Table 1.

Patient-level data for adults with SCD and HSCT

IDGenotypeTransplant indicationsAge (y), sexDMTHaplo-BMT Hb (g/dL) (before, after)Haplo-BMT HbS% (before, after)Stem cell donor relation, genotypeMyeloid
engraftment (% donor)
Change in mRS and mRS scores before/after transplantTransplant 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 
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. 
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. 
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 
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 
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 
IDGenotypeTransplant indicationsAge (y), sexDMTHaplo-BMT Hb (g/dL) (before, after)Haplo-BMT HbS% (before, after)Stem cell donor relation, genotypeMyeloid
engraftment (% donor)
Change in mRS and mRS scores before/after transplantTransplant 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 
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. 
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. 
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 
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 
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.

or Create an Account

Close Modal
Close Modal