Table 3.
Reference | Conditioning Regimen |
Disease/ n |
HSC source/n |
Median Age (years) |
Incidence of TRM |
Incidence of Grades II-IV GVHD |
Graft Failure |
Probability of OS/EFS |
Outcomes and Conclusions |
---|---|---|---|---|---|---|---|---|---|
Del Toro et al (2004) | Flu based Plus: Bu, Cy, melph, or VP- 16 Plus: ATG or C1-H |
HL/6 NHL/1 NBL/2 Wilms/1 CML/1 AML/1 MDS/2 WAS/1 Thal/2 SAA/2 HLH/1 FA/1 |
UCB/14 (100% mm) RBM/2 RPBSC/4 RBM+PBSC/ 1 |
13 | 10% (n=2) (100 days) |
Acute: 38% Chronic: 0% (median f/u 149 days) |
n=5 (24%) (UCB: 3 early GF, 1 late GF) |
OS: 73% (1 -year) Good risk: 89% High risk: 40% |
• RIC in kids is feasible (≤ 25% GF) and results in = ≥85% of recipients initially achieving >50% donor chimerism. |
Jacobsohn et al (2004) | Flu/Bu based +/−ATG |
SCD/3 Thal/1 MPS/3 Imm/6 |
UCB/2 PBSC/6 RPBSC/5 |
5.2 | 15% (n=2) (100 days) |
Acute: 8% (n=1) Chronic: 37% (n=3) |
Primary: n=2 Late: n=1 |
OS: 84% (1 -year) |
• RIC provides a good alternative to MA transplant for children with non-malignant disorders, except for hemoglobinopathi es where engraftment is poor. |
Majhail et al (2006a) | Bu/Flu/TBI(20 0) or Cy/Flu/TBI(20 0) |
HL/21 | UCB/9 (100% mm) MSD/12 (1 mm) |
28 42 |
n=1 (100 days) n=2 (100 days) |
*Acute: 33% Chronic: 11% *Acute: 33% Chronic: 33% |
n=0 n=0 |
OS: 51% EFS: 20% (2-year) OS: 49% EFS: 25% (2-year) |
• Comparable outcomes with UCB and MSD after RIC. |
Majhail et al (2008) | Flu/Cy/TBI(20 0) Flu/Bu/TBI (n=16) Flu/Clad/TBI (n=5) |
AML/29 ALL/4 CML/3 CLL/5 MDS/16 NHL/21 HL/2 MM/6 Other/2 |
UCB/5 dUCB/38 (93% mm) RPBSC/44 RBM/1 RBM+PBSC/ 2 |
59 58 |
28% 23% (180 days) |
Acute: 49% Chronic: 17% Acute: 42% Chronic: 40% |
89% 100% (Sustain ed donor engraft ment at 42 days) |
OS: 34% EFS: 34% OS: 43% EFS: 30% (3-year) |
• Survival and TRM following RIC UCB and MSD transplant are comparable in adults over 55 years. • Graft type had no impact on TRM or survival. |
Grades III-IV acute GVHD
HSC(T) = haematopoietic stem cell (transplantation), TRM = treatment-related mortality, GVHD = graft-versus-host disease LFS = leukemia-free survival, OS = overall survival, Flu = fludarabine, Bu = busulfan, Cy = cytoxan, melph = melphalan, VP-16 = etoposide, ATG = antithymocyte globulin, C1-H = campath, TBI = total body irradiation, Clad = cladribine, HL = Hodgkin lymphoma, NHL = Non-Hodgkin lymphoma, NBL = neuroblastoma, Wilms = Wilms tumour. CML = chronic myeloid leukaemia,AML = acute myeloid leukaemia, MDS = myelodysplastic syndrome, WAS = Wiskott-Aldrich syndrome, Thal = thalassaemia, SAA = severe aplastic anemia, HLH = haemophagocytic lymphohistiocytosis, FA = Fanconi anaemia,SCD = sickle cell disease, Imm = immune deficiency, MPS = mucopolysaccharidoses, ALL = acute lymphoblastic leukaemia, CLL = chronic lymphocytic leukaemia, MM = multiple myeloma, UCB = unrelated umbilical cord blood, RBM = related bone marrow, (R)PBSC = (related) peripheral blood stem cells, MSD = matched sibling donor, dUCB = double unrelated umbilical cord blood, mm = mismatch, f/u = follow-up, GF = graft failure, RIC = reduced intensity conditioning, MA = myeloablative.