评价小儿朗格汉斯细胞组织细胞增生症(LCH)患者胸腺受累的特点及治疗效果。
我们回顾性地描述了临床,生物,以及2016年9月至2019年12月在我们中心治疗的19例有胸腺累及的儿童LCH患者的影像学特征.我们进一步分析了接受化疗或靶向治疗的患者的治疗反应和结果。
在433个连续的儿科LCH队列中,有4.4%的人发现胸腺受累;所有LCH胸腺受累均表现为多系统疾病。胸腺受累的患者通常更年轻,与没有胸腺受累的人相比,肺和甲状腺受累更多,骨骼受累更少。大多数胸腺受累患者的免疫能力改变,T淋巴细胞亚群和免疫球蛋白G水平降低。总的来说,47.1%的患者在诱导治疗6周后表现出反应,对一线治疗无反应的患者中92.3%在二线和/或靶向治疗后胸腺消退.转用二线治疗的患者和转用dabrafenib的患者之间的进展/复发率没有差异(33.3%vs25%,P=1.000)。胸腺受累患者的生存率与无胸腺受累患者的生存率没有差异。接受二线化疗的患者比接受dabrafenib的患者更多(88.9%vs0,P<0.001)。
在LCH中很少观察到胸腺受累,并且具有特定的临床特征。化疗可以解决大多数胸腺病变,对于BRAF-V600E突变的婴儿,BRAF抑制剂可能提供毒性较小的有前景的治疗选择.
http://www。chictr.org.cn,标识符:ChiCTR2000030457(BCH-LCH2014研究);ChiCTR2000032844(dabrafenib研究)。
To evaluate the characteristics and treatment outcomes of patients with pediatric Langerhans cell histiocytosis (
LCH) with thymic involvement.
We retrospectively described the clinical, biological, and imaging characteristics of a series of 19 patients with pediatric
LCH with thymic involvement in our center between September 2016 and December 2019. We further analyzed the treatment response and outcomes of patients treated with chemotherapy or targeted therapy.
Thymic involvement was found in 4.4% of a 433-consecutive pediatric
LCH cohort; all
LCH-thymic involvement presented with multisystem disease. Patients with thymic involvement were typically younger, harboring more lung and thyroid involvement and less bone involvement than those without thymic involvement. Most patients with thymic involvement had alteration of immunocompetence with decreased numbers of T-lymphocyte subsets and immunoglobulin G levels. Overall, 47.1% of patients demonstrated a response after 6 weeks of induction therapy, and 92.3% of the patients who did not respond to the first-line treatment had resolution of thymus after the second-line and/or targeted therapy. The progression/relapse rate showed no difference between patients who shifted to second-line therapy and those to dabrafenib (33.3% vs 25%, P = 1.000). The survival for patients with thymic involvement did not differ from those without thymic involvement. More patients treated with second-line chemotherapy had severe adverse events than those given dabrafenib (88.9% vs 0, P < .001).
Thymic involvement was observed rarely in
LCH and had specific clinical characteristics. Chemotherapy could resolve most thymic lesions, and BRAF inhibitors might provide a promising treatment option with less toxicity for infants with BRAF-V600E mutation.
http://www.chictr.org.cn, identifier: ChiCTR2000030457 (BCH-
LCH 2014 study); ChiCTR2000032844 (dabrafenib study).