PFDD Pre-Meeting Survey

Share your experiences living with lipodystrophy. Your voice is what matters most in communicating the burdens of lipodystrophy to the FDA. Thank you for taking the time to share!

EL-PFDD Pre-Meeting Survey

Who is completing this survey?

I am a patient living with lipodystrophy

I am a care partner caring for someone with lipodystrophy

About You

What is your sex assigned at birth?

Male

Female

Other

Prefer not to say

Which country do you currently live in?

Which US state or territory do you currently live in?

Diagnosis

What is your specific lipodystrophy diagnosis?

CGL (Congenital Generalized Lipodystrophy)

AGL (Acquired Generalized Lipodystrophy)

FPLD (Familial Partial Lipodystrophy)

APL (Acquired Partial Lipodystrophy)

Other

I am unsure

I don't have an official diagnosis yet

Please describe

What type of clinician(s) did you see before getting the correct lipodystrophy diagnosis? (Select all that apply)

Family Medicine / Primary Care Physician

Endocrinologist

Cardiologist

Gastroenterologist

Hepatologist

Dermatologist

OB/GYN

Other

Please provide your previous physician's specialty

Please provide your diagnosing physician's specialty

Please provide your prior misdiagnoses

Symptoms

In the past 12 months, how often have you experienced the following symptoms related to your lipodystrophy?

	Never	Rarely	Sometimes	Often	Always
Extreme hunger (hyperphagia)
Fatigue / Low energy
Widespread pain
Abdominal pain
Feeling self-conscious about appearance
Brain fog / Difficulty concentrating
Muscle weakness
Muscle cramps
Joint pain
Skin changes (e.g. acanthosis nigricans: dark skin patches)
Liver problems / Abdominal swelling
Irregular periods / fertility issues
Episodes of acute pancreatitits

Please provide the most burdensome symptom you've experienced in the past 12 months that was not listed in the table above.

If you experience hyperphagia (extreme hunger), how does it affect you? (Select all that apply)

Constant preoccupation with food

Binge eating episodes

Waking up at night to eat

Irritability or food anxiety when food is restricted

Seeking or hoarding food

Feeling hungry shortly after a full meal

Difficulty concentrating due to hunger

Feeling out of control with eating

I do not experience hyperphagia

Mental Health & Social Impact

Have you been diagnosed with any of the following mental health conditions? (Select all that apply)

Depression

Generalized Anxiety Disorder (GAD)

Panic Disorder

ADHD

Obsessive-Compulsive Disorder (OCD)

Adjustment Disorder

Eating Disorder

None of the above

Prefer not to say

Daily Life

During the past 7 days, how much did lipodystrophy make it harder for you to do your usual daily activities?

Not at all

A little

Somewhat

Quite a bit

Extremely

Do the effects of lipodystrophy on your activities tend to stay about the same, or do they come and go?

Stay about the same

They come and go

Not sure

Current Treatment

Which of the following treatments are you currently using? (Select all that apply)

Leptin replacement therapy

Insulin

Statins (e.g. atorvastatin)

Fibrates (e.g. fenofibrate)

GLP-1 receptor agonists (e.g. semaglutide, tirzepatide)

Dietary management only

Other medication

No treatment currently

Please list other medications

Please provide your most burdensome symptom

How well does your current treatment control the symptom you identified above?

Not at all controlled

Poorly controlled

Moderately controlled

Well controlled

Completely controlled

I am not receiving treatment for this symptom

What are the most significant downsides to your current treatments? (Select up to 3)

Frequent or painful injections

Need for constant refrigeration / Travel logistics

Side effects (e.g. nausea, skin reactions)

Time devoted to managing treatments

High cost or insurance hurdles

Extremely restrictive diet

Not effective enough at controlling symptoms

Treatment loses effectiveness over time, requiring dose increases or frequent medication changes.

Have you ever stopped a treatment because the side effects were worse than the disease symptoms?

Yes

I considered it but decided against it

Future Treatment & Clinical Trials

Which ONE of the following would represent the most meaningful improvement from a new treatment?

Significant reduction in hunger / food noise

Increased energy levels

Stabilization of blood sugar / triglycerides

Improved physical appearance (fat redistribution)

Better quality sleep

Ability to work or socialize normally

Reduction in chronic pain

Other

Describe the most meaningful improvement not listed above

What percent of change in your disease and/or symptom management would be a meaningful change?

What matters MOST to you in an ideal future treatment? (Select ONE)

Long-lasting effect (more time on the same medication or same doses)

Oral medication instead of injection

Zero or minimal side effects

Addresses the root cause of fat loss

Easy to access regardless of location

Affordable and covered by insurance

Reduces the need for a restrictive diet

Other

What matters most in an ideal future treatment that is not listed above?

How willing would you be to participate in a clinical trial that required a liver biopsy?

Very unwilling

Somewhat unwilling

Neutral

Somewhat willing

Very willing

What is the maximum time you would be willing and able to travel for a clinical trial visit?

Under 1 hour (local)

1-2 hours (regional)

3-5 hours (domestic flight or long drive)

International flight, if needed

Would you consider taking a drug that carried a risk of side effects if it significantly reduced your hyperphagia?

Yes

It depends on the severity of the side effects

Provide the factor that would make it easier to participate in a trial.

What factor is most important to you that is not listed above?

Closing

If you could tell the FDA ONE thing about what it is really like to live with lipodystrophy, what would it be?

Is there anything about living with lipodystrophy that you feel is not captured in this survey? Please share in your own words.

About You

Which country do you currently live in?

Which US state or territory do you currently live in?

About the person you care for

What is their sex assigned at birth?

Male

Female

Other

Prefer not to say

What is their specific lipodystrophy diagnosis?

CGL (Congenital Generalized Lipodystrophy)

AGL (Acquired Generalized Lipodystrophy)

FPLD (Familial Partial Lipodystrophy)

APL (Acquired Partial Lipodystrophy)

Other

I am unsure

I don't have an official diagnosis yet

Please describe

Please provide your diagnosing physician's specialty

Please provide your prior misdiagnoses

In the past 12 months, how often has the person you care for experienced the following symptoms related to their lipodystrophy?

	Never	Rarely	Sometimes	Often	Always
Extreme hunger (hyperphagia)
Fatigue / Low energy
Widespread pain
Abdominal pain
Feeling self-conscious about appearance
Brain fog / Difficulty concentrating
Muscle weakness
Muscle cramps
Joint pain
Skin changes (e.g. acanthosis nigricans: dark skin patches)
Liver problems / Abdominal swelling
Irregular periods / fertility issues
Episodes of acute pancreatitits

Please provide the most burdensome symptom that has impacted your life in the past 12 months that was not listed in the table above.

Current Treatment

Which of the following treatments is the person you care for currently using? (Select all that apply)

Leptin replacement therapy

Insulin

Statins (e.g. atorvastatin)

Fibrates (e.g. fenofibrate)

GLP-1 receptor agonists (e.g. semaglutide, tirzepatide)

Dietary management only

Other medication

No treatment currently

Please list other medications

As a caregiver, what are the most significant downsides of your loved one's current treatments? (Select all that apply)

Administering frequent or painful injections to my loved one

Managing refrigeration and travel logistics for medications

Watching my loved one experience side effects (e.g. nausea, skin reactions)

Time I spend managing and coordinating treatments

High cost or insurance hurdles affecting access to treatment

Enforcing or preparing an extremely restrictive diet

Treatments not being effective enough at controlling my loved one's symptoms

Treatments losing effectiveness over time, requiring dose increases or frequent medication changes

Caregiver Burden

Please provide your most burdensome symptom

How well does their current treatment control the symptom you identified above?

Not at all controlled

Poorly controlled

Moderately controlled

Well controlled

Completely controlled

They are not receiving treatment for this symptom

Which ONE of the following would represent the most meaningful improvement from a new treatment?

Significant reduction in hunger / food noise

Increased energy levels

Stabilization of blood sugar / triglycerides

Improved physical appearance (fat redistribution)

Better quality sleep

Ability to work or socialize normally

Reduction in chronic pain

Other

Provide your most meaningful improvement not listed above

How much does caring for the person with lipodystrophy affect the following areas of YOUR life?

	Not at all	Slightly	Moderately	Significantly	Extremely
Controlling access to food and the kitchen
Seeing the person I care for suffer or feel embarrassed
Managing daily injections or medical equipment
Managing insurance approvals and specialist center visits
Fear of the future (e.g. organ failure or sudden pancreatitis)
Explaining the condition to teachers, friends, or extended family

Clinical Trials

Which of the following factors would most influence your decision about whether to enroll the person you care for in a clinical trial? (Select up to 3)

Time commitment (taking time off work or school)

Travel distance to the study site

Types of procedures required (e.g. liver biopsy, frequent blood draws)

Chance of receiving placebo (no active drug)

Potential safety risks or unknown side effects

If you were to accompany your loved one to a clinical trial visit, what is the maximum travel time you would be willing and able to manage?

Under 1 hour (local)

1-2 hours (regional)

3-5 hours (domestic flight or long drive)

International flight, if needed

Provide the factor that would make it easier to decide to participate in a trial.

What factor is most important to you that is not listed above?

Future Treatment

Which of the following changes would you most like to see for the person you care for? (Select ONE)

Reduction in their hunger / food-seeking behavior

An oral treatment rather than an injection

More energy and stamina for daily life

Lower risk of emergency events like acute pancreatitis

Other

Provide the change not listed above

What percent of change in your loved one's disease and/or symptom management would be a meaningful change?

Daily Life

Thinking about a recent day when lipodystrophy affected the person you care for a lot, what did you OBSERVE?

Thinking about a recent day when lipodystrophy affected them very little, what did you observe that was different?

On days when their lipodystrophy affects them a lot, how does caring for them affect YOUR own activities or responsibilities? Please give an example.

If you could tell the FDA ONE thing about caring for someone with lipodystrophy, what would it be?

Is there anything about caring for someone with lipodystrophy that you feel is not captured in this survey? Please share in your own words.

PFDD Pre-Meeting Survey

Share your experiences living with lipodystrophy. Your voice is what matters most in communicating the burdens of lipodystrophy to the FDA. Thank you for taking the time to share!

About You

Diagnosis

Symptoms

Mental Health & Social Impact

Daily Life

Current Treatment

Future Treatment & Clinical Trials

Closing

About You

About the person you care for

Current Treatment

Caregiver Burden

Clinical Trials

Future Treatment

Daily Life

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