From the Guidelines
The risk of Graft-Versus-Host Disease (GVHD) in pediatric patients undergoing Stem Cell Transplant (SCT) is significant, with an incidence of acute GVHD (grade 2-4) of 12.6% and chronic GVHD of 14.6% in patients ≤ 16 years of age, as reported in the most recent guidelines from 2021 1.
GVHD Risk Factors
The risk of GVHD is a major concern in pediatric stem cell transplantation, and several factors contribute to this risk, including:
- Age: Patients > 16 years of age have a higher incidence of acute and chronic GVHD, with rates of 16% and 23%, respectively 1.
- Donor type: HLA-identical sibling donors have a lower risk of GVHD compared to alternative donors, such as HLA-mismatched or unrelated donors.
- Conditioning regimen: Myeloablative conditioning regimens have a higher risk of GVHD compared to less intense conditioning regimens.
GVHD Prevention and Treatment
Prevention and treatment of GVHD are crucial to minimize its impact on transplant outcomes and quality of life. Standard prophylaxis typically includes a calcineurin inhibitor (tacrolimus or cyclosporine) combined with methotrexate or mycophenolate mofetil. For acute GVHD treatment, first-line therapy consists of systemic corticosteroids, usually methylprednisolone at 2 mg/kg/day, with gradual tapering based on response 1.
Supportive Care
Supportive care is essential in managing GVHD and includes:
- Antimicrobial prophylaxis
- Nutritional support
- Organ-specific management Early intervention is crucial, and treatment must be tailored to the child's age, weight, and specific manifestations, with careful monitoring for medication side effects, which can be more pronounced in pediatric patients.
From the Research
Graft-Versus-Host Disease (GVHD) Risk in Pediatric Patients Undergoing Stem Cell Transplant (SCT)
- The risk of GVHD in pediatric patients undergoing SCT is a significant concern, with various studies investigating different prophylaxis regimens to minimize this risk 2, 3, 4, 5, 6.
- A study from 2022 found that post-transplant cyclophosphamide (PTCy) is a potentially safe and effective GVHD prophylaxis strategy in pediatric patients with acute myeloid leukemia who received matched-donor HSCT 5.
- Another study from 2017 compared the combination of cyclosporine and mycophenolate mofetil to cyclosporine and methotrexate for GVHD prophylaxis after stem-cell transplantation from unrelated donors, and found that the latter combination is more effective in preventing acute GVHD 6.
- The incidence of acute and chronic GVHD varies depending on the prophylaxis regimen used, with reported rates ranging from 16% to 43% for acute GVHD and 28% to 53% for chronic GVHD 2, 3, 4.
- The use of low-dose mycophenolate mofetil in combination with cyclosporine and methotrexate has been shown to be effective in reducing the risk of GVHD in unrelated donor allogeneic stem cell transplantation 4.
- The choice of GVHD prophylaxis regimen should be individualized based on the patient's specific needs and risk factors, with consideration of the potential benefits and risks of each regimen 2, 3, 4, 5, 6.