DOCSLIB.ORG

Hematology 101 Hematocrits Normal Peripheral Blood Aplastic Anemia

Total Page：16

File Type：pdf, Size：1020Kb

Download full-text PDF Read full-text
Hematology 101 Hematocrits Normal Peripheral Blood Aplastic Anemia Hematocrits Hematology 101 Plasma White cells Red cells Blanche P Alter, MD, MPH, FAAP Clinical Genetics Branch Division of Cancer Epidemiology and Genetics Bethesda, MD Normal, Hemorrhage, IDA, Leukemia, Hemolysis, B12, P Vera Normal Peripheral Blood Aplastic Anemia Peripheral Blood Red blood cells Platelets White blood cells Red Cells Reticulocytes • Contain a red pigment, hemoglobin Red cells • Carry oxygen from the lung to other newly tissues that need it released • Muscles, liver, kidney, heart, brain from bone • Normally live 4 months marrow 1 Fetal Hemoglobin Platelets Kleihauer- • Help blood clot Betke stain • Live 7-10 days • Low numbers can lead to: • Bruising • Petechiae (tiny red dots) • Nosebleeds • Internal bleeding Platelets Types of White Cells (Leukocytes) White Blood Cell Functions Type Life Span • Neutrophils - eat bacteria and fungus Phagocytes (eaters): • Lymphocytes - direct the other cells and Neutrophil hours make antibodies Monocyte days • Monocytes - eat particles coated with Eosinophil hours antibody Basophil hours • Eosinophils - allergies and fight parasites Lymphocytes months-years • Basophils - allergies Neutrophils (Phagocytes) Lymphocytes • Polymorphonuclear (PMN), segmented, • Regulate other white cells granulocytes • Bands, juveniles = early forms • Make antibodies • First line of defense against bacterial • Proteins that act as flags to infection is intact skin and lining of the stic k to bac ter ia an d v iruses mouth, throat and intestines • Tell other cells to eat things • Second line of defense is neutrophils, which eat bacteria and kill them • Low neutrophil number increases susceptibility to bacterial and fungal infections 2 Monocytes CBC Machine • Phagocytes • Draw blood from the • Become tissue macrophages tube into an electronic • Cells in the tissues that eat counter particles tagged with antibodies • Result is called the complete blood count (CBC) Y Bacterium tagged by an antibody Blood Counts Red Cells • Hemoglobin (Hb, Hgb) WBC RBC • 12-15 grams/100 ml (g/dl) [lower Hb for children] Hct MCV • Hematocrit (Hct) MCH • 35 to 45% MCHC RDW Plat Anemia = Low Hb/Hct (H/H) MPV Platelets White Blood Cells (Leukocytes) • Platelet count (Plt) • WBC = white blood cell count • 150,000 to 400,000/μl • 5000–10,000/μl, 5 – 10 thousand/μl • WBC differential Thrombocytopenia = low platelets • % Neutrophils, bands, lymphocytes, monocytes, eosinophils, basophils Leukopenia = low WBC 3 Absolute Neutrophil Count (ANC(ANC) CBC Summary • ANC = WBC x % Neutrophils • Quick and easy assessment of numbers • e.g. WBC = 5000/μl, 30% neutrophils of blood cells • ANC = 5000 x 0.30 = 1500/μl • Relatively inexpensive • Normal: above 1500/μl • No single test tells us more about a blood • OK: above 500 disorder • Low: 200-500 • Measures all three cell types (RBC, WBC, • Very low: below 200 platelets) Neutropenia = low neutrophils • Provides other valuable details Causes of Anemia Definitions • Decreased production • Aplastic Anemia (AA): • Decreased reticulocytes • Pancytopenia due to decreased • Increased destruction production • Hypocellular bone marrow • Increased reticulocytes • Leukemia: • Blood loss • Malignant proliferation of immature cells • Increased reticulocytes • Myelodysplastic syndrome (MDS): • Cytopenia with hypercellular bone marrow Bone Marrow Equipment Bone Marrow Biopsy Normal Aplastic Aspirate Biopsy 4 Aplastic Anemia Bone Marrow Normal Bone Marrow Aspirate Aspirate Erythroid (red cells) Myeloid (white cells) Bone Marrow Iron Leukemia Bone Marrow Hematopoiesis • Formation and development of blood cells • Takes place in the bone marrow • Involves “stem cells” 5 Hematopoiesis BM Cultures • CFU-E: colony-forming unit, erythroid • BFU-E: burst-forming unit, erythroid • CFU-C: colony-forming unit in culture • CFU-GM: colony-forming unit, granulocyte-macrophage CFUCFU--EE and BFUBFU--EE Dysplastic Marrow in MDS • Erythroid: megaloblastic, multinucleation, nuclear fragments, increased immature forms, ring sideroblasts • MlidMyeloid: iditfincreased immature forms, hypo/hyper-granulation • Megakaryocytes: hypo-/hyper-lobulated, small forms, increased nuclear- cytoplasmic ratio Bone Marrow in MDS - myeloid and Bone Marrow in MDS - Erythroid megakaryocytic 6 FAB CLASSIFICATION WHO Classification RCUD: refractory cytopenia with unilineage • No MDS dysplasia (>10% cells in one lineage) • RA = refractory anemia RA, RN, RT: anemia, neutropenia, thrombocytopenia • RARS = ring sideroblasts RARS: ring sideroblasts • RAEB = RA with excess blasts (5-20%) RCMD: multilineage dysplasia (>2 lineages) • CMML = chronic myelomonocytic RAEB-1: 5-9% blasts leukemia, PB monocytes >1000/μL RAEB-2: 10-19% blasts • RAEBT = RA in transformation MDS-U: unclassified: dysplasia <10% cells, + clone MDS del(5q): anemia with isolated del(5q) French-American-British Hematopoiesis Blood and Marrow MDS Study • Aspirate: Morphology • Biopsy: Cellularity • Cytochemistry: PAS, MPO, dual esterase, iron • Flow cytometry: Lymphocytes , granulocytes • Oncogenes: p53, p21 • Cytogenetics: • Classical G banding, • FISH (fluorescence in situ hybridization), • CGH (comparative genomic hybridization) Prepared by www.marrowfailure.cancer.gov Blanche P Alter, MD, MPH National Cancer Institute Greg Kato , MD National Heart, Lung, and Blood Institute M Tarek Elghetany, MD University of Texas Medical Branch 7.
Load more

Recommended publications
  • White Blood Cells and Severe COVID-19: a Mendelian Randomization Study
    Journal of Personalized Medicine Article White Blood Cells and Severe COVID-19: A Mendelian Randomization Study Yitang Sun 1 , Jingqi Zhou 1,2 and Kaixiong Ye 1,3,* 1 Department of Genetics, Franklin College of Arts and Sciences, University of Georgia, Athens, GA 30602, USA; [email protected] (Y.S.); [email protected] (J.Z.) 2 School of Public Health, Shanghai Jiao Tong University School of Medicine, Shanghai 200025, China 3 Institute of Bioinformatics, University of Georgia, Athens, GA 30602, USA * Correspondence: [email protected]; Tel.: +1-706-542-5898; Fax: +1-706-542-3910 Abstract: Increasing evidence shows that white blood cells are associated with the risk of coronavirus disease 2019 (COVID-19), but the direction and causality of this association are not clear. To evaluate the causal associations between various white blood cell traits and the COVID-19 susceptibility and severity, we conducted two-sample bidirectional Mendelian Randomization (MR) analyses with summary statistics from the largest and most recent genome-wide association studies. Our MR results indicated causal protective effects of higher basophil count, basophil percentage of white blood cells, and myeloid white blood cell count on severe COVID-19, with odds ratios (OR) per standard deviation increment of 0.75 (95% CI: 0.60–0.95), 0.70 (95% CI: 0.54–0.92), and 0.85 (95% CI: 0.73–0.98), respectively. Neither COVID-19 severity nor susceptibility was associated with white blood cell traits in our reverse MR results. Genetically predicted high basophil count, basophil percentage of white blood cells, and myeloid white blood cell count are associated with a lower risk of developing severe COVID-19.
    [Show full text]
  • WHITE BLOOD CELLS Formation Function ~ TEST YOURSELF
    Chapter 9 Blood, Lymph, and Immunity 231 WHITE BLOOD CELLS All white blood cells develop in the bone marrow except Any nucleated cell normally found in blood is a white blood for some lymphocytes (they start out in bone marrow but cell. White blood cells are also known as WBCs or leukocytes. develop elsewhere). At the beginning of leukopoiesis all the When white blood cells accumulate in one place, they grossly immature white blood cells look alike even though they're appear white or cream-colored. For example, pus is an accu- already committed to a specific cell line. It's not until the mulation of white blood cells. Mature white blood cells are cells start developing some of their unique characteristics larger than mature red blood cells. that we can tell them apart. There are five types of white blood cells. They are neu- Function trophils, eosinophils, basophils, monocytes and lymphocytes (Table 9-2). The function of all white blood cells is to provide a defense White blood cells can be classified in three different ways: for the body against foreign invaders. Each type of white 1. Type of defense function blood cell has its own unique role in this defense. If all the • Phagocytosis: neutrophils, eosinophils, basophils, mono- white blood cells are functioning properly, an animal has a cytes good chance of remaining healthy. Individual white blood • Antibody production and cellular immunity: lympho- cell functions will be discussed with each cell type (see cytes Table 9-2). 2. Shape of nucleus In providing defense against foreign invaders, the white • Polymorphonuclear (multilobed, segmented nucleus): blood cells do their jobs primarily out in the tissues.
    [Show full text]
  • PATHOLOGY RESIDENT HEMATOLOGY ROTATION (North Florida/South Georgia Veterans Health Care System): Rotation Director: William L
    PATHOLOGY RESIDENT HEMATOLOGY ROTATION (North Florida/South Georgia Veterans Health Care System): Rotation Director: William L. Clapp, M.D., Chief, Hematology Section, Gainesville VAMC; Consultants: Neil S. Harris, M.D., Director, Laboratory Hematology/Coagulation, University of Florida and Shands Hospital and Raul C. Braylan, M.D., Director, Hematopathology, University of Florida and Shands Hospital 1. Description of the Rotation: In this rotation, the resident will gain experience in laboratory hematology, which will include (1) peripheral blood studies to evaluate a variety of hematologic disorders, including anemias, lymphoproliferative and myeloproliferative disorders and leukemias. The emphasis on a multidisciplinary approach to diagnose hematologic disorders (including correlation of the peripheral blood studies with bone marrow and lymph node studies) provides an opportunity for the resident to also gain additional experience in (2) traditional histopathology, (3) immunohistochemistry, (4) electron microscopy, (5) protein electrophoresis, (6) flow cytometry, (7) cytogenetics and (8) molecular genetics which may be performed on the peripheral blood, bone marrow or lymph nodes of patients. The residents will acquire valuable experience by independently performing some bone marrow procedures. In addition, the resident will gain experience in coagulation testing. The residents will become familiar with the instrumentation in the hematology laboratory, including the operating principles and trouble-shooting (medical knowledge). The availability of assembled case study sets and reading materials (medical knowledge) will enhance the resident’s experience. Participation in CAP surveys, continuing education and hematology conferences is a component of the rotation (practice-based learning). Management issues and computer applications will be discussed (practice-based learning). As appropriate to the individual case or consultation under review, the ethical, socioeconomic, medicolegal and cost-containment issues will be reviewed and discussed.
    [Show full text]
  • Section 8: Hematology CHAPTER 47: ANEMIA
    Section 8: Hematology CHAPTER 47: ANEMIA Q.1. A 56-year-old man presents with symptoms of severe dyspnea on exertion and fatigue. His laboratory values are as follows: Hemoglobin 6.0 g/dL (normal: 12–15 g/dL) Hematocrit 18% (normal: 36%–46%) RBC count 2 million/L (normal: 4–5.2 million/L) Reticulocyte count 3% (normal: 0.5%–1.5%) Which of the following caused this man’s anemia? A. Decreased red cell production B. Increased red cell destruction C. Acute blood loss (hemorrhage) D. There is insufficient information to make a determination Answer: A. This man presents with anemia and an elevated reticulocyte count which seems to suggest a hemolytic process. His reticulocyte count, however, has not been corrected for the degree of anemia he displays. This can be done by calculating his corrected reticulocyte count ([3% × (18%/45%)] = 1.2%), which is less than 2 and thus suggestive of a hypoproliferative process (decreased red cell production). Q.2. A 25-year-old man with pancytopenia undergoes bone marrow aspiration and biopsy, which reveals profound hypocellularity and virtual absence of hematopoietic cells. Cytogenetic analysis of the bone marrow does not reveal any abnormalities. Despite red blood cell and platelet transfusions, his pancytopenia worsens. Histocompatibility testing of his only sister fails to reveal a match. What would be the most appropriate course of therapy? A. Antithymocyte globulin, cyclosporine, and prednisone B. Prednisone alone C. Supportive therapy with chronic blood and platelet transfusions only D. Methotrexate and prednisone E. Bone marrow transplant Answer: A. Although supportive care with transfusions is necessary for treating this patient with aplastic anemia, most cases are not self-limited.
    [Show full text]
  • Acquired Hemophilia A: Pathogenesis and Treatment
    Bleeding disorders Acquired hemophilia A: pathogenesis and treatment P.W. Collins ABSTRACT Arthur Bloom Haemophilia Centre, Acquired hemophilia A is an autoimmune disease caused by an inhibitory antibody to factor VIII. The School of Medicine, severity of bleeding varies but patients remain at risk of life-threatening bleeding until the inhibitor Cardiff University, Heath Park, has been eradicated. The cornerstones of management are rapid and accurate diagnosis, control of Cardiff, UK bleeding, investigation for an underlying cause, and eradication of the inhibitor by immunosuppres - sion. Patients should be managed jointly with a specialist center even if they present without signifi - cant bleeding. Despite an extensive literature, few controlled data are available and management Hematology Education: guidelines are based on expert opinion. Recombinant factor VIIa and activated prothrombin complex the education program for the concentrate are equally efficacious for treating bleeds and both are superior to factor VIII or desmo - annual congress of the European pressin. Immunosuppression should be started as soon as the diagnosis is made. Commonly used reg - Hematology Association imens are steroids alone or combined with cytotoxic agents. Rituximab is being used more commonly but current evidence does not suggest that it improves outcomes or reduces side effects. 2012;6:65-72 Introduction Pathogenesis Acquired hemophilia A (AHA) is a bleed - AHA is associated with autoimmune dis - ing disorder caused by polyclonal IgG1 and eases, such as rheumatoid arthritis, polymyal - IgG4 autoantibodies to the factor VIII ( FVIII ) gia rheumatic, and systemic lupus erythe - A2 and C2 domain. Morbidity and mortality matosis; malignancy; pregnancy and dermato - are high secondary to age, underlying dis - logical disorders, such as pemphigoid.
    [Show full text]
  • A Distant Gene Deletion Affects Beta-Globin Gene Function in an Atypical Gamma Delta Beta-Thalassemia
    A distant gene deletion affects beta-globin gene function in an atypical gamma delta beta-thalassemia. P Curtin, … , A D Stephens, H Lehmann J Clin Invest. 1985;76(4):1554-1558. https://doi.org/10.1172/JCI112136. Research Article We describe an English family with an atypical gamma delta beta-thalassemia syndrome. Heterozygosity results in a beta-thalassemia phenotype with normal hemoglobin A2. However, unlike previously described cases, no history of neonatal hemolytic anemia requiring blood transfusion was obtained. Gene mapping showed a deletion that extended from the third exon of the G gamma-globin gene upstream for approximately 100 kilobases (kb). The A gamma-globin, psi beta-, delta-, and beta-globin genes in cis remained intact. The malfunction of the beta-globin gene on a chromosome in which the deletion is located 25 kb away suggests that chromatin structure and conformation are important for globin gene expression. Find the latest version: https://jci.me/112136/pdf A Distant Gene Deletion Affects ,8-Globin Gene Function in an Atypical '6y5-Thalassemia Peter Curtin, Mario Pirastu, and Yuet Wai Kan Howard Hughes Medical Institute and Department ofMedicine, University of California, San Francisco, California 94143 John Anderson Gobert-Jones Department ofPathology, West Suffolk County Hospital, Bury St. Edmunds IP33-2QZ, Suffolk, England Adrian David Stephens Department ofHaematology, St. Bartholomew's Hospital, London ECIA-7BE, England Herman Lehmann Department ofBiochemistry, University ofCambridge, Cambridge CB2-lQW, England Abstract tologic picture of f3-thalassemia minor in adult life. Globin syn- thetic studies reveal a ,3 to a ratio of -0.5, but unlike the usual We describe an English family with an atypical 'yS6-thalassemia fl-thalassemia heterozygote, the levels of HbA2 (and HbF) are syndrome.
    [Show full text]
  • Anemia in Heart Failure - from Guidelines to Controversies and Challenges
    52 Education Anemia in heart failure - from guidelines to controversies and challenges Oana Sîrbu1,*, Mariana Floria1,*, Petru Dascalita*, Alexandra Stoica1,*, Paula Adascalitei, Victorita Sorodoc1,*, Laurentiu Sorodoc1,* *Grigore T. Popa University of Medicine and Pharmacy; Iasi-Romania 1Sf. Spiridon Emergency Hospital; Iasi-Romania ABSTRACT Anemia associated with heart failure is a frequent condition, which may lead to heart function deterioration by the activation of neuro-hormonal mechanisms. Therefore, a vicious circle is present in the relationship of heart failure and anemia. The consequence is reflected upon the pa- tients’ survival, quality of life, and hospital readmissions. Anemia and iron deficiency should be correctly diagnosed and treated in patients with heart failure. The etiology is multifactorial but certainly not fully understood. There is data suggesting that the following factors can cause ane- mia alone or in combination: iron deficiency, inflammation, erythropoietin levels, prescribed medication, hemodilution, and medullar dysfunc- tion. There is data suggesting the association among iron deficiency, inflammation, erythropoietin levels, prescribed medication, hemodilution, and medullar dysfunction. The main pathophysiologic mechanisms, with the strongest evidence-based medicine data, are iron deficiency and inflammation. In clinical practice, the etiology of anemia needs thorough evaluation for determining the best possible therapeutic course. In this context, we must correctly treat the patients’ diseases; according with the current guidelines we have now only one intravenous iron drug. This paper is focused on data about anemia in heart failure, from prevalence to optimal treatment, controversies, and challenges. (Anatol J Cardiol 2018; 20: 52-9) Keywords: anemia, heart failure, intravenous iron, ferric carboxymaltose, quality of life Introduction g/dL in men) (2).
    [Show full text]
  • Apheresis Donation This Quick Reference Guide Will Help You Identify the Best Donation for Your Unique Blood Type
    Apheresis Donation This quick reference guide will help you identify the best donation for your unique blood type. Donors now have the opportunity to make an apheresis (ay-fur-ee-sis) donation and donate just platelets, red cells, or plasma at blood drives. These individual components are vital for local patients in need. Platelets Control Bleeding Red Cells Deliver Oxygen Plasma transports blood cells & controls bleeding Donation Type Blood Types Requirements Donation Time A+, B+, O+ Over 75% of population has one of these blood types. Platelet Donation: Be healthy, weigh at least 114 lbs 2 hours cancer & surgery patients no aspirin for 48 hours Platelets only last five days after donation so the need is constant. O-, O+, A-, B- Special height, weight, Double Red: O-Negative is the 1 hour and hematocrit requirements. surgery, trauma patients, universal red cell donor. +25 min Please call us or see a staff member accident, & burn victims Only 17% of population has one of these negative blood types Plasma: AB+, AB- Trauma patients, burn Universal Plasma Donors 1 hour Be healthy, weigh at least 114 lbs victims, & patients with +30 min serious illness or injuries Only 4% of population How Apheresis works: Blood is drawn from the donor’s arm and the components are separated. Only the components being donated are collected while the remaining components are safely returned to the donor How to Schedule an Appointment: Please call 800-398-7888 or visit schedule.bloodworksnw.org. Walk-ins are also welcome at some blood drives, so be sure to ask our staff when you stop in.
    [Show full text]
  • Stem Cell Or Bone Marrow Transplant
    cancer.org | 1.800.227.2345 Stem Cell or Bone Marrow Transplant A stem cell transplant, also called a bone marrow transplant, can be used to treat certain types of cancer. This procedure might be called peripheral stem cell transplant or cord blood transplant, depending on where the stem cells come from. Here we’ll explain stem cells and stem cell transplant, cover some of the issues that come with transplants, and describe what it's like to donate stem cells. ● How Stem Cell and Bone Marrow Transplants Are Used to Treat Cancer ● Types of Stem Cell and Bone Marrow Transplants ● Donating Stem Cells and Bone Marrow ● Getting a Stem Cell or Bone Marrow Transplant ● Stem Cell or Bone Marrow Transplant Side Effects How Stem Cell and Bone Marrow Transplants Are Used to Treat Cancer What are stem cells? All of the blood cells in your body - white blood cells, red blood cells, and platelets - start out as young (immature) cells called hematopoietic stem cells. Hematopoietic means blood-forming. These are very young cells that are not fully developed. Even though they start out the same, these stem cells can mature into any type of blood cell, depending on what the body needs when each stem cell is developing. 1 ____________________________________________________________________________________American Cancer Society cancer.org | 1.800.227.2345 Stem cells mostly live in the bone marrow (the spongy center of certain bones). This is where they divide to make new blood cells. Once blood cells mature, they leave the bone marrow and enter the bloodstream. A small number of the immature stem cells also get into the bloodstream.
    [Show full text]
  • Automatic White Blood Cell Measuring Aid for Medical Diagnosis
    Automatic White Blood Cell Measuring Aid for Medical Diagnosis Pramit Ghosh, Debotosh Bhattacharjee, Mita Nasipuri and Dipak Kumar Basu Abstract— Blood related invasive pathological investigations increases. It turns into a vicious cycle. On the other hand, a play a major role in diagnosis of diseases. But in India and low count of white blood cells indicates viral infections, low other third world countries there are no enough pathological immunity and bone marrow failure [3]. A severely low white infrastructures for medical diagnosis. Moreover, most of the blood count that is the count of less than 2500 cells per remote places of those countries have neither pathologists nor micro-litre is a cause for a critical alert and possesses a high physicians. Telemedicine partially solves the lack of physicians. But the pathological investigation infrastructure can’t be risk of sepsis [4]. integrated with the telemedicine technology. The objective of In conventional procedure, glass slides containing blood this work is to automate the blood related pathological samples are dipped into Lisman solution before placing it investigation process. Detection of different white blood cells into microscope [5]. Microscope enlarges the pictures of has been automated in this work. This system can be deployed blood samples for manual detection of different white blood in the remote area as a supporting aid for telemedicine technology and only high school education is sufficient to cells; but this manual process totally depends on pathologist. operate it. The proposed system achieved 97.33% accuracy for Some auto cell counting units exist like Cellometer [15], the samples collected to test this system.
    [Show full text]
  • Patient Information Leaflet – Plasma Exchange Procedure
    Therapeutic Apheresis Services Patient Information Leaflet – Plasma Exchange Procedure Introduction Antibodies, which normally help to protect you from infection, can begin to attack your own This leaflet has been written to give patients healthy cells, or an over production of proteins can information about plasma exchange (sometimes cause your blood to become thicker and slow down called plasmapheresis). If you would like any more the blood flow throughout your body. A plasma information or have any questions, please ask the exchange can help improve your symptoms, doctors and nurses involved in your treatment at although this may not happen immediately. the NHS Blood and Transplant (NHSBT) Therapeutic Apheresis Services Unit. Although plasma exchange may help with symptoms, it will not normally cure your condition When you have considered the information given as it does not switch off the production of the in this leaflet, and after we have discussed the harmful antibodies or proteins. It is likely that procedure and its possible risks with you, we will this procedure will form only one part of your ask you to sign a consent form to indicate that you treatment. are happy for the procedure to go ahead. Before any further procedures we will again check that you are happy to proceed. How do we perform Plasma Exchange? What is a plasma exchange? Plasma exchange is performed using a machine Blood is made up of red cells, white cells and called a Blood Cell Separator which can separate platelets which are carried around in fluid called blood into its various parts. The machine separates plasma.
    [Show full text]
  • Haemophilia a Is the Most Common Form – Affecting
    Haemophilia is an inherited, serious It can dramatically reduce bleeding disorder where a person’s the quality of life of people blood does not clot properly, leading affected, as well as their family, to uncontrolled bleeding which can friends and caregivers1. occur spontaneously or after minor trauma. Haemophilia A is the most common form – affecting 50-60% of whom have severe haemophilia4. blood of a person In a healthy person, proteins called clotting factors work together to form a blood clot and help stop bleeding. People with haemophilia A either lack or do not have enough of a clotting factor called which leads to their blood not being able to clot properly. Bruising Repeated bleeding into muscles and joints, which can lead to long term disability or joint disease5 Spontaneous bleeding, which can be life threatening if it occurs in vital organs, such as the brain Prolonged and uncontrolled bleeding following injury or surgery6,7 Life for people with haemophilia and their caregivers is often centred on treatment infusions, taking up a large amount of time and having a significant impact on their lives8. People with haemophilia A report difficulty balancing treatment with daily life, so compliance can be a challenge9,10 leaving them vulnerable to potentially dangerous bleeds. The mainstay of current treatment for haemophilia A is factor VIII replacement therapy, which is taken on-demand (as needed to treat bleeds), or on an ongoing basis (to prevent bleeds). It is short-acting and so needs to be administered frequently (at least twice a week)2 by the patient or a caregiver and for some, especially children, finding a vein for medicine infusion can be difficult11.
    [Show full text]