Table 3.
Studies with PAH-targeted therapy medications for SLE-PAH
| Reference | Year | N (SLE) | Baseline characteristics | Intervention | Outcome | Notes/side effects | |
|---|---|---|---|---|---|---|---|
| Robbins et al53 | 2000 | 6 | Age mPASP PVR NYHA |
26–35 years 57±9 mmHg 14±7 WU III, IV |
IV epoprostenol | Significant improvement in hemodynamics (PAP reduced by 38±21%, PVR reduced by 58±12%), and NYHA functional class from III–IV to I–II for all patients | SLE relapsed in one patient |
| Rubin et al43 | 2002 | 213 (16) | Age mPAP PVR NYHA 6MWD |
49± 16 years 55 ±16 mmHg 12.7±8.5 WU III, IV 330±74 m |
Bosentan | Significant improvement in exercise capacity (6MWD increased by 36 m in cases, decreased by 8 m in controls), NYHA functional class (42% in bosentan group), and time to clinical worsening | Double-blind, placebo-controlled trial No significant differences in side effects between groups |
| Oudiz et al45 | 2004 | 90 (25) | Age mPASP NYHA 6MWD |
54±2 years 52 ±2 mmHg II, III, IV 280±13 m |
SC treprostinil | Improved exercise capacity (6MWD increased by 25 m), dyspnea, hemodynamics (PVR decreased by 4±2 WU), and trend toward improved quality of life | Double-blind, placebo-controlled trial Patients with MCTD and SSc were also included Minor side effects (infusion site pain) only |
| Mok et al56 | 2007 | 4 | Age mPAP PVR NYHA 6MWD |
42±8 years 39.5±9.2 mmHg 8.75±5.43 WU I, II, III 442±54 m |
Bosentan | 6MWD significantly improved in 3 (+24.8 m), 6 (+26.2 m), 9 (+54 m), and 12 (+62.7 m) months | Liver toxicity in one patient, PASP assessed with TTE |
| Badesch et al44 | 2007 | 284(19) | Age mPAP PVR NYHA 6MWD |
53±15 years 47±11 mmHg 10.1 ±5.5 WU II, III, IV 342±76 m |
Sildenafil 20, 40, or 80 mg/d | Patients with CTD-associated PAH had improved exercise capacity (6MWD increased by 42 m in cases and decreased by 13 m in controls), hemodynamics, and NYHA functional class (29%–42% improvement, 5% for placebo) after 12 weeks of treatment with sildenafil 20 mg/day | 12-week double-blind study (SUPER-1) Subgroup analysis of 84 patients with CTD-associated PAH Patients with SSc and other CTDs were also included in the analysis No significant side effects |
| Shirai et al54 | 2013 | 16 (6) | Age mPAP PVR NYHA |
43 ±14 years 56 ±9 mmHg 21 ±9 WU III, IV |
IV epoprostenol | mPAP (26% reduction from baseline), PVR (41% reduction from baseline), and functional class were improved after 6 months. Treated patients had better survival (3-year survival 55% vs 6%) | Patients with MCTD and SSc were also included All patients had treatment-related side effects (headache, flushing, diarrhea) Seven of 16 patients had catheter-related infections and required admission |
Abbreviations: IV, intravenous; PAP, pulmonary artery pressure; mPAP, mean PAP; PAH, pulmonary arterial hypertension; SLE-PAH, SLE-associated PAH; SLE, systemic lupus erythematosus; SC, subcutaneous; SSc, systemic sclerosis; PASP, pulmonary artery systolic pressure; mPASP, mean PASP; PVR, pulmonary vascular resistance; NYHA, New York Heart Association; 6MWD, 6-minute walk distance; CTD, connective tissue disease; MCTD, mixed CTD; TTE, transthoracic echocardiogram; WU, Woods units.