Bosse Raphael C, Franke Aaron J, Paul Skelton William, Woody Lindsey E, Bishnoi Rohit, Wang Yu, Bhaduri-McIntosh Sumita, Rajderkar Dhanashree, Shih Renata, Dang Nam H, Slayton William B
Division of Hematology & Oncology, Department of Medicine, University of Florida, Gainesville, Florida, USA.
Division of Hematology/Oncology, H. Lee Moffitt Cancer Center & Research Institute, University of South Florida, Gainesville, Florida, USA.
Pediatr Transplant. 2020 Aug;24(5):e13747. doi: 10.1111/petr.13747. Epub 2020 Jun 4.
PTLD is the most frequent malignancy following SOT in children and the second most common SOT complication in adults. However, factors determining outcomes in children are poorly understood due to its relative rarity.
This study was performed at the University of Florida. Univariate and multivariate analyses were used to identify prognostic factors in pediatric patients diagnosed with PTLD.
We reviewed records of 54 pediatric (younger than 18 years old at diagnosis) patients diagnosed with PTLD from 1994 to 2017. The median follow-up was 28.8 months. The estimated 5-year survival rate was 87.6% (95% CI 74.3-94.2%). Univariate analysis showed that organ transplanted (specifically heart transplant), poor response to initial treatment, allograft rejection, and low Karnofsky score were statistically significant for negative prognostic factors in determining survival. Multivariate analysis determined progression in response to initial treatment and presence of allograft rejection as statistically significant prognostic factors affecting overall survival. We found no statistically significant impact of EBV serological status on PTLD prognosis.
Disease progression and allograft rejection were strong negative prognostic indicators in our study cohort. Close attention to graft status and development of therapies that protect the graft from rejection while bolstering anti-EBV immunity will be essential to further improving PTLD outcomes in children.
移植后淋巴增殖性疾病(PTLD)是儿童实体器官移植(SOT)后最常见的恶性肿瘤,也是成人SOT后第二常见的并发症。然而,由于其相对罕见,决定儿童PTLD预后的因素尚不清楚。
本研究在佛罗里达大学进行。采用单因素和多因素分析确定诊断为PTLD的儿科患者的预后因素。
我们回顾了1994年至2017年诊断为PTLD的54例儿科患者(诊断时年龄小于18岁)的记录。中位随访时间为28.8个月。估计5年生存率为87.6%(95%CI 74.3-94.2%)。单因素分析显示,器官移植(特别是心脏移植)、对初始治疗反应不佳、移植排斥和低卡诺夫斯基评分在决定生存的负面预后因素方面具有统计学意义。多因素分析确定,对初始治疗的反应进展和移植排斥的存在是影响总生存的统计学显著预后因素。我们发现EBV血清学状态对PTLD预后没有统计学显著影响。
在我们的研究队列中,疾病进展和移植排斥是强烈的负面预后指标。密切关注移植状态以及开发既能保护移植免受排斥又能增强抗EBV免疫力的治疗方法,对于进一步改善儿童PTLD的预后至关重要。