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2.
Biochem Biophys Res Commun ; 628: 49-56, 2022 Nov 05.
Artigo em Inglês | MEDLINE | ID: mdl-36081278

RESUMO

The coagulation factor 9 gene (FIX) point mutation contributes to most hemophilia B cases, providing ideal gene correction models. Here we identified the frequent mutation G20519A (R226Q) in FIX, which resulted in many severe and moderate hemophilia B patients. This study aimed to investigate the effect of HDR and base editing in correcting FIX mutant. We first constructed HEK293 and liver-derived cell lines Huh7 cells stabling carrying mutated FIX containing G20519A (HEK293-FIXmut and Huh7-FIXmut). Then, CRISPR/Cas9-based homology-directed repair (HDR) and base editing were used for the correction of this mutated point. We used Cas9 nickase (nCas9) mediated HDR and the advanced base editor ABE8e to correct G20519A and then measured the concentration and activity of FIX. Furthermore, we used the star-shaped poly(lysine) gene nanocarriers to deliver the ABE8e correction systems into HEK293-FIXmut and Huh7-FIXmut stem cells to correct mutated FIX. As a result, we found that gRNAs directed inefficient HDR in correcting G20519A. The ABE8e corrected the mutation efficiently in both HEK293-FIXmut and Huh7-FIXmut stem cells. In addition, the star-shaped poly(lysine) carriers delivered non-viral vectors into stem cells efficiently. The nanocarriers-delivered ABE8e system corrected mutated FIX in stem cells, and the stem cells secreted active FIX in high concentration. In conclusion, our study provides a potential alternative for correcting mutated FIX in hemophilia B patients.


Assuntos
Hemofilia A , Hemofilia B , Fatores de Coagulação Sanguínea/genética , Fatores de Coagulação Sanguínea/metabolismo , Sistemas CRISPR-Cas/genética , Desoxirribonuclease I/metabolismo , Edição de Genes/métodos , Células HEK293 , Hemofilia A/genética , Hemofilia A/metabolismo , Hemofilia B/genética , Hemofilia B/terapia , Humanos , Mutação , Mutação de Sentido Incorreto , Polilisina , Células-Tronco/metabolismo
4.
Lancet Haematol ; 9(9): e689-e697, 2022 Sep.
Artigo em Inglês | MEDLINE | ID: mdl-36055333

RESUMO

Highly effective treatment of haemophilia A and B is primarily available to 15% of the world's population, in high-income countries. In low-income countries (LICs) and lower-middle-income countries (LMICs), morbidity and mortality are high because of greatly reduced access to diagnosis, care, and treatment. We report the challenges and impact after the first 5 years (mid-2015-2020) of the expanded World Federation of Hemophilia (WFH) Humanitarian Aid Program (HAP). WFH HAP donated coagulation products were used to treat more than 250 000 acute bleeding episodes, manage approximately 4000 surgeries, and establish bleeding preventive prophylaxis in about 2000 patients in 73 countries. Health-care providers worldwide learned optimal management of patients with complex needs through virtual and in-person training. In response to the programme, some governments increased investment in haemophilia care, including independent purchases of small amounts of treatment products. With unparalleled scope and complexity, and substantial benefits to people with haemophilia and society in general, the WFH HAP is an exemplar of partnership between for-profit and not-for-profit organisations advancing health-care equity in LICs and LMICs, which could be replicated by other organisations supporting people with different monogenic diseases.


Assuntos
Hemofilia A , Socorro em Desastres , Países em Desenvolvimento , Hemofilia A/epidemiologia , Hemorragia , Humanos , Renda
5.
Hum Gene Ther ; 33(17-18): 879-888, 2022 Sep.
Artigo em Inglês | MEDLINE | ID: mdl-36082993

RESUMO

Gene therapy is an exciting therapeutic concept that offers the promise of a cure for an array of inherited and acquired disorders. The liver has always been a key target for gene therapy as it controls essential biological processes including digestion, metabolism, detoxification, immunity, and blood coagulation. Metabolic disorders of hepatic origin number several hundreds, and for many, liver transplantation remains the only cure. Liver-targeted gene therapy is an attractive treatment modality for many of these conditions. After years of failure, substantial progress in this field in the past decade has resulted in promising clinical efficacy and safety in patients with monogenetic disorders with Valoctocogene roxaparvovec (Roctavian), the first gene therapy for treatment for hemophilia A, to be approved in Europe. Another, Etranacogene dezaparvovec (AMT-061) for hemophilia B is also in the final stages of approval. A number of other liver targeted gene therapy products are at an advanced stage of development, thus heralding a new era of potentially curative molecular medicine. This review explores the recent clinical advances in liver targeted gene therapy as well as the challenges that need to be overcome for the widespread adoption of this new treatment paradigm.


Assuntos
Hemofilia A , Hemofilia B , Terapia Genética/métodos , Vetores Genéticos , Hemofilia A/genética , Hemofilia A/terapia , Hemofilia B/genética , Hemofilia B/terapia , Humanos , Fígado
7.
PLoS One ; 17(9): e0273775, 2022.
Artigo em Inglês | MEDLINE | ID: mdl-36084067

RESUMO

BACKGROUND: Several innovative drugs liable to lead to changes in healthcare organization are or soon will be available for the management of hemophilia. Analyzing their implementation can shed further light on healthcare decision-making, to anticipate changes and risk of breakdown in the patient's care pathway. METHODS: Multiple criteria decision analysis (MCDA), based on ISPOR recommendations, was used to assess the organizational impact of innovation in hemophilia care management. The MCDA process designed for this specific context involved ten French experts in hemophilia care management (physicians, nurses, pharmacist, physiotherapist and psychologist) in the hemophilia care center of Chambéry, in the Rhône-Alpes Region of France. This pilot study involved seven steps: (i) defining the decision problem; (ii) selecting and structuring criteria; (iii) assessing the relative weight of each criterion with software-assisted simulation based on pairwise comparisons of different organizational change scenarios; (iv) measuring the performance of the selected innovations; (v) scoring alternatives; (vi) calculating aggregate scores; (vii) discussion. The endpoint was to determine the expected overall organizational impact on a 0-100 scale. RESULTS: Seven organizational criteria were selected. "Acceptability for patient/caregiver/association" was the most heavily weighted. Factor VIII by subcutaneous route obtained the highest aggregate score: i.e., low impact on care organization (88.8 out of 100). The innovation with strongest organizational impact was gene therapy (27.3 out of 100). CONCLUSION: This approach provided a useful support for discussion, integrating organizational aspects in the treatment decision-making process, at healthcare team level. The study needs repeating in a few years' time and in other hemophilia centers.


Assuntos
Hemofilia A , Procedimentos Clínicos , Técnicas de Apoio para a Decisão , Hemofilia A/terapia , Humanos , Inovação Organizacional , Projetos Piloto
8.
Am J Case Rep ; 23: e937312, 2022 Sep 18.
Artigo em Inglês | MEDLINE | ID: mdl-36116005

RESUMO

BACKGROUND Combined factor V and factor VIII deficiency (F5F8D) is a rare bleeding disorder with an incidence of 1: 1 000 000. The identified mutations were observed in LMAN1 and MCFD2 genes. This case report presents the cases of 3 Saudi siblings with the genetic mutation of LMAN1 causing F5F8D, and highlights the challenges in diagnosis and treatment. CASE REPORT Patient X, a 7-year-old boy, was misdiagnosed with hemophilia A after a history of prolonged circumcision bleeding and epistaxis. He was referred to our clinic for pre-operative assessment. Blood workup showed prolonged PT and aPTT, which were normalized by mixing studies. Since his previous diagnosis could not explain a prolonged PT, further investigations were performed, revealing low levels of FVIII and FV. Genetic testing confirmed a c.822G>A homozygous LMAN1 mutation. The other 2 siblings (patient Y and Z), who were 5- and 12-year-old, respectively, girls, were also assessed. They both had a history of epistaxis. The younger sibling also had an episode of bleeding after tooth extraction, and physical examination of this patient revealed a bruise over her left thigh. The older sibling had menorrhagia. Blood workup of both revealed prolonged PT and aPTT, with complete correction by mixing study, and low levels of FV and FVIII. The patients' backgrounds and lab results were highly suggestive of F5F8D. CONCLUSIONS This case report describes an extremely rare bleeding disorder. More attention should be directed toward this disease, and a careful evaluation of suspicious cases should be performed to better diagnose and manage these patients.


Assuntos
Fator V , Irmãos , Criança , Pré-Escolar , Epistaxe/etiologia , Fator V/genética , Deficiência do Fator V , Feminino , Hemofilia A , Humanos , Masculino , Lectinas de Ligação a Manose/genética , Proteínas de Membrana/genética , Mutação , Arábia Saudita , Proteínas de Transporte Vesicular/genética
9.
BMC Med Res Methodol ; 22(1): 215, 2022 08 05.
Artigo em Inglês | MEDLINE | ID: mdl-35931967

RESUMO

BACKGROUND: Long-term prophylactic therapy is considered the standard of care for hemophilia A patients. This study models the long-term clinical and cost outcomes of two factor VIII (FVIII) products using a pharmacokinetic (PK) simulation model in a Chinese population. METHODS: Head-to-head PK profile data of BAY 81-8973 (KOVALTRY®) and antihemophilic factor (recombinant) plasma/albumin-free method (rAHF-PFM, ADVATE®) were applied to a two-state (alive and dead) Markov model to simulate blood FVIII concentrations at a steady state in prophylactically-treated patients with hemophilia A. Worsening of the Pettersson score was simulated and decline was associated with the probability of having orthopaedic surgery. The only difference between the compounds was FVIII concentration at a given time; each subject was treated with 25 IU/kg every 3 days. The model used a lifetime horizon, with cycle lengths of 1 year. RESULTS: Cumulative bleeding events, joint bleeding events, and major bleeding events were reduced by 19.3% for BAY 81-8973 compared to rAHF-PFM. Hospitalizations and hospitalization days were also reduced by 19.3% for BAY 81-8973 compared to rAHF-PFM. BAY 81-8973 resulted in both cost savings and a gain in quality adjusted life years (QALYs) compared to rAHF-PFM. CONCLUSION: Based on modeled head-to-head comparisons, differences in PK-properties between BAY 81-8973 and rAHF-PFM result in a reduced number of bleeding events, leading to reduced costs and increased quality of life for BAY 81-8973. These results should be used to inform clinical practice in China when caring for patients with severe hemophilia A.


Assuntos
Fator VIII , Hemofilia A , Atenção à Saúde , Fator VIII/farmacocinética , Fator VIII/uso terapêutico , Hemofilia A/complicações , Hemofilia A/tratamento farmacológico , Hemorragia/tratamento farmacológico , Hemorragia/prevenção & controle , Humanos , Qualidade de Vida , Proteínas Recombinantes/efeitos adversos , Proteínas Recombinantes/farmacocinética , Albumina Sérica/uso terapêutico
10.
BMC Surg ; 22(1): 303, 2022 Aug 06.
Artigo em Inglês | MEDLINE | ID: mdl-35933357

RESUMO

BACKGROUND: Cerebral infarction (CI) is an unusual complication in patients with bleeding disorders. To our knowledge, this is the first case of postoperative internal border-zone infarction (I-BZI) from Hemophilia A. CASE PRESENTATION: We present a case of Hemophilia A developing I-BZI, after surgical treatment of giant hemophilic pseudotumor. A 36-year-old man was introduced from other hospital by Hemophilia with giant hemophilic pseudotumor in his left thigh. Patient and his relatives did not have a history of thrombophilia. After excluding the relevant surgical contraindications, we performed the operation of pseudotumor resection. Prior to surgery, blood tests revealed hemoglobin of 137 g/L. FVIII activity was 1.5%. Activated partial thromboplastin time (APTT) was 71.50 s and D-dimer was 3.33 mg/L FEU. Immediately before surgery, the patient received an intravenous infusion of FVIII products (Xyntha®) at a dose of 3500 IU for his body weight of 80 kg. Post-operative day two (POD2), patient developed vomiting, decreased response, and dysarthria. Hemoglobin was 54 g/L with blood pressure of 110/70 mmHg. Magnetic resonance imaging of the brain showed there were multiple acute cerebral infarctions in bilateral lateral ventricles (internal border zone) and multiple ischemic foci in the white matter areas and basal ganglia of the bilateral cerebral hemispheres. This case suggested that acute severe anemia can be one of the causes of I-BZI. CONCLUSIONS: For the treatment of I-BZI caused by acute anemia from Hemophilia A, volume expansion, red blood cell supplement and continuous improvement of coagulation with suitable dose of factor VIII (FVIII) should be considered to improve prognosis.


Assuntos
Hemofilia A , Adulto , Infarto Cerebral/etiologia , Infarto Cerebral/cirurgia , Hemofilia A/complicações , Humanos , Imageamento por Ressonância Magnética , Masculino , Coxa da Perna
11.
BMJ Case Rep ; 15(8)2022 Aug 29.
Artigo em Inglês | MEDLINE | ID: mdl-36038157

RESUMO

We detail the case of a man in his 80s who was a Jehovah's Witness, presenting to hospital for the second time in 1 week with atraumatic, severe bruising affecting his right thigh and flank. He was subsequently diagnosed with idiopathic acquired haemophilia A (AHA) and was urgently treated with recombinant factor concentrate and immunosuppressive therapy. Management of his bleeding disorder and resultant severe anaemia was adapted in line with his religious beliefs. AHA is a rare bleeding disorder which should be considered in patients with an isolated prolonged activated partial thromboplastin time and a history of recent or acute bleeding. Prompt diagnosis and management are essential as delays may result in increased mortality. Given that this patient declined blood transfusion, management of his bleeding disorder presented a unique challenge.


Assuntos
Hemofilia A , Testemunhas de Jeová , Idoso de 80 Anos ou mais , Transfusão de Sangue , Hemofilia A/complicações , Hemofilia A/diagnóstico , Hemofilia A/psicologia , Hemofilia A/terapia , Hemorragia/complicações , Hemorragia/terapia , Humanos , Testemunhas de Jeová/psicologia , Masculino
12.
BMC Pediatr ; 22(1): 487, 2022 08 15.
Artigo em Inglês | MEDLINE | ID: mdl-35965332

RESUMO

PURPOSE: Real-world data and study data regarding therapy with Emicizumab in pediatric cohorts with haemophilia A is scarce. Especially, data on previously untreated pediatric patients (PUPs) and minimally treated patients (MTPs) are missing. METHODS: Thirteen pediatric patients with haemophilia A and treatment with Emicizumab were retrospectively evaluated for Annual Bleeding Rates (ABR) pre-and post-Emicizumab treatment. Safety data and data on management of minor surgery as well as laboratory results were collected. Additionally, we describe the clinical features of two PUPs and one MTP that are included in our cohort. RESULTS: Median age at initiation of Emicizumab was 5.3 (range: 0.26-17.5) years, three patients were younger than one year at initiation of treatment with Emicizumab. Median follow-up time on Emicizumab was 23.8 (range: 0.7-40) months. Total ABR (p = 0.009) as well as spontaneous (p = 0.018), traumatic (p = 0.018), and joint (p = 0.027) ABR reduced significantly post-Emicizumab transition. Safety profile was favourable as only one local site reaction occurred; no cessation of treatment was necessary. Surgery was successfully performed in three patients receiving rFVlla pre- and post-surgery. Emicizumab trough levels showed a median of 43.2 µg/ml (range: 23.9-56.8) after three doses of 3 mg/kg and 51.9 µg/ml (range: 30.4-75) at first follow-up with 1.5 mg/kg. CONCLUSION: Emicizumab is safe and efficient in pediatric patients with and without inhibitors. More data on larger multicenter cohorts and especially on PUPs/MTPs are still needed.


Assuntos
Anticorpos Biespecíficos , Hemofilia A , Anticorpos Biespecíficos/uso terapêutico , Anticorpos Monoclonais Humanizados/uso terapêutico , Criança , Hemofilia A/tratamento farmacológico , Humanos , Estudos Retrospectivos
13.
Biochem Med (Zagreb) ; 32(3): 030801, 2022 Oct 01.
Artigo em Inglês | MEDLINE | ID: mdl-35966257

RESUMO

The acquired hemophilia A (AHA) is a life-threatening condition. The incidence of AHA is extremely low, which requires a multidisciplinary approach to diagnosis and treatment. This is case report of 73-year-old man who presented with AHA secondary to severe acute respiratory syndrome Coronavirus 2 (SARS-CoV-2) pneumonia. The patient had extensive skin bleeding and hematomas. In the coagulation screening tests activated partial thromboplastin time (APTT) was prolonged with normal prothrombin time (PT), which was indication for further investigation. The APTT in a mixing study with normal plasma did not correct so clotting factors inhibitors were suspected. With signs of bleeding, extremely low factor VIII (FVIII) activity (2%) and presence of FVIII inhibitors, AHA was diagnosed and treatment initiated. Patient was treated with factor eight inhibitor bypassing agent (FEIBA) for three days, followed by long-term corticosteroid and cyclophosphamide therapy. Malignant and autoimmune diseases as the most common causes of AHA were ruled out. The patient had a good response to therapy with gradual normalization of APTT and FVIII activity. To the best of our knowledge, the present case is the first reported case of de novo AHA after SARS-CoV-2 pneumonia. The diagnosis of AHA should be suspected in a patient with bleeding into the skin and mucous membranes without a previous personal and family history of bleeding, and with isolated prolonged APTT. It is important to investigate any isolated prolongation of APTT in cooperation with clinical laboratory experts.


Assuntos
COVID-19 , Hemofilia A , Pneumonia , Idoso , COVID-19/complicações , Hemofilia A/complicações , Hemofilia A/diagnóstico , Hemofilia A/tratamento farmacológico , Humanos , Masculino , SARS-CoV-2
14.
Front Immunol ; 13: 894411, 2022.
Artigo em Inglês | MEDLINE | ID: mdl-35967311

RESUMO

Conjugation to polyethylene glycol (PEG) is commonly used to enhance drug delivery and efficacy by extending the half-life of the drug molecule. This has important implications for reducing treatment burden in diseases that require chronic prophylaxis, such as hemophilia. Clearance of PEG molecules with high molecular weights (≥ 40 kDa) has been reported to cause cellular vacuolation in mammals. Rurioctocog alfa pegol (PEGylated recombinant coagulation factor VIII) contains a 20-kDa PEG. This study investigated the effects of exposure to 20-kDa PEG (10 µg/ml to 10 mg/ml) on the morphology and function of human monocyte-derived macrophages (MDMs) in vitro. Exposure to PEG for 24 hours was associated with significant vacuolation only at concentrations of 1 mg/ml or more, which far exceed the levels associated with clinically relevant doses of rurioctocog alfa pegol. Immunofluorescence staining of PEG was detected in the cytoplasm of MDMs, indicating uptake into the cells. No impairment of MDM phagocytic activity (ability to ingest fluorescently labeled Escherichia coli) was observed with 24-hour exposure to PEG, even at concentrations associated with significant vacuolation. Furthermore, PEG exposure did not have significant effects on cytokine secretion in resting or lipopolysaccharide-stimulated MDMs, or on the expression of cell surface markers in stimulated MDMs. Cell viability was not affected by 24-hour exposure to PEG. In conclusion, vacuolation of human MDMs after exposure to 20-kDa PEG only occurred with PEG concentrations far in excess of those equivalent to clinically relevant doses of rurioctocog alfa pegol and did not affect MDM viability or functionality. Together, these results support the concept that PEG-mediated vacuolation is an adaptive cellular response rather than a toxic effect.


Assuntos
Hemofilia A , Polietilenoglicóis , Animais , Hemofilia A/tratamento farmacológico , Humanos , Macrófagos/metabolismo , Mamíferos/metabolismo , Peso Molecular , Polietilenoglicóis/metabolismo , Proteínas Recombinantes/farmacologia , Proteínas Recombinantes/uso terapêutico
15.
Adv Pediatr ; 69(1): 133-147, 2022 Aug.
Artigo em Inglês | MEDLINE | ID: mdl-35985706

RESUMO

Hemophilia A is an inherited insufficiency of Factor VIII (FVIII), one of the critical clotting factors. The gold standard for the management of moderate-to-severe hemophilia A is prophylaxis using regular replacement therapy with clotting factor concentrates. Compared with conventional treatment, extended half-life products reduce the burden of frequent factor replacement injections. Of note, up to 30% of patients with hemophilia A receiving prophylactic factor infusions develop "inhibitors," neutralizing anti-FVIII autoantibodies. Therapeutic options for patients with hemophilia A and inhibitors include the immune tolerance induction (ie, eradication of inhibitors) and the management of acute bleeds with bypassing agents and/or emicizumab. Emicizumab is a biphasic monoclonal antibody mimicking activated FVIII, approved for patients with hemophilia A with/without inhibitors. Gene therapy is an emerging therapy for hemophilia A, essentially curing patients with hemophilia A or transforming them to a milder phenotype by establishing continuous endogenous expression of FVIII after one-time treatment.


Assuntos
Hemofilia A , Hemofilia A/tratamento farmacológico , Humanos , Tolerância Imunológica
16.
Perm J ; 26(2): 153-157, 2022 06 29.
Artigo em Inglês | MEDLINE | ID: mdl-35933672

RESUMO

Introduction Acquired hemophilia A (AHA) is a rare bleeding disorder caused by autoantibodies against clotting factor VIII. It occurs most commonly in the elderly population. AHA is associated with malignancy, autoimmune diseases, pregnancy, and drugs. However, up to 50% of reported cases remain idiopathic. AHA is very rarely associated with multiple sclerosis. Case Presentation A 56-year-old patient with a history of MS presented with thigh and arm swelling and associated pain. Imaging revealed diffuse iliopsoas hematoma. Laboratory studies showed that prothombin time was not elevated and there was prolongation of activated partial thromboplastin time that was not corrected by mixing studies. Clotting factor VIII activity level was reduced, and the inhibitor level was elevated. He was treated with factor eight inhibitor bypassing agents, rituximab, and steroids. Conclusion AHA should be suspected in any patient with new-onset bleeding in the presence of a prolonged activated partial thromboplastin time. Prompt diagnosis and treatment are important as AHA carries a high mortality rate.


Assuntos
Hemofilia A , Esclerose Múltipla , Idoso , Fator VIII/uso terapêutico , Feminino , Hemofilia A/complicações , Hemofilia A/tratamento farmacológico , Hemorragia , Humanos , Masculino , Pessoa de Meia-Idade , Esclerose Múltipla/complicações , Tempo de Tromboplastina Parcial , Gravidez
17.
Zhongguo Shi Yan Xue Ye Xue Za Zhi ; 30(4): 1301-1304, 2022 Aug.
Artigo em Chinês | MEDLINE | ID: mdl-35981403

RESUMO

Traditional replacement therapy is the main treatment method of hemophilia, while inhibitor generation makes replacement therapy ineffective. The emergence of non-factor therapy brings new hope for the treatment of patients with inhibitor. Non-factor products mainly achieve therapeutic purpose by imitating the function of coagulation factor Ⅷ, inhibiting the function of anti-tissue factor pathway inhibitors, the expression of antithrombin mRNA, and the function of activated protein C. This paper reviews the latest research progress of non-factor products in the treatment of hemophilia.


Assuntos
Hemofilia A , Anticorpos Monoclonais Humanizados/efeitos adversos , Fator VIII/uso terapêutico , Hemofilia A/terapia , Humanos
18.
Clin Radiol ; 77(10): 730-737, 2022 Oct.
Artigo em Inglês | MEDLINE | ID: mdl-35985846

RESUMO

Haemophilia is a common hereditary cause of bleeding diathesis and the musculoskeletal system is frequently affected. Repeated episodes of haemarthrosis initiate a cascade towards haemophilic arthropathy, a disabling and deforming joint disease with both degenerative and inflammatory features, which include articular cartilage loss, bone erosions, and synovitis. Haemophilic pseudotumour and intra-muscular haematoma make up the remainder of the musculoskeletal manifestations of this systemic condition. Radiological assessment is vital in the assessment and follow-up of these haemophilic complications and MRI is the reference standard. This article summarises the radiological findings relevant to the diagnosis and monitoring of this complex patient group.


Assuntos
Artrite , Cartilagem Articular , Hemofilia A , Sinovite , Hemartrose/complicações , Hemartrose/etiologia , Hemofilia A/complicações , Hemofilia A/diagnóstico por imagem , Humanos , Sinovite/complicações
20.
Pol Arch Intern Med ; 132(7-8)2022 08 22.
Artigo em Inglês | MEDLINE | ID: mdl-35925049

RESUMO

INTRODUCTION: Patient adherence to a prophylactic regimen is important for optimal benefit of hemophilia treatment. Despite a growing number of adults with hemophilia in Poland receiving secondary prophylaxis, data on adherence to the regimen are limited. OBJECTIVES: The aim of the study was to assess adherence to secondary prophylaxis in Polish adults with severe hemophilia. PATIENTS AND METHODS: Patients were recruited in 18 hemophilia treatment centers in Poland. Adherence to prophylaxis was assessed with the Validated Hemophilia Regimen Treatment Adherence Scale Prophylaxis (VERITAS­Pro) questionnaire. RESULTS: Data on 270 men on the prophylactic regimen (median [interquartile range, IQR] age, 37 [18-75] years; mean [SD], 38.2 [13.3] years) were analyzed. Median (IQR) VERITAS­Pro score for the study population was 36 (24-76) years; mean (SD), 37.7 (9.9) years, indicating general adherence to the prophylactic regimen. The median subscale scores ranged from 4 for Dosing to 8 for Planning (means, 5.6 and 7.7, respectively). The most pronounced difference in the subscale scores between adherent and nonadherent patients was recorded for Dosing (median, 4 vs 10; mean, 5.3 vs 9.3) and Remembering (median, 5 vs 11; mean, 5.7 vs 10.7). The overall adherence rate was 94%. CONCLUSIONS: Our results show a high rate of adherence to hemophilia prophylaxis by Polish adults. Problems with the management of clotting factor stocks and remembering about the injection of the clotting factor were identified as potential barriers to adherence in adults with hemophilia in Poland.


Assuntos
Hemofilia A , Hemofilia B , Adolescente , Adulto , Idoso , Fatores de Coagulação Sanguínea/uso terapêutico , Fator VIII , Hemofilia A/complicações , Hemofilia A/tratamento farmacológico , Hemofilia B/complicações , Humanos , Masculino , Pessoa de Meia-Idade , Polônia , Adulto Jovem
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