Article
Immunology
Diego Medina-Valencia, Daniela Cleves, Estefania Beltran, Natalia Builes, Alexis A. Franco, Andres Felipe Escobar-Gonzalez, Manuela Olaya
Summary: Primary hemophagocytic lymphohistiocytosis is a severe and uncommon disease affecting pediatric patients, with genetic abnormalities linked to altered apoptosis and exaggerated inflammatory reactions. This study describes a novel treatment strategy involving haploidentical hematopoietic stem cell transplantation, yielding positive outcomes for all pediatric patients treated. The results suggest this strategy could be a promising alternative in cases where other donor options are limited.
JOURNAL OF CLINICAL IMMUNOLOGY
(2021)
Article
Immunology
Maryam Barkhordar, Amir Kasaeian, Ghasem Janbabai, Hossein Kamranzadeh Fumani, Sahar Tavakoli, Amir Abbas Rashidi, Seied Asadollah Mousavi, Ardeshir Ghavamzadeh, Mohammad Vaezi
Summary: This study evaluated the long-term outcomes of haploidentical peripheral blood stem cell transplantation using the combination of anti-thymocyte globulin and post-transplant cyclophosphamide (ATG/PTCy). The results showed that the ATG/PTCy combination had a lower risk of graft-versus-host disease, but a higher risk of relapse compared to the standard ATG regimen. However, the combination had no significant effect on long-term outcomes.
FRONTIERS IN IMMUNOLOGY
(2022)
Article
Hematology
Nelli Bejanyan, Joseph A. Pidala, Xuefeng Wang, Ram Thapa, Taiga Nishihori, Hany Elmariah, Aleksandr Lazaryan, Farhad Khimani, Marco L. Davila, Asmita Mishra, Rawan Faramand, Michael D. Jain, Leonel Ochoa, Lia Elena Perez, Hien Liu, Melissa Alsina, Mohamed A. Kharfan-Dabaja, Hugo Fernandez, Michael L. Nieder, Frederick L. Locke, Claudio Anasetti, Ernesto Ayala
Summary: The study demonstrates that GVHD prophylaxis with sirolimus/MMF following PTCy effectively prevents grade II-IV aGVHD after PB haplo-HCT, highlighting the need for a prospective comparison of sirolimus vs tacrolimus in combination with MMF for GVHD prophylaxis after PB HCT.
Article
Oncology
Remy Dulery, Razan Mohty, Myriam Labopin, Simona Sestili, Florent Malard, Eolia Brissot, Giorgia Battipaglia, Clemence Mediavilla, Anne Banet, Zoe Van de Wyngaert, Annalisa Paviglianiti, Ramdane Belhocine, Francoise Isnard, Simona Lapusan, Rosa Adaeva, Anne Vekhoff, Tounes Ledraa, Ollivier Legrand, Ariel Cohen, Agnes Bonnin, Stephane Ederhy, Mohamad Mohty
Summary: This study compared clinical outcomes between patients who received post-transplant cydophosphamide (PT-Cy) and patients who did not, finding a higher incidence of early cardiac events in patients who received PT-Cy.
JACC: CARDIOONCOLOGY
(2021)
Article
Hematology
Kaitai Yang, Susu Gong, Tiebin Jiang, Xinquan Liang, Jian Hu, Ping Zhu, Lin Nie, Yajing Xu, Bin Fu
Summary: The study investigated a novel approach to HSCT, haploidentical peripheral blood stem cell transplantation, for patients with SAA who lack HLA-identical siblings or matched unrelated donors. The results showed promising outcomes with low rates of graft-versus-host disease, indicating the potential for safely expanding the donor pool for young patients with severe aplastic anemia.
TRANSPLANTATION AND CELLULAR THERAPY
(2021)
Article
Immunology
Xingying Li, Jun Yang, Yu Cai, Chongmei Huang, Xiaowei Xu, Huiying Qiu, Jiahua Niu, Kun Zhou, Ying Zhang, Xinxin Xia, Yu Wei, Chang Shen, Yin Tong, Baoxia Dong, Liping Wan, Xianmin Song
Summary: The novel low-dose ATG/PTCy-based regimen demonstrates promising activity in preventing acute GVHD in haplo-PBSCT.
FRONTIERS IN IMMUNOLOGY
(2023)
Article
Oncology
Siddharth Kunte, Lisa Rybicki, Auro Viswabandya, Roni Tamari, Asad Bashey, Alla Keyzner, Madiha Iqbal, Michael R. Grunwald, Bhagirathbhai Dholaria, Hany Elmariah, Michael Ozga, Anurag Singh, Sameem Abedin, Amy E. DeZern, Richard J. Jones, Vikas Gupta, Aaron T. Gerds, Tania Jain
Summary: The study demonstrates that haplo-BMT with PTCy is effective in treating myelofibrosis, but splenomegaly >= 22 cm and bone marrow grafts are associated with an increased risk of relapse.
Article
Oncology
Ling Wang, Lining Wang, Xing Fan, Wei Tang, Jiong Hu
Summary: The study evaluated the safety and efficacy of PT-Cy combined with cyclosporine for GVHD prophylaxis in lymphoid malignancies after transplantation, showing the potential of this strategy in reducing the incidence of chronic GVHD.
FRONTIERS OF MEDICINE
(2021)
Article
Immunology
Cong Zeng, Yan Chen, Juan Hua, Yi Liu, Ting-ting Cheng, Xia Ma, Xu Chen, Shi-yu Wang, Ya-jing Xu
Summary: This retrospective study analyzed the outcomes of 80 patients with hematologic malignancies who received haploidentical peripheral blood stem cell transplantation (haplo-PBSCT) combined with unrelated cord blood (UCB). Satisfactory results were obtained with high engraftment rates and relatively low rates of transplant-related complications.
FRONTIERS IN IMMUNOLOGY
(2022)
Article
Biochemistry & Molecular Biology
Jaroslaw Dybko, Malgorzata Sobczyk-Kruszelnicka, Sebastian Makuch, Siddarth Agrawal, Krzysztof Dudek, Sebatian Giebel, Lidia Gil
Summary: This study compared the outcomes of patients who received post-transplant cyclophosphamide (PTCy) or conventional immunosuppressants after allogeneic hematopoietic cell transplantation. The study found that patients receiving PTCy had lower rates of acute graft-versus-host disease and cytomegalovirus reactivation, and higher survival rates. Further research with a larger sample size is needed to confirm these results.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2023)
Article
Biophysics
Lorenzo Lazzari, Aitana Balaguer-Rosello, Juan Montoro, Raffaella Greco, Rafael Hernani, Maria Teresa Lupo-Stanghellini, Marta Villalba, Fabio Giglio, Ana Facal, Francesca Lorentino, Manuel Guerreiro, Alessandro Bruno, Ariadna Perez, Elisabetta Xue, Daniela Clerici, Simona Piemontese, Jose Luis Pinana, Miguel Angel Sanz, Carlos Solano, Javier de la Rubia, Fabio Ciceri, Jacopo Peccatori, Jaime Sanz
Summary: GvHD prophylaxis with Sir-PTCy, with or without MMF, is safe and effective in patients with AML undergoing myeloablative allo-HSCT, resulting in low rates of transplant-related mortality, relapse/progression, and acute and chronic GvHD in all donor settings.
BONE MARROW TRANSPLANTATION
(2022)
Article
Biophysics
Elmariah Hany, Syeda Mahrukh Hussnain Naqvi, Jongphil Kim, Taiga Nishihori, Asmita Mishra, Lia Perez, Rawan Faramand, Aleksandr Lazaryan, Hien D. Liu, Farhad Khimani, Michael Nieder, Claudio Anasetti, Joseph Pidala, Nelli Bejanyan
Summary: Higher infused total nucleated cell dose in allogeneic bone marrow transplant with post-transplant cyclophosphamide is associated with improved overall survival. The study suggests that infused CD34+ cell doses >= 5 x 10(6) cells/kg may result in improved survival in patients receiving allogeneic PBSCT with PTCy. Targeting a CD34+ cell dose of >= 5 x 10(6) cells/kg for allogeneic PBSCT with PTCy is supported by these findings.
BONE MARROW TRANSPLANTATION
(2021)
Article
Biochemistry & Molecular Biology
Rong-Long Chen, Peng Peng Ip, Jy-juinn Shaw, Yun-Hsin Wang, Li-Hua Fan, Yi-Ling Shen, Nithila A. Joseph, Tsen-Erh Chen, Liuh-Yow Chen
Summary: Most cases of AA, including the SAMD9L-inherited subtype, are immune-mediated. The modified PTCy-based regimen presented in this study is efficient and safe for salvage.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2022)
Article
Biophysics
Remy Dulery, Florent Malard, Eolia Brissot, Anne Banet, Simona Sestili, Ramdane Belhocine, Martina Calabro, Zoe van de Wyngaert, Agnes Bonnin, Tounes Ledraa, Ollivier Legrand, Myriam Labopin, Elodie Capderou, Ariel Cohen, Stephane Ederhy, Mohamad Mohty
Summary: Reducing PT-Cy total dose to 70 mg/kg is a safe and valid approach for elderly patients and those with cardiac comorbidities undergoing haploidentical HCT with PBSCs and low-dose antithymocyte globulin. The reduced PT-Cy dose is associated with improved hematological count recovery, lower incidence of toxicities, and higher GVHD-free, relapse-free survival.
BONE MARROW TRANSPLANTATION
(2023)
Article
Immunology
Ashley D. Hadjis, Natalia S. Nunes, Shanzay M. Khan, Rochelle E. Fletcher, Alessandra de Paula Pohl, David J. Venzon, Michael A. Eckhaus, Christopher G. Kanakry
Summary: Post-transplantation cyclophosphamide (PTCy) is a drug used to treat graft-versus-host disease (GVHD) following hematopoietic cell transplantation (HCT). It works by impairing and suppressing antigen-specific T cells. In this study, reduced proliferation of CD4(+) T cells at day +7 and preferential recovery of CD4(+)CD25(+)Foxp3(+) regulatory T cells (T-regs) at day +21 were identified as potential biomarkers associated with optimal PTCy dosing and timing.
FRONTIERS IN IMMUNOLOGY
(2022)
Article
Biochemistry & Molecular Biology
Angela Genchi, Elena Brambilla, Francesca Sangalli, Marta Radaelli, Marco Bacigaluppi, Roberto Furlan, Annapaola Andolfo, Denise Drago, Cinzia Magagnotti, Giulia Maria Scotti, Raffaella Greco, Paolo Vezzulli, Linda Ottoboni, Marco Bonopane, Daniela Capilupo, Francesca Ruffini, Daniela Belotti, Benedetta Cabiati, Stefania Cesana, Giada Matera, Letizia Leocani, Vittorio Martinelli, Lucia Moiola, Luca Vago, Paola Panina-Bordignon, Andrea Falini, Fabio Ciceri, Anna Uglietti, Maria Pia Sormani, Giancarlo Comi, Mario Alberto Battaglia, Maria A. A. Rocca, Loredana Storelli, Elisabetta Pagani, Giuseppe Gaipa, Gianvito Martino
Summary: This study conducted a phase 1 clinical trial evaluating the feasibility, safety and tolerability of intrathecally transplanted human fetal neural precursor cells (hfNPCs) in patients with progressive multiple sclerosis (PMS). The results demonstrated that hfNPC therapy is feasible, safe and tolerable in PMS patients.
Article
Clinical Neurology
Giacomo Boffa, Alessio Signori, Luca Massacesi, Alice Mariottini, Elvira Sbragia, Salvatore Cottone, Maria Pia Amato, Claudio Gasperini, Lucia Moiola, Stefano Meletti, Anna Maria Repice, Vincenzo Brescia Morra, Giuseppe Salemi, Francesco Patti, Massimo Filippi, Giovanna De Luca, Giacomo Lus, Mauro Zaffaroni, Patrizia Sola, Antonella Conte, Riccardo Nistri, Umberto Aguglia, Franco Granella, Simonetta Galgani, Luisa Maria Caniatti, Alessandra Lugaresi, Silvia Romano, Pietro Iaffaldano, Eleonora Cocco, Riccardo Saccardi, Emanuele Angelucci, Maria Trojano, Giovanni Luigi Mancardi, Maria Pia Sormani, Matilde Inglese
Summary: This study compared the effects of autologous hematopoietic stem cell transplantation (AHSCT) with other anti-inflammatory disease-modifying therapies (DMTs) on long-term disability worsening in active secondary progressive multiple sclerosis (SPMS). The results showed that AHSCT was associated with a slower disability progression and a higher likelihood of disability improvement compared to standard immunotherapy.
Article
Biophysics
Jakob R. R. Passweg, Helen Baldomero, Fabio Ciceri, Selim Corbacioglu, Rafael de la Camara, Harry Dolstra, Bertram Glass, Raffaella Greco, Donal P. P. McLornan, Benedicte Neven, Regis Peffault de Latour, Zinaida Peric, Annalisa Ruggeri, John A. A. Snowden, Anna Sureda
Summary: In 2021, there were 47,412 HCT cases reported by 694 European centers, with 42% being allogeneic and 58% autologous. CAR-T treatments showed a significant increase of 35%, and allogeneic and autologous HCT increased by 5.4% and 3.9% respectively, particularly in non-malignant disorders. The main indications for HCT were myeloid malignancies, lymphoid malignancies, and non-malignant disorders. The use of haploidentical donors decreased while unrelated and sibling donors increased. The report highlights the impact of the SARS-CoV-2 pandemic on transplant activities and the importance of healthcare resource planning.
BONE MARROW TRANSPLANTATION
(2023)
Article
Biophysics
Riccardo Saccardi, Hein Putter, Dirk-Jan Eikema, Maria Paula Busto, Eoin McGrath, Bas Middelkoop, Gillian Adams, Marina Atlija, Francis Ayuketang Ayuk, Helen Baldomero, Yves Beguin, Rafael de la Camara, Angel Cedillo, Anna Maria Sureda Balari, Christian Chabannon, Selim Corbacioglu, Harry Dolstra, Rafael F. Duarte, Remy Dulery, Raffaella Greco, Andreu Gusi, Nada Hamad, Michelle Kenyon, Nicolaus Kroeger, Myriam Labopin, Julia Lee, Per Ljungman, Lynn Manson, Florence Mensil, Noel Milpied, Mohamad Mohty, Elena Oldani, Kim Orchard, Jakob Passweg, Rachel Pearce, Regis Peffault de Latour, Helene A. Poirel, Tuula Rintala, J. Douglas Rizzo, Annalisa Ruggeri, Carla Sanchez-Martinez, Fermin Sanchez-Guijo, Isabel Sanchez-Ortega, Marie Trnkova, David Valcarcel Ferreiras, Leonie Wilcox, Liesbeth C. de Wreede, John A. Snowden
Summary: From 2016, EBMT and JACIE developed an international risk-adapted benchmarking program for HSCT outcome. The program aims to assure quality and meet accreditation requirements for EBMT Centers. With input from previous experience, the program established criteria for patient and Center selection and a statistical model based on key clinical variables. The experience so far has shown the feasibility, acceptability, and reliability of the system, while also identifying limitations and future challenges.
BONE MARROW TRANSPLANTATION
(2023)
Article
Biophysics
Ibrahim Yakoub-Agha, Raffaella Greco, Francesco Onida, Rafael de la Camara, Fabio Ciceri, Selim Corbacioglu, Harry Dolstra, Bertram Glass, Michelle Kenyon, Donal P. McLornan, Benedicte Neven, Regis Peffault de Latour, Zinaida Peric, Annalisa Ruggeri, John A. Snowden, Anna Sureda, Isabel Sanchez-Ortega
Summary: Clinical patient care for hematopoietic cell transplantation and cellular therapy varies between countries and centers. To harmonize clinical practices, European guidelines will be developed by the EBMT PH&G committee through workshops with experts. These guidelines aim to provide clear and practical recommendations in the absence of international consensus.
BONE MARROW TRANSPLANTATION
(2023)
Letter
Biophysics
Raffaella Greco, Jorinde D. Hoogenboom, Edouard F. Bonneville, Achilles Anagnostopoulos, Angela Cuoghi, Jean-Hugues Dalle, Eva M. Weissinger, Peter Lang, Federica Galaverna, Massimo Martino, Alexei Maschan, Christine Mauz-Korholz, Maddalena Noviello, Jakob Passweg, Jacopo Peccatori, Montserrat Rovira, Carlos Solano, Hendrik Veelken, Andrea Velardi, Eva Maria Wagner-Drouet, Xi Zhang, Fabio Ciceri, Chiara Bonini, Luca Vago, Annalisa Ruggeri, Christian Chabannon
BONE MARROW TRANSPLANTATION
(2023)
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
Oncology
Giuseppina Arbore, Luca Albarello, Gabriele Bucci, Marco Punta, Andrea Cossu, Lorella Fanti, Aurora Maurizio, Francesco Di Mauro, Vito Bilello, Gianluigi Arrigoni, Silvia Bonfiglio, Donatella Biancolini, Francesco Puccetti, Ugo Elmore, Luca Vago, Stefano Cascinu, Giovanni Tonon, Riccardo Rosati, Giulia Casorati, Paolo Dellabona
Summary: Current treatment for locally advanced esophageal adenocarcinoma involves neoadjuvant chemotherapy (nCT) before surgery, but response rates are low. This study examines the immune mechanisms involved in nCT response and identifies potential immune signatures that could be used for personalized treatment. The results show that patients with a complete response to nCT have increased immune response and proliferation, along with specific immune cell populations and cytokine profiles. These findings suggest that baseline tumor immunity plays a crucial role in the clinical activity of nCT and could be used to stratify patients for tailored treatments.
Review
Allergy
Alessandro Bruno, Carmelo Gurnari, Tobias Alexander, John A. Snowden, Raffaella Greco
Summary: VEXAS is a novel disease with a variety of clinical features, mainly affecting men and presenting symptoms in the fifth to sixth decade of life. It has attracted medical interest due to its association with multiple medical conditions. However, its recognition in everyday clinical practice is challenging and requires close collaboration between different medical specialists. The treatment and diagnostic guidelines for VEXAS are exploratory and include rheumatological and supportive care treatments. Allogeneic hematopoietic stem cell transplantation may be curative, but its position in the treatment algorithm is yet to be defined.
JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY
(2023)
Article
Immunology
Raffaella Greco, John A. Snowden, Nina Simone Knelange, Gloria Tridello, Carlotta Cacciatore, Alienor Xhaard, Fabio Ciceri, Matthew Collin, Christelle Ferra, Ann De Becker, Manuela Badoglio, Dina Averbuch, Tobias Alexander, Per Ljungman, Rafael De la Camara
Summary: This multicenter observational study reported the clinical data on COVID-19 in 11 patients affected by severe autoimmune diseases treated with HSCT. All patients had symptoms during the initial phase of the SARS-CoV-2 infection. Four cases developed lower respiratory tract disease. Hospitalization was required in 6 cases, but none needed ICU admission or ventilation/supplemental oxygen. Different interventions were adopted and all patients survived and resolved the infection at the last follow-up.
JOURNAL OF AUTOIMMUNITY
(2023)
Article
Oncology
Elisabetta Sauta, Marie Robin, Matteo Bersanelli, Erica Travaglino, Manja Meggendorfer, Lin-Pierre Zhao, Juan Carlos Caballero Berrocal, Claudia Sala, Giulia Maggioni, Massimo Bernardi, Carmen Di Grazia, Luca Vago, Giulia Rivoli, Lorenza Borin, Saverio D'Amico, Cristina Astrid Tentori, Marta Ubezio, Alessia Campagna, Antonio Russo, Daniele Mannina, Luca Lanino, Patrizia Chiusolo, Luisa Giaccone, Maria Teresa Voso, Marta Riva, Esther Natalie Oliva, Matteo Zampini, Elena Riva, Olivier Nibourel, Marilena Bicchieri, Niccolo' Bolli, Alessandro Rambaldi, Francesco Passamonti, Victor Savevski, Armando Santoro, Ulrich Germing, Shahram Kordasti, Valeria Santini, Maria Diez-Campelo, Guillermo Sanz, Francesc Sole, Wolfgang Kern, Uwe Platzbecker, Lionel Ades, Pierre Fenaux, Torsten Haferlach, Gastone Castellani, Matteo Giovanni Della Porta
Summary: The study validates the effectiveness of the Molecular International Prognostic Scoring System (IPSS-M) in predicting clinical outcomes of patients with myelodysplastic syndromes (MDS). IPSS-M improves the prognostic discrimination of the Revised International Prognostic Scoring System (IPSS-R) and enhances the accuracy of selecting candidates for hematopoietic stem cell transplantation (HSCT).
JOURNAL OF CLINICAL ONCOLOGY
(2023)
Article
Multidisciplinary Sciences
Matteo Maria Naldini, Gabriele Casirati, Matteo Barcella, Paola Maria Vittoria Rancoita, Andrea Cosentino, Carolina Caserta, Francesca Pavesi, Erika Zonari, Giacomo Desantis, Diego Gilioli, Matteo Giovanni Carrabba, Luca Vago, Massimo Bernardi, Raffaella Di Micco, Clelia Di Serio, Ivan Merelli, Monica Volpin, Eugenio Montini, Fabio Ciceri, Bernhard Gentner
Summary: Relapse in acute myeloid leukemia may be driven by the presence of leukemia stem cells. The authors use single-cell RNA sequencing to identify and characterize leukemia stem cells, and find that miR-126 is a potential marker of resistance. miR-126 high-expressing leukemia stem cells are enriched in chemotherapy-refractory AML and at relapse, and their transcriptional signature can robustly stratify patients for survival.
NATURE COMMUNICATIONS
(2023)
Article
Immunology
Chiara Oltolini, Andrea Acerbis, Giorgio Orofino, Sara Racca, Maddalena Noviello, Stefania Dispinseri, Nicola Clementi, Simona Piemontese, Elisabetta Xue, Fabio Giglio, Maria Teresa Lupo Stanghellini, Elisa Diral, Alessandro Bruno, Elena Tassi, Valeria Beretta, Ilaria Marzinotto, Gabriella Scarlatti, Vito Lampasona, Anna Ardemagni, Michela Sampaolo, Chiara Bonini, Consuelo Corti, Jacopo Peccatori, Antonella Castagna, Fabio Ciceri, Raffaella Greco
Summary: This report describes a case of a 34-year-old patient with active SARS-CoV-2 infection and high-risk leukemia who underwent allogeneic hematopoietic stem cell transplantation. Timely anti-SARS-CoV-2 preventive therapies and prompt management of transplant-related complications resulted in a favorable outcome.
FRONTIERS IN IMMUNOLOGY
(2023)
