Hemlibra shows long-term efficacy and safety in hemophilia

Long-term prevention of bleeding episodes

Dr. Steven Pipe, Professor of Pediatric Hematology-Oncology at the University of Michigan C. S. Mott Children's Hospital

”Results from a 5-year study of 191 patients with hemophilia A... annual bleeding rate reduced to 0.8 episodes“ 

”Bleeding rate remained low even with high-intensity exercise... preventive therapy improves patients' daily lives”

 “The hemophilia patients’ only desire is to lead a normal life like everyone else.

 

Hemlibra (emicizumab) has been found to be effective in preventing bleeding and safe in long-term follow-up studies.

 

Moreover, it demonstrates clear bleeding prevention effects even during high-intensity exercise, significantly helping patients lead normal lives.” Dr.

 

Steven Pipe, Professor of Pediatric Hematology-Oncology at the University of Michigan C.

 

S.

 

Mott Children's Hospital in the United States, said so while participating at the 'HAVEN Symposium' held on the 9th at the Sofitel Ambassador Seoul Hotel in Songpa-gu, Seoul.

 

Dr.

 

Pipe visited Korea to present the results of long-term administration of Hemlibra in patients with hemophilia A.

 

He led the 'HAVEN3' and 'HAVEN4’ trials on Hemlibra.

 

HAVEN3 is a study involving 151 hemophilia A patients who received emicizumab at a dose of 1.5mg/kg weekly or 3mg/kg every two weeks.

 

HAVEN4 is a study involving 40 patients with hemophilia A who received emicizumab 6 mg every four weeks.

 

Five years of follow-up data on the 191 patients showed that the annual bleeding rate (ABR) was 2.0 during the initial treatment period (weeks 1–24).

 

At the long-term treatment stage (217–240 weeks), the annual bleeding rate fell to 0.8 episodes.

 

Joint bleeds, a common complication in hemophilia A patients, also decreased in the long-term follow-up.

 

The annual joint bleeding rate (AJBR) at the 217–240-week mark was 0.9 episodes.

 

The proportion of patients who did not experience any bleeding during Hemlibra treatment increased from 62.2% at weeks 1–24 to 78.8% at weeks 217–240.

 

Only 1 patient discontinued treatment over the five-year period.

 

This case was a mild adverse reaction, and no association with the drug was identified.

 

Twelve patients experienced inadequate bleeding control, and these patients continued treatment with an increased weekly dose of 3 mg/kg.

 

Dr.

 

Pipe highlighted the bleeding prevention effect of Hemlibra during various sports and physical activities.

 

Similar to healthy individuals, he explained that there is little concern about bleeding even with high-intensity physical activity.

 

The long-term follow-up results also showed that the annual bleeding rate (ABR) during sports and physical activities remained low at 0.91.

 

Dr.

 

Pipe said, “What hemophilia patients want most is ‘zero bleeding.’ They want to live their daily lives without any bleeding.

 

Especially, they want to engage in high-intensity physical activities, including intense exercise, without worrying about bleeding.” In this sense, Hemlibra showed long-term efficacy as a preventive therapy.

 

For example, at our hospital, 80% of patients diagnosed with hemophilia in childhood are currently receiving Hemlibra, and the drug shows definite bleeding prevention effects even during high-intensity exercise.” He also explained that the fact that Hemlibra maintains a higher level of clotting factor concentration for a longer period compared to existing treatments contributes to improving the patients' quality of life.

 

Dr.

 

Pipe said, “With previous medications, it was difficult to maintain consistent concentrations throughout the day, so patients had to take additional doses before intense exercise, which was inconvenient.

 

In contrast, Hemlibra maintains consistent concentration levels, allowing patients to live their daily lives without such inconveniences.” Dr.

 

Pipe plans to expand research on the long-term effects of Hemlibra in infants.

 

He is conducting a long-term observational study (HAVEN 7) on the joint damage prevention effects of Hemlibra prophylaxis in 55 infants under one year of age with severe hemophilia A who have not developed antibodies to Hemlibra.

 

R.

 

Pipe stated, “Based on the results so far, infants receiving Hemlibra also exhibit a low annual bleeding rate.

 

Even when bleeding occurs, it typically presents with traumatic bleeding patterns similar to those observed in infants of the same age.

 

This is why we anticipate that Hemlibra will continue to demonstrate high bleeding prevention efficacy in the long term.”