Division of Hematology and Oncology, Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota.
Division of Hematology and Oncology, Department of Pediatrics, Rainbow Babies and Children's Hospital, Cleveland, Ohio.
Pediatr Blood Cancer. 2019 Jun;66(6):e27626. doi: 10.1002/pbc.27626. Epub 2019 Feb 10.
Hematopoietic stem cell transplant (HSCT) can cure or alleviate a wide range of nonmalignant childhood conditions. However, few studies have examined longitudinal national trends of frequency or short-term complications of HSCT before 2006 when an HSCT became a reportable procedure by US law. By using a US nationally representative database, we conducted nationwide longitudinal analyses on demographics, in-hospital mortality, and short-term complications in nonmalignant HSCT from 2000 to 2012.
We analyzed 2504 admissions for children < 20 years old who underwent an allogeneic HSCT for a nonmalignant condition by using the Kids' Inpatient Database for the years 2000, 2003, 2006, 2009, and 2012. Changes in in-hospital mortality and other outcomes were assessed over the study period using weighted analyses, which enabled generation of national estimates in each year.
The number of admissions for HSCT increased from 334 to 667 from 2000 to 2012, respectively; among them, the use of bone marrow decreased (66.5% to 34.1%, P < 0.001). In-hospital mortality declined (13.4% to 7.1%, P = 0.04), as did bacteremia (28.7% to 10.1%, P < 0.001) and vascular catheter infections (18.8% to 8.7%, P = 0.006), but cytomegalovirus infections increased (4.9% to 15.9%, P < 0.001), as did adenovirus infections (1.8% to 6.9%, P < 0.001) from 2000 to 2012.
Population-based analyses demonstrated a substantial expansion of the utilization of HSCT occurred for pediatric nonmalignancies from 2000 to 2012 in the United States, whereas the in-hospital mortality declined by approximately a half. Further research is needed to identify distinct contributing factors.
造血干细胞移植（HSCT）可治愈或缓解多种非恶性儿童疾病。然而，在 2006 年 HSCT 成为美国法律规定的报告程序之前，很少有研究对其进行过频率或短期并发症的全国性纵向趋势研究。本研究利用美国全国代表性数据库，对 2000 年至 2012 年间非恶性 HSCT 的人口统计学、院内死亡率和短期并发症进行了全国性纵向分析。
我们对 2000 年、2003 年、2006 年、2009 年和 2012 年接受非恶性疾病异基因 HSCT 的 2504 名<20 岁儿童的住院记录进行了分析。通过加权分析评估研究期间院内死亡率和其他结局的变化，从而每年生成全国性估计值。
2000 年至 2012 年，HSCT 入院人数从 334 人增加到 667 人；其中骨髓的使用率从 66.5%降至 34.1%（P<0.001）。院内死亡率从 13.4%降至 7.1%（P=0.04），菌血症（从 28.7%降至 10.1%，P<0.001）和血管导管感染（从 18.8%降至 8.7%，P=0.006）的发生率也有所下降，但巨细胞病毒感染（从 4.9%增至 15.9%，P<0.001）和腺病毒感染（从 1.8%增至 6.9%，P<0.001）的发生率从 2000 年至 2012 年有所上升。
基于人群的分析表明，2000 年至 2012 年期间，美国非恶性儿童疾病 HSCT 的应用显著扩大，而院内死亡率下降了约一半。需要进一步研究以确定不同的促成因素。
