Vanderbilt-Ingram Cancer Center, Nashville, TN.
Stem Cell Transplantation Clinic I for Internal Medicine, University of Cologne, Köln, Germany.
Blood Adv. 2019 Jul 23;3(14):2218-2229. doi: 10.1182/bloodadvances.2019000145.
The investigation of extracorporeal photopheresis (ECP) plus standard of care (SoC) (SoC+ECP) in chronic graft-versus-host disease (cGVHD) within prospective, randomized clinical studies is limited, despite its frequent clinical use. This phase 1/pilot study was the first randomized, prospective study to investigate ECP use as first-line therapy in cGVHD, based on the 2015 National Institutes of Health (NIH) consensus criteria for diagnosis and response assessment. Adult patients with new-onset (≤3 years of hematopoietic stem cell transplantation) moderate or severe cGVHD were randomized 1:1 to 26 weeks of SoC+ECP vs SoC (corticosteroids and cyclosporine A/tacrolimus) between 2011 and 2015. The primary endpoint was overall response rate (ORR), defined as complete or partial response, at week 28 in the intention-to-treat population (ITT). Other outcomes included quality of life (QoL) measures and safety. Sixty patients were randomized; ITT included 53 patients (SoC+ECP: 29; SoC: 24). Week 28 ORR was 74.1% (SoC+ECP) and 60.9% (SoC). Investigator-assessed ORR was 56.0% (SoC+ECP) and 66.7% (SoC). Patients treated with SoC experienced a decline in QoL over the 28-week study period; QoL remained unchanged in SoC+ECP patients. Most frequent treatment-emergent adverse events (TEAEs) in SoC+ECP patients were hypertension (31.0%), cough (20.7%), dyspnea (17.2%), and fatigue (17.2%). Seventeen patients (SoC+ECP: 8; SoC: 9) experienced 35 serious adverse events (SAEs). No TEAEs or SAEs were considered related to the ECP instrument or methoxsalen. The encouraging short-term results of this study could inform the design of subsequent studies. This trial was registered at www.clinicaltrials.gov as #NCT01380535.
体外光分离术（ECP）联合标准治疗（SoC）（SoC+ECP）在慢性移植物抗宿主病（cGVHD）中的研究仅限于前瞻性随机临床试验，尽管其临床应用广泛。这项基于 2015 年美国国立卫生研究院（NIH）诊断和反应评估共识标准的新发病例（≤3 年造血干细胞移植）中重度 cGVHD 患者的一线 ECP 治疗的 1 期/初步研究是第一项随机前瞻性研究。成人患者按 1:1 比例随机分配至 26 周的 SoC+ECP 或 SoC（皮质类固醇和环孢素 A/他克莫司）治疗，时间为 2011 年至 2015 年。主要终点是意向治疗人群（ITT）中第 28 周的总体缓解率（ORR），定义为完全或部分缓解。其他结果包括生活质量（QoL）测量和安全性。60 例患者被随机分配；ITT 纳入 53 例患者（SoC+ECP：29 例；SoC：24 例）。第 28 周 ORR 为 74.1%（SoC+ECP）和 60.9%（SoC）。研究者评估的 ORR 为 56.0%（SoC+ECP）和 66.7%（SoC）。接受 SoC 治疗的患者在 28 周研究期间 QoL 下降；SoC+ECP 患者 QoL 保持不变。SoC+ECP 患者最常见的治疗相关不良事件（TEAEs）为高血压（31.0%）、咳嗽（20.7%）、呼吸困难（17.2%）和疲劳（17.2%）。17 例患者（SoC+ECP：8 例；SoC：9 例）发生 35 例严重不良事件（SAE）。无 TEAEs 或 SAE 被认为与 ECP 仪器或甲氧沙林有关。这项研究的短期结果令人鼓舞，可为后续研究提供设计依据。该试验在 www.clinicaltrials.gov 注册，编号为 #NCT01380535。
