Jorgensen Jessica J, Joe Galen O, Jiménez-Silva Rafael, Ho Pei-Shu, Dunigan Tiara, Mitchell Sandra A, Pavletic Steven Z, Curtis Lauren M, Comis Leora E
Rehabilitation Medicine Department, Clinical Center, National Institutes of Health, Bethesda, Maryland; Department of Medicine, University of Wisconsin School of Medicine and Public Health, Madison, WI, USA.
Rehabilitation Medicine Department, Clinical Center, National Institutes of Health, Bethesda, Maryland.
Transplant Cell Ther. 2025 Jun;31(6):361.e1-361.e15. doi: 10.1016/j.jtct.2025.02.023. Epub 2025 Mar 5.
Deficits in motor performance and functional abilities represent a severe complication for individuals with steroid refractory chronic graft versus host disease (cGVHD) and is associated with decreased survival and high morbidity. The objective of this study was to characterize the impact of pomalidomide on motor and functional outcomes in patients with cGVHD. Thirty-four adult patients with cGVHD were enrolled in a randomized and unblinded trial. Pomalidomide was administered orally at two dose levels: low (0.5 mg/d) or high (initial 0.5 mg/d, escalating 0.5 mg/d every 2 weeks to a maximum 2 mg/d). Efficacy was assessed primarily by the Activity Card Sort (ACS), 2 Minute Walk Test (2MWT), Medical Outcomes Study Short Form 36 (SF-36), Active Range of Motion (AROM), Disabilities of the Arm, Shoulder, and Hand (DASH), and Manual Abilities Measure 36 (MAM). Compared to baseline, the pooled sample of study participants at 6 months showed improvement in hand skills (MAM, P = .01), upper extremity (UE) function (DASH, P = .01), and health related quality of life (SF-36 Physical Component Summary score (PCS), P = .02). Though no statistically meaningful differences between the two dose groups were found, the low-dose group had greater improvements in AROM, walk distance, UE and hand function, and in the high-demand physical leisure and social subdomains of the ACS as compared to the high-dose group. Responders to pomalidomide performed better than nonresponders on most measures at the 6-month endpoint. The study suggests pomalidomide, at both dose levels, may improve several aspects of motor and functional abilities. However, further study is warranted to determine if the trends found in this study, are sustained over time in larger, and in more diverse cGVHD populations. The findings highlight the potential utility of administering functional and motor tests, such as the ACS, DASH, and MAM, to patients with cGVHD, to fully elucidate the efficacy of treatment options for persons with steroid refractory cGVHD.
运动表现和功能能力的缺陷是类固醇难治性慢性移植物抗宿主病（cGVHD）患者的严重并发症，与生存率降低和高发病率相关。本研究的目的是描述泊马度胺对cGVHD患者运动和功能结局的影响。34例成年cGVHD患者参加了一项随机非盲试验。泊马度胺以两种剂量水平口服给药：低剂量（0.5mg/d）或高剂量（初始0.5mg/d，每2周递增0.5mg/d至最大2mg/d）。主要通过活动卡片分类（ACS）、2分钟步行试验（2MWT）、医学结局研究简明健康调查36项量表（SF-36）、主动关节活动度（AROM）、上肢、肩部和手部功能障碍（DASH）以及手动能力测量36项量表（MAM）评估疗效。与基线相比，研究参与者在6个月时的合并样本显示手部技能（MAM，P = 0.01）、上肢（UE）功能（DASH，P = 0.01）和健康相关生活质量（SF-36身体成分汇总评分（PCS），P = 0.02）有所改善。虽然两个剂量组之间未发现统计学上有意义的差异，但与高剂量组相比，低剂量组在AROM、步行距离、UE和手部功能以及ACS的高需求身体休闲和社会子领域有更大改善。在6个月终点时，泊马度胺反应者在大多数指标上的表现优于无反应者。该研究表明，两个剂量水平的泊马度胺均可改善运动和功能能力的多个方面。然而，有必要进行进一步研究，以确定本研究中发现的趋势在更大、更多样化的cGVHD人群中是否能长期持续。这些发现突出了对cGVHD患者进行功能和运动测试（如ACS、DASH和MAM）的潜在效用，以充分阐明类固醇难治性cGVHD患者治疗方案的疗效。
