Management Of Hemophilia Patients Research Articles

Occupational therapy in the management of haemophilia patients: A scoping review.

Haemophilia is a haematological disease, although most haemorrhages occur in the locomotor system. Patients are physically disabled from an early age and have a poorer perception of quality of life. In the day-to-day lives of patients and their families, psychosocial well-being, the disease's physical, personal, and social impact, as well as work-related problems are the most complicated aspects of the disease that need to be addressed. To identify the role of occupational therapy in managing patients with haemophilia and to analyse the therapeutic potential of occupational therapy in treating these patients. A scoping review was conducted to identify the role of occupational therapy in managing patients with haemophilia and to analyse the therapeutic potential of occupational therapy in treating these patients. The review was registered in the international registry PROSPERO (Id: CRD42022319637). The databases consulted were SCOPUS, PubMed, PsycINFO, Web of Science and Science Direct, including all studies published until 14 August 2023. No single study was found that specifically developed an occupational therapy intervention for patients with haemophilia. Measurement instruments have been identified, specific for patients with haemophilia and generic, that can be useful for the functional evaluation of these patients in the occupational therapy approach. Different studies showed the importance of multidisciplinary treatment, including occupational therapy. The use of occupational therapy could be effective in improving autonomy and quality of life in haemophilia patients. Therefore, it is of paramount importance to conduct research studies within the field of occupational therapy.

Translated article] Consensus recommendations for the improvement of inter- and intra-centre care coordination in the management of hemophilia

ObjectiveDefine consensus recommendations to improve care coordination between Hospital Pharmacy, Hematology and Nursing, inter- and intra-center, in the care of hemophilia patients. MethodRecommendations for the improvement of care coordination in the management of hemophilia patients were identified and assessed by a multidisciplinary panel of professionals with experience in this field (Hospital Pharmacy, Hematology and Nursing) and supported by scientific evidence. The identified recommendations were assessed by Rand/UCLA consensus methodology (Delphi-adapted) based on their appropriateness and, subsequently, on their necessity. In both cases, it was used ordinal Likert scale. Data were statistically analyzed through different metrics. ResultsFifty-three recommendations for the improvement of care coordination between Hospital Pharmacy, Hematology and Nursing in the management of hemophilia patients were identified, grouped into eight areas of action: i) Hemophilia units, reference centers and multidisciplinary care; ii) Role of Hematology, Hospital Pharmacy and Nursing in the patient journey of hemophilia patients; iii) Telepharmacy and telemedicine; iv) Pharmacokinetic monitoring; v) Transition to adult patient regimen; vi) Patient health education; vii) Surgery, emergency room and hospital admission; and viii) Outcome evaluation. All recommendations were assessed as appropriate and necessary by the external expert panel. ConclusionsHemophilia patient journey is complex and depends on different variables. It also requires the involvement of different healthcare professionals who must act in a coordinated and integrated manner at all stages of the patient's life, adapted to their individual needs. On this matter, the identified and agreed recommendations may improve continuity and quality of care, as they facilitate the integration and coordination of the professionals involved in the management of this pathology, especially Hospital Pharmacy, Hematology and Nursing.

Consenso de recomendaciones para la mejora de la coordinación asistencial inter e intra-centros en el abordaje de la hemofilia

ObjectiveDefine consensus recommendations to improve care coordination between Hospital Pharmacy, Haematology and Nursing, inter- and intra-center, in the care of haemophilia patients. MethodRecommendations for the improvement of care coordination in the management of haemophilia patients were identified and assessed by a multidisciplinary panel of professionals with experience in this field (Hospital Pharmacy, Haematology and Nursing) and supported by scientific evidence. The identified recommendations were assessed by Rand/UCLA consensus methodology (Delphi-adapted) based on their appropriateness and, subsequently, on their necessity. In both cases, it was used ordinal Likert scale. Data were statistically analysed through different metrics. ResultsFifty-three recommendations for the improvement of care coordination between Hospital Pharmacy, Haematology and Nursing in the management of haemophilia patients were identified, grouped into eight areas of action: i) Haemophilia units, reference centers and multidisciplinary care; ii) Role of Haematology, Hospital Pharmacy and Nursing in the patient journey of haemophilia patients; iii) Telepharmacy and telemedicine; iv) Pharmacokinetic monitoring; v) Transition to adult patient regimen; vi) Patient health education; vii) Surgery, emergency room and hospital admission; and viii) Outcome evaluation. All recommendations were assessed as appropriate and necessary by the external expert panel. ConclusionsHaemophilia patient journey is complex and depends on different variables. It also requires the involvement of different healthcare professionals who must act in a coordinated and integrated manner at all stages of the patient's life, adapted to their individual needs. On this matter, the identified and agreed recommendations may improve continuity and quality of care, as they facilitate the integration and coordination of the professionals involved in the management of this pathology, especially Hospital Pharmacy, Haematology and Nursing.

EE180 Budget Impact Model of Feiba in the Management of Hemophilia Patients With Inhibitors in the Egyptian Public Healthcare System

EE180 Budget Impact Model of Feiba in the Management of Hemophilia Patients With Inhibitors in the Egyptian Public Healthcare System

Knowledge and Awareness regarding Dental Management of Hemophilic Patients among Students of a Malaysian University: A Cross-Sectional Study

Background: Improper management of bleeding disorders in the dental setting can lead to complications that can endanger the lives of such patients. Objective: This study aimed to assess the knowledge and awareness of dental management of hemophilia among undergraduate students of a Malaysian university. Methodology: A cross-sectional survey was conducted in the year 2018-2019 to collect the data. A 23-item questionnaire developed and validated by the authors was used to collect data on the knowledge and awareness regarding dental management of hemophilia patients. A total of 216 dental students participated in this research. Unpaired t-test and one-way ANOVA were used to compare the knowledge score between gender and academic year. Results: Only 21% of the participants were confident in treating a hemophilia patient. About 97% of the participants believed that the dental curriculum must be modified to emphasize the dental management of bleeding disorders. The overall mean of the knowledge score was 47.38% and awareness was 73.7% regarding the management of patients with hemophilia. Conclusion: The study revealed significant scope for improvement in the dental students' knowledge, which can be achieved by modifying the dental curriculum suitably to provide more emphasis on the management of bleeding disorders.

An Update on the Dental Management of Children with Haemophilia.

Children with haemophilia present a bleeding risk and a challenge for dentists. Guidelines on the dental management of haemophilia patients are largely based on expert consensus. Many existing guidelines also provide generic guidance mainly for adult patients, which have been adapted for children. However, children have unique needs that require additional considerations. With limited evidence available, it is important that dentists have an understanding of the principles of both medical and dental management and have a close collaboration with the haematologist at all times. Therefore, this paper provides some key principles related to various aspects of dental management of children with haemophilia. Furthermore, there has been a recent update to the World Federation of Haemophilia (WFH) Guidelines for the Management of Haemophilia,1 with references to novel medical therapies for haemophilia. Hence, this paper also aims to inform dentists with the standard and newer medical therapies for haemophilia, including a specific focus on the novel agent Emicizumab and the associated dental considerations.

Assessment of Chromolena odorata potential ability of hemostasis in patients with and without Haemophilia A Inhibitors

Haemophilia is a chronic bleeding illness that causes several difficulties in the patient's daily life in various aspects. Inhibitor development is presently the most important treatment complication observed in patients with hemophilia. Treatment modalities of Haemophilia A with either plasma or recombinant FVIII regimes results in the development of inhibitors and the risk of complications in patients. Hence, newer treatment strategies involving innovative hemostatic therapy are warranted for better management of hemophilia patients. The aim and objective of this study was to assess the efficacy of Chromolena odorata mediated hemostasis in patients with hemophilia A inhibitors. It was a cohort study constituting 147 clinically diagnosed hemophilic A patients. All the patients underwent treatment with FVIII A regimes (specific or multiple). The severity of hemophilia and clotting time was assessed by standard FVIII and aPTT assays, respectively. Modified Bethesda assay method was used to detect FVIII inhibitors. The hemostatic capacity of Chromolena odorata was evaluated by pooling the patient’s plasma with Chromolena extract concentrate. Younger age group (15-30) were more prevalent (56.4%) among hemophilia pathogenesis. Hemarthrosis was the significant co-morbidity observed in 58.5% of patients. Majority of the patients (72.7%) were diagnosed with severe hemophilia A. We identified 10 clinically evident patients to possess inhibitors for FVIII. Evaluation of Chromolena odorata hemostatic potential showed the improvement of clotting capacity with optimal concentrations of extract in patients with inhibitors. The findings of the study revealed the hemostatic properties of Chromolena odorata that can be recommended in the treatment strategies of Haemophilia patients.

Extended Half-Life Coagulation Factors: A New Era in the Management of Hemophilia Patients

Despite effective factor replacement and various treatment schedules, there remain several challenges and unmet needs in the prophylactic treatment of hemophilia limiting its adoption and thereby posing an increased risk of spontaneous bleeding. In this regard, extended half-life (EHL) recombinant factor VIII (rFVIII) and factor IX (rFIX) products promise optimal prophylaxis by decreasing the dose frequency, increasing the compliance, and improving the quality of life without compromising safety and efficacy. EHL products might lead to higher trough levels without increasing infusion frequency, or could facilitate the ability to maintain trough levels while reducing infusion frequency. This paper aims to provide a comprehensive review of the rationale for developing EHL coagulation factors and their utility in the management of hemophilia, with special emphasis on optimal techniques for half-life extension and criteria for defining EHL coagulation factors, as well as indications, efficacy, and safety issues of the currently available EHL-rFVIII and EHL-rFIX products. Potential impacts of these factors on quality of life, health economics, and immune tolerance treatment will also be discussed alongside the challenges in pharmacokinetic-driven prophylaxis and difficulties in monitoring the EHL products with laboratory assays.

Implementation of a hemophilia management program improves clinical outcomes

IntroductionHemophilia is a rare, inherited, coagulation disorder that impairs the body's ability to cease bleeding, making hemophiliacs susceptible to bleeding episodes. These patients often require multiple doses of clotting factor concentrates to help maintain hemostasis. In fiscal year (FY) 2016, Rush University Medical Center's drug expense for clotting factor concentrates totaled $4.6 million, which accounted for approximately 15% of the total drug budget. The Department of Pharmacy developed a Hemophilia Management Program (HMP) to improve care, reduce spending, and expand clinical pharmacy services. Prior to the HMP, the Department of Pharmacy completed drug acquisition and dispensing and no role existed for pharmacists as part of the comprehensive care team for the hemophilia population. The primary objective of this study was to evaluate clinical outcomes after implementation of clinical pharmacy services into the management of hemophilia patients in an interdisciplinary HMP.MethodsSeveral areas for improved care and efficiency were identified prior to the implementation of the HMP: interdisciplinary education, clinical management and continuity of care, and formulary management.ResultsIn total, implementation of the HMP resulted in a net savings of $2.7 million for clotting factor expenses for FY 2017. The cost savings based on clotting factor expenses resulted in a return on investment (ROI) of 20:1, exceeding the initial projections of a modest ROI of 3:1. In addition, these costs savings were realized with the average length of stay per patient staying within the previous margins. Finally, the HMP effectively reduced the need for blood transfusions per patient.DiscussionDesign and implementation of the HMP resulted in significant cost savings, improved efficiency, and enhanced clinical services for patients with hemophilia. This expansion of pharmacy services has become the first pharmacy‐led program in the country to not only reduce costs, but also improve clinical outcomes.

PSY1 - Efficacy and Inhibitor Development of Prophylactic vs On-Demand Factor VIII and IX in the Management of Haemophilia Patients from Southeast Asia: A Systematic Review

PSY1 - Efficacy and Inhibitor Development of Prophylactic vs On-Demand Factor VIII and IX in the Management of Haemophilia Patients from Southeast Asia: A Systematic Review

A Generic Version of Recombinant FVIIa Is Similar to the Branded Product (NovoSeven)

Background: Commercially available recombinant factor VIIa (Novoseven) is widely used in the management of hemophilia patients with inhibitors. Recently several generic versions of recombinant VIIa (rFVIIa) have become available. The generic versions of rFVIIa are claimed to be biosimilar to the barnded Novoseven (Novo Nordisk, Copenhagen, Denmark). The purpose of this study is to compare the US and European Novoseven products with a generic version of rFVIIa namely, Aryoseven (Aryogen, Tehran, Iran).Methods: Four commercially available random lots of Novoseven were obtained from the US and European sources. Four different batches of Aryoseven were obtained from Aryogen. All individual rFVIIa preparations were diluted to obtain working concentrations of 100, 10, 1 and 0.1 ug/ml. Protein content (Lowry's method), molecular profile using surface enhanced laser desorption ionization (SELDI), gel electrophoretic profile (GEP), factor VII related antigen level (FVII:Ag), factor VII correction studies in depleted plasma and thrombin generation (TG) studies were carried out. In addition, VIIa/tissue factor mediated thrombin generation studies were carried out in various prothrombin complex concentrates such as Beriplex and Prothromplex.Results: The protein content and SELDI mass spectrophotometric profile of all 4 rFVIIa preparations were comparable. There was no differences in the Novoseven obtained from the US and European sources. The GEP of the two groups of agents showed a comparable profile with distinct peaks at 50 KDa and 25 KDa. The FVII related antigen levels were also comparable in the Novoseven and Aryoseven preparations. Supplementation of both the Novoseven and Aryoseven preparations at 10 and 100 ug/ml resulted in a comparable correction of the factor VII deficient plasma as measured by PT(INR). Thrombin generation was comparable in the branded and generic product.Conclusions: These results demonstrate that the US and European Aryoseven are comparable. Four batches of Novoseven and 4 individual clinical batches of Aryoseven were found to be comparable. When the US purchased Novoseven preparation was compared with the European Novoseven product, no differences were noted. Thus, the generic Aryoseven is biosimilar to barnded Novoseven and warrant in vivo validation studies. DisclosuresNo relevant conflicts of interest to declare.

Antibody profile in Indian severe haemophilia A patients with and without FVIII inhibitors

Diagnosis and management of haemophilia patients with inhibitors is often tricky due to the heterogeneous nature of the antibodies with regard to their kinetics, as well as the co-existence of other interfering antibodies. Plasma samples from severe haemophilia A patients from India with and without FVIII inhibitors were analysed for the presence of possible co-existing antibodies such as lupus anticoagulants (LA), anti-cardiolipin antibodies (ACLA), anti-β2-glycoprotein-I (anti-β2-GP-I) antibodies, viral transfusion transmitted disease (HIV, HBsAg, HCV) related antibodies, anti-cyclic citrullinated peptides (anti-CCP), and anti-nuclear antibodies. A high incidence of LA and anti-HCV antibodies was detected in Indian haemophilia A patients similar to earlier reports. More importantly, a relatively high incidence of autoantibodies to nuclear antigens (18.62%) and anti-CCP antibodies (1.38%) associated with autoimmune disorders was also seen in these congenital haemophilia A patients with and without inhibitors. Knowledge on the antibody profile in these haemophilia patients especially in those with FVIII inhibitors along with correlation with the clinical manifestations and other risk factors for inhibitor development could possibly shed more light on the complex immune response in these patients.

Management of Hemophilia Patients in Dentistry

Medically compromised patients are occasionally dealt with in dentistry. Sometimes patients with bleeding and clotting disorders may also be encountered in our day to day practice. Hemophilia being one of those clotting disorder which is commonly seen in males whereas females are carriers of the disease. The management of such patients in various specialties of dentistry is discussed in this article.

Utility of a Wireless, Handheld Monitoring System in the Management of Hemophilia Patients

Optimal management of hemophilia requires precise monitoring and thorough compliance with prescribed treatment. However, keeping track of these data using traditional paper log books is often challenging for young patients. We conducted a pilot study with 52 patients at three hemophilia treatment centers to determine whether the use of a wireless, handheld monitoring system would improve patients' record keeping and communication with their healthcare team. At the conclusion of the 3-month testing period, surveys completed by all participating patients and clinicians demonstrated that such a device can facilitate and enhance both patient self-management and communication between clinician and patient.

The need for speed in the management of haemophilia patients with inhibitors

Rapid control of bleeding is the key to reducing bleeding complications and thereby preserving joint and musculoskeletal function in haemophilia patients with inhibitors. However, this requires early diagnosis following the onset of bleeding and strategies for rapid treatment in an outpatient setting. Overarching themes on the need for speed in managing bleeds in haemophilia patients were examined by a panel of clinicians experienced in managing inhibitor patients and joint disease during the Third Zürich Haemophilia Forum on 8 May 2009. This report summarizes the opinions of the panel on how to achieve rapid bleeding control in inhibitor patients and areas that were identified by the panel for future research or as needing new consensus guidelines. The consensus was that home treatment should be established for haemophilia patients with inhibitors, as it is associated with a faster time to treatment, as well as improvements in the quality of life of patients and their carers. In addition, as improved haemostatic control now allows inhibitor patients to participate in a wider range of physical activities, specific guidelines are required on which types of sport and work are appropriate. It was agreed that clear, systematic approaches are needed for early diagnosis of joint and muscle bleeds in inhibitor patients, which could facilitate rapid treatment. There may be opportunities for exploiting new diagnostic techniques from osteoarthritis to enable earlier diagnosis of haemophilic arthropathy. Overall, it was concluded that greater emphasis should be placed on education and patients' psychological needs, to enable inhibitor patients to cope up more effectively with their disease.

The use of central venous catheters in haemophilia patients

Summary. Central venous access devices (CVADs) play an important role in the management of haemophilia patients requiring repeated and/or urgent administration of coagulation factor concentrates. In this article, we summarize current knowledge regarding the use of central venous catheters in these patients, indicating advantages and disadvantages of both fully implantable and external tunnelled CVADs. Finally, we describe our personal experience on the use of the external tunnelled catheter Broviac.

Optimising immune tolerance induction strategies in the management of haemophilia patients with inhibitors: a cost-minimisation analysis

ABSTRACTBackground: The objective of this study was to model the economic impact of optimal dosing of immune tolerance induction (ITI) in haemophilia patients with inhibitors. Evidence based research suggests that, in the right patient population characterised by ITI risk status, the use of high-dose ITI regimen in ‘poor risk’ patients, and the low-dose regimen in ‘good risk’ patients, would be the cost effective strategy. The model also explored the impact of anamnestic response (AR), a phenomenon which worsens patients’ ITI risks.Method: A cost-minimisation technique was used to compare the cost of managing inhibitor patients whose bleeds were managed pre-ITI with an immunogenic bypassing agent, activated prothrombin complex concentrate (APCC), with patients previously on a non-immunogenic product, recombinant activated factor VII (rFVIIa). Patients were subsequently offered a low-dose or high-dose ITI regime depending on their ITI risk status. The study perspective was that of the United Kingdom NHS, hence, all resources used were based on UK costs.Results: The model estimated the mean cost of managing inhibitor patients from detection of titres through ITI to be £959 250.39 and £770 834.17 in the APCC and rFVIIa treatment options, respectively. Meanwhile, the costs per effectively tolerised patients were £1 505 279 and £1 196 706 for APCC and rFVIIa treated patients, respectively. Of the incremental cost in the APCC-treated patients in the model, £129 367 (68%) represents additional ITI cost attributable to anamnestic response to earlier treatment with an immunogenic bypassing agent (APCC).Conclusion: The study concludes that decreasing factor VIII usage during ITI, through the identification and management of ‘good risk’ ITI patients with low-dose protocol, while managing ‘poor risk’ patients with high-dose regimen, will significantly lower the cost of ITI. Furthermore, avoiding AR prior to ITI by using non-immunogenic bypassing agents to manage spontaneous bleeds also has the potential for significant cost savings.

The Economic Impact of Anamnestic Response in the Management of Hemophilia Patients with Inhibitors Undergoing Immune Tolerance Induction in Germany.

The aim of this analysis was to estimate the economic impact of bleed control treatments prior to immune tolerance induction (ITI) in patients with hemophilia with inhibitors in Germany. “Poor risk” hemophilia patients presenting at ITI are more costly to manage as they require higher-dose factor VIII treatment over a longer period, compared to “good risk” patients. Thus, factors that improve patient risk status for ITI, and permit the lowering of factor usage during ITI while maintaining clinical efficacy, are expected to result in cost-effective outcomes. The phenomenon of anamnestic response (AR) which occurs following treatment of hemophilia bleeds with immunogenic products results in significant elevation of titer levels, conferring a “poor risk” status on these patients at ITI. We therefore hypothesize that the prevention of AR pre-ITI, by avoiding immunogenic agents, would result in fewer “poor risk” patients requiring high-dose regimen during ITI, resulting in a cost-effective outcome. A cost-minimization model compared two strategies for the management of patients with severe hemophilia A with inhibitors. One strategy involved management of bleeds with a bypassing agent (activated prothrombin complex concentrates; aPCC) prior to ITI, while the other involved management of bleeds by use of a product which does not provoke AR (recombinant activated factor VII; rFVIIa) prior to ITI. Data on resource use and probabilities of clinical outcomes were derived from published literature, and the model validated by clinical experts with experience in managing inhibitor patients. The mean cost of ITI in inhibitor patients previously treated with rFVIIa is €1,073,327 compared to €1,420,825 for patients previously treated with aPCC. The model also estimated the incremental cost attributable to anamnesis in the management of a hemophilia patient who has undergone ITI to be €186,665. Sensitivity analyses suggest that the model is robust to the price of aPCC, but sensitive to the proportion of patients with AR post-aPCC treatment and the cost and dosage of FVIII at ITI. Anamnestic response is an unpredictable but avoidable consequence of managing bleeding episodes in inhibitor patients and confers significant additional costs to the management of high-responding inhibitor patients undergoing ITI. Avoiding AR prior to ITI by managing spontaneous bleeding episodes with bypassing agents which do not provoke AR has the potential for significant cost savings.

Management of haemophilia patients in Thailand

Management of haemophilia patients in Thailand

Haemophilia therapies

In the last few decades dramatic improvements in the management of haemophilia patients have occurred. Haemophilia has moved from a fatal or disabling disease to a hereditary disorder with available treatment and much better clinical outcomes. The safety of antihaemophilic factor concentrates has been dramatically improved and, in a multidisciplinary environment including haematologists, orthopaedic surgeons, paediatrics, infectiologists, specialised nurses and physiotherapists, complications related to haemophilia are now limited, markedly improving the quality of life of haemophiliacs. One can even think that the cure of haemophilia through gene therapy migth occur in the next decades. Keeping this ultimate aim in mind, efforts at present are mainly focused on bioengineered Factor VIII/Factor IX concentrates with increased efficacy or longer half-life or decreased immunogenicity. In addition, several preclinical and clinical studies are being carried out for optimising and individually tailoring the therapeutic regimens of antihaemophilic therapies using global haemostasis tests in combination with the routine coagulation assays.