Page 19 - Haematologica Vol. 107 - September 2022

REVIEW ARTICLE
Diagnosis of immune thrombocytopenia, including secondary forms, and selection of second-line treatment
James B. Bussel1 and Christine A. Garcia2
1Weill Cornell Medicine, Division of Hematology-Oncology, Department of Pediatrics, New York, NY and 2Weill Cornell Medicine, Division of Hematology and Medical Oncology, New York, NY, USA
Abstract
This article summarizes our approach to the diagnosis of immune thrombocytopenia (ITP), its secondary forms, and choice of second-line treatment options. We very briefly summarize first-line treatment and then utilize a case-based approach. We first explore persistent, chronic ITP in a younger female. We consider many possibilities beyond primary ITP e.g., hypogammaglobulinemia, chronic infection, and anemia, and how to approach their diagnosis and management. The journey continues throughout pregnancy and the post-partum period and eventually includes fourth-line treatment after a late relapse. We then consider an older male, emphasizing differences in diagnostic considerations and management. The focus is on initiation and continuation of second-line treatment, the pros and cons of each option, and briefly the impact of treatment choices related to the endemic presence of severe acute respiratory syndrome coronavirus 2. During the review of potential second-line treatment options, we also briefly touch upon novel treatments. Finally, there is a short section on refractory disease drawn from our previous extensive review published in February 2020.1 The clinical nature of the discussions, replete with figures and tables and with the interspersion of pearls regarding efficacy and toxicity at different ages and genders, will serve the reader in the management of “typical” adult patients who develop persistent and chronic ITP.
Introduction
Immune thrombocytopenia (ITP) is a complicated disease because of its heterogeneity and lack of diagnostic markers making selection of treatment difficult. Perhaps the most straightforward part of management is at pres- entation of ITP. If the platelet count is very low and no other findings are present, the worldwide consensus treatment is steroids. Whether dexamethasone, predni- sone/prednisolone (prednis[ol]one), or intravenous (IV) methylprednisolone is used, the response rate and side effects are relatively predictable. IV methylprednisolone or dexamethasone increases the platelet counts faster and may have fewer side effects than have weeks of pred- nis(ol)one treatment.2 Questions revolve around whether to add IV immunoglobulin (IVIG), and/or platelet trans- fusion. While the latter is rarely appropriate if there is seri- ous bleeding and/or the diagnosis is unclear, an analysis based on medical records in the USA identified that as many as 25% of ITP patients receive platelet transfusion,3 which is far too many.
The management of ITP becomes more complicated if
other findings arise, if patients do not respond to steroids, or if patients continue to require treatment. Both the American Society of Hematology guidelines and an inter- national consensus report emphasize that continued ste- roid use beyond 6 weeks is to be avoided.4,5 Compliance with this strong recommendation entails earlier use of “second-line” therapy in patients with ITP, a practice al- ready gaining traction. However, the definition of “early” remains fluid; “early” can be at 1 month of steroid treat- ment to allow discontinuation of steroids. “Early” can also be in the first 3 months when ITP is “newly diagnosed.6 These ill-defined distinctions are one reason for substan- tial variation in the management of ITP. Another is the un- certainty of diagnosis. A third, the focus of this discussion, is how to select second-line treatment.
This review focuses on the initiation of second-line treat- ment reviewing the pros and cons of different agents util- izing a case-based approach by first exploring ITP in a young female and continuing throughout her pregnancy. The review then outlines diagnostic considerations and management in an older male with particular attention to secondary ITP in both patients.
Haematologica | 107 September 2022
2018
Correspondence: J.B. Bussel jbussel@med.cornell.edu
Received: Accepted: Prepublished:
February 23, 2022. June 7, 2022. June 16, 2022.
https://doi.org/10.3324/haematol.2021.279513
©2022 Ferrata Storti Foundation Published under a CC-BY-NC license