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2023-03-20T14:54:31.000Z

Visual abstract | Vitamin A for the prevention of GvHD in pediatric patients undergoing allo-HSCT

Mar 20, 2023
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Learning objective: After reading this article, learners will be able to cite a new clinical development in pediatric GvHD.

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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
(n = 40)

Placebo
(n = 40)

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.
*Adapted from Khandelwal.1

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


Expert Opinion

We asked, Do you think this result will translate into adult patients?

“We plan to open another study of oral vitamin A in adult BMT patients to see if our findings translate to patients of all ages.”

Visual abstract

To download this visual abstract, click below.

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  1. Khandelwal P. Vitamin A prevents acute and chronic graft versus host disease in children and young adults: A single-center randomized double-blinded placebo-controlled clinical trial. Oral abstract #117. 2023 Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR; Feb 16, 2023; Orlando, US.

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