Treatment Options in Hemophilia

Miesbach, Wolfgang; Schwäble, Joachim; Müller, Markus; Seifried, Erhard

doi:10.3238/arztebl.2019.0791

Cited by 14 publications

(10 citation statements)

References 58 publications

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“…In recent years, several new therapies for treatment of hemophilia-both factor-and non-factor-based-have received marketing authorization, while more are undergoing clinical trials [30,31] In novel factor products, so-called extended half-life (EHL) products, plasma half-life was prolonged up to 1.5-fold for FVIII and 2.4-4.8-fold for FIX by modification of recombinant FVIII and FIX proteins [32]. EHL products were first introduced to the German market in 2016 (FIX, Alprolix [33] and Idelvion [34]).…”

Section: Inclusion Of New Hemophilia Therapiesmentioning

confidence: 99%

The German Hemophilia Registry: Growing with Its Tasks

Duda

Hesse

Haschberger

et al. 2020

JCM

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Hemophilia is a rare heredity bleeding disorder that requires treatment for life. While few therapeutic options were available in the past, multiple recent breakthroughs have fundamentally altered and diversified hemophilia therapy, with even more new therapeutic options forthcoming. These changes are mirrored by significant regulatory and legal changes, which have redefined the role of hemophilia registries in the European Union (EU). This dual paradigm shift poses new regulatory, scientific but also structural requirements for hemophilia registries. The aim of this manuscript is to enumerate these significant challenges and to demonstrate their incorporation into the redesign of the German Hemophilia Registry (Deutsches Hämophilieregister, dhr). To identify the spectrum of hemophilia therapies and the degree of regulatory changes, a horizon screening was performed. Consequently, a core dataset for the dhr was defined by harmonization with regulatory guidelines as well as other hemophilia registries and by heeding the needs of different stakeholders (patients, clinicians, regulators, and scientists). Based on this information, a new registry structure was established, which is optimized for capturing data on new and established hemophilia therapies in a changing therapeutic and regulatory landscape

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Section: Inclusion Of New Hemophilia Therapiesmentioning

confidence: 99%

The German Hemophilia Registry: Growing with Its Tasks

Duda

Hesse

Haschberger

et al. 2020

JCM

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“…The current treatment for patients with severe hemophilia includes regular administration of coagulation factors as prophylaxis rather than on-demand [ 27 , 28 ]. From a global point of view, the high cost of coagulation factors hinders the availability of this treatment.…”

Section: Discussionmentioning

confidence: 99%

The Application of Focused Medium-Energy Extracorporeal Shockwave Therapy in Hemophilic A Arthropathy

Sheen

Chen

et al. 2022

Healthcare

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Hemophilic arthropathy causes the damage of synovium, cartilage, and subchondral bone. The present study evaluated the safety and the effect of extracorporeal shockwave therapy (ESWT), a safe treatment widely used in musculoskeletal conditions in patients with hemophilic arthropathy. Between 1 August 2019 and 31 July 2020, seven hemophilia A patients were enrolled and treated with medium-energy ESWT on the knee joint in the first two months after prophylactic coagulation factor administration. At the beginning of the study and at 1-, 2-, 3-, and 6-month follow-ups, the Hemophilia Joint Health Score (HJHS), visual analog scale score (VAS), and Hemophilia Early Arthropathy Detection with Ultrasound score (HEAD-US) were evaluated for therapeutic effectiveness and safety, while serum bone morphogenetic protein 2 (BMP-2) and von Willebrand factor (vWF) levels were analyzed for assessing chondroprotection and bone healing. Magnetic resonance imaging (MRI) of the knee was performed at the beginning of the study and the 6-month follow-ups. As a result, a non-significant decrease in VAS scores (p = 0.151) but not HJHS after treatment was noticed. At the 3-month follow-up, there was a non-significant increase in BMP2 levels (p = 0.171) but not vWF. Ultrasonography showed no disease activity score elevation in five patients and no further disease damage in all patients. Repeated MRI examinations in three patients showed no structural progression during the 6-month follow-up. As to adverse events, redness, local heat, and mild swelling were noted in five patients without breakthrough bleeding. We concluded that medium-energy ESWT might be safe for hemophilic arthropathy once prophylactic coagulation factors are administered.

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“…Patients of severe hemophilia are severely de cient in coagulation factors and spontaneous joint or muscle bleeding often occurs. As to current treatment for severe hemophilia patients, coagulation factors are administrated regularly as prophylaxis rather than administrated as on demand [1].…”

Section: Introductionmentioning

confidence: 99%

The Safety of Extracorporeal Shockwave Therapy in Hemophilic Arthropathy

Lo¹,

Sheen²,

Chen³

et al. 2021

Preprint

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BackgroundThe hemophilic arthropathy was caused by repeated joint bleeding, which resulted in joint synovium hyperplasia, cartilage damage and bone deformity. Extracorporeal shockwave treatment (ESWT) is a non-invasive treatment with high safety and has been widely used in the treatment of various musculoskeletal conditions. This study was aimed to evaluate the safety of ESWT in hemophilic arthropathy of the knee.MethodsThis is a prospective single blind randomized control study conducted between 2019/08/01 and 2020/07/31. Hemophilia and Von Willebrand disease patients were enrolled and randomized for extracorporeal shock wave therapy to the left or right knee joint and energy-free sham therapy to the other knee joint at the 1st and 2nd month after prophylactic coagulation factor administration. As safety evaluation, hemophilia joint health score (HJHS), knee score scale (KSS), visual analog scale (VAS) and ultrasound (HEAD-US) were checked at the beginning, 1st, 2nd, 3rd and 6th month. Knee MRI was examined at the beginning and 6th month.ResultsHJHS score of the knee receiving real ESWT did not elevate in 9 patients (75%). KSS demonstrated neither significant improvement nor deterioration after ESWT. VAS declined after two sessions of ESWT. Ultrasound and MRI showed no breakthrough joint bleeding nor progression of previously existed effusion and hemarthrosis.ConclusionESWT might be a safe treatment for hemophilic arthropathy once prophylactic coagulation factors are adequately administrated before ESWT. Further well-controlled study enrolling more hemophilia patients might be needed for the effectiveness of ESWT on these patients.

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Treatment Options in Hemophilia

Cited by 14 publications

References 58 publications

The German Hemophilia Registry: Growing with Its Tasks

The German Hemophilia Registry: Growing with Its Tasks

The Application of Focused Medium-Energy Extracorporeal Shockwave Therapy in Hemophilic A Arthropathy

The Safety of Extracorporeal Shockwave Therapy in Hemophilic Arthropathy

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