journal
https://read.qxmd.com/read/38911444/rapid-and-sustained-response-to-luspatercept-and-eltrombopag-combined-treatment-in-one-case-of-clonal-cytopenias-of-undetermined-significance-with-prior-failure-to-cyclosporin-and-androgen-therapy-a-case-report
#1
JOURNAL ARTICLE
Jing Xu, Yixin Yan, Siwen Zong, Wencan Ye, Jifu Zheng, Chao Min, Qingming Wang, Zhenjiang Li
Clonal cytopenia of undetermined significance (CCUS) has the characteristics of high-risk transformation into myelodysplastic syndromes. At present, there are few effective treatments for CCUS, and there is no consensus or evidence-based recommendation. We present a case demonstrating a rapid, significant and sustained response to combined treatment with luspatercept and eltrombopag, following the failure of cyclosporin and androgen therapy. Even after discontinuing luspatercept for 10 months, trilineage haematopoiesis remained normal with the use of cyclosporin and other haematopoietic stimulants...
2024: Therapeutic Advances in Hematology
https://read.qxmd.com/read/38883164/updates-of-primary-central-nervous-system-lymphoma
#2
REVIEW
Jiaying Wu, Delian Zhou, Xiaojian Zhu, Yicheng Zhang, Yi Xiao
Lymphoma occurring in the central nervous system is considered primary central nervous system lymphoma (PCNSL), usually without systematic lesions. Over the last few decades, a deep understanding of PCNSL has been lacking due to the low incidence rate, and the overall survival and progression-free survival of patients with PCNSL are lower than those with other types of non-Hodgkin lymphoma. Recently, there have been several advancements in research on PCNSL. Advances in diagnosis of the disease are primarily reflected in the promising diagnostic efficiency of novel biomarkers...
2024: Therapeutic Advances in Hematology
https://read.qxmd.com/read/38883163/integrated-genetic-epigenetic-and-immune-landscape-of-tp53-mutant-aml-and-higher-risk-mds-treated-with-azacitidine
#3
JOURNAL ARTICLE
Amer M Zeidan, Jan Philipp Bewersdorf, Vanessa Hasle, Rory M Shallis, Ethan Thompson, Daniel Lopes de Menezes, Shelonidta Rose, Isaac Boss, Stephanie Halene, Torsten Haferlach, Brian A Fox
BACKGROUND: TP53 mutations are associated with an adverse prognosis in acute myeloid leukemia (AML) and higher-risk myelodysplastic syndromes (HR-MDS). However, the integrated genetic, epigenetic, and immunologic landscape of TP53 -mutated AML/HR-MDS is not well defined. OBJECTIVES: To define the genetic, epigenetic, and immunologic landscape of TP53- mutant and TP53 wild-type AML and HR-MDS patients. DESIGN: Post hoc analysis of TP53- mutant and TP53 wild-type patients treated on the randomized FUSION trial with azacitidine ± the anti-PD-L1 antibody durvalumab...
2024: Therapeutic Advances in Hematology
https://read.qxmd.com/read/38883162/successful-maintenance-of-a-sustained-molecular-response-in-cml-patients-receiving-low-dose-tyrosine-kinase-inhibitors
#4
JOURNAL ARTICLE
Yan Li, Pu Kuang, Huanling Zhu, Ling Pan, Tian Dong, Ting Lin, Yu Chen, Yunfan Yang
BACKGROUND: The development of tyrosine kinase inhibitor (TKI) therapy has positively impacted the survival rates of patients with chronic myeloid leukemia (CML). It is common in medical practice to adjust the dosage of TKI downward because of TKI-associated adverse events, financial burden, comorbidity, or an attempt at treatment-free remission. OBJECTIVES: This investigation sought to explore the feasibility of employing a reduced dosage of TKI for treating CML...
2024: Therapeutic Advances in Hematology
https://read.qxmd.com/read/38832237/a-systematic-literature-review-of-74-chinese-blastic-plasmacytoid-dendritic-cell-neoplasm-patients
#5
REVIEW
Chen Gong, Ying Liu, Mingzhi Zhang
BACKGROUND: Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare and aggressive hematological cancer. Due to its low incidence, researchers struggle to gather sufficient prospective data to inform clinical treatment. OBJECTIVES: We sought to summarize the clinical characteristics and current treatment methods of BPDCN and provide more specific guidance on treatment options. DESIGN: A systematic literature review using data from 74 Chinese BPDCN patients...
2024: Therapeutic Advances in Hematology
https://read.qxmd.com/read/38828002/intensive-chemotherapy-with-dual-induction-and-all-like-consolidation-for-childhood-acute-myeloid-leukemia-a-respective-report-from-multiple-centers-in-china
#6
JOURNAL ARTICLE
Jia-Nan Li, Yi-Jun Chen, Zhong Fan, Qiao-Ru Li, Liu-Hua Liao, Zhi-Yong Ke, Yu Li, Li-Na Wang, Cui-Yun Yang, Xue-Qun Luo, Yan-Lai Tang, Xiao-Li Zhang, Li-Bin Huang
BACKGROUND: Pediatric acute myeloid leukemia (AML) has poor prognosis and high rate of relapse and mortality, and exploration of new treatment options is still critically needed. OBJECTIVES: To summarize the outcome of our new treatment strategies for pediatric AML, which is characterized by dual induction and acute lymphoblastic leukemia (ALL) elements consolidation. DESIGN: Retrospective, single-arm study. METHODS: From July 2012 to December 2019, an intensive chemotherapy protocol was used for newly diagnosed children with AML, which contains dual induction, three courses of consolidations based on high-dose cytarabine, and two courses of consolidations composed of high-dose methotrexate, vincristine, asparaginase, and mercaptopurine (ALL-like elements)...
2024: Therapeutic Advances in Hematology
https://read.qxmd.com/read/38737006/simoctocog-alfa-nuwiq-%C3%A2-in-children-early-steps-in-life-s-journey-for-people-with-severe-hemophilia-a
#7
REVIEW
Anna Klukowska, Robert F Sidonio, Guy Young, Maria Elisa Mancuso, María Teresa Álvarez-Román, Neha Bhatnagar, Martina Jansen, Sigurd Knaub
People with severe hemophilia A usually experience their first bleed early in life. In children with severe hemophilia A, primary prophylaxis is recommended to prevent recurrent and potentially life-threatening bleeds that significantly impact day-to-day life. Factor VIII (FVIII) prophylaxis is well-established in children and has been shown to reduce the development of hemophilic arthropathy. However, a major challenge of FVIII therapy is the development of neutralizing anti-FVIII antibodies (FVIII inhibitors)...
2024: Therapeutic Advances in Hematology
https://read.qxmd.com/read/38737005/development-of-a-nomogram-to-predict-the-risk-of-secondary-failure-of-platelet-recovery-in-patients-with-%C3%AE-thalassemia-major-after-hematopoietic-stem-cell-transplantation-a-retrospective-study
#8
JOURNAL ARTICLE
Yanni Xie, Gaohui Yang, Lin Pan, Zhaoping Gan, Yumei Huang, Yongrong Lai, Rongrong Liu
BACKGROUND: Secondary failure of platelet recovery (SFPR) is a common complication that influences survival and quality of life of patients with β-thalassemia major (β-TM) after hematopoietic stem cell transplantation (HSCT). OBJECTIVES: A model to predict the risk of SFPR in β-TM patients after HSCT was developed. DESIGN: A retrospective study was used to develop the prediction model. METHODS: The clinical data for 218 β-TM patients who received HSCT comprised the training set, and those for another 89 patients represented the validation set...
2024: Therapeutic Advances in Hematology
https://read.qxmd.com/read/38721038/treatment-of-multiple-myeloma-what-is-the-impact-on-t-cell-function
#9
REVIEW
Chenggong Li, Xindi Wang, Jia Xu, Jiachen Liu, Heng Mei
Treatment of multiple myeloma (MM) has evolved remarkably over the past few decades. Autologous stem cell transplantation, as well as proteasome inhibitors, immunomodulatory drugs, and monoclonal antibodies, has substantially improved the prognosis of patients with MM. Novel therapies, including chimeric antigen receptor-T cells, bispecific T-cell engagers, antibody-drug conjugates, histone deacetylase inhibitors, and nuclear export inhibitors, have provided more options. However, MM remains incurable. T cells are the principal weapons of antitumor immunity, but T cells display a broad spectrum of dysfunctional states during MM...
2024: Therapeutic Advances in Hematology
https://read.qxmd.com/read/38633898/improved-survival-outcomes-with-anakinra-over-etoposide-based-therapies-for-the-management-of-adults-with-hemophagocytic-lymphohistiocytosis-a-retrospective-multicenter-research-network-study
#10
JOURNAL ARTICLE
Benjamin J Lee
BACKGROUND: Hemophagocytic lymphohistiocytosis (HLH) is a rare life-threatening, hyperinflammatory syndrome for which etoposide-based regimens have historically been the standard of care. Recent reports have described positive outcomes with the utilization of ruxolitinib or anakinra although these studies are often limited to small samples. OBJECTIVES: We aimed to compare the efficacy of ruxolitinib, anakinra, and etoposide-based therapies for the management of HLH in adult patients...
2024: Therapeutic Advances in Hematology
https://read.qxmd.com/read/38628436/advancements-in-minimal-residual-disease-detection-a-practical-approach-using-single-cell-droplet-pcr-for-comprehensive-monitoring-in-hematological-malignancy
#11
JOURNAL ARTICLE
Satoshi Uchiyama, Kentaro Fukushima, Seiichiro Katagiri, Junichi Tsuchiya, Tomohiro Kubo, SungGi Chi, Yosuke Minami
The identification of chromosomal abnormalities accompanied by copy number alterations is important for understanding tumor characteristics. Testing methodologies for copy number abnormality have limited sensitivity, resulting in their use only for the sample provided at the time of diagnosis or recurrence of malignancy, but not for the monitoring of minimal residual disease (MRD) during and after therapy. We developped the "DimShift" technology which enable to measure the copy number of target gene/chromosome in each cell, which is given by the single cell droplet PCR...
2024: Therapeutic Advances in Hematology
https://read.qxmd.com/read/38558826/research-advances-on-short-chain-fatty-acids-in-gastrointestinal-acute-graft-versus-host-disease
#12
REVIEW
Xinping Song, Jing Lao, Lulu Wang, Sixi Liu
Gastrointestinal acute graft- versus -host disease (GI-aGVHD) is a severe early complication following allogeneic hematopoietic stem cell transplantation (allo-HSCT). It has been shown that the intestinal microbiota plays a critical role in this process. As metabolites of the intestinal microbiota, short-chain fatty acids (SCFAs) are vital for maintaining the host-microbiota symbiotic equilibrium. This article provides an overview of the protective effect of SCFAs in the gastrointestinal tract, emphasizes their association with GI-aGVHD, and explores relevant research progress in prevention and treatment research...
2024: Therapeutic Advances in Hematology
https://read.qxmd.com/read/38481949/progress-and-prospect-of-asct-combined-with-car-t-therapy-in-the-treatment-of-multiple-myeloma
#13
REVIEW
Shihui Yuan, Ying Chen, Huasheng Liu
Multiple myeloma (MM) is a hematological cancer characterized by abnormal proliferation of plasma cells in bone marrow. In recent years, autologous stem cell transplantation (ASCT) has become the cornerstone of MM treatment. At the same time, immunotherapy, such as monoclonal antibody therapy and chimeric antigen receptor T cell (CAR-T) has also emerged, in which CAR-T is the most attractive focus. ASCT and its myeloablative preconditioning will turn its immune microenvironment into an inhibited state, which may provide an opportunity for the expansion of CAR-T cells so as to further clear the residual lesions after ASCT and reduce the recurrence rate after ASCT...
2024: Therapeutic Advances in Hematology
https://read.qxmd.com/read/38481948/avatrombopag-for-the-salvage-treatment-of-platelet-transfusion-refractoriness
#14
JOURNAL ARTICLE
Yuehong Qin, Yu Wang, Yujiao Zhang, Yingying Jiao, Jieyu Ye
BACKGROUND: Platelet transfusion refractoriness (PTR) is a life-threatening and intractable condition in hematological patients. Thrombopoietin receptor agonists such as avatrombopag promote platelet production and modulate immune intolerance. However, its application in PTR has not been extensively studied. OBJECTIVES: We aimed to compare the platelet response (PR) as well as bleeding events and mortality rate between the best available therapies (BATs) and avatrombopag (Ava) treatments in refractory PTR patients...
2024: Therapeutic Advances in Hematology
https://read.qxmd.com/read/38481947/a-phase-i-trial-of-pevonedistat-in-combination-with-ruxolitinib-for-the-treatment-of-myelofibrosis
#15
JOURNAL ARTICLE
Tim Kong, Nicole Gaudin, Karyn Gordon, Maggie J Cox, Amy W Zhou, Stephen T Oh
Janus kinase 2 (JAK2) inhibitors such as ruxolitinib have become standard-of-care therapy for patients with myeloproliferative neoplasms (MPNs); however, activation of alternate oncogenic pathways including nuclear factor kappa-light-chain-enhancer of activated B cells (NFκB) has limited durable response as single-agent therapy. With the rationale of targeting both pathways, we conducted a phase I dose escalation trial of pevonedistat in combination with ruxolitinib for the treatment of patients with myelofibrosis (NCT03386214)...
2024: Therapeutic Advances in Hematology
https://read.qxmd.com/read/38456078/an-occurrence-of-eosinophilic-folliculitis-and-alopecia-associated-with-a-sustained-complete-response-to-mogamulizumab-in-s%C3%A3-zary-syndrome-a-case-report
#16
Jean-Matthieu L'Orphelin
Mogamulizumab is a monoclonal antibody that binds to C-C chemokine receptor 4 (CCR4), initiating antibody-dependent cellular cytotoxicity. CCR4 is highly expressed in the cutaneous T-cell lymphoma subtypes mycosis fungoides and Sézary syndrome (SS), and mogamulizumab has been shown to be effective in patients with these conditions who were refractory to at least one prior systemic treatment. One of the more common adverse events encountered with mogamulizumab is rash, which may mimic disease progression and lead to premature discontinuation...
2024: Therapeutic Advances in Hematology
https://read.qxmd.com/read/38380373/interferons-in-the-treatment-of-myeloproliferative-neoplasms
#17
REVIEW
Pankit Vachhani, John Mascarenhas, Prithviraj Bose, Gabriela Hobbs, Abdulraheem Yacoub, Jeanne M Palmer, Aaron T Gerds, Lucia Masarova, Andrew T Kuykendall, Raajit K Rampal, Ruben Mesa, Srdan Verstovsek
Interferons are cytokines with immunomodulatory properties and disease-modifying effects that have been used to treat myeloproliferative neoplasms (MPNs) for more than 35 years. The initial use of interferons was limited due to difficulties with administration and a significant toxicity profile. Many of these shortcomings were addressed by covalently binding polyethylene glycol to the interferon structure, which increases the stability, prolongs activity, and reduces immunogenicity of the molecule. In the current therapeutic landscape, pegylated interferons are recommended for use in the treatment of polycythemia vera, essential thrombocythemia, and primary myelofibrosis...
2024: Therapeutic Advances in Hematology
https://read.qxmd.com/read/38371314/the-effectiveness-and-safety-of-octocog-alfa-in-patients-with-hemophilia-a-up-to-7-year-follow-up-of-the-real-world-ahead-international-study
#18
JOURNAL ARTICLE
Margareth C Ozelo, Cedric Hermans, Manuel Carcao, Benoît Guillet, Joan Gu, Randy Guerra, Leilei Tang, Kate Khair
BACKGROUND: Real-world data assessing treatment outcomes in patients with hemophilia A in routine clinical practice are limited. OBJECTIVE: To evaluate the effectiveness and safety of octocog alfa in patients with moderate/severe hemophilia A receiving treatment in clinical practice. DESIGN: The international Antihemophilic Factor Hemophilia A Outcome Database study is an observational, noninterventional, prospective, multicenter study. METHODS: This planned interim data read-out was conducted following 7 years of observation of patients receiving octocog alfa (cut-off, 30 June 2020)...
2024: Therapeutic Advances in Hematology
https://read.qxmd.com/read/38357251/when-idiopathic-multicentric-castleman-disease-meets-covid-19-a-multicenter-retrospective-study-from-china
#19
JOURNAL ARTICLE
Yi Liu, Xuejiao Yin, Dan Xu, Yunfei Lv, Li Zhu, Min Yang, Qiumei Yao, Jie Jin, Li Huang, Haitao Meng, Liangshun You
Idiopathic multicentric Castleman disease (iMCD) is a rare and cytokine storm-driven inflammatory disorder. The exact cause of iMCD is still unknown, although several hypotheses have been proposed. However, regardless of the underlying cause, the ultimate result is the activation of the inflammatory pathway, which can lead to damage in multiple organs. Currently, there have been several reports highlighting the intricate link between coronavirus disease 2019 (COVID-19) and iMCD. To better understand the impact of COVID-19-induced immune storm on iMCD, we conducted a multicenter retrospective study in three hospitals in China...
2024: Therapeutic Advances in Hematology
https://read.qxmd.com/read/38186638/outcomes-and-effect-of-somatic-mutations-after-erythropoiesis-stimulating-agents-in-patients-with-lower-risk-myelodysplastic-syndromes
#20
JOURNAL ARTICLE
Juan Carlos Caballero, Julio Dávila, María López-Pavía, Esperanza Such, Teresa Bernal, Fernando Ramos, Marisa Calabuig, Jesús María Hernández Sánchez, Helena Pomares, Mercedes Sánchez Barba, María Abáigar, Bernardo González, Brayan Merchán, Reyes Sancho-Tello, Marta Callejas, Carolina Muñoz-Novas, Carlos Cerveró, Guillermo Sanz, Jesús María Hernández Rivas, María Díez Campelo
BACKGROUND: Erythropoiesis stimulating agents (ESAs) are the first-line therapy in patients with lower-risk myelodysplastic syndromes (LR-MDS). Some predictive factors for ESAs response have been identified. Type and number of somatic mutations have been associated with prognosis and response to therapies in MDS patients. OBJECTIVES: The objective was to evaluate the outcomes after ESAs in patients with LR-MDS and to address the potential predictive value of somatic mutations in ESAs-treated patients...
2024: Therapeutic Advances in Hematology
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