Department of Hematology, The First Affiliated Hospital of Nanjing Medical University, Jiangsu Province Hospital, Nanjing, 210029, China.
Key Laboratory of Hematology of Nanjing Medical University, Nanjing, 210029, China.
Sci Rep. 2020 Oct 1;10(1):16357. doi: 10.1038/s41598-020-73131-y.
Paraneoplastic pemphigus (PNP) is a severe autoimmune syndrome commonly triggered by neoplasms. The prognosis of CLL-associated PNP is dismal due to its refractory course and secondary infection and no standard treatment was recommended. We retrospectively reported six CLL with PNP cases from 842 cases of CLL including diagnosis, treatment and prognosis. The median time between the initial of CLL to PNP was 36 months while the median overall survival from the diagnosis of PNP was 26 months. And three cases died of lung infection while 5 developed pulmonary symptoms. And 5 cases received fludarabine-based chemotherapy before developing PNP, which suggesting fludarabine was one of potential causes of PNP. For the treatment, five patients were rescued by combined regimens including rituximab, methylprednisolone, immunoglobulin, fresh frozen plasma and the last received ibrutinib combined with short-term prednisone. Fludarabine-based regimen may be one of the potential causes of PNP. The combined regimen might shed a new light, while ibrutinib is a promising drug for CLL with PNP, but needs much more evidence. PNP should be carefully treated to guide early diagnosis and intervention for a better prognosis.
副肿瘤天疱疮（PNP）是一种常见的由肿瘤引发的严重自身免疫综合征。由于其难治性病程和继发感染，CLL 相关性 PNP 的预后较差，且没有标准的治疗方法。我们回顾性报告了 6 例 CLL 合并 PNP 病例，这些病例均来自 842 例 CLL 患者，包括诊断、治疗和预后。CLL 出现 PNP 的中位时间为 36 个月，从 PNP 诊断到死亡的中位总生存期为 26 个月。有 3 例死于肺部感染，5 例出现肺部症状。在发生 PNP 之前，有 5 例接受了氟达拉滨为基础的化疗，这表明氟达拉滨可能是 PNP 的潜在原因之一。在治疗方面，5 例患者接受了包括利妥昔单抗、甲基强的松龙、免疫球蛋白、新鲜冷冻血浆在内的联合方案治疗，最后 1 例接受了伊布替尼联合短期泼尼松治疗。氟达拉滨为基础的方案可能是 PNP 的潜在原因之一。联合方案可能会带来新的希望，而伊布替尼可能是治疗 CLL 合并 PNP 的一种有前途的药物，但需要更多的证据。PNP 应谨慎治疗，以指导早期诊断和干预，以获得更好的预后。
