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.