The purpose of this study is to determine whether long-acting octreotide is safe and effective in the treatment of patients with Rendu-Osler-Weber (e.g. HHT). The study hypothesis is that octreotide is safe and will reduce transfusion requirements and endoscopy frequency in ROW patients with refractory anaemia due to bleeding gastrointestinal telangiectasias.
Rationale: Rendu-Osler-Weber (ROW) is an autosomal dominant hereditary disease which affects 1 / 5-8000 individuals. It is characterized by arteriovenous malformations (AVMs) and telangiectasias in multiple organs, including the gastrointestinal tract. Patients can be transfusion dependent due to severe gastrointestinal bleeding from those telangiectasias. Endoscopy is not as effective due to the recurrent character of the telangiectasias. Based on literature in patients with non-ROW AVMs and telangiectasias, octreotide might be beneficial for these patients to decrease their transfusion needs. Objective: To assess the efficacy of octreotide in decreasing the need for transfusions and endoscopic intervention in patients ROW with refractory anaemia due to gastrointestinal bleeding telangiectasias. Study design: Multicenter, open-label uncontrolled pilot study. Study population: Patients with ROW and symptomatic gastrointestinal bleeding telangiectasias, who are transfusion and/or endoscopy dependent: 1. Transfusion dependent: at least 2 blood and/or iron infusions in the 6 months before inclusion. 2. Endoscopy dependent: at least one endoscopic intervention with argon plasma coagulation (APC) after the initial/first endoscopic treatment after diagnosis in the half year before inclusion or unsuitable for endoscopy. Intervention: The intervention is 20 mg Sandostatin long-acting release (LAR) once every four weeks for 26 weeks on top of standard of care. Main study parameters/endpoints: Primary outcome is response to treatment defined as: * complete: no endoscopic intervention or transfusion requirements * partial: a reduction in endoscopic intervention or transfusion requirements * non-response: an equal or increase in endoscopy frequency or transfusions Important secondary outcomes are the percent change in the number of rebleeds from baseline to endpoint and the number of epistaxis episodes.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
15
Radboudumc
Nijmegen, Gelderland, Netherlands
St Antonius Hospital
Nieuwegein, Utrecht, Netherlands
The percentage of patients who are full responder, partial responder and non-responder at the end of the treatment period
Full responder: no endoscopy and no blood/iron transfusions during treatment period. * Partial responder: a decrease in number of blood/iron transfusions and/or endoscopy during the treatment period compared with the 6 months prior to inclusion. * Non-responder: no decrease in number of blood/iron transfusions and endoscopy during the treatment period compared with the 6 months prior to inclusion.
Time frame: Comparing the 6 months before inclusion and the study period (26 weeks)
The percentual decrease in blood and iron requirements
Time frame: Comparing the 6 months prior to inclusion and the treatment period of 6 months.
The percentual decrease in the number of endoscopic interventions
Time frame: Comparing the 6 months prior to inclusion and the treatment period of 6 months.
The mean/median decrease on the epistaxis severity score (ESS)
Comparing baseline and the end of treatment visit (week 26)
Time frame: Comparing the 6 months prior to inclusion and the treatment period of 6 months.
Change in quality of life using the Short Form (SF)-36 questionnaire
Time frame: Comparing baseline and end of treatment visit
The number, type and severity of adverse events
Time frame: Study period
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