1. Outcome and its Predictors in Neonatal Vein of Galen Malformation Great Ormond Street Hospital, London, UK Adam Rennie, Anne Schmitt, Lakshmi Kanagarajah, Francesca Lecce, Claire Toolis, Stefan Brew, Jo Bhattacharya, Fergus Robertson, Vijeya Ganesan

2. Disclosures All medical devices are off license in children
No disclosures

3. Neonatal Vein of Galen 4 neonates but what outcome?
Good, poor, Poor, good
>12 Bectre

4. Neonatal Outcomes Neonates:
But what does this mean for the parents that we meet pre-procedure?
Neonates:
All small series, short F/U av. 2 yrs, heterogeneous
48% good outcome, 22% poor outcome, 27% mortality

5. Bicêtre score
McSweeny et al 2010

6. VGM in the UK Since 2006 all UK cases treated in one of 2 centres
All patients assessed
All offered treatment other than those with clearly irreversible brain injury

7. Not Treated

8. Neonatal Outcomes NIHR funded study
To describe neurocognitive outcomes in an unselected national cohort of children with neonatal VGM
To identify early clinical and radiological predictors of outcome

9. Methods Retrospective amalgamation of clinical data in all patients
Survivors aged >1 year invited to undergo standardised assessments of neurocognitive function
Structured analysis of brain imaging and angiography

10. Study Recruitment 87 neonates 54 survivors 13 uncontactable
35 participated
6 refused
33 non-survivors
(12 not treated)
4 fold

11. Research participants (n = 34) Non-survivors
All survivors
(n = 51)
Research participants
(n = 34)
Non-survivors
(n= 34)
Antenatal diagnosis
23 (45%)
16 (47%)
14 (41%)
Age (days) at post-natal diagnosis
median (range)
2 (1-28)
2.5 (1-27)
2 (1-5)
Gestation
At term
39 (77%)
26 (76%) 30
Preterm
23 (23%)
8 (24%) 4
Presentation
Antenatal
Severe cardiac failure
28 (55%)
18 (53%)
20 (59%)
Bicetre score
Median (range)
18 (11-23)
17 (9-23)
7 (4-21)
No of embolizations
3 (1-6)
3 (1-4)
1 (1-3)
No of treatment complications
20 (39%)
11 (32%)
14/19 (74%)

12. Neonatal Neurocognitive Outcomes
Good
Poor
Neurological outcome
19 (54%)
16 (46%)
Cognitive outcome
15 (43%)
20 (57%)
Language development
Neuromotor development
17 (48.5%)
18 (51.5%)
Adaptive skills (ABAS)
Conceptual skills (ABAS)
Social skills (ABAS)
21 (60%)
14 (40%)
Practical skills (ABAS)
11 (31%)
24 (69%)
Emotional and behavioral development (SDQ)
21/33 (63%)
11/33 (33%)
Executive functions (BRIEF)
8/17 (47%)
9/17 (53%)
Heterogeneous group, however, categorisation into “good” and “poor” tended to hold true across categories for individual patients
Survivors roughly evenly split between these two outcomes
Poor outcomes – high percentage of autistic spectrum

13. Clinical variables vs. outcome
Predictor
Good outcome
(n = 15)
Poor outcome
(n = 20)
P value
Age at follow up
6.1
5.2
0.4
Bicetre 18 13
0.01
Head circumference (centile)
75.2
36.6
< 0.01
Periprocedural haemorrhage/ischaemic event
2 (13%)
9 (45%)
0.04
Antenatal diagnosis
7 (47%)
10 (50%)
0.73

14. MRI variables vs. outcome
Predictor
Good outcome
(n = 15)
Poor outcome
(n = 19)
P value
Normal scan at presentation
13 (87%)
7 (37%)
< 0.01
Cortical injury at presentation
1 (6%)
6 (31%)
1.04
Cortical injury at follow up
3 (20%)
10 (53%)
0.07
White matter injury at presentation
2 (13%)
11 (58%)
0.01
White matter injury at follow up
9 (60%)
18 (95%)
0.03
Ventricular index at follow up
0.31
0.41

15. Angiographic variables vs. outcome
Analysed
Choroidal vs. mural
Feeders including presence of MCA steal
Symmetry of shunt
Sac size/falcine sinus size
Shunt speed
Brain perfusion
The only determinant of significance was whether or not the shunt was closed at follow up
Good outcome 13 (87%), poor outcome 9 (47%) (p 0.02)

16. Physiological predictors Comparison of AN and symptomatic NN patients
Birth weight and MBP significantly lower in AN group
First Bicetre score significantly predicted survival in both AN and NN
Post embolisation lactate predicted outcome
All with Bicetre <8 died or had poor outcome
Bicetre score
<8
8-12
>12
Survived 1 9 12
Did not survive 4 8 2
Good outcome 3
Poor outcome 5 14

17. Conclusions
Early indices of systemic organ failure (Bicetre) useful predictors of survival and outcome
Neonates with normal initial scans are more likely to do well
White matter, rather than grey matter injury, appears more important for prognosis

18. Neonatal Vein of Galen 4 neonates but what outcome?
Pre-treatment discussions:
Neonates with VGM, 1/3rd of children will die, 1/3rd good and 1/3rd poor outcome
Poor outcomes have varying severity of behavioural/cognitive problems
Good, poor, Poor, good
>12 Bectre

19. Acknowledgements F Lecce A Kuczynski F Vargha-Khadem F Robertson
A Rennie
A Schmitt
L Kangarajah
P Lister
M Peters
V Ganesan
C Toolis
S Bhate
F Ahmad
A-M Heuchan (RHSC)