Table 1.
Characteristics of Included Patients.
| All patients (N=315) | |
|---|---|
| Sex, female (%, n) | 40.3% (127) |
| Median age at HCT (years, IQR) | 10.4 (5.0 – 16.5) |
| Year of transplantation | |
| – 2008 – 2011 | 41.9% (132) |
| – 2012 – 2014 | 30.2% (95) |
| – 2015 – 2017 | 27.9% (88) |
| Diagnosis | |
| – Malignancy | 69.8% (220) |
| – AML | 25.1% (79) |
| – ALL | 31.4% (99) |
| – Other (CML, JMML, Leukemia other, FAζ, MDS, NHL/HD, Sarcoma) | 13.3% (42) |
| – Non-malignant | 30.2% (95) |
| – Group 1: ALD, SSD, Thal, SCN, CGD, | 5.4% (17) |
| – Group 2: DBA, CMT, DC,FA, SAA, PNH, SAA, SDS | 12.1% (38) |
| – Group 3: CID, SCID, HLH, LAD, WAS, AHA | 12.7% (40) |
| Disease type malignancy ALL vs AML | |
| – ALL in CR1/CR2 | 27.0% (85) |
| – ALL in CR3+ or other | 4.4% (14) |
| – AML in CR1 | 14.0% (44) |
| – AML in CR2/CR3+/other | 11.1% (35) |
| Stem cell source (%, n) | |
| – Unmodified (T-replete) graft | 18.7% (59) |
| -Bone Marrow + Cord Blood | 2.5% (8) |
| – T cell depleted graft* | 66.0% (208) |
| – Cord blood | 12.7% (40) |
| Donor relation / match | |
| – MMRD | 8.9% (28) |
| – MMUD | 24.4% (77) |
| – MRD | 28.6% (90) |
| – MUD | 25.4% (80) |
| – Cord blood | 12.7% (40) |
| CMV serostatus recipient/donor | |
| – R-/D- | 37.1% (117) |
| – R-/D+ | 12.4% (39) |
| – R+/D- | 19.7% (62) |
| – R+/D+ | 30.8% (97) |
| Conditioning | |
| – Ablative | 95.2% (300) |
| – Non-ablative | 3.5% (11) |
| – None | 1.3% (4) |
| Conditioning regimen | |
| – Chemotherapy based± | 63.2% (199) |
| – TBI basedº | 35.6% (112) |
| – None | 1.3% (4) |
| Follow-up time (years, IQR) | 3.2 (1.3 – 6.0) |
±
Chemotherapy include different combination of following agents
Busulfan/Cyclophosphamide/Clofarabine/Fludarabine/Melphalan/Mesna/Thio-TEPA
º
In combination with chemotherapy
ζ
Nine FA (2.9%) patients with malignancy were included in the malignancy group.
*
the following techniques for ex vivo TCD were used: Soybean Agglutination (SBA-E-; n=30), Isolex/E- (n=33) and CliniMACs (n=145; see methods).