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Experience with Pediatric Chronic Immune Thrombocytopenia over 30 Years in the Era before Eltrombopag
Begum S Koc1, Gul Nihal Ozdemir2, Javid Alakbarli1
1Department of Pediatric Hematology and Oncology, Cerrahpasa Medical Faculty, Istanbul University, Istanbul 34696, Turkey.
Insights
Nearly one-third of children with chronic immune thrombocytopenia (ITP) achieve spontaneous remission within 3 years. Splenectomy is effective for severe cases, offering insights into childhood ITP natural history before eltrombopag.
Area of Science:
- Pediatric Hematology
- Immunology
- Clinical Research
Background:
- Limited data exists on the natural progression of chronic immune thrombocytopenia (ITP) in pediatric populations.
- Understanding ITP's natural course is crucial for guiding treatment decisions.
Purpose of the Study:
- To characterize the clinical and demographic features of children with chronic ITP.
- To describe the natural history of chronic ITP in children prior to widespread eltrombopag use.
Main Methods:
- Retrospective analysis of 86 children diagnosed with chronic ITP between 1978 and 2014.
- Data collected included demographics, laboratory values, clinical presentation, bleeding severity, and remission times.
Main Results:
- Median age at diagnosis was 7 years; median initial platelet count was 10 × 10^9/L.
- Petechiae/ecchymoses (86%) and mucosal bleeding (39.5%) were most common; severe bleeding occurred in 5%.
- Spontaneous remission occurred in 29% (median 3 years); 70% achieved remission after splenectomy.
Conclusions:
- Approximately 30% of children with chronic ITP experience spontaneous remission within 3 years.
- Splenectomy demonstrated positive outcomes for severe chronic ITP cases.
- This study provides valuable insights into the natural history of childhood chronic ITP before the availability of eltrombopag.
Background:
There is limited information on the natural course of chronic ITP in children. We aimed to evaluate the clinical and demographic characteristics of children with chronic ITP in the era before the availability of eltrombopag.
Methods:
A total of 86 children with chronic ITP between 1978-2014 were included. Demographic findings, laboratory results, clinical signs, bleeding scores, response time and time of complete remission were recorded.
Results:
The male/female ratio was 1.09, and median follow-up time was 3 years (range: 1.5-17 years). The median age at diagnosis of chronic ITP was 7 years (range: 2-17), and the median initial platelet count was 10 × 109/L (range: 1-66 × 109/L). Petechiae/ecchymoses were the most common clinical sign (86%) and followed by mucosal bleeding (39.5%). Severe bleeding was seen in 5% of the patients. None of them had intracranial hemorrhage. Twenty patients underwent splenectomy, and the rate of complete remission was 70%. Spontaneous complete remission was seen in 29% of the patients, and the median time to spontaneous complete remission was 3 years.
Conclusions:
Our study showed that almost one-third of patients with chronic ITP experienced spontaneous complete remission in an average of 3 years, and splenectomy provided satisfactory results in severe cases. This study demonstrates the natural history of chronic ITP in childhood before the era of eltrombopag.
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