NEXUS NARCOLEPSY REGISTRY

Mark Patterson, MD, PhDNarcolepsy Network, Inc.Carilion Children’s Hospital22 October 2016

WHAT IS A PATIENT REGISTRY?

• At it’s simplest, a patient registry is a database of information on people dealing with a similar medical condition.

• Can also be known as a medical or disease registry. • Information is keep confidential and the data is de-

identified in order to protect the privacy of the individual patients.

HOW DO REGISTRIES HELP PATIENTS?

• As noted by the National Institutes of Health, “Registries can provide health care professionals and researchers with first-hand information about people with certain conditions”.

• The goal is to increase understanding of those conditions amongst those who can help.

• Especially true in uncommon conditions.

PATIENTCROSSROADS• PatientCrossroads (www.patientcrossroads.com) is an

organization which maintains Connect registries on 64 medical conditions.

• https://vimeo.com/110811116• These registries help patients and their families,

advocacy organizations, researchers, and industry.

NEXUS NARCOLEPSY REGISTRY (NNR)

• In June 2015, PatientCrossroads launched the NNR.• They hope to enroll up to 1,500 people age 18 and

over who have been diagnosed with Type 1 or Type 2 narcolepsy.

• Following the initial survey, data will be updated every 6 months for up to 3 years.

• https://narcolepsy.patientcrossroads.org/

NNR STEERING COMMITTEE

• Dr. Michael Thorpy, Albert Einstein College of Medicine.

• Dr. Thomas Scammell, Harvard Medical School.• Dr. Mark Patterson, Carilion Children’s Hospital.• David Pasta, ICON plc.• Dr. Jed Black, Jazz Pharmaceuticals.• Dr. Maurice Ohayon, Stanford University.• Kathleen Villa, Jazz Pharmaceuticals.• Monica Gow, Wake Up Narcolepsy.

NNR PROTOCOL• All patient data is de-identified.• The participants may withdraw at any time.• Those who complete the survey can compare their

answers to others in the registry. • Findings from the database will be submitted for

publication in medical journals and shared at medical meetings.

ENROLLMENT TO DATE• As of 9 August 2016, 902 people have joined.• Of these, 746 have completed Wave 1 (the initial

survey).• 535 participants are eligible for Wave 2 (you are eligible

182 days after completion of previous survey).• Wave 2 has been completed by 261 patients. • Most participants have been women; we need more

men to join!

PARTICIPANT DISTRIBUTION

TOPICS COVERED IN QUESTIONS

• Survey attempts to cover as many aspects of narcolepsy’s impact as possible.

• Initial survey longest, others much shorter.• Basic demographics.• Diagnosis timing, missteps.• Medications.• Effect on quality of life (family, career).• Personal narratives.

PATIENT DEMOGRAPHICS• Mean age of participants is 39.• Average symptom onset was at 19 years old.• First average physician consult for narcolepsy

symptoms was at 27 years of age.• Average age at diagnosis was 31, representing a delay

of 12 years from symptom onset!• 32% of patients reported seeing 5 or more physicians

before being correctly diagnosed.

SYMPTOMS LEADING TO 1ST CONSULT

0102030405060708090

100

Excessive Sleepiness Poor ConcentrationWeakness Spells Poor School/Work PerfromanceVivid Dreams/Hallucinations Sleep Paralysis

INITIAL MISDIAGNOSES

0

5

10

15

20

25

30

35

Depression Anxiety Disorder ADHD Insomnia HypersomniaSleep Apnea Bipolar Disorder Epilepsy Schizophrenia

NON-MENTAL HEALTH CONDITIONS

0

5

10

15

20

25

30

35

Headache/Migraine Obesity Sleep ApneaRestless Leg Syndrome High Blood Pressure High CholesterolDiabetes

CURRENT MENTAL HEALTH CONDITIONS

0

10

20

30

40

50

60

Depression Anxiety None Panic Attacks Mood Disorder Bipolar Disorder

WEEKLY DRIVING FREQUENCY

0

10

20

30

40

50

60

Daily 4-6 Days/Week 1-3 Days/Week <1 Day/Week Never

No Cataplexy Cataplexy

PATIENT NARRATIVES• “Communicating my condition to others is stressful and frustrating. They

usually don't understand, which is emotionally difficult. The first reaction is typically laughter, until I explain what narcolepsy actually is. Many times people still think I am just either lazy or not getting enough sleep (hours).”

• “Whilst I am a generally happy person and I am well supported by my family, narcolepsy makes my life very lonely and causes me to feel isolated at times. I can no longer work so I don't have the same level of interaction with people that I did for all of my working life.”

ADDITIONAL RESPONSES• “The effects I suffer from most are foggy brain, automatic behavior,

procrastination, slow moving, always late, feeling stuck, spacey, forgetful.”

• “Each & every day is a battle to make sure I get my first dose taken the second I wake up or I'll fall back asleep & then it might be 3-4 hours before I wake up again, hopefully awake enough the second time around to take my dose or the cycle starts all over again. I really hope all these questions help.”

GOING FORWARD• Short, additional surveys will be sent out every 6 months

for up to 3 years. • Will allow for monitoring changes over time, investigate

other topics.• Additional areas with focus on impact on quality of life,

efficacy of medication combinations, issues associated with anesthesia.

WHAT YOU CAN DO• Join the registry!• https://narcolepsy.patientcrossroads.org/• Complete the follow up surveys when they are sent to

you. • Compare your responses to others with narcolepsy (all

anonymous).

• Thank You!!

QUESTIONS?