scholarly journals THE EVOLVING PHARMACOTHERAPEUTIC LANDSCAPE FOR THE TREATMENT OF SICKLE CELL DISEASE

2020 ◽  
Vol 12 (1) ◽  
pp. e2020010 ◽  
Author(s):  
Samir Ballas
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
The United States ◽  
Mechanisms Of Action ◽  
Chemotherapeutic Agents ◽  
Cell Disease ◽  
The Third ◽  
United States Food ◽  
States Food ◽  
Second Category

Sickle cell disease (SCD) is an extremely heterogeneous disease that has been associated with global morbidity and early mortality. More effective and inexpensive therapiesare needed. During the last five years the landscape of the pharmacotherapy of SCD has changed dramatically. Currently, there are at least 50 drugs that have been used or under consideration to use for the treatment of SCD. These fall into 3 categories: the first category includes the three drugs (Hydroxyurea, L-Glutamine and Crizanlizumab tmca) that have been approved by the United States Food and Drug Administration (FDA) based on successful clinical trials. The second category includes 22 drugs that failed, discontinued or terminated for now and the third category includes 25 drugs that are actively being considered for the treatment of SCD. New therapies targeting multiple pathways in its complex pathophysiology have been achieved or are under continued investigation. The emerging trend seems to be the use of multimodal drugs (i.e. drugs that have different mechanisms of action) to treat SCD similar to the use of multiple chemotherapeutic agents to treat cancer.

Red cell storage age policy for patients with sickle cell disease: A survey of transfusion service directors in the United States

2016 ◽  
Vol 54 (1) ◽  
pp. 158-162 ◽  
Author(s):  
Matthew S. Karafin ◽  
Arun K. Singavi ◽  
Mehraboon S. Irani ◽  
Kathleen E. Puca ◽  
Lisa Baumann Kreuziger ◽  
...  
Keyword(s):  
United States ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
The United States ◽  
Red Cell ◽  
Cell Disease ◽  
Cell Storage

scholarly journals Antimicrobial Resistance and Sickle Cell Protein Analysis in Invasive Non-Typhoidal Salmonella Isolated From Outpatient and Hospitalized Children Below 16 Years in Informal Settlements in Nairobi Kenya

2020 ◽  
Author(s):  
Susan Kavai ◽  
Cecilia Mbae ◽  
Celestine Wanjiku ◽  
Ronald Ngetich ◽  
Zillah Wakio ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Salmonella Typhimurium ◽  
Sickle Cell ◽  
Informal Settlements ◽  
The United States ◽  
Sub Saharan Africa ◽  
Cell Protein ◽  
Cell Disease ◽  
Resistance Phenotype ◽  
Stool Samples

Abstract Background: Invasive Non-typhoidal Salmonella (iNTS) disease continues to be a major public health problem, especially in sub Saharan Africa where incidence rates are 227 cases [range 152-341] per 100,000 population. Populations at risk of iNTS include adults with HIV infection, malnourished children, those with recent malaria or sickle-cell anaemia (SCA). Individuals with SCA are at an increased risk of invasive bacterial infections with the proportion of deaths from infection reported to be as high as 38% in the United States and 29% in Jamaica. In Kenya, iNTS disease is particularly a major challenge in poor informal settlements with infants and young children less than 5 years of age being the most affected; mortality rates can be 20-25% unless prompt treatment is administered. Methods Our study was conducted in 3 outpatient sites and 1 inpatient site, the outpatient sites were all located within Mukuru informal settlement, a densely populated slum, 15km East of Nairobi City. Blood and stool samples from children with fever alone and with fever and diarrhea were collected for processing for presence of iNTS using basic microbiology procedures. Dry blood spots were also taken and processed for sickle cell protein markers using High performance liquid chromatography (HPLC). Results A total of 22,246 blood and stool samples were collected from children < 16 years of age with fever/with or without diarrhea, for a period of 6 years and subjected to microbiological culture and detection of bacterial pathogens. Out of these 741 (3.3%) tested positive for Salmonella species. A total of 338/741(41%) NTS were isolated across all the sites; these consisted of 158/741(21%) Salmonella Enteritidis and 180/741 (24%) Salmonella Typhimurium. The most common resistance phenotype was ampicillin, cotrimoxazole and chloramphenicol (35.03%). We had 12/338 (3.6%) isolates (11 of them being Salmonella Typhimurium) that were ESBL producers conferring resistance to 3rd generation cephalosporins (Amp C β-lactamases) while only 0.3% were resistant to ciprofloxacin. A total of 118 (35.03%) isolates were MDR. Out of 2684 dry blood samples subjected to HPLC for investigation of sickle cell disease traits, 1820/2684 (67%) had normal hemoglobin (Hb AA/ Hb AF); (162/2684 (6%) tested positive for Sickle Cell Traits (Hb AS/Hb AFS); while 4/2684 (0.2%) tested positive for Sickle cell disease (Hb FS). Conclusion The high MDR resistance phenotype in iNTS isolates and emerging resistance to third generation cephalosporins is of great concern in management of iNTS in our settings. Sickle cell disease was not a major factor among children with iNTS disease and no significant association with iNTS was observed.

Newborn Screening for Sickle Cell Disease and Other Hemoglobinopathies

PEDIATRICS ◽  
1989 ◽  
Vol 83 (5) ◽  
pp. 813-814
Author(s):  
DORIS WETHERS ◽  
HOWARD PEARSON ◽  
MARILYN GASTON
Keyword(s):  
Sickle Cell Disease ◽  
Early Diagnosis ◽  
Sickle Cell ◽  
Sickle Cell Anemia ◽  
Case Fatality ◽  
The United States ◽  
Early Mortality ◽  
Cell Disease ◽  
Newborn Period ◽  
The Past

Hemoglobinopathies represent one of the major health problems in the United States and constitute the most common genetic disorders in some populations. Sickle cell disease (SS, SC, S-β-thalassemia) alone affects about one in 400 American black newborns, as well as persons of African, Mediterranean, Asian, Caribbean, Middle Eastern, and South and Central American origins. For the past 20 years, the medical profession has known that children with sickle cell anemia have an increased susceptibility to severe bacterial infection, particularly due to Streptococcus pneumoniae. The risk of major infection and death posed by this organism is greatest in the first 3 years of life and can occur as early as 3 months of age. In fact, this infection may be the first clinical manifestation of disease. The infection can be fulminant, progressing from the onset of fever to death in a matter of hours, and the case fatality rate is reported as high as 30%. In addition, acute splenic sequestration, another acute catastrophic event, contributes to early mortality in children with sickle cell anemia and may occur as early as 5 months of age. It has been proposed that early diagnosis to identify infants with major sickle hemoglobinopathies, who have a high risk of early mortality and morbidity, is essential to institute appropriate ongoing care and effective measures of prophylaxis and intervention. Early diagnosis of hemoglobinopathies should be in the newborn period. Even though the technology to screen infants in the newborn period has been available for the past 15 to 20 years, screening has not received widespread acceptance.

scholarly journals Psychological Predictors of Pain in Children and Adolescents With Sickle Cell Disease: A Scoping Review

2018 ◽  
Vol 36 (2) ◽  
pp. 150-159
Author(s):  
Clare Donohoe ◽  
Ellen Lavoie Smith
Keyword(s):  
Sickle Cell Disease ◽  
Children And Adolescents ◽  
Sickle Cell ◽  
Middle Eastern ◽  
The United States ◽  
Health Science ◽  
Psychological Characteristics ◽  
Cell Disease ◽  
Pain Frequency ◽  
Psychological Risk

Objective: Sickle cell disease (SCD) is a common red blood cell disorder that disrupts the lives of many African Americans and those of Middle Eastern heritage within the United States due to frequent pain. There is limited research quantifying biopsychosocial factors, specifically psychological characteristics, that influence pain in children and adolescents with SCD. The aim of this literature review was to identify psychological characteristics that are predictive or associated with pain in children and adolescents with SCD. Method: This review was conducted using PRISMA guidelines. Four databases, PubMed, CINAHL, PsycINFO, and Scopus, were searched using specific terms to address the aim of the review (SCD, pain, pediatrics and adolescents, and psychological characteristics). Results: The review identified a lack of consensus regarding the definitions and measurement of pain frequency and intensity. A variety of psychological characteristics were associated with pain including coping strategies, anxiety, depressive symptoms, catastrophizing, and stress. Overall, the study designs restricted the ability to fully identify psychological characteristics that predict pain. Conclusions: Health science researchers must strive for a deeper understanding about the presentation of SCD pain and psychological risk factors associated with increased pain to provide targeted screening and treatment.

scholarly journals The Economic Burden of Sickle Cell Disease in the United States

2018 ◽  
Vol 21 ◽  
pp. S108 ◽  
Author(s):  
J Huo ◽  
H Xiao ◽  
M Garg ◽  
C Shah ◽  
DJ Wilkie ◽  
...  
Keyword(s):  
United States ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Economic Burden ◽  
The United States ◽  
Cell Disease

Childhood Hearing Loss in Patients With Sickle Cell Disease in the United States

2019 ◽  
Vol 41 (2) ◽  
pp. 124-128 ◽  
Author(s):  
Heather K. Schopper ◽  
Christopher F. D’Esposito ◽  
John S. Muus ◽  
Julie Kanter ◽  
Ted A. Meyer
Keyword(s):  
United States ◽  
Hearing Loss ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
The United States ◽  
Cell Disease ◽  
Childhood Hearing Loss

scholarly journals Liver Transplantation in Patients with Sickle Cell Disease in the United States

2020 ◽  
Vol 255 ◽  
pp. 23-32 ◽  
Author(s):  
Rachel Hogen ◽  
Michelle Kim ◽  
Yelim Lee ◽  
Mary Lo ◽  
Navpreet Kaur ◽  
...  
Keyword(s):  
United States ◽  
Liver Transplantation ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
The United States ◽  
Cell Disease

scholarly journals Survey of Staff Positions in Academic Hematology in 1971

Blood ◽  
1972 ◽  
Vol 40 (4) ◽  
pp. 574-584
Author(s):  
Helen M. Ranney ◽  
Louis Kinyon ◽  
Harry A. Sultz
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Medical Schools ◽  
The United States ◽  
Cell Disease ◽  
Military Hospitals ◽  
Research Areas ◽  
Grant Support ◽  
Staffing Needs ◽  
Salary Support

Abstract A minimal estimate of the number of positions in academic hematology in the United States was 1159 of which 10% were unfilled in the fall of 1971. Exclusive of positions in federal institutions (Veterans Administration and military hospitals), 72% of the salary support for filled positions was derived from "hard money", and similar nogrant sources were available for the support of 74% of the salaries for unfilled positions. Private institutions had slightly less "hard money" salary support than did public institutions. A probable maximum of one-third of the positions now supported by grants would be supported by "hard money" were grant support to terminate. The numbers of positions in academic hematology tripled in the 11 yr from 1960 to 1971. Because of the staffing needs for new medical schools, and the expansion of classes in existing medical schools as well as the need for physician-scientists in the developing programs in oncology and sickle cell disease, the growth of hematology will probably continue, albeit at a slower rate. Academic hematologists’ research interests were divided among several areas. The primary interests of the largest numbers of researchers were oncology, red blood cells, hemostasis and coagulation, and immunohematology. Attention was directed to the rather small numbers of academic hematologists with research interests in blood banking, clinical laboratories, and nutrition. It is not clear whether efficient development of larger programs in oncology and sickle cell disease will require more academic hematologists with experience in these areas than are now available. These data do not provide information about the overall national needs for hematologists in service laboratories in nonacademic institutions or in the delivery of health care. Nor do they provide the basis for any recommendations about the size or composition of divisions of hematology in medical schools. They do provide the first overall appraisal of the size, funding, and research areas in academic hematology in this country.

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