Immunogenicity of idursulfase and clinical outcomes in very young patients (16 months to 7.5 years) with mucopolysaccharidosis II (Hunter syndrome)

Pano, Arian; Barbier, Ann J; Bielefeld, Bonnie; Whiteman, David AH; Amato, David A

doi:10.1186/s13023-015-0265-2

Orphanet Journal of Rare Diseases

Table 2 SAEs by patient

From: Immunogenicity of idursulfase and clinical outcomes in very young patients (16 months to 7.5 years) with mucopolysaccharidosis II (Hunter syndrome)

Patient information			SAE information
ID	Genotype group	Ab status overall	Preferred term	Relationship to study drug	IRAE	Included in landmark analyses? ^a
1	FS/SSM	PNAb+	Bronchopneumonia	Not related	No	Yes
1	FS/SSM	PNAb+	Bronchospasm	Not related	No	Yes
2	CD/LR	PAb+	Respiratory distress	Probably related	Yes	No^b
2	CD/LR	PAb+	Pulmonary hypertension	Not related	No	Yes
3	MS	Ab−	Bronchopneumonia	Not related	No	Yes
			Hypoxia	Not related	No	Yes
			Respiratory distress	Not related	No	Yes
4	FS/SSM	PNAb+	Microcytic anemia	Not related	No	Yes
4	FS/SSM	PNAb+	Pneumonia	Not related	No	Yes
5	CD/LR	PNAb+	Rash	Probably related	Yes	No^b
			Pyrexia	Probably related	Yes	No^b
			Pyrexia	Probably related	Yes	Yes
			Pyrexia	Probably related	Yes	No^c
			Pyrexia	Probably related	Yes	No^c
			Irritability^d	Not related	No	Yes
6	MS	Ab−	Skin hypertrophy	Not related	No	Yes
7	CD/LR	PNAb+	Urticaria	Possibly related	Yes	No^b
			Pyrexia	Possibly related	Yes	No^b
			Urticaria	Possibly related	Yes	No^b
			Respiratory tract infection	Not related	No	Yes
			Respiratory tract infection	Not related	No	No^c
			Ear infection	Not related	No	Yes
			Viral pharyngitis	Not related	No	Yes
			Pneumonia	Not related	No	Yes
			Urticaria	Not related	No	Yes
			Atonic seizures	Not related	No	Yes
			Edema peripheral	Not related	No	Yes
8	FS/SSM	Ab+	Gastrointestinal infection^d	Not related	No	Yes
9	Unknown	PNAb+	Food poisoning^d	Not related	No	Yes
			Upper respiratory tract infection	Not related	No	Yes
			Muscle contracture	Not related	No	Yes
10	CD/LR	PNAb+	Pyrexia^d	Probably related	Yes	Yes
11	CD/LR	PNAb+	Asthma	Not related	No	Yes
			Bronchopneumonia	Not related	No	Yes
			Otitis media	Not related	No	Yes
12	CD/LR	PNAb+	Hematoma	Not related	No	Yes
13	FS/SSM	PNAb+	Catheter site hematoma	Not related	No	No^b
13	FS/SSM	PNAb+	Otitis media	Not related	No	Yes

Ab+, antibody positive; Ab−, antibody negative; CD/LR, complete deletion/large rearrangement; FS/SSM, frameshift/splice site mutation; IRAE, infusion-related adverse event; landmark, estimated time point of change in Ab status; MS, missense mutation; PAb+, persistently antibody positive; PNAb+, persistently neutralizing antibody positive, SAEs, serious adverse events.
^aOnly 28 of the 38 total SAEs that occurred during HGT-ELA-038 were included in the landmark analyses investigating the relationship between Ab status, genotype, and risk of SAEs. SAEs were excluded from the landmark analyses if they occurred before the landmark point (the estimated time point of change in Ab status; see Methods) or if they were not the first occurrence of an SAE for a given patient.
^bOccurred before the landmark point.
^cNot the first occurrence for this patient.
^dPatients were hospitalized for social reasons (i.e., convenience) rather than for medical reasons.

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ISSN: 1750-1172

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