Paroxysmal Nocturnal Hemoglobinuria (PNH)
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Predictors for improvement in patient-reported outcomes: post hoc analysis of a phase 3 randomized, open-label study of eculizumab and ravulizumab in complement inhibitor-naive patients with paroxysmal nocturnal hemoglobinuria
Paroxysmal nocturnal hemoglobinuria Paroxysmal nocturnal hemoglobinuria: (pa
Clonal Hematopoiesis, Inflammation, and Hematologic Malignancy
Somatic or acquired mutations are postzygotic genetic variations that can occur within any tissue. These mutations accumulate during aging and have classically been linked to malignant processes. Tremendous advancements over the past years have led to a deeper understanding of the role of somatic mutations in benign and malignant age-related diseases. Here, we review the somatic mutations that accumulate in the blood and their connection to disease states, with a particular focus on inflammatory diseases and myelodysplastic syndrome.
What are Registries?
In the medical field, registries are systematic collections of data related to specific diseases, conditions, or treatments. They are databases designed to gather and store detailed information about patients' medical histories, diagnostic information, treatments, outcomes, and various other factors. The primary goal of medical registries is to advance medical research, improve patient care, and enhance healthcare practices by providing comprehensive and reliable data.
Types of Research
Medical research is classified into primary and secondary research. There are three main types of primary research: basic (experimental), clinical, and epidemiological research. Secondary research provides a summary of available studies through reviews and meta-analyses.
The Medical Research Process
The medical research process is a structured and methodical approach used to generate new knowledge and insights about various aspects of medicine and healthcare. It involves several stages, each aimed at addressing specific research questions, testing hypotheses, and contributing to advancements in medical science. Below are components of the typical medical research process:
A Journey from Blood Cells to Genes and Back
I was attracted to hematology because by combining clinical findings with the use of a microscope and simple laboratory tests, one could often make a diagnosis. I was attracted to genetics when I learned about inherited blood disorders, at a time when we had only hints that somatic mutations were also important. It seemed clear that if we understood not only what genetic changes caused what diseases but also the mechanisms through which those genetic changes contribute to cause disease, we could improve management.
Mission and History
Since 1983, the Aplastic Anemia Aplastic Anemia: (ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cel
