Quality of Life in Children with Acute Immune Thrombocytopenia Is Related to the Course of the Disease and Not to Treatment Modality or Bleeding Tendency
Authors: Heitink-Pollé Katja M.J. , Haverman Lotte , Annink Kim V. , de Haas Masja , Bruin Marrie C. A. 
Published: October 2019

Abstract
Abstract 1098

Introduction:
Acute immune thrombocytopenia (ITP) is characterized by isolated thrombocytopenia and a sudden onset of bruising in an otherwise healthy child. Most children recover within 6–12 months and only 3–6% of children experience severe bleeding. To prevent bleeding complications, children with acute ITP are restricted in their activities by doctors and parents. We hypothesized that these restrictions in activities and fear of bleeding have an important impact on health related quality of life (HRQoL) of both children and parents and that treatment might improve HRQoL. However, HRQoL studies in childhood ITP are scarce. The TIKI study (Therapy with or without Intravenous Immunoglobulin for Kids with acute ITP) is a multicenter randomized clinical trial to determine if early intravenous immunoglobulin (IVIg) administration can prevent a chronic course of the disease. Children receive either a single dose of IVIg or careful observation and treatment only in case of severe bleeding. As part of this study, HRQoL was measured at diagnosis and during follow up.

Patients and methods:
Children aged 3 months to 16 years with newly diagnosed ITP were included in the TIKI study. Parents of all included children and children aged 7 years and older completed a generic HRQoL questionnaire, the Pediatric Quality of Life Inventory (PedsQL, HRQoL of the child reported by proxy and by children themselves) and the recently developed and validated disease specific Kids' ITP Tools (KIT, HRQoL of children (proxy and self) and parents) at diagnosis, after 1 week, 1 month, 3 months, 6 months and 12 months. Bleeding tendency was scored according to the adapted Buchanan bleeding score. We compared PedsQL scores of (1) children diagnosed with acute ITP and healthy children, (2) children with persisting ITP beyond 6 months and children with other chronic conditions. The KIT scores of children (proxy and self) and parents were compared between: (3) children that received IVIg and children randomized to the observational arm; (4) children with bleeding score 0, 1, 2, 3 and 4; (5) children that recovered within 3–6 months and children with persisting thrombocytopenia beyond 3–6 months. Mann Whitney tests were performed to analyze differences between two groups, ANOVA tests to analyze data regarding bleeding score. P values < 0.05 were considered significant. HRQoL PedsQL norm scores were available for healthy Dutch children and children with chronic conditions, like asthma and eczema.

Results:
At time of analysis, HRQoL data were available for 74 patients, 37 boys and 37 girls with a median age of 41 months (9–198 months). HRQoL of (1) children with acute ITP at diagnosis did not differ significantly from healthy children (median score proxy: 83 vs. 87, p 0.26; child: 79 vs. 83, p 0.45); (2) HRQoL of children with persisting ITP after 6 months was comparable with HRQoL of children with other chronic conditions. There were no significant differences between (3) HRQoL of children and parents in the IVIg group and in the observational group after 1 week (median score child 55 vs. 69, p 0.23; proxy 76 vs. 74, p 0.54; parent 57 vs. 64, p 0.21) and 1 month (median score child 75 vs 86, p 0.33; proxy 92 vs 84, p 0.09; parent 74 vs. 70, p 0.69). (4) No significant differences in HRQoL were found between children with bleeding score 0,1,2,3 or 4. (5)Parents of children that recovered within 3 or 6 months showed a significant better HRQoL than parents of children with persisting ITP beyond 3 or 6 months (3 months: median score parents 81 vs. 66, p 0.004; 6 months: 88 vs. 70, p 0.002). HRQoL of children reported by proxy at 6 months but not at 3 months showed a significant difference as well (median score proxy 95 vs. 88, p 0.048).

Conclusion:
HRQoL of children with acute ITP measured 1 week and 1 month after diagnosis does not differ between children that received careful observation or IVIg treatment. HRQoL is also not influenced by bleeding tendency, but only by clinical course: parents of children that have recovered within 3 and within 6 months have a significant better HRQoL than parents of children with persisting ITP beyond 3 and 6 months. HRQoL of children reported by proxy at 6 months is also better in the recovered group. To improve HRQoL, preventing a chronic course of the disease seems to have the most beneficial effect. The clinical results of our trial are awaited to determine if early IVIg adminstration can prevent a chronic course of the disease.

Disclosures:
No relevant conflicts of interest to declare.