Table 4

Related and unrelated cord blood donors for SCD

ReferenceTransplant regimenMedian TNC dose, × 107/kg (range)HLA matchNumber of patients (age range)Alive without SCDAcute GVHDChronic GVHDDeath (cause)
Related cord blood donors         
    Brichard et al, 199664  Bu 16 mg/kg, Cy 200 mg/kg, ATG, CSA 4.6 6/6 1 (5) N/A 
    Miniero et al, 199865  Bu 16 mg/kg, Cy 200 mg/kg, CSA ± MTX (3.5-6.0) 6/6 3 (3-11) 2 (Gr 1) N/A 
    Gore et al, 200066  Bu 726 mg/m2, Cy 200 mg/kg, ATG, CSA 2.3 6/6 1 (9) N/A 
    Locatelli et al, 200367  Mostly Bu, Cy, ATG/ALG, CSA ± MTX 4.0 (1.2-10)* 6/6 41 pts, 5/6 3 pts* 11 (1-20)* 10 11% (Gr 2)* 6% (limited)* N/A 
    Walters et al, 200568  NR NR 6/6 42 pts, 4/6 4 pts 8 (NR) NR NR 1 (intractable seizures) 
Unrelated cord blood donors         
    Adamkiewicz et al, 200769  Mixed, 4 pts myeloablative, 3 pts reduced intensity (1.5-9.3) 5/6 2 pts, 4/6 5 pts 7 (3.4-16.8) 1 (Gr 1); 2 (Gr 2); 1 (Gr 3); 1 (Gr 4) 1 (extensive) 1 (multiorgan failure) 
    Mazur et al, 200670  Rituximab, alemtuzumab, thiotepa (600 mg/m2), 600 cGy TBI, tac + MMF 4.2 4/6 1 (9) Gr 1 N/A 
    Sauter et al, 201071  Reduced intensity 3.8 and 2.0§ 5/6 1 (22) N/A 
ReferenceTransplant regimenMedian TNC dose, × 107/kg (range)HLA matchNumber of patients (age range)Alive without SCDAcute GVHDChronic GVHDDeath (cause)
Related cord blood donors         
    Brichard et al, 199664  Bu 16 mg/kg, Cy 200 mg/kg, ATG, CSA 4.6 6/6 1 (5) N/A 
    Miniero et al, 199865  Bu 16 mg/kg, Cy 200 mg/kg, CSA ± MTX (3.5-6.0) 6/6 3 (3-11) 2 (Gr 1) N/A 
    Gore et al, 200066  Bu 726 mg/m2, Cy 200 mg/kg, ATG, CSA 2.3 6/6 1 (9) N/A 
    Locatelli et al, 200367  Mostly Bu, Cy, ATG/ALG, CSA ± MTX 4.0 (1.2-10)* 6/6 41 pts, 5/6 3 pts* 11 (1-20)* 10 11% (Gr 2)* 6% (limited)* N/A 
    Walters et al, 200568  NR NR 6/6 42 pts, 4/6 4 pts 8 (NR) NR NR 1 (intractable seizures) 
Unrelated cord blood donors         
    Adamkiewicz et al, 200769  Mixed, 4 pts myeloablative, 3 pts reduced intensity (1.5-9.3) 5/6 2 pts, 4/6 5 pts 7 (3.4-16.8) 1 (Gr 1); 2 (Gr 2); 1 (Gr 3); 1 (Gr 4) 1 (extensive) 1 (multiorgan failure) 
    Mazur et al, 200670  Rituximab, alemtuzumab, thiotepa (600 mg/m2), 600 cGy TBI, tac + MMF 4.2 4/6 1 (9) Gr 1 N/A 
    Sauter et al, 201071  Reduced intensity 3.8 and 2.0§ 5/6 1 (22) N/A 

ALG indicates antilymphocyte globulin; ATG, antithymocyte globulin; Bu, busulfan; cGy, centi-Gray; CSA, cyclosporine A; Cy, cyclophosphamide; Gr, grade; MMF, mycophenolate mofetil; MTX, methotrexate; N/A, not applicable; pts, patients; NR, not reported; SCD, sickle cell disease; tac, tacrolimus; TBI, total body irradiation; and TNC, total nucleated cell.

*

Includes 44 patients in the entire cohort: 33 patients with thalassemia and 11 patients with SCD.

Includes 47 patients in the entire cohort: 14 patients with thalassemia, 8 patients with SCD, and 25 other.

This represents a second unrelated umbilical cord blood transplantation.

§

Patient received a double umbilical cord unit transplantation.

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