A justice‐based argument for including sickle cell disease in CRISPR/Cas9 clinical research

Bioethics ◽  
2019 ◽  
Vol 33 (6) ◽  
pp. 661-668
Author(s):  
Marilyn S. Baffoe-Bonnie
Keyword(s):  
Sickle Cell Disease ◽  
Clinical Research ◽  
Sickle Cell ◽  
Cell Disease

Survey of Pain Management Therapy Provided for Children With Sickle Cell Disease

1992 ◽  
Vol 31 (4) ◽  
pp. 211-214 ◽  
Author(s):  
Charles H. Pegelow
Keyword(s):  
Pain Management ◽  
Sickle Cell Disease ◽  
Clinical Research ◽  
Sickle Cell ◽  
Special Interest ◽  
Pain Control ◽  
Control Methods ◽  
Cell Disease ◽  
Narcotic Analgesics ◽  
Principal Investigators

A questionnaire was sent to principal investigators of NIH-sponsored clinical research in sickle cell disease. Twenty of 21 respondents indicated they used parenteral narcotic analgesics for pain episodes sufficiently severe to warrant hospitalization. Eleven used meperidine; seven, morphine; and one each, nalbuphine, hydromorphone, and acetaminophen with codeine. They gave the agents at frequent, regular intervals or by continuous infusion. A total of 41 of more than 3,500 patients required chronic transfusion for pain control. Complications included meperidine-associated convulsions reported by nine respondents and addiction by six. This information indicates that vigorous pain-control methods are used at institutions having a special interest in providing medical care for children with sickle cell disease.

A 2-Dose Schedule of 13-Valent Pneumococcal Conjugate Vaccine (PCV13) Given to Children with Sickle Cell Disease Previously Immunized with 23-Valent Pneumococcal Polysaccharide Vaccine (PPSV23): Results of a Phase 3 Study

Blood ◽  
2012 ◽  
Vol 120 (21) ◽  
pp. 3212-3212
Author(s):  
Mariane De Montalembert ◽  
Miguel R. Abboud ◽  
Anne Fiquet ◽  
Adlette Inati ◽  
Jeffrey D. Lebensburger ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Research And Development ◽  
Clinical Research ◽  
Conjugate Vaccine ◽  
Sickle Cell ◽  
Cell Disease ◽  
Phase 3 ◽  
Local Pain ◽  
Increased Risk ◽  
Global Research

Abstract Abstract 3212 Introduction: Children with sickle cell disease (SCD) have a significantly increased risk of invasive pneumococcal disease (IPD) and mortality, primarily attributable to functional asplenia. Penicillin prophylaxis and PPSV23 have been shown to dramatically reduce those risks, but strain resistance to penicillin has increased. Furthermore, PPSV23 is less effective in children <2 years and the protection declines within 3 years after administration. The introduction in 2000 of the 7-valent conjugate vaccine (PCV7) induced a further reduction of IPD in children with SCD, but was accompanied by the emergence of non-PCV7 serotypes-related IPD, especially 19A. A new PCV13 incorporating the new serotypes 1, 3, 5, 6A, 7F and 19A to those in PCV7 was recently licensed. This study was designed to determine whether PCV13 has the potential to offer immunologic benefit to children with SCD. Methods: We conducted a phase 3, open-label, single-arm study to evaluate the safety, tolerability, and immunogenicity of 2-doses of PCV13 given 6 months apart to children 6 to less than 18 years of age with SCD previously immunized with PPSV23 more than 6 months prior to study entry. Blood samples were collected prior to and 1 month after each dose. Serotype-specific immunoglobulin (IgG) geometric mean concentrations (GMCs) and fold rises (GMFRs) were determined. Local pain, redness and swelling, and fever, vomiting, diarrhea, headache, fatigue, muscle pain and joint pain were recorded in an ediary for 7 days after each dose. Serious adverse events (SAEs) were reported throughout the study. Results: We enrolled 158 subjects; 51.9% were males. All subjects were previously vaccinated with PPSV23; 12 subjects had received 2 to 6 doses. The mean age at enrollment was 13.3 (±3.08) years. All subjects received dose 1; 146 subjects received dose 2. One of the 12 subjects who did not complete the vaccination phase withdrew due to local pain and systemic events reported after dose 1. Subjects responded very well to a single dose of PCV13 (see Table). The prevaccination/postdose 1 IgG GMFRs ranged from 1.73 (serotype 5) to 7.01 (serotype 4). The antibodies declined over 6 months and subjects responded well to a second dose of PCV13; however the IgG GMCs were generally lower than after the first dose (see Table). The postdose 2/postdose 1 IgG GMFRs were <1.0 for all serotypes except 19F and the 95% confidence intervals did not include 1.0 except for serotypes 6A, 6B, 19F and 23F, suggesting the postdose 2 responses were significantly lower than postdose 1. 91.7% and 88.5% of subjects reported local reactions, and 87.5% and 89.3% of subjects reported systemic events after dose 1 or dose 2, respectively. Most of the SAEs were due to vaso occlusive crisis and only one was assessed by the investigator as related to the study vaccine. No deaths occurred. Conclusions: Children with SCD previously vaccinated with PPSV23 responded well to PCV13. A second dose did not enhance the immune response, likely due to the short interval (6 months) and high levels of persisting antibody. Pending analysis of functional antibody responses assessed by opsonophagocytic activity (OPA) will further describe the immune responses of PCV13 in this population. Disclosures: De Montalembert: Novartis: Speakers Bureau. Abboud:Novartis: Speakers Bureau. Fiquet:Vaccines Clinical Research Pfizer Global Research and Development : Employment. Piga:Novartis: Membership on an entity's Board of Directors or advisory committees, Research Funding. Jiang:Pfizer Inc: Employment. Pereira:Vaccines Clinical Research Pfizer Global Research and Development: Employment. Emini:Pfizer Inc.: Employment, Equity Ownership. Gruber:Pfizer: Employment. Gurtman:Pfizer: Employment. Scott:Pfizer: Employment.

scholarly journals Advances in clinical research in sickle cell disease

2008 ◽  
Vol 0 (0) ◽  
pp. 080313115903451-??? ◽  
Author(s):  
Ward Hagar ◽  
Elliott Vichinsky
Keyword(s):  
Sickle Cell Disease ◽  
Clinical Research ◽  
Sickle Cell ◽  
Cell Disease

scholarly journals A community-centered approach to sickle cell disease and clinical trials participation: an evaluation of perceptions, facilitators, and barriers

2021 ◽  
Author(s):  
LaTasha H Lee ◽  
LaShanta H Whisenton ◽  
Jasmine Benger ◽  
Sophie Lanzkron
Keyword(s):  
United States ◽  
Clinical Trial ◽  
Clinical Trials ◽  
Sickle Cell Disease ◽  
Clinical Research ◽  
Sickle Cell ◽  
Treatment Options ◽  
The United States ◽  
Personal Health Information ◽  
Cell Disease

Sickle cell disease (SCD) is the most common inherited red blood cell disorder in the United States, affecting 70,000-100,000 Americans and causing a range of serious medical complications. Although the cause of SCD was established decades ago, existing therapies have varied effectiveness and side effects, and novel therapies have been slow to develop. The limitations of existing treatment options highlight the need for new therapies that are aligned with the desires of the community. To date, little has been done to systematically seek and report the opinions and experiences of people with SCD regarding clinical research. In 2019, the American Society of Hematology Research Collaborative (ASH RC) conducted eight community workshops across the United States engaging 472 people, including persons with SCD and caregivers of those living with the disease. The workshop goals included assessing understanding, awareness, and perceptions of clinical research; and identifying the most critical clinical trial considerations of this community. Participants were asked about their experiences living with SCD and their satisfaction with treatment options. Pain and fatigue were reported as symptoms requiring better therapies. Although few participants reported being asked to enroll in a clinical trial, they expressed conditional willingness to participate. A majority were willing to share personal health information to further research and improve health outcomes. To actively engage the SCD community and increase enrollment and retention in clinical trials, researchers should address the treatment priorities of this population and ensure they have access to trusted information about clinical research and opportunities for participation.

Sickle Cell Clinical Research and Intervention Program (SCCRIP): A lifespan cohort study for sickle cell disease progression from the pediatric stage into adulthood

2018 ◽  
Vol 65 (9) ◽  
pp. e27228 ◽  
Author(s):  
Jane S. Hankins ◽  
Jeremie H. Estepp ◽  
Jason R. Hodges ◽  
Martha A. Villavicencio ◽  
Leslie L. Robison ◽  
...  
Keyword(s):  
Cohort Study ◽  
Sickle Cell Disease ◽  
Clinical Research ◽  
Disease Progression ◽  
Sickle Cell ◽  
Intervention Program ◽  
Cell Disease

Hemolysis in sickle cell disease

1974 ◽  
Vol 133 (4) ◽  
pp. 624-631 ◽  
Author(s):  
T. A. Bensinger
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Cell Disease
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