ASH Clinical News ACN_4.2_FULL_ISSUE | Page 9

to the editor :
Loved this editorial ! I would have written it myself , but I was clearly too busy signing lab values , doing Firecrest training , and driving to meetings with site monitors to hear them say , “ Great job , nothing to report .”
Jonathan Ducore , MD , MPH Co-Director of Hemophilia
Treatment Center University of California , Davis
I feel for you about the insanity of using trial-specific equipment ! I have a room full of electrocardiogram machines ( 14 at last count – as well as all the boxes that they were shipped in that I have to store so I can eventually ship them back to the sponsor ), trial-specific pregnancy tests , blood pressure cuffs , needles , scales and 10 kg weights to calibrate the scales ( we use one as a doorstop ), and all other kinds of equipment that I eventually give to medical missions or – God forbid – have to throw away . I wish I could trade it all in for a decent intravenous infusion pump .
I have a laptop from one company that has never been used , but the company won ’ t let us send back ; it is still sitting in my office .
Also , I have at least one of every size of battery known to man .
Catherine Hubert , RN , BSN Manager of Clinical Research
Mercy Research Springfield , Missouri
Clinical study results in children and adults receiving prophylactic treatment over a 6-month period 1 , 2
The efficacy , safety and PK of ADYNOVATE were evaluated in 2 multicenter , open-label clinical studies . The pediatric study of children < 12 years of age ( N = 66 ) evaluated the immunogenicity , efficacy , PK ( as compared to ADVATE ® [ Antihemophilic Factor ( Recombinant )]), and safety of ADYNOVATE twice-weekly prophylaxis ( 40-60 IU / kg ) and determined hemostatic efficacy in the treatment of bleeding episodes for 6 months . The pivotal trial of children and adults ≥12 years ( N = 137 ) evaluated ADYNOVATE twice-weekly prophylaxis ( 40-50 IU / kg ) vs on-demand ( 10-60 IU / kg ) treatment , and determined hemostatic efficacy in the treatment of bleeding episodes for 6 months . 1-3
Proven prophylaxis with ADYNOVATE

ZERO

MEDIAN ABR FOR JOINT & SPONTANEOUS 1
In pediatric patients < 12 years : Joint : 0.0 ( IQR : 1.9 ) median ABR 3 ; Spontaneous : 0.0 ( IQR : 1.9 ) median ABR . 3
In adults and children ≥12 years : Joint : Prophylaxis 0.0 ( IQR : 2.0 ) median ABR vs on-demand 38.1 ( IQR : 20.1 ) median ABR . 1 , 3 Spontaneous : Prophylaxis 0.0 ( IQR : 2.2 ) median ABR vs on-demand 21.6 ( IQR : 22.0 ) median ABR . 1 , 3
Consistent dosing1
+ In the clinical studies , the majority of children and adults did not have a dose adjustment 1 , 2
+ 98 % of adults and children ( 12 years and older ) did not have a dose adjustment ( 118 of 120 ) 1 — Two subjects increased their dose to 60 IU / kg due to bleeding in target joints
+ 91 % of children ( less than 12 years ) did not have a dose adjustment ( 60 of 66 ) 2 — Reported reasons for dose adjustment included FVIII trough levels < 1 %, increased risk of bleeding , and bleeding episodes
Neutralizing Antibodies
Formation of neutralizing antibodies ( inhibitors ) to factor VIII can occur following administration of ADYNOVATE . Monitor patients regularly for the development of factor VIII inhibitors by appropriate clinical observations and laboratory tests . Perform an assay that measures factor VIII inhibitor concentration if the plasma factor VIII level fails to increase as expected , or if bleeding is not controlled with expected dose .
ADVERSE REACTIONS
The most common adverse reactions ( ≥1 % of subjects ) reported in the clinical studies were headache and nausea .
Please see the following page for the Brief Summary of the ADYNOVATE full Prescribing Information .
For full Prescribing Information , visit www . ADYNOVATEPRO . com .
References : 1 . ADYNOVATE Prescribing Information . 2 . Mullins ES , Stasyshyn O , Alvarez-Román MT , et al . Extended half-life pegylated , full-length recombinant factor VIII for prophylaxis in children with severe haemophilia A . Haemophilia . 2016 Nov 27 . doi : 10.1111 / hae . 13119 [ Epub ahead of print ]. 3 . Data on file .
© 2017 Shire US Inc ., Lexington , MA 02421 . All rights reserved . 1-800-828-2088 . SHIRE and the Shire Logo are registered trademarks of Shire Pharmaceutical Holdings Ireland Limited or its affiliates . ADVATE and ADYNOVATE are trademarks or registered trademarks of Baxalta Incorporated , a wholly owned , indirect subsidiary of Shire plc . S31608 05 / 17