HEREDITARY HEMORRHAGIC TELANGIECTASIA (HHT) MedDRA version: 9.1 Level: SOC Classification code 10005329
Conditions
Sponsors
AZIENDA OSPEDALIERA OSPEDALE POLICLINICO CONSORZIALE
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: ? Patients diagnosed with HHT, in particular HHT2, for whom the severity of this disease presents a life-damaging risk ? Adult volunteer patients, in possession of full mental capacity ? Patients with frequent and long-lasting epistaxis episodes ? Patients with untreatable gastrointestinal bleeding ? Patients with hemoglobin values =65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: ? Patients with elevated risk factor for hemorrhagic diseases ? Patients with cerebral arteriovenous fistulas ? Female patients during age of fertility, during pregnancy, or breast-feeding ? Patients with severe diabetic decompensation ? Patients at elevated risk for thromboembolic disease, in particular previous episodes of brain stroke, deep venous thrombosis, pulmonary embolism and myocardial ischemia ? Patients diagnosed with current neoplasms ? Patients subjected to surgical procedures less than one month previously or who are shortly scheduled for surgery ? Patients with uncontrollable hypertension ? Patients with epilepsy ? Patients with proteinuria (> 2 gr/24h) ? Patients with chronic intestinal inflammatory disease
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: ? To evaluate the efficacy of bevacizumab for treatment of severe chronic nasal and/or gastrointestinal bleeding in HHT patients. ? To establish the most effective dosage, according to a risk/benefit ratio.;Secondary Objective: ? To assess the increase of the hemoglobulin concentration (Hb, MCH, MCHC) in the blood of HHT patients during and after the administration of bevacizumab, and the eventual necessity of transfusions. ? To monitor the evolution of pulmonary and hepatic fistulas with non-invasive procedures of proven accuracy in HHT patients during and after the period of bevacizumab administration. ? To monitor the alterations of cardiovascular parameters (arterial blood pressure, cardiac output, right and left teledyastolic volume) by means of echocardiography of HHT patients during and after administration of bevacizumab therapy.;Primary end point(s): hemoglobulin concentration,evolution of pulmonary and hepatic fistulas, cardiovascular parameters (arterial blood pressure, cardiac output, right and left teledyastolic volume) | — |
Countries
Italy
Outcome results
None listed