Chronic immune thrombocytopenic purpura in children: assessment of rituximab treatment

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/m 2 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.     

Recurrent immune thrombocytopenic purpura in children

Recurrent immune thrombocytopenic purpura (ITP) is defined as the recurrence of ITP after at least 3 months of remission sustained without treatment. Among 340 children with ITP, 14 had recurrent ITP (4.1%). Ten were females. The initial course was acute in 8 patients and chronic in 6. The median time to recurrence was 33 months (range 4-120). Only 1 patient had a second recurrence. Twelve (86%) achieved complete (n = 10) or partial (n = 2) remission, two of them after splenectomy. One patient continued to require treatment at 10 months from recurrence. One child died of intracranial hemorrhage despite aggressive treatment including splenectomy and craniotomy.     

Danazol for children with immune thrombocytopenic purpura

Ten patients with steroid-dependent or refractory immune thrombocytopenic purpura were treated with danazol in an attempt to improve their platelet counts. Nine of the ten children exhibited an excellent initial response to the drug, with five achieving a complete, unmaintained remission. Two others were able to have their prednisone doses tapered with a resultant disappearance of steroid-induced adverse effects. No significant adverse effects of danazol were noted. Danazol seems to be effective in the treatment of children with immune thrombocytopenic purpura and would warrant additional studies to determine its proper role in the management of this common pediatric ailment.     

Dapsone therapy for children with immune thrombocytopenic purpura

Dapsone has been shown to be effective in treating adults with immune thrombocytopenic purpura (ITP). This retrospective review describes the authors' experience using dapsone in children with refractory, symptomatic ITP. Seven children were treated with dapsone. Dapsone was discontinued in two patients because of methemoglobinemia. In the remaining five patients, three achieved platelet counts of more than 100 x 10(3)/microL. Discontinuation resulted in a rapid decline in platelet counts in all three patients. Two of the three responded to a second round of treatment. Additional study of dapsone in children is warranted. Children receiving dapsone should be monitored for methemoglobinemia.     

Intended management of children with acute idiopathic thrombocytopenic purpura: a national survey

OBJECTIVE: In Australia acute idiopathic thrombocytopenic purpura (ITP) is mainly treated by paediatricians (either general paediatricians or paediatric haematologists/oncologists). A survey was conducted to gauge the current practice of treating children with acute ITP in Australia. METHODS: All practising Australian paediatricians registered by the Royal Australasian College of Physicians were surveyed regarding their intended management of children with acute ITP. The questionnaire, adapted from a study of paediatric haematologists/oncologists in North America, presented four clinical scenarios of children with acute ITP with a platelet count of 3000 x 10(9)/L, with and without mucosal bleeding (wet and dry purpura, respectively). Questionnaires were returned by mail or filled in online at a dedicated webpage. RESULTS: Five hundred and sixty-three of 1097 (51%) paediatricians responded to the survey. Data from 140 who had treated at least one child with ITP in the previous 12 months were analysed. Respondents indicated that children with acute ITP are usually or always hospitalised (58-92%) and that 48% would be given active treatment, even with dry purpura. Various regimens of i.v. immunoglobulin or corticosteroids are used when treatment is administered. In comparing Australian and North American management of acute ITP there were many similarities, although Australian paediatricians were less likely to arrange a bone marrow aspirate if corticosteroids were prescribed. CONCLUSIONS: There is great variation in the intended management of children with acute ITP in Australia. Previously published management recommendations regarding investigation and treatment have had little impact on intended practice. Prospective studies are required to evaluate hypotheses so as to produce evidence-based recommendations for treatment of patients with acute ITP.     

小儿特发性血小板减少性紫癜临床治疗体会

小儿特发性血小板减少性紫癜(idiopathic thrombocytopenic purpura,ITP)是儿科最常见的出血性疾病之一,其病因呈多样性,疗效也与多种因素相关,现将我院近4年收治的92例ITP患儿的临床特点、诊治情况分析如下。1资料与方法1.1一般资料92例患儿年龄从新生儿到14岁以下,其中新生儿1例,3岁以下58例,占63.0%,4~6岁26例,占28.3%,7~14岁8例,占8.7%,男44例,女48例。男女比例为1.1∶1.2。入院时轻度出血13例,占14.1%;中度出血63例,占68.5%;重度出血12例,占13.0%;极重度出血4例,占4.4%,其中消化道大出血合并颅内出血1例。所有病例都有皮肤出血症状,…     

Treatment of chronic immune thrombocytopenic purpura with rituximab in children

Objective  

To evaluate the rituximab treatment in children with chronic immune thrombocytopenic purpura     

细胞因子在免疫性血小板减少性紫癜发病机制中的作用

免疫性血小板减少性紫癜(immune thrombo-cytopenic purpura,ITP)是儿童常见的出血性疾病,以皮肤黏膜的自发性出血、血小板减少、骨髓巨核细胞数量正常或增多,伴成熟障碍为特征.目前其确切发病机制尚未完全清楚,但可以肯定的是与机体免疫功能紊乱密切相关.     

Management of immune thrombocytopenic purpura

Childhood immune thrombocytopenic purpura (ITP) is an uncommon, generally self-limiting, heterogeneous condition usually with a good outlook. Most children recover quickly without serious bleeding complications irrespective of specific treatment to raise the platelet count. The low platelet count was thought to equate to a risk of serious bleeding, but several population studies have confirmed that this risk is low, around 3–4%, and intracranial haemorrhage is rare. Most children do not require active treatment. Assessment of bleeding by a clinical score is required together with assessment of quality of life issues that help to determine whether invasive investigations and treatment are worse for the child than the illness itself. Advances have been made in understanding the pathogenesis, and new treatments have been developed, some of which have been tried with success in children.     

Intracranial hemorrhage in immune thrombocytopenic purpura: a retrospective analysis

PURPOSE: To ascertain characteristics of children with immune thrombocytopenic purpura (ITP) and intracranial hemorrhage (ICH). METHODS: The authors identified 75 published cases of ICH in children with ITP by review of the literature from 1954 to 1998. Data pertaining to the ICH was recorded for age, gender, time from diagnosis of ITP (to ICH), platelet count, head trauma or arteriovenous malformation, concomitant medications, associated infections, other bleeding manifestations, prior treatment, and outcome.RESULTS Sixty-two cases represented 6 months to 20 years of age; 65% of patients were female. The median time from the diagnosis of ITP to ICH was 32 days (range 0 days to 8 years). Fifty of 69 ICH cases (72%) occurred within 6 months of diagnosis, but only 7 (10%) occurred within 3 days of diagnosis. The platelet count was less than 10000/microL in 71.4% of the cases. Treatment prior to the ICH was primarily steroids but also included intravenous immune globulin (IVIG), splenectomy, and others (interferon, azathioprine, or vincristine). There was no difference in mortality of patients before (56%) or after (54%) 1980. CONCLUSIONS: A very low platelet count appears permissive but not sufficient for ICH to occur in children with ITP. ICH occurs more commonly in acute ITP but can occur years after diagnosis. A significant number of patients develop an ICH despite having already initiated steroid treatment of ITP.     

Efficacy and safety of anti-D for immune thrombocytopenic purpura in children

This study was conducted in 20 children (16 males) (mean age 9.2 ± 4.34y) with immune thrombocytopenic purpura (ITP) to assess the response to anti-D immunoglobulin. Six patients had newly diagnosed ITP, 6 had persistent ITP and 8 had chronic ITP. The overall response rate was 70% (14/20). The median time to response was 3 days (1–13 days). Response to anti-D was not related to age, sex, severity of bleeding, platelet counts at presentation, ABO blood group, or prior steroid or IVIG response.