Health related Quality of Life in patients with inherited unconjugated hyperbilirubinemia (Crigler-Najjar syndrome): a multicentre study.

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

Dutch, German, French, Italian

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

Study period/begin/stage: Study start: 01-11-2020
  • Open for inclusions: yes*

Contact details:
Dr P.J.Bosma;