Clinical trial NCT03369314: Observational Study of the Use of octaplasLG®.
Official title: Observational, Real-life Study of the Use of octaplasLG®.
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Thrombotic Thrombocytopenic Purpura
Lead sponsor: Octapharma
Sponsor class: INDUSTRY
First posted: 2017-12-12
Enrollment: 263
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Analyses will be carried out on all patients included, i.e. who have received at least one unit of octaplasLG®. This population will be stratified based on the different product indications.

For the population defined as that of patients with TTP, only those patients for whom the investigator has indicated a confirmed diagnosis of idiopathic TTP will be included in the efficacy analysis (see secondary objective).

Brief summary: This observational study enrolls patients who have received at least one infusion of octaplasLG®. OctaplasLG® will be administered standard of care and observation occurs during the treatment and 24 hours after the end of treatment. Characteristics of the use of octaplasLG®, tolerance criteria, and efficacy criteria will be collected.

Detailed description: Prospective, non-interventional, multicentre study meeting a post-marketing need. The data will be collected in all patients who have received at least one infusion of octaplasLG®.

The duration of follow-up is limited to the duration of treatment with octaplasLG® plus 24 h of monitoring after cessation of treatment.

Characteristics of the use of octaplasLG® (indication for the transfusion, degree of urgency, characteristics of plasma infusions, patient characteristics, number of iso-group and/or compatible units administered, tolerance criteria (adverse effects), and efficacy criteria (for patients with confirmed diagnosis of TTP--time to normalization of platelet count) will be collected.

Eligibility criteria: Inclusion Criteria:

1. The patient is treated with octaplasLG® (receiving at least 1 unit of 200 ml octaplasLG®)
2. In accordance with Reference Methodology MR-003, the patient of legal age must be individually informed through an information sheet, and must not oppose participating in this non-interventional study (with no collection of consent). If the patient is unable to receive the information and express any opposition, family members or the trusted person receive the information and must not oppose the collection of data\*. For minor patients, one of the holders of parental authority receive the information and must not oppose the collection of data.

   * However, the patient in question will be informed if his/her condition later allows.

Exclusion Criteria:

1) Patient's refusal to participate

Primary outcomes:
measure: Indication for the transfusion of octaplasLG®
timeFrame: From start of treatment to 24 hours following treatment with octaplasLG®
measure: Degree of urgency - Ordering department of octaplasLG®
timeFrame: From start of treatment to 24 hours following treatment with octaplasLG®
measure: Characteristics of the plasma infusions performed (transfusion or PE, administration rate, volume, date)
timeFrame: From start of treatment to 24 hours following treatment with octaplasLG®
measure: Patient characteristics (socio-demographic criteria, blood group, dominant pathology, co-morbidities including allergies, prior treatments)
timeFrame: From start of treatment to 24 hours following treatment with octaplasLG®
measure: Number of iso-group and/or compatible units administered for octaplasLG®
timeFrame: From start of treatment to 24 hours following treatment with octaplasLG®
Secondary outcomes:
measure: Tolerance of octaplasLG®: Adverse effects (with notion of Severity and Imputability, time to onset/start of infusion) associated with the plasma
timeFrame: From start of treatment to 24 hours following treatment with octaplasLG®
measure: Efficacy criteria in the treatment of TTP including time to normalisation of platelet count (time between first PE procedure and a confirmed platelet count > 150 x 109/L on at least 2 consecutive days)
timeFrame: From start of treatment to 24 hours following treatment with octaplasLG®
Other outcomes:
Locations:
city: Avignon
country: France
facility: CH Henri Duffaut
city: Brest
country: France
facility: Hôpital de La Cavale Blanche
city: Bron
country: France
facility: Hôpital Louis Pradel
city: Caluire-et-Cuire
country: France
facility: Infirmerie Protestante de Lyon
city: Lyon
country: France
facility: Hospices Civils de Lyon Hôpital Edouard Herriot
city: Marseille
country: France
facility: Hôpital de la Conception
city: Paris
country: France
facility: APHP Hopital Pitié salpêtrière
city: Paris
country: France
facility: APHP Hopital Cochin
city: Rouen
country: France
facility: CHU Hôpitaux de Rouen
city: Saint-Étienne
country: France
facility: CHRU Hôpital Nord
city: Suresnes
country: France
facility: Hôpital Foch
city: Toulouse
country: France
facility: Clinique Pasteur
city: Tours
country: France
facility: CHRU Bretonneau
city: Vandœuvre-lès-Nancy
country: France
facility: CHU de Nancy
Study officials:
References: