Clinical trial NCT02134171: Early Predictive Factors of Cardiac and Cerebral Involvement in TMA
Official title: Identification of Early Predictive Factors of Cardiac and Cerebral Involvement in Thrombotic Microangiopathies
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Thrombotic Microangiopathies
Thrombotic Thrombocytopenic Purpura
Keywords: Thrombotic microangiopathy,
haemolytic uremic syndrome,
thrombotic thrombocytopenic purpura,
ADAMTS13,
troponin,
plasma exchange.
Lead sponsor: Assistance Publique - Hôpitaux de Paris
Sponsor class: OTHER
First posted: 2014-05-09
Enrollment: 119
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: DIAGNOSTIC

Brief summary: The aim of this study is to determine the frequency of cardiac and cerebral involvements in patients with idiopathic thrombotic microangiopathies on diagnosis. Patients will be assessed for cardiac involvement (troponin Ic level and cardiac ultrasonography) and cerebral involvement (cerebral MRI). The investigators will assess whether serum troponin Ic on diagnosis can predict morbidity and mortality of patients with a thrombotic microangiopathy at the acute phase.

The primary outcome measurement is the event free survival at day 30, as defined by death, myocardial ischemia, arrhythmia, severe cerebral injury and disease exacerbation. An increase in troponin Ic on diagnosis is defined as at least one result above 0.2 ng/ml among the three daily analyses performed after TMA diagnosis.

Detailed description: After TMA diagnosis, patients will be treated in emergency according to standard National recommendations. Patient will be included in the study as soon as the diagnosis of TMA is performed.

From day 1 to day 3, specific blood tests will be performed (serum troponin Ic and brain natriuretic peptide \[BNP\]). A cardiac ultrasonography within the 4 first days and a cerebral MRI within the first 7 days after TMA diagnosis will be performed.

Our hypothesis is that an increased serum troponin Ic level on diagnosis (\> 0.2 ng/ml) is a predictive feature of cardiac events or worsening at the acute phase.

At 6 months, a control cardiac ultrasonography and cerebral MRI will be performed in patients with cardiac and/or cerebral involvement on diagnosis.

122 patients are expected to be included among 30 recruiting centres in France. The total duration of inclusions is 2.5 years, and the total duration of the study is of 3 years.

Eligibility criteria: Inclusion Criteria:

* A diagnosis of thrombotic microangiopathy on the following criteria :
* A microangiopathic haemolytic anaemia (Hb\< 12 g/dl, with presence of schistocytes on blood smear);
* A thrombocytopenia \<150 G/l;
* No associated (precipitating) disease (HIV infection, cancer, chemotherapy, transplantation) or pregnancy;
* A written consent obtained from the patient, or from a relative for patients unable to provide the informed consent (because of cerebral involvement for example);
* Affiliation at the social insurance regimen.
* Major person

Exclusion Criteria:

* A TMA associated with an associated condition: infection with HIV (HIV) in AIDS stage, , chemotherapy, malignancy, transplantation, or pregnancy.

Primary outcomes:
measure: 30-day event-free survival
timeFrame: At 30 days
Secondary outcomes:
measure: Cardiac trouble frequency and type at diagnosis
timeFrame: From day 1 to day 3 after diagnosis
measure: Cerebral trouble frequency and type at diagnosis
timeFrame: From day 1 and day 7 after diagnosis
measure: Comparison of cerebral and cardiac trouble at diagnosis between thrombotic microangiopathies type
timeFrame: Baseline
measure: Description of cardiac and cerebral sequelae at M6 and reversibility frequency of diagnosis cardiac and cerebral lesions at M6
timeFrame: At 6 months
measure: Determination of cardiac and cerebral sequelae prognostic factors at M6
timeFrame: At 6 months
Other outcomes:
Locations:
city: Paris
country: France
facility: Saint Antoine
Study officials:
affiliation: Assistance Publique
name: Paul Coppo, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: