Article
Hematology
Jiapei Liu, Zhiping Fan, Na Xu, Jieyu Ye, Yanqiu Chen, Ruoyang Shao, Yiming Sun, Qiaoyuan Wu, Qifa Liu, Hua Jin
Summary: This study evaluated the effects of ruxolitinib compared with basiliximab in the treatment of steroid-refractory acute graft-versus-host disease (SR-aGVHD). The results showed that ruxolitinib had a higher overall response rate and effectively prevented chronic GVHD (cGVHD), leading to improved survival rates.
ANNALS OF HEMATOLOGY
(2023)
Article
Hematology
Jiapei Liu, Zhiping Fan, Na Xu, Jieyu Ye, Yanqiu Chen, Ruoyang Shao, Yiming Sun, Qiaoyuan Wu, Qifa Liu, Hua Jin
Summary: This study retrospectively evaluated the effects of ruxolitinib compared with basiliximab for steroid-refractory acute graft-versus-host disease (SR-aGVHD). The results showed that ruxolitinib had higher overall response and complete response rates, as well as lower incidence of chronic GVHD. Ruxolitinib also improved overall survival and failure-free survival rates. In addition, ruxolitinib significantly reduced the occurrence of EBV viremia. Therefore, ruxolitinib should be recommended for the treatment of SR-aGVHD.
ANNALS OF HEMATOLOGY
(2023)
Article
Medicine, General & Internal
Robert Zeiser, Nicola Polverelli, Ron Ram, Shahrukh K. Hashmi, Ronjon Chakraverty, Jan Moritz Middeke, Maurizio Musso, Sebastian Giebel, Ant Uzay, Peter Langmuir, Norbert Hollaender, Maanasa Gowda, Tommaso Stefanelli, Stephanie J. Lee, Takanori Teshima, Franco Locatelli
Summary: The study suggests that ruxolitinib can significantly improve overall response, failure-free survival, and symptom response in patients with glucocorticoid-refractory or -dependent chronic graft-versus-host disease (GVHD), although it may increase the incidence of thrombocytopenia and anemia.
NEW ENGLAND JOURNAL OF MEDICINE
(2021)
Review
Immunology
Shuang Fan, Wen-Xuan Huo, Yang Yang, Meng-Zhu Shen, Xiao-Dong Mo
Summary: This study reports the updated results of ruxolitinib as treatment for SR-GVHD and compares the efficacy and safety between children and adults. The findings suggest that ruxolitinib is an effective and safe treatment for SR-GVHD, and both children and adults can benefit from it.
FRONTIERS IN IMMUNOLOGY
(2022)
Article
Hematology
Sameem Abedin, Nahid Rashid, Mark Schroeder, Rizwan Romee, Mary Nauffal, Muhamad Alhaj Moustafa, Mohamed A. Kharfan-Dabaja, Jeanne Palmer, William Hogan, Mehrdad Hefazi, Samantha Larson, Shernan Holtan, Zachariah DeFilipp, Reena Jayani, Bhagirathbhai Dholaria, Joseph Pidala, Farhad Khimani, Michael R. Grunwald, Candace Butler, Mehdi Hamadani
Summary: Among 307 patients with SR-aGVHD, approximately 1/5 of patients showed resistance or intolerance to Ruxolitinib. The overall response rate to additional therapy was 36% in resistant patients with a median survival of 21 days; while 50% of intolerant patients continued to improve with additional therapy, with a median survival of 50 days. These data provide a baseline for future studies on SR-aGVHD.
BRITISH JOURNAL OF HAEMATOLOGY
(2021)
Article
Immunology
Dong Wang, Yin Liu, Xiaoxuan Lai, Jia Chen, Qiao Cheng, Xiao Ma, Zhihong Lin, Depei Wu, Yang Xu
Summary: The study showed that ruxolitinib is potentially a safe and effective treatment for patients with steroid-refractory chronic GVHD, with an overall response rate of 74.3%. The main adverse event was cytopenia. Treatment with ruxolitinib increased the percentage of CD4 cells, decreased the percentages of NK cells and regulatory T cells, and increased the proportion of total B cells.
FRONTIERS IN IMMUNOLOGY
(2021)
Article
Oncology
Elisabetta Xue, Francesca Lorentino, Francesca Pavesi, Andrea Assanelli, Jacopo Peccatori, Massimo Bernardi, Consuelo Corti, Fabio Ciceri, Maria Teresa Lupo Stanghellini
Summary: Chronic Graft versus Host Disease (GvHD) is a serious complication of allogeneic hematopoietic stem cell transplant that severely impacts quality of life and long-term survival. Ruxolitinib has been shown to be a safe and effective salvage treatment option for advanced stages of chronic GvHD, with an overall response rate of 59% at three months and 62% at six months. Further prospective analyses on larger cohorts are ongoing to evaluate the durability of response.
Article
Chemistry, Medicinal
Wenli Yang, Guanghua Zhu, Maoquan Qin, Zhigang Li, Bin Wang, Jun Yang, Tianyou Wang
Summary: The study found that Ruxolitinib is effective for acute and chronic graft-versus-host disease in children. Patients with chronic GVHD had a higher overall response rate and better prognosis after treatment compared to those with acute GVHD.
DRUG DESIGN DEVELOPMENT AND THERAPY
(2021)
Article
Chemistry, Medicinal
Cong Wei, Xiaoting Zhang, Dan Liang, Jilong Yang, Jingwen Du, Chunyan Yue, Lan Deng
Summary: Ruxolitinib has been shown to be a safe and effective treatment for steroid-refractory GVHD in a Chinese population, with high overall response rates in acute GVHD patients and comparable overall survival rates between responders and non-responders in chronic GVHD patients. Common adverse events include cytopenia, cytomegalovirus reactivation, and infections, particularly in patients with acute GVHD.
DRUG DESIGN DEVELOPMENT AND THERAPY
(2021)
Article
Dentistry, Oral Surgery & Medicine
Martina Kaurinovic, Konstantina Delli, Ana-Mae E. Jonk, Anouschka Biswana, Carin L. E. Hazenberg, Goda Choi, Marco R. de Groot, Linde M. Morsink, Arjan Vissink, Mar Bellido
Summary: The study aimed to assess the effect of ruxolitinib on the oral mucosa of SR-cGVHD patients with oral involvement. In an observational longitudinal patient study, 53 patients with SR-cGVHD and oral involvement were treated with ruxolitinib. The baseline condition of the oral mucosa was compared to its condition at 4 and 12 weeks after starting ruxolitinib.
CLINICAL ORAL INVESTIGATIONS
(2022)
Review
Immunology
Fathima A. Mohamed, Govindarajan Thangavelu, Stephanie Y. Rhee, Peter T. Sage, Roddy S. O'Connor, Jeffrey C. Rathmell, Bruce R. Blazar
Summary: The therapeutic efficacy of allo-HSCT is limited by the development of GVHD, with immune dysregulation leading to expansion of alloreactive cells and reduction in regulatory cells. New therapies are needed to restore the balance between effector and regulatory immune cells while preserving the beneficial graft-versus-tumor effect.
FRONTIERS IN IMMUNOLOGY
(2021)
Review
Immunology
Makoto Murata, Takanori Teshima
Summary: This article discusses the potential of MSC therapy in the treatment of acute GVHD, along with a comparison of studies on the two products, Remestemcel-L and Temcell. While Temcell's real-world efficacy was found to be equivalent to that observed in a prospective study, the precise position of Remestemcel-L/Temcell therapy in the treatment of acute GVHD remains to be determined.
FRONTIERS IN IMMUNOLOGY
(2021)
Article
Surgery
Bin Pan, Longmei Shang, Cong Liu, Jun Gao, Fan Zhang, Mengdi Xu, Lingling Li, Zengtian Sun, Zhenyu Li, Kailin Xu
Summary: The combined therapy of PD-1 antibody and ruxolitinib increased graft-versus-lymphoma effects without increasing acute graft-versus-host disease, leading to improved survival of recipients challenged by A20 lymphoma cells.
AMERICAN JOURNAL OF TRANSPLANTATION
(2021)
Article
Biophysics
Kristina Maas-Bauer, Chrissoula Kiote-Schmidt, Hartmut Bertz, Petya Apostolova, Ralph Waesch, Gabriele Ihorst, Juergen Finke, Robert Zeiser
Summary: The combination of Ruxolitinib and extracorporeal photopheresis has shown activity in treating glucocorticoid refractory chronic graft-versus-host disease, with a best response rate of 74%. However, some patients may experience newly diagnosed cytopenia and CMV reactivation. Further prospective trials are needed to validate the safety and efficacy of this combination therapy.
BONE MARROW TRANSPLANTATION
(2021)
Review
Oncology
Robert Q. Le, Xin Wang, Hongfei Zhang, Hongshan Li, Donna Przepiorka, Jonathon Vallejo, Ruby Leong, Lian Ma, Kirsten B. Goldberg, Richard Pazdur, Marc R. Theoret, Angelo De Claro
Summary: The Food and Drug Administration approved ruxolitinib for the treatment of chronic graft-versus-host disease (cGVHD) after the failure of systemic therapy. Ruxolitinib demonstrated higher response rates and longer duration of response compared to best available therapy (BAT), with manageable adverse reactions.
Review
Microbiology
Emilio Bouza, Miguel Martin, Jose Enrique Ales, Nuria Aragones, Begona Barragan, Rafael de la Camara, Jose Luis Del Pozo, Valentin Garcia-Gutierrez, Ramon Garcia-Sanz, Diego Gracia, Vicente Guillem, Victor Jimenez-Yuste, Mari Cruz Martin-Delgado, Joaquin Martinez, Rafael Lopez, Alvaro Rodriguez-Lescure, Julian Ruiz Galiana, Ana Maria Sureda, Francisco Tejerina-Picado, Antoni Trilla, Antonio Zapatero, Esteban Palomo, Jesus San-Miguel
Summary: The exact number of solid organ tumors diagnosed each year in Spain is unknown, making it difficult to determine if the pandemic has led to a decrease in cancer diagnoses. However, indirect data suggests that the pandemic has resulted in later-stage diagnoses for some non-hematological neoplasms. Although there is a lack of strong evidence, oncology patients who contract COVID-19 appear to have poorer outcomes. The immune response to infection in cancer patients is influenced by factors such as the type of neoplasia, treatment received, and timing of treatment.
REVISTA ESPANOLA DE QUIMIOTERAPIA
(2023)
Article
Hematology
Sara Redondo Velao, Irene Garcia Cadenas, Ma Angeles Cuesta, Isabel Sanchez-Ortega, Francesc Fernandez-Aviles, Elisa Roldan, Anna Torrent, M. Cruz Viguria, Sara Villar, Leyre Bento, Lucrecia Yanez, Rodrigo Martino, Jose Luis Pinana
Summary: This study investigated the occurrence of acute diarrhea in recipients of allogeneic hematopoietic stem cell transplantation. The main causes were lower GI-aGVHD, infections, and drug-related toxicity. Infectious causes were identified in only a small percentage of cases. The overall survival rate at 1 year was 69%.
ACTA HAEMATOLOGICA
(2023)
Article
Biophysics
Arnon Nagler, Myriam Labopin, Ryszard Swoboda, Alexander Kulagin, Andrea Velardi, Jaime Sanz, Helene Labussiere-Wallet, Victoria Potter, Juergen Kuball, Simona Sica, Elena Parovichnikova, Wolfgang Bethge, Natacha Maillard, Uwe Platzbecker, Friedrich Stoelzel, Fabio Ciceri, Mohamad Mohty
Summary: This study evaluated the outcome of second transplantation (HSCT2) for the treatment of primary graft failure (pGF) in 243 patients with acute leukemia. The median age was 44.8 years. 73.7% of patients achieved engraftment after HSCT2. The 5-year nonrelapse mortality was 51.6%, relapse incidence was 18.8%, leukemia-free survival was 29.6%, and overall survival was 30.7%.
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)
Article
Oncology
Rama Al Hamed, Maud Ngoya, Jacques-Emmanuel Galimard, Henrik Sengeloev, Tobias Gedde-Dahl, Aleksandr Kulagin, Uwe Platzbecker, Ibrahim Yakoub-Agha, Jenny L. Byrne, Thomas Valerius, Gerard Socie, Nicolaus Kroeger, Didier Blaise, Ali Bazarbachi, Jaime Sanz, Fabio Ciceri, Arnon Nagler, Mohamad Mohty
Summary: This retrospective study investigates the impact of time on allogeneic hematopoietic cell transplantation (allo-HCT) outcomes for patients with acute myeloid leukemia (AML) in second complete remission (CR2). The results show that outcomes of allo-HCT in CR2 have significantly improved over time, with the most favorable outcomes achieved with a matched sibling donor (MSD).
Article
Oncology
Guadalupe Onate, Marta Pratcorona, Ana Garrido, Alicia Artigas-Baleri, Alex Bataller, Mar Tormo, Montserrat Arnan, Susana Vives, Rosa Coll, Olga Salamero, Ferran Vall-Llovera, Antonia Sampol, Antoni Garcia, Marta Cervera, Sara Garcia Avila, Joan Bargay, Xavier F. Ortin, Josep Nomdedeu, Jordi Esteve, Jorge Sierra
Summary: This study analyzed 227 patients with FLT3-mutated acute myeloid leukemia (AML) and found that the addition of midostaurin to treatment improved outcomes, especially in patients with NPM1 mutations.
BLOOD CANCER JOURNAL
(2023)
Article
Pharmacology & Pharmacy
Yaiza Nunez, Annabel Garcia-Leon, Aida Falgas, Naroa Serna, Laura Sanchez-Garcia, Ana Garrido, Jorge Sierra, Alberto Gallardo, Ugutz Unzueta, Esther Vazquez, Antonio Villaverde, Ramon Mangues, Isolda Casanova
Summary: Researchers developed a protein nanoparticle, T22-PE24-H6, which can selectively deliver the exotoxin A to CXCR4(+) leukemic cells, and showed potent antitumor activity without toxicity.
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)
Article
Biophysics
Jaime Sanz, Myriam Labopin, Thomas Pabst, Jurjen Versluis, Gwendolyn Van Gorkom, Ellen Meijer, Tobias Gedde-Dahl, Juan Montoro, William Arcese, Jose Antonio Perez-Simon, Nicolaas Schaap, Johan Maertens, Radovan Vrhovac, Francesco Lanza, Norbert Claude Gorin, Mohamad Mohty, Fabio Ciceri
Summary: We compared the impact of different conditioning regimens, including BEA, BUCY, and BUMEL, on the outcomes of adult AML patients in CR1 undergoing ASCT. The BEA regimen showed better overall survival compared to BUCY and BUMEL. This retrospective study suggests that the high-dose myeloablative combination chemotherapy with BEA is a more effective treatment option for AML patients in CR1.
BONE MARROW TRANSPLANTATION
(2023)
Article
Biophysics
Enrico Maffini, Maud Ngoya, Jacques-Emmanuel Galimard, Samia Harbi, Nicolaus Kroeger, Uwe Platzbecker, Henrik Sengeloev, Charles Craddock, Victoria Potter, Goda Choi, Patrice Chevallier, Friedrich Stolzel, Eleni Tholouli, Johan Maertens, Fabio Ciceri, Jan Cornelissen, Jaime Sanz, Alexandros Spyridonidis, Francesco Lanza, Arnon Nagler, Mohamad Mohty
Summary: Allogeneic HCT programs are becoming accessible for older AML patients. Patients aged ≥70 years who received HCT from matched sibling, unrelated, or haploidentical donors showed good clinical outcomes, with reduced relapse rates and improved leukemia-free survival in haploidentical and unrelated donor transplantations compared to matched sibling transplantation.
BONE MARROW TRANSPLANTATION
(2023)
Article
Hematology
Frederic Baron, Arnon Nagler, Jacques-Emmanuel Galimard, Jaime Sanz, Jurjen Versluis, Edouard Forcade, Patrice Chevallier, Anne Sirvent, Chloe Anthias, Jurgen Kuball, Sabine Furst, Alessandro Rambaldi, Jorge Sierra, Peter A. von Dem Borne, Maria Pilar Gallego Hernanz, Thomas Cluzeau, Stephen Robinson, Anna Maria Raiola, Helene Labussiere-Wallet, Jenny L. Byrne, Jean-Valere Malfuson, Annalisa Ruggeri, Mohamad Mohty, Fabio Ciceri
Summary: We investigated whether secondary versus de novo AML would be associated with poor outcomes in adult acute AML patients in first complete remission (CR1) receiving unrelated cord blood transplantation (CBT). We observed non-significantly different leukaemia-free survival (LFS) and relapse incidence (RI) in de novo versus secondary AML patients.
BRITISH JOURNAL OF HAEMATOLOGY
(2023)
Letter
Oncology
Elena Sebastian, Laura Andres-Zallo, Marta Gonzalez-Vicent, Maria Jara, Blanca Molina, Josune Zubicaray, Eva Galvez, Alberto Orfao, Julian Sevilla
PEDIATRIC BLOOD & CANCER
(2023)
Article
Hematology
Isabel Regalado-Artamendi, Marta Garcia-Fasanella, Laura Medina, Jesus Fernandez-Sojo, Albert Esquirol, Irene Garcia-Cadenas, Rodrigo Martino, Javier Briones, Jorge Sierra, Silvana Novelli
Summary: This study describes transfusion support in lymphoma patients undergoing autologous stem cell transplant (ASCT) and identifies predictors of increased or prolonged transfusion requirement. Factors such as age over 60, pre-transplant anemia, pre-transplant thrombocytopenia, and CD34(+) cell dose <4x10(6)/kg are associated with increased transfusion requirements. These factors categorize patients into three groups with significant differences in time until transfusion independence.
Article
Hematology
Jorge Sierra, Pau Montesinos, Xavier Thomas, Laimonas Griskevicius, Thomas Cluzeau, Denis Caillot, Ollivier Legrand, Clara Minotti, Mario Luppi, Firas Farkas, Bourras-Rezki Bengoudifa, Geralyn Gilotti, Sejla Hodzic, Alessandro Rambaldi, Adriano Venditti
Summary: The pivotal RATIFY study demonstrated that midostaurin combined with standard chemotherapy significantly reduced mortality in adult patients with newly diagnosed FLT3(mut) acute myeloid leukemia. This open-label, multicenter phase 3b trial aimed to further assess the safety and efficacy of midostaurin plus chemotherapy in different age groups. The results showed that the safety and efficacy of midostaurin remained consistent with previous findings regardless of age, sex, or induction regimen.
