Graft Vs Host Disease, Vitamin A Deficiency, Vitamin D Deficiency
Conditions
Brief summary
The investigators hypothesize that single oral high dose supplementation with vitamin A will reduce the incidence of moderate-severe chronic graft-versus-host disease (GVHD) compared with placebo.
Detailed description
Hematopoietic stem cell transplantation (HSCT) is an effective treatment strategy for many malignancies, marrow failure syndromes, and immune deficiencies in children, adolescents, and adults. Vitamin A and its derivatives regulate growth and differentiation of intestinal cells, and vitamin A deficiency is associated with increased susceptibility to infection in both human and animal models. The investigators' preliminary data suggest lower vitamin A levels were associated with an increased incidence of gastrointestinal graft versus host disease (GI GVHD) in patients undergoing HSCT. This study is a randomized double blinded comparison of vitamin A supplementation comparing a single large dose of vitamin A with a placebo.
Interventions
DRUGVitamin A
Enrolled subjects will receive one observed oral vitamin A dose, prior to their HSCT, in the outpatient clinic or inpatient Bone Marrow Transplant (BMT) floor.
OTHERPlacebo
Placebo pills containing microcrystalline cellulose will be dispensed in patients who are randomized to the placebo arm.
Sponsors
Children's Hospital Medical Center, Cincinnati
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Be 18 years of age or older * Be scheduled for allogeneic stem cell transplant. * Have a vitamin A level \< upper limit of normal for age. * Be able to tolerate enteral vitamin dose administration. * Have a total bilirubin level \< 1.5x ULN and an AST and/or ALT\< 3xULN for age * Receiving PBSCs as stem cell graft
Exclusion criteria
* Ongoing raised intracranial pressure (see appendix D for definition of raised intracranial pressure) * Liver cirrhosis (see appendix C for definition of liver cirrhosis) * Patients will be excluded if they are currently pregnant. * Patients will be excluded if they have a fish (not shellfish) allergy.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of moderate-severe chronic graft versus host disease (GVHD) | 1 year after transplant | Incidence of moderate-severe chronic GVHD |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of acute gastrointestinal graft versus host disease (GI GVHD) | 2 years after transplant | Incidence of acute GI GVHD |
| Incidence of relapse | 2 years after transplant | Incidence of relapse |
| Overall survival. | 2 years after transplant | Overall survival. |
Countries
United States
Contacts
CONTACTCeleste Dourson, MS, CCRP
PRINCIPAL_INVESTIGATORPooja Khandelwal, MD
Children's Hospital Medical Center, Cincinnati
Outcome results
None listed