Table 1. Modifications made to transplant regimens to improve outcomes.

Ref	Median follow-up	Recipient / transplant characteristics	Modification in preparative regimen	OS (%)	DFS (%)	Graft rejection (%)
22	36 months	HLA-matched related donors Risk class 3 Age < 17 years	Transfusions chelation (desferroxamine) hydroxyurea azathioprine + busulfan / cyclophosphamide	93	85	8 (Eventual risk 15%)
7	39 months	Unrelated donors Recipient risk class 1-3	Busulfan + fludarabine / cyclophosphamide Thiotepa added for GR	79 96 (C 1–2) 65 (C 3)	65 80 (C 1–2) 55 (C 3)	20 (C 1–2) 11 (C 3)
13	64 months	Related donors Class 3	Low dose busulfan, fludarabine, TLI, and ATG	100	77	23
12	41 months	HLA-matched related donors Risk class 3 Age < 17 years	Transfusions, chelation (desferoxamine) hydroxyurea azathioprine + busulfan /fludarabine/ cyclophosphamide/thiotepa	92	92	0
14	39 months	High risk<br≫10 years Hepatomegaly Unrelated donors	A subset received RIC: fludarabine / dex × 2; then busulfan/fludarabine/ATG	94	82	0
23	36 months	Related and unrelated donors	Treosulfan, thiotepa and fludarabine	93	82	9
18	24 months	Unrelated donor group PBSC transplants	Busulfan, cyclophosphamide, fludarabine	92	90	1.9
19	36 months	Unrelated donor cord	Busulfan, cyclophosphamide, antilymphocyte globulin	88	73	17
21	41 months	Unrelated donor cord or marrow	HU, alemtuzumab, fludarabine, melphalan, thiotepa	87	83	1.9

Risk factors: Inadequate iron chelation, hepatomegaly > 2cm, portal fibrosis on liver biopsy.

Risk class 1: No risk factors; risk class 2: one or two factors; risk class 3: all 3 risk factors present.

GR, graft rejection; C, risk class; HU, hydroxyurea; RIC, reduced-intensity conditioning; Dex, dexamethasone; Bu, busulfan; Flu, fludarabine; TLI, total lymphoid irradiation; ATG, antithymocyte globulin; Dex, dexamethasone; PBSC, peripheral blood stem cell; OS, overall survival; DFS, disease-free survival; FU, follow-up