Standards and definitions


Sumit Parikh is the AHCDO ABDR Senior Research Fellow

Sumit and Australian haemophilia nurses Alex Connolly, Megan Walsh and Andrew Atkins at the AHCDO posters

ISTH 2017 was all about showcasing and discussing the most recent advances in prevention, diagnosis and treatment of thrombotic and bleeding disorders.

AHCDO had two research posters at ISTH this year: ‘To characterise current inhibitor status of patients with haemophilia A in Australia’ and ‘The impact of switching recombinant factor VIII product concentrates on inhibitor development among haemophilia A patients in Australia’. Both the posters received very good feedback.

Among the many engaging sessions presented that were relevant to the bleeding disorder community there was considerable interest in the Scientific and Standardisation Committee (SSC) sessions.

Standardisation of post-registration surveillance

Post-registration surveillance of a treatment product occurs after a medicine has been approved by a regulatory authority. The current method to evaluate post-registration surveillance does not include registration of all side effects related to existing and upcoming treatment products. It was highlighted that evaluating safety/efficacy of novel drugs after product registration in a harmonised and standardised way could help to understand how to optimise the data collection. This will improve the safety surveillance system knowledgebase and thus the provision of comprehensive care services to the patient and could be achieved by:

  • setting a minimum set of data necessary to bring enough information on safety and efficacy of each single product
  • and by approval of this template by regulatory agencies and institutions
  • and performing an observational study for at least 5 years.

With so many novel treatment and therapies round the corner, this brings the focus back to the importance of recording data and interactions between patients and their HTC. 

Consensus definitions and recommendations for ITI in haemophilia with inhibitors

Management of haemophilia with inhibitor patients is complex. ITI (immune tolerance induction) has been highly regarded as an effective method of eradicating inhibitors. This means it is particularly important to establish definitions of ITI outcome and response and to standardise the methodologies to assess treatment endpoints.  The characteristics of patient receiving ITI form an integral part of the ITI outcome definition and are essential to be able to provide treatment recommendations and guidelines. This includes information about the patient and their treatment such as:

  • age at inhibitor development, and at ITI start
  • inhibitor titre, historical inhibitor peak, and peak on ITI
  • type of product and treatment dose. 

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