For Rare Disease Day 2023, SJIA Foundation organized a Social hour for SJIA and Still's Disease patients

1. In Celebration of Rare Disease Day
SJIA & Still’s Social Hour

2. Housekeeping: How to use Zoom during event
During Talks / Patient Stories
● All attendees will be muted
● You can use Chat to chat with other
attendees and ask questions of Speakers.
○ Turn off “Fullscreen Mode” (under View) so that
Chat stays in a separate window to the right (does
not pop-up over the meeting window).

3. Renaming yourself in Zoom:
Click in the 3 dots, in your window, then in rename and change your name.
Click rename.

4. Housekeeping: Asking for Help
● For any issues with the Zoom Conference, please chat with
Gabriel_Help for assistance.
● For questions about the program, post in chat.

5. Conference Videos
● Most sessions will be recorded and available post-conference on our YouTube
channel: https://www.youtube.com/sjiafoundation
● Subscribe to channel to be notified.
○ Those on our mailing list / registered for this conference will also receive a link to videos via
email.
● Founded in 2014 by Jon Boutelle and
Rashmi Sinha, parents of a child
diagnosed with SJIA, MAS and Lung
Disease
● Goal to find new treatments and
ultimately a cure
● Focus on refractory patients
● Science based approach.
● Built on the passion and resilience of
many parents and patients.
About the SJIA Foundation

6. Work with Patients
● Build awareness among patients about
options if disease is refractory
● Help patients with second opinions to
research centers
● Encourage patients to share experiences
with new treatments
● Encourage patients to participate in
research studies
● Encourage patients to get specialized labs
(e.g., IL18, CXCL9)
Strategy for Accelerating
Treatments
Work with Physicians & Scientists
● Help them understand patient needs, especially
that of refractory patients
○ Share patient stories
● Help identify smaller subsets of refractory
patients who have similar disease
● Help them gather patient input via focus groups,
surveys etc.

7. The NextGen Conference
● Founded in 2014 by Jon Boutelle and Rashmi Sinha
● Parents of a child with SJIA
● •Goal to find new treatments and ultimately a cure – focus on refractory patients
● •We support, promote science and scientists to research and find a cure for this harsh disease.
● •We do this via our events –
● •Annual Family Conference– for patient education & to drive research
● •NextGen Therapies for SJIA, Still’s & MAS – to bring together researchers, patients, regulators and
pharma to drive towards a cure
In 2016, 2017, 2019 & 2022
Bring together patients, physicians, regulators & pharma to discuss next
treatments
Representatives from FDA / EMA have attended every meeting
Several treatments for SJIA /
Still’s were first discussed at
NextGen. Patients &
Clinicians learnt about the
options.
● Jak-inhibitors (2017)
● emapalumab
● BMT for refractory
SJIA
● IL-18 inhibitors

8. The SJIA Family Conference
● 3 SJIA Family conferences (2018, 2019,
2020)
○ 2020 was online
● Helped families meet others in similar
situations and form lifelong friendships
● Also organized second opinions for
refractory patients during conference

9. Grant from
Chan-Zuckerberg Foundation
● SJIA Foundation is part of the Rare as One Network
○ One of 50 patient-driven Patient-driven Foundations who received $475k to
drive cures for Rare Diseases
● Grant helped fund
○ Accelerate: Study focused on children with refractory SJIA, in order to accelerate
development of new therapies for this disorder.
■ Multi-center study: 7 US Centers (Cincinnati CHildren’s, Boston Children’s, CHOP, Texas
Children’s, Univ of Alabama, Univ of Minnesota, HSS)
■ Will coordinate with European SJIA projects as well

10. Our Support of
Research in Europe
● SJIA / Still’s being rare diseases require international perspective to understand.
○ Seed grant to UMC Utretch for Resyst (an international project to investigate
refractory SJIA)
○ Seed grant to study SJIA-Lung Disease in Europe
■ A retrospective and prospective cohort study (Bambino Gesu Ospedale)
● Increasing European Patients in our network:
○ Planning an event for International Patients in collaboration with European SJIA
experts

11. Find Clinical Trials in SJIA,
Stills & MAS on our website
https://www.systemicjia.org/clinical-trials-in-sjia-aosd-mas/
Summary of Open Clinical Trials
● In SJIA & AOSD
● In Mas, Primary / Secondary
HLH
● For Inflammasome
Inhibitors in
Autoinflammatory Diseases
Full data downloadable (google
spreadsheets)
Compiled over months by Luciana
Peixoto

12. Coming soon:
Open Research Studies
Org / Coordinating
Center
Researchers / Main
Contact
What patients are eligible? What centers
CARRA Registry CARRA Yukiko Kimura . Newly diagnosed sJIA (criteria?)
. When starting a new medication
76 centers, mostly US, a few
international
CARRA (STARS) Study CARRA Michael Ombrello,
Lauren Henderson
Patients enrolled in CARRA SJIA
Registry at some sites
16 US centers
NIH Natural History of
SJIA & Still's Study
NIH Michael Ombrello Diagnosis of SJIA / AOSD by a
physician
NIH, Maryland
CARRA SJIA-LD study CARRA Grant Schulert,
Yukiko Kimura
Patients diagnosed with SJIA and
probable/definite SJAI-LD
16 US centers
Accelerate (Accelerating
Therapies for Refractory
Patients)
SJIA Foundation +
Cincinnati Childrens
and 6 other research
centers
Grant Schulert,
Alexei Grom and
PI's from each of
the 6 centers
What SJIA criteria?
. Children <18 with refractory SJIA
7 US Centers + Coordination with
Europe
European SJIA-LD study Bambino Gesù
Children’s Hospital,
Rome, Italy
Claudia Bracaglia ILAR / CARRA / PRINTO new
classification criteria
- Diagnosis of sJIA
- Diagnosis of LD
- Disease onset before 18 years
All the European Paediatric
Rheumatology
centres involved in MAS/sJIA Working
Party (PReS) are participating

13. How to get involved:
Donate
● Donate!
○ As a mostly volunteer organization, we keep costs low (&
cover through grants). All donated money goes to research.
○ Do a Birthday Fundraiser on Facebook!
■ Last year, patients & their families did 57 fundraisers
■ Raised a total of $8,800
● Do a Fundraiser on Bonfire!
○ Several parents regularly run tshirt campaigns
● Run an event
○ One volunteer is planning a golf tournament
○ Other options are raffles, silent auctions…

14. How to get involved:
Volunteer
● Translation of articles & surveys: As we expand in Europe, we need translation help (as
we expand to more patients internationally). If you can help with below (or other
languages) get in touch at contact@systemicjia.org
○ French, German, Spanish, Portuguese, Italian, Dutch…
● Adult Patient volunteers to share patient patient journeys: We have several stories of
pediatric patients on our website. Need more adult stories.
● Other Skills based volunteering:
○ Fundraising expertise
○ Run mailchimp, Help manage Wordpress site

15. With Thanks to…
SJIA Parents
● Luciana Peixoto
● Anna Carlson
● Leah Bush
● Kari Cupp
● Zulayka Martinez
● Ronny Bachrach
● Vincent Delgaizo
● Sheila Taylor
● Katlin Reardon
● and many more…
Physicians we collaborate with
● Alexei Grom
● Scott Canna
● Fabrizio DeBenedetti
● Claudia Bracaglia
● Grant Schulert
● Bas Vastert
● Hermine Brunner
● Dan Lovell
● Peter Nigrovic
● Yuki Kimura
● Bella Mehta
● And many more…

16. Housekeeping: Breakout Rooms
Start Video &
Unmute for
Breakout Group
First Turn on your Camera and Unmute to speak!

17. Show your name & Parent / Patient
To rename yourself,
Click “Participants”
then “More”>
Rename

18. Click on Breakout Rooms
Click icon in
toolbar which
looks like this

19. Click “Join” for the
group you want to be
part of.
Then click “Yes”

20. You will be directed
to the Room you
selected

21. To leave the Group,
click on “Leave”, and
then “Leave Breakout
Group”

22. You will return to the Main Session:

23. Session 1: Break Out Groups
● Older Teens & Young adults (15 & up) - Patients
Room 1
● Older Teens & Young adults (15 & up) Parents/Caregivers
Room 2
● Managing Macrophage Activation Syndrome
Room 3
● Role of Physical / Occupational Therapy /
Room 4

24. Session 2: Break Out Groups
● Managing Stubborn Joint Issues (arthritis, hypermobility etc.)
Room 7
● Newer Meds, such as jak-inhibitors / emapalumab / IL18 inhibitors
Room 8
● Parent / Caregiver Support Chat
Room 9
● AOSD Patients Chat
Room 10

25. Thank You!

26. 1 Sandra Enciso sandiaenciso@gmai
l.com
c) I am a Physician /
Scientist
Hospital
español
mexico city Mexico
2 AnnaCarin Horne annacarin.horne@re
gionstockholm.se
c) I am a Physician /
Scientist
Stockholm Sweden
3 Sayyora Akhmedova drsayyora@gmail.co
n
c) I am a Physician /
Scientist
Tashkent Uzbekistan
4 Daniel Lovell daniel.lovell@cchmc
.org
c) I am a Physician /
Scientist
Cncinnati United States
5 Aryan Jalal aryan.jalal87@yaho
o.com
c) I am a Physician /
Scientist
Doctor
rheumatology
Erbil Iraq
6 Rodrigo Suárez reumatologiadoc202
2@gmail.com
c) I am a Physician /
Scientist
Montevideo Uruguay
7 Sheila Olioveira sheila_knupp@hotm
ail.com
c) I am a Physician /
Scientist
Rio de Janeiro Brazil
8 Susan Prockop susan.prockop@chil
drens.harvard.edu
c) I am a Physician /
Scientist
Boston United States
9 Tracy Augustine gsusa1912@aol.co
m
Other Babylon United States
10 Dalwoo Jung avbcu@aprilbio.com d) I work in the
healthcare field
Seoul South Korea
11 Joana Araújo JOANA.ARAUJO@
LILLY.COM
d) I work in the
healthcare field
Eli Lilly Alcobendas Spain
12 Trish Reynolds reynoldsp2@nih.gov d) I work in the
healthcare field
RN/writer-
editor
Bethesda United States