Patient preferences in the treatment of hemophilia A: impact of storage conditions on product choice

Objectives: To gain insights into the usage of factor VIII (FVIII) products by patients diagnosedwith moderate/severe hemophilia A, and to assess the impact and perceived importance ofproduct storage.Methods: In this study, 200 patients diagnosed with moderate or severe hemophilia A acrossseven countries participated. Data were collected via a 30-minute, face-to-face interview in sixcountries and via a web-based survey in the seventh country. The questionnaire evaluated theeffect of six features associated with FVIII products on the choice of the product; the structureand flow of data collection was designed to eliminate potential bias.Results: Two-thirds of the respondents were using recombinant FVIII products. Only 17%were generally dissatisfied with current FVIII products, whereas 40% of the respondentswere dissatisfied with frequency of administration and storage issues when traveling. Themajority noted restrictions in their daily activities, particularly travel and sports. Most of them(85%), stored their product in the refrigerator and of these, 88% believed that it should alwaysbe stored there. These patients were also less satisfied with the product overall, more concernedabout storage temperature, more restricted in daily activities, and spent more time on preparationand injection compared with patients who stored their product at room temperature. Conjointanalysis revealed that origin of FVIII (plasma-derived vs recombinant) was the strongestdriver of product choice among all respondents, followed by storage flexibility (temperature),reconstitution device, and administration frequency. In this study, we did not investigate theefficacy and safety of the product.Conclusion: Not refrigerating FVIII products was associated with greater patient satisfaction and less restriction on daily activities. If efficacy and safety are unaffected, then storingFVIII at room temperature might have a positive impact on product choice. Few patients wereaware that FVIII can be stored without refrigeration, suggesting that health care professionalswho treat hemophilia should communicate this aspect to the patient (depending on the labeledoption); this approach might offer patients greater flexibility when traveling and require lesstime for reconstitution.