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. Author manuscript; available in PMC: 2010 Oct 1.
Published in final edited form as: Br J Haematol. 2009 Oct;147(2):246–261. doi: 10.1111/j.1365-2141.2009.07828.x

Table 3.

UCB and RIC Transplant

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.