Article
Biophysics
Juergen Finke, Claudia Schmoor, Matthias Stelljes, Andreas Burchert, Peter Dreger, Ute Hegenbart, Eva-Maria Wagner-Drouet, Martin Bornhaeuser, Kristina Sohlbach, Natalie Schub, Christian Reicherts, Guido Kobbe, Bertram Glass, Hartmut Bertz, Olga Grishina
Summary: This study aimed to investigate the efficacy of a second allogeneic HCT using Thiotepa, Fludarabine, and Treosulfan (TFT) as conditioning therapy in patients with AML relapse > 6 months after the first allogeneic HCT. The results showed that this approach is feasible and has a high anti-leukemic efficacy, but relapse rates and non-relapse mortality remain a challenge.
BONE MARROW TRANSPLANTATION
(2022)
Article
Hematology
Ioanna Sakellari, Eleni Gavriilaki, Despina Mallouri, Ioannis Batsis, Christos Varelas, Sofia Tagara, Zoi Bousiou, Maria Papathanasiou, Anna Vardi, Apostolia Papalexandri, Chrysanthi Vadikoliou, Anastasia Athanasiadou, Chrysavgi Lalayanni, Asimina Fylaktou, Konstantinos Antoniadis, Achilles Anagnostopoulos
Summary: Our study confirms the advantages of using FluTreo in HCT compared to previous regimens, showing favorable outcomes in older patients with higher comorbidity index. A reevaluation is needed in the selection of HCT for patients of a rather older age and comorbidity index.
TRANSPLANTATION AND CELLULAR THERAPY
(2021)
Article
Hematology
Krischan Braitsch, Alix Schwarz, Katrin Koch, Mara Hubbuch, Helge Menzel, Ulrich Keller, Katharina S. Goetze, Florian Bassermann, Peter Herhaus, Mareike Verbeek
Summary: This retrospective analysis compares the efficacy and tolerability of reduced intensity conditioning regimens, Flu/Treo and FLAMSA-RIC, in patients with myeloid malignancies undergoing alloSCT. The results indicate that there is no significant difference in engraftment, survival, relapse, transplant-related mortality, and graft-versus-host disease between the two regimens.
ANNALS OF HEMATOLOGY
(2022)
Article
Oncology
Ally Fraser, Paul Castillo, Erika Cascio, Giselle Moore-Higgs, Nosha Farhadfar, John Fort, William Slayton, Jatinder Lamba, Biljana Horn
Summary: This study aimed to determine the minimum effective dose (MED) of thiotepa in combination with reduced-dose busulfan, fludarabine or cyclophosphamide, and rATG to achieve engraftment in >90% of HSCT recipients with non-malignant disorders. The results showed that all six patients achieved engraftment prior to Day +42 and none of them experienced disease recurrence.
PEDIATRIC BLOOD & CANCER
(2023)
Article
Hematology
Roswitha Luftinger, Natalia Zubarovskaya, Jacques-Emmanuel Galimard, Annamaria Cseh, Elisabeth Salzer, Franco Locatelli, Mattia Algeri, Akif Yesilipek, Josu de la Fuente, Antonella Isgro, Amal Alseraihy, Emanuele Angelucci, Frans J. Smiers, Giorgia La La Nasa, Marco Zecca, Tunc Fisgin, Emel Unal, Katharina Kleinschmidt, Christina Peters, Arjan Lankester, Selim Corbacioglu
Summary: Significant advances in supportive care have improved the life expectancy of patients with transfusion-dependent thalassemia major (TDT), but iron overload remains a barrier to long-term survival. This retrospective analysis compared the outcomes of TDT patients undergoing hematopoietic stem cell transplantation (HSCT) with different conditioning regimens, and found that both busulfan-fludarabine-based and treosulfan-fludarabine-based conditioning resulted in high cure rates. The main causes of death were infections, graft-versus-host disease, and rejection.
ANNALS OF HEMATOLOGY
(2022)
Article
Hematology
Akanksha Chichra, Lingaraj Nayak, Rushabh Kothari, Siddhesh Kalantri, Avinash Bonda, Anant Gokarn, Sachin Punatar, Sumeet Mirgh, Nishant Jindal, Bhausaheb Bagal, Sadhana Kannan, Libin Mathew, Navin Khattry
Summary: This study compares the toxicity and outcomes of two reduced-intensity conditioning regimens, Flu-Mel and Flu-Treo, in different donor groups for transplantation. The results show that Flu-Treo has lower toxicity in terms of severe mucositis and diarrhea compared to Flu-Mel. Additionally, Flu-Treo provides comparable outcomes to Flu-Mel in all donor transplants.
INTERNATIONAL JOURNAL OF HEMATOLOGY
(2023)
Article
Biophysics
Jesus Duque-Afonso, Gabriele Ihorst, Miguel Waterhouse, Robert Zeiser, Ralph Waesch, Hartmut Bertz, Mehtap Yuecel, Thomas Koehler, Joachim Mueller-Quernheim, Reinhard Marks, Juergen Finke
Summary: Conditioning protocols based on FBM and FTM show adequate antineoplastic efficacy and are suitable for patients with impaired lung function. Factors such as age, lung function, performance, etc., do not significantly impact overall survival.
BONE MARROW TRANSPLANTATION
(2021)
Article
Biophysics
Jochen J. Frietsch, Jenny Miethke, Paul Linke, Carl C. Crodel, Ulf Schnetzke, Sebastian Scholl, Andreas Hochhaus, Inken Hilgendorf
Summary: The study evaluated the efficacy and safety of conditioning with Treo/Flu compared to TEAM in patients with B-cell Non-Hodgkin lymphoma (NHL) before autologous hematopoietic stem cell transplantation (auto-HSCT). The results showed no significant differences in progression-free survival, event-free survival, and overall survival between the two groups. However, the Treo/Flu group had a lower dependence on platelet transfusions, older age, and fewer infectious complications, mucositis, and stomatitis. This study highlights the potential value of Treo/Flu as an alternative conditioning regimen for elderly patients with B-cell NHL.
BONE MARROW TRANSPLANTATION
(2022)
Article
Biophysics
Stephen Maurer, Kathleen A. Linder, Carol A. Kauffman, Philip McDonald, Jonathan T. Arcobello, Jon Karl D. Velasco, Pranatharthi Chandrasekar, Sanjay Revankar, Marisa H. Miceli
Summary: We conducted a retrospective review of the infectious complications and outcomes over a 2-year follow-up period of adult patients who received a second allogeneic hematopoietic cell transplant (2nd allo-HCT) during a five-year period at two cancer centers in Michigan. Sixty patients, of whom 44 (73%) had acute leukemia or myelodysplastic syndrome, were studied. The majority (n = 37,62%) received a 2nd allo-HCT because of relapsed leukemia. Infection episodes after the 2nd allo-HCT totaled 112. Bacteria were identified in 76 episodes, the majority of which occurred pre-engraftment. The most common infecting organisms were Enterococcus species and Clostridioides difficile. Viral infections, predominantly cytomegalovirus, accounted for 59 infection episodes and occurred mostly in pre-engraftment and early post-engraftment periods. There were 16 proven/probable fungal infections, of which 9 were invasive aspergillosis or candidiasis. Mortality was 45% (n = 27) at one year and 65% (n = 39) at 2 years after transplant, and 16 deaths (41%) were due to infection. Of those 16 infection deaths, 8 were bacterial, 4 fungal, 2 both bacterial and fungal, and 2 viral. Failure to engraft neutrophils or platelets was significantly associated with decreased survival, p < 0.0001 and p < 0.001, respectively. Infections are common after a 2nd allo-HCT and are associated with a high mortality rate.
BONE MARROW TRANSPLANTATION
(2022)
Review
Immunology
Asim Saha, Bruce R. Blazar
Summary: Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is an effective treatment option for hematological malignancies. However, conventional allo-HSCT methods have adverse effects and can lead to graft-versus-host-disease. Recent developments in targeted radiotherapy and antibody-based conditioning show promise in improving the outcomes of allo-HSCT.
FRONTIERS IN IMMUNOLOGY
(2022)
Article
Hematology
Avichai Shimoni, Marie Robin, Simona Iacobelli, Dietrich Beelen, Ghulam J. Mufti, Fabio Ciceri, Wolfgang Bethge, Liisa Volin, Didier Blaise, Arnold Ganser, Thomas Luft, Patrice Chevallier, Rainer Schwerdtfeger, Linda Koster, Theo de Witte, Nicolaus Kroeger, Arnon Nagler, Ibrahim Yakoub-Agha
Summary: FT regimen is associated with similar low relapse rates as MAC and similar low NRM as RIC, resulting in improved OS.
BRITISH JOURNAL OF HAEMATOLOGY
(2021)
Article
Biophysics
Marjan Cruijsen, Jacobien R. Hilberink, Walter J. F. M. van der Velden, Joop H. Jansen, Brigitte Bar, Nicolaas P. M. Schaap, Anton de Haan, Andre B. Mulder, Marco R. de Groot, Frederic Baron, Edo Vellenga, Nicole N. M. Blijlevens, Gerwin Huls
Summary: The combination of decitabine with a standard non-myeloablative conditioning regimen prior to allogeneic hematopoietic cell transplantation can effectively reduce relapse incidence in poor risk AML patients. The study demonstrated a one-year relapse rate of 23% and a one-year overall survival of 70% in this patient population.
BONE MARROW TRANSPLANTATION
(2021)
Article
Biophysics
Jesus Duque-Afonso, Juergen Finke, Myriam Labopin, Charles Craddock, Rachel Protheroe, Panagiotis Kottaridis, Eleni Tholouli, Jenny L. Byrne, Kim Orchard, Urpu Salmenniemi, Inken Hilgendorf, Hannah Hunter, Emma Nicholson, Adrian Bloor, John A. Snowden, Mareike Verbeek, Andrew Clark, Bipin N. Savani, Alexandros Spyridonidis, Arnon Nagler, Mohamad Mohty
Summary: This study compared the outcomes of two commonly used conditioning protocols, FluMel and FluTreo, for patients with acute myeloid leukemia. While both groups had similar overall survival rates, the FluMel group had a lower relapse incidence and slightly higher non-relapse mortality.
BONE MARROW TRANSPLANTATION
(2022)
Article
Biophysics
Yoshimitsu Shimomura, Masahiko Hara, Hisashi Yamamoto, Naoyuki Uchida, Toshiro Kawakita, Takashi Ashida, Satoru Takada, Takashi Ikeda, Satoshi Morishige, Yumiko Maruyama, Atsushi Wake, Tatsuo Ichinohe, Takahiro Fukuda, Minoko Takanashi, Yoshiko Atsuta, Takayuki Ishikawa
Summary: The combination of Fludarabine and a myeloablative dose of busulfan (Flu/Bu4) with melphalan (Mel) in allogeneic hematopoietic stem cell transplantation (HSCT) may improve overall survival and reduce the risk of relapse. The study found that adding Mel to Flu/Bu4 was associated with a better 5-year overall survival rate and a lower incidence of relapse, without impacting non-relapse mortality.
BONE MARROW TRANSPLANTATION
(2021)
Review
Oncology
Xue Yang, Dongjun Li, Yao Xie
Summary: ATG significantly reduced the incidence and severity of GVHD, improved GRFS, and reduced the number and dose of immunosuppressants in patients undergoing allo-HSCT.
FRONTIERS IN ONCOLOGY
(2021)
Article
Oncology
Valeria Ceolin, Erica Brivio, Harm van Tinteren, Susan R. Rheingold, Allison Leahy, Britta Vormoor, Maureen M. O'Brien, Jeremy D. Rubinstein, Krzysztof Kalwak, Barbara De Moerloose, Elad Jacoby, Peter Bader, Monica Lopez-Duarte, Bianca F. Goemans, Franco Locatelli, Peter Hoogerbrugge, Friso G. Calkoen, Christian Michel Zwaan
Summary: A retrospective analysis of 39 children and young adults who received inotuzumab ozogamicin (InO) before and/or after CAR T-cell therapy showed that the efficacy and survival rates of patients treated with InO prior to or after CAR T-cell therapy were similar to those without prior InO treatment.
Article
Hematology
Kerstin Schuetze, Miriam Gross, Kerstin Cornils, Katharina Wustrau, Sonja Schneppenheim, Henning Lenhartz, G. Christoph Korenke, Gritta Janka, Svea Ledig, Ingo Mueller, Stephan Ehl, Kai Lehmberg
Summary: Multiple hereditary forms of hemophagocytic lymphohistiocytosis (HLH) are caused by defects of cytotoxicity, including vesicle trafficking disorder Griscelli syndrome type 2 (GS2) and MADD deficiency, resulting in various syndromic features. This study demonstrates that MADD deficiency leads to a defect in cytotoxicity, contributing to the pathogenesis of HLH.
Article
Hematology
Fanny Delehaye, Jeremie Rouger, David Brossier, Meinolf Suttorp, Adalet Meral Gunes, Petr Sedlacek, Birgitta Versluys, Chi Kong Li, Krzysztof Kalwak, Birgitte Lausen, Culic Srdjana, Michael Dworzak, Andrea Hraskova, Barbara De Moerloose, Farah Roula, Anais Briant, Jean-Jacques Parienti, Frederic Millot
Summary: The clinical presentation of chronic myeloid leukemia (CML) differs in children compared to adults. Anemia at diagnosis is frequent in pediatric CML and may be considered as a prognostic factor. Moderate and severe anemia at diagnosis in children with CML is significantly associated with younger age, asthenia, splenomegaly, increased leukocyte and basophil counts, delayed major and deep molecular responses, and more frequent failure of imatinib treatment. However, the hemoglobin level is not significantly associated with survival.
ANNALS OF HEMATOLOGY
(2023)
Article
Biophysics
Moshe Yeshurun, Uri Rozovski, Liat Shargian, Oren Pasvolsky, Steffie van der Werf, Gloria Tridello, Nina Knelange, Malgorzata Mikulska, Jan Styczynski, Diana Averbuch, Rafael de la Camara
Summary: We conducted a study to describe the infection prevention practices among EBMT centers. Questionnaires were sent to all 553 centers and responses from 127 centers in 32 countries were obtained. The study revealed significant variations in antimicrobial prophylaxis practices, medications employed and duration of preventive therapy among the centers.
BONE MARROW TRANSPLANTATION
(2023)
Article
Hematology
G. A. Amos Burke, Luciana Vinti, Edita Kabickova, Auke Beishuizen, Nurdan Tacyildiz, Anne Uyttebroeck, Hyoung Jin Kang, Flavio Luisi, Veronique Minard-Colin, Birgit Burkhardt, Monelle Tamegnon, Steven Sun, Madeliene Curtis, Sanjay Deshpande, Kerri Nottage, Angela Howes, Srimathi Srinivasan, Deepa Bhojwani, Robin Norris, Mitchell Cairo
Summary: The results of the SPARKLE trial showed that continued assessment of ibrutinib with modified rituximab or rituximab, vincristine, ifosfamide, carboplatin, idarubicin, and dexamethasone in pediatric patients with relapsed/refractory mature B-cell non-Hodgkin lymphoma (B-NHL) did not provide a significant benefit in event-free survival. The overall response rate and overall survival were slightly higher in the ibrutinib plus chemotherapy group compared to chemotherapy alone. However, all patients in both treatment arms experienced grade >= 3 treatment-emergent adverse events. Ibrutinib exposure in pediatric patients was within the target range observed in adults.
Article
Biophysics
Jan Styczynski, Gloria Tridello, Linda Koster, Nina Knelange, Lotus Wendel, Anja van Biezen, Steffie van der Werf, Malgorzata Mikulska, Lidia Gil, Catherine Cordonnier, Per Ljungman, Diana Averbuch, Simone Cesaro, Helen Baldomero, Christian Chabannon, Selim Corbacioglu, Harry Dolstra, Bertram Glass, Raffaella Greco, Nicolaus Kroeger, Regis Peffault de Latour, Mohamad Mohty, Benedicte Neven, Zinaida Peric, John A. Snowden, Anna Sureda, Ibrahim Yakoub-Agha, Rafael de la Camara
Summary: This study analyzed the incidence and specific causes of death in HCT patients, focusing on infectious deaths. The results showed a decrease in mortality from infections in early phases but an increase in mortality from bacterial infections in the late phase.
BONE MARROW TRANSPLANTATION
(2023)
Article
Pharmacology & Pharmacy
Xieran Li, Krzysztof Kalwak, Rita Beier, Jochen Kehne, Ann-Kristin Moeller, Joachim Baumgart, Dietrich W. Beelen, Ralf A. Hilger, Ajay Vora, Karl -Walter Sykora
Summary: A population pharmacokinetic (PK) model was developed based on historical trials to support treosulfan dose recommendations for children in prospective trials. The model was updated with additional pediatric PK data from these trials, resulting in a modified dose recommendation for children based on body surface area (BSA). The simplified BSA-dependent dose recommendation ensures comparable treosulfan exposure in children and adults without individual therapeutic drug monitoring.
DRUG METABOLISM AND PHARMACOKINETICS
(2023)
Review
Biotechnology & Applied Microbiology
Agata Marjanska, Jan Styczynski
Summary: Post-transplant lymphoproliferative disorders (PTLD) are a diverse group of diseases caused by uncontrolled proliferation of lymphoid or plasmacytic cells resulting from T-cell immunosuppression after transplantation. These are mainly caused by latent Epstein-Barr virus (EBV), and the risk of EBV recurrence depends on the level of immune system incompetency.
EXPERT OPINION ON BIOLOGICAL THERAPY
(2023)
Article
Oncology
Tomasz Styczynski, Jagoda Sadlok, Monika Richert-Przygonska, Robert Debski, Patrycja Zalas-Wiecek, Krzysztof Czyzewski, Jan Styczynski
Summary: This paper reports two cases of pediatric patients with invasive fungal disease caused by Saprochaete clavata after allogeneic hematopoietic cell transplantation, and summarizes the literature data on this infection. With quick diagnosis and treatment, the outcome of therapy was successful.
JOURNAL OF PEDIATRIC HEMATOLOGY ONCOLOGY
(2023)
Article
Biophysics
Jan Styczynski, Gloria Tridello, Lotus Wendel, Nina Knelange, Simone Cesaro, Lidia Gil, Per Ljungman, Malgorzata Mikulska, Dina Averbuch, Rafael de la Camara
Summary: The aim of this study was to examine the current approach to EBV-driven post-transplant complications in EBMT transplant centers. The study found that most centers conduct routine serology testing before HCT, while some also routinely test for pretransplant EBV-DNA. Monitoring for EBV infection is feasible in almost all centers, with some using standardized PCR. Regular post-HCT monitoring is performed in the majority of centers. Prophylaxis with rituximab is used in a small percentage of centers. The prevalence of csEBV-DNA-emia was 7.4%, with higher rates in children. The frequency of EBV-PTLD was 1.6%, with higher rates in children. Rituximab and reduction of immunosuppressive therapy were the first-line treatments for EBV-driven complications. The failure rate of first-line preemptive treatment was 12.0%. EBV-specific T-lymphocytes were available in a portion of centers. Experimental therapies were given to patients with resistant/refractory PTLD. Overall, the prevalence of EBV-DNA-emia and EBV-PTLD decreased in comparison to historical data.
BONE MARROW TRANSPLANTATION
(2023)
Letter
Biophysics
Giorgia Battipaglia, Jarl E. Mooyaart, Ralf Meyer, Mohamad Mohty, Alicja Sadowska-Klasa, Oleg Goloshchapov, Franco Locatelli, Jan Styczynski, Jiri Pavlu, Jaroslaw Dybko, Gleb Bronin, Urpu Salmenniemi, Pavel Jindra, Jorinde D. Hoogenboom, Jurgen Kuball, Annalisa Ruggeri, Florent Malard
BONE MARROW TRANSPLANTATION
(2023)
Article
Biochemistry & Molecular Biology
Kinga Musial, Jakub Stojanowski, Justyna Miskiewicz-Bujna, Krzysztof Kalwak, Marek Ussowicz
Summary: This study assessed the serum concentrations of KIM-1, IL-18, and NGAL in children undergoing HSCT and their usefulness as predictors of kidney damage. The results showed that serum KIM-1, IL-18, and NGAL concentrations before HSCT can serve as markers of renal dysfunction after the procedure.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2023)
Letter
Oncology
Simone Cesaro, Malgorzata Mikulska, Hans H. Hirsch, Jan Styczynski, Sylvain Meylan, Catherine Cordonnier, Davide Navarro, Marie von Lilienfeld-Toal, Varun Mehra, Francesco Marchesi, Caroline Besson, Raul Cordoba Masculano, Gernot Beutel, Herman Einsele, Johan Maertens, Rafael de la Camara, Per Ljungman, Livio Pagano
Article
Biochemistry & Molecular Biology
Anne Kruchen, Pascal-David Johann, Laura Rekowski, Ingo Mueller
Summary: Mesenchymal stromal cells (MSC) play a crucial role in maintaining the homeostasis of hematopoietic stem cells and regulating immune effector cells. However, they may also protect malignant cells from therapeutic drugs and immune effector cells. Modulating these mechanisms could potentially enhance therapeutic efficacy.
CURRENT ISSUES IN MOLECULAR BIOLOGY
(2023)
Article
Immunology
Aleksandra Grzegorczyk, Zuzanna Marczynska, Rafal Matkowski, Marcin Zietek, Wojciech Pietras, Elzbieta Latos-Grazynska, Krzysztof Kalwak, Iwona Dachowska-Kalwak
Summary: This study presents a case of a 17-year-old female patient who had previously undergone surgical resection of melanoma and was administered PD-1 inhibitor nivolumab as therapy. The patient experienced several complications during treatment but follow-up showed no evidence of metastasis or lymph node enlargement.
CENTRAL EUROPEAN JOURNAL OF IMMUNOLOGY
(2023)
