Nonalcoholic fatty liver disease (NAFLD) is a spectrum of liver conditions associated with fat accumulation that range from benign, non-progressive liver fat accumulation to severe liver injury, cirrhosis, and liver failure. NAFLD is highly prevalent within the United States and is most common in adults who are overweight or have diabetes, insulin resistance, or hyperlipidemia. However, the disease also occurs in children and in persons who are not obese or diabetic. The Nonalcoholic Steatohepatitis Clinical Research Network (NASH CRN) was initiated by the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) in 2002 to conduct multicenter, collaborative studies on the etiology, contributing factors, natural history, complications and treatment of NASH.
The NAFLD Pediatric Database 2 was a multicenter, prospective follow-up study of patients with NAFLD or nonalcoholic steatohepatitis (NASH) which aimed to investigate the etiology, pathogenesis, natural history, diagnosis, treatment, and prevention of NAFLD and NASH. The study included longitudinal follow-up of participants enrolled in earlier NASH CRN studies and recruited new participants. The study population included pediatric patients 2- 17 years old at the time of enrollment with histologically confirmed NAFLD or NASH located in the United States. Comprehensive data, including demographics, medical history, symptoms, medication use, alcohol use and routine laboratory studies was collected on all participants at entry and at follow-up visits every 48 weeks from enrollment. A standard of care liver biopsy was collected at baseline if not previously collected, and specimens were collected every 48 weeks during follow-up.
Primary objective(s): The NAFLD Pediatric Database 2 study aimed to elucidate, through the cooperative effort of a multidisciplinary and multicenter group of collaborators, the etiology, natural history, diagnosis, treatment, and prevention of NAFLD, and in particular its more severe form of NASH and its complications. Additional primary objectives included enrolling at least 650 pediatric patients with a diagnosis of NAFLD; increasing the population diversity of the NAFLD Database to provide greater representation of Hispanic, Native American, African American, and Asian patients; and expanding the specimen bank comprised of liver tissue, serum, plasma, and DNA obtained from patients undergoing a liver biopsy.
Secondary objective(s): The secondary objectives of the NAFLD Pediatric Database 2 were to continue the analysis of the data obtained in the previous NAFLD Database study; to add to current NASH CRN resources for developing clinical and pathological criteria for standardizing diagnostic and staging criteria for NAFLD or NASH-related cirrhosis; to develop Magnetic Resonance Imaging (MRI) or Magnetic Resonance Spectroscopy (MRS) protocols to evaluate the utility of these diagnostic modalities for the non-invasive staging and grading of NAFLD; and to add to NASH CRN resources for ancillary studies of the pathogenesis, diagnosis or diagnostic biomarker development, genomic, proteomic and lipidomic characterization, natural history and treatment of NAFLD or NASH-related cirrhosis.
The following measures were used to assess primary and secondary outcomes of interest: liver histology scores (derived from central reading of standard of care biopsy done during screening or follow-up), change in ALT and AST levels, change in glucose and insulin levels, change in lipid profiles, and change in body mass index (BMI) and anthropometric data.
Inclusion criteria:
Exclusion criteria:
A total of 971 participants (101 of which were continuing participants from prior NASH CRN studies) were included in the NAFLD Pediatric Database 2. Data collection began on October 5, 2009 and concluded on May 31, 2020. The histologic scoring system for review and grading of liver biopsies was further refined and expanded, and participants are being followed into adulthood, to better understand the natural history of NAFLD during the adolescent to adult transition. Participants will continue to be followed in NAFLD Pediatric Database 3 study (DB3), the third phase of the longitudinal NAFLD database study.
Liver Disease
Observational
2010-01
2020-05
Metabolic Dysfunction-Associated Steatotic Liver Disease, Metabolic Dysfunction-Associated Steatohepatitis, Cirrhosis of Liver, Fatty Liver Disease, Fibrotic Liver Disease
Liver Histology Scores , Glucose, Disease Etilogy, Cirrhosis, Nonalcoholic Fatty Liver Disease, Fat Accumulation, Non-Alcoholic Steatohepatitis
DDN
Dataset Name | Description | # of Records | # of Variables | File Format(s) |
---|---|---|---|---|
LS - Laboratory Results - Tests Done only During Screening (Revision 3) Data | Data collected to record archival and current results of laboratory tests done only during screening | 44 | sas7bdat (128 KB); csv (6.7 KB) | |
IE - Interim Event Report (Revision 2) Data | Data collected to document events that occur after registration that impact on the patient’s participation in the NAFLD Database 2 Study (eg, mild or moderate liver biopsy complications). Complete this form if there has been an incident cirrhosis, hepatocellular carcinoma (HCC), hospitalization, Emergency Room visit, liver transplant, an event associated with a study-related procedure, or death | 3 | sas7bdat (128 KB); csv (335 B) | |
RG - Registration Data | Data collected to register patients as candidates for enrollment in the NAFLD Database 2 study and to assign a patient ID number | 857 | sas7bdat (320 KB); csv (57.59 KB) | |
LR - Laboratory Results - Tests Done During Screening and Follow-up (Revision 2) Data | Data collected to record archival and current laboratory test results for tests done during both screening and follow-up | 1737 | sas7bdat (640 KB); csv (260.47 KB) | |
LR - Laboratory Results - Tests Done During Screening and Follow-up (Revision 1) Data | Data collected to record archival and current laboratory test results for tests done during both screening and follow-up | 135 | sas7bdat (128 KB); csv (19.96 KB) | |
IE - Interim Event Report (Revision 3) Data | Data collected to document events that occur after registration that impact on the patient’s participation in the NAFLD Database 2 Study (eg, mild or moderate liver biopsy complications). Complete this form if there has been an incident cirrhosis, hepatocellular carcinoma (HCC), hospitalization, Emergency Room visit, liver transplant, an event associated with a study-related procedure, or death | 289 | sas7bdat (128 KB); csv (22.33 KB) | |
BG - Baseline History (Revision 1) Data | Data collected on baseline medical history information about the patient | 91 | sas7bdat (312 KB); csv (43.82 KB) | |
IR - Liver Imaging Studies Report Data | Data collected to record liver imaging results | 473 | sas7bdat (320 KB); csv (43.9 KB) | |
CG - Genetic Consent and Blood Collection Documentation (Revision 1) Data | Data collected to document options selected for use of blood samples for genetic research | 682 | sas7bdat (128 KB); csv (18.79 KB) | |
PE - Physical Examination Data | Data collected to record physical exam findings of NAFLD Database 2 patients | 3144 | sas7bdat (896 KB); csv (393.45 KB) | |
AD - Alcohol Use Disorders Identification Test (AUDIT) Data | Data collected to screen for current heavy drinking and/or active alcohol abuse or dependence | 540 | sas7bdat (128 KB); csv (16.1 KB) | |
LS - Laboratory Results - Tests Done only During Screening (Revision 1) Data | Data collected to record archival and current results of laboratory tests done only during screening | 636 | sas7bdat (320 KB); csv (85.29 KB) | |
LD - Lifetime Drinking History (Skinner) Data | Data collected to obtain quantitative indices of the patient's alcohol consumption patterns from the onset of regular drinking | 8 | sas7bdat (128 KB); csv (2.8 KB) | |
CG - Genetic Consent and Blood Collection Documentation (Revision 2) Data | Data collected to document options selected for use of blood samples for genetic research | 223 | sas7bdat (128 KB); csv (6.39 KB) | |
HI - Follow-Up Medical History Data | Data collected to record follow-up medical history information about the patient | 2317 | sas7bdat (1.31 MB); csv (591.18 KB) | |
Central Histology Review (Revision 4) Data | Data collected to record results of the NASH CRN Pathology Committee review of liver biopsy slides archived at the Histology Review Center | 3 | sas7bdat (128 KB); csv (386 B) | |
Central Histology Review (Revision 2) Data | Data collected to record results of the NASH CRN Pathology Committee review of liver biopsy slides archived at the Histology Review Center | 454 | sas7bdat (128 KB); csv (37.08 KB) | |
BQ - Beverage Questionnaire (BEVQ-15) Data | Data collected to obtain the patient's beverage intake | 1146 | sas7bdat (192 KB); csv (85.66 KB) | |
IE - Interim Event Report (Revision 1) Data | Data collected to document (1) events that occur after registration but before enrollment, or between regular follow-up visits, that impact on the patient’s participation in the NAFLD Database 2 Study (eg, mild or moderate liver biopsy complications), or (2) other event that clinical center staff feel should be reported now rather than wait until the next follow-up visit and that is not recorded on another NAFLD Database 2 form | 115 | sas7bdat (128 KB); csv (7.12 KB) | |
LS - Laboratory Results - Tests Done only During Screening (Revision 2) Data | Data collected to record archival and current results of laboratory tests done only during screening | 191 | sas7bdat (192 KB); csv (28.5 KB) | |
LR - Laboratory Results - Tests Done During Screening and Follow-up (Revision 3) Data | Data collected to record archival and current laboratory test results for tests done during both screening and follow-up | 1264 | sas7bdat (512 KB); csv (196.51 KB) | |
BG - Baseline History (Revision 2) Data | Data collected on baseline medical history information about the patient | 766 | sas7bdat (1.16 MB); csv (351.23 KB) | |
Central Histology Review (Revision 3) Data | Data collected to record results of the NASH CRN Pathology Committee review of liver biopsy slides archived at the Histology Review Center | 356 | sas7bdat (128 KB); csv (23.51 KB) |
Specimen | Count |
---|---|
Plasma | 21181 |
Serum | 52376 |