Comparison of the efficacy and safety of 12‐month low‐dose factor VIII tertiary prophylaxis vs on‐demand treatment in severe haemophilia A children

Chozie, Novie Amelia; Primacakti, Fitri; Gatot, Djajadiman; Setiabudhy, Rahajuningsih D.; Tulaar, Angela Bm; Prasetyo, Marcel

doi:10.1111/hae.13770

Cited by 27 publications

(44 citation statements)

References 29 publications

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“…Prophylactic infusion of factor concentrate is the standard of care for adult and pediatric patients with severe hemophilia A and B, as well as those with a clinically severe bleeding phenotype, despite moderate or mild deficiency by laboratory assessment, to prevent life‐threatening bleeding and reduce chronic disability from recurrent intramuscular bleeding and hemarthrosis 1‐3 . Even countries with extremely limited access to factor concentrates have leveraged use of strategies, including population pharmacokinetics (popPK) modeling, to facilitate low‐dose prophylaxis with demonstrated musculoskeletal benefit in children 4‐6 . The introduction of several engineered factor VIII (FVIII) and factor IX (FIX) clotting factor concentrates through use of Fc‐fusion, PEGylation, and albumin fusion has created a class of factor concentrates with an extended half‐life (EHL), compared to conventional or standard half‐life (SHL) products 7‐12 .…”

Section: Current Management Of Hemophiliamentioning

confidence: 99%

2021 clinical trials update: Innovations in hemophilia therapy

Croteau

Wang²,

Wheeler

2020

American J Hematol

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Therapies engineered to prolong clotting factor protein circulation time, manipulate the balance of pro‐coagulant and anti‐coagulant proteins, or introduce new genetic material to enable endogenous factor protein production dominate the clinical trial landscape of hemophilia. The availability of clotting factor concentrates and the establishment of primary prophylaxis have dramatically improved health outcomes for hemophilia patients. But, the burden of hemostatic therapy remains significant, and many barriers to consistent longitudinal use of prophylaxis exist. Several types of emerging therapeutics including engineered factor concentrates, substitutive therapies, rebalancing therapies, and gene transfer/editing all aim to reduce the challenges of current hemophilia treatment. Emerging treatment options may reduce treatment frequency or need for intravenous administration. They may also introduce new challenges in laboratory assessment of hemostasis. These novel therapies must not introduce significant new health risks and continue to support similar or improved outcomes. The potential ramifications of rebalancing the coagulation cascade, particularly in a stress or inflammatory state, or introduction of new genetic material are not trivial. The focus of this review is to provide an overview of active and recently completed clinical trials as well as emerging preclinical data investigating new therapeutic possibilities for hemophilia patients and potentially other rare bleeding disorders.

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Section: Current Management Of Hemophiliamentioning

confidence: 99%

2021 clinical trials update: Innovations in hemophilia therapy

Croteau

Wang²,

Wheeler

2020

American J Hematol

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“…Low‐dose prophylaxis was shown to reduce bleeding rates by 75%‐80% with substantial gains in quality of life in terms of daily activities, school and job attendance, and community engagement. The benefits of low‐dose prophylaxis were confirmed by studies in other economically constrained economies, including India, 20 Thailand, 21 Indonesia 22 and Iran 23 . The annual mean factor consumption of China's low‐dose prophylaxis regimen is approximately 1000 IU/kg/y, 19 far less than the 5000‐6000 U/kg/y based on the standard dose.…”

Section: Achievements In the Clinical Evaluation And Treatment Of Haementioning

confidence: 93%

Haemophilia care in China: Achievements in the past decade

2020

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There is a large population of people with haemophilia in China. However, the development of haemophilia care in China lagged far behind developed countries and was unbalanced across different regions. With the establishment of the Hemophilia Treatment Center Collaborative Network of China (HTCCNC) in 2004 and its collaboration with the Chinese government, the World Federation of Haemophilia (WFH), non-profit foundations, industries, and experts from other countries, haemophilia care in China have made considerable strides in building capacity. The HTCCNC helped develop clinics throughout the country, promote multidisciplinary care, promote prophylaxis (albeit low-dose), advance clinical evaluation and treatment, and research. With many collaborative efforts in the past decade, haemophilia care in China has entered a new stage of development. In this review, we summarize these major achievements and efforts within the last decade.

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“…Many countries such as China, India, Tunisia, Thailand, Indonesia, Algeria and Egypt practise low‐dose prophylaxis as a primary, secondary or tertiary prophylaxis.…”

Section: Low‐dose Prophylaxis Studiesmentioning

confidence: 99%

Show me the evidence: Effectiveness of low‐dose prophylaxis

Gouider

2020

Haemophilia

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Prophylaxis is the gold standard for haemophilia treatment, by reducing bleeding events and improving joint status and quality of life. 1 In fact, it does require more amount of clotting factor concentrates than for on-demand treatment, and so a higher cost, that could represent a barrier to introduce prophylaxis in countries with limited resources. In emerging countries, efficiency of prophylaxis was also demonstrated. 2 If we consider the history of prophylaxis, low-dose prophylaxis is the first step of prophylaxis. 3 In the 1960s, Swedish started prophylaxis with low doses, and then, the evolution of the regimen led to gradually intensified prophylaxis protocols, with the aim of zero bleed, and early start. Low-dose prophylaxis is an alternative that could be effective and recommended for countries with limited resources. 4 Is low-dose prophylaxis acceptable nowadays? In countries where on-demand therapy is the treatment option, because full-dose prophylaxis could not be applied, low-dose prophylaxis should be considered, at least for children, as a first step. There are data of evidence showing the superiority of low-dose prophylaxis than episodic treatment. Low-dose prophylaxis has positive outcomes with improvement of quality of life and reduction ofbleeding events. Moreover, Srivastava 4 reported calculations of no increase of used clotting factor concentrates. Outcome measurements should be used in order to have objective data of the effectiveness of low-dose prophylaxis. Annual bleeding rate and Haemophilia Joint Health score are the easier and cost effective tools. Many countries such as China, 5 India, 6 Tunisia, 7 Thailand, 8 Indonesia, 9 Algeria and Egypt practise low-dose prophylaxis as a primary, secondary or tertiary prophylaxis. Published studies on low-dose prophylaxis have shown superiority of such protocol over episodic treatment in terms of bleed reduction, and quality of life, with improved physical activity, inde-Correspondence AbstractProphylaxis is the gold standard treatment for haemophilia but requires more amount of clotting factor concentrates, than on demand therapy. Low dose prophylaxis is an alternative for countries with limited resources. There are data of evidence showing the superiority of low-dose prophylaxis than episodic treatment. Studies from China, India, Tunisia, Thailand and Indonesia reported experiences with low dose prophylaxis using outcome assessment. These studies have shown the effectiveness of various protocols regimen with once, twice or thrice injection of 10-15 UI Kg-1 per injection. These protocols allow reduction of joint bleeds and at least delay of joint damages. There is not enough long-term data nowadays, but low dose prophylaxis is certainly better than on demand therapy and should be considered as a first step of prophylaxis in some countries but not the final goal. K E Y W O R D SLow dose prophylaxis, Hemophilia, Prophylaxis, Hemophilia joint health score

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Comparison of the efficacy and safety of 12‐month low‐dose factor VIII tertiary prophylaxis vs on‐demand treatment in severe haemophilia A children

Cited by 27 publications

References 29 publications

2021 clinical trials update: Innovations in hemophilia therapy

2021 clinical trials update: Innovations in hemophilia therapy

Haemophilia care in China: Achievements in the past decade

Show me the evidence: Effectiveness of low‐dose prophylaxis

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