- 作者：Ruosha Li ; PhD1 ; Steven H. Belle ; PhD ; MScHyg2 ; Simon Horslen ; MB ; ChB3 ; Ling-wan Chen ; MS4 ; Song Zhang ; MS5 ; Robert H. Squires ; MD6 ; squiresr@upmc.edu" class="auth_mail" title="E-mail the corresponding author ; on behalf of the Pediatric Acute Liver Failure Study Group&lowast ; ; Kathryn Bukauskas ; RN ; CCRC ; Michael R. Narkewicz ; MD ; Michelle Hite ; MA ; CCRC ; Kathleen M. Loomes ; MD ; Elizabeth B. Rand ; MD ; David Piccoli ; MD ; Deborah Kawchak ; MS ; RD ; Rene Romero ; MD ; Saul Karpen ; MD ; PhD ; Liezl de la Cruz-Tracy ; CCRC ; Vicky Ng ; MD ; Kelsey Hunt (Clinical Research Coordinator) ; Girish C. Subbarao ; MD ; Ann Klipsch ; RN ; Estella M. Alonso ; MD ; Lisa Sorenson ; PhD ; Susan Kelly ; RN ; BSN ; Dhey Delute ; RN ; CCRC ; Katie Neighbors ; MPH ; CCRC ; Philip J. Rosenthal ; MD ; Shannon Fleck (Clinical Research Coordinator) ; Mike A. Leonis ; MD ; PhD ; John Bucuvalas ; MD ; Tracie Horning (Clinical Research Coordinator) ; Norberto Rodriguez Baez ; MD ; Shirley Montanye ; RN (Clinical Research Coordinator) ; Margaret Cowie (Clinical Research Coordinator) ; Karen Murray ; MD ; Melissa Young (Clinical Research Coordinator) ; Heather Vendettuoli (Clinical Research Coordinator) ; David A. Rudnick ; MD ; PhD ; Ross W. Shepherd ; MD ; Kathy Harris (Clinical Research Coordinator) ; Saul J. Karpen ; MD ; PhD ; Alejandro De La Torre (Clinical Research Coordinator) ; Dominic Dell Olio ; MD ; Deirdre Kelly ; MD ; Carla Lloyd (Clinical Research Coordinator) ; Steven J. Lobritto ; MD ; Sumerah Bakhsh ; MPH (Clinical Research Coordinator) ; Maureen Jonas ; MD ; Scott A. Elifoson ; MD ; Roshan Raza ; MBBS ; Kathleen B. Schwarz ; MD ; Wikrom W. Karnsakul ; MD ; Mary Kay Alford ; RN ; MSN ; CPNP ; Anil Dhawan ; MD ; Emer Fitzpatrick ; MD ; Nanda N. Kerkar ; MD ; Brandy Haydel ; CCRC ; Sreevidya Narayanappa (Clinical Research Coordinator) ; M. James Lopez ; MD ; PhD ; Victoria Shieck ; RN ; BSN
- 关键词：APAP ; Acetaminophen overdose ; BIC ; Bayesian information criteria ; BLRT ; Bootstrap likelihood ratio test ; GMM ; Growth mixture modeling ; HE ; Hepatic encephalopathy ; IND ; Indeterminate final diagnosis ; INR ; International normalized ratio ; KCHC ; King's College Hospital Criteria ; LTx ; Liver transplantation ; MAR ; Missing at random ; non-IND ; Determined final diagnosis ; PALF ; Pediatric acute liver failure
- 刊名：The Journal of Pediatrics
- 出版年：2016
- 出版时间：April 2016
- 年：2016
- 卷：171
- 期：Complete
- 页码：163-170.e3
- 全文大小：475 K
Study design
We studied participants enrolled in the PALF registry study with indeterminate final diagnosis. Growth mixture modeling was used to analyze participants' international normalized ratio, total bilirubin, and hepatic encephalopathy trajectories in the first 7 days following enrollment. Participants with at least 3 values for 1 or more of the measurements were included. We examined the association between the resulting latent subgroup classification with participants' characteristics and disease outcomes. Data from participants with PALF of specified etiologies were used to investigate the potential diagnostic value of the latent subgroups.
Results
In this sample of 380 participants with indeterminate final diagnosis, 115 (30%) experienced mild and quickly improving disease trajectories and another 48 (13%) started with severe disease but improved by day 7. The majority of participants (216, 57%) had disease trajectories that worsened over time. The identified patterns of disease trajectories are predictive of outcome (P < .001). The trajectory patterns are associated with the underlying disease etiology (P < .001) for the 488 participants with PALF of specified etiologies.
Conclusions
The clinical courses of participants with PALF of indeterminate disease etiology exhibit distinct trajectory patterns, which have important prognostic and potentially diagnostic value.