Sickle cell disease has emerged as a public health concern. Some drugs may conflict with curative therapies, yet they may be useful as a bridge to HSCT and gene therapy

2021 ◽  
Author(s):  
Moataz Dowaidar
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Low Income ◽  
Low Income Countries ◽  
Health Concern ◽  
Haematopoietic Stem Cell ◽  
Cell Disease ◽  
Public Health Concern ◽  
Disease Modifying Drugs ◽  
Matched Sibling

Sickle cell disease has resurfaced as a health-care priority in high-income nations and low-income countries (LMICs). Transplantation results with haploidentical haematopoietic stem cell transplantation (HSCT) are improving, increasing the likelihood of a curative treatment for the majority of patients. The indications for HSCT and for disease-modifying drugs, for example, must be determined. There are still a few things to think about, including biomarkers for systemic vasculopathy. Some medications may compete with curative treatments, but they might potentially be an important bridge treatment to HSCT. One of the most difficult hurdles yet ahead is reaching out to general practitioners and haematologists to bridge the awareness gap about curative alternatives such as matched sibling donor HSCT so that patients and their families may be identified early.

Advances in Sickle Cell Disease Treatments

2020 ◽  
Vol 27 ◽  
Author(s):  
Aline Renata Pavan ◽  
Jean Leandro dos Santos
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Low Income ◽  
New Drugs ◽  
Low Income Countries ◽  
High Morbidity ◽  
Single Mutation ◽  
Cell Disease ◽  
Hematopoietic Stem ◽  
Therapeutic Alternatives

: Sickle Cell Disease (SCD) is an inherited disorder of red blood cells that is caused by a single mutation in the βglobin gene. The disease, which afflicts millions of patients worldwide mainly in low income countries, is characterized by high morbidity, mortality and low life expectancy. The new pharmacological and non-pharmacological strategies for SCD is urgent in order to promote treatments able to reduce patient’s suffering and improve their quality of life. Since the FDA approval of HU in 1998, there have been few advances in discovering new drugs; however, in the last three years voxelotor, crizanlizumab, and glutamine have been approved as new therapeutic alternatives. In addition, new promising compounds have been described to treat the main SCD symptoms. Herein, focusing on drug discovery, we discuss new strategies to treat SCD that have been carried out in the last ten years to discover new, safe, and effective treatments. Moreover, nonpharmacological approaches, including red blood cell exchange, gene therapy and hematopoietic stem cell transplantation will be presented.

scholarly journals Pain and opioid use after reversal of sickle cell disease following HLA ‐matched sibling haematopoietic stem cell transplant

2018 ◽  
Vol 184 (4) ◽  
pp. 690-693 ◽  
Author(s):  
Deepika S. Darbari ◽  
Jaquette Liljencrantz ◽  
Austin Ikechi ◽  
Staci Martin ◽  
Marie Claire Roderick ◽  
...  
Keyword(s):  
Stem Cell ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Stem Cell Transplant ◽  
Haematopoietic Stem Cell Transplant ◽  
Haematopoietic Stem Cell ◽  
Cell Transplant ◽  
Cell Disease ◽  
Opioid Use ◽  
Matched Sibling

scholarly journals SICKLE CELL DISEASE AND INFECTIONS IN HIGH- AND LOW-INCOME COUNTRIES

2019 ◽  
Vol 11 (1) ◽  
pp. 02019042 ◽  
Author(s):  
Giovanna Cannas
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Host Defense ◽  
Low Income ◽  
Defense Mechanisms ◽  
Developed Countries ◽  
Infectious Complications ◽  
Low Income Countries ◽  
Cell Disease ◽  
Salmonella Infections

Infections, especially pneumococcal septicemia, meningitis, and Salmonella osteomyelitis, are a major cause of morbidity and mortality in patients with sickle cell disease (SCD). SCD increased susceptibility to infection, while infection leads to SCD-specific pathophysiological changes. The risk of infectious complications is highest in children with a palpable spleen before 6 months of age. Functional splenectomy, the results of repeated splenic infarctions, appears to be an important host-defense defect. Infection is the leading cause of death, particularly in less developed countries. Defective host-defense mechanisms enhance the risk of pneumococcal complications. Susceptibility to Salmonella infections can be explained at least in part by a similar mechanism. In high-income countries, the efficacy of the pneumococcal vaccine has been demonstrated in this disease. A decreased in infection incidence has been noted in SCD patients treated prophylactically with daily oral penicillin. Studies in low-income countries suggest the involvement of a different spectrum of etiological agents.

scholarly journals Choice of Donor Source and Conditioning Regimen for Hematopoietic Stem Cell Transplantation in Sickle Cell Disease

2019 ◽  
Vol 8 (11) ◽  
pp. 1997
Author(s):  
Emily Limerick ◽  
Courtney Fitzhugh
Keyword(s):  
Stem Cell ◽  
Sickle Cell Disease ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Sickle Cell ◽  
Cell Disease ◽  
Hematopoietic Stem ◽  
Matched Sibling

In the United States, one out of every 500 African American children have sickle cell disease (SCD), and SCD affects approximately 100,000 Americans. Significant advances in the treatment of this monogenetic disorder have failed to substantially extend the life expectancy of adults with SCD over the past two decades. Hematopoietic stem cell transplantation (HSCT) remains the only curative option for patients with SCD. While human leukocyte antigen (HLA) matched sibling HSCT has been successful, its availability is extremely limited. This review summarizes various conditioning regimens that are currently available. We explore recent efforts to expand the availability of allogeneic HSCT, including matched unrelated, umbilical cord blood, and haploidentical stem cell sources. We consider the use of nonmyeloablative conditioning and haploidentical donor sources as emerging strategies to expand transplant availability, particularly for SCD patients with complications and comorbidities who can undergo neither matched related transplant nor myeloablative conditioning. Finally, we show that improved conditioning agents have improved success rates not only in the HLA-matched sibling setting but also alternative donor settings.

scholarly journals Risk score to predict event-free survival after hematopoietic cell transplant for sickle cell disease

Blood ◽  
2020 ◽  
Vol 136 (5) ◽  
pp. 623-626 ◽  
Author(s):  
Ruta Brazauskas ◽  
Graziana M. Scigliuolo ◽  
Hai-Lin Wang ◽  
Barbara Cappelli ◽  
Annalisa Ruggeri ◽  
...  
Keyword(s):  
Risk Factors ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Risk Score ◽  
Unrelated Donor ◽  
Cell Disease ◽  
Event Free Survival ◽  
Matched Unrelated Donor ◽  
Free Survival ◽  
Matched Sibling

Abstract We developed a risk score to predict event-free survival (EFS) after allogeneic hematopoietic cell transplantation for sickle cell disease. The study population (n = 1425) was randomly split into training (n = 1070) and validation (n = 355) cohorts. Risk factors were identified and validated via Cox regression models. Two risk factors of 9 evaluated were predictive for EFS: age at transplantation and donor type. On the basis of the training cohort, patients age 12 years or younger with an HLA-matched sibling donor were at the lowest risk with a 3-year EFS of 92% (score, 0). Patients age 13 years or older with an HLA-matched sibling donor or age 12 years or younger with an HLA-matched unrelated donor were at intermediate risk (3-year EFS, 87%; score, 1). All other groups, including patients of any age with a haploidentical relative or HLA-mismatched unrelated donor and patients age 13 years or older with an HLA-matched unrelated donor were high risk (3-year EFS, 57%; score, 2 or 3). These findings were confirmed in the validation cohort. This simple risk score may guide patients with sickle cell disease and hematologists who are considering allogeneic transplantation as a curative treatment relative to other available contemporary treatments.

scholarly journals Newborn Screening for Sickle Cell Disease Using Point-of-Care Testing in Low-Income Setting

PEDIATRICS ◽  
2019 ◽  
Vol 144 (4) ◽  
pp. e20184105 ◽  
Author(s):  
Ofelia A. Alvarez ◽  
Tally Hustace ◽  
Mimose Voltaire ◽  
Alejandro Mantero ◽  
Ulrick Liberus ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Newborn Screening ◽  
Sickle Cell ◽  
Low Income ◽  
Point Of Care ◽  
Cell Disease ◽  
Point Of Care Testing ◽  
Low Income Setting

Managing Sickle Cell Disease in Low-Income Families.

1996 ◽  
Vol 25 (3) ◽  
pp. 420
Author(s):  
Essie Manuel Rutledge ◽  
Shirley A. Hill
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Low Income ◽  
Cell Disease ◽  
Low Income Families
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