journal
https://read.qxmd.com/read/37869360/rilzabrutinib-versus-placebo-in-adults-and-adolescents-with-persistent-or-chronic-immune-thrombocytopenia-luna-3-phase-iii-study
#21
JOURNAL ARTICLE
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 platelet production to prevent bleeding. Rilzabrutinib is an oral, reversible, potent Bruton tyrosine kinase inhibitor that was specifically designed to treat immune-mediated diseases and mediates its therapeutic effect through a dual mechanism of action: (1) inhibiting B-cell activation and (2) interrupting antibody-coated cell phagocytosis by Fc gamma receptor in spleen and liver...
2023: Therapeutic Advances in Hematology
https://read.qxmd.com/read/37859645/infrastructural-considerations-of-implementing-gene-therapy-for-hemophilia-in-the-nordic-context
#22
JOURNAL ARTICLE
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 an effective infrastructure for gene therapy introduction. DESIGN: Literature review and consensus discussion among Hemophilia Comprehensive Care centers (HCCCs) in the Nordic region...
2023: Therapeutic Advances in Hematology
https://read.qxmd.com/read/37854355/the-history-of-oral-decitabine-cedazuridine-and-its-potential-role-in-acute-myeloid-leukemia
#23
REVIEW
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 a superior overall response rate (ORR) when compared with 'treatment choice' [consisting of low-dose cytarabine (80%) and supportive care (20%)] as a frontline treatment for elderly patients with acute myeloid leukemia (AML)...
2023: Therapeutic Advances in Hematology
https://read.qxmd.com/read/37822572/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
#24
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 of PML was established conclusively based on findings from a brain biopsy. The patient was treated with recombinant interleukin 2 (IL-2) and showed rapid clinical improvement...
2023: Therapeutic Advances in Hematology
https://read.qxmd.com/read/37822571/survival-outcomes-in-patients-with-relapsed-refractory-or-mrd-positive-b-cell-acute-lymphoblastic-leukemia-treated-with-blinatumomab
#25
REVIEW
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 and TOWER for adults with Philadelphia chromosome (Ph)-negative R/R B-cell ALL, ALCANTARA for adults with Ph-positive R/R B-cell ALL, MT-103-203 for adults with MRD-positive B-cell ALL, and MT-103-205 for pediatric patients with R/R B-cell ALL], with the median RFS and OS from retrospective analyses, country or ethnicity-specific studies, and studies based on real-world evidence (RWE) identified from a literature search...
2023: Therapeutic Advances in Hematology
https://read.qxmd.com/read/37780945/alterations-of-erythropoiesis-in-covid-19-patients-prevalence-of-positive-coombs-tests-and-iron-metabolism
#26
JOURNAL ARTICLE
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 Respiratory Syndrome Coronavirus 2 (SARS-CoV-2) and its correlation with complications, and then investigate the impact of the infection on iron metabolism...
2023: Therapeutic Advances in Hematology
https://read.qxmd.com/read/37719987/comparison-of-eltrombopag-and-avatrombopag-in-the-treatment-of-refractory-relapsed-aplastic-anemia-a-single-center-retrospective-study-in-china
#27
JOURNAL ARTICLE
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 study, patients with relapsed/refractory AA who had been treated with ELT ( N  = 45) or AVA ( N  = 30) alone and had compatible baseline hematological parameters were compared...
2023: Therapeutic Advances in Hematology
https://read.qxmd.com/read/37693118/impact-on-mental-health-disease-management-and-socioeconomic-modifications-in-hematological-patients-during-the-covid-19-pandemic-in-italy
#28
JOURNAL ARTICLE
Marianna De Muro, Annelot Julia Janssen, Sergio Amadori, Paolo de Fabritiis, Dante Sabatino, Pasquale Niscola, Lorenza Torti, Malgorzata Monika Trawinska, Cristiano Tesei, Felice Bombaci, Mario Tarricone, Monica Bocchia, Carmen Fava, Sara Galimberti, Alessandra Iurlo, Luigia Luciano, Elisabetta Abruzzese
BACKGROUND: Hematological patients are a highly vulnerable population with an increased risk of developing severe COVID-19 symptoms due to their immunocompromised status. COVID-19 has proven to cause serious mental health issues, such as stress, anxiety, and depression in the general population. However, data on the psycho-social impact of COVID-19 on hematological patients are lacking. OBJECTIVES: This study aims to examine the psychological well-being of hematological patients in Italy during the initial period of the COVID-19 pandemic...
2023: Therapeutic Advances in Hematology
https://read.qxmd.com/read/37664800/a-new-beginning-can-omidubicel-emerge-as-the-next-viable-alternative-donor-source
#29
REVIEW
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 such as HLA-mismatched URD, UCB, or haploidentical donor grafts. While CBT demonstrates an excellent graft- versus -leukemia (GVL) effect, use of UCB as a graft source is limited due to a lower cell dose that can result in delayed engraftment and an immature immune system with increased infectious risk as a consequence...
2023: Therapeutic Advances in Hematology
https://read.qxmd.com/read/37654523/anemia-is-associated-with-long-term-exposure-to-pm-2-5-and-its-components-a-large-population-based-study-in-southwest-china
#30
JOURNAL ARTICLE
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 general population and to identify the most critical PM2...
2023: Therapeutic Advances in Hematology
https://read.qxmd.com/read/37645382/luspatercept-for-transfusion-dependent-%C3%AE-thalassemia-time-to-get-real
#31
EDITORIAL
Khaled M Musallam, Sujit Sheth, Maria Domenica Cappellini, Antonis Kattamis, Kevin H M Kuo, Ali T Taher
No abstract text is available yet for this article.
2023: Therapeutic Advances in Hematology
https://read.qxmd.com/read/37575175/non-invasive-prenatal-testing-for-fetal-ss-kidd-and-ctl2-blood-group-prediction-by-multiplex-digital-droplet-pcr
#32
JOURNAL ARTICLE
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 and sensitivity in detecting fetal single nucleotide variation. OBJECTIVES: The objective is to predict fetal Ss, Kidd, and CTL2 blood groups using multiplex ddPCR...
2023: Therapeutic Advances in Hematology
https://read.qxmd.com/read/37564898/treating-accelerated-and-blast-phase-myeloproliferative-neoplasms-progress-and-challenges
#33
REVIEW
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 of ⩾20% blasts in the blood or bone marrow and is almost always preceded by an accelerated phase (MPN-AP) defined as ⩾10-19% blasts in the blood or bone marrow...
2023: Therapeutic Advances in Hematology
https://read.qxmd.com/read/37547364/immune-dysregulation-and-potential-targeted-therapy-in-myelodysplastic-syndrome
#34
REVIEW
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 MDS with aberrant cytokine secretion in the bone marrow microenvironment. It is also clear that immune dysregulation plays an important role in the occurrence and progression of MDS, especially the destruction of the bone marrow microenvironment, including hematopoiesis and stromal components...
2023: Therapeutic Advances in Hematology
https://read.qxmd.com/read/37529276/real-world-data-in-patients-with-congenital-hemophilia-and-inhibitors-final-data-from-the-feiba-global-outcome-feiba-go-study
#35
JOURNAL ARTICLE
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 of bleeding episodes and perioperative management in patients with acquired hemophilia A...
2023: Therapeutic Advances in Hematology
https://read.qxmd.com/read/37492394/reflect-prospective-multicenter-non-interventional-study-evaluating-the-effectiveness-and-safety-of-sandoz-rituximab-sdz-rtx-rixathon-%C3%A2-in-combination-with-chop-for-the-treatment-of-patients-with-previously-untreated-cd20-positive-diffuse-large-b-cell-lymphoma
#36
JOURNAL ARTICLE
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, vincristine, and prednisone (R-CHOP) in patients with treatment-naïve CD20‑positive DLBCL...
2023: Therapeutic Advances in Hematology
https://read.qxmd.com/read/37465396/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
#37
JOURNAL ARTICLE
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 the benefit of PK-guided prophylaxis with rurioctocog alfa pegol targeting two FVIII trough levels in specific patient subgroups in a post hoc analysis using data from PROPEL...
2023: Therapeutic Advances in Hematology
https://read.qxmd.com/read/37465395/avatrombopag-treatment-response-in-patients-with-immune-thrombocytopenia-the-real-ava-1-0-study
#38
JOURNAL ARTICLE
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; however, limited real-world effectiveness data have been reported to date. OBJECTIVES: To describe demographic and clinical characteristics, treatment patterns, and outcomes following the initiation of AVA in patients with ITP in the United States...
2023: Therapeutic Advances in Hematology
https://read.qxmd.com/read/37426835/use-and-positioning-of-fostamatinib-in-the-management-of-primary-chronic-immune-thrombocytopenia-an-italian-expert-opinion
#39
REVIEW
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 this drug in patient's therapeutic sequence is undefined within the most recent international guidelines. The conclusions from a consensus meeting between Italian experts, whose task was to outline the profile of the ideal candidate to receive fostamatinib, are reported here. A modified Delphi methodology was used to achieve shared statements, which were reported in a narrative form...
2023: Therapeutic Advances in Hematology
https://read.qxmd.com/read/37359893/real-world-patient-characteristics-and-treatment-patterns-of-polycythemia-vera-in-taiwan-between-2016-and-2017-a-nationwide-cross-sectional-study
#40
JOURNAL ARTICLE
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 reported in the literature. DESIGN: This is a nationwide cross-sectional study...
2023: Therapeutic Advances in Hematology
journal
journal
43357
2
3
Fetch more papers »
Fetching more papers... Fetching...
Remove bar
Read by QxMD icon Read
×

Save your favorite articles in one place with a free QxMD account.

×

Search Tips

Use Boolean operators: AND/OR

diabetic AND foot
diabetes OR diabetic

Exclude a word using the 'minus' sign

Virchow -triad

Use Parentheses

water AND (cup OR glass)

Add an asterisk (*) at end of a word to include word stems

Neuro* will search for Neurology, Neuroscientist, Neurological, and so on

Use quotes to search for an exact phrase

"primary prevention of cancer"
(heart or cardiac or cardio*) AND arrest -"American Heart Association"

We want to hear from doctors like you!

Take a second to answer a survey question.