Characteristics of study participants
| Variable . | Total (n = 70) . | Pediatric (n = 32) <18 y . | Adult (n = 38) ≥18 y . | P value∗ . |
|---|---|---|---|---|
| Age at transplant, median (IQR), y | 19.1 (14.1-25.0) | 13.8 (8.0-16.0) | 24.9 (20.4-31.3) | NA |
| Sex (male), n (%) | 31 (44.3) | 14 (43.8) | 17 (44.7) | .934 |
| SCD genotype (SS and Sβ0-thalassemia), n (%) | 68 (97.1) | 32 (100.0) | 36 (94.7) | .234† |
| Follow-up time, median (IQR), y | 2.4 (1.5-3.9) | 2.2 (0.5-3.8) | 3.1 (1.7-4.7) | .088 |
| Transplantation indication, n (%)‡ | ||||
| Stroke (overt and silent infarcts) | 37 (52.9) | 21 (65.6) | 16 (42.1) | .050 |
| Acute chest syndrome | 36 (51.4) | 13 (40.6) | 23 (60.5) | .097 |
| Osteonecrosis | 7 (10.0) | 3 (9.4) | 4 (10.5) | 1.000† |
| Sickle nephropathy | 1 (1.4) | 0 (0.0) | 1 (2.6) | 1.000† |
| Pulmonary hypertension | 4 (5.7) | 0 (0.0) | 4 (10.5) | .120† |
| Alloimmunization | 16 (22.9) | 5 (15.6) | 11 (28.9) | .186 |
| Donor characteristics | ||||
| Donor age, mean (SD) | 34.4 (12.1) | 36.8 (10.9) | 32.4 (12.8) | .127 |
| Donor sex, male, n (%) | 33 (47.1) | 16 (50.0) | 17 (44.7) | .660 |
| Donor relationship, n (%) | .003† | |||
| Mother | 19 (27.1) | 13 (40.6) | 6 (15.8) | |
| Father | 15 (21.4) | 10 (31.2) | 5 (13.2) | |
| Sibling | 33 (47.1) | 9 (28.1) | 24 (63.2) | |
| Other | 3 (4.3) | 0 (0.0) | 3 (7.9) | |
| Sickle cell trait (AS) | 56 (80.0) | 30 (93.8) | 26 (68.4) | .008 |
| Donor/recipient sex match, n (%) | .473 | |||
| Sex-matched transplant | 36 (51.4) | 14 (43.8) | 22 (57.9) | |
| Female donor, male recipient | 16 (22.9) | 8 (25.0) | 8 (21.1) | |
| Male donor, female recipient | 18 (25.7) | 10 (31.3) | 8 (2115) | |
| ABO incompatibility, n (%), (n = 68) | .586† | |||
| No incompatibility | 52 (76.5) | 22 (73.3) | 30 (79.0) | |
| Minor incompatibility | 10 (14.7) | 4 (13.3) | 6 (15.8) | |
| Major incompatibility | 6 (8.8) | 4 (13.3) | 2 (5.3) | |
| CMV serostatus, n (%) | .134† | |||
| CMV-seronegative recipient and donor | 4 (5.7) | 2 (6.3) | 2 (5.3) | |
| CMV-seropositive recipient and donor | 47 (67.1) | 19 (59.4) | 28 (73.7) | |
| CMV-seronegative recipient and CMV- seropositive donor | 14 (20.0) | 10 (31.3) | 4 (10.5) | |
| CMV-seropositive recipient and CMV-seronegative donor | 5 (7.1) | 1 (3.1) | 4 (10.5) | |
| Degree of HLA match, n (%) | .008† | |||
| 5/10 | 24 (34.3) | 7 (21.9) | 17 (44.7) | |
| 6/10 | 11 (15.7) | 4 (12.5) | 7 (18.4) | |
| 7/10 | 1 (1.4) | 0 (0.0) | 1 (2.6) | |
| 8/10 | 2 (2.9) | 1 (3.1) | 1 (2.6) | |
| 9/10 | 2 (2.9) | 0 (0.0) | 2 (5.3) | |
| 6/12 | 22 (31.4) | 16 (50.0) | 6 (15.8) | |
| 7/12 | 4 (5.7) | 1 (3.1) | 3 (7.9) | |
| 8/12 | 3 (4.3) | 3 (9.4) | 0 (0.0) | |
| 9/12 | 1 (1.4) | 0 (0.0) | 1 (2.6) | |
| DSA positive, n (%) | 7 (10.0) | 6 (21.9) | 0 (0.0) | .003† |
| TNC dose (108/kg), median (IQR) | 5.9 (3.6-8.7) | 5.7 (2.8-8.6) | 6.4 (3.8-9.1) | .361 |
| CD34+ cell dose (106/kg), median (IQR) | 4.1 (2.5-5.9) | 4.2 (2.7-7.5) | 3.8 (2.4-5.6) | .275 |
| Graft source, n (%) | .166† | |||
| BM | 61 (87.1) | 30 (93.8) | 31 (81.6) | |
| Primed BM | 9 (12.9) | 2 (6.3) | 7 (18.4) |
| Variable . | Total (n = 70) . | Pediatric (n = 32) <18 y . | Adult (n = 38) ≥18 y . | P value∗ . |
|---|---|---|---|---|
| Age at transplant, median (IQR), y | 19.1 (14.1-25.0) | 13.8 (8.0-16.0) | 24.9 (20.4-31.3) | NA |
| Sex (male), n (%) | 31 (44.3) | 14 (43.8) | 17 (44.7) | .934 |
| SCD genotype (SS and Sβ0-thalassemia), n (%) | 68 (97.1) | 32 (100.0) | 36 (94.7) | .234† |
| Follow-up time, median (IQR), y | 2.4 (1.5-3.9) | 2.2 (0.5-3.8) | 3.1 (1.7-4.7) | .088 |
| Transplantation indication, n (%)‡ | ||||
| Stroke (overt and silent infarcts) | 37 (52.9) | 21 (65.6) | 16 (42.1) | .050 |
| Acute chest syndrome | 36 (51.4) | 13 (40.6) | 23 (60.5) | .097 |
| Osteonecrosis | 7 (10.0) | 3 (9.4) | 4 (10.5) | 1.000† |
| Sickle nephropathy | 1 (1.4) | 0 (0.0) | 1 (2.6) | 1.000† |
| Pulmonary hypertension | 4 (5.7) | 0 (0.0) | 4 (10.5) | .120† |
| Alloimmunization | 16 (22.9) | 5 (15.6) | 11 (28.9) | .186 |
| Donor characteristics | ||||
| Donor age, mean (SD) | 34.4 (12.1) | 36.8 (10.9) | 32.4 (12.8) | .127 |
| Donor sex, male, n (%) | 33 (47.1) | 16 (50.0) | 17 (44.7) | .660 |
| Donor relationship, n (%) | .003† | |||
| Mother | 19 (27.1) | 13 (40.6) | 6 (15.8) | |
| Father | 15 (21.4) | 10 (31.2) | 5 (13.2) | |
| Sibling | 33 (47.1) | 9 (28.1) | 24 (63.2) | |
| Other | 3 (4.3) | 0 (0.0) | 3 (7.9) | |
| Sickle cell trait (AS) | 56 (80.0) | 30 (93.8) | 26 (68.4) | .008 |
| Donor/recipient sex match, n (%) | .473 | |||
| Sex-matched transplant | 36 (51.4) | 14 (43.8) | 22 (57.9) | |
| Female donor, male recipient | 16 (22.9) | 8 (25.0) | 8 (21.1) | |
| Male donor, female recipient | 18 (25.7) | 10 (31.3) | 8 (2115) | |
| ABO incompatibility, n (%), (n = 68) | .586† | |||
| No incompatibility | 52 (76.5) | 22 (73.3) | 30 (79.0) | |
| Minor incompatibility | 10 (14.7) | 4 (13.3) | 6 (15.8) | |
| Major incompatibility | 6 (8.8) | 4 (13.3) | 2 (5.3) | |
| CMV serostatus, n (%) | .134† | |||
| CMV-seronegative recipient and donor | 4 (5.7) | 2 (6.3) | 2 (5.3) | |
| CMV-seropositive recipient and donor | 47 (67.1) | 19 (59.4) | 28 (73.7) | |
| CMV-seronegative recipient and CMV- seropositive donor | 14 (20.0) | 10 (31.3) | 4 (10.5) | |
| CMV-seropositive recipient and CMV-seronegative donor | 5 (7.1) | 1 (3.1) | 4 (10.5) | |
| Degree of HLA match, n (%) | .008† | |||
| 5/10 | 24 (34.3) | 7 (21.9) | 17 (44.7) | |
| 6/10 | 11 (15.7) | 4 (12.5) | 7 (18.4) | |
| 7/10 | 1 (1.4) | 0 (0.0) | 1 (2.6) | |
| 8/10 | 2 (2.9) | 1 (3.1) | 1 (2.6) | |
| 9/10 | 2 (2.9) | 0 (0.0) | 2 (5.3) | |
| 6/12 | 22 (31.4) | 16 (50.0) | 6 (15.8) | |
| 7/12 | 4 (5.7) | 1 (3.1) | 3 (7.9) | |
| 8/12 | 3 (4.3) | 3 (9.4) | 0 (0.0) | |
| 9/12 | 1 (1.4) | 0 (0.0) | 1 (2.6) | |
| DSA positive, n (%) | 7 (10.0) | 6 (21.9) | 0 (0.0) | .003† |
| TNC dose (108/kg), median (IQR) | 5.9 (3.6-8.7) | 5.7 (2.8-8.6) | 6.4 (3.8-9.1) | .361 |
| CD34+ cell dose (106/kg), median (IQR) | 4.1 (2.5-5.9) | 4.2 (2.7-7.5) | 3.8 (2.4-5.6) | .275 |
| Graft source, n (%) | .166† | |||
| BM | 61 (87.1) | 30 (93.8) | 31 (81.6) | |
| Primed BM | 9 (12.9) | 2 (6.3) | 7 (18.4) |
A summary depicting the clinical characteristics of 70 evaluable participants who received the conditioning regimen as per protocol, and their donors.
CMV, cytomegalovirus; DSA, donor-specific antigens; SD, standard deviation; TNC, total nucleated cell dose.
χ2 test for the count, t test for mean, or Mann-Whitney test for the median, unless otherwise indicated.
Fisher exact test.
Some patients had >1 indication.