Anemia, Sickle Cell
Conditions
Keywords
Hemoglobin S Disease, Sickle Cell Anemia
Brief summary
Children with sickle cell anemia (SCA) seem to have higher energy needs than children who do not have the disease. This may be the reason why children and teenagers with sickle cell anemia tend to be smaller, weigh less, and have less fat and muscle than children and teens that do not have the disease. This study is being done to find out if giving a supplement called glutamine will help children with sickle cell anemia by lowering their energy needs and improving their growth and strength. Children will be randomly assigned (like a flip of a coin) to one of two groups. One group will take glutamine and one group will take a placebo (a protein mixture that looks like glutamine but may not have the same effect in the body). No one will know which group is taking which supplement until the study has been completed. Children will be in the study for 12 months.
Detailed description
1. The study will compare the effect of glutamine and placebo on resting energy expenditure (REE) in children with sickle cell anemia (SCA) by comparing the change in REE ratio between baseline and 12 months. 2. The study investigates the effect of oral glutamine and placebo on body composition in children with SCA by comparing the difference in body mass indexes (BMIs) and percent of body fat (DEXA Scan) between baseline and 12 months of treatment in the two groups. 3. This Study will investigate the effect of oral glutamine and placebo on growth in children with SCA by comparing the Z scores for one year before baseline to 1 year while on study. 4. This study will investigate the effect of oral glutamine and placebo in children with SCA by comparing the difference in the levels of plasma and red blood cell glutamine between baseline and 12 months of treatment in the two groups. 5. This study will investigate the clinical effects (strength and exercise endurance) of oral glutamine and placebo in children with SCA by comparing the difference between baseline and 12 months of treatment in the two groups. 6. This study will evaluate quality of life in children with SCA who have glutamine or placebo for 12 months. 7. This study will evaluate the changes in REE over time in a small group of patients that will have REE measurement at months 3, 6, and 9. This objective will be offered to all patients, but will be additional studies that are not required to participate in the protocol.
Interventions
DRUGGlutamine
0.6 gm/kg of oral glutamine per day, in two doses for one year.
DRUGPlacebo
Placebo
Sponsors
Thrasher Research Fund
St. Jude Children's Research Hospital
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
PREVENTION
Masking
TRIPLE (Subject, Caregiver, Investigator)
Eligibility
Sex/Gender
ALL
Age
5 Years to 18 Years
Healthy volunteers
No
Inclusion criteria
Active patient (presently receiving medical care for SCA) at the St. Jude Comprehensive Sickle Cell Center (SJCSCC) or an affiliate or alliance of St Jude. Patient must not be in the high risk category as defined by the SJCSCC. High risk is defined as follows: * 3 or more admissions and/or emergency department visits for pain within the past 12 months, or; * 2 or more episodes of acute chest syndrome within the past 24 months, or; * A combination of pain and ACS events \> 3 within the past 12 months. * 5-18 years of age * Diagnosis of Hb SS or HB0Thal * Weight \>15 kg * \<50th percentile for height/age or weight/age or weight/ height, or \<90% ideal body weight, or \<90% of BMI for age/ gender. * Is willing to sign informed consent
Exclusion criteria
* Patients receiving hydroxyurea or any other anti-sickling agent, chronic transfusion, or nutrition supplements.A nutrition supplement is any high calorie or high protein food additive or oral supplement being used for the purpose of weight gain. * History of poor compliance (Missing two or more clinic appointments in the past year). * Renal or liver dysfunction * Renal dysfunction as defined by serum creatinine \>1.5 times normal for age based on testing lab. * Hepatic dysfunction as defined by alanine aminotransferase (ALT) \>2 times the upper limit of normal for age based on testing lab. * Breastfeeding * Pregnancy.Females of childbearing potential must have negative serum or urine pregnancy test (record date of test). * Patients enrolled on previous glutamine protocol (SCDGLU). * Patients who are considered high risk
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in Resting Energy Expenditure From Baseline to 12 Months | Baseline and 12 months | To compare the effect of glutamine and placebo on resting energy expenditure (REE) in children with sickle cell anemia (SCA) by comparing the change in REE ratio between baseline and 12 months. REE was measured by indirect calorimetry, using a metabolic cart.REE Ratio =(REE Measured/REE Predicted)x 100).Change was defined as 12 Month REE Ratio minus Baseline REE Ratio.The REE Ratio was evaluated at baseline and 12 months.The REE Ratio is calculated as (REE Measured / REE Predicted) x 100).REE units are measured as (Kcal / day).Change was defined as 12 Month REE Ratio minus Baseline REE Ratio. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in Red Blood Cell Glutamine From Baseline to 12 Months | Baseline and 12 months | To investigate the effect of oral glutamine and placebo in children with Sickle Cell Anemia (SCA) by comparing the difference in the levels of red blood cell glutamine between baseline and 12 months of treatment in the two groups. |
| Change in Quality of Life Measures From Baseline to 12 Months.Scores for Each Subcategory Range From 0 (Best) to 4 (Worst).This is True for Both Patient and Parent Reports. | Baseline and 12 Months | Evaluation of quality of life at baseline and 12 months in the glutamine versus placebo group using the PedsQL Version 4.0 inventory. This instrument measures individual well being across physical, emotional, social, and school function categories using patient self-reports and/or parent reports. The tool contains a 15-question, age-specific, self-report inventory (for children age 5-7 years, 8-12 years, and 13-18 years) and a corresponding parent inventory. Lower scores indicate a better quality of life. |
| Change in Height Z-score From Baseline to 12 Months | Baseline and 12 months | To investigate the effect of oral glutamine and placebo on height Z-score in children with Sickle Cell Anemia (SCA) between baseline and 12 months on treatment. |
| Change in Body Mass Index From Baseline to 12 Months | Baseline and 12 months | To investigate the effect of oral glutamine and placebo on body composition in children with SCA by comparing the difference in body mass indexes (BMI) between baseline and 12 months of treatment in the two groups. |
| Change in Weight Percentile From Baseline to 12 Months | Basline and 12 months | To investigate the effect of oral glutamine and placebo on weight in children with Sickle Cell Anemia (SCA) between baseline and 12 months on treatment. |
| Change in Pulse Rate From Baseline to 12 Months | Baseline and 12 months | To investigate the clinical effects of oral glutamine and placebo on pulse rate in children with Sickle Cell Anemia (SCA) by comparing the difference between baseline and 12 months of treatment between the two groups. |
| Change in Hand Grip From Baseline to 12 Months. | Baseline and 12 months | To investigate the clinical effects of oral glutamine and placebo on hand grip in children with Sickle Cell Anemia (SCA) by comparing the difference between baseline and 12 months of treatment between the two groups. Hand grip strength is a measure of muscle strength.Units are measured in Kg.Muscle strength is measured using a hydraulic hand-held dynamometer.Change was defined as 12 Month measure minus baseline.Muscle strength is measured using the hand grip strength via a hydraulic hand-held dynamometer (Kg). |
| Change in Height Percentile From Baseline to 12 Months | Baseline and 12 months | To investigate the effect of oral glutamine and placebo on height percentile in children with Sickle Cell Anemia (SCA) between baseline and 12 months on treatment. |
Countries
United States
Participant flow
Recruitment details
27 participants were recruited from July, 2004 through October, 2007.
Pre-assignment details
25 of the 27 participants were randomized to either the Glutamine or Placebo arm.
Participants by arm
| Arm | Count |
|---|---|
| Placebo A mixture of non-essential amino acids containing glycine, alanine, and serine. | 12 |
| Glutamine Glutamine dosage of 0.60 gm/kg body weight per day divided into two doses. | 13 |
| Total | 25 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Disease progression | 0 | 2 |
| Overall Study | Non-Compliance | 2 | 4 |
| Overall Study | Withdrawal by Subject | 1 | 2 |
Baseline characteristics
| Characteristic | Placebo | Glutamine | Total |
|---|---|---|---|
| Age, Continuous | 9.1 years STANDARD_DEVIATION 2.9 | 9.1 years STANDARD_DEVIATION 3.6 | 9.1 years STANDARD_DEVIATION 3.2 |
| Sex: Female, Male Female | 6 Participants | 9 Participants | 15 Participants |
| Sex: Female, Male Male | 6 Participants | 4 Participants | 10 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 0 / 12 | 0 / 13 |
| serious Total, serious adverse events | 0 / 12 | 0 / 13 |
Outcome results
Primary
Change in Resting Energy Expenditure From Baseline to 12 Months
To compare the effect of glutamine and placebo on resting energy expenditure (REE) in children with sickle cell anemia (SCA) by comparing the change in REE ratio between baseline and 12 months. REE was measured by indirect calorimetry, using a metabolic cart.REE Ratio =(REE Measured/REE Predicted)x 100).Change was defined as 12 Month REE Ratio minus Baseline REE Ratio.The REE Ratio was evaluated at baseline and 12 months.The REE Ratio is calculated as (REE Measured / REE Predicted) x 100).REE units are measured as (Kcal / day).Change was defined as 12 Month REE Ratio minus Baseline REE Ratio.
Time frame: Baseline and 12 months
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Placebo | Change in Resting Energy Expenditure From Baseline to 12 Months | -5.5 REE ratio |
| Glutamine | Change in Resting Energy Expenditure From Baseline to 12 Months | 7.1 REE ratio |
Comparison: We tested for a significant difference in the median change in REE ratio between the placebo and glutamine groups. The study was designed to provide 80% power at an alpha level of 0.05 for this objective. Due to slow accrual, the sample size of 46 participants required to obtain the designed power of the study was not realized.p-value: 0.17Wilcoxon (Mann-Whitney)
Secondary
Change in Body Mass Index From Baseline to 12 Months
To investigate the effect of oral glutamine and placebo on body composition in children with SCA by comparing the difference in body mass indexes (BMI) between baseline and 12 months of treatment in the two groups.
Time frame: Baseline and 12 months
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Placebo | Change in Body Mass Index From Baseline to 12 Months | 0.1 kg/m2 |
| Glutamine | Change in Body Mass Index From Baseline to 12 Months | 0.8 kg/m2 |
Comparison: We tested for a significant difference in the median change of body mass index between the placebo and glutamine groups.p-value: 0.53Wilcoxon (Mann-Whitney)
Secondary
Change in Hand Grip From Baseline to 12 Months.
To investigate the clinical effects of oral glutamine and placebo on hand grip in children with Sickle Cell Anemia (SCA) by comparing the difference between baseline and 12 months of treatment between the two groups. Hand grip strength is a measure of muscle strength.Units are measured in Kg.Muscle strength is measured using a hydraulic hand-held dynamometer.Change was defined as 12 Month measure minus baseline.Muscle strength is measured using the hand grip strength via a hydraulic hand-held dynamometer (Kg).
Time frame: Baseline and 12 months
Population: Analyzed patients had a height measurement at both baseline and 12 months.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Placebo | Change in Hand Grip From Baseline to 12 Months. | 12 kg |
| Glutamine | Change in Hand Grip From Baseline to 12 Months. | 5 kg |
Comparison: We tested for a significant difference in the median change in hand grip between the placebo and glutamine groups.p-value: 0.4Wilcoxon (Mann-Whitney)
Secondary
Change in Height Percentile From Baseline to 12 Months
To investigate the effect of oral glutamine and placebo on height percentile in children with Sickle Cell Anemia (SCA) between baseline and 12 months on treatment.
Time frame: Baseline and 12 months
Population: Analyzed patients had a height measurement at both baseline and 12 months.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Placebo | Change in Height Percentile From Baseline to 12 Months | 0.0 Percentile |
| Glutamine | Change in Height Percentile From Baseline to 12 Months | 0.0 Percentile |
Comparison: We tested the null hypothesis that the median difference in height percentile between baseline and 12 months is equal to zero versus the alternative hypothesis that the difference is not zero.p-value: 0.69Wilcoxon Signed Rank Test
Comparison: We tested the null hypothesis that the median difference in height percentile between baseline and 12 months is equal to zero versus the alternative hypothesis that the difference is not zero.p-value: 0.75Wilcoxon Signed Rank Test
Comparison: We test the null hypothesis that the median difference in height percentile between baseline and 12 months differs between the Glutamine and Placebo groups.p-value: 0.93Wilcoxon (Mann-Whitney)
Secondary
Change in Height Z-score From Baseline to 12 Months
To investigate the effect of oral glutamine and placebo on height Z-score in children with Sickle Cell Anemia (SCA) between baseline and 12 months on treatment.
Time frame: Baseline and 12 months
Population: Analyzed patients had a height measurement at both baseline and 12 months.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Placebo | Change in Height Z-score From Baseline to 12 Months | -0.1 Z-score |
| Glutamine | Change in Height Z-score From Baseline to 12 Months | 0.0 Z-score |
Comparison: We tested the null hypothesis that the median difference in height Z-Score betweeen baseline and 12 months is equal to zero versus the alternative hypothesis that the difference is not zero.p-value: 0.2Wilcoxon Signed Rank Test
Comparison: We tested the null hypothesis that the median difference in height Z-Score betweeen baseline and 12 months is equal to zero versus the alternative hypothesis that the difference is not zero.p-value: 0.81Wilcoxon Signed Rank Test
Comparison: We tested the null hypothesis that the median difference in height Z-Score betweeen baseline and 12 months differs between the Glutamine and Placebo groups.p-value: 0.7Wilcoxon (Mann-Whitney)
Secondary
Change in Pulse Rate From Baseline to 12 Months
To investigate the clinical effects of oral glutamine and placebo on pulse rate in children with Sickle Cell Anemia (SCA) by comparing the difference between baseline and 12 months of treatment between the two groups.
Time frame: Baseline and 12 months
Population: Analyzed patients had a height measurement at both baseline and 12 months.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Placebo | Change in Pulse Rate From Baseline to 12 Months | 11 Beats per minute (BPM) |
| Glutamine | Change in Pulse Rate From Baseline to 12 Months | 17 Beats per minute (BPM) |
Comparison: We tested for a significant difference in the median change in pulse rate between the placebo and glutamine groups.p-value: 0.83Wilcoxon (Mann-Whitney)
Secondary
Change in Quality of Life Measures From Baseline to 12 Months.Scores for Each Subcategory Range From 0 (Best) to 4 (Worst).This is True for Both Patient and Parent Reports.
Evaluation of quality of life at baseline and 12 months in the glutamine versus placebo group using the PedsQL Version 4.0 inventory. This instrument measures individual well being across physical, emotional, social, and school function categories using patient self-reports and/or parent reports. The tool contains a 15-question, age-specific, self-report inventory (for children age 5-7 years, 8-12 years, and 13-18 years) and a corresponding parent inventory. Lower scores indicate a better quality of life.
Time frame: Baseline and 12 Months
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Placebo | Change in Quality of Life Measures From Baseline to 12 Months.Scores for Each Subcategory Range From 0 (Best) to 4 (Worst).This is True for Both Patient and Parent Reports. | 12 Months Parent Reported Physical Function | 2.5 Units on a scale |
| Placebo | Change in Quality of Life Measures From Baseline to 12 Months.Scores for Each Subcategory Range From 0 (Best) to 4 (Worst).This is True for Both Patient and Parent Reports. | 12 Months Participant Reported Physical Function | 1.0 Units on a scale |
| Placebo | Change in Quality of Life Measures From Baseline to 12 Months.Scores for Each Subcategory Range From 0 (Best) to 4 (Worst).This is True for Both Patient and Parent Reports. | Baseline Participant Reported Physical Function | 0.9 Units on a scale |
| Placebo | Change in Quality of Life Measures From Baseline to 12 Months.Scores for Each Subcategory Range From 0 (Best) to 4 (Worst).This is True for Both Patient and Parent Reports. | 12 Months Participant Reported Emotional Function | 0.8 Units on a scale |
| Placebo | Change in Quality of Life Measures From Baseline to 12 Months.Scores for Each Subcategory Range From 0 (Best) to 4 (Worst).This is True for Both Patient and Parent Reports. | 12 Months Parent Reported Social Function | 2.0 Units on a scale |
| Placebo | Change in Quality of Life Measures From Baseline to 12 Months.Scores for Each Subcategory Range From 0 (Best) to 4 (Worst).This is True for Both Patient and Parent Reports. | 12 Months Participant Reported Social Function | 1.2 Units on a scale |
| Placebo | Change in Quality of Life Measures From Baseline to 12 Months.Scores for Each Subcategory Range From 0 (Best) to 4 (Worst).This is True for Both Patient and Parent Reports. | Baseline Participant Reported Emotional Function | 0.8 Units on a scale |
| Placebo | Change in Quality of Life Measures From Baseline to 12 Months.Scores for Each Subcategory Range From 0 (Best) to 4 (Worst).This is True for Both Patient and Parent Reports. | 12 Months Participant Reported School Function | 1.6 Units on a scale |
| Placebo | Change in Quality of Life Measures From Baseline to 12 Months.Scores for Each Subcategory Range From 0 (Best) to 4 (Worst).This is True for Both Patient and Parent Reports. | 12 Months Parent Reported Emotional Function | 1.4 Units on a scale |
| Placebo | Change in Quality of Life Measures From Baseline to 12 Months.Scores for Each Subcategory Range From 0 (Best) to 4 (Worst).This is True for Both Patient and Parent Reports. | Baseline Parent Reported Physical Function | 1.4 Units on a scale |
| Placebo | Change in Quality of Life Measures From Baseline to 12 Months.Scores for Each Subcategory Range From 0 (Best) to 4 (Worst).This is True for Both Patient and Parent Reports. | Baseline Participant Reported Social Function | 1.0 Units on a scale |
| Placebo | Change in Quality of Life Measures From Baseline to 12 Months.Scores for Each Subcategory Range From 0 (Best) to 4 (Worst).This is True for Both Patient and Parent Reports. | Baseline Parent Reported Emotional Function | 1.0 Units on a scale |
| Placebo | Change in Quality of Life Measures From Baseline to 12 Months.Scores for Each Subcategory Range From 0 (Best) to 4 (Worst).This is True for Both Patient and Parent Reports. | 12 Months Parent Reported School Function | 1.8 Units on a scale |
| Placebo | Change in Quality of Life Measures From Baseline to 12 Months.Scores for Each Subcategory Range From 0 (Best) to 4 (Worst).This is True for Both Patient and Parent Reports. | Baseline Parent Reported Social Function | 0.6 Units on a scale |
| Placebo | Change in Quality of Life Measures From Baseline to 12 Months.Scores for Each Subcategory Range From 0 (Best) to 4 (Worst).This is True for Both Patient and Parent Reports. | Baseline Participant Reported School Function | 1.6 Units on a scale |
| Placebo | Change in Quality of Life Measures From Baseline to 12 Months.Scores for Each Subcategory Range From 0 (Best) to 4 (Worst).This is True for Both Patient and Parent Reports. | Baseline Parent Reported School Function | 1.8 Units on a scale |
| Glutamine | Change in Quality of Life Measures From Baseline to 12 Months.Scores for Each Subcategory Range From 0 (Best) to 4 (Worst).This is True for Both Patient and Parent Reports. | Baseline Participant Reported School Function | 1.3 Units on a scale |
| Glutamine | Change in Quality of Life Measures From Baseline to 12 Months.Scores for Each Subcategory Range From 0 (Best) to 4 (Worst).This is True for Both Patient and Parent Reports. | 12 Months Parent Reported Physical Function | 2.1 Units on a scale |
| Glutamine | Change in Quality of Life Measures From Baseline to 12 Months.Scores for Each Subcategory Range From 0 (Best) to 4 (Worst).This is True for Both Patient and Parent Reports. | 12 Months Parent Reported Emotional Function | 1.2 Units on a scale |
| Glutamine | Change in Quality of Life Measures From Baseline to 12 Months.Scores for Each Subcategory Range From 0 (Best) to 4 (Worst).This is True for Both Patient and Parent Reports. | 12 Months Parent Reported Social Function | 1.2 Units on a scale |
| Glutamine | Change in Quality of Life Measures From Baseline to 12 Months.Scores for Each Subcategory Range From 0 (Best) to 4 (Worst).This is True for Both Patient and Parent Reports. | 12 Months Parent Reported School Function | 2.4 Units on a scale |
| Glutamine | Change in Quality of Life Measures From Baseline to 12 Months.Scores for Each Subcategory Range From 0 (Best) to 4 (Worst).This is True for Both Patient and Parent Reports. | Baseline Participant Reported Physical Function | 1.0 Units on a scale |
| Glutamine | Change in Quality of Life Measures From Baseline to 12 Months.Scores for Each Subcategory Range From 0 (Best) to 4 (Worst).This is True for Both Patient and Parent Reports. | Baseline Participant Reported Emotional Function | 1.2 Units on a scale |
| Glutamine | Change in Quality of Life Measures From Baseline to 12 Months.Scores for Each Subcategory Range From 0 (Best) to 4 (Worst).This is True for Both Patient and Parent Reports. | Baseline Participant Reported Social Function | 1.2 Units on a scale |
| Glutamine | Change in Quality of Life Measures From Baseline to 12 Months.Scores for Each Subcategory Range From 0 (Best) to 4 (Worst).This is True for Both Patient and Parent Reports. | Baseline Parent Reported School Function | 2.0 Units on a scale |
| Glutamine | Change in Quality of Life Measures From Baseline to 12 Months.Scores for Each Subcategory Range From 0 (Best) to 4 (Worst).This is True for Both Patient and Parent Reports. | 12 Months Participant Reported Physical Function | 1.0 Units on a scale |
| Glutamine | Change in Quality of Life Measures From Baseline to 12 Months.Scores for Each Subcategory Range From 0 (Best) to 4 (Worst).This is True for Both Patient and Parent Reports. | 12 Months Participant Reported Emotional Function | 1.2 Units on a scale |
| Glutamine | Change in Quality of Life Measures From Baseline to 12 Months.Scores for Each Subcategory Range From 0 (Best) to 4 (Worst).This is True for Both Patient and Parent Reports. | 12 Months Participant Reported Social Function | 1.0 Units on a scale |
| Glutamine | Change in Quality of Life Measures From Baseline to 12 Months.Scores for Each Subcategory Range From 0 (Best) to 4 (Worst).This is True for Both Patient and Parent Reports. | 12 Months Participant Reported School Function | 1.8 Units on a scale |
| Glutamine | Change in Quality of Life Measures From Baseline to 12 Months.Scores for Each Subcategory Range From 0 (Best) to 4 (Worst).This is True for Both Patient and Parent Reports. | Baseline Parent Reported Physical Function | 2.0 Units on a scale |
| Glutamine | Change in Quality of Life Measures From Baseline to 12 Months.Scores for Each Subcategory Range From 0 (Best) to 4 (Worst).This is True for Both Patient and Parent Reports. | Baseline Parent Reported Emotional Function | 2.0 Units on a scale |
| Glutamine | Change in Quality of Life Measures From Baseline to 12 Months.Scores for Each Subcategory Range From 0 (Best) to 4 (Worst).This is True for Both Patient and Parent Reports. | Baseline Parent Reported Social Function | 1.8 Units on a scale |
Comparison: We tested for a significant difference in the median change between baseline parent reported physical function in the placebo and glutamine groups.p-value: 0.62Wilcoxon (Mann-Whitney)
Comparison: We tested for a significant difference in the median change between baseline parent reported emotional function in the placebo and glutamine groups.p-value: 0.14Wilcoxon (Mann-Whitney)
Comparison: We tested for a significant difference in the median change between baseline parent reported social function in the placebo and glutamine groups.p-value: 0.2Wilcoxon (Mann-Whitney)
Comparison: We tested for a significant difference in the median change between baseline parent reported school function in the placebo and glutamine groups.p-value: 0.62Wilcoxon (Mann-Whitney)
Comparison: We tested for a significant difference in the median change between 12 month parent reported physical function in the placebo and glutamine groups.p-value: 0.61Wilcoxon (Mann-Whitney)
Comparison: We tested for a significant difference in the median change between 12 month parent reported emotional function in the placebo and glutamine groups.p-value: 0.65Wilcoxon (Mann-Whitney)
Comparison: We tested for a significant difference in the median change between 12 month parent reported social function in the placebo and glutamine groups.p-value: 0.55Wilcoxon (Mann-Whitney)
Comparison: We tested for a significant difference in the median change between 12 month parent reported school function in the placebo and glutamine groups.p-value: 0.69Wilcoxon (Mann-Whitney)
Comparison: We tested for a significant difference in the median change between baseline patient reported physical function in the placebo and glutamine groups.p-value: 0.82Wilcoxon (Mann-Whitney)
Comparison: We tested for a significant difference in the median change between baseline patient reported emotional function in the placebo and glutamine groups.p-value: 0.99Wilcoxon (Mann-Whitney)
Comparison: We tested for a significant difference in the median change between baseline patient reported social function in the placebo and glutamine groups.p-value: 0.3Wilcoxon (Mann-Whitney)
Comparison: We tested for a significant difference in the median change between baseline patient reported school function in the placebo and glutamine groups.p-value: 0.24Wilcoxon (Mann-Whitney)
Comparison: We tested for a significant difference in the median change between 12 month patient reported physical function in the placebo and glutamine groups.p-value: 0.5Wilcoxon (Mann-Whitney)
Comparison: We tested for a significant difference in the median change between 12 month patient reported emotional function in the placebo and glutamine groups.p-value: 0.45Wilcoxon (Mann-Whitney)
Comparison: We tested for a significant difference in the median change between 12 month patient reported social function in the placebo and glutamine groups.p-value: 0.46Wilcoxon (Mann-Whitney)
Comparison: We tested for a significant difference in the median change between 12 month patient reported school function in the placebo and glutamine groups.p-value: 0.84Wilcoxon (Mann-Whitney)
Secondary
Change in Red Blood Cell Glutamine From Baseline to 12 Months
To investigate the effect of oral glutamine and placebo in children with Sickle Cell Anemia (SCA) by comparing the difference in the levels of red blood cell glutamine between baseline and 12 months of treatment in the two groups.
Time frame: Baseline and 12 months
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Placebo | Change in Red Blood Cell Glutamine From Baseline to 12 Months | 43.5 nmol/mg creatinine |
| Glutamine | Change in Red Blood Cell Glutamine From Baseline to 12 Months | 100.8 nmol/mg creatinine |
Comparison: We tested for a significant difference in the median change in red blood cell glutamine between the placebo and glutamine groups.p-value: 0.24Wilcoxon (Mann-Whitney)
Secondary
Change in Weight Percentile From Baseline to 12 Months
To investigate the effect of oral glutamine and placebo on weight in children with Sickle Cell Anemia (SCA) between baseline and 12 months on treatment.
Time frame: Basline and 12 months
Population: Analyzed patients had a height measurement at both baseline and 12 months.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Placebo | Change in Weight Percentile From Baseline to 12 Months | 0.0 Percentile |
| Glutamine | Change in Weight Percentile From Baseline to 12 Months | 7.5 Percentile |
Comparison: We tested the null hypothesis that the median difference in weight percentile between baseline and 12 months is equal to zero versus the alternative hypothesis that the difference is not zero.p-value: 0.56Wilcoxon Signed Rank Test
Comparison: We tested the null hypothesis that the median difference in weight percentile between baseline and 12 months is equal to zero versus the alternative hypothesis that the difference is not zero.p-value: 1Wilcoxon Signed Rank Test
Comparison: We test the null hypothesis that the median difference in weight percentile between baseline and 12 months differs between the Glutamine and Placebo groups.p-value: 0.61Wilcoxon (Mann-Whitney)