Background

Punch biopsy is the gold standard for diagnosis and subtyping of basal cell carcinoma. The aim of this study was to assess whether use of optical coherence tomography (OCT), a non-invasive imaging tool, might avoid the need for biopsy.

Methods

In a multicentre, randomised, non-inferiority trial, patients (aged ≥18 years) with an indication for biopsy of a suspected basal cell carcinoma outside the H-zone (high-risk zone) of the face were randomly assigned (1:1) to receive either OCT or punch biopsy (regular care) via a web-based randomisation system. Patients were enrolled from three participating centres in the Netherlands: Maastricht University Medical Centre+, Catharina Hospital Eindhoven, and Zuyderland Medical Centre Heerlen. Stratification factors for randomisation were participating centre and the grade of clinical basal cell carcinoma suspicion (high vs low). The primary endpoint was the proportion of patients free from a recurrent or residual lesion (malignant or premalignant) 12 months after treatment. Modified intention-to-treat and per-protocol analyses were conducted, with a predefined non-inferiority margin of –10%. This trial is registered with ClinicalTrials.gov number, NCT03848078, and is complete.

Findings

Between Feb 25, 2019, and Sept 2, 2020, 598 patients were enrolled and randomly assigned to either the regular care group (n=299) or the OCT group (n=299). Data on the primary endpoint were available in 553 patients (n=268 in the regular care group, n=285 in the OCT group). After median follow-up of 12·7 months (IQR 11·2–14·1) in the OCT group and 12·6 months (10·8–14·3) in the regular care group, 253 (94%) of 268 patients in the OCT group and 266 (93%) of 285 patients in the regular care group were free from recurrent or residual lesions (malignant or pre-malignant) 12 months after treatment. According to our modified intention-to-treat analysis, the absolute difference (OCT vs regular care) was 1·07% (95% CI –2·93 to 5·06; one-sided p=0·30), with the lower limit of the 95% CI not exceeding the predefined non-inferiority margin of –10%. Per-protocol analyses led to proportions free from a residual or recurrent lesion (premalignant or malignant) of 95% (250 of 263) in the OCT group and 94% (262 of 278) in the regular care group, and an absolute difference of 0·81% (95% CI –2·98 to 4·60; one-sided p=0·34).

Interpretation

OCT-guided diagnosis and treatment of basal cell carcinoma is non-inferior to regular care punch biopsy. Implementation of OCT for diagnosis of basal cell carcinoma could reduce the number of consultations and invasive procedures.

Funding

The Netherlands Organization for Health Research and Development and Maurits en Anna de Kock Stichting.

中文翻译：

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光学相干断层扫描对比穿孔活检诊断基底细胞癌：一项多中心、随机、非劣效性试验

背景

穿刺活检是基底细胞癌诊断和分型的金标准。本研究的目的是评估使用光学相干断层扫描 (OCT)，一种非侵入性成像工具，是否可以避免活检的需要。

方法

在一项多中心、随机、非劣效性试验中，对面部 H 区（高风险区）外疑似基底细胞癌进行活检的患者（年龄≥18 岁）随机分配（1:1 ) 通过基于网络的随机化系统接受 OCT 或穿孔活检（常规护理）。患者来自荷兰的三个参与中心：马斯特里赫特大学医学中心+、埃因霍温凯瑟琳娜医院和 Zuyderland 医学中心 Heerlen。随机化的分层因素是参与中心和临床基底细胞癌怀疑的等级（高vs低的）。主要终点是治疗后 12 个月没有复发或残留病变（恶性或癌前病变）的患者比例。进行了改良的意向治疗和符合方案分析，预先确定的非劣效性为–10%。该试验已在 ClinicalTrials.gov 注册号 NCT03848078 注册，并且已完成。

发现

在 2019 年 2 月 25 日至 2020 年 9 月 2 日期间，共招募了 598 名患者，并随机分配到常规护理组 (n=299) 或 OCT 组 (n=299)。553 名患者可获得主要终点数据（常规护理组 n=268，OCT 组 n=285）。在 OCT 组中位随访 12·7 个月 (IQR 11·2–14·1) 和常规护理组 12·6 个月 (10·8–14·3) 后，253 (94%) OCT 组的 268 名患者和常规护理组的 285 名患者中的 266 名（93%）在治疗 12 个月后没有复发或残留病变（恶性或癌前病变）。根据我们改良的意向治疗分析，绝对差异（OCT vs常规护理）为 1·07%（95% CI –2·93 至 5·06；单侧 p=0·30），95% CI 的下限不超过预先定义的非劣效性界值—— 10%。符合方案分析导致 OCT 组中无残留或复发病变（癌前病变或恶性病变）的比例为 95%（263 例中的 250 例）和常规护理组中的 94%（278 例中的 262 例），绝对差异为0·81%（95% CI –2·98 至 4·60；单侧 p=0·34）。

解释

OCT 引导的基底细胞癌诊断和治疗不劣于常规护理穿孔活检。实施 OCT 诊断基底细胞癌可以减少会诊次数和侵入性手术次数。

资金

荷兰卫生研究与发展组织和 Maurits en Anna de Kock Stichting。