Best practice & research. Clinical haematology
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Best Pract Res Clin Haematol · Jan 2005
ReviewThe blue cross blue shield assessment technology review: summary of findings.
Epoetin (EPO; erythropoietin) treatment offers an attractive but costly alternative to red blood cell transfusion for managing anemia associated with cancer therapy. The goal of this paper is to review the findings of the 2001 Blue Cross/Blue Shield Association Technology Evaluation Center overview on EPO use in oncology, which served the foundation for the 2002 ASH/ASCO clinical guideline on this subject. The BC/BS review had two major aims: (1) to quantify the effects of EPO on the likelihood of transfusion and on quality of life in patients with cancer treatment-related anemia, and (2) to evaluate whether outcomes are superior when EPO treatment is initiated at higher hemoglobin thresholds. ⋯ The review concluded that EPO reduced the odds of transfusion for cancer patients undergoing therapy. Evidence was insufficient to determine whether initiating EPO earlier spares more patients from transfusion or results in better quality of life than waiting until hemoglobin concentrations decline to nearly 10g/dL. An update of this review, which includes information on quality of life, transfusion requirment, survival and tumor response, for first and second-generation epoetin products is ongoing.
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Best Pract Res Clin Haematol · Jan 2005
ReviewEvidence-based medicine: can it be applied to stimulation of erythropoiesis for patients with malignancy?
Health care decision-making is affected by the values of patients and providers, available resources, and information substantiating effectiveness (or efficacy) of a particular therapy. A number of factors contribute to our growing need for evidence-based decision-making. Our aging population generally requires greater medical attention in a system with limited resources devoted to health care. ⋯ Physicians are increasingly turning to evidence-based medicine resources (guidelines, systematic reviews) to inform their decisions regarding application of new therapies. Evidence-based medicine offers health care decision-makers the opportunity for quality improvement, efficient resource allocation and utilization, informed policy-making and reimbursement, and identification of future research priorities. Judicious use of erythropoietic stimulants guided by evidence-based decision-making should ensure treatment of patients who can be reasonably expected to benefit with appropriate dose regimens, while preserving valuable health care resources in those situations where patients are not expected to derive significant health care benefit.
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Best Pract Res Clin Haematol · Sep 2004
ReviewGlobin gene transfer for treatment of the beta-thalassemias and sickle cell disease.
The beta-thalassemias and sickle cell disease are severe congenital anemias that are caused by mutations that alter the production of the beta chain of hemoglobin. Allogeneic hematopoietic stem cell (HSC) transplantation is curative, but this therapeutic option is not available to the majority of patients. The transfer of a functional globin gene in autologous HCSs thus represents a highly attractive alternative treatment. ⋯ Several groups have now confirmed and extended these findings in various mouse models of severe hemoglobinopathies, thus generating enthusiasm for a genetic treatment based on globin gene transfer. Furthermore, globin vectors represent a general paradigm for the regulation of transgene function and the improvement of vector safety by restricting transgene expression to the differentiated progeny within a single lineage, thereby reducing the risk of activating oncogenes in hematopoietic progenitors. Here we review the principles underlying the genesis of regulated vectors for stem cell therapy.
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Best Pract Res Clin Haematol · Mar 2004
ReviewNatural anticoagulant inhibitors: activated Protein C.
Protein C is a vitamin-K-dependent zymogen, whose congenital deficiency state leads to increased risk for venous thrombosis. Activated Protein C (aPC) exerts its anticoagulant function by inhibiting the cofactors in the clotting cascade, Factors Va and VIIIa. In addition, aPC displays anti-inflammatory, anti-apoptotic and profibrinolytic activities. ⋯ The major toxicity associated with treatment is bleeding. Appropriate use of rhAPC depends on an understanding of its mechanisms of action and risk:benefit profile. The goals of this review are: to describe the Protein C pathway; to discuss the definitions, epidemiology and pathophysiology of severe sepsis; to provide a conceptual framework for understanding the role of rhAPC in this syndrome; and to address frequently asked questions about the day-to-day use of this agent.
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Best Pract Res Clin Haematol · Sep 2003
ReviewRetinoic acid syndrome: manifestations, pathogenesis, and treatment.
All-trans retinoic acid (ATRA) is a potent differentiation agent that is effective therapy in acute promyelocytic leukaemia. Although ATRA is generally well tolerated, some patients develop retinoic acid syndrome. ⋯ However, if identified early enough, effective therapy can be administered. This chapter discusses the clinical aspects and pathogenesis of retinoic acid syndrome.