hugues, Diaz-de-Heredia, Cristina, Trigoso, Eugenia, Falkenberg, Ulrike, Hartmann, Mihaela, Deiana, Marco, Canesi, Marta, Broggi, Chiara, Bertaina, Alice, Gibson, Brenda, Krivan, Gergely, Vettenranta, Kim, Matic, Toni, Büchner, Jochen, Lawitschka, Anita, Peters, Christina, Yesilipek, Akif, Yalçin, Koray, Lucchini, Giovanna, Bakhtiar, Shahrzad, Turkiewicz, Dominik, Niinimäki, Riitta, Wachowiak, Jacek, Cesaro, Simone, Dalissier, Arnaud, Corbacioglu, Selim, Willasch, Andre Manfred, and Bader, Peter

Abstract

Specific protocols define eligibility, conditioning, donor selection, graft composition and prophylaxis of graft vs. host disease for children and young adults undergoing hematopoietic stem cell transplant (HSCT). However, international protocols rarely, if ever, detail supportive care, including pharmaceutical infection prophylaxis, physical protection with face masks and cohort isolation or food restrictions. Supportive care suffers from a lack of scientific evidence and implementation of practices in the transplant centers brings extensive restrictions to the child's and family's daily life after HSCT. Therefore, the Board of the Pediatric Diseases Working Party (PDWP) of the European Society for Blood and Marrow Transplantation (EBMT) held a series of dedicated workshops since 2017 with the aim of initiating the production of a set of minimal recommendations. The present paper describes the consensus reached within the field of infection prophylaxis.

Published
2021

46. Stem Cell Transplantation for Diamond–Blackfan Anemia. A Retrospective Study on Behalf of the Severe Aplastic Anemia Working Party of the European Blood and Marrow Transplantation Group (EBMT)

Author

Miano, Maurizio, Eikema, Dirk Jan, de la Fuente, Josu, Bosman, Paul, Ghavamzadeh, Ardeshir, Smiers, Frans, Sengeløv, Henrik, Yesilipek, Akif, Formankova, Renata, Bader, Peter, Díaz Pérez, Miguel Ángel, Bertrand, Yves, Niemeyer, Charlotte, Diallo, Safiatou, Ansari, Marc, Bykova, Tatiana A., Faraci, Maura, Bonanomi, Sonia, Gozdzik, Jolanta, Satti, Tariq Mahmood, Bodova, Ivana, Wölfl, Matthias, Rocha, Vanderson G., Mellgren, Karin, Rascon, Jelena, Holter, Wolfgang, Lange, Andrzej, Meisel, Roland, Beguin, Yves, Mozo, Yasmina, Kriván, Gergely, Sirvent, Anne, Bruno, Benedicte, Dalle, Jean Hugues, Onofrillo, Daniela, Giardino, Stefano, Risitano, Antonio M., de Latour, Régis Peffault, Dufour, Carlo, Miano, Maurizio, Eikema, Dirk Jan, de la Fuente, Josu, Bosman, Paul, Ghavamzadeh, Ardeshir, Smiers, Frans, Sengeløv, Henrik, Yesilipek, Akif, Formankova, Renata, Bader, Peter, Díaz Pérez, Miguel Ángel, Bertrand, Yves, Niemeyer, Charlotte, Diallo, Safiatou, Ansari, Marc, Bykova, Tatiana A., Faraci, Maura, Bonanomi, Sonia, Gozdzik, Jolanta, Satti, Tariq Mahmood, Bodova, Ivana, Wölfl, Matthias, Rocha, Vanderson G., Mellgren, Karin, Rascon, Jelena, Holter, Wolfgang, Lange, Andrzej, Meisel, Roland, Beguin, Yves, Mozo, Yasmina, Kriván, Gergely, Sirvent, Anne, Bruno, Benedicte, Dalle, Jean Hugues, Onofrillo, Daniela, Giardino, Stefano, Risitano, Antonio M., de Latour, Régis Peffault, and Dufour, Carlo

Abstract

Data on stem cell transplantation (SCT) for Diamond–Blackfan Anemia (DBA) is limited. We studied patients transplanted for DBA and registered in the EBMT database. Between 1985 and 2016, 106 DBA patients (median age, 6.8 years) underwent hematopoietic stem cell transplantation from matched-sibling donors (57%), unrelated donors (36%), or other related donors (7%), using marrow (68%), peripheral blood stem cells (20%), both marrow and peripheral blood stem cells (1%), or cord blood (11%). The cumulative incidence of engraftment was 86% (80% to 93%), and neutrophil recovery and platelet recovery were achieved on day +18 (range, 16 to 20) and +36 (range, 32 to 43), respectively. Three-year overall survival and event-free survival were 84% (77% to 91%) and 81% (74% to 89%), respectively. Older patients were significantly more likely to die (hazard ratio, 1.4; 95% confidence interval, 1.06 to 1.23; P <.001). Outcomes were similar between sibling compared to unrelated-donor transplants. The incidence of acute grades II to IV of graft-versus-host disease (GVHD) was 30% (21% to 39%), and the incidence of extensive chronic GVHD was 15% (7% to 22%). This study shows that SCT may represent an alternative therapeutic option for transfusion-dependent younger patients.

Published
2021

47. The impact of donor type on the outcome of pediatric patients with very high risk acute lymphoblastic leukemia. A study of the ALL SCT 2003 BFM-SG and 2007-BFM-International SG

Author

Dalle, Jean-Hugues; https://orcid.org/0000-0002-8406-0793, Balduzzi, Adriana; https://orcid.org/0000-0002-5879-0610, Bader, Peter, Pieczonka, Anna; https://orcid.org/0000-0001-5922-4242, Yaniv, Isaac, Lankester, Arjan, Bierings, Marc; https://orcid.org/0000-0002-0192-3080, Yesilipek, Akif, Sedlacek, Petr, Ifversen, Marianne, Svec, Peter; https://orcid.org/0000-0002-7647-2253, Toporski, Jacek; https://orcid.org/0000-0001-6012-0651, Güngör, Tayfun; https://orcid.org/0000-0002-3261-1186, Wachowiak, Jacek, Glogova, Evgenia, Poetschger, Ulrike, Peters, Christina; https://orcid.org/0000-0003-0369-8515, Dalle, Jean-Hugues; https://orcid.org/0000-0002-8406-0793, Balduzzi, Adriana; https://orcid.org/0000-0002-5879-0610, Bader, Peter, Pieczonka, Anna; https://orcid.org/0000-0001-5922-4242, Yaniv, Isaac, Lankester, Arjan, Bierings, Marc; https://orcid.org/0000-0002-0192-3080, Yesilipek, Akif, Sedlacek, Petr, Ifversen, Marianne, Svec, Peter; https://orcid.org/0000-0002-7647-2253, Toporski, Jacek; https://orcid.org/0000-0001-6012-0651, Güngör, Tayfun; https://orcid.org/0000-0002-3261-1186, Wachowiak, Jacek, Glogova, Evgenia, Poetschger, Ulrike, and Peters, Christina; https://orcid.org/0000-0003-0369-8515

Abstract

Allogeneic HSCT represents the only potentially curative treatment for very high risk (VHR) ALL. Two consecutive international prospective studies, ALL-SCT-(I)BFM 2003 and 2007 were conducted in 1150 pediatric patients. 569 presented with VHR disease leading to any kind of HSCT. All patients >2 year old were transplanted after TBI-based MAC. The median follow-up was 5 years. 463 patients were transplanted from matched donor (MD) and 106 from mismatched donor (MMD). 214 were in CR1. Stem cell source was unmanipulated BM for 330 patients, unmanipulated PBSC for 135, ex vivo T-cell depleted PBSC for 62 and cord-blood for 26. There were more advanced disease, more ex vivo T-cell depletion, and more chemotherapy based conditioning regimen for patients transplanted from MMD as compared to those transplanted from MSD or MD. Median follow up (reversed Kaplan Meier estimator) was 4.99 years, median follow up of survivals was 4.88, range (0.01–11.72) years. The 4-year CI of extensive cGvHD was 13 ± 2% and 17 ± 4% (p = NS) for the patients transplanted from MD and MMD, respectively. 4-year EFS was statistically better for patients transplanted from MD (60 ± 2% vs. 42 ± 5%, p < 0.001) for the whole cohort. This difference does not exist if considering separately patients treated in the most recent study. There was no difference in 4-year CI of relapse. The 4-year NRM was lower for patients transplanted from MD (9 ± 1% vs. 23 ± 4%, p < 0.001). In multivariate analysis, donor-type appears as a negative risk-factor for OS, EFS, and NRM. This paper demonstrates the impact of donor type on overall results of allogeneic stem cell transplantation for very-high risk pediatric acute lymphoblastic leukemia with worse results when using MMD stem cell source.

Published
2021

48. Outcome of Children with B-Cell Precursor Acute Lymphoblastic Leukemia (BCP-ALL) with Hypodiploidy or BCR-ABL1 Fusion Given Allogeneic Hematopoietic Stem Cell Transplantation (HSCT): Results from the Prospective Forum Study

Author

Buechner, Jochen, Poetschger, Ulrike, Kurzmann, Paulina, Bader, Peter, Dalle, Jean-Hugues, Yesilipek, Akif, Pichler, Herbert, Palma, Julia, Staciuk, Raquel, Sedlacek, Petr, Krivan, Gergely, Ifversen, Marianne, Güngör, Tayfun, Kitra Rousou, Vassiliki, Kalwak, Krzysztof, Toporski, Jacek, Shaw, Peter J., Renard, Marleen, Díaz De Heredia, Cristina, Matic, Toni, Bierings, Marc, Svec, Peter, Meisel, Roland, Balduzzi, Adriana, Locatelli, Franco, Peters, Christina, and Stein, Jerry

Published
2023
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49. Deviating from safety guidelines during deferiprone therapy in clinical practice may not be associated with higher risk of agranulocytosis

Author

Elalfy, Mohssen, Wali, Yasser A., Qari, Mohamad, Al Damanhouri, Ghazi, Al-Tonbary, Youssef, Yazman, Dilek, Al Hawsawi, Zakaria, Karakas, Zeynep, Kilinc, Yurdanur, Yesilipek, Akif M., Badr, Mohamed, Elsafy, Usama, Salama, Mostafa, Rahman, Yousryeia Abdel, Shebl, Shebl, Stilman, Anne, Temin, Noemi Toiber, and Tricta, Fernando

Published
2014
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