Subcutaneous Emicizumab Reduces Number of Injections Needed to Control Hemophilia

MedicalResearch.com Interview with:

Dr. Johnny Mahlangu  MBBCh Faculty of Health Sciences University of the Witwatersrand and National Health Laboratory Service Johannesburg, South Africa

Dr. Mahlangu

Dr. Johnny Mahlangu  MBBCh
Faculty of Health Sciences
University of the Witwatersrand and National Health Laboratory Service
Johannesburg, South Africa

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: Current unmet needs in patients with haemophilia without inhibitors are the high disease burden imposed by the frequent injections which have to be given intravensously .

Emicizumab which is given subcutaneously weekly or fortnightly aims to address these unmet needs.

MedicalResearch.com: What should readers take away from your report?

Response: Emicizumab prophylaxis reduces the bleed rate by 96% or 97% when given at mantainance dose of 1.5 mg /kg weekly or every second week respectively. Emicizumb prophylaxis reduced the bleed rate by 56% or 60% when given weekly or severy second week when compared to currently available FVIII products used for prophylaxis. Elicizumab is safe with mild injections site reactions but no thrombosis or thrombotic microangiopathy 

MedicalResearch.com: What recommendations do you have for future research as a result of this work?

Response: Explore giving Emicizumab once a month which is supported by its pharmacokinetic profile of a half life of more than 30 days 

MedicalResearch.com: Is there anything else you would like to add?

Response: Emicizumab prophylaxis may represent a paradigm shift in the prevention of bleeds in hemophilia patients without inhibitors

Citation:

Emicizumab Prophylaxis in Patients Who Have Hemophilia A without Inhibitors

Johnny Mahlangu, M.B., B.Ch., M.Med., Johannes Oldenburg, M.D., Ph.D., Ido Paz-Priel, M.D., Claude Negrier, M.D., Ph.D., Markus Niggli, Ph.D., M. Elisa Mancuso, M.D., Ph.D., Christophe Schmitt, Pharm.D., Victor Jiménez-Yuste, M.D., Ph.D., Christine Kempton, M.D., Christophe Dhalluin, Ph.D., Michael U. Callaghan, M.D., Willem Bujan, M.D., et al.

August 30, 2018
N Engl J Med 2018; 379:811-822
DOI: 10.1056/NEJMoa1803550 

Sep 7, 2018 @ 12:12 pm

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1 thought on “Subcutaneous Emicizumab Reduces Number of Injections Needed to Control Hemophilia

  1. Great publication! Odd that there was no observed statistically significant difference in the Haem-A-QoL scores between any of the groups. That is unexpected given “the high disease burden imposed by the frequent injections which have to be given intravensously”. Is there a plan to study this again to hopefully demonstrate a difference in disease burden?

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