Krupa Sivamurthy

Analysis of long-term clinical and cost impact of etranacogene dezaparvovec for the treatment of hemophilia B population in the United States

Songkai Yan, Cheryl McDade, Kris Thiruvillakkat, Robert W. Rouse et autres

INTRODUCTION: Etranacogene dezaparvovec (EDZ), Hemgenix, is a gene therapy recently approved for people with hemophilia B (PwHB). OBJECTIVE: To estimate long-term clinical impact and cost of EDZ in the United States (US). METHODS: A decision-analytic model was developed to evaluate the long-term …

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Assessing health plan payer’s budget impact of etranacogene dezaparvovec for the treatment of hemophilia B in the United States

Michele Wilson, Cheryl McDade, Kris Thiruvillakkat, Robert W. Rouse et autres

BACKGROUND: Etranacogene dezaparvovec is a recently approved gene therapy for people with hemophilia B (PwHB). Current standard of care is prophylaxis with factor IX (FIX) to prevent bleeding. Etranacogene dezaparvovec increases blood FIX levels such that FIX prophylaxis could be eliminated. OBJECTIVE: …

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Featured Cover

Wolfgang Miesbach, Annette von Drygalski, Clive A. Smith, Krupa Sivamurthy et autres

The cover image is based on the Review The current challenges faced by people with hemophilia B by Wolfgang Miesbach et al., https://doi.org/10.1111/ejh.14135

The current challenges faced by people with hemophilia B

Wolfgang Miesbach, Annette von Drygalski, Clive A. Smith, Krupa Sivamurthy et autres

Hemophilia B (HB) is a rare, hereditary disease caused by a defect in the gene encoding factor IX (FIX) and leads to varying degrees of coagulation deficiency. The prevailing treatment for people with HB (PWHB) is FIX replacement product. The advent of …

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OC 52.1 Assessing the Safety Profile of AMT-060 and Etranacogene Dezaparvovec Gene Therapies Across Clinical Trials in People with Severe/Moderately Severe Hemophilia B

Wolfgang Miesbach, Michael Recht, Nigel S. Key, R. Guillen-Gonzalez et autres

Aims: We conducted a prospective hepatological screening to detect any relevant risk-factor of liver-complication in PWH after HCV-clearance.Methods: We evaluated 119 patients (median age: 54 years; range:36-87) with previous history of HCV-infection: 108/11 hemophilia A/B.Any active factor of chronic liver damage was …

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Durability of Factor IX activity and bleeding rate in people with severe or moderately severe haemophilia B after long-term follow-up in the phase 1/2 Study of AMT-060, and phase 2b and phase 3 studies of etranacogene dezaparvovec (AMT-061)

Wolfgang Miesbach, Michael Recht, Nigel S. Key, Krupa Sivamurthy et autres

Introduction Gene therapy for haemophilia B (HB) appears to have a durable response, with data presented at 5 years [ 1 ] and 8 years [ 2 ] post-dose. We review the durability of AMT-060 and etranacogene dezaparvovec (formerly AMT-061), defined by …

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Durability of Factor IX Activity and Bleeding Rate in People with Severe or Moderately Severe Hemophilia B after 5 Years of Follow-up in the Phase 1/2 Study of AMT-060, and after 3 Years of Follow-up in the Phase 2b and 2 Years of Follow-up in the Phase 3 Studies of Etranacogene Dezaparvovec (AMT-061)

Wolfgang Miesbach, Michael Recht, Nigel S. Key, Krupa Sivamurthy et autres

Introduction: Gene therapy for people with hemophilia B appears to have a durable response, with data presented over 5 years (Samelson-Jones. Blood 2021; 138_suppl 1: 3975) and 8 years (Nathwani. Blood 2018; 132_suppl 1: 491) post-administration. Here we review the durability of …

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Comprehensive analysis and prediction of long-term durability of factor IX activity following etranacogene dezaparvovec gene therapy in the treatment of hemophilia B

Jinesh Shah, Hongseok Kim, Krupa Sivamurthy, Paul E. Monahan et autres

OBJECTIVE: Congenital hemophilia B is a rare bleeding disorder caused by defects in the gene encoding factor IX (FIX) leading to coagulation deficiency. Recurrent bleeds may cause chronic pain, disability, and reduced quality of life. Phase 2 b and 3 single-arm, open-label, …

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