Patient follow-up data
| Patient . | Sex . | Age at HSCT, y . | Donor . | Diagnosis . | Follow-up, mo . | Hematopoietic chimerism analysis . | Time of last BM chimerism, mo . | Time of last PB chimerism, mo . | Outcome . |
|---|---|---|---|---|---|---|---|---|---|
| 1 | M | 15.5 | Mother | Refractory AML | +7† | 100% donor | +6 | NE | Died in CR due to Candida sepsis |
| 2* | M | 2 | Father | X-LPD | +28 | 100% donor | +24 | +26 | Alive and well |
| 3* | M | 2.3 | Father | X-LPD | +24 | 100% donor on PMNs, 88% donor on CD3+ cells | +20 | +24 | Alive and well |
| 4 | M | 13.1 | Mother | Refractory AML | +4† | 100% donor | +3 | +3 | Died due to relapse |
| 5 | F | 8.8 | Father | Fanconi anemia | +16 | 100% donor | +14 | +11 | Alive and well, transfusion independent |
| 6 | F | 3.7 | Father | ALL CR2 | +7† | 100% donor | +3 | +3 | Died due to relapse |
| 7 | F | 13.3 | Father | Refractory AML with monosomy 7 | +4† | 100% donor | +2 | +2 | Died in CR due to adenovirus hepatitis |
| 8 | F | 7.1 | Mother | AML CR2 | +12 | 100% donor | +7 | +10 | Alive and well, CR |
| 9 | M | 5 | Father | ALL-T CR4 | +10 | 100% donor | +8 | +7 | Alive and well, CR |
| 10 | M | 5.3 | Mother | ALL CR2 | +8 | 100% donor | +7 | +8 | Alive and well, CR |
| 11 | F | 8.4 | Sister | ALL CR2 | +7 | 100% donor | +5 | +6 | Alive and well, CR |
| 12 | F | 8.1 | Mother | AML CR2 | +6 | 95% donor on BM 80% donor on PB 30% donor on PB CD4+/CD8+ cells | +5 | +6 | Alive and well, CR |
| 13 | M | 16 | Father | AML CR2 | +3 | 100% donor | +3 | +4 | Alive and well, CR |
| 14 | M | 12 | Father | AML 2CR | +2 | 100% donor | +3 | +3 | Alive and well, CR |
| Patient . | Sex . | Age at HSCT, y . | Donor . | Diagnosis . | Follow-up, mo . | Hematopoietic chimerism analysis . | Time of last BM chimerism, mo . | Time of last PB chimerism, mo . | Outcome . |
|---|---|---|---|---|---|---|---|---|---|
| 1 | M | 15.5 | Mother | Refractory AML | +7† | 100% donor | +6 | NE | Died in CR due to Candida sepsis |
| 2* | M | 2 | Father | X-LPD | +28 | 100% donor | +24 | +26 | Alive and well |
| 3* | M | 2.3 | Father | X-LPD | +24 | 100% donor on PMNs, 88% donor on CD3+ cells | +20 | +24 | Alive and well |
| 4 | M | 13.1 | Mother | Refractory AML | +4† | 100% donor | +3 | +3 | Died due to relapse |
| 5 | F | 8.8 | Father | Fanconi anemia | +16 | 100% donor | +14 | +11 | Alive and well, transfusion independent |
| 6 | F | 3.7 | Father | ALL CR2 | +7† | 100% donor | +3 | +3 | Died due to relapse |
| 7 | F | 13.3 | Father | Refractory AML with monosomy 7 | +4† | 100% donor | +2 | +2 | Died in CR due to adenovirus hepatitis |
| 8 | F | 7.1 | Mother | AML CR2 | +12 | 100% donor | +7 | +10 | Alive and well, CR |
| 9 | M | 5 | Father | ALL-T CR4 | +10 | 100% donor | +8 | +7 | Alive and well, CR |
| 10 | M | 5.3 | Mother | ALL CR2 | +8 | 100% donor | +7 | +8 | Alive and well, CR |
| 11 | F | 8.4 | Sister | ALL CR2 | +7 | 100% donor | +5 | +6 | Alive and well, CR |
| 12 | F | 8.1 | Mother | AML CR2 | +6 | 95% donor on BM 80% donor on PB 30% donor on PB CD4+/CD8+ cells | +5 | +6 | Alive and well, CR |
| 13 | M | 16 | Father | AML CR2 | +3 | 100% donor | +3 | +4 | Alive and well, CR |
| 14 | M | 12 | Father | AML 2CR | +2 | 100% donor | +3 | +3 | Alive and well, CR |
Full donor chimerism was defined as greater than 98% donor cells in peripheral blood and bone marrow. Chimeric analysis was routinely performed at 1 to 2 weekly intervals in the first months after transplantation on peripheral blood mononuclear cells. Other serial controls of chimerism were subsequently performed at variable intervals. The last known analysis is shown for all study patients (+ months after HSCT). Two patients (number 3 and 12) show stable mixed chimerism at 2 years and 6 months, respectively.
mo indicates months; BM, bone marrow; PB, peripheral blood; M, male; NE, not evaluated; X-LPD,X-linked lymphoproliferative disorder; F, female.
Patients number 2 and 3 are identical twins that received transplants from their haploidentical father.
Dead.