Endorsed Domain Definitions

Presenteeism refers to (the consequences of) being present at work while sick, which can be defined whether as decreased ability to perform tasks at work (worker’s perspective) or as the actual decrease in work output (products, services) due to health (societal/employer perspective).

Diagnosis of new or worsening bone fragility

A new diagnosis of hypertension or worsening blood pressure after initiation of glucocorticoid. Blood pressure elevation that is of concern to the clinician, the patient, or both.

The ability to perform basic and desired activities of daily living that is affected by limited functioning of muscles, mobility and instrumental acts of daily living due to myositis

Range of symptoms from mild subjective feelings of tiredness to an overwhelming debilitating, and sustained sense of exhaustion that likely decreases one’s ability to execute daily activities and function normally* in family or social roles

*Note: the term ‘normally’ is sometimes hard to define – here it relates to what is typical or usual for you as an individual.

Fatigue including tiredness and feeling wiped out

1. Effect of fatigue on daily functioning
2. Effect of fatigue on participation (social activity, work or relationships)
3. Effect of fatigue on feeling independent

A feeling of extreme tiredness or exhaustion attributable to myositis, limiting someone to perform his/her usual and meaningful daily activities

A term referring to the health aspects of quality of life, generally considered to reflect the impact of disease and treatment on disability and daily functioning. It has also been considered to reflect the impact of perceived health on an individual’s ability to live a fulfilling life.

Aching, soreness, or tenderness thought to be attributable to myositis (DM, PM, or IMNM) relating to joints, muscles, skin, or generalized, preventing someone to do what you normally do or from doing it as well as you normally would.

Impact of weight, appetite, and weight gain includes:
1. Personal perception of self and impact on mental health (including feelings of embarrassment, shame, or low self-esteem)
2. Influence on social participation and public social identity
3. Financial implications such as weekly grocery bill and purchasing new clothes

Diagnosis of recurrent, atypical or serious infection

Inflammatory markers (ESR, CRP, plasma fibrinogen) measured in people with PMR with a view to aiding assessment of disease activity and guiding treatment decisions

1) The impact of PMR on limiting a person’s physical function in their day-to-day life

or

2) Limitation to physical activity affecting a person’s ability to carry out activities of daily living, that a person attributes to their PMR

?ability to perform physical tasks person wants to complete

Mood lability or presence of anhedonia, depression, anxiety, or euphoria.

Component 1: anxiety – e.g., worry, stress, feeling overwhelmed
Component 2: depression – e.g., sadness, hopelessness
Component 3: lability – e.g., personality changes, irritability, impatience, anger
Component 4: anhedonia – e.g., avolition, apathy
Component 5: elevation – e.g., euphoria, hypomania, mania, psychosis

Note this target domain does not include: fatigue, cognitive deficits, life impact of mood disturbance
Suicidal ideation, although not formally associated with glucocorticoid medicines, is ordinarily assessed as a safety parameter in clinical trials of conditions or medications linked to depressed mood.

Formal diagnoses of mood or anxiety disorders, if made, and (changed) use of psychotropic medicines should be recorded in clinical trials as adverse events and concomitant medications. Hospitalization for treatment of a mood or anxiety disorder, even if voluntary, during a clinical trial should be reported as a serious adverse event

The intensity of pain (unpleasant, distressing sensation) a patient attributes to their PMR, at the worst time of day (usually the early morning) over the last 24-hour period – the intensity tends to be worst on movement.

How much a person’s shoulder hurts, reflecting the overall magnitude of the pain experience (ie, at rest, during and after activity, at night)

The intensity of the sensation of pain, encompassing the entire spectrum from a complete absence of pain to the most extreme levels of discomfort.

Consequences of pain on relevant aspects of one’s life. This includes the extent to which pain hinders engagement with social, cognitive, emotional, physical and recreational activities.

Patient-reported global rating of the status of the shoulder

Patient perception of disease activity includes the aspects of the disease that in the personal judgment of the patient (or parent/caregiver) directly relate to the JIA (such as joint
swelling, pain, stiffness), and may possibly respond to treatment/medication.
This assessment would not include the following: symptoms or physical findings thought to be due to another disease process or diagnosis, or due to treatment, or due to irreversible damage from JIA.

Patient perception of overall well-being as relates to disease (JIA) captures the view (of the patient or parent/caregiver) of the aspects of the patient’s life impacted by the patient’s JIA. It may be affected by the level of disease activity, burden of illness on health (physical, mental, or social), and impact of treatment/medications (positive changes, (e.g., improved mobility), or negative changes (e.g., side effects and administration of medication).

An outcome that represents the patient voice evaluating the symptomatic treatment-related adverse events for people with rheumatic and musculoskeletal diseases enrolled in clinical trials.
This would include severity, frequency, and interference of symptoms (i.e., distinct from ‘signs’) from different body systems.

A person’s ability to carry out daily physical activities required to meet basic needs (for example, bathing and brushing hair), and more complex activities that require a combination of skills (for example, driving a car).

Note that there is a separate participation domain which includes recreational activities and work (not part of physical function).

Physical functioning is defined as one’s ability to carry out various activities that require physical capability, ranging form self-care (activities of daily living) to more vigorous activities that require increasing degrees of mobility, strength, or endurance.

The “impediment of movement” (difficulty in moving) which a patient attributes to their PMR, at the worst time of day (usually the early morning) over the last 24-hour period

Weight, appetite, and weight gain includes:

1. Change in weight
2. Appetite and dietary changes, including food preferences and eating patterns, (i.e. overwhelming need to eat, eating more frequently or binging)

Flare in OA defined “a transient state, different from the usual state of the condition, with a duration of a few days, characterized by onset, worsening of pain, swelling, stiffness, impact on sleep, activity, functioning, and psychological aspects that can resolve spontaneously or lead to a need to adjust therapy” (Guillemin et al., 2019; p1189)

“Blood glucose (sugar), high blood glucose, development of diabetes mellitus and/or worsening control” that are of concern to the patient and clinician

Glucocorticoid therapy may contribute to the risk of development, or exacerbation, of diabetes. The pathophysiology of glucocorticoid induced diabetes is still unclear.

New or worsening glucose intolerance/insulin sensitivity

New diagnosis of Type 2 Diabetes Mellitus

Worsening existing diabetes

Worsening diabetic control

Worsening diabetic complications

Acute complications of symptomatic hyperglycaemia

Death – any causes of death should be documented 

A feeling of extreme tiredness or exhaustion attributable to IIM, interfering with usual and meaningful daily activities

Aching, soreness, or tenderness thought to be attributable to myositis (DM, PM, or IMNM) relating to joints, muscles, skin, or generalized, preventing someone from doing what you normally do or from doing it as well as you normally would. 

The ability to perform basic and desired activities of daily living that is affected by decreased use of muscles or other organ systems affected by IIM. 

The ability to perform basic to complex activities of daily living in a satisfactory manner that is affected by limited functioning due to myositis.

Definition:

The study participant follows through with the chosen option

Description:

This domain includes two descriptions of adherence depending on the type of decision (A and B) which include various components, as well as a separate component (5.5) to assess reasons for non-adherence if that is a concern:

A. Adherence for options that patients follow over time (e.g., medication, exercise routine):

Component 5.1:

The study participant starts using the chosen option (initiation phase)

Component 5.2:

The study participant uses the chosen option in the manner that was agreed upon (timing, frequency, dosing) with their clinicians (implementation phase)

Component 5.3:

The study participant continues using the chosen option for the intended duration, if applicable (persistence phase)

or

B. Adherence for one-time procedures or tests (e.g., surgery, injection, diagnostic test):

Component 5.4:

Proceed to have the procedure/test

Component 5.5:

-Reasons for non-adherence

Many factors may impact whether study participants use the chosen option, and should be taken into account if there is a concern for non-adherence (e.g., occurrence of side effects, costs, limited health literacy)

Definition:

The study participant feels sure or certain of the chosen option. They feel sure they made the best decision for themselves.

Description:

This domain includes one component:

The study participant’s perceived certainty in the chosen option.

How sure is the study participant that they chose the best option for themselves?

Definition:

The study participant’s chosen option* matches with the study participant’s values**. The study participant chooses the option that best matches the features (e.g., benefits, harms, mode of administration, timing) that they value most.

Description:

This domain includes three components:

Component 2.1:

-The chosen option(s)* after using the SDM intervention (A).

Component 2.2:

-The study participant’s values** (B).

Component 2.3:

-The extent to which there is a concordance between (A) and (B).

* The chosen option means any option that the participant chose when using the SDM intervention. It can include a wide variety of healthcare options.

**In this context, values represent how the study participant rates each of the features (e.g., benefits, harms, mode of administration, timing) of the healthcare options.

The study participant is knowledgeable and has a perception of being informed about the options. If probabilities are available, the study participant is knowledgeable about how likely the benefits and harms are to happen.

Description:

This domain includes two components:

Component 1.1:

-The study participant’s knowledge of the options, their potential benefits and harms, and if probabilities are available, how likely they are to happen.

Is the study participant able to correctly answer questions on the options, benefits and harms?

Component 1.2:

-The study participant’s feeling of being informed about the options, their potential benefits and harms.
Does the study participant feel informed about the options, benefits and harms?

The study participant feels satisfied about the way they made the decision and about their level of engagement in the decision-making process.

Description:

This domain includes two components:

Component 4.1:

-The extent to which the study participant feels satisfied about the way they made the decision (e.g., identifying the decision, exchanging information, clarifying values, deliberating, feeling prepared to make a decision).

Component 4.2:

-The extent to which the study participant feels satisfied about their level of engagement in the decision-making process, meaning how involved they were (e.g., feeling heard, talked about what was important to them, asked questions to better understand the options).

Potential negative consequences that may arise from using the SDM intervention, such as being difficult to use, stressful, or requiring too much time or money.

Description:

This domain includes two components in life impact and adverse effects and two components in resource use:

In life impact and adverse effects:

Component 6.1:

-Difficulty for the study participant to use the SDM intervention (e.g., requires high literacy to use, not available in participant’s language, requires technical skills or technology)

Component 6.2:

-The study participant’s perceived emotional distress related to the use of the SDM intervention or decision regret

In resource use:

Component 6.3:

-Time required to use the SDM intervention

Component 6.4:

-Cost of using the SDM intervention (cost of using the SDM intervention and cost stemming from the use of the chosen options)

-Physical space and technology required to use the SDM intervention