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Topic Review
Wilms’ Tumor Gene
WT1 was initially identified as a tumor-suppressor gene involved in the pathogenesis of childhood renal Wilms’ tumor. The gene is located on chromosome 11 (band 11p13) and encodes for a zinc finger DNA-binding protein with four major isoforms, each of which plays a significant role in normal gene function.
  • 316
  • 22 Jun 2022
Topic Review
Von Willebrand Factor, ADAMTS13 and Cardiac Disease
This entry briefly describes the involvement of VWF (von Willebrand factor) and ADAMTS13 (a disintegrin and metalloproteinase with thrombospondin type 1 motif-13) in the pathophysiology of cardiac disease.
  • 505
  • 24 Nov 2021
Topic Review
Venous Thromboembolism Prevention in Multiple Myeloma
Multiple myeloma (MM) is associated with an increased risk of thrombotic complications, which remains substantial despite the implementation of thromboprophylaxis. The procoagulant state that characterizes the disease is multifactorial, and a greater understanding of the underlying pathophysiology is required to inform appropriate thrombosis prevention. There is a shift towards using direct oral anticoagulants (DOACs) in this setting; head-to-head comparisons in the context of controlled clinical trials between class agents are still missing. MM-specific venous thromboembolism (VTE) risk assessment scores have been developed to optimize management and minimize the associated mortality/morbidity. Their clinical utility remains to be evaluated. 
  • 467
  • 29 Dec 2022
Topic Review
Venetoclax in the Treatment of Younger AML Patients
The combination approach based on venetoclax (VEN) with azacytidine (AZA) has significantly improved outcomes for elderly patients with acute myeloid leukemia (AML). This innovative approach has led to higher rates of overall response, measurable residual disease (MRD)-negative remissions, and overall survival compared with AZA monotherapy. As a result, this combination has emerged as the gold-standard treatment for elderly or unfit patients with AML who are not eligible for intensive therapy. In younger, fit patients with AML, intensive induction and consolidation chemotherapy is commonly used as a first-line approach; however, relapse continues to be the main reason for treatment failure in approximately 30–40% of patients. Efforts to improve MRD-negative response rates and to facilitate the transition to allogeneic hematopoietic stem cell transplantation, particularly in high-risk AML, have inspired trials exploring the combination of intensive chemotherapy with targeted agents. VEN, a first-in-class anti-BCL2 agent, combined with intensive chemotherapy regimens has shown deep MRD-negative remissions, producing prolonged event-free survival and enhancing the transition to allogeneic transplant in first-complete-remission patients. These benefits support the incremental advantages of adding VEN to intensive chemotherapy approaches across ELN risk subcategories, and provides a robust benchmark to design future trials.
  • 86
  • 09 Jan 2024
Topic Review
Venetoclax in Relapsed/Refractory Acute Myeloid Leukemia
Relapsed and/or refractory (R/R) acute myeloid leukemia (AML) represents one of the most challenging scenarios in hematology, with a 5-year survival of only 10%. Venetoclax is a small molecule that acts as a potent, selective inhibitor of BCL-2. Given its satisfactory safety profile, VEN-based combinations are increasingly being investigated as a part of the therapeutic strategy for R/R AML. On the other hand, the issue of overcoming resistance is one of the most important fields to be addressed in upcoming clinical research.
  • 426
  • 22 May 2023
Topic Review
Venetoclax Combination Therapies in Pediatric Hematological Malignancies
Venetoclax is a strongly effective B-cell lymphoma-2 inhibitor (BCL-2) with an ability to selectively restore the apoptotic potential of cancerous cells. It has been proven that in combination with immunotherapy, targeted therapies, and lower-intensity therapies such as hypomethylating agents (HMAs) or low-dose cytarabine (LDAC), the drug can improve overall outcomes for adult patients with acute myeloid leukemia (AML), chronic lymphocytic leukemia (CLL), and multiple myeloma (MM), amongst other hematological malignancies.
  • 60
  • 07 Feb 2024
Topic Review
Updates in Anticoagulation Therapy Monitoring
Anticoagulation is indicated in a broad range of clinical scenarios, including (but not limited to) the management of venous and/or arterial thromboembolism, treatment of disseminated intravascular coagulation, the flushing of lines such as in hemodialysis, cardiopulmonary bypass, or extracorporeal membrane oxygenation (ECMO).
  • 564
  • 20 Apr 2021
Topic Review
Treatment Strategies in Chronic Myeloid Leukaemia
Chronic myeloid leukaemia (CML) is a type of blood cancer that is currently well-managed with drugs that target cancer-causing proteins. However, a significant proportion of CML patients do not respond to those drug treatments or relapse when they stop those drugs because the cancer cells in those patients stop relying on that protein and instead develop a new way to survive. Therefore, new treatment strategies may be necessary for those patients.
  • 535
  • 21 Jul 2022
Topic Review
Treatment Strategies for Older Myeloma Patients
While novel therapies have improved outcomes in multiple myeloma (MM), physicians are calling for greater caution when managing this hematologic malignancy in older patients due to their fragility, which increases their vulnerability to toxic events. Additionally, this patient population may be excluded from clinical trials due to comorbidities, whereby available data are not always applicable in real-word clinical practice.
  • 163
  • 08 Jun 2023
Topic Review
Treatment of Acute Myeloid Leukemia
Acute myeloid leukemia (AML) is a heterogenous disease with a broad spectrum of cytogenetic and molecular aberrations contributing to the definition of distinct AML subgroups. Treatment options for patients suffering from AML are continuously expanding and targeted therapies are available for distinct molecularly defined subgroups. Nevertheless, AML treatment remains challenging; in particular, patients with high-risk AML not eligible for intensive treatment or allogeneic hematopoietic stem cell transplantation (alloHSCT) are characterized by an unfavorable outcome.
  • 365
  • 02 Dec 2021
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