Management Of Severe Hemophilia Research Articles

Management of severe hemophilia with inhibitors to factor VIII with recurrent retroperitoneal bleed

Management of severe hemophilia with inhibitors to factor VIII with recurrent retroperitoneal bleed

Homoeopathic Management of Severe Haemophilia in Pediatric age group: Twin Case Study

Both the patients were relieved of their other complaints with homoeopathy.

Assessing Patient and Provider Perspectives, Clinical Practice, Behaviors, and Knowledge on Hemophilia A Care

BACKGROUND: The management of severe hemophilia A with and without inhibitors is evolving rapidly. Clinicians who manage patients with hemophilia A may lack confidence in how to optimally utilize extended half-life factor replacement agents as well as non-factor replacement agents for prophylaxis, and in managing complications such as inhibitor development in those with severe hemophilia. Patients with hemophilia and their families are burdened by the complexity of managing their care and would benefit from improved understanding of the disease state, treatment options, how to decrease bleeding episodes, and optimize their quality of life. For these reasons, a web-based joint educational initiative for patients/caregivers and HCPs was created. METHODS: In collaboration with Hemophilia Federation of America, National Hemophilia Foundation, and National Organization for Rare Disorders, a 1-hour online video-based CME activity for HCPs and 1-hour healthcare education activity for patients were created in June, 2020 to address identified practice and knowledge needs among HCPs, and knowledge, communication and self-efficacy behaviors among patients. Each activity consisted of slides, polling and live questions, and remains on-demand for 12 months at www.OMedLive.com and RareDiseaseLive.com, and on Facebook video for patients and caregivers. Test questions were administered at 3 time points (pre-, immediate post-, and 2 mos. post-activity). Data from these test questions, responses to live polling questions, and learner-submitted questions during live Q&A were analyzed to determine engagement, lessons learned and continuing education gaps. RESULTS: As of August 2020, 235 HCPs and 2,384 patients took part in the ongoing activities. Post education, HCP learners (n=79) anticipated the education would positively impact patient clinical outcomes (84%) and their practice behavior (80%). Improvements in pre/post knowledge and case-based competence questions were realized in all 4 administered HCP pre/post questions. For patients and caregivers, knowledge improvements were observed in 3 out of 4 pre/post questions, and a shift was also observed towards preference for more shared models of decision making, and towards increased confidence with HCP communication and managing their own care/the care of the person for whom they care. Differences were observed in patient versus provider perceptions of hemophilia-related challenges. Behavioral changes indicated by patients and providers following the educational activities will be reported and grouped by theme. Two-month follow up post-tests will be administered to HCPs and patient-caregivers who took part in the educational programs, and statistical significance of change and effect size for all repeated test questions will be reported. CONCLUSIONS: Joint CME and patient education can yield insights into clinical practice behaviors; patient healthcare communication, preferences, and confidence; and treatment knowledge of Hemophilia A. Together, aligned education directed to HCPs and patients can improve the ability to provide evidence-based treatment for patients. Disclosures Croteau: Bayer: Consultancy; Genentech: Consultancy; Pfizer: Consultancy; Novo Nordisk: Research Funding; CSL-Behring: Consultancy; Spark Therapeutics: Research Funding; ATHN: Research Funding; Sigilon Therapeutics: Consultancy; National Hemophilia Foundation: Honoraria; Hemophilia Federation of America: Honoraria. Pipe:Apcintex, Bayer, BioMarin, Catalyst Biosciences, CSL Behring, HEMA Biologics, Freeline, Novo Nordisk, Pfizer, F. Hoffmann-La Roche Ltd/Genentech, Inc., Sangamo Therapeutics, Sanofi, Takeda, Spark Therapeutics, uniQure: Consultancy; Siemens: Research Funding; Medical and Scientific Advisory Council to the National Hemophilia Foundation; Medical Advisory Board to World Federation of Hemophilia: Membership on an entity's Board of Directors or advisory committees. Tickle:National Hemophilia Foundation: Other, Research Funding; Platform Q Health: Honoraria.

Challenges in Management of Severe Hemophilia with Acute Hematologic Malignancies

Childhood Leukemia treatment is one of the most common malignancies seen across the globe in the pediatric age group. Severe Hemophilia A is still considered a rare bleeding disorder. Only 13 patients with hemophilia A or B have been reported in literature to also be diagnosed with acute leukemia in childhood . This rarity appeared again in a 13 year boy with severe hemophilia A who presented with worsening bone pain and joint swelling, weight loss and leukocytosis. Morphology and molecular diagnostics confirmed Acute Pre B Lymphoblastic Leukemia . The patient happens to be the first case of Acute Lymphoblastic Leukemia in a patient with severe Hemophilia with Inhibitors. Severe hemophilia with inhibitors pose challenge in clinical management given their propensity of bleeding and poor response to traditional therapies due to a neutralizing antibody as is. Treatment of acute lymphoblastic leukemia requires an intensive treatment with systemic and intrathecal chemotherapy. Medications are commonly administered through a central line, in many cases an implantable catheter. Coexistence of both these life threatening disorders posed a unique practical challenge in providing standard care and our discussion aims at highlighting strategies in management of Severe Hemophilia in challenging clinical scenarios. Disclosures No relevant conflicts of interest to declare.

Tailored Prophylaxis in Mexican Pediatric Patients with Hemophilia a By Determining the Pharmacokinetics of Factor VIII

Introduction. Prophylactic replacement of factor VIII (FVIII) is the gold standard for the management of severe hemophilia, preventing bleeding and its complications. One of the most commonly used methods in developing countries is the Swedish regimen that consist in the administration of 25-40 IU/kg of FVIII three times per week. However, the actual required levels may vary considerably, these depend on the dose administered, the dosage interval and the pharmacokinetic profile of the patient. Pharmacokinetic calculations based on bayesian estimation are useful for designing an optimal dosage of the minimum required factor levels for each individual patient, nevertheless in our country it is not accessible to all patients mainly due to the cost of the multiple samples needed to obtain the pharmacokinetic profile. This report describes our experience determining the pharmacokinetics of FVIII in a cohort of pediatric patients with hemophilia A in order to establish a personalized prophylaxis and to compare the infusion frequency and factor consumption with the dosage regimen previously mentioned. Methods. Pediatric patients with severe and moderate hemophilia A were included, with one or more years in prophylaxis and monthly follow-up at the hemophilia clinic of our centre. Patients with inhibitors and/or documented renal and/or hepatic alterations were excluded. We administered 50 U/kg of FVIII to all patients and 4 blood samples were collected for the estimation of FVIII phamacokinetics (0, 4, 24 and 48 hours after the application). FVIII was measured using one-stage assay. The FVIII pharmacokinetics of each patient were obtained using the WAPPS-Hemo app. Results. The pharmacokinetics of FVIII were determined in 10 male patients (9 severe, 1 moderate), with a median age of 9 years (2-13) and a mean BMI of 18.6 (15.1-23.6). One patient was excluded for having an atypical t1/2 for FVIII, not within the population curve, in him the existence of an inhibitor was suspected and later corroborated. We observed an average estimated time of 70.6 hr (53.5-89.5) in which the FVIII concentration reached <1% and an average half-life of 9.3 hr (6.25-14.25). Using the clinical calculator of the application, a prophylactic regimen was obtained for each patient in order to maintain FVIII trough levels ≥1%. In 22.2% the factor dose was not modified and in the remaining 77.8% it was necessary to elevate the dose with an average increase of 23.2 IU / kg / week. Conclusions. Tailored prophylaxis is easy to obtain with the currently available digital tools. In our study, unlike what is reported in previous similar studies, almost 80% of patients required an increase in the dose of factor according to their pharmacokinetics profile. We also found a clearance time lower than that reported in other populations groups maily based in adults cases, but very similiar to what was previously reported in a pediatric population from southern Mexico. Most of our patients had not reported clinically significant bleeding, but it is possible that they had had subclinical bleeding during the time they maintained a trough level of FVIII <1% with their previous prophylactic regimen. The impact of the implementation of prophylaxis by pharmacokinetics and its cost-effectiveness is currently being evaluated prospectively. Disclosures No relevant conflicts of interest to declare. Disclosures Gomez-Almaguer: Celgene: Consultancy, Speakers Bureau; Takeda: Consultancy, Speakers Bureau; Teva: Consultancy, Speakers Bureau; Janssen: Consultancy, Speakers Bureau; Amgen: Consultancy, Speakers Bureau.

Management of severe haemophilia A during surgery: case report

Abstract We describe the case of a patient with severe haemophilia A and significant comorbidities who underwent surgery to remove a large intra-abdominal haematoma first diagnosed 12 years previously. The haemophilia team was instrumental in coordinating care, building a strong rapport with surgical and other medical teams to manage bleeding risk with continuous infusion of factor VIII (FVIII). Medical teams adjusted their working hours according to clinical need. Haemophilia nurses were available to offer support at all times, and developed management procedures and educated staff on haemophilia and its treatment. Perioperative complications included a thrombus occluding the right internal jugular vein, infection and paraesthesia. However, surgery was effective and the patient was pleased with the overall outcome. Haemophilia nurses established strong and rewarding relationships with other teams that will enhance the delivery of care in the future.

Treatment decision-making among Canadian youth with severe haemophilia: a qualitative approach.

The first generation of young men using primary prophylaxis is coming of age. Important questions regarding the management of severe haemophilia with prophylaxis persist: Can prophylaxis be stopped? At what age? To what effect? Can the regimen be individualized? The reasons why some individuals discontinue or poorly comply with prophylaxis are not well understood. These issues have been explored using predominantly quantitative research approaches, yielding little insight into treatment decision-making from the perspectives of persons with haemophilia (PWH). Positioning the PWH as a source of expertise about their condition and its management, we undertook a qualitative study: (i) to explore and understand the lived experience of young men with severe haemophilia A or B and (ii) to identify the factors and inter-relationships between factors that affect young men's treatment decision-making. This manuscript reports primarily on the second objective. A modified Straussian, grounded theory methodology was used for data collection (interviews) and preliminary analysis. The study sample, youth aged 15-29, with severe haemophilia A or B, was chosen selectively and recruited through three Canadian Haemophilia Treatment Centres. We found treatment decision-making to be multi-factorial and used the Framework method to analyze the inter-relationships between factors. A typology of four distinct approaches to treatment was identified: lifestyle routine prophylaxis, situational prophylaxis, strict routine prophylaxis and no prophylaxis. Standardized treatment definitions (i.e.: 'primary' and 'secondary', 'prophylaxis') do not adequately describe the ways participants treat. Naming the variation of approaches documented in this study can improve PWH/provider communication, treatment planning and education.