Understanding the Association between Transfusion Status (TS) and Overall Survival (OS), and Other Clinical Outcomes in Patients with Myelodysplastic Syndromes (MDS) from the Physicians' Perspective: A Pilot Survey
Authors: Gupta Shaloo , Li Zhiyi , Schroeder Thomas , Kulasekararaj Austin , Matos Joana E. , Berk Alexandra , Tang Derek , Grisolano Jay 
Published: November 2019

Introduction: Approximately 50% of patients with myelodysplastic syndromes (MDS) are anemic at initial diagnosis, with many becoming transfusion dependent. Based on several clinical trial analyses and observational studies, transfusion dependence is associated with worse outcomes in patients with MDS (e.g. overall survival [OS], hematologic improvement, etc.) compared with transfusion independence. However, no studies have examined the physicians' perspective with regard to the relationship between transfusion dependence and outcomes in MDS. The current study investigates physicians' understanding of the impact of transfusion status (TS) on OS and other clinical outcomes.
Methods: Data were derived from a brief 5-minute web-based physician survey via the M3 Global Research physician panel. Physicians had to be a hematologist/oncologist or hematologist to participate in the survey. In total, 100 physicians completed the survey across the EU5 countries (France, Germany, Italy, Spain, and United Kingdom; n = 15 in each country) and the USA (n = 25). The survey asked physicians for their opinion on the impact that TS has on OS, as well as on other clinical outcomes, such as hematologic improvement or serum ferritin level. Both items were ranked with a 7-point Likert scale ranging from 0 "very low impact" to 7 "very high impact". Physicians were also asked to rate their level of certainty for each of these assessments, with a 7-point Likert scale ranging from 0 "not at all certain" to 7 "completely certain". Lastly, respondents were asked to select from several options regarding what they based these decisions upon in terms of their sources of knowledge of the relationships between TS and OS or other outcomes (respondents could choose more than one option). All results were described descriptively with frequency counts and percentages.
Results: A total of 80 hematologist/oncologists and 20 hematologists completed the survey. A majority (85%) of physicians selected a score ≥ 5 (7 being "very high impact") on the item "In your opinion, what is the impact of TS on OS in MDS patients, holding other characteristics constant (e.g. disease severity, comorbidities)?". A total of 84% of physicians selected a score ≥ 5 (7 being "completely certain") on the certainty of their answer, with 8% reporting being "completely certain". Similarly, 87% of physicians selected a score ≥ 5 on the item "In your opinion, what is the impact of TS on other clinical outcomes (e.g. hematologic improvement, serum ferritin level) in MDS patients, holding other characteristics constant (e.g. disease severity, comorbidities)?". Some 85% of physicians selected a score ≥ 5 on the certainty of their answer, with 15% reporting being "completely certain". A majority (79%) of respondents reported using their clinical experience with their own patients as the basis for their assessment of the association between TS and OS or other clinical outcomes. More than half (57%) of physicians reported basing their assessment on knowledge of the literature, and 24% reported using the opinions of their colleagues or others in the field. These findings were numerically similar across the EU5 countries and the USA.
Conclusions: In this global pilot survey, almost all physicians reported TS as having more than a moderate level of impact on OS and other clinical outcomes in patients with MDS; almost all physicians also reported more than a moderate level of certainty to their perceptions of these relationships between TS and OS or other clinical outcomes. Physicians' own clinical experience was the key driver in understanding the association between TS and OS or other clinical outcomes. Physicians' level of knowledge and confidence was similar across geographical areas.

Disclosures
Gupta: Kantar: Employment; Celgene Corporation: Consultancy. Li:Celgene Corporation: Consultancy; Sun Pharma: Consultancy; BMS: Consultancy; Novartis: Consultancy; Asclepius Analytics: Employment. Schroeder:Celgene Corporation: Consultancy, Honoraria, Research Funding. Kulasekararaj:Alexion: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Celgene Corporation: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Novartis: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Amgen: Honoraria, Membership on an entity's Board of Directors or advisory committees; Achilleon: Consultancy; Ra Pharma: Honoraria, Membership on an entity's Board of Directors or advisory committees; Akari Therapeutics: Consultancy. Matos:Celgene Corporation: Consultancy; Kantar: Employment. Berk:Kantar: Employment; Celgene Corporation: Consultancy. Tang:Celgene Corporation: Employment, Equity Ownership. Grisolano:Celgene Corporation: Consultancy; Kantar: Employment.