Characteristics of stem cell transplantation
| Patient no. . | Age at HSCT, y . | Donor/stem cell source . | Conditioning . | GVHD and VOD prophylaxis . | Time to engraftment* . | GVHD . | Infection/toxicity (treatment) . | Hypercalcemia (treatment) . | Other complications . | Follow-up, y . | Present status/chimerism . |
|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | 14 | MUD (10/10)/PBSC | Treo (42)/flud (150)/thio (10) | ATG/tacr/MMF | Neut d+18 plt d+28 | No | EBV-PTLD (rituximab ×4) | Y (forced diuresis) | N | 4 | A&W, school attendance/100% donor |
| 2 | 9 | MFD (10/10, uncle)/BM | Treo (42)/flud (150)/thio (10) | ATG/tacr/MMF | Neut d+29 plt d+33 | No | CMV reactivation | N | N | 3 | A&W, school attendance, cure of osteomyelitis/100% donor |
| 3 | 12 | MFD (10/10, aunt)/BM | Treo (42)/flud (150) | CSA/MMF/defi | Neut d+17 plt d+39 last ET/PT d+55/+37 | AGVHD L2 (skin L3); limited cGVHD, resolved (ECP) | FUO/renal insufficiency (transitional hemofiltration) | Severe, symptomatic hypercalcemia at time of engraftment (calcitonin, hemofiltration, denosumab for several mo) | Pathological fractures (ribs, forearm) until about 1 y post-BMT | 8 | A&W, no pathological fractures, no infections, school attendance/100% donor |
| 4 | 30 | MSD/BM | Treo (42)/flud (160)/thio (10) | ATG/tacr/MMF/defi | Neut d+22 plt +48 last ET/PT d +35/+31 | AGVHD L1 (skin L1); no cGVHD | Bacteremia in BM aplasia, resolved, CMV reactivation | Moderate hypercalcemia shortly after engraftment (denosumab for several mo) | Pathological fractures, hip pseudarthrosis until about 1 y post-BMT | 5 | A&W, no pathological fractures, no infections/100% donor |
| 5 | 7 | MFD (10/10, uncle)/BM | Treo (42)/flud (150)/thio (8) | CSA/MMF/defi | Neut d+32 plt +42 last ET/PT d+20/d+24 | No | Bacteremia in BM aplasia, resolved | N | N | 4 | A&W, no pathological fractures, no infections, school attendance/100% donor |
| 6 | 5 | Haplo-identical father/PBSC | Busu (19.2)/ flud (150)/ thio (10) | T-cell depletion/Campath/defi | NA (rejection)/2nd HSCT same donor → rejection | No | Sepsis, CMV infection/pulmonary hypertension | N | Respiratory failure in aplasia | NE | Death after 3rd HSCT (haplo mother) without engraftment |
| 7 | 28 | MUD (9/10)/PBSC | Treo (42)/flud (150)/thio (10) | Campath/CSA/MMF/posttransplant cyclo | Neut d+28 plt d+34 | No | Sepsis, CMV | Y (forced diuresis) | N | 1 | A&W, no pathological fractures, no infections/100% donor |
| Patient no. . | Age at HSCT, y . | Donor/stem cell source . | Conditioning . | GVHD and VOD prophylaxis . | Time to engraftment* . | GVHD . | Infection/toxicity (treatment) . | Hypercalcemia (treatment) . | Other complications . | Follow-up, y . | Present status/chimerism . |
|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | 14 | MUD (10/10)/PBSC | Treo (42)/flud (150)/thio (10) | ATG/tacr/MMF | Neut d+18 plt d+28 | No | EBV-PTLD (rituximab ×4) | Y (forced diuresis) | N | 4 | A&W, school attendance/100% donor |
| 2 | 9 | MFD (10/10, uncle)/BM | Treo (42)/flud (150)/thio (10) | ATG/tacr/MMF | Neut d+29 plt d+33 | No | CMV reactivation | N | N | 3 | A&W, school attendance, cure of osteomyelitis/100% donor |
| 3 | 12 | MFD (10/10, aunt)/BM | Treo (42)/flud (150) | CSA/MMF/defi | Neut d+17 plt d+39 last ET/PT d+55/+37 | AGVHD L2 (skin L3); limited cGVHD, resolved (ECP) | FUO/renal insufficiency (transitional hemofiltration) | Severe, symptomatic hypercalcemia at time of engraftment (calcitonin, hemofiltration, denosumab for several mo) | Pathological fractures (ribs, forearm) until about 1 y post-BMT | 8 | A&W, no pathological fractures, no infections, school attendance/100% donor |
| 4 | 30 | MSD/BM | Treo (42)/flud (160)/thio (10) | ATG/tacr/MMF/defi | Neut d+22 plt +48 last ET/PT d +35/+31 | AGVHD L1 (skin L1); no cGVHD | Bacteremia in BM aplasia, resolved, CMV reactivation | Moderate hypercalcemia shortly after engraftment (denosumab for several mo) | Pathological fractures, hip pseudarthrosis until about 1 y post-BMT | 5 | A&W, no pathological fractures, no infections/100% donor |
| 5 | 7 | MFD (10/10, uncle)/BM | Treo (42)/flud (150)/thio (8) | CSA/MMF/defi | Neut d+32 plt +42 last ET/PT d+20/d+24 | No | Bacteremia in BM aplasia, resolved | N | N | 4 | A&W, no pathological fractures, no infections, school attendance/100% donor |
| 6 | 5 | Haplo-identical father/PBSC | Busu (19.2)/ flud (150)/ thio (10) | T-cell depletion/Campath/defi | NA (rejection)/2nd HSCT same donor → rejection | No | Sepsis, CMV infection/pulmonary hypertension | N | Respiratory failure in aplasia | NE | Death after 3rd HSCT (haplo mother) without engraftment |
| 7 | 28 | MUD (9/10)/PBSC | Treo (42)/flud (150)/thio (10) | Campath/CSA/MMF/posttransplant cyclo | Neut d+28 plt d+34 | No | Sepsis, CMV | Y (forced diuresis) | N | 1 | A&W, no pathological fractures, no infections/100% donor |
aGVHD, acute graft-versus-host disease; A&W, alive and well; BM, bone marrow; BMT, bone marrow transplantation; busu, busulfan; cGVHD, chronic graft-versus-host disease; cyclo, cyclophosphamide; flud, fludarabine; defi, defibrotide; EBV-PTLD, Epstein Barr virus–related lymphoproliferative disease; ECP, extracorporeal photopheresis; ET, erythrocyte transfusion; FUO, fever of unknown origin; L, level; MFD, matched family donor; MSD, matched sibling donor; N, no; NA, not available; neut, neutrophil; NE, not evaluable; PBSC, peripheral blood stem cell; PT, platelet transfusion; tacr, tacrolimus; thio, thiotepa; treo, treosulfan.
Definition criteria: neutrophils >0.5 × 109/L, platelets >50 × 109/L.