Clinical and hematological features of patients with IAA investigated at diagnosis before any treatment
| Patients with IAA . | Sex/age . | Hb g/L . | ANC (×10−9/L) . | Platelets (×10−9/L) . | Retics (×10−9/L) . | LDH (×UNL)* . | GPI(−)N (%)† . | IAA severity‡ . | Treatment . | Response/maintenance . | T cells (%) . | ||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| GPI-specific¶ . | IFN-γ producing§ . | GPI-reactive IFN-γ producing‖ . | |||||||||||
| 1AA | F/33 | 8.5 | 250 | 6 | 13 | 0.8 | <0.001 | SAA | hATG+CyA | CR/no | 0.80 | 2.67 | 2.19 |
| 2AA | M/19 | 4.5 | 590 | 12 | 37 | 0.8 | <0.001 | SAA | hATG+CyA | CR/no | 1.90 | 6.00 | 5.90 |
| 4AF | M/25 | 7.4 | 200 | 17 | 7 | 0.9 | 1.36 | SAA | hATG+CyA | CR/no** | 0.50 | 3.90 | 3.50 |
| 5AF | F/72 | 6.7 | 550 | 7 | 28 | 1.0 | 4.50 | NSAA | No therapy | na/no | <0.004 | 0.43 | 0.20 |
| 6AF | F/56 | 8.5 | 520 | 14 | 32 | 0.9 | 3.50 | NSAA | hATG+CyA | NR/no | <0.004 | 0.89 | 0.19 |
| 7AF | F/23 | 9.6 | 350 | 4 | 18 | 0.8 | 0.50 | SAA | hATG+CyA | CR/no | 0.95 | 5.60 | 4.30 |
| 72F | F/18 | 5.2 | 378 | 16 | 43 | 2.2 | 60.00 | NSAA | hATG+CyA | CR/no | 0.98 | 1.44 | 1.03 |
| 11J | F/58 | 7.6 | 1000 | 3.5 | 38 | 0.7 | <0.001 | NSAA | hATG+CyA | PR/CyA | 0.60 | 9.80 | 7.15 |
| 17J | M/77 | 7.2 | 470 | 12 | 10 | 0.9 | 0.008 | SAA | rATG+CyA | PR/CyA | 1.80 | 1.90 | 1.7 |
| 20J | F/68 | 6.8 | 540 | 16 | 34 | 0.9 | 0.096 | SAA | rATG+CyA | PR/CyA | 3.30 | 6.60 | 6.4 |
| 21J | F/82 | 7.5 | 1230 | 33 | 35 | 0.9 | 0.045 | NSAA | CyA | NR/CyA | 1.40 | 1.90 | 0.9 |
| 22J | F/82 | 6.8 | 542 | 13 | 45 | 1.0 | 0.11 | NSAA | no therapy | na/no | 0.80 | 3.60 | 2.75 |
| 24J | M/31 | 5.3 | 741 | 19 | 31 | 1.6 | <0.001 | NSAA | no therapy | na/no | <0.004 | 3.10 | 0.15 |
| 25J | M/79 | 7.8 | 945 | 17 | 49 | 1.0 | 0.001 | NSAA | no therapy | na/no | 0.70 | 3.00 | 0.95 |
| 28J | M/61 | 12.6 | 940 | 49 | 36 | 0.8 | 0.007 | NSAA | CyA | CR | 0.40 | 1.30 | 0.50 |
| 29J | M/60 | 5.7 | 624 | 4 | 34 | 0.9 | 9.10 | NSAA | rATG+CyA | PR/no | <0.004 | 3.00 | −1.20 |
| 30J | F/51 | 12.2 | 410 | 24 | 60 | 1.1 | 0.015 | NSAA | CyA | PR/CyA | 1.20 | 4.70 | 3.6 |
| Patients with IAA . | Sex/age . | Hb g/L . | ANC (×10−9/L) . | Platelets (×10−9/L) . | Retics (×10−9/L) . | LDH (×UNL)* . | GPI(−)N (%)† . | IAA severity‡ . | Treatment . | Response/maintenance . | T cells (%) . | ||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| GPI-specific¶ . | IFN-γ producing§ . | GPI-reactive IFN-γ producing‖ . | |||||||||||
| 1AA | F/33 | 8.5 | 250 | 6 | 13 | 0.8 | <0.001 | SAA | hATG+CyA | CR/no | 0.80 | 2.67 | 2.19 |
| 2AA | M/19 | 4.5 | 590 | 12 | 37 | 0.8 | <0.001 | SAA | hATG+CyA | CR/no | 1.90 | 6.00 | 5.90 |
| 4AF | M/25 | 7.4 | 200 | 17 | 7 | 0.9 | 1.36 | SAA | hATG+CyA | CR/no** | 0.50 | 3.90 | 3.50 |
| 5AF | F/72 | 6.7 | 550 | 7 | 28 | 1.0 | 4.50 | NSAA | No therapy | na/no | <0.004 | 0.43 | 0.20 |
| 6AF | F/56 | 8.5 | 520 | 14 | 32 | 0.9 | 3.50 | NSAA | hATG+CyA | NR/no | <0.004 | 0.89 | 0.19 |
| 7AF | F/23 | 9.6 | 350 | 4 | 18 | 0.8 | 0.50 | SAA | hATG+CyA | CR/no | 0.95 | 5.60 | 4.30 |
| 72F | F/18 | 5.2 | 378 | 16 | 43 | 2.2 | 60.00 | NSAA | hATG+CyA | CR/no | 0.98 | 1.44 | 1.03 |
| 11J | F/58 | 7.6 | 1000 | 3.5 | 38 | 0.7 | <0.001 | NSAA | hATG+CyA | PR/CyA | 0.60 | 9.80 | 7.15 |
| 17J | M/77 | 7.2 | 470 | 12 | 10 | 0.9 | 0.008 | SAA | rATG+CyA | PR/CyA | 1.80 | 1.90 | 1.7 |
| 20J | F/68 | 6.8 | 540 | 16 | 34 | 0.9 | 0.096 | SAA | rATG+CyA | PR/CyA | 3.30 | 6.60 | 6.4 |
| 21J | F/82 | 7.5 | 1230 | 33 | 35 | 0.9 | 0.045 | NSAA | CyA | NR/CyA | 1.40 | 1.90 | 0.9 |
| 22J | F/82 | 6.8 | 542 | 13 | 45 | 1.0 | 0.11 | NSAA | no therapy | na/no | 0.80 | 3.60 | 2.75 |
| 24J | M/31 | 5.3 | 741 | 19 | 31 | 1.6 | <0.001 | NSAA | no therapy | na/no | <0.004 | 3.10 | 0.15 |
| 25J | M/79 | 7.8 | 945 | 17 | 49 | 1.0 | 0.001 | NSAA | no therapy | na/no | 0.70 | 3.00 | 0.95 |
| 28J | M/61 | 12.6 | 940 | 49 | 36 | 0.8 | 0.007 | NSAA | CyA | CR | 0.40 | 1.30 | 0.50 |
| 29J | M/60 | 5.7 | 624 | 4 | 34 | 0.9 | 9.10 | NSAA | rATG+CyA | PR/no | <0.004 | 3.00 | −1.20 |
| 30J | F/51 | 12.2 | 410 | 24 | 60 | 1.1 | 0.015 | NSAA | CyA | PR/CyA | 1.20 | 4.70 | 3.6 |
ANC, absolute neutrophil count; CR, complete remission; CyA, cyclosporine A; GPI(−), GPI negative; hATG, equine anti-thymocyte globulin; Hb, hemoglobin; N, neutrophils; na, not applicable; NR, no response; Plts, platelets; PR, partial remission; rATG, rabbit anti-thymocyte globulin; Retics, reticulocytes.
Because lactate dehydrogenase (LDH) reference values are different for different laboratories, we are reporting LDH level as the ratio between the actual value and the upper normal level (×UNL).
The percentage of PNH N reflects the relative size of the PNH cell population more accurately than the percentage of PNH red blood cells, because the latter will be grossly underestimated as consequence of selective hemolysis; this may be further complicated by blood transfusion.
The severity of IAA (severe [SAA] vs nonsevere [NSAA]) has been classified according to Camitta’s criteria.25
GPI-specific T cells: CD8+ T cells that bind human CD1d dimer loaded with human GPI molecule on culture on APC competent for GPI synthesis.
IFN-γ-producing T cells: IFN-γ-producing CD8+ T cells on culture on APC competent for GPI synthesis.
GPI-reactive IFN-γ-producing T cells: difference between the frequency of CD8+IFN- -producing T cells observed after culture on GPI-positive APC and the frequency of CD8+ IFN-γ-producing T cells observed after culture on GPI-negative APC.
Relapse after more than 1 year, followed by allogeneic hematopoietic stem cell transplantation. At relapse, the frequencies of GPI-specific, GPI-reactive, and ∆IFN-γ+ T cells was 0.9%, 2.65%, and 1.25%, respectively.