Table 1.
Clinical features of study subjects
| Characteristics | Number | % |
|---|---|---|
| Number of patients (male/female) | 55 (42/13) | |
| Age at transplant (mo), median (range) | 5 (1-111) | |
| Diagnosis | ||
| Primary immune deficiency | 45 | 82% |
| SCID | 37 | 67% |
| Pre-NBS | 11 | 30% |
| IL2RG | 10 | 28% |
| RAG1,2 | 7 | 19% |
| IL7R | 5 | 14% |
| CD3D | 2 | 6% |
| ADA/PNP | 4/1 | 14% |
| Zap70 | 1 | 3% |
| Jak3 | 1 | 3% |
| RAC | 1 | 3% |
| other | 5 | 10% |
| IPEX syndrome | 2 | 4% |
| Wiskott-Aldrich syndrome | 2 | 4% |
| Leukocyte adhesion defect 1 | 2 | 4% |
| Chronic granulomatous disease | 2 | 4% |
| Metabolic disorders | 5 | 9% |
| Krabbes | 2 | |
| Alpha-mannosidosis | 1 | |
| Adrenoleukodystrophy | 1 | |
| Hurler | 1 | |
| HLH | 1 | 2% |
| Hematological diseases | 4 | 7% |
| Diamond-Blackfan Anemia | 2 | |
| Bone marrow failure | 2 | |
| Degree of HLA matching | ||
| 5/6 | 35 | 64% |
| 6/6 | 20 | 36% |
| Total nucleated cell dose median | 13×107/kg (5.1-27) | |
| Number of patients with pretransplant infections (active at time of transplant) | 46 (16) | |
| Viral | 26 (13)∗ | |
| CMV | 8 (5) | |
| RSV | 6 (3) | |
| Paraflu3 | 4 (3) | |
| VZV | 1 (2) | |
| Adv. | 1 (1) | |
| Rhino | 5 | |
| Norovirus/sapovirus | 3 (1) | |
| PJP | 5 | |
| Bacterial | 9 | |
| Mycobacterial | 1(1) | |
| Fungal | 5 (1) | |
| Number of patients with pretransplant pneumonia | 22 | 40% |
| Number of patients with pretransplant used of assisted mechanical ventilation | 10 | 18% |
Adv, Adenovirus.
∗
One patient had 2 viruses.