-
A phase I trial of pevonedistat in combination with ruxolitinib for the treatment of myelofibrosis Ther. Adv. Hematol. (IF 3.4) Pub Date : 2024-03-13 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
-
Avatrombopag for the salvage treatment of platelet transfusion refractoriness Ther. Adv. Hematol. (IF 3.4) Pub Date : 2024-03-13 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
-
Progress and prospect of ASCT combined with CAR-T therapy in the treatment of multiple myeloma Ther. Adv. Hematol. (IF 3.4) Pub Date : 2024-03-13 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
-
An occurrence of eosinophilic folliculitis and alopecia associated with a sustained complete response to mogamulizumab in Sézary syndrome: a case report Ther. Adv. Hematol. (IF 3.4) Pub Date : 2024-03-06 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
-
Interferons in the treatment of myeloproliferative neoplasms Ther. Adv. Hematol. (IF 3.4) Pub Date : 2024-02-20 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
-
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 Ther. Adv. Hematol. (IF 3.4) Pub Date : 2024-02-16 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
-
When idiopathic multicentric Castleman disease meets COVID-19: a multicenter retrospective study from China Ther. Adv. Hematol. (IF 3.4) Pub Date : 2024-02-14 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
-
Treatment of multiple myeloma with selinexor: a review Ther. Adv. Hematol. (IF 3.4) Pub Date : 2024-01-05 Qianlei Huang, Ranran Zhao, Lu Xu, Xinbao Hao, Shi Tao
Over the last 20 years, breakthroughs in accessible therapies for the treatment of multiple myeloma (MM) have been made. Nevertheless, patients with MM resistant to immunomodulatory drugs, proteasome inhibitors, and anti-CD38 monoclonal antibodies have a very poor outcome. Therefore, it is necessary to explore new drugs for the treatment of MM. This review summarizes the mechanism of action of selinexor
-
Outcomes and effect of somatic mutations after erythropoiesis stimulating agents in patients with lower-risk myelodysplastic syndromes Ther. Adv. Hematol. (IF 3.4) Pub Date : 2024-01-04 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
-
Low-dose decitabine for previously untreated acute myeloid leukemia ineligible for intensive chemotherapy aged 65 years or older: a prospective study based on comprehensive geriatric assessment. Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-11-28 Ru Feng,Shuai Zhang,Jiang-Tao Li,Ting Wang,Chun-Li Zhang,Jie-Fei Bai,Lei Yang,Li-Ru Wang,Hong-Mei Jing,Hui Liu
Background The outcome of patients with acute myeloid leukemia (AML) aged ⩾65 years is poor. Effective treatment options are limited for patients with AML who cannot tolerate intensive chemotherapy. Objectives We aimed to evaluate the efficacy of low-dose decitabine in previously untreated patients with AML aged ⩾65 years who were ineligible for intensive chemotherapy based on a comprehensive geriatric
-
Prognostic and clinicopathological impacts of systemic immune-inflammation index on patients with diffuse large B-cell lymphoma: a meta-analysis. Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-11-10 Zaijing Fan,Lihong Shou
Background The systemic immune-inflammation index (SII) represents the immunoinflammatory score and can be considered as a prognostic marker; however, its relevance to the prognosis in patients with diffuse large B-cell lymphoma (DLBCL) remains unclear. Objectives The present meta-analysis was conducted to comprehensively evaluate the relationship between the SII and prognosis in patients with DLBCL
-
The clinical experience of compassionate use program for avapritinib: implications for drug positioning in the therapeutic scenario of systemic mastocytosis. Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-11-02 Francesco Mannelli,Francesca Crupi,Roberta Zanotti,Livio Pagano,Davide Rapezzi,Ilaria Tanasi,Marianna Criscuolo,Massimiliano Bonifacio,Alberto Fresa,Paola Guglielmelli,Alessandro M Vannucchi
In systemic mastocytosis, cytoreductive treatment is indicated for advanced systemic mastocytosis (AdvSM) variants. The treatment scenario is rapidly diversifying especially with the introduction of KIT tyrosine kinase inhibitors. Avapritinib is a second-generation potent and selective inhibitor of the mutant KIT D816V that, based on the results of pivotal clinical trials, was approved for the treatment
-
Effect of BCR::ABL1 transcript type and droplet digital polymerase chain reaction on successful treatment-free remission in chronic myeloid leukemia patients who discontinued tyrosine kinase inhibitor. Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-11-02 Hyunkyung Park,Hyeong-Joon Kim,Sang-Kyun Sohn,Yoonsuk Baik,Dongho Kim,Sung-Yeoun Lee,Jee Hyun Kong,Hawk Kim,Dong-Yeop Shin,Jae-Sook Ahn,Jinny Park,Seonyang Park,Inho Kim
Background Droplet digital polymerase chain reaction (ddPCR) is an exact method of measurement. Objectives We conducted this study to identify the prognostic factors for successful treatment-free remission in patients with chronic-phase chronic myeloid leukemia who discontinued tyrosine kinase inhibitors (TKIs). We also aimed to validate ddPCR for predicting molecular relapse. Design This is a prospective
-
Assessment of risk factors for acute graft-versus-host disease post-hematopoietic stem cell transplantation: a retrospective study based on a proportional odds model using a nonlinear mixed-effects model. Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-10-21 Ling Xue,Lin Song,Xun Yu,Xiao Yang,Fan Xia,Xiaoliang Ding,Chenrong Huang,Depei Wu,Liyan Miao
Background Acute graft-versus-host disease (aGVHD) is a major complication following hematopoietic stem cell transplantation (HSCT). Objective This study aimed to explore the risk factors for the incidence of aGVHD in patients post-HSCT. Design This was a retrospective study. Methods A total of 407 patients were enrolled. The patients' data were recorded from the medical records. The exposure of cyclosporine
-
Rilzabrutinib versus placebo in adults and adolescents with persistent or chronic immune thrombocytopenia: LUNA 3 phase III study. Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-10-18 David J Kuter,James B Bussel,Waleed Ghanima,Nichola Cooper,Terry Gernsheimer,Michele P Lambert,Howard A Liebman,Michael D Tarantino,Michelle Lee,Hailing Guo,Ahmed Daak
Background Immune thrombocytopenia (ITP) is characterized by primarily autoantibody-mediated platelet destruction and impaired platelet production resulting in thrombocytopenia and an increased risk of bleeding. Other manifestations include increased risk of thrombosis and diminished quality of life. Current treatment approaches are directed toward lowering the rate of platelet destruction or stimulating
-
Infrastructural considerations of implementing gene therapy for hemophilia in the Nordic context. Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-10-17 Jan Astermark,Fariba Baghaei,Karin Strandberg,Petra Gabric Toplican,Maj Friberg Birkedal,Emma Engman Grahn,Charlotta Hansson,Peter Kampmann,Anna-Elina Lehtinen,Kinga Täckström,Pål Andre Holme,Maria Magnusson
Background Despite improvements in hemophilia care, challenges remain, including treatment burden and impaired quality of life. Gene therapy may overcome these. However, its introduction presents a challenge. Objectives To outline a function-based gene therapy working model describing critical milestones associated with gene therapy handling, administration, and follow-up to facilitate and implement
-
The history of oral decitabine/cedazuridine and its potential role in acute myeloid leukemia. Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-10-17 Robert Briski,Guillermo Garcia-Manero,Hagop Kantarjian,Farhad Ravandi
Decitabine, a member of the 5-azanucleosides, has a dose-dependent mechanism of action in vitro: termination of DNA replication at high doses, and inhibition of DNA methyltransferase at low doses. The alteration of DNA methylation patterns by low-dose decitabine is hypothesized to upregulate genes, which promote myeloblast differentiation. In a phase III clinical trial, low-dose decitabine achieved
-
Survival outcomes in patients with relapsed/refractory or MRD-positive B-cell acute lymphoblastic leukemia treated with blinatumomab. Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-10-09 Hagop M Kantarjian,Aaron C Logan,Faraz Zaman,Nicola Gökbuget,Ralf C Bargou,Yi Zeng,Gerhard Zugmaier,Franco Locatelli
Blinatumomab has demonstrated significant efficacy in adult and pediatric patients with relapsed/refractory B-cell acute lymphoblastic leukemia (R/R B-cell ALL) and patients with measurable residual disease (MRD). This review aimed to compare median relapse-free survival (RFS) and median overall survival (OS) in adult and pediatric patients with R/R or MRD-positive B-cell ALL from pivotal studies [MT-103-211
-
Long-term outcome of progressive multifocal leukoencephalopathy with recombinant interleukin-2 treatment and an associated increase in the number of HPyV-2-specific T-cells: a case report. Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-10-09 Fieke W Hoff,John Rolwes,Paula A Hardeman,Molly Perkins,Eugene O Major,Daniel Douek,Robert H Collins,Benjamin M Greenberg
Progressive multifocal leukoencephalopathy (PML) is a demyelinating disease caused by reactivation of the human polyomavirus 2 (HPyV-2). PML is associated with a high morbidity and mortality rate and there is currently no standard curative therapy. We report short-term immunologic response and long-term clinical outcomes in a patient diagnosed with follicular lymphoma (FL) who developed PML. Diagnosis
-
Alterations of erythropoiesis in Covid-19 patients: prevalence of positive Coombs tests and iron metabolism. Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-09-28 Léa Schmitz,Michelle Pirotte,Alizée Lebeau,Marie Ernst,Marianne Fillet,Anais Devey,Justine Schmitt,Gaël Cobraiville,Marilène Binsfeld,Stéphanie Gofflot,Yves Beguin,Gaëlle Vertenoeil
Background For more than 2 years medical practice has been dealing with the Covid-19 pandemic. Atypical symptoms, such as frostbites and acrosyndromes, have appeared, and autoimmune anemias (some of which with cold agglutinins) have been described. Objectives We planned to study the prevalence of positive direct Coombs tests (DCTs) and hemolytic autoimmune anemia in patients infected with Severe Acute
-
Comparison of eltrombopag and avatrombopag in the treatment of refractory/relapsed aplastic anemia: a single-center retrospective study in China. Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-09-14 Zhuxin Zhang,Qinglin Hu,Chen Yang,Miao Chen,Bing Han
Background Eltrombopag (ELT), a thrombopoietin receptor agonist (TPO-RA), has been approved for relapsed/refractory aplastic anemia (AA). However, data on avatrombopag (AVA), another TPO-RA, are limited, and the comparisons between the two TPO-RAs are lacking. Objectives We aimed to compare the efficacy and safety between ELT and AVA in relapsed/refractory AA patients. Design In this retrospective
-
A new beginning: can omidubicel emerge as the next, viable alternative donor source? Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-08-30 Arpita P Gandhi,Laura F Newell,Richard T Maziarz
Umbilical cord blood (UCB) transplantation (CBT) has been an important alternative donor option for patients lacking matched related donor (MRD) or unrelated donor (URD) grafts. Only 30% of patients with high-risk hematologic malignancies have a human leukocyte antigen (HLA)-identical sibling; subjects without a MRD option are referred for HLA-matched URD selection, or utilize alternative donor sources
-
Anemia is associated with long-term exposure to PM2.5 and its components: a large population-based study in Southwest China. Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-08-28 Congyuan He,Linshen Xie,Lingxi Gu,Hongyu Yan,Shiyu Feng,Chunmei Zeng,Wangjiu Danzhen,Xuehui Zhang,Mingming Han,Zhifeng Li,Zhuoma Duoji,Bing Guo,Juying Zhang,Feng Hong,Xing Zhao
Background Anemia is linked to PM2.5 (particulate matter with aerodynamic diameters of ⩽2.5 μm) exposure, which can increase the risk of various negative health outcomes. It remains unclear which PM2.5 components are associated with anemia and the respective contribution of each component to this association. Objective This study aimed at investigating the association between PM2.5 and anemia in the
-
Luspatercept for transfusion-dependent β-thalassemia: time to get real. Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-08-26 Khaled M Musallam,Sujit Sheth,Maria Domenica Cappellini,Antonis Kattamis,Kevin H M Kuo,Ali T Taher
-
Non-invasive prenatal testing for fetal Ss, Kidd, and CTL2 blood group prediction by multiplex digital droplet PCR. Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-08-11 Yufeng Wang,Xiaoyue Chu,Xihui Chen,Anan Yin,Yan Yao,Li Wang,Hua Xu,Fangfang Liu,Kun Chen,Yuanming Wu
Background Some blood groups, such as S and s blood groups in the MNS blood group system, and Kidd and CTL2 blood group systems, can cause severe fetal and newborn alloimmune disorders. Non-invasive prenatal testing (NIPT) to predict fetal blood groups and knowledge of local blood group gene frequency are both important for pregnancy management decisions. Droplet digital PCR (ddPCR) has high specificity
-
Immune dysregulation and potential targeted therapy in myelodysplastic syndrome. Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-08-02 Xiaoying Zhang,Xingcheng Yang,Ling Ma,Yicheng Zhang,Jia Wei
Myelodysplastic syndrome (MDS) is a heterogeneous group of clonal hematological diseases and a high risk for transformation to acute myeloid leukemia (AML). The identification of key genetic alterations in MDS has enhanced our understanding of the pathogenesis and evolution. In recent years, it has been found that both innate and adaptive immune signaling are activated in the hematopoietic niche of
-
Real-world data in patients with congenital hemophilia and inhibitors: final data from the FEIBA Global Outcome (FEIBA GO) study. Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-07-24 Carmen Escuriola Ettingshausen,Cedric Hermans,Pål A Holme,Ana R Cid,Kate Khair,Johannes Oldenburg,Claude Négrier,Jaco Botha,Aurelia Lelli,Jerzy Windyga
Background The bypassing agent, activated prothrombin complex concentrate [aPCC, FEIBA (factor VIII inhibitor bypass activity); Baxalta US Inc, a Takeda company, Lexington, MA, USA], is indicated for the treatment of bleeding episodes, perioperative management, and routine prophylaxis in patients with hemophilia A or B with inhibitors. In certain countries, aPCC is also indicated for the treatment
-
REFLECT: prospective multicenter non-interventional study evaluating the effectiveness and safety of Sandoz rituximab (SDZ-RTX; Rixathon®) in combination with CHOP for the treatment of patients with previously untreated CD20-positive diffuse large B-cell lymphoma. Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-07-22 Manfred Welslau,Boris Kubuschok,Julian Topaly,Burkhard Otremba,Thomas Wolff,Galyna Bryn
Background REFLECT is the first prospective study of Sandoz biosimilar rituximab (SDZ-RTX) in patients with diffuse large B-cell lymphoma (DLBCL). Objective To evaluate the 2-year effectiveness and safety of SDZ-RTX as first-line treatment for DLBCL. Design Real-world, multicenter, open-label, single-arm, non-interventional, post-approval study of SDZ-RTX in combination with cyclophosphamide, doxorubicin
-
Targeting an elevated FVIII level using personalized rurioctocog alfa pegol prophylaxis in specific patient populations with hemophilia A: post hoc subanalysis of the randomized, phase 3 PROPEL study. Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-07-15 Carmen Escuriola-Ettingshausen,Robert Klamroth,Miguel Escobar,Oleksandra Stasyshyn,Srilatha Tangada,Werner Engl,Ivan Honauer,Hye-Youn Lee,Pratima Chowdary,Jerzy Windyga
Background The phase 3, prospective PROPEL study demonstrated that pharmacokinetic (PK)-guided prophylaxis targeting elevated factor VIII (FVIII) troughs in patients with hemophilia A resulted in lower annualized bleeding rates (ABRs) and a higher proportion of patients experiencing zero bleeds in the second 6 months of treatment when targeting a FVIII trough of 8-12% versus 1-3%. Objective To investigate
-
Avatrombopag treatment response in patients with immune thrombocytopenia: the REAL-AVA 1.0 study. Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-07-13 Abiola Oladapo,Scott Kolodny,Michael Vredenburg,Elyse Swallow,Debbie Goldschmidt,Kirthana Sarathy,Priscilla Lopez,Hillary Maitland,John Yee
Background Thrombopoietin-receptor agonists (TPO-RAs) are used to treat immune thrombocytopenia (ITP), a disorder characterized by prolonged low platelet counts (PCs) that pose a risk of serious bleeding episodes. Avatrombopag (AVA) is the most recently approved TPO-RA for the treatment of chronic ITP. A high proportion of patients responded to AVA in clinical trials, and treatment was well-tolerated;
-
Treating accelerated and blast phase myeloproliferative neoplasms: progress and challenges. Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-06-22 Helen O Ajufo,Julian A Waksal,John O Mascarenhas,Raajit K Rampal
Myeloproliferative neoplasms (MPNs) are a group of clonal hematologic malignancies that include polycythemia vera (PV), essential thrombocythemia (ET), and myelofibrosis (MF). MPNs are characterized by activating mutations in the JAK/STAT pathway and an increased risk of transformation to an aggressive form of acute leukemia, termed MPN-blast phase (MPN-BP). MPN-BP is characterized by the presence
-
Real-world patient characteristics and treatment patterns of polycythemia vera in Taiwan between 2016 and 2017: a nationwide cross-sectional study. Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-06-15 Tsung-Hsien Tsai,Lennex Hsueh-Lin Yu,Ming-Sun Yu,Shih-Hao Huang,Alex Jia-Hong Lin,Kuan-Der Lee,Min-Chi Chen
Background Polycythemia vera (PV) patients often experience constitutional symptoms and are at risk of thromboembolism as well as disease progression to myelofibrosis or acute myeloid leukemia. Not only is PV often overlooked but treatment options are also limited, however. Objectives To explore the patient characteristics and treatment pattern of PV patients in Taiwan, and compare with other countries
-
Epigenetic targets in B- and T-cell lymphomas: latest developments. Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-05-30 Marcelo Lima Ribeiro,Salvador Sánchez Vinces,Laura Mondragon,Gael Roué
Non-Hodgkin's lymphomas (NHLs) comprise a diverse group of diseases, either of mature B-cell or of T-cell derivation, characterized by heterogeneous molecular features and clinical manifestations. While most of the patients are responsive to standard chemotherapy, immunotherapy, radiation and/or stem cell transplantation, relapsed and/or refractory cases still have a dismal outcome. Deep sequencing
-
Phase I/II results of ceralasertib as monotherapy or in combination with acalabrutinib in high-risk relapsed/refractory chronic lymphocytic leukemia. Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-05-30 Wojciech Jurczak,Nagah Elmusharaf,Christopher P Fox,William Townsend,Amanda G Paulovich,Jeffrey R Whiteaker,Fanny Krantz,Chuan-Chuan Wun,Graeme Parr,Shringi Sharma,Veerendra Munugalavadla,Richa Manwani,Emma Dean,Talha Munir
Background Patients with relapsed/refractory (R/R) chronic lymphocytic leukemia (CLL) have limited treatment options. Ceralasertib, a selective ataxia telangiectasia and Rad-3-related protein (ATR) inhibitor, demonstrated synergistic preclinical activity with a Bruton tyrosine kinase (BTK) inhibitor in TP53- and ATM-defective CLL cells. Acalabrutinib is a selective BTK inhibitor approved for treatment
-
Recombinant factor IX Fc prophylaxis reduces pain and increases levels of physical activity, with sustained, long-term improvements in patients with hemophilia B: post hoc analysis of phase III trials using patient-reported outcomes. Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-05-29 Jan Astermark,Cédric Hermans,Monia Ezzalfani,Alaeddine Sidhom,Sylvaine Barbier,Nana Kragh,Aletta Falk,Daniel Eriksson
Background Pain is a common symptom of hemophilia that may adversely affect patients' quality of life (QoL). Previous post hoc analyses of prophylaxis with recombinant factor IX Fc fusion protein (rFIXFc) have been published for adults and adolescents, demonstrating improvements in health-related QoL (HRQoL) when assessed by the haemophilia-specific QoL (HaemAQoL) questionnaire. Objective To describe
-
Cost-effectiveness analysis of axicabtagene ciloleucel as a second-line treatment for diffuse large B-cell lymphoma in China and the United States. Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-05-23 Na Li,Jianying Lei,Jiahao Zhang,Hongfu Cai,Bin Zheng,Ting Yang,Maobai Liu,Jianda Hu
Background Axicabtagene ciloleucel (Axi-cel) is the first Chimeric Antigen Receptor T-Cell Immunotherapy (CAR-T) product approved in China for treating adult patients with relapsed or refractory large B-cell lymphoma after receiving second-line or above systemic therapy. However, it cannot be widely used in clinical practice due to its high price. Objectives To evaluate the economic value of Axi-cel
-
Innovations in hematopoietic stem-cell mobilization: a review of the novel CXCR4 inhibitor motixafortide. Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-05-23 Zachary D Crees,Michael P Rettig,John F DiPersio
Hematopoietic stem-cell transplantation (HCT) and stem-cell-based gene therapies rely on the ability to collect sufficient CD34+ hematopoietic stem and progenitor cells (HSPCs), typically via peripheral blood mobilization. Commonly used HSPC mobilization regimens include single-agent granulocyte colony-stimulating factor (G-CSF), plerixafor, chemotherapy, or a combination of these agents. These regimens
-
Risk factors and prognosis of thrombocytopenia in people living with HIV/AIDS. Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-05-19 Xiaoyan Lv,Pengpeng Li,Pengpeng Yue,Ping Tang,Fuling Zhou
Background Thrombocytopenia is a common hematological manifestation in people living with human immunodeficiency virus (HIV) or acquired immune deficiency syndrome (AIDS; PLWHA). Data on the prognostic relationship and associated factors of thrombocytopenia and HIV infection in China are limited. Objectives We assessed the prevalence of thrombocytopenia, its association with prognosis, and analyzed
-
Efgartigimod alfa for the treatment of primary immune thrombocytopenia Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-05-10 Catherine Broome
Primary immune thrombocytopenia (ITP) is an acquired autoimmune disorder characterized by isolated thrombocytopenia. Most patients with ITP have antiplatelet antibodies of the immunoglobulin G (IgG...
-
The impacts of total body irradiation on umbilical cord blood hematopoietic stem cell transplantation Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-05-03 Hao Wang, Kristin N. Berger, Elizabeth L. Miller, Wei Fu, Larisa Broglie, Frederick D. Goldman, Heiko Konig, Su Jin Lim, Arthur S. Berg, Julie-An Talano, Melanie A. Comito, Sherif S. Farag, Jeffrey J. Pu
Background:Umbilical cord blood hematopoietic stem cells are commonly used for hematopoietic system reconstitution in recipients after umbilical cord blood transplantation (UCBT). However, the opti...
-
Brain iron content and cognitive function in patients with β-thalassemia Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-04-28 Meiru Bu, Xi Deng, Yu Zhang, Sean W. Chen, Muliang Jiang, Bihong T. Chen
Patients with β-thalassemia (β-TM) may have brain iron overload from long-term blood transfusions, ineffective erythropoiesis, and increased intestinal iron absorption, leading to cognitive impairm...
-
Efficacy and safety of chimeric antigen receptor T-cell (CAR-T) therapy in hematologic malignancies: a living systematic review on comparative studies Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-04-27 Luis Carlos Saiz, Leire Leache, Marta Gutiérrez-Valencia, Juan Erviti, María Ximena Rojas Reyes
Background:Chimeric antigen receptor T-cell (CAR-T) cell therapies have been claimed to be curative in responsive patients. Nonetheless, response rates can vary according to different characteristi...
-
Efficacy and safety of damoctocog alfa pegol prophylaxis in patients ⩾40 years with severe haemophilia A and comorbidities: post hoc analysis from the PROTECT VIII study Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-04-22 Mark T. Reding, Ingrid Pabinger, Pål Andrè Holme, Monika Maas Enriquez, Maria Elisa Mancuso, Shadan Lalezari, Wolfgang Miesbach, Giovanni Di Minno, Robert Klamroth, Cedric Hermans
Background:Advances in treatment have enabled patients with haemophilia A to live longer and therefore may be subjected to comorbidities associated with ageing, in addition to disease-associated mo...
-
Considerations for shared decision management in previously untreated patients with hemophilia A or B Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-04-17 Jan Astermark, Jan Blatný, Christoph Königs, Cédric Hermans, Victor Jiménez-Yuste, Daniel P. Hart
Recent advances in therapeutics are now providing a wide range of options for adults and children living with hemophilia. Although therapeutic choices are also increasing for the youngest individua...
-
Challenges in the treatment of melanoma with BRAF and MEK inhibitors in patients with sickle cell disease: case report and review of the literature Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-03-15 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,...
-
Outcomes of SARS-CoV-2 infection in Ph-neg chronic myeloproliferative neoplasms: results from the EPICOVIDEHA registry Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-03-11 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
Background:Patients with Philadelphia-negative chronic myeloproliferative neoplasms (MPN) typically incur high rates of infections and both drugs and comorbidities may modulate infection risk.Objec...
-
Efficacy and safety of venetoclax-based regimens for the treatment of relapsed/refractory multiple myeloma: a systemic review and meta-analysis Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-03-06 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), d...
-
Superior survival outcome of blinatumomab compared with conventional chemotherapy for adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia: a propensity score–matched cohort analysis Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-03-06 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 pre...
-
Use and positioning of fostamatinib in the management of primary chronic immune thrombocytopenia: an Italian expert opinion Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-02-28 Alessandro Lucchesi, Bruno Fattizzo, Valerio De Stefano, Marco Ruggeri, Sergio Siragusa, Nicola Vianelli, Francesco Zaja, Francesco Rodeghiero
Fostamatinib, a spleen tyrosine kinase (Syk) inhibitor, represents a new therapeutic opportunity for patients with immune thrombocytopenia (ITP) in Europe and Italy. However, the positioning of thi...
-
Application and investigation of thrombopoiesis-stimulating agents in the treatment of thrombocytopenia Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-02-27 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 proc...
-
Update on the role of gemtuzumab-ozogamicin in the treatment of acute myeloid leukemia Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-02-09 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...
-
Role of abatacept in the prevention of graft-versus-host disease: current perspectives Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-02-09 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...
-
Treatment de-escalation in Philadelphia chromosome–positive B-cell acute lymphoblastic leukemia: the emerging role of chemotherapy-free regimens Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-02-03 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...
-
Genetic lesions and targeted therapy in Hodgkin lymphoma Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-01-12 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 abnormalit...
-
Antihuman T lymphocyte porcine immunoglobulin combined with cyclosporine as first-line immunosuppressive therapy for severe aplastic anemia in China: a large single-center, 10-year retrospective study Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-01-12 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) si...
-
How to translate and implement the current science of gene therapy into haemophilia care? Ther. Adv. Hematol. (IF 3.4) Pub Date : 2023-01-12 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 (FVI...
-
Current state of CAR-T therapy for T-cell malignancies Ther. Adv. Hematol. (IF 3.4) Pub Date : 2022-12-26 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 applicatio...
-
Eltrombopag increases the hematopoietic supporting ability of mesenchymal stem/stromal cells Ther. Adv. Hematol. (IF 3.4) Pub Date : 2022-12-26 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...
-
CAR T-cell therapy for follicular lymphoma and mantle cell lymphoma Ther. Adv. Hematol. (IF 3.4) Pub Date : 2022-12-16 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-...
-
Monocyte or white blood cell counts and β2 microglobulin predict the durable efficacy of daratumumab with lenalidomide Ther. Adv. Hematol. (IF 3.4) Pub Date : 2022-12-13 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
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 daratu...