Delving into the Latest Updates on Human coagulation factor VIII (Rongsheng) with Synapse

Overview

Basic Info

Drug Type

Non-recombinant coagulation factor

Synonyms-

Target

F10

Mechanism

F10 stimulants(Coagulation factor X stimulants)

Therapeutic Areas

Congenital DisordersHemic and Lymphatic DiseasesOther Diseases

Active Indication

Hemophilia A

Inactive Indication-

Originator Organization

Chengdu Rongsheng Pharmaceutical Co., Ltd.

Active Organization

Chengdu Rongsheng Pharmaceutical Co., Ltd.

Inactive Organization-

Drug Highest PhaseApproved

First Approval Date

CN (01 Jan 2000),

Hemophilia A

Regulation-

Login to view timeline

INTRODUCTIONMultiple studies have demonstrated the effectiveness of pharmacokinetic (PK)-guided individualized prophylaxis with human coagulation factor VIII (FVIII) compared with standard prophylaxis, but no studies have evaluated the economics of PK-guided prophylaxis in China. Hence, we conducted this study to assess the cost-effectiveness of PK-guided prophylaxis with recombinant FVIII (rFVIII) versus standard prophylaxis in Chinese adult patients with severe hemophilia A.METHODSA discrete event simulation model was developed to simulate 10,000 patients with hemophilia A who received rFVIII treatment over a 1-year time horizon. The standard prophylaxis rFVIII dose was 30 IU/kg by intravenous injection. The PK-guided prophylaxis dosage was adjusted for each patient to maintain FVIII trough level at 1-5 IU/dL. Dosing interval for both approaches was kept fixed at 48 h. The health outcomes included annual joint bleed rate (AJBR) and quality-adjusted life years (QALYs). The model considered the costs of drug. Incremental cost-effectiveness ratio (ICER) was estimated and scenario analysis was performed.RESULTSA total of 94.3% of patients receiving PK-guided individualized prophylaxis achieved the goal of maintaining the trough concentration at 1-5 IU/dL compared with 62.7% on standard prophylaxis. AJBR and QALYs gained in PK-guided and standard prophylaxis were 1.527 vs 1.601, and 0.8384 vs 0.8383, respectively. Costs of drug prophylaxis and costs of treatment for bleeding events in PK-guided prophylaxis (148,641.47 USD; 4546.43 USD) were lower than those in standard prophylaxis (159,620.93 USD; 4753.39 USD). An average saving of USD 11,186.47 was obtained by the PK-guided approach. The prophylaxis treatment scenarios were the most influential factors.CONCLUSIONPK-guided individualized prophylaxis appeared to be a dominant treatment compared with standard prophylaxis, with slightly higher QALYs but lower total costs.

20 Apr 2022·Zhonghua shao shang yu chuang mian xiu fu za zhi

[Treatment of chronic refractory wound with autologous platelet-rich plasma in a hemophilia A patient after knee joint surgery].

Article

Author: Zhou, R X ; Zhou, W B ; Dong, Y S

On December 22, 2017, a 35-year-old male hemophilia A patient with a secondary chronic refractory wound after left knee joint surgery was transferred from the Department of Hematology of Maoming People's Hospital to the Department of Burns and Plastic Surgery in the same hospital. The physical examination revealed that the patient's left knee joint was swollen, with a full-thickness skin defect wound of 4 cm×4 cm on the lateral side of the joint and a large number of dark red blood clots at the bottom of the wound. The wound bleeding was controlled by intravenous infusion of plasma, cryoprecipitate, and human coagulation factor Ⅷ. After con- ventional debridement and dressing changes until the wound infection was controlled and necrotic tissue was removed, a subcutaneous cavity wound of 2 cm×2 cm in area and 3 cm in depth remained in the left knee joint and was difficult to heal. Nineteen days after transfer, the patient received autologous platelet-rich plasma (PRP) treatment, and 32 days after PRP treatment, the wound in left knee joint was healed with epithelialization. This case suggests that autologous PRP therapy would be a good option for hemophilia complicated chronic refractory wounds when they could not be repaired by surgery.

01 Jan 2021·Pharmeuropa bio & scientific notes

Calibration of the Ph. Eur. human coagulation Factor VIII Concentrate BRP batch 6.

Article

Author: Costanzo, A ; Le Tallec, D ; Raut, S

The European Pharmacopoeia (Ph. Eur.) Biological Reference Preparation (BRP) for human coagulation Factor VIII (FVIII) Concentrate is used as working standard for potency determination of human coagulation FVIII preparations by chromogenic assay. BRP batch 5 was established in 2015 and its stocks were running low. Therefore, the European Directorate for the Quality of Medicines & HealthCare (EDQM) initiated a project (BSP156) for the calibration of a replacement batch. The potency of BRP batch 6 was assigned during an international collaborative study involving 16 laboratories worldwide, with reference to the WHO 8th International Standard (IS) and BRP batch 5. Participants were instructed to perform 3 independent FVIII potency assays following their own routine validated methods for the chromogenic assay, which is the assay prescribed by the Ph. Eur. As an outcome of the study, Ph. Eur. human coagulation FVIII Concentrate BRP batch 6 was assigned a consensus potency of 9.9 IU/ampoule for the chromogenic assay. The Ph. Eur. BRP batch 6 is a freeze-dried, plasma-derived concentrate. Based on accelerated degradation studies, the stability of the material is suitable for a reference preparation. The Ph. Eur. BRP batch 6 was adopted at the 167th session of the Ph. Eur. Commission in June 2020 and is available from the EDQM under product code H0920000.

100 Deals associated with Human coagulation factor VIII (Rongsheng)

Login to view more data