One year follow‐up of children and adolescents with chronic immune thrombocytopenic purpura (ITP) treated with rituximab

Mueller, Brigitta U.; Bennett, Carolyn M.; Feldman, Henry A.; Bussel, James B.; Abshire, Thomas C.; Moore, Theodore B.; Sawaf, Hadi; Loh, Mignon L.; Rogers, Zora R.; Glader, Bertil; McCarthy, Michaël; Mahoney, Donald H.; Olson, Thomas A.; Feig, Stephen A.; Lorenzana, Adonis; Mentzer, William C.; Buchanan, George R.; Neufeld, Ellis J.

doi:10.1002/pbc.21757

Cited by 49 publications

(29 citation statements)

References 18 publications

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“…IVIG is suggested as the drug of choice by the American Society of Hematology, although it has no benefit over corticosteroids (31). There are no data showing that the treatment affects either short or long term clinical outcome of ITP (30,32). In our study, there was no significant difference between the prescribed medications regarding time to remission and rise of platelet counts after one month.…”

Section: Discussioncontrasting

confidence: 63%

Idiopathic Thrombocytopenic Purpura Is More Severe in Children with a Recent History of Vaccination

Shafiee

Nazari

Hashemiaghdam

et al. 2013

Arch Pediatr Infect Dis

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Background: Idiopathic thrombotic thrombocytopenia (ITP) the most common cause of acute onset thrombocytopenia in children, can be classified as primary (idiopathic) or secondary to different causal agents (drugs, vaccination, infections). Objectives: This study is aimed to observe the severity of ITP based on the probable cause of the disease, specifically vaccination. Patients and Methods: This retrospective observational study surveyed the records of all patients, aged 1 to6 months, who were admitted with the diagnosis of ITP at Mofid Children's hospital in a 5 year period (2005)(2006)(2007)(2008)(2009)(2010). Based on history of recent vaccination (< 6 weeks), patients were divided into two groups. The severity of ITP, described by the platelet count, was then compared between the recently vaccinated and not recently vaccinated groups. Severe ITP was defined as platelet count below 20,000/mm 3 . All ITP patients with concomitant diseases, diagnoses other than ITP, or simultaneous history of vaccination and probable virus infection were excluded. Results: 51 patients were enrolled in this study (mean age = 3.86 ± 1.53 months; males = 32 [62.7%]). In 33 (64.7%) patients the platelets level was below 20000 /mm 3 . Thrombocytopenia following vaccination was observed in 25 (49%) patients. The number of patients with platelet count below 20000 /mm 3 was significantly higher in the recently-vaccinated group (P value = 0.006). ITP was more frequent in the recently vaccinated children under 3 months of age (P Value = 0.03). Conclusions: In this study, a higher rate of more severe ITP in recently vaccinated young children was observed in comparison with other probable etiologies. Further investigations are needed to explain this finding.

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Section: Discussioncontrasting

confidence: 63%

Idiopathic Thrombocytopenic Purpura Is More Severe in Children with a Recent History of Vaccination

Shafiee

Nazari

Hashemiaghdam

et al. 2013

Arch Pediatr Infect Dis

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“…In the 1-year follow-up of a prospective, multicenter trial of 4 weekly doses of rituximab (375 mg/m 2 ) 35 only 8 of 36 patients maintained their platelet counts above 50 ϫ 10 9 /L. 36 Higher response rates were found in some other trials [37][38][39][40] including one that allowed doubling of the dose if there was no response to the initial therapy. 39 Serum sickness has occurred in some patients 35,37,39 and the rate of more significant long-term adverse events such as progressive multifocal leukoencephalopathy remains uncertain.…”

Section: 3b We Suggestmentioning

confidence: 97%

The American Society of Hematology 2011 evidence-based practice guideline for immune thrombocytopenia

Neunert

Lim

Crowther

et al. 2011

Blood

1,650

1,783

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Immune thrombocytopenia (ITP) is commonly encountered in clinical practice. In 1996 the American Society of Hematology published a landmark guidance paper designed to assist clinicians in the management of this disorder. Since 1996 there have been numerous advances in the management of both adult and pediatric ITP. These changes mandated an update in the guidelines. This guideline uses a rigorous, evidence-based approach to the location, interpretation, and presentation of the available evidence. We have endeavored to identify, abstract, and present all available methodologically rigorous data informing the treatment of ITP. We provide evidence-based treatment recommendations using the GRADE system in those areas in which such evidence exists. We do not provide evidence in those areas in which evidence is lacking, or is of lower quality-interested readers are referred to a number of recent, consensus-based recommendations for expert opinion in these clinical areas. Our review identified the need for additional studies in many key areas of the therapy of ITP such as comparative studies of "front-line" therapy for ITP, the management of serious bleeding in patients with ITP, and studies that will provide guidance about which therapy should be used as salvage therapy for patients after failure of a first-line intervention. (Blood. 2011;117(16):4190-4207)

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“…11 Because only 5%-10% of all pediatric ITP patients will have severe, chronic, and/or refractory disease, experience with treating these children is quite limited and there are no randomized clinical trials of treatments for these patients. Therapeutic agents for childhood ITP-corticosteroids, intravenous immunoglobulin, and anti-D immunoglobulin [12][13][14][15][16] ; azathioprine and rituximab [17][18][19] ; and splenectomy 15,17,[20][21][22][23][24] -may be effective but may have limitations with respect to long-term efficacy and/or safety. [12][13][14][15][16][17][19][20][21][22][23][24] Two new thrombopoietin (TPO) receptor agonists that stimulate platelet production, romiplostim and eltrombopag, are now approved for the treatment of adults with chronic ITP in the United States, Europe, Australia, Japan, and elsewhere.…”

Section: Introductionmentioning

confidence: 99%

A randomized, double-blind study of romiplostim to determine its safety and efficacy in children with immune thrombocytopenia

Bussel

Buchanan

Nugent

et al. 2011

Blood

Self Cite

191

193

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Romiplostim, a thrombopoietin-mimetic peptibody, increases and maintains platelet counts in adults with immune thrombocytopenia (ITP). In this first study of a thrombopoietic agent in children, patients with ITP of > 6 months' duration were stratified by age 1:2:2 (12 months-< 3 years; 3-< 12 years; 12-< 18 years). Children received subcutaneous injections of romiplostim (n ‫؍‬ 17) or placebo (n ‫؍‬ 5) weekly for 12 weeks, with dose adjustments to maintain platelet counts between 50 ؋ 10 9 /L and 250 ؋ 10 9 /L. A platelet count > 50 ؋ 10 9 /L for 2 consecutive weeks was achieved by 15/17 (88%) patients in the romiplostim group and no patients in the placebo group (P ‫؍‬ .0008). Platelet counts > 50 ؋ 10 9 /L were maintained for a median of 7 (range, 0-11) weeks in romiplostim patients and 0 (0-0) weeks in placebo patients (P ‫؍‬ .0019). The median weekly dose of romiplostim at 12 weeks was 5 g/kg. Fourteen responders received romiplostim for 4 additional weeks for assessment of pharmacokinetics. No patients discontinued the study. There were no treatment-related, serious adverse events. The most commonly reported adverse events in children, as in adults, were headache and epistaxis. In this short-term study, romiplostim increased platelet counts in 88% of children with ITP and was well-tolerated and apparently safe.

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One year follow‐up of children and adolescents with chronic immune thrombocytopenic purpura (ITP) treated with rituximab

Cited by 49 publications

References 18 publications

Idiopathic Thrombocytopenic Purpura Is More Severe in Children with a Recent History of Vaccination

Idiopathic Thrombocytopenic Purpura Is More Severe in Children with a Recent History of Vaccination

The American Society of Hematology 2011 evidence-based practice guideline for immune thrombocytopenia

A randomized, double-blind study of romiplostim to determine its safety and efficacy in children with immune thrombocytopenia

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