Background/Objectives: This study involved an analysis of clinical data, histological types, genetic predisposition, treatment and outcomes in PPB in children.Patients and methods: We conducted a retrospective review of children treated for PPB at Polish pediatric oncology centers between 2011 and 2024.Results:A total of fifteen children (seven boys, eight girls; median age of 39 months; range: 27–64 months) were included. Type II solid/cystic PPB and type III solid PPB were diagnosed in six and eight children, respectively (one not known). Overall, 93% of patients were diagnosed at up to 4 years of age. Metastatic disease at diagnosis was confirmed in three (20%) patients, localized in bones, bone marrow and lymph nodes. Diagnosis was confirmed via central pathology review in 11 patients (73%). DICER1 pathogenic variants were identified in eight patients. All children presented with respiratory symptoms. The tumor dimensions were >10 cm (n = 7), 5–10 cm (n = 5) and <5 cm (n = 2). No bilateral lung involvement was observed. Tumor biopsy was performed in six children (40%), with subsequent resection (R0) in five patients. Primary resection (R0) was achieved in three patients (20%) with type II (n = 1) or type III (n = 2). In the other six patients, non-radical resection was performed: R1 in four (27%) children (with a tumor rupture in one patient) and R2 (subtotal resection) in two children (13%). All patients received postoperative chemotherapy. Maintenance chemotherapy was given to two patients. No patient received radiotherapy as first-line treatment. Progressive disease occurred in two patients in the CNS and lungs. Relapsed disease appeared in three patients, all with CNS involvement.Conclusions: PPB is a rare, malignant tumor of early childhood with an uncertain prognosis. Despite multimodal treatment, patients remain at risk of progression or CNS relapse. Complete surgical resection remains a key prognostic factor.
背景/目的：本研究旨在分析儿童胸膜肺母细胞瘤（PPB）的临床资料、组织学类型、遗传易感性、治疗方案及预后。 患者与方法：我们对2011年至2024年间在波兰儿科肿瘤中心接受治疗的PPB患儿进行了回顾性分析。 结果：共纳入15名儿童（7名男孩，8名女孩；中位年龄39个月；范围：27-64个月）。其中6名患儿诊断为II型（实性/囊性）PPB，8名诊断为III型（实性）PPB（1例分型不明）。总体而言，93%的患者在4岁前确诊。3名（20%）患者在诊断时已发生转移，转移部位包括骨骼、骨髓和淋巴结。11名（73%）患者通过中心病理会诊确诊。8名患者检出DICER1致病性变异。所有患儿均出现呼吸道症状。肿瘤最大径＞10 cm者7例，5-10 cm者5例，＜5 cm者2例。未观察到双侧肺受累。6名（40%）患儿接受了肿瘤活检，其中5例后续实现了根治性切除（R0）。3名（20%）患儿（II型1例，III型2例）实现了原发性肿瘤根治性切除（R0）。其余6名患儿接受非根治性切除：4名（27%）为R1切除（其中1例术中出现肿瘤破裂），2名（13%）为R2切除（次全切除）。所有患者术后均接受化疗，其中2名患者接受了维持化疗。无一例患者将放疗作为一线治疗。2名患者出现疾病进展（中枢神经系统和肺部）。3名患者出现疾病复发，均累及中枢神经系统。 结论：PPB是一种罕见的儿童早期恶性肿瘤，预后不确定。尽管采用多模式治疗，患者仍面临疾病进展或中枢神经系统复发的风险。完全手术切除仍是关键预后因素。
Pleuropulmonary Blastoma in Children: A Nationwide Multicenter Study
肿瘤(癌症)患者之家