Trial Outcomes & Findings for A Study to Learn About Sickle Cell Disease In Adult Patients (NCT NCT05407805)

Participants with stable sickle cell disease (SCD) (hemoglobin S inherited from both parents \[HbS/S\] or hemoglobin S inherited from one parent and hemoglobin beta thalassemia inherited from the other parent \[HbS/beta-zero-thalassemia\] genotype) were enrolled in two concurrent groups and were observed for 6 months in this study. This was an observational study, and under this study no therapeutic study interventions were administered.

Participants were asked to complete a daily SCD electronic patient reported outcome (ePRO) diary entry to report on their experience in the past 24 hours from Day 1 to Day 180.

A Study to Learn About Sickle Cell Disease In Adult Patients

Physician-reported MU VOC: defined as an acute episode of pain with no other cause other than a VOC event that required a medical facility visit or contact with a health care professional and treatment with oral or parenteral narcotics, or non-steroidal anti-inflammatory drugs. Acute chest syndrome, hepatic sequestration, splenic sequestration, and priapism (requiring a visit to a medical facility) were also considered MU VOC. Contact with healthcare professional included: called healthcare provider (or telemedicine visit) and received treatment, went to clinic and received treatment, went to emergency department and received treatment, admitted to the hospital and received treatment. VOC rate was derived as annualized rate. Annualized MU VOC rate = (Number of MU VOC events \* 365)/ (number of days in the observation period).

A VOC was a complication of SCD characterized by vaso-occlusion presenting as recurrent pain episodes. VOC day was a self-report by the participant of experiencing a VOC during the past 24 hours. This was assessed through a dichotomous (Yes/No) item on the SCD ePRO system, "Did you have a pain crisis in the past 24 hours?" A response of "Yes" indicated a VOC day. VOC day rate was derived as annualized rate. VOC day rate = (Number of VOC days \* 365)/ (number of days in the observation period).

A VOC was a complication of SCD characterized by vaso-occlusion presenting as recurrent pain episodes. Patient reported VOC Event is used to define a sequence of VOC days that could also include single intervening days with no pain crisis. The subsequent occurrence of two consecutive days with no pain crisis operationally defines the end of the respective VOC event. Rate were derived as annualized rate. Annualized VOC event rate = (Number of VOC events \* 365)/ (number of days in the observation period).

A VOC was a complication of SCD characterized by vaso-occlusion presenting as recurrent pain episodes. VOC day was defined as the day on which a SCD participant self-reports sickle pain crisis that was recorded in the SCD ePRO. A non-VOC day was defined as the day on which a SCD participant does not self-reports sickle pain crisis. Participants rated their pain by selecting the one number that best described their pain at its worst in the past 24 hours from 0-10, where 0= no pain and 10= as bad as you can imagine. Higher scores indicated worse pain. Data in this outcome measure was presented separately for VOC state and non-VOC state.

A VOC was a complication of SCD characterized by vaso-occlusion presenting as recurrent pain episodes. VOC day was defined as the day on which a SCD participant self-reports sickle pain crisis that was recorded in the SCD ePRO. A non-VOC day was defined as the day on which a SCD participant does not self-reports sickle pain crisis. Participants rated their tiredness by selecting the one number that best described their tiredness at its worst in the past 24 hours from 0-10, where 0= no tiredness and 10= as bad as you can imagine. Higher scores indicated worse tiredness. Data in this outcome measure was presented separately for VOC state and non-VOC state.

A VOC was a complication of SCD characterized by vaso-occlusion presenting as recurrent pain episodes. VOC day was defined as the day on which a SCD participant self-reports sickle pain crisis that was recorded in the SCD ePRO. A non-VOC day was defined as the day on which a SCD participant does not self-reports sickle pain crisis. A measure of participant's ability to perform their UPA (e.g. walking, climbing stairs, or household chores) during a VOC event was assessed on SCD ePRO recorded by participants using a scale ranging from 1-4 scale, where 1= able to perform with no difficulty and 4= unable to perform usual physical activities, where higher score indicated worse status. Data in this outcome measure was presented separately for VOC state and non-VOC state.

MU VOC: acute episode of pain with no other cause other than VOC event that required medical facility visit/contact with health care professional and treatment with oral/parenteral narcotics/NSAIDs. Acute chest syndrome,hepatic/splenic sequestration, priapism also considered MU VOC. MU VOC derived as annualized rate by:(Number of MU VOC events\*365)/(number of days in observation period). VOC day rate: VOC day was self-report by participant experiencing VOC during past 24 hours. It was assessed by dichotomous (Yes/No) item on SCD ePRO system, "Did you have pain crisis in past 24 hours?" Response "Yes" indicated VOC day. VOC day rate derived as annualized rate by: (Number of VOC days\*365)/(number of days in observation period). Parameter of interest was % change of MU VOC rate per unit of change in VOC Day rate. It was estimated via Negative Binomial model to evaluate association between physician-reported MU VOC rate and SCD ePRO VOC Day rate and was reported with corresponding 95% CI.

MU VOC: acute episode of pain with no other cause other than VOC event that required medical facility visit/contact with health care professional and treatment with oral/parenteral narcotics/NSAIDs. Acute chest syndrome, hepatic/splenic sequestration, priapism also considered MU VOC. MU VOC derived as annualized rate by:(Number of MU VOC events\*365)/(number of days in observation period). VOC Event rate: Patient-reported VOC Event is used to define a sequence of VOC days. The subsequent occurrence of two consecutive days with no pain crisis operationally defines the end of the respective VOC event. Rate were derived as annualized rate by: (Number of VOC events \* 365)/ (number of days in the observation period). Parameter of interest was % change of MU VOC rate per unit of change in VOC Event rate. It was estimated via Negative Binomial model to evaluate association between physician-reported MU VOC rate and SCD ePRO VOC Event rate and was reported with corresponding 95% CI.