Cindy E. Neunert

Despite immune thrombocytopenia (ITP) being one of the most common conditions encountered by the pediatric hematologist, relatively few prospective investigations of ITP exist, and treatment approaches remain highly variable. The ideal prospective clinical trial would use a reduction in severe bleeding as an outcome; however, recent data shows that due to the rarity of severe bleeding events such a trial is not feasible due to the large number of children needed to be enrolled on such studies. Therefore, platelet count is often used a surrogate risk for bleeding severity and an increase in platelet count as the primary outcome in clinical trials. Reliance solely on the platelet count fails to account for relevant patient-related outcomes, among them bleeding severity. Bleeding assessment instruments published to date lack validity and reliability. Therefore, rigorous methods to design and analysis of patient-related outcome measurement tools need to be applied. Once designed appropriately, these measures can be applied to enhance research and help to guide treatment for those patients who truly need it.

Controversy exists regarding management of children newly diagnosed with immune thrombocytopenic purpura (ITP). Drug treatment is usually administered to prevent severe hemorrhage, although the definition and frequency of severe bleeding are poorly characterized. Accordingly, the Intercontinental Childhood ITP Study Group (ICIS) conducted a prospective registry defining severe hemorrhage at diagnosis and during the following 28 days in children with ITP. Of 1106 ITP patients enrolled, 863 were eligible and evaluable for bleeding severity assessment at diagnosis and during the subsequent 4 weeks. Twenty-five children (2.9%) had severe bleeding at diagnosis. Among 505 patients with a platelet count less than or equal to 20 000/mm3 and no or mild bleeding at diagnosis, 3 (0.6%), had new severe hemorrhagic events during the ensuing 28 days. Subsequent development of severe hemorrhage was unrelated to initial management (P = .82). These results show that severe bleeding is uncommon at diagnosis in children with ITP and rare during the next 4 weeks irrespective of treatment given. We conclude that it would be difficult to design an adequately powered therapeutic trial aimed at demonstrating prevention of severe bleeding during the first 4 weeks after diagnosis. This finding suggests that future studies of ITP management should emphasize other outcomes.

We report two pediatric patients with rifampin‐induced hemolysis following treatment with low daily dose rifampin for methicillin‐resistant Staphylococcus aureus (MRSA). With the increased use of rifampin to treat MRSA, physicians should be aware that patients receiving rifampin therapy are at risk for hemolysis, even at low daily doses. Pediatr Blood Cancer 2008;51:821–823.

Background: Physician attitudes regarding management of children with severe chronic immune thrombocytopenic purpura (ITP) have not been recently characterized.Procedure: We designed a survey of members of the American Society of Pediatric Hematology‐Oncology (ASPHO) that described a 5‐year‐old female with ITP for 1 year who was unresponsive to steroids, IVIG, and anti‐D immune globulin and having frequent epistaxis causing interference with her daily activities. A 13‐item questionnaire evaluated physician decision‐making in this setting.Results: Two hundred and ninety‐seven surveys (35% response rate) were returned, and 295 were evaluable. Thirty‐three percent of respondents stated that they would recommend splenectomy for such a child. Of those who would not recommend splenectomy, 67% reported that they would instead treat with rituximab. If initial drug therapy failed, 47% would proceed with splenectomy. Those who reported treating with rituximab initially were more likely to recommend splenectomy following failure than those who preferred other drug therapy (P < 0.0001).Conclusions: Physician management of patients with chronic ITP is diverse. With the advent of new treatments such as rituximab and thrombopoetic agents it is critically important to compare their cost, adverse effects and efficacy with splenectomy in order to optimally guide treatment practices. Pediatr Blood Cancer 2008;51:513–516.

Summary. Elective surgical procedures involving central venous access devices (CVADs) in patients with haemophilia are often necessary for adequate factor delivery but there are few data regarding haemostatic coverage and acute complication rates accompanying these procedures. To describe experience with CVAD insertion, revision and removal in young haemophilia patients at our institution and in the literature and to assess acute complications following CVAD procedures. PubMed, Medline and Cochrane databases were searched for articles, which included a description of factor coverage during CVAD procedures. A retrospective review of our comprehensive haemophilia database identified patients undergoing CVAD placement, revision and removal between January 1993 and August 2005. Manual and electronic searches of the published literature yielded 14 articles, which met inclusion criteria. Peri‐operative factor administration varied greatly among the reports. Mean acute infection and haematoma rates were 8% and 12.5% respectively. A retrospective review identified 49 CVAD placements, revisions, or removals meeting inclusion criteria. Most patients received outpatient bolus factor replacement to achieve a level of 100% preoperatively, immediately postoperatively and on postoperative days 1, 2, 3, 5 and 7. Thirty‐six procedures were performed without hospitalization. Ten patients developed 11 (22%) minor haematomas postoperatively. Major haemorrhage, acute infection, or pneumothorax was not encountered. Few published data exist regarding haemostatic coverage and complications following CVAD procedures. Our institutional experience using a consistent management approach was favourable. Further studies are required to define optimal haemostatic coverage during minor surgical procedures in haemophilia.