TY - JOUR
T1 - Prophylaxis and treatment with mycophenolate mofetil in children with graft-versus-host disease undergoing allogeneic hematopoietic stem cell transplantation
T2 - a nationwide survey in Japan
AU - Kawashima, Nozomu
AU - Iida, Minako
AU - Suzuki, Ritsuro
AU - Fukuda, Takahiro
AU - Atsuta, Yoshiko
AU - Hashii, Yoshiko
AU - Inoue, Masami
AU - Kobayashi, Masao
AU - Yabe, Hiromasa
AU - Okada, Keiko
AU - Adachi, Souichi
AU - Yuza, Yuki
AU - Kawa, Keisei
AU - Kato, Koji
N1 - Funding Information:
The authors would like to thank the following collaborating institutions for collecting patient data: Hokkaido University, Sapporo Medical University, Jichi Medical University, Narita Red Cross Hospital, Tokyo Metropolitan Children’s Medical Center, Tokyo Medical Dentinal University, Yokohama City University, Kanagawa Cancer Center, Tokai University, Niigata University, Kanazawa University, Shinshu University, Hamamatsu Medical University, Shizuoka Children’s Hospital, Kyoto University, Kinki University, Osaka University, Osaka City University, Osaka City General Hospital, Osaka Women’s and Children’s Hospital, Matsushita Memorial Hospital, Kobe University, Okayama University, Hiroshima University, Yamaguchi University, Kagawa Children’s Hospital, Kyushu University, and Kyushu Cancer Center (listed from north to south).
PY - 2019/4/5
Y1 - 2019/4/5
N2 - 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.
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.
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U2 - 10.1007/s12185-019-02601-5
DO - 10.1007/s12185-019-02601-5
M3 - Article
C2 - 30694451
AN - SCOPUS:85063904659
VL - 109
SP - 491
EP - 498
JO - International Journal of Hematology
JF - International Journal of Hematology
SN - 0925-5710
IS - 4
ER -