Table 4 Domain-specific observations and comments for PaPaS for assessment of patients

1.1 Trajectory of disease and impact on daily activities of the child (in comparison with the child’s own baseline) (with reference to the last 4 weeks)

Stable

(0)

1. Though the two measured constructs (disease trajectory and impact on daily activities) are related, they can vary independently. Both should be scored separately.

2. Children with life-limiting conditions generally have longer disease trajectories. It is recommended that the reference baseline for Activities of Daily Living be extended to 3 months (instead of just 4 weeks) to maximise detection of change.

Slowly deteriorating without impact on daily activities

(1)

Unstable; with impact on and restriction of daily activities

(2)

Significant deterioration with severe restriction of daily activities

(4)

1.2 Increase of hospital admissions, (>  50% within 3 months, compared to previous periods)

No

(0)

1. As hospital admissions are used here as a proxy for disease severity and worsening trajectory, the purpose and type of hospital stay should be considered. Elective admissions should not be included, as they could be for routine work-up (e.g. sleep studies).

2. Instead of 3 months, admissions over the last 6 months is suggested to better capture medical instability for patients with predominantly longer prognoses and variable trajectories in PPC.

Yes

(3)

2.1 Treatment directed at the disease, (does not mean treatment of disease related complications, such as pain, dyspnoea or fatigue)

… is curative

(0)

1. To assess disease acuity based on potential treatment options, it may be helpful to qualify with an additional statement like “treatments directed at the disease, even if not administered”; as it was observed that many patients might not have received treatments, either due to unavailability, or they were declined by caregivers.

… controls disease and prolongs life with good quality of life

(1)

… does not cure or control but has a positive effect on quality of life

(2)

… does not control and has no effect on quality of life

(4)

2.2 Burden of treatment, (Burden means side effects of treatment and additional burdens such as stay in hospital in the patient’s or family’s view)

No or minimal burden or no treatment is envisioned

(0)

1. It may be helpful to qualify further “Burden of treatments, both disease-directed and symptom-directed”. Treatments that are not directed at the underlying disease should also be included for consideration, as they do exact a toll on the patient and caregiver (e.g. newly ventilator-dependent) [28,29,30,31,32].

2. Burden is often contingent on the intensity and caregiver skills required to administer treatment. Points of reference should be provided to ensure agreement between assessors for each level of burden (e.g. invasive treatments should be scored as high level of burden).

Low level of burden

(1)

Medium level of burden

(2)

High level of burden

(4)

3.1 Symptom intensity or difficulty of symptom control (over the last 4 weeks)

Patient is asymptomatic

(0)

1. Similar to Item 1.1, the two measured constructs (severity of symptoms and difficulty in management) should be scored separately, to account for differential variations.

2. The reference baseline should be extended to “within the past 3 months”, to maximise detection of symptom related issues.

Symptom(s) are mild and easy to control

(1)

Any symptom is moderate and controllable

(2)

Any symptom is severe or difficult to control (unplanned hospitalisation or outpatient visits, symptom crises)

(4)

3.2 Psychological distress of patient related to symptoms

Absent

(0)

1. While psychological distress is inherent in complex care [33, 34], it may be challenging to assess this accurately as these young children may struggle to articulate distress, are non-communicative, or cognitively impaired [33]. A proxy report – via either caregivers or the healthcare professional – is often required. If appropriate, this can be explicitly specified.

Mild

(1)

Moderate

(2)

Significant

(4)

3.3Psychological distress of parents or family related to symptoms and suffering of the child

Absent

(0)

1. To be accurately evaluated, psychological distress may require professional assessment. It may be difficult for most clinicians to score distress in caregivers objectively or ensure consistency between observers in that case.

Mild

(1)

Moderate

(2)

Significant

(4)

5.1 Estimated life expectancy

Several years

(0)

1. There is ambiguity to the terms ‘weeks’ and ‘months’ as it is in the original scale. For example, it is unclear whether children with a prognosis of three to 4 weeks should be scored 3 or 4. Life expectancies can be better clarified using defined cut-offs, e.g. “3 months to a year”, “3 weeks to 3 months”, “less than 3 weeks”.

Months to 1–2 years

(1)

Weeks to months Please skip 5.2

(3)

Days to weeks Please skip 5.2

(4)

5.2 “Would you be surprised if this child were to suddenly die in 6 months’ time?”

Yes

(0)

1. The word “suddenly” presents difficulty as it may be challenging to determine what changes in trajectories are considered sudden. It may exclude patients who die after slow deterioration, even within 6 months, if literally interpreted. Removing the term ensures that all patients who are at-risk of dying within 6 months are accounted for during scoring.

No

(2)