ABSTRACT
BACKGROUND: Treatment choice in pediatric immune thrombocytopenia (ITP) is arbitrary, because few studies are powered to identify predictors of therapy response. Increasingly, rituximab is becoming a treatment of choice in those refractory to other therapies. METHODS: The objective of this study was to evaluate univariate and multivariable predictors of platelet count response to rituximab. After local IRB approval, 565 patients with chronic ITP enrolled and met criteria for this study in the longitudinal, North American Chronic ITP Registry (NACIR) between January 2004 and October 2010. Treatment response was defined as a post-treatment platelet count ≥ 50,000/µl within 16 weeks of rituximab and 14 days of steroids. Treatment response data were captured both retrospectively at enrollment and then prospectively. RESULTS: Eighty (14.2%) patients were treated with rituximab with an overall response rate of 63.8% (51/80). Univariate correlates of response to rituximab included the presence of secondary ITP and a positive response to steroids. In multivariable analysis, response to steroids remained a strong correlate of response to rituximab, OR 6.8 (95% CI 2.0-23.0, P = 0.002). Secondary ITP also remained a strong predictor of response to rituximab, OR 5.6 (95% CI 1.1-28.6, P = 0.04). Although 87.5% of patients who responded to steroids responded to rituximab, 48% with a negative response to steroids did respond to rituximab. CONCLUSION: In the NACIR, response to steroids and presence of secondary ITP were strong correlates of response to rituximab, a finding not previously reported in children or adults.
Subject(s)
Antibodies, Monoclonal, Murine-Derived/therapeutic use , Antineoplastic Agents/therapeutic use , Prednisone/therapeutic use , Thrombocytopenia/drug therapy , Thrombocytopenia/immunology , Adolescent , Child , Child, Preschool , Chronic Disease , Cohort Studies , Female , Follow-Up Studies , Glucocorticoids/therapeutic use , Humans , Immunoglobulins, Intravenous/therapeutic use , Immunologic Factors/therapeutic use , Infant , Infant, Newborn , Longitudinal Studies , Male , Platelet Count , Prospective Studies , Retrospective Studies , Rituximab , Treatment OutcomeABSTRACT
OBJECTIVES/HYPOTHESIS: :To understand Glanzmann's thrombasthenia and provide insight to the management of epistaxis in children with this disease. STUDY DESIGN: Retrospective chart review. METHODS: All children diagnosed with Glanzmann's thrombasthenia and treated for epistaxis at Children's Hospitals and Clinics of Minnesota were identified and a retrospective chart review was performed. Outpatient charts, hospitalization records, and operative reports were reviewed from 1999 up to 2009 and appropriate data were extracted. The episodes of epistaxis, therapy used, complications, and success rates for controlling hemorrhage were noted. RESULTS: Five children with a total of 63 clinical encounters for epistaxis were identified. Forty-seven encounters required hospitalization, close to half of which necessitated medical care in the intensive care unit. Nearly all encounters required infusion of hemostatic therapy with either single or multiple agents. Seventy-one procedures for life-threatening hemorrhage were performed. The most common (n = 24) intervention was administration of bovine collagen matrix, which was successful only half of the time (50%). Anterior and posterior nasal packing with or without hemostatic material completely resolved hemorrhage in 35% of the procedures. CONCLUSIONS: To date, no form of medical or surgical intervention in children with this condition has consistently demonstrated its ability resolve nasal hemorrhage. The role of the otolaryngologist is to control bleeding during major episodes of nasal hemorrhage that do not respond to medical management. These patients usually have remarkable improvement in the frequency and severity of epistaxis in adolescence and then require much less aggressive therapy.
Subject(s)
Collagen/administration & dosage , Epistaxis/therapy , Hemostatic Techniques , Thrombasthenia/complications , Administration, Topical , Child, Preschool , Epistaxis/blood , Epistaxis/etiology , Follow-Up Studies , Humans , Infant , Infant, Newborn , Retrospective Studies , Thrombasthenia/blood , Thrombasthenia/therapy , Time Factors , Treatment OutcomeABSTRACT
In newborns with hemophilia, intracranial hemorrhage (ICH) is a potentially life-threatening event and in those who survive, there is a risk of long-term neurologic sequelae. A single-center retrospective chart review was performed to look at the utility of central nervous system screening of babies who were diagnosed with moderate or severe hemophilia in the first 7 days of life. Twenty of the twenty-two eligible babies had either a head ultrasound or computerized tomography of the head performed as soon as possible after the diagnosis of hemophilia was confirmed. ICH was found in 3 of the 20 newborns. All patients with ICH had instrument-assisted deliveries with extracranial signs of bleeding/trauma. On the basis of this series we found screening to be safe and effective in recognizing ICH. As a result of this approach, all ICHs were found when they were radiographically small and before the babies had neurologic symptoms.
