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IV immunoglobulin does not prevent chronic immune thrombocytopenia among children
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IV immunoglobin treatment at immune thrombocytopenia diagnosis did not lead to a lower rate of chronic immune thrombocytopenia among children, according to results from a randomized clinical trial.
“This will influence clinical care in daily practice; IV immunoglobin treatment should only be given based on individual patient characteristics, like for toddlers who fall often or teenage girls who have had their menarche in order to stop or prevent bleeding,” Katja M.J. Heitink-Pollé, MD, from the department of pediatric hematology at University Medical Center Utrecht in The Netherlands, told HemOnc Today. “But, we have also shown that observation is a good alternative to IV immunoglobin treatment, information that is particularly useful to counsel parents and patients and for clinicians in low-income countries that cannot afford IV immunoglobin.”
Observation — for skin bleeding only — or immunomodulatory treatment, for severe bleeding, have remained standard care for children with newly diagnosed immune thrombocytopenia (ITP). Most children with ITP recover within 3 to 12 months; however, 20% to 25% develop chronic ITP.
Previous observational studies have suggested IV immunoglobulin (IVIg) treatment can lead to lower risk for chronic ITP in children; however, randomized clinical trials are limited.
Researchers randomly assigned 206 children aged 3 months to 16 years with newly diagnosed ITP and mild to moderate bleeding to receive a dose of 0.8 g/kg IVIg (Nanogam, Sanquin; n = 102) or careful observation with immunomodulatory treatment in the instance of severe bleeding (n = 104).
Incidence of chronic ITP from baseline — defined as a platelet count less than 150 × 109/L after 6 months — served as the study’s primary outcome. Researchers also reported platelet counts less than 100 × 109/L — the current definition for chronic ITP — at 1 year.
Secondary outcomes included safety and efficacy of both groups, rate of recovery, the impact of treatment on bleeding score, platelet counts, health-related quality of life, and biological factors related to response to IVIg and recovery.
Overall, chronic ITP occurred among 18.6% of children in the IVIg group and 28.9% in the observation group, for a RR of 0.64 (95% CI, 0.38-1.08).
Ten percent of children in the IVIg group had platelet counts less than 100 × 109/L at 1 year compared with 12% of children in the observation group (RR = 0.83; 95% CI 0.38-1.84).
All infants (n = 8) showed complete response at 6 months. Among children aged 1 to 10 years (n = 154), the complete response rate was 82.5% at 6 months and 89% at 1 year.
Among children aged 10 years and older (n = 38), the complete response rate was 71.1% at at 6 months and 86.8% at 1 year.
Although overall response (82.8% vs. 61.6%) and complete response (64.6% vs. 41.4%; P = .001 for both) rates were significantly higher in the IVIg group than in the observation group at 1 month, only complete response rates appeared significantly higher at 3 months (81% vs. 65.3%; P = .01).
Factors associated with early complete response among children in both groups included IgG- Fc receptor IIb genetic variations.
In the observation group, researchers reported 18 admissions due to bleeding in 13 different patients. Nine percent of children in the observation group experienced grade 4 to grade 5 bleeding compared with 1% of children in the IVIg group.
Severe adverse events included epistaxis and menorrhagia, which appeared transient with recovery after medical intervention within a few days, according to the researchers.
“Our current research focuses on prediction of response to IV immunoglobin and prediction of recovery. If we are able to predict this, IV immunoglobin can be given in a more cost-effective way,” Heitink-Pollé said. “If we are able to predict clinical course in individual patients, we can counsel parents and children more specifically and could make better choices in management. We believe that in the near future we will have promising results regarding both issues.” – by Melinda Stevens
For more information:
Katja M.J. Heitink-Pollé, MD, can be reached at University Medical Center Utrecht, Heidelberglaan 100, 3584 CX Utrecht, Netherlands; email: k.m.j.heitink-polle@umcutrecht.nl.
Disclosures: Heitink-Pollé reports no relevant financial disclosures. Two other authors are employed by Sanquin Blood Supply, the manufacturer of IV immunoglobin.
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