journal
https://read.qxmd.com/read/38380373/interferons-in-the-treatment-of-myeloproliferative-neoplasms
#1
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
#2
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
#3
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
#4
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
https://read.qxmd.com/read/38186637/treatment-of-multiple-myeloma-with-selinexor-a-review
#5
REVIEW
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, relevant primary clinical trials, and recent developments in both patients with relapsed/refractory myeloma and patients with newly diagnosed myeloma...
2024: Therapeutic Advances in Hematology
https://read.qxmd.com/read/38145059/twenty-years-of-evolution-of-cml-therapy-how-the-treatment-goal-is-moving-from-disease-to-patient
#6
REVIEW
Domenico Russo, Michele Malagola, Nicola Polverelli, Mirko Farina, Federica Re, Simona Bernardi
The introduction of imatinib in 2000 opened the era of tyrosine kinase inhibitors (TKIs) for CML therapy and has revolutionized the life expectancy of CML patients, which is now quite like the one of the healthy aged population. Over the last 20 years, both the TKI therapy itself and the objectives have undergone evolutions highlighted and discussed in this review. The main objective of the CML therapy in the first 10 years after TKI introduction was to abolish the disease progression from the chronic to the blastic phase and guarantee the long-term survival of the great majority of patients...
2023: Therapeutic Advances in Hematology
https://read.qxmd.com/read/38105771/efficacy-of-complement-inhibitors-for-patients-with-paroxysmal-nocturnal-hemoglobinuria-a-systematic-review-and-meta-analysis
#7
REVIEW
Jiyeon Lee, Haeseon Lee, Siin Kim, Hae Sun Suh
BACKGROUND: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired hematological disease. The development of complement inhibitors such as eculizumab, ravulizumab, and pegcetacoplan has revolutionized the management of PNH, leading to improvements in overall survival and quality of life for patients. OBJECTIVES: This systematic review aims to provide comprehensive evidence of the efficacy of complement inhibitors in relation to treatment duration. DESIGN: This is a systematic review and meta-analysis...
2023: Therapeutic Advances in Hematology
https://read.qxmd.com/read/38105770/therapy-for-patients-with-chronic-phase-chronic-myeloid-leukemia-previously-treated-with-%C3%A2-2-tyrosine-kinase-inhibitors-a-systematic-literature-review
#8
REVIEW
Ehab Atallah, Lovneet Saini, Rodrigo Maegawa, Tanvi Rajput, Regina Corbin, Ricardo Viana
BACKGROUND: ATP-competitive tyrosine kinase inhibitors (TKIs) are the current standard of care for patients with chronic phase-chronic myeloid leukemia (CP-CML) in the first-line and second-line (2 L) setting. Treatment after 2 L is not clearly established. OBJECTIVE: The objective of this study was to summarize the available evidence to compare the efficacy and safety of interventions in the treatment of CP-CML patients who had received ⩾2 prior TKIs. DESIGN: A systematic literature review was performed...
2023: Therapeutic Advances in Hematology
https://read.qxmd.com/read/38033755/low-dose-decitabine-for-previously-untreated-acute-myeloid-leukemia-ineligible-for-intensive-chemotherapy-aged-65%C3%A2-years-or-older-a-prospective-study-based-on-comprehensive-geriatric-assessment
#9
JOURNAL ARTICLE
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 assessment. DESIGN: We performed a prospective, multicenter, open-label, and non-randomized study...
2023: Therapeutic Advances in Hematology
https://read.qxmd.com/read/37954483/prognostic-and-clinicopathological-impacts-of-systemic-immune-inflammation-index-on-patients-with-diffuse-large-b-cell-lymphoma-a-meta-analysis
#10
JOURNAL ARTICLE
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. DESIGN: This meta-analysis was conducted according to the Preferred Reporting Items for Systematic Reviews and Meta-Analyses statement...
2023: Therapeutic Advances in Hematology
https://read.qxmd.com/read/37929079/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
#11
JOURNAL ARTICLE
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, multicenter study. METHODS: We enrolled patients treated with TKIs for at least 3 years with a confirmed sustained deep molecular response (DMR) for at least 1 year...
2023: Therapeutic Advances in Hematology
https://read.qxmd.com/read/37929078/the-clinical-experience-of-compassionate-use-program-for-avapritinib-implications-for-drug-positioning-in-the-therapeutic-scenario-of-systemic-mastocytosis
#12
JOURNAL ARTICLE
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 of adults with AdvSM by the regulatory agencies US FDA and EMA. The present article reports the experience of treating SM patients with avapritinib in an Italian compassionate use program...
2023: Therapeutic Advances in Hematology
https://read.qxmd.com/read/37872970/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
#13
JOURNAL ARTICLE
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 was estimated based on a population pharmacokinetics model...
2023: Therapeutic Advances in Hematology
https://read.qxmd.com/read/37869360/rilzabrutinib-versus-placebo-in-adults-and-adolescents-with-persistent-or-chronic-immune-thrombocytopenia-luna-3-phase-iii-study
#14
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
#15
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
#16
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
#17
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
#18
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
#19
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
#20
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
journal
journal
43357
1
2
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.