Emicizumab in children: bleeding episodes and outcome before and after transition to Emicizumab.

ABSTRACT:

Purpose

Real-world data and study data regarding therapy with Emicizumab in pediatric cohorts with haemophilia A is scarce. Especially, data on previously untreated pediatric patients (PUPs) and minimally treated patients (MTPs) are missing.

Methods

Thirteen pediatric patients with haemophilia A and treatment with Emicizumab were retrospectively evaluated for Annual Bleeding Rates (ABR) pre-and post-Emicizumab treatment. Safety data and data on management of minor surgery as well as laboratory results were collected. Additionally, we describe the clinical features of two PUPs and one MTP that are included in our cohort.

Results

Median age at initiation of Emicizumab was 5.3 (range: 0.26-17.5) years, three patients were younger than one year at initiation of treatment

SUBMITTER: Glonnegger H 

PROVIDER: S-EPMC9377120 | biostudies-literature | 2022 Aug

REPOSITORIES: biostudies-literature