RESEARCH QUESTION: Are somatostatin analogues (SSA) effective in decreasing the transfusion requirements in patients with hereditary hemorrhagic telangiectasia (HHT) and gastrointestinal (GI) bleeding who are refractory to APC therapy?
HYPOTHESIS SSA are effective in decreasing transfusion requirements and improving quality of life while being cost-effective.
STUDY DESIGN A phase III, randomized, open-label, parallel-group, superiority, multicenter trial.
STUDY POPULATION HHT with GI bleeding and transfusion dependency (=4 blood units and/or IV iron in the previous 26 weeks).
INTERVENTION Short-acting octreotide subcutaneously 0.1mg twice daily for a period of 26 weeks, on top of standard of care.
OUTCOME MEASURES The difference between the octreotide and observational arm in patients with a clinical relevant successful decrease in number of IV iron and blood transfusions. Clinical relevant successful decrease defined as =50% in IV iron and/or blood transfusions. Secondary outcomes: quality of life, level of fatigue, epistaxis severity, number of endoscopic APC treatments and cost-effectiveness.
SAMPLE SIZE/DATA-ANALYSIS It is conservatively hypothesized that the intervention arm with octreotide will have a success rate of 40% (40% in this arm will have a decrease of =50% in IV iron and/or blood transfusions) compared to 3.7% in the placebo arm. With an alpha-level of 0.05, a power of 0.80 a total sample size of 34 patients is required. Accounting for a 10% drop-out, 38 patients should be randomized.