Gamma Glutamyltransferase Reduction Is Associated With Favorable Outcomes in Pediatric Primary Sclerosing Cholangitis

Show full item record



Deneau , M R , Mack , C , Abdou , R , Amin , M , Amir , A , Auth , M , Bazerbachi , F , Broderick , A M , Chan , A , DiGuglielmo , M , El-Matary , W , El-Youssef , M , Ferrari , F , Furuya , K N , Gottrand , F , Gupta , N , Homan , M , Jensen , M K , Kamath , B M , Kim , K M , Kolho , K-L , Konidari , A , Koot , B , Iorio , R , Martinez , M , Mohan , P , Palle , S , Papadopoulou , A , Ricciuto , A , Saubermann , L , Sathya , P , Shteyer , E , Smolka , V , Tanaka , A , Valentino , P L , Varier , R , Venkat , V , Vitola , B , Vos , M B , Woynarowski , M , Yap , J & Miloh , T 2018 , ' Gamma Glutamyltransferase Reduction Is Associated With Favorable Outcomes in Pediatric Primary Sclerosing Cholangitis ' , Hepatology communications , vol. 2 , no. 11 , pp. 1369-1378 .

Title: Gamma Glutamyltransferase Reduction Is Associated With Favorable Outcomes in Pediatric Primary Sclerosing Cholangitis
Author: Deneau, Mark R.; Mack, Cara; Abdou, Reham; Amin, Mansi; Amir, Achiya; Auth, Marcus; Bazerbachi, Fateh; Broderick, Anne Marie; Chan, Albert; DiGuglielmo, Matthew; El-Matary, Wale; El-Youssef, Mounif; Ferrari, Federica; Furuya, Katryn N.; Gottrand, Frederic; Gupta, Nitika; Homan, Matjaz; Jensen, M. K.; Kamath, Binita M.; Kim, Kyung Mo; Kolho, Kaija-Leena; Konidari, Anastasia; Koot, Bart; Iorio, Raffaele; Martinez, Mercedes; Mohan, Parvathi; Palle, Sirish; Papadopoulou, Alexandra; Ricciuto, Amanda; Saubermann, Lawrence; Sathya, Pushpa; Shteyer, Eyal; Smolka, Vratislav; Tanaka, Atsushi; Valentino, Pamela L.; Varier, Raghu; Venkat, Veena; Vitola, Bernadette; Vos, Miriam B.; Woynarowski, Marek; Yap, Jason; Miloh, Tamir
Contributor organization: Children's Hospital
University of Helsinki
HUS Children and Adolescents
Date: 2018-11
Language: eng
Number of pages: 10
Belongs to series: Hepatology communications
ISSN: 2471-254X
Abstract: Adverse clinical events in primary sclerosing cholangitis (PSC) happen too slowly to capture during clinical trials. Surrogate endpoints are needed, but no such validated endpoints exist for children with PSC. We evaluated the association between gamma glutamyltransferase (GGT) reduction and long-term outcomes in pediatric PSC patients. We evaluated GGT normalization (<50 IU/L) at 1 year among a multicenter cohort of children with PSC who did or did not receive treatment with ursodeoxycholic acid (UDCA). We compared rates of event-free survival (no portal hypertensive or biliary complications, chola ngiocarcinoma, liver transplantation, or liver-related death) at 5 years. Of the 287 children, mean age of 11.4 years old, UDCA was used in 81% at a mean dose of 17 mg/kg/day. Treated and untreated groups had similar GGT at diagnosis (314 versus 300, P = not significant[NS]). The mean GGT was reduced at 1 year in both groups, with lower values seen in treated (versus untreated) patients (99 versus 175, P = 0.002), but 5-year event-free survival was similar (74% versus 77%, P = NS). In patients with GGT normalization (versus no normalization) by 1 year, regardless of UDCA treatment status, 5-year eventfree survival was better (91% versus 67%, P <0.001). Similarly, larger reduction in GGT over 1 year (>75% versus <25% reduction) was also associated with improved outcome (5-year event-free survival 88% versus 61%, P = 0.005). Conclusion: A GGT <50 and/or GGT reduction of > 75% by 1 year after PSC diagnosis predicts favorable 5-year outcomes in children. GGT has promise as a potential surrogate endpoint in future clinical trials for pediatric PSC.
3121 General medicine, internal medicine and other clinical medicine
Peer reviewed: Yes
Rights: cc_by_nc_nd
Usage restriction: openAccess
Self-archived version: publishedVersion

Files in this item

Total number of downloads: Loading...

Files Size Format View
Deneau_et_al_2018_Hepatology_Communications.pdf 498.7Kb PDF View/Open

This item appears in the following Collection(s)

Show full item record