Experimental Transplantation and Immunology Branch, National Cancer Institute, National Institutes of Health, Bethesda, Maryland.
Biostatistics and Data Management Section, National Cancer Institute, National Institutes of Health, Bethesda, Maryland.
Biol Blood Marrow Transplant. 2017 Nov;23(11):1980-1988. doi: 10.1016/j.bbmt.2017.08.005. Epub 2017 Aug 7.
Predicting the duration of systemic therapy in patients with chronic graft-versus-host disease (cGVHD) is of critical clinical importance when counseling patients and for treatment planning. cGVHD characteristics associated with this outcome have not been studied in severely affected patients. The National Institutes of Health (NIH) cGVHD scoring provides a standardized set of organ severity measures that could represent clinically useful and reproducible predictive characteristics. We analyzed 227 previously treated patients most with moderate (n = 54) or severe (n = 170) cGVHD defined by NIH criteria who were prospectively enrolled in a natural history protocol (NCT00092235). Patients received a median of 4 prior systemic therapy regimens and were seen at the NIH for a single time-point visit and were then monitored for survival and ability to discontinue cGVHD systemic therapy. With a median follow-up of 71.1 months, the cumulative incidence of systemic therapy discontinuation was 9.5% (95% confidence interval, 6.0% to 13.9%) at 2 years and 27.7% (95% confidence interval, 20.9% to 34.8%) by 5 years after the initial visit. Factors associated with a higher incidence of immunosuppression discontinuation included lower NIH global severity (P = .019) and lung (P = .030) scores and less extensive deep sclerosis (<37% body surface area, P = .024). Lower patient- and clinician-reported 0 to 10 severity NIH scores and noncyclosporine prophylaxis regimens were also associated with higher incidence of immunosuppression discontinuation (P <.05). In conclusion, we found low success rates for immune suppression discontinuation in previously treated patients who were severely affected with cGVHD. NIH scoring and clinical measures provide new standardized disease-specific tools to predict discontinuation of systemic therapy.
预测慢性移植物抗宿主病（cGVHD）患者系统治疗的持续时间对于患者咨询和治疗计划非常重要。尚未研究与该结果相关的 cGVHD 特征在严重受影响的患者中。美国国立卫生研究院（NIH）cGVHD 评分提供了一套标准化的器官严重程度测量方法，这些方法可能代表具有临床意义和可重复性的预测特征。我们分析了 227 名先前接受过治疗的患者，这些患者大多数患有中度（n=54）或重度（n=170）cGVHD，根据 NIH 标准定义，这些患者前瞻性地入组了一项自然史研究方案（NCT00092235）。患者接受了中位数为 4 种的系统治疗方案，在 NIH 接受了一次单时间点就诊，然后监测其生存和停止 cGVHD 系统治疗的能力。中位随访 71.1 个月后，在初始就诊后 2 年和 5 年时，停止系统治疗的累积发生率分别为 9.5%（95%置信区间，6.0%至 13.9%）和 27.7%（95%置信区间，20.9%至 34.8%）。与更高的免疫抑制停药发生率相关的因素包括 NIH 全球严重程度评分较低（P=.019）、肺部评分较低（P=.030）和深部硬化程度较低（<37%体表面积，P=.024）。较低的患者和临床医生报告的 NIH 0 至 10 严重程度评分以及非环孢素预防方案也与更高的免疫抑制停药发生率相关（P<.05）。总之，我们发现先前患有严重 cGVHD 的治疗患者停止免疫抑制的成功率较低。NIH 评分和临床指标为预测系统治疗停药提供了新的标准化疾病特异性工具。
