International Journal of Hematology

DOI: 10.1007/s12185-019-02601-5 Pages: 491-498

Prophylaxis and treatment with mycophenolate mofetil in children with graft-versus-host disease undergoing allogeneic hematopoietic stem cell transplantation: a nationwide survey in Japan

1. Children’s Medical Center, Japanese Red Cross Nagoya First Hospital, Department of Hematology and Oncology

2. Nagoya University Graduate School of Medicine, Department of Pediatrics

3. Aichi Medical University School of Medicine, Department of Promotion for Blood and Marrow Transplantation

4. Shimane University Hospital Innovative Cancer Center, Department of Oncology and Hematology

5. National Cancer Center Hospital, Department of Hematopoietic Stem Cell Transplantation

6. Japanese Data Center for Hematopoietic Cell Transplantation

7. Osaka University Graduate School of Medicine, Department of Pediatrics

8. Osaka Women’s and Children’s Hospital, Department of Hematology/Oncology

9. Hiroshima University Graduate School of Biomedical & Health Sciences, Department of Pediatrics

10. Tokai University School of Medicine, Department of Cell Transplantation and Regenerative Medicine

11. Osaka City General Hospital, Department of Pediatric Hematology/Oncology

12. Graduate School of Medicine, Kyoto University, Department of Human Health Sciences

13. Tokyo Metropolitan Children’s Medical Center, Department of Hematology and Oncology

Correspondence to:
Nozomu Kawashima
Tel: +81-52-744-2294



We investigated the safety and efficacy of mycophenolate mofetil (MMF) in the prevention and treatment of graft-versus-host disease (GVHD) using a nationwide retrospective survey in Japanese children undergoing hematopoietic stem cell transplantation (HSCT). Overall, 141 children undergoing allogeneic HSCT for hematological malignancy (n = 84), non-malignancy (n = 52), and solid tumors (n = 5) were administered MMF orally (median 8 years; range 0–15 years; 89 males and 52 females) during 1995–2011. Donors were primarily unrelated and mismatched related. In the GVHD prophylaxis group, 29% and 8.6% of patients developed grade II–IV and III–IV GVHD, respectively. Of the 32 evaluable patients, 16% developed chronic [limited (n = 4) and extensive (n = 1)] GVHD. In the acute GVHD treatment group, 61% had decreased grade. In the chronic GVHD treatment group, 36% had improved symptoms. Combined immunosuppressant was reduced or discontinued in 61% patients. Major adverse events (AEs) were neutropenia (4.3%), infection (3.5%), thrombocytopenia (2.1%), myelosuppression (2.1%), and diarrhea (1.4%). MMF dosage was reduced in two children due to grade ≥ 3 AEs; two children died from infection. MMF thus may be well tolerated in children, and may be an effective option for prophylaxis and treatment of acute and chronic GVHD.

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