Table 1 Patients’ characteristics
Characteristic | All patients | Responder | Non Responder | p value |
|---|---|---|---|---|
Demographics | ||||
Number of patients | 32 | 19 | 13 | |
Female [no.[%)] | 20 (63) | 14 (74) | 6 (46) | 0.15 |
Median age (range) | 40 (19–57) | 39 (19–53) | 42 (19–57) | 0.51 |
Disease characteristics [mean, (range)] | ||||
Disease duration (months) | 37 (3-125) | 33 (3–87) | 43 (6-125) | 0.35 |
Anti-Scl70 positive (no.[%]) | 21 (66) | 12 (63) | 9 (77) | 0.47 |
mRSS | 20 (2–35) | 20 (3–35) | 18 [2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19,20,21,22,23,24,25,26,27,28,29,30] | 0.52 |
No. of organs involved Skin [no.[%)] Lung [no.[%)] Gastrointestinal [no.[%)] Heart [no.[%)] Musculoskeletal [no.[%)] Kidney [no.[%)] | 32 (100) 30 (94) 17 (53) 9 [27] 6 [19] 1 [3] | 19 (100) 17 (89) 8 (42) 6 (32) 3 [16] 1 [5] | 13 (100) 13 (100) 9 (69) 3 [22] 3 [22] 0 | 0.34 > 0.9 0.50 0.17 0.70 0.67 > 0.9 |
No. of prior therapies | 2.0 (0–8) | 1.6 (0–4) | 2.5 (0–8) | 0.12 |
Treatment [mean (range)] | ||||
Reinfused autologous cells Purity [%] CD34 + stem cells x106/kg CD3 + cells x104/kg | 89 (41–99) 4.5 (2.3–9.2) 0.3 (0.1–2.2) | 89 (46–99) 4.5 (2.3–9.2) 0.4 (0.1–2.2)† | 89 (41–99) 4.3 (2.3–6.9) 0.2 (0.1–0.5) | 0.97 0.56 0.11 |
Cyclophosphamide dose Mobilization therapy (g/m²) Conditioning regimen (mg/kg) | 3.2 (0–4) 188 (0-200) | 3.1 (0–4) 195 (150–200) | 3.3 (0–4) 177 (0-200) | 0.72 0.22 |
ATG dose (mg/kg) | 34 (2.5–40) | 34 (2.5–40) | 35 (2.5–40) | 0.63 |
Treatment plan including thiothepa | 6 (19%) | 4 (21%) | 2 (15%) | > 0.9 |