More than half with hemophilia had gaps in prescribed factor treatment

More than half of people with hemophilia receiving prophylactic (preventive) factor replacement therapies at two treatment centers in Brazil did not collect enough medication to follow their prescribed treatment regimen, a real-world study has found.

This gap between “prescription and practice” was evident even though Brazil provides universal, free access to clotting factor concentrates through its public health system, suggesting that access alone does not ensure people can maintain their prescribed preventive treatment.

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Sept. 25, 2026 News by Marisa Horak, MS

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“These findings indicate that, although access to prophylaxis is well established within the Brazilian Health System, sustained execution of the therapeutic regimen remains a relevant clinical challenge,” the researchers wrote.

The study, “Gaps Between Prescription and Practice: Pharmacy-Based Measurement of Prophylaxis Adherence in Haemophilia,” was published in Haemophilia.

Hemophilia A and B are inherited bleeding disorders caused by a deficiency of clotting factor VIII (FVIII) or factor IX (FIX), respectively. Without enough of these proteins, blood does not clot properly, which can lead to prolonged or spontaneous bleeding.

Regular prophylaxis with FVIII or FIX replaces the missing clotting factor, helping prevent bleeds and the joint damage that repeated bleeds can cause. But keeping up with treatment can be challenging, particularly when it requires frequent intravenous (into-the-vein) infusions.

“Although Brazil provides universal and free access to clotting factor concentrates (CFC) through the public health system, real-world adherence remains insufficiently explored,” the researchers wrote.

To take a closer look, researchers in Brazil analyzed data from 224 people with hemophilia A or B who received care in 2024 at two specialized treatment centers, Hemocentro UNICAMP in São Paulo and HEMOES in Espírito Santo.

All had been receiving preventive FVIII or FIX treatment for at least one year. Nearly all (99.6%) were male, and the median age was 27. Most (78.6%) had hemophilia A. Severe disease was common, affecting about 85% of those with hemophilia A and 75% of those with hemophilia B.

Pharmacy records reveal gaps in prescribed treatment

In Brazil, factor replacement therapy is prescribed by the treating physician and dispensed by pharmacies within hemophilia treatment centers. Researchers compared the amount of factor each person was expected to use, based on the prescribed dose and infusion schedule, with the amount actually dispensed. Because the study measured pharmacy dispensing rather than directly observing infusions, the researchers noted that the records may not fully reflect how much treatment participants actually used.

People who had more than 85% of their prescribed factor dispensed were considered optimally adherent. Those who had 75%-85% dispensed were considered suboptimally adherent, while those who had less than 75% dispensed were classified as non-adherent.

Overall, more than half of participants (52.7%) were classified as non-adherent, 78 (34.8%) as optimally adherent, and 28 (12.5%) as suboptimally adherent.

The gap between “prescription and practice” was particularly evident among young adults. About two-thirds (66.7%) of those ages 20-39 were non-adherent, compared with 22.8% of children younger than 10. By contrast, nearly two-thirds (63.6%) of children younger than 10 had optimal adherence.

The researchers suggested that caregiver involvement may help explain the better adherence seen in young children. As people move through adolescence and early adulthood, greater independence, school and work demands, and feeling less at risk when they have no symptoms may make it harder to stay on schedule with treatment.

Treatment burden may also contribute. All participants were receiving conventional intravenous factor prophylaxis, which can require frequent infusions. The researchers noted that other studies have linked treatments that require less frequent administration with better adherence.

Adherence differed by treatment center

Adherence did not differ significantly between people with hemophilia A and B, despite differences in their treatment regimens. However, adherence did differ between the two treatment centers.

Non-adherence was more common at HEMOES than at UNICAMP — 60.3% versus 43.7%. The authors noted that at UNICAMP, “patients are actively reminded to collect their prophylactic factor concentrate,” a practice that may have contributed to better adherence there. They suggested that “structured interventions involving active participation of the multidisciplinary team” may help reduce gaps between prescribed and actual treatment.

Despite differences in adherence, the median annualized rates of total and joint bleeding were zero across all three adherence groups, with no significant differences among them. Individual results varied, however, and some people classified as adherent experienced multiple bleeds.

The researchers cautioned that the zero median bleeding rates should be interpreted carefully. Bleeding can also be influenced by disease severity, existing joint damage, physical activity, and differences in how each person’s body processes replacement factors.

“In summary, the results demonstrate that, even in a setting of broad access to prophylaxis, significant challenges related to treatment adherence persist,” the researchers wrote. “The routine incorporation of objective monitoring metrics, combined with multidisciplinary strategies particularly targeted at young adults, may contribute to optimizing clinical outcomes.”