Treatment
Current therapy mainly consists of phototherapy (10-12 hours per day) to reduce bilirubin levels, and consequently the risk to develop kernicterus. Patient's survival is dependent on the indefinite continuation of this therapy. Although initially very effective, phototherapy is inconvenient and the efficacy of this treatment is reduced with ageing due to increased thickness of skin and body surface/weight ratio; thus, patients are again at risk for kernicterus around time of puberty.
To improve effectiveness of phototherapy it is best to:
Hepatocyte transplantation (transplantation of hepatic cells, i.e. hepatocytes, rather than whole liver) has been performed on a limited number of patients as an experimental therapeutic procedure (you might find some reports in the news page). Major limitations hampering a more generalized clinical application of this option include the transient benefit in the correction of the disorder, the need of suitable sources for cell isolation and technical problems such as criopreservation of isolated cells.
Other treatments aiming at reducing bilirubin entero-hepatic circulation have been proposed including oral administration of calcium phosphate, cholestyramine and agar. During 2007 results of a clinical trial using oral administration of Orlistat have been published. These data indicated an increased fecal excretion of fat and unconjugated bilirubin with concomitant decreased levels of plasmatic unconjugated bilirubin.
Gene therapy is considered a promising experimental option. During 2017-2018 several clinical trials have been proposed (you might find more information in the Gene Therapy Clinical Trials page below)
To improve effectiveness of phototherapy it is best to:
- Change lamps after about 1,000-1,500 hours of use (approximately every four to six months)
- Keep the light source close to the body (about 15-20 centimeters, 6-8 inches)
- Maximize skin exposure to light
- Use solid white sheets
- Place reflective surfaces (mirrors and emergency blankets) around the bed (you might find more information in the Phototherapy page)
Hepatocyte transplantation (transplantation of hepatic cells, i.e. hepatocytes, rather than whole liver) has been performed on a limited number of patients as an experimental therapeutic procedure (you might find some reports in the news page). Major limitations hampering a more generalized clinical application of this option include the transient benefit in the correction of the disorder, the need of suitable sources for cell isolation and technical problems such as criopreservation of isolated cells.
Other treatments aiming at reducing bilirubin entero-hepatic circulation have been proposed including oral administration of calcium phosphate, cholestyramine and agar. During 2007 results of a clinical trial using oral administration of Orlistat have been published. These data indicated an increased fecal excretion of fat and unconjugated bilirubin with concomitant decreased levels of plasmatic unconjugated bilirubin.
Gene therapy is considered a promising experimental option. During 2017-2018 several clinical trials have been proposed (you might find more information in the Gene Therapy Clinical Trials page below)
