Table 1 Detailed characteristics of xenografted MDS patients.

From: Preclinical evaluation of eltrombopag in a PDX model of myelodysplastic syndromes

Patient

Age (years)

Sex

WHO 2016

IPSS-R

Karyotype

Mutations

Blasts in BM (%)

PLT number (×10E9/L)

Prior treatment

P01

59

M

MDS-EB-2

Very high

Complex aberrant

CBL, DNMT3A, EZH2

12

29

AZA, PT, RCT

P02

82

M

MDS-EB-2

Very high

Complex aberrant

TET2, TP53

13

7

AZA, PT, RCT

P03

65

M

tMDS-EB-1

Very high

Complex aberrant

BCOR, KRAS

5

15

AZA, PT, RCT

P04

71

M

tMDS-MLD

Low

46,XY,r(6) (p21q26) [14]/46,XY [6]

ASXL1, RUNX1, SRSF2, TET2

1

24

RCT

P05

69

M

MDS-EB-1

Very high

Complex aberrant

TP53

7

15

AZA, PT, RCT

P06

82

M

MDS-EB-2

Very high

47,XY,+8 [4]/46,XY [21]

ASXL1, RUNX1, STAG2, ZRSR2

10

15

None

P07

83

F

MDS-MLD

int

46,XX

ASXL1, BCOR, CBL, DNMT3A, EZH2, TET2, WT1

4

24

G-CSF

P08

80

M

MDS-U

int

47,XY,+8 [3]/46,XY [17]

ASXL1, IDH2, SRSF2, STAG2

4

80

ESA

P09

77

M

MDS with isolated del(5q)

Very low

Del5q

ASXL1, ETV6, EZH2

1

74

Deferoxamine, Lenalidomide, PT, RCT

  1. AZA 5-Azacitidine, BM bone marrow, EB with excess blasts, ESA erythropoiesis-stimulating agent, G-CSF granulocyte colony-stimulating factor, int intermediate, IPSS-R International Prognostic Scoring System–revised, MLD multilineage dysplasia, PLT platelet, PT platelet transfusion, RCT red cell transfusion, U unclassifiable.