Implementation of national blood conservation recommendations at an adult sickle cell center.

BACKGROUND: Due to the national blood supply crisis caused by the COVID-19 pandemic, the American Society of Hematology proposed guidance to decrease blood utilization for sickle cell patients on chronic transfusion therapy (CTT). Little evidence exists to support the efficacy and safety of these blood conservation strategies. STUDY DESIGN AND METHODS: Through retrospective analysis, we sought to describe outcomes following implementation of these recommendations in 58 adult sickle cell patients on chronic exchange transfusions. The strategies employed included: relaxing the goal fraction of cells remaining (FCR) to 30%-50%, utilizing depletion exchanges in select patients, and transitioning select patients to monthly simple transfusions. We compared hemoglobin S%, hemoglobin values, and other laboratory parameters, acute care visits, and red blood cell usage during the first year of the COVID-19 pandemic to the year prior using Wilcoxon signed rank test. RESULTS: Of 53 patients who remained on chronic exchanges during the pandemic, use of depletion exchange increased (15%-23%) and FCR increased (34.9 [SD 4.7] vs. 37.6 [SD 4.5], p < .05). These changes resulted in 854 units conserved without clinically significant changes to pre-exchange laboratory parameters, including hemoglobin S%, or number of acute care presentations. In contrast, five patients who transitioned to predominantly simple transfusions, experienced difficulty maintaining hemoglobin S% less than 30 and worsening anemia. DISCUSSION: Our data suggest that in a blood shortage crisis, optimizing the exchange procedure itself may be the safest means of conserving blood in a population of adult patients with sickle cell disease.

Patient perception of voxelotor treatment benefit in sickle cell disease.

Patients with sickle cell disease (SCD) experience a range of clinical symptoms, including acute and chronic pain, fatigue, and respiratory problems, as well as chronic organ complications that can lead to disability and accelerated mortality. Voxelotor is a first-in-class therapy that targets sickle hemoglobin polymerization, the root cause of SCD. It is approved by the US Food and Drug Administration for treatment of SCD in patients aged 4 years and older and in the European Union and United Arab Emirates for the treatment of SCD in patients aged 12 years and older. Here, we report the single-center experience of both clinician-determined and patient-reported benefits of voxelotor in 27 consecutive patients treated for at least 8 weeks. Clinical Global Impression of Change and Patient Global Impression of Change rating scales were used to capture clinicians' and patients' perceptions of change in overall patient health-related quality-of-life with voxelotor treatment. Laboratory data were also collected to assess clinical response to treatment. As observed in previous clinical studies, hemoglobin concentrations and markers of hemolysis were improved in patients treated with voxelotor. Most patients reported marked improvement in disease symptoms, which correlated well with the clinicians' assessments. Although limited by the retrospective open-label study design, these findings suggest that voxelotor use has a positive impact on outcomes in patients with SCD.