Estimation Of The Quality Of Life Benefits Associated With Treatment For Spinal Muscular Atrophy

Figures & data

Figure 1 Study methods flow chart.

Figure 2 Example case study description (SMA Type I baseline).

Table 1 Type I Health States And Associated HRQoL Scores

Health State	N=5
	EQ-5D	PedsQoL NMM
	Mean (SD)	Domain	Mean (SD)
Baseline	−0.12 (0.19)	About	29.6 (6.5)
		Communication	9.4 (8.8)
		Family	24.1 (13.9)
Worsened	−0.24 (0.14)
Improvement	−0.17 (0.17)
Sits without support^a	−0.04 (0.12)
Stands with assistance	0.04 (0.09)
Walks with assistance^b	0.52 (0.22)
Stands/walks unaided^b,c	0.71 (0.14)
SMA after scoliosis surgery	−0.22 (0.22)
Gastric/NG tube	−0.17 (0.17)
Requires ventilation	−0.33 (0.27)

Notes: ^aReclassified as Type II. ^bDenotes health states where two index scores were calculated by one of the participants. ^cReclassified as Type IIIa.

Table 2 Type II Health States And Associated HRQoL Scores

Health State	N=5
	EQ-5D	PedsQoL NMM
	Mean (SD)	Domain	Mean (SD)
Baseline	0.04 (0.10)	About	43.8 (7.3)
		Communication	56.7 (16.0)
		Family	44.0 (8.0)
Worsened	−0.13 (0.06)
Mild improvement	0.04 (0.11)
Moderate improvement	0.10 (0.09)
Stands/walks with assistance*	0.39 (0.29)
Stands/walks unaided*	0.72 (0.12)
Loss of ambulation with/without assistance*	−0.12 (0.16)

Note: *Denotes health states where two index scores were calculated for one of the participants.