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39 Impact of Pain and Functional Impairment in US Adult People with Hemophilia (PWH): Patient-Reported Outcomes and Musculoskeletal Evaluation in the Pain, Functional Impairment, and Quality of Life (P-FiQ) Study

Health Services and Outcomes Research – Non-Malignant Conditions
Program: Oral and Poster Abstracts
Type: Oral
Session: 901. Health Services and Outcomes Research – Non-Malignant Conditions: Health Outcomes in Inherited and Acquired Bleeding Disorders
Saturday, December 5, 2015: 8:00 AM
W230, Level 2 (Orange County Convention Center)

Christine L Kempton, MD, MSc1, Michael Recht, MD, PhD2*, Anne Neff, MD3, Michael Wang, MD4, Tyler W. Buckner, MD, MSc5, Amit Soni, MD6*, Doris Quon, MD, PhD7, Michelle Witkop, DNP, FNP-BC8*, Lisa Boggio, MD9 and David L Cooper, MD, MBA10*

1Emory University, Atlanta, GA
2The Hemophilia Center, Oregon Health & Science University, Portland, OR
3Cleveland Clinic, Cleveland, OH
4University of Colorado School of Medicine, Aurora, CO
5Divisions of Hematology and Pediatric Hematology/Oncology, Hemophilia and Thrombosis Center, University of Colorado School of Medicine, Aurora, CO
6Children's Hospital of Orange County, Orange, CA
7Orthopaedic Hemophilia Treatment Center, Los Angeles, CA
8Munson Medical Center, Traverse city, MI
9Rush University Medical Center, Chicago, IL
10Novo Nordisk Inc, Plainsboro, NJ

Introduction: Pain and functional impairment associated with joint disease are major problems affecting adults with congenital hemophilia. Various standardized and disease-specific patient-reported outcome (PRO) instruments have been used in clinical studies of PWH or other diseases, but the use of these tools in the hemophilia comprehensive care setting for individual patient assessment or overall outcome tracking is limited and inconsistent. P-FiQ was designed to assess the impact of pain on functional impairment and quality of life (QoL) in adult PWH.

Objectives: To assess pain and functional impairment in nonbleeding adult PWH through 5 PRO instruments and a clinical joint health evaluation.

Methods: Adult males with mild to severe hemophilia and a history of joint pain or bleeding were enrolled from 15 US sites between October 2013 and October 2014. During routine clinical care visits, participants completed a pain history and 5 PROs (EQ-5D-5L with visual analog scale [VAS], Brief Pain Inventory v2 Short Form [BPI], International Physical Activity Questionnaire [IPAQ], SF-36v2, Hemophilia Activities List [HAL]) and underwent a Hemophilia Joint Health Score v2.1 (HJHS) evaluation completed by a trained physical therapist.

Results: Overall 381 patients were enrolled, with a median age of 34.0 years. PRO and HJHS summary scores are presented in the table below. On EQ-5D-5L, most participants reported problems with mobility, usual activities, and pain/discomfort. On BPI, median worst pain was 6.0, least pain 1.0, average pain 3.0, and current pain 2.0. Ankles were most frequently reported as the joints with the most pain (37.4%), followed by knees (23.7%) and elbows (18.9%). On IPAQ, approximately half of participants (51.0%) reported no physical activity in the prior week. Median SF-36v2 subscores were lower for the 4 physical health domains than for the 4 mental health domains. Among HAL domains, self-care was the least impacted (median, 100.0) and functions of the legs (median, 66.7) and lying/sitting/kneeling/standing (median, 67.5) were the most impacted. On HJHS, elbow, knee, and ankle scores did not differ appreciably (median, 4.0, 4.0, and 6.0, respectively).

 

Assessment Tool

Median (Q1, Q3)

Range

EQ-5D-5L

VAS

Health index

 

80.0 (66.0, 90.0)

0.796 (0.678, 0.861)

 

0 to 100a

-0.11 to 1.0a

BPI

Pain severity

Pain interference

 

3.3 (1.3, 5.0)

2.7 (0.6, 5.4)

 

0 to 10b

 

IPAQ

Total physical activity

 

530.2 (264.0, 1039.5)

Metabolic equivalents of task (MET)/minutes per week:

Walking = 3.3 METs

Moderate activities = 4.0 METs

Vigorous activities = 8.0 METs

SF-36v2

Physical functioning

Role physical

Bodily pain

General health

Vitality

Social functioning

Role emotional

Mental health

Physical health summary

Mental health summary

Overall health

 

44.4 (29.7, 52.8)

44.6 (32.4, 56.9)

41.8 (37.2, 51.1)

45.8 (35.3, 52.9)

49.0 (42.7, 55.2)

45.6 (34.9, 56.4)

55.9 (36.4, 55.9)

52.8 (41.6, 58.5)

39.2 (29.5, 49.4)

50.7 (41.4, 55.7)

3.0 (2.0, 4.0)

 

0 to 100a

 

 

 

 

 

 

 

 

 

1 to 5a

HAL

Upper extremity activities

Basic lower extremity activities

Complex lower extremity activities

Overall sum score

 

88.9 (73.3, 97.8)

73.3 (46.7, 96.7)

55.6 (30.0, 88.9)

76.6 (58.0, 94.3)

 

0 to 100a

 

HJHS

Elbow

Knee

Ankle

Global gait

Total score

 

4.0 (0.0, 11.0)

4.0 (0.0, 10.0)

6.0 (1.0, 15.0)

3.0 (0.0, 4.0)

19.5 (6.0, 36.0)

 

0 to 40c

 

 

 

0 to 124c

aHigher scores indicate better QoL or functional status

bLower scores indicate less pain severity

cLower scores indicate better function

Conclusions: Results of this analysis demonstrated challenges of lower extremity pain and functional impairment in US adult PWH. Pain was frequently observed, and it impacted physical function and quality of life across PROs and HJHS. Further analyses are underway to correlate assessments of pain and function across different PROs and with the exam-based HJHS.

Disclosures: Kempton: CSL Behring: Consultancy ; Biogen: Consultancy ; Baxter: Consultancy . Recht: Baxalta: Research Funding ; Kedrion: Consultancy . Neff: Novo Nordisk: Other: Advisory Board ; Kedrion: Other: Advisory Board . Wang: Biogen: Membership on an entity’s Board of Directors or advisory committees ; CSL Behring: Membership on an entity’s Board of Directors or advisory committees ; Novo Nordisk: Membership on an entity’s Board of Directors or advisory committees ; Baxalta: Membership on an entity’s Board of Directors or advisory committees . Buckner: Novo Nordisk: Consultancy ; Baxalta, Inc. US: Consultancy . Soni: Bayer: Other: member of the Global Emerging HEmophilia Panel (GEHEP) ; Novo Nordisk: Speakers Bureau . Quon: Bayer: Other: Advisory Board ; Grifols: Speakers Bureau ; Novo Nordisk: Other: Advisory Board , Speakers Bureau ; Biogen: Other: Advisory Board , Speakers Bureau ; Baxter: Other: Advisory Board , Speakers Bureau . Witkop: Pfizer: Other: Advisory Board , Research Funding ; Baxter Bioscience: Other: Advisory Board ; Novo Nordisk: Other: Advisory Board , Speakers Bureau . Boggio: Baxter: Consultancy , Research Funding ; OctaPharma: Consultancy , Research Funding ; OPKO: Research Funding ; CSL Behring: Consultancy , Research Funding ; Novo Nordisk: Consultancy , Research Funding ; Selexys: Research Funding ; Bayer: Consultancy , Research Funding . Cooper: Novo Nordisk: Employment .

*signifies non-member of ASH