Blood:利妥昔单抗联合苯达莫司汀治疗冷凝集素病的长期预后

2020-05-16 QQY MedSci原创

CAD的最大规模研究发现,寒冷和温暖气候之间的患病率和发病率存在4倍的差异。 利妥昔单抗-苯达莫司汀治疗的有效率为78%,完全缓解率为53%,持续缓解事件>88个月,并可降晚期恶性肿瘤风险。

冷凝集素病(CAD)是指低温时冷凝集素引起肢体末端血管内红细胞凝集,发生皮肤微循环障碍,或伴轻度溶血性贫血为特征的自身免疫性疾病。

本研究回顾性研究了5个国家24个中心的232位冷凝集素病患者。在挪威和意大利北部地区,这项研究几乎是基于人口的。本研究首次发现CAD在寒冷和温暖气候地区的患病率(20例与5例/百万)和发病率存在4倍差异(1.9 vs 0.48 例/10万人·年)。

受试患者的平均基线血红蛋白水平为9.3 g/dL,但有27%患者的血红蛋白<8 g/dL。通过集中活检评估发现,骨髓中“CAD相关淋巴增生性疾病”的典型特征的识别大大增加。CAD还似乎与静脉血栓栓塞的风险轻度增加有关。

本研究包括后续的治疗随访,重点关注利妥昔单抗联合苯达莫司汀和利妥昔单抗联合氟达拉滨方案的长期预后。妥昔单抗联合苯达莫司汀疗法的缓解率为78%(35/45),其中53%(24例)的患者获得完全缓解(CR)。

有趣的是,这些比率仍然高于最初(2017年)的前瞻性试验,而且研究人员还发现,随着时间的推移,患者的缓解会越来越深。这可以解释为许多患者缓解时间的延长,可能与存活时间长的浆细胞有关。

从利妥昔单抗-苯达莫司汀治疗获得缓解的患者,88个月后中位缓解持续时间仍未达到,估计5年持续缓解率为77%。而且该方案对于晚期恶性肿瘤的安全性貌似良好。相比之下,利妥昔单抗联合氟达拉滨疗法似乎具有较高的长期不良反应风险。

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