Authors' objectives: To collect international indicators for assessing hemophilia treatment effectiveness and analyze patient characteristics, treatment types, treatment efficacy, and the medical resource consumption of hemophilia A and B patients in our country.

Authors' results and conclusions: Registry systems in various countries primarily record epidemiological information and lack efficacy indicators; hence, all countries encourage patients to record their treatment status. The patient populations in the examined studies mainly have severe hemophilia A with a median annual bleeding rate ranging from one to three times. However, there are significant differences in bleeding episodes and drug consumption among studies. In Taiwan, based on the national registry data, in 2020, there were 1,414 patients with hemophilia A or B in our country, with 523 and 108 patients having severe hemophilia A or B, respectively; the treatment rates were 98.3% and 100%, respectively. The proportion of patients with inhibitors was 16.8% and 13%, respectively. The median annual bleeding rates for patients with severe hemophilia A receiving prophylactic treatment or on-demand treatment during bleeding were three times (IQR: 0ˇV8.1) and 18 times (IQR: 8ˇV34.9), respectively, while for patients with severe hemophilia B, they were 3.3 times (IQR: 0ˇV10) and nine times (IQR: 5ˇV20) respectively. Comparing domestic data with international data shows that treatment rates, clotting factor consumption, and the number of bleeding episodes for patients with severe hemophilia in our country are already close to international levels.

Authors' recommendations: Finally, this study suggests that the authorities could add fields for patient treatment plans and other examination data to the home treatment record form to collect more comprehensive and detailed data for deeper analysis and discussion in the future.

Authors' methods: This study is a systematic literature review on recommendations and indicators for treating severe hemophilia A and B. And It also analyzes major illness files, National Health Insurance claim data, and home treatment records to analyze this countryˇ¦s hemophilia A and B patientsˇ¦ basic characteristics, treatment modalities, number of bleeding episodes, number of hospital visits, and the amount and cost of hemophilia treatment drugs they claim. It also compares domestic and international data to examine the effectiveness of hemophilia treatment in our country.

Project Status: Completed

Year Published: 2023

URL for published report: https://www.cde.org.tw/HTA/achievements

URL for additional information: English (Abstract) Full HTA report in Chinese (Mandarin)

English language abstract: An English language summary is available

Publication Type: Full HTA

Organisation Name: Center for Drug Evaluation

Contact Address: 3F.,No.465, Sec.6, Zhongxiao E. Rd., Taipei 11557, Taiwan. R.O.C.

Contact Name: Li Ying (Grace) Huang

Contact Email: lyhuang277@cde.org.tw