Allergies, Food Protein-Induced Enterocolitis Syndrome, Pediatric Disorder
Conditions
Keywords
Safe foods, Trigger foods, Vomiting
Brief summary
The aim of this study is to validate a blood test that can identify safe foods for food protein-induced enterocolitis syndrome (FPIES). This study proposes a solution to the problems of FPIES by developing a new blood assay that screens a large number of foods (more than 20) in a culture plate. If this blood test is successful it may be able to identify safe foods more quickly. The study will recruit 10 participants that will have more than 2 trigger foods.
Detailed description
Participants will complete surveys and have blood draws during the study. Additionally, participants will be asked to keep track of their diet as well as introduce safe foods identified by the researchers.
Interventions
DIAGNOSTIC_TESTBlood test assay
Participants will have their blood drawn and be evaluated with a new blood assay that screens a large number of foods (more than 20) in a culture plate. Participants will be asked to eat the identified safe foods by the the blood assay.
Sponsors
University of Michigan
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
DIAGNOSTIC
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
1 Months to 7 Years
Healthy volunteers
No
Inclusion criteria
* Diagnosis of FPIES * Have to have documented reactions to 2-3 trigger foods with recurrent delayed vomiting or documented reactions to 4 or more trigger foods with recurrent delayed vomiting.
Exclusion criteria
* Patients who are currently on medications that suppress the immune system * Patients who do not have at least 2 trigger foods identified. * Patients who have a history of an organic Gastrointestinal (GI) disease (e.g., inflammatory bowel disease, celiac disease, biliary disorders, bowel resection), cardiac, pulmonary, neurologic, renal, endocrine, or gynecological pathology * Lack of parental or guardian informed consent.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Negative Predictive Value (NPV), Defined as the Percentage of Test-predicted Safe Foods That Are Actually Safe Foods. | Up to 7 weeks from the first blood trial, on average 11 weeks | A Receiver operating characteristic (ROC) curve was built to estimate the NPV. A random-effects logit model was used to model the binary outcome (safe or trigger food) as a function of the 9 biomarker measurements in the assay. (Expression Value = Relative fold change of 9-gene expression panel in response to food treatment, divided by fold change in response to LPS treatment, multiplied by 1000). The random effect in the logit model took into consideration the correlated data measured within the same subject. A cluster ROC curve analysis was used to assess the precision of the assay. Specifically, the area was computed under the cluster ROC curve (AUC). A threshold to obtain the NPV was selected based on inspection of the ROC curve. At the initial visit, participants had their blood drawn, which was assayed. On average, participants came in up to 4 weeks later after the test results were ready. Participants were then asked to trial a new food each week for up to 7 weeks. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Positive Predictive Value (PPV), Defined as the Percentage of Test-predicted Unsafe Foods That Are Actually Unsafe Foods. | Up to 7 weeks from the first blood trial, on average 11 weeks | The same ROC curve described for the primary outcome was used to derive the PPV. At the initial visit, participants had their blood drawn, which was assayed. On average, participants came in up to 4 weeks later after the test results were ready. Participants were then asked to trial a new food each week for up to 7 weeks. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Blood Test With Assays 10 participants with FPIES exhibiting reactions to more than 2 foods were recruited.
Blood test assay: Participants had their blood drawn and evaluated with a new blood assay that screened a large number of foods (more than 20) in a culture plate. Participants were asked to eat the foods identified as safe by the the blood assay. | 10 |
| Total | 10 |
Baseline characteristics
| Characteristic | Blood Test With Assays |
|---|---|
| Age, Continuous | 0.75 years |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 10 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 1 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 9 Participants |
| Region of Enrollment United States | 10 Participants |
| Sex: Female, Male Female | 6 Participants |
| Sex: Female, Male Male | 4 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 10 |
| other Total, other adverse events | 0 / 10 |
| serious Total, serious adverse events | 0 / 10 |
Outcome results
Primary
Negative Predictive Value (NPV), Defined as the Percentage of Test-predicted Safe Foods That Are Actually Safe Foods.
A Receiver operating characteristic (ROC) curve was built to estimate the NPV. A random-effects logit model was used to model the binary outcome (safe or trigger food) as a function of the 9 biomarker measurements in the assay. (Expression Value = Relative fold change of 9-gene expression panel in response to food treatment, divided by fold change in response to LPS treatment, multiplied by 1000). The random effect in the logit model took into consideration the correlated data measured within the same subject. A cluster ROC curve analysis was used to assess the precision of the assay. Specifically, the area was computed under the cluster ROC curve (AUC). A threshold to obtain the NPV was selected based on inspection of the ROC curve. At the initial visit, participants had their blood drawn, which was assayed. On average, participants came in up to 4 weeks later after the test results were ready. Participants were then asked to trial a new food each week for up to 7 weeks.
Time frame: Up to 7 weeks from the first blood trial, on average 11 weeks
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Blood Test With Assays | Negative Predictive Value (NPV), Defined as the Percentage of Test-predicted Safe Foods That Are Actually Safe Foods. | NPV <29.24 Threshold | 98.5 percentage |
| Blood Test With Assays | Negative Predictive Value (NPV), Defined as the Percentage of Test-predicted Safe Foods That Are Actually Safe Foods. | Area Under the ROC Curve | 73.16 percentage |
p-value: <0.0001Regression, non-linear mixed effects
Secondary
Positive Predictive Value (PPV), Defined as the Percentage of Test-predicted Unsafe Foods That Are Actually Unsafe Foods.
The same ROC curve described for the primary outcome was used to derive the PPV. At the initial visit, participants had their blood drawn, which was assayed. On average, participants came in up to 4 weeks later after the test results were ready. Participants were then asked to trial a new food each week for up to 7 weeks.
Time frame: Up to 7 weeks from the first blood trial, on average 11 weeks
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Blood Test With Assays | Positive Predictive Value (PPV), Defined as the Percentage of Test-predicted Unsafe Foods That Are Actually Unsafe Foods. | 31.1 percentage |
p-value: <0.0001Regression, non-linear mixed effects