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Table 1 Patient demographics, transplant-related and survival characteristics

From: Long-term outcomes after allogeneic hematopoietic stem cell transplantation for metachromatic leukodystrophy: the largest single-institution cohort report

Cohort ID

Gender

Time from symptom onset to HSCT (y,m)

Age at HSCT (y,m)

Transplant Year

Conditioning regimen

Donor type

HLA matching

% Donor engraftment (y,m)c

% ARSA Activity (y,m)c

Time to follow-up (y,m)c

Cause of death

Late infantile

           

1

M

X

0,4

1996

MA (Bu/Cy)

cRD

6/6

100 (0,4)

—

0,3a

VOD

2

M

X

0,5

2004

MA (Bu/Cy)

UCB

5/6

100 (2,11)

100 (2,10)

4,10

 

3

F

X

0,8

1995

MA (Bu/Cy)

cRD

6/6

100 (8,4)

60 (6,10)

19,6

 

6

F

0,6

2,10

1995

MA (Bu/Cy)

URD

5/6

100 (1,6)

100 (1,0)

1,6a

unknown

Juvenile

           

4

F

1,10

4,10

1984

MA (Bu/Cy)

RD

6/6

100 (15,1)

100 (25,2)

30,6

 

9

F

1,2

4,2b

2001

MA (Cy/TBI)

URD

6/6

100 (4,9)

100 (3,5)

13,3

 

5

F

X

1,3b

1994

MA (Bu/Cy)

URD

6/6

100 (0,3)

—

2,2a

cGvHD

7

F

X

2,8

1994

MA (Bu/Cy)

URD

6/6

100 (0,2)

—

0,3a

VOD

8

F

X

3,5

1994

MA (Bu/Cy)

URD

6/6

97 (6.5)

100 (6,5)

8,2

 

22

F

X

11,1

2004

MA (Bu/Cy)

dUCB

4/6 + 4/6

100 (3,2)

100 (0,6)

10,7

 

10

F

0,5

5,8

1996

MA (Cy/TBI)

URD

6/6

60 (11,3)

38 (11,3)

18,7

 

11

F

0,10

5,10b

1998

MA (Cy/TBI)

cRD

5/6

100 (2,6)

25 (2,6)

7,2a

MLD

12

F

1,1

5,11

2008

RIC

UCB

6/6

2 (0,1)

8 (0,1)

3,1a

MLD

13

F

0,11

6,4

1989

MA (Bu/Cy)

RD

6/6

—

73 (0,1)

0,2a

MSOF

14

F

2,3

6,3

2002

MA (Bu/Cy)

UCB

4/6

100 (2,0)

100 (2,0)

2,0

 

15

F

0,0

6,7

1997

MA (Bu/Cy)

URD

6/6

100 (1,7)

100 (1,7)

7,3

 

16

F

3,6

7,6

1999

MA (Cy/TBI)

URD

5/6

67 (3,5)

59 (1,9)

15,7

 

17

M

2,8

7,8

2003

MA (Bu/Cy)

UCB

5/6

100 (0,6)

100 (0,6)

11,9

 

18

M

0,10

8,4

1997

MA (Bu/Cy)

URD

6/6

100 (0,7)

100 (0,6)

0,7a

Sepsis

19

M

0,9

8,7

2002

MA (Bu/Cy)

UCB

5/6

100 (0,5)

100 (0,5)

10,6a

cGvHD

20

F

2,4

11,1

2005

MA (Bu/Cy)

dUCB

4/6 + 4/6

100 (0,3)

100 (0,3)

0,3a

Sepsis

21

F

2,0

10,4

1995

MA (Bu/Cy)

RD

6/6

100 (0,1)

—

0,2a

aGvHD

23

M

2,7

11,6

1991

MA (Bu/Cy)

URD

6/6

—

50 (0,7)

0,7a

Sepsis

24

F

1,5

14,8

1989

MA (Bu/Cy)

RD

6/6

100 (16,4)

100 (16,4)

16,4

 

25

F

1,7

16,4

2004

MA (Bu/Cy)

dUCB

6/6 + 6/6

100 (0,4)

100 (0,2)

0,5a

TTP

26

F

1,10

16,2

1994

MA (Bu/Cy)

cRD

6/6

100 (9,5)

100 (11,0)

12,6

 

27

F

5,1

16,1

2004

MA (Bu/Cy)

UCB

4/6

100 (3,2)

100 (3,2)

10,7

 

28

M

3,11

16,5

2002

MA (Bu/Cy)

UCB

4/6

100 (0,4)

100 (0,4)

0,8a

Sepsis

30

M

10,5

20,5

2000

MA (Cy/TBI)

URD

5/6

100 (0,2)

100 (0,2)

0,6a

Sepsis

32

F

16,3

27,7

1996

MA (Cy/TBI)

RD

6/6

44 (2,0)

32 (2,0)

18,2

 

43

M

5,8

19,8

2013

RIC

RD

6/6

100 (1,1)

100 (1,1)

1,6

 

Adult

           

42

F

X

20,1

1995

MA (Bu/Cy)

URD

5/6

100 (0,1)

—

0,1a

VOD

31

M

9,1

27,1

2003

MA (Bu/Cy)

UCB

4/6

100 (0,2)

—

0,3a

Graft Failure

33

F

10,10

29,10

1999

MA (Cy/TBI)

URD

6/6

100 (3,0)

100 (3,0)

10,0

 

34

M

3,7

33,7

2000

MA (Cy/TBI)

URD

5/6

100 (1,5)

64 (0,1)

10,0

 

36

F

5,4

38,1

1999

MA (Cy/TBI)

RD

6/6

35 (2,3)

76 (1,0)

1,1

 

38

F

0,11

40,7

2001

MA (Cy/TBI)

UCB

5/6

100 (0,3)

100 (0,3)

0,4a

Sepsis

39

M

5,5

42,5

2007

RIC

dUCB

5/6 + 5/6

100 (0,6)

100 (1,3)

7,6

 

40

F

3,5

42,6

2007

RIC

dUCB

5/6 + 6/6

80 (2,9)

100 (1,2)

6,5a

unknown

41

F

3,2

44,2

2007

RIC

dUCB

4/6 + 5/6

0 (1,1)

50 (0,2)

1,1

 
  1. y Year, m Month, X Asymptomatic, MA Myeloablative, Bu Busulfan, Cy Cyclophosphamide, TBI Total-body irradiation, RIC Reduced-intensity conditioning, RD Related marrow donor (sibling), URD Unrelated marrow donor, UCB Umbilical cord blood, d Double cord, c MLD-carrier marrow donor, a Deceased, VOD Hepatic veno-occlusive disease, MSOF Multi-system organ failure, aGVHD Acute graft-vs-host disease, cGvHD Chronic graft-vs-host disease, b Age denoted is time of first transplant, c Most recent time point available, expressed relative to date of HSCT. Patients 29, 35 and 37 were removed due to lack of confirmatory MLD diagnostic data on retrospective review