Table 2.
Retrospective or prospective non-randomised studies of HSCT in NMOSD patients.
| Study [Reference] |
Greco, R., et al., 2015 [43] |
Burt, R.K., et al., 2018 [3] |
Burton, J.M., et al., 2021 [44] |
|---|---|---|---|
| Baseline features | |||
| N patients; % F | 16; 81% F | 12; 92% F | 3; 67% F |
| Age, y | 37 (20–57) | 42 (19–51) | 34 (28–39) |
| Disease duration, y | 2 (<1–17) | 7 (1–19.7) | 8.3 (3–13) |
| EDSS | 6.5 (2.0–8.5) | 4.3 (2–6.5) | 4 (3.0–4.5) |
| ARR before AHSCT | N.R. | 4.3 (2–10) | 3.4 (1.3–5) in the y pre-AHSCT |
| Anti-AQP4 ab positive | 10/13 tested (62%) | 92% | 67% |
| AHSCT protocol | |||
| Mobilisation of HSCs | Cy (2–4 g/m2) + G-CSF (+ RTX 375 mg/m2 in 2 cases) | Cy 2 g/m2 + G-CSF | Cy 2 g/m2 + RTX 375 mg/m2 + G-CSF |
| Conditioning | BEAM + ATG (n = 9); thiotepa-Cy (n = 3); Cy 200 mg/Kg + ATG (n = 4) |
Cy 200 mg/Kg + ATG + RTX 500 mgx2 + plasmapheresis 1 | Cy 200 mg/Kg + ATG + RTX 375 mg/m2 |
| Outcomes | |||
| Progression-free survival | 48% at y 3–5 | 90% at y 5 | 67% at last follow-up |
| EDSS | improved in 56% 2 | 3.0 (0–6.5) at y 5 | improved in 67% 2 |
| Relapse-free survival | 31% at y 3; 10% at y 5 |
80% at y 5 | 33% at last follow-up |
| IS-medications free | 19% | 83% | 33% |
| Anti-AQP4 ab positive | 8/8 tested (100%) | 17% | 67% |
| Severe adverse events and death | one death due to disease progression at month 14; one grade 4 neutropenia | no grade 4 toxicities | 1 death due to disease progression at y 3.5 |
| Secondary autoimmunity | 1 thyroiditis | 1 MG; 1 hyperthyroidism | N.R. |
| Follow-up duration, y | 4 (1.7–10.7) | 4.7 (2–5) | 7.5 (3.5–10) |
1 performed before hospital admission; 2 comparisons between the last follow-up and baseline. Continuous variables are reported as median/mean (range), where applicable. Abbreviations: AQP4 ab, anti-aquaporin 4 antibodies; ARR, annualised relapse rate; ATG, anti-thymocyte globulin; Cy, cyclophosphamide; G-CSF, granulocyte-colony stimulating factor; IS, immunosuppressive; MG, myasthenia gravis; N.R., not reported; RTX, rituximab; y, years.