The purpose of this multi-country, retrospective data collection study (chart review) is to describe the effectiveness and safety of caplacizumab in pediatric patients with iTTP.
Pediatric patients who received caplacizumab will be identified for enrollment in the chart review. The eligibility period starts on August 30, 2018 in the United Kingdom (UK) and France and February 6, 2019 for the United States (US). Data collection is fully retrospective and will be anchored to the patient's index event date. The index event date is defined as the date the patient initiated caplacizumab treatment. The study period begins at the index date and ends at the earliest date of chart abstraction initiation, 12 weeks after last dose of caplacizumab treatment, date of death, or loss to follow-upwhich ever comes first .
Study Type
OBSERVATIONAL
Enrollment
4
as in real world practice
Sanofi-Aventis
Chilly-Mazarin, France
Platelet count response
defined as time from caplacizumab initiation to initial platelet count ≥ 150×109/L with subsequent stop of daily plasma exchange (PE) within 5 days
Time frame: From index date up to 12 weeks after last dose of caplacizumab
Proportion of subjects with refractory iTTP
defined as lack of doubling of platelet count after four days of caplacizumab treatment and a lactate dehydrogenase (LDH) level that remained above the upper limit of normal (ULN) range
Time frame: From index date up to 12 weeks after last dose of caplacizumab
Proportion of subjects with recurrent disease
Proportion of subjects with iTTP exacerbation (defined as recurrence within 30 days after last PE) and Proportion of subjects with iTTP relapse (defined as recurrence more than 30 days after last PE)
Time frame: From index date up to 12 weeks after last dose of caplacizumab
Time to normalization of organ damage marker levels
Defined asLDH ≤ 2 x ULN, Serum creatinine ≤ 1 x ULN, Cardiac troponin I ≤ 1 x ULN
Time frame: From index date up to 12 weeks after last dose of caplacizumab
Total duration of hospitalization stays
Time frame: From index date up to 12 weeks after last dose of caplacizumab
Duration of intensive care unit (ICU) stay
Time frame: From index date up to 12 weeks after last dose of caplacizumab
Duration of therapeutic PE
Time frame: From index date up to 12 weeks after last dose of caplacizumab
Proportion of patients achieving clinical response
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defined as a normal platelet countand LDH \< 2 ULN for at least 48 hours following initial normalization or response of platelet count
Time frame: From index date up to 12 weeks after last dose of caplacizumab
Time to ADAMTS13 activity ≥ 20%
where available and feasible
Time frame: From index date up to 12 weeks after last dose of caplacizumab
Number of participants with Adverse event
including serious adverse events
Time frame: From index date up to 12 weeks after last dose of caplacizumab
Treatment pattern of caplacizumab therapy
Dosing and duration
Time frame: From index date up to 12 weeks after last dose of caplacizumab
Types and duration of concomitant medications
Time frame: From index date up to 12 weeks after last dose of caplacizumab