Study Examines Pain and Quality of Life in Hemophilia Patients

Findings may help individualize care and optimize patient well-being.

Newly validated patient-reported outcomes (PROs) in hemophilia correlate well with social and demographic data, according to a recent study. Could measuring PROs during clinical encounters help assess the impact of patient characteristics on important health outcomes?

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Researchers say yes, these findings could help individualize care and improve outcomes. Using linear and logistic regression models to analyze data from the Pain, Functional Impairment and Quality of Life (P-FiQ) study, researchers found a correlation between PROs and social/demographic data, such as educational level and employment status. Results of the study were presented at the 2016 American Society of Hematology Annual Meeting in San Diego.

Hemophilia-associated morbidity — including bone and joint problems, associated pain and functional impairment, and related impact on quality of life (QoL) — has been catalogued in the medical literature. What this study adds is a rigorous validation of several self-reported assessment tools that evaluate chronic pain, activity level and QoL based on health-related issues.

“These data help us to tease out who to target for more specific interventions, questions or probing, and who are more likely to have problems and perhaps less likely to report them or know how to voice those concerns,” says Anne Neff, MD, a specialist in hemophilia and other non-malignant blood disorders who was involved in the study at Vanderbilt University prior to joining the Cleveland Clinic staff.

Study population

The P-FiQ study enrolled 381 men with hemophilia (types A and B, and all severities) at 15 sites around the United States. Median age was 34 years, though differing hemophilia treatment eras were represented, as evidenced by the rates of comorbidities and viral infection: cardiovascular disease, 19 percent; HIV, 16 percent; and HCV, 32 percent. Depression was documented in 19 percent. A majority had severe hemophilia and half reported a history of joint procedures; over the previous six months, 85 percent experienced pain and 67 percent had restrictions in school/work or recreational activities.

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Patient-reported outcomes

During routine visits, patients completed a pain history and five patient-reported questionnaires:

  • EQ-5D-5L with visual analog scale (VAS): Measures health-related quality of life with five dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression)
  • Brief Pain Inventory v2 Short Form (BPI): Assesses the severity of pain and its impact on functioning.
  • International Physical Activity Questionnaire (IPAQ): Tool for surveillance of physical activity.
  • SF-36v2: Quality of life questionnaire that asks 36 questions to measure functional health and well-being from the patient’s point of view.
  • Hemophilia Activities List (HAL): Tool that measures the impact of hemophilia on self-perceived abilities.

Findings and ramifications

Not surprisingly to researchers, increased pain and decreased function were associated with older age, having the disease for a longer period of time and having a history of joint procedures.

Lack of college education, however, was one previously unexplored characteristic that was associated with poor functioning, increased pain and decreased quality of life, potentially indicating a patient subpopulation that might benefit from dedicated assistance from the treating physician, according to Dr. Neff.

Other factors associated with suboptimal PROs included unemployment, viral disease (HIV, hepatitis C), comorbidities (diabetes, cardiovascular disease, depression, anxiety), and severe hemophilia.

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In short, this set of tools for patient self-assessment is now validated in the hemophilia population, and the information provided may be useful in determining an individual patient’s needs, Dr. Neff states.

Takeaways for clinicians

“I think one of the main takeaways is that physicians should probe and ask patients about these particular problems,” Dr. Neff says. “It’s so easy during the clinic visit to focus solely on the medical aspects and not delve further into how the disease is affecting patients’ lives and what we might be able to do to improve their quality of life.”

For hemophilia physicians, this study may also offer a glimpse into the future. For instance, younger patients in this study had fewer joint problems, partially attributable to the younger age, but also reflecting the newer treatment era. Today, patients are prescribed clotting factor prophylaxis from a very early age, which helps prevent joint damage. Preventing joint damage prevents pain, increases QoL and decreases disability. “We also need to help patients understand why prevention is so important and why they need to stick with the treatment,” Dr. Neff says.

“The old adage ‘an ounce of prevention is worth a pound of cure’ is very evident in a study like this,” she concludes.