Children with inhibitors8
n=88
1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W
“The decision to prescribe HEMLIBRA was made together with the patient’s family. They were excited about a subcutaneous option and the potential for better efficacy and less bleeding.”1
—Michael Callaghan, MD, Children’s Hospital of Michigan
Based on a randomized trial (N=65) with a mean observation period of 49 months.
*Results from a multicenter, randomized, open-label trial of young boys (<30 months old) with severe hemophilia A without inhibitors randomized to regular prophylactic recombinant FVIII infusions or to an enhanced episodic infusion schedule. Clinical assessments of index joints were performed with plain-film x-rays or magnetic resonance imaging at age 6.20
The HAVEN 2 study evaluated patient-reported hemophilia-related symptoms (painful swellings and presence of joint pain) and physical functioning (pain with movement) using the Physical Health Score of the Hemophilia-specific Quality of Life Short Form (Haemo-QoL-SF) questionnaire for patients ≥8 to <12 years of age.
INTRA-INDIVIDUAL COMPARISON
HAVEN 2†: Individual patient ABR at a median study duration of 89 weeks in QW cohort (N=15)‡1
†Based on HAVEN 2: Non-randomized, multicenter, open-label clinical trial in pediatric patients (age <12 years, or 12–17 years who weigh <40 kg) with hemophilia A with FVIII inhibitors. All patients received HEMLIBRA prophylaxis at 3 mg/kg QW for the first 4 weeks. Sixty-eight patients received 1.5 mg/kg QW, 10 patients received 3 mg/kg Q2W, and 10 patients received 6 mg/kg Q4W thereafter.
‡A comparison of data from HAVEN 2 vs data in the NIS prior to enrollment.
Intraindividual comparison of 15 participants who previously took BPA prophylaxis showed that emicizumab prophylaxis reduced the ABR by 99% (95% CI: 97.4; 99.4).§8
§ABR was calculated with a negative binomial regression model, accounting for different follow-up times.
As observed over at least 52 weeks (HAVEN 2) and at least 24 weeks (HAVEN 4‖).16
‖Based on HAVEN 4: A single-arm, multicenter, open-label trial in 41 adult and adolescent males with hemophilia A with or without FVIII inhibitors who previously received either on-demand or prophylactic treatment with FVIII or BPAs. All patients received HEMLIBRA prophylaxis at 3 mg/kg QW for the first 4 weeks followed by 6 mg/kg Q4W thereafter.5
HEMLIBRA Q2W prophylaxis
(95% Cl: 0.6; 2.9)
(n=6)
HEMLIBRA Q4W prophylaxis
(95% Cl: 0.2; 2.6)
(n=7)
§ABR was calculated with a negative binomial regression model, accounting for the different follow-up times.
¶HOHOEMI was a non-randomized study in pediatric patients (Japanese children <12 years) with hemophilia A without FVIII inhibitors. All patients received HEMLIBRA prophylaxis at 3 mg/kg once weekly for the first 4 weeks. Six patients received 3 mg/kg Q2W and 7 patients received 6 mg/kg Q4W thereafter.12
Frequently Asked Questions
Get your questions about HEMLIBRA answered
ABR=annualized bleed rate. ABR calculated with a negative binomial regression model, which accounts for different follow-up times15; BPA=bypassing agent; CI=confidence interval; FVIII=factor VIII; MASAC=Medical and Scientific Advisory Council; NIS=non-interventional study; QW=once weekly; Q2W=once every 2 weeks; Q4W=once every 4 weeks.
Help you determine whether HEMLIBRA is right for your patient.
Data on file. Genentech, Inc.
Data on file. Genentech, Inc.
Our commitment to provide transparent and timely safety information about emicizumab-kxwh. Genentech, Inc. https://www.emicizumabinfo.com/content/emicizumabinfo/en_us/patient.html#. Accessed August 25, 2020.
Our commitment to provide transparent and timely safety information about emicizumab-kxwh. Genentech, Inc. https://www.emicizumabinfo.com/content/emicizumabinfo/en_us/patient.html#. Accessed August 25, 2020.
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Jiménez-Yuste V, Klamroth R, Castaman G, et al. Second interim analysis results from the STASEY trial: a single-arm, multicenter, open-label, phase III clinical trial to evaluate the safety and tolerability of emicizumab prophylaxis in people with hemophilia A (PwHA) with FVIII inhibitors. Poster presented at: International Society on Thrombosis and Haemostasis 2020 Virtual Congress; July 12–14, 2020.
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Callaghan M, Negrier C, Paz-Priel I, et al. Emicizumab treatment is efficacious and well tolerated long term in persons with haemophilia (PwHA) with or without FVIII inhibitors: pooled data from four HAVEN studies. Slide deck presented at: International Society on Thrombosis and Haemostasis 2019 Congress; July 6–10, 2019; Melbourne, Australia.
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Oldenburg J, Mahlangu JN, Kim B, et al. Emicizumab prophylaxis in hemophilia A with inhibitors. N Engl J Med. 2017;377(9):809-818.
Young G, Liesner R, Sidonio R Jr, et al. Emicizumab prophylaxis provides flexible and effective bleed control in children with hemophilia A with inhibitors: results from the HAVEN 2 study. Presented at: ASH 60th Annual Meeting of the American Society of Hematology; December 1–4, 2018; San Diego, CA.
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