Initial experience of Emicizumab prophylaxis in children with inhibitor positive severe hemophilia A from a single center in India

Radhakrishnan, Nita; Shankar, Ravi; Maji, Manideepa; Rana, Megha

doi:10.1016/j.phoj.2021.03.004

Cited by 7 publications

(6 citation statements)

References 10 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…By February 2022 Twin A and Twin B completed 15 and 9 months of therapy respectively .The average bleed frequency reduced from 10 and 12 bleeds per year to Zero bleeds after initiation of emicizumab prophylaxis and there were no adverse events. The results are similar to the outcomes described in HAVEN 1-4 clinical trials and in studies evaluating real world effectiveness of Emicizumab [9,10].…”

supporting

confidence: 82%

Outcome of Emicizumab prophylaxis in twins with severe hemophilia A and inhibitors.

T.A.

Sundaran

Vincent

2022

Preprint

View full text Add to dashboard Cite

show abstract

supporting

confidence: 82%

Outcome of Emicizumab prophylaxis in twins with severe hemophilia A and inhibitors.

T.A.

Sundaran

Vincent

2022

Preprint

View full text Add to dashboard Cite

show abstract

“…In a single‐center study from India, emicizumab prophylaxis was given to four children with inhibitor‐positive severe HA for a median of 34 weeks. Bleeding frequency decreased from 2 to 3.5 times per month to zero, and no significant adverse events were reported 34 . Although evidence on the effectiveness of emicizumab from an Indian setting is limited, emicizumab appears to be a promising agent for prophylaxis of patients with severe HA 2–4 …”

Section: Discussionmentioning

confidence: 94%

“…Bleeding frequency decreased from 2 to 3.5 times per month to zero, and no significant adverse events were reported. 34 Although evidence on the effectiveness of emicizumab from an Indian setting is limited, emicizumab appears to be a promising agent for prophylaxis of patients with severe HA. [2][3][4] Emicizumab, approved globally and in India, is a first-in-class prophylactic regimen for severe HA with several advantages over current treatments.…”

Section: Discussionmentioning

confidence: 99%

Cost‐effectiveness analysis of emicizumab prophylaxis in patients with haemophilia A in India

Seth,

John,

Chakrabarti

et al. 2023

Haemophilia

Self Cite

View full text Add to dashboard Cite

IntroductionEmicizumab is the initial subcutaneously administered bispecific antibody approved as a prophylactic treatment for patients with haemophilia A (PwHA).AimThis study assessed the economic evaluation of emicizumab treatment for non‐inhibitor severe haemophilia A (HA) patients in India.MethodsA Markov model evaluated the cost‐effectiveness of emicizumab prophylaxis compared to on‐demand therapy (ODT), low‐dose prophylaxis (LDP; 1565 IU/kg/year), intermediate‐dose prophylaxis (IDP; 3915 IU/kg/year) and high‐dose prophylaxis (HDP; 7125 IU/kg/year) for HA patients without factor VIII inhibitors. Inputs from HAVEN‐1 and HAVEN‐3 trials included transition probabilities of different bleeding types. Costs and benefits were discounted at a 3.5% annual rate.ResultsIn the base‐case analysis, emicizumab was cost‐effective compared to HDP, with an incremental cost‐effectiveness ratio (ICER) per quality‐adjusted life‐years (QALY) of Indian rupees (INR) 27,869. Compared to IDP, ODT and LDP, emicizumab prophylaxis could be considered a cost‐effective option if the paying threshold is >1 per capita gross domestic product (GDP) with ICER/QALY values of INR 264,592, INR 255,876 and INR 305,398, respectively. One‐way sensitivity analysis (OWSA) highlighted emicizumab cost as the parameter with the greatest impact on ICERs. Probabilistic sensitivity analysis (PSA) indicated that emicizumab had a 94.7% and 49.4% probability of being cost‐effective at willingness‐to‐pay (WTP) thresholds of three and two‐times per capita GDP.ConclusionEmicizumab prophylaxis is cost‐effective compared to HDP and provides value for money compared to ODT, IDP, and LDP for severe non‐inhibitor PwHA in India. Its long‐term humanistic, clinical and economic benefits outweigh alternative options, making it a valuable choice in resource‐constrained settings.

show abstract

“…Its cost (INR 87,000 for 60 mg injection) makes its availability to the general population of India a challenge. There is a lack of published literature on the use of emicizumab in India, but it has been shown to be effective and safe in Indian children and has the potential to improve the quality of life of inhibitorpositive patients who otherwise have high morbidity [23] .…”

Section: Discussionmentioning

confidence: 99%

A creative approach to managing a case of haemophilia A with inhibitors in a low-resource country: case report

Bhatia,

Archana,

Bhat

et al. 2023

The Journal of Haemophilia Practice

View full text Add to dashboard Cite

The development of antibodies to therapeutic factor VIII, known as inhibitors, is a leading cause of morbidity and mortality in people with haemophilia. This is particularly challenging in areas where access to novel treatment options is limited. This case report presents a clinical scenario in South Western India involving a 16-year-old male with severe haemophilia A with high titre inhibitors, who sustained a tibia-fibula shaft fracture necessitating emergency surgical intervention. The successful management of this patient required a multidisciplinary approach, encompassing haemostasis optimisation, innovative factor replacement strategies to work around financial constraints, and a comprehensive rehabilitation plan. The patient’s history of multiple factor VIII concentrate transfusions contributed to inhibitor development. Limited funds for bypassing agents prompted the utilisation of fresh frozen plasma to achieve haemostasis before access to FEIBA and recombinant factor VII. Emicizumab, a subcutaneously administered bispecific antibody, was used to assist perioperative haemostasis. A comprehensive rehabilitation plan with regular physiotherapy was followed. Emicizumab prophylaxis was initiated and the patient now shows improvement. This case emphasises the importance of novel treatment options such as FEIBA and emicizumab in dealing with complications in haemophilia such as inhibitors. In resource-limited settings, there is still a need to improve the availability of these life-saving interventions to optimise surgical outcomes in such cases.

show abstract

Initial experience of Emicizumab prophylaxis in children with inhibitor positive severe hemophilia A from a single center in India

Cited by 7 publications

References 10 publications

Outcome of Emicizumab prophylaxis in twins with severe hemophilia A and inhibitors.

Outcome of Emicizumab prophylaxis in twins with severe hemophilia A and inhibitors.

Cost‐effectiveness analysis of emicizumab prophylaxis in patients with haemophilia A in India

A creative approach to managing a case of haemophilia A with inhibitors in a low-resource country: case report

Contact Info

Product

Resources

About