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Original Article

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Blood Res 2020; 55(4):

Published online December 31, 2020

https://doi.org/10.5045/br.2020.2020225

© The Korean Society of Hematology

Safety and effectiveness of Rixubis in patients with hemophilia B: a real-world, prospective, postmarketing surveillance study in South Korea

Eun Jin Choi1, Tai ju Hwang2, Yong Mook Choi3, Hugh Chul Kim4, Myung Chul Yoo5, Haylee Song6, Kayode Badejo7

Author Info. +

1Department of Pediatrics, Daegu Catholic University Medical Center, Daegu, 2Korea Hemophilia Foundation Clinic, Gwangju, 3Korea Hemophilia Foundation Clinic, 4HughChul Kim Internal Medicine Clinic, 5Sunchun Chung Hospital, Seoul, Korea, 6Hematology, Growth and Emerging Markets, Takeda Pharmaceutical International, Singapore, 7Global Patient Safety Evaluation, Takeda Pharmaceutical Company Ltd, Cambridge, MA, USA

Correspondence to :
*This study was supported by a grant from Shire, a member of the Takeda group of companies.

Kayode Badejo, M.D.
Global Patient Safety Evaluation, Takeda Pharmaceutical Company Ltd, 650 E. Kendall Street, Cambridge, MA 02142, USA
E-mail: Kayode.Badejo@takeda.com

Received: August 31, 2020; Accepted: October 30, 2020

Abstract

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Background
Rixubis (recombinant factor IX, nonacog gamma) is indicated for the control and prevention of bleeding episodes, perioperative management, and routine prophylaxis in hemophilia B patients. This real-world, postmarketing surveillance study aimed to evaluate the safety and effectiveness of Rixubis in adult and pediatric hemophilia B patients in South Korea.
Methods
This prospective, observational, multicenter study (clinicaltrials.gov identifier: NCT029 22231) was conducted in hemophilia B patients between April 2015 and April 2019, who were observed for up to 6 months after the initiation of Rixubis treatment. Safety was evaluated based on the number and severity of adverse events (AEs) and serious AEs (SAEs). Hemostatic effectiveness was assessed by physicians and patients by using a four-point scale and rated as excellent, good, fair, or no response based on treatment type.
Results
In all, 58 patients were enrolled from four centers by seven physicians during the study period. The safety and effectiveness analysis sets included 57 and 54 patients, respectively. Overall, 11 AEs were reported in eight patients (14.0%), of which three were SAEs and occurred in three patients (5.3%). All 11 AEs were reported as unexpected and mild in severity, with no anaphylactic reaction, and 10 AEs (90.9%) resolved. The majority of AEs (10) were unrelated to Rixubis. Of the 142 hemostatic effectiveness assessments, 123 (86.6%) were reported as good or excellent.
Conclusion
Rixubis demonstrated an acceptable safety and effectiveness profile in the treatment of bleeding, perioperative management, and prophylaxis in hemophilia B patients in a real-world setting in South Korea.

Keywords Hemophilia B, Rixubis, Safety, Effectiveness, South Korea, Bleeding

Article

Original Article

Blood Res 2020; 55(4): 246-252

Published online December 31, 2020 https://doi.org/10.5045/br.2020.2020225

Copyright © The Korean Society of Hematology.

Safety and effectiveness of Rixubis in patients with hemophilia B: a real-world, prospective, postmarketing surveillance study in South Korea

Eun Jin Choi1, Tai ju Hwang2, Yong Mook Choi3, Hugh Chul Kim4, Myung Chul Yoo5, Haylee Song6, Kayode Badejo7

1Department of Pediatrics, Daegu Catholic University Medical Center, Daegu, 2Korea Hemophilia Foundation Clinic, Gwangju, 3Korea Hemophilia Foundation Clinic, 4HughChul Kim Internal Medicine Clinic, 5Sunchun Chung Hospital, Seoul, Korea, 6Hematology, Growth and Emerging Markets, Takeda Pharmaceutical International, Singapore, 7Global Patient Safety Evaluation, Takeda Pharmaceutical Company Ltd, Cambridge, MA, USA

Correspondence to:
*This study was supported by a grant from Shire, a member of the Takeda group of companies.

Kayode Badejo, M.D.
Global Patient Safety Evaluation, Takeda Pharmaceutical Company Ltd, 650 E. Kendall Street, Cambridge, MA 02142, USA
E-mail: Kayode.Badejo@takeda.com

Received: August 31, 2020; Accepted: October 30, 2020

Abstract

Background
Rixubis (recombinant factor IX, nonacog gamma) is indicated for the control and prevention of bleeding episodes, perioperative management, and routine prophylaxis in hemophilia B patients. This real-world, postmarketing surveillance study aimed to evaluate the safety and effectiveness of Rixubis in adult and pediatric hemophilia B patients in South Korea.
Methods
This prospective, observational, multicenter study (clinicaltrials.gov identifier: NCT029 22231) was conducted in hemophilia B patients between April 2015 and April 2019, who were observed for up to 6 months after the initiation of Rixubis treatment. Safety was evaluated based on the number and severity of adverse events (AEs) and serious AEs (SAEs). Hemostatic effectiveness was assessed by physicians and patients by using a four-point scale and rated as excellent, good, fair, or no response based on treatment type.
Results
In all, 58 patients were enrolled from four centers by seven physicians during the study period. The safety and effectiveness analysis sets included 57 and 54 patients, respectively. Overall, 11 AEs were reported in eight patients (14.0%), of which three were SAEs and occurred in three patients (5.3%). All 11 AEs were reported as unexpected and mild in severity, with no anaphylactic reaction, and 10 AEs (90.9%) resolved. The majority of AEs (10) were unrelated to Rixubis. Of the 142 hemostatic effectiveness assessments, 123 (86.6%) were reported as good or excellent.
Conclusion
Rixubis demonstrated an acceptable safety and effectiveness profile in the treatment of bleeding, perioperative management, and prophylaxis in hemophilia B patients in a real-world setting in South Korea.

Keywords: Hemophilia B, Rixubis, Safety, Effectiveness, South Korea, Bleeding

Fig 1.

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Figure 1.Overview of the study population.
Abbreviation: CRF, case report form.
Blood Research 2020; 55: 246-252https://doi.org/10.5045/br.2020.2020225

Table 1 . Baseline demographic and clinical characteristics of the study participants..

CharacteristicsN=57
Sex, N (%)
Male57 (100.0)
Age, mean±SD34.6±19.0
Age group (yr), N (%)
<63 (5.3)
≥6 to <126 (10.5)
≥12 to <194 (7.0)
≥1944 (77.2)
BMI (kg/m2), mean±SDa)24.0±3.7
Hepatic impairment, N (%)
Yes6 (10.5)
No51 (89.5)
History of allergic reactions, N (%)
Yes1 (1.8)
No55 (96.5)
Unknown1 (1.8)
Family history of inhibitor development, N (%)
Yes3 (5.3)
No51 (89.5)
Unknown3 (5.3)
Bleeding episodes by severity (number) within the last 12 mo (mean±SD)
Minor (N=41)22.4±24.8
Moderate (N=14)2.5±3.1
Major (N=4)1.3±0.5
Use of FIX before Rixubis treatment, N (%)
Yes54 (94.7)
No3 (5.3)
Total number of days of FIX product use after hemophilia B diagnosis, N (%)b)
1–4 days4 (7.4)
5–20 days4 (7.4)
21–50 days4 (7.4)
51–100 days16 (29.6)
101–150 days1 (1.9)
>150 days24 (44.4)
Current medical status, N (%)
Acquired immunodeficiency syndrome3 (5.3)
Chronic hepatitis C3 (5.3)
Chronic hepatitis B2 (3.5)
Hepatitis C1 (1.8)
Hypertension4 (7.0)
Arthralgia1 (1.8)
Hemophilic arthropathy1 (1.8)
Osteonecrosis1 (1.8)
Arthroscopy1 (1.8)
Diabetes mellitus1 (1.8)
Bronchitis chronic1 (1.8)
Rehabilitation therapy1 (1.8)
Duration of hemophilia B (yr), mean±SDb)18.1±10.2

a)N=48 (data are missing for nine patients). b)N=53 (data are missing for four patients)..

Abbreviations: BMI, body mass index; FIX, factor IX; SD, standard deviation..

Table 2 . Information of study drug administration..

Treatment typeTreatment dose (IU), mean±SDTreatment dose (IU/kg), mean±SDN (%)
Treatment of bleeding (on-demand)36 (63.2)
Total32,241.7±39,659.9482.3±486.1
Average2,831.5±1,341.243.8±14.4
Surgery and/or perioperative coverage2 (3.5)
Total44,040.0±2,658.7630.4±25.7
Average3,145.7±189.945.0±1.8
Prophylaxis45 (78.9)
Total2,388,776.9±4,020,208.133,712.0±51,060.2
Average2,603.5±881.741.9±11.1

Treatment of bleeding and surgery and/or preoperative coverage:

Total treatment dose (IU/kg)=sum of total dose per infusion for each treatment regimen/body weight.

Average treatment dose (IU/kg)=(total treatment dose for each treatment regimen/body weight)/Total number of infusions for each treatment regimen.

Prophylaxis:

Total treatment dose (IU/kg)=sum of [(total number of infusions)×(total dose per infusion/body weight)].

✓ Total number of infusions=(actual Dosing interval)×integer of {(stop date of treatment regimen-start date of treatment regimen+1)/7} +[7×decimal of {(stop date of treatment regimen-start date of treatment regimen+1)/7}]/(dosing interval).

✓ Actual dosing interval=stop date of treatment regimen-start date of treatment regimen.

Average treatment dose (IU/kg)=(total treatment dose/body weight)/total number of infusions.

Abbreviations: IU, international unit; SD, standard deviation..

Table 3 . Summary of adverse events (safety analysis set, N=57)..

AEs (PTa))N of patients (%)N of AEsUnexpected AEsSerious AEs
Hemophilic arthropathy2 (3.5)222
Cataract1 (1.8)110
Upper abdominal pain1 (1.8)110
Aspergillus infection1 (1.8)110
Tinea pedis1 (1.8)110
Procedural pain1 (1.8)110
Anti-factor IX antibody increasedb)1 (1.8)111
Hyperuricemia1 (1.8)110
Insomnia1 (1.8)110
Hematuria1 (1.8)110
Total8 (14.0)11113

a)Dictionary: MedDRA (version: 21.1). b)Confirmatory test was negative for this..

Abbreviations: AE, adverse events; PT, preferred term..

Table 4 . Assessment of hemostatic effectiveness (efficacy analysis set, N=54)..

Treatment typeN of assessments (%)N of patients (%)b)
Treatment of bleedinga)
Total6625c)
None0 (0.0)0 (0.0)
Fair11 (16.7)7 (28.0)
Good50 (75.8)17 (68.0)
Excellent5 (7.6)4 (16.0)
Effective (excellent+good)55 (83.3)18 (72.0)
Prophylaxisa)
Total7640c)
None0 (0.0)0 (0.0)
Fair8 (10.5)5 (12.5)
Good47 (61.8)32 (80.0)
Excellent21 (27.6)17 (42.5)
Effective (excellent+good)68 (89.5)35 (87.5)
Overall treatment typea)
Total14254c)
None0 (0.0)0 (0.0)
Fair19 (13.4)12 (22.2)
Good97 (68.3)43 (79.6)
Excellent26 (18.3)20 (37.0)
Effective (excellent+good)123 (86.6)42 (77.8)

a)In case an assessment is performed by the patient as well as by the physician, then the worst-case assessment is considered. b)For the ratings “None,” “Fair,” “Good,” or “Excellent,” each patient could rate more often. For the rating “Good” or “Excellent,” in case a patient had more than one assessment, the worst-case assessment is considered for this patient. c)Number of unique patients with an effectiveness assessment available in the respective “Treatment type/Assessor.”.
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