Table 1.
Baseline patient and graft characteristics
| UPIN | Diagnosis | Age (yrs) | Sex | Minority (Y/N) | Pre-transplant Co-morbidities | Wt (kg) on day 0 | HLA Match 6 allele A, B, DRB1 | HLA Match 8 allele Hi Res A, B, C, DRB1 | CMV Pt | TNCC (×10e7/kg reinfused | CD34+ (×10e5/kg reinfused) |
|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | Hunter | 2.03 | M | Y | Cardiomyopathy, obstructive sleep apnea | 25.3 | 4/6 | 6/8 | Neg | 7.91 | 3.70 |
| 2 | Sanfilippo B Syndrome | 3.35 | M | N | 15.1 | 6/6 | 8/8 | Neg | 5.18 | 3.78 | |
| 3 | Sanfilippo B Syndrome | 3.32 | F | N | 15.8 | 5/6 | 6/8 | Neg | 4.14 | 0.87 | |
| 4 | Cartilage Hair Hypoplasia | 1.30 | M | N | CMV viremia, transfusion dependent anemia, chronic neutropenia | 8.2 | 6/6 | 7/8 | Pos | 13.90 | 4.31 |
| 5 | MLD | 6.76 | M | N | 20.4 | 5/6 | 6/8 | Neg | 4.58 | 2.43 | |
| 6 | Zap 70 deficiency | 3.32 | F | Y | Resp: MAI, Pseudomonas, Parainfluenza 1, Candida, bronchiectasis; Enteroviral meningitis, hydrocephalus, VP shunt | 11.2 | 5/6 | 5/8 | Neg | 6.60 | 2.6 |
| 7 | HLH | 0.45 | F | N | 7.9 | 5/6 | 5/8 | Neg | 15.3 | 3.67 | |
| 8 | HLH | 0.58 | F | Y | Adenoviremia, adenovirus in stool and urine | 6.20 | 4/6 | 4/8 | Neg | 11.80 | 2.48 |
| 9 | β thalassemia major | 1.56 | M | Y | 9.5 | 5/6 | 6/8 | Pos | 8.17 | 5.10 | |
| 10 | CID | 1.14 | F | N | ITP, HHV-6 pneumonitis, Enterovirus encephalitis, colitis | 10.1 | 5/6 | 6/8 | Neg | 7.80 | 2.73 |
| 11 | Krabbe | 3.09 | F | Y | Blindness | 12.2 | 4/6 | 5/8 | Neg | 14.30 | 4.58 |
| 12 | Krabbe | 3.25 | F | N | Ataxia | 19.1 | 4/6 | 4/8 | Neg | 7.36 | 1.55 |
| 13 | DBA | 2.56 | M | N | Iron overload | 15.9 | 5/6 | 7/8 | Neg | 6.22 | 1.50 |
| 14 | Hurler | 1.71 | F | Y | Obstructive sleep apnea. Stool adenovirus +, h/o Parainfluenza 3 infection | 11.8 | 4/6 | 4/8 | Neg | 10.8 | 4.86 |
| 15 | ALD | 8.02 | M | Y | 26.9 | 4/6 | 3/8 | Neg | 6.99 | 3.7 | |
| 16 | βThalassemia major | 3.65 | M | Y | 14.2 | 4/6 | 5/8 | Neg | 7.07 | 1.63 | |
| 17 | β thalassemia major | 5.44 | F | Y | Prior myeloablative transplant; h/o severe hemorrhagic cystitis | 18 | 5/6 | 6/8 | Pos | 11 | 3.5 |
| 18 | Omenn syndrome | 0.45 | M | Y | 5.26 | 5/6 | 6/8 | Pos | 22 | 7.72 | |
| 19 | βThalassemia major | 2.58 | M | Y | 13.2 | 6/6 | 6/8 | Neg | 5.04 | 4.58 | |
| 20 | CD40L deficiency | 3.38 | M | N | 14.1 | 5/6 | 7/8 | Neg | 9.46 | 3 | |
| 21 | Omenn syndrome | 0.31 | M | N | Erythroderma, FTT, CMV | 6 | 6/6 | 8/8 | Pos | 15.8 | 2.4 |
| 22 | PNP deficiency | 4.27 | M | N | FTT, food intolerance, chronic enteritis, CMV urine, adenovirus in stool | 16.6 | 5/6 | 5/8 | Pos | 5.58 | 1.3 |
ALD = adrenoleukodystrophy; CID = combined immunodeficiency; CMV = cytomegalovirus; DBA = Diamond Blackfan anemia; F = female; FTT = failure to thrive; HHV-6 = human herpes virus 6; Hi res = high resolution; HLH = Hemophagocytic lymphohistiocytosis; h/o = history of; ITP = immune thrombocytopenic purpura; kg = kilograms; M = male; MAI = Mycobacterium avium; MLD = metachromatic leukodystrophy; Neg = negative; PNP = purine nucleoside phosphorylase; Pos = positive; Pt = patient; UPIN = patient number; VP = ventriculoperitoneal; Wt = weight; Y/N = Yes/No; yrs = years; ZAP 70 =zeta chain associated protein kinase 70;
Inherited metabolic disorders (IMD) = Hunter, Hurler, Krabbe, Sanfilippo B, Metachromatic leukodystrophy (MLD), adrenoleukodystrophy (ALD);
Primary immunodeficiency diseases (PID) = Cartilage Hair Hypoplasia, Zap 70 deficiency, HLH, CID, Omenn syndrome, CD40Ligand deficiency, PNP deficiency)