. 2021 Jun 5;110(6):1435–1454. doi: 10.1002/cpt.2247

Table 1.

Included studies

	Author, year (reference)	Study design	Sample size	Follow‐up duration	Treatment	Age, mean (range)	Country	NICE/QUIPS ROB assessment
Studies in Type 1 SMA population	Aragon‐Gawinska, 2018 ⁶⁷	POS	33	6 m	Nusinersen	21.3 m (8.3–113.1) ^a	France	Low
	D’Amico, 2008 ⁶²	ROS	38	NR	Unspecified	NR	Italy	Low
	Finkel, 2017 ENDEAR ⁵³	RCT (Phase 3, DB)	122	13 m	Nusinersen vs. sham procedure	Nusinersen: 163 d (52–242) ^a Placebo: 181 d (30–262) ^a	Multinational	Low
	Pechmann, 2018 ⁶⁸	POS	61	6 m	Nusinersen	21.08 m (1–93) ^a	Germany	Low
	Dabbous, 2019 ⁶⁴	ITC	92	NR	Onasemnogene abeparvovec vs. nusinersen	Onasemnogene abeparvovec: 3.4 m (0.9–7. 9) ^a Nusinersen: 5.3 m (1.7–7.9) ^a	Multinational	High
	De Sanctis, 2018 ⁶⁹	ROS	20	NR	Unspecified	NR	Italy	High
	Feldkoetter, 2002 ⁶	ROS	113	NR	Unspecified	NR	Germany	High
	Finkel, 2014 ¹⁸	POS	79	36 m	Unspecified	Type 1b SMA (median): 2.5–184 m ^b Type 1c SMA (median): 6–78 m ^b	US	High
	Gregoretti, 2013 ⁶³	ROS	194	NR	TV vs. continuous NRA	NR	Italy	High
	Kaneko, 2017 ²⁶	ROS	47	NR	Unspecified	7 m to 57 y ^b	Japan	High
	Oskoui, 2007 ⁵⁸	POS	143	49.9 m	Unspecified		US	High
	Rudnik‐Schoneborn, 2009 ⁵⁶	ROS	66	NR	Valproic acid	NR	Germany	High
Studies in Types 2 and 3 SMA population	Bertini, 2017 ⁴⁹	RCT (Phase 2, DB)	165	24 m	Olesoxime vs. placebo	Olesoxime: 9.1 y (SD: 5.5) Placebo: 11.2 y (SD: 6.0)	Multinational	Low
	Mercuri, 2018 CHERISH ⁵²	RCT (Phase 3, DB)	126	15 m	Nusinersen vs. sham	Nusinersen: 4 y (range: 2–9) Sham: 3 y (range: 2–7)	Multinational	Low
	Mazzone, 2013 ⁷⁴	POS	38	12 m	Unspecified	14.07 y (SD: 12.43)	Europe (6 countries)	Low
	Mercuri, 2016 ⁷³	ROS	268	12 m	Unspecified	10.65 y (range: 2.5–55.5)	US, Italy, UK, Belgium	Low
	Montes, 2018 ⁷⁰	POS	73	108 m	Unspecified	13.5 y (SD: 12.4)	US, Italy, UK	Low
	Pera, 2019 ⁷¹	POS	114	12 m	Unspecified	13.3 y (SD: 10.1)	US, Italy, UK	Low
	Kaufmann, 2012 ⁶⁶	POS	79	25 m	Unspecified	11.3 y (SD: 9.4)	US	High
Studies in a mixed SMA population (Types 1, 2 and 3 SMA)	Farrar, 2013 ²⁸	ROS	70	NR	Unspecified	NR	Australia	Low
	Swoboda, 2005 ⁶⁵	POS	89	NR	Unspecified	Type 1 SMA: 18.9 m (1.08–263) ^b Type 2 SMA: 87.1 m (0.43–589) ^b Type 3 SMA: 146.6 m (28.4–604.8) ^b	US	Low
	Qu, 2015 ⁵⁴	POS	232	NR	Unspecified	NR	China	Low
	Vuillerot, 2013 ⁷²	ROS	112	21.6 m	Unspecified	Type 2 SMA: 11.5 y (SD: 5) Type 3 SMA: 18.7 y (SD: 12.3)	France, Belgium, Switzerland	Low
	Belter, 2018 ⁵⁹	ROS	1966	NR	Unspecified	NR	US	High
	Bladen, 2014 ¹⁷	ROS	5068	NR	Unspecified	NR	North America, Australasia, Europe	High
	Chung, 2004 ³⁰	POS	83	NR	Unspecified	Type 2 SMA: 0.96 y (SD: 0.58) Type 3a SMA: 1.31 y (SD: 0.62) Type 3b SMA: 6.93 y (SD: 8.03)	Hong Kong	High
	Ge, 2012 ⁵⁷	ROS	237	6 m	Unspecified	Type 1 SMA: 5.3 y (SD: 4.7) ^b Type 2 SMA: 12.8 y (SD: 10.0) ^b Type 3 SMA: 32.8 y (SD: 27.7) ^b	China	High
	Madrid Rodríguez, 2015 ⁶⁰	ROS	37	NR	Unspecified	NR	Spain	High
	Mannaa, 2009 ⁶¹	ROS	40	NR	Unspecified	NR	US	High
	Petit, 2011 ⁵⁵	ROS	103	NR	Unspecified	NR	France	High
	Yuan, 2015 ⁹⁸	ROS	132	49 m	Unspecified	Type 1 SMA: 7.8 y (SD: 5.7) ^b Type 2 SMA: 36.2 y (SD: 23.0) ^b Type 3 SMA: 110.3 y (SD: 63.2) ^b	China	High

All studies included in the systemic review. Studies were divided into Type 1 and Types 2 and 3 SMA populations and mixed SMA populations that reported on patients with Types 1, 2 and 3 SMA.

d, days; DB, double‐blind; ITC, indirect treatment comparison; m, months; NICE, National Institute of Health and Care Excellence; NR, not reported; NRA, non‐invasive respiratory muscle aid; POS, prospective observational study; QUIPS, Quality in Prognosis Studies; RCT, randomized controlled trial; ROB, risk of bias; ROS, retrospective observational study; SMA, spinal muscular atrophy; SD, standard deviation; TV, tracheostomy and invasive mechanical ventilation; y, years.

^{^a}

Age at treatment initiation.

^{^b}

Age at enrollment/first visit.