Building a network for hemophilia care in China: 15 years of achievement for the Hemophilia Treatment Center Collaborative Network of China

Yang, Renchi; Poon, Man‐Chiu; Luke, Koon Hung; Zhao, Yongqiang; Sun, Jing; Wang, Xuefeng; Wu, Runhui; Chen, Lixia; Zhang, Xinsheng; Wu, Jingsheng

doi:10.1182/bloodadvances.2019gs121524

Cited by 17 publications

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“…For example, informed by the HTCCNC, the Chinese government began development of a hierarchical medical system in 2004, setting up designated medical institutions at the municipal, county and provincial levels 19 . Furthermore, in 2013, the Chinese Ministry of Health recognized haemophilia as one of 20 major diseases occurring in China and included it in health insurance that provided partial coverage for FVIII concentrates 19 . This initiative has contributed substantially to filling the knowledge gap regarding real‐world data on haemophilia and has contributed to an in‐depth understanding of the current care and clinical characteristics of patients with haemophilia A in China.…”

Section: Discussionmentioning

confidence: 99%

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An overview of patients with haemophilia A in China: Epidemiology, disease severity and treatment strategies

Song

Zhong²,

Xue

et al. 2020

Haemophilia

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Introduction Haemophilia A (HA) is a rare X chromosome‐linked bleeding disorder resulting in missing or defective clotting factor VIII (FVIII) and causes large disease burden. Aim As a member of World Federation of Hemophilia, China seeks to understand the current epidemiology, disease profile and treatment landscape of patients with HA through the Hemophilia Treatment Center Collaboration Network of China (HTCCNC). Methods The HTCCNC enabled data collection on patients with HA from 166 member hospitals (2007–2019) across China. The distribution of patients across 31 divisions was summarized using a heat map. Patient demographics, disease severity and clinical and treatment information were summarized using descriptive statistics. Results HTCCNC identified 17,779 patients with HA during 2007–2019. Patients were predominantly male (99.99%), and 28.3% had a known family history of haemophilia. Among patients with lab‐measured disease severity (N = 13,116), 6,519 had severe HA (49.7%), 4,788 had moderate HA (36.5%), and 1,809 had mild HA (13.8%). Among patients with information on the delays, delays in diagnosis and in treatment initiation were observed in 1,437 (28.8%) and 1,750 (39.2%) patients, respectively. On average, those patients had an 8.4 years gap between the first bleed and HA diagnosis and a delay of 8.6 years from the first bleed to treatment initiation. Additionally, 44.33% of patients relied solely on episodic treatments, and 16.2% received any prophylaxis treatments. Conclusions Using data from the largest haemophilia registry in China, this study indicated that delayed diagnosis and treatment, together with low utilization of prophylaxis, are key challenges for patients with HA.

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Section: Discussionmentioning

confidence: 99%

“…Besides providing systematic training and medical education, 19 the HTCCNC established the first haemophilia registry in China. Leveraging its data, this study summarized the up‐to‐date epidemiology, disease severity and treatment landscape of patients with haemophilia A.…”

Section: Discussionmentioning

confidence: 99%

An overview of patients with haemophilia A in China: Epidemiology, disease severity and treatment strategies

Song

Zhong²,

Xue

et al. 2020

Haemophilia

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“…The HTCCNC has published several guidelines and consensuses to standardize areas of haemophilia care, including haemophilic arthropathy, inhibitors, paediatric haemophilia, acquired haemophilia and HTC establishment 13 …”

Section: Achievements In Capacity Building and Development Of A Haemomentioning

confidence: 99%

Haemophilia care in China: Achievements in the past decade

2020

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There is a large population of people with haemophilia in China. However, the development of haemophilia care in China lagged far behind developed countries and was unbalanced across different regions. With the establishment of the Hemophilia Treatment Center Collaborative Network of China (HTCCNC) in 2004 and its collaboration with the Chinese government, the World Federation of Haemophilia (WFH), non-profit foundations, industries, and experts from other countries, haemophilia care in China have made considerable strides in building capacity. The HTCCNC helped develop clinics throughout the country, promote multidisciplinary care, promote prophylaxis (albeit low-dose), advance clinical evaluation and treatment, and research. With many collaborative efforts in the past decade, haemophilia care in China has entered a new stage of development. In this review, we summarize these major achievements and efforts within the last decade.

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“…According to a 2019 report, the network includes 115 regional hospitals and has registered approximately 18,000 patients with hemophilia, including 16,083 patients with hemophilia A. 3 However, it is estimated that the actual number of people with hemophilia A and B in China is 65,000 to 130,000, 3 suggesting that only a small percentage of patients are diagnosed and registered. [4][5][6] Despite improvements in care over the last decade, management of hemophilia in China remains a challenge because of factors such as insufficient health care infrastructure, inadequate awareness and knowledge of the disease, high costs, lack of medical insurance for a majority of patients, and limited availability or access to factor replacement products.…”

Section: Introductionmentioning

confidence: 99%

“…2,[4][5][6][7][8][9] To mitigate treatment costs and limited access to resources, several studies led by the HTCCNC investigated the potential benefit of using lowdose prophylaxis to reduce the number of joint bleeding events and to improve quality of life. 3,9 Moroctocog alfa is a B-domain-deleted recombinant FVIII product manufactured using an albumin-free cell culture (AF-CC) process. [10][11][12] Moroctocog alfa (AF-CC; Xyntha in China, the United States, Canada, and certain other regions, or ReFacto AF in Europe; Wyeth Pharmaceuticals, Inc. [Pfizer Inc], Philadelphia, PA, USA) has an enhanced viral safety profile relative to its predecessor product, moroctocog alfa (ReFacto), utilizing a nanofiltration to eliminate animal-and human-derived proteins.…”

Section: Introductionmentioning

confidence: 99%

<p>Safety and Efficacy of Moroctocog Alfa (AF-CC) in Chinese Patients with Hemophilia A: Results of Two Open-Label Studies</p>

Yang

Zhao

Wang

et al. 2020

JBM

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Introduction: Moroctocog alfa albumin-free cell culture (AF-CC) increases plasma levels of factor VIII (FVIII) activity and, in China, is indicated for the control and prevention of bleeding episodes in patients with hemophilia A. This study aimed to evaluate the efficacy, safety, and recovery data of moroctocog alfa (AF-CC) in patients with hemophilia participating in two open-label studies, both conducted in China. Methods: The authorization study (clinicaltrials.gov identifier NCT00868530) enrolled patients aged ≥6 years, previously treated with ≥1 exposure day of FVIII replacement therapy. The real-world study (clinicaltrials.gov identifier NCT02492984) enrolled patients of any age who were previously untreated or requiring surgical prophylaxis. In both studies, on-demand treatment was administered over 6 months. Key assessments included response to treatment, FVIII inhibitor development, and recovery. Results: In the authorization study (N = 53; mean age, 23.2 years; severe hemophilia, 23%), response was excellent/good for 90% of infusions at 24 hours. Seven patients developed inhibitors. Mean (SD) FVIII recovery at the initial and final visits was 1.77 (0.50) and 1.67 (0.45) (IU/dL)/(IU/kg), respectively. In the real-world study (N = 85; mean age, 9.5 years; severe hemophilia, 58%), response was rated as excellent or good for most (87%) on-demand infusions and for all surgical prophylaxis patients (n = 14). Seven patients developed FVIII inhibitors. Mean (SD) FVIII recovery at the initial and final visits was 1.71 (0.50) and 1.68 (0.31) (IU/dL)/(IU/kg), respectively. No new safety signals were observed in either study. Conclusion: On-demand treatment and surgical prophylaxis with moroctocog alfa (AF-CC) is safe and effective for both previously treated and previously untreated Chinese patients with hemophilia A.

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Building a network for hemophilia care in China: 15 years of achievement for the Hemophilia Treatment Center Collaborative Network of China

Cited by 17 publications

References 0 publications

An overview of patients with haemophilia A in China: Epidemiology, disease severity and treatment strategies

An overview of patients with haemophilia A in China: Epidemiology, disease severity and treatment strategies

Haemophilia care in China: Achievements in the past decade

<p>Safety and Efficacy of Moroctocog Alfa (AF-CC) in Chinese Patients with Hemophilia A: Results of Two Open-Label Studies</p>

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