辅助化疗是否比单独使用皮质激素更能降低单灶性或多灶性骨组织细胞增多症的缓解率？
Does adjunctive chemotherapy reduce remission rates compared to cortisone alone in unifocal or multifocal histiocytosis of bone?
机构信息
Department of Orthopaedics, University of São Paulo, Rua Barata Ribeiro, 490 Conjunto 33, Bela Vista, São Paulo 01308-000, Brazil.
出版信息
Clin Orthop Relat Res. 2012 Mar;470(3):663-9. doi: 10.1007/s11999-011-2162-x.
BACKGROUND
Langerhans cell histiocytosis (LCH) is a rare disorder that can affect almost any organ, including bone. Treatment options include local corticosteroid infiltration in isolated bone lesions and oral corticosteroids and chemotherapy in multifocal bone lesions. Several studies show local corticosteroid injection in unifocal bone lesions heal in more than 75% of patients with minimal side effects. Therefore, it is unclear whether chemotherapy adds materially to the healing rate.
QUESTIONS/PURPOSES: We therefore compared overall survival, remission rate, and recurrence rate in patients with bone LCH treated with chemotherapy and corticosteroids or corticosteroids alone.
METHODS
We retrospectively reviewed the records of 198 patients with LCH since 1950. Median age at diagnosis was 5 years, male-to-female ratio was 1.33, and the most frequent symptom was local pain (95%). We recorded the disease presentation, demographics, treatment, and clinical evolution of each patient. Minimum followup was 4 months (median, 24 months; range, 4-360 months).
RESULTS
The survival rate of the systemic disease group was 76.5% (65 of 85) while the survival rate in the unifocal and multifocal bone involvement groups was 100% at a median 5-year followup. All patients with unifocal bone involvement and 40 of 43 (93%) with multifocal bone involvement had complete remission. One of 30 patients with multifocal bone involvement treated with chemotherapy and oral corticosteroids did not achieve remission whereas two of six receiving only corticosteroids did not achieve remission.
CONCLUSIONS
Our observations suggest intralesional corticosteroid injection without adjunctive chemotherapy achieves remission in unifocal bone LCH but may not do so in multifocal single-system bone involvement. Larger series would be required to confirm this observation.
LEVEL OF EVIDENCE
Level IV, therapeutic study. See Guidelines for Authors for a complete description of levels of evidence.
背景
朗格汉斯细胞组织细胞增生症（LCH）是一种罕见的疾病，几乎可影响任何器官，包括骨骼。治疗方案包括孤立性骨病变的局部皮质类固醇浸润以及多发性骨病变的口服皮质类固醇和化疗。多项研究表明，局部皮质类固醇注射治疗单一骨病变，75%以上的患者可治愈，且副作用极小。因此，尚不清楚化疗是否能显著提高治愈率。
问题/目的：我们比较了接受化疗和皮质类固醇或仅皮质类固醇治疗的骨 LCH 患者的总生存率、缓解率和复发率。
方法
我们回顾性分析了自 1950 年以来 198 例 LCH 患者的记录。诊断时的中位年龄为 5 岁，男女比例为 1.33，最常见的症状是局部疼痛（95%）。我们记录了每位患者的疾病表现、人口统计学特征、治疗方法和临床转归。最小随访时间为 4 个月（中位数 24 个月；范围 4-360 个月）。
结果
全身性疾病组的生存率为 76.5%（85 例中的 65 例），而单一和多灶性骨受累组的生存率在中位 5 年随访时均为 100%。所有单一骨受累的患者和 43 例多灶性骨受累患者中的 40 例（93%）均达到完全缓解。接受化疗和口服皮质类固醇治疗的 30 例多灶性骨受累患者中有 1 例未缓解，而仅接受皮质类固醇治疗的 6 例中有 2 例未缓解。
结论
我们的观察结果表明，单一骨 LCH 病变内皮质类固醇注射无需辅助化疗即可缓解，但多灶性单一系统骨受累可能无法缓解。需要更大的系列来证实这一观察结果。
证据水平
IV 级，治疗研究。请参阅作者指南以获取完整的证据水平描述。
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