Table 1 Characteristics of patients and donors undergoing T-depleted haplo-HSCT and MSD HSCT.

From: αß T-cell depleted haploidentical stem cell transplantation for pediatric and young adult patients with transfusion-dependent thalassemia

Pat.

Age (years)

Sex

ß-Thal subtype

Thal-associated complications/ secondary diagnosis

MRI Iron (µmol/g)

Pesaro class

Ishak

Donor age (years)

Donor sex

Donor relation

HLA match

T-cell depletion

BG compatibility

Haplo-HSCT

1b

23

F

β0/β0

ED, CD, HSM, S

158

II

n.a.

43

F

Mother

5/10

CD3/CD19

Compatible

2aa

10

M

β0/β0

CD

35

I

n.a.

50

M

Father

5/10

CD3/CD19

Major incomp.

2ba

11

M

β0/β0

CD

35

I

n.a.

39

F

Mother

5/10

CD3/CD19

Minor incomp.

3b

8

M

Β+/β+

–

99

II

n.a.

28

F

Mother

6/10

TCRαβ/CD19

Compatible

4b

3

M

β0/β+

CD, GF

114

II

0

40

M

Father

5/10

TCRαβ/CD19

Major incomp.

5

21

M

β0/β0

ED, H, S, IT

n.a.

II

5

41

F

Mother

5/10

TCRαβ/CD19

Compatible

6

6

F

β0/β0

N

n.a.

I

n.a.

41

F

Mother

5/10

TCRαβ/CD19

Major incomp.

7

2

F

β0/β+

–

n.a.

I

n.a.

36

M

Father

6/10

TCRαβ/CD19

Compatible

8

11

F

β0/β0

ED, HSM

n.a.

I

n.a.

37

M

Father

7/10

TCRαβ/CD19

Major incomp.

9b

6

M

β0/β0

ED, GF, H

268

II

1

37

F

Mother

5/10

TCRαβ/CD19

Minor incomp.

10

11

M

β0/β0

ED

13

II

n.a.

38

M

Father

7/10

TCRαβ/CD19

Compatible

11

11

M

β0/β0

Allo-I, HSM

179

II

n.a.

35

F

Mother

7/10

TCRαβ/CD19

Minor incomp.

12b

8

M

β0/β0

–

64

II

n.a.

36

F

Mother

6/10

TCRαβ/CD19

Compatible

13

13

M

β0/β0

S

84

II

n.a.

36

F

Mother

5/10

TCRαβ/CD19

Minor incomp.

MSD-HSCT

1

4

M

β+/β°

HSM

n.a.

I

n.a.

15

M

Brother

10/10

–

Minor incomp.

2b

11

M

β°/β°

ED

n.a.

III

n.a.

21

F

Sister

10/10

–

Compatible

3

7

M

β+/β°

–

n.a.

I

n.a.

12

M

Brother

10/10

–

Compatible

4b

10

F

β+/β°

ED, H, HSM

429

III

2

16

M

Brother

10/10

–

Minor incomp.

5

12

M

β°/β°

ED, IT, N

240

II

0

13

M

Brother

10/10

–

Compatible

6b

12

F

β+/β°

H, HSM, N

571

III

2

25

M

Brother

10/10

–

Compatible

7b

10

F

β°/β°

ED, HSM

140

II

n.a.

14

M

Brother

10/10

–

Compatible

  1. Allo-I allo-immunization, β° ß+ ß-thalassemia genotype variants, CD cardiac dysfunction (including cardiomegaly and impaired cardiac pump function, pulmonary stenosis), ED endocrine dysfunction (including diabetes mellitus type 1, hypoparathyroidism, hypothyroidism, adrenal insufficiency, growth retardation, hypogonadism, pubertas tarda), F female, GF graft failure after 1st SCT, H hepatopathy (including liver fibrosis/ cirrhosis and/or functional impairment), HLA human leucocyte antigens, HSM hepato- and/or splenomegaly, IT immune thrombocytopenia, M male, N nephropathy, n.a. non applicable, S splenectomy.
  2. aSame patient
  3. bPatients received intensified iron chelation therapy with deferasirox (Exjade®) and deferiprone (Ferriprox®) (*1) or deferoxamine (Desferal®) (*2); *3documented incompliance. Shadowed patients undergoing 2nd SCT.
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