From: The need for a holistic approach for SSc-ILD – achievements and ambiguity in a devastating disease
Publication | No. patients included | Treatment | Primary endpoint | Secondary endpoint |
---|---|---|---|---|
Distler et al. NEJM 2019 [28] | 576 | Phase III Nintedanib 150 mg × 2 | Annual rate of Decline in FVC | Absolute changes in: • mRSS • SGRQ at week 52 • FVC (ml) • %DLCO • Net digital ulcer burden • HAQ-DI • FACIT–Dyspnea questionnaire Annual rate of decline in %FVC Time to death from any cause |
Sircar et al. Rheumatology 2018 [20] | 64 | Phase II Monthly pulses of CYC 500 mg/m2 or RTX 1000 mg × 2 doses at 0, 15 days | %FVC at 6 months | Absolute change in: • FVC (l) • mRSSa • 6MWTD • Medsgers scorea • New onset/ worsening of pulmonary hypertension |
Hsu et al. J Rheumatol 2018 [43] | 23 | Phase II Pomalidomide 1 mg q.d. | FVC Total UCLA SCTC GIT V2.0 score mRSS | BDI/TDI Pulse oximetry (SpO2) UCLA SCTC GIT 2.0 subscale scores SHAQ |
Khanna et al. Ann Rheum. Dis. 2017 [22] | 87 | Phase II Tocilizumab 162 mg sc | mRSS | %FVC %DLCO VAS (Clinician Global) HAQ-DI, Patient Global VAS FACIT-Fatigue Score Pruritus 5-D Itch Scale. |
Tashkin et al. LRM 2016 [16] | 142 | Phase II Oral CYC 2 mg/kg/day or MMF 1500 mg b.i.d. | %FVC at 24 months | %DLCO TDI mRSS LCQ Change in HRCT extent |
Khanna et al. J Rheumatol 2016 [27] | 63 | Phase II Pirfenidone 801 mg t.i.d. | Safety | UCLA SCTC GIT V2.0 score FVC DLCO Mahlers dyspnea score TDI HAQ-DI PtGA mRSS |
Burt et al. Lancet Resp. Med. 2011 [44] | 19 | Phase II HSCT vs. monthly pulses of IV CYC 1 g/m2 | mRSS, 25% decrease FVC, 10% improvement | CT volume of lung disease DLCO TLC |
Spiera et al. Ann Rheum Dis 2011 [45] | 30 | Phase IIa Imatinib 400 mg q.d. | mRSS | FVC DLCO SF36 SHAQ-DI VAS (global, SOB, pain, Raynaud) |
Seibold et al. Arthrit. Rheum. 2010 [46] | 163 | Phase II Bosentan 125 (62.5 mg) mg b.i.d. | 6MWTD | Death FVC DLCO BDI mRSS Medsgers score SHAQ-DI VAS |
Denton et al. Arthrit. Rheum. 2007 [47] | 45 | Phase I/II Recombinant Human Anti–Transforming Growth Factor Antibody Therapy (CAT-192) | mRSS | FVC TLC DLCO HAQ VAS (global, Raynaud, lung disease, GO disease, digital ulcers) |
Tashkin et al. NEJM 2006 [14] | 158 | Phase II Oral CYC 1–2 mg/kg q.d. | FVC | DLCO TLC HAQ SF36 Mahler dyspnea score VAS breathing |
Hoyles et al. Arthrit. Rheum. 2006 [15] | 45 | Phase II 20 mg oral prednisolone (alternate days), 6 monthly IV pulses of CYC 600 mg/m2 followed by AZA 2.5 mg/kg/day | FVC DLCO | Dyspnea score Change in HRCT extent and pattern |
Nadashkevich et al. Clin Rheumatol. 2006 [48] | 60 | Phase I/II Oral CYC 2 mg/kg for 12 months, then 1 mg/kg for 12 months vs. Aza 2.5 mg/kg for 12 months, then 2 mg/kg for 18 months | mRSSb Raynaud frequency %FVC %DLCO | |
Binks et al. Ann. Rheum. Dis. 2001 [49] | 41 | Phase I/II HSCT | Mortality Disease progression | mRSS VC DLCO LVEF by echocardiography |