Patient and HCT characteristics of SCD vs non-SCD
| . | SCD (n = 52), n (%) . | Non-SCD (n = 63), n (%) . | P value . |
|---|---|---|---|
| Diagnosis | 52 (100) | — | — |
| SCD | 16 (25) | ||
| Acute lymphoblastic leukemia (B-, T-, mixed phenotype leukemia) | 16 (25) | ||
| Acute myelogenous leukemia/myelodysplastic syndrome | 1 (2) | ||
| Juvenile myelomonocytic leukemia | 11 (17) | ||
| Immune deficiency/dysregulation | 2 (3.5) | ||
| Lymphoma | 3 (5) | ||
| β-thalassemia severe aplastic anemia | 7 (11) | ||
| Congenital neutropenia | 5 (8) | ||
| Bone marrow failure | 2 (3.5) | ||
| Sex∗ | .19 | ||
| Female | 19 (37) | 31 (49) | |
| Male | 33 (63) | 32 (51) | |
| Age, median (interquartile range), y† | 8 (7) | 10 (12) | .33 |
| Race‡ | <.0001 | ||
| Black | 50 (96) | 11 (17) | |
| White | 2 (4) | 33 (52) | |
| Asian | 0 (0) | 7 (11) | |
| American Indian | 0 (0) | 2 (3) | |
| Hawaiian/Pacific Islander | 0 (0) | 1 (2) | |
| Unknown | 0 (0) | 9 (14) | |
| Ethnicity∗ | <.0001 | ||
| Hispanic/Latino | 2 (4) | 21 (33) | |
| Non-Hispanic/Latino | 50 (96) | 42 (67) | |
| Conditioning intensity∗ | .05 | ||
| Myeloablative | 51 (98) | 47 (75) | |
| Reduced intensity conditioning | 1 (2) | 16 (25) | |
| Preparative regimen agents‡ | <.0001 | ||
| Total body irradiation (<400 Gy) | 5 (10) | 6 (10) | |
| Total body irradiation (≥400 Gy) | 0 (0) | 19 (30) | |
| Busulfan containing | 40 (77) | 24 (38) | |
| Acute GVHD prophylaxis‡ | <.0001 | ||
| Calcineurin inhibitor | 47 (90) | 56 (89) | |
| Methotrexate | 45 (87) | 34 (54) | |
| Mycophenolate | 0 (0) | 20 (32) | |
| Sirolimus | 6 (12) | 3 (5) | |
| Posttransplant cyclophosphamide | 6 (12) | 14 (22) | |
| Abatacept | 1 (2) | 0 (0) | |
| αβ T-cell depletion | 0 (0) | 4 (6) | |
| HLA matching∗ | .04 | ||
| 10/10 MSD | 46 (88) | 45 (71) | |
| Haploidentical | 6 (12) | 18 (29) | |
| Stem cell source‡ | .0008 | ||
| Bone marrow | 52 (100) | 46 (73) | |
| PBSC | 0 (0) | 14 (22) | |
| UC‡ | 1 (2)§ | 3 (5) | |
| Cytomegalovirus serostatus‡ (recipient/donor) | .003 | ||
| Positive/positive | 13 (25) | 38 (60) | |
| Negative/positive | 7 (13) | 5 (8) | |
| Positive/negative | 7 (13) | 10 (16) | |
| Negative/negative | 25 (48) | 10 (16) | |
| TA-TMA∗ | 7(13) | 1 (2) | .002 |
| TA-TMA treatment‡ | .56 | ||
| Eculizumab treatment | 3 (43) | 1 (100) | |
| Change GVHD prophylaxis | 3 (43) | 0 (0) | |
| Supportive care | 1 (14) | 0 (0) | |
| VOD∗ | 2 (4) | 11 (17) | .02 |
| Defibrotide | |||
| Treatment | 1 (50) | 4 (36) | .71 |
| Prophylaxis | 0 (0) | 2 (18) | |
| Posterior reversible encephalopathy syndrome∗ | 3 (6) | 1 (2) | .22 |
| Acute GVHD‡ | .058 | ||
| Grade 0 | 43 (83) | 43 (69) | |
| Grade 1-2 | 5 (10) | 17 (27) | |
| Grade 3-4 | 4 (8) | 3 (5) | |
| Infections within 1 y after HCT∗ | .067 | ||
| Bacteremia|| | 26 (50) | 30 (48) | |
| Viral|| | 27 (52) | 23 (37) | |
| Fungal|| | 0 (0) | 5 (8) | |
| Estimated overall survival, %¶ | .008 | ||
| d 100 | 100 | 100 | |
| d 180 | 100 | 90 | |
| 1 y | 100 | 87 | |
| Estimated NRM, %# | .12 | ||
| d 100 | 0 | 2 | |
| d 180 | 0 | 3 | |
| 1 y | 0 | 5 |
| . | SCD (n = 52), n (%) . | Non-SCD (n = 63), n (%) . | P value . |
|---|---|---|---|
| Diagnosis | 52 (100) | — | — |
| SCD | 16 (25) | ||
| Acute lymphoblastic leukemia (B-, T-, mixed phenotype leukemia) | 16 (25) | ||
| Acute myelogenous leukemia/myelodysplastic syndrome | 1 (2) | ||
| Juvenile myelomonocytic leukemia | 11 (17) | ||
| Immune deficiency/dysregulation | 2 (3.5) | ||
| Lymphoma | 3 (5) | ||
| β-thalassemia severe aplastic anemia | 7 (11) | ||
| Congenital neutropenia | 5 (8) | ||
| Bone marrow failure | 2 (3.5) | ||
| Sex∗ | .19 | ||
| Female | 19 (37) | 31 (49) | |
| Male | 33 (63) | 32 (51) | |
| Age, median (interquartile range), y† | 8 (7) | 10 (12) | .33 |
| Race‡ | <.0001 | ||
| Black | 50 (96) | 11 (17) | |
| White | 2 (4) | 33 (52) | |
| Asian | 0 (0) | 7 (11) | |
| American Indian | 0 (0) | 2 (3) | |
| Hawaiian/Pacific Islander | 0 (0) | 1 (2) | |
| Unknown | 0 (0) | 9 (14) | |
| Ethnicity∗ | <.0001 | ||
| Hispanic/Latino | 2 (4) | 21 (33) | |
| Non-Hispanic/Latino | 50 (96) | 42 (67) | |
| Conditioning intensity∗ | .05 | ||
| Myeloablative | 51 (98) | 47 (75) | |
| Reduced intensity conditioning | 1 (2) | 16 (25) | |
| Preparative regimen agents‡ | <.0001 | ||
| Total body irradiation (<400 Gy) | 5 (10) | 6 (10) | |
| Total body irradiation (≥400 Gy) | 0 (0) | 19 (30) | |
| Busulfan containing | 40 (77) | 24 (38) | |
| Acute GVHD prophylaxis‡ | <.0001 | ||
| Calcineurin inhibitor | 47 (90) | 56 (89) | |
| Methotrexate | 45 (87) | 34 (54) | |
| Mycophenolate | 0 (0) | 20 (32) | |
| Sirolimus | 6 (12) | 3 (5) | |
| Posttransplant cyclophosphamide | 6 (12) | 14 (22) | |
| Abatacept | 1 (2) | 0 (0) | |
| αβ T-cell depletion | 0 (0) | 4 (6) | |
| HLA matching∗ | .04 | ||
| 10/10 MSD | 46 (88) | 45 (71) | |
| Haploidentical | 6 (12) | 18 (29) | |
| Stem cell source‡ | .0008 | ||
| Bone marrow | 52 (100) | 46 (73) | |
| PBSC | 0 (0) | 14 (22) | |
| UC‡ | 1 (2)§ | 3 (5) | |
| Cytomegalovirus serostatus‡ (recipient/donor) | .003 | ||
| Positive/positive | 13 (25) | 38 (60) | |
| Negative/positive | 7 (13) | 5 (8) | |
| Positive/negative | 7 (13) | 10 (16) | |
| Negative/negative | 25 (48) | 10 (16) | |
| TA-TMA∗ | 7(13) | 1 (2) | .002 |
| TA-TMA treatment‡ | .56 | ||
| Eculizumab treatment | 3 (43) | 1 (100) | |
| Change GVHD prophylaxis | 3 (43) | 0 (0) | |
| Supportive care | 1 (14) | 0 (0) | |
| VOD∗ | 2 (4) | 11 (17) | .02 |
| Defibrotide | |||
| Treatment | 1 (50) | 4 (36) | .71 |
| Prophylaxis | 0 (0) | 2 (18) | |
| Posterior reversible encephalopathy syndrome∗ | 3 (6) | 1 (2) | .22 |
| Acute GVHD‡ | .058 | ||
| Grade 0 | 43 (83) | 43 (69) | |
| Grade 1-2 | 5 (10) | 17 (27) | |
| Grade 3-4 | 4 (8) | 3 (5) | |
| Infections within 1 y after HCT∗ | .067 | ||
| Bacteremia|| | 26 (50) | 30 (48) | |
| Viral|| | 27 (52) | 23 (37) | |
| Fungal|| | 0 (0) | 5 (8) | |
| Estimated overall survival, %¶ | .008 | ||
| d 100 | 100 | 100 | |
| d 180 | 100 | 90 | |
| 1 y | 100 | 87 | |
| Estimated NRM, %# | .12 | ||
| d 100 | 0 | 2 | |
| d 180 | 0 | 3 | |
| 1 y | 0 | 5 |
Calcineurin inhibitors included tacrolimus or cyclosporine. GVHD, graft versus host disease; gy, gray; NRM, non-relapse mortality; PBSC, peripheral blood stem cells; UC, umbilical cord; VOD, veno-occlusive disease.
Defibrotide treatment and prophylaxis were given for VOD. There were no missing data. Significant values <0.05 are bolded.
Fisher exact test.
Wilcoxon rank-sum test.
χ2 test.
This patient received a bone marrow and UC stem cell product.
Some patients had multiple infections.
Estimated using the Kaplan-Meier curve.
Estimated using the cumulative incidence curve.