1. Basic information and transplant characteristics of children: The basic characteristics of the 118 children are shown in Table S1. Among them, kids with WAS and CGD were statistically different in terms of the age at transplant, nucleated cell infusion volume, CD34 + cell infusion volume, graft type, and ATG use. The transplantation age of children in the WAS group was apparently younger compared with that in the CGD group; the nucleated cell infusion volume and CD34 + cell infusion volume in the CGD group had been greater compared with that in the WAS group; the proportion of UCBT in the WAS group was apparently higher compared with that in the CGD group; and the proportion of patients receiving ATG pretreatment in the CGD group used to be considerably greater compared with that in the WAS group. There were also differences in infection between the WAS and CGD groups before transplantation: the number of CMV infections in the WAS group was higher than that in the CGD group, and the numbers of EBV and fungal infections in the CGD group were higher than those in the WAS group. All children in the WAS and CGD groups were male, 1 child in the WAS group was transplanted with CB + BM, and the rest were transplanted with a single graft. As of December 31, 2022, one child in the WAS group died of respiratory and circulatory failure due to severe pulmonary infection in the 11th month after allo-HSCT (2013/7/2), and the remaining 117 children survived. The median follow-up time was 78.1 months for the surviving children in the WAS group (69 children) and 52 months for the surviving children in the CGD group (48 children).
2. Reconstruction of lymphocyte subsets in the WAS and CGD groups (shown in Table S2 and Fig.S1 (A, B, C, D, E)).
On Day 15 after allo-HSCT, the WAS group had significantly higher NK cell counts than the CGD group [233.47 (59.69-395.78) ×107/L vs. 129.25 (28.41-217.53) ×107/L, P = 0.022]. On Days 30, 100 and 180 after allo-HSCT, the WAS group had notably higher CD4 + T-cell counts than the CGD group [305.47 (117.81–427.50) ×107/L vs. 134.77 (62.74-244.97) ×107/L, P < 0.001], [312.80 (127.00-546.95) ×107/L vs. 148.64 (111.06-282.32) ×107/L, P = 0.005], [397.93 (228.15-631.74) ×107/L vs. 179.85(120.61-366.11)×107/L, P = 0.004]. On Days 100 and 180 after allo-HSCT, the WAS group had considerably higher B-cell counts than the CGD group [32.28 (12.24-231.74) ×107/L vs. 18.43 (8.39–55.82) ×107/L, P = 0.027], [132.05 (43.04–418.20) ×107/L vs. 37.32 (14.96-132.36) ×107/L, P = 0.002].
3. The reconstitution of lymphocyte subsets and analysis of graft types in children aged 1–3 years in the WAS and CGD groups (shown in Table S3 and Table S4, Fig.S2 (A, B, C, D, E)).
We selected the age group with the largest number of transplants in WAS and CGD groups, i.e., children aged 1–3 years who underwent allo-HSCT, and we analyzed the reconstruction of lymphocyte subpopulations in these children at different time points after allo-HSCT. The results are as follows.
On Day 15 posttransplantation, NK cell counts in the WAS group were considerably higher than those in the CGD group among children aged 1–3 years who underwent transplants [265.60 (61.98-375.97) ×107/L vs. 113.46 (26.85-223.29) ×107/L, P = 0.050]. On Days 30 and 180 posttransplantation, the WAS group had notably higher CD4 + T-cell counts than the CGD group among kids aged 1–3 years who underwent transplants [325.80 (143.29–465.00) ×107/L vs. 133.93 (63.94-241.75) ×107/L, P = 0.011], [308.20 (201.21–526.40) ×107/L vs. 174.39 (129.96-282.96) ×107/L, P = 0.025]. On Day 180 posttransplantation, B-cell counts in the WAS group were consistently higher than those in the CGD group among children aged 1–3 years who underwent transplants [131.20 (95.70-266.64) ×107/L vs. 28.02 (13.10-104.23) ×107/L, P = 0.003]. On Day 360 posttransplantation, the CGD group had notably higher CD8 + T-cell counts than the WAS group among kids aged 1–3 years who underwent transplants [1278.28 (674.62-3540.85)×107/L vs. 578.54 (454.63-803.18)×107/L, P = 0.021].
In addition, the study compared the graft types of children in the WAS and CGD groups who underwent allo-HSCT at the age of 1–3 years. The results showed that there were no significant differences in graft type between children in the WAS and CGD groups who underwent allo-HSCT at the age of 1–3 years (P = 0.100).
4. Reconstruction of lymphocyte subsets after umbilical cord blood transplantation (UCBT) and non-UCBT in the WAS group (shown in Table S5 and Fig.S3 (A, B, C, D, E)).
On Days 15 and 30 posttransplantation, children who underwent non-UCBT had significantly higher B-cell counts than children who underwent UCBT in the WAS group [15.55 (5.69–60.66) ×107/L vs. 0.48 (0.00-1.41) ×107/L, P < 0.001], [14.63 (8.48–25.46) ×107/L vs. 1.24 (0.20–2.23) ×107/L, P < 0.001]. On Days 100 and 180 posttransplantation, children who underwent UCBT had apparently higher B-cell counts than children who underwent non-UCBT in the WAS group [250.80 (125.50-552.30) ×107/L vs. 24.99 (11.18–89.16) ×107/L, P = 0.001], [445.600 (131.20-712.71) ×107/L vs. 96.60 (39.24-191.95) ×107/L, P < 0.001]. On Day 30 posttransplantation, kids who underwent UCBT had notably higher CD3 + T-cell counts than kids who underwent non-UCBT in the WAS group [1427.40 (961.69-2080.18) ×107/L vs. 914.92 (282.17-1614.18) ×107/L, P = 0.026]. On Days 30, 100 and 180 posttransplantation, kids who underwent UCBT had obviously higher CD4 + T-cell counts than kids who underwent non-UCBT in the WAS group [507.04 (218.31-705.45) ×107/L vs. 244.39 (97.21-401.69) ×107/L, P = 0.003], [466.82 (289.30-734.57) ×107/L vs. 260.25 (118.72-479.53) ×107/L, P = 0.038], [571.29 (384.52–832.00) ×107/L vs. 308.20 (168.48–488.70) ×107/L, P = 0.002]. On Day 360 posttransplantation, children who underwent UCBT had markedly higher NK cell counts than children who underwent non-UCBT in the WAS group [668.62 (515.46-901.64) ×107/L vs. 346.13 (229.56-488.45) ×107/L, P = 0.007].
5. Reconstruction of lymphocyte subsets after non-UCBT in the WAS and CGD groups (shown in Table S6 and Fig.S4 (A, B, C, D, E)).
On Day 15 posttransplantation, NK cell counts were probably higher in the non-cord-blood-transplanted kids with WAS compared to the non-cord-blood-transplanted kids with CGD [233.47 (63.09-438.78) ×107/L vs. 129.69 (28.02-222.26) ×107/L, P = 0.043]. On Days 30 and 100 posttransplantation, CD4 + T-cell counts were significantly higher in the non-cord-blood-transplanted kids with WAS compared to the non-cord-blood-transplanted kids with CGD [244.39 (97.21-401.69) ×107/L vs. 139.22 (63.26-253.12) ×107/L, P = 0.024], [260.25 (118.72-479.53) ×107/L vs. 148.64 (111.06-282.32) ×107/L, P = 0.049]. On Day 30 posttransplantation, B-cell counts were notably higher in the non-cord-blood-transplanted kids with WAS compared to the non-cord-blood-transplanted kids with CGD [14.63 (8.48–25.46) ×107/L vs. 4.51 (2.21–11.52) ×107/L, P < 0.001].
6. Immunoglobulin reconstitution in the WAS and CGD groups (shown in Table S7 and Fig.S5 (A, B, C, D, E, F)).
On Day 100 after allo-HSCT, the CGD group had higher C3 levels than the WAS group [0.9000 (0.8075–1.0750) ×g/L vs. 0.7650 (0.6900-0.9825) ×g/L, P = 0.002]. On Day 360 after allo-HSCT, the CGD group had higher IgA and C4 levels than the WAS group [0.4930 (0.3595–0.76975) ×g/L vs. 0.4030 (0.2740–0.5320) ×g/L, P = 0.033], [0.2000 (0.1475-0.2200) ×g/L vs. 0.150 (0.130–0.180) ×g/L, P = 0.004]. There was no statistically significant change in immunoglobulin levels between the both groups on Days 15, 30 and 180 after allo-HSCT (P > 0.05). Among the patients who underwent allo-HSCT in our center, due to the influence of regular infusions of gamma globulin (IVIG), the IgG levels of the WAS and CGD groups of patients at various time points posttransplantation are not of comparative value.
7. Infection and aGVHD after allo-HSCT in the WAS and CGD groups (shown in Table S8)
In this paper, children in the WAS and CGD groups were divided into different age groups (< 1 year, 1–3 years, 3–5 years, and > 5 years), and we compared the numbers of posttransplantation infections and occurrences of aGVHD in children in different age groups. In children younger than 1 year of age, the numbers of posttransplantation EBV and fungal infections were higher in the CGD group than in the WAS group. In children 1–3 years of age, the numbers of posttransplantation EBV and CMV infections were higher in the CGD group than in the WAS group. In children older than 5 years, the number of posttransplantation fungal infections was higher in the CGD group than in the WAS group.