This issue of Hematology/Oncology Clinics, guest edited by Dr. Elliott Vichinsky, is devoted to Sickle Cell Disease, and focuses on pathophysiology of hemoglobinopathies, therapeutic targets, and new approaches to correcting ineffective erythropoiesis and iron dysregulation. Articles in this issue include Polymerization and red cell membrane changes; Overview on reperfusion injury in the pathophysiology of SCD; Regulation of ineffective erythropoiesis in iron metabolism; Altering oxygen affinity; Cellular adhesion and the endothelium; Arginine therapy; Role of the hemostatic system on SCD pathophysiology and potential therapeutics; Adenosine signaling and novel therapies; New approaches to correcting ineffective erythropoiesis and iron dysregulation; New approaches to correcting ineffective erythropoiesis and iron dysregulation; Fetal hemoglobin induction; Gene therapy for hemoglobinopathies; and Oxidative injury and the role of antioxidant therapy.
Sickle Cell Pain is a panoramic, in-depth exploration of every scientific, human, and social dimension of this cruel disease. This comprehensive, definitive work is unique in that it is the only book devoted to sickle cell pain, as opposed to general aspects of the disease. The 752-page book links sickle cell pain to basic, clinical, and translational research, addressing various aspects of sickle pain from molecular biology to the psychosocial aspects of the disease. Supplemented with patient narratives, case studies, and visual art, Sickle Cell Pain’s scientific rigor extends through its discussion of analgesic pharmacology, including abuse-deterrent formulations. The book also addresses in great detail inequities in access to care, stereotyping and stigmatization of patients, the implications of rapidly evolving models of care, and recent legislation and litigation and their consequences.
This issue of Hematology/Oncology Clinics, guest edited by Drs. Matthew Heeney and Alan Cohen, is devoted to Iron Disorders. Articles in this issue include: Hereditary Hemochromatosis (HFE and Non-HFE); Iron Refractory Iron Deficiency Anemia (IRIDA); Sideroblastic Anemia; Anemia of Chronic Disease/Inflammation; Pathophysiology of Transfusional Iron Overload; Transfusional Iron Overload and Iron Chelation Therapy; Iron Overload and its Management in Non-Transfusion-Dependent Thalassemia; Treatment of Iron Deficiency Anemia; and Iron Overload Assessment.
This issue of Hematology/Oncology Clinics, guest edited by Dr. F. Stephen Hodi, is devoted to Melanoma. Articles in this issue include: The current state of Melanoma; Understanding the Biology of Melanoma Development and Therapeutic Implications; Surgical Management of Melanoma; Targeted Therapies for Cutaneous Melanoma; Treatments for Non-cutaneous Melanoma; Resistant Mechanisms and Therapeutic Implications; The Role of the Immune System in Melanoma Development and Treatment; Vaccines and Melanoma; IL-2, Interferon, and Cytokines; Immune Checkpoint Blockade; Adjuvant Treatments, Chance for Cure in Melanoma; and Combinatorial Approach to Treatment of Melanoma.
This issue of Hematology/Oncology Clinics of North America, devoted to Multiple Myeloma, is edited by Dr. Kenneth C. Anderson. Articles in this issue include: Monoclonal Gammopathy of Undetermined Significance and Smoldering Multiple Myeloma; Diagnosis and Risk Stratification in Myeloma; Treatment of Newly Diagnosed Transplant Eligible Patients; Treatment of Newly Diagnosed Transplant Ineligible Patients; Treatment of Relapsed/Refractory Myeloma; Maintenance Therapy; Novel Targeted Therapies; Novel Immune-based Therapies; Allotransplantation in Myeloma; and Waldenstrom’s Macroglobulinemia.
This issue of Hematology/Oncology Clinics is focused on Hodgkin's Lymphoma and covers such topics as First HRS-cell line L428 and the detection of the CD-30 Antigen , “Normal“ CD30-B-lymphocytes, CD-30-Antigen, Combination-Chemo-Radiotherapy, Early intensification (escBEACOPP), Customized/Targeted Therapy, and more.
Newborn Screening for Sickle Cell Disease and other Haemoglobinopathies is a Special Issue of the International Journal of Neonatal Screening. Sickle cell disease is one of the most common inherited blood disorders, with a huge impact on health care systems due to high morbidity and high mortality associated with the undiagnosed disease. Newborn screening helps to make the diagnosis early and to prevent fatal complications and diagnostic odysseys. This book gives an overview of diagnostic standards in newborn screening for sickle cell disease and examples of existing newborn screening programs.
#1 Best Seller on Sickle Cell Disease (SCD). Sickle cell disease is a group of blood disorders passed down from parents to children. Sickle cell anemia shortens life expectancy by 30 years via bacterial infections, painful swellings, fever, arthritis, leg ulcers, eye, lung & heart damage. Over 100,000 people, mostly African-Americans, in the United States have sickle cell disease. Over 2 million people have sickle cell trait in America. It is estimated that more than 300,000 children are born each year with SCD around the world. This edition of The Management of Sickle Cell Disease (SCD) is organized into four parts: 1. Diagnosis and Counseling 2. Health Maintenance 3. Treatment of Acute and Chronic Complications 4. Special Topics. The original intent was to incorporate evidence-based medicine into each chapter, but there was variation among evidence-level scales, and some authors felt recommendations could be made, based on accepted practice, without formal trials in this rare disorder. The best evidence still is represented by randomized, controlled trials (RCTs), but variations exist in their design, conduct, endpoints, and analyses. It should be emphasized that selected people enter a trial, and results should apply in practice specifically to populations with the same characteristics as those in the trial. Randomization is used to reduce imbalances between groups, but unexpected factors sometimes may confound analysis or interpretation. In addition, a trial may last only a short period of time, but long-term clinical implications may exist. Another issue is treatment variation, for example, a new pneumococcal vaccine developed after the trial, which has not been tested formally in a sickle cell population. Earlier trial results may be accepted, based on the assumption that the change is small. In some cases, RCTs cannot be done satisfactorily (e.g., for ethical reasons, an insufficient number of patients, or a lack of objective measures for sickle cell "crises"). Thus the bulk of clinical experience in SCD still remains in the moderately strong and weaker categories of evidence. Not everyone has an efficacious outcome in a clinical trial, and the frequency of adverse events, such as with long-term transfusion programs or hematopoietic transplants, might not be considered. Thus, an assessment of benefit-to-risk ratio should enter into translation of evidence levels into practice recommendations. A final issue is that there may be two alternative approaches that are competitive (e.g., transfusions and hydroxyurea). In this case the pros and cons of each course of treatment should be discussed with the patient. This book is B&W copy of the government agency publication.
The most comprehensive, current sickle cell disease resource—for both clinicians and researchers A Doody's Core Title for 2023! The first and only resource of its kind, Sickle Cell Disease examines this blood disorder through both clinical and research lenses. More than 80 dedicated experts in the field present their combined clinical knowledge of basic mechanisms, screening, diagnosis, management, and treatment of myriad complex complications of a single base point mutation in the human genome. Case studies with “How I Treat” authoritative insights provide overviews of common and rare complications, and Key Facts offer at-a-glance high-yield information. Filled with clinical photos, illustrations, numerous original diagrams, and with free updates available online, this unmatched resource covers: Mechanisms of sickle cell disease Historic and current research approaches The latest work in gene therapy and editing Guidelines for patient care, diagnosis, unique cases, and therapies Rare and common complications, including domestic and internationally relevant topics Psychosocial and supportive care The newest standards of therapy and future treatment options in children and adults Cardiopulmonary complications
Recognizing the potential design complexities and ethical issues associated with clinical trials for gene therapies, the Forum on Regenerative Medicine of the National Academies of Sciences, Engineering, and Medicine held a 1-day workshop in Washington, DC, on November 13, 2019. Speakers at the workshop discussed patient recruitment and selection for gene-based clinical trials, explored how the safety of new therapies is assessed, reviewed the challenges involving dose escalation, and spoke about ethical issues such as informed consent and the role of clinicians in recommending trials as options to their patients. The workshop also included discussions of topics related to gene therapies in the context of other available and potentially curative treatments, such as bone marrow transplantation for hemoglobinopathies. This publication summarizes the presentation and discussion of the workshop.
