Today, Novo Nordisk presented new data detailing the impact hemophilia has on both caregivers and those living with the disease. Results from 2 separate studies presented at the International Society on Thrombosis and Haemostasis (ISTH) 2015 Congress in Toronto, Canada, showed many caregivers experience significant emotional stress and concern over the financial burden of treatment, and indicated a high prevalence of pain among people living with hemophilia. These findings provide insights into the quality of life associated with hemophilia and add to the body of knowledge uncovered in the company's Hemophilia Experiences, Results, and Opportunities (HERO) study, the largest-ever multinational, comprehensive study that aimed to increase understanding of psychosocial issues impacting people with hemophilia.
"These wide-ranging data uncover the tremendous burden hemophilia has on both patients and caregivers and reinforces the importance of an individualized treatment approach for this chronic disease," said David Cooper, MD, Senior Director, Medical Affairs, Novo Nordisk.
HEMOCAB™ Scale Pilot Test
Results from a pilot test of the "HEMOphilia associated CAregiver Burden" scale (HEMOCAB™) found that among the domains surveyed, emotional stress, financial burden, perception of child, and impact on the caregivers themselves were reported to be the most burdensome. The findings cite 74% of caregivers frequently feeling afraid that their child might get injured when they are not around to provide help. Additionally, 73% of caregivers expressed feelings of sadness about informing a child of what he/she can and cannot do due to his/her illness. Fifty-three percent of caregivers reported the cost of treating a child with hemophilia impacts family life. Importantly, the study found that caregiver burden is greatest among individuals caring for a child who has/had inhibitory antibodies against clotting factor replacement.1
The HEMOCAB™ questionnaire was provided to US caregivers of children with hemophilia with and without inhibitors younger than 22 years of age, with the objective to conduct a pilot test consisting of 13 domains to identify burdens associated with decreasing health-related quality of life. Forty caregivers were recruited, a majority of whom had a child with hemophilia A without inhibitors.1
Findings from initial analyses of the Pain, Functional Impairment and Quality of Life
(P-FiQ) study substantiated the high prevalence of pain in adults with hemophilia, with the majority of participants (85.2%) having reported they experienced acute and/or chronic (persistent) pain over the past 6 months.2 Data demonstrated pain and functional impairment were ubiquitous and impacted patients' daily activities, especially mobility and quality of life.3 The investigators validated patient-reported outcome (PRO) instruments through these data, noting the reliability of the methodology and highlighting its capacity to consistently evaluate pain and functional impairments.4
The P-FiQ study was provided to adult male patients with hemophilia (mild to severe) with a history of joint pain or bleeding to assess pain and functional impairment through 5 PRO instruments to provide insight to individualizing treatment plans. During routine visits over the course of a year, participants completed a pain history and 5 PROs. Of the 381 patients enrolled from October 2013 to October 2014, 164 completed the retest. Ongoing analysis of the complete study population will include individual question responses and assessment of different domains.2-4
"Pain and discomfort are significant challenges for people with hemophilia. These results emphasize the importance of providing comprehensive care and support beyond traditional therapy to people living with bleeding disorders," said Michael Recht, MD, PhD, Oregon Health Sciences University.
"We are committed to further research to better understand the unique and ongoing challenges patients and their loved ones face in managing the disease," said Natalia Holot, MD, Executive Director, Medical Affairs, Novo Nordisk.