Table 2. Changes in NTDT-PRO SoB domain scores by anchor response category from baseline to weeks 13–24.

Anchor and statistic	Improvement				No change	Worsening by ≥1 level
	4 levels	3 levels	2 levels	1 level
PGI-S	≤−4	>−4 to ≤−3	>−3 to ≤−2	>−2 to ≤−1	>−1 to <1	≥1
N	3	7	10	15	73	23
Mean	−4.40	−3.75	−1.59	−1.40	−0.14	0.94
95% CI	−7.54 to –1.26	−5.00 to –2.51	−2.55 to –0.63	−1.90 to –0.90	−0.34 to 0.07	0.43 to 1.45
Median	−4.49	−3.34	−1.97	−1.49	−0.01	1.19
ES*	−1.92	1.63	−0.69	−0.61	−0.06	0.41
FACIT-F FS	N/A	N/A	≥8	≥4 to <8	>−4 to <4	≤−4
N	–	–	20	15	68	22
Mean	–	–	−2.37	−1.33	−0.06	0.20
95% CI	–	–	−3.14 to –1.61	−2.19 to 0.48	−0.36 to 0.23	−0.30 to 0.70
Median	–	–	−2.17	−1.09	−0.02	0.30
ES*	–	–	−1.03	−0.58	−0.03	0.09
FACIT-F item HI12	≥4	≥3 to <4	≥2 to <3	≥1 to <2	>−1 to <1	≤−1
N	0	0	10	18	71	26
Mean	–	–	−2.90	−1.25	−0.32	0.27
95% CI	–	–	−4.22 to –1.58	−2.21 to –0.30	−0.62 to –0.03	−0.18 to 0.71
Median	–	–	−2.44	−1.53	−0.07	0.28
ES*	–	–	−1.26	−0.55	−0.14	0.12
FACIT-F item An2	≥4	≥3 to <4	≥2 to <3	≥1 to <2	>−1 to <1	≤−1
N	0	0	11	23	71	20
Mean	–	–	−3.10	−0.93	−0.19	0.07
95% CI	–	–	−4.48 to –1.73	−1.55 to –0.32	−0.48 to 0.10	−0.56 to 0.69
Median	–	–	−2.92	−0.83	−0.07	0.29
ES*	–	–	−1.35	−0.41	−0.08	0.03
SF-36v2 vitality	N/A	N/A	≥13.4	≥6.7 to <13.4	>−6.7 to <6.7	≤−6.7
N	–	–	9	19	81	15
Mean	–	–	−2.04	−1.34	−0.37	0.43
95% CI	–	–	−3.69 to –0.40	−2.19 to –0.49	−0.68 to –0.06	−0.13 to 1.00
Median	–	–	−1.49	−1.70	−0.12	0.41
ES*	–	–	−0.89	−0.58	−0.16	0.19

^*ES was calculated as the mean change from baseline within the group divided by the overall SD of the NTDT-PRO SoB score at baseline.

ES, effect size; FACIT-F, Functional Assessment of Chronic Illness Therapy–Fatigue; FS, Fatigue Subscale; N/A, not applicable; NTDT-PRO, non-transfusion-dependent beta-thalassaemia patient-reported outcome; PGI-S, Patient Global Impression of Severity; SF-36v2, Short Form Health Survey version 2; SoB, shortness of breath