Institute of Hematology, Catholic University, Rome, Italy.
Haematologica. 2013 Feb;98(2):239-46. doi: 10.3324/haematol.2012.072645. Epub 2012 Oct 12.
The objective of this study was to evaluate the clinical features, prognostic factors, and efficacy of treatments in patients with blastic plasmacytoid dendritic cell neoplasm with a leukemic presentation at onset of the disease. In order to do this, a retrospective multicenter study was performed from 2005-2011 in 28 Italian hematology divisions in which 43 cases were collected. Forty-one patients received an induction therapy, consisting of an acute myeloid leukemia-type regimen in 26 patients (60%) and acute lymphoid leukemia/lymphoma-type regimen in 15 patients (35%). Six patients (14%) underwent allogeneic hematopoietic stem cell transplantation. Seventeen patients (41%) achieved a complete remission: seven after acute myeloid leukemia-type treatment and 10 after an acute lymphoid leukemia/lymphoma-type regimen, with a significant advantage for acute lymphoid leukemia/lymphoma-type chemotherapy (P=0.02). Relapse occurred in six of the 17 patients (35%) who achieved complete remission, more frequently after acute lymphoid leukemia/lymphoma-type chemotherapy. The median overall survival was 8.7 months (range, 0.2-32.9). The patients treated with an acute myeloid leukemia-type regimen had an overall survival of 7.1 months (range, 0.2-19.5), whereas that of the patients receiving acute lymphoid leukemia/lymphoma-type chemotherapy was 12.3 months (range, 1-32.9) (P=0.02). The median overall survival of the allogeneic hematopoietic stem cell transplant recipients was 22.7 months (range, 12-32.9), and these patients had a significant survival advantage compared to the non-transplanted patients (median 7.1 months, 0.2-21.3; P=0.03). In conclusion, blastic plasmacytoid dendritic cell neoplasm with bone-marrow involvement is an aggressive subtype of high-risk acute leukemia. The rarity of this disease does not enable prospective clinical trials to identify the better therapeutic strategy, which, at present, is based on clinicians' experience.
本研究旨在评估以疾病发病时的白血病表现为特征的原始浆细胞样树突细胞肿瘤患者的临床特征、预后因素和治疗效果。为此，我们于 2005 年至 2011 年在意大利 28 个血液学中心进行了一项回顾性多中心研究，共收集了 43 例病例。41 例患者接受了诱导治疗，其中 26 例（60%）接受急性髓系白血病样治疗方案，15 例（35%）接受急性淋巴母细胞白血病/淋巴瘤样治疗方案。6 例（14%）接受异基因造血干细胞移植。17 例（41%）患者获得完全缓解：7 例采用急性髓系白血病样治疗方案，10 例采用急性淋巴母细胞白血病/淋巴瘤样治疗方案，后者具有显著优势（P=0.02）。在 17 例获得完全缓解的患者中，有 6 例（35%）发生了复发，其中更多的患者在接受急性淋巴母细胞白血病/淋巴瘤样化疗后复发。中位总生存期为 8.7 个月（范围：0.2-32.9）。采用急性髓系白血病样治疗方案的患者总生存期为 7.1 个月（范围：0.2-19.5），而采用急性淋巴母细胞白血病/淋巴瘤样化疗的患者总生存期为 12.3 个月（范围：1-32.9）（P=0.02）。接受异基因造血干细胞移植的患者中位总生存期为 22.7 个月（范围：12-32.9），与未接受移植的患者相比，这些患者具有显著的生存优势（中位生存期 7.1 个月，0.2-21.3；P=0.03）。总之，骨髓受累的原始浆细胞样树突细胞肿瘤是一种侵袭性高风险急性白血病亚型。由于这种疾病的罕见性，无法进行前瞻性临床试验来确定更好的治疗策略，目前的治疗策略是基于临床医生的经验。
