1

Eneida Mioshi, PhD Department of Psychiatry University of Cambridge School of Clinical Medicine

Knopman et al, Brain, 2008

Disease stage?

Outcome

measures?

Power?

Longitudinal

changes?

2

FRS – Frontotemporal Dementia

Rating Scale

(Also known as FTD-FRS)

3

Very mild

Mild

Moderate

Severe

Very severe

Profound

3.35

1.68

-0.59

-3.09

-6.66

Mioshi et al, Neurology, 2010 4

FRS

Mioshi et al, Neurology, 2010

Disease progression

differs across

FTD variants

5

FRS

CDR can

underestimate disease

severity in FTD

Mioshi et al, Neurology, 2010 6

*

Baseline Follow up-2

-1

0

1

2

Sem Dem

PNFA

bv-FTDLogi

t sco

re d

eclin

e

Mioshi et al, Neurology, 2010 7

Significant decline for all variants

FRS

• 130 patient-carer dyads

• Patients Demographics • 84 males • 63.8±9.1 years • 4.7 years disease duration •34 (26%) bulbar; 96 (74%) limb

RESULTS • Stages:

• Mild (30.8%) • Moderate (63.1%) • Severe (6.2%)

Hsieh et al, ALSFTD, 2013 8

FRS

De Silva et al (in prep)

ALSFRS-R

ALSFRS-R

FTDFRS

FTDFRS

9

FRS

FTLD

(n=252) AD (n=243)

Early susceptibility to

decline in frontal-

dependent abilities

Early susceptibility

to decline in

memory

Different profiles on CDR-FTLD for AD and FTLD

Mioshi & Knopman (in prep)

Late decline in

language and

behaviour

10

Lima-Silva et al, Dementia&Neuropsychologia, 2013

CDR 1 = Mild

11

R = - 0.636

p < 0.001

Mioshi et al, ADAD, 2012

Global improvements

Naming

Activity engagement

Family carer burden

Family carer appraisal of

problems

12

FRS

Mixed ANOVA

Time: F(1,20)

= 5.851, p< 0.05

Group: F(1,20)

= 40.380, p< 0.001

Behaviour: n.s.

Time*group: F(1,20)

= 5.631, p< 0.05

Mioshi et al (in prep)

How much did it bother you?

13

DISINHIBITION

APATHY

FRS: disease staging; longitudinal changes; FTDMND

GENFI: validation in

other European

countries/languages

TRx-237-007; FRM-0334 Reviewing scoring system

Other considerations for

non-pharmacological

trials 14

Australia John Hodges Olivier Piguet Matthew Kiernan Sharpley Hsieh Jashelle Caga Colleen McKinnon Claire O’Connor Cassandra Kaizik James Burrell Emma Devenney

USA David Knopman Brazil Thais Lima-Silva Monica Yassuda Paulo Caramelli Ricardo Nitrini

UK

Michael Hornberger James Rowe Kate Dawson Rachel Winson Julieta de la Llosa Camilla Zugman

All patients and their family carers

All FRS Collaborators

15

em341@medschl.cam.ac.uk

15