Division of Pediatric Gastroenterology, Hepatology and Nutrition, Necker-Enfants Malades University of Paris-UFR Paris Descartes, Certified Center for Home Parenteral Nutrition, Reference Center for Rare Digestive Diseases in Children, Paris, France.
Division of Pediatric Gastroenterology, Hepatology and Nutrition, Purpan University Hospital, Certified Center for Home Parenteral Nutrition, Toulouse, France.
Clin Nutr. 2021 Oct;40(10):5278-5287. doi: 10.1016/j.clnu.2021.08.002. Epub 2021 Aug 14.
Home Parenteral Nutrition (HPN) is the cornerstone management for children suffering from chronic intestinal failure (CIF). In France, HPN is organized from a network of 7 certified centers located in University Hospitals spread across the national territory. This study aims to review the data involving children on HPN over a 6-years period in France to outline the global and continuous improvement in care.
This cross-sectional study included all children enrolled in any of the 7 French HPN certified centers from January 1st, 2014 to December 31st, 2019. Data was recorded from annual databases provided by each center regarding: age at inclusion, indication and duration of HPN, type of intravenous lipid emulsion (ILE), outcome [PN weaning off, transfer to adult center, death, intestinal transplantation (ITx)], rate of catheter-related bloodstream infections (CRSBIs) for 1000 days of HPN, Taurolidine lock procedure (TLP) use and prevalence of cholestasis defined as conjugated bilirubin ≥20 μmol/l.
The number of patients increased by 43.6% from 268 in 2014 to 385 in 2019. According to the year of follow up, the indications for HPN were short bowel syndrome (SBS) (42.3-46.6%), congenital enteropathies (CE) (18.5-22.8%), chronic intestinal pseudo-obstruction syndrome (CIPOS) (13.0-16.3%), long segment Hirschsprung's disease (LSHD) (9.7-13.3%), Crohn's disease (CD) (1.6-2.6%) and other non-primary digestive diseases (NPDD) such as immune deficiency, cancer or metabolic disease (4.0-9.2%). The median age at discharge on HPN decreased from 11.7 months in 2014 to 8.3 months in 2019 (p < .001). By December 31st, 2019, 44.8% of children had left the HPN program after a median duration ranging between 39.9 and 66.4 months. Among these patients, 192 (74.2%) were weaned off PN (94.7% SBS), 41 (15.8%) were transferred to adult centers for CIPOS (42%), SBS (31%) or CE (27%), 21 died (8.1%) - mostly in relation to cancer or immune deficiency - and 5 were transplanted (1.9%): 4 underwent combined liver-intestine transplantation for LSHD (n = 2), SBS, CE and one multivisceral Tx for CIPOS. The use of a composite fish-oil based ILE increased from 67.4% in 2014 to 88.3% in 2019 (p < 0.001). CRBSIs dropped from 1.04 CRSBIs per 1000 days HPN in 2014 to 0.61 in 2019 (p < 0.001) while meantime, the percentage of children receiving TLP increased from 29.4% to 63.0% (p < 0.001). The prevalence of cholestasis (conjugated bilirubin ≥ 20 μmol/l) was low and stable between 4.1 and 5.9% of children during the study period.
In France, the number of children enrolled in a HPN program continuously increased over a 6 years period. SBS is the leading cause of CIF requiring HPN. The rate of CRBSIs dropped dramatically as the use of TLP increased. Mortality rate was low and mainly in relation to the underlying disease (cancer, immune deficiency). Cholestasis and intestinal Tx remained very rare.
家庭肠外营养（HPN）是治疗慢性肠衰竭（CIF）儿童的基石管理方法。在法国，HPN 由分布在全国的 7 个位于大学医院的认证中心网络组织，这些中心负责管理 HPN。本研究旨在回顾法国在 6 年期间接受 HPN 治疗的儿童的数据，以概述整体和持续的护理改善。
本回顾性研究纳入了 2014 年 1 月 1 日至 2019 年 12 月 31 日期间在法国任何一个 7 个 HPN 认证中心接受治疗的所有儿童。通过每个中心提供的年度数据库记录以下数据：纳入时的年龄、HPN 的适应证和持续时间、静脉内脂肪乳剂（ILE）的类型、结局[PN 脱机、转至成人中心、死亡、肠移植（ITx）]、HPN 1000 天内导管相关血流感染（CRBSI）的发生率、牛磺胆酸锁程序（TLP）的使用情况和定义为结合胆红素≥20 μmol/L 的胆汁淤积的患病率。
患者数量从 2014 年的 268 例增加到 2019 年的 385 例，增长了 43.6%。根据随访时间，HPN 的适应证为短肠综合征（SBS）（42.3-46.6%）、先天性肠病（CE）（18.5-22.8%）、慢性肠假性梗阻综合征（CIPOS）（13.0-16.3%）、长段先天性巨结肠（LSHD）（9.7-13.3%）、克罗恩病（CD）（1.6-2.6%）和其他非原发性消化疾病（NPDD），如免疫缺陷、癌症或代谢疾病（4.0-9.2%）。HPN 出院时的中位年龄从 2014 年的 11.7 个月下降到 2019 年的 8.3 个月（p<0.001）。截至 2019 年 12 月 31 日，在中位持续时间为 39.9 至 66.4 个月后，44.8%的儿童停止了 HPN 治疗。在这些患者中，192 例（74.2%）成功脱机（94.7%为 SBS），41 例（15.8%）转至成人中心，其中 CIPOS（42%）、SBS（31%）或 CE（27%）为主要转归，21 例死亡（8.1%），主要与癌症或免疫缺陷有关，5 例进行了移植（1.9%）：4 例接受 LSHD（n=2）、SBS、CE 和 1 例多脏器移植的联合肝肠移植，1 例接受 LSHD、SBS、CE 和 1 例多脏器移植的联合肝肠移植。复合鱼油为基础的 ILE 的使用率从 2014 年的 67.4%增加到 2019 年的 88.3%（p<0.001）。CRBSI 从 2014 年的每 1000 天 HPN 1.04 例降至 2019 年的 0.61 例（p<0.001），与此同时，接受 TLP 的儿童比例从 29.4%增加到 63.0%（p<0.001）。在研究期间，胆汁淤积（结合胆红素≥20 μmol/L）的患病率较低且稳定，在 4.1%至 5.9%的儿童中。
在法国，接受 HPN 治疗的儿童人数在 6 年内持续增加。SBS 是导致需要 HPN 的 CIF 的主要原因。随着 TLP 的使用增加，CRBSI 的发生率显著下降。死亡率较低，主要与基础疾病（癌症、免疫缺陷）有关。胆汁淤积和肠移植仍然非常罕见。
