Managing Fatigue in Patients with Paroxysmal Nocturnal Hemoglobinuria: A Patient-Focused Perspective

Figures & data

Table 1 Quality of Life (QoL) and Paroxysmal Nocturnal Hemoglobinuria (PNH) Therapy: Eculizumab and Ravulizumab

Drug	Study Name	Number of Participants	Main Message	Ref
Eculizumab	Triumph	87 (43 eculizumab)	FACIT-F score significantly increased from baseline in eculizumab arm	[^Citation16, ^Citation51]
	Shepherd	97	Both FACIT-F score and EORTC-QLQ-C30 improved from baseline to week 52	[^Citation14, ^Citation51]
	Japanese experience	54	Both FACIT-F score and EORTC-QLQ-C30 meaningfully increased	[^Citation52]
Ravulizumab	301	246 (125 ravulizumab, 121 eculizumab)	Improvement in FACIT-F score and EORTC-QLQ-C30 similar in the ravulizumab and eculizumab group	[^Citation33]
	302	195 (97 ravulizumab, 98 eculizumab)	Change in FACIT-F score from the baseline significantly higher in the ravulizumab group; change in EORTC-QLQ-C30 similar in both groups	[^Citation29]
	302 extension	191 (96 ravulizumab, 95 eculizumab to ravulizumab)	Mean FACIT-F score remained stable through study period; mean EORTC-QLQ-C30 was similar in both groups	[^Citation53]
	302s	95	Ravulizumab preferred over eculizumab by a significant proportion of patients	[^Citation54]

Abbreviations: EORTC, European Organisation for Research and Treatment of Cancer-QLQ-C30 score; FACIT-F, the Functional assessment of chronic illness-Fatigue.

Table 2 Quality of Life (QoL) and Paroxysmal Nocturnal Hemoglobinuria (PNH) Therapy: New Drugs

Drug	Study Name	Number of Participants	Main Message	Ref
Pegcetacoplan	Pegasus	80 (41 pegcetacoplan, 39 eculizumab)	FACIT-F score increased from baseline in pegcetacoplan arm	[^Citation55]
Crovalimab	Composer	29	Both FACIT-F score and EORTC-QLQ-C30 improved from baseline	[^Citation56]
Danicopan	NCT03053102	10	Significant increase of mean FACIT–Fatigue score from baseline at days 28 and day 84	[^Citation57]
	NCT03472885	12	Mean FACIT-Fatigue score increased by 11 points at week 24	[^Citation58]
Iptacopan	NCT03439839	10	The FACIT-F score improved significantly in most patients	[^Citation59, ^Citation60]

Abbreviations: EORTC, European Organisation for Research and Treatment of Cancer-QLQ-C30 score; FACIT-F, the Functional assessment of chronic illness-Fatigue.

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