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 | [55] |
| Crovalimab | Composer | 29 | Both FACIT-F score and EORTC-QLQ-C30 improved from baseline | [56] |
| Danicopan | NCT03053102 | 10 | Significant increase of mean FACIT–Fatigue score from baseline at days 28 and day 84 | [57] |
| NCT03472885 | 12 | Mean FACIT-Fatigue score increased by 11 points at week 24 | [58] | |
| Iptacopan | NCT03439839 | 10 | The FACIT-F score improved significantly in most patients | [59, 60] |
Abbreviations: EORTC, European Organisation for Research and Treatment of Cancer-QLQ-C30 score; FACIT-F, the Functional assessment of chronic illness-Fatigue.