Table 2.
Published procedures and outcomes of allo-HSCT in sJIA.
| Number of patients (references) | 13 (10–12) |
| Age at HSCT, years | 6.2 (2.7–16.8) |
| Disease duration at HSCT, years | 3.3 (1.3–15.3) |
| Donor type (N) | MUD (5); mMUD (2); MSD (5); HAPLO (1) |
| Conditioning regimen (N) | F/M/A (8); F/T/A (3) B/F/A/RTX (1); F/M/THIO/A (1) |
| GVHD prophylaxis | MMF/CsA a CYC/CsA/MMF (1); Tac/MMF (1) |
| Donor chimerism, % (N) b | 100
c (7) 89 d (1); 88 d (1); 67 d (1); 55 d (1) <10 d (1) |
| Time to stop IS, years [N] e | 0.6 (0.5–2.2) [10] Ongoing (2) |
| Follow-up post-HSCT, years | 2.0 (0.2–7.0) |
| Remission (N) | CR (11)
f PR (1) |
| Flare (N) | 2 |
| Death (N) | N = 1 |
Data is shown as median (range) unless otherwise specified. A, Alemtuzumab; B, Busulfan; CR, Complete remission; CS, Corticosteroids; CsA, Cyclosporin; CYC, Cyclophosphamide; F, Fludarabine; GVHD, Graft-versus-host disease; HAPLO, Haploidentical donor; HSCT, Hematopoietic stem cell transplantation; IS, Immunosuppression; M, Melphalan; MAS, Macrophage activation syndrome; MMF, Mycophenolate mofetil; mMUD, Mismatched unrelated donor; MSD, Matched sibling donor; MUD, Matched unrelated donor; N/A, Not available; PR, Partial remission; RIC, Reduced intensity conditioning; RTX, Rituximab; T, Treosulfan; Tac, Tacrolimus; THIO, Thiotepa.
The paper by Silva et al. mentions that “most patients received MMF and cyclosporin” but individual treatment was not specified.
Data available for N = 12 patients and timing of measurement not specified for most patients.
Performed on peripheral blood/whole blood for patients reported by Silva et al.
Performed on CD3.
Data unavailable for one patient.
One patient is in CR of his sJIA symptoms but remains with lung disease which is described as significantly improved.