Disease Severity and Age, Not Inhibitors Alone, Influence Quality of Life in Patients with Hemophilia

Severity of disease and patient age, not the presence of an inhibitor alone, influenced the health-related quality of life (HRQoL) of patients with hemophilia, according to the results of a new study published in SAGE Open Medicine.

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Hemophilia is most often treated with a coagulation factor replacement therapy, which has shown excellent efficacy with the use of recombinant factors. However, a challenge that has emerged in some patients is the development of inhibitory alloantibodies that compromise the mainstay of treatment. They can make replacement therapy fail in 25%-30% of severe hemophilia cases and can cause pain and disability in addition to higher mortality and morbidity.

Though treatments have been developed to treat patients with inhibitors, the high cost of treatment prevented many patients from achieving standard care and management, leading to an increased physical and emotional burden and reduced quality of life (QoL).

The investigators—based on the reality of sanctions and poor access to medication in Iran, as well as a previously conducted study from Bastani et al. that compared HRQoL in Iranian patients who had severe hemophilia with inhibitor and those without it—designed a study to compare the HRQoL in a group of Iranian patients with hemophilia A with and without inhibitor using the 36 item Short Form (SF-36) questionnaire.

Inclusion criteria for the study were male patients with hemophilia. All patients with hemophilia with inhibitors (n = 11) were referred to hemophilia centers affiliated with Shiraz of Zahedan University of Medical Sciences were included during the 6-month study period. For each patient with inhibitor, 3 patients without were chosen, eventually totaling 30 patients without inhibitor.

Demographic and clinical characteristics of the patients show a mean age of 36.9 +/- 13.2 years and that approximately half of the patients (51.2%) had severe hemophilia A, whereas 26.8% were inhibitor-positive. All patients included in the study had a severe version of the disease, and 9 patients (22%) had at least 1 type of blood-borne infection.

Patients who had severe hemophilia showed significantly lower scores in physical fitness (PF) (p < 0.001), physical health (PH) dimension (p = 0.018), and total SF-36 score (p = 0.031) than those who had mild and moderate hemophilia, the results of the study show.

Additionally, PF, mental health (MH) dimension, and total SF-36 scores were significantly lower in the inhibitor-positive group in comparison to inhibitor-negative patients (p < 0.001, p = 0.0045, p = 0.035 respectively).

The researchers conducted a multiple linear regression analysis that considered age, disease severity, and inhibitor as independent variables and HRQoL scores as dependent variables. The results of the analysis indicated that the presence of inhibitor alone was not an independent influencing factor on HRQoL in hemophilia patients.

Concurrently, disease severity and age were shown to be the independent influential factors on HRQoL of the patients. Severe type of hemophilia had a negative significant relationship with scores in PF (p = 0.001), role physical (RP) (p = 0.015), general health (p = 0.006), PH dimension (p = 0.006) and marginally in total SF-36 score (p = 0.054), the investigators found.

Some limitations observed in the study were the lack of a control group and the small number of hemophilia patients with inhibitor due to the rarity of inhibitor development complications. Further, QoL can depend on several factors that can vary between countries, which could make accurate comparisons between different countries difficult.

Reduced HRQoL was also observed in hemophilia patients with higher annual bleeding rate, blood-borne infections, and in unemployed patients, the study authors found.

“Precise attention should be considered in the prevention and management of bleeding symptoms in these patients. On the other hand, improving the social environment of these patients by providing educational facilities and creating a suitable job situation can improve the physical and MH of these patients,” the investigators concluded.

Reference

Haghpanah S, Naderi M, Kamalian S, et al. The impact of inhibitors on the quality of life in patients with hemophilia. SAGE Open Med. 2023;11. doi:10.1177/20503121231182284