SSIEM 2019: Annual Symposium of the Society for the Study of Inborn Errors of Metabolism, Rotterdam, Hollanda, 3 - 06 Eylül 2019, ss.11-12
Background: In some inherited metabolic disorders (IMD) liver transplantation (LT) offers an alternative to conven- tional treatment. Between 1990 and 2018, 34 IMD children on diet treatment received a LT at Birmingham Children's Hospital, UK.
Aim: A retrospective review examining survival, hospital admissions, growth and feeding in IMD children post LT. Methods/Case Report: Medical and dietetic records were reviewed retrospectively
Results: Subject disorders were GSD n = 8 (7 = GSD1b,1Ia), UCD neonatal n = 8 (UCDn), UCD late onset n = 6 (UCDL), MMAn=2,PAn=9,andMSUDn=1.Mediantransplant age was 3.1 y (1.8-8.6). Patients were followed up post LT for a median of 6 y (1-19). 70% (n = 24) of children survived LT (100% GSD/UCDL, 88% UCDn30% PA, 0% MMA/MSUD). From pre to post LT, weight z scores deteriorated in GSD, UCDn, and PA, while UCDL showed a small non-significant increase. Post LT, median height z scores improved slightly in GSD, but deteriorated in UCDn, UCDLand PA [pre transplant GSD (range) −1.5 (−0.8, −2.2), UCDn0.1(−1.0, 0.5), UCDL0.1 (−1.8, 1.1), PA -0.6 (−0.1, −0.6); post LT, GSD −1.1 (−2.9, 0.2), UCDn −1.2 (−2.4, −0.3), UCDL−0.8 (−1, −0.2), PA n = 1 −1.3)]. Complications post LT were rejection n = 14, perianal abscess, chest infections n = 3, second trans- plant, pleural effusion and chylothorax n = 3, diabetes n = 1, renal dialysis. n = 1. The mean number of annual hospital admissions for all conditions were: 12m pre-LT, 4/y; and 3y post LT only 1/y. Before LT all GSD, UCDn, PA and UCDL(n = 2) were tube fed meeting full nutritional require- ments. 3 y post LT, tube feeding use was decreased in all con- ditions except PA. Diet restrictions were relaxed except for PA who maintained a controlled protein intake (2g/kg). Discussion: LT prevents metabolic decompensation decreas- ing hospital admissions. Post LT faltering growth is evident, possibly due to rapid cessation of tube feeding and inade- quate nutritional intake in children who had limited experi- ence of oral feeding.
Conflict of Interest declared.