Table 1. Changes in NTDT-PRO T/W 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	−5.12	−4.08	−2.43	−2.14	−0.26	0.94
95% CI	−9.37 to –0.86	−5.50 to –2.66	−3.02 to –1.84	−2.58 to –1.71	−0.47 to –0.06	0.38 to 1.49
Median	−4.98	−3.56	−2.56	−1.98	−0.11	1.21
ES*	−2.33	−1.86	−1.11	−0.98	−0.12	0.43
FACIT-F FS	N/A	N/A	≥8	≥4 to <8	>−4 to <4	≤−4
N	–	–	20	15	68	22
Mean	–	–	−2.95	−1.86	−0.25	0.37
95% CI	–	–	−3.75 to –2.14	−2.70 to –1.02	−0.54 to 0.04	−0.25 to 0.99
Median	–	–	−2.93	−1.76	−0.11	0.14
ES*	–	–	−1.34	−0.85	−0.11	0.17
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	–	–	−3.51	−1.87	−0.53	0.41
95% CI	–	–	−5.03 to –1.98	−2.73 to –1.01	−0.83 to –0.23	−0.12 to 0.94
Median	–	–	−3.43	−2.06	−0.37	0.33
ES*	–	–	−1.60	−0.85	−0.24	0.19
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	–	–	−3.86	−1.31	−0.32	−0.03
95% CI	–	–	−5.20 to –2.51	−1.94 to –0.67	−0.61 to –0.03	−0.82 to 0.76
Median	–	–	−3.61	−1.47	−0.11	0.18
ES*	–	–	−1.76	−0.59	−0.15	−0.01
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	–	–	−2.95	−1.68	−0.55	0.47
95% CI	–	–	−4.73 to –1.17	−2.55 to –0.82	−0.88 to –0.21	−0.07 to 1.00
Median	–	–	−2.46	−1.76	−0.29	0.72
ES*	–	–	−1.34	−0.77	−0.25	0.21

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

ESeffect sizeFACIT-FFunctional Assessment of Chronic Illness Therapy–FatigueFSFatigue SubscaleN/Anot applicableNTDT-PROnon-transfusion-dependent beta-thalassaemia patient-reported outcomePGI-SPatient Global Impression of SeveritySF-36v2Short Form Health Survey version 2T/Wtiredness/weakness