{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,11,14]],"date-time":"2026-11-14T16:40:16Z","timestamp":1794674416491,"version":"4.9.1"},"reference-count":40,"publisher":"MDPI AG","issue":"12","license":[{"start":{"date-parts":[[2020,11,30]],"date-time":"2020-11-30T00:00:00Z","timestamp":1606694400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["K01HL135466"],"award-info":[{"award-number":["K01HL135466"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]},{"name":"PDSA","award":["no number"],"award-info":[{"award-number":["no number"]}]},{"name":"Imperial College NIHR Bioresources","award":["no number"],"award-info":[{"award-number":["no number"]}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Medicina"],"abstract":"<jats:p>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\u2019s 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\u2019s 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\u2019s 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\u2019s perspective.<\/jats:p>","DOI":"10.3390\/medicina56120667","type":"journal-article","created":{"date-parts":[[2020,11,30]],"date-time":"2020-11-30T20:10:22Z","timestamp":1606767022000},"page":"667","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":63,"title":["Immune Thrombocytopenia (ITP): Current Limitations in Patient Management"],"prefix":"10.3390","volume":"56","author":[{"given":"Deirdra R.","family":"Terrell","sequence":"first","affiliation":[{"name":"Department of Biostatistics and Epidemiology, Hudson College of Public Health, University of Oklahoma Health Sciences Center, Oklahoma City, OK 73104, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cindy E.","family":"Neunert","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Columbia University Irving Medical Center, New York, NY 10032, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nichola","family":"Cooper","sequence":"additional","affiliation":[{"name":"Centre for Haematology, Department of Medicine, Hammersmith Hospital, Imperial College London, London SW7 2BU, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Katja M.","family":"Heitink-Poll\u00e9","sequence":"additional","affiliation":[{"name":"Department of Pediatric Hemato-oncology, Princess Maxima Center, 3584 Utrecht, The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Caroline","family":"Kruse","sequence":"additional","affiliation":[{"name":"Platelet Disorder Support Association, Cleveland, OH 44141, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paul","family":"Imbach","sequence":"additional","affiliation":[{"name":"Medical Faculty, University of Basel, 4051 Basel, Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7387-7028","authenticated-orcid":false,"given":"Thomas","family":"K\u00fchne","sequence":"additional","affiliation":[{"name":"University Children\u2019s Hospital, Oncology\/Hematology, 4056 Basel, Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Waleed","family":"Ghanima","sequence":"additional","affiliation":[{"name":"Departments of Hemato-oncology and Research, \u00d8stfold Hospital, 1714 Gr\u00e5lum, Norway"},{"name":"Department of Hematology, Oslo University Hospital and Institute of Clinical Medicine, University of Oslo, 0318 Oslo, Norway"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2020,11,30]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"3829","DOI":"10.1182\/bloodadvances.2019000966","article-title":"American Society of Hematology 2019 guidelines for immune thrombocytopenia","volume":"3","author":"Neunert","year":"2019","journal-title":"Blood Adv."},{"key":"ref_2","doi-asserted-by":"crossref","first-page":"3780","DOI":"10.1182\/bloodadvances.2019000812","article-title":"Updated international consensus report on the investigation and management of primary immune thrombocytopenia","volume":"3","author":"Provan","year":"2019","journal-title":"Blood Adv."},{"key":"ref_3","doi-asserted-by":"crossref","first-page":"493","DOI":"10.1002\/ajh.24702","article-title":"Newly diagnosed immune thrombocytopenia adults: Clinical epidemiology, exposure to treatments, and evolution. 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