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. Author manuscript; available in PMC: 2019 Jan 22.
Published in final edited form as: Adv Exp Med Biol. 2017;1013:123–153. doi: 10.1007/978-1-4939-7299-9_5

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