Demographics of the eculizumab-treated cohort
| . | Total participants . |
|---|---|
| Male sex | 15 (71%) |
| Age at transplant (y) | 11.8 (IQR, 4-16.6; range, 0.5-34) |
| Weight (kg) | 36.5 (IQR, 16.2-58.5; range, 10.3-87.5) |
| HCT number∗ | |
| First | 14 (66.7%) |
| Second | 5 (23.8%) |
| Third | 2 (9.5%) |
| Received solid organ transplantation before HCT | 1 (4.7%) |
| Race | |
| White | 14 (67%) |
| African American | 2 (9.5%) |
| Asian | 2 (9.5%) |
| White/African American | 3 (14.3%) |
| Diagnosis | |
| Malignancy | 11(52.4%) |
| Immune deficiency | 5 (23.8%) |
| Marrow failure | 4 (19.1%) |
| Benign hematology | 1 (4.7%) |
| Stem cell donor type | |
| Related (MRD, MMRD, haplo) | 4 (19%) |
| Unrelated (MUD, MMUD) | 12 (57.1%) |
| Autologous† | 5 (23.8%) |
| Stem cell donor gender and age (y) | |
| Female donors | 43.7% (7 of 16) |
| Donor age, y | 25 (IQR, 14-37) |
| Stem cell source | |
| Bone marrow | 9 (42.9%) |
| Peripheral blood (PBSCs) | 10 (47.6%) |
| Cord blood | 2 (9.5%) |
| HLA match (allogeneic HCT, n = 16) | |
| Fully matched (MRD, MUD) | 11 (68.8%) |
| Mismatched (MMR, MMUD, haplo) | 5 (31.2%) |
| Conditioning regimen type | |
| Myeloablative | 15 (71.4%) |
| Reduced intensity | 6 (28.6%) |
| Total body radiation | 4 (19%) |
| GVHD prophylaxis (allogeneic HCT, n = 16) | |
| CNIs (CSA, Tacro) | 12 (75%) |
| T-cell depletion | 4 (25%) |
| Pretransplant cystatin-C GFR (mL/min per 1.73 m2) | 104 (IQR, 91-114) |
| Pretransplant serum creatinine (mg/dL) | 0.3 (IQR, 0.3-0.6) |
| rUPCR (mg/mg) | |
| Before transplantation | 0.3 (IQR, 0.2-0.5) |
| At hrTMA diagnosis | 3.4 (IQR, 1.6-5.2) |
| Max rUPCR during active hrTMA | 6.3 (IQR, 4.8-15) |
| Terminal complement activation, sC5b-9, (normal <244 ng/mL) | |
| Pretransplant sC5b-9 (ng/mL) | 117 (IQR, 99-128) |
| At hrTMA diagnosis, sC5b-9 (ng/mL) | 367 ng/mL (IQR, 277-479) |
| sC5b-9 change (Δ,delta) from pre-HCT baseline to hrTMA diagnosis (times increase) | 3.1 times (IQR, 2.4-3.8) |
| . | Total participants . |
|---|---|
| Male sex | 15 (71%) |
| Age at transplant (y) | 11.8 (IQR, 4-16.6; range, 0.5-34) |
| Weight (kg) | 36.5 (IQR, 16.2-58.5; range, 10.3-87.5) |
| HCT number∗ | |
| First | 14 (66.7%) |
| Second | 5 (23.8%) |
| Third | 2 (9.5%) |
| Received solid organ transplantation before HCT | 1 (4.7%) |
| Race | |
| White | 14 (67%) |
| African American | 2 (9.5%) |
| Asian | 2 (9.5%) |
| White/African American | 3 (14.3%) |
| Diagnosis | |
| Malignancy | 11(52.4%) |
| Immune deficiency | 5 (23.8%) |
| Marrow failure | 4 (19.1%) |
| Benign hematology | 1 (4.7%) |
| Stem cell donor type | |
| Related (MRD, MMRD, haplo) | 4 (19%) |
| Unrelated (MUD, MMUD) | 12 (57.1%) |
| Autologous† | 5 (23.8%) |
| Stem cell donor gender and age (y) | |
| Female donors | 43.7% (7 of 16) |
| Donor age, y | 25 (IQR, 14-37) |
| Stem cell source | |
| Bone marrow | 9 (42.9%) |
| Peripheral blood (PBSCs) | 10 (47.6%) |
| Cord blood | 2 (9.5%) |
| HLA match (allogeneic HCT, n = 16) | |
| Fully matched (MRD, MUD) | 11 (68.8%) |
| Mismatched (MMR, MMUD, haplo) | 5 (31.2%) |
| Conditioning regimen type | |
| Myeloablative | 15 (71.4%) |
| Reduced intensity | 6 (28.6%) |
| Total body radiation | 4 (19%) |
| GVHD prophylaxis (allogeneic HCT, n = 16) | |
| CNIs (CSA, Tacro) | 12 (75%) |
| T-cell depletion | 4 (25%) |
| Pretransplant cystatin-C GFR (mL/min per 1.73 m2) | 104 (IQR, 91-114) |
| Pretransplant serum creatinine (mg/dL) | 0.3 (IQR, 0.3-0.6) |
| rUPCR (mg/mg) | |
| Before transplantation | 0.3 (IQR, 0.2-0.5) |
| At hrTMA diagnosis | 3.4 (IQR, 1.6-5.2) |
| Max rUPCR during active hrTMA | 6.3 (IQR, 4.8-15) |
| Terminal complement activation, sC5b-9, (normal <244 ng/mL) | |
| Pretransplant sC5b-9 (ng/mL) | 117 (IQR, 99-128) |
| At hrTMA diagnosis, sC5b-9 (ng/mL) | 367 ng/mL (IQR, 277-479) |
| sC5b-9 change (Δ,delta) from pre-HCT baseline to hrTMA diagnosis (times increase) | 3.1 times (IQR, 2.4-3.8) |
sC5b-9, also known as membrane attack complex.
haplo, haploidentical; MMRD, mismatched related; MMUD, mismatched unrelated; MRD, matched related; MUD, matched unrelated; PBSCs, peripheral blood stem cells; Tacro, tacrolimus.
Second transplantation: autologous tandem transplant for neuroblastoma (n = 4), and allogeneic HCT (n = 1). Third transplantation: all allogeneic HCT (n = 2);
Neuroblastoma after second tandem HCT (n = 4) and rhabdoid tumor (n = 1).