Hereditary hemorrhagic teleangiectasia (HHT) Rendu-Osler-Weber syndrome
Conditions
Interventions
Self-administerend subcutaneous injection with 0.1 mg short-acting Octreotide
twice daily for 26 weeks.
Sponsors
Radboud Universitair Medisch Centrum
Eligibility
Age
18 Years to 99 Years
Inclusion criteria
Inclusion criteria: - Patients older than 18 years with written informed consent. - Diagnosis of HHT: either confirmed by genetic testing or the Curacao criteria (definitediagnosis). - Presence of IDA in combination with the presence endoscopic proven GI AVM manifestations / telangiectasias confirmed within the last 12 months (upper and/or lower endoscopy and/or capsule endoscopy). - Endoscopic refractory: at least 1 endoscopic APC / laser /other endoscopic treatment modality performed in the past 5 years. - Substantial transfusion dependency: at least 4 blood units and / or intravenous iron in the 6 months prior to study inclusion with a: o At least one serum ferritin below
Exclusion criteria
Exclusion criteria: - liver cirrhosis child-pugh C. - symptomatic cholecystolithiasis (possible side-effect octreotide). - pregnancy or nursing women or women having a pregnancy wish during the study period.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 'Successful response', defined as a decrease of >=50% in the amount of units intravenous iron and/or blood transfusions given. | — |
Secondary
| Measure | Time frame |
|---|---|
| The percentual or mean/median difference between the half year prior to inclusion and the treatment period of a half year between the treatment and observational arm in: - blood and intravenous iron requirements - PROM*s: quality of life (SF-36, EQ-5D), level of fatigue (MFI-20), epistaxis severity (ESS tool), and patient satisfaction - hemoglobin and ferritin levels - number of endoscopic treatments - cost-effectiveness - Safety | — |
Countries
Netherlands
Outcome results
None listed