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

BACKGROUND: 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. PATIENTS AND METHODS: 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. RESULTS: 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. CONCLUSIONS: 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.