Definitions for haemophilia prophylaxis and its outcomes: The Canadian Consensus Study

Abstract

The creation of acceptable standard definitions for terms used in the care and assessment of haemophilia patients has become increasingly important, as a growing number of international clinical studies have been initiated. The Delphi approach has been used in health research to reach consensus in large groups and can be used to develop definitions by using several iterations of surveys eliciting opinions from specialists in the field. Three consecutive surveys were designed based on the Delphi approach and distributed to specialist physicians, nurses and physiotherapists in order to develop definitions for seven haemophilia terms: 'primary prophylaxis', 'secondary prophylaxis', 'target joint', 'joint bleed', 'significant soft-tissue bleed', 'superficial soft-tissue bleed' and 'mucosal bleed'. Suggestions were solicited, compiled into a subsequent survey and fed back to the group to rank-order the importance of each suggested component of the definition. Final definitions were created using the top-ranked suggestions and sent back to the experts for approval. Five of the seven terms were highly endorsed with greater than 90% agreement. Some differences in agreement were found when analysed by profession. Haemophilia terms were successfully defined using the Delphi approach. Further refinement from members of the international haemophilia community will ensure that comprehensive standard definitions can be used in multicentre studies in the future.

The Jamaican Haemophilia Registry: Describing the burden of disease

“…An estimate of total bleeds in the year prior to enrolment of cases into the study was determined through interviews with patients or with parents/guardians of PWH. Target joints were defined as index joints into which 3‐4 bleeds occurred within a consecutive 3‐ to 6‐month period …”

Recombinant long‐acting glycoPEGylated factor IX (nonacog beta pegol) in haemophilia B: assessment of target joints in multinational phase 3 clinical trials

“…The ISTH definition of a newly developed target joint considers spontaneous bleeds only and is therefore less conservative than the PEDNET definition which considers all bleeds. The ISTH definition is also less conservative compared to a number of previously proposed definitions . For a current target joint, up to two bleeds within 12 months would still allow for this joint to cease being classified as a target joint.…”

Clinical evaluation of bleeds and response to haemostatic treatment in patients with acquired haemophilia: A global expert consensus statement

“…The Delphi methodology uses a series of surveys distributed to a group of individuals to ascertain consensus on statements relating to a domain of expertise. [22][23][24][25][26] After three successive survey rounds, a definitive list of statements that did and did not derive consensus is generated.…”

“…Finally, while the benefits of using Delphi methodology for generating consensus are well documented, [22][23][24][25][26] this approach may have also avoided the potential bias that could have occurred using a face-to-face assessments, utilized in other consensus methodologies. Finally, while the benefits of using Delphi methodology for generating consensus are well documented, [22][23][24][25][26] this approach may have also avoided the potential bias that could have occurred using a face-to-face assessments, utilized in other consensus methodologies.…”