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Updated: Aug 18, 2026
Bone Marrow Transplantation Platform to Investigate the Role of Dendritic Cells in Graft-versus-Host Disease
Published on: March 17, 2020
[Prophylaxis and treatment of chronic graft versus host disease]
Ke Huang1, Yang Li, Shao-liang Huang
1Department of Pediatrics, Second Affiliated Hospital, Sun Yat-Sen University, Guangzhou 510120, China.
Insights
A combination therapy of methylprednisolone (MP), mycophenolate mofetil (MMF), and either tacrolimus (FK506) or cyclosporine A (CSA) is safe and effective for treating chronic graft-versus-host disease (cGVHD) in children. This treatment achieved a 100% response rate, with 78% of patients surviving event-free longer than 3 years.
Area of Science:
- Pediatric Hematology
- Immunology
- Transplantation Medicine
Background:
- Chronic graft-versus-host disease (cGVHD) is a significant complication following allogeneic hematopoietic stem cell transplantation (allo-HSCT), impacting long-term survival.
- Conventional therapies for cGVHD have limited efficacy, with approximately 50% complete response rates.
- Emerging immunosuppressants like tacrolimus (FK506) and mycophenolate mofetil (MMF) have shown promise in improving treatment outcomes.
Purpose of the Study:
- To evaluate the efficacy and safety of combined immunosuppressive therapies for cGVHD in pediatric patients.
- To compare the effectiveness of regimens involving methylprednisolone (MP), MMF, and either FK506 or cyclosporine A (CSA).
- To identify an optimal treatment strategy for cGVHD in children undergoing allo-HSCT.
Main Methods:
- A cohort of 45 patients undergoing allo-HSCT (32 UCBT, 13 PBSCT) was analyzed.
- GVHD prophylaxis included CSA, MP, and MMF.
- Patients diagnosed with cGVHD received treatment with a combination of MP, MMF, and either FK506 or CSA.
Main Results:
- The overall incidence of cGVHD was 30% (9/30 engrafted patients), with a higher rate observed after peripheral blood stem cell transplantation (PBSCT) (46%) compared to umbilical cord blood transplantation (UCBT) (18%).
- The combined MP, MMF, and FK506/CSA regimen demonstrated a 100% overall response rate for cGVHD.
- Event-free survival (EFS) beyond 3 years was achieved in 78% of patients, with infection being the primary cause of mortality.
Conclusions:
- The incidence of cGVHD in children is relatively low, but higher following PBSCT than UCBT.
- Acute graft-versus-host disease (aGVHD) is identified as a significant risk factor for developing cGVHD.
- Combined therapy using MP, MMF, and FK506 or CSA is a safe and effective treatment for pediatric cGVHD.
Objective:
Chronic graft versus host disease (cGVHD) is the most common late complication after allogeneic hematopoietic stem cell transplantation (allo-HSCT) and it represents the major cause of mortality in long-term survivors. Over the past decade, although conventional therapy has achieved complete responses in approximately 50% of patients, the prophylaxis and treatment of cGVHD are still not satisfactory. In the late years, utilization of new immunosuppressant such as tacrolimus (FK506), mycophenolate mofetil (MMF) on cGVHD improved the curative effects. This study tried to analyze the results of combination of methylprednisolone (MP), MMF and FK506 or cyclosporine A (CSA) as immunosuppressive therapies for cGVHD and to explore the effective regimen for children.
Methods:
Forty-five patients received allo-HSCT. Among them 32 received UCBT and 13 received PBSCT. The conditional regimen mainly consisted of busalphan, cyclophosphamide, antihuman thymocyte globulin, fludarabin, melphalan, thiotepa and total lymph node irradiation. Prophylaxis of GVHD consisted of CSA, MP and MMF. Patients with cGVHD received a regimen with combination of MP, MMF and FK506 or CSA.
Results:
Seventeen out of 32 patients who received UCBT were engrafted. while 9 out of 13 patients who received PBSCT were engrafted. Nine cases of the 30 engrafted patients developed cGVHD (morbidity 30%). Among the 17 patients who received UCBT, 3 developed cGVHD (18%). Among the 13 patients who received PBSCT, 6 developed cGVHD (46%). Six cGVHD continued from aGVHD (6/9). One patient was given CSA plus MMF, and 8 were given three-drug regimen with MP, MMF and FK506. The overall response rate was 100%. Two patients died of CMV-IP or septicemia (mortality 20%). Seven (78%) patients survived (event free survival, EFS) longer than 3 years. The side effects included hepatotoxicity, nephrotoxicity, hypertension, articular capsulitis and arrhythmia. The main complication and the major causes of death were infection.
Conclusion:
The incidence of cGVHD is low in children. The incidence of cGVHD after PBSCT is higher than that after UCBT. aGVHD is a highly dangerous factor. Combined therapy of MP plus MMF and FK506 or CSA is safe and effective for the treatment of cGVHD in children.
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