Article
Pharmacology & Pharmacy
Yuan-Yuan Shi, Zeng-Yan Liu, Gui-Xin Zhang, Yi He, Ming-Zhe Han, Si-Zhou Feng, Rong-Li Zhang, Er-Lie Jiang
Summary: This study explores the safety and effectiveness of a cladribine-based conditioning regimen for autologous hematopoietic stem cell transplantation in patients with acute myeloid leukemia. The results indicate that this regimen is a successful and safe approach for AML treatment.
FRONTIERS IN PHARMACOLOGY
(2023)
Review
Biochemistry & Molecular Biology
Carmine Liberatore, Mauro Di Ianni
Summary: The management of relapsed acute myeloid leukemia (AML) post allogeneic hematopoietic stem cell transplantation (HSCT) remains a clinical challenge. Hypomethylating agents (HMAs) have become the standard therapeutic approach due to favorable tolerability and their ability to be combined with other anti-leukemic agents. The addition of donor lymphocytes infusions (DLI) generally leads to improved outcomes, while the introduction of novel targeted drugs offers new possibilities in salvage regimens.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2023)
Article
Oncology
Ziying Li, Wei Shi, Xuan Lu, Hui Lu, Xiena Cao, Liang Tang, Han Yan, Zhaodong Zhong, Yong You, Linghui Xia, Yu Hu, Huafang Wang
Summary: This study found that decitabine-intensified myeloablative conditioning could reduce the incidence of acute graft-versus-host disease and improve the overall survival and GVHD-free relapse-free survival in patients with acute myeloid leukemia after related donor hematopoietic stem cell transplantation.
FRONTIERS IN ONCOLOGY
(2022)
Review
Immunology
Justin Loke, Richard Buka, Charles Craddock
Summary: While most AML patients achieve complete remission with intensive chemotherapy, many face a risk of relapse if treated with chemotherapy alone. Allogeneic stem cell transplant has become a key treatment strategy for AML patients, especially for those in first complete remission and advanced stages. Despite a decrease in transplant-related mortality, there remains a need to address the risk of disease relapse.
FRONTIERS IN IMMUNOLOGY
(2021)
Article
Immunology
Hyery Kim, Beom Hee Lee, Hyo-Sang Do, Gu-Hwan Kim, Sunghan Kang, Kyung-Nam Koh, Ho Joon Im
Summary: Mevalonic aciduria (MA) is the most severe clinical subtype of mevalonate kinase deficiency (MKD) caused by an inherited defect in the mevalonate pathway. The treatment of MKD focuses on suppressing recurrent hyperinflammatory attacks using anti-inflammatory drugs. Allogeneic hematopoietic stem cell transplantation (HCT) has been shown to successfully ameliorate autoinflammatory attacks in patients with MKD who have MA.
FRONTIERS IN IMMUNOLOGY
(2021)
Review
Oncology
Li Xuan, Qifa Liu
Summary: Relapse is still the main reason for treatment failure in AML patients undergoing allo-HSCT. Evidence suggests that maintenance therapy post-transplantation, particularly targeted drugs like hypomethylating agents, FLT3 inhibitors, and isocitrate dehydrogenase inhibitors, could benefit AML patients, especially those at high risk.
JOURNAL OF HEMATOLOGY & ONCOLOGY
(2021)
Article
Multidisciplinary Sciences
Troy M. Robinson, Robert L. Bowman, Sonali Persaud, Ying Liu, Rosemary Neigenfind, Qi Gao, Jingping Zhang, Xiaotian Sun, Linde A. Miles, Sheng F. Cai, Adam Sciambi, Aaron Llanso, Christopher Famulare, Aaron Goldberg, Ahmet Dogan, Mikhail Roshal, Ross L. Levine, Wenbin Xiao
Summary: A single-cell MRD assay was developed in this study, which can detect residual cancer cells with high sensitivity and provide clone-specific information on clonal architecture and immunophenotype.
Article
Biophysics
Eleni Gavriilaki, Myriam Labopin, Ioanna Sakellari, Urpu Salmenniemi, Ibrahim Yakoub-Agha, Victoria Potter, Ana Berceanu, Alessandro Rambaldi, Inken Hilgendorf, Nicolaus Kroeger, Stephan Mielke, Tsila Zuckerman, Jaime Sanz, Alessandro Busca, Hakan Ozdogu, Achilles Anagnostopoulos, Bipin Savani, Sebastian Giebel, Ali Bazarbachi, Alexandros Spyridonidis, Arnon Nagler, Mohamad Mohty
Summary: This study compared the outcomes of treosulfan plus fludarabine and busulfan in different patient groups, and found that busulfan had better outcomes in younger patients.
BONE MARROW TRANSPLANTATION
(2022)
Article
Medicine, General & Internal
Sunita Goyal, John Tisdale, Manfred Schmidt, Julie Kanter, Jennifer Jaroscak, Dustin Whitney, Hans Bitter, Philip D. Gregory, Geoffrey Parsons, Marianna Foos, Ashish Yeri, Maple Gioia, Sarah B. Voytek, Alex Miller, Jessie Lynch, Richard A. Colvin, Melissa Bonner
Summary: A woman developed acute myeloid leukemia 5.5 years after receiving LentiGlobin therapy for sickle cell disease, but investigation revealed that the leukemia was unlikely related to vector insertion. Other genetic mutations and inadequate disease control after treatment may contribute to the development of leukemia in sickle cell disease patients.
NEW ENGLAND JOURNAL OF MEDICINE
(2022)
Review
Oncology
Michael Loschi, Pierre Fenaux, Thomas Cluzeau
Summary: TP53-mutated acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) are characterized by poor prognosis. In this review, we analyze the prognosis of these diseases, focusing on the extent of TP53 mutation status and its correlation with cytogenetic complexity. We discuss the potential improvement in outcome based on recent results obtained with new drugs (especially eprenetapopt and magrolimab). The impact of allogeneic hematopoietic stem cell transplantation (aHSCT) and post-transplantation treatment is also emphasized.
Review
Oncology
Ying-Jun Chang, Xiang-Yu Zhao, Xiao-Jun Huang
Summary: Haploidentical stem cell transplantation is an effective alternative donor source for AML patients, with outcomes comparable to traditional transplantation methods for various types of AML. Relapse and infections post haplo-SCT remain key challenges in improving clinical outcomes for AML patients. Areas of active research include relapse prophylaxis, intervention, treatment, as well as infection prevention and therapy.
FRONTIERS IN ONCOLOGY
(2021)
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
Oncology
Ekaterina Gurevich, Michael Hayoz, Yolanda Aebi, Carlo R. Largiader, Behrouz Mansouri Taleghani, Ulrike Bacher, Thomas Pabst
Summary: This study compared the use of busulfan/melphalan regimen with treosulfan/melphalan regimen in AML patients. The treosulfan/melphalan regimen was found to be safer and more effective, with no neurotoxicity and irreversible alopecia. There was considerable interindividual biovariability in treosulfan serum levels.
Article
Oncology
Juan Chen, Li Liu, Runzhi Ma, Aiming Pang, Donglin Yang, Xin Chen, Jialin Wei, Yi He, Rongli Zhang, Weihua Zhai, Qiaoling Ma, Erlie Jiang, Mingzhe Han, Jiaxi Zhou, Sizhou Feng
Summary: This study evaluated the efficacy of autologous hematopoietic stem cell transplantation (auto-HSCT) in patients with favorable-risk acute myeloid leukemia in first remission. The results showed that auto-HSCT had excellent outcomes in these patients and could be an effective treatment for them.
CANCER CELL INTERNATIONAL
(2022)
Article
Oncology
Samantha O'Hagan Henderson, Jochen J. Frietsch, Inken Hilgendorf, Andreas Hochhaus, Claus-Henning Koehne, Jochen Casper
Summary: Treosulfan, fludarabine, and cytarabine (Treo/Flu/AraC) conditioning regimen shows tolerable, feasible, and effective outcomes in high-risk AML, MDS, and MPN patients undergoing allogeneic SCT. The incidence of non-relapse mortality and relapse is acceptable in this heavily pre-treated population with advanced disease. Further prospective trials with larger sample sizes are needed to confirm these promising initial findings.
JOURNAL OF CANCER RESEARCH AND CLINICAL ONCOLOGY
(2022)
Article
Biophysics
Sebastian Giebel, Myriam Labopin, Mohamed Houhou, Denis Caillot, Juergen Finke, Didier Blaise, Nathalie Fegueux, Mark Ethell, Jan J. Cornelissen, Edouard Forcade, Ibrahim Yakoub-Agha, Federico Lussana, Johan Maertens, Jean Henri Bourhis, Pavel Jindra, Norbert Claude Gorin, Arnon Nagler, Mohamad Mohty
Summary: For elderly patients with acute lymphoblastic leukemia (ALL) in first complete remission (CR1), autologous hematopoietic cell transplantation (auto-HCT) may be considered as an alternative to reduced intensity conditioning allogeneic HCT (RIC-allo-HCT). A retrospective study compared outcomes of RIC-allo-HCT from matched sibling donors (MSD) or matched unrelated donors (MUD) with auto-HCT for patients aged 55 years or more. While the probabilities of leukemia-free survival (LFS) and overall survival (OS) were not significantly different between RIC-allo-HCT and auto-HCT, the risk of non-relapse mortality (NRM) was higher for both MSD-HCT and MUD-HCT, and for MUD-HCT, the chance of LFS and OS was lower.
BONE MARROW TRANSPLANTATION
(2023)
Letter
Biophysics
Tamim Alsuliman, Nicolas Stocker, Elise Corre, Remy Dulery, Simona Sestili, Laure Ricard, Florent Malard, Mohamad Mohty, Paul Coppo, Zora Marjanovic
Summary: Patients with high-risk lymphoma received R-COPADM and CYVE treatment, followed by ASCT if suitable. The study showed the efficacy and acceptable safety profile of this regimen, with a 87% overall survival rate and a 83% progression-free survival rate at 46 months of follow-up. These results suggest the potential benefit of this treatment approach for high-risk lymphoma patients.
BONE MARROW TRANSPLANTATION
(2023)
Article
Hematology
Alexandros Spyridonidis, Myriam Labopin, Bipin Savani, Sebastian Giebel, Gesine Bug, Stefan Schoenland, Nicolaus Kroeger, Matthias Stelljes, Thomas Schroeder, Andrew McDonald, Igor-Wolfgang Blau, Martin Bornhaeuser, Montse Rovira, Wolfgang Bethge, Andreas Neubauer, Arnold Ganser, Jean Henri Bourhis, Matthias Edinger, Bruno Lioure, Gerald Wulf, Kerstin Schaefer-Eckart, Mutlu Arat, Zinaida Peric, Christoph Schmid, Ali Bazarbachi, Fabio Ciceri, Arnon Nagler, Mohamad Mohty
Summary: In this registry-based study, the outcomes of allogeneic hematopoietic cell transplantation (allo-HCT) in adult patients with acute lymphoblastic leukemia (ALL) were compared. The study found that the total dose of total body irradiation (TBI) did not influence the survival outcomes for patients, regardless of whether they received a standard 12-Gray or lower 8-Gray dose.
Article
Biophysics
Iman Abou Dalle, Myriam Labopin, Nicolaus Kroeger, Thomas Schroeder, Juergen Finke, Matthias Stelljes, Andreas Neubauer, Didier Blaise, Ibrahim Yakoub-Agha, Urpu Salmenniemi, Edouard Forcade, Maija Itala-Remes, Peter Dreger, Gesine Bug, Jakob Passweg, Michael Heuser, Goda Choi, Eolia Brissot, Sebastian Giebel, Arnon Nagler, Fabio Ciceri, Ali Bazarbachi, Mohamad Mohty
Summary: Pre-transplant detectable measurable residual disease (MRD) is still associated with high risk of relapse and poor outcomes in acute myeloid leukemia (AML). The impact of disease burden on prediction of relapse and survival in patients receiving allogeneic hematopoietic cell transplantation (allo-HCT) in first remission (CR1) was evaluated. The study found that patients in CR1 and MRD positive at time of transplant could still be salvaged by allo-HCT, with significantly better outcomes than patients transplanted with active disease.
BONE MARROW TRANSPLANTATION
(2023)
Article
Biophysics
Olivier Tournilhac, Michel van Gelder, Dirk-Jan Eikema, Nienke Zinger, Peter Dreger, Martin Bornhaeuser, Vladan Vucinic, Christof Scheid, Jan J. Cornelissen, Thomas Schroeder, Pavel Jindra, Henrik Sengeloev, Stephanie Nguyen Quoc, Matthias Stelljes, Igor Wolfgang Blau, Jiri Mayer, Shankara Paneesha, Patrice Chevallier, Edouard Forcade, Nicolaus Kroeger, Didier Blaise, John Gribben, Bendt Nielsen, Jan-Erik Johansson, Charalampia Kyriakou, Yves Beguin, Pietro Pioltelli, Antonia Sampol, Donal P. McLornan, Johannes Schetelig, Patrick J. Hayden, Ibrahim Yakoub-Agha
Summary: A study analyzed the use of allo-HCT in CLL patients in Europe and found that the treatment is still being applied in selected patients, despite the emergence of targeted therapies. However, the use of long-term control strategies using targeted therapies is becoming more common, especially for patients who have failed immunochemotherapy or have TP53 gene mutations.
BONE MARROW TRANSPLANTATION
(2023)
Article
Biophysics
Arnon Nagler, Myriam Labopin, Stephan Mielke, Jakob Passweg, Didier Blaise, Tobias Gedde-Dahl, Jan J. J. Cornelissen, Urpu Salmenniemi, Ibrahim Yakoub-Agha, Peter Remenyi, Gerard Socie, Gwendolyn van Gorkom, Helene Labussiere-Wallet, Xiao-Jun Huang, Marie Therese Rubio, Jenny Byrne, Charles Craddock, Laimonas Griskevicius, Fabio Ciceri, Mohamad Mohty
Summary: This study compared the outcomes of hematopoietic stem cell transplants in acute myeloid leukemia patients in first complete remission from different donor sources. The results showed that acute graft-versus-host disease (GVHD) was more common in all non-sibling donor groups. Chronic GVHD rates were also higher in certain unrelated donor groups. However, overall survival and leukemia-free survival did not differ significantly between donor types.
BONE MARROW TRANSPLANTATION
(2023)
Article
Biophysics
Arnon Nagler, Myriam Labopin, Ryszard Swoboda, Alexander Kulagin, Helene Labussiere-Wallet, Montserrat Rovira, Didier Blaise, Jan Vydra, Ibrahim Yakoub-Agha, Goda Choi, Peter Remenyi, Yener Koc, Jaime Sanz, Fabio Ciceri, Mohamad Mohty
Summary: Retrospective evaluation of transplant outcomes in adult AML patients undergoing allo-HCT in CR2 showed that post-transplant cyclophosphamide is an effective method for GVHD prophylaxis.
BONE MARROW TRANSPLANTATION
(2023)
Article
Biophysics
Frederic Baron, Annalisa Ruggeri, Christophe Peczynski, Myriam Labopin, Jean-Henri Bourhis, Mauricette Michallet, Patrice Chevallier, Jaime Sanz, Edouard Forcade, Riccardo Saccardi, Victoria Potter, Eliane Gluckman, Arnon Nagler, Mohamad Mohty
Summary: This study assessed the outcomes of patients who experienced graft failure after umbilical cord blood transplantation (CBT). It included a total of 87 patients and found that the cumulative incidence of relapse and nonrelapse mortality at 1-year was 35% and 37% respectively. Among patients who underwent salvage second transplantation, the 1-year and 2-year overall survival rates were 41% and 34% respectively. The study also identified complete remission and reduced-intensity conditioning as factors associated with better overall survival.
BONE MARROW TRANSPLANTATION
(2023)
Article
Hematology
Arnon Nagler, Myriam Labopin, Bhagirathbhai Dholaria, Didier Blaise, Sergey Bondarenko, Jan Vydra, Goda Choi, Montserrat Rovira, Peter Remenyi, Ellen Meijer, Claude Eric Bulabois, J. L. Diez-Martin, Ibrahim Yakoub-Agha, Eolia Brissot, Alexandros Spyridonidis, Jaime Sanz, Amit Patel, Mutlu Arat, Ali Bazarbachi, Gesine Bug, Bipin N. Savani, Sebastian Giebel, Fabio Ciceri, Mohamad Mohty
Summary: In this study, the impact of measurable residual disease (MRD) on the outcomes of post-transplant cyclophosphamide (PTCy)-based allo-HCT in acute myeloid leukemia (AML) patients in first complete remission (CR1) was assessed. The results showed that MRD positive (MRD+) patients had higher relapse rates and lower survival rates compared to MRD negative (MRD-) patients.
BRITISH JOURNAL OF HAEMATOLOGY
(2023)
Review
Multidisciplinary Sciences
Huisheng Ai, Nelson J. Chao, David A. Rizzieri, Xiaojun Huang, Thomas R. Spitzer, Jianxiang Wang, Mei Guo, Armand Keating, Elizabeth F. Krakow, Didier Blaise, Jun Ma, Depei Wu, John Reagan, Usama Gergis, Rafael F. Duarte, Preet M. Chaudhary, Kaixun Hu, Changlin Yu, Qiyun Sun, Ephraim Fuchs, Bo Cai, Yajing Huang, Jianhui Qiao, David Gottlieb, Kirk R. Schultz, Mingyao Liu, Xiequn Chen, Wenming Chen, Jianmin Wang, Xiaohui Zhang, Jianyong Li, He Huang, Zimin Sun, Fei Li, Linhua Yang, Liansheng Zhang, Lijuan Li, Kaiyan Liu, Jie Jin, Qifa Liu, Daihong Liu, Chunji Gao, Chuanbo Fan, Li Wei, Xi Zhang, Liangding Hu, Weijing Zhang, Yuyang Tian, Weidong Han, Jun Zhu, Zhijian Xiao, Daobin Zhou, Bolong Zhang, Yongqian Jia, Yongqing Zhang, Xiaoxiong Wu, Xuliang Shen, Xuzhang Lu, Xinrong Zhan, Xiuli Sun, Yi Xiao, Jingbo Wang, Xiaodong Shi, Bo Zheng, Jieping Chen, Banghe Ding, Zhao Wang, Fan Zhou, Mei Zhang, Yizhuo Zhang, Jie Sun, Bing Xia, Baoan Chen, Liangming Ma
Summary: Recent studies have shown that microtransplant (MST) could improve outcome of patients with elderly acute myeloid leukemia (EAML). To further standardize the MST therapy and improve outcomes in EAML patients, the International Microtransplant Interest Group provides recommendations and considerations for MST in the treatment of EAML, based on analysis of the literature on MST, especially MST with EAML from January 1st, 2011 to November 30th, 2022. The recommendations address key issues including therapeutic principle, candidate selection, chemotherapy regimens, post-remission therapy, and other aspects such as donor screening and complications.
Article
Biophysics
Tapani Ruutu, Christophe Peczynski, Mohamed Houhou, Emmanuelle Polge, Mohamad Mohty, Nicolaus Kroeger, Ivan Moiseev, Olaf Penack, Nina Salooja, Helene Schoemans, Rafael F. Duarte, Thomas Schroeder, Jakob Passweg, Gerald G. Wulf, Arnold Ganser, Simona Sica, Mutlu Arat, Urpu Salmenniemi, Annoek E. C. Broers, Jean Henri Bourhis, Alessandro Rambaldi, Johan Maertens, Kazimierz Halaburda, Tsila Zuckerman, Helene Labussiere-Wallet, Grzegorz Basak, Christian Koenecke, Zinaida Peric
Summary: The study investigated the incidence, diagnostic policy, management, and outcome of VOD/SOS at EBMT centers. It included 71 participating centers with a total of 2886 allogeneic transplantations and 93 cases of VOD/SOS in 2018. The findings showed a low incidence of VOD/SOS and a predominance of severe and very severe grades, indicating the importance of early diagnosis and treatment to improve outcomes.
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
Hematology
Alicia Rovo, Luuk Gras, Brian Piepenbroek, Nicolaus Kroeger, H. Christian Reinhardt, Aleksandar Radujkovic, Didier Blaise, Guido Kobbe, Riitta Niityvuopio, Uwe Platzbecker, Katja Sockel, Mathilde Hunault-Berger, J. J. Cornelissen, Edouard Forcade, Jean Henri Bourhis, Yves Chalandon, Francesca Kinsella, Stephanie Nguyen-Quoc, Johan Maertens, Ahmet Elmaagacli, Nicola Mordini, Patrick Hayden, Kavita Raj, Joanna Drozd-Sokolowska, Liesbeth C. de Wreede, Donal P. Mclornan, Marie Robin, Ibrahim Yakoub-Agha, Francesco Onida
Summary: This study compared the outcomes of CMML and MDS patients after hematopoietic cell transplantation. The results showed that CMML patients had significantly worse survival rates, progression-free survival, and relapse rates compared to MDS patients, while non-relapse mortality did not differ significantly. This study demonstrates the poor outcomes of CMML patients after hematopoietic cell transplantation.
AMERICAN JOURNAL OF HEMATOLOGY
(2023)
Article
Biophysics
A. Mussetti, B. Rius-Sansalvador, V. Moreno, C. Peczynski, E. Polge, J. E. Galimard, N. Kroeger, D. Blaise, R. Peffault de Latour, A. Kulagin, A. Mousavi, M. Stelljes, R. M. Hamladji, J. M. Middeke, U. Salmenniemi, H. Sengeloev, E. Forcade, U. Platzbecker, P. Remenyi, E. Angelucci, P. Chevallier, I. Yakoub-Agha, C. Craddock, F. Ciceri, T. Schroeder, M. Aljurf, Koenecke Ch, I. Moiseev, O. Penack, H. Schoemans, M. Mohty, B. Glass, A. Sureda, G. Basak, Z. Peric
Summary: This study aimed to develop a new prognostic score for overall mortality (OM) and non-relapse mortality (NRM) in patients undergoing allogeneic haematopoietic cell transplantation (alloHCT). AI techniques were applied, and the analysis included data from 33,927 patients. The final prognostic model effectively predicted the risk of mortality and non-relapse mortality in these patients.
BONE MARROW TRANSPLANTATION
(2023)
Meeting Abstract
Oncology
Nirav N. Shah, Ian Flinn, Mi Kwon, Ulrich Jaeger, Javier Briones, Emmanuel Bachy, Didier Blaise, Nicolas Boissel, Koji Kato, Peter A. Riedell, Matthew J. Frigault, Leyla O. Shune, Takanori Teshima, Fabio Ciceri, Shaun A. Fleming, Silvia Ferrari, David Pearson, Jeanne Whalen, Aiesha Zia, Jaclyn Davis, Aisha Masood, Michael Dickinson, Pere Barba
CLINICAL LYMPHOMA MYELOMA & LEUKEMIA
(2023)