Hematology
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During the last four decades, much progress has been made in understanding the molecular pathogenesis of acute myeloid leukemia and in identifying prognostic factors predictive of outcome. However, progress in therapy has been much slower. Since the initial description of the combination of an anthracycline and cytarabine for induction, few major advances have changed the standard of care. ⋯ Daunorubicin dose intensification improves outcome in younger patients. Finally, allogeneic hematopoietic cell transplantation is an effective strategy for many patients in first CR. The discovery of drugs with novel mechanisms of action which are directly at specific molecular targets is among the most exciting areas of research and holds great promise for the development of effective treatment.
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The telomeropathies are a newly described group of human diseases based on the genetics and molecular biology of the telomeres, the ends of chromosomes. Telomeres are repeated hexanucleotides and their associated proteins; the protect chromosomes from recognition as damaged DNA, and their inevitable gradual loss with DNA replication is harmless as they are noncoding. However, when telomeres become critically short in a cell, senescence, apoptosis, or, rarely malignant transformation results. ⋯ Chromosome instability is a result of critical shortening of telomeres and cancer. For example, short telomeres are the major prognostic risk factor for clonal evolution to myelodysplasia and acute leukemia. Practically, hematologists need to recognize the multisystem presentation of telomere disease, implications for outcomes, and options for therapy.
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In order to maximize the impact and outcome of comprehensive care, it is important to track the identification of people with bleeding disorders and to evaluate their health outcomes over a long-term period. ⋯ The challenges of answering government and payer demands for evidence-based medicine and cost justification for the introduction and enhancement of treatment and care are ever-present and growing. To sustain and continue the expansion of access to care globally it is critical to build the body of outcome data for individual patients, within HTCs, nationally, regionally and globally. Doing so will not only improve clinical practices and support the allocation of scare resources, but most importantly, the well-being of patients will improve as well.
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Public health surveillance is the ongoing collection, analysis, and dissemination of health related data to provide information that can be used to monitor and improve the health of populations. Such surveillance systems can be established in many settings to study a variety of populations and conditions. ⋯ Data from surveillance systems have been used to identify risk factors for complications that, once identified, have been modified through public health interventions. The effectiveness of these interventions can be assessed by continued surveillance, thereby assuring improvement in care of people affected by hemophilia around the world.