Cargando…

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...

Descripción completa

Detalles Bibliográficos
Autores principales: Terrell, Deirdra R., Neunert, Cindy E., Cooper, Nichola, Heitink-Pollé, Katja M., Kruse, Caroline, Imbach, Paul, Kühne, Thomas, Ghanima, Waleed
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2020
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
_version_ 1783627576662556672
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
work_keys_str_mv AT terrelldeirdrar immunethrombocytopeniaitpcurrentlimitationsinpatientmanagement
AT neunertcindye immunethrombocytopeniaitpcurrentlimitationsinpatientmanagement
AT coopernichola immunethrombocytopeniaitpcurrentlimitationsinpatientmanagement
AT heitinkpollekatjam immunethrombocytopeniaitpcurrentlimitationsinpatientmanagement
AT krusecaroline immunethrombocytopeniaitpcurrentlimitationsinpatientmanagement
AT imbachpaul immunethrombocytopeniaitpcurrentlimitationsinpatientmanagement
AT kuhnethomas immunethrombocytopeniaitpcurrentlimitationsinpatientmanagement
AT ghanimawaleed immunethrombocytopeniaitpcurrentlimitationsinpatientmanagement