Octreotide in Patients With GI Bleeding Due to Rendu-Osler-Weber

What Was Tested

This registered phase 2 trial tested Octreotide for anemia.

It had 1 arm (groups): Active comparator: Octreotide LAR (experimental).

The registry classes the interventions as drug.

More on the Plan

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.

Study population: Patients with ROW and symptomatic gastrointestinal bleeding telangiectasias, who are transfusion and/or endoscopy dependent: 1.

Who Took Part

15 planned participants were the target.

It was open to people of any sex aged from 18 years with no upper age limit.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: Patients with Rendu-Osler-Weber. Symptomatic gastrointestinal bleeds out of telangiectasias. Transfusion and / or endoscopy dependent:.

The full record lists 3 inclusion and 9 exclusion criteria; among the exclusions: liver cirrhosis child-pugh c or acute liver failure; severe diseases with life expectancy \< 1 year; patients with left ventricular assist devices (lvad's).

How the Study Was Run

Design: single-group and open-label.

Nothing was hidden in this open-label Octreotide study: the anemia participants and staff both knew the treatment.

Everyone received the same intervention, so there was no separate comparison group inside the trial.

It started in 2016-10 with completion listed for 2018-07.

It ran at 2 sites in Netherlands (2).

Its stated primary purpose is treatment.

What Was Measured

Primary outcome: The percentage of patients who are full responder, partial responder and non-responder at the end of the treatment period (time frame: Comparing the 6 months before inclusion and the study period (26 weeks)).

It also tracked 5 secondary outcomes, for example Change in quality of life using the Short Form (SF)-36 questionnaire and The number, type and severity of adverse events.

What This Means in Plain Language

Put simply, this is a phase 2 trial of Octreotide in 15 people with anemia.

Its size is small, so on its own it can only give an early look.

Its status is unknown; no results are posted to the registry yet, so nothing here describes outcomes.

More context: the Octreotide compound page, Anemia research.