Establishing clinical meaning and defining important differences for Patient-Reported Outcomes Measurement Information System (PROMIS^®) measures in juvenile idiopathic arthritis using standard setting with patients, parents, and providers

Abstract

Background

Patient-Reported Outcomes Measurement Information System (PROMIS) measures are used increasingly in clinical care. However, for juvenile idiopathic arthritis (JIA), scores lack a framework for interpretation of clinical severity, and minimally important differences (MID) have not been established, which are necessary to evaluate the importance of change.

Methods

We identified clinical severity thresholds for pediatric PROMIS measures of mobility, upper extremity function (UE), fatigue, and pain interference working with adolescents with JIA, parents of JIA patients, and clinicians, using a standard setting methodology modified from educational testing. Item parameters were used to develop clinical vignettes across a range of symptom severity. Vignettes were ordered by severity, and panelists identified adjacent vignettes considered to represent upper and lower boundaries separating category cut-points (i.e., from none/mild problems to moderate/severe). To define MIDs, panelists reviewed a full score report for the vignettes and indicated which items would need to change and by how much to represent “just enough improvement to make a difference.”

Results

For fatigue and UE, cut-points among panels were within 0.5 SD of each other. For mobility and pain interference, cut-scores among panels were more divergent, with parents setting the lowest cut-scores for increasing severity. The size of MIDs varied by stakeholders (parents estimated largest, followed by patients, then clinicians). MIDs also varied by severity classification of the symptom.

Conclusions

We estimated clinically relevant severity cut-points and MIDs for PROMIS measures for JIA from the perspectives of multiple stakeholders and found notable differences in perspectives.

Appendices

Appendix 1: Clinical vignettes

(Note: T score locations are provided, but were not printed on participant copies)

Fatigue

Anne’s fatigue (T score = 27.5)

In the last 7 days, Anne never got tired easily and never had trouble starting things because she was too tired. She felt that she was never so tired that it was hard for her to focus on work. She never found it hard to get out of bed in the morning because she was too tired and she never felt too tired to do sports or exercise.

In summary, Anne reports:

• Never getting tired easily.

• She never had trouble starting things because she was too tired.

• Never feeling so tired that it was hard for her to focus on work.

• It was never hard to get out of bed in the morning because of being too tired.

• Never feeling too tired to do sports or exercise.

Taylor’s fatigue (T score = 37.5)

In the last 7 days, Taylor sometimes felt tired. She was never too tired to go up and down a lot of stairs and she never had trouble finishing things because she was too tired. It was never hard for her to play or go out with her friends as much as she likes, and being tired never made it hard for her to keep up with her schoolwork.

In summary, Taylor reports:

• Sometimes feeling tired

• Never being too tired to go up and down a lot of stairs.

• She never had trouble finishing things because she was too tired.

• Never being so tired that it was hard for her to play or go out with her friends as much as she would like.

• It was never hard for her to keep up with her schoolwork due to being tired.

David’s fatigue (T score = 42.5)

In the last 7 days, David sometimes felt tired but almost never got tired easily. He was never too tired to do things outside or to enjoy the things that he likes to do. Similarly, he was never so tired that it was hard for him to focus on his work.

In summary, David reports:

• Sometimes feeling tired.

• He almost never got tired easily.

• He was never too tired to do things outside.

• He was never too tired to enjoy things that he likes to do.

• It was never hard to focus on work due to being tired.

Zoe’s fatigue (T score = 47.5)

In the last 7 days, Zoe was never too tired to read or to watch television. She almost never felt weak, but sometimes felt more tired than usual when she woke up in the mornings. She also mentioned that it was sometimes hard for her to get out of bed in the morning because she was too tired.

In summary, Zoe reports:

• She was never too tired to read.

• Never being too tired to watch television.

• Almost never feeling weak.

• Sometimes she felt more tired than usual when waking up in the morning.

• Sometimes it was hard to get out of bed in the morning because she was too tired.

Katie’s fatigue (T score = 52.5)

In the last 7 days, Katie sometimes got tired easily. Being tired never made it hard for her to keep up with her schoolwork, and she almost never had trouble starting things because she was too tired. In addition, she was never too tired to take a bath or shower but sometimes she was so tired that it was hard for her to pay attention.

In summary, Katie reports:

• Sometimes she got tired easily.

• Being tired never made it hard for her to keep up with her schoolwork.

• She almost never had trouble starting things because of tiredness.

• She was never too tired to take a bath or shower.

• Sometimes she was so tired that it was hard for her to pay attention.

Jessica’s fatigue (T score = 57.5)

In the last 7 days, Jessica sometimes was too tired to do things outside. Sometimes she had trouble finishing things because she was too tired, and being tired sometimes kept her from having fun. She was also sometimes too tired to read. She never felt too tired to eat.

In summary, Jessica reports:

• Sometimes she was too tired to do things outside.

• Sometimes she had trouble finishing things because she was too tired.

• Being tired sometimes kept her from having fun.

• Sometimes she felt too tired to read.

• She never felt too tired to eat.

Nevaeh’s fatigue (T score = 62.5)

In the last 7 days, Nevaeh sometimes was too tired to go up and down a lot of stairs and sometimes she was too tired to do sports or exercise. While she was almost never too tired to go out with her family, sometimes she needed to sleep during the day. Being tired sometimes made it hard for her to keep up with her school work.

In summary, Nevaeh reports she:

• Sometimes she was too tired to go up and down a lot of stairs.

• Sometimes she was too tired to do sports or exercise.

• Almost never felt too tired to go out with her family.

• Sometimes needed to sleep during the day.

• Sometimes being tired made it hard to keep up with schoolwork.

Grace’s fatigue (T score = 67.5)

In the last 7 days, Grace was sometimes too tired to do things outside. It was almost always hard for her to get out of bed in the morning and she often had trouble finishing things because she was too tired. She sometimes felt weak. Being tired sometimes made it hard for her to play or go out with her friends as much as she would like.

In summary, Grace reports she:

• Sometimes was too tired to do things outside.

• Almost always found it hard to get out of bed in the morning because of tiredness.

• Often had trouble finishing things because of tiredness.

• Sometimes felt weak.

• Sometimes because of being tired she found it hard to play or go out with friends as much as she would have liked.

Isabel’s fatigue (T score = 72.5)

In the last 7 days, Isabel almost always felt more tired than usual when she woke up in the morning. She often felt tired and sometimes felt too tired to spend time with her friends. Isabel was sometimes so tired that it was hard to focus on her work and she was almost always too tired to read.

In summary, Isabel reports she:

• Almost always felt more tired than usual when waking up in the morning.

• Often felt tired.

• Sometimes felt too tired to spend time with her friends.

• Sometimes was so tired that is was hard to focus on work.

• Almost always was too tired to read.

Owen’s fatigue (T score = 77.5)

In the last 7 days, Owen was sometimes too tired to eat and almost always too tired to do sports and exercise. He often had trouble starting things because he was too tired and felt that being tired almost always kept him from having fun. Owen almost always found being tired made it hard to play or go out with friends as much as he’d like.

In summary, Owen reports:

• Sometimes being too tired to eat.

• Almost always being too tired to do sports and exercise.

• He often had trouble starting things because of being tired.

• Being tired almost always kept him from having fun.

• It was almost always hard to play or go out with friends as much as he would have liked due to being tired.

Leah’s fatigue (T score = 82.5)

In the last 7 days, Leah was sometimes too tired to watch television, and almost always too tired to take a bath or shower. She was also almost always too tired to go out with her family, or enjoy the things that she liked to do. Being tired almost always made it hard for Leah to keep up with her schoolwork.

In summary, Leah reports she:

• Sometimes was too tired to watch television.

• Almost always was too tired to take a bath or shower.

• Almost always was too tired to go out with her family.

• Almost always was too tired to enjoy the things that she likes to do.

• Almost always had a hard time keeping up with her schoolwork.

Upper extremity function

Ella’s upper extremity (T score = 7.5)

In the last 7 days, Ella was not able to cut paper with scissors, nor was she able to lift a cup to drink. She was unable to open her clothing drawers and she could not take a bath without help. She also was not able to put on her shoes unassisted.

In summary, Ella reported she:

• Could not cut paper with scissors.

• She was not able to lift a cup to drink.

• Could not open her clothing drawers.

• Almost always needed help with a bath.

• Was not able to put on her shoes by herself.

Mary Kate’s upper extremity (T score = 12.5)

In the last 7 days, Mary Kate had a lot of trouble writing with a pen or a pencil. She not able to zip up her clothes, nor was she able to unlock a door with a key. She was also not able to open a jar or pour a drink from a full pitcher by herself.

In summary, Mary Kate reported she:

• Could write with a pen or pencil only with a lot of trouble.

• Was unable to zip up her clothes.

• Could not unlock a door with a key.

• Could not open a jar by herself.

• Was not able to pour a drink from a full pitcher by herself.

Chloe’s upper extremity (T Score = 17.5)

In the last 7 days, Chloe could put her shoes on by herself with a lot of trouble, but was not able to put on her socks. She could lift a cup to drink with some trouble. She was not able to open the rings in school binders nor was she able to open heavy doors.

In summary, Chloe reported she:

• Could put on her shoes by herself with a lot of trouble.

• Was unable to put on her socks by myself.

• Could lift a cup to drink, but with some trouble.

• Was unable to open the rings in school binders.

• Could not pull open heavy doors.

Brianna’s upper extremity (T score = 22.5)

In the last 7 days, Brianna had some trouble with opening jars. She had a little trouble with cutting paper with scissors, a little trouble with opening her clothing drawers, and a little trouble zipping up her clothes. However, she could not put toothpaste on her toothbrush by herself.

In summary, Brianna reported she:

• Could open a jar with some trouble.

• Was able to cut paper with scissors with a little trouble.

• Could open her clothing drawers with a little trouble.

• Could zip up her clothes with a little trouble.

• Was unable to put toothpaste on her toothbrush by herself.

Emma’s upper extremity (T score = 27.5)

In the last 7 days, Emma never needed help with a bath. She could dry her back with a towel with a little trouble, and with a little trouble she could put on her socks by herself. She had a little trouble unlocking a door with a key. She had no trouble with lifting a cup to drink.

In summary, Emma reports she:

• Never needed help with a bath.

• Could dry her back with a towel with a little trouble.

• Could put on socks with a little trouble.

• Had a little trouble using a key to unlock a door.

• Could lift a cup to drink with no trouble.

Sophia’s upper extremity (T score = 32.5)

In the last 7 days, Sophia has had a little trouble with opening the rings in her binders for school. She also had a little trouble with pouring a drink from a full pitcher. She had no trouble washing her face with a cloth, opening her clothing drawers, or writing with a pen or pencil.

In summary, Sophia reports she:

• Could open the rings in school binders with a little trouble.

• Could pour a drink from a full pitcher with a little trouble.

• Had no trouble washing her face with a cloth.

• Had no trouble opening her clothing drawers.

• Could write with a pen or pencil with no trouble.

Allison’s upper extremity (T score = 37.5)

In the last 7 days, Allison could put on her shoes by herself, pull on and fasten her seat belt, and dial a phone with no trouble. She also could put toothpaste on her toothbrush by herself with no trouble. She had a little trouble with opening a jar.

In summary, Allison reports she:

• Could put on her shoes by herself with no trouble.

• Had no trouble pulling on and fastening her seat belt.

• Had no trouble dialing a phone.

• Could put toothpaste on her toothbrush by herself without trouble.

• Could open a jar with a little trouble.

Hailey’s upper extremity (T score = 42.5)

In the last 7 days, Hailey could use a key to unlock a door with no trouble, and had no trouble pulling open heavy doors. She could brush her teeth by herself. She also could zip up her clothes and put on her socks by herself with no trouble.

In summary, Hailey reported she:

• Could unlock a door with a key with no trouble.

• Could pull open heavy doors with no trouble.

• Had no trouble with brushing her teeth by herself.

• Could zip up her clothes with no trouble.

• Could put on socks with no trouble.

Pain interference

Andrea’s pain (T score = 42.5)

In the last 7 days, Andrea never found it hard to get along with other people or pay attention when she had pain. She never hurt a lot and never found it hard to walk one block when she had pain. It was also never hard for Andrea to stay standing when she had pain.

In summary, Andrea reports:

• It was never hard to get along with other people when she had pain.

• It was never hard to pay attention when she had pain.

• She never hurt a lot.

• Never finding it hard to walk one block when she has pain.

• It was never hard to stay standing when she has pain.

Claire’s pain (T score = 47.5)

In the last 7 days, Claire sometimes found it hard to run when she was in pain. But her pain never caused her to feel angry. She never found it hard to have fun when in pain. She also never had trouble sleeping and never missed school when she had pain.

In summary, Claire reports:

• Sometimes finding it hard to run when she had pain.

• Never feeling angry when she had pain.

• It was never hard for her to have fun when she has pain.

• She never had trouble sleeping when she has pain.

• She never missed school when she has pain.

Maya’s pain (T score = 52.5)

In the last 7 days, Maya never hurt all over her body, but sometimes had trouble sleeping when she had pain. She never found it hard to walk one block when in pain. Maya almost never had trouble doing schoolwork when she had pain, but sometimes found it hard to pay attention when she had pain.

In summary, Maya reports she:

• Never hurt all over her body.

• Sometimes has trouble sleeping when in pain.

• Never found it hard to walk one block when in pain.

• Never has trouble doing schoolwork when in pain.

• Sometimes has a hard time paying attention when in pain.

Addison’s pain (T score = 57.5)

In the last 7 days, Addison found it sometimes hard to have fun when she had pain. It was never hard for her to remember things when she had pain. She sometimes felt angry and sometimes had trouble doing schoolwork when she had pain. She sometimes found it hard to stay standing when she had pain.

In summary, Addison reports:

• Sometimes it was hard to have fun when in pain.

• It was never hard to remember things when in pain.

• Sometimes feeling angry when she had pain.

• Sometimes she had trouble doing schoolwork when in pain.

• Sometimes it was hard to stay standing when she had pain.

Jacob’s pain (T score = 62.5)

In the last 7 days, Jacob often found it hard to run when he had pain. He sometimes hurt all over his body and found that when he had pain he sometimes missed school. It was also sometimes hard for him to walk one block or remember things when he had pain.

In summary, Jacob reports:

• Often found it hard to run when in pain.

• Sometimes hurt all over his body.

• Sometimes missed school when having pain.

• Sometimes it was hard for him to walk one block when he had pain.

• Sometimes it was hard to remember things when in pain.

Anna’s pain (T score = 67.5)

In the last 7 days, Anna almost always had trouble sleeping and often had trouble doing schoolwork when she was in pain. She sometimes hurt a lot, and it was almost always hard for her to stay standing when she had pain. She sometimes felt it was hard to get along with other people when she had pain.

In summary, Anna reports she:

• Almost always had trouble sleeping when in pain.

• Often had trouble doing schoolwork when in pain.

• Sometimes hurt a lot.

• Almost always had a hard time staying standing when she had pain.

• Sometimes finds it hard to get along with other people when in pain.

Julia’s pain (T score = 72.5)

In the last 7 days, Julia almost always felt angry when she had pain and almost always found it hard have fun. She often found it hard to remember things when she had pain. She sometimes missed school when in pain. It was almost always hard for her to walk one block when in pain.

In summary, Julia reports she:

• Almost always feels angry when in pain.

• Almost always finds it hard to have fun when in pain.

• Often finds it hard to remember things when in pain.

• Sometimes misses school when in pain.

• Almost always finds it hard to walk one block when in pain.

Kristen’s pain (T score = 77.5)

In the last 7 days, Kristen almost always hurt a lot and almost always hurt all over her body. She often found it hard to remember things when she had pain. Kristen also reported that it was almost always hard for to run and get along with other people she had pain.

In summary, Kristen reports she:

• Almost always hurts a lot.

• Almost always hurts all over her body.

• Often has a hard time remembering things when in pain.

• Almost always finds it hard to run when in pain.

• Almost always has a hard getting along with other people when she has pain.

Mobility

Lily’s mobility (T score = 7.5)

In the past 7 days, Lily was unable to move her legs. She could not get into bed by herself, nor stand up by herself. She was unable to turn her head all the way to the side and could not bend over to pick something up.

In summary, Lily reports she:

• Could not move her legs.

• Was unable to get into bed by herself.

• Was unable to stand up by herself.

• Could not turn her head all the way to the side.

• Was unable to bend over to pick something up.

Kylie’s mobility (T score = 12.5)

In the past 7 days, Kylie was unable to keep up with other kids her age when they played together. With a lot of trouble, she could go up one step. She was unable to stand on tiptoes and could not walk more than one block. She could not get in and out of a car.

In summary, Kylie reports that she:

• Could not keep up when playing with other kids.

• Could go up one step, but with a lot of trouble.

• Could not stand on her tiptoes.

• Could not walk more than one block.

• Was unable to get in and out of a car.

Madison’s mobility (T score = 17.5)

In the past 7 days, Madison was able to get out of bed by herself with a little trouble. While she could bend over to pick something up with some trouble, she found she was not able to get up from the floor. Madison almost always used a wheelchair to get around, and she was physically unable to do the activities she enjoys the most.

In summary, Madison reports she:

• Could get out of bed by herself with a little trouble.

• Could bend over to pick something up with some trouble.

• Was unable to get up from the floor.

• Almost always used a wheelchair to get around.

• Was not physically able to do the activities she enjoys most.

Chase’s mobility (T score = 22.5)

In the past 7 days, Chase had a lot of trouble with moving his legs. However, he was able to go up one step with a little trouble, and he could get up from a regular toilet with no trouble. Chase was unable to get down on to his knees without support and he could not do sports and exercise that other kids his age could do.

In summary, Chase reports he:

• Could move his legs with a lot of trouble.

• Could go up one step with a little trouble.

• Was able to get up from a regular toilet with no trouble.

• Could not get down on his knees without holding onto something.

• Was unable to do sports and exercise other kids his age could do.

Emily’s mobility (T score = 27.5)

In the past 7 days, Emily found that she was unable to ride a bike. With some trouble she could walk more than one block and stand up on her tip toes. Emily could get in and out of a car with a little trouble and never needed to use a walker, cane or crutches to get around.

In summary, Emily reports she:

• Was unable to ride a bike.

• With some trouble, she could walk more than one block.

• With some trouble, she could stand up on her tip toes.

• She could get in and out of a car with a little trouble.

• Never needed to use a walker, cane or crutches to get around.

Evelyn’s mobility (T score = 32.5)

In the past 7 days, Evelyn reports she could turn her head all the way to the side and get into bed by herself with no trouble. With a little trouble, Evelyn could get down on her knees unassisted, or walk up stairs without holding onto anything. However, she had some trouble with running a mile.

In summary, Evelyn reports she:

• With no trouble could turn her head all the way to the side.

• With no trouble could get into bed by herself.

• With a little trouble could get down on her knees without holding onto something.

• With a little trouble could walk up stairs without holding onto anything.

• Could run a mile with some trouble.

Olivia’s mobility (T score = 37.5)

In the past 7 days, Olivia had a little trouble with physically doing the activities she enjoys most. She found it was no trouble to stand up by herself and had no trouble moving her legs. She also had no trouble carrying her books in her backpack. Olivia had some trouble with doing sports and exercise that other kids her age could do.

In summary, Olivia reports she:

• With a little trouble, she could physically do the activities she enjoys most.

• Could stand up by herself with no trouble.

• Could move her legs with no trouble.

• Carried her books in her backpack with no trouble.

• With some trouble could do sports and exercise that other kids her age could do.

Lauren’s mobility (T score = 42.5)

In the past 7 days, Lauren found that she could get out of bed with no trouble. It was also no trouble for her to get up from the floor or to bend over to pick something up. She could ride a bike with no trouble; however, she had a little trouble keeping up when she played with the other kids.

In summary, Lauren reports she:

• Could get out of bed by herself with no trouble.

• Had no trouble getting up from the floor.

• Could bend over to pick something up from the floor with no trouble.

• Could ride a bike with no trouble.

• Had a little trouble keeping up when she played with the other kids.

Samantha’s mobility (T score = 47.5)

In the past 7 days, Samantha found herself able to get in and out of a car with no trouble, and also had no trouble with going up one step, or standing on her tiptoes. It was no trouble for her to do sports and exercise other kids her age could do. However, she did have a little trouble with running a mile.

In summary, Samantha reports she:

• Could get in and out of a car with no trouble.

• Could go up one step with no trouble.

• Had no trouble standing on her tiptoes.

• Could do sports and exercise that other kids her age can do, with no trouble.

• Was able to run a mile with a little trouble.

Caroline’s mobility (T score = 57.5)

In the past 7 days, Caroline found that she could keep up with the other kids when playing. She had no trouble walking up the stairs without holding on to anything, or getting down on her knees without holding on to something. She could run a mile with no trouble and has been physically able to do the activities she enjoys the most.

In summary, Caroline reports she:

• Could keep up with the other kids when playing with no trouble.

• Was able to walk up stairs without holding on to anything with no trouble.

• Could get down on her knees without holding on to something with no trouble.

• Had no trouble running a mile.

• Had no trouble being physically able to do the activities she enjoys the most.

Appendix 2: R-code to generate most likely item responses for a given T-score

Created by Dr. Ryoungsun Park, http://coe.wayne.edu/profile/fy3504