Michelle Witkop

A national study of pain in the bleeding disorders community: a description of haemophilia pain

Summary.  The National Pain Study was a prospective, computer‐based, descriptive survey of the pain experience of persons with a bleeding disorder conducted in the United States over a 28 month period from 2007 to 2009. The aim of this study was to (i) determine the language used by patients to describe and differentiate acute and persistent pain, (ii) describe pharmacological and non‐pharmacological strategies utilized to control pain, (iii) assess the perceived effectiveness of current pain management on quality of life and, (iv) to determine who provides pain management to this population. One thousand, one hundred and four surveys were received. Only the responses of the 764 respondents who reported having hemophilia A or B were evaluated for this paper. Thirty nine percent of participants reported their pain was not well treated. The average acute pain score associated with a bleed reported was 5.97/10 while the average persistent pain score reported was 4.22/10. The most frequently reported word descriptors for acute pain were: throbbing, aching, sharp, tender and miserable. The most frequently reported word descriptors for persistent pain were aching, nagging, tiring, sharp, and tender. The most frequently reported pain strategies for acute and persistent pain included factor, rest, ice, elevation, and compression. Alcohol and illicit drugs were reportedly used to manage both acute pain as well as persistent pain. Primarily, short‐acting opioids and acetaminophen were reported to treat both acute and persistent pain. Hematologists and primary care providers provide the majority of pain management for persons with hemophilia (PWH). Quality of life (QOL) scores were lowest in the domains of pain, energy/fatigue and physical problems indicating disruption of QOL. This substantiates under‐recognition and under‐treatment of pain in the hemophilia population when combined with the 39% of respondents who felt their pain was not well treated and literature in the general pain population of wide spread under‐treatment of pain. Recommendations: The NPS is an initial step in recognizing the prevalence and description of pain in PWH. HTC providers should educate themselves in pain management techniques to better serve this population. Further research is necessary to develop specific pain management guidelines for the bleeding disorders population that include multimodal holistic treatment plans.

Assessment of acute and persistent pain management in patients with haemophilia

Summary.  A descriptive survey was conducted in Region V‐E of the United States to bridge the gap in available information on pain issues in the bleeding disorders population. The aim of this study was to a) determine language used by patients to describe and differentiate acute and persistent pain, b) describe pharmacological and non‐pharmacological strategies utilized to control pain, c) determine the providers of pain management to this population and d) evaluate quality of life incorporating the SF‐36 QOL tool. A total of 202 surveys were returned. For the purposes of this paper, it was decided to analyse only haemophilia data (n = 114). Average persistent daily pain levels were 5/10 (P < 0.001). The three most common word descriptors for both acute and persistent pain were the same ‐ achy, throbbing and tender; the most utilized pain medications were NSAIDs and acetaminophen. Factor replacement was used for what respondents described as acute pain management 79% of the time and for persistent pain management 38% of the time. Participants described acute and persistent pain with the same pain descriptors leading to the conclusion that patients have difficulty distinguishing between acute and persistent pain. This lack of differentiation was further displayed by the use of factor replacement to treat persistent pain associated with arthritic discomfort (38%) which would be viewed as inappropriate, as well as lack of factor replacement use by 21% of respondents who identified pain as from an acute bleed. Opportunities exist to improve pain management through patient and provider‐directed educational programs.

Better adherence to prescribed treatment regimen is related to less chronic pain among adolescents and young adults with moderate or severe haemophilia

Little data exist, especially for adolescent and young adult (AYA) persons with haemophilia (PWH), about the relationship between adherence to prescribed treatment regimen and chronic pain. We examined this relationship among PWH (moderate or severe) aged 13–25 via cross‐sectional survey. Adherence was assessed using the Validated Hemophilia Regimen Treatment Adherence Scale (VERITAS)‐Pro and VERITAS‐PRN for prophylactic and on‐demand participants respectively. VERITAS scores range from 24 (most adherent) to 120 (least adherent). Chronic pain was measured using the FPS‐R and was dichotomized as high for FPS‐R scores ≥4 and low for <4. Logistic regression models were constructed to assess factors associated with having high (vs. low) chronic pain. Of 80 AYA respondents (79 men), most had severe disease (91%), infused prophylactically (86%) and had haemophilia A (91%). Fifty‐one per cent were aged 13–17 and most were white (76%), non‐Hispanic (88%) and never married (93%). Chronic pain was reported as high for 35% of respondents. Mean VERITAS‐Pro scores for those with high and low chronic pain were 53.6 ± 12.3 vs. 47.4 ± 12.9, P = 0.05. VERITAS‐PRN scores were similar across chronic pain status. Logistic regression revealed that for each 10‐point reduction (i.e. increase in adherence) in the combined VERITAS (Pro and PRN) and VERITAS‐Pro scores there was a 35% (OR = 0.65; 95%CI = 0.44, 0.96; P = 0.03) and 39% (OR = 0.61; 95%CI = 0.39, 0.96; P = 0.03) reduction in odds of having high chronic pain respectively. Among AYA PWHs, better adherence was associated with significantly lower odds of having high chronic pain. Moreover, non‐whites were >4 times as likely as whites to report high chronic pain.

Associations of quality of life, pain, and self-reported arthritis with age, employment, bleed rate, and utilization of hemophilia treatment center and health care provider services: results in adults with hemophilia in the HERO study

Introduction Severe hemophilia and subsequent hemophilic arthropathy result in joint pain and impaired health-related quality of life (HRQoL). Assessment of HRQoL in persons with hemophilia (PWH), including underlying factors that drive HRQoL differences, is important in determining health care resource allocation and in making individualized clinical decisions. Aim To examine potential associations between HRQoL, pain interference, and self-reported arthritis and age, employment, activity, bleed frequency, and hemophilia treatment center and health care professional utilization. Methods PWH (age ≥18 years) from ten countries completed a 5-point Likert scale on pain interference over the previous 4 weeks, the EQ-5D-3L scale (mobility, usual activities, self-care, pain/discomfort, anxiety/depression) including a health-related visual analog scale (0–100, coded as an 11-point categorical response). Results Pain interference (extreme/a lot) was higher in PWH aged >40 years (31%) compared to those aged 31–40 years (27%) or ≤30 years (21%). In an analysis of eight countries with home treatment, PWH who reported EQ-5D mobility issues were less likely to be employed (53% vs 79%, with no mobility issues). Median annual bleed frequency increased with worsening EQ-5D pain or discomfort. The percentage of PWH with inhibitors reporting visual analog scale scores of 80–90–100 was lower (20%) than those without inhibitors (34%). Median bleed frequency increased with pain. Globally, nurse and social worker involvement increased with disability and pain; physiotherapist utilization was moderate regardless of the extent of disability or pain. Conclusion Increased disability and pain were associated with increased age, lower employment, higher reported bleed frequency, and lower HRQoL.

Self-reported prevalence, description and management of pain in adults with haemophilia: Methods, demographics and results from the Pain, Functional Impairment, and Quality of life (P-FiQ) study

Haemophilia is characterized by frequent haemarthrosis, leading to acute/chronic joint pain.

Treatment outcomes, quality of life, and impact of hemophilia on young adults (aged 18–30 years) with hemophilia

The Hemophilia Experiences, Results and Opportunities (HERO) initiative assessed psychosocial issues reported by people with moderate to severe hemophilia and was led by a multidisciplinary international advisory board. This analysis reports data from young adult respondents (aged 18–30 years), including both US and overall global (including US respondents) results, and investigates treatment outcomes, quality of life, and impacts of hemophilia on relationships. More young adults in HERO received prophylaxis than on‐demand treatment, although a majority reported not using factor products exactly as prescribed, and 50% of global respondents and 26% of US respondents reported issues with access to factor replacement therapy in the previous 5 years. Many young adults with hemophilia reported comorbidities, including bone/skeletal arthritis, chronic pain, and viral infections, and nearly half of young adults reported anxiety/depression. Most reported pain interference with daily activities in the past 4 weeks, although a majority reported participating in lower‐risk activities and approximately half in intermediate‐risk activities. Most young adults were very or quite satisfied with the support of partners/spouses, family, and friends, although roughly one‐third reported that hemophilia affected their ability to develop close relationships with a partner. A majority of young adults reported that hemophilia has had a negative impact on employment, and 62% of global respondents and 78% of US respondents were employed at least part‐time. Together these data highlight the psychosocial issues experienced by young adults with hemophilia and suggest that increased focus on these issues may improve comprehensive care during the transition to adulthood. Am. J. Hematol. 90:S3–S10, 2015.

Unmet needs in the transition to adulthood: 18- to 30-year-old people with hemophilia

Young adults with hemophilia face unique challenges during the transition to adulthood, including issues associated with switching from pediatric to adult hematology care, building mature interpersonal relationships, and establishing an independent career with an assurance of medical insurance coverage. A greater understanding of these challenges is essential for developing effective strategies to address the specific needs of this population. These challenges may be differentiated from those of older adults with hemophilia in large part because of more extensive childhood prophylaxis and safer factor products, resulting in fewer joint problems and lower rates of HIV and HCV infections. This analysis of the changing nature and unmet needs of todays young adults entering into adult hemophilia treatment centers, as well as potential strategies for optimally addressing these needs, was developed following roundtable discussions between patients, caregivers, hematologists, and other health care professionals participating in comprehensive care. Challenges identified among young adults with hemophilia include psychosocial issues related to maturity, personal responsibility, and increased independence, as well as concerns regarding when and with whom to share information about ones hemophilia, limited awareness of educational and financial resources, and a low perceived value of regular hematology care. The initiatives proposed herein highlight important opportunities for health care professionals at pediatric and adult hemophilia treatment centers, as well as national organizations, community groups, and career counselors, to address key unmet needs of this patient population. Am. J. Hematol. 90:S17–22, 2015.

Predictors of non-adherence to prescribed prophylactic clotting-factor treatment regimens among adolescent and young adults with a bleeding disorder

Adherence to clotting‐factor treatment regimens, especially among adolescents and young adults (AYAs), is under‐researched.

Medical co-morbidities of patients with haemophilia: pain, obesity and hepatitis C

Clinical care of patients with haemophilia (PWH) has progressed rapidly over the past decade. Current therapy has allowed patients with haemophilia to live longer and many patients are now experiencing the co‐morbidities of the general population. In this review article, we focus on three common diseases states that affect PWH: chronic pain, obesity and hepatitis C. Pain has been a co‐morbidity for many years and PWH often have unusual needs for chronic pain relief compared to the general population. Obesity is not only increasing in the general population but also in patients with hereditary bleeding disorders. The co‐morbidity of obesity not only causes increased pain progression and joint damage but also affects the dosing of factor concentrates. Finally, hepatitis C is known to have infected the majority of patients who received non‐virally inactivated pooled factor concentrates in the past. New treatment regimens have been developed that allow the nearly uniform cure of chronic hepatitis C with a short course of oral medications.

Impact of pain and functional impairment in US adults with haemophilia: Patient-reported outcomes and musculoskeletal evaluation in the pain, functional impairment and quality of life (P-FiQ) study

Standardized and disease‐specific patient‐reported outcome (PRO) instruments assessing pain, functional impairment and health‐related quality of life (HRQoL) in people with haemophilia (PWH) have been used in studies, but infrequently in comprehensive care settings for individual assessment or treatment planning.