Comparison between low-dose chemotherapy and surgery for the treatment of extremity-associated solitary bone lesions in children with Langerhans cell histiocytosis in South China: A case-control study.
Comparison between low-dose chemotherapy and surgery for the treatment of extremity-associated solitary bone lesions in children with Langerhans cell histiocytosis in South China: A case-control study.
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华南地区朗格汉斯细胞组织细胞增多症儿童四肢孤立性骨病变低剂量化疗与手术治疗的比较：病例对照研究。
DOI：
10.1016/j.jbo.2018.02.003
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发表时间：
2018-09
影响因子：
3.4
通讯作者：
Huang G
中科院分区：
文献类型：
--
作者：
Li H;Xie X;Yin J;Tu J;Wang X;Liu W;Zhang J;Li H;Zou C;Wang Y;Shen J;Huang G
儿童四肢朗格汉斯细胞组织细胞增生症孤立性骨病变（SBL - LCH）的治疗方案仍存在争议。我们进行了一项回顾性病例对照研究，以比较低剂量化疗（LDC）和手术治疗儿童四肢SBL - LCH的可行性。
本研究将43例开始接受低剂量化疗的儿童患者与一个手术对照组（n = 44）进行比较，两组在性别、年龄、随访时间以及病变部位和大小方面相匹配，这些患者于2001年至2015年在我们机构接受治疗。对每种治疗策略的住院时间（HS）、症状缓解时间（TTSR）、恢复时间（RT）、并发症、无复发生存率（RFS）、健康相关生活质量（HRQOL）以及成本效益进行了分析。
低剂量化疗组的住院时间、症状缓解时间和恢复时间均短于手术组（p < 0.01）。化疗相关并发症包括恶心（16.30%）、转氨酶升高（9.30%）、轻度脱发（11.63%）、免疫功能下降（23.26%）、生长迟缓（16.30%）和满月脸（9.30%）。化疗相关副作用较轻且耐受性良好。手术患者出现病理性骨折（6.81%）、内固定物松动（6.00%）、手术部位感染（4.00%）和骨移植排斥（9.09%）。低剂量化疗治疗的无复发生存期（87个月）比单纯手术（59个月）更长（p = 0.011）。此外，根据欧洲癌症研究与治疗组织QLQ - C30®调查，与手术患者相比，低剂量化疗组患者在随访3个月时，在身体、角色、情感和社会功能领域的健康相关生活质量更好（分别为p < 0.001、p = 0.001、p < 0.001和p = 0.003）。然而，两组在随访2年时的健康相关生活质量评分相似。低剂量化疗相对于手术的增量成本 - 效果比（ICER）为 - 137,030元/质量调整生命年（QALY）。
与手术相比，低剂量化疗促进更快恢复，侵入性更小，具有更高的安全性和更好的健康相关生活质量，是儿童四肢SBL - LCH患者更具成本效益的治疗策略。
The treatment algorithm for solitary bone lesions of Langerhans cell histiocytosis (SBL-LCH) in children extremities still remains controversial. We conducted a retrospective case-control study to compare the feasibility of low-dose chemotherapy (LDC) and surgery for SBL-LCH in children extremities. This study compares 43 pediatric patients starting LDC with a surgery control group (n = 44), matched for gender, age, follow-up time, and lesion sites and sizes, treated between 2001 and 2015 at our institution. Hospital stay (HS), time to symptom relief (TTSR), recovery time (RT), complications, relapse-free survival (RFS), health-related quality of life (HRQOL) and cost-effectiveness were analyzed for each strategy. HS, TTSR and RT in the LDC group were shorter than those in the surgery group (p < 0.01). Chemotherapy-related complications included nausea (16.30%), aminotransferase elevation (9.30%), slight hair loss (11.63%), decline in immune function (23.26%), growth retardation (16.30%), and moon face (9.30%). Chemotherapy-related side effects were mild and well tolerated. Pathologic fractures (6.81%), loosening of instrumentation (6.00%,), surgical site infection (4.00%) and rejection of bone grafting (9.09%) developed in surgery patients. LDC treatment resulted in a longer RFS (87 months) than surgery alone (59 months) (p = 0.011). Furthermore, compared with surgery patients, patients in the LDC group had a better HRQOL at 3 months’ follow-up for the physical, role, emotional and social function domains assessed (p < 0.001, p = 0.001, p < 0.001 and p = 0.003, respectively) according to the European Organisation for Research and Treatment of Cancer QLQ-C30® survey. However, HRQOL scores at 2 years’ follow-up were similar between the two groups. The incremental cost-effectiveness ratio (ICER) was ¥−137,030/quality-adjusted life year (QALY) for LDC versus surgery. Compared with surgery, LDC promotes more rapid recovery, is less invasive, is characterized by increased safety and a superior HRQOL, and is a more cost-effective treatment strategy for pediatric patients with SBL-LCH in the extremities.
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DOI：
10.1084/jem.20130977
发表时间：
2014-04-07
期刊：
The Journal of experimental medicine
影响因子：
--
作者：
Berres ML;Lim KP;Peters T;Price J;Takizawa H;Salmon H;Idoyaga J;Ruzo A;Lupo PJ;Hicks MJ;Shih A;Simko SJ;Abhyankar H;Chakraborty R;Leboeuf M;Beltrão M;Lira SA;Heym KM;Bigley V;Collin M;Manz MG;McClain K;Merad M;Allen CE
通讯作者：
Allen CE
影响因子：
8.4
作者：
Eisenhauer, E. A.;Therasse, P.;Verweij, J.
通讯作者：
Verweij, J.
影响因子：
4.5
作者：
McKenzie, Lynda;van der Pol, Marjon
通讯作者：
van der Pol, Marjon
影响因子：
20.3
作者：
Badalian-Very, Gayane;Vergilio, Jo-Anne;Rollins, Barrett J.
通讯作者：
Rollins, Barrett J.
DOI：
10.2106/00004623-199802000-00009
发表时间：
1998-02-01
影响因子：
5.3
作者：
Yasko, AW;Fanning, CV;Murray, JA
通讯作者：
Murray, JA