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

BMC Pediatrics

Table 1 Patient characteristics

Patient number	Age (years)	Ethnicity	Inhibitors	Prior regimen	treatment duration Pre-Emicizumab	treatment duration Post-Emicizumab	Reason to transition to Emicizumab	Comorbidities	Motor activity level
1	0.4	Caucasian	none	MTP^a	0	0.7 mo	difficult venous access, parental decision	none	normal
2	2	Caucasian	none	PUP^b	0	12 mo	difficult venous access, parental decision	none	normal
3	2	Caucasian	none	PUP^b	0	0.9 mo	parental decision	none	normal
4	3	Caucasian	none	proph. FVlll^c	4 mo	25 mo	difficult venous access, parental decision	none	normal
5	6	Caucasian	none	proph. FVlll^c	4 y, 6 mo	12 mo	increasing problems with FVlll administration	autism	normal
6	9	Caucasian	none	proph. FVlll^c	8 y, 1 mo	3 mo	parental decision, better feasibility (reduction of intravenous punctures and physician visits)	linguistic development delay	normal, very careful parents
7	12	African	none	proph. FVlll^c	10 y, 4 mo	24 mo	parental decision	Juvenile idiopathic arthritis, asthma	normal
8	15	Caucasian	none	proph. FVlll^c	12 y, 7 mo	3 mo	parental decision, reduced compliance due to puberty	hyperreactive bronchitis	normal
9	20	Caucasian	none	proph. FVlll^c	16 y, 5 mo	28 mo	change of FVlll required due to availability	none	reduced
10	3	Caucasian	high-titre	proph. FVlll^c	2 mo	29 mo	inhibitors	none	normal
11	6	Caucasian	high-titre	ITI^d, FEIBA^e	3 y, 2 mo	24 mo	inhibitors	ADHD^f	high
12	9	Caucasian	high-titre	ITI^d, FEIBA^e	5 y, 5 mo	33 mo	inhibitors	none	normal
13	15	Caucasian	high-titre	failed ITI^d, FEIBA^e	11 y	40 mo	inhibitors	none	normal

^aMTP Minimally treated patient, ^bPUP Previously untreated patient, proph. ^cFVlll Prophylactic treatment with FVlll, ^dITI Immune tolerance induction, ^eFEIBA Factor Eight Inhibitor Bypassing Activity , ^fADHD Attention Deficit Hyperactivity Disorder

ISSN: 1471-2431