1. Combining thrombopoietin receptor agonists with immunosuppressive drugs in adult patients with multirefractory immune thrombocytopenia, an update on the French experience.
- Author
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Crickx, Etienne, Ebbo, Mikael, Rivière, Etienne, Souchaud‐Debouverie, Odile, Terriou, Louis, Audia, Sylvain, Ruivard, Marc, Asli, Bouchra, Marolleau, Jean‐Pierre, Méaux‐Ruault, Nadine, Gerfaud‐Valentin, Mathieu, Audeguy, Philippe, Hamidou, Mohamed, Corm, Selim, Delbrel, Xavier, Fontan, Jean, Lebon, Delphine, Mausservey, Christelle, Moulis, Guillaume, and Limal, Nicolas
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THROMBOPOIETIN receptor agonists , *IMMUNOSUPPRESSIVE agents , *IDIOPATHIC thrombocytopenic purpura , *PLATELET count , *DRUG efficacy - Abstract
Summary: Combining drugs could be an effective option for treating multirefractory ITP, that is, patients not responding to rituximab, thrombopoietin receptor agonists (TPO‐RA) and splenectomy. We conducted a retrospective, multicenter, observational study including multirefractory ITP patients who received a combination of a TPO‐RA and an immunosuppressive drug. We included 39 patients (67% women, median age 59 years [range 21–96]), with a median ITP duration of 57 months [3–393] and a median platelet count at initiation of 10 × 109/L [1–35]. The combination regimen was given for a median duration of 12 months [1–103] and included eltrombopag (51%) or romiplostim (49%), associated with mycophenolate mofetil (54%), azathioprine (36%), cyclophosphamide (5%), cyclosporin (3%) or everolimus (3%). Overall, 30 patients (77%) achieved at least a response (platelet count ≥30 × 109/L and at least doubling baseline during at least 3 months), including 24 complete responses (platelet count >100 × 109/L during at least 3 months) with a median time to response of 30 days [7–270] and a median duration of response of 15 months [4–63]. Severe adverse event related to ITP treatment was observed in 31%. In conclusion, this study confirms that some patients with multirefractory ITP can achieve long lasting response with this combination. [ABSTRACT FROM AUTHOR]
- Published
- 2023
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