Transplant characteristics and outcome
| Patient no. . | HLA donor/ conditioning . | Source: CD34 (×106/kg); CD3 (×108/kg) . | Ne D > 0.5 × 109/kg . | Chimerism at last follow-up . | Complications . | Time to stop IS . | Outcome . |
|---|---|---|---|---|---|---|---|
| 1 | 9/10 (1C) mMUD Flu/Mel/Alem | PB: 6.3; 3.2 | 11 | PB/WB: 100% | Grade IV aGVHD (skin and gut); shingles; adenoviremia; EBV viremia; BKV encephalitis. | Ongoing at the last follow-up | Died of sepsis (20 mo post-HSCT). In CR. |
| 2 | 10/10 MUD Flu/Mel/Alem | BM: N/A | 9 | N/A | Grade III aGVHD (skin, liver and gut); invasive fungal infection. | Ongoing at the last follow-up | Died of pulmonary hemorrhage 85 d post-HSCT |
| 3 | 10/10 MUD Flu/Mel/Alem | PB: 13.83; 7.7 | 26 | PB/WB: 100%; CD19: 100% | CMV pneumonitis + T-cell sequestration; adenoviremia; thrombocytopenia, neutropenia (2× CD34 top-ups), eltrombopag and splenectomy | Off CsA (11 mo), corticosteroids (24 mo) | CR 7 y f/u |
| 4 | 10/10 MUD Flu/Mel/Alem | PB: 7.1; 15.5 | 10 | PB/WB: 100% | Adenoviremia, requiring 1xCTLs | 7 mo | CR 5 y 10 mo f/u |
| 5 | 11/12 MUD Flu/Treo/Alem | PB: 7.1; 2.7 | 12 | PB/WB: 100% | HHV6 enteritis; transient Guillain-Barré syndrome secondary to shingles (VZV) | 7 mo | CR; then relapsed at 5 y f/u Episode of shingles (VZV) Polyarthritis (feet, knees, hands, shoulder); uveitis; on tocilizumab and IA corticosteroids; on antidepressants |
| 6 | 12/12 MUD Flu/Treo/Alem | PB: 21.2; 5.2 | 18 | CD3: 94%; CD15: 90%; CD19: 61% | Grade II skin aGVHD (skin, transient) | 4 mo | CR 3.5 y f/u |
| 7 | 10/10 MSD Flu/Treo/Alem | BM: 1.7; 2.6 | 25 | CD3: 67%; CD15: 24%;CD19: 51% | BKV virus hemorrhagic cystitis; | 6 mo | CR 2.5 y f/u CMV and adenoviremia Episode of shingles/varicella zoster virus |
| 8 | 10/10 MUD Flu/Mel/Alem | PB: 12.2; 2.66 | 13 | CD3: 95%; CD15: 95%;CD19: 100% | Alloimmune neutropenia and thrombocytopenia | 6 mo | CR 2 y 9 mo f/u |
| 9 | 10/10 MSD Flu/Mel/Alem | PB: 26; 5 | 20 | CD3: 55%; CD15: 94% CD19: 82% | Grade I aGVHD upper gut EBV viremia; CMV viremia | 7 mo | Developed fevers with raised inflammatory markers. Rx corticosteroids 1 mg/kg. Off IS since 26 mo post-HSCT and in CR. 2 y 4 mo f/u |
| 10 | 9/10 (1DQ1) mMUD Flu/Mel/Alem | PB: 6.8; 1.52 | 65 | CD3: <10%CD15: 0%CD19: 0% | Pneumomediastinum and pneumothorax, | 6 mo | CR 2 y 2 mo f/u |
| 11 | 10/10 MUD Flu/Mel/Alem | PB: 9.2; 3 | 10 | PB/WB: 100% | Grade I aGVHD (skin/upper gut) adenoviremia requiring 1× CTLs; Serratia fasciitis; shingles; AVN both hips | 10 mo | CR 2 y f/u |
| 12 | 10/10 MUD Flu/Treo/Alem | PB: 8.2; 3.3 | 14 | PB/WB: 100% | None | Ongoing at 20 mo post | PR 20 mo f/u |
| 13 | 10/10 MSD Flu/Mel/Alem | BM: 19; 0.26 | 12 | PB/WB: 100% | None | 6 mo | CR 22 mo f/u |
| 14 | 9/10 mMUD Flu/Mel/Alem | PB: 15.4; 2.9 | 11 | PB/WB: 100% | Autoimmune thrombocytopenia Rx rituximab, hence still on Immunoglobulin replacement | 7 mo | CR 13 mo f/u |
| 15 | 10/10 MSD Flu/Treo/Alem | BM: 6.4; 5.8 | 19 | CD3: 88%; CD15: 97%; CD19: 96% | Grade I aGVHD (skin), Thyrotoxicosis | 6 mo | CR 14 mo f/u Hypothyroid, on thyroxine replacement |
| 16 | 12/12 MUD Flu/Treo/Alem | PB: 8.7; 32.2 | 12 | CD3: 89%; CD15: 86% | Adenovirus, HHV6 | Ongoing | 10 mo post-HSCT PR; 2 mo post-HSCT hands/wrist synovitis (responded to corticosteroids); 6 mo post-HSCT disease flare (polyarthritis, fever, cytopenias) MAS-like episode following cessation of CsA (responded to MP, rituximab, high-dose IVIG and anakinra) |
| Patient no. . | HLA donor/ conditioning . | Source: CD34 (×106/kg); CD3 (×108/kg) . | Ne D > 0.5 × 109/kg . | Chimerism at last follow-up . | Complications . | Time to stop IS . | Outcome . |
|---|---|---|---|---|---|---|---|
| 1 | 9/10 (1C) mMUD Flu/Mel/Alem | PB: 6.3; 3.2 | 11 | PB/WB: 100% | Grade IV aGVHD (skin and gut); shingles; adenoviremia; EBV viremia; BKV encephalitis. | Ongoing at the last follow-up | Died of sepsis (20 mo post-HSCT). In CR. |
| 2 | 10/10 MUD Flu/Mel/Alem | BM: N/A | 9 | N/A | Grade III aGVHD (skin, liver and gut); invasive fungal infection. | Ongoing at the last follow-up | Died of pulmonary hemorrhage 85 d post-HSCT |
| 3 | 10/10 MUD Flu/Mel/Alem | PB: 13.83; 7.7 | 26 | PB/WB: 100%; CD19: 100% | CMV pneumonitis + T-cell sequestration; adenoviremia; thrombocytopenia, neutropenia (2× CD34 top-ups), eltrombopag and splenectomy | Off CsA (11 mo), corticosteroids (24 mo) | CR 7 y f/u |
| 4 | 10/10 MUD Flu/Mel/Alem | PB: 7.1; 15.5 | 10 | PB/WB: 100% | Adenoviremia, requiring 1xCTLs | 7 mo | CR 5 y 10 mo f/u |
| 5 | 11/12 MUD Flu/Treo/Alem | PB: 7.1; 2.7 | 12 | PB/WB: 100% | HHV6 enteritis; transient Guillain-Barré syndrome secondary to shingles (VZV) | 7 mo | CR; then relapsed at 5 y f/u Episode of shingles (VZV) Polyarthritis (feet, knees, hands, shoulder); uveitis; on tocilizumab and IA corticosteroids; on antidepressants |
| 6 | 12/12 MUD Flu/Treo/Alem | PB: 21.2; 5.2 | 18 | CD3: 94%; CD15: 90%; CD19: 61% | Grade II skin aGVHD (skin, transient) | 4 mo | CR 3.5 y f/u |
| 7 | 10/10 MSD Flu/Treo/Alem | BM: 1.7; 2.6 | 25 | CD3: 67%; CD15: 24%;CD19: 51% | BKV virus hemorrhagic cystitis; | 6 mo | CR 2.5 y f/u CMV and adenoviremia Episode of shingles/varicella zoster virus |
| 8 | 10/10 MUD Flu/Mel/Alem | PB: 12.2; 2.66 | 13 | CD3: 95%; CD15: 95%;CD19: 100% | Alloimmune neutropenia and thrombocytopenia | 6 mo | CR 2 y 9 mo f/u |
| 9 | 10/10 MSD Flu/Mel/Alem | PB: 26; 5 | 20 | CD3: 55%; CD15: 94% CD19: 82% | Grade I aGVHD upper gut EBV viremia; CMV viremia | 7 mo | Developed fevers with raised inflammatory markers. Rx corticosteroids 1 mg/kg. Off IS since 26 mo post-HSCT and in CR. 2 y 4 mo f/u |
| 10 | 9/10 (1DQ1) mMUD Flu/Mel/Alem | PB: 6.8; 1.52 | 65 | CD3: <10%CD15: 0%CD19: 0% | Pneumomediastinum and pneumothorax, | 6 mo | CR 2 y 2 mo f/u |
| 11 | 10/10 MUD Flu/Mel/Alem | PB: 9.2; 3 | 10 | PB/WB: 100% | Grade I aGVHD (skin/upper gut) adenoviremia requiring 1× CTLs; Serratia fasciitis; shingles; AVN both hips | 10 mo | CR 2 y f/u |
| 12 | 10/10 MUD Flu/Treo/Alem | PB: 8.2; 3.3 | 14 | PB/WB: 100% | None | Ongoing at 20 mo post | PR 20 mo f/u |
| 13 | 10/10 MSD Flu/Mel/Alem | BM: 19; 0.26 | 12 | PB/WB: 100% | None | 6 mo | CR 22 mo f/u |
| 14 | 9/10 mMUD Flu/Mel/Alem | PB: 15.4; 2.9 | 11 | PB/WB: 100% | Autoimmune thrombocytopenia Rx rituximab, hence still on Immunoglobulin replacement | 7 mo | CR 13 mo f/u |
| 15 | 10/10 MSD Flu/Treo/Alem | BM: 6.4; 5.8 | 19 | CD3: 88%; CD15: 97%; CD19: 96% | Grade I aGVHD (skin), Thyrotoxicosis | 6 mo | CR 14 mo f/u Hypothyroid, on thyroxine replacement |
| 16 | 12/12 MUD Flu/Treo/Alem | PB: 8.7; 32.2 | 12 | CD3: 89%; CD15: 86% | Adenovirus, HHV6 | Ongoing | 10 mo post-HSCT PR; 2 mo post-HSCT hands/wrist synovitis (responded to corticosteroids); 6 mo post-HSCT disease flare (polyarthritis, fever, cytopenias) MAS-like episode following cessation of CsA (responded to MP, rituximab, high-dose IVIG and anakinra) |
AVN, avascular necrosis; BKV, BK virus; BM, bone marrow; CTL, cytotoxic T lymphocyte; EBV, Epstein-Barr virus; Flu/Mel/Alem, fludarabine/melphalan/alemtuzumab; Flu/Treo/Alem, fludarabine/treosulfan/alemtuzumab; f/u, follow-up; IA, intra-articular; mMUD, mismatched unrelated donor; MP, methylprednisolone; MSD, matched sibling donor; MUD, matched unrelated donor; N/A, not available; Ne D > 0.5 × 109/kg, number of days to achieve neutrophils >0.5 × 109/kg; PB, peripheral blood; PB/WB, peripheral blood/whole blood; Rx, received; VZV, varicella zoster virus.