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Prophylactic Tx Stems Bleeds in Hemophilia A with Inhibitors

Prophylactic Tx Stems Bleeds in Hemophilia A with Inhibitors

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Action Points

  • Note that this study was published as an abstract and presented at a conference. These data and conclusions should be considered to be preliminary until published in a peer-reviewed journal.
  • Emicizumab (Hemlibra) prophylaxis prevented or significantly reduced bleeds, and was well tolerated, in pediatric patients with hemophilia A with factor VIII inhibitors.
  • In November 2017, the FDA approved emicizumab for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients with hemophilia A with factor VIII inhibitors.

ATLANTA – Emicizumab (Hemlibra) prophylaxis prevented or significantly reduced bleeds, and was well tolerated, in pediatric patients with hemophilia A with factor VIII inhibitors, researchers reported here.

After a median of 38.1 weeks, 94.7% of 53 children in the study had zero treated bleeds, according to Guy Young, MD, of the Children’s Hospital of Los Angeles, and colleagues.

Overall, 41 bleeds, which were mostly minor bruising episodes that did not require treatment, were reported among 15 of 23 patients followed for at least 12 weeks, Young said at a press conference at the American Society of Hematology (ASH) meeting. Three of those bleeds required treatment, he added.

Young said the annualized bleeding rate was reduced 99%, to 0.2 bleeds per year for the entire population in the trial, compared with the rate of bleeds that occurred in the patients prior to treatment with emicizumab. The calculated annualized bleeding rate (ABR) before subcutaneous administration of emicizumab was as high as 30 bleeds per year among some of the children in the study.

“Emicizumab, a bispecific humanized monoclonal antibody administered subcutaneously, bridges FIXa and FX to restore the function of missing FVIIIa,” the authors explained.

In November 2017, the FDA approved emicizumab for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients with hemophilia A with factor VIII inhibitors. Approval was based on data from HAVEN 1, an adult and adolescent trial, and HAVEN 2, a pediatric trial.

HAVEN 1 was a randomized, multicenter, open-label, phase III trial in 109 adult and adolescent males (ages 12-75 years; weight >40 kg) with hemophilia A with FVIII inhibitors who previously received either episodic (on-demand) or prophylactic treatment with bypassing agents.

Interim results of the HAVEN 2 trial presented at the 2017 International Society on Thrombosis and Haemostatis meeting. The results in 20 patients, ages 2-12 years, showed that subcutaneous, once-weekly emicizumab prophylaxis successfully prevented or reduced bleeds, provided clinically meaningful reductions in ABR versus prior bypassing agent (BPA) treatment, and was well tolerated.

The current findings are from 60 male children with a median age of 7 years. Three children ages >12 were enrolled because they weighed less than 40 kg. The vast majority (95%) of the children were diagnosed with severe hemophilia A, and they averaged six bleeds in the previous 24 weeks.

A total of 65 bleeds were reported in 20 patients, with eight occurring in a joint, two occurring in a muscle, and 55 classified as “other.” Of the latter, 40.0% were spontaneous, 55.4% were traumatic, and 4.6% were because of procedure/surgery, the authors explained.

Young told MedPage Today that all the patients kept bleeding diaries in which they recorded any kind of a bleed, including bruises that might have occurred in a fall. “They would record bleed, but they would also note that they didn’t treat that bleed. So the number of total bleeds will always exceed the number of treated bleeds,” he said.

When bleeds need treatment the patients generally use BPA to stop the bleeding, he said. “None of these bleeds became any sort of problem for the patients. Those bleeds were treated just the way they would if they were not on emicizumab, and they responded.”

Young noted that about 30% of patients who are treated with factor VIII for hemophilia developed neutralizing alloantibodies against the factor concentrate, and that can increase morbidity, as well as decrease health-related quality of life.

Patients with inhibitors have to undergo immune tolerance induction or use BPA. These treatments include the need for frequent intravenous dosing and often requiring emplacement of central lines which can lead to adverse events, such as infections and thrombosis.

Young said that in a proportion of these patients, immune tolerance fails to work, so that “any point in time in the United States, about 8% to 10% of people with hemophilia A have inhibitors. It is for these patients that we really need other treatments. They are often young people in their teens or early 20s. And these patients do not have other options.”

ASH press conference moderator Margaret Ragni, MD, of the University of Pittsburgh, said, “This is an exciting time in the management of these patients, not only in dealing with clotting issues but also in improvement of the quality of their life because of these advancements.”

“This study offers the potential to change the standard of care for both children with hemophilia A,” she noted. “In the study…95% of the patients experienced no bleeding events requiring treatment. This is outstanding and unusual. It suggests the drug is highly effective in preventing bleeding associated with hemophilia A.”

The trial was supported by Genentech/Roche.

Young disclosed relevant relationships with CSL Behring and Novo Nordisk.

Ragni disclosed relevant relationships with Alynlam, Bayer, Bioverativ, Biomarin, Genentech/Roche, MOGAM, Novo Nordisk, Sangamo, Shire and SPARK.

  • Reviewed by
    Robert Jasmer, MD Associate Clinical Professor of Medicine, University of California, San Francisco and Dorothy Caputo, MA, BSN, RN, Nurse Planner


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