Transplant characteristics and outcome
| UPN . | Donor/HLA-mismatch . | Preparative regimen . | Transplanted cells . | Hematopoietic reconstitution (day after SCT) . | Major complications . | Chimerism (last study) . | Outcome . | |||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| CD34+ cells (× 106/kg) . | T-cells (× 104/kg) . | Neutro/μL . | Reti . | Platelets/μL . | ||||||||||
| Initial graft . | Boost . | Initial graft . | Boost . | > 500 . | > 1000 . | > 1.0% . | > 20 000 . | > 100 000 . | ||||||
| 275 | Mother/1 Ag (A) | Bu (16), Thio (10), Cy (200), ATG | 25 | 40 (d + 42) | 2.8 | 2.6 | 27 | 44 | 45 | 49 | 75 | Respiratory insufficiency, CMV-disease, VOD (liver) | Full donor (5 y) | a/w (>60 mo) minor visual impairment |
| 292 (1st HSCT) | Father/3 Ag (A, B, DR-B1) | Bu (20), Thio (10), Cy (120), ATG | 36 | — | 1.1 | — | — | — | — | — | — | Rejection | NE | |
| 292 (2nd HSCT) | Mother/3 Ag (A, B, DR-B1) | 5 Gy splenic radiation, OKT-3 | 56 | — | 9.1 | — | — | — | — | — | — | CMV-pneumonitis, ARDS, VOD (liver) | NE | dead (d + 34) |
| 302 | Father/3 Ag (A, B, DR-B1) | Bu (16), Thio (10), Cy (200), ATG | 20 | 10 (d + 11) | 0.8 | 0.4 | 22 | 33 | 24 | 44 | 130 | — | Full donor (4 y) | a/w (>48 mo) visual impairment |
| 303 | Father/1 Ag (DR-B1) | Bu (20), Thio (10), Cy (200), ATG | 36 | 24 (d + 10) | 11.1 | 7.4 | NE† | 9 | 10 | 51 | 330 | Respiratory insufficiency, VOD (liver) | Full donor (3 y) | a/w (>47 mo) visual impairment, mental retardation |
| 323 | Father/3 Ag (A, B, DR-B1) | Bu (20), Thio (10), Cy (200), ATG | 12 | 12 (d + 10) | 1.2 | 1.2 | 22 | — | — | 70 | — | Respiratory insufficiency, VOD (lung and liver) | Full donor (+37 d) | dead (d + 84) |
| 332 | Father/3 Ag (A, B, DR-B1) | Bu (20), Thio (10), Flu,*ATG | 43 | 2 × 43 (d + 7, + 42) | 1.7 | 2 × 1, 7 | 17 | 68 | 50 | 53 | 57 | — | Full donor (2 y) | a/w (>36 mo) |
| 364 | Aunt/1 Ag (DR-B1) | Bu (20), Thio (10), Flu,* OKT-3 | 15 | 15 (d + 10) | 1.6 | 1.6 | 14 | 55 | 13 | 50 | 58 | — | Full donor (1.5 y) | a/w (>25 mo) visual impairment |
| UPN . | Donor/HLA-mismatch . | Preparative regimen . | Transplanted cells . | Hematopoietic reconstitution (day after SCT) . | Major complications . | Chimerism (last study) . | Outcome . | |||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| CD34+ cells (× 106/kg) . | T-cells (× 104/kg) . | Neutro/μL . | Reti . | Platelets/μL . | ||||||||||
| Initial graft . | Boost . | Initial graft . | Boost . | > 500 . | > 1000 . | > 1.0% . | > 20 000 . | > 100 000 . | ||||||
| 275 | Mother/1 Ag (A) | Bu (16), Thio (10), Cy (200), ATG | 25 | 40 (d + 42) | 2.8 | 2.6 | 27 | 44 | 45 | 49 | 75 | Respiratory insufficiency, CMV-disease, VOD (liver) | Full donor (5 y) | a/w (>60 mo) minor visual impairment |
| 292 (1st HSCT) | Father/3 Ag (A, B, DR-B1) | Bu (20), Thio (10), Cy (120), ATG | 36 | — | 1.1 | — | — | — | — | — | — | Rejection | NE | |
| 292 (2nd HSCT) | Mother/3 Ag (A, B, DR-B1) | 5 Gy splenic radiation, OKT-3 | 56 | — | 9.1 | — | — | — | — | — | — | CMV-pneumonitis, ARDS, VOD (liver) | NE | dead (d + 34) |
| 302 | Father/3 Ag (A, B, DR-B1) | Bu (16), Thio (10), Cy (200), ATG | 20 | 10 (d + 11) | 0.8 | 0.4 | 22 | 33 | 24 | 44 | 130 | — | Full donor (4 y) | a/w (>48 mo) visual impairment |
| 303 | Father/1 Ag (DR-B1) | Bu (20), Thio (10), Cy (200), ATG | 36 | 24 (d + 10) | 11.1 | 7.4 | NE† | 9 | 10 | 51 | 330 | Respiratory insufficiency, VOD (liver) | Full donor (3 y) | a/w (>47 mo) visual impairment, mental retardation |
| 323 | Father/3 Ag (A, B, DR-B1) | Bu (20), Thio (10), Cy (200), ATG | 12 | 12 (d + 10) | 1.2 | 1.2 | 22 | — | — | 70 | — | Respiratory insufficiency, VOD (lung and liver) | Full donor (+37 d) | dead (d + 84) |
| 332 | Father/3 Ag (A, B, DR-B1) | Bu (20), Thio (10), Flu,*ATG | 43 | 2 × 43 (d + 7, + 42) | 1.7 | 2 × 1, 7 | 17 | 68 | 50 | 53 | 57 | — | Full donor (2 y) | a/w (>36 mo) |
| 364 | Aunt/1 Ag (DR-B1) | Bu (20), Thio (10), Flu,* OKT-3 | 15 | 15 (d + 10) | 1.6 | 1.6 | 14 | 55 | 13 | 50 | 58 | — | Full donor (1.5 y) | a/w (>25 mo) visual impairment |
Abbreviations: Bu: busulfan; Thio: thiotepa; Cy: cyclophosphamide (numbers in parenthesis indicate total doses in mg/kg body weight).
Flu: fludarabine 200 mg/m2 body surface. ATG: antithymocyte globulin. For details of application see text. Ag: HLA-antigen; NE: not evaluable; a/w: alive and well.
Patient 4 (UPN 303) received granulocyte transfusions on day + 5, + 7, + 10.