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2555 Early Treatment Use of Fostamatinib for Immune Thrombocytopenia (ITP): Efficacy and Safety As Second/Third ITP Treatment Lines in Clinical Practice in Spain

Program: Oral and Poster Abstracts
Session: 311. Disorders of Platelet Number or Function: Clinical and Epidemiological: Poster II
Hematology Disease Topics & Pathways:
Research, Clinical Practice (Health Services and Quality), Clinical Research, Real-world evidence, Registries
Sunday, December 8, 2024, 6:00 PM-8:00 PM

Tomas Jose Gonzalez-Lopez, MD1, Nuria Bermejo-Vega2*, Rocío Cardesa-Cabrera3*, Natalia Acedo Dominguez4*, Josefa Luis-Navarro5*, Sunil Lakhwani6*, Silvia Bernat7*, Guillermo Enrique Haces-Tirado8*, Pilar Galán9*, Maria Luisa Lozano10*, Daniel Martinez Carballeira11*, Roberto Hernandez12*, Reyes Jimenez Barcenas13*, Begoña Navas-Elorza14*, Shally Marcellini15*, Ana Torres-Tienza16*, Alvaro Perona Blazquez17*, Carmen Benet18*, Dolores Fernandez-Jimenez19*, Carolina López-Santamaría Castro20*, Isidro Jarque21*, Violeta Martínez Robles22*, Isabel González-Gascón-y-Marín23* and Gloria García-Donas Gabaldón24*

1Department of Hematology, Hospital Universitario de Burgos, Burgos, Spain
2Department of Hematology, Complejo Hospitalario Universitario de Cáceres, Caceres, ESP
3Hospital Público Comarcal de La Merced, Osuna, Seville, ESP
4Hospital Universitario de La Princesa, Madrid, ESP
5Hospital General de Riotinto, Minas de Riotinto, Huelva, Spain
6Hospital Universitario de Canarias, La Laguna, Spain
7Hospital Universitario de La Plana, Villareal, Castellón, Spain, Villareal, Spain
8Hospital Clínico Universitario Lozano Blesa, Zaragoza, Spain
9Complejo Asistencial de Segovia, Segovia, ESP
10Hospital Morales Meseguer, Murcia, ESP
11Hospital Universitario Central de Asturias, Oviedo (Asturias), Spain
12Complejo Asistencial de Zamora, Zamora, Spain
13Hospital de la Serranía de Ronda, Ronda, Málaga., Ronda (Málaga), Spain
14Hospital Clínico San Carlos, Madrid, ESP
15Complejo Asistencial de Segovia, Segovia, Spain
16Hospital General de Segovia, Segovia, Spain, Segovia, Spain
17Complejo Hospitalario Universitario de Albacete, Albacete, Spain
18Hospital Arnau de Vilanova, Valencia, Spain
19Hospital Universitario San Cecilio, Granada, Spain, Granada, Spain
20Hospital Universitario de Badajoz, Badajoz, Spain
21Hematology Department, Hospital Universitari i Politècnic La Fe, Centro de Investigación Biomédica en Red de Cáncer – CIBERONC, Valencia, Spain
22Complejo Asistencial Universitario de León, León, Spain
23Hospital Universitario Infanta Leonor, Madrid, Spain
24Hospital Universitario Virgen Macarena, Sevilla, Spain

Introduction: Fostamatinib is a splenic tyrosine kinase (SYK) inhibitor that prevents antibody-mediated platelet destruction. This drug has been shown to be effective and safe in immune thrombocytopenia (ITP) and is recommended as a second-line treatment with robust evidence by the International Working Group Consensus (IWG; 2019). Nevertheless, limited real-world data exist on its use as an early therapy for ITP (second/third ITP treatment lines).

Aims: The aim of this study was to evaluate data from daily clinical practice data on the treatment of adult ITP patients with fostamatinib in early lines of treatment: second (after steroids +/- intravenous immunoglobulin use [IVIG]) and third lines (after steroids +/- IVIG and another additional ITP therapy).

Methods: Multicentre, retrospective and prospective observational study at national level to evaluate the management of fostamatinib (Tavlesse®) in adult patients with ITP. An analysis of the medical records of all patients with ITP treated with fostamatinib at each center was performed. A total of 157 adult patients with ITP from 42 Spanish centers were treated with fostamatinib. Response was defined as a platelet count ≥ 30 x 109/L and at least two-fold increase the baseline count and absence of bleeding. Complete response was defined as a platelet count ≥ 100 x 109/L and absence of bleeding.

Results: We evaluated 64 of the 157 patients, in which fostamatinib was used as a second (n=21) or third ITP treatment lines (n=43). The median age of our cohort was 65 years (interquartile range, IQR, 57-77). 35/64 (54.7%) were female. Fifty-seven of them were primary ITP, 7 secondary ITP (2 lymphoproliferative disorders, 1 MGUS, 1 SLE, 2 SAF and 1 ITP secondary to HIV/HCV). 31/64 (48.4%) had ≥ 1 comorbidity of the Charlson Comorbidity lndex. At the time of ITP diagnosis, the median platelet count was 20 x 109 /L (IQR, 7-39) while 40 (62.5%) patients had hemorrhages (37/40 were mucocutaneous). The median time with a diagnosis of ITP at initiation of fostamatinib was 19 months (IQR, 4-114). 16 (25%) and 11 (17.2%) patients were newly-diagnosed and persistent ITP respectively, whereas 37 patients were diagnosed as chronic ITP.

Of the entire cohort of 64 patients, 27 (42.2%) were exposed to eltrombopag, 22 (34.4%) to IVIG, 6 (9.3%) to romiplostim and 4 (6.2%) to rituximab. Only 3 (4.7%) and 2 (3.1%) patients received splenectomy and avatrombopag prior to fostamatinib treatment, respectively. Nineteen patients (29.7%) had signs/symptoms of bleeding in the month prior to treatment initiation. 37/64 (57.8%) patients were treated with fostamatinib monotherapy throughout ITP. The median platelet count at baseline on fostamatinib was 25 x 109 /L (IQR, 12-57), while the median platelet count to best response achieved after fostamatinib treatment was 140 x 109 /L (IQR, 75-230 ). The median time from initiation of fostamatinib to maximum response to treatment was 63 days (IQR, 16-130). Similarly, the median duration of treatment with fostamatinib was 278 days (IQR, 130-449). 36 (56.2%) patients increased fostamatinib dose to 150 mg twice a day during fostamatinib therapy. The median time from initiation of fostamatinib to this dose increase was 26 days (IQR, 15-42).

A total of 55/64 patients (85.9%) responded to fostamatinib with 45/64 patients (70.3%) achieving a complete response. Regarding second line treatment, 17/21 patients (80.9%) responded to fostamatinib with 14 (66.6%) attaining complete responses. 38/43 patients (88.4%) responded as third line therapy with 31 (72.1%) achieving complete responses.

Twenty-nine patients (45.3%) experienced adverse events (AEs). The most frequent AEs were mild and moderate: diarrhea (n=11), hypertension (n=9) and cephalea (n=5). Fifteen patients (23.4%) discontinued fostamatinib treatment due to the following reasons: inefficacy to drug treatment (n=6, 9.4%), toxicity/severe adverse events (n=5, 7.8%), sustained response off-treatment (SROT) (n=3, 4.7%), and exitus non-related to fostamatinib use (n=1, 1.6%).

Conclusions: In this real-world study, fostamatinib showed high response rates when used as early treatment in ITP. In addition, fostamatinib showed a favourable safety profile in unselected patients with ITP.

Disclosures: Gonzalez-Lopez: Grifols: Honoraria, Research Funding; Amgen: Honoraria, Research Funding; Novartis: Honoraria, Research Funding; Sobi: Honoraria, Research Funding; Momenta: Honoraria; Alpine: Honoraria; Argenx: Honoraria. Lozano: Amgen: Honoraria; Terumo: Honoraria; Alexion: Honoraria; Argenx: Honoraria; Celgene: Honoraria; GlaxoSmithKline: Honoraria; Grifols: Honoraria; Novartis: Honoraria; Sobi: Honoraria; UCB Biopharma: Honoraria. Jimenez Barcenas: Sobi: Honoraria; Roche: Honoraria; Pfizer: Honoraria; Novo Nordisk: Honoraria; Grifols: Honoraria; CSL Behring: Honoraria; Amgen: Honoraria. Jarque: Grifols: Honoraria; Gilead: Honoraria; Regeneron Pharmaceuticals, Inc.: Research Funding; Janssen: Honoraria, Research Funding; Incyte: Honoraria, Research Funding; BeiGene: Research Funding; Takeda: Consultancy, Honoraria, Research Funding; Sobi: Consultancy, Honoraria, Research Funding; Pfizer: Consultancy; Novartis: Consultancy; Kyowa Kirin: Consultancy; AstraZeneca: Consultancy, Honoraria, Research Funding; Amgen: Consultancy, Research Funding.

*signifies non-member of ASH