TORONTO—When children with hemophilia develop inhibitors, their caregivers shoulder a greater burden, according to a pilot study.
Researchers surveyed 40 subjects on the burden of caring for a child with hemophilia and found that inhibitor development significantly increased the burden of care.
But other factors—such as the number of bleeds a child had experienced in the last 12 months—had no significant impact.
Sylvia von Mackensen, PhD, of the University Medical Centre Hamburg-Eppendorf in Hamburg, Germany, and her colleagues presented these findings at the ISTH 2015 Congress (abstract PO256-WED).
The study was a pilot test of the HEMOCAB questionnaire, which consists of 59 questions mapped to 13 domains. Caregivers were asked to select the response that best qualified their burden and were scored on a scale of 0 to 100, with higher values corresponding with greater burden.
Forty caregivers completed the questionnaire. All of them had children with hemophilia (n=40), with or without inhibitors, who were younger than 22 years of age.
Three-quarters of the caregivers were mothers, 17.5% were fathers, and 7.5% were grandmothers. The mean age of caregivers was 39.32 ± 8.9 years (range, 27-66), and the mean age of the hemophilia patients was 10.98 ± 5.5 years (range, 1-21).
Most of the patients had hemophilia A (95%), and most had severe disease (77.5%). Six children (15%) had inhibitors. Overall, patients had experienced an average of 4.83 ±8.9 bleeds (range, 0-52) in the previous 12 months.
Most of the patients (88.5%) were receiving prophylaxis. Caregivers said they spent 8.69 ± 7.7 hours per month on infusion and 3.84 ± 6.7 hours (range, 0-30) per month traveling to hemophilia treatment facilities.
Size of burden
Caregivers said the most burdensome aspects of care were the caregivers’ perception of the child (38%), emotional stress (36%), financial burden (34%), and the impact of care on the caregiver (31%).
For nearly all of the domains assessed—emotional stress, personal sacrifice, medical management, work situation, etc.—a caregivers’ burden was significantly higher if a child had inhibitors. The only exception was school-related burden.
When the researchers analyzed the impact of other factors on care burden, they found that only inhibitor development had a significant impact.
There was no impact for orthopedic joint score, age of the caregiver, age of the child, time for infusion, time traveling to a hemophilia treatment center, the number of bleeds in the past 12 months, the number of children with hemophilia per household, home treatment, caregiver marital status, location, or caregiver education.
Frequency of burden
The HEMOCAB questionnaire also included scales assessing the frequency of burden. Dr von Mackensen and her colleagues presented data from these scales for the caregivers’ perception of their child, emotional stress, and finances.
Sixty percent of caregivers said they sometimes, often, or always feel their child’s condition is a difficult situation. Seventy-three percent of caregivers expressed feelings of sadness about informing their child of what he can and cannot do due to his illness.
Seventy-four percent of caregivers said they sometimes, often, or always feel afraid their child might get injured when they are not around to help. Forty-six percent of caregivers reported feeling afraid their child’s condition might worsen, and 36% said they feared their child might die from his condition.
Forty-six percent of caregivers said their child’s hemophilia sometimes, often, or always causes financial problems. And 33% of caregivers said that, at least sometimes, their family does not have enough money because of their child’s hemophilia.
HEMOCAB is a trademark of Novo Nordisk Health Care AG. Dr von Mackensen received a consulting fee from Novo Nordisk for developing the HEMOCAB questionnaire. Leonard A. Valentino, MD, of Rush University Medical Center in Chicago, Illinois, was involved in developing the questionnaire as well but did not participate in the pilot study.
The other researchers involved in the study have received funding/consulting fees from—or are employees of—Novo Nordisk, Baxter, Bayer, OctaPharma, CSL Behring, OPKO Health, and Selexys.