Patients with the autoimmune disease ITP suffer from bleeding events as a result of low platelet counts. These events may manifest as bruises, petechiae, blood blisters, bloody stools, blood in urine, or even bleeding in the brain. Treatments vary by severity of disease but include medications (steroids, immunosuppressants, IVIG, TPO-receptor agonists) and splenectomy. Both the disease and the treatments themselves impact quality of life for these patients, who commonly state concerns of anxiety and fatigue.
In 2017, PDSA in collaboration with NORD launched the ITP Natural History Registry to understand patient characteristics, their disease, disease management, and quality of life. Here, we describe the demographics and quality of life for registrants to date.
The PDSA Registry contains 6 surveys covering patient demographics, medical and diagnostic information, treatment utilization, disease progression, and quality of life. As of February 2019, 538 patients have completed 1,975 surveys.
Patients in the registry are mostly white (89%, 470/528), female (76%, 399/528), and reside in the US. 34% (170/499) of patients have Medicare, Medicaid, or both, while 62% (311/499) have commercial insurance. Average (median) age at diagnosis was 32 (31) years with 25% (125/502) younger than 18 years. Time from onset of symptoms to diagnosis was more than one year for 32% (169/528) of patients. In response to specific questions regarding anxiety and fatigue, 11% (32/285) of patients stated they were often or always feeling like they needed help for their anxiety and 36% (99/277) said that their fatigue bothered them quite a bit or very much. Of the 538 registrants, 288 provided data on treatment and 27% (78/288) indicated receiving a splenectomy. For patients who received a splenectomy, quality of life was rated as poor to fair for 23% (18/78) of patients. For patients who had not had a splenectomy, 19% (40/210) rated their quality of life as poor to fair, which is improved but not statistically different from the splenectomy group (z = 0.758, p = 0.447).
Respondents in the PDSA ITP Natural History Registry are predominantly female, white, and diagnosed while young, with a fourth of patients under 18 at time of diagnosis. Disease awareness is suboptimal as indicated by the prolonged time to diagnosis, >1 year, for a third of registrants. QoL is impacted in patients with ITP with it directionally worse, though not significantly different, for those who have had a splenectomy compared to those without. Data collection continues through the registry with the intent of raising disease awareness and understanding the impact of the disease.