Patient characteristics and outcomes
| Patient . | Age, y/sex . | Indication . | Stem cell source/HLA match . | Number of abatacept doses received . | Myeloid chimerism at last evaluation, % . | aGVHD/cGVHD (cGVHD NIH grade) . | Follow up, y . | Organ toxicity/outcome . | Systemic immune suppression (time withdrawn) . | Performance score pre/at last follow up . |
|---|---|---|---|---|---|---|---|---|---|---|
| 1 | 10/F | Stroke | Bone marrow/A antigen | 4* | 95 | 0/limited (mild) | 5.5 | SJS/resolved | Off (1 y, 9 mo) | 100/100 |
| 2 | 13/F | VOE | Bone marrow/DRB1 antigen | 4* | 100 | Gr 1/extensive (severe) | 4.8 | PRES/resolved | On | 100/70 |
| 3 | 21/M | Stroke | Bone marrow/none | 8 | 95 | 0/limited (mild) | 4.5 | Pancreatic insufficiency/ongoing | Off (1 y) | 100/100 |
| 4 | 18/F | VOE | Bone marrow/ C allele | 8 | 100 | 0/limited (mild) | 3.3 | None | Off (1 y, 1 mo) | 80/100 |
| 5 | 19/M | VOE, ACS | Bone marrow/A allele | 8 | 100 | Gr 2/0 | 2.9 | AKI/resolved | Off (1 y, 1 mo) | 90/100 |
| 6 | 7/M | ACS | Bone marrow/none | 8 | 96 | 0/limited (mild) | 2.1 | None | Off (1 y, 5 mo) | 90/100 |
| 7 | 18/F | VOE, ACS | Bone marrow/A antigen | 8 | 100 | Gr 2/extensive (severe) | 1.6 | Peripheral neuropathy/resolved | On | 90/80 |
| 8 | 16/F | VOE, RBC antibodies | Bone marrow/none | 8 | 96 | 0/limited (mild) | 1.5 | None | Off (1 y) | 100/100 |
| 9 | 9/M | VOE, ACS | Cord blood/DRB1 | 4 | 100 | Gr2/0 | 1.5 | Transient red cell aplasia, AKI, cholelithiasis/resolved | Off (1 y, 6 mo) | 90/90 |
| 10 | 8/F | Stroke | Bone marrow/B allele | 8 | 100 | Gr 4/limited (mild) | 1.5 | None | On | 80/90 |
| 11 | 13/F | Stroke | Bone marrow/none | 4 | 0 | NE | GR | None | NE | 90/90 |
| 12 | 21/M | Stroke | Bone marrow/none | 8 | 100 | 0/0 | 1.4 | None | Off (1 y) | 100/100 |
| 13 | 2/M | ACS | Cord blood/B antigen | 4 | 100 | 0/0 | 1 | AKI/resolved | Off (1 y) | 100/100 |
| 14 | 7/M | Stroke | Bone marrow/A antigen | 8 | 100 | 0/0 | 1 | None | Off (1 y) | 70/90 |
| Patient . | Age, y/sex . | Indication . | Stem cell source/HLA match . | Number of abatacept doses received . | Myeloid chimerism at last evaluation, % . | aGVHD/cGVHD (cGVHD NIH grade) . | Follow up, y . | Organ toxicity/outcome . | Systemic immune suppression (time withdrawn) . | Performance score pre/at last follow up . |
|---|---|---|---|---|---|---|---|---|---|---|
| 1 | 10/F | Stroke | Bone marrow/A antigen | 4* | 95 | 0/limited (mild) | 5.5 | SJS/resolved | Off (1 y, 9 mo) | 100/100 |
| 2 | 13/F | VOE | Bone marrow/DRB1 antigen | 4* | 100 | Gr 1/extensive (severe) | 4.8 | PRES/resolved | On | 100/70 |
| 3 | 21/M | Stroke | Bone marrow/none | 8 | 95 | 0/limited (mild) | 4.5 | Pancreatic insufficiency/ongoing | Off (1 y) | 100/100 |
| 4 | 18/F | VOE | Bone marrow/ C allele | 8 | 100 | 0/limited (mild) | 3.3 | None | Off (1 y, 1 mo) | 80/100 |
| 5 | 19/M | VOE, ACS | Bone marrow/A allele | 8 | 100 | Gr 2/0 | 2.9 | AKI/resolved | Off (1 y, 1 mo) | 90/100 |
| 6 | 7/M | ACS | Bone marrow/none | 8 | 96 | 0/limited (mild) | 2.1 | None | Off (1 y, 5 mo) | 90/100 |
| 7 | 18/F | VOE, ACS | Bone marrow/A antigen | 8 | 100 | Gr 2/extensive (severe) | 1.6 | Peripheral neuropathy/resolved | On | 90/80 |
| 8 | 16/F | VOE, RBC antibodies | Bone marrow/none | 8 | 96 | 0/limited (mild) | 1.5 | None | Off (1 y) | 100/100 |
| 9 | 9/M | VOE, ACS | Cord blood/DRB1 | 4 | 100 | Gr2/0 | 1.5 | Transient red cell aplasia, AKI, cholelithiasis/resolved | Off (1 y, 6 mo) | 90/90 |
| 10 | 8/F | Stroke | Bone marrow/B allele | 8 | 100 | Gr 4/limited (mild) | 1.5 | None | On | 80/90 |
| 11 | 13/F | Stroke | Bone marrow/none | 4 | 0 | NE | GR | None | NE | 90/90 |
| 12 | 21/M | Stroke | Bone marrow/none | 8 | 100 | 0/0 | 1.4 | None | Off (1 y) | 100/100 |
| 13 | 2/M | ACS | Cord blood/B antigen | 4 | 100 | 0/0 | 1 | AKI/resolved | Off (1 y) | 100/100 |
| 14 | 7/M | Stroke | Bone marrow/A antigen | 8 | 100 | 0/0 | 1 | None | Off (1 y) | 70/90 |
ACS, acute chest syndrome; AKI, acute kidney injury; F, female; Gr, grade; GR, graft rejection; M, male; NE, not evaluable; NIH, National Institutes of Health; RBC, red blood cell; SJS, Stevens-Johnson syndrome; VOE, vaso-occlusive episodes.
The first 2 patients were treated on an earlier version of the protocol and received 4 doses of abatacept. Abatacept administration was extended to 8 doses in bone marrow transplants subsequently.