The aim of the study is to evaluate the efficacy of a personalized caplacizumab regimen based on ADAMTS13 activity monitoring in adult acquired thrombotic thrombocytopenic purpura (aTTP): This study is a phase II, prospective, multicenter non-inferiority single-arm study.
ADAMTS13 activity will be evaluated on day 7 after the end of daily PE and every 7 days until ADAMTS13 activity ≥ 20% is reached. In case of persistent severe ADAMTS13 deficiency (≤ 20%), caplacizumab administration could be extended for a maximum of 58 days after the end of the daily PE period and should be accompanied by an adjusted immunosuppressive therapy as needed. This duration is in accordance with the HERCULES study protocol and its SmPC.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
125
Analyse of ADAMTS13 activity in patients with aTTP treated with Caplacizumab in order to help adapting the treatment with caplacizumab in TTP patients
To evaluate the feasibility of a personalized caplacizumab regimen in aTTP based on ADAMTS13 activity monitoring assessed by a composite criteria including mortality, refractoriness and/or exacerbation at 30 days post-PE treatment
composite endpoint defined by the occurrence of at least one the following events during the 30 days post-PE treatment: death, refractoriness or exacerbation.
Time frame: 30 days post-PE treatment
Response to treatment (platelet count recovery)
to platelet count recovery (as defined by a platelet count ≥ 150 G/L with a subsequent interruption of daily PE within 5 days)
Time frame: 30 days post-PE treatment
Durable remission achievement
Occurrence of durable remission achievement (platelet count ≥ 150 G/L for ≥ 30 consecutive days following PE interruption);
Time frame: 90 days post-PE treatment
Mortality at D90 post-PE treatment
Occurrence of death within 90 days post-PE treatment
Time frame: 90 days post-PE treatment
Refractoriness at D30 post-PE treatment
Occurrence of refractoriness at D30 post-PE treatment;
Time frame: Day 30 post-PE treatment
Exacerbation at D30 post-PE treatment
Occurrence of exacerbations at D30 post-PE treatment
Time frame: Day 30 post-PE treatment
Duration of plasma exchange (PE) treatment and the associated plasma volumes
Duration of daily PE with the corresponding plasma volume
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Time frame: 30 days
Duration of plasma exchange (PE) treatment and the associated plasma
Total number of PE and the corresponding plasma volume during the full study drug treatment period
Time frame: 30 days
Occurrence of neurological sequelae treatment
Neurological assessment based on Rankin score
Time frame: Day 90 post-PE treatment
Evaluate the Quality of life
Quality of life based on global post-traumatic score (PCL-S SCALE) at baseline, D90 post-PE treatment
Time frame: Day 90 post-PE treatment
Evaluate the cost of the strategy
Costs of the patients' management (Direct hospital medical expenses, Suppléments, direct costs of home care, caplacizumab injections, rehospitalizations) of patients treated with the regimen according to the study
Time frame: Day 90 post-PE treatment
To perform a safety analysis
Occurrence of AE and SAE during the study
Time frame: 90 days post-PE treatment
Occurrence of cognitive sequelae treatment
Cognitive assessment based on MMS score
Time frame: Day 90 post-PE treatment