The purpose of this clinical trial is to evaluate the performance of the sickle cell disease (SCD) electronic diary in people with SCD who are on treatment that will change SCD and those not on such a treatment. SCD is a type of condition when there are fewer red blood cells to carry oxygen around the body. This disease can be passed on from parent to child and may cause pain, infections and damage to organs. This study is seeking participants who: * are confirmed with SCD * are on a stable regimen of disease changing treatment or have not received any disease changing treatment before the start of the study and do not plan any changes in their treatment during the 6-month study observation period For 6 months, participants will be asked to complete a daily electronic diary to report on their experience in the past 24 hours with sickle cell pain crisis (if they got any treatment and what medications they took), worst pain, worst tiredness, and their ability to perform usual physical activities. We will compare the experiences of people who are taking SCD-modifying therapy to those that are not taking a SCD-modifying therapy.
Study Type
OBSERVATIONAL
Enrollment
98
Participants will be asked to complete a daily electronic patient reported outcome diary entry to report on their experience in the past 24 hours.
Foundation for Sickle Cell Disease Research
Hollywood, Florida, United States
Mid-Atlantic Permanente Medical Group Largo Medical Center
Largo, Maryland, United States
Mid-Atlantic Permanente Medical Group Largo Medical Center
Upper Marlboro, Maryland, United States
Sanguine Biosciences, Inc.
Waltham, Massachusetts, United States
Cohen Children's Medical Center
New Hyde Park, New York, United States
Physician-reported Medical Utilization vaso-occlusive crisis (VOC) rate
Confirmation that the population is suitable for assessing responsiveness of electronic patient reported outcomes based upon a lower frequency rate of Physician reported Medical Utilization VOCs in the SCD disease modifying treatment group.
Time frame: Day 1 to 180
VOC Day rate
Responsiveness of electronic patient reported outcomes between participants treated with SCD disease modifying treatment or no disease modifying treatment.
Time frame: Day 1 to 180
Patient-reported VOC Event rate
Responsiveness of electronic patient reported outcomes between participants treated with SCD disease modifying treatment or no disease modifying treatment.
Time frame: Day 1 to 180
Average SCD ePRO daily worst pain score during VOC days
Responsiveness of electronic patient reported outcomes between participants treated with SCD disease modifying treatment or no disease modifying treatment.
Time frame: Day 1 to 180
Average SCD ePRO daily worst pain score during non-VOC days
Responsiveness of electronic patient reported outcomes between participants treated with SCD disease modifying treatment or no disease modifying treatment.
Time frame: Day 1 to 180
Average SCD ePRO daily worst tiredness score during VOC days
Responsiveness of electronic patient reported outcomes between participants treated with SCD disease modifying treatment or no disease modifying treatment.
Time frame: Day 1 to 180
Average SCD ePRO daily worst tiredness score during non-VOC days
Responsiveness of electronic patient reported outcomes between participants treated with SCD disease modifying treatment or no disease modifying treatment.
Time frame: Day 1 to 180
Average SCD ePRO daily rating for ability to perform usual physical activity during a VOC day
Responsiveness of electronic patient reported outcomes between participants treated with SCD disease modifying treatment or no disease modifying treatment.
Time frame: Day 1 to 180
Average SCD ePRO daily rating for ability to perform usual physical activity during a non-VOC day
Responsiveness of electronic patient reported outcomes between participants treated with SCD disease modifying treatment or no disease modifying treatment.
Time frame: Day 1 to 180
Quantitative relationship between VOC Day rate and Physician-reported Medical Utilization VOC
Quantitative measure of association between VOC Day rate and Physician-reported Medical Utilization VOC.
Time frame: Day 1 to 180
Quantitative relationship between VOC Day rate and Physician-reported Medical Utilization VOC rate across treatment groups
Comparison of quantitative measures of treatment effect between VOC Day rate and Physician-reported Medical Utilization VOC rate.
Time frame: Day 1 to 180
Quantitative relationship between Patient-reported VOC Event rate and Physician-reported Medical Utilization VOC rate
Quantitative measure of association between Patient-reported VOC Event rate and Physician-reported Medical Utilization VOC rate.
Time frame: Day 1 to 180
Quantitative relationship between Patient-reported VOC Event rate and Physician-reported Medical Utilization VOC rate across treatment groups
Comparison of quantitative measures of treatment effect between Patient-reported VOC Event rate and Physician-reported Medical Utilization VOC rate.
Time frame: Day 1 to 180
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