Key Clinical Summary: Integrating PROs & HRQoL for Personalized Sickle Cell Care

This is a micro-learning module summary of the SCD Education session which you can find here.

Before participating please read our CME and disclosure information which can be found here. This program was supported by an independent medical education grant from Novo Nordisk. This content is intended for US Healthcare Professionals only.

Introduction:

This educational summary outlines the critical role of patient-reported outcome measures (PROs) and health-related quality of life (HRQoL) assessments in personalizing care for individuals with sickle cell disease (SCD). Pain in SCD is deeply intertwined with a patient's HRQoL, affecting sleep, fatigue, and psychosocial and social health. Integrating validated PRO tools into routine practice allows clinicians to move beyond symptom management to a more holistic approach, enabling better risk stratification, shared decision-making, and justification for transformative therapies.

The Disproportionate Impact of SCD on Quality of Life:

Data clearly demonstrates that the burden of SCD on a patient's quality of life is severe, often more so than other serious chronic illnesses.

• Comparative HRQoL: On standardized scales, adults and children with SCD report a lower HRQoL than patients with cancer or cystic fibrosis
• Domain-Specific Impact: The PISCES project showed that on the SF-36 assessment, patients with SCD scored particularly poorly on domains of bodily pain, physical function, and general health.
• Pain and Sleep: In children and adolescents, pain and sleep have a significant, bidirectional relationship. High daily pain is linked to poor sleep quality, which in turn predicts higher pain the following day, creating a vicious cycle.

Validated PRO Tools for Comprehensive Assessment:

A suite of validated tools is available to systematically measure the diverse impacts of SCD. While traditional tools like the SF-36, Brief Pain Inventory (BPI), and Pittsburgh Sleep Quality Index (PSQI) are useful, newer systems offer more comprehensive and SCD-specific insights.

• PROMIS® (Patient-Reported Outcomes Measurement Information System): A widely used set of measures assessing domains like anger, anxiety, depression, fatigue, and pain impact across various chronic diseases.
• ASCQ-Me® (Adult Sickle Cell Quality of Life Measurement Information System): This is a powerful, SCD-specific tool developed with and for patients with SCD. It is designed to be comprehensive and evaluates physical, mental, and social well-being, and is unique in its ability to help assess the effectiveness of the healthcare delivery system itself.

From Data to Decision: Using PROs to Guide Care:

The primary value of PROs lies in their ability to translate patient experiences into actionable clinical insights. By interpreting PRO signals, clinicians can stratify risk and engage in meaningful, evidence-based shared decision-making.

• Signal: Worsening Pain Scores (e.g., ASCQ-Me Pain Impact, PROMIS Pain Interference).
• Clinical Interpretation: This indicates suboptimal pain control and high disease activity, placing the patient at elevated risk for emergency department visits and hospitalizations.
• Actionable Step: Initiate a discussion about treatment optimization. For example:
• "Your reports show pain is impacting your life more. Let's discuss our options, like optimizing your hydroxyurea dose or considering newer therapies, to find what best fits your goals.".
• Signal: Low Functional Scores & High Fatigue (e.g., PROMIS Physical Function, PROMIS Fatigue).
• Clinical Interpretation: This suggests significant functional impairment, a high overall disease burden, and possible progression of end-organ damage.
• Actionable Step: Use the data to justify advanced treatment. For example:
• "This level of impact on your daily life is a serious concern. It strengthens the clinical justification for considering transformative therapies, such as stem cell transplant or gene therapy. This data helps us make the case to payers that such a step is medically necessary.".
• Signal: Worsening Emotional Health Scores (e.g., ASCQ-Me Emotional Impact, PROMIS Depression).
• Clinical Interpretation: This identifies significant psychological comorbidity, which is a known barrier to treatment adherence and a contributor to poor overall HRQoL.
• Actionable Step: Facilitate mental health support. For example:
• "These feelings are a very real and understandable part of living with sickle cell. Let's talk about connecting you with our psychology or social work team to add another layer of support to your care plan.".

A 5-Step Workflow for Clinical Integration:

Successfully incorporating PROs into a busy clinical setting requires a systematic approach. A 5-step workflow can help overcome implementation barriers and ensure the data is used effectively.

1. Pre-Visit Data Collection: Use electronic PROs (ePROMs) to gather information before the appointment to improve data quality and reduce staff burden.
2. Automated Scoring & EHR Integration: Automate data scoring and entry into the electronic health record to ensure information is readily accessible at the point of care.
3. Point-of-Care Data Visualization: Use dashboards to display PRO results visually, allowing for rapid clinical assessment and trend analysis.
4. Clinician Pre-Consult Review: The clinician reviews the PRO dashboard before seeing the patient to identify key concerns and structure the visit's agenda around what matters most to the patient.
5. PRO-Informed Dialogue: Use the data to start a patient-centered conversation, validating their experience and building trust. For example:
6. "I see your fatigue has been worse over the last three months. Let's start by talking about that.".

Conclusion:

The integration of PROs is fundamental to modern, personalized SCD management. Studies confirm that worse psychosocial health—including higher stress, greater use of emotion-focused coping, and lower social support—is strongly associated with worse pain and functioning as measured by both PROMIS and ASCQ-Me. Conversely, better self-efficacy is linked to better mental health and lower pain frequency and severity. By systematically collecting and acting on PRO data, clinicians can better understand the global impact of SCD, foster patient-centered communication, and make more informed decisions that improve overall health and quality of life.