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Panagiotis T Diamantopoulos, Amalia Anastasopoulou, Maria Dimopoulou, Michalis Samarkos, Helen Gogas
Patients with sickle cell disease (SCD) suffer from complications due to anemia, inflammation, and vaso-occlusion. Factors that trigger sickling and/or inflammation may initiate such complications, while treatment with hydroxyurea (HU) reduces their emergence and prolongs survival. On the contrary, inhibition of the BRAF-MEK-ERK pathway with BRAF and MEK inhibitors (BRAF/MEKi) has revolutionized treatment of melanoma but their use has been correlated with inflammatory adverse events. Thus, treatment of patients with SCD with BRAF/MEKi may be quite challenging and pyrexia in those patients should be managed as a medical emergency...
2023: Therapeutic Advances in Hematology
#2
JOURNAL ARTICLE
Monia Marchetti, Jon Salmanton-García, Shaimaa El-Ashwah, Luisa Verga, Federico Itri, Zdeněk Ráčil, Julio Dávila-Valls, Sonia Martín-Pérez, Jaap Van Doesum, Francesco Passamonti, Ghaith Abu-Zeinah, Francesca Farina, Alberto López-García, Giulia Dragonetti, Chiara Cattaneo, Maria Gomes Da Silva, Yavuz M Bilgin, Pavel Žák, Verena Petzer, Andreas Glenthøj, Ildefonso Espigado, Caterina Buquicchio, Valentina Bonuomo, Lucia Prezioso, Stef Meers, Rafael Duarte, Rui Bergantim, Ozren Jaksic, Natasha Čolović, Ola Blennow, Martin Cernan, Martin Schönlein, Michail Samarkos, Maria Enza Mitra, Gabriele Magliano, Johan Maertens, Marie-Pierre Ledoux, Moraima Jiménez, Fatih Demirkan, Graham P Collins, Alba Cabirta, Stefanie K Gräfe, Anna Nordlander, Dominik Wolf, Elena Arellano, Raul Cordoba, Michaela Hanakova, Giovanni Paolo Maria Zambrotta, Raquel Nunes Rodrigues, Giulia Limberti, Francesco Marchesi, Oliver A Cornely, Livio Pagano
BACKGROUND: Patients with Philadelphia-negative chronic myeloproliferative neoplasms (MPN) typically incur high rates of infections and both drugs and comorbidities may modulate infection risk. OBJECTIVES: The present study aims to assess the effect of immunosuppressive agents on clinical outcomes of MPN patients affected by the coronavirus disease 2019 (COVID-19). DESIGN: This is an observational study. METHODS: We specifically searched and analyzed MPN patients collected by EPICOVIDEHA online registry, which includes individuals with hematological malignancies diagnosed with COVID-19 since February 2020...
2023: Therapeutic Advances in Hematology
#3
JOURNAL ARTICLE
Linchun Xu, Shaoze Lin, Xueyang Xing, Yongzhong Su
BACKGROUND: Patients with relapsed/refractory multiple myeloma (RRMM) usually have dismal prognostic outcomes. Venetoclax, a selective inhibitor of antiapoptotic protein B-cell lymphoma-2 (BCL-2), demonstrates antimyeloma activity in plasma cells with t(11;14) or high BCL-2 expression. OBJECTIVES: This meta-analysis aimed to investigate the efficacy and safety of venetoclax-based therapy in RRMM. DESIGN: This is a meta-analysis study. DATA SOURCES AND METHODS: PubMed, Embase, and Cochrane were searched for studies published up to 20 December 2021...
2023: Therapeutic Advances in Hematology
#4
JOURNAL ARTICLE
Jae-Ho Yoon, Daehun Kwag, Jong-Hyuk Lee, Gi June Min, Sung-Soo Park, Silvia Park, Sung-Eun Lee, Byung-Sik Cho, Ki-Seong Eom, Yoo-Jin Kim, Hee-Je Kim, Chang-Ki Min, Seok-Goo Cho, Jong Wook Lee, Seok Lee
BACKGROUND: Blinatumomab showed a higher complete remission (CR) rate and a safe bridging to allogeneic hematopoietic cell transplantation (allo-HCT) in adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (R/R BCP-ALL). OBJECTIVES: We tried to analyze the outcome of blinatumomab compared with the real-world historical data. We expected superior outcome of blinatumomab compared with historical conventional chemotherapy. DESIGN: We conducted a retrospective study using real-world data in the Catholic Hematology Hospital...
2023: Therapeutic Advances in Hematology
#5
REVIEW
Lejun Huang, Jianxuan Xu, Huaying Zhang, Mengfan Wang, Yiyue Zhang, Qing Lin
Platelets, derived from a certain subpopulation of megakaryocytes, are closely related to hemostasis, coagulation, metastasis, inflammation, and cancer progression. Thrombopoiesis is a dynamic process regulated by various signaling pathways in which thrombopoietin (THPO)-MPL is dominant. Thrombopoiesis-stimulating agents could promote platelet production, showing therapeutic effects in different kinds of thrombocytopenia. Some thrombopoiesis-stimulating agents are currently used in clinical practices to treat thrombocytopenia...
2023: Therapeutic Advances in Hematology
#6
REVIEW
Mahesh Swaminathan, Jorge E Cortes
Gemtuzumab-ozogamicin (GO) is an antibody-drug conjugate (ADC) in which a monoclonal antibody targeting CD33 is covalently linked to the toxin calicheamicin. GO was initially approved by the United States Food and Drug Administration (FDA) for the treatment of adult patients with CD33+ acute myeloid leukemia (AML) in 2000. However, GO was recalled from the US market due to the lack of efficacy, and higher incidence of hepatotoxicities, including hepatic veno-occlusive disease (VOD), observed in phase 3 SWOG-0106 study...
2023: Therapeutic Advances in Hematology
#7
REVIEW
Alexander Ngwube, Hemalatha Rangarajan, Niketa Shah
Administration of abatacept following transplantation has been reported to inhibit graft rejection and graft- versus -host-disease (GvHD) in mouse models associated with allogeneic hematopoietic stem cell transplant (HSCT). This strategy has recently been adopted in clinical practice for GvHD prevention in human allogeneic HSCT and offers a unique approach to optimizing GvHD prophylaxis following alternative donor HSCTs. When combined with calcineurin inhibitors and methotrexate, abatacept had shown to be safe and effective in preventing moderate to severe acute GvHD in myeloablative HSCT using human leukocyte antigen (HLA) unrelated donors...
2023: Therapeutic Advances in Hematology
#8
REVIEW
Fadi G Haddad, Jacki Sawyers, Nicholas J Short
The management of Philadelphia chromosome-positive (Ph-positive) acute lymphoblastic leukemia (ALL) has witnessed major progress over the past two decades. Initially, the incorporation of the first-generation BCR::ABL1 tyrosine kinase inhibitor (TKI) imatinib into intensive chemotherapy regimens improved outcomes compared with chemotherapy alone. The combinations of chemotherapy with second- or third-generation TKIs further improved outcomes, with higher rates of complete molecular remission (CMR) and superior survival...
2023: Therapeutic Advances in Hematology
#9
REVIEW
Cedric Hermans, Yves Gruel, Laurent Frenzel, Evelien Krumb
Gene-based therapy opens an entirely new paradigm in managing people with haemophilia (PWH), offering them the possibility of a functional cure by enabling continuous expression of factor VIII (FVIII) or factor IX (FIX) after transfer of a functional gene designed to replace the PWH's own defective gene. In recent years, significant advances in gene therapy have been made, resulting in clotting factor activity attaining near-normal levels, as reflected by 'zero bleeding rates' in previously severely inflicted patients following a single administration of adeno-associated viral (AAV) vectors...
2023: Therapeutic Advances in Hematology
#10
REVIEW
Zhe Li, Wei Mu, Min Xiao
Hodgkin lymphoma is a special type of lymphoma in which tumor cells frequently undergo multiple genetic lesions that are associated with accompanying pathway abnormalities. These pathway abnormalities are dominated by active signaling pathways, such as the JAK-STAT (Janus kinase-signal transducer and activator of transcription) pathway and the NFκB (nuclear factor kappa-B) pathway, which usually result in hyperactive survival signaling. Targeted therapies often play an important role in hematologic malignancies, such as CAR-T therapy (chimeric antigen receptor T-cell immunotherapy) targeting CD19 and CD22 in diffuse large B-cell lymphoma, while in Hodgkin lymphoma, the main targets of targeted therapies are CD30 molecules and PD1 molecules...
2023: Therapeutic Advances in Hematology
#11
JOURNAL ARTICLE
Wenrui Yang, Xu Liu, Xin Zhao, Li Zhang, Guangxin Peng, Lei Ye, Kang Zhou, Yuan Li, Jianping Li, Huihui Fan, Yang Yang, Youzhen Xiong, Liping Jing, Fengkui Zhang
BACKGROUND: Antihuman T lymphocyte porcine immunoglobulin (p-ATG) has been the most common ATG preparation in immunosuppressive therapy (IST) in Chinese patients with severe aplastic anemia (SAA) since 2009. OBJECTIVES: This study aimed to evaluate the early hematologic response and long-term outcomes of a large cohort of patients with SAA who received p-ATG plus cyclosporine (CsA) as first-line therapy from 2010 to 2019. DESIGN: This is a single-center retrospective study of medical records...
2023: Therapeutic Advances in Hematology
#12
REVIEW
Liangkui Luo, Xuan Zhou, Lijuan Zhou, Zhao Liang, Jilong Yang, Sanfang Tu, Yuhua Li
Chimeric antigen receptor T-cell (CAR-T) therapy has been approved for relapsed/refractory B-cell lymphomas and greatly improves disease outcomes. The impressive success has inspired the application of this approach to other types of tumors. The relapsed/refractory T-cell malignancies are characteristic of high heterogeneity and poor prognoses. The efficacy of current treatments for this group of diseases is limited. CAR-T therapy is a promising solution to ameliorate the current therapeutic situation. One of the major challenges is that normal T-cells typically share mutual antigens with malignant cells, which causes fratricide and serious T-cell aplasia...
2022: Therapeutic Advances in Hematology
#13
JOURNAL ARTICLE
Sandra Muntión, Silvia Preciado, Elena Sánchez-Luis, Luis Corchete, María Díez-Campelo, Lika Osugui, Gerardo-Javier Martí-Chillón, María-Belén Vidriales, Almudena Navarro-Bailón, Javier De Las Rivas, Fermín Sánchez-Guijo
BACKGROUND: Eltrombopag (EP) is a small molecule that acts directly on hematopoietic stem cells (HSCs) and megakaryocytes to stimulate the hematopoietic process. Mesenchymal stem/stromal cells (MSCs) are key hematopoietic niche regulators. OBJECTIVES: We aimed to determine whether EP has any effect on MSC function and properties (especially on their hematopoietic-supporting ability) and if so, what changes (e.g. genome-wide transcriptomic alterations) are induced in MSC after EP treatment...
2022: Therapeutic Advances in Hematology
#14
REVIEW
Razan Mohty, Mohamed A Kharfan-Dabaja
Patients with relapsed and/or refractory (R/R) follicular lymphoma (FL) and mantle cell lymphoma (MCL) have a poor prognosis with anticipated short progression-free and overall survivals. Two CD19-directed chimeric antigen receptor T-cell (CAR T) therapies are approved in the United States for R/R FL, namely, axicabtagene ciloleucel (axi-cel) and tisagenlecleucel. The results of ZUMA-5 and ELARA studies led to the approval of axi-cel and tisagenlecleucel, respectively, after demonstrating high overall (ORR) and complete response (CR) rates in this high-risk population of FL patients who had received a median of 3 (range = 2-4) and 4 (range = 2-13) prior lines of therapies, respectively...
2022: Therapeutic Advances in Hematology
#15
JOURNAL ARTICLE
Yutaka Shimazu, Junya Kanda, Hitomi Kaneko, Kazunori Imada, Ryosuke Yamamura, Satoru Kosugi, Yuji Shimura, Tomoki Ito, Shin-Ichi Fuchida, Hitoji Uchiyama, Kentaro Fukushima, Satoshi Yoshihara, Hitoshi Hanamoto, Hirokazu Tanaka, Nobuhiko Uoshima, Kensuke Ohta, Hideo Yagi, Hirohiko Shibayama, Yoshiyuki Onda, Yasuhiro Tanaka, Yoko Adachi, Mitsuhiro Matsuda, Masato Iida, Takashi Miyoshi, Toshimitsu Matsui, Ryoichi Takahashi, Teruhito Takakuwa, Masayuki Hino, Naoki Hosen, Shosaku Nomura, Chihiro Shimazaki, Itaru Matsumura, Akifumi Takaori-Kondo, Junya Kuroda
BACKGROUND: Daratumumab is one of the most widely used treatments for relapsed/refractory multiple myeloma (MM) patients. However, not all patients achieve a lasting therapeutic response with daratumumab. OBJECTIVES: We hypothesized that a durable response to daratumumab could be predicted by the balance between the MM tumor burden and host immune status. DESIGN: We conducted a retrospective study using the real-world data in the Kansai Myeloma Forum (KMF) database...
2022: Therapeutic Advances in Hematology
#16
REVIEW
Omar Albanyan, Julio Chavez, Javier Munoz
For approximately three decades, autologous hematopoietic cell transplantation (auto-HCT) has been the standard of care for patients with relapsed/refractory (R/R) diffuse large B-cell lymphoma (DLBCL) after frontline therapy. This approach is limited due to the intensity of chemotherapy and the proportion of patients who relapse after auto-HCT. Since the approval of anti-CD19 chimeric antigen receptor T-cell (CAR-T) therapy and novel agents, the treatment paradigm for DLBCL has changed remarkably. Anti-CD19 CAR-T therapy was first approved for relapsed DLBCL after two or more previous lines of therapy with long-lasting responses, with over 50% of patients still alive at 5-year follow-up...
2022: Therapeutic Advances in Hematology
#17
JOURNAL ARTICLE
Antonio Piga, Filomena Longo, Maria Rita Gamberini, Ersi Voskaridou, Paolo Ricchi, Vincenzo Caruso, Antonello Pietrangelo, Xiaosha Zhang, Jeevan K Shetty, Kenneth M Attie, Immacolata Tartaglione
BACKGROUND: β-thalassemia is a hereditary blood disorder resulting in ineffective erythropoiesis and anemia. Management of anemia with regular blood transfusions is associated with complications including iron overload. Here, we report long-term safety and efficacy results of the first clinical study of luspatercept in β-thalassemia, initiated in 2013, enrolling adults with both nontransfusion-dependent (NTD) and transfusion-dependent (TD) β-thalassemia. OBJECTIVES: The objective was to report long-term safety data, for up to 5 years of treatment, for 64 patients with TD or NTD β-thalassemia, and long-term efficacy data for a subset of 63 patients with β-thalassemia who received high-dose luspatercept (0...
2022: Therapeutic Advances in Hematology
#18
JOURNAL ARTICLE
Di Yao, Yuanyuan Tian, Jie Li, Bohan Li, Jun Lu, Jing Ling, Defei Zheng, Yanhua Yao, Peifang Xiao, Lijun Meng, Shaoyan Hu
BACKGROUND: Haploidentical hematopoietic stem cell transplantation (haplo-HSCT) based on granulocyte colony-stimulating factor plus anti-thymocyte regimens ('Beijing Protocol') provides a salvage treatment for patients of acquired severe aplastic anemia (SAA) in China. However, graft- versus -host disease (GVHD) is a major impediment of haplo-HSCT due to human leukocyte antigen disparity. Recently, haplo-HSCT combined with umbilical cord blood (UCB) (haplo-cord HSCT) is performed in clinical trials to potentially reduce the risk of severe GVHD...
2022: Therapeutic Advances in Hematology
#19
JOURNAL ARTICLE
Nan Zhang, Xiaoyan Liu, Jinxian Wu, Xinqi Li, Qian Wang, Guopeng Chen, Linlu Ma, Sanyun Wu, Fuling Zhou
Background: The clinical risk classification of acute myelocytic leukemia (AML) is largely based on cytogenetic and molecular genetic detection. However, the optimal treatment for intermediate-risk AML patients remains uncertain. Further refinement and improvement of prognostic stratification are therefore necessary. Objectives: The aim of this study was to identify serum protein biomarkers to refine risk stratification in AML patients. Design: This study is a retrospective study...
2022: Therapeutic Advances in Hematology
#20
JOURNAL ARTICLE
Anna Maria Frustaci, Giovanni Del Poeta, Andrea Visentin, Paolo Sportoletti, Alberto Fresa, Candida Vitale, Roberta Murru, Annalisa Chiarenza, Alessandro Sanna, Francesca Romana Mauro, Gianluigi Reda, Massimo Gentile, Marzia Varettoni, Claudia Baratè, Chiara Borella, Antonino Greco, Marina Deodato, Giulia Zamprogna, Roberta Laureana, Alessandra Cipiciani, Andrea Galitzia, Angelo Curto Pelle, Francesca Morelli, Lucio Malvisi, Marta Coscia, Luca Laurenti, Livio Trentin, Marco Montillo, Roberto Cairoli, Alessandra Tedeschi
BACKGROUND: The question of which parameters may be informative on venetoclax outcome in chronic lymphocytic leukemia (CLL) is still unclear. Furthermore, the choice to treat with venetoclax can be challenging in patients with baseline characteristics or comorbidities that may potentially favor some specific adverse events. OBJECTIVES: This study was aimed to evaluate whether age, fitness status, patients'/disease characteristics, or concomitant medications may predict outcomes in CLL patients receiving venetoclax...
2022: Therapeutic Advances in Hematology
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