Although the use of bypassing agents has dramatically improved the management of haemophilia in patients with inhibitors, questions remain regarding optimal dosing regimens and methodology for monitoring their clinical effectiveness. In this study, we evaluated the efficacy and safety of two different doses of recombinant activated factor VIIa (rFVIIa) in patients with haemophilia and inhibitors and assessed the feasibility of using thromboelastography (TEG) and thrombin generation assays (TGA) for monitoring the response to rFVIIa. Six patients aged 9-49 years with congenital or acquired haemophilia with inhibitors who experienced a total of nine bleeding episodes were included. Seven episodes were treated with conventional rFVIIa dosing (72.7-109.1 μg/kg), and two episodes were treated with a single high-dose regimen (254.6-264.0 μg/kg). Clinical and haemostatic responses were evaluated. Haemostasis was assessed by prothrombin time (PT), activated partial thromboplastin time (aPTT), factor VII coagulant activity (FVII:C), TEG, and TGA. Six out of seven (85.7%) bleeding episodes responded to conventional rFVIIa dosing, and half (50%) responded to the high-dose regimen. No relationships between PT, aPTT, and FVII:C levels and clinical outcome were observed. However, changes in TEG and TGA parameters tended to correspond to clinical response, although large inter-individual variation in rFVIIa efficacy was noted. A good response was seen with rFVIIa in treating acute bleeding episodes in patients with haemophilia and inhibitors. Because changes in TEG and TGA may correlate with clinical outcomes of rFVIIa, TEG and TGA may be useful for monitoring rFVIIa activity in inhibitor-positive haemophilia.

Full-dose prophylaxis treatment for persons with haemophilia is not affordable in China due to its economic constraints, particularly in adults requiring higher clotting factor (CFC) doses. Low-dose tertiary prophylaxis for adults with severe haemophilia A (SHA) in Beijing became feasible and implemented when government insurance covering 85% CFC cost in Beijing began in December 2009. The aim of this study was to evaluate the benefits of low-dose tertiary prophylaxis in SHA adults. Analysis of data on 33 patients on low-dose tertiary prophylaxis (5-10 IU/kg, two to three times per week) at the Haemophilia Treatment Center, Peking Union Medical College Hospital between December 2009 and December 2013. The 33 patients (age 18-60 years, mean 33.4) were on prophylaxis for 20.8 ± 9.9 months (compared with prior on-demand therapy for 20.0 ± 11.7 months). Prophylaxis resulted in significant decrease in annual bleeding rate (ABR, 11.8 ± 7.6 vs. 41.5 ± 20.7, 71.1% reduction, P < 0.0001), and significant improvement in Functional Independence Score in Haemophilia (FISH) measurement reflecting improvement in self-care and mobility. Radiologic (Pettersson) joint score was neither improved nor deteriorated. Ten of the 33 patients originally wheel chair and bed-bound began to walk and function independently in their daily lives. Low-dose tertiary prophylaxis for adults with SHA in China is feasible and beneficial. Although the average ABR remained high, a significant improvement in self-care and mobility measured by FISH was observed. These promising clinical experiences form the basis for further formal studies with more defined therapeutic protocol and outcome measures for affordable prophylaxis regimens in haemophilia adults in China.

Introduction: An increased bleeding tendency has been shown in female haemophilia carriers compared to healthy females. Bleeding assessment tools (BATs) have mainly been performed in western cultures. It is unclear how they perform in populations with different healthcare, health/wellness concepts and awareness, as well as family planning practices. Aim: To (a) describe and compare the bleeding symptoms in carriers with healthy females, particularly for bleeding after surgical abortion and intrauterine device (IUD) placement which are performed frequently for family planning in China; (b) quantify scores of International Society on Thrombosis and Haemostasis Bleeding Assessment Tool (ISTH-BAT) and Chinese-BAT (C-BAT) developed to include surgical abortion and IUD placement as separate categories in Chinese haemophilia carriers;(c) correlate bleeding scores (BS) with factor levels. Methods:We conducted a multicentre, cross-sectional study on obligate haemophilia carriers and healthy controls using ISTH-BAT and C-BAT. Results:We enrolled 125 haemophilia carriers and 106 controls. Carriers, compared to controls, had significantly higher median BS (3 vs 1 by both ISTH-BAT and C-BAT) and lower factor level (63.5 vs 101.8 IU/dL). Bleeding after surgical abortion and IUD placement was significantly associated with carrier status. Bleeding scores from neither ISTH-BAT nor C-BAT showed significant correlation with factor levels. Conclusion: Haemophilia carriers in China experienced abnormal bleeding. Uniqueto the Chinese carriers is significant bleeding after surgical abortion (3rd highest incidence of bleeding symptom) and IUD placement (4th highest). However, both ISTH-BAT and C-BAT exhibited no correlation between BS and factor levels in this population and neither could identify carriers with low factor level (of <50 IU/dL).

Severe haemophilia A is a hereditary disease with clotting factor VIII (FVIII) <1%. These severe patients suffer spontaneous bleeding in muscles and joints. 1,2 Recurrent joints bleeding can lead to arthropathy. Iron and haemoglobin deposited on the surface of the joint synovium activate inflammatory reactions, leading to synovitis. 3,4 As a result, the joint is more prone to recurrent bleeding, becoming a target joint. Eventually, cartilage and subchondral bone damage develop. These pathogenic processes result in fibrotic joints, loss of joint function, and even disability, which seriously reduces the quality of life (QOL) of the affected children with severe haemophilia. 5,6 In the past, due to the limitation of drug sources and economic conditions, most children with haemophilia in China were treated on

Haemophilia nursing roles continue to develop alongside nursing as a profession. There are now nurses who practice autonomously, much like a medical practitioner, and many who have extended their roles to deliver direct patient care, education and research. There has been little, if any, comparison with haemophilia nurse roles internationally, nor of the impact of these roles on patient reported outcomes. This paper reports the results of an international survey, of 297 haemophilia nurses from 22 countries, describing current day practice and care. Many nurses work above and beyond their funded hours to improve care through research and evidence-based practice. While some are able to attend international meetings to report and discover this evidence, many due to financial constraints, are not. Others reported difficulty with communicating in English, which limited congress attendance. With on-line learning capability, sharing of best practice is now possible, and this approach should be a platform developed in coming years to further enhance haemophilia nursing practice and ultimately patient care.

Introduction Physiotherapy plays a very important role in comprehensive care of patients with haemophilia. Due to limited resources and a lack of understanding among medical personnel, physiotherapy has not become a standard component of comprehensive management of haemophilia in China. Aim To investigate the understanding and practice of physiotherapy in the comprehensive management of haemophilia by non‐physiotherapy department medical staff engaged in haemophilia work in China. Methods A specifically designed questionnaire was used to investigate the medical staff who participated in the 10th World Federation of Hemophilia—Hemophilia Conference China held in Xi'an, China, from 14 October to 16 October 2016. Results Among all respondents, 97.2% believed that haemophilia patients needed physiotherapy. However, only 17.5% of the respondents were found to have a very accurate understanding of the timing of physiotherapeutic interventions for haemophilia patients; 83.9% of the respondents worried that physiotherapy would increase the incidence of new haemorrhagic episodes in haemophilia patients, and 11.1% of the respondents never recommended physiotherapy for haemophilia patients. Factors that independently affected the respondents’ knowledge of the timing of physiotherapy in haemophilia patients were their professional rank and participation in continuing education that included haemophilia physiotherapy. One factor that independently affected respondents’ recommendation of physiotherapy to patients was participation in continuing education that included haemophilia physiotherapy. Conclusion Haemophilia continuing education covering physiotherapy should be strengthened for new non‐physiotherapy medical staff in the haemophilia field.

Background: In countries with limited resource, haemophilia patients have to choose low-dose prophylaxis or on-demand treatment (ODT) because of economic constraints. Whether low-dose prophylaxis can achieve better joint function outcome than ODT over long-term remains unclear. Aim: To investigate the long-term effect of low-dose tertiary prophylaxis versus ODT on joint health in severe haemophilia A children. Methods: This was a retrospective study. We enrolled and followed 34 severe haemophilia boys in China receiving on-demand treatment (n = 18) or low-dose prophylaxis (10-15 IU/kg, 2-3 times per week) for a medium-term (6-18 months, n = 9) or longer-term (19-30 months, n = 7). We evaluated their haemophilia joint health score (HJHS) 2.1 and functional independence score in haemophilia (FISH) at baseline and at their 6-year follow-up visits. Their annual bleeding rate (ABR) and annual joint bleeding rate (AJBR) were also recorded. Results: During the 6-year follow-up period, ABR and AJBR were similar between the 2 prophylaxis groups, with each of the 2 prophylaxis group rates being significantly better (lower) than the ODT group (P < .05). Compared to baseline values, evaluation at 6-year follow-up showed HJHS improvement in both prophylaxis groups, although significantly (P < .05) only in the longer-term prophylaxis group. The FISH score showed insignificant change in patients in each prophylaxis cohort, compared to significant worsening (P < .05) in the ODT group. Conclusion: Low-dose tertiary prophylaxis reduced ABR and AJBR of children with severe haemophilia and better maintained their functional independence by the FISH over the long term. Longer-term prophylaxis also improved their joint health status by the HJHS.

Introduction Women and girls with haemophilia (WGH) may have spontaneous/traumatic bleeding similar to that in males with haemophilia, and in addition excessive bleeding during menstruation and delivery. Aim To characterize WGH in China and provide guidance for better management. Methods We retrospectively analysed the characteristics of WGH registered in the Haemophilia Treatment Center Collaborative Network of China (HTCCNC) Registry, including demographics, diagnosis and treatment, bleeding characteristics, obstetrical and gynaecological experiences, and surgical history. Results A total of 61 females had confirmed haemophilia. Diagnosis and treatment were typically delayed, longer in mild haemophilia than in severe and moderate. The most frequently reported bleeding manifestations were haemarthrosis in severe and moderate patients, and cutaneous bleeding in mild patients. Among 45 postmenarcheal WGH, 21 (46.7%) had history of heavy menstrual bleeding, but only three received treatments. Prenatal diagnosis and management of perinatal haemorrhage were inadequate. Of 34 deliveries in 30 women, nine deliveries were complicated by postpartum haemorrhage, and 22 offspring carried mutations causing haemophilia. Forty‐four surgical procedures were performed in 29 patients. Those procedures receiving preoperative coagulation factors coverage were significantly less likely to have excessive bleeding than those who did not (P = .003). Conclusion This is the first and largest study describing WGH in China. There are currently deficiencies in the identification, diagnosis, and management of these patients. Improving health insurance policies, establishing haemophilia centres, and multidisciplinary teams for bleeding and perinatal or perioperative management will help reduce morbidity and mortality.