St John's Institute of Dermatology, King's College London, London, UK.
Br J Dermatol. 2012 Sep;167(3):678-87. doi: 10.1111/j.1365-2133.2012.11156.x.
Psoralen plus ultraviolet A (PUVA) is the standard treatment for early stages of mycosis fungoides. There have been no adequate randomized controlled trials with sufficient power comparing this modality with other therapies.
To assess disease response and to compare the response rates of patients treated with PUVA alone or PUVA and bexarotene.
EORTC 21011 (NCT 00056056) was a randomized phase III study comparing combined bexarotene (Targretin(®) ) and PUVA vs. PUVA alone in patients with stage IB and IIA mycosis fungoides (MF). The primary endpoint was the overall response rate [complete clinical response (CCR) plus partial response (PR)].
The study was prematurely closed due to low accrual after 93 of 145 required patients (65%) were randomized. Of the 93 randomized patients, 87 started treatment, 41 received PUVA and 46 received PUVA + bexarotene. Total UVA doses received were 107 J cm(-2) (range 1·4-489·9) in the PUVA arm vs. 101·7 J cm(-2) (0·2-529·9) in the combination arm. The safety profile was acceptable with few grade 3-4 toxicities observed in either arm. More drop-outs due to toxicity were observed in the combination arm compared with the PUVA-alone arm. The best overall response (CCR + PR) rate was 71% for PUVA alone and 77% for the combination arm (P = 0·57). The median duration of response was 9·7 months for PUVA vs. 5·8 months for the combination arm (P = 0·33). CCR was seen in 25 patients of whom 10 received PUVA alone (CCR 22%) and 15 received combination therapy (CCR 31%) (P = 0·45). CCR was sustained in 25% of patients regardless of therapy. There was a trend towards fewer PUVA sessions needed to achieve CCR in the combination arm (median 22) compared with the PUVA arm (median 27·5) (P = 0·11). Similarly, a trend towards lower UVA dose required to achieve CCR in the combination arm (median 55·8 J cm(-2) ) compared with the PUVA arm alone (median 117·5 J cm(-2) ) (P = 0·5) was observed.
No significant difference in response rate or response duration was observed in this study. However, there was a trend towards fewer PUVA sessions and lower UVA dose required to achieve CCR in the combination arm (PUVA + bexarotene) but this did not achieve statistical significance due to insufficient power.
补骨脂素加紫外线 A（PUVA）是蕈样肉芽肿早期阶段的标准治疗方法。目前尚无足够的、有足够效能的随机对照试验来比较这种治疗方法与其他治疗方法的疗效。
评估疾病的缓解情况，并比较单独接受 PUVA 治疗或 PUVA 联合贝沙罗汀治疗的患者的缓解率。
EORTC 21011（NCT 00056056）是一项随机 III 期研究，比较了联合贝沙罗汀（Targretin®）和 PUVA 与单独接受 PUVA 治疗在蕈样肉芽肿 IB 期和 IIA 期患者中的疗效。主要终点是总缓解率[完全临床缓解（CCR）加部分缓解（PR）]。
由于入组人数较少（93 例患者中有 145 例患者需要入组），该研究提前终止。93 例随机分组的患者中，87 例接受了治疗，41 例接受了 PUVA 治疗，46 例接受了 PUVA+贝沙罗汀治疗。PUVA 组接受的总 UVA 剂量为 107 J cm(-2)（范围为 1.4-489.9），联合治疗组为 101.7 J cm(-2)（0.2-529.9）。两组的安全性状况均可接受，仅观察到少数 3-4 级毒性。与单独接受 PUVA 治疗相比，联合治疗组的毒性相关停药率更高。单独接受 PUVA 治疗的患者最佳总缓解（CCR+PR）率为 71%，联合治疗组为 77%（P=0.57）。接受 PUVA 治疗的患者的中位缓解持续时间为 9.7 个月，而联合治疗组为 5.8 个月（P=0.33）。接受 CCR 的患者有 25 例，其中 10 例接受了单独的 PUVA 治疗（CCR 22%），15 例接受了联合治疗（CCR 31%）（P=0.45）。无论治疗方式如何，CCR 均可维持 25%的患者。与接受单独的 PUVA 治疗相比，联合治疗组达到 CCR 所需的 PUVA 治疗次数（中位数为 22 次）较少（P=0.11）。同样，与单独接受 PUVA 治疗相比，联合治疗组达到 CCR 所需的 UVA 剂量（中位数为 55.8 J cm(-2)）较低（P=0.5）。
与单独接受 PUVA 治疗相比，联合治疗组的缓解率或缓解持续时间没有显著差异。然而，联合治疗组（PUVA+贝沙罗汀）达到 CCR 所需的 PUVA 治疗次数和 UVA 剂量较少，但由于效能不足，这并未达到统计学意义。
