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Promis bundles

Below are PROMIS Pediatric domain bundles that are commonly considered fit-for-purpose across three overlapping disease areas: pediatric neurology, chronic pain, and rare disease. Each bundle is structured to balance measurement precision, respondent burden, and regulatory defensibility, and is suitable for clinical trials or longitudinal cohorts.

I indicate Core (recommended for most studies) versus Optional (condition-specific or exploratory) domains, and note implementation considerations (CAT vs short form).

1. PROMIS Pediatric – Neurology-Focused Bundle

(e.g., epilepsy, migraine, neuromuscular disorders, neuroinflammatory disease)

Core Domains

  1. Physical Function – Mobility
  2. Captures gait, endurance, and motor limitations
  3. Highly responsive in neuromuscular and CNS disorders
  4. Fatigue
  5. One of the most sensitive domains in pediatric neurology
  6. Strong associations with disease activity and treatment burden
  7. Pain Interference
  8. Preferable to pain intensity alone
  9. Captures functional impact of headaches, neuropathic pain, spasticity
  10. Anxiety
  11. Common across epilepsy, migraine, chronic neurologic disease
  12. Responsive to both disease control and psychosocial interventions
  13. Peer Relationships
  14. Captures participation and social integration, often affected even when physical symptoms are mild

Optional / Condition-Specific

  • Depressive Symptoms – if mood disorders are prevalent or treatment-relevant
  • Upper Extremity Function – for disorders affecting fine motor control
  • Cognitive Function (Pediatric v2) – if cognitive complaints are central (note: newer and less widely deployed)

Implementation Notes

  • CAT preferred for Fatigue, Pain Interference, Anxiety
  • Short forms acceptable for Physical Function if CAT is not feasible
  • Suitable as secondary endpoints in drug or device trials

2. PROMIS Pediatric – Chronic Pain–Centered Bundle

(e.g., primary headache disorders, CRPS, sickle cell pain, juvenile fibromyalgia)

Core Domains

  1. Pain Interference
  2. Primary outcome candidate in many pain trials
  3. More informative than intensity alone
  4. Pain Intensity
  5. Single-item numeric scale
  6. Useful anchor for clinical interpretation
  7. Fatigue
  8. Strong mediator between pain, disability, and quality of life
  9. Physical Function – Mobility
  10. Sensitive to activity avoidance and deconditioning
  11. Depressive Symptoms
  12. Strongly associated with pain chronicity and disability
  13. Anxiety
  14. Particularly relevant in headache and centralized pain syndromes

Optional / Adjunctive

  • Sleep Disturbance – if sleep is a hypothesized mechanism
  • Anger – sometimes relevant in adolescents with chronic pain
  • Peer Relationships – if social withdrawal is a concern

Implementation Notes

  • CAT is strongly recommended for Pain Interference and Fatigue
  • Bundle is appropriate for primary or co-primary endpoints, depending on intervention
  • Well aligned with NIH and FDA patient-focused drug development guidance

3. PROMIS Pediatric – Rare Disease / Multisystem Bundle

(e.g., metabolic, genetic, inflammatory, ultra-rare disorders)

Core Domains

  1. Global Health (Pediatric Global Health 7 or 10)
  2. Provides a broad anchor for overall well-being
  3. Useful when disease manifestations are heterogeneous
  4. Physical Function – Mobility
  5. High face validity across many rare diseases
  6. Fatigue
  7. Consistently elevated across rare disease populations
  8. Pain Interference
  9. Captures burden even when pain is not the primary symptom
  10. Anxiety
  11. Reflects uncertainty, disease unpredictability, and treatment burden

Optional / Customizable

  • Upper Extremity Function – if relevant to disease phenotype
  • Depressive Symptoms – if psychosocial burden is substantial
  • Peer Relationships – for conditions affecting school attendance or visibility
  • Family Relationships – sometimes useful in rare pediatric conditions

Implementation Notes

  • Global Health + 3–4 domains is often optimal to limit burden
  • Particularly suitable for ** small samples , ** natural history studies , and platform trials
  • Domains can be justified as clinically meaningful regardless of molecular heterogeneity

Cross-Cutting Design Guidance (Important)

  • Age range: Self-report (8–17), Parent proxy (5–17) — predefine transition rules
  • Scoring: Use T-scores; document directionality per domain
  • Change thresholds: Pre-specify MID assumptions (commonly 2–5 points)
  • Multiplicity: Avoid over-interpretation of multiple domains without hierarchy
  • EDC: PROMIS CAT integrates cleanly with REDCap and other modern systems

Summary Recommendation

If I had to recommend one robust default bundle for a pediatric neurology or rare-disease trial:

Fatigue + Pain Interference + Physical Function (Mobility) + Anxiety + Peer Relationships ,

with Global Health added when overall well-being is a key objective.

If helpful, I can next:

  • Draft SAP-ready endpoint language for one of these bundles
  • Recommend primary vs secondary endpoint hierarchies
  • Tailor the bundle to a specific condition (e.g., pediatric migraine, epilepsy, neuromuscular disease, autonomic disorders)
  • Align the bundle with regulatory or HTA expectations (FDA, EMA, CADTH)