Great Attention Needed in Care of Women and Girls With Hemophilia

Although hemophilia treatments are generally effective, women and girls with this disease have specific medical challenges, such as excessive menstrual bleeding, that must be addressed for better clinical care.

A study evaluated the symptoms, bleeding management, and clinical outcomes related to menstruation, childbirth, dental procedures, surgeries, and other bleeding events among female patients visiting hemophilia treatment centers (HTCs) in the U.S.

Women and girls with hemophilia, a disorder more common to boys and men, is a term used to describe females who are disease carriers — because they have one gene copy with disease-causing mutations — with evident symptoms due to low levels of the relevant clotting factor.

The study, “Women and girls with haemophilia and bleeding tendencies: Outcomes related to menstruation, pregnancy, surgery and other bleeding episodes from a retrospective chart review,” was published in the journal Haemophilia.

Beyond concerns common to all hemophilia patients, women and girls with hemophilia (WGH) also experience issues related to reproductive health. These include heavier and prolonged bleeding during menstruation, as well as during and after childbirth.

Current treatment guidelines, however, reflect a general lack of knowledge regarding bleeding management specific to female patients.

A team of researchers reviewed medical records of patients being treated at three pediatric and adult HTCs in the U.S. between April 2012 and November 2018.

They sought to describe the characteristics of WGH, and evaluate their bleeding management and clinical outcomes during spontaneous bleeding episodes both particular to girls and women, and those particular to all with this disease.

Among the 47 patients identified, 37 had factor VIII (FVIII) deficiency (hemophilia A) and 10 had FIX deficiency (hemophilia B).

Median age at diagnosis was 25.3 for those with hemophilia A and 5.7 for those with hemophilia B, while median age at first HTC visit was 28.3 for the hemophilia A group, and 6.7 for hemophilia B patients. A “gap”  appeared to exist between diagnosis and the start of specialty care, the researchers noted.

Most (37 patients, or 79%) had mild hemophilia, while 4% had moderate and 2% had severe forms. Three patients (6%) had normal factor levels, while those for the remaining patients were unknown.

Family history or genetic counseling, spontaneous or traumatic bleeds, and heavy menstrual bleeding were the three most common reasons for hemophilia A patients to make a first HTC visit.

Among those with hemophilia B, a family history or genetic counseling, treatment for a family member, and upcoming surgery or dental procedures were main reasons for a first visit.

Of those whose first HTC visit was for a spontaneous or traumatic bleed, 10 (59%) arrived for easy bruising, six (35%) came for nosebleeds, five (29%) had joint bleeds, four (24%) experienced excessive dental bleeding. Another four had other bleeds.

Among these 47 patients, 38 (81%) had begun menstruating and 14 of them — 11 with hemophilia A and three with hemophilia B — were treated for heavy menstrual bleeding. Both groups experienced a median number of seven days of bleeding per month, and a median of three days of heavy bleeding.

Antifibrinolytics — medicines that promote blood clotting — were the most frequently prescribed medications to women and girls with heavy menstrual bleeds. Bleeding stopped or was controlled in six patients, while the other eight (seven with hemophilia A and one with hemophilia B) continued to experience worse-than-expected menstrual bleeding.

Pregnancies were reported in 25 patients, accounting for 49 births. Of these, data on the most recent delivery were available for seven, and all were live births. Three mothers with hemophilia A and one with hemophilia B received clotting factor concentrates either before or during delivery, and two needed blood transfusions following delivery.

Bleeding in women given factor concentrates before or during delivery often fell to levels expected for a normal delivery. Of those treated with factor concentrates within six weeks of giving birth, one stopped bleeding, two experienced normal postpartum bleeding, and two reported bleeding that was worse than expected.

Twenty women — 15 with hemophilia A and five with hemophilia B — had surgeries and/or major dental procedures. Five were hospitalized following surgery for a median stay of two days. Factor concentrates were the most commonly prescribed medications before or during surgery, while FVIII concentrates and antifibrinolytics were those most frequently administered in the two weeks following surgery.

These treatments generally controlled bleeds as expected, stopping or reducing them within two weeks of surgery.

Five hemophilia B patients and 18 with hemophilia A reported spontaneous, traumatic, or joint bleeds. Traumatic bleeds were the most common, occurring in 44% cases. Factor concentrates were the most commonly administered type of medication for these cases and generally reduced bleeding.

Overall, heavy menstrual bleeding proved the most difficult to control among the women and girls in this study.

Excessive bleeding reported during or after delivery, including in cases of miscarriage and stillbirth, are consistent with other reports covering women with bleeding disorders, the researchers noted. In total, nine stillbirths or miscarriages were reported among women with hemophilia A, and four for those with hemophilia B.

Little data are available to assess the risk of miscarriage in the WGH population, the investigators said.

They added that current treatment guidelines offer limited information on managing hemophilia in women and girls. The bleeding events that these patients experience at regular intervals, for instance, highlight the increased need to monitor their coagulation status and their potential need for specialty care, the team said.

“Furthering clinical care for WGH,” the scientists concluded, “is needed to reduce the psychosocial, emotional and economic impacts of a bleeding disorder diagnosis.”