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The GvHD Hub is happy to present a visual abstract summarizing key findings from a prospective, double-blind trial evaluating the efficacy of high-dose vitamin A for graft-versus-host disease (GvHD) in pediatric patients undergoing an allogeneic hematopoietic stem cell transplant, as presented during the 2023 Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR.
Baseline patient characteristics are shown in Table 1. Despite a higher proportion of patients in the vitamin A cohort having characteristics that predispose them to GvHD (HLA mismatching, donor relation, and the use of unmanipulated peripheral blood stem cells), patients in the vitamin A cohort had a lower incidence of GvHD.1
Table 1. Baseline patient characteristics*
Characteristic, n (unless otherwise stated) |
Vitamin A |
Placebo |
p value |
---|---|---|---|
Median age (range), years |
6.9 (2–20) |
8.5 (1.1–28) |
0.2 |
Underlying diagnosis |
|
|
0.6 |
Malignancy |
8 |
9 |
|
Primary immune deficiency |
8 |
6 |
|
Bone marrow failure |
14 |
17 |
|
Hemoglobinopathy |
8 |
8 |
|
Metabolic disorder |
2 |
0 |
|
Conditioning regimen |
|
|
0.8 |
Myeloablative |
25 |
27 |
|
Reduced intensity |
15 |
13 |
|
Serotherapy |
24 |
28 |
0.4 |
Radiation in conditioning |
4 |
2 |
0.6 |
HLA match |
|
|
0.1 |
≥2 antigen mismatch |
5 |
2 |
|
9/10 |
11 |
6 |
|
10/10 |
24 |
32 |
|
Donor relation |
|
|
0.1 |
Related (excluding ≥2 antigen mismatch) |
8 |
15 |
|
Related (≥2 antigen mismatch) |
4 |
1 |
|
Unrelated |
28 |
24 |
|
GvHD prophylaxis |
|
|
0.4 |
Calcineurin inhibitor + MMF |
9 |
5 |
|
Calcineurin inhibitor + MTX |
1 |
5 |
|
Calcineurin inhibitor + prednisone |
2 |
2 |
|
T-cell depletion |
12 |
10 |
|
αβ CD19 TCR depletion |
0 |
2 |
|
Abatacept to standard prophylaxis |
15 |
14 |
|
PTCy |
1 |
1 |
|
GvHD, graft-versus-host disease; HLA, human leukocyte antigen; MMF, mycophenolate mofetil; MTX, methotrexate; PTCy, post-transplant cyclophosphamide; TCR, T-cell receptor. |
The use of high-dose vitamin A is already recommended by the World Health Organization (WHO) for treatment of children with acute measles.1 Vitamin A is involved in gut homeostasis by maintaining barrier function and expression of junction proteins in the intestinal epithelium. The level of CCR9+ CD8+ effector memory T-cells were significantly higher in the placebo arm at Day 30 posttransplant compared with the vitamin A arm; therefore, this shows that gut T-cell trafficking was reduced in patients treated with vitamin A (p = 0.01).1
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