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; LIMAL, Nicolas; MICHEL, Marc; GODEAU, Bertrand; MAHÉVAS, Matthieu

doi:10.1111/bjh.18893

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CRICKX, Etienne
Institut Necker Enfants-Malades [INEM - UM 111 (UMR 8253 / U1151)]
Institut Mondor de Recherche Biomédicale [IMRB]

EBBO, Mikael
Centre d'Immunologie de Marseille - Luminy [CIML]

RIVIÈRE, Etienne
Biologie des maladies cardiovasculaires = Biology of Cardiovascular Diseases

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Ce document a été publié dans

British Journal of Haematology. 2023-05

Résumé en anglais

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.< Réduire

Mots clés

Article clinique

Mots clés en anglais

ITP

TPO-RA

combination therapy

immunosuppressive drug