Long-term safety and outcomes in hereditary tyrosinaemia type 1 with nitisinone treatment: a 15-year non-interventional, multicentre study
This study (n=339; median nitisinone treatment duration of 11.2 years) found low incidences of hepatic, renal, ophthalmic, haematological, or cognitive or developmental adverse events. The majority (87%) had good overall clinical condition throughout treatment.
Source:
The Lancet Diabetes & Endocrinology
SPS commentary:
A related commentary notes that hereditary tyrosinaemia type 1, a disorder of tyrosine degradation, has a high morbidity and mortality in the early years of life, primarily due to life-threatening hepatic disease including liver failure and hepatocellular carcinoma. Previously, management of children with this condition was limited to dietary restriction of phenylalanine and tyrosine, which was remarkably ineffective, followed by liver transplantation. Since the EU approval of nitisinone in 2005, prognosis for patients with hereditary tyrosinaemia type 1 has changed dramatically, with patients living with the disease now reaching adulthood for the first time in history.
However, although nitisinone has become the standard of care for hereditary tyrosinaemia type 1, long-term follow-up data on safety are still needed to determine the potential consequences of prolonged nitisinone use. These data provide some reassurance, with no new safety signals reported, and a suggestion that starting treatment early can help to prevent life-limiting hepatic disease (no cases of liver transplantation or death in those who started treatment at age <28 days v 13% in those who started treatment later).