OMS Patient Registry – Natural History Study Title Layout Details on OMS Onset to Date – August 20, 2017 OMS Patient Registry & Natural History Study OMSLife Foundation Caregivers Conference SUBTITLE September 29, 2018
The OMSLife Foundation - Who we are Founded in 2012 Our mission is ➢ Raise awareness of OMS ➢ Provide a support network for patients and caregivers ➢ Fund OMS research We now work with a network of 1,025 OMS patients in 56 countries We have hosted seven OMS caregivers conferences since 2014 We co-host the OMS Medical conference in Abingdon England And now, we are building the first OMS Patient Reported Natural History Study OMS Patient Registry – Natural History Study 2
Before we discuss our Registry – A special thank you… Our doctors / presenters: Dr. Mark Gorman Kitty Petty Dr. Angela Waanders Dr. Wendy Mitchell Dr. Yasmin Khakoo Sue Rossov Brian Cisneros Our Board of Directors: Christy Dennis Hank Holley Becky Jennings Bill Bradley Shandi Riccioti Billy Enochs Anja Norman OMS Patient Registry – Natural History Study 3
Methodology – The OMS Patient Registry ▪ The OMS Patient Reported Registry is a natural history study over time chronicling changes in treatments, therapies, behaviors, relapses, education, and overall quality of life. ▪ Registry funding is supported by a 5 year cooperative agreement between National Organization of Rare Disorders (NORD) and the U.S. Food and Drug Administration (FDA). ▪ Data input is through an online portal housed and maintained by NORD. The portal allows customized data prompts and questions. ▪ Data are stored in a manner that meet all US government mandated standards for data security and PHI protection. ▪ The data collection forms, processes, and outputs were reviewed and approved by Hummingbird IRB. ▪ Data collection initiated Feb 2017. Data views provided in this presentation were as of April 2018. OMS Patient Registry – Natural History Study 4
Our Survey Process - The OMS Patient Registry ▪ We had 2 teams assist in the survey generation: ▪ Development team – 4 OMS caregivers/stakeholders ▪ Medical team – Mitchell, Lotze, Gorman, Tardieu, Berg ▪ Surveys were created by development team / modified by medical team ▪ Surveys and reports are sent for IRB approval ▪ Programmed by NORD ▪ Then launched ▪ Analytics for this presentation were done with assistance from Trio Health OMS Patient Registry – Natural History Study 5
What the Registry means to you OMS Specialists Researchers ▪ Provides view on larger sample sets than they ▪ Provide secondary mechanism to validate can otherwise access findings ▪ Research ▪ Allows opportunities to have surveys ▪ Other OMS registries developed for trends they are seeing ▪ ▪ Provide a base of potential patients for clinical Allows quick turnaround on patient data studies Patients and Caregivers Clinicians ▪ Provide resource information for discussions ▪ Provides reference materials on a variety of with clinicians OMS topics ▪ Provide data for fighting insurance claims ▪ Give alternative options when typical ▪ Give realistic view into patient outcomes protocols are not working OMS Patient Registry – Natural History Study 6
What the Registry means to you OMS Specialists Researchers ▪ Provides view on larger sample sets than they ▪ Provide secondary mechanism to validate can otherwise access findings ▪ Research ▪ Allows opportunities to have surveys ▪ Other OMS registries developed for trends they are seeing ▪ ▪ Provide a base of potential patients for clinical Allows quick turnaround on patient data studies Patients and Caregivers Clinicians ▪ Provide resource information for discussions ▪ Provides reference materials on a variety of with clinicians OMS topics ▪ Provide data for fighting insurance claims ▪ Give alternative options when typical ▪ Give realistic view into patient outcomes protocols are not working OMS Patient Registry – Natural History Study 7
Numbers have increased since February 2017 launch OMS Patient Registry – Natural History Study 8
OMS Natural History Study Forms and Fields 148 respondents across 4 primary forms as of April 2018.. Drug Treatments Demographics Onset & Diagnosis Therapies (n=104) (n=139) (n=123) (n=97) • Type • Age • Age at time • Type • IVIG • Race • Dates • Speech • Chemo/Immuno • Ethnicity • Symptoms • Physical • Steroids • Nationality • Diagnosis • Occupational • Other • Gender • How • Behavioral • Route • Sexual Preference • Who • Reason for therapy • Frequency • Occupation • Where • Start/Stop Dates • Duration • Family • Severity • Frequency • Side effects • Income • Effectiveness • Size • Care Agencies • Education • Insurance Type OMS Patient Registry – Natural History Study 9
Patient Demographics (n=139) Gender Birth Country No. (%) Australia 2/139 (2%) Female Male Brazil 1/139 (1%) 53% 47% Canada 5/139 (3%) Germany 5/139 (3%) Ireland 1/139 (1%) Race Netherlands 1/139 (1%) American Paraguay 1/139 (1%) Indian or Alaska Native 1% Other Spain 1/139 (1%) 10% White 85% Sweden 1/139 (1%) Asian 2% United Kingdom 3/139 (2%) Black United States 118/139 (84%) 2% OMS Patient Registry – Natural History Study 10
Patient Demographics (n=139) Mother’s Education Father’s Education Household Annual Income No Answer 2% No Answer 4% Not reported 17% Don't Know 2% Don't Know 5% Post College work 23% $200+ 8% Post College work 18% College Grad 35% College Grad 35% $150K<$200K 6% Went to College 17% Went to College 15% Trade school 9% $100K<$150K 18% Trade school 5% High School Grad 9% High School Grad 13% $50K<$100K 27% GED 2% GED 3% <$50K 24% Did not graduate 1% Did not graduate 2% 0% 10% 20% 30% 40% 0% 10% 20% 30% 0% 10% 20% 30% 40% Primary Insurance Health Insurance Family Size No. (%) Don't know 27% No SCHIP… 1% 2 family members 4/139 (3%) 1% Other… 1% No Reply 3 family members 31/139 (22%) Military health… 4% 3% 4 family members 48/139 (36%) Medicaid|Privat… 4% 5 family members 35/139 (25%) Medicaid 10% State-… 4% 6 family members 7/119 (5%) Yes Private health… 49% 96% 7 or more family members 8/119 (5%) 0% 20% 40% 60% Not Reported 6/139 (4%) OMS Patient Registry – Natural History Study 11
87% of patients were ≤36 months at symptoms onset; median age of pediatric onset was 18 months Age at Onset (months) Number Summary Overall Pediatric Age at Onset min 2.0 2.0 N=123 25th percentile 14.0 14.0 40% median 18.0 18.0 36% 35% mean 32.3 24.2 75th percentile 29.8 29.0 30% max 420.0 120.0 25% n 123 120 20% 15% Who diagnosed patient n=123 15% 13% 12% 10% Other 10% 7% 5% Oncologist 4% 5% 3% 11% 0% years months months months months months months Adult 3-17 13-18 19-24 25-30 31-36 7-12 0-6 Neurologist 84% OMS Patient Registry – Natural History Study 12
Days to Diagnose has decreased; Initial Diagnosis has become more Accurate Days to Diagnosis OMS Days to Diagnose OMS (n=118) 200 Onset Date Before 2010- 2015 – 168 180 2010 2014 Present 160 Mean Min 1 1 1 140 112 120 25 th Per Median 19 16 5 100 Median 61 38 14 80 61 60 Mean 112.2 167.6 26.8 38 27 40 75 th Per 157 127.5 31 14 20 Max 922 2038 280 0 Pre 2010 2010-2014 2015-Present n 33 40 45 Initial Diagnosis Trends 100% Initial Diagnosis (n=118*) OMS Acute Cerebellar Ataxia Other Onset Date Before 2010- 2015 – 80% 2010 2014 Present 58% OMS 9 17 26 60% 45% 44% Misdiagnosed 24 22 19 40% 33% 28% 27% - Acute Cerebellar Ataxia 15 13 10 23% 22% 20% 20% - Other 9 9 9 n 33 39 45 0% *Note – five people did not respond Pre 2010 2010-2014 2015-Present OMS Patient Registry – Natural History Study 13
Ataxia was the most common symptom at onset, indicated in 85% of patients % Patients with a given symptom at onset (n=123) 100% 85% 80% 60% 58% 60% 46% 44% 37% 40% 26% 20% 14% 13% 9% 0% Headache Fever Other* Vomiting Temper Sleep Tremors Opsoclonus Myoclonus Loss of balance tantrums disturbances (ataxia) *Other includes Clinginess (2), Depression (2), Diarrhea, Excessive Drooling (2), Head Tilt, Hypotonia, Loss of Appetite, Muscle Spams, Nystagmus, Seizures, and Spaced Out. OMS Patient Registry – Natural History Study 14
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