Assessing Functional Capacity in Sickle Cell Patients Receiving Voxelotor
Voxelotor is a novel hemoglobin polymerization inhibitor which has been demonstrated to reduce hemolysis and improve hemoglobin levels. There have been numerous studies examining the clinical impact of voxelotor in sickle cell disease (SCD) patients, but there are few published reports on the effects of treatment on physical function in patients with SCD. The hypothesis to be tested is that anemic SCD patients will have improvements in performance after 6 months of voxelotor treatment.
• Provision of signed and dated informed consent form
• Stated willingness to comply with all study procedures and availability for the duration of the study
• Male or female,
• aged 18-65 years
• Diagnosis of sickle cell disease with HbSS, HbSc, HbS-beta thalassemia genotypes
• Hemoglobin level of 6 g/dL - 10.5 g/dL
• Between 0 and 10 vaso-occlusive (VOC) episodes in previous 12 months
• Ability to take oral medication and be willing to adhere to the voxelotor regimen
• For females of reproductive potential, they will be educated to avoid pregnancy during study period and for one month after due to limited data surrounding pregnancy and voxelotor
⁃ For males of reproductive potential: use of condoms or other methods to ensure effective contraception with partner
⁃ Agreement to adhere to Lifestyle Considerations (see section 5.3) throughout study duration