Abstract Number: PB1363
Meeting: ISTH 2020 Congress
Background: Eltrombopag (El) and Romiplostim (Rom) (TPO-RAs) represent second/third-line agents for pITP therapy with high success rate, but long-term treatment is generally required, because platelet (plt) level return to baseline within 2 weeks after discontinuation. However, literature data indicate the possibility of maintaining response after stop of both drugs.
Aims: To evaluate sustained response rate after discontinuation of TPO-RAs, in persistent or chronic adult pITP patients (pts) >18y who failed one or more therapy lines, including splenectomy.
Methods: Retrospective, observational, multicentre study. Definitions: Response (R): plt count ≥30×10^9/L and at least 2-fold increase baseline count and no bleeding; Complete Response (CR): plt count ≥100×10^9/L without bleeding, both in absence of concomitant medications and rescue therapy in the preceding 4 weeks; Sustained R (SR) and Sustained CR (SCR): responses assessed as the first plt count, at more than 4 weeks after discontinuation of TPO-RAs, that satisfy the response definitions.
Results: Characteristics of patients are reported in table 1 and table 2. Enrolment period 2015-2016. Responses evaluation in 139 pts: 69 pts (46.64%) achieved a CR, 41 a R (29.50%), 29 (20.86%) were unresponsive. Ninety pts suspended TPO-RAs (Rom 58, El 32), 34/90 (37.8%) in CR or R. Out of 31 evaluable pts, 29 obtained SR or SCR, (Rom 22/24; El 7/7) for a median time duration of 135.4 weeks (range 4.7-317.0).
Conclusions: 29/147 (19.72%) pts treated with TPO-RAs, reached a SR or SCR maintained for a median time of 2.1 years, in agreement with published data. Our results highlight that TPO-RAs therapy can be successfully suspended in a subset of pITP pts.
|Participating GIMEMA Centers||19|
|Median age, years||58 (17-83)|
|Median plt count at therapy start (x 10^9/L)||16 (1-134)|
|Concomitant therapies (IvIg, Corticosteroids, other)|
|Median number of previous therapies||4 (1-8)|
|Splenectomised patients||50/147 (34.01%)|
|Reasons of TPO-RAs start:|
|no response to splenectomy||8.70%|
|relapse after splenectomy||27.54%|
|contraindication to splenectomy and failure of one or more therapy lines||30.43%|
|refusal of splenectomy and failure of one or more therapy lines||23.19%|
|no eligibility to splenectomy||10.14%|
To cite this abstract in AMA style:Mazzucconi MG, Paoloni F, Carpenedo M, De Stefano V, Cantoni S, Vianelli N, Iodice M, Baldacci E, Zaja F, Bosi A, Schinco P, Fazi P, Rodeghiero F, Santoro C. Response Rate and Response Duration after Discontinuation of Treatment with Thrombopoietin Receptor Agonists (TPO-RAS) in Patients Affected by Primary Immune Thrombocytopenia (pITP): Retrospective Study. Preliminary Results. GIMEMA Protocol ITP0714 [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/response-rate-and-response-duration-after-discontinuation-of-treatment-with-thrombopoietin-receptor-agonists-tpo-ras-in-patients-affected-by-primary-immune-thrombocytopenia-pitp-retrospective-stu/. Accessed November 30, 2021.
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