Pediatric Hematology, Oncology, Marrow and Blood Cell Transplantation, Children's Hospital at Montefiore, 3411 Wayne Avenue, 9th Floor, Bronx, NY 10467, USA.
Pediatric Hematology, Oncology and Hematopoietic Stem Cell Transplantation, City of Hope, 1500 East Duarte Road, Duarte, CA 91010, USA.
Hematol Oncol Clin North Am. 2020 Jun;34(3):601-612. doi: 10.1016/j.hoc.2020.01.008. Epub 2020 Mar 9.
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare, aggressive hematological malignancy, derived from plasmacytoid dendritic cells. It mainly occurs in older adults, but has been reported across all age groups. Most patients present with skin lesions with or without marrow involvement and leukemic dissemination. Treatment with high-risk acute lymphoblastic leukemia therapy regimens with central nervous system prophylaxis is recommended in pediatric patients. Stem cell transplant in children is recommended for relapsed/refractory disease or high-risk disease at presentation. New targeted therapies including the recently FDA-approved anti-CD123 cytotoxin show great promise in improving the response rate.
原始滤泡性淋巴瘤（BPDCN）是一种罕见的侵袭性血液系统恶性肿瘤，来源于浆细胞样树突状细胞。它主要发生在老年人中，但也有报道发生在所有年龄组。大多数患者表现为皮肤病变，伴有或不伴有骨髓受累和白血病播散。推荐在儿科患者中采用高危急性淋巴细胞白血病治疗方案并进行中枢神经系统预防。对于复发/难治性疾病或初诊时高危疾病，建议进行干细胞移植。包括最近获得美国食品和药物管理局批准的抗 CD123 细胞毒素在内的新型靶向治疗方法显示出极大的改善反应率的潜力。
