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Table 1 Patient-reported outcomes collected in the Ig20Gly North American and European phase 2/3 trials

From: Patient experience with subcutaneous immunoglobulin 20%, Ig20Gly, for primary immunodeficiency diseases: a prespecified post hoc analysis of combined data from 2 pivotal trials

Instrument

Domain score assessed

Concept

Specific item-level score assessed

Study included

North American

European

LQIa

Treatment interference (scaled score range: 0–100)

• Interference with social/family life

• Time waiting

• Treatment is worthwhile

• Dependency on others

• Freedom to take trips or move

• Scheduled according to patient’s convenience

Scheduled according to patient’s convenience (score range: 1–7)

x

x

Therapy-related problems (scaled score range: 0–100)

• Convenience

• Painfulness

• Health improvement

• Anxiety or nervousness

Convenience (score range: 1–7)

x

x

Therapy setting (scaled score range: 0–100)

• Interference with work/school

• Given in a comfortable place

• Given in a pleasant atmosphere

Interference with work/school (score range: 1–7)

x

x

TSQM-9b

Effectiveness (score range: 0–100)

• Ability of the medication to prevent or treat conditions

• The way the medication relieves symptoms

• Time before the medication works

–

x

–

Convenience (score range: 0–100)

• Easy/difficult to use the medication in its current form

• Easy/difficult to plan when to use the medication each time

• Convenient/inconvenient to take the medication as instructed

Convenient/inconvenient to take medication as instructed (score range: 1–7)

x

–

Global satisfaction (score range: 0–100)

• Overall confidence that taking this medication is a good thing

• Certainty that good things about medication outweigh bad things

• Overall satisfaction/dissatisfaction with medication

Overall satisfaction/dissatisfaction with medication (score range: 1–7)

x

–

Treatment preferencec

–

• Like/dislike aspects of administration

• Preference to continue Ig20Gly

Multiple items

–

x

  1. aIn the European study, the LQI was self-administered by patients aged ≥14 years (observer: patient) and completed by parents/caregivers for patients aged 2–13 years (observer: parent/caregiver). In the North American study, the LQI was self-administered by patients aged ≥13 years (observer: patient) and completed by parents/caregivers for patients aged 2–12 years (observer: parent/caregiver)
  2. bIn the North American study, the TSQM-9 was self-administered by patients aged ≥13 years (observer: patient) and completed by parents/caregivers for patients aged 2–12 years (observer: parent/caregiver)
  3. cIn the European study, the treatment preference questionnaire was self-administered by patients aged ≥14 years (observer: patient) and completed by parents/caregivers for patients aged 2–13 years (observer: parent/caregiver).
  4. Ig20Gly, immune globulin subcutaneous (human) 20%: LQI, Life Quality Index; TSQM-9, Treatment Satisfaction Questionnaire for Medication-9