Retrospective Study on Caplacizumab-treated Pediatric Patients With Immune-mediated Thrombocytopenic Purpura (iTTP)

Study Overview
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Brief Summary
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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.

Detailed Description
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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 .

Official Title
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Retrospective Data Collection of Pediatric Patients With Immune-mediated Thrombotic Thrombocytopenic Purpura (iTTP) Treated With Caplacizumab

Conditions
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Immune-mediated Thrombocytopenic Purpura

Intervention / Treatment
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* Drug: Caplacizumab


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patient's aged ≤18 years at start of caplacizumab treatment initiation Patient has a diagnosis of iTTP documented in the medical records Patient was treated with caplacizumab within the eligibility period Exclusion Criteria: Patient declined use of data for study (where local regulations require patient notification of planned study) Patient's medical chart is missing or not retrievable

Ages Eligible for Study
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Maximum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Intervention/Treatment |
| --- |
|Drug: Caplacizumab|as in real world practice|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 | 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 | 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) | 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 | From index date up to 12 weeks after last dose of caplacizumab | 
| Total duration of hospitalization stays |  | From index date up to 12 weeks after last dose of caplacizumab | 
| Duration of intensive care unit (ICU) stay |  | From index date up to 12 weeks after last dose of caplacizumab | 
| Duration of therapeutic PE |  | From index date up to 12 weeks after last dose of caplacizumab | 
| Proportion of patients achieving clinical response | defined as a normal platelet countand LDH < 2 ULN for at least 48 hours following initial normalization or response of platelet count | From index date up to 12 weeks after last dose of caplacizumab | 
| Time to ADAMTS13 activity ≥ 20% | where available and feasible | From index date up to 12 weeks after last dose of caplacizumab | 
| Number of participants with Adverse event | including serious adverse events | From index date up to 12 weeks after last dose of caplacizumab | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Treatment pattern of caplacizumab therapy | Dosing and duration | From index date up to 12 weeks after last dose of caplacizumab | 
| Types and duration of concomitant medications |  | From index date up to 12 weeks after last dose of caplacizumab |