Table 5.5.
Mismatch related marrow or matched unrelated donor transplantation for patients with thalassemia major
Refs. | Transplant regimen | Graft type | Pesaro Class | No. of patients (age) | Alive without TM | Acute GvHD (Gr 2–4) | Chronic GvHD (extensive) | Death |
---|---|---|---|---|---|---|---|---|
Sullivan et al. [63] | Bu, Cy, MTX, CSA | Unrelated | Class 2 and 3 | 4(1–16) | 2 | NR | NR | 1 |
La Nasa et al. [68, 69] | Bu 14 mg/kg, Cy 200 mg/kg, TT 10 mg/kg, ATG, CSA, MTX, | Unrelated BM, 10 of 10 match 85% | Class 1, 14 | 68(2–37) | Class 1+2: 24(80%) | 24(35%, gr 2–4) | 5 | 16(most were in class 3) |
Class 2, 16 | Class 3: 21(55%) | |||||||
Class 3, 38 | ||||||||
Feng et al. [93] | Bu 16 mg/kg, Cy 200 mg/kg, ALG | Unrelated, BM, includes 1 and 2 alleles mismatch | Class 1,3 | 9(1–9) | 7(78%) | 6 | 1 | 1 |
Class 2, 4 | ||||||||
Class 3, 2 | ||||||||
Resnick et al. [94] | Bu 16 mg/kg, Flu 180 mg/kg, ATG | Unrelated, 1 BM, 1 PBSC | Both Class 1 | 2(2 and 4) | 1 | NR | NR | NR |
Li et al. [70] | Bu 12–13.2 mg/kg, Cy 100–120 mg/kg, TT 5 mg/kg, Flu 160 mg/m2, ATG, MTX, CSA, MMF | Unrelated, PBSC 8/8: 32, 7/8: 19, 6/8: 1 | Class 1,9 | 52(2–15) | 50(96%) | 4(8%, grade 3–4) | 0 | 4, 2 directly from GvHD |
Class 2, 82 | ||||||||
Class 3,9 | ||||||||
Total | Backbone of Bu, Cy, ATG | 135 | 105(78%) | 26%(of total) | 5%(of total) | 17%(of total) | ||
33%(of engrafted) | 6%(of engrafted) |
Abbreviations: SCD sickle cell disease, GvHD graft-versus-host disease, Bu busulfan, Cy cyclophosphamide, ATG anti-thymocyte globulin, CSA cyclosporine A, MTX methotrexate, Flu fiudarabine, Mel melphalan, MMF mycophenolate mofetil, ALG TT thiotepa, Gr grade, NR not reported, Gy gray