Elsevier

The Journal of Pediatrics

Volume 146, Issue 2, February 2005, Pages 217-221
The Journal of Pediatrics

Original Article
Chronic immune thrombocytopenic purpura in children: Assessment of rituximab treatment

https://doi.org/10.1016/j.jpeds.2004.09.004Get rights and content

Objectives

This study examined the efficacy and safety of rituximab in children with chronic immune thrombocytopenic purpura.

Study design

Twenty-four patients, 2 to 19 years of age, with platelet counts <30,000/mcL (microliter 2), received 375 mg/m2 rituximab in 4 weekly doses. Platelet response was characterized as complete (CR) if a count >150,000/mcL was achieved; partial (PR) if 50,000 to 150,000/mcL; minimal (MR) if the count increased by >20,000/mcL to a peak count >30,000/mcL but <50,000/mcL; or no response (NR).

Results

Fifteen of 24 patients (63%) achieved a CR lasting 4 to 30 months, 9 of which are ongoing. Two had PRs lasting 4 and 6 months; 2 had MRs lasting 5 and 8 months, and 5 did not respond. Pruritus, urticaria, and throat tightness (but no respiratory distress) occurred with the first infusion in a small number of children. Three patients had serum sickness after the first, second, and third infusions, respectively. No increased frequency or severity of infections was seen, although immunoglobulin levels decreased to below the normal range in 6 of 14 cases.

Conclusions

Rituximab may be a useful treatment for chronic immune thrombocytopenic purpura in children with a >50% CR rate lasting an average of 13 months, with 9 of 15 CRs ongoing (8 lasted 6 months or longer). There was no substantial toxicity other than transient serum sickness.

Section snippets

Patients

Eligibility for this study included chronic ITP (duration >6 months) with platelet counts off other treatment of less than 30,000/mcL (Table I). Three patients had platelet counts greater than 30,000/mcL in the 4 weeks before the study as a result of steroid treatment for low platelet counts. The age, sex, duration of ITP, and previous therapies are listed in Table I. Patient ages ranged from 2 to 19 years; 4 patients had previously undergone splenectomy. Nine patients were treated at the New

Results

Patients enrolled in this pilot study had chronic ITP lasting from 6 to 120 months. All patients had had only transient responses after 1 to 7 other therapies before the initiation of this study. Four patients had already failed splenectomy (Table I).

Discussion

This is the largest report of rituximab treatment in children with hematologic disease to date. Rituximab treatment was initiated for 24 patients, 18 of whom were 10 years of age or older. All had ITP that had failed to respond to between 1 and 7 different treatments, including 4 patients who had failed splenectomy. The CR rate was 15 of 24 (63%); there were also 2 PRs and 2 MRs, for an overall response rate of 78%. Fifteen of the 17 good responders (CRs and PRs) had platelet increases to above

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    Symptoms of RISS began 6.63 ± 3.83 d following infusion. The common dosing regimen of rituximab used was 375 mg/m2 (n = 21, 63.6%) [3–5,11–18,24,27,29] every week for 4 wks per cycle and 1000 mg for 2 doses 15 d apart (n = 5, 15.2%) [10,19,22,23]. A reduced dose of 180 mg/m2 for the fifth cycle was given in 1 case due to infusion reactions during third and fourth cycles [2].

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Supported in part by the Platelet Disorders Center of the Children's Blood Foundation and by The Pediatric Clinical Research Center at the Weill Medical College of Cornell University (GCRC# 0103-641).

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