Abstract Number: PB0826
Meeting: ISTH 2021 Congress
Theme: Platelet Disorders, von Willebrand Disease and Thrombotic Microangiopathies » Acquired Thrombocytopenias
Background: Since their license in 2008 for use in immune thrombocytopenic purpura, thrombopoietin receptor agonists (TPO-RA), due to their favorable efficacy and safety profile, are becoming candidates for the management of thrombocytopenia in different settings.
Aims: The study aimed to report the response rates and safety profile of the TPO-RA used off-label.
Methods: Observational retrospective study including consecutive patients aged >18 years and receiving off-label TPO-RA at our hospital between January 2010 and June 2020.
Results: A total of 81 patients have been treated with a TPO-RA (eltrombopag or romiplostim) at our center. Sixty-seven patients received the TPO-RA for in-label conditions – ITP (54 patients) or aplastic anemia (13 patients) – while 14 patients received a TPO-RA for unlabeled conditions
(Figure 1). Among the latter group, 4 patients had a transplant-associated thrombocytopenia, 8 suffered from myelodysplastic syndrome and 2 had thrombocytopenia associated to a lymphoproliferative neoplasm. All patients were treated with eltrombopag, except for one who received romiplostim. Figure 2 shows median platelet trends over time among the different groups. Overall, TPO-RA proved effective in all these conditions achieving responses also in heavily pretreated patients. Overall response rate (ORR) was 100% in patients with thrombocytopenia after transplantation and in those with lymphoproliferative diseases and 75% in patients with myelodysplastic syndromes. Median duration of therapy was 285 days (range 93-1513 days). Four patients (29%) discontinued treatment, because of lack of response (n=2) or a sustained response (n=2). No grade 3-4 adverse events occurred, particularly no thrombosis.
Conclusions: In our real-life experience, TPO-RA were effective and safe and proved of value in the challenging management of patients with refractory thrombocytopenia associated with different conditions.
To cite this abstract in AMA style:
Capecchi M, Serpenti F, Giannotta J, Pettine L, Reda G, Barcellini W, Fattizzo B, Martinelli I, Artoni A, Peyvandi F. Off-label Use of Thrombopoietin Receptor Agonists: Case Series and Review of the Literature [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/off-label-use-of-thrombopoietin-receptor-agonists-case-series-and-review-of-the-literature/. Accessed March 22, 2024.« Back to ISTH 2021 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/off-label-use-of-thrombopoietin-receptor-agonists-case-series-and-review-of-the-literature/