HEMLIBRA was studied in infants and children with and without inhibitors
CHILDREN WITH FACTOR VIII INHIBITORS: HAVEN 2 STUDY
of children taking HEMLIBRA once a week had 0 bleeds (50/65)
of people taking HEMLIBRA once every 2 weeks had 0 bleeds (7/10)
of people taking HEMLIBRA once every 4 weeks had 0 bleeds (6/10)
Children: less than 12 years of age. | Infants ≤12 months of age.
The average number of treated bleeds per year (ABR, annualized bleed rate) for HAVEN 2: once every week 0.3, once every 2 weeks 0.2, once every 4 weeks 1.8.
The median time on HEMLIBRA was 58 weeks (once every week), 67 weeks (once every 2 weeks), and 67 weeks (once every 4 weeks). A median is a numeric middle. That means that in a list of numbers, the median is the number in the middle. This analysis was based on a descriptive study not designed to determine a definitive benefit.
A majority of people on HEMLIBRA had zero bleeds requiring treatment, regardless of their age, dosing option, or inhibitor status in the HAVEN 1-4 clinical trials
- HAVEN 3: 60% of adults and adolescents (ages 12 and older) without factor VIII inhibitors had zero treated bleeds
- HAVEN 4: 56% of adults and adolescents (ages 12 and older) with or without FVIII inhibitors taking HEMLIBRA once every 4 weeks had zero treated bleeds
Every bleed matters, regardless of age
That’s why Genentech designed the HAVEN 7 trial to study the use of HEMLIBRA in infants ≤12 months of age, including those previously untreated.
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OF INFANTS IN HAVEN 7 HAD ALREADY EXPERIENCED AT LEAST 1 BLEED PRIOR TO ENROLLMENT IN THE STUDY (36/55)
Of the total bleeds reported in these infants, 1/3 of them were spontaneous (25/77)
Why does this matter?
- The National Bleeding Disorders Foundation (NBDF) Medical and Scientific Advisory Council (MASAC) recommends that infants should be considered for prophylaxis with HEMLIBRA or factor VIII at any time after birth
- However, many infants with severe hemophilia A do not receive prophylaxis until >1 year of age, despite diagnosis at birth
*Defined as an infant with ≤5 exposure days to factor VIII.
Findings from our descriptive study on HEMLIBRA in infants
IN HAVEN 7, MORE THAN HALF OF INFANTS HAD ZERO BLEEDS REQUIRING TREATMENT (30/55)
- The average number of bleeds requiring treatment per year: 0.4
- 25 of 55 infants had a total of 42 treated bleeds, and all were caused by injuries
- There were 140 bleeds from injuries that did not need treatment
- The average number of joint bleeds requiring treatment per year: 0
- The median age at time of analysis was 29 months (range: 12-39 months)†
- The median time on treatment was about 100 weeks†
Infants had spontaneous bleeds requiring additional treatment while on HEMLIBRA
What’s a descriptive study?
Researchers are simply looking at what happened in a clinical trial, without determining a definitive benefit. Therefore, results should be interpreted with caution.
†A median is a numeric middle. That means, in a list of numbers, the median is the number in the middle.
HAVEN 7 Safety Profile
No new safety concerns were found in HAVEN 7 study.
ZERO BRAIN BLEEDS WERE REPORTED
People with a history of intracranial hemorrhage (ICH), otherwise known as a brain bleed, weren’t included in the study. Therefore, no conclusive data have been collected about the effects of HEMLIBRA regarding ICH prevention.
- No side effects led to withdrawal from HEMLIBRA or dose modification or interruption
- Of the 24 infants tested for factor VIII inhibitors following factor VIII exposure, 2 previously untreated patients tested positive, both of whom were less than 3 months of age at enrollment
- These data are a descriptive analysis and therefore should be interpreted with caution
‡One participant had 1 Grade 2 allergic reaction due to an egg allergy, and was found to be not related to HEMLIBRA.
§ISRs included redness, tenderness, warmth, or itching at the site of an injection.
IINone of the serious side effects were considered to be related to HEMLIBRA and all were considered serious because of hospitalization.
¶The severity of side effects are graded according to the World Health Organization Scoring System and range from 1 (mild) to 4 (most severe).
is an 8-year commitment from Genentech to build on the understanding of the safety and efficacy of HEMLIBRA in infants.
HEMLIBRA was also studied in children without factor VIII inhibitors
A study in Japan called HOHOEMI measured the average number of treated bleeds per year (ABR, annualized bleed rate) in children without factor VIII inhibitors using HEMLIBRA. This was a descriptive study with a small population (13 children). In a descriptive analysis, researchers are simply looking at what happened in the clinical trial, without determining a definitive benefit.
Children using HEMLIBRA every 2 weeks (6 people)
Children using HEMLIBRA every 4 weeks (7 people)
Children who were included in HOHOEMI were in the study for at least 24 weeks. ABRs shown are for treated bleeds.
The median time on HEMLIBRA every 2 weeks and HEMLIBRA every 4 weeks was 39 weeks and 32 weeks, respectively. A median is a numeric middle. That means that in a list of numbers, the median is the number in the middle.
Frequently Asked Questions
Have questions about HEMLIBRA? We have answers.
Individual results may vary. Not all patients in the clinical trials had zero bleeds. See results for clinical trial patients with or without factor VIII inhibitors at the top of this page.
When starting HEMLIBRA, a “loading dose” is taken once weekly for 4 weeks. After that, HEMLIBRA offers 3 regular dosing options: once weekly, once every 2 weeks, or once every 4 weeks. Talk to your doctor about dosing.
See How HEMLIBRA Makes a Difference in Our Younger Populations
"Hemophilia will always be part of our family, but it doesn't have to be the first thing on our minds all the time."
—Olivia, caregiver to Arlo
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