Background and Aims
Patients with Immune Thrombocytopenia (ITP) may have bleeding because of low platelet counts; many also have reduced quality of life (QoL). Course of therapy can vary by duration and severity of disease but includes steroids and thrombopoietin receptor agonists [TPO-RA]. Both symptoms of ITP and side effects from treatments impact patient QoL, commonly anxiety and fatigue. In 2017, PDSA in collaboration with the National Organization for Rare Diseases launched the ITP Registry to understand patient characteristics, their disease, disease management, and QoL. Here, we describe the QoL for registrants, as it relates to mental health and therapy options.
Methods
The registry contains five questionnaires covering patient demographics, medical history and diagnostic information, treatments, and QoL in adults and children. Patients are asked to update their responses every six months to document the natural history of ITP. As of February 2019, 538 patients completed in total 1,975 surveys. Not all patients enrolled completed all five surveys; 385 patients completed the survey on treatment, and 310 patients completed the survey on quality of life in adults.
Results
Registry respondents (n=385) reported use of 16 different treatments to manage their ITP. The most commonly used medications were prednisone (65%), IVIG (42%), and rituximab (33%); 10% of participants reported no use of medication.
Most patients reported significant anxiety and fatigue as a result of ITP. 89% of patients (n=276) were fatigued within the past week. 47% of participants reported feeling like they needed help with their anxiety within the past week, 53% of participants reported finding it hard to focus on anything because of anxiety during the same time period.
Participant degree of fatigue did not differ based on medication type; similarly, no trend was seen in frequency of fatigue based on treatment type. The number of participants using and not using steroids reported similar rates of fatigue (p=0.674). The same was true comparing on and off TPO agents and fatigue (p=0.682).
Conversely, participant anxiety differed significantly based on medication type. Patients who reported anxiety while taking steroids was significantly greater than patients who reported anxiety while using TPO-RAs (p < 0 .00001).
QoL reported as poor or fair in 20% of affected patients (80% of participants reported QoL as good, very good, or excellent) did not differ significantly by medication type (steroid use compared to total p=0.889; TPO agent compared to total: p=0.435).
Discussion
Patients with ITP feel the impact of disease beyond the physical manifestations like bleeding and bruising, extending to the emotional impact of ITP. In part this is due to the uncertainty of symptoms, treatment efficacy and fear of the unknown. This is reflected in the high rates of anxiety experienced by patients while receiving steroids. Patients with newer ITP may have higher levels of anxiety due to their new diagnosis and uncertainty of disease course and are commonly treated with steroids first. Conversely, fatigue is not correlated with treatment type. Surprisingly, despite high levels of fatigue and anxiety, 4/5 respondents still report at least good quality of life. This could be in part because patients become acclimated to living with their disease, or a belief that their ITP could manifest more severely. Additionally, treatments of ITP which increase the platelet count (not confirmed here) may improve overall QoL even though fatigue and anxiety do not disappear or may even be increased as shown by the more than 4/5 respondents who report these. Further studies may clarify the complex nature of QoL, anxiety and fatigue in ITP.
Disclosures
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. Bussel:GSK: Honoraria, Membership on an entity's Board of Directors or advisory committees; Tranquil: 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; RallyBio: 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; 3S Bio: Speakers Bureau; Regeneron: Consultancy, 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; Dova Pharmaceuticals: Consultancy, 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; 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.
Patient-rated mental health needs and quality of life improvement