Hematopoietic stem cell transplantation for sickle cell disease.
| Location . | Belgium43 . | Pesaro69 . | France42,70 . | Multicenter31,46,49 . | Other US/Europe5,43 ,71,72,73,74,75,76 . | Total . | |
|---|---|---|---|---|---|---|---|
| Regimen | BU,CY (30) | BU,CY (17) | BU,CY (12) | BU,CY,ATG | BU,CY,ATG (13) | ||
| BU,CY,TLI (6) | BU,CY,TL1 (1) | CY/TBI (3) | |||||
| BU,CY,ATG (14) | BU,CY,ATG (21) | ||||||
| Number of patients | 36 | 14 | 19 | 34¶ | 59 | 16 | 175¶ |
| Median age (range) | 8.6 (1.7-23) | 2.0 (0.9-15) | 7 (4-38) | 8.6 (2.3-17.2) | 9.9 (3.3-15.9) | NA | |
| Clinical Status: | |||||||
| Asymptomatic | 0 | 14 | 3 | 0 | 0 | NS | |
| Stroke/CNS | 6 | 0 | 1 | 16 | 31 | 2 | |
| ACS | 20 | 0 | NS | 15 | 20 | 1 | |
| VOC | 36 | 0 | 15 | 18 | 8 | 1 | |
| Other/unknown | — | — | — | — | — | 11 | |
| MM donor | — | — | 1 | — | — | ||
| Survival (%) | 34 (94) | 14 (100) | 14 (74) | 31 (91) | 55 (94) | 13 (81) | 159 (91) |
| Deaths | 2 | 0 | 5 | 3¶ | 4 | 3 | 16¶ |
| Graft rejection/recurrent SCD (%) | 4 | 1 | 1 | 4 | 5 | 1 | 16 (9) |
| Stable mixed chimerism | 6** | — | 5 | 1 | 10 | — | 11% |
| Disease-free survival (%) | 30 (83)* | 13 (93) | 13 (68) | 27 (79)* | 50 (85) | 12 (80)* | 143 (82) |
| aGVHD | # 15## | 5 | 4 | 6 (>grade II) | 11 (Grade I-III) | 2 | 25% |
| cGVHD | 8 | 2 | 2 | 2 | 5 | 1 | 12% |
| Seizures | 18 | 1 | 7/26 | 13 | 1 | 25% | |
| Abbreviations: ACS, acute chest syndrome; aGVHD, acute graft-versus-host disease; ATG, antithymocyte globulin; BU, busulfan; cGVHD, chronic graft-versus-host disease; CNS, central nervous system; CY, cyclophosphamide; MM, mismatched; SCD, sickle cell disease; TLI, total lymphoid irradiation; TBI, total body irradiation, VOC, vasoocclusive crisis; NS, not stated; NA, not available; HSCT, hematopoietic stem cell transplantation | |||||||
| Location . | Belgium43 . | Pesaro69 . | France42,70 . | Multicenter31,46,49 . | Other US/Europe5,43 ,71,72,73,74,75,76 . | Total . | |
|---|---|---|---|---|---|---|---|
| Regimen | BU,CY (30) | BU,CY (17) | BU,CY (12) | BU,CY,ATG | BU,CY,ATG (13) | ||
| BU,CY,TLI (6) | BU,CY,TL1 (1) | CY/TBI (3) | |||||
| BU,CY,ATG (14) | BU,CY,ATG (21) | ||||||
| Number of patients | 36 | 14 | 19 | 34¶ | 59 | 16 | 175¶ |
| Median age (range) | 8.6 (1.7-23) | 2.0 (0.9-15) | 7 (4-38) | 8.6 (2.3-17.2) | 9.9 (3.3-15.9) | NA | |
| Clinical Status: | |||||||
| Asymptomatic | 0 | 14 | 3 | 0 | 0 | NS | |
| Stroke/CNS | 6 | 0 | 1 | 16 | 31 | 2 | |
| ACS | 20 | 0 | NS | 15 | 20 | 1 | |
| VOC | 36 | 0 | 15 | 18 | 8 | 1 | |
| Other/unknown | — | — | — | — | — | 11 | |
| MM donor | — | — | 1 | — | — | ||
| Survival (%) | 34 (94) | 14 (100) | 14 (74) | 31 (91) | 55 (94) | 13 (81) | 159 (91) |
| Deaths | 2 | 0 | 5 | 3¶ | 4 | 3 | 16¶ |
| Graft rejection/recurrent SCD (%) | 4 | 1 | 1 | 4 | 5 | 1 | 16 (9) |
| Stable mixed chimerism | 6** | — | 5 | 1 | 10 | — | 11% |
| Disease-free survival (%) | 30 (83)* | 13 (93) | 13 (68) | 27 (79)* | 50 (85) | 12 (80)* | 143 (82) |
| aGVHD | # 15## | 5 | 4 | 6 (>grade II) | 11 (Grade I-III) | 2 | 25% |
| cGVHD | 8 | 2 | 2 | 2 | 5 | 1 | 12% |
| Seizures | 18 | 1 | 7/26 | 13 | 1 | 25% | |
| Abbreviations: ACS, acute chest syndrome; aGVHD, acute graft-versus-host disease; ATG, antithymocyte globulin; BU, busulfan; cGVHD, chronic graft-versus-host disease; CNS, central nervous system; CY, cyclophosphamide; MM, mismatched; SCD, sickle cell disease; TLI, total lymphoid irradiation; TBI, total body irradiation, VOC, vasoocclusive crisis; NS, not stated; NA, not available; HSCT, hematopoietic stem cell transplantation | |||||||
Three patients were reported twice in the two French series42,70 ; one of them died after HSCT and was also counted twice among the deaths. These three patients are counted only once in the totals.
1 patient was disease-free after a second HSCT
3 had > 30% recipient cells
1 patient had Morquio's disease and 2 had acute leukemia concomitantly.
Only 1 had > grade II GVHD