A Delphi Consensus by Hematologists on the Burden of Treatment on Patients with Severe Hemophilia in Mexico.

Abstract

Purpose: Current treatment of hemophilia still represents an important burden for patients and caregivers. As a consequence, adherence can be poor, thus reducing efficiency of treatment. Then, a modified Delphi method was used to assess the burden of hemophilia treatment in Mexico.

Methods: In the first round, the experts answered online a questionnaire with 32 statements structured in eight sections: overall efficacy of recombinant coagulation factors, delivery of recombinant factors, adherence to treatment with recombinant factors, emotional burden, physical load, interference with daily life, impact of adherence on efficacy, and satisfaction with treatment. Each statement was qualified from "Totally disagree" to "Totally agree". In the second round, those statements with less than 70% agreement between experts were discussed.

Results: Consensus was reached in more than half of the questions. There was a consensus on the limited efficacy of current treatments, the complexity of preparation and administration of the recombinant coagulation factors, and the negative effect of the emotional burden, among other statements. Answers to the section related to the impact of adherence on efficacy showed the concern of experts about this issue.

Conclusion: The burden of prophylactic hemophilia treatment on patients and caregivers in Mexico is substantial. Its negative effects on adherence and efficacy of treatment require evaluation of new approaches to hemophilia treatment.