Background: Immune thrombocytopenia (ITP) is an autoimmune disorder causing reduced platelet counts <100,000/µL, with potential bleeding consequences and reduced quality of life. Treatment is generally reserved for adults with platelet counts <30,000/µL.
Methods: ITP patients from the Platelet Disorder Support Association were recruited to complete a one-time cross-sectional survey online. Eligible adults from the United States self-reported that they had been diagnosed with ITP and received at least 1 treatment. Patients completed a 30-45-minute online survey about demographics, diagnosis experience, symptoms, disease management and treatment.
Results: Seventy-six patients completed the survey. Two-thirds had been diagnosed at least 10 years previously. Events leading to diagnosis were petechiae (21%), general check-up (19%), bleeding events (18%), and bruising (16%). Patients with ITP felt well informed about ITP (87.5% reported a score ≥5 on a 7-point Likert scale with 7 being highly knowledgeable) and 97% knew their current platelet count. At the time of diagnosis, platelet counts were < 10 x 109/L in 47% and 10 - 30 x 109/L in 27%. At the time of survey response, platelet count was < 30 x 109/L in 4.62% of participants; 47.69% of respondents had platelet counts > 100 x 109/L.
Using a Likert scale (7 = very strongly agree, 1 = very strongly disagree), 58% of patients reported a score ≥5 that ITP-related fatigue interfered with their work, family, or social life. Prior to being treated, 35% reported fatigue daily and 13% reported it twice a week. Despite treatment, a similar proportion reported fatigue daily (39%) or twice a week (16%).
Overall, the results of the survey did not suggest that bleeding was a concern for most patients: 59% reported a score ≤3 suggesting that bleeding does not interfere with their work, family or social life. Additionally, only 3% of patients reported that they experienced bleeding related to their ITP daily or twice weekly prior to treatment. However, fear of bleeding interfered with work, family or social life in 47% of patients with score ≥5 ; in contrast an equal number, 47%, reported a score ≤3.
45% of respondents underwent splenectomy without clinical improvement; an additional 31% were offered but declined surgery. Almost 90% of patients had prior treatment with steroids: 72% prednisone first and 15% dexamethasone first. Most had received rituximab (72%) and eltrombopag (55%); 40% received romiplostim. Additional information regarding aspects related to treatment will be presented, including sequencing and preferences associated with different therapies.
Conclusions:
This survey demonstrates some of the effects of fatigue, bleeding, and treatment on the lives of patients with ITP. On the one hand, almost half the patients had failed splenectomy, although currently very few patients undergo this procedure. Thus those taking the survey were not "random" ITP patients but may likely represent an atypical group that is refractory to treatment or dissatisfied with the course of their ITP despite half the patients having normal platelet counts at the time of the survey. In the course of their ITP, patients received a variety of treatments beyond steroids and results for side effects experienced and satisfaction with treatment will be analyzed and presented. The most surprising finding was that, even in those in whom treatment raised the platelet count, fatigue was not always alleviated. These issues and others remain to be clarified as we further unravel the complexity of ITP.
Bussel:Tranquil: Honoraria, Membership on an entity's Board of Directors or advisory committees; UCB: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Regeneron: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Kezar Life Sciences: Consultancy, Membership on an entity's Board of Directors or advisory committees; Physician Education Resource: Speakers Bureau; Amgen: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Momenta Pharmaceuticals: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Novartis: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Dova Pharmaceuticals: Consultancy, Membership on an entity's Board of Directors or advisory committees; GSK: Honoraria, Membership on an entity's Board of Directors or advisory committees; argenx: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; RallyBio: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Rigel: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; 3S Bio: Speakers Bureau. Kruse:Novartis: Consultancy; UCB: Honoraria; Amgen: Research Funding; Argenx: Research Funding; Dova: Research Funding; Novartis: Research Funding; Momenta: Research Funding; Principia: Research Funding; Octapharma: Research Funding; CSL Behring: Research Funding; UCB: Research Funding. Aggarwal:Dova Pharmaceuticals: Employment. Vredenburg:Dova Pharmaceuticals: Employment, Other: Shareholder. McCrae:Dova Pharmaceuticals: Membership on an entity's Board of Directors or advisory committees; Pfizer Pharmaceutical: Membership on an entity's Board of Directors or advisory committees; Rigel Pharmaceutical: Membership on an entity's Board of Directors or advisory committees; Sanofi Pharmaceuticals: Membership on an entity's Board of Directors or advisory committees.
Author notes
Asterisk with author names denotes non-ASH members.
