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Presentation Details
| A Retrospective US Claims Database Study Assessing Treatment Patterns and Bleeding Episodes in Patients with Previously Treated Primary Immune Thrombocytopenia Joao Da Silva1, Yu-Chen Yeh2, Firas Dabbous3, Carlos E Bertaglia Goncalves4, Sarah Hale4, Qian Li3, Mei Lu4, Sean Sana2, Dorothy Romanus2. 1Thermo Fisher Scientific, Azores, Portugal.2Takeda Development Center Americas, Inc., Cambridge, MA, USA.3Thermo Fisher Scientific, Bethesda, MD, USA.4Takeda Pharmaceuticals U.S.A., Inc, Lexington, MA, USA |
Abstract
Background: Primary immune thrombocytopenia (ITP) is a rare disorder in which autoimmune destruction of platelets causes low platelet counts and high bleeding risk. ITP can be persistent (continuing 3–12 months after diagnosis) or chronic (continuing >12 months after diagnosis), and most commonly manifests as mucocutaneous bleeding. Severe gastrointestinal, intracranial, or urinary tract bleeds can be life-threatening. Different treatment options are available, with oral corticosteroids (OCS) generally used as first-line therapy, followed by thrombopoietin receptor agonists (TPO-RAs), fostamatinib, rituximab, immunosuppressants, other medications, or splenectomy. Ongoing bleeds can be managed with intravenous (IV) immunoglobulin (Ig) and anti-D Ig. However, patients may still experience bleeding episodes despite these therapies.
Objectives: This analysis is aimed to understand clinical characteristics, treatment patterns, and bleeding episodes among previously treated adult patients with primary ITP in the US. Methods: This retrospective cohort study used data from a US administrative claims database (HealthVerity) collected between October 2015 and May 2022. Eligible adults had ≥1 inpatient or ≥2 outpatient ITP diagnosis codes (ICD-10-CM code D69.3), no secondary thrombocytopenia, and no ITP-specific claims in the 6 months before diagnosis. Previously treated patients were defined as currently receiving ITP treatment, with 1 prior therapy of OCS, IVIg, IV anti-D, or platelet transfusion, and ≥1 prior therapy of TPO-RA, rituximab, fostamatinib, anti-CD38 monoclonal antibodies, immunosuppressants, danazol, dapsone, bortezomib, vinca alkaloid, or splenectomy after the initial diagnosis. The index date was defined as start of the next ITP regimen after meeting the previously treated definition criteria. Patients were followed up until end of study (May 31, 2022) or end of enrollment, whichever came first. ITP treatment and rescue therapies were recorded; bleeding episodes were identified based on diagnosis codes and classified as major (hospitalization), moderate (emergency room visit), or mild (outpatient visit alone).
Results: In total, 34 patients (median age 49.5 [range: 22.0–75.0] years; 71% female) with previously treated ITP were included and followed up for a median of 16.3 (range: 1.5–56.6) months. At the index date, the median time since ITP diagnosis was 10.1 (range: 2.6–57.5) months. Approximately 47% of patients had persistent ITP, 44% had chronic ITP, and 9% were newly diagnosed. Before the index date, 100% of patients received OCS, 35% received TPO-RA-containing regimens, 38% received rituximab-containing regimens, and 3% had undergone splenectomy. Prior treatments by line of therapy are shown in Figure 1. At the index date, 47% of patients initiated TPO-RA-containing regimens (21% TPO-RA monotherapy; 27% TPO-RA combination therapy), 35% initiated OCS monotherapy, and 18% initiated immunosuppressants without TPO-RAs. During follow-up, 59% of patients required rescue therapy and 35% experienced bleeding episodes classified as major (26%), moderate (12%) or mild (29%). Event rates for bleeding episodes by severity and site or type are shown in Table 1.
Conclusions: According to this US-based claims study, many patients with primary ITP experience bleeding episodes requiring treatment with rescue therapies, despite receiving first- and second-line treatments, implying a clinically significant decrease in platelets, and highlighting the unmet need for more effective therapies for patients with primary ITP.
No part of this publication may be reproduced, distributed, or transmitted in any form or by any means, including photocopying, recording, or other electronic or mechanical methods, without the prior written permission of the author.
Objectives: This analysis is aimed to understand clinical characteristics, treatment patterns, and bleeding episodes among previously treated adult patients with primary ITP in the US. Methods: This retrospective cohort study used data from a US administrative claims database (HealthVerity) collected between October 2015 and May 2022. Eligible adults had ≥1 inpatient or ≥2 outpatient ITP diagnosis codes (ICD-10-CM code D69.3), no secondary thrombocytopenia, and no ITP-specific claims in the 6 months before diagnosis. Previously treated patients were defined as currently receiving ITP treatment, with 1 prior therapy of OCS, IVIg, IV anti-D, or platelet transfusion, and ≥1 prior therapy of TPO-RA, rituximab, fostamatinib, anti-CD38 monoclonal antibodies, immunosuppressants, danazol, dapsone, bortezomib, vinca alkaloid, or splenectomy after the initial diagnosis. The index date was defined as start of the next ITP regimen after meeting the previously treated definition criteria. Patients were followed up until end of study (May 31, 2022) or end of enrollment, whichever came first. ITP treatment and rescue therapies were recorded; bleeding episodes were identified based on diagnosis codes and classified as major (hospitalization), moderate (emergency room visit), or mild (outpatient visit alone).
Results: In total, 34 patients (median age 49.5 [range: 22.0–75.0] years; 71% female) with previously treated ITP were included and followed up for a median of 16.3 (range: 1.5–56.6) months. At the index date, the median time since ITP diagnosis was 10.1 (range: 2.6–57.5) months. Approximately 47% of patients had persistent ITP, 44% had chronic ITP, and 9% were newly diagnosed. Before the index date, 100% of patients received OCS, 35% received TPO-RA-containing regimens, 38% received rituximab-containing regimens, and 3% had undergone splenectomy. Prior treatments by line of therapy are shown in Figure 1. At the index date, 47% of patients initiated TPO-RA-containing regimens (21% TPO-RA monotherapy; 27% TPO-RA combination therapy), 35% initiated OCS monotherapy, and 18% initiated immunosuppressants without TPO-RAs. During follow-up, 59% of patients required rescue therapy and 35% experienced bleeding episodes classified as major (26%), moderate (12%) or mild (29%). Event rates for bleeding episodes by severity and site or type are shown in Table 1.
Conclusions: According to this US-based claims study, many patients with primary ITP experience bleeding episodes requiring treatment with rescue therapies, despite receiving first- and second-line treatments, implying a clinically significant decrease in platelets, and highlighting the unmet need for more effective therapies for patients with primary ITP.
No part of this publication may be reproduced, distributed, or transmitted in any form or by any means, including photocopying, recording, or other electronic or mechanical methods, without the prior written permission of the author.