Patient and transplant characteristics, ungrouped and by alemtuzumab exposure
| Characteristic . | Ungrouped . | By median exposure . | ||
|---|---|---|---|---|
| N = 53∗ . | Low exposure, n = 27∗ . | High exposure, n = 26∗ . | P value† . | |
| Diagnosis | .012 | |||
| ADA2 deficiency | 1 (1.9%) | 0 (0%) | 1 (3.8%) | |
| APDS type 1 | 1 (1.9%) | 1 (3.7%) | 0 (0%) | |
| CGD | 8 (15%) | 5 (19%) | 3 (12%) | |
| CID | 1 (1.9%) | 0 (0%) | 1 (3.8%) | |
| Congenital severe thrombopenia | 1 (1.9%) | 1 (3.7%) | 0 (0%) | |
| CTLA-4 deficiency | 1 (1.9%) | 0 (0%) | 1 (3.8%) | |
| DOCK8 mutation | 1 (1.9%) | 1 (3.7%) | 0 (0%) | |
| HLH | 7 (13%) | 4 (15%) | 3 (12%) | |
| Hyper-IgE syndrome | 1 (1.9%) | 1 (3.7%) | 0 (0%) | |
| IFNgR1 deficiency | 7 (13%) | 5 (19%) | 2 (7.7%) | |
| IL10RB deficiency | 2 (3.8%) | 2 (7.4%) | 0 (0%) | |
| IPEX | 2 (3.8%) | 2 (7.4%) | 0 (0%) | |
| MKL1 mutation | 1 (1.9%) | 1 (3.7%) | 0 (0%) | |
| SAA | 6 (11%) | 0 (0%) | 6 (23%) | |
| SCID | 10 (19%) | 2 (7.4%) | 8 (31%) | |
| Shwachman-Diamond syndrome | 1 (1.9%) | 1 (3.7%) | 0 (0%) | |
| STIM1 | 1 (1.9%) | 1 (3.7%) | 0 (0%) | |
| WAS | 1 (1.9%) | 0 (0%) | 1 (3.8%) | |
| Diagnosis category | .14 | |||
| Hematological | 8 (15%) | 2 (7.4%) | 6 (23%) | |
| IEI | 45 (85%) | 25 (93%) | 20 (77%) | |
| Sex | .5 | |||
| Female | 20 (38%) | 9 (33%) | 11 (42%) | |
| Male | 33 (62%) | 18 (67%) | 15 (58%) | |
| Age at HSCT, y | 4.4 (0.8-8.7) | 4.0 (0.7-8.6) | 4.9 (1.4-9.8) | .5 |
| Alemtuzumab treatment | ||||
| Cumulative dose mg/kg, | 0.60 (0.60-1.00) | 0.60 (0.55-0.60) | 0.90 (0.60-1.00) | .01 |
| Start day before HSCT | −11.0 (−14.0 to −8.0) | −12.0 (−14.0 to −10.0) | −8.0 (−11.8 to −7.2) | .003 |
| No. of doses | 4 (2-7) | 3 (3-4) | 4 (3-5) | .069 |
| Conditioning | .021 | |||
| Busulfan, fludarabine | 12 (23%) | 5 (19%) | 7 (27%) | |
| Fludarabine, cyclophosphamide | 6 (11%) | 0 (0%) | 6 (23%) | |
| Fludarabine, treosulfan | 18 (34%) | 10 (37%) | 8 (31%) | |
| Fludarabine, treosulfan, thiotepa | 17 (32%) | 12 (44%) | 5 (19%) | |
| Intensity of conditioning | .2 | |||
| Myeloablative | 21 (40%) | 13 (48%) | 8 (31%) | |
| Reduced | 32 (60%) | 14 (52%) | 18 (69%) | |
| GVHD prophylaxis | .3 | |||
| Cyclosporine | 7 (13%) | 2 (7%) | 5 (19%) | |
| Cyclosporine, MMF | 15 (28%) | 7 (26%) | 8 (31%) | |
| Cyclosporine, MTX | 23 (43%) | 12 (44%) | 11 (42%) | |
| MTX, MMF | 1 (2%) | 1 (4%) | 0 (0%) | |
| Cyclosporine, MMF, post-cyclophosphamide | 5 (9%) | 5 (19%) | 0 (0%) | |
| No | 2 (4%) | 0 (0%) | 2 (8%) | |
| Graft source | .7 | |||
| BM | 39 (74%) | 21 (78%) | 18 (69%) | |
| CB | 6 (11%) | 2 (7.4%) | 4 (15%) | |
| PBSC | 8 (15%) | 4 (15%) | 4 (15%) | |
| Graft manipulation | .7 | |||
| CD34 selection | 5 (10%) | 2 (7%) | 3 (12%) | |
| No | 48 (91%) | 25 (93%) | 23 (88%) | |
| Donor type | .025 | |||
| MRD | 12 (23%) | 10 (37%) | 2 (8%) | |
| HAPLO | 2 (3.8%) | 0 (0%) | 2 (8%) | |
| MMD | 17 (32%) | 6 (22%) | 11 (42%) | |
| MUD | 22 (42%) | 11 (41%) | 11 (42%) | |
| Follow-up post-HSCT, y | 3.3 (2.5-8.0) | 2.8 (2.0-3.7) | 6.8 (2.8-10.6) | .005 |
| Characteristic . | Ungrouped . | By median exposure . | ||
|---|---|---|---|---|
| N = 53∗ . | Low exposure, n = 27∗ . | High exposure, n = 26∗ . | P value† . | |
| Diagnosis | .012 | |||
| ADA2 deficiency | 1 (1.9%) | 0 (0%) | 1 (3.8%) | |
| APDS type 1 | 1 (1.9%) | 1 (3.7%) | 0 (0%) | |
| CGD | 8 (15%) | 5 (19%) | 3 (12%) | |
| CID | 1 (1.9%) | 0 (0%) | 1 (3.8%) | |
| Congenital severe thrombopenia | 1 (1.9%) | 1 (3.7%) | 0 (0%) | |
| CTLA-4 deficiency | 1 (1.9%) | 0 (0%) | 1 (3.8%) | |
| DOCK8 mutation | 1 (1.9%) | 1 (3.7%) | 0 (0%) | |
| HLH | 7 (13%) | 4 (15%) | 3 (12%) | |
| Hyper-IgE syndrome | 1 (1.9%) | 1 (3.7%) | 0 (0%) | |
| IFNgR1 deficiency | 7 (13%) | 5 (19%) | 2 (7.7%) | |
| IL10RB deficiency | 2 (3.8%) | 2 (7.4%) | 0 (0%) | |
| IPEX | 2 (3.8%) | 2 (7.4%) | 0 (0%) | |
| MKL1 mutation | 1 (1.9%) | 1 (3.7%) | 0 (0%) | |
| SAA | 6 (11%) | 0 (0%) | 6 (23%) | |
| SCID | 10 (19%) | 2 (7.4%) | 8 (31%) | |
| Shwachman-Diamond syndrome | 1 (1.9%) | 1 (3.7%) | 0 (0%) | |
| STIM1 | 1 (1.9%) | 1 (3.7%) | 0 (0%) | |
| WAS | 1 (1.9%) | 0 (0%) | 1 (3.8%) | |
| Diagnosis category | .14 | |||
| Hematological | 8 (15%) | 2 (7.4%) | 6 (23%) | |
| IEI | 45 (85%) | 25 (93%) | 20 (77%) | |
| Sex | .5 | |||
| Female | 20 (38%) | 9 (33%) | 11 (42%) | |
| Male | 33 (62%) | 18 (67%) | 15 (58%) | |
| Age at HSCT, y | 4.4 (0.8-8.7) | 4.0 (0.7-8.6) | 4.9 (1.4-9.8) | .5 |
| Alemtuzumab treatment | ||||
| Cumulative dose mg/kg, | 0.60 (0.60-1.00) | 0.60 (0.55-0.60) | 0.90 (0.60-1.00) | .01 |
| Start day before HSCT | −11.0 (−14.0 to −8.0) | −12.0 (−14.0 to −10.0) | −8.0 (−11.8 to −7.2) | .003 |
| No. of doses | 4 (2-7) | 3 (3-4) | 4 (3-5) | .069 |
| Conditioning | .021 | |||
| Busulfan, fludarabine | 12 (23%) | 5 (19%) | 7 (27%) | |
| Fludarabine, cyclophosphamide | 6 (11%) | 0 (0%) | 6 (23%) | |
| Fludarabine, treosulfan | 18 (34%) | 10 (37%) | 8 (31%) | |
| Fludarabine, treosulfan, thiotepa | 17 (32%) | 12 (44%) | 5 (19%) | |
| Intensity of conditioning | .2 | |||
| Myeloablative | 21 (40%) | 13 (48%) | 8 (31%) | |
| Reduced | 32 (60%) | 14 (52%) | 18 (69%) | |
| GVHD prophylaxis | .3 | |||
| Cyclosporine | 7 (13%) | 2 (7%) | 5 (19%) | |
| Cyclosporine, MMF | 15 (28%) | 7 (26%) | 8 (31%) | |
| Cyclosporine, MTX | 23 (43%) | 12 (44%) | 11 (42%) | |
| MTX, MMF | 1 (2%) | 1 (4%) | 0 (0%) | |
| Cyclosporine, MMF, post-cyclophosphamide | 5 (9%) | 5 (19%) | 0 (0%) | |
| No | 2 (4%) | 0 (0%) | 2 (8%) | |
| Graft source | .7 | |||
| BM | 39 (74%) | 21 (78%) | 18 (69%) | |
| CB | 6 (11%) | 2 (7.4%) | 4 (15%) | |
| PBSC | 8 (15%) | 4 (15%) | 4 (15%) | |
| Graft manipulation | .7 | |||
| CD34 selection | 5 (10%) | 2 (7%) | 3 (12%) | |
| No | 48 (91%) | 25 (93%) | 23 (88%) | |
| Donor type | .025 | |||
| MRD | 12 (23%) | 10 (37%) | 2 (8%) | |
| HAPLO | 2 (3.8%) | 0 (0%) | 2 (8%) | |
| MMD | 17 (32%) | 6 (22%) | 11 (42%) | |
| MUD | 22 (42%) | 11 (41%) | 11 (42%) | |
| Follow-up post-HSCT, y | 3.3 (2.5-8.0) | 2.8 (2.0-3.7) | 6.8 (2.8-10.6) | .005 |
Based on the median alemtuzumab concentration at day of HSCT (0.77 μg/mL), patients were stratified into the high-exposure (>0.77 μg/mL) or low-exposure groups (≤0.77 μg/mL).
ADA2, adenosine deaminase; APDS, activated PI3 kinase delta syndrome; BM, bone marrow; CB, cord blood; CGD, chronic granulomatous disease; CTLA4, cytotoxic T-lymphocyte associated protein 4; CID, combined immunodeficiency; DOCK8, dedicator of cytokinesis 8; HAPLO, haploidentical; HLH, hemophagocytic lymphohistiocytosis; IgG, immunoglobulin G; IEI, inborn errors of immunity; IFNgR1, interferon γ receptor 1; IL10RB, interleukin 10 receptor subunit β; IPEX, immunodysregulation polyendocrinopathy enteropathy X-linked syndrome; MMD, mismatched donors; MMF, mycophenolate mofetil; MRD, matched related donors; MTX, methotrexate; MUD, matched unrelated donors; PBSC, peripheral blood stem cells; SAA, severe aplastic anemia; SCID, severe combined immunodeficiency; STIM1, stromal interaction molecule; WAS, Wiskott-Aldrich syndrome.
n (%); median (interquartile range).
Fisher exact test; Pearson χ2 test; Wilcoxon rank-sum test.