Improving Sickle Cell Disease Outcomes in the U.S. and Africa
Webcasts at the bottom
3 Realizing Effectiveness across Continents with Hydroxyurea (REACH): A Prospective Multi-National Trial of Hydroxyurea for Sickle Cell Anemia in Sub-Saharan Africa
Leon Tshilolo, et al.
Conclusion: The NHLBI-funded multi-national REACH trial provides the first prospective data on hydroxyurea treatment for children with SCA in sub-Saharan Africa. These results document the feasibility, safety, and benefits of daily oral hydroxyurea in the area of greatest global burden. With evidence that hydroxyurea can reduce sickle-related clinical events, transfusions, malaria, and even death, wider access to hydroxyurea in Africa can be planned to provide treatment where it is most needed.
NEJM: ORIGINAL ARTICLE
L. Tshilolo and Others
162 Significantly Improved Long Term Health Related Quality of Life (HRQL) and Neurocognition Following Familial Haploidentical Stem Cell Transplantation (HISCT) Utilizing CD34 Enrichment and Mononuclear (CD3) Addback in High Risk Patients with Sickle Cell Disease (SCD)
Mitchell Cairo, et al.
MAC and familial HISCT utilizing parental donors and CD34 enrichment and MNC addback (2 x 105 CD3/kg) in high risk patients with SCD was well tolerated, resulted in rapid hematological reconstitution, long term stable WBC and RBC (CD71) donor chimerism, low cumulative incidence of A+CGVHD and stable to improved pulmonary and cardiac function. There was also a significant improvement in processing speed and emotional and physical HRQL 2yrs post HISCT. This study was registered at clinicaltrials.gov (NCT02675959) and conducted under IND (14359) (MSC) and supported in large part by FDA R01FD004090.
315 Opioid Use Is NOT Associated with in-Hospital Mortality Among Patients with Sickle CELL Disease in the United States: Findings from the National Inpatient Sample
Oladimeji Akinola Akinboro, et al.
Hospitalization rates are rising among most age-groups of adults with SCD. The reasons for this finding are unclear but the rising rates may reflect the fragmentation of care for SCD in adults as well as age-related increases in pain-related comorbidities and SCD complications as SCD patients live longer. However, there has been no associated increase in-hospital SCD mortality, supporting extant data which suggest that the rate of opioid-related deaths in SCD is low, and the use of opioids for pain control may be considered relatively safe in the SCD population.
1023 Flipping the Switch: Initial Results of Genetic Targeting of the Fetal to Adult Globin Switch in Sickle Cell Patients
Erica B. Esrick, et al.
These early results show: (1) feasibility of enrollment, cell procurement, and GMP manufacturing of gene modified CD34+ cells in 3 adult SCD patients; (2) the first proof of principle demonstrating shRNAmiR-based gene knockdown in humans, and (3) successful rapid induction of HbF in the first patient infused, with marked attenuation of hemolysis in the early phase of autologous reconstitution. Based on the trajectory of increasing HbF/(HbF+HbS), near full suppression of the SCD phenotype is expected.