Sickle Cell Anemia, Sickle Cell Disease
Conditions
Keywords
Red Cell Survival, Biotin, Sickle Cell Anemia, Sickle Cell Disease, Sickle Cell Trait
Brief summary
Background: Sickle cell disease (SCD) is an inherited blood disorder. It results from a single genetic change (mutation) in red blood cells (RBCs). RBCs are the cells that carry oxygen to the body. In people with SCD, some RBCs are abnormal and die early. This leaves a shortage of healthy RBCs. Researchers want to learn more about how long RBCs live in the human body. Objective: To study how long RBCs live in people with and without SCD. Eligibility: People age 18 and older who either have SCD, had SCD but were cured with a bone marrow transplant, have the sickle cell trait (SCT), or are a healthy volunteer without SCD or SCT Design: Participants will be screened with a medical history and physical exam. They will give a blood sample. Participants will have a small amount of blood drawn from a vein. In the laboratory, the blood will be mixed with a vitamin called biotin. Biotin sticks to the outside of RBCs without changing their function, shape, or overall lifetime. This process is known as biotin labeling of RBCs. The biotin labeled RBCs will be returned to the participant via vein injection. Participants will give frequent blood samples. Their RBCs will be studied to see how many biotin labeled RBCs remain over time. This shows how long the RBCs live. Participants will give blood samples until no biotin labeled RBCs can be detected. During the study visits, participants will report any major changes to their health. Participation lasts for up to 6 months.
Detailed description
Study Description: This study will use biotin-labeling of red blood cells (RBCs) to determine the mean potential lifespan (MPL) of RBCs in patients with sickle cell disease (SCD) compared to patients who have successfully undergone curative bone marrow transplantation (BMT, allogeneic or autologous), participants with sickle cell trait, and healthy donors without SCD. Previous studies have corroborated the MPL of healthy donor RBCs to be approximately 115 days while RBCs from patients with SCD have a much more variable but consistently shorter MPL of approximately 32 days. Allogeneic BMT is a curative therapy for the treatment of severe SCD with stable, mixed donor recipient chimerism after BMT sufficient to reverse the sickle cell phenotype by virtue of improved donor red cell survival compared to the ineffective erythropoiesis of SCD. We predict that the hematologic variables associated with red cell survival among patients with SCD vs. participants with SCT and healthy donors can be used to determine the necessary amount of corrected hemoglobin required to overcome the red cell pathology of SCD. Data generated will be used to determine the utility of performing a population study of RBC lifespan in gene therapy treated patients to ultimately target the percentage of transferred globin gene needed to reverse SCD. The data generated will refine our understanding of the degree of correction necessary to reverse the phenotype of SCD. Objectives: Primary Objective: To determine and compare red blood cell survival in patients with SCD, patients with SCD who have undergone BMT, participants with SCT, and healthy donors, and validate the association of red cell survival with known markers of increased red cell survival. Secondary Objectives: To evaluate correlation of markers of hemolysis (reticulocyte count), number of alpha globin genes, and fetal hemoglobin with RBC survival. Endpoints: Primary Endpoint: Red blood cell survival Secondary Endpoints: Relationship of red blood cell survival to hematologic parameters. Antibody detection to biotin.
Interventions
DRUGBiotin label
Autologous cells will be collected and biotin-labeled ex vivo and reinfused to measure red cell survival
Sponsors
National Heart, Lung, and Blood Institute (NHLBI)
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
BASIC_SCIENCE
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 99 Years
Healthy volunteers
Yes
Inclusion criteria
* Age 18 or greater with a confirmed diagnosis of homozygous SCD (HbSS, HbSC, HbSB0), sickle cell trait (HbAS), or healthy volunteer (HbA) * Normal renal function: creatinine \<1.5 mg/dL * Negative direct antiglobulin test (DAT) * Ability to give informed consent to participate in the protocol
Exclusion criteria
* Any uncontrolled chronic illness other than sickle cell disease * Active viral, bacterial, fungal, or parasitic infection * Consumption of biotin supplements or raw eggs within 30 days * Blood loss within the previous 8 weeks \>540mL * Pregnancy * Pre-existing, naturally occurring antibodies against biotin
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Mean Red Blood Cells Lifespan in Participants | Sickle Cell Disease Pre-Transplantation cohort time frame is as follows: baseline, twice weekly up to week 3 then weekly up to week 22. All other cohorts, time frame is as follows: baseline, weekly up to week 4, then every other week up to week 22. | Mean Days of Red Blood Cells (RBC) survival in participants with Sickle Cell Disease (SCD), participants with SCD who have undergone stem cell transplant, participants with Sickle Cell Trait, and healthy volunteers. Peripheral blood samples were analyzed by flow cytometry until biotin was not detectable on RBC. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Mean White Blood Cell Count | Sickle Cell Disease Pre-Transplantation cohort time frame is as follows: baseline, twice weekly up to week 3 then weekly up to week 22. All other cohorts, time frame is as follows: baseline, weekly up to week 4, then every other week up to week 22. | Mean White Blood Cell (WBC) count between Sickle Cell Disease Pre-transplantation and Sickle Cell Disease Post-Transplantation |
| Mean Red Blood Cell Count | Sickle Cell Disease Pre-Transplantation cohort time frame is as follows: baseline, twice weekly up to week 3 then weekly up to week 22. All other cohorts, time frame is as follows: baseline, weekly up to week 4, then every other week up to week 22. | Mean Red Blood Cell (RBC) Count between Sickle Cell Disease Pre-transplantation and Sickle Cell Disease Post-Transplantation |
| Mean Hemoglobin Value | Sickle Cell Disease Pre-Transplantation cohort time frame is as follows: baseline, twice weekly up to week 3 then weekly up to week 22. All other cohorts, time frame is as follows: baseline, weekly up to week 4, then every other week up to week 22. | Mean Hemoglobin (Hb) Value between Sickle Cell Disease Pre-transplantation and Sickle Cell Disease Post-Transplantation |
| Mean Hematocrit Value | Sickle Cell Disease Pre-Transplantation cohort time frame is as follows: baseline, twice weekly up to week 3 then weekly up to week 22. All other cohorts, time frame is as follows: baseline, weekly up to week 4, then every other week up to week 22. | Mean Hematocrit (Hct) value between Sickle Cell Disease Pre-transplantation and Sickle Cell Disease Post-Transplantation |
| Mean Value of Mean Corpuscular Volume | Sickle Cell Disease Pre-Transplantation cohort time frame is as follows: baseline, twice weekly up to week 3 then weekly up to week 22. All other cohorts, time frame is as follows: baseline, weekly up to week 4, then every other week up to week 22. | Mean Value of Mean Corpuscular Volume (MCV) between Sickle Cell Disease Pre-transplantation and Sickle Cell Disease Post-Transplantation |
| Mean Absolute Reticulocyte Count | Sickle Cell Disease Pre-Transplantation cohort time frame is as follows: baseline, twice weekly up to week 3 then weekly up to week 22. All other cohorts, time frame is as follows: baseline, weekly up to week 4, then every other week up to week 22. | Mean Absolute Reticulocyte Count (ARC) between Sickle Cell Disease Pre-transplantation and Sickle Cell Disease Post-Transplantation |
| Number of Participants With Antibody Detection | Baseline, 3 months, 6 months | Number of participants with Antibody detection to biotin |
| Mean Total Bilirubin Value | Sickle Cell Disease Pre-Transplantation cohort time frame is as follows: baseline, twice weekly up to week 3 then weekly up to week 22. All other cohorts, time frame is as follows: baseline, weekly up to week 4, then every other week up to week 22. | Mean Total Bilirubin value between Sickle Cell Disease Pre-transplantation and Sickle Cell Disease Post-Transplantation |
| Mean Lactate Dehydrogenase Value | Sickle Cell Disease Pre-Transplantation cohort time frame is as follows: baseline, twice weekly up to week 3 then weekly up to week 22. All other cohorts, time frame is as follows: baseline, weekly up to week 4, then every other week up to week 22. | Mean Lactate Dehydrogenase (LDH) value between Sickle Cell Disease Pre-transplantation and Sickle Cell Disease Post-Transplantation |
| Mean Adult Hemoglobin Percentage | Sickle Cell Disease Pre-Transplantation cohort time frame is as follows: baseline, twice weekly up to week 3 then weekly up to week 22. All other cohorts, time frame is as follows: baseline, weekly up to week 4, then every other week up to week 22. | Mean Adult Hemoglobin (HbA) percentage between Sickle Cell Disease Pre-transplantation and Sickle Cell Disease Post-Transplantation |
| Mean Sickle Hemoglobin Percentage | Sickle Cell Disease Pre-Transplantation cohort time frame is as follows: baseline, twice weekly up to week 3 then weekly up to week 22. All other cohorts, time frame is as follows: baseline, weekly up to week 4, then every other week up to week 22. | Mean Sickle Hemoglobin (HbS) Percentage between Sickle Cell Disease Pre-transplantation and Sickle Cell Disease Post-Transplantation |
| Mean Fetal Hemoglobin Percentage | Sickle Cell Disease Pre-Transplantation cohort time frame is as follows: baseline, twice weekly up to week 3 then weekly up to week 22. All other cohorts, time frame is as follows: baseline, weekly up to week 4, then every other week up to week 22. | Mean Fetal Hemoglobin (HbF) Percentage between Sickle Cell Disease Pre-transplantation and Sickle Cell Disease Post-Transplantation |
| Mean Aspartate Aminotransferase Value | Sickle Cell Disease Pre-Transplantation cohort time frame is as follows: baseline, twice weekly up to week 3 then weekly up to week 22. All other cohorts, time frame is as follows: baseline, weekly up to week 4, then every other week up to week 22. | Mean Aspartate Aminotransferase (AST) value between Sickle Cell Disease Pre-transplantation and Sickle Cell Disease Post-Transplantation |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Sickle Cell Disease Pre-Transplantation Autologous cells will be collected in participants with Sickle Cell Disease Pre-Transplantation and biotin-labeled ex vivo and reinfused to measure red cell survival | 6 |
| Sickle Cell Disease Post-Transplantation Autologous cells will be collected in participants with Sickle Cell Disease Post-Transplantation and biotin-labeled ex vivo and reinfused to measure red cell survival | 6 |
| Sickle Cell Trait (HbAS) Autologous cells will be collected in participants with Sickle Cell Trait (HbAS) and biotin-labeled ex vivo and reinfused to measure red cell survival | 7 |
| HbAA (Healthy Volunteers) Autologous cells will be collected in participants with HbAA (Healthy volunteers) and biotin-labeled ex vivo and reinfused to measure red cell survival | 3 |
| Total | 22 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 | FG002 | FG003 |
|---|---|---|---|---|---|
| Overall Study | Withdrawal by Subject | 0 | 0 | 1 | 0 |
Baseline characteristics
| Characteristic | Total | Sickle Cell Disease Pre-Transplantation | Sickle Cell Disease Post-Transplantation | Sickle Cell Trait (HbAS) | HbAA (Healthy Volunteers) |
|---|---|---|---|---|---|
| Age, Categorical <=18 years | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical >=65 years | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical Between 18 and 65 years | 22 Participants | 6 Participants | 6 Participants | 7 Participants | 3 Participants |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 22 Participants | 6 Participants | 6 Participants | 7 Participants | 3 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 22 Participants | 6 Participants | 6 Participants | 7 Participants | 3 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Region of Enrollment United States | 22 participants | 6 participants | 6 participants | 7 participants | 3 participants |
| Sex: Female, Male Female | 14 Participants | 4 Participants | 2 Participants | 6 Participants | 2 Participants |
| Sex: Female, Male Male | 8 Participants | 2 Participants | 4 Participants | 1 Participants | 1 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk |
|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 6 | 0 / 6 | 0 / 7 | 0 / 3 |
| other Total, other adverse events | 0 / 6 | 0 / 6 | 0 / 7 | 0 / 3 |
| serious Total, serious adverse events | 0 / 6 | 0 / 6 | 0 / 7 | 0 / 3 |
Outcome results
Primary
Mean Red Blood Cells Lifespan in Participants
Mean Days of Red Blood Cells (RBC) survival in participants with Sickle Cell Disease (SCD), participants with SCD who have undergone stem cell transplant, participants with Sickle Cell Trait, and healthy volunteers. Peripheral blood samples were analyzed by flow cytometry until biotin was not detectable on RBC.
Time frame: Sickle Cell Disease Pre-Transplantation cohort time frame is as follows: baseline, twice weekly up to week 3 then weekly up to week 22. All other cohorts, time frame is as follows: baseline, weekly up to week 4, then every other week up to week 22.
Population: Analysis includes participants that have completed study and had no exchange or simple red blood cell transfusions.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Sickle Cell Disease Pre-Transplantation | Mean Red Blood Cells Lifespan in Participants | 64.1 day |
| Sickle Cell Disease Post-Transplantation | Mean Red Blood Cells Lifespan in Participants | 113.4 day |
| Sickle Cell Trait (HbAS) | Mean Red Blood Cells Lifespan in Participants | 126.0 day |
| HbAA (Healthy Volunteers) | Mean Red Blood Cells Lifespan in Participants | 123.7 day |
p-value: <0.001Wilcoxon (Mann-Whitney)
Secondary
Mean Absolute Reticulocyte Count
Mean Absolute Reticulocyte Count (ARC) between Sickle Cell Disease Pre-transplantation and Sickle Cell Disease Post-Transplantation
Time frame: Sickle Cell Disease Pre-Transplantation cohort time frame is as follows: baseline, twice weekly up to week 3 then weekly up to week 22. All other cohorts, time frame is as follows: baseline, weekly up to week 4, then every other week up to week 22.
Population: Pre-specified in the protocol to only assess this Outcome Measure in the Sickle Cell Disease Arms/Groups.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Sickle Cell Disease Pre-Transplantation | Mean Absolute Reticulocyte Count | 192.7 K/mcL | Standard Deviation 84.8 |
| Sickle Cell Disease Post-Transplantation | Mean Absolute Reticulocyte Count | 152.9 K/mcL | Standard Deviation 96.4 |
p-value: 0.48t-test, 2 sided
Secondary
Mean Adult Hemoglobin Percentage
Mean Adult Hemoglobin (HbA) percentage between Sickle Cell Disease Pre-transplantation and Sickle Cell Disease Post-Transplantation
Time frame: Sickle Cell Disease Pre-Transplantation cohort time frame is as follows: baseline, twice weekly up to week 3 then weekly up to week 22. All other cohorts, time frame is as follows: baseline, weekly up to week 4, then every other week up to week 22.
Population: Pre-specified in the protocol to only assess this Outcome Measure in the Sickle Cell Disease Arms/Groups.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Sickle Cell Disease Pre-Transplantation | Mean Adult Hemoglobin Percentage | 2.8 % of Adult Hemoglobin (HbA) | Standard Deviation 6.9 |
| Sickle Cell Disease Post-Transplantation | Mean Adult Hemoglobin Percentage | 59.6 % of Adult Hemoglobin (HbA) | Standard Deviation 8.2 |
p-value: <0.01t-test, 2 sided
Secondary
Mean Aspartate Aminotransferase Value
Mean Aspartate Aminotransferase (AST) value between Sickle Cell Disease Pre-transplantation and Sickle Cell Disease Post-Transplantation
Time frame: Sickle Cell Disease Pre-Transplantation cohort time frame is as follows: baseline, twice weekly up to week 3 then weekly up to week 22. All other cohorts, time frame is as follows: baseline, weekly up to week 4, then every other week up to week 22.
Population: Pre-specified in the protocol to only assess this Outcome Measure in the Sickle Cell Disease Arms/Groups.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Sickle Cell Disease Pre-Transplantation | Mean Aspartate Aminotransferase Value | 26.1 IU/L | Standard Deviation 12.7 |
| Sickle Cell Disease Post-Transplantation | Mean Aspartate Aminotransferase Value | 26.1 IU/L | Standard Deviation 5.5 |
p-value: 0.99t-test, 2 sided
Secondary
Mean Fetal Hemoglobin Percentage
Mean Fetal Hemoglobin (HbF) Percentage between Sickle Cell Disease Pre-transplantation and Sickle Cell Disease Post-Transplantation
Time frame: Sickle Cell Disease Pre-Transplantation cohort time frame is as follows: baseline, twice weekly up to week 3 then weekly up to week 22. All other cohorts, time frame is as follows: baseline, weekly up to week 4, then every other week up to week 22.
Population: Pre-specified in the protocol to only assess this Outcome Measure in the Sickle Cell Disease Arms/Groups.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Sickle Cell Disease Pre-Transplantation | Mean Fetal Hemoglobin Percentage | 10.4 % of Fetal Hemoglobin | Standard Deviation 9.1 |
| Sickle Cell Disease Post-Transplantation | Mean Fetal Hemoglobin Percentage | .5 % of Fetal Hemoglobin | Standard Deviation 0.8 |
p-value: 0.04t-test, 2 sided
Secondary
Mean Hematocrit Value
Mean Hematocrit (Hct) value between Sickle Cell Disease Pre-transplantation and Sickle Cell Disease Post-Transplantation
Time frame: Sickle Cell Disease Pre-Transplantation cohort time frame is as follows: baseline, twice weekly up to week 3 then weekly up to week 22. All other cohorts, time frame is as follows: baseline, weekly up to week 4, then every other week up to week 22.
Population: Pre-specified in the protocol to only assess this Outcome Measure in the Sickle Cell Disease Arms/Groups.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Sickle Cell Disease Pre-Transplantation | Mean Hematocrit Value | 25.0 % of Hematocrit | Standard Deviation 6.3 |
| Sickle Cell Disease Post-Transplantation | Mean Hematocrit Value | 38.3 % of Hematocrit | Standard Deviation 5.8 |
p-value: 0.01t-test, 2 sided
Secondary
Mean Hemoglobin Value
Mean Hemoglobin (Hb) Value between Sickle Cell Disease Pre-transplantation and Sickle Cell Disease Post-Transplantation
Time frame: Sickle Cell Disease Pre-Transplantation cohort time frame is as follows: baseline, twice weekly up to week 3 then weekly up to week 22. All other cohorts, time frame is as follows: baseline, weekly up to week 4, then every other week up to week 22.
Population: Pre-specified in the protocol to only assess this Outcome Measure in the Sickle Cell Disease Arms/Groups.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Sickle Cell Disease Pre-Transplantation | Mean Hemoglobin Value | 8.8 g/dL | Standard Deviation 2.1 |
| Sickle Cell Disease Post-Transplantation | Mean Hemoglobin Value | 13.0 g/dL | Standard Deviation 2.2 |
p-value: 0.01t-test, 2 sided
Secondary
Mean Lactate Dehydrogenase Value
Mean Lactate Dehydrogenase (LDH) value between Sickle Cell Disease Pre-transplantation and Sickle Cell Disease Post-Transplantation
Time frame: Sickle Cell Disease Pre-Transplantation cohort time frame is as follows: baseline, twice weekly up to week 3 then weekly up to week 22. All other cohorts, time frame is as follows: baseline, weekly up to week 4, then every other week up to week 22.
Population: Pre-specified in the protocol to only assess this Outcome Measure in the Sickle Cell Disease Arms/Groups.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Sickle Cell Disease Pre-Transplantation | Mean Lactate Dehydrogenase Value | 323.9 unit/L | Standard Deviation 254.1 |
| Sickle Cell Disease Post-Transplantation | Mean Lactate Dehydrogenase Value | 253.7 unit/L | Standard Deviation 323.9 |
p-value: 0.43t-test, 2 sided
Secondary
Mean Red Blood Cell Count
Mean Red Blood Cell (RBC) Count between Sickle Cell Disease Pre-transplantation and Sickle Cell Disease Post-Transplantation
Time frame: Sickle Cell Disease Pre-Transplantation cohort time frame is as follows: baseline, twice weekly up to week 3 then weekly up to week 22. All other cohorts, time frame is as follows: baseline, weekly up to week 4, then every other week up to week 22.
Population: Pre-specified in the protocol to only assess this Outcome Measure in the Sickle Cell Disease Arms/Groups.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Sickle Cell Disease Pre-Transplantation | Mean Red Blood Cell Count | 3.0 M/mcL | Standard Deviation 1 |
| Sickle Cell Disease Post-Transplantation | Mean Red Blood Cell Count | 4.3 M/mcL | Standard Deviation 0.8 |
p-value: 0.04t-test, 2 sided
Secondary
Mean Sickle Hemoglobin Percentage
Mean Sickle Hemoglobin (HbS) Percentage between Sickle Cell Disease Pre-transplantation and Sickle Cell Disease Post-Transplantation
Time frame: Sickle Cell Disease Pre-Transplantation cohort time frame is as follows: baseline, twice weekly up to week 3 then weekly up to week 22. All other cohorts, time frame is as follows: baseline, weekly up to week 4, then every other week up to week 22.
Population: Pre-specified in the protocol to only assess this Outcome Measure in the Sickle Cell Disease Arms/Groups.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Sickle Cell Disease Pre-Transplantation | Mean Sickle Hemoglobin Percentage | 68.7 % of Sickle Hemoglobin (HbS) | Standard Deviation 17.1 |
| Sickle Cell Disease Post-Transplantation | Mean Sickle Hemoglobin Percentage | 36.7 % of Sickle Hemoglobin (HbS) | Standard Deviation 7.5 |
p-value: 0.01t-test, 1 sided
Secondary
Mean Total Bilirubin Value
Mean Total Bilirubin value between Sickle Cell Disease Pre-transplantation and Sickle Cell Disease Post-Transplantation
Time frame: Sickle Cell Disease Pre-Transplantation cohort time frame is as follows: baseline, twice weekly up to week 3 then weekly up to week 22. All other cohorts, time frame is as follows: baseline, weekly up to week 4, then every other week up to week 22.
Population: Pre-specified in the protocol to only assess this Outcome Measure in the Sickle Cell Disease Arms/Groups.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Sickle Cell Disease Pre-Transplantation | Mean Total Bilirubin Value | 7.1 mg/dL | Standard Deviation 1 |
| Sickle Cell Disease Post-Transplantation | Mean Total Bilirubin Value | 1.1 mg/dL | Standard Deviation 1.7 |
p-value: 0.39t-test, 2 sided
Secondary
Mean Value of Mean Corpuscular Volume
Mean Value of Mean Corpuscular Volume (MCV) between Sickle Cell Disease Pre-transplantation and Sickle Cell Disease Post-Transplantation
Time frame: Sickle Cell Disease Pre-Transplantation cohort time frame is as follows: baseline, twice weekly up to week 3 then weekly up to week 22. All other cohorts, time frame is as follows: baseline, weekly up to week 4, then every other week up to week 22.
Population: Pre-specified in the protocol to only assess this Outcome Measure in the Sickle Cell Disease Arms/Groups.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Sickle Cell Disease Pre-Transplantation | Mean Value of Mean Corpuscular Volume | 86.7 fL | Standard Deviation 12.8 |
| Sickle Cell Disease Post-Transplantation | Mean Value of Mean Corpuscular Volume | 89.4 fL | Standard Deviation 2.3 |
p-value: 0.65t-test, 2 sided
Secondary
Mean White Blood Cell Count
Mean White Blood Cell (WBC) count between Sickle Cell Disease Pre-transplantation and Sickle Cell Disease Post-Transplantation
Time frame: Sickle Cell Disease Pre-Transplantation cohort time frame is as follows: baseline, twice weekly up to week 3 then weekly up to week 22. All other cohorts, time frame is as follows: baseline, weekly up to week 4, then every other week up to week 22.
Population: Pre-specified in the protocol to only assess this Outcome Measure in the Sickle Cell Disease Arms/Groups.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Sickle Cell Disease Pre-Transplantation | Mean White Blood Cell Count | 9.0 K/mcL | Standard Deviation 4.5 |
| Sickle Cell Disease Post-Transplantation | Mean White Blood Cell Count | 6.5 K/mcL | Standard Deviation 2.4 |
p-value: 0.28t-test, 2 sided
Secondary
Number of Participants With Antibody Detection
Number of participants with Antibody detection to biotin
Time frame: Baseline, 3 months, 6 months
Population: Analysis includes participants that have completed study and had no exchange or simple red blood cell transfusions.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Sickle Cell Disease Pre-Transplantation | Number of Participants With Antibody Detection | 0 Participants |
| Sickle Cell Disease Post-Transplantation | Number of Participants With Antibody Detection | 0 Participants |
| Sickle Cell Trait (HbAS) | Number of Participants With Antibody Detection | 0 Participants |
| HbAA (Healthy Volunteers) | Number of Participants With Antibody Detection | 0 Participants |