Transplant characteristics and outcomes
| ID . | Indication for HSCT . | Sex, age at HSCT (y) . | Stem cell source . | Donor . | Conditioning regimen . | Serotherapy . | GVHD prophylaxis . | Neutrophil engraftment (>500/μL), d . | Thrombocyte engraftment (unsupported, >20 000/μL), d . | T cells at day 100 (CD3+/μL) . | Chimerism . | GVHD . |
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | Immunodeficiency, G-CSF nonresponder | m, 1.6 | BM | MSD | FLU (5.2 mg/kg), TT (5mg/kg), MEL (4.6 mg/kg) | ATG Fresenius (60 mg/kg) | CSA | +17 | _ | 593 | Autologous | _ |
| 1 | Autologous reconstitution | m, 2.5 | BM | MSD | BU (12.8 mg/kg), CY (120 mg/kg) | ATG Fresenius (60 mg/kg) | CSA | +15 | +56 | 242 | Mixed (30% autologous) | _ |
| 2 | MDS/AML M2 | f, 19 | BM | MUD | BU (8.75 mg/kg), CY (120 mg/kg) | Campath (1,8 mg/kg) | MTX, CSA | +15 | +15 | 832 | Complete | _ |
| 12 | Infections, severe periodontitis, G-CSF nonresponder | m, 11.3 | PBSC | Haplo, brother | FLU (150 mg/m2), TREO (42 g/m2), TT (5 mg/kg) | none | MTX, CSA, Post-Cy day +3 / +4 (50 mg/kg), MMF | +21 | +25 | n.a. | Complete | _ |
| 14_1 | Severe infections | m, 10.3 | PBSC | Haplo, mother | BU (15,5 mg/kg)∗, CY(54 mg/kg), TT(9 mg/kg) | ATG Fresenius (30 mg/kg) | MMF | _ | _ | _ | Autologous | _ |
| 14_2 | Graft failure | m, 10.3 | PBSC | Haplo, father | FLU (120 mg/m2), CY (60 mg/kg), TT (5 mg/kg), TBI (2 × 2 Gy), rituximab (2 × 375 mg/m2), bortezomib (2 × 1.3 mg/m2) | ATG thymoglobulin (4.5 mg/kg) | MMF | +19 | +12 | n.a. | _ | |
| 19 | Severe infections | m, 3.3 | BM | MSD | FLU (160 mg/m2), TREO (42 g/m2), TT (5 mg/kg) | ATG Fresenius (15 mg/kg) | MTX, CSA | +19 | +25 | 231 | Complete | _ |
| 27 | Neutropenia, severe infections, aspergillosis | m, 0.8 | n.a. | n.a. | n.a. | n.a. | n.a. | n.a. | n.a. | n.a. | n.a. | n.a. |
| ID . | Indication for HSCT . | Sex, age at HSCT (y) . | Stem cell source . | Donor . | Conditioning regimen . | Serotherapy . | GVHD prophylaxis . | Neutrophil engraftment (>500/μL), d . | Thrombocyte engraftment (unsupported, >20 000/μL), d . | T cells at day 100 (CD3+/μL) . | Chimerism . | GVHD . |
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | Immunodeficiency, G-CSF nonresponder | m, 1.6 | BM | MSD | FLU (5.2 mg/kg), TT (5mg/kg), MEL (4.6 mg/kg) | ATG Fresenius (60 mg/kg) | CSA | +17 | _ | 593 | Autologous | _ |
| 1 | Autologous reconstitution | m, 2.5 | BM | MSD | BU (12.8 mg/kg), CY (120 mg/kg) | ATG Fresenius (60 mg/kg) | CSA | +15 | +56 | 242 | Mixed (30% autologous) | _ |
| 2 | MDS/AML M2 | f, 19 | BM | MUD | BU (8.75 mg/kg), CY (120 mg/kg) | Campath (1,8 mg/kg) | MTX, CSA | +15 | +15 | 832 | Complete | _ |
| 12 | Infections, severe periodontitis, G-CSF nonresponder | m, 11.3 | PBSC | Haplo, brother | FLU (150 mg/m2), TREO (42 g/m2), TT (5 mg/kg) | none | MTX, CSA, Post-Cy day +3 / +4 (50 mg/kg), MMF | +21 | +25 | n.a. | Complete | _ |
| 14_1 | Severe infections | m, 10.3 | PBSC | Haplo, mother | BU (15,5 mg/kg)∗, CY(54 mg/kg), TT(9 mg/kg) | ATG Fresenius (30 mg/kg) | MMF | _ | _ | _ | Autologous | _ |
| 14_2 | Graft failure | m, 10.3 | PBSC | Haplo, father | FLU (120 mg/m2), CY (60 mg/kg), TT (5 mg/kg), TBI (2 × 2 Gy), rituximab (2 × 375 mg/m2), bortezomib (2 × 1.3 mg/m2) | ATG thymoglobulin (4.5 mg/kg) | MMF | +19 | +12 | n.a. | _ | |
| 19 | Severe infections | m, 3.3 | BM | MSD | FLU (160 mg/m2), TREO (42 g/m2), TT (5 mg/kg) | ATG Fresenius (15 mg/kg) | MTX, CSA | +19 | +25 | 231 | Complete | _ |
| 27 | Neutropenia, severe infections, aspergillosis | m, 0.8 | n.a. | n.a. | n.a. | n.a. | n.a. | n.a. | n.a. | n.a. | n.a. | n.a. |
Complete donor chimerism was defined as donor chimerism ≥99%.
BM, bone marrow; CY, cyclophosphamide; f, female; FLU, fludarabine; Haplo, haploidentical; m, male; MEL, melphalane; MSD, matched sibling donor; MTX, methotrexate; MUD, matched unrelated donor; n.a., not available; PBSC, peripheral blood stem cell; postCY, posttransplant cyclophosphamide; TBI, total body irradiation; TREO, treosulfan; TT, thiotepa.
AUC (area under the concentration-vs-time curve) 85 mg x h/L.