A Pilot Study to Assess Quality of Life in Older Children and Adolescents with Primary Immune Thrombocytopenia

Background: Primary immune thrombocytopenia (ITP) is characterized by immune-mediated platelet destruction and impaired platelet production which may result in severe and chronic, symptomatic thrombocytopenia in a considerable minority of patients. The impact of chronic ITP (cITP) on the health-related quality of life (HRQoL), as it occurs in children, is not well defined.

Methods: We administered a validated, age-appropriate HRQoL instrument, the Kids ITP Tools (KIT) (Klaassen, et al. 2007) consisting of 27 questions to assess the HRQoL of older children and adolescents with cITP and the burden it places on their caregivers. Numerical KIT scores, ranging from 0-100 are inversely related to HRQoL. The Platelet Disorder Support Association (PDSA) invited participants (8 to 18 years of age) and their parents to complete an online version of the KIT survey and a background demographic and clinical questionnaire.

Results: The survey and questionnaire was completed and returned by 74 children, 168 parents and 90 parents (as proxies for their children) and were evaluable in 70, 155, and 80, respectively. Pediatric participants had male-to-female ratio of 0.98, a mean age and duration of ITP of 12.8 and 5.6 years, respectively. Bleeding symptoms were reported in 97% and drug treatment for ITP in 94%; 44% received an ITP treatment within the last month. Team sports and extracurricular club participation was reported in 42.9% and 54.3%of children, respectively. Twenty percent reported having been bullied. The relationship to being bullied and other determinants of HRQoL were not studied in this pilot study. No significant difference in KIT scores was noted by age, recent treatment or history of bleeding symptoms. The younger cohort (8-12 years), reported a significantly worse HRQoL than the older cohort, aged 12-18 years (p=0.042). In addition, a worse HRQoL was reported by children with ITP < 3 years compared to children with ITP ≥ 3 years (p=0.031), children with > 5 treatment side effects compared to children with ≤ 5 treatment side effects (p=0.010), children with no sports participation compared to children with sports participation (p=0.009). Parents-as-proxy reported a worse HRQoL for children with the shorter duration of ITP (p=0.008), > 5 treatment side effects (p=0.003), co-morbid health conditions* (p=0.009), and no sports participation (p=0.002). Parental burden was reported as worse if their child with ITP was younger (p=0.025), non-Caucasian (p=0.025), had ITP < 3 years (p=0.026), had co-morbidities (p=0.048), or didn’t participate in social clubs (p=0.046). A high degree of correlation, r = 0.8, was noted between the patient and parent-proxy answers. Areas of non-concordance between KIT scores and degree of parental burden included race, number of treatment side effects, and sports or social club participation. The study was limited by potential for ascertainment bias and survey fatigue.

C onclusions: This pilot study of the HRQoL in older children and adolescents with cITP showed that the HRQoL results are largely well correlated among the children, parent-proxy and parental burden results. Worse HRQoL was reported in children less than 12 years of age, those having 5 or more side effects to prior ITP treatment, and individuals not participating in sports. The study provides insight to the somewhat unpredictable determinants of parental burden and will facilitate larger cross-sectional HRQoL study of children and adolescents with cITP.

*thyroid disease, inflammatory bowel disorders, stomach ulcer, hearing loss, kidney problems, skin disorders, genetic syndromes, asthma, diabetes, anemia, recurrent infections, allergies, seizures, heart conditions