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Immune Thrombocytopenia (ITP): Current Limitations in Patient Management
Primary immune thrombocytopenia (ITP) is an acquired autoimmune disorder characterized by isolated thrombocytopenia caused by increased platelet destruction and impaired platelet production. First-line therapies include corticosteroids, intravenous immunoglobulin, and anti-D immunoglobulin. For pati...
Autores principales: | , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2020
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7761470/ https://www.ncbi.nlm.nih.gov/pubmed/33266286 http://dx.doi.org/10.3390/medicina56120667 |
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author | Terrell, Deirdra R. Neunert, Cindy E. Cooper, Nichola Heitink-Pollé, Katja M. Kruse, Caroline Imbach, Paul Kühne, Thomas Ghanima, Waleed |
author_facet | Terrell, Deirdra R. Neunert, Cindy E. Cooper, Nichola Heitink-Pollé, Katja M. Kruse, Caroline Imbach, Paul Kühne, Thomas Ghanima, Waleed |
author_sort | Terrell, Deirdra R. |
collection | PubMed |
description | Primary immune thrombocytopenia (ITP) is an acquired autoimmune disorder characterized by isolated thrombocytopenia caused by increased platelet destruction and impaired platelet production. First-line therapies include corticosteroids, intravenous immunoglobulin, and anti-D immunoglobulin. For patients who are refractory to these therapies, those who become corticosteroid dependent, or relapse following treatment with corticosteroid, options include splenectomy, rituximab, and thrombopoietin-receptor agonists, alongside a variety of additional immunosuppressive and experimental therapies. Despite recent advances in the management of ITP, many areas need further research. Although it is recognized that an assessment of patient-reported outcomes in ITP is valuable to understand and guide treatment, these measures are not routinely measured in the clinical setting. Consequently, although corticosteroids are first-line therapies for both children and adults, there are no data to suggest that corticosteroids improve health-related quality of life or other patient-related outcomes in either children or adults. In fact, long courses of corticosteroids, in either children or adults, may have a negative impact on a patient’s health-related quality of life, secondary to the impact on sleep disturbance, weight gain, and mental health. In adults, additional therapies may be needed to treat overt hemorrhage, but unfortunately the results are transient for the majority of patients. Therefore, there is a need to recognize the limitations of current existing therapies and evaluate new approaches, such as individualized treatment based on the probability of response and the size of effect on the patient’s most bothersome symptoms and risk of adverse effects or complications. Finally, a validated screening tool that identifies clinically significant patient-reported outcomes in routine clinical practice would help both patients and physicians to effectively follow a patient’s health beyond simply treating the laboratory findings and physical symptoms of ITP. The goal of this narrative review is to discuss management of newly diagnosed and refractory patients with ITP, with a focus on the limitations of current therapies from the patient’s perspective. |
format | Online Article Text |
id | pubmed-7761470 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2020 |
publisher | MDPI |
record_format | MEDLINE/PubMed |
spelling | pubmed-77614702020-12-26 Immune Thrombocytopenia (ITP): Current Limitations in Patient Management Terrell, Deirdra R. Neunert, Cindy E. Cooper, Nichola Heitink-Pollé, Katja M. Kruse, Caroline Imbach, Paul Kühne, Thomas Ghanima, Waleed Medicina (Kaunas) Review Primary immune thrombocytopenia (ITP) is an acquired autoimmune disorder characterized by isolated thrombocytopenia caused by increased platelet destruction and impaired platelet production. First-line therapies include corticosteroids, intravenous immunoglobulin, and anti-D immunoglobulin. For patients who are refractory to these therapies, those who become corticosteroid dependent, or relapse following treatment with corticosteroid, options include splenectomy, rituximab, and thrombopoietin-receptor agonists, alongside a variety of additional immunosuppressive and experimental therapies. Despite recent advances in the management of ITP, many areas need further research. Although it is recognized that an assessment of patient-reported outcomes in ITP is valuable to understand and guide treatment, these measures are not routinely measured in the clinical setting. Consequently, although corticosteroids are first-line therapies for both children and adults, there are no data to suggest that corticosteroids improve health-related quality of life or other patient-related outcomes in either children or adults. In fact, long courses of corticosteroids, in either children or adults, may have a negative impact on a patient’s health-related quality of life, secondary to the impact on sleep disturbance, weight gain, and mental health. In adults, additional therapies may be needed to treat overt hemorrhage, but unfortunately the results are transient for the majority of patients. Therefore, there is a need to recognize the limitations of current existing therapies and evaluate new approaches, such as individualized treatment based on the probability of response and the size of effect on the patient’s most bothersome symptoms and risk of adverse effects or complications. Finally, a validated screening tool that identifies clinically significant patient-reported outcomes in routine clinical practice would help both patients and physicians to effectively follow a patient’s health beyond simply treating the laboratory findings and physical symptoms of ITP. The goal of this narrative review is to discuss management of newly diagnosed and refractory patients with ITP, with a focus on the limitations of current therapies from the patient’s perspective. MDPI 2020-11-30 /pmc/articles/PMC7761470/ /pubmed/33266286 http://dx.doi.org/10.3390/medicina56120667 Text en © 2020 by the authors. Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (http://creativecommons.org/licenses/by/4.0/). |
spellingShingle | Review Terrell, Deirdra R. Neunert, Cindy E. Cooper, Nichola Heitink-Pollé, Katja M. Kruse, Caroline Imbach, Paul Kühne, Thomas Ghanima, Waleed Immune Thrombocytopenia (ITP): Current Limitations in Patient Management |
title | Immune Thrombocytopenia (ITP): Current Limitations in Patient Management |
title_full | Immune Thrombocytopenia (ITP): Current Limitations in Patient Management |
title_fullStr | Immune Thrombocytopenia (ITP): Current Limitations in Patient Management |
title_full_unstemmed | Immune Thrombocytopenia (ITP): Current Limitations in Patient Management |
title_short | Immune Thrombocytopenia (ITP): Current Limitations in Patient Management |
title_sort | immune thrombocytopenia (itp): current limitations in patient management |
topic | Review |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7761470/ https://www.ncbi.nlm.nih.gov/pubmed/33266286 http://dx.doi.org/10.3390/medicina56120667 |
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