Vitamin A Replacement in Patients Undergoing HSCT and Its Role on MBI-LCBI Rates

Study Overview
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Brief Summary
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The primary objective of this study is to establish that single dose vitamin A supplementation is feasible and safe in pediatric and young adult bone marrow transplant recipients until day +30 (± 7 days) after hematopoietic stem cell transplantation.

Detailed Description
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The investigators' preliminary data suggest that low levels of vitamin A directly impact risk of mucosal barrier injury laboratory-confirmed bloodstream infection (MBI-LCBI), likely via decreased gut permeability, and they hypothesize supplemental vitamin A at the time of HSCT can reduce the risk of MBI-LCBI and gastrointestinal graft versus host disease (GI GVHD). A 3x3 dose escalation/de-escalation study design will be used to determine the safety and dosing required to maintain vitamin A levels in the upper quartile of normal range for age at day +30 (± 7 days) with single dose vitamin A supplementation prior to hematopoietic stem cell transplantation.

Official Title
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Single, High Dose Vitamin A Replacement in Patients Undergoing Hematopoietic Stem Cell Transplantation and Its Role on MBI-LCBI Rates

Conditions
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Hematopoietic Stem Cell Transplant

Intervention / Treatment
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* Dietary Supplement: Vitamin A


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Any patient undergoing HSCT. Exclusion Criteria: Vitamin A hypersensitivity or allergy. Age less than 1 year at time of transplant. Baseline pre-HSCT vitamin A levels higher than the upper quartile of normal range for age. Enteral feeding or medication intolerance. Pregnancy.

Ages Eligible for Study
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Minimum Age: 1 Year

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Other: Vitamin A<br>Participants receive single dose vitamin A supplementation prior to HSCT. A 3x3 dose escalation/de-escalation design will be used for this study. | Dietary Supplement: Vitamin A<br>* Dose administration will start with 2500 IU/kg with maximum dose of 250,000 IU orally.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Vitamin A Level | Vitamin A level will be measured 30 days after HSCT. | 30 days after HSCT | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of mucosal barrier injury laboratory-confirmed bloodstream infection (MBI-LCBI) | Incidence of mucosal barrier injury laboratory-confirmed bloodstream infection (MBI-LCBI) 100 days after HSCT. | 100 days after HSCT | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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hematopoietic stem cell transplant, vitamin a