Huang Yuzhou, Jiang Xianyong, Han Bing
Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing, China.
Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Shuaifuyuan No. 1, Dongcheng District, Beijing 100730, China.
Ther Adv Hematol. 2020 Jul 20;11:2040620720940144. doi: 10.1177/2040620720940144. eCollection 2020.
Acquired pure red cell aplasia (aPRCA) is a kind of anemia characterized by severe reticulocytopenia and reduced bone marrow erythroblastic cells. For patients who are refractory to the first-line therapy (cyclosporin A with/without glucocorticoids), second-line therapy is considered less effective. We report on a patient with primary aPRCA who was refractory to cyclosporin A, glucocorticoids, and several second-line regimens. The patient was treated with sirolimus for 10 months with no improvement in hemoglobin but complete response was achieved after adding eltrombopag at a dosage of 25 mg/day. Eltrombopag was well tolerated with no evidence of clonal evolution at the end of follow up. This case provided a new attempt at treating patients with refractory/relapse aPRCA with eltrombopag, probably in combination with sirolimus.
获得性纯红细胞再生障碍性贫血（aPRCA）是一种以严重网织红细胞减少和骨髓幼红细胞减少为特征的贫血。对于一线治疗（环孢素A联合/不联合糖皮质激素）难治的患者，二线治疗被认为效果较差。我们报告了一名原发性aPRCA患者，该患者对环孢素A、糖皮质激素及几种二线治疗方案均难治。该患者接受西罗莫司治疗10个月，血红蛋白无改善，但在加用剂量为25mg/天的艾曲泊帕后获得完全缓解。艾曲泊帕耐受性良好，随访结束时无克隆演变的证据。该病例为使用艾曲泊帕（可能联合西罗莫司）治疗难治性/复发性aPRCA患者提供了新的尝试。
