Kawashima Nozomu, Iida Minako, Suzuki Ritsuro, Fukuda Takahiro, Atsuta Yoshiko, Hashii Yoshiko, Inoue Masami, Kobayashi Masao, Yabe Hiromasa, Okada Keiko, Adachi Souichi, Yuza Yuki, Kawa Keisei, Kato Koji
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.
Department of Pediatrics, Nagoya University Graduate School of Medicine, 65 Tsurumai-cho, Showa-ku, Nagoya, 466-8550, Japan.
Int J Hematol. 2019 Apr;109(4):491-498. doi: 10.1007/s12185-019-02601-5. Epub 2019 Jan 29.
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.
我们通过一项针对日本接受造血干细胞移植（HSCT）的儿童的全国性回顾性调查，研究了霉酚酸酯（MMF）在预防和治疗移植物抗宿主病（GVHD）方面的安全性和有效性。总体而言，1995年至2011年期间，141名接受异基因HSCT治疗血液系统恶性肿瘤（n = 84）、非恶性疾病（n = 52）和实体瘤（n = 5）的儿童口服了MMF（中位年龄8岁；范围0至15岁；男89例，女52例）。供者主要为无关供者和不相合亲属供者。在GVHD预防组中，分别有29%和8.6%的患者发生了II - IV级和III - IV级GVHD。在32例可评估的患者中，16%发生了慢性GVHD[局限性（n = 4）和广泛性（n = 1）]。在急性GVHD治疗组中，61%的患者病情分级降低。在慢性GVHD治疗组中，36%的患者症状改善。61%的患者联合免疫抑制剂减少或停用。主要不良事件（AE）为中性粒细胞减少（4.3%）、感染（3.5%）、血小板减少（2.1%）、骨髓抑制（2.1%）和腹泻（1.4%）。由于发生了≥3级AE，两名儿童减少了MMF剂量；两名儿童死于感染。因此，MMF在儿童中可能耐受性良好，并且可能是预防和治疗急性及慢性GVHD的有效选择。
