A patient satisfaction survey for haemophilia treatment centres

The importance of patient satisfaction has continued to grow such that patient satisfaction is now viewed as a vital component of health-care delivery. This is evidenced by the expanding body of research in the area and the use of measures of patient satisfaction as indicators of health-care quality. The value of patient satisfaction is particularly apparent in the setting of chronic disease where medical care utilization is high, compliance with therapy is critical and the patient-provider relationship is often long-term. Although several validated tools exist to quantify general measures of patient satisfaction, there is a recognized need for disease-specific instruments. Not only are there issues that are unique to haemophilia, but many patients receive care via a specialized comprehensive clinic model. The authors were unaware of an instrument that could adequately address patient satisfaction issues specific to haemophilia; thus, they undertook to develop one. The patient satisfaction survey presented here contains fixed-choice, Likert-scale and open-ended questions adapted from previously validated questionnaires. Assessment of face validity and internal consistency indicate that the survey is measuring one underlying construct - patient satisfaction. Information acquired through this survey will provide a quantitative assessment of patient satisfaction within a clinic population of persons with bleeding disorders and could be used to guide decisions regarding provision of health-care services.     

A survey of factor prophylaxis in boys with haemophilia followed in North American haemophilia treatment centres

Summary.  A survey was conducted in 2002 to determine the pattern of factor prophylaxis use in boys ≤18 years of age with haemophilia followed in North American treatment centres. Responses were obtained from 4553 cases (74% haemophilia A, 26% haemophilia B). The frequency of prophylaxis, defined as factor infusion greater than or equal to once per week for ≥45 weeks per year, was significantly higher for haemophilia A vs. haemophilia B cases (51% vs. 32%, P < 0.0001), and for boys with severe haemophilia A living in Canada vs. the USA (77% vs. 47%, P < 0.0001). Use of full-dose prophylaxis, defined as the infusion of 25–40 IU kg⁻¹ of factor VIII on alternate days (minimum three times per week) or 25–40 IU kg⁻¹ of factor IX twice weekly, was similar for boys ≤5 years of age in both Canada and the USA (30% and 33% haemophilia A and 35% and 13% haemophilia B). Reasons for initiating prophylaxis included a history of joint bleeding (88%) and age ≤2 years (23%). For prophylaxis triggered by joint bleeding, 38% of haemophilia treatment centres indicated that they would initiate prophylaxis after the first joint bleed and 66% after a history of target joint bleeding, defined most frequently as 2–4 bleeds over a 3–6 consecutive month period. A central venous line was used to ensure easy venous access for full-dose prophylaxis therapy in 80% of boys ≤5 years of age. These data offer a basis for projecting long-term factor concentrate needs for persons with haemophilia living in North America.     

Identifying non‐responsive bleeding episodes in patients with haemophilia and inhibitors: a consensus definition

Summary. Assessing response to treatment with bypassing agents presents a substantial challenge in the treatment of patients with haemophilia and inhibitors. Rapid and accurate identification of bleeding episodes that are non‐responsive to bypassing therapy with either Factor Eight Inhibitor Bypassing Activity (FEIBA; Baxter AG) or recombinant activated factor VII (rFVIIa; NovoSeven^®, Novo Nordisk A/S) is essential to guide treatment decisions and optimize patient outcomes through early intervention. Although both bypassing agents are effective, differential responses to therapy necessitate multiple therapeutic options. This article provides a consensus definition for non–life‐threatening joint and muscle bleeds that are non‐responsive to bypassing agents. An international panel of seven physicians met in December 2008 to develop the consensus definition using a modified National Institutes of Health Consensus Development Conference method. The consequent definition of non–life‐threatening bleeding episodes that are non‐responsive to bypassing treatment provides a global picture of the condition of the patient during such an event. Identification of non‐responsiveness is based on various criteria: pain, swelling/tension, mobility, patient perception and laboratory parameters. Criteria can be assessed subjectively by the patient/parent and/or objectively by the clinician. Although the precise timing of each determination should be at the discretion of the physician, bleeds should be considered non‐responsive if the clinical situation meets the specified criteria 24 h from the start of treatment. Although it is not intended to replace clinical judgment, this definition can guide the optimal course of treatment for patients with haemophilia and inhibitors.     

Consensus recommendations for the use of FEIBA® in haemophilia A patients with inhibitors undergoing elective orthopaedic and non‐orthopaedic surgery

A growing number of publications have described the efficacy and safety of FEIBA as a first‐line haemostatic agent for surgical procedures in haemophilia A patients with high‐responding FVIII inhibitors. The aim of this study was to provide practical guidance on patient management and selection and also to communicate a standardized approach to the dosing and monitoring of FEIBA during and after surgery. A consensus group was convened with the aims of (i) providing an overview of the efficacy and safety of FEIBA in surgery; (ii) sharing best practice; (iii) developing recommendations based on the outcome of (i) and (ii). To date there have been 17 publications reporting on the use of FEIBA in over 210 major and minor orthopaedic and non‐orthopaedic surgical procedures. Haemostatic outcome was rated as ‘excellent’ or ‘good’ in 78–100% of major cases. The reporting of thromboembolic complications or anamnestic response to FEIBA was very rare. Key to the success of FEIBA as haemostatic cover in surgery is to utilize the preplanning phase to prepare the patient both for surgery and also for rehabilitation. Haemostatic control with FEIBA should be continued for an adequate period postoperatively to support wound healing and to cover what can in some patients be an extended period of physiotherapy. Published data have demonstrated that FEIBA can provide adequate, well tolerated, peri and postoperative haemostatic cover for a variety of major and minor surgical procedures in patients with haemophilia A. The consensus recommendations provide a standardized approach to the dosing and monitoring of FEIBA.     

Combined treatment with APCC (FEIBA®) and tranexamic acid in patients with haemophilia A with inhibitors and in patients with acquired haemophilia A – a two‐centre experience

The management of bleeding in haemophilia patients with inhibitors can be challenging when using monotherapy with either activated prothrombin complex concentrate (APCC) or recombinant activated FVII (rFVIIa) fail. The antifibrinolytic agent tranexamic acid (TXA) increases clot stability and is used concomitantly with coagulation factor replacement to improve haemostasis in haemophilia patients without inhibitors in many countries in Europe. Combined treatment with TXA and rFVIIa is not contraindicated in haemophilia patients with inhibitors. However, the combined approach of TXA and APCC has not been investigated due to safety concerns of increased risk of thrombosis or disseminated intravasal coagulation (DIC). The aim of this study is to report our experience of concomitant use of APCC and TXA in haemophilia A patients with inhibitor and in patients with acquired haemophilia A with respect to safety and efficacy. Seven (n = 6) haemophilia A patients with inhibitors and one (n = 1) with acquired haemophilia A from Oslo (Norway) and Stockholm (Sweden) were included in the study. The APCC was given at doses consistent to the manufacturers' recommendation. TXA was administered concomitantly either 10 mg kg(-1) every 6-8 h intravenously or 20 mg kg(-1) every 6-8 h orally. Haemostatic response was assessed by thromboelastography (TEG) and thrombin generation assay (TGA) in three of the patients. A total number of three bleeding episodes and two minor and six major surgical procedures were performed under the coverage with APCC and TXA. Haemostatic outcome was rated excellent or good in 10 of 11 (91%) treatment episodes. One episode was rated with poor effect. No episodes of arterial, venous thrombosis or DIC occurred during or after the treatment. Data from TEG and TGA analysis showed no signs of hypercoagulability following the combined treatment. This report demonstrates that, in a limited number of patients, combined treatment with APCC and TXA seemed to be safe, tolerated and relatively effective in management of bleeding episodes and in preventing haemorrhage during surgery in haemophilia patients with inhibitors and in a patient with acquired haemophilia A. Further studies should be performed to confirm these data.     

Home treatment of haemophilia patients with inhibitors

Summary. Bleeding episodes in patients with inhibitors can be challenging to treat. Clinical guidelines recognize the importance of early treatment, ideally within 2 h of the onset of bleeding. On‐demand haemophilia care at home has been shown to reduce the time between recognition of the symptoms of bleeding and initiation of treatment. Rapid resolution of bleeding is associated with longer‐term benefits for the patient. Effective haemophilia care at home depends on patients and carers taking greater responsibility for treatment; however, many find this difficult. Education can help raise awareness of haemophilia treatment at home and provide helpful information for patients/carers. The haemophilia nurse has a key role in providing this support and education. This review discusses a number of recent guidelines and educational materials for haemophilia home care identified during a literature survey. The survey shows that most materials were not validated. In addition, the survey shows limited effectiveness data on techniques for training haemophilia patients about home care. Further education resources and research in the treatment of haemophilia at home are required.     

Practice patterns in haemophilia A therapy – a survey of treatment centres in the United States

A survey was conducted to ascertain practice patterns for haemophilia A therapy in the United States. Questionnaire data were supplied by 52 haemophilia centres with a total of 4129 patients under treatment. Most participating centres were affiliated with academic/teaching hospitals or institutions. Patients below 5 years comprised 17% of the study population, 6-18 years 41% and >18 years 42%, and the apportionment across severity categories was 53% severe, 17% moderate and 30% mild. Among patients with severe haemophilia, 49% were receiving on-demand treatment, while 44% were receiving some form of prophylaxis (13% primary, 20% secondary and 11% tertiary). Primary prophylaxis was the most common type in children below 5 years of age, accounting for 25% of this age group. In children 6-18 years old, 58% were on some type of prophylactic regimen, while on-demand treatment was most frequent among adults. Difficulties of venous access were the most frequently cited barrier to instituting prophylaxis. Catheters were in use among 37% of the patients under primary prophylaxis and 14% of those on secondary prophylaxis. No major differences were observed in forms of therapy used between larger and smaller centres. These observations provide an extensive characterization of haemophilia A practice patterns in the United States.     

A survey of patients with acquired haemophilia in a haemophilia centre over a 28‐year period

Data of patients with acquired inhibitors to clotting factors seen in a haemophilia centre from 1970–98 was collected. Twenty‐four patients with anti‐factor VIII antibodies and four with acquired von Willebrand disease case records were evaluable. Two‐thirds of the patients were females and their age ranged from 2 to 92 years (median 69). There was no correlation between the inhibitor titres and the severity or the pattern of bleeding. Porcine factor VIII and activated prothrombin complex were both effective for acute bleeds. Prednisolone and cyclophosphamide proved valuable for suppression of the antibodies without adverse effects even in the elderly group of patients. Three deaths were directly related to bleeding. This review has shown the life‐threatening nature of this acquired bleeding disorder, its variable clinical presentation and the importance of early referral to a specialist centre for appropiate therapy.