Table 2 Description of previous treatments, clinically significant mutations and outcomes of patients receiving therapeutic intervention during the study

From: Feasibility of functional precision medicine for guiding treatment of relapsed or refractory pediatric cancers

Patient ID

General diagnosis

Sample type

Sex

Age (years)

Previous therapy lines

Previous treatment

Previous objective response

Previous PFS (weeks)

Clinical mutations

FPM-guided

Additional intervention

Study treatment

Current objective response

Current PFS (weeks)

EV013

AML

Peripheral blood

M

5

3

Cyclophosphamide + busulfan + sorafenib

Complete response

60

FLT3-ITD, KDM6A

Yes

Allogeneic bone marrow transplant

Fludarabine + cytarabine, midostaurin

Complete response*

112

EV009

OS

Excised tumor

F

9

3

High-dose ifosfamide + larotrectinib

Stable disease

17

CDKN2A/B, TP53, ATRX, ZFHX4

Yes

Resection

Idarubicin + montelukast

Complete response

84

EV010

EWS

Excised tumor

M

21

5

Irinotecan + temozolomide + vigil vaccine

Complete response

58

CDKN2A/B, EWSR1-FLI1, ESR2, AD21, TOP2A

Yes

None

Topotecan + cyclophosphamide + vincristine

Complete response

61

EV002

AML

Peripheral blood

M

16

2

Cytarabine

Progressive disease

N/A

CEBPA, ASLX2, SETD2

Yes

None

Cytarabine + idarubicin + dexamethasone

Complete response

48

EV004

RMS

Excised tumor

F

7

3

Pazopanib + nivolumab

Progressive disease

2

CDKN2A/B, GNAC, NRAS

Yes

None

Vincristine, temozolomide + irinotecan

Partial response

24

EV008

OS

Excised tumor

F

17

2

Ifosfamide + mifamurtide

Progressive disease

N/A

CDKN2A/B, TERT, CCDN3

Yes

Resection

Sirolimus

Stable disease

15

EV011

RMS

Excised tumor

M

4

3

Irinotecan + cyclophosphamide

Progressive disease

N/A

BCOR, PAX3-FOXO1, SMARCA4

No

None

Cyclophosphamide + vinorelbine + temsirolimus

Partial response

28

EV021

NB

Excised tumor

F

4

2

Temozolomide + irinotecan + dinutuximab

Stable disease

45

None

No

None

Cyclophosphamide + topotecan, temozolomide + irinotecan + dinutuximab

Stable disease

18

EV005

OS

Biopsy of metastatic nodule

F

7

2

Ifosfamide

Complete response

7

CDKN2A/B, MYC, TP53, 9p24.1, NOTCH3, NF1

No

None

Sorafenib + everolimus

Progressive disease

N/A

EV007

GBM

Excised tumor

M

11

2

Bevacizumab + lomustine

Progressive disease

N/A

CREBBP2, TP53, PTEN

No

Radiation

Everolimus + fenofibrate + metformin + celecoxib + etoposide + cyclophosphamide

Progressive disease

N/A

EV019

MB

Excised tumor

F

12

2

Cisplatin + vincristine + cyclophosphamide

Progressive disease

N/A

TP53, GLI2, MYCN, ATM

No

None

Temozolomide + irinotecan + temsirolimus

Progressive disease

N/A

EV022

AML

Bone marrow aspirate

M

1.94

5

Clofarabine + cytarabine

Progressive disease

N/A

N/A

No

None

Azacitidine + nivolumab

Progressive disease

N/A

EV023

ALL

Peripheral blood

M

0.97

2

Vincristine + daunorubicin + cytarabine

Progressive disease

N/A

NRAS, KMT2A-EPS15, EPHA3, PALB2

No

None

Blinatumomab + methotrexate

Progressive disease

N/A

EV025

RMS

Excised tumor

F

8

2

Vincristine

Progressive disease

N/A

PAX3-FOXO1

No

None

Doxorubicin + ifosfamide + mesna

Progressive disease

N/A

  1. ALL, acute lymphoblastic leukemia; AML, acute myeloid leukemia; EWS, Ewing sarcoma; GBM, glioblastoma multiforme; MB, medulloblastoma; NB, neuroblastoma; OS, osteosarcoma; RMS, rhabdomyosarcoma. *indicates the patient received a bone marrow transplant.