Patient-level data for adults with SCD and HSCT
| ID . | Genotype . | Transplant indications . | Age (y), sex . | DMT . | Haplo-BMT Hb (g/dL) (before, after) . | Haplo-BMT HbS% (before, after) . | Stem cell donor relation, genotype . | Myeloid engraftment (% donor) . | Change in mRS and mRS scores before/after transplant . | Transplant complications . |
|---|---|---|---|---|---|---|---|---|---|---|
| 1∗ | HbSS | ACS and osteomyelitis | 20.8, M | HU | 6.7, 12.9 | 80.5, 32.1 | Sibling, HbAS | 100% | None, 0/0 | N/A |
| 2∗ | HbSS | Overt stroke and moyamoya | 34.4, M | HU† | 6.7, 14.2 | 86.9, 32.8 | Sibling, HbAS | 100% | None, 3/3 | HHV6 encephalitis treated with Foscarnet. |
| 3∗ | HbSS | SCI, ACS, VOC, and iron overload | 39.9, F | HU | 5.2, 10.9 | 60.6, 31.6 | Sibling, HbAS | 100% | None, 0/0 | N/A |
| 4 | HbSS | Overt stroke, moyamoya, and ACS | 24.6, M | CBT | 9.4, 13.1 | 44.4, 34.8 | Parent, HbAS | 100% | None, 1/1 | First transplant did not engraft. Second transplant engrafted. |
| 5∗ | HbSS | Overt stroke and moyamoya | 39.5, F | HU + CBT | 7.4, 11.2 | 18.0, 0.0 | Sibling, HbAA | 100% | None, 1/1‡ | New SCI at 8.5 months after transplant (MRI timing for participant convenience, not symptoms) and died of COVID-19 3.5 y after transplant. |
| 6 | HbSS | Overt stroke and moyamoya | 25.9, F | CBT | 10.4, 8.1 | 35.1, 0.0 | Sibling, HbAA | 100% | None, 3/3 | Major ABO-incompatibility with transplant resulting in pure red cell aplasia. Eventually able to treat with monoclonal antibody daratumumab at 4 doses; current Hb level at 12.9 g/dL. |
| 7 | HbSS | SCI, ACS, and pulmonary HTN | 43.7, F | HU + CBT | 10.4, 14.3 | 15.2, 38.8 | Sibling, HbAS | 100% | None, 0/0 | N/A |
| 8 | HbSThal+ | VOC requiring chronic transfusions, and hyperhemolytic phenotype | 22.4, M | CBT | 10.2, 19.2 | 34.9, 38.2 | Sibling, HbAS | 100% | None, 0/0 | N/A |
| 9 | HbSC | Overt stroke, SCI, ACS, and systemic lupus | 43.7, F | CBT | 11.1, 13.1 | 31.3, 0.0 | Sibling, HbAA | 100% | Improved, 1/0 | Significant ocular GVHD. |
| 10 | HbSC | ACS, pulmonary embolus, and retinal hemorrhage | 41.8, M | HU | 10.4, 12.9 | 12.9, 33.4 | Child, HbAS | 100% | None, 0/0 | N/A |
| 11 | HbSS | ACS and AVN of hips with hip replacements | 29.6, F | HU | 8.4, 15.0 | 73.8, 39.8 | Sibling, HbAS | 100% | None, 0/0 | N/A |
| ID . | Genotype . | Transplant indications . | Age (y), sex . | DMT . | Haplo-BMT Hb (g/dL) (before, after) . | Haplo-BMT HbS% (before, after) . | Stem cell donor relation, genotype . | Myeloid engraftment (% donor) . | Change in mRS and mRS scores before/after transplant . | Transplant complications . |
|---|---|---|---|---|---|---|---|---|---|---|
| 1∗ | HbSS | ACS and osteomyelitis | 20.8, M | HU | 6.7, 12.9 | 80.5, 32.1 | Sibling, HbAS | 100% | None, 0/0 | N/A |
| 2∗ | HbSS | Overt stroke and moyamoya | 34.4, M | HU† | 6.7, 14.2 | 86.9, 32.8 | Sibling, HbAS | 100% | None, 3/3 | HHV6 encephalitis treated with Foscarnet. |
| 3∗ | HbSS | SCI, ACS, VOC, and iron overload | 39.9, F | HU | 5.2, 10.9 | 60.6, 31.6 | Sibling, HbAS | 100% | None, 0/0 | N/A |
| 4 | HbSS | Overt stroke, moyamoya, and ACS | 24.6, M | CBT | 9.4, 13.1 | 44.4, 34.8 | Parent, HbAS | 100% | None, 1/1 | First transplant did not engraft. Second transplant engrafted. |
| 5∗ | HbSS | Overt stroke and moyamoya | 39.5, F | HU + CBT | 7.4, 11.2 | 18.0, 0.0 | Sibling, HbAA | 100% | None, 1/1‡ | New SCI at 8.5 months after transplant (MRI timing for participant convenience, not symptoms) and died of COVID-19 3.5 y after transplant. |
| 6 | HbSS | Overt stroke and moyamoya | 25.9, F | CBT | 10.4, 8.1 | 35.1, 0.0 | Sibling, HbAA | 100% | None, 3/3 | Major ABO-incompatibility with transplant resulting in pure red cell aplasia. Eventually able to treat with monoclonal antibody daratumumab at 4 doses; current Hb level at 12.9 g/dL. |
| 7 | HbSS | SCI, ACS, and pulmonary HTN | 43.7, F | HU + CBT | 10.4, 14.3 | 15.2, 38.8 | Sibling, HbAS | 100% | None, 0/0 | N/A |
| 8 | HbSThal+ | VOC requiring chronic transfusions, and hyperhemolytic phenotype | 22.4, M | CBT | 10.2, 19.2 | 34.9, 38.2 | Sibling, HbAS | 100% | None, 0/0 | N/A |
| 9 | HbSC | Overt stroke, SCI, ACS, and systemic lupus | 43.7, F | CBT | 11.1, 13.1 | 31.3, 0.0 | Sibling, HbAA | 100% | Improved, 1/0 | Significant ocular GVHD. |
| 10 | HbSC | ACS, pulmonary embolus, and retinal hemorrhage | 41.8, M | HU | 10.4, 12.9 | 12.9, 33.4 | Child, HbAS | 100% | None, 0/0 | N/A |
| 11 | HbSS | ACS and AVN of hips with hip replacements | 29.6, F | HU | 8.4, 15.0 | 73.8, 39.8 | Sibling, HbAS | 100% | None, 0/0 | N/A |
ACS, acute chest syndrome; AVN, avascular necrosis; CBT, chronic blood transfusion therapy; CSVT, cerebral sinovenous thrombosis; DMT, disease-modifying therapy; DVT, deep vein thrombosis; GVHD, graft-versus-host disease; HHV-6, human herpes virus-6; HTN, hypertension; HU, hydroxyurea; SCI, silent cerebral infarct; TIA, transient ischemic attack; VOC, vaso-occlusive crisis.
Previously reported.12
CBT declined.
mRS at the time of neuroimaging, before death from infection.