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Childhood immune thrombocytopenia: A nationwide cohort study on condition management and outcomes
Lamiae Grimaldi-Bensouda1, Clémentine Nordon1, Thierry Leblanc2,3
1LASER, Paris, France.
Insights
In children with immune thrombocytopenia (ITP), low platelet counts and bleeding symptoms often lead to treatment initiation. Younger children generally have a better prognosis, with factors like female gender and older age predicting chronicity.
Area of Science:
- Pediatric Hematology
- Immunology
- Clinical Research
Background:
- Primary immune thrombocytopenia (ITP) is an autoimmune disorder affecting platelet counts in children.
- Management strategies for pediatric ITP involve watchful waiting or active treatment.
- Understanding factors influencing treatment decisions and long-term outcomes is crucial for clinical practice.
Purpose of the Study:
- To identify factors associated with initiating treatment versus watchful waiting in children with primary ITP.
- To determine predictors of chronic ITP at 12 months in a real-world clinical setting.
Main Methods:
- A nationwide prospective cohort study was conducted in France between 2008 and 2013.
- 257 children (6 months-18 years) with primary ITP were consecutively enrolled across 23 centers.
- Multivariate logistic regression analyses were performed to identify significant factors and predictors.
Main Results:
- Treatment was initiated in 81% of children, primarily driven by platelet counts below 10 × 10⁹/L and bleeding symptoms.
- At 12 months, 74% of children had recovered.
- Predictors of chronicity included female gender (OR=2.2), age ≥10 years (OR=2.6), and platelet counts ≥10 × 10⁹/L (OR=3.2).
Conclusions:
- Watchful waiting in pediatric ITP is influenced by platelet counts, even without bleeding symptoms, leading to early treatment in most cases.
- Younger children with ITP generally have a favorable prognosis.
- Female gender, older age, and higher platelet counts at 12 months are associated with a higher likelihood of chronic ITP.
Objectives:
Nationwide prospective cohort study exploring (i) the factors associated with treatment initiation (vs. watchful waiting) in children with primary immune thrombocytopenia (ITP) followed in routine clinical practice and (ii) the predictors of chronicity at 12 months.
Procedure:
Between 2008 and 2013, 23 centers throughout France consecutively included 257 children aged 6 months-18 years and diagnosed with primary ITP over a 5-year period. Data on ITP clinical features along with medical management were collected at baseline and 12 months. Multivariate logistic regressions were used to determine (i) and (ii) as defined above, providing odds ratio (OR) with 95% confidence interval (95% CI).
Results:
One hundred thirty-seven (53%) children were males, median age was 4.6 years, median platelet count was 7 × 109/l, and 214 (81%) patients initiated medication. Factors independently associated with treatment initiation included platelet counts <10 × 109/l (P < 0.0001) and mucocutaneous bleeding symptoms at baseline (P < 0.001). At 12 months, data were available for 211 (82%) children, of whom 160 (74%) had recovered. Predictors of chronicity included female gender (OR = 2.2; 95% CI = 1.0-4.8), age ≥10 years (OR = 2.6; 95% CI = 1.1-6.0), and platelet counts ≥10 × 109 /l (OR = 3.2; 95% CI = 1.5-6.9).
Conclusions:
In routine clinical practice, the decision to apply a watchful waiting strategy seems to be driven by platelet counts even in the absence of bleeding symptoms, resulting in treatment being initiated in more than 80% of the children surveyed. Overall, younger children with ITP showed good prognosis, with lower platelet counts and, to a lesser extent, male gender predicting more favorable outcomes.
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