Transfusion Evidence Library

1. Indirect Treatment Comparison of Damoctocog Alfa Pegol versus Turoctocog Alfa Pegol as Prophylactic Treatment in Patients with Hemophilia A

Vashi P, Batt K, Klamroth R, Mancuso ME, Majewska R, Tiede A, Mantovani LG

Journal of blood medicine. 2021;12:935-943

Abstract

PURPOSE To assess the efficacy and FVIII consumption of BAY 94-9027 versus N8-GP in prophylaxis in adolescent and adult patients with severe hemophilia A (HA). PATIENTS AND METHODS A systematic literature review was conducted to identify studies on the efficacy of BAY-94-9027 and N8-GP for prophylaxis in patients with HA aged ≥12 years without a history of inhibitors. Eight studies met systematic literature review inclusion criteria, but only data from PROTECT VIII on BAY 94-9027 and PATHFINDER 2 on N8-GP could be used for an indirect comparison. Matching-adjusted indirect comparison (MAIC) and simulated treatment comparison were performed. RESULTS No significant differences (unadjusted and adjusted) were observed in the mean annualized bleeding rate (ABR) for any bleed and proportion of patients with zero bleeds when comparing BAY 94-9027 to N8-GP. The adjusted treatment difference [incidence rate ratio (IRR)] in terms of ABR was 1.11 (95% CI, 0.85-1.44). The odds ratio (OR) of any bleed, measuring the relative effect of BAY 94-9027 versus N8-GP on the proportion of patients with zero bleeds, was 1.03 (95% CI, 0.60-1.77). FVIII consumption was significantly lower in BAY 94-9027 [mean adjusted difference=-1292.57 IU/kg/year (95% CI, ‒2152.44 to ‒432.70)]; a 26.7% reduction in consumption of BAY-94-9027. The results of the sensitivity analyses were similar to the main analysis for mean ABRs, percentages of patients with zero bleeds, and significant reduction in rFVIII consumption. For patients on BAY 94-9027 every-5-days and every-7-days, no differences versus every-4-days N8-GP were observed for the mean ABR for any bleed [IRR=0.90 (95% CI, 0.68‒1.20)] and proportion of patients with zero bleeds [OR=1.06 (95% CI, 0.56‒2.02)]. CONCLUSION BAY 94-9027 prophylaxis demonstrated 26.7% lower annual consumption when compared to N8-GP with similar efficacy in terms of ABR and percentage of patients with zero bleeds.

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2. Comparative evaluation of the safety and efficacy of recombinant FVIII in severe hemophilia A patients

Abolghasemi H, Panahi Y, Ahmadinejad M, Toogeh G, Karimi M, Eghbali A, Mirbehbahani NB, Dehdezi BK, Badiee Z, Hoorfar H, et al

Journal of Pharmacopuncture.. 2018;21((2)):76-81.

Abstract

Objective: This study compared the safety and efficacy of Safacto versus xyntha in patients with severe hemophilia A. Methods: Thirty-three male patients with severe hemophilia A were randomly divided into two groups. Seventeen patients received Safacto and 16 patients received Xyntha for four consecutive times. The dosage of FVIII was 40-50 IU/kg for each injection. Plasma level of FVIII activity was evaluated before every injection, 15 minutes after the injection and one month after the start of the trial. The rate of factor VIII activity, pain and joint motion were also assessed before and after the treatment. Results: Plasma level of FVIII clotting activity in Safacto and Xyntha were 1.96+/-0.5 IU/dl and 1.63+/-0.5 IU/dl and increased to 88.84+/-25.2 IU/dl and 100.09+/-17.8 IU/dl, respectively (P<0.001). Pain score and range of motion improvement were 9.3+/-0.9 and 8.7+/-0.1 in Safacto (P=0.17); and 9.4+/-0.8 and 8.8+/-0.3 in Xyntha (P=0.35), respectively. No allergic or other unfavorable reactions was observed with either of the preparations. Conclusion: This study showed that Safacto has a favorable efficacy and safety profile.

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3. Source of factor VIII replacement (PLASMATIC OR RECOMBINANT) and incidence of inhibitory alloantibodies in previously untreated patients with severe hemophilia a: the multicenter randomized sippet study

Peyvandi F, Mannucci PM, Garagiola I, Elalfy M, El-Beshlawy A, Ramanan MV, Eshghi P, Hanagavadi S, Varadarajan R, Karimi M, et al

Blood. 2015;126((23)): Abstract No. 5.

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4. Effect on joint health of routine prophylaxis with Bayer’s sucrose-formulated recombinant factor VIII (rFVIII-FS) in adolescents and adults previously treated on demand: MRI analyses from the 3-Year Spinart study

Lundin B, Hong W, Raunig D, Engelen S, Peterfy C, Werk, Manco-Johnson MJ

Blood. 2014;124((21)): Abstract No. 2854

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5. A comparison of efficacy between recombinant activated factor VII (ARYOSEVEN TM) and NOVOSEVEN® in patients with hereditary FVIII deficiency with inhibitor

Faranoush M, Abolghassemi H, Toogeh G, Karimi M, Eshghi P, Managhchi M, Hoorfar H, Keikhaei DB, Mehrvar B, Khoein B, et al

Blood. 2014;124((21)): Abstract No. 4299

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6. Economic assessment of recombinant factor IX in prophylaxis for hemophilia B in young patients in Mexico

Mucino-Ortega E, Salinas-Escudero G, Galindo-Suarez RM

Value in Health. 2013;16((3):):A118.. Abstract No. PSY42.

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7. Evaluation of two secondary prophylaxis regimens of recombinant factor IX (r-IX) in moderately severe to severe (FIX <=2%) hemophilia B patients

Rendo P, Barrette-Grischow M-K, Smith L, Korth-Bradley JM, Charnigo R, Shafer FE

Blood. 2012;120((21):): Abstract No. 4628.

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8. A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT Study)

Gringeri A, Lundin B, Von Mackensen S, Mantovani L, Mannucci PM

Journal of Thrombosis and Haemostasis. 2011;9((4):):700-10.

Abstract

Background: Prevention of arthropathy is a major goal of hemophilia treatment. While studies in adults have demonstrated an impact of prophylaxis on the incidence of joint bleeds and patients' well-being in terms of improved quality of life (QoL), it is unclear whether or not prophylaxis influences the outcome and perception of well- of children with hemophilia. Objective:This randomized controlled study compared the efficacy of prophylaxis with episodic therapy in preventing hemarthroses and image-proven joint damage in children with severe hemophilia A (factor VIII <1%) over a 10-year time period. Methods: Forty-five children with severe hemophilia A, aged 1-7years (median 4), with negative clinical-radiologic joint score at entry and at least one bleed during the previous 6 months, were consecutively randomized to prophylaxis with recombinant factor VIII (25IUkg-1 3x week) or episodic therapy with >=25IUkg-1 every 12-24h until complete clinical bleeding resolution. Safety, feasibility, direct costs and QoL were also evaluated. Results:Twenty-one children were assigned to prophylaxis, 19 to episodic treatment. Children on prophylaxis had fewer hemarthroses than children on episodic therapy: 0.20 vs. 0.52 events per patient per month (P<0.02). Plain-film radiology showed signs of arthropathy in six patients on prophylaxis (29%) vs. 14 on episodic treatment (74%) (P< 0.05). Prophylaxis was more effective when started early (<=36 months), with patients having fewer joint bleeds (0.12 joint bleeds per patient per month) and no radiologic signs of arthropathy. Conclusion:This randomized trial confirms the efficacy of prophylaxis in preventing bleeds and arthropathy in children with hemophilia, particularly when it is initiated early in life. 2011 International Society on Thrombosis and Haemostasis.