Table 1 Characteristics of 25 clinical studies that provided data on 12 outcomes of interest in patients with MPSII who received intravenous infusions of ERT (Elaprase)

From: Treatment of mucopolysaccharidosis type II (Hunter syndrome): results from a systematic evidence review

Author, year	Location	Study type	Number of patients	Mean age (range)	MPS II phenotype	Number of groups	Previous ERT (%)	Enzyme dose (mg/kg)	Follow-up (months)	uGAG levels	Liver volume	Spleen volume	6-min walk test	Growth	Joint mobility	Pulmonary function	Cardiac function	Sleep disorder	Disability index/QoL^a	Safety: IRRs/SAEs	Safety: antibodies	Potential COI^b
Muenzer (2006)¹⁷	US, Europe, SA	RCT^c	96	13 (5–31)	A	3	0	0, 0.5 EOW, 0.5 W	12	•	•	•	•			•				•	•	F, A
Muenzer (2007)²⁰	US	RCT to open label	12	14 (6–20)	A	4	0	0, 0.5 EOW, 0.5 W, 1.0 W	6, 12	•	•	•	•			•	•	•		•	•	F, A
Okuyama (2010)²¹	Japan	OBS, open label	10	30 (21–53)	A	1	0	0.5 W	12	•	•	•	•		•	•	•	•		•	•
Burton (2011)²⁶	HOS^d	RET cases	63	9 (2–39)	A, S	1	0	NR	12												•	F, A
Glamuzina (2011)²⁸	England	RET cases	11	4 (<1–4)	A, S	1	0	0.5 W	5–35	•					•						•
Muenzer (2011)¹⁹	US, Europe, SA	OBS, open label ext.	94^e	15 (6–26)	A	3	100	0.5 W	>24	•	•	•	•	•	•	•			•	•	•	A
Schultze-Frenking (2011)²⁵	Europe	RET cases	18	10 (6–19)	A	2	100	0.5 W after 1 year	>24					•								A
Tylkl-Szymanska (2012)³⁸	Poland	Case^f, twins	1	0.25	S	NA	0	0.5 W	33		•
Wyatt (2012)²³	US	OBS cohort	39	10 (2–21)	A, S	1	95	Var	Var					•							•
Barbier (2013)¹⁸	Europe, SA	Muenzer^{17, 19} reanalysis	94	15 (6–26)	A	3	100	0.5 W	>24											•	•	F, A
Jones (2013)³⁰	HOS^d	OBS, open label	133	14 (8–15)	A, S	1	100	Var	Var					•
Kim (2013)³¹	Korea	OBS, open label	34	12 (3–38)	A, S	1	0	0.5 W	3–45											•	•
Sohn (2013)²²	South Korea	RCT, drug comparison^g	31	11 (6–35)	A	3	100	0.5 WE, 0.5 WH, 1.0 WH	6	•			•		•	•	•				•
Cho (2014)²⁷	South Korea	OBS, open label	32	7 (3–23)	A, S	1	100	0.5 W	>24					•
Tajima (2013)³⁷	Japan	Affected siblings^h	2	0.3, 3.0	S	NA	0	0.3–0.4 W, 0.5 W	32–33	•
Giugliani (2014)²⁴	SA, Asia, Europe	OBS, open label	28	4 (1.4–7)	A	1	0	0.5 W	12	•	•	•							•	•	•	F, A
Lampe (2014)³⁶	US, SA, Europe	RET case reviews	22	7 (1–21)	S	1	100	0.5 W	24–72	•											•	A
Lampe (2014)⁴⁰	US, SA, Europe	Case series	8	0.25 (<0.6)	NR	1	0	0.5 W	1.5–66											•		A
Patel (2014)³²	Japan, US	RCT	26	≤ 8	7 A, 19 S	1	NR	1.0 W	12					•								F, A
Tomanin (2014)³⁵	Italy	OBS, open label	27	5.3 (1–27)	A, S	1	0	0.5 W	~42	•				•								A
Zuber (2014)³⁹	Poland	RET/historical cases	13, 50ⁱ	3.6 (<1–4)	S	2	NA	0.5 W	12–288					•
Guffon (2015)²⁹	France	RCT, open label^j	52	12.4 (1.3–51)	16 A, 36 S	1	100	Mean 0.6±0.2 W	6, 12										•			F, A
Pano (2015)⁴¹	Korea	OBS, open label	28	4.0 (1.3–7.5)	NR	1	0	0.5 W	12	•	•	•								•	•	F, A
Sohn (2015)³³	Korea	RET cases	6	4.1 (3.2–5.6)	A, S	1	100	0.5 WH	5–35	•					•						•
Tanjuakio (2015)³⁴	Japan, US	OBS, open label^k	48	5 (<8) 18 (≥8)	19 A, 29 S	1	0	0.5 W	>24										•
Total										12	7	6	5	8	5	6	3	2	3	9	13	14

A, attenuated MPSII phenotype; Ab, antibody; COI, conflict of interest; E, Elaprase (idursulfase); EOW, every other week; ERT, enzyme replacement therapy; H, Hunterase (idursulfase beta); HOS, Hunter Outcome Survey; IRR, infusion-related reaction; MPS II, mucopolysaccharidosis type II; OBS, observational study; QoL, quality of life; RCT, randomized controlled trial; RET, retrospective study; S, severe MPSII phenotype; SAE, severe adverse event; uGAG, urinary glycosaminoglycan; Var, various; W, weekly.
Articles are listed alphabetically for each publication year.
^aDisability index/quality of life.
^bPotential conflicts of interest related to study funding or authors from the enzyme supplier: A, authors are paid employees and/or consultants; F, study funding.
^cRCT with placebo group.
^dHunter Outcome Survey (HOS) registry data.
^e85 patients remaining at 2 years tested using Child Health Assessment Questionnaire Disability Index Score.
^fOne affected and one unaffected twin; ERT started in affected twin while asymptomatic at 3 months. Older sister has severe phenotype (with mental retardation). Family mutation 1568 A>G not previously described.
^gRCT, single-blinded drug-controlled comparison of patients treated with Elaprase (mean 15 months) converted to idursulfase beta (Hunterase=H) after 2-week “washout” period. Denver Developmental Screen II for disability.
^hAffected siblings with severe phenotype treated beginning at 4 months and 3 years of age.
ⁱ13 patients on ERT and 50 historical never-treated patients.
^jClinical/Patient Global Impression-Improvement (CGI-I/PGI-I), KIDSCREEN-27, EuroQoL-5D for disability.
^kActivity of Daily Living questionnaire for disability.

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Table 1 Characteristics of 25 clinical studies that provided data on 12 outcomes of interest in patients with MPSII who received intravenous infusions of ERT (Elaprase)

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