Ito T, Suno M, Sakamoto K, Yoshizaki Y, Yamamoto K, Nakanishi R, Hirano Y, Irie M, Kurosaki T, Otani S, Yamane M, Sugimoto S, Miyoshi K, Oto T
Division of Oncology Pharmaceutical Care & Science, Graduate School of Medicine, Dentistry and Pharmaceutical Sciences, Okayama University, Japan.
Division of Oncology Pharmaceutical Care & Science, Graduate School of Medicine, Dentistry and Pharmaceutical Sciences, Okayama University, Japan.
Transplant Proc. 2016 Jan-Feb;48(1):271-4. doi: 10.1016/j.transproceed.2015.12.021.
Sirolimus (SRL) is used to treat pulmonary lymphangioleiomyomatosis (P-LAM). There is limited evidence that SRL has systemic efficacy for the patients with extrapulmonary lymphangioleiomyomatosis (E-LAM) remaining after lung transplantation (LT) for P-LAM. This report examines the efficacy of SRL treatment for the patient with E-LAM remaining after an LT for P-LAM.
The course of the patient's recovery from an LT for P-LAM was complicated by lymphedema in the left femoral region that was caused by two E-LAM lesions remaining in the left pelvic cavity and in the retroperitoneal area. After the LT was performed, the patient started SRL treatment to reduce the E-LAM lesions. The daily SRL dose, selected based on the standard SRL dose for P-LAM, was initiated at 1 mg/d and was maintained at 2 mg/d. The remaining E-LAM lesions and lymphedema in the left femoral region improved in approximately 9 months after the LT with the administration of both SRL and the standard immunosuppressive therapy used by Okayama University Hospital, including tacrolimus, mycophenolate mofetil, and prednisolone. The SRL and tacrolimus trough concentrations in whole blood were maintained within the therapeutic window for the next 1.5 years after initiation of SRL treatment. The patient experienced no severe adverse events that required discontinuation of the SRL treatment during this time.
The patients with remaining E-LAM lesions may receive SRL treatment to improve the quality of life after LT for P-LAM as effective therapy in cases where the patient's recovery is complicated by E-LAM lesions.
西罗莫司（SRL）用于治疗肺淋巴管平滑肌瘤病（P-LAM）。对于因P-LAM接受肺移植（LT）后仍存在肺外淋巴管平滑肌瘤病（E-LAM）的患者，SRL具有全身疗效的证据有限。本报告探讨了SRL治疗因P-LAM接受LT后仍存在E-LAM的患者的疗效。
该患者因P-LAM接受LT后的恢复过程因左盆腔和腹膜后区域残留的两个E-LAM病变导致左股部淋巴水肿而复杂化。LT术后，患者开始接受SRL治疗以减少E-LAM病变。根据P-LAM的标准SRL剂量选择的每日SRL剂量起始为1mg/d，并维持在2mg/d。在LT术后约9个月，在给予SRL以及冈山大学医院使用的标准免疫抑制疗法（包括他克莫司、霉酚酸酯和泼尼松龙）后，残留的E-LAM病变和左股部淋巴水肿有所改善。在开始SRL治疗后的接下来1.5年里，全血中的SRL和他克莫司谷浓度维持在治疗窗内。在此期间，患者未发生需要停用SRL治疗的严重不良事件。
对于因E-LAM病变使恢复过程复杂化的病例，存在残留E-LAM病变的患者在因P-LAM接受LT后可接受SRL治疗以改善生活质量，作为一种有效的治疗方法。
