Zhang Lele, Chen Nafei, Xu Zhipeng, Liang Qian, Pan Hong, Zhao Jingyu, Fang Liwei, Shi Jun
Regenerative Medicine Clinic, State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin 300020, China.
Xingtai People's Hospital, Hebei Medical University Affiliated Hospital, Hebei 054000, China.
Blood Cells Mol Dis. 2021 Jul;89:102573. doi: 10.1016/j.bcmd.2021.102573. Epub 2021 Apr 27.
Pure red cell aplasia (PRCA) is a rare syndrome characterized by severe anemia and absence of erythroid precursors. PRCA associated to monoclonal gammopathy of undetermined significance (MGUS) is a scarce condition with less than five cases reported so far. There is no agreement on the treatment of MGUS associated PRCA and treatment- free survival (TFS) is an unmet clinical need. In this report, for the first time, we demonstrated two patients with MGUS associated PRCA obtained rapid remission and maintained TFS after accepting intensive short-term bortezomib plus dexamethasone. The first case was refractory to cyclosporine and prednisone, but achieved complete remission after ten doses of bortezomib. Moreover, he has kept TFS for 12 months. The other case initiated bortezomib plus dexamethasone as soon as making a definite diagnosis. She obtained complete remission after twelve doses of bortezomib and she has maintained a normal level of haemoglobin for 8 months.
纯红细胞再生障碍性贫血（PRCA）是一种罕见的综合征，其特征为严重贫血且无红系前体细胞。与意义未明的单克隆丙种球蛋白病（MGUS）相关的PRCA是一种罕见病症，迄今为止报道的病例不足五例。对于MGUS相关PRCA的治疗尚无共识，无治疗生存期（TFS）是一项尚未满足的临床需求。在本报告中，我们首次证明了两名MGUS相关PRCA患者在接受短期密集硼替佐米联合地塞米松治疗后迅速缓解并维持了TFS。第一例患者对环孢素和泼尼松耐药，但在接受十剂硼替佐米治疗后实现完全缓解。此外，他已保持TFS达12个月。另一例患者在确诊后立即开始使用硼替佐米联合地塞米松治疗。她在接受十二剂硼替佐米治疗后实现完全缓解，并且已维持血红蛋白正常水平达8个月。
