There Is Much to Consider and Discuss in Previously Untreated Hemophilia

Treatment options have improved care, but also increased the need for patient education.

Advances in the treatment of hemophilia A and B mean that physicians and other health care providers need to be proactive and thorough when explaining disease management and treatment options to caregivers for young patients, according to a new review published in Therapeutic Advances in Hematology.

The article outlined some of the key considerations for physicians and discussed emerging treatment options.

In families with a history of hemophilia, physicians should initiate conversations with prospective parents even before pregnancy, if possible, said the study authors. Doctors can provide genetic counseling and give an overview of the investigations and options available during pregnancy.

“This will help known or potential carrier women and their partners to understand the implications of hemophilia and proactively prepare for the possibility of having a child with the condition,” they said.

Once a pregnancy occurs, tests can help inform patient and child care and physicians and be used to help plan for delivery, the authors added. Careful coordination between a patient's obstetrics team and the hemophilia team is critical, they added, in order to ensure all parties understand the birth plan and are prepared for potential complications.

Of course, some cases of hemophilia are sporadic, meaning there is no family history of the disease. In those cases, it is not possible to plan ahead, so the investigators said physicians will need to explain to caregivers how to recognize and treat bleeding and begin conversations about the range of therapeutic options now available. The authors said ideally patients would begin prophylaxis before age 2 years.

They then reviewed some of the treatment options available to infants and other patients with hemophilia. Clotting factor concentrates have been the “treatment of choice,” they said, and several concentrate products are now available. However, they noted that the therapy is burdensome.

“Clotting factor products require intravenous administration, and venous access is a particularly important consideration when treating small children with hemophilia,” they said.

The authors said newer concentrates have extended half-lives, which can reduce the treatment burden; however, they said it is important to carefully monitor patients to optimize treatment. Newer nonfactor therapies have also been investigated and may be appropriate options for some patients.

One of them is emicizumab (Hemlibra), a monoclonal antibody approved to treat hemophilia A. The authors said the drug works by mimicking the function of missing activated factor VIII and can serve as effective prophylaxis in patients with hemophilia A and inhibitors. In some cases, the therapy is also available for patients without inhibitors. One advantage of emicizumab is its subcutaneous administration subcutaneously. Still, the authors said there are not yet enough data to fully understand how the therapy works in the youngest patients. They said an ongoing clinical trial should help clear up some of the remaining questions.

The investigators noted that a number of other nonfactor treatments are also under investigation. Those include gene therapy, although the authors said that treatment is currently not an option for children.

The investigators concluded by emphasizing the importance of shared decision-making, even when such a model requires considerable patient education.

“The pathophysiology of the disease needs to be considered, as do the consequences of bleeding, together with the benefits and risks associated with the different options available for bleed prevention and management,” they said.

The investigators said providing easy access to care, support, and information will also help encourage patient adherence.


Astermark J, Blatný J, Königs C, Hermans C, Jiménez-Yuste V, Hart DP. Considerations for shared decision management in previously untreated patients with hemophilia A or B. Ther Adv Hematol. Published online April 17, 2023. doi:10.1177/20406207231165857

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