Low dose emicizumab prophylaxis in haemophilia a patients: A pilot study from India

Bansal, Shweta; Donners, Anouk A. M. T.; Fischer, Kathelijn; Fischer, Kathelijn; Rangarajan, Savita; Phadke, Varsha; Mhatre, Shrutika; Sontate, Bharati; Silva, Magdelene D'; Ansari, Shahana; Shetty, Shrimati

doi:10.1111/hae.14785

Cited by 14 publications

(4 citation statements)

References 8 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…A previous case series report 22 showed the effectiveness of monthly emicizumab prophylaxis dose as low as 1.05-1.66 mg/kg and even without loading doses, yet their median ABR still decreased from a baseline of 27.0-4.0. In a pilot study from India 23 including eight patients with severe HA on low-dose emicizumab prophylaxis (.84-2.6 mg/kg/4 weeks), all patients had zero treated bleeds. In addition, earlier Japanese 24 and Finnish studies 25…”

Section: Discussionmentioning

confidence: 99%

Reduced doses of emicizumab achieve good efficacy: Results from a national‐wide multicentre real‐world study in China

Xu,

Wang,

et al. 2024

Haemophilia

View full text Add to dashboard Cite

IntroductionReduced doses of emicizumab improve the affordability among patients in developing countries. However, the relationship between variant dose selection and efficacy in the real world of China is still unclear.AimThis study aimed to investigate the efficacy and safety of emicizumab especially in those on reduced dose regimens in a real‐world setting.MethodsWe carried out a multicentre study from 28 hospitals between June 2019 and June 2023 in China and retrospectively analysed the characteristics including demographics, diagnosis, treatment, bleeding episodes, and surgical procedures.ResultsIn total, 127 patients with haemophilia A, including 42 with inhibitors, were followed for a median duration of 16.0 (IQR: 9.0–30.0) months. Median age at emicizumab initiation was 2.0 (IQR: 1.0–4.0) years. Median (IQR) consumption for loading and maintenance was 12.0 (8.0–12.0) and 4.2 (3.0–6.0) mg/kg/4 weeks, respectively. While on emicizumab, 67 (52.8%) patients had no bleeds, whereas 60 (47.2%) patients had any bleeds, including 26 with treated bleeds. Compared to previous treatments, patients on emicizumab had significantly decreased annualized bleeding rate, annualized joint bleeding rate, target joints and intracerebral haemorrhage. Different dosages had similar efficacy except the proportion of patients with treated spontaneous bleeds and target joints. Adverse events were reported in 12 (9.4%) patients. Postoperative excessive bleeding occurred following two of nine procedures.ConclusionThis is the largest study describing patients with HA receiving emicizumab prophylaxis on variant dose regimens in China. We confirmed that nonstandard dose is efficacious and can be considered where full‐dose emicizumab is ill affordable.

show abstract

Section: Discussionmentioning

confidence: 99%

Reduced doses of emicizumab achieve good efficacy: Results from a national‐wide multicentre real‐world study in China

Xu,

Wang,

et al. 2024

Haemophilia

View full text Add to dashboard Cite

show abstract

“…Reduction of the cost to less than 25% of the conventional emicizumab prophylaxis cost with relatively good clinical outcome will allow more patients to be on prophylaxis with this highly effective drug. 18,19…”

Section: Neutralizing Antibodiesmentioning

confidence: 99%

“…In some Asian countries with resource‐constrained settings such as Thailand and India, lower dosage of emicizumab has been used with good clinical efficacy. Reduction of the cost to less than 25% of the conventional emicizumab prophylaxis cost with relatively good clinical outcome will allow more patients to be on prophylaxis with this highly effective drug 18,19 …”

Section: Management Of People With Haemophiliamentioning

confidence: 99%

Haemophilia care in Asia: Learning from clinical practice in some Asian countries

Angchaisuksiri,

Amurao‐Abiera,

Chou

et al. 2024

Haemophilia

View full text Add to dashboard Cite

BackgroundThe healthcare systems in Asia vary greatly due to the socio‐economic and cultural diversities which impact haemophilia management.MethodsAn advisory board meeting was conducted with experts in haemophilia care from Asia to understand the heterogeneity in clinical practices and care provision in the region.FindingsThe overall prevalence of haemophilia in Asia ranges between 3 and 8.58/100,000 patients. Haemophilia A was more prevalent as compared to haemophilia B with a ratio of around 5:1. There is under‐diagnosis in the region due to lack of diagnosis, registries and/or lack of appropriate facilities in suburban areas. Most patients are referred to the haematologists by their families or primary care physicians, while some are identified during bleeding episodes. Genetic testing faces obstacles like resource constraints, services available at limited centres and unwillingness of patients to participate. Prophylaxis is offered for people with haemophilia (PWH) with a severe bleeding phenotype. Recombinant factors are approved in most countries across the region and are the preferred therapy. The challenges highlighted for not receiving a high standard of care include patients’ reluctance to use an intravenous treatment, poor patient compliance due to frequency of infusions, budget constraints and lack of funding, insurance, availability and accessibility of factor concentrates. Prevalence of neutralizing antibodies ranged from 5% to 20% in the region. Use of immune tolerance induction and bypassing agents to treat inhibitors depends on their cost and availability.ConclusionHaemophilia care in Asia has evolved to a great extent. However, some challenges remain for which a strategic approach along with multi‐stakeholder involvement are needed.

show abstract

“…Low-dose emicizumab was effective in patients with HA, with or without inhibitors, who performed low bleeding risk activities [ 52 ]. A recent single-center, pilot study conducted in India on a small group of eight patients with HA showed that emicizumab administration at a low dose of 0.84-2.6 mg/kg once every four weeks was effective in reducing bleed rates throughout the follow-up period of one year; there was no evidence of reported bleeding events during the prophylaxis period [ 53 ]. In this study, the patients were exposed to low-impact physical activities such as swimming, cycling, and walking.…”

Section: Reviewmentioning

confidence: 99%

Expert Opinions on the Management of Hemophilia A in India: The Role of Emicizumab

Gupta,

Dutta,

Ahmed

et al. 2024

Cureus

View full text Add to dashboard Cite

Hemophilia A (HA) is a genetic disorder of hemostasis associated with a deficiency or reduced activity of clotting factor VIII (FVIII). This disorder remains unacceptably underdiagnosed in India. Early diagnosis and appropriate management of HA can substantially prevent morbidity and mortality. Currently, HA is managed with regular replacement therapy using standard or extended half-life FVIII concentrates or non-factor drug products. The challenges associated with FVIII concentrates include plateauing of drug effect, issues with its administration and adherence to treatment, breakthrough bleeds, and the development of inhibiting antibodies against administered clotting factors. Emicizumab is a bispecific antibody, launched in India in April 2019, for managing patients with HA. To investigate the role of emicizumab in Indian patients with HA, opinions were sought from 13 eminent hematologists and experts from India on the effectiveness of emicizumab in preventing all bleeds, spontaneous bleeds, perioperative bleeds, and intracranial hemorrhage; resolving target joints; and reducing the rate of hospitalizations and fatality associated with HA in children and adults, with or without inhibitors. The benefits of emicizumab over traditional FVIII concentrates include the subcutaneous route of delivery, less frequent dosing, and a lack of inhibitor development, in addition to providing sustained hemostasis without in-depth monitoring. It is a safe and effective management option for all HA patients, especially for patients with certain archetypes, such as those with inhibitors, those with high annualized bleed rates, those living far away from hemophilia care centers, pediatric patients and infants with intravenous access challenges, and those with a history of life-threatening bleeding events.

show abstract

Low dose emicizumab prophylaxis in haemophilia a patients: A pilot study from India

Cited by 14 publications

References 8 publications

Reduced doses of emicizumab achieve good efficacy: Results from a national‐wide multicentre real‐world study in China

Reduced doses of emicizumab achieve good efficacy: Results from a national‐wide multicentre real‐world study in China

Haemophilia care in Asia: Learning from clinical practice in some Asian countries

Expert Opinions on the Management of Hemophilia A in India: The Role of Emicizumab

Contact Info

Product

Resources

About