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[Clinical Analysis of Hospitalized Children with Primary Immune Thrombocytopenia]
Rong-Wei Li1, Rong-Feng Fu1, Yun-Fei Chen1
1State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin Laboratory of Blood Disease Gene Therapy, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Tianjin 300020, China.
Insights
Insidious onset of symptoms in childhood primary immune thrombocytopenia (ITP) increases chronicity risk, while anti-platelet antibodies may reduce it. Intravenous immunoglobulin (IVIG) or IVIG with steroids are more effective first-line treatments than steroids alone for ITP.
Area of Science:
- Pediatric Hematology
- Immunology
- Clinical Medicine
Background:
- Childhood primary immune thrombocytopenia (ITP) is an autoimmune bleeding disorder.
- Understanding factors influencing ITP chronicity is crucial for patient management.
- Optimizing first-line treatment strategies can improve outcomes for children with ITP.
Purpose of the Study:
- To identify factors associated with the development of chronic ITP in children.
- To compare the effectiveness of different first-line treatment regimens for newly diagnosed and persistent ITP.
Main Methods:
- Retrospective analysis of 301 children diagnosed with ITP between September 2013 and October 2018.
- Assessment of chronicity based on disease duration and treatment outcomes.
- Multivariable logistic regression used to identify risk factors for chronicity.
Main Results:
- The study included 301 children with ITP, with 32.9% developing chronic disease.
- Insidious symptom onset significantly increased the risk of chronicity (OR=3.754).
- Positive anti-platelet membrane glycoprotein antibodies were associated with a reduced risk of chronicity (OR=0.446).
- First-line treatment with intravenous immunoglobulin (IVIG) alone or combined with steroids showed better efficacy than steroids alone (P=0.028).
Conclusions:
- Insidious onset is a risk factor for chronic ITP in children.
- Anti-platelet antibodies may have a protective role against ITP chronicity.
- IVIG-based regimens are more effective than steroid monotherapy for initial ITP treatment.
Objective:
To investigate the factors affecting the chronicity of childhood primary immune thrombo-cytopenia (ITP) and compare the efficiency of different first-line treatment regimens.
Methods:
Children with ITP hospitalized in our hospital from September 2013 to October 2018 were retrospectively analyzed.
Results:
Three hundred and one children (150 males and 151 females) were included in this study, with a median age of 8 (0.17-17) years old, and 110 (36.5%), 92 (30.6%), and 99 (32.9%) cases were grouped into newly diagnosed, persistent, and chronic ITP, respectively. The median of follow-up was 41.92 (1.07-74.03) months. At the end of the follow-up (October 2019), among the 202 newly diagnosed/persistent ITP children, 79 cases (59 newly diagnosed and 20 persistent ITP) achieved remission within 1 year after initial diagnosis, with a remission rate of 39.3%; 122 cases (50 newly diagnosed and 72 persistent ITP) developed chronic disease, with a chronicity rate of 60.7%; one case underwent splenectomy. In 99 cases with chronic ITP, 5 cases underwent splenectomy. Multivariable logistic regression analysis showed that, the insidious onset of symptoms (OR=3.754, 95%CI: 1.882-7.488, P=0.000) increased the risk of chronicity, while the positive antibody to anti-platelet membrane glycoprotein (OR=0.446, 95%CI: 0.224-0.888, P=0.021) might reduce the risk of chronicity. And no difference was found by the analysis of subtype of anti-platelet membrane glycoprotein (P=0.305). The efficacy of the first-line treatment of intravenous immunoglobulin (IVIG) alone or combined with steroid was better than that of steroid alone (P=0.028, 0.028), however, the efficiency was not significantly different between IVIG alone and combined with steroid (P=0.086).
Conclusion:
Insidious onset of symptoms in pediatric ITP increases the risk of chronicity, while the positive titer of anti-platelet membrane glycoprotein may reduce the risk. In the first-line treatment for the newly diagnosed/persistent children. The efficacy of IVIG alone or combined with steroid is better than that of steroid alone.
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