Thrombopoietin Receptor Agonists in Children with Immune Thrombocytopenia: A New Therapeutic Era

2020 ◽  
Vol 20 ◽  
Author(s):  
Giuseppe Lassandro ◽  
Valentina Palladino ◽  
Giovanni Carlo Del Vecchioa ◽  
Viviana Valeria Palmieri ◽  
Paola Carmela Corallo ◽  
...  
Keyword(s):  
Immune Thrombocytopenia ◽  
Treatment Options ◽  
Thrombopoietin Receptor Agonists ◽  
Receptor Agonists ◽  
Thrombopoietin Receptor ◽  
Related Quality ◽  
Health Related

Background and Objective: Immune thrombocytopenia (ITP) is a common bleeding disorder in childhood. The management of ITP in children is controversial, requiring personalized assessment of patients and therapeutic choices. Thrombopoietin receptor agonists (TPO-RAs), eltrombopag and romiplostim, have been shown to be safety and effective for the treatment of pediatric ITP. The aim of our research is defining the role of thrombopoietin receptor agonists in the management of pediatric ITP. Method: This review focuses on the use of TPO-RAs in pediatric ITP, in randomized trials and in clinical routine, highlighting their key role in management of the disease. Results: Eltrombopag and romiplostim appear effective treatment options for children with ITP. Several clinical studies have assessed that the use of TPO-RAs increases platelet count, decreases bleeding symptoms and improves health-related quality of life. Moreover, TPO-RAs are well tolerated with minor side effects. Conclusion: Although TPO-RAs long term efficacy and safety still require further investigations, their use is gradually expanding in clinical practice of children with ITP.

scholarly journals Changes in health-related quality of life with long-term eltrombopag treatment in adults with persistent/chronic immune thrombocytopenia: Findings from the EXTEND study

2018 ◽  
Vol 94 (2) ◽  
pp. 200-208 ◽  
Author(s):  
Abderrahim Khelif ◽  
Mansoor N. Saleh ◽  
Abdulgabar Salama ◽  
Maria do Socorro O. Portella ◽  
Mei Sheng Duh ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Immune Thrombocytopenia ◽  
Health Related Quality ◽  
Related Quality ◽  
Health Related ◽  
Chronic Immune Thrombocytopenia

Introduction: Life after the ICU

2014 ◽  
Author(s):  
Margaret Herridge
Keyword(s):  
Critical Illness ◽  
Distress Syndrome ◽  
The Family ◽  
Related Quality ◽  
Health Related ◽  
Risk Modifier

Chapter 1 introduces a section about recovery from critical illness, and discusses the magnitude and burden of critical illness (including acute respiratory distress syndrome (ARDS) and post-ARDS residual pulmonary disease, and compromised health-related quality of life (HRQoL)), its mortality, detailed morbidity (ICU-based risk factors for long-term disability), and costs. It also examines the central role of the family caregiver as outcome and risk modifier.

scholarly journals Childhood immune thrombocytopenia: role of rituximab, recombinant thrombopoietin, and other new therapeutics

Hematology ◽  
2012 ◽  
Vol 2012 (1) ◽  
pp. 444-449 ◽  
Author(s):  
Janna M. Journeycake
Keyword(s):  
Immune Thrombocytopenia ◽  
Therapeutic Option ◽  
Immunosuppressive Agents ◽  
First Year ◽  
Refractory Disease ◽  
Thrombopoietin Receptor Agonists ◽  
Receptor Agonists ◽  
Hematologic Disorder ◽  
Thrombopoietin Receptor

Abstract Childhood immune thrombocytopenia (ITP) is often considered a benign hematologic disorder. However, 30% of affected children will have a prolonged course and 5%-10% will develop chronic severe refractory disease. Until recently, the only proven therapeutic option for chronic severe ITP was splenectomy, but newer alternatives are now being studied. However, because immunosuppressive agents such as rituximab are not approved for use in ITP and the thrombopoietin receptor agonists are not yet approved in children, the decision to use alternatives to splenectomy needs to be considered carefully. This review describes the factors that should affect decisions to treat ITP at diagnosis and compares the options for the occasional child in whom ITP does not resolve within the first year.

scholarly journals Long-term experience in the treatment of α1-antitrypsin deficiency: 25 years of augmentation therapy

2015 ◽  
Vol 24 (135) ◽  
pp. 46-51 ◽  
Author(s):  
Helmut Teschler
Keyword(s):  
Transplant Recipients ◽  
Augmentation Therapy ◽  
Lung Fluid ◽  
Antitrypsin Deficiency ◽  
Related Quality ◽  
Health Related ◽  
Lung Transplant Recipients

Although it is often under-recognised, α1-antitrypsin deficiency (AATD) represents one of the most common genetic respiratory disorders worldwide. Since the publication of studies in the late 1980s, which demonstrated that plasma-derived augmentation therapy with intravenous α1-antitrypsin (AAT) can reverse the biochemical deficiencies in serum and lung fluid that characterise emphysema, augmentation therapy has become the cornerstone of patient management. This article, with a focus on experience gained in clinical practice in Germany, provides an overview of some of the research highlights and clinical experience gained in the use of augmentation therapy for AATD during the past 25 years, and briefly discusses the potential role of AAT augmentation therapy in lung transplant recipients. Additionally, the goals of AAT augmentation therapy will be discussed, namely to delay the progression of emphysema, reduce the frequency of exacerbations and improve health-related quality of life. Beyond pulmonary disease, there is recent growing evidence to indicate that AATD could also play a role in rare disorders such as panniculitis, granulomatosis with polyangiitis and ulcerative colitis.

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