Health related Quality of Life in patients with inherited unconjugated hyperbilirubinemia (Crigler-Najjar syndrome): a multicentre study.
Background/aims:
Development of Novel treatment strategies, including gene therapy, evaluation of the HRQL in to evaluate the potential beneficial effects of the novel treatment strategies.
Study design/Methods or/PROMS:
- Baseline study in all and in candidates before and after treatment paper-pencil-survey, Castor database
- PROMS: General questions, SF-34 in adults and the PredsQL in children
Target population:
Patients with Crigler-Najjar syndrome and their family. Aim to include > 50 patients
Languages:
Dutch, German, French, Italian
Countries:
AmsterdamUMC and Erasmus MC (The Netherlands), Hospital Papa Giovanni XXIII Bergamo and TIGEM, University Federico II, Naples, Italy; Hannover Medical School, Germany; Hopital Antoine-Béclère Paris
Inclusion and exclusion criteria:
- Inclusion criteria: definite or probable Crigler-Najjar syndrome
- Exclusion criteria: None
- Open for inclusions: yes*
Contact details:
Dr P.J.Bosma;
Email: p.j.bosma@amsterdamumc.nl