Survey Results
There were 25 respondents from the 50 ICON sites (50% response). Questions were posed as 7 case descriptions followed by 5 general questions about types of patients (Supplemental Appendix B). Information about individual providers were not collected. Most respondents completed all questions (20/25). There was little agreement amongst providers for both the case scenarios and patient descriptions as to which patients would be considered refractory. Only one question achieved unanimity amongst respondents: “Is a patient with ongoing symptoms (fatigue) regardless of platelet count responses refractory?” to which respondents universally agreed “no.” Only 55% of respondents felt that patients who fail to respond to initial second line therapy are refractory (Figure 2A). There was a wide variation in the identification of refractory ITP amongst the respondents (Figure 2B). The majority, 96%, of respondents agreed that a patient with chronic ITP and minimal or transient IVIG response and lack of TPO-RA response could be considered refractory. Additionally, 85% said a newly diagnosed patient who failed to respond to IVIG, steroids or TPO-RA and required platelet transfusions for bleeding could be considered refractory (based on case scenarios). Seventy-five percent of respondents agreed that a transient response to IVIG alone was not sufficient to label a patient refractory.