PMID- 30694451 OWN - NLM STAT- MEDLINE DCOM- 20190417 LR - 20200225 IS - 1865-3774 (Electronic) IS - 0925-5710 (Linking) VI - 109 IP - 4 DP - 2019 Apr TI - Prophylaxis and treatment with mycophenolate mofetil in children with graft-versus-host disease undergoing allogeneic hematopoietic stem cell transplantation: a nationwide survey in Japan. PG - 491-498 LID - 10.1007/s12185-019-02601-5 [doi] AB - 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. FAU - Kawashima, Nozomu AU - Kawashima N AUID- ORCID: 0000-0001-5700-4481 AD - Department of Hematology and Oncology, Children's Medical Center, Japanese Red Cross Nagoya First Hospital, 3-35 Michishita-cho, Nakamura-ku, Nagoya, 453-8511, Japan. kwnozomu@med.nagoya-u.ac.jp. AD - Department of Pediatrics, Nagoya University Graduate School of Medicine, 65 Tsurumai-cho, Showa-ku, Nagoya, 466-8550, Japan. kwnozomu@med.nagoya-u.ac.jp. FAU - Iida, Minako AU - Iida M AD - Department of Promotion for Blood and Marrow Transplantation, Aichi Medical University School of Medicine, Nagakute, Japan. FAU - Suzuki, Ritsuro AU - Suzuki R AD - Department of Oncology and Hematology, Shimane University Hospital Innovative Cancer Center, Izumo, Japan. FAU - Fukuda, Takahiro AU - Fukuda T AD - Department of Hematopoietic Stem Cell Transplantation, National Cancer Center Hospital, Tokyo, Japan. FAU - Atsuta, Yoshiko AU - Atsuta Y AD - Japanese Data Center for Hematopoietic Cell Transplantation, Nagoya, Japan. FAU - Hashii, Yoshiko AU - Hashii Y AD - Department of Pediatrics, Osaka University Graduate School of Medicine, Suita, Japan. FAU - Inoue, Masami AU - Inoue M AD - Department of Hematology/Oncology, Osaka Women's and Children's Hospital, Izumi, Japan. FAU - Kobayashi, Masao AU - Kobayashi M AD - Department of Pediatrics, Hiroshima University Graduate School of Biomedical & Health Sciences, Hiroshima, Japan. FAU - Yabe, Hiromasa AU - Yabe H AD - Department of Cell Transplantation and Regenerative Medicine, Tokai University School of Medicine, Isehara, Japan. FAU - Okada, Keiko AU - Okada K AD - Department of Pediatric Hematology/Oncology, Osaka City General Hospital, Osaka, Japan. FAU - Adachi, Souichi AU - Adachi S AD - Department of Human Health Sciences, Graduate School of Medicine, Kyoto University, Kyoto, Japan. FAU - Yuza, Yuki AU - Yuza Y AD - Department of Hematology and Oncology, Tokyo Metropolitan Children's Medical Center, Fuchu, Japan. FAU - Kawa, Keisei AU - Kawa K AD - Department of Hematology/Oncology, Osaka Women's and Children's Hospital, Izumi, Japan. FAU - Kato, Koji AU - Kato K AD - Department of Hematology and Oncology, Children's Medical Center, Japanese Red Cross Nagoya First Hospital, 3-35 Michishita-cho, Nakamura-ku, Nagoya, 453-8511, Japan. LA - eng PT - Clinical Trial PT - Journal Article PT - Multicenter Study DEP - 20190129 PL - Japan TA - Int J Hematol JT - International journal of hematology JID - 9111627 RN - HU9DX48N0T (Mycophenolic Acid) SB - IM MH - Adolescent MH - Allografts MH - Child MH - Child, Preschool MH - Female MH - Graft vs Host Disease/*prevention & control MH - Hematologic Neoplasms/*therapy MH - *Hematopoietic Stem Cell Transplantation MH - Humans MH - Infant MH - Infant, Newborn MH - Japan MH - Male MH - Mycophenolic Acid/*administration & dosage/adverse effects MH - *Registries OTO - NOTNLM OT - Graft-versus-host disease OT - Mycophenolate mofetil OT - Pediatric OT - Prophylaxis OT - Treatment EDAT- 2019/01/30 06:00 MHDA- 2019/04/18 06:00 CRDT- 2019/01/30 06:00 PHST- 2018/11/29 00:00 [received] PHST- 2019/01/17 00:00 [accepted] PHST- 2019/01/16 00:00 [revised] PHST- 2019/01/30 06:00 [pubmed] PHST- 2019/04/18 06:00 [medline] PHST- 2019/01/30 06:00 [entrez] AID - 10.1007/s12185-019-02601-5 [pii] AID - 10.1007/s12185-019-02601-5 [doi] PST - ppublish SO - Int J Hematol. 2019 Apr;109(4):491-498. doi: 10.1007/s12185-019-02601-5. Epub 2019 Jan 29.