Somatostatin analogues, somatostatine analoga, SSA, Octreotide, Thalidomide, Bevacizimab, transfusion requirement, transfusiebehoefte, hereditary hemorrhagic telangiectasia, Hereditaire hemorragische teleangië
Conditions
Interventions
STANDARD OF CARE
- Iron supplementation:
o Occult gastrointestinal bleeding with iron deficiency anemia: oral iron supplementation
o Acute gastrointestinal bleeding with hospitalization or when ora
Sponsors
Radboudumc
Eligibility
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 (definite diagnosis). - 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 < 30 ug/l within the last 6 months requiring iron infusion above or equal to 1 g and/or o Hemoglobin below 5.6 mmol/l (9.0 g/dl) or are in need of transfusions due to anemia related symptoms within the last 6 months requiring blood transfusion above.
Exclusion criteria
Exclusion criteria: - liver cirrhosis child-pugh C. - symptomatic cholecystolithiasis (possible side-effect octreotide). - pregnancy or nursing women or women have a pregnancy wish during the study period.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| ¡®Successful response¡¯, defined as a decrease of ¡Ý50% in the amount of units intravenous iron and/or blood transfusions given between the number of transfusions at baseline (26 weeks prior to study inclusion) and during the treatment study period (26 weeks). | — |
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 (multidimensional fatigue inventory (MFI)-20), epistaxis severity (ESS tool), and patient satisfaction - hemoglobin and ferritin levels - number of endoscopic treatments - cost-effectiveness - Safety: All adverse events occurring during the study will be recorded in the patient's medical records. | — |
Contacts
Public ContactE.J.M. van Geenen
Department of Gastroenterology & Hepatology - Radboud University Medical Center
Outcome results
None listed